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1. Allogeneic Hematopoietic Stem Cell Transplantation in Immunodeficiency—Centromeric Instability—Facial Dysmorphism (ICF) Syndrome: an EBMT/ESID Inborn Errors Working Party Study

Author

Berghuis, Dagmar, Mehyar, Lubna S., Abu-Arja, Rolla, Albert, Michael H., Barnum, Jessie L., von Bernuth, Horst, Elfeky, Reem, Lewalle, Philippe, Laberko, Alexandra, Ghosh, Sujal, Slatter, Mary A., Weemaes, Corry M. R., Yesilipek, Akif, Sirait, Tiarlan, Neven, Bénédicte, Gennery, Andrew R., and Lankester, Arjan C.

Published
2024
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2. Blinatumomab as a Curative Therapy Option for Relapsed/Refractory Infant Acute Lymphoblastic Leukemia Post-Hematopoietic Stem Cell Transplantation - Case Report

Author

Suleimen Zhumatayev, Koray Yalcin, Safiye Suna Celen, Vedat Uygun, Gulsun Karasu, and Mehmet Akif Yesilipek

Subjects
blinatumomab, infant all, hematopoietic stem cell transplantation, Pediatrics, RJ1-570, Internal medicine, RC31-1245, Neoplasms. Tumors. Oncology. Including cancer and carcinogens, RC254-282
Abstract

Acute lymphoblastic leukemia (ALL) is the most common type of cancer in children, with a particularly poor prognosis in infants under one year of age. Hematopoietic stem cell transplantation (HSCT) is an effective therapy for relapsed or refractory ALL; however, relapse after HSCT remains a significant challenge. Many children cannot undergo HSCT because of serious adverse events from previous treatment. In this case report, we present the case of an infant with relapsed/refractory ALL who received blinatumomab as salvage therapy after a second haploidentical HSCT and remained in remission for 15 months without subsequent HSCT. The patient was a 4-month-old male diagnosed with high-risk infant B-cell ALL with KMT2A/AFF1. He received induction chemotherapy according to the INTERFANT-06 protocol and achieved complete remission. He underwent 10/10 matched-sibling bone marrow transplantation but experienced an isolated marrow relapse 2 months post-transplant and then received a second haploidentical HSCT. He was treated with one cycle of blinatumomab after the relapse that occurred after the second HSCT. Due to toxicity, the patient did not receive a third transplant but was followed up after blinatumomab. And the patient remained in complete remission for 15 months after the blinatumomab therapy. Blinatumomab has been known as a bridging therapy. We suggest that blinatumomab could be a promising curative therapy option for patients who cannot receive further HSCT.

Published
2024
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3. Outcomes of hematopoietic stem cell transplantation in 813 pediatric patients with Fanconi anemia

Author

Lum, Su Han, Eikema, Dirk-Jan, Piepenbroek, Brian, Wynn, Robert F., Samarasinghe, Sujith, Dalissier, Arnaud, Kalwak, Krysztof, Ayas, Mouhab, Hamladji, Rose-Marie, Yesilipek, Akif, Dalle, Jean-Hugues, Uckan-Cetinkaya, Duygu, Bierings, Marc, Kupesiz, Alphan, Halahleh, Khalid, Skorobogatova, Elena, Öztürk, Gülyüz, Faraci, Maura, Renard, Cecile, Evans, Pamela, Corbacioglu, Selim, Locatelli, Franco, Dufour, Carlo, Risitano, Antonio, and Peffault de Latour, Régis

Published
2024
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4. Low rate of nonrelapse mortality in under-4-year-olds with ALL given chemotherapeutic conditioning for HSCT: a phase 3 FORUM study

Author

Bader, Peter, Pötschger, Ulrike, Dalle, Jean-Hugues, Moser, Laura M., Balduzzi, Adriana, Ansari, Marc, Buechner, Jochen, Güngör, Tayfun, Ifversen, Marianne, Krivan, Gergely, Pichler, Herbert, Renard, Marleen, Staciuk, Raquel, Sedlacek, Petr, Stein, Jerry, Heusel, Jan Robert, Truong, Tony, Wachowiak, Jacek, Yesilipek, Akif, Locatelli, Franco, and Peters, Christina

Published
2024
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5. Influence of invasive aspergillosis during acute leukaemia treatment on survival after allogeneic stem cell transplantation: a prospective study of the EBMT Infectious Diseases Working Party

Author

Penack, Olaf, Tridello, Gloria, Salmenniemi, Urpu, Martino, Rodrigo, Khanna, Nina, Perruccio, Katia, Fagioli, Franca, Richert-Przygonska, Monika, Labussière-Wallet, Hélène, Maertens, Johan, Jubert, Charlotte, Aljurf, Mahmoud, Pichler, Herbert, Kriván, Gergely, Kunadt, Desiree, Popova, Marina, Gabriel, Melissa, Calore, Elisabetta, Blau, Igor Wolfgang, Benedetti, Fabio, Itäla-Remes, Maija, de Kort, Elizabeth, Russo, Domenico, Faraci, Maura, Ménard, Anne-Lise, Borne, Peter von dem, Poiré, Xavier, Yesilipek, Akif, Gozdzik, Jolanta, Yeğin, Zeynep Arzu, Yañez, Lucrecia, Facchini, Luca, Van Gorkom, Gwendolyn, Thurner, Lorenz, Kocak, Ulker, Sampol, Antònia, Zuckerman, Tsila, Bierings, Marc, Mielke, Stephan, Ciceri, Fabio, Wendel, Lotus, Knelange, Nina, Mikulska, Malgorzata, Averbuch, Dina, Styczynski, Jan, Camara, Rafael de la, and Cesaro, Simone

Published
2024
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6. Influence of invasive aspergillosis during acute leukaemia treatment on survival after allogeneic stem cell transplantation: a prospective study of the EBMT Infectious Diseases Working PartyResearch in context

Author

Olaf Penack, Gloria Tridello, Urpu Salmenniemi, Rodrigo Martino, Nina Khanna, Katia Perruccio, Franca Fagioli, Monika Richert-Przygonska, Hélène Labussière-Wallet, Johan Maertens, Charlotte Jubert, Mahmoud Aljurf, Herbert Pichler, Gergely Kriván, Desiree Kunadt, Marina Popova, Melissa Gabriel, Elisabetta Calore, Igor Wolfgang Blau, Fabio Benedetti, Maija Itäla-Remes, Elizabeth de Kort, Domenico Russo, Maura Faraci, Anne-Lise Ménard, Peter von dem Borne, Xavier Poiré, Akif Yesilipek, Jolanta Gozdzik, Zeynep Arzu Yeğin, Lucrecia Yañez, Luca Facchini, Gwendolyn Van Gorkom, Lorenz Thurner, Ulker Kocak, Antònia Sampol, Tsila Zuckerman, Marc Bierings, Stephan Mielke, Fabio Ciceri, Lotus Wendel, Nina Knelange, Malgorzata Mikulska, Dina Averbuch, Jan Styczynski, Rafael de la Camara, and Simone Cesaro

Subjects
Invasive, Aspergillosis, Stem cell transplantation, Mortality, Medicine (General), R5-920
Abstract

Summary: Background: Infections are the main reason for mortality during acute leukaemia treatment and invasive aspergillosis (IA) is a major concern. Allogeneic stem cell transplantation (alloSCT) is a standard therapy and often is the only live-saving procedure in leukaemia patients. The profound immunodeficiency occurring after alloSCT led to high IA-associated mortality in the past. Therefore, patients with IA were historically considered transplant-ineligible. Recently, there has been improvement of anti-fungal management including novel anti-fungal agents. As a result, more leukaemia patients with IA are undergoing alloSCT. Outcome has not been prospectively assessed. Methods: We performed a prospective study in acute leukaemia patients undergoing alloSCT to analyse the impact of a prior history of probable or proven IA (pre-SCT IA). The primary endpoint was 1-year non-relapse mortality (NRM). Relapse free survival and overall survival were analysed as secondary endpoints. Findings: 1439 patients were included between 2016 and 2021. The incidence of probable or proven pre-SCT IA was 6.0% (n = 87). The cumulative incidence of 1-year NRM was 17.3% (95% CI 10.2–26.0) and 11.2% (9.6–13.0) for patients with and without pre-SCT IA. In multivariate analyses the hazard ratio (HR) for 1-year NRM was 2.1 (1.2–3.6; p = 0.009) for patients with pre-SCT IA. One-year relapse-free survival was inferior in patients with pre-SCT IA (59.4% [48.3–68.9] vs. 70.4 [67.9–72.8]; multivariate HR 1.5 [1.1–2.1]; p = 0.02). Consequently, 1-year overall survival was lower in patients with pre-SCT IA: (68.8% [57.8–77.4] vs. 79.0% [76.7–81.1]; multivariate HR 1.7 [1.1–2.5]; p = 0.01). Interpretation: Pre-SCT IA remains to be significantly associated with impaired alloSCT outcome. On the other hand, more than two thirds of patients with pre-SCT IA were alive at one year after alloSCT. IA is not anymore an absolute contraindication for alloSCT because the majority of patients with IA who undergo alloSCT benefit from this procedure. Funding: There was no external funding source for this study.

Published
2024
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7. Artemis deficiency: A large cohort including a novel variant with increased radiosensitivity

Author

Meric, Zeynep, primary, Gemici Karaaslan, Betul, additional, Yalcin Gungoren, Ezgi, additional, Bektas Hortoglu, Melika, additional, Cavas, Tolga, additional, Aydemir, Sezin, additional, Bilgic Eltan, Sevgi, additional, Firtina, Sinem, additional, Kendir Demirkol, Yasemin, additional, Eser, Metin, additional, Cekic, Sukru, additional, Kilic, Suar, additional, Karasu, Gulsun, additional, Yesilipek, Mehmet Akif, additional, Eke Gungor, Hatice, additional, Karakoc‐Aydiner, Elif, additional, Ozen, Ahmet, additional, Baris, Safa, additional, Yucel, Esra, additional, Cokugras, Haluk, additional, and Kiykim, Ayca, additional

Published
2024
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9. Thalassemia-free and graft-versus-host-free survival: outcomes of hematopoietic stem cell transplantation for thalassemia major, Turkish experience

Author

Yesilipek, M. Akif, Uygun, Vedat, Kupesiz, Alphan, Karasu, Gulsun, Ozturk, Gulyuz, Ertem, Mehmet, Şaşmaz, İlgen, Daloğlu, Hayriye, Güler, Elif, Hazar, Volkan, Fisgin, Tunç, Sezgin, Gülay, Kansoy, Savaş, Kuşkonmaz, Barış, Akıncı, Burcu, Özbek, Namık, İnce, Elif Ünal, Öztürkmen, Seda, Küpesiz, Funda Tayfun, Yalçın, Koray, Anak, Sema, Bozkurt, Ceyhun, Karakükçü, Musa, Küpeli, Serhan, Albayrak, Davut, Öniz, Haldun, Aksoylar, Serap, Okur, Fatma Visal, Albayrak, Canan, Yenigürbüz, Fatma Demir, Bozkaya, İkbal Ok, İleri, Talia, Gürsel, Orhan, Karagün, Barbaros Şahin, Kintrup, Gülen Tüysüz, Çelen, Suna, Elli, Murat, Aksoy, Basak Adaklı, Yılmaz, Ebru, Tanyeli, Atila, Akyol, Şule Turan, Siviş, Zuhal Önder, Özek, Gülcihan, Uçkan, Duygu, Kartal, İbrahim, Atay, Didem, Akyay, Arzu, Bilir, Özlem Arman, Çakmaklı, Hasan Fatih, Kürekçi, Emin, Malbora, Barış, Akbayram, Sinan, Demir, Hacı Ahmet, Kılıç, Suar Çakı, Güneş, Adalet Meral, Zengin, Emine, Özmen, Salih, and Antmen, Ali Bülent

Published
2022
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10. Low Density Granulocytes and Dysregulated Neutrophils Driving Autoinflammatory Manifestations in NEMO Deficiency

Author

Surucu Yilmaz, Naz, Bilgic Eltan, Sevgi, Kayaoglu, Basak, Geckin, Busranur, Heredia, Raul Jimenez, Sefer, Asena Pinar, Kiykim, Ayca, Nain, Ercan, Kasap, Nurhan, Dogru, Omer, Yucelten, Ayse Deniz, Cinel, Leyla, Karasu, Gulsun, Yesilipek, Akif, Sozeri, Betul, Kaya, Goksu Gokberk, Yilmaz, Ismail Cem, Baydemir, Ilayda, Aydin, Yagmur, Cansen Kahraman, Deniz, Haimel, Matthias, Boztug, Kaan, Karakoc-Aydiner, Elif, Gursel, Ihsan, Ozen, Ahmet, Baris, Safa, and Gursel, Mayda

Published
2022
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11. Busulfan–fludarabine- or treosulfan–fludarabine-based myeloablative conditioning for children with thalassemia major

Author

Lüftinger, Roswitha, Zubarovskaya, Natalia, Galimard, Jacques-Emmanuel, Cseh, Annamaria, Salzer, Elisabeth, Locatelli, Franco, Algeri, Mattia, Yesilipek, Akif, de la Fuente, Josu, Isgrò, Antonella, Alseraihy, Amal, Angelucci, Emanuele, Smiers, Frans J., La La Nasa, Giorgia, Zecca, Marco, Fisgin, Tunc, Unal, Emel, Kleinschmidt, Katharina, Peters, Christina, Lankester, Arjan, and Corbacioglu, Selim

Published
2022
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13. Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study.

Author

Barzaghi, Federica, Amaya Hernandez, Laura Cristina, Neven, Benedicte, Ricci, Silvia, Kucuk, Zeynep Yesim, Bleesing, Jack J, Nademi, Zohreh, Slatter, Mary Anne, Ulloa, Erlinda Rose, Shcherbina, Anna, Roppelt, Anna, Worth, Austen, Silva, Juliana, Aiuti, Alessandro, Murguia-Favela, Luis, Speckmann, Carsten, Carneiro-Sampaio, Magda, Fernandes, Juliana Folloni, Baris, Safa, Ozen, Ahmet, Karakoc-Aydiner, Elif, Kiykim, Ayca, Schulz, Ansgar, Steinmann, Sandra, Notarangelo, Lucia Dora, Gambineri, Eleonora, Lionetti, Paolo, Shearer, William Thomas, Forbes, Lisa R, Martinez, Caridad, Moshous, Despina, Blanche, Stephane, Fisher, Alain, Ruemmele, Frank M, Tissandier, Come, Ouachee-Chardin, Marie, Rieux-Laucat, Frédéric, Cavazzana, Marina, Qasim, Waseem, Lucarelli, Barbarella, Albert, Michael H, Kobayashi, Ichiro, Alonso, Laura, Diaz De Heredia, Cristina, Kanegane, Hirokazu, Lawitschka, Anita, Seo, Jong Jin, Gonzalez-Vicent, Marta, Diaz, Miguel Angel, Goyal, Rakesh Kumar, Sauer, Martin G, Yesilipek, Akif, Kim, Minsoo, Yilmaz-Demirdag, Yesim, Bhatia, Monica, Khlevner, Julie, Richmond Padilla, Erick J, Martino, Silvana, Montin, Davide, Neth, Olaf, Molinos-Quintana, Agueda, Valverde-Fernandez, Justo, Broides, Arnon, Pinsk, Vered, Ballauf, Antje, Haerynck, Filomeen, Bordon, Victoria, Dhooge, Catharina, Garcia-Lloret, Maria Laura, Bredius, Robbert G, Kałwak, Krzysztof, Haddad, Elie, Seidel, Markus Gerhard, Duckers, Gregor, Pai, Sung-Yun, Dvorak, Christopher C, Ehl, Stephan, Locatelli, Franco, Goldman, Frederick, Gennery, Andrew Richard, Cowan, Mort J, Roncarolo, Maria-Grazia, Bacchetta, Rosa, and Primary Immune Deficiency Treatment Consortium (PIDTC) and the Inborn Errors Working Party (IEWP) of the European Society for Blood and Marrow Transplantation (EBMT)

Subjects
Primary Immune Deficiency Treatment Consortium (PIDTC) and the Inborn Errors Working Party (IEWP) of the European Society for Blood and Marrow Transplantation, Humans, Genetic Diseases, X-Linked, Diabetes Mellitus, Type 1, Immune System Diseases, Diarrhea, Disease-Free Survival, Hematopoietic Stem Cell Transplantation, Survival Rate, Retrospective Studies, Follow-Up Studies, Mutation, Adolescent, Adult, Child, Child, Preschool, Infant, Female, Male, Forkhead Transcription Factors, Allografts, Immunosuppression Therapy, FOXP3, IPEX, Treg cells, enteropathy, genetic autoimmunity, hematopoietic stem cell transplantation, immunosuppression, neonatal diabetes, primary immune deficiency, rapamycin, Clinical Research, Stem Cell Research, Regenerative Medicine, Pediatric, Genetics, Transplantation, Aetiology, 2.1 Biological and endogenous factors, Immunology, Allergy
Abstract

BackgroundImmunodysregulation polyendocrinopathy enteropathy x-linked (IPEX) syndrome is a monogenic autoimmune disease caused by FOXP3 mutations. Because it is a rare disease, the natural history and response to treatments, including allogeneic hematopoietic stem cell transplantation (HSCT) and immunosuppression (IS), have not been thoroughly examined.ObjectiveThis analysis sought to evaluate disease onset, progression, and long-term outcome of the 2 main treatments in long-term IPEX survivors.MethodsClinical histories of 96 patients with a genetically proven IPEX syndrome were collected from 38 institutions worldwide and retrospectively analyzed. To investigate possible factors suitable to predict the outcome, an organ involvement (OI) scoring system was developed.ResultsWe confirm neonatal onset with enteropathy, type 1 diabetes, and eczema. In addition, we found less common manifestations in delayed onset patients or during disease evolution. There is no correlation between the site of mutation and the disease course or outcome, and the same genotype can present with variable phenotypes. HSCT patients (n = 58) had a median follow-up of 2.7 years (range, 1 week-15 years). Patients receiving chronic IS (n = 34) had a median follow-up of 4 years (range, 2 months-25 years). The overall survival after HSCT was 73.2% (95% CI, 59.4-83.0) and after IS was 65.1% (95% CI, 62.8-95.8). The pretreatment OI score was the only significant predictor of overall survival after transplant (P = .035) but not under IS.ConclusionsPatients receiving chronic IS were hampered by disease recurrence or complications, impacting long-term disease-free survival. When performed in patients with a low OI score, HSCT resulted in disease resolution with better quality of life, independent of age, donor source, or conditioning regimen.

Published
2018

16. Allogeneic hematopoietic stem cell transplantation in leukocyte adhesion deficiency type I and III

Author

Bakhtiar, Shahrzad, Salzmann-Manrique, Emilia, Blok, Henric-Jan, Eikema, Dirk-Jan, Hazelaar, Sheree, Ayas, Mouhab, Toren, Amos, Goldstein, Gal, Moshous, Despina, Locatelli, Franco, Merli, Pietro, Michel, Gerard, Öztürk, Gülyüz, Schulz, Ansgar, Heilmann, Carsten, Ifversen, Marianne, Wynn, Rob F., Aleinikova, Olga, Bertrand, Yves, Tbakhi, Abdelghani, Veys, Paul, Karakukcu, Musa, Kupesiz, Alphan, Ghavamzadeh, Ardeshir, Handgretinger, Rupert, Unal, Emel, Perez-Martinez, Antonio, Gokce, Muge, Porta, Fulvio, Aksu, Tekin, Karasu, Gülsün, Badell, Isabel, Ljungman, Per, Skorobogatova, Elena, Yesilipek, Akif, Zuckerman, Tsila, Bredius, Robbert R.G., Stepensky, Polina, Shadur, Bella, Slatter, Mary, Gennery, Andrew R., Albert, Michael H., Bader, Peter, and Lankester, Arjan

Published
2021
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17. Low rate of nonrelapse mortality in under-4-year-olds with ALL given chemotherapeutic conditioning for HSCT: a phase 3 FORUM study

Author

Bader, Peter; https://orcid.org/0000-0003-4554-0265, Pötschger, Ulrike, Dalle, Jean-Hugues; https://orcid.org/0000-0002-8406-0793, Moser, Laura M, Balduzzi, Adriana; https://orcid.org/0000-0002-5879-0610, Ansari, Marc; https://orcid.org/0000-0002-9649-6498, Buechner, Jochen; https://orcid.org/0000-0001-5848-4501, Güngör, Tayfun; https://orcid.org/0000-0002-3261-1186, Ifversen, Marianne, Krivan, Gergely, Pichler, Herbert; https://orcid.org/0000-0001-5107-5123, Renard, Marleen, Staciuk, Raquel, Sedlacek, Petr; https://orcid.org/0000-0001-7895-2886, Stein, Jerry, Heusel, Jan Robert; https://orcid.org/0000-0002-5753-0817, Truong, Tony, Wachowiak, Jacek, Yesilipek, Akif, Locatelli, Franco; https://orcid.org/0000-0002-7976-3654, Peters, Christina; https://orcid.org/0000-0003-0369-8515, Bader, Peter; https://orcid.org/0000-0003-4554-0265, Pötschger, Ulrike, Dalle, Jean-Hugues; https://orcid.org/0000-0002-8406-0793, Moser, Laura M, Balduzzi, Adriana; https://orcid.org/0000-0002-5879-0610, Ansari, Marc; https://orcid.org/0000-0002-9649-6498, Buechner, Jochen; https://orcid.org/0000-0001-5848-4501, Güngör, Tayfun; https://orcid.org/0000-0002-3261-1186, Ifversen, Marianne, Krivan, Gergely, Pichler, Herbert; https://orcid.org/0000-0001-5107-5123, Renard, Marleen, Staciuk, Raquel, Sedlacek, Petr; https://orcid.org/0000-0001-7895-2886, Stein, Jerry, Heusel, Jan Robert; https://orcid.org/0000-0002-5753-0817, Truong, Tony, Wachowiak, Jacek, Yesilipek, Akif, Locatelli, Franco; https://orcid.org/0000-0002-7976-3654, and Peters, Christina; https://orcid.org/0000-0003-0369-8515

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) is highly effective for treating pediatric high-risk or relapsed acute lymphoblastic leukemia (ALL). For young children, total body irradiation (TBI) is associated with severe late sequelae. In the FORUM study (NCT01949129), we assessed safety, event-free survival (EFS), and overall survival (OS) of 2 TBI-free conditioning regimens in children aged <4 years with ALL. Patients received fludarabine (Flu), thiotepa (Thio), and either busulfan (Bu) or treosulfan (Treo) before HSCT. From 2013 to 2021, 191 children received transplantation and were observed for ≥6 months (median follow-up: 3 years). The 3-year OS was 0.63 (95% confidence interval [95% CI], 0.52-0.72) and 0.76 (95% CI, 0.64-0.84) for Flu/Thio/Bu and Flu/Thio/Treo (P = .075), respectively. Three-year EFS was 0.52 (95% CI, 0.41-0.61) and 0.51 (95% CI, 0.39-0.62), respectively (P = .794). Cumulative incidence of nonrelapse mortality (NRM) and relapse at 3 years were 0.06 (95% CI, 0.02-0.12) vs 0.03 (95% CI: <0.01-0.09) (P = .406) and 0.42 (95% CI, 0.31-0.52) vs 0.45 (95% CI, 0.34-0.56) (P = .920), respectively. Grade >1 acute graft-versus-host disease (GVHD) occurred in 29% of patients receiving Flu/Thio/Bu and 17% of those receiving Flu/Thio/Treo (P = .049), whereas grade 3/4 occurred in 10% and 9%, respectively (P = .813). The 3-year incidence of chronic GVHD was 0.07 (95% CI, 0.03-0.13) vs 0.05 (95% CI, 0.02-0.11), respectively (P = .518). In conclusion, both chemotherapeutic conditioning regimens were well tolerated and NRM was low. However, relapse was the major cause of treatment failure. This trial was registered at www.clinicaltrials.gov as #NCT01949129.

Published
2024

18. The role of haematopoietic stem cell transplantation for sickle cell disease in the era of targeted disease-modifying therapies and gene editing

Author

Amrolia, Persis, Ansari, Marc, Balduzzi, Adriana, Dalassier, Arnaud, Dalle, Jean-Hugues, Hereda Diaz, Cristina, Feuchtinger, Tobias, Locatelli, Franco, Lucchini, Giovanna, Galimard, Jaques-Emmanuel, Gonzalez Vincent, Marta, Handgretinger, Rupert, Kleinschmidt, Katharina, Lawitschka, Anita, Perez Martinez, Antonio, Peters, Christina, Rocha, Vanderson, Ruggeri, Annalisa, Sedlacek, Petr, Svec, Peter, Toporski, Jacek, Yesilipek, Akif, de la Fuente, Josu, Gluckman, Eliane, Makani, Julie, Telfer, Paul, Faulkner, Lawrence, and Corbacioglu, Selim

Published
2020
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19. The impact of donor type on the outcome of pediatric patients with very high risk acute lymphoblastic leukemia. A study of the ALL SCT 2003 BFM-SG and 2007-BFM-International SG

Author

Dalle, Jean-Hugues, Balduzzi, Adriana, Bader, Peter, Pieczonka, Anna, Yaniv, Isaac, Lankester, Arjan, Bierings, Marc, Yesilipek, Akif, Sedlacek, Petr, Ifversen, Marianne, Svec, Peter, Toporski, Jacek, Gungor, Taifun, Wachowiak, Jacek, Glogova, Evgenia, Poetschger, Ulrike, and Peters, Christina

Published
2021
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23. 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)

Author

Nava, Tiago, Ansari, Marc, Dalle, Jean-Hugues, de Heredia, Christina Diaz, Güngör, Tayfun, Trigoso, Eugenia, Falkenberg, Ulrike, Bertaina, Alice, Gibson, Brenda, Jarisch, Andrea, Balduzzi, Adriana, Boenig, Halvard, Krivan, Gergely, Vettenranta, Kim, Matic, Toni, Buechner, Jochen, Kalwak, Krzysztof, Lawitschka, Anita, Yesilipek, Akif, Lucchini, Giovanna, Peters, Christina, Turkiewicz, Dominik, Niinimäki, Riitta, Diesch, Tamara, Lehrnbecher, Thomas, Sedlacek, Petr, Hutt, Daphna, Dalissier, Arnaud, Wachowiak, Jacek, Yaniv, Isaac, Stein, Jerry, Yalçin, Koray, Sisinni, Luisa, Deiana, Marco, Ifversen, Marianne, Kuhlen, Michaela, Meisel, Roland, Bakhtiar, Shahrzad, Cesaro, Simone, Willasch, Andre, Corbacioglu, Selim, and Bader, Peter

Published
2020
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24. Transplantation in Children and Adolescents with Acute Lymphoblastic Leukemia from a Matched Donor versus an HLA-Identical Sibling: Is the Outcome Comparable? Results from the International BFM ALL SCT 2007 Study

Author

Balduzzi, Adriana, Dalle, Jean-Hugues, Wachowiak, Jacek, Yaniv, Isaac, Yesilipek, Akif, Sedlacek, Petr, Bierings, Marc, Ifversen, Marianne, Sufliarska, Sabina, Kalwak, Krzysztof, Lankester, Arjan, Toporski, Jacek, Di Maio, Lucia, Glogova, Evgenia, Poetschger, Ulrike, and Peters, Christina

Published
2019
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25. 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)

Author

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

Subjects
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.

Published
2021
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26. Allogeneic Stem Cell Transplantation from HLA-Mismatched Donors for Pediatric Patients with Acute Lymphoblastic Leukemia Treated According to the 2003 BFM and 2007 International BFM Studies: Impact of Disease Risk on Outcomes

Author

Dalle, Jean-Hugues, Balduzzi, Adriana, Bader, Peter, Lankester, Arjan, Yaniv, Isaac, Wachowiak, Jacek, Pieczonka, Anna, Bierings, Marc, Yesilipek, Akif, Sedlaçek, Petr, Ifversen, Marianne, Sufliarska, Sabina, Toporski, Jacek, Glogova, Evgenia, Poetschger, Ulrike, and Peters, Christina

Published
2018
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28. Clinical Features and HSCT Outcome for SCID in Turkey

Author

Ikinciogullari, Aydan, Cagdas, Deniz, Dogu, Figen, Tugrul, Tuba, Karasu, Gulsum, Haskologlu, Sule, Aksoylar, Serap, Uygun, Vedat, Kupesiz, Alphan, Yildiran, Alisan, Gursel, Orhan, Ates, Can, Elhan, Atilla, Kansoy, Savas, Yesilipek, Akif, Tezcan, Ilhan, and on behalf of Turkish Pediatric Bone Marrow Transplantation Sub Group (TPBMT-SG)

Published
2019
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29. Hematopoietic Stem Cell Transplantation Using Preimplantation Genetic Diagnosis and Human Leukocyte Antigen Typing for Human Leukocyte Antigen–Matched Sibling Donor: A Turkish Multicenter Study

Author

Kurekci, Emin, Küpesiz, Alphan, Anak, Sema, Öztürk, Gülyüz, Gürsel, Orhan, Aksoylar, Serap, Ileri, Talia, Kuşkonmaz, Barış, Eker, İbrahim, Cetin, Mualla, Tezcan Karasu, Gülsün, Kaya, Zühre, Fışgın, Tunç, Ertem, Mehmet, Kansoy, Savaş, and Yeşilipek, Mehmet Akif

Published
2017
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30. Comparison of tacrolimus vs. cyclosporine in pediatric hematopoietic stem cell transplantation for thalassemia.

Author

Zhumatayev, Suleimen, Yalcin, Koray, Celen, Safiye Suna, Karaman, Irem, Daloglu, Hayriye, Ozturkmen, Seda, Uygun, Vedat, Karasu, Gulsun, and Yesilipek, Akif

Subjects
HEPATIC veno-occlusive disease, STEM cell transplantation, HEMATOPOIETIC stem cell transplantation, TACROLIMUS, CYCLOSPORINE, THALASSEMIA, CHILD patients, GRAFT versus host disease
Abstract

Objectives: Graft‐versus‐host disease (GvHD) is one of the leading causes of morbidity and mortality in patients undergoing allogeneic HSCT, and effective prevention of GvHD is critical for the success of the HSCT procedure. Calcineurin inhibitors (CNI) have been used for decades as the backbone of GvHD prophylaxis. In this study, the efficacy and safety of Cyclosporine A (CsA) and tacrolimus (TCR) were compared in pediatric HSCT for thalassemia. Materials and Methods: This is a retrospective analysis of 129 pediatric patients who underwent HSCT with the diagnosis of thalassemia at Medicalpark Göztepe and Antalya Hospitals between January 2017 and December 2020. Results: Despite the GvHD prophylaxis, grade II–IV acute GvHD developed in 29 patients. Of these patients, 12 had only gut, 10 had only skin, 6 had combined gut and skin, and one had only liver GvHD. Fifteen of these 29 patients were in the CsA group, and 14 of them were in the TCR group. There was no significant difference between the groups in terms of acute GvHD occurrence, GvHD stage, or involvement sites. In terms of CNI‐related toxicity, neurotoxicity in 15 (CsA n = 9, TCR n = 6) and nephrotoxicity in 18 (CsA n = 4, TCR n = 14) patients were observed. While there was no difference between the two groups in terms of neurotoxicity, more nephrotoxicity developed in patients using TCR (p =.013). There was no significant difference between the groups in terms of engraftment syndrome, veno‐occlusive disease, CMV reactivation, PRES, or graft rejection. Conclusion: Regarding GvHD, there was no difference in efficacy between TCR and CsA usage. Patients taking TCR experienced noticeably higher nephrotoxicity in terms of adverse effects. This difference should be considered according to the patient's clinical situation while choosing a CNI. [ABSTRACT FROM AUTHOR]

Published
2024
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31. Different Kinetics and Risk Factors for Isolated Extramedullary Relapse after Allogeneic Hematopoietic Stem Cell Transplantation in Children with Acute Leukemia

Author

Hazar, Volkan, Öztürk, Gülyüz, Yalçın, Koray, Uygun, Vedat, Aksoylar, Serap, Küpesiz, A., Ok Bozkaya, İkbal, Karagün, Barbaros Şahin, Bozkurt, Ceyhun, İleri, Talia, Atay, Didem, Koçak, Ülker, Karasu, Gülsün Tezcan, Yeşilipek, Akif, Gökçe, Müge, Kansoy, Savaş, Kintrup, Gülen Tüysüz, Karakükcü, Musa, Okur, Fatma Visal, Ertem, Mehmet, Kaya, Zühre, Gürsel, Orhan, Yaman, Yöntem, Özbek, Namık, Antmen, Bülent, Tüfekçi, Özlem, Albayrak, Canan, Adaklı Aksoy, Başak, Sezgin, Gülay, Albayrak, Davut, Evim, Melike Sezgin, Zengin, Emine, and Pekpak, Esra

Published
2021
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32. Haematopoietic stem cell transplantation for severe autoimmune diseases in children: A review of current literature, registry activity and future directions on behalf of the autoimmune diseases and paediatric diseases working parties of the European Society for Blood and Marrow Transplantation

Author

Achini-Gutzwiller, F, Snowden, J, Corbacioglu, S, Greco, R, Alexander, T, Badoglio, M, Labopin, M, Abinun, M, Apte, S, Arnold, R, Domenech, A, Brierley, C, Burman, J, Castilla-Llorente, C, Cooper, N, Daghia, G, Daikeler, T, del Papa, N, de Vries-Bouwstra, J, Farge, D, Finke, J, Hagglund, H, Hawkey, C, Henes, J, Hiepe, F, Jessop, H, Kiely, D, Kazmi, M, Kirgizov, K, Kramer, E, Mancardi, G, Marjanovic, Z, Martin, R, Martin, T, Ma, D, Moore, J, Miller, P, Muraro, P, Oliveira, M, Polushin, A, Onida, F, Simoes, B, Puyade, M, Resnick, I, Ricart, E, Rovira, M, Saccardi, R, Saif, M, Sakellari, I, Sharrack, B, Snarski, E, Scherer, H, Sossa, C, Withers, B, Wulffraat, N, Zaccara, E, Amrolia, P, Ansari, M, Balduzzi, A, Dalassier, A, Dalle, J, Diaz, C, Feuchtinger, T, Locatelli, F, Lucchini, G, Galimard, J, Vincent, M, Handgretinger, R, Kleinschmidt, K, Lawitschka, A, Martinez, A, Peters, C, Rocha, V, Ruggeri, A, Sedlacek, P, Svec, P, Toporski, J, Yesilipek, A, Achini-Gutzwiller F. R., Snowden J. A., Corbacioglu S., Greco R., Alexander T., Snowden J., Badoglio M., Labopin M., Abinun M., Apte S., Arnold R., Domenech A., Brierley C., Burman J., Castilla-Llorente C., Cooper N., Daghia G., Daikeler T., del Papa N., de Vries-Bouwstra J., Farge D., Finke J., Hagglund H., Hawkey C., Henes J., Hiepe F., Jessop H., Kiely D., Kazmi M., Kirgizov K., Kramer E., Mancardi G., Marjanovic Z., Martin R., Martin T., Ma D., Moore J., Miller P., Muraro P., Oliveira M. -C., Polushin A., Onida F., Simoes B., Puyade M., Resnick I., Ricart E., Rovira M., Saccardi R., Saif M., Sakellari I., Sharrack B., Snarski E., Scherer H. U., Sossa C., Withers B., Wulffraat N., Zaccara E., Amrolia P., Ansari M., Balduzzi A., Dalassier A., Dalle J. -H., Diaz C. H., Feuchtinger T., Locatelli F., Lucchini G., Galimard J. -E., Vincent M. G., Handgretinger R., Kleinschmidt K., Lawitschka A., Martinez A. P., Peters C., Rocha V., Ruggeri A., Sedlacek P., Svec P., Toporski J., Yesilipek A., Achini-Gutzwiller, F, Snowden, J, Corbacioglu, S, Greco, R, Alexander, T, Badoglio, M, Labopin, M, Abinun, M, Apte, S, Arnold, R, Domenech, A, Brierley, C, Burman, J, Castilla-Llorente, C, Cooper, N, Daghia, G, Daikeler, T, del Papa, N, de Vries-Bouwstra, J, Farge, D, Finke, J, Hagglund, H, Hawkey, C, Henes, J, Hiepe, F, Jessop, H, Kiely, D, Kazmi, M, Kirgizov, K, Kramer, E, Mancardi, G, Marjanovic, Z, Martin, R, Martin, T, Ma, D, Moore, J, Miller, P, Muraro, P, Oliveira, M, Polushin, A, Onida, F, Simoes, B, Puyade, M, Resnick, I, Ricart, E, Rovira, M, Saccardi, R, Saif, M, Sakellari, I, Sharrack, B, Snarski, E, Scherer, H, Sossa, C, Withers, B, Wulffraat, N, Zaccara, E, Amrolia, P, Ansari, M, Balduzzi, A, Dalassier, A, Dalle, J, Diaz, C, Feuchtinger, T, Locatelli, F, Lucchini, G, Galimard, J, Vincent, M, Handgretinger, R, Kleinschmidt, K, Lawitschka, A, Martinez, A, Peters, C, Rocha, V, Ruggeri, A, Sedlacek, P, Svec, P, Toporski, J, Yesilipek, A, Achini-Gutzwiller F. R., Snowden J. A., Corbacioglu S., Greco R., Alexander T., Snowden J., Badoglio M., Labopin M., Abinun M., Apte S., Arnold R., Domenech A., Brierley C., Burman J., Castilla-Llorente C., Cooper N., Daghia G., Daikeler T., del Papa N., de Vries-Bouwstra J., Farge D., Finke J., Hagglund H., Hawkey C., Henes J., Hiepe F., Jessop H., Kiely D., Kazmi M., Kirgizov K., Kramer E., Mancardi G., Marjanovic Z., Martin R., Martin T., Ma D., Moore J., Miller P., Muraro P., Oliveira M. -C., Polushin A., Onida F., Simoes B., Puyade M., Resnick I., Ricart E., Rovira M., Saccardi R., Saif M., Sakellari I., Sharrack B., Snarski E., Scherer H. U., Sossa C., Withers B., Wulffraat N., Zaccara E., Amrolia P., Ansari M., Balduzzi A., Dalassier A., Dalle J. -H., Diaz C. H., Feuchtinger T., Locatelli F., Lucchini G., Galimard J. -E., Vincent M. G., Handgretinger R., Kleinschmidt K., Lawitschka A., Martinez A. P., Peters C., Rocha V., Ruggeri A., Sedlacek P., Svec P., Toporski J., and Yesilipek A.

Abstract

Although modern clinical management strategies have improved the outcome of paediatric patients with severe autoimmune and inflammatory diseases over recent decades, a proportion will experience ongoing or recurrent/relapsing disease activity despite multiple therapies often leading to irreversible organ damage, and compromised quality of life, growth/development and long-term survival. Autologous and allogeneic haematopoietic stem cell transplantation (HSCT) have been used successfully to induce disease control and often apparent cure of severe treatment-refractory autoimmune diseases (ADs) in children. However, transplant-related outcomes are disease-dependent and long-term outcome data are limited in respect to efficacy and safety. Moreover, balancing risks of HSCT against AD prognosis with continually evolving non-transplant options is challenging. This review appraises published literature on HSCT strategies and outcomes in individual paediatric ADs. We also provide a summary of the European Society for Blood and Marrow Transplantation (EBMT) Registry, where 343 HSCT procedures (176 autologous and 167 allogeneic) have been reported in 326 children (<18 years) for a range of AD indications. HSCT is a promising treatment modality, with potential long-term disease control or cure, but therapy-related morbidity and mortality need to be reduced. Further research is warranted to establish the position of HSCT in paediatric ADs via registries and prospective clinical studies to support evidence-based interspeciality guidelines and recommendations.

Published
2022

33. Outcomes of Unmanipulated Haploidentical Transplantation Using Post-Transplant Cyclophosphamide (PT-Cy) in Pediatric Patients With Acute Lymphoblastic Leukemia

Author

Ruggeri, Annalisa, Galimard, Jacques-Emmanuel, Paina, Olesya, Fagioli, Franca, Tbakhi, Abdelghani, Yesilipek, Akif, Navarro, José Maria Fernandez, Faraci, Maura, Hamladji, Rose-Marie, Skorobogatova, Elena, Al-Seraihy, Amal, Sundin, Mikael, Herrera, Concepcion, Rifón, Jose, Dalissier, Arnaud, Locatelli, Franco, Rocha, Vanderson, and Corbacioglu, Selim

Published
2021
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34. Vaccine Adherence and Postvaccination Serological Status of Pediatric Allogeneic Hematopoietic Stem Cell Transplant Recipients: A Single-center Experience

Author

Oyku Ozboru Askan, Tulin Ayse Ozden, Gulsun Karasu Tezcan, Gonca Keskindemirci, Alev Bakir, Deniz Tugcu, Fugen Pekun, Akif Yesilipek, and Emine Gulbin Gokcay

Subjects
Oncology, Pediatrics, Perinatology and Child Health, Hematology
Abstract

Despite developing consensus guidelines addressing immunization after hematopoietic stem cell transplantation (HSCT), studies showed deviations from recommended immunization practices commonly occur. Difference between the ideal scenario presented in guidelines and real-life scenarios is one of the most recognized barriers to implementing recommended practices. Therefore, this study aimed to evaluate pediatric allogeneic HSCT recipients' adherence to revaccination schedule and evaluate the serological status after immunization. Transplant and vaccination records of children who were followed up at least 2 years after HSCT, postvaccination antibody results of vaccine-preventable diseases were evaluated retrospectively. Total of 173 patients have enrolled in this study. Median revaccination onset time was post-transplant 15 months. Adherence to revaccination program was 30% for inactive and 11.4% for live vaccines. Oral polio vaccine was given to 22 patients, and Bacille-Calmette-Guerin vaccine was applied to 3. Seropositivity after revaccination was90% for Hepatitis B, Hepatitis A, pertussis, and measles, and it was 88.5% for rubella, 80% for mumps and varicella. Measles seropositivity was low in children with hemoglobinopathy. In subgroup assessments of pertussis, patients vaccinated with low antigen-containing pertussis vaccine (Tdap) had higher seropositivity of adenylate cyclase toxin. Our findings revealed the importance of careful monitoring of current practices in pediatric HSCT recipients.

Published
2022

36. Relapse Is the Most Common Treatment Failure Post HSCT in Children with ALL below 4 Years of Age Given a Chemo-Based Conditioning Regimen. Results from the Prospective Multinational Forum-Trial

Author

Christina Peters, Ulrike Poetschger, Jean-Hugues Dalle, Akif Yesilipek, Jerry Stein, Herbert Pichler, Adriana Balduzzi, Franco Locatelli, Petr Sedlacek, Jacek Wachowiak, Tayfun Güngör, Marianne Ifversen, Marleen Renard, Tony H Truong, Raquel Staciuk, Peter J. Shaw, Gergely Kriván, Jochen Büchner, and Peter Bader

Subjects
Immunology, Cell Biology, Hematology, Biochemistry
Published
2022

37. 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

Author

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

Subjects
Immunology, Cell Biology, Hematology, Biochemistry
Published
2022

38. Superior Graft-Versus-Leukemia Effect in Matched Unrelated Donor Versus HLA-Identical Sibling Pediatric Recipients Transplanted for Acute Lymphoblastic Leukemia within the Forum Study

Author

Jean-Hugues Dalle, Peter Bader, Akif Yesilipek, Franco Locatelli, Julia Palma, Jacek Wachowiak, Herbert Pichler, Marianne Ifversen, Gergely Kriván, Jochen Buechner, Cristina Díaz-de-Heredia, Marc Bierings, Raquel Staciuk, Tayfun Gungor, Jacek Toporski, Adriana Balduzzi, Ulrike Poetschger, Christina Peters, and Petr Sedlacek

Subjects
Immunology, Cell Biology, Hematology, Biochemistry
Published
2022

39. Hematopoietic stem cell transplantation in children with mucopolysaccharidosis IVA: single center experience

Author

Yalcin, Koray, Uygun, Vedat, Ozturk Hismi, Burcu, Celen, Suna, Ozturkmen, Seda, Zhumatayev, Suleimen, Daloglu, Hayriye, Karasu, Gülsün, and Yesilipek, Akif

Abstract

Mucopolysaccharidosis IVA (MPS IVA; Morquio syndrome) is a lysosomal storage disorder and features systemic skeletal dysplasia that is caused by defective Nacetylgalactosamine-6-sulfate sulfatase (GALNS). Although there are convincing data for hematopoietic stem cell transplantation (HSCT) in certain types of MPS, the studies are limited for MPS IVA and more data is still pending to show the efficacy/safety of HSCT. This study included 3 girls and 7 boys, with a median age of 75,5 months (35–186 months), who underwent allogeneic HSCT for severe MPS IVA between February 12, 2021, and March 10, 2023. Enzyme levels, height growth, the most involved organs (ear, eye, and heart), and the activities of daily living (ADL) scoring system were monitored to assess the benefit of HSCT. In a median follow-up of 20 months (9–34 months), there is no severe transplant-related adverse event was observed. In all cases, normal enzyme levels were reached after HSCT. During the short follow-up period, our cases showed an increase in stature and improvement in daily activity functions. Here we present the data of our HSCT experience in MPS IVA with promising results regarding both safety and efficacy. Although there are signs of amelioration with HSCT, we need more data and long-term follow-up to comment properly on the benefits of HSCT in MPS IVA.

Published
2024
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40. Haematopoietic stem cell transplantation for severe autoimmune diseases in children : a review of current literature, registry activity and future directions on behalf of the autoimmune diseases and paediatric diseases working parties of the European Society for Blood and Marrow Transplantation

Author

Achini‐Gutzwiller, FR, Snowden, JA, Corbacioglu, S, Greco, R, Alexander, T, Snowden, J, Badoglio, M, Labopin, M, Abinun, M, Apte, S, Arnold, R, Domenech, A, Brierley, C, Burman, J, Castilla‐Llorente, C, Cooper, N, Daghia, G, Daikeler, T, del Papa, N, de Vries‐Bouwstra, J, Farge, D, Finke, J, Hagglund, H, Hawkey, C, Henes, J, Hiepe, F, Jessop, H, Kiely, D, Kazmi, M, Kirgizov, K, Kramer, E, Mancardi, G, Marjanovic, Z, Martin, R, Martin, T, Ma, D, Moore, J, Miller, P, Muraro, P, Oliveira, M, Polushin, A, Onida, F, Simoes, B, Puyade, M, Resnick, I, Ricart, E, Rovira, M, Saccardi, R, Saif, M, Sakellari, I, Sharrack, B, Snarski, E, Scherer, HU, Sossa, C, Withers, B, Wulffraat, N, Zaccara, E, Amrolia, P, Ansari, M, Balduzzi, A, Dalassier, A, Dalle, J, Diaz, CH, Feuchtinger, T, Locatelli, F, Lucchini, G, Galimard, J, Vincent, MG, Handgretinger, R, Kleinschmidt, K, Lawitschka, A, Martinez, AP, Peters, C, Rocha, V, Ruggeri, A, Sedlacek, P, Svec, P, Toporski, J, Yesilipek, A, Achini-Gutzwiller, F, Snowden, J, Corbacioglu, S, Greco, R, Alexander, T, Badoglio, M, Labopin, M, Abinun, M, Apte, S, Arnold, R, Domenech, A, Brierley, C, Burman, J, Castilla-Llorente, C, Cooper, N, Daghia, G, Daikeler, T, del Papa, N, de Vries-Bouwstra, J, Farge, D, Finke, J, Hagglund, H, Hawkey, C, Henes, J, Hiepe, F, Jessop, H, Kiely, D, Kazmi, M, Kirgizov, K, Kramer, E, Mancardi, G, Marjanovic, Z, Martin, R, Martin, T, Ma, D, Moore, J, Miller, P, Muraro, P, Oliveira, M, Polushin, A, Onida, F, Simoes, B, Puyade, M, Resnick, I, Ricart, E, Rovira, M, Saccardi, R, Saif, M, Sakellari, I, Sharrack, B, Snarski, E, Scherer, H, Sossa, C, Withers, B, Wulffraat, N, Zaccara, E, Amrolia, P, Ansari, M, Balduzzi, A, Dalassier, A, Dalle, J, Diaz, C, Feuchtinger, T, Locatelli, F, Lucchini, G, Galimard, J, Vincent, M, Handgretinger, R, Kleinschmidt, K, Lawitschka, A, Martinez, A, Peters, C, Rocha, V, Ruggeri, A, Sedlacek, P, Svec, P, Toporski, J, and Yesilipek, A

Subjects
autoimmune diseases, haematopoietic stem cell transplantation, paediatric, ddc:610, paediatric, surgical procedures, operative, Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, haematopoietic stem cell transplantation, 610 Medizin, autoimmune diseases, autoimmune disease, Hematology
Abstract

Although modern clinical management strategies have improved the outcome of paediatric patients with severe autoimmune and inflammatory diseases over recent decades, a proportion will experience ongoing or recurrent/relapsing disease activity despite multiple therapies often leading to irreversible organ damage, and compromised quality of life, growth/development and long-term survival. Autologous and allogeneic haematopoietic stem cell transplantation (HSCT) have been used successfully to induce disease control and often apparent cure of severe treatment-refractory autoimmune diseases (ADs) in children. However, transplant-related outcomes are disease-dependent and long-term outcome data are limited in respect to efficacy and safety. Moreover, balancing risks of HSCT against AD prognosis with continually evolving non-transplant options is challenging. This review appraises published literature on HSCT strategies and outcomes in individual paediatric ADs. We also provide a summary of the European Society for Blood and Marrow Transplantation (EBMT) Registry, where 343 HSCT procedures (176 autologous and 167 allogeneic) have been reported in 326 children (

Published
2022

41. Stem cell transplantation in severe congenital neutropenia: an analysis from the European Society for Blood and Marrow Transplantation

Author

Fioredda, Francesca, Iacobelli, Simona, van Biezen, Anja, Gaspar, Bobby, Ancliff, Phil, Donadieu, Jean, Aljurf, Mahmoud, Peters, Christina, Calvillo, Michaela, Matthes-Martin, Susanne, Morreale, Giuseppe, van 't Veer-Tazelaar, Nelleke, de Wreede, Liesbeth, Al Seraihy, Amal, Yesilipek, Akif, Fischer, Alain, Bierings, Marc, Ozturk, Gulyuz, Smith, Owen, Veys, Paul, Ljungman, Per, Peffault de Latour, Régis, Sánchez de Toledo Codina, José, Or, Reuven, Ganser, Arnold, Afanasyev, Boris, Wynn, Robert, Kalwak, Krzysztof, Marsh, Judith, and Dufour, Carlo

Published
2015
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42. Correction: ABO incompatibile graft management in pediatric transplantation

Author

Balduzzi, Adriana, Bönig, Halvard, Jarisch, Andrea, Nava, Tiago, Ansari, Marc, Cattoni, Alessandro, Prunotto, Giulia, Lucchini, Giovanna, Krivan, Gergely, Matic, Toni, Kalwak, Krzyzstof, Yesilipek, Akif, Ifversen, Marianne, Svec, Peter, Buechner, Jochen, Vettenranta, Kim, Meisel, Roland, Lawitschka, Anita, Peters, Christina, Gibson, Brenda, Dalissier, Arnaud, Corbacioglu, Selim, Willasch, André, Dalle, Jean-Hugues, and Bader, Peter

Published
2021
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43. Prophylaxis vs. on‐demand treatment with BAY 81‐8973, a full‐length plasma protein‐free recombinant factor VIII product: results from a randomized trial (LEOPOLD II)

Author

Kavakli, K., Yang, R., Rusen, L., Beckmann, H., Tseneklidou‐Stoeter, D., Maas Enriquez, M., Yang, Renchi, Zhao, Yongqiang, Sun, Jing, Wang, Xuefeng, Wu, Depei, Hlusi, Antonin, Fukutake, Katsuyuki, Hanabusa, Hideji, Fujii, Teruhisa, Ramírez, Oscar Pérez, Alvarado, Blanca Salazar, Serban, Margit, Rusen, Luminita, Uscatescu, Valentina, Truica, Cristina, Kostic, Gordana, Konstantinidis, Nada, Igrutinovic, Zoran, Perina, Farida, Andreeva, Tatiana, Kavakli, Kaan, Antmen, Bulent, Sasmaz, Ilgen, Kupesiz, Alphan, Yesilipek, Mehmet Akif, Peng, Ching‐Tien, French, James, II, Escobar, Miguel, Mahlangu, Johnny, and Pool, Roger

Published
2015
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44. Fertility preservation issues in pediatric hematopoietic stem cell transplantation: practical approaches from the consensus of the Pediatric Diseases Working Party of the EBMT and the International BFM Study Group

Author

Balduzzi, A, Dalle, J-H, Jahnukainen, K, von Wolff, M, Lucchini, G, Ifversen, M, Macklon, K T, Poirot, C, Diesch, T, Jarisch, A, Bresters, D, Yaniv, I, Gibson, B, Willasch, A M, Fadini, R, Ferrari, L, Lawitschka, A, Ahler, A, Sänger, N, Corbacioglu, S, Ansari, M, Moffat, R, Dalissier, A, Beohou, E, Sedlacek, P, Lankester, A, De Heredia Rubio, C D, Vettenranta, K, Wachowiak, J, Yesilipek, A, Trigoso, E, Klingebiel, T, Peters, C, and Bader, P

Published
2017
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45. 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

Author

Dalle, J-H, Lucchini, G, Balduzzi, A, Ifversen, M, Jahnukainen, K, Macklon, K T, Ahler, A, Jarisch, A, Ansari, M, Beohou, E, Bresters, D, Corbacioglu, S, Dalissier, A, Diaz 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

Published
2017
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46. Pre‐transplantation vitamin D deficiency increases acute graft‐versus‐host disease after hematopoietic stem cell transplantation in thalassemia major patients

Author

Daloğlu, Hayriye, Uygun, Vedat, Ozturkmen, Seda, Yalcın, Koray, Karasu, Gulsu, Yesilipek, Akif, İstinye Üniversitesi, Tıp Fakültesi, Dahili Tıp Bilimleri Bölümü, Uygun, Vedat, and AGH-4534-2022

Subjects
Transplantation, Acute Graft Versus Host Disease, Hematopoetic Stem Cell Transplantation, Vitamin D, Children, Thalassemia Major
Abstract

Background: Although there are many studies on the role of vitamin D deficiency (VDD) in hematopoetic stem cell transplantation (HSCT), outcomes have often reported conflicting results because of the heterogeneity of the patients in the studies. Methods: We investigated the association between VDD prior to HSCT and outcomes after HSCT in a relatively homogenous group of patients with thalassemia major (TM) who received identical treatment for TM before transplantation, and the same conditioning regimen and GVHD prophylaxis during and after transplantation. All patients, including the patients with normal vitamin D3 levels received 400 to 800 IU per day of vitamin D for the first 6 months after HSCT. Results: Pre-HSCT VDD increased the frequency of aGVHD after transplantation, particularly in HSCTs performed with PBSC for the stem cell source. Pre-transplant low vitamin D3 levels had no association with transplant outcomes such as engraftment, viral infections, alloimmunization, chronic GvHD, total days of hospitalization, and success in terms of transfusion independence. Conclusions: Low vitamin D3 levels before HSCT carry a significant risk for aGVHD. All patients with TM should be screened for VDD before HSCT, and every effort should be made to supplement vitamin D before the transplant in VDD patients 36461145

Published
2022

47. 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)

Author

Nava, Tiago, Ansari, Marc, Dalle, Jean-Hugues, de Heredia, Christina Diaz, Güngör, Tayfun, Trigoso, Eugenia, Falkenberg, Ulrike, Bertaina, Alice, Gibson, Brenda, Jarisch, Andrea, Balduzzi, Adriana, Boenig, Halvard, Krivan, Gergely, Vettenranta, Kim, Matic, Toni, Buechner, Jochen, Kalwak, Krzysztof, Lawitschka, Anita, Yesilipek, Akif, Lucchini, Giovanna, Peters, Christina, Turkiewicz, Dominik, Niinimäki, Riitta, Diesch, Tamara, Lehrnbecher, Thomas, Sedlacek, Petr, Hutt, Daphna, Dalissier, Arnaud, Wachowiak, Jacek, Yaniv, Isaac, Stein, Jerry, Yalçin, Koray, Sisinni, Luisa, Deiana, Marco, Ifversen, Marianne, Kuhlen, Michaela, Meisel, Roland, Bakhtiar, Shahrzad, Cesaro, Simone, Willasch, Andre, Corbacioglu, Selim, and Bader, Peter

Published
2020
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48. Superior Graft-Versus-Leukemia Effect in Matched Unrelated Donor Versus HLA-Identical Sibling Pediatric Recipients Transplanted for Acute Lymphoblastic Leukemia within the Forum Study

Author

Dalle, Jean-Hugues, primary, Bader, Peter, additional, Yesilipek, Akif, additional, Locatelli, Franco, additional, Palma, Julia, additional, Wachowiak, Jacek, additional, Pichler, Herbert, additional, Ifversen, Marianne, additional, Kriván, Gergely, additional, Buechner, Jochen, additional, Díaz-de-Heredia, Cristina, additional, Bierings, Marc, additional, Staciuk, Raquel, additional, Gungor, Tayfun, additional, Toporski, Jacek, additional, Balduzzi, Adriana, additional, Poetschger, Ulrike, additional, Peters, Christina, additional, and Sedlacek, Petr, additional

Published
2022
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49. 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

Author

Lum, Su Han, primary, Samarasinghe, Sujith, additional, Eikema, Dirk-Jan, additional, Piepenbroek, Brian, additional, Dalissier, Arnaud, additional, Ayas, Mouhab, additional, Mousavi, Ashrafsadat, additional, Hamladji, Rose-Marie, additional, Yesilipek, Akif, additional, Dalle, Jean-Hugues, additional, Kansoy, Savas, additional, Locatelli, Franco, additional, Çetinkaya, Duygu, additional, Bierings, Marc, additional, Kupesiz, O. Alphan, additional, Tbakhi, Abdelghani, additional, Skorobogatova, Elena, additional, Öztürk, Gülyüz, additional, Faraci, Maura, additional, Bertrand, Yves, additional, Evans, Pamela, additional, Carbacioglu, Selim, additional, Dufour, Carlo, additional, Risitano, Antonio, additional, Wynn, Robert F, additional, and Peffault de Latour, Régis, additional

Published
2022
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50. Relapse Is the Most Common Treatment Failure Post HSCT in Children with ALL below 4 Years of Age Given a Chemo-Based Conditioning Regimen. Results from the Prospective Multinational Forum-Trial

Author

Peters, Christina, primary, Poetschger, Ulrike, additional, Dalle, Jean-Hugues, additional, Yesilipek, Akif, additional, Stein, Jerry, additional, Pichler, Herbert, additional, Balduzzi, Adriana, additional, Locatelli, Franco, additional, Sedlacek, Petr, additional, Wachowiak, Jacek, additional, Güngör, Tayfun, additional, Ifversen, Marianne, additional, Renard, Marleen, additional, Truong, Tony H, additional, Staciuk, Raquel, additional, Shaw, Peter J., additional, Kriván, Gergely, additional, Büchner, Jochen, additional, and Bader, Peter, additional

Published
2022
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