37 results on '"Arnaud Dalissier"'
Search Results
2. Matched unrelated donor transplantation versus haploidentical transplantation with post-transplant cyclophosphamide in children with acute myeloid leukemia: a PDWP-EBMT study
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Annalisa Ruggeri, Nicole Santoro, Jacques-Emmanuel Galimard, Krzysztof Kalwak, Mattia Algeri, Ludmila Zubarovskaya, Krzysztof Czyzewski, Elena Skorobogatova, Petr Sedlacek, Caroline Besley, Adriana Balduzzi, Yves Bertrand, Julia Peristeri, Franca Fagioli, Mariane Ifversen, Jolanta Gozdzik, Christina Peters, Birgitta Versluijs, Alessandra Biffi, Arcangelo Prete, Maura Faraci, Ibrahim Ghemlas, Ivana Bodova, Olga Aleinikova, Arnaud Dalissier, Vanderson Rocha, and Selim Corbacioglu
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Diseases of the blood and blood-forming organs ,RC633-647.5 - Abstract
In children with acute myeloid leukemia (AML) who lack an HLA identical sibling, the donor can be replaced with an HLA matched unrelated donor (MUD) or a haploidentical donor (haplo). We compared outcomes of patients
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- 2024
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3. Supportive Care During Pediatric Hematopoietic Stem Cell Transplantation: Prevention of Infections. A Report From Workshops on Supportive Care of the Paediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT)
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Marianne Ifversen, Roland Meisel, Petr Sedlacek, Krzysztof Kalwak, Luisa Sisinni, Daphna Hutt, Thomas Lehrnbecher, Adriana Balduzzi, Tamara Diesch, Andrea Jarisch, Tayfun Güngör, Jerry Stein, Isaac Yaniv, Halvard Bonig, Michaela Kuhlen, Marc Ansari, Tiago Nava, Jean-Hugues Dalle, Cristina Diaz-de-Heredia, Eugenia Trigoso, Ulrike Falkenberg, Mihaela Hartmann, Marco Deiana, Marta Canesi, Chiara Broggi, Alice Bertaina, Brenda Gibson, Gergely Krivan, Kim Vettenranta, Toni Matic, Jochen Buechner, Anita Lawitschka, Christina Peters, Akif Yesilipek, Koray Yalçin, Giovanna Lucchini, Shahrzad Bakhtiar, Dominik Turkiewicz, Riitta Niinimäki, Jacek Wachowiak, Simone Cesaro, Arnaud Dalissier, Selim Corbacioglu, Andre Manfred Willasch, and Peter Bader
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infection precaution ,allogeneic hematological stem cell transplantation ,children ,antibiotic prophylactic therapy ,vaccination ,Pediatrics ,RJ1-570 - Abstract
Specific protocols define eligibility, conditioning, donor selection, graft composition and prophylaxis of graft vs. host disease for children and young adults undergoing hematopoietic stem cell transplant (HSCT). However, international protocols rarely, if ever, detail supportive care, including pharmaceutical infection prophylaxis, physical protection with face masks and cohort isolation or food restrictions. Supportive care suffers from a lack of scientific evidence and implementation of practices in the transplant centers brings extensive restrictions to the child's and family's daily life after HSCT. Therefore, the Board of the Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT) held a series of dedicated workshops since 2017 with the aim of initiating the production of a set of minimal recommendations. The present paper describes the consensus reached within the field of infection prophylaxis.
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- 2021
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4. Specialized Pediatric Palliative Care Services in Pediatric Hematopoietic Stem Cell Transplant Centers
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Hilda Mekelenkamp, Teija Schröder, Eugenia Trigoso, Daphna Hutt, Jacques-Emmanuel Galimard, Anne Kozijn, Arnaud Dalissier, Marjola Gjergji, Sarah Liptrott, Michelle Kenyon, John Murray, Selim Corbacioglu, Peter Bader, on behalf of the EBMT-Nurses Group, and Paediatric Diseases Working Party
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hematopoietic stem cell transplantation ,palliative care ,palliative care services ,pediatric ,Pediatrics ,RJ1-570 - Abstract
Hematopoietic stem cell transplantation (HSCT) is widely used in pediatric patients as a successful curative therapy for life-threatening conditions. The treatment is intensive, with risks of serious complications and lethal outcomes. This study aimed to provide insight into current data on the place and cause of death of transplanted children, the available specialized pediatric palliative care services (SPPCS), and what services HSCT professionals feel the SPPCS team should provide. First, a retrospective database analysis on the place and cause of death of transplanted pediatric HSCT patients was performed. Second, a survey was performed addressing the availability of and views on SPPCS among HSCT professionals. Database analysis included 233 patients of whom the majority died in-hospital: 38% in the pediatric intensive care unit, 20% in HSCT units, 17% in other hospitals, and 14% at home or in a hospice (11% unknown). For the survey, 98 HSCT professionals from 54 centers participated. Nearly all professionals indicated that HSCT patients should have access to SPPCS, especially for pain management, but less than half routinely referred to this service at an early stage. We, therefore, advise HSCT teams to integrate advance care planning for pediatric HSCT patients actively, ideally from diagnosis, to ensure timely SPPCS involvement and maximize end-of-life preparation.
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- 2021
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5. Specific Cytogenetic Abnormalities at Diagnosis Predict Survival after Hematopoietic Cell Transplant in Poor-Risk Pediatric Acute Myeloid Leukemia: A PDWP/EBMT Study
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Akshay Sharma, Jacques-Emmanuel Galimard, Angharad Pryce, Senthil Bhoopalan, Arnaud Dalissier, Jean-Hugues Dalle, Franco Locatelli, Charlotte Jubert, Oana Mirci-Danicar, Vassiliki Kitra-Roussou, Yves Bertrand, Franca Fagioli, Fanny Rialland, Alessandra Biffi, Robert F Wynn, Gerard Michel, Francesco Paolo P Tambaro, Ali Abdallah Ahmari, Abdelghani Tbakhi, Caroline Furness, Miguel Ángel Díaz Pérez, Petr Sedlacek, Ivana Bodova, Maura Faraci, Kanchan Rao, Marie Thérèse Rubio, Brenda Gibson, Neel S. Bhatt, and Selim Corbacioglu
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Immunology ,Cell Biology ,Hematology ,Biochemistry - Published
- 2022
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6. Outcome of Haematopoietic Cell Transplantation in 813 Children with Fanconi Anaemia: A Study on Behalf of the EBMT Severe Aplastic Anaemia Working Party and Paediatric Disease Working Party
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Su Han Lum, Sujith Samarasinghe, Dirk-Jan Eikema, Brian Piepenbroek, Arnaud Dalissier, Mouhab Ayas, Ashrafsadat Mousavi, Rose-Marie Hamladji, Akif Yesilipek, Jean-Hugues Dalle, Savas Kansoy, Franco Locatelli, Duygu Çetinkaya, Marc Bierings, O. Alphan Kupesiz, Abdelghani Tbakhi, Elena Skorobogatova, Gülyüz Öztürk, Maura Faraci, Yves Bertrand, Pamela Evans, Selim Carbacioglu, Carlo Dufour, Antonio Risitano, Robert F Wynn, and Régis Peffault de Latour
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Immunology ,Cell Biology ,Hematology ,Biochemistry - Published
- 2022
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7. The Impact of Pre-Transplant Extramedullary Disease on the Outcome of Allogeneic Hematopoietic Cell Transplantation for Acute Myeloid Leukemia in Children- on Behalf of PDWP/EBMT
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Juliana Silva, Jacques-Emmanuel Galimard, Jean-Hugues Dalle, Franco Locatelli, Hawazen S. Alsaedi, Miguel Angel Diaz, Charlotte Jubert, Yves Bertrand, Giuseppina Simone, Peter Bader, Franca Fagioli, Vassiliki Kitra Roussou, Caroline Furness, Robert Wynn, Birgit Burkhardt, Alessandra Biffi, Marc Bierings, Cristina Díaz de Heredia, Arcangelo Prete, Amos Toren, Katharine Patrick, Wolfgang Holter, Arnaud Dalissier, Kanchan Rao, and Selim Corbacioglu
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Immunology ,Cell Biology ,Hematology ,Biochemistry - Published
- 2022
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8. Use of eculizumab in children with allogeneic haematopoietic stem cell transplantation associated thrombotic microangiopathy-a multicentre retrospective PDWP and IEWP EBMT study
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Peter Svec, Reem Elfeky, Jacques-Emmanuel Galimard, Christine S. Higham, Arnaud Dalissier, Troy C. Quigg, David Bueno Sanchez, Su Han Lum, Maura Faraci, Theresa Cole, Herbert Pichler, Maria Isabel Benítez-Carabante, Julia Horakova, Marta Gonzalez -Vicent, Asaf Yanir, Franca Fagioli, Matthias Wölfl, Nicolas von der Weid, Rachel Protheroe, Gergely Krivan, Carsten Speckmann, Beki James, Simona Lucija Avcin, Yves Bertrand, Marta Verna, Petr Riha, Katharine Patrick, Simone Cesaro, Krzysztof Kalwak, Marc Bierings, Jochen Büchner, Karin Mellgren, Zoltán Prohászka, Bénédicte Neven, Arjan Lankester, and Selim Corbacioglu
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Transplantation ,Hematology - Abstract
Terminal complement blockade by humanised monoclonal antibody eculizumab has been used to treat transplantation-associated thrombotic microangiopathy (TA-TMA) in recent years. This retrospective international study conducted by the Paediatric Diseases (PDWP) and Inborn Error Working Party (IEWP) of the European Society for Blood and Marrow Transplantation (EBMT) describes outcome and response of 82 paediatric patients from 29 centres who developed TA-TMA and were treated with eculizumab between January 2014 and May 2019. The median time from hematopoietic stem cell transplantation (HSCT) to TA-TMA manifestation was 92 days (range: 7-606) and from TA-TMA diagnosis to the start of eculizumab treatment 6 days (range: 0-135). Most patients received eculizumab weekly (72%, n = 55) with a standard weight (kg)-based dose (78%, n = 64). Six months from beginning of eculizumab therapy, the cumulative incidence of TA-TMA resolution was 36.6% (95% CI: 26.2-47) and the overall survival (OS) was 47.1% (95% CI: 35.9-57.5). All 43 patients with unresolved TA-TMA died. The cause of death was HSCT-related in 41 patients. This study also documents poor outcome of patients without aGvHD and their frequent concomitant viral infections. Considering recent publications, intensified eculizumab dosing and complement monitoring could potentially improve upon outcomes observed in this study.
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- 2022
9. Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation for Congenital Amegakaryocytic Thrombocytopenia, a PDWP/EBMT Study
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Clemence Aldebert, Mony Fahd, Jacques-Emmanuel Galimard, Ibrahim A. Ghemlas, Marco Zecca, Juliana Silva, Alexander Mohseny, Alphan Kupesiz, Rose-Marie Hamladji, Nuno Miranda, Tayfun Gungor, Robert F Wynn, Pietro Merli, Mikael Sundin, Maura Faraci, Cristina Díaz-de-Heredia, Birgit Burkhardt, Victoria Bordon, Charlotte Jubert, Peter Bader, Marianne Ifversen, Concepcion Herrera Arroyo, Natalia Maximova, Susana Riesco, Jerry Stein, Arnaud Dalissier, Franco Locatelli, Krzysztof Kalwak, Jean-Hugues Dalle, and Selim Corbacioglu
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Immunology ,Cell Biology ,Hematology ,Biochemistry - Published
- 2022
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10. Supportive care during pediatric hematopoietic stem cell transplantation: beyond infectious diseases. A report from workshops on supportive care of the Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT)
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Krzysztof Kałwak, Peter Bader, Alice Bertaina, Riitta Niinimäki, Tayfun Güngör, Brenda Gibson, Marianne Ifversen, Marc Ansari, Christina Diaz de Heredia, Akif Yesilipek, Tiago Nava, Jerry Stein, Jochen Buechner, E. Trigoso, Jacek Wachowiak, Marco Deiana, Koray Yalcin, Christina Peters, Halvard Boenig, Simone Cesaro, Isaac Yaniv, Gergely Kriván, Kim Vettenranta, Toni Matic, Dominik Turkiewicz, Roland Meisel, Michaela Kuhlen, Giovanna Lucchini, Shahrzad Bakhtiar, Andre Willasch, Luisa Sisinni, Petr Sedlacek, Daphna Hutt, Thomas Lehrnbecher, Anita Lawitschka, Adriana Balduzzi, Jean Hugues Dalle, Tamara Diesch, Arnaud Dalissier, Selim Corbacioglu, Andrea Jarisch, Ulrike Falkenberg, Nava, T, Ansari, M, Dalle, J, de Heredia, C, Güngör, T, Trigoso, E, Falkenberg, U, Bertaina, A, Gibson, B, Jarisch, A, Balduzzi, A, Boenig, H, Krivan, G, Vettenranta, K, Matic, T, Büchner, J, Kalwak, K, Lawitschka, A, Yesilipek, A, Lucchini, G, Peters, C, Turkiewicz, D, Niinimäki, R, Diesch, T, Lehrnbecher, T, Sedlacek, P, Hutt, D, Dalissier, A, Wachowiak, J, Yaniv, I, Stein, J, Yalçin, K, Sisinni, L, Deiana, M, Ifversen, M, Kuhlen, M, Miesel, R, Bakhtiar, S, Cesaro, S, Willasch, A, Corbacioglu, S, and Bader, P
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HEPATIC VENOOCCLUSIVE DISEASE ,medicine.medical_specialty ,bone marrow transplantation ,medicine.medical_treatment ,MEDLINE ,ENTERAL NUTRITION ,Hematopoietic stem cell transplantation ,ORAL MUCOSITIS ,Communicable Diseases ,03 medical and health sciences ,bone marrow transplantation, pediatric, supportive care ,0302 clinical medicine ,Bone Marrow ,Internal medicine ,IRON OVERLOAD ,Mucositis ,Humans ,Medicine ,Child ,Intensive care medicine ,SYNDROME/VENO-OCCLUSIVE DISEASE ,ANTICIPATORY NAUSEA ,Transplantation ,Hematology ,business.industry ,Marrow transplantation ,Research ,Hematopoietic Stem Cell Transplantation ,SEVERITY CRITERIA ,medicine.disease ,supportive care, hematopoietic stem cell transplantation, pediatric ,URSODEOXYCHOLIC ACID ,Europe ,supportive care ,Leukemia ,pediatric ,ANTINEOPLASTIC MEDICATION ,surgical procedures, operative ,Parenteral nutrition ,medicine.anatomical_structure ,030220 oncology & carcinogenesis ,Bone marrow ,business ,CHEMOTHERAPY-INDUCED NAUSEA ,030215 immunology - Abstract
Hematopoietic stem cell transplantation (HSCT) is currently the standard of care for many malignant and nonmalignant blood diseases. As several treatment-emerging acute toxicities are expected, optimal supportive measurements critically affect HSCT outcomes. The paucity of good clinical studies in supportive practices gives rise to the establishment of heterogeneous guidelines across the different centers, which hampers direct clinical comparison in multicentric studies. Aiming to harmonize the supportive care provided during the pediatric HSCT in Europe, the Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT) promoted dedicated workshops during the years 2017 and 2018. The present paper describes the resulting consensus on the management of sinusoidal obstructive syndrome, mucositis, enteral and parenteral nutrition, iron overload, and emesis during HSCT.
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- 2020
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11. Safe transfer of pediatric patients from hematopoietic stem cell transplant unit into the pediatric intensive care unit: views of nurses and physicians
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Daphna, Hutt, Eugenia Trigoso, Arjona, Hilda, Mekelenkamp, Jacques-Emmanuel, Galimard, Anne, Kozijn, Teija, Schröder, Marjola, Gjergji, Arnaud, Dalissier, Sarah J, Liptrott, John, Murray, Michelle, Kenyon, J P J, van Gestel, Selim, Corbacioglu, Peter, Bader, and Andre, Willasch
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Cross-Sectional Studies ,Risk Factors ,Physicians ,Hematopoietic Stem Cell Transplantation ,Humans ,Child ,Intensive Care Units, Pediatric - Abstract
Sufficient communication between hematopoietic stem cell transplantation (HSCT) and pediatric intensive care unit (PICU) teams is pivotal for a successful advanced support in the PICU for HSCT-related complications. We evaluated perceived communication and barriers between both teams with the aim of providing recommendations for improvement. In this cross-sectional survey, a self-designed online questionnaire was distributed among ESPNIC and EBMT members. Data were analyzed using descriptive statistics. Over half of HSCT respondents employed a transfer indication protocol and patient assessment tool, but less structured checklist prior to patient transfer. Nearly all PICU respondents perceived this checklist as improvement for communication. Most HSCT and PICU physicians have daily rounds upon patient transfer while this is mostly missing between nursing teams. Half of both HSCT and PICU nurses indicated that HSCT training for PICU nurses could improve communication and patient transfer. Most respondents indicated that structured meetings between HSCT and PICU nurses could improve communication. Overall there is good communication between HSCT and PICU units, although barriers were noted between members of both teams. Based on our findings, we recommend use of a structured and specific checklist by HSCT teams, HSCT training for PICU personnel, and structured meetings between HSCT and PICU nurses.
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- 2021
12. Pregnancy and pregnancy outcomes after hematopoietic stem cell transplantation in childhood: a cross-sectional survey of the EBMT Pediatric Diseases Working Party
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Herbert Pichler, Brenda Gibson, Alicia Rovó, M. Faraci, J E Galimard, P Sedlacek, Selim Corbacioglu, Peter Bader, F Fagioli, I Bodova, Arnaud Dalissier, Gabor Szinnai, Fabienne Gumy-Pause, Adriana Balduzzi, X Poiré, V K Roussou, Jean Hugues Dalle, T Diesch-Furlanetto, UCL - SSS/IREC/SLUC - Pôle St.-Luc, UCL - (SLuc) Centre du cancer, UCL - (SLuc) Service d'hématologie, Diesch-Furlanetto, T, Rovo, A, Galimard, J, Szinnai, G, Dalissier, A, Sedlacek, P, Bodova, I, Roussou, V, Gibson, B, Poire, X, Fagioli, F, Pichler, H, Faraci, M, Gumy-Pause, F, Dalle, J, Balduzzi, A, Bader, P, and Corbacioglu, S
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Counseling ,Adult ,Male ,medicine.medical_specialty ,Pediatrics ,Adolescent ,fertility preservation ,Population ,Reproductive medicine ,Hematopoietic stem cell transplantation ,cryopreservation ,ovarian reserve ,Interquartile range ,Pregnancy ,medicine ,Humans ,Fertility preservation ,education ,610 Medicine & health ,Child ,Retrospective Studies ,Cryopreservation ,Pediatric ,education.field_of_study ,business.industry ,Ovarian reserve ,Rehabilitation ,Hematopoietic Stem Cell Transplantation ,Pregnancy Outcome ,Obstetrics and Gynecology ,Total body irradiation ,medicine.disease ,Transplantation ,Cross-Sectional Studies ,counseling ,pediatric ,Reproductive Medicine ,Female ,business ,Live birth ,General Economics, Econometrics and Finance ,Live Birth - Abstract
STUDY QUESTION What are the characteristics of patients with conceptions transplanted in childhood and adolescence? SUMMARY ANSWER Insemination and conception after hematopoietic stem cell transplantation (HCT) in childhood or adolescence was possible, even after myeloablative conditioning regimes, although some patients required reproductive medicine support. WHAT IS KNOWN ALREADY Preparative regimens of HCT are highly gonadotoxic, which leads to gonadal failure and pubertal development disorders. There are few population-based studies assessing the risk of future infertility in children after HCT. STUDY DESIGN, SIZE, DURATION We conducted a retrospective study to investigate natural or assisted conceptions and their outcomes in patients PARTICIPANTS/MATERIALS, SETTING, METHODS Detailed information concerning pregnancy occurrences and outcomes were obtained by a separate questionnaire. Quantitative variables were presented as medians with their interquartile range (IQR) or range, and categorical variables were presented as frequencies and percentages. MAIN RESULTS AND THE ROLE OF CHANCE In total, 62 988 pediatric patients received a first HCT in EBMT centers between 1995 and 2016. Pregnancy was reported in 406 patients in the database. The median age at transplantation was 15.7 (range: 0.7–18) years, and the median age at declared conception was 25.0 (range: 16.3–38.8) years. Details concerning the first pregnancy and pregnancy outcome were obtained from 99 patients (24%) from the returned questionnaires. The median age at delivery or pregnancy interruption of the females was 23.0 (IQR: 20.8–27) years, with a median time after transplant of 10.7 (IQR: 6.6–15.4) years. Compared with the mean age of healthy women at their first child’s birth (29 years old), the transplanted women delivered 5 years earlier (mean: 24.3 years). In terms of conception modality, 13/25 (52%) females conditioned with total body irradiation (TBI) and 50/52 (96%) of those conditioned without TBI conceived naturally. All seven male patients who had been conditioned with TBI achieved fatherhood but required assisted fertilization or used their cryopreserved sperm. In the females, 63/70 (90%) of all conceptions resulted in a live birth, 49/63 (84.5%) were at term and 43/46 (93%) had normal birthweight. Cesarean delivery was performed in 9/61 (15%) especially in women who had received a myeloablative regimen. LIMITATIONS, REASONS FOR CAUTION In the EBMT pediatric dataset, the age at last follow-up or death was WIDER IMPLICATIONS OF THE FINDINGS Reproductive health surveillance and fertility preservation counseling are important in younger transplanted patients. Our results showed that there is a window of opportunity to conceive naturally or with reproductive medicine support. STUDY FUNDING/COMPETING INTEREST(S) Funding was provided by the ‘Stiftung für krebskranke Kinder Regio Basiliensis’, Basel, Switzerland. All authors have no conflicts of interest to declare. TRIAL REGISTRATION NUMBER N/A.
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- 2021
13. Outcomes of Unmanipulated Haploidentical Transplantation Using Post-Transplant Cyclophosphamide (PT-Cy) in Pediatric Patients With Acute Lymphoblastic Leukemia
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Selim Corbacioglu, Arnaud Dalissier, Annalisa Ruggeri, Akif Yesilipek, Mikael Sundin, José M. Fernández Navarro, Franco Locatelli, Rose-Marie Hamladji, Vanderson Rocha, Maura Faraci, Concepción Herrera, O.V. Paina, Jose Rifón, E V Skorobogatova, Jacques-Emmanuel Galimard, Abdelghani Tbakhi, Franca Fagioli, and Amal Al-Seraihy
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medicine.medical_specialty ,Transplantation Conditioning ,Haploidentical transplantation ,Disease ,Acute lymphoblastic leukemia ,Gastroenterology ,Internal medicine ,medicine ,Immunology and Allergy ,Humans ,Cumulative incidence ,Post-transplantation cyclophosphamide ,Child ,Childhood Acute Lymphoblastic Leukemia ,Cyclophosphamide ,Retrospective Studies ,Transplantation ,business.industry ,Hazard ratio ,Cell Biology ,Hematology ,Precursor Cell Lymphoblastic Leukemia-Lymphoma ,Childhood ,surgical procedures, operative ,medicine.anatomical_structure ,Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA ,Transplantation, Haploidentical ,Molecular Medicine ,Bone marrow ,Stem cell ,business - Abstract
HLA-haploidentical transplantation (haplo-HCT) using post-transplantation-cyclophosphamide (PT-Cy) is a feasible procedure in children with malignancies. However, large studies on Haplo-HCT with PT-Cy for childhood acute lymphoblastic leukemia (ALL) are lacking. We analyzed haplo-HCT outcomes in 180 children with ALL. Median age was 9 years, and median follow-up was 2.7 years. Disease status was CR1 for 24%, CR2 for 45%, CR+3 for 12%, and active disease for 19%. All patients received PT-Cy day +3 and +4. Bone marrow (BM) was the stem cell source in 115 patients (64%). Cumulative incidence of 42-day engraftment was 88.9%. Cumulative incidence of day-100 acute graft-versus-host disease (GVHD) grade II-IV was 28%, and 2-year chronic GVHD was 21.9%. At 2 years, cumulative incidence of nonrelapse mortality (NRM) was 19.6%. Cumulative incidence was 41.9% for relapse and 25% for patients in CR1. Estimated 2-year leukemia free survival was 65%, 44%, and 18.8% for patients transplanted in CR1, CR2, CR3+ and 3% at 1 year for active disease. In multivariable analysis for patients in CR1 and CR2, disease status (CR2 [hazard ratio {HR} = 2.19; P = .04]), age at HCT older than 13 (HR = 2.07; P = .03) and use of peripheral blood stem cell (PBSC) (HR = 1.98; P = .04) were independent factors associated with decreased overall survival. Use of PBSC was also associated with higher NRM (HR = 3.13; P = .04). Haplo-HCT with PT-Cy is an option for children with ALL, namely those transplanted in CR1 and CR2. Age and disease status remain the most important factors for outcomes. BM cells as a graft source is associated with improved survival.
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- 2021
14. Pediatric acute graft-versus-host disease prophylaxis and treatment : surveyed real-life approach reveals dissimilarities compared to published recommendations
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Brenda Gibson, Christina Peters, Dorothea Bauer, Isaac Yaniv, Adriana Balduzzi, Brigitte Strahm, Selim Corbacioglu, Roland Meisel, Kim Vettenranta, Cristina Díaz de Heredia, Jacek Wachowiak, Peter Bader, Jean-Hugues Dalle, Arnaud Dalissier, Anita Lawitschka, Giovanna Lucchini, Victoria Bordon, HUS Children and Adolescents, Lastentautien yksikkö, Children's Hospital, University of Helsinki, Lawitschka, A, Lucchini, G, Strahm, B, Dalle, J, Balduzzi, A, Gibson, B, Diaz De Heredia, C, Wachowiak, J, Dalissier, A, Vettenranta, K, Yaniv, I, Bordon, V, Bauer, D, Bader, P, Meisel, R, Peters, C, and Corbacioglu, S
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Transplantation Conditioning ,BLOOD ,Graft vs Host Disease ,CHILDREN ,Disease ,030230 surgery ,0302 clinical medicine ,immune system diseases ,Surveys and Questionnaires ,hemic and lymphatic diseases ,Extracorporeal Photopheresis ,Child ,MYCOPHENOLATE-MOFETIL ,Response rate (survey) ,Acute leukemia ,treatment ,Hematopoietic Stem Cell Transplantation ,3. Good health ,surgical procedures, operative ,Acute Disease ,Original Article ,030211 gastroenterology & hepatology ,prophylaxis ,Adult ,medicine.medical_specialty ,pediatrics ,ACUTE GVHD ,03 medical and health sciences ,CENTER STRATEGIES ,Clinical Research ,Internal medicine ,Acute graft versus host disease ,medicine ,Humans ,EXTRACORPOREAL PHOTOPHERESIS ,hematopoietic cell transplantation ,MARROW-TRANSPLANTATION ,ACUTE-LEUKEMIA ,Sibling ,Antilymphocyte Serum ,Transplantation ,EUROPEAN GROUP ,Hematopoietic cell ,business.industry ,prophylaxi ,STEM-CELL TRANSPLANTATION ,3126 Surgery, anesthesiology, intensive care, radiology ,pediatric ,business - Abstract
Pediatric allogeneic hematopoietic cell transplantation (HCT) practices differ from those of adults, particularly the heterogeneity of transplantable nonmalignant diseases and the lower incidence of graft-versus-host disease (GVHD). Several guidelines regarding the management of acute (a) GVHD in adult HCT have been published. We aimed to capture the real-life approaches for pediatric aGVHD prophylaxis/treatment, and data from 75/193 (response rate 39%) EBMT centers (26 countries) were included, representing half (48%) of the pediatric EBMT-HCT activity. Results with >= 75% approval from respondents (74/75) for GVHD prophylaxis after myeloablative HCT for malignancies partially contradict published guidelines: Single-agent cyclosporine A (CsA) was used for matched sibling donor HCT in 47%; blood CsA levels were reported lower; the relapse risk in malignant diseases influenced GVHD prophylaxis with early withdrawal of CsA; distinct longer duration of CsA was employed in nonmalignant diseases. Most centers used additional anti-thymocyte globulin for matched unrelated and mismatched donor HCT, but not for matched siblings. Regarding prophylaxis in nonmyeloablative conditioning (mainly for nonmalignant diseases), responses showed broad heterogeneity. High conformity was found for first-line treatment; however, results regarding steroid-refractory aGVHD indicate an earlier diagnosis in children. Our findings highlight the need for standardized pediatric approaches toward aGVHD prophylaxis/treatment differentiated for malignant and nonmalignant underlying diseases.
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- 2020
15. Myeloablative conditioning for allo-HSCT in pediatric ALL
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Adriana Balduzzi, Olga Aleinikova, Damir Nemet, Thomas Klingebiel, Ain Kaare, Sophie Dupont, Manuel Abecasis, E V Skorobogatova, Peter Bader, Jacek Wachowiak, Vassiliki Kitra-Roussou, Gérard Michel, Akif Yesilipek, Arjan C. Lankester, Antonio Campos, Arnaud Dalissier, Tayfun Güngör, Petr Sedlacek, Arcangelo Prete, Cristina Diaz-de-Heredia, Myriam Labopin, Yves Bertrand, K. Nagy, Gergely Kriván, Rose-Marie Hamladji, Jochen Buechner, Amir Ali Hamidieh, Kim Vettenranta, Alphan Kupesiz, Marc Bierings, Ardeshir Ghavamzadeh, Sabina Sufliarska, Jean-Hugues Dalle, Mikael Sundin, Jelena Rascon, Boris V. Afanasyev, Christina Peters, Stephen P. Robinson, Jacques-Emmanuel Galimard, Alicja Chybicka, Amal Al-Seraihy, Selim Corbacioglu, Reuven Or, Paul Veys, Jan Styczyński, Franco Locatelli, Franca Fagioli, Marianne Ifversen, Andre Willasch, Marc Ansari, Herbert Pichler, Alice Bertaina, Willasch, A, Peters, C, Sedláček, P, Dalle, J, Kitra-Roussou, V, Yesilipek, A, Wachowiak, J, Lankester, A, Prete, A, Hamidieh, A, Ifversen, M, Buechner, J, Kriván, G, Hamladji, R, Diaz-de-Heredia, C, Skorobogatova, E, Michel, G, Locatelli, F, Bertaina, A, Veys, P, Dupont, S, Or, R, Güngör, T, Aleinikova, O, Sufliarska, S, Sundin, M, Rascon, J, Kaare, A, Nemet, D, Fagioli, F, Klingebiel, T, Styczynski, J, Bierings, M, Nagy, K, Abecasis, M, Afanasyev, B, Ansari, M, Vettenranta, K, Alseraihy, A, Chybicka, A, Robinson, S, Bertrand, Y, Kupesiz, A, Ghavamzadeh, A, Campos, A, Pichler, H, Dalissier, A, Labopin, M, Corbacioglu, S, Balduzzi, A, Galimard, J, and Bader, P
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Oncology ,medicine.medical_specialty ,Transplantation Conditioning ,medicine.medical_treatment ,Allo hsct ,Hematopoietic stem cell transplantation ,acute lymphoblastic leukemia ,hematopoietic stem cell transplantation, acute lymphoblastic leukemia, total body irradiation ,Article ,03 medical and health sciences ,0302 clinical medicine ,Internal medicine ,medicine ,Humans ,Transplantation, Homologous ,Child ,Etoposide ,Myeloablative conditioning for allo-HSCT ,residual neoplasm ,pre B lymphocyte ,Retrospective Studies ,Transplantation ,Chemotherapy ,Precursor Cell Lymphoblastic Leukemia-Lymphoma / therapy ,ddc:618 ,Acute lymphocytic leukaemia ,business.industry ,Incidence (epidemiology) ,Myeloablative conditioning ,Hematopoietic Stem Cell Transplantation ,Hematology ,MED/38 - PEDIATRIA GENERALE E SPECIALISTICA ,Total body irradiation ,Survival Analysis ,Stem-cell research ,Leukemia, Myeloid, Acute ,medicine.anatomical_structure ,Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA ,030220 oncology & carcinogenesis ,Bone marrow ,business ,Whole-Body Irradiation ,030215 immunology ,medicine.drug - Abstract
Although most children with acute lymphoblastic leukemia (ALL) receive fractionated total body irradiation (FTBI) as myeloablative conditioning (MAC) for allogeneic hematopoietic stem cell transplantation (allo-HSCT), it is an important matter of debate if chemotherapy can effectively replace FTBI. To compare outcomes after FTBI versus chemotherapy-based conditioning (CC), we performed a retrospective EBMT registry study. Children aged 2–18 years after MAC for first allo-HSCT of bone marrow (BM) or peripheral blood stem cells (PBSC) from matched-related (MRD) or unrelated donors (UD) in first (CR1) or second remission (CR2) between 2000 and 2012 were included. Propensity score weighting was used to control pretreatment imbalances of the observed variables. 3.054 patients were analyzed. CR1 (1.498): median follow-up (FU) after FTBI (1.285) and CC (213) was 6.8 and 6.1 years. Survivals were not significantly different. CR2 (1.556): median FU after FTBI (1.345) and CC (211) was 6.2 years. Outcomes after FTBI were superior as compared with CC with regard to overall survival (OS), leukemia-free survival (LFS), relapse incidence (RI), and nonrelapse mortality (NRM). However, we must emphasize the preliminary character of the results of this retrospective “real-world-practice” study. These findings will be prospectively assessed in the ALL SCTped 2012 FORUM trial.
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- 2020
16. Second Hematopoietic Stem Cell Transplantation for Post-Transplantation Relapsed Acute Leukemia in Children: A Retrospective EBMT-PDWP Study
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Petr Sedlacek, Stefania Varotto, Miguel Angel Diaz, Arnaud Dalissier, Isaac Yaniv, Christina Peters, Boris V. Afanasyev, Peter Bader, Eric Beohou, Selim Corbacioglu, Krzysztof Kałwak, Aviva Krauss, Massimo Berger, and Marco Zecca
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Male ,Reoperation ,medicine.medical_specialty ,Adolescent ,medicine.medical_treatment ,Graft vs Host Disease ,Disease ,Hematopoietic stem cell transplantation ,03 medical and health sciences ,Myelogenous ,0302 clinical medicine ,Recurrence ,hemic and lymphatic diseases ,Internal medicine ,medicine ,Humans ,Nonrelapse mortality ,Child ,Retrospective Studies ,Transplantation ,Acute leukemia ,Marrow transplantation ,business.industry ,Hematopoietic Stem Cell Transplantation ,Hematology ,Precursor Cell Lymphoblastic Leukemia-Lymphoma ,Prognosis ,medicine.disease ,Survival Analysis ,Post transplant ,Leukemia, Myeloid, Acute ,Leukemia ,Child, Preschool ,030220 oncology & carcinogenesis ,Female ,business ,030215 immunology - Abstract
Outcome data were collected from the European Society for Blood and Marrow Transplantation registry on 373 children from 120 centers with relapsed leukemia (214 with acute lymphoblastic leukemia [ALL] and 159 with acute myelogenous leukemia [AML]) who underwent second allogeneic hematopoietic stem cell transplantation (HSCT) between 2004 and 2013. Overall survival (OS) was 38% at 2 years and 29% at 5 years, and leukemia-free survival (LFS) was 30% at 2 years and 25% at 5 years. Median follow-up after second HSCT was 36.4 months in the ALL group and 50.2 months in the AML group. In the ALL group, OS was 43% at 2 years and 33% at 5 years, and LFS was 34% at 2 years and 31% at 5 years. In the AML group, OS was 32% at 2 years and 24% at 5 years, and LFS was 24% at 2 years and 17% at 5 years. The 2-year nonrelapse mortality (NRM) rate was 22% in the ALL group and 18% in the AML group. Favorable prognostic factors (P .05) for OS and LFS included12 months between transplantations and chronic graft-versus-host disease after the first HSCT (in both groups), complete response before the second HSCT (ALL group only), and age12 years (AML group only). Findings were more consistent over time in the ALL group, with no significant differences between 2-year and 5-year rates of relapse, NRM, and LFS. Children with relapsed acute leukemias have a substantial likelihood of long-term survival following second HSCT. Given the many novel targeted and immunomodulation therapies currently under development, it is important to identify specific patient subpopulations that may benefit from a second HSCT compared with those better suited to new approaches.
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- 2018
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17. Outcome of Allogeneic HSCT after Chemo-Based Conditioning in Infants with Acute Myeloid Leukemia in First Complete Remission: A Multicenter EBMT-PDWP Study
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Amos Toren, Franco Locatelli, Adriana Balduzzi, Gérard Michel, Alessandra Biffi, Andre Willasch, Dragana Vujic, Charlotte Jubert, Maud Ngoya, José M. Moraleda, Maura Faraci, Tracey A. O'Brien, Vassiliki Kitra-Roussou, Selim Corbacioglu, Arnaud Dalissier, Marco Zecca, Mikael Sundin, Mariacristina Menconi, Franca Fagioli, Jacques-Emmanuel Galimard, Peter Bader, Fanny Rialland, Christina Peters, Paul Veys, Jean-Hugues Dalle, Cécile Pochon, Yves Bertrand, Stephen P. Robinson, Jacques-Olivier Bay, Ottavio Ziino, and Amal Al-Seraihy
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Oncology ,0303 health sciences ,medicine.medical_specialty ,business.industry ,Immunology ,Complete remission ,Myeloid leukemia ,Cell Biology ,Hematology ,Biochemistry ,3. Good health ,03 medical and health sciences ,0302 clinical medicine ,Internal medicine ,Allogeneic hsct ,medicine ,business ,health care economics and organizations ,030304 developmental biology ,030215 immunology - Abstract
Background: Pediatric patients younger than two years of age with acute myeloid leukemia (AML) commonly receive a chemotherapy-based myeloablative conditioning regimen before allogeneic hematopoietic stem cell transplantation (HSCT). The optimal choice of cytotoxic agents is still controversial. Methods: A retrospective EBMT-registry based study was conducted to investigate the impact of different chemotherapy-based conditionings on the outcomes in young children. Children younger than two years of age receiving a first HSCT of bone marrow (BM), peripheral blood stem cells (PBSC) or cord blood (CB) from matched siblings (MSD) or unrelated donors (UD) in first complete remission (CR1) between 2000 and 2019 were included. Busulfan/Cyclophosphamide (BuCy) and BuCy/Melphalan (BuCyMel) were the most frequent combinations on which this analysis focused. The primary endpoint was leukemia-free survival (LFS). Multivariate analysis adjusting for differences between the conditioning regimens and risk factors influencing outcome was performed using the Cox's proportional hazards regression model. Results: 289 patients (56% male) transplanted at a median age of 1.2 years (IQR 0.9-1.6) after BuCy (164, 57%) or BuCyMel (125, 43%) were included. 184 (64%) patients received BM, 71 (24%) CB and 34 (12%) PBSC from UD (201, 70%) and MSD (88, 30%). In-vivo T-cell-depletion (TCD) was performed in 160 (58%, missing data 14) of the HSCTs with anti-thymocyte-globulin (ATG, 153) or alemtuzumab (7). Ex-vivo TCD was performed in 13 (5%, missing data 3) of the HSCTs. Graft-versus-host-disease (GvHD)-prophylaxis was Cyclosporin-A-based in 90% of the HSCTs. Median follow-up (FU) was 4.9 years (95% CI 3.9-5.5). After a median FU of 4 years, 4-y-LFS after BuCyMel (74.3%, 95% CI 65.1-81.4) was significantly better compared to BuCy (59.7%, 95% CI 51.2-67.2), hazard ratio (HR) 0.56 (95% CI 0.35-0.90, P=0.02). Overall survival (4-y-OS) after BuCyMel (77.2%, 95% CI 68.1-84.0) was significantly better compared to BuCy (66.6%, 95% CI 58.0-73.8), HR=0.58 (95% CI 0.35-0.97, P=0.04). No significant differences were found in the probability of relapse (4-y-RI (whole cohort) 26.2% (95% CI 21.0-31.7), HR of BuCyMel 0.59 (95% CI 0.34-1.02), P=0.06), non-relapse mortality (4-y-NRM (whole cohort) 7.8% (95% CI 5.0-11.4), HR of BuCyMel 0.49 (95% CI 0.19-1.24), P=0.13) and incidence of acute grade II-IV GvHD at day 100 (day-100-aGvHD II-IV (whole cohort) 36.8% (95% CI 31.2-42.5), HR of BuCyMel 0.59 (95% CI 0.35-1.01), P=0.06). Incidence of chronic GvHD (4-y-cGvHD (whole cohort)) was 9.8% (95%-CI 6.3-14.2). The donor type had no significant influence on the outcome. Conclusion: Bu-based conditionings of HSCT for infants with AML at high risk of relapse offer a high probability of cure. Conditioning with three alkylators (BuCyMel) resulted in better LFS and OS compared with two alkylators (BuCy) without significantly increasing the risk of both NRM and aGvHD. Future trials will evaluate the impact of the more recently introduced alkylator Treosulfan within the conditioning of HSCT in pediatric AML. Disclosures Peters: Amgen: Membership on an entity's Board of Directors or advisory committees, Other: Travel grants. Locatelli: Amgen: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Novartis: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Miltenyi: Speakers Bureau; Medac: Speakers Bureau; Jazz Pharamceutical: Speakers Bureau; Takeda: Speakers Bureau. Moraleda: Pfizer: Other: Educational Grants, Research Funding; Sanofi: Other: Educational Grants, Research Funding; MSD: Other: Educational Grants, Research Funding; ROCHE: Consultancy, Honoraria, Other: Educational Grants, Research Funding; Takeda: Consultancy, Honoraria, Other: Educational Grants, Research Funding; Sandoz: Consultancy, Honoraria; Novartis: Consultancy, Honoraria, Other: Educational Grants, Research Funding; Gilead: Consultancy, Honoraria, Other: Educational Grants, Research Funding; Jazz Pharmaceuticals: Consultancy, Honoraria, Other: Educational Grants, Research Funding; NovoNordisk: Other: Educational Grants, Research Funding; Janssen: Other: Educational Grants, Research Funding; Celgene: Other: Educational Grants, Research Funding; Amgen: Other: Educational Grants, Research Funding. Biffi: BlueBirdBio: Consultancy, Other: Advisory Board. Corbacioglu: Gentium/Jazz Pharmaceuticals: Consultancy, Honoraria.
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- 2021
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18. Correction: Myeloablative conditioning for allo-HSCT in pediatric ALL: FTBI or chemotherapy?—A multicenter EBMT-PDWP study
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Cristina Diaz-de-Heredia, Rose-Marie Hamladji, Ardeshir Ghavamzadeh, Sabina Sufliarska, Thomas Klingebiel, Sophie Dupont, Alicja Chybicka, Tayfun Güngör, Amal Al-Seraihy, Christina Peters, Akif Yesilipek, Jacek Wachowiak, Gérard Michel, Arcangelo Prete, Boris V. Afanasyev, Myriam Labopin, Kitra-Roussou, Reuven Or, Adriana Balduzzi, Ain Kaare, Olga Aleinikova, Paul Veys, Franco Locatelli, Franca Fagioli, Amir Ali Hamidieh, Yves Bertrand, K. Nagy, Marc Ansari, Alice Bertaina, Jochen Buechner, Jean-Hugues Dalle, Arnaud Dalissier, Peter Bader, Stephen P. Robinson, Herbert Pichler, Petr Sedlacek, A Campos, Selim Corbacioglu, Jacques-Emmanuel Galimard, E V Skorobogatova, Gergely Kriván, Arjan C. Lankester, Andre Willasch, Kim Vettenranta, Manuel Abecasis, Damir Nemet, Marianne Ifversen, Alphan Kupesiz, Mikael Sundin, Jelena Rascon, Marc Bierings, and Jan Styczyński
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Oncology ,medicine.medical_specialty ,Transplantation Conditioning ,Bone marrow transplantation ,medicine.medical_treatment ,Allo hsct ,Internal medicine ,medicine ,Humans ,Transplantation, Homologous ,Child ,Retrospective Studies ,Transplantation ,Chemotherapy ,Acute lymphocytic leukaemia ,business.industry ,Myeloablative conditioning ,Hematopoietic Stem Cell Transplantation ,Correction ,Hematology ,Precursor Cell Lymphoblastic Leukemia-Lymphoma ,Survival Analysis ,Stem-cell research ,Leukemia, Myeloid, Acute ,business ,Whole-Body Irradiation - Abstract
Although most children with acute lymphoblastic leukemia (ALL) receive fractionated total body irradiation (FTBI) as myeloablative conditioning (MAC) for allogeneic hematopoietic stem cell transplantation (allo-HSCT), it is an important matter of debate if chemotherapy can effectively replace FTBI. To compare outcomes after FTBI versus chemotherapy-based conditioning (CC), we performed a retrospective EBMT registry study. Children aged 2-18 years after MAC for first allo-HSCT of bone marrow (BM) or peripheral blood stem cells (PBSC) from matched-related (MRD) or unrelated donors (UD) in first (CR1) or second remission (CR2) between 2000 and 2012 were included. Propensity score weighting was used to control pretreatment imbalances of the observed variables. 3.054 patients were analyzed. CR1 (1.498): median follow-up (FU) after FTBI (1.285) and CC (213) was 6.8 and 6.1 years. Survivals were not significantly different. CR2 (1.556): median FU after FTBI (1.345) and CC (211) was 6.2 years. Outcomes after FTBI were superior as compared with CC with regard to overall survival (OS), leukemia-free survival (LFS), relapse incidence (RI), and nonrelapse mortality (NRM). However, we must emphasize the preliminary character of the results of this retrospective "real-world-practice" study. These findings will be prospectively assessed in the ALL SCTped 2012 FORUM trial.
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- 2021
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19. High dose chemotherapy and autologous hematopoietic cell transplantation for Wilms tumor: a study of the European Society for Blood and Marrow Transplantation
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Arnaud Dalissier, Jean Michon, C Peters, Gianni Bisogno, Andre Willasch, Franco Locatelli, Ulrike Pötschger, Peter Bader, Norbert Graf, Sandro Dallorso, M van den Heuvel Eibrink, Filippo Spreafico, and Daniel Yeomanson
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Male ,Melphalan ,Oncology ,medicine.medical_specialty ,Platelet Engraftment ,HIGH DOSE CHEMOTHERAPY ,medicine.medical_treatment ,Population ,ThioTEPA ,Transplantation, Autologous ,Bone Marrow ,Internal medicine ,WILMS TUMOR ,Antineoplastic Combined Chemotherapy Protocols ,medicine ,Humans ,clear cell sarcoma ,nephroblastoma ,patient with Wilms' tumor ,Child ,education ,Transplantation ,Chemotherapy ,education.field_of_study ,business.industry ,Hematopoietic Stem Cell Transplantation ,Wilms' tumor ,Hematology ,medicine.disease ,Combined Modality Therapy ,Kidney Neoplasms ,Regimen ,Treatment Outcome ,Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA ,Neoplasm Recurrence, Local ,business ,medicine.drug - Abstract
Survival for subgroups of patients with Wilms tumor (WT), such as those who suffer from relapse, is disappointing. Some patients' treatment plans include high-dose chemotherapy (HDT) with autologous hematopoietic cell transplantation (aHCT), although proof for its benefit is lacking. To increase the level of evidence regarding children with WT receiving aHCT as consolidation of first or second remission (after first relapse), we extracted relevant data from the European Blood and Marrow Transplantation Registry concerning 69 patients. Different HDT regimens were administered, mostly either melphalan-containing (n = 34) or thiotepa-containing (n = 14). For the whole population, 5-year overall survival (OS) and event-free survival (EFS) probabilities were 0.67 (±0.06) and 0.63 (±0.06), respectively (median observation time 7.8 years); for children transplanted in first remission, OS and EFS were 0.69 (±0.09) and 0.72 (±0.08). In univariate analysis, male gender and relapse in multiple sites were associated with lower OS probabilities. The use of a given pretransplant regimen (i.e. melphalan alone versus regimens with multiple drugs) did not seem to influence EFS/OS probability after aHCT, but significantly influenced platelet engraftment (more delayed with thiotepa). We here provide further data to improve the basis for future evidence-based clinical decision-making when using HDT and aHCT in relapsed/refractory WT.
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- 2020
20. Solid organ transplantation after hematopoietic stem cell transplantation in childhood: A multicentric retrospective survey
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Petr Sedlacek, Jaap Jan Boelens, Joern S Kühl, Edoardo Lanino, Birgit Burkhardt, Bernd Gruhn, Susanne Matthes-Martin, Audrey Guilmatre, Isabel Badell Serra, Alice Bertaina, Cristina Diaz-de-Heredia, Arjan C. Lankester, Marianne Ifversen, Eric Beohou, Manuel Abecasis, Stefania Varotto, Ansgar Schulz, Peter Bader, Maura Faraci, Birgit Garwer, Arnaud Dalissier, Andrew R. Gennery, Ana Sastre, and Catherine Paillard
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Male ,medicine.medical_treatment ,Graft vs Host Disease ,heart failure ,Hematopoietic stem cell transplantation ,Disease ,030230 surgery ,Cohort Studies ,Liver disease ,0302 clinical medicine ,Surveys and Questionnaires ,Immunology and Allergy ,Pharmacology (medical) ,organ transplantation in general ,Autografts ,Child ,dysfunction ,Allografts ,practice ,Europe ,Survival Rate ,Treatment Outcome ,kidney failure/injury ,surgical procedures, operative ,medicine.anatomical_structure ,Child, Preschool ,hematopoietic stem cell transplantation ,Female ,medicine.symptom ,liver disease ,graft-vs-host disease (GVHD) ,Lung Transplantation ,medicine.medical_specialty ,bone marrow ,Adolescent ,pediatrics ,injury ,clinical research/practice ,heart failure/injury ,03 medical and health sciences ,Retrospective survey ,Internal medicine ,medicine ,Humans ,bone marrow/hematopoietic stem cell transplantation ,Proportional Hazards Models ,Retrospective Studies ,Transplantation ,Lung ,business.industry ,Organ dysfunction ,Infant ,Organ Transplantation ,medicine.disease ,Kidney Transplantation ,Confidence interval ,lung (allograft) function ,Liver Transplantation ,kidney failure ,clinical research ,lung (allograft) function/dysfunction ,Heart Transplantation ,business ,Solid organ transplantation - Abstract
We report data obtained from a retrospective multicenter pediatric survey on behalf of the European Society for Blood and Marrow Transplantation (EBMT). Information on solid organ transplantation (SOT) performed in pediatric recipients of either autologous or allogeneic hematopoietic stem cell transplantation (HSCT) between 1984 and 2016 was collected in 20 pediatric EBMT Centers (25.6%). Overall, we evaluated data on 44 SOTs following HSCT including 20 liver (LTx), 12 lung (LuTx), 6 heart (HTx), and 6 kidney (KTx) transplantations. The indication for SOT was organ failure related to intractable graft-vs-host disease in 16 children (36.3%), acute or chronic HSCT-related toxicity in 18 (40.9%), and organ dysfunction related to the underlying disease in 10 (22.8%). The median follow-up was 10.9 years (95% confidence interval: 1.7-29.5). The overall survival rate at 1 and 5 years after SOT was 85.7% and 80.4%, respectively: it was 74% and 63.2% after LTx, 83.2% after HTx, and 100% equally after LuTx and KTx. This multicenter survey confirms that SOT represents a promising option in children with severe organ failure occurring after HSCT. Additional studies are needed to further establish the effectiveness of SOT after HSCT and to better understand the mechanism underlying this encouraging success.
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- 2019
21. More chronic GvHD and non-relapse mortality after peripheral blood stem cell compared with bone marrow in hematopoietic transplantation for paediatric acute lymphoblastic leukemia: a retrospective study on behalf of the EBMT Paediatric Diseases Working Party
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J.-H. Dalle, Adriana Balduzzi, M. Labopin, Arnaud Dalissier, Peter Bader, M. Simonin, Andre Willasch, Marco Zecca, Christoph Peters, Alicja Chybicka, Liisa Volin, A. Mouhab, Simonin, M, Dalissier, A, Labopin, M, Willasch, A, Zecca, M, Mouhab, A, Chybicka, A, Balduzzi, A, Volin, L, Peters, C, Bader, P, and Dalle, J
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Male ,medicine.medical_specialty ,Adolescent ,Graft vs Host Disease ,03 medical and health sciences ,0302 clinical medicine ,Allograft ,Retrospective Studie ,hemic and lymphatic diseases ,Internal medicine ,Humans ,Medicine ,030212 general & internal medicine ,Progenitor cell ,Child ,Retrospective Studies ,Bone Marrow Transplantation ,Peripheral Blood Stem Cell Transplantation ,Transplantation ,Hematology ,business.industry ,Infant ,Retrospective cohort study ,Precursor Cell Lymphoblastic Leukemia-Lymphoma ,Allografts ,medicine.disease ,humanities ,Haematopoiesis ,surgical procedures, operative ,Graft-versus-host disease ,medicine.anatomical_structure ,Child, Preschool ,030220 oncology & carcinogenesis ,Immunology ,Female ,Bone marrow ,Stem cell ,business ,Human - Abstract
More chronic GvHD and non-relapse mortality after peripheral blood stem cell compared with bone marrow in hematopoietic transplantation for paediatric acute lymphoblastic leukemia: a retrospective study on behalf of the EBMT Paediatric Diseases Working Party
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- 2017
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22. Correction: ABO incompatibile graft management in pediatric transplantation
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Toni Matic, Alessandro Cattoni, Marianne Ifversen, Christina Peters, Kim Vettenranta, Jean-Hugues Dalle, Arnaud Dalissier, Andre Willasch, Peter Svec, Akif Yesilipek, Halvard Bonig, Giovanna Lucchini, K. Kałwak, Peter Bader, Marc Ansari, Jochen Buechner, Adriana Balduzzi, Anita Lawitschka, Brenda Gibson, Andrea Jarisch, Roland Meisel, Selim Corbacioglu, Tiago Nava, Gergely Kriván, and Giulia Prunotto
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Transplantation ,Pediatrics ,medicine.medical_specialty ,business.industry ,Pediatric transplantation ,ABO blood group system ,MEDLINE ,Medicine ,Hematology ,business - Published
- 2020
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23. Transplant center practices for psychosocial assessment and management of pediatric hematopoietic stem cell donors
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Bronwen E. Shaw, Lori Wiener, Brent R. Logan, Galen E. Switzer, Chloe Anthias, Wendy Pelletier, Andre Willasch, Jennifer A. Sees, Nirali N. Shah, Dennis L. Confer, Jennifer A. Hoag, Jessica G. Bruce, Gregory M.T. Guilcher, Arnaud Dalissier, Michael A. Pulsipher, and Peter Bader
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Male ,medicine.medical_specialty ,Adolescent ,Cross-sectional study ,Emotions ,MEDLINE ,03 medical and health sciences ,0302 clinical medicine ,Surveys and Questionnaires ,Living Donors ,Medicine ,Humans ,Sibling ,Child ,Transplantation ,business.industry ,Follow up studies ,Hematopoietic Stem Cell Transplantation ,Hematology ,Hematopoietic Stem Cells ,Clinical trial ,Cross-Sectional Studies ,Multicenter study ,030220 oncology & carcinogenesis ,Donation ,Family medicine ,Female ,business ,Psychosocial ,030215 immunology ,Follow-Up Studies - Abstract
Understanding the potential emotional and psychological risks of pediatric sibling HSC donation is an area of research that remains in its infancy. A cross-sectional survey was distributed electronically to directors at all CIBMTR and EBMT centers to describe current transplant center practices for obtaining assent, preparation for the physical/emotional experiences of donation, and monitoring the post-donation well-being of pediatric donors (
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- 2019
24. Correction: Supportive care during pediatric hematopoietic stem cell transplantation: beyond infectious diseases. A report from workshops on supportive care of the Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT)
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Isaac Yaniv, Jochen Buechner, Petr Sedlacek, Anita Lawitschka, U Falkenberg, Toni Matic, Luisa Sisinni, Marc Ansari, Jacek Wachowiak, Jerry Stein, Gergely Kriván, Roland Meisel, Halvard Boenig, Riitta Niinimäki, Shahrzad Bakhtiar, Andrea Jarisch, Thomas Lehrnbecher, Krzysztof Kałwak, E. Trigoso, Akif Yeşilipek, Daphna Hutt, Arnaud Dalissier, Michaela Kuhlen, Tiago Nava, J.-H. Dalle, Kim Vettenranta, Koray Yalcin, Adriana Balduzzi, Selim Corbacioglu, Marco Deiana, Marianne Ifversen, Tamara Diesch, Simone Cesaro, Giovanna Lucchini, C D de Heredia, Brenda Gibson, Alice Bertaina, Andre Willasch, Tayfun Güngör, Christoph Peters, Dominik Turkiewicz, and Peter Bader
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Transplantation ,medicine.medical_specialty ,Bone marrow transplantation ,Marrow transplantation ,business.industry ,medicine.medical_treatment ,medicine ,Hematology ,Hematopoietic stem cell transplantation ,Intensive care medicine ,business - Abstract
An amendment to this paper has been published and can be accessed via a link at the top of the paper.
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- 2020
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25. Haematopoietic stem cell transplantation for refractory Langerhans cell histiocytosis: outcome by intensity of conditioning
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R. Maarten Egeler, Paul J. Orchard, Mary Eapen, Giuseppe Bandini, Paul Veys, Jeffrey H. Davis, Susanne Matthes, Gretchen Eames, Olle Ringdén, K. Scott Baker, Herbert Jürgens, Alexandra H. Filipovich, Paul G. Schlegel, Vasanta Nanduri, Anna Pieczonka, Alain Fischer, Wing Leung, Anne Sirvent, Andrea Biondi, Robert A. Krance, Edoardo Lanino, Wensheng He, Gérard Michel, Kim Vettenranta, Arnaud Dalissier, Veys, P, Nanduri, V, Baker, K, He, W, Bandini, G, Biondi, A, Dalissier, A, Davis, J, Eames, G, Egeler, R, Filipovich, A, Fischer, A, Jürgens, H, Krance, R, Lanino, E, Leung, W, Matthes, S, Michel, G, Orchard, P, Pieczonka, A, Ringdén, O, Schlegel, P, Sirvent, A, Vettenranta, K, and Eapen, M
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Adult ,Male ,medicine.medical_specialty ,Transplantation Conditioning ,Allogeneic transplantation ,Survival ,Adolescent ,medicine.medical_treatment ,Graft vs Host Disease ,Hematopoietic stem cell transplantation ,Article ,Conditioning regimen intensity ,Young Adult ,Refractory ,Langerhans cell histiocytosis ,Retrospective Studie ,hemic and lymphatic diseases ,medicine ,Humans ,Transplantation, Homologous ,Child ,Transplantation, Homologou ,Retrospective Studies ,Chemotherapy ,business.industry ,Langerhans cell histiocytosi ,Hematopoietic Stem Cell Transplantation ,Infant ,Hematology ,medicine.disease ,3. Good health ,Surgery ,Transplantation ,Histiocytosis, Langerhans-Cell ,Treatment Outcome ,surgical procedures, operative ,Treatment failure ,Child, Preschool ,Cytarabine ,Female ,business ,Human ,medicine.drug - Abstract
Patients with Langerhans cell histiocytosis (LCH) refractory to conventional chemotherapy have a poor outcome. There are currently two promising treatment strategies for high-risk patients: the first involves the combination of 2-chlorodeoxyadenosine and cytarabine; the other approach is allogeneic haematopoietic stem cell transplantation (HSCT). Here we evaluated 87 patients with high-risk LCH who were transplanted between 1990 and 2013. Prior to the year 2000, most patients underwent HSCT following myeloablative conditioning (MAC): only 5 of 20 patients (25%) survived with a high rate (55%) of transplant-related mortality (TRM). After the year 2000 an increasing number of patients underwent HSCT with reduced intensity conditioning (RIC): 49/67 (73%) patients survived, however, the improved survival was not overtly achieved by the introduction of RIC regimens with similar 3-year probability of survival after MAC (77%) and RIC transplantation (71%). There was no significant difference in TRM by conditioning regimen intensity but relapse rates were higher after RIC compared to MAC regimens (28% vs. 8%, P = 0·02), although most patients relapsing after RIC transplantation could be salvaged with further chemotherapy. HSCT may be a curative approach in 3 out of 4 patients with high risk LCH refractory to chemotherapy: the optimal choice of HSCT conditioning remains uncertain.
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- 2015
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26. State-of-the-art fertility preservation in children and adolescents undergoing haematopoietic stem cell transplantation: a report on the expert meeting of the Paediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT) in Baden, Austria, 29-30 September 2015
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A.C. Lankester, Dorine Bresters, C. Diaz de Heredia Rubio, Catherine Poirot, Akif Yeşilipek, Brenda Gibson, Isaac Yaniv, Rebecca Moffat, Peter Bader, K. T. Macklon, Marianne Ifversen, Selim Corbacioglu, M. von Wolff, T. Klingebiel, Kirsi Jahnukainen, E. Trigoso, Anita Lawitschka, Petr Sedlacek, A. Ahler, J.-H. Dalle, Giovanna Lucchini, Christoph Peters, Arnaud Dalissier, Andre Willasch, Jacek Wachowiak, Tamara Diesch, Marc Ansari, Andrea Jarisch, Kim Vettenranta, N. Saenger, Adriana Balduzzi, Eric Beohou, Dalle, J, Lucchini, G, Balduzzi, A, Ifversen, M, Jahnukainen, K, Macklon, K, Ahler, A, Jarisch, A, Ansari, M, Beohou, E, Bresters, D, Corbacioglu, S, Dalissier, A, de Heredia Rubio, C, Diesch, T, Gibson, B, Klingebiel, T, Lankester, A, Lawitschka, A, Moffat, R, Peters, C, Poirot, C, Saenger, N, Sedlacek, P, Trigoso, E, Vettenranta, K, Wachowiak, J, Willasch, A, von Wolff, M, Yaniv, I, Yesilipek, A, and Bader, P
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Male ,Infertility ,medicine.medical_specialty ,Adolescent ,media_common.quotation_subject ,medicine.medical_treatment ,Reproductive medicine ,Fertility ,Hematopoietic stem cell transplantation ,03 medical and health sciences ,0302 clinical medicine ,medicine ,Humans ,Fertility preservation ,Child ,Intensive care medicine ,Infertility, Male ,Societies, Medical ,media_common ,Transplantation ,030219 obstetrics & reproductive medicine ,business.industry ,Female infertility ,Hematopoietic Stem Cell Transplantation ,Hematology ,Congresses as Topic ,medicine.disease ,3. Good health ,Surgery ,Europe ,surgical procedures, operative ,Graft-versus-host disease ,Austria ,030220 oncology & carcinogenesis ,Female ,fertility preservation, hematopoietic stem cell transplantation, pediatric, late effects ,business ,Infertility, Female - Abstract
Nowadays, allogeneic haematopoietic stem cell transplantation (allo-HSCT) is a well-established treatment procedure and often the only cure for many patients with malignant and non-malignant diseases. Decrease in short-term complications has substantially contributed to increased survival. Therefore long-term sequelae are reaching the focus of patient care. One of the most important risks of stem cell transplant survivors is infertility. As well as in the field of allo-HSCT also the field of reproductive medicine has achieved substantial advances to offer potential options for fertility preservation in both boys and girls. Access to these procedures as well as their financing differs significantly throughout Europe. As all European children and adolescents should have the same possibility, the Paediatric Diseases Working Party of the European Society for Blood and Marrow Transplantation organised an expert meeting in September 2015. This manuscript describes the recommendations for the diagnosis and pre-emptive procedures that should be offered to all children and adolescents in Europe who have to undergo an allo-HSCT.
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- 2017
27. Use of defibrotide to treat transplant-associated thrombotic microangiopathy: a retrospective study of the Paediatric Diseases and Inborn Errors Working Parties of the European Society of Blood and Marrow Transplantation
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Annemieke Willekens, Michel Duval, Cornelio Uderzo, W. H. Y. Hwang, Andrew R. Gennery, Shin Yeu Ong, Peter Bader, Mary Slatter, Sonia Bonanomi, Laura Yeates, Ansgar Shulz, Francesca Lorraine Wei Inng Lim, Walter Barberi, Carsten Heilmann, and Arnaud Dalissier
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Adult ,Male ,medicine.medical_specialty ,Thrombotic microangiopathy ,medicine.medical_treatment ,Hematopoietic stem cell transplantation ,Defibrotide ,03 medical and health sciences ,0302 clinical medicine ,Polydeoxyribonucleotides ,medicine ,Humans ,Intensive care medicine ,Child ,Societies, Medical ,Bone Marrow Transplantation ,Retrospective Studies ,Transplantation ,business.industry ,Marrow transplantation ,Thrombotic Microangiopathies ,Genetic Diseases, Inborn ,Hematopoietic Stem Cell Transplantation ,Infant, Newborn ,Infant ,Retrospective cohort study ,Hematology ,Middle Aged ,medicine.disease ,Allografts ,humanities ,Surgery ,Europe ,surgical procedures, operative ,Graft-versus-host disease ,Multicenter study ,030220 oncology & carcinogenesis ,Child, Preschool ,Female ,business ,030215 immunology ,medicine.drug - Abstract
Use of defibrotide to treat transplant-associated thrombotic microangiopathy: a retrospective study of the Paediatric Diseases and Inborn Errors Working Parties of the European Society of Blood and Marrow Transplantation
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- 2017
28. Fertility preservation practices in pediatric and adolescent cancer patients undergoing HSCT in Europe: a population-based survey
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Marta Pillon, Arnaud Dalissier, Alicia Rovó, J.-H. Dalle, Tamara Diesch, Maura Faraci, N. X. von der Weid, and Peter Bader
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Male ,Infertility ,Pediatrics ,medicine.medical_specialty ,Adolescent ,medicine.medical_treatment ,media_common.quotation_subject ,MEDLINE ,Fertility ,Hematopoietic stem cell transplantation ,03 medical and health sciences ,0302 clinical medicine ,medicine ,Humans ,Fertility preservation ,Child ,610 Medicine & health ,Infertility, Male ,media_common ,Transplantation ,030219 obstetrics & reproductive medicine ,business.industry ,Female infertility ,Hematopoietic Stem Cell Transplantation ,Hematology ,Allografts ,medicine.disease ,Europe ,Clinical trial ,030220 oncology & carcinogenesis ,Female ,business ,Infertility, Female - Abstract
Chemotherapy and irradiation can affect the gonads, leading to impairment of pubertal development and/or infertility. Fertility preservation (FP) is therefore a crucial endeavor in hematopoietic stem cell transplantation (HSCT) because of the severe impact of infertility on the quality of life of long-term survivors. Despite the existence of different international guidelines, FP counseling and procedures are not routinely implemented as part of patient care. We present herein a survey conducted by the Pediatric Working Party of the European Society for Blood and Marrow Transplantation (EBMT), which aims to analyze and compare different FP practices for children and adolescents across EBMT centers in 2013. A total of 177 pediatric centers reporting to the EBMT were contacted; of this number, 38 centers (21%) located in 16 different countries responded. These centers reported 834 patients receiving HSCT in 2013 (73% prepubertal), corresponding to 22% of all children (n=3789) undergoing HSCT in EBMT reporting centers. Overall, 39% of the reported patients received counseling and 29% received an FP procedure. The increased need for FP programs, extended education for patient-care teams, and more personal resources and funding emerged from this survey as pivotal factors necessary to support and implement such programs.Bone Marrow Transplantation advance online publication, 23 January 2017; doi:10.1038/bmt.2016.363.
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- 2017
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29. Stem Cell Transplantation for Pediatric Patients with Non-Anaplastic Peripheral T-Cell Lymphoma on Behalf of the EBMT-Pediatric Diseases Working Party
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Birgit Burkhardt, Peter Bader, Eric Beohou, Andre Willasch, Karin Mellgren, Wilhelm Woessmann, Udo Kontny, Olga Moser, and Arnaud Dalissier
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Oncology ,medicine.medical_specialty ,business.industry ,medicine.medical_treatment ,Immunology ,Cell Biology ,Hematology ,Hematopoietic stem cell transplantation ,medicine.disease ,Natural killer T cell ,Biochemistry ,Peripheral T-cell lymphoma ,Lymphoma ,Transplantation ,Radiation therapy ,hemic and lymphatic diseases ,Internal medicine ,medicine ,T-cell lymphoma ,Stem cell ,business - Abstract
Peripheral T-cell lymphomas (PTCL) other than anaplastic large cell lymphoma are rare in children, and data about outcome and treatment results especially regarding the role of stem cell transplantation (SCT) are scarce. Here we present the results of a retrospective study of SCT for pediatric patients with PTCL within the European Group of Bone marrow transplantation (EBMT). Out of 125 patients aged After SCT the 5-year overall survival (OS) and lymphoma-free survival (LFS) probability was 57% (95% CI 44.5-72.5) and 56% (95% CI 43.4-71.2), respectively. Relapse incidence was 25% (95% CI 14.1-37.9), whereas the non-relapse mortality (NRM) rate was 18% (95% CI 9.4-29.7). Patients with younger age (0-9 years, vs. 10-18 years) at diagnosis of PTCL had a tendency for better outcome (OS 80% vs. 50%, respectively p=0.069), whereas age at SCT and gender had no influence on outcome (p=0.2 and p=0.6, respectively). Year of SCT (1995-2007 vs. 2008-2015) was of no significance for the outcome (p=0.31). A tendency for better outcome (OS/LFS) was observed in patients who received RT in the pre-transplant treatment, and for patients who achieved first/second complete remission (CR1/CR2) before SCT, as compared to those with later/ without CR prior to SCT (OS 72% vs 46%, p=0.1). Patients receiving MAC conditioning had a better OS compared to patients receiving RIC (66% vs. 33%, p=0.07). LFS was inferior after ASCT compared to allogeneic SCT, resulting in non-significant difference in OS . Acute Graft-versus host disease (aGvHD) grade III/IV was seen in 6 (14.6%) patients. 6 patients suffered from chronic GvHD, 2 of them from the extensive form. In conclusion, SCT for pediatric patients with PTCL in CR1/CR2 is a valid option of treatment. MAC may currently be chosen for conditioning in order to possibly prevent relapses. Disclosures Bader: Neovii: Research Funding; Cellgene: Consultancy; Riemser: Research Funding; Novartis: Consultancy, Speakers Bureau; Medac: Patents & Royalties, Research Funding.
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- 2018
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30. Hematopoietic Cell Transplantation in Thalassemia and Sickle Cell Disease: Report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry: 2000-2017
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Donatella Baronciani, Ardeshir Ghavamzadeh, Gérard Socié, Peter Bader, Gian Luca Forni, O. Alphan Kupesiz, Arjan C. Lankester, Abdelghani Tbakhi, Josu de la Fuente, Marco Zecca, Amal Al-Seraihy, Javid Gaziev, Ariane Boumendil, Emanuele Angelucci, Vassiliki Kitra-Roussou, and Arnaud Dalissier
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Bone marrow transplantation ,Hematopoietic cell ,business.industry ,Thalassemia ,Immunology ,Cell ,Cell Biology ,Hematology ,Disease ,medicine.disease ,Biochemistry ,Transplantation ,03 medical and health sciences ,0302 clinical medicine ,medicine.anatomical_structure ,Hemoglobinopathy ,030220 oncology & carcinogenesis ,medicine ,business ,030215 immunology - Abstract
INTRODUCTION: Allogeneic hematopoietic cell transplantation (HCT) is a diffuse curative option for transfusion dependent thalassemia (TDT) and sickle cell disease (SCD). To verify transplant activity, distribution, demography, policies and outcomes the Hemoglobinopathy Registry was established inside the European Group for Blood and Marrow Transplantation (EBMT). After a previous analysis limited to TDT for the 2000-2010 period data (BMT 2016; 51:536-41), we performed an updated report considering TDT and SCD patients transplanted in the last eighteen years (years 2000-2017). METHODS: Data on pediatric patients transplanted between Jan 1st, 2000 through Dec 31st, 2017 were extracted by the EBMT promise Hemoglobinopathy registry database. Only first transplants were considered. Data are expressed as median with range unless specifically indicated. Survival probabilities were calculated with the method of Kaplan and Meier and expressed as means and 95% confidence intervals (95%CI). Differences between survival probabilities were tested by means of the log-rank test. RESULTS: In the above-specified period 3856 consecutive pediatric patients affected by TDT (2936, 76%) or SCD (920, 24%) were transplanted in 166 HCT centers distributed in 36 countries in Europe, Asia and Africa. Median age at transplant was 7.2 years (range 0.48-17.9). 3342 (87%) transplants were performed on patients Figure 1 reports pediatric transplant activity inside the EBMT showing an increased numbers of patients transplanted after year 2010. After a median follow up of 24 months, the 2 years overall survival (OS) and event-free survival (EFS) were 91% (95%CI 90-92) and 86% (95%CI 85-87) for the entire population. In TDT, OS and EFS were 90% (95%CI 89-92) and 84% (95%CI 82-85), respectively. In SCD, OS and EFS were 94% (95%CI 92-96) and 92% (95%CI 90-94), respectively. In both diseases no outcome difference was recorded on the basis of year of transplant (data not shown). Source of hematopoietic cells was bone marrow in 70% of TDT transplants and 81% of SCD transplants. In both diseases better results were recorded with the use of bone marrow versus peripheral blood [OS 91% versus 85% (P< 0.001); 95% versus 84% (P= 0.004) for TDT and SCD, respectively]. Similar results were recorded when EFS was analyzed. 176 patients received hematopoietic cells from single cord blood donors. Of them only 21 were from an unrelated donor. 119 patients received cord blood + bone marrow. Results of related HLA identical cord blood were similar to that of HLA identical related bone marrow (data not shown). Transplantation from an HLA identical sibling offered the best results in OS and EFS compared to other donor options (P< 0.001), both for TDT and SCD. Transplant results by donor type are reported in table 1. The threshold age for optimal transplant outcomes is confirmed 14 years, with OS 92% (95%CI 91-93), EFS 87% (95%CI 85-88) versus OS 85% (95%CI 82-89), EFS 81% (95%CI 77-85) ( P The two years incidence of cumulative extensive chronic graft versus host disease was 4.1% in TDT and 4.4 % in SCD (P=ns). CONCLUSIONS: Allogeneic HCT for TDT and SCD is a widely available curative approach; the procedure has been increasing and internationally performed during the years with excellent results. The emerging gene therapy approach will have to be compared to these well-established results. Disclosures Zecca: Chimerix: Honoraria. Forni:Novartis: Other: travel expenses, Research Funding; Celgene: Research Funding; Roche: Consultancy; Shire: Research Funding; Apopharma: Other: DSM Board. Bader:Medac: Patents & Royalties, Research Funding; Neovii: Research Funding; Novartis: Consultancy, Speakers Bureau; Riemser: Research Funding; Cellgene: Consultancy. Angelucci:Vertex Pharmaceuticals Incorporated (MA) and CRISPR CAS9 Therapeutics AG (CH): Other: Chair DMC; Novartis: Honoraria, Other: Chair Steering Comiittee TELESTO Protocol; Roche Italy: Other: Local (national) advisory board; Jazz Pharmaceuticals Italy: Other: Local ( national) advisory board; Celgene: Honoraria, Other: Chair DMC.
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- 2018
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31. Pregnancy Rates and Pregnancy Outcomes after Hematopoietic Stem Cell Transplantation in Childhood: a Cross-Sectional Survey of the EBMT Pediatric WP
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Alicia Rovó, Tamara Diesch, Peter Bader, Adriana Balduzzi, Arnaud Dalissier, Gabor Szinnai, and Fabienne Gumy-Pause
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Infertility ,Pediatrics ,medicine.medical_specialty ,education.field_of_study ,Pregnancy ,Cross-sectional study ,business.industry ,Incidence (epidemiology) ,Immunology ,Population ,Cell Biology ,Hematology ,medicine.disease ,Biochemistry ,Transplantation ,03 medical and health sciences ,Pregnancy rate ,0302 clinical medicine ,030220 oncology & carcinogenesis ,medicine ,Live birth ,education ,business ,030215 immunology - Abstract
Introduction: In the last decade transplant activity increased constantly and consequently the number of long-term survivors. Most applied conditioning regimes are known to impair normal puberty development and can lead to infertility. Pregnancies in women who had undergone a HSCT as well as in partners of transplanted men have been documented. However, the true incidence of pregnancies and its related complications in patients transplanted during their childhood remains unknown. The Pediatric WP of the EBMT conducted a study aiming to analyze pregnancy rates and to evaluate pregnancy outcomes in survivors of childhood HSCT. Material and Methods: In this study, we first evaluate the available data on the EBMT registry. To collect additional data a survey was conducted between July 2017- June 2018. 203 centers with pediatric activity reported to the EBMT patients were asked to fill-out the survey with 36 items related to treatment plan, pregnancies and pregnancies outcome. Adoptions were not considered in the study. Results: Data obtained from the EBMT database "Promise" reported that 62'988 pediatric patients were transplanted in the EBMT centers from 1995 to end of 2016. In 484 (0.76%) patients a conception after HSCT has been reported. 341/484 patients (70%) had an allograft, 134 of them (40%) were males. 143/484 had an autologous, 53 of them (37%) were male. The median age of patients was 15.02 years (range 0.6 - 17.9 years), was similar for male and females regardless of whether they were auto or allogeneic transplanted. 283 patients (58%) had conditioning regimes containing alkylating agents, irradiation or both. Data obtained from the conducted survey provide precise information of 27 (13 %) EBMT centers from 16 countries about 114 pregnancies of 99 patients and their partners. Pregnancies were in 30/99 (30%) from partners of post HSCT male patients and 60/99 (70%) were of post HSCT females. Underlying diseases for HSCT were reported in 82 of all 99 patients. 47/82 (57%) had a malignant disease and35/82 (43%) had a non-malignant disease. 29/99 (29%) had a reduced conditioning regime, 70/99 (71%) a myeloablative conditioning regime. 25/82 (30%) received TBI as part of the conditioning regimes. Regarding conception in patients post TBI, 12/23 (52%) became naturally pregnant (2/12 had 2 Gy only), 9/23(39%) required assisted fertilization and 2 used cryopreserved sperms. In patients conditioned without TBI, 54/59 (92%) had a natural conception, 4/59 (7%) required assisted fertilization and 1 used cryopreserved sperms. In 90/99 (90%) patients pregnancy resulted in a livebirth. 13 of the 90 livebirths (14%) were preterm, with a higher proportion than in normal the population (5.5-11%) and 6 of the 90 livebirths (6%) presented a low birthweight which is comparable to the normal population (6.5%). Conclusion: This study confirms a low pregnancy rate in survivors of childhood HSCT. Natural conception was possible when patients are conditioned without TBI. After TBI, pregnancy assisted fertilization techniques may increase pregnancy rate. The number of live births and complications indicate the need to advise survivors of childhood HSCT about the potential risks and outcome of pregnancy and advise them appropiately. Disclosures Bader: Cellgene: Consultancy; Neovii: Research Funding; Medac: Patents & Royalties, Research Funding; Riemser: Research Funding; Novartis: Consultancy, Speakers Bureau.
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- 2018
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32. Second Allogeneic Hematopoietic Stem Cell Transplantation for Post-Transplant Relapsed Acute Leukemia in Children - an EBMT PDWP Retrospective Study
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Miguel Angel Diaz, Peter Bader, Massimo Berger, Petr Sedlacek, Christina Peters, Selim Corbacioglu, Eric Beohou, Aviva C. Krauss, Stefania Varotto, Arnaud Dalissier, Isaac Yaniv, Krzysztof Kałwak, Marco Zecca, and Boris V. Afanasyev
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Oncology ,medicine.medical_specialty ,Acute leukemia ,business.industry ,medicine.medical_treatment ,Immunology ,Retrospective cohort study ,Cell Biology ,Hematology ,Hematopoietic stem cell transplantation ,medicine.disease ,Biochemistry ,Transplantation ,Leukemia ,Graft-versus-host disease ,Median follow-up ,hemic and lymphatic diseases ,Internal medicine ,Cohort ,medicine ,business - Abstract
Introduction Using the EBMT registry, we retrospectively analyzed outcomes for 373 pediatric patients who underwent second allogeneic transplant for relapsed acute leukemia at 120 centers in 32 countries, between the years 2004 and 2013, in an attempt to assess relapse, survival, GVHD and other outcomes, as well as identify factors correlating with prognosis in this cohort of patients. To our knowledge, this is the largest analysis of pediatric patients undergoing second allogeneic HSCT for relapsed acute leukemia to date. This allowed for an independent analysis of each disease, including 214 patients with ALL and 159 with AML. Patients and Methods Centers received a questionnaire completing data already available in the ProMISe database on patients between 0-18 years of age treated between 2004 and 2013. Results A total of 387 patients received a second SCT after relapse. 373 have been included in the analysis, 214 for ALL and 159 for AML. Detailed data were available for 201 patients from 48 centers; for the remainder, analysis was based on the registry. For the entire cohort overall survival (OS) at 2 and 5 years were 38% and 29%, and leukemia free survival (LFS) 30% and 25% respectively. ALL: With a median follow up from 2nd SCT of 36.4 months, OS at 1 and 5 years were 47% and 28% respectively. LFS was 39% and 28% respectively. NRM at 2 years was 22%. In multivariate analyses favorable prognostic factors for both OS and LFS were: CR prior to 2nd SCT (p=0.0001), interval > 12 months between transplants (p=0.0007), use of myeloablative conditioning (p=0.039) and the presence of cGvHD after the first SCT (p=0.0001). Good prognostic factor for low NRM was interval of more than 12 months between transplants (p=0.0002). AML: With a median follow up from 2nd SCT of 50 months, OS at 1 and 5 years were 44% and 15% respectively. LFS was 28% and 15% respectively. NRM at 2 years was 18%. In multivariate analyses, favorable prognostic factors for OS as well as LFS were: CR prior to 2nd SCT (p=0.031;0.044 respectively), interval > 6 months between transplants (p=0.0003;0.0001 respectively), and having cGvHD after the first SCT (p=0.0001). Most patients experience disease relapse or NRM within the first year after their second transplant. This observation seems to be more consistent in patients transplanted for ALL, with more changes over time in patients with AML. For ALL in particular, the 2-year incidences of relapse, NRM and LFS were not different from those at 5-years. Even in the relapse setting, survival rates for patients with ALL remain superior to patients with AML, consistent with the prognostic differences at diagnosis. Our findings, consistent for the AML and ALL subgroups, suggest that cGHVD prior to second HSCT is associated with better outcome. The identification of cGHVD prior to second transplant has not been heretofore described as a favorable prognostic factor. This strong correlation merits further study, specifically as to the underlying biology for this association. Conclusion Children with relapsed acute leukemias have a substantial chance to become long term survivors following a second SCT. CR prior to second SCT, longer interval between transplants and the presence of cGvHD after the first transplant, are favorable prognostic factors for ALL and AML. Our findings may help physicians in discussing the risk-benefit of a second transplant. These results are particularly relevant in an era where an explosion of new therapies, specifically targeted therapies and those that modulate the immune response, behoove us to carefully identify subpopulations of patients for whom specific therapies are appropriate. Novel approaches are needed to minimize relapse risk as well as short and long term morbidity in these pediatric patients while considering a second SCT for relapsed acute leukemia. Disclosures Corbacioglu: Jazz Pharmaceuticals: Consultancy, Honoraria. Bader: Novartis, Medac, Amgen, Riemser, Neovii: Consultancy, Honoraria, Research Funding.
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- 2017
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33. Myeloablative Conditioning for First Allogeneic Hematopoietic Stem Cell Transplantation in Children with ALL: Total Body Irradiation or Chemotherapy? - a Multicenter EBMT-PDWP Study
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Marianne Ifversen, Peter Bader, Christina Peters, Jacek Wachowiak, Marc Bierings, Gérard Michel, Boris V. Afanasyev, Myriam Labopin, Paul Veys, Rose-Marie Hamladji, Jochen Büchner, K. Nagy, Sabina Sufliarska, Marc Ansari, Thomas Klingebiel, Sophie Dupont, Arcangelo Prete, Jan Styczyński, Eric Beohou, Andre Willasch, E V Skorobogatova, Tayfun Guengoer, Alice Bertaina, Arjan C. Lankester, Damir Nemet, Ain Kaare, Petr Sedlacek, Mikael Sundin, Jelena Rascon, Stelios Graphakos, Amir Ali Hamidieh, Gergely Kriván, Jean-Hugues Dalle, Manuel Abecasis, Akif Yeşilipek, Reuven Or, Franca Fagioli, Cristina Díaz de Heredia, Franco Locatelli, Olga Aleinikova, and Arnaud Dalissier
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Oncology ,medicine.medical_specialty ,Univariate analysis ,Chemotherapy ,Cyclophosphamide ,business.industry ,medicine.medical_treatment ,Immunology ,Cell Biology ,Hematology ,Hematopoietic stem cell transplantation ,Total body irradiation ,Biochemistry ,Chemotherapy regimen ,Internal medicine ,medicine ,business ,Busulfan ,Etoposide ,medicine.drug - Abstract
Introduction: Most pediatric patients (pts) with ALL receive total body irradiation (TBI) for myeloablative conditioning of allogeneic hematopoietic stem cell transplantation (allo-HSCT). It is unproven whether TBI can be replaced by chemotherapy (CHT). Methods: To compare the outcomes of TBI- versus (vs.) CHT-based conditioning, we performed a retrospective EBMT-registry based study. Children between 2 and 18 years of age (y.) after myeloablative conditioning for first allo-HSCT of bone marrow (BM) or peripheral blood SC (PBSC) from matched sibling (MSD) or unrelated donors (UD) in first (CR1) or second remission (CR2) between 2000 and 2012 were included. Propensity score weighting was used to consider the covariate "distribution of TBI recipients" for pts who did not receive TBI. Results: In total 3071 pts (CR1: 1504 (49%), CR2: 1567 (51%)) were included. CR1: 1045 pts (69%) received BM and 459 pts (31%) PBSC from MSD (760 (51%)) or UD (744 (49%)). CR2: 1067 pts (68%) received BM and 500 pts (32%) PBSC from MSD (675 (43%)) or UD (892 (57%)). Overall, conditioning was TBI- in 2647 (86%) and CHT-based in 424 pts (14%). Busulfan/Cyclophosphamide (Bu/Cy) and Bu/Cy/Etoposide (Bu/Cy/Eto) were the two most frequently applied CHT combinations in CR1 (68 (32%), 66 (31%)) and CR2 (68 (32%), 52 (25%)). The remaining conditionings included 5 different combinations of chemotherapeutics (other chemo). 1504 pts in CR1 were conditioned with TBI (1291), Bu/Cy/Eto (66) or other chemo (147) with a median follow-up of 4.4, 3.4 and 2.4 y. In weighted univariate analysis no significant differences were detected for LFS (5-y.-LFS, range: 62.4 to 67.5%) and relapse incidence (5-y.-RI, range: 24.0 to 29.0%). In pairwise testing, OS after Bu/Cy/Eto was significantly better compared with TBI (5-y.-OS, 78.7 vs. 66.8%, P=.006). Non-relapse mortality was significantly higher after other chemo (5-y.-NRM, 12.7%, P Other significant influencing factors on LFS were age (5-y.-LFS, 2-11 y. 65.8 vs. 12-18 y. 58.0%, P=.009), y. of HSCT (2008-2012 65.9 vs. 2000-2007 59.6%, P=.035) and donor type (MSD 64.9 vs. UD 59.5%, P=.007). RI was influenced by y. of HSCT (5-y.-RI, 2008-2012 21.7 vs. 2000-2007 26.8%. P=.026). OS was influenced by age (5-y.-OS, 2-11 y. 72.7 vs. 12-18 y. 61.7%, P In weighted multivariable Cox model clustered on EBMT centers, TBI-based conditioning in CR1 was associated with lower RI (HR 0.70, P=.047), lower OS (HR 1.54, P=.017) and higher NRM (HR 3.97, P 1567 pts in CR2 were conditioned with TBI (1356), Bu/Cy (68) or other chemo (143) with a median follow-up of 3.9, 3.2 and 3.1 y. In weighted univariate analysis highly significant differences of survival were detected. TBI-based conditioning resulted in highest 5-y.-LFS of 52.2% (Bu/Cy 41.0%, other chemo 18.4%, P Other significant influencing factors on LFS were age (5-y.-LFS, 2-11 y. 53.5 vs. 12-18 y. 43.0%, P In weighted multivariable Cox model clustered on EBMT centers, TBI-based conditioning in CR2 was associated with higher LFS (HR 0.48, P Conclusion: Conditioning by TBI demonstrated clear superiority in comparison to CHT for children with ALL undergoing HSCT in CR2. For pts in CR1, TBI- and CHT-based conditioning showed similar results. This retrospective data is currently re-evaluated in a prospective, randomized, international trial (ALL SCTped 2012 FORUM). Disclosures Büchner: Novartis Pharmaceuticals Corporation: Consultancy; Pfizer: Consultancy. Veys: Bellicum: Research Funding; Servier: Research Funding. Bader: Novartis, Medac, Amgen, Riemser, Neovii: Consultancy, Honoraria, Research Funding.
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- 2017
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34. Fertility Preservation in Pediatric and Adolescent Patients Undergoing HSCT: a Cross-Sectional Survey of the EBMT Pediatric WP
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Maura Faraci, Alicia Rovó, Peter Bader, Tamara Diesch, Arnaud Dalissier, Nicolas von der Weid, Marta Pillon, and Jean-Hugues Dalle
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Infertility ,Pediatrics ,medicine.medical_specialty ,business.industry ,Cross-sectional study ,Immunology ,Cell Biology ,Hematology ,medicine.disease ,Biochemistry ,Chemotherapy regimen ,Transplantation ,Pelvic irradiation ,medicine ,Fertility preservation ,business ,Standard operating procedure - Abstract
Introduction: Fertility preservation is crucial in the setting of hematopoietic stem cell transplantation (HSCT) due to the severe impact of infertility on quality of life in long term survivors. Many chemotherapy agents as well as CNS or pelvic irradiation can affect gonads leading to impairment of pubertal development and/or infertility. In the last years different fertility preservation guidelines especially for cancer patients have been published. Despite these recommendations options for fertility preservation are not well established in children, it's discussed only in about 42% (Terenziani et al. PBC 2014) of all eligible patients. We present here a survey conducted by the Pediatric WP of the EBMT. Aim: to analyze and compare the different fertility preservation practices for children and adolescents across the EBMT centers and to identify hence the unmet needs in the field. Material and Methods: The survey was conducted between September 2014 and December 2015. 177 pediatric centers reporting to the EBMT were asked to fill-out a form with 24 questions related to fertility preservation strategies. Questions focused on institutional availability of a fertility preservation program and its options, number of patients included in the program, payers and suggestions for improvement. Results: 38 (21%) centers from 16 countries replied. In 2013 the number of HSCT per center ranges from 2 to 60. Totally, 834 children were transplanted in the 38 centers, 22% of the reported HSCT in children (3789) in 2013 from the EBMT centers. Of the 834 patients 612 (73%) were prepubertal and 203 (25%) postpubertal. 322/834 (39%) patients received a fertility preservation counselling and 243 (29%) of them had a fertility preservation procedure. Half of the 38 centers reported to have a fertility preservation program with standard operating procedure (SOP) for counselling. Centers with SOP counselled patients significantly more often than those without (Figure 1A). Regarding the number of applied procedures, no significant difference was found between centers with and without a SOP (Figure 1B). Centers with SOP counselled earlier (at the beginning of treatment/diagnosis; 61% versus 29%), and an interdisciplinary counselling team was largely used (60%). This team involved hematologists, gynecologists and /or reproductive medicine specialists. The most frequently cited reasons for missing counselling were lack of time (59%), refusal by parents (35%) and expected poor prognosis (29%). Reasons explaining parents/patients refusal were psychological distress (52%) and overwhelming feeling (50%) in this life-threatening moment. All responding centers considered the topic of fertility preservation as very relevant. Better education, more resources and financial funding emerged as pivotal factors to support and implement such programs. Conclusion: This EBMT survey showed the great interest for fertility preservation in participating centers. The collected data indicated that less than half of pediatric and adolescent patients undergoing HSCT in 2013 received a counselling on fertility preservation and/ or underwent fertility preservation procedures. This emphasizes the need for enhanced education of the transplant team and active multidisciplinary collaboration in the field. Fertility preservation becomes a standard of care; this data represents the basis to further efforts toward fertility preservation standardization in the EBMT community. Figure 1 Upper figures: Percentage of consulted patients in centers with and without SOP. Lower figures: Percentage of treated patients in centers with and without SOP Figure 1. Upper figures: Percentage of consulted patients in centers with and without SOP. Lower figures: Percentage of treated patients in centers with and without SOP Disclosures Bader: Servier: Consultancy, Honoraria; Neovii Biotech: Research Funding; Riemser: Research Funding; Medac: Consultancy, Research Funding; Novartis: Consultancy, Honoraria.
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- 2016
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35. Incidence and severity of crucial late effects after allogeneic HSCT for malignancy under the age of 3 years : TBI is what really matters
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Gérard Michel, C Peters, Miguel Angel Diaz, Patrick Lutz, Petr Sedlacek, Roderick Skinner, A. B. Versluys, Dorine Bresters, Peter J. Shaw, Anita Lawitschka, Maura Faraci, A. Jenkins, Brenda Gibson, Amal Al-Seraihy, Arnaud Dalissier, Nina Salooja, Chiara Cugno, Ulrike Pötschger, Kim Vettenranta, Alison D. Leiper, Mikael Sundin, Peter Bader, Tracey A. O'Brien, Brno University of Technology [Brno] (BUT), Institute of education, and Queen Mary University of London (QMUL)
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Male ,Pediatrics ,medicine.medical_specialty ,Time Factors ,Cross-sectional study ,[SHS.PSY]Humanities and Social Sciences/Psychology ,Graft vs Host Disease ,Malignancy ,03 medical and health sciences ,0302 clinical medicine ,Sex Factors ,Risk Factors ,medicine ,Journal Article ,Humans ,Transplantation, Homologous ,Cumulative incidence ,Registries ,Transplantation ,business.industry ,Incidence (epidemiology) ,Incidence ,Hematopoietic Stem Cell Transplantation ,Infant ,Hematology ,Total body irradiation ,medicine.disease ,[SHS.ECO]Humanities and Social Sciences/Economics and Finance ,3. Good health ,Graft-versus-host disease ,Cross-Sectional Studies ,030220 oncology & carcinogenesis ,[SDV.MHEP.PSM]Life Sciences [q-bio]/Human health and pathology/Psychiatrics and mental health ,Child, Preschool ,Hematologic Neoplasms ,Quality of Life ,[SDV.SPEE]Life Sciences [q-bio]/Santé publique et épidémiologie ,Female ,Thyroid function ,business ,Whole-Body Irradiation ,030215 immunology ,Follow-Up Studies - Abstract
Younger children are considered to be more vulnerable to late effects (LE), which prompted us to study LE in patients after haematopoietic stem cell transplantation (HSCT) for a haematological malignancy before the age of 3. In this multicentre EBMT study, cumulative incidence (CI) and severity of endocrine LE, central nervous system complications and secondary malignancies at 5, 10, 15 and 20 years of follow-up were assessed. Risk factors (RF) like gender, diagnosis, age at and year of HSCT, TBI- or chemo-conditioning and GVHD were analysed. CI of any LE was 0.30, 0.52, 0.66 and 0.72 at 5, 10, 15 and 20 years after HSCT, respectively. In 25% of the patients, LE were severe at a median follow-up of 10.4 years. In multivariate analysis, only TBI was a RF for having any LE and for thyroid dysfunction and growth disturbance. Female gender was a RF for delayed pubertal development. Some more insight could be gained by descriptive analysis regarding the role of TBI and GVHD on the severity of LE. Although only five selected LE have been studied and median follow-up is relatively short, the incidence and severity of these LE are considerable but not different from what has been found in older children and TBI is the main RF.Bone Marrow Transplantation advance online publication, 27 June 2016; doi:10.1038/bmt.2016.139.
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- 2016
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36. Myeloablative Chemo-Conditioning for First Hematopoietic STEM CELL Transplantation in Children with ACUTE Lymphoblastic Leukemia in First or Second Remission
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Petr Sedlacek, António Campos, Gergely Kriván, Jacek Wachowiak, Christina Peters, Franca Fagioli, Peter Bader, Marianne Ifversen, Arjan C. Lankester, Jochen Büchner, M. Akif Yesilipek, Arnaud Dalissier, Andrea Pession, Stelios Graphakos, Amir Ali Hamidieh, Jean-Hugues Dalle, and Myriam Labopin
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Melphalan ,medicine.medical_specialty ,business.industry ,medicine.medical_treatment ,Immunology ,Cell Biology ,Hematology ,Hematopoietic stem cell transplantation ,ThioTEPA ,Total body irradiation ,Biochemistry ,Gastroenterology ,Fludarabine ,Transplantation ,Internal medicine ,medicine ,business ,Etoposide ,Busulfan ,medicine.drug - Abstract
Christina Peters, Petr Sedlacek, Jean Hugues Dalle, Stelios Graphakos, Antonio Campos, Akif Yesilipek, Jacek Wachowiak, Arjan Lankester, Andrea Pession, Amir Ali Hamidieh, Marianne Ifversen, Jochen Büchner, Gergely Krivan, Franca Fagioli, Arnaud Dalissier; Myriam Labopin; Peter Bader on behalf of the EBMT Pediatric Diseases Working Party Most children with acute lymphoblastic leukemia (ALL) with indication for allogeneic hematopoietic stem cell transplantation (HSCT) receive myeloablative conditioning with a total body irradiation (TBI)-containing regimen. To investigate the outcomes of patients (pts) who did not undergo TBI, we performed a retrospective registry based study on children below 18 years who received a myeloablative chemo-conditioning for a first allogeneic HSCT from different donors between 2000 and 2012. In this analysis, only chemotherapeutic regimens with more than 30 applications were included. In total, 732 pts were included: 313 pts who received bone marrow (BM) or peripheral blood stem cells (PBSC) in 1st CR, 247 pts with BM/PBSC transplantation in CR2, 85 pts and 52 pts who received umbilical cord blood (CB) in 1st or 2nd CR, respectively. The most commonly applied myeloablative chemo-combinations were: Busulfan (Bu)/Cyclophosphamide (Cy) (n=202), Bu/Cy/Etoposide (VP) (n=189), Bu/Cy/Melphalan (Mel) (n=93), Bu/AraC/Mel (n=80), Bu/Fludarabine (Flu)/Thiotepa (Thio) (n=62), Bu/Cy/Thio (n=53, Bu/Cy/Thio (n=53), and Bu/Flu (n=53). 313 pts received either BM or PBSC in CR1 with a median follow up of 26 months (1-156) and we compared Bu/Cy/VP vs the other chemo-conditioning regimens. The Bu/Cy/VP cohort had a longer follow up (med 37 vs. 20 months, p=0.002), pts were younger (med 3,6 vs. 6,5 years, p=0.003) and the median year of transplant was earlier (med 2009 vs. 2010, p=0.03). Donor type, CMV match, gender match, stem cell were comparable. In univariate analysis, conditioning with Bu/Cy/VP was better than all other combinations: relapse incidence (RI) 21% vs 32% (p=0.05), leukemia-free survival (LFS) 72 vs 54% (p=0.004), overall survival (OS) 79 vs 68% (p=0.03) and chronic GVHD (cGVHD) 9% vs 19% (p=0.014). Engraftment and incidence and severity of acute GVHD were similar and non- relapse mortality (NRM) was 7% vs 13% (p=0.10). Other significant influencing factors were: interval between diagnosis and transplantation below or beyond 208 days (NRM 6% vs 16%, p=0.015), donor sibling vs other (RI 35% vs 23%, p=0.01, NRM 5% vs 16%, p=0.001) and in vivo T cell depletion (TCD) vs no TCD (RI 35% vs. 19%, p=0.003; NRM 20% vs 4%, p=0.0001). In the cox model, conditioning type (Bu/CY/VP vs other), age, year of transplantation, interval from diagnosis to transplant, donor type, stem cell source and in vivo TCD were evaluated. For LFS only BU/CY/VP was associated with better outcome (p=0.004, HR .52), RI was lower after Bu/Cy/VP (HR .54, p=0.02), NRM was higher in pts older than 4,6 years (p=0.02, HR 2,48) and after TCD HSCT (p=0.01, HR 9,13) and OS was best after Bu/Cy/VP (p=0.03, HR 0.57). We conclude that omission of TBI is feasible for children who undergo first allogeneic HSCT in first or second complete remission. The combination of busulfan, cyclophosphamide and etoposide resulted in better LFS and OS with less NRM and RI for children who received bone marrow or peripheral blood stem cells in CR1. These observations should be the basis for prospective trials in homogenous patient groups. Disclosures No relevant conflicts of interest to declare.
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- 2014
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37. Myeloablative conditioning for first allogeneic HSCT in pediatric all: FTBI or chemotherapy? - An update of the retrospective multicenter EBMT-PDWP study
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Andre Manfred Willasch, Christina Peters, Petr Sedlacek, Jean-Hughes Dalle, Vassiliki Kitra-Roussou, Akif Yesilipek, Jacek Wachowiak, Arjan Lankester, Arcangelo Prete, Amir Ali Hamidieh, Marianne Ifversen, Jochen Buechner, Gergely Krivan, Rose-Marie Hamladji, Cristina Diaz de Heredia, Elena Skorobogatova, Gerard Michel, Franco Locatelli, Alice Bertaina, Paul Veys, Sophie Dupont, Reuven Or, Tayfun Gungor, Olga Aleinikova, Sabina Sufliarska, Mikael Sundin, Jelena Rascon, Ain Kaare, Damir Nemet, Franca Fagioli, Thomas Erich Klingebiel, Jan Styczyński, Marc Bierings, Thomas Nagy, Manuel Abecasis, Boris Afanasyev, Marc Ansari, Kim Venntenranta, Amal AlSeraihy, Alicja Chybicka, Stephen Robinson, Yves Bertrand, Alphan Kupesiz, Ardeshir Ghavamzadeh, Antonio Campos, Arnaud Dalissier, Myriam Labopin, Selim Corbacioglu, Jaques Emmanuel Galimard, and Peter Bader
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