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51. Hematopoietic stem cell transplantation does not increase the risk of infection‐related complications for pediatric patients with Hodgkin and non‐Hodgkin lymphomas: A multicenter nationwide study

Author

Zając‐Spychała, O., primary, Wachowiak, J., additional, Czyżewski, K., additional, Dziedzic, M., additional, Wysocki, M., additional, Zalas‐Więcek, P., additional, Szmydki‐Baran, A., additional, Hutnik, Ł., additional, Matysiak, M., additional, Małas, Z., additional, Badowska, W., additional, Gryniewicz–Kwiatkowska, O., additional, Gietka, A., additional, Dembowska‐Bagińska, B., additional, Semczuk, K., additional, Dzierżanowska‐Fangrat, K., additional, Bartnik, M., additional, Ociepa, T., additional, Urasiński, T., additional, Frączkiewicz, J., additional, Salamonowicz, M., additional, Kałwak, K., additional, Gorczyńska, E., additional, Chybicka, A., additional, Irga‐Jaworska, N., additional, Bień, E., additional, Drożyńska, E., additional, Chełmecka‐Wiktorczyk, L., additional, Balwierz, W., additional, Zak, I., additional, Pierlejewski, F., additional, Młynarski, W., additional, Urbanek‐Dądela, A., additional, Karolczyk, G., additional, Stolpa, W., additional, Sobol‐Milejska, G., additional, Płonowski, M., additional, Krawczuk‐Rybak, M., additional, Musiał, J., additional, Chaber, R., additional, Gamrot‐Pyka, Z., additional, Woszczyk, M., additional, Tomaszewska, R., additional, Szczepański, T., additional, Kowalczyk, J., additional, and Styczyński, J., additional

Published
2020
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56. Multidrug‐resistant bacterial infections in children undergoing haematopoietic stem cell transplantation over a 6‐year period: analysis of the Polish Pediatric Group for Hematopoietic Stem Cell Transplantation

Author

Zając‐Spychała, O., primary, Wachowiak, J., additional, Frączkiewicz, J., additional, Salamonowicz, M., additional, Kałwak, K., additional, Gorczyńska, E., additional, Chybicka, A., additional, Czyżewski, K., additional, Dziedzic, M., additional, Wysocki, M., additional, Zalas‐Wiącek, P., additional, Zaucha‐Prażmo, A., additional, Kowalczyk, J.R., additional, Goździk, J., additional, and Styczyński, J., additional

Published
2019
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57. Mediastinal germ cell tumours in children and adolescents: Report from the Polish Pediatric Solid Tumor Group

Author

Stefanowicz, J., primary, Adamkiewicz-Drożyńska, E., additional, Jezierska, M., additional, Polczynska, K., additional, Izycka-Swieszewska, E., additional, Balwierz, W., additional, Chełmecka-Wiktorczyk, L., additional, Dębski, R., additional, Peregut-Pogorzelski, J., additional, Sobol, G., additional, Urasinski, T., additional, Wachowiak, J., additional, Wachowiak-Szajdak, K., additional, and Wawryków, P., additional

Published
2019
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58. The Results of Treatment of Acute Lymphoblastic Leukemia Relapses in the Polish Children’s Leukemia/Lymphoma Study Group

Author

Bogusławska-Jaworska, J., primary, Pisarek, J., additional, Chybicka, A., additional, Kazanowska, B., additional, Armata, J., additional, Balwierz, W., additional, Bubała, H., additional, Jackowska, T., additional, Michalewska, D., additional, Ochocka, M., additional, Radwanska, U., additional, Milewska, R. Rokicka, additional, Rytlewska, M., additional, Skomra, S., additional, Sladkowska, G., additional, Wachowiak, J., additional, Wieczorek, M., additional, Zelenay, E., additional, Sońta-Jakimczuk, D., additional, and Woźniak, A., additional

Published
1992
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59. Diagnosis and severity criteria for sinusoidal obstruction syndrome/veno-occlusive disease in pediatric patients: A new classification from the European society for blood and marrow transplantation

Author

Corbacioglu, S., Carreras, E., Ansari, M., Balduzzi, A., Cesaro, S., Dalle, J. -H., Dignan, F., Gibson, B., Guengoer, T., Gruhn, B., Lankester, A., Locatelli, Franco, Pagliuca, A., Peters, C., Richardson, P. G., Schulz, A. S., Sedlacek, P., Stein, J., Sykora, K. -W., Toporski, J., Trigoso, E., Vetteranta, K., Wachowiak, J., Wallhult, E., Wynn, R., Yaniv, I., Yesilipek, A., Mohty, M., Bader, P., Locatelli F. (ORCID:0000-0002-7976-3654), Corbacioglu, S., Carreras, E., Ansari, M., Balduzzi, A., Cesaro, S., Dalle, J. -H., Dignan, F., Gibson, B., Guengoer, T., Gruhn, B., Lankester, A., Locatelli, Franco, Pagliuca, A., Peters, C., Richardson, P. G., Schulz, A. S., Sedlacek, P., Stein, J., Sykora, K. -W., Toporski, J., Trigoso, E., Vetteranta, K., Wachowiak, J., Wallhult, E., Wynn, R., Yaniv, I., Yesilipek, A., Mohty, M., Bader, P., and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

The advances in hematopoietic cell transplantation (HCT) over the last decade have led to a transplant-related mortality below 15%. Hepatic sinusoidal obstruction syndrome/veno-occlusive disease (SOS/VOD) is a life-threatening complication of HCT that belongs to a group of diseases increasingly identified as transplant-related, systemic endothelial diseases. In most cases, SOS/VOD resolves within weeks; however, severe SOS/VOD results in multi-organ dysfunction/failure with a mortality rate >80%. A timely diagnosis of SOS/VOD is of critical importance, given the availability of therapeutic options with favorable tolerability. Current diagnostic criteria are used for adults and children. However, over the last decade it has become clear that SOS/VOD is significantly different between the age groups in terms of incidence, genetic predisposition, clinical presentation, prevention, treatment and outcome. Improved understanding of SOS/VOD and the availability of effective treatment questions the use of the Baltimore and Seattle criteria for diagnosing SOS/VOD in children. The aim of this position paper is to propose new diagnostic and severity criteria for SOS/VOD in children on behalf of the European Society for Blood and Marrow Transplantation.

Published
2018

60. Outcome of relapse after allogeneic HSCT in children with ALL enrolled in the ALL-SCT 2003/2007 trial

Author

Kuhlen, M, Willasch, A, Dalle, J, Wachowiak, J, Yaniv, I, Ifversen, M, Sedlacek, P, Guengoer, T, Lang, P, Bader, P, Sufliarska, S, Balduzzi, A, Strahm, B, von Luettichau, I, Hoell, J, Borkhardt, A, Klingebiel, T, Schrappe, M, von Stackelberg, A, Glogova, E, Poetschger, U, Meisel, R, Peters, C, Kuhlen, Michaela, Willasch, Andre M., Dalle, Jean-Hugues, Wachowiak, Jacek, Yaniv, Isaac, Ifversen, Marianne, Sedlacek, Petr, Guengoer, Tayfun, Lang, Peter, Bader, Peter, Sufliarska, Sabina, Balduzzi, Adriana, Strahm, Brigitte, von Luettichau, Irene, Hoell, Jessica I., Borkhardt, Arndt, Klingebiel, Thomas, Schrappe, Martin, von Stackelberg, Arend, Glogova, Evgenia, Poetschger, Ulrike, Meisel, Roland, Peters, Christina, Kuhlen, M, Willasch, A, Dalle, J, Wachowiak, J, Yaniv, I, Ifversen, M, Sedlacek, P, Guengoer, T, Lang, P, Bader, P, Sufliarska, S, Balduzzi, A, Strahm, B, von Luettichau, I, Hoell, J, Borkhardt, A, Klingebiel, T, Schrappe, M, von Stackelberg, A, Glogova, E, Poetschger, U, Meisel, R, Peters, C, Kuhlen, Michaela, Willasch, Andre M., Dalle, Jean-Hugues, Wachowiak, Jacek, Yaniv, Isaac, Ifversen, Marianne, Sedlacek, Petr, Guengoer, Tayfun, Lang, Peter, Bader, Peter, Sufliarska, Sabina, Balduzzi, Adriana, Strahm, Brigitte, von Luettichau, Irene, Hoell, Jessica I., Borkhardt, Arndt, Klingebiel, Thomas, Schrappe, Martin, von Stackelberg, Arend, Glogova, Evgenia, Poetschger, Ulrike, Meisel, Roland, and Peters, Christina

Abstract

Relapse remains the major cause of treatment failure in children with high-risk acute lymphoblastic leukaemia (ALL) undergoing allogeneic haematopoietic stem-cell transplantation (allo-SCT). Prognosis is considered dismal but data on risk factors and outcome are lacking from prospective studies. We analysed 242 children with recurrence of ALL after first allo-SCT enrolled in the Berlin-Frankfurt-Munster (BFM) ALL-SCT-BFM 2003 and ALL-SCT-BFM international 2007 studies. Median time from allo-SCT to relapse was 7·7 months; median follow-up from relapse after allo-SCT until last follow-up was 3·4 years. The 3-year event-free survival (EFS) was 15% and overall survival (OS) was 20%. The main cause of death was disease progression or relapse (86·5%). The majority of children (48%) received salvage therapy without second allo-SCT, 26% of the children underwent a second allo-SCT and 25% received palliative treatment only. In multivariate analyses, age, site of relapse, time to relapse and type of salvage therapy were identified as significant prognostic factors for OS and EFS, whereas factors associated with first SCT were not statistically significant. Combined approaches incorporating novel immunotherapeutic treatment options and second allo-SCT hold promise to improve outcome in children with post allo-SCT relapse.

Published
2018

62. Multidrug‐resistant bacterial infections in children undergoing haematopoietic stem cell transplantation over a 6‐year period: analysis of the Polish Pediatric Group for Hematopoietic Stem Cell Transplantation.

Author

Zając‐Spychała, O., Wachowiak, J., Frączkiewicz, J., Salamonowicz, M., Kałwak, K., Gorczyńska, E., Chybicka, A., Czyżewski, K., Dziedzic, M., Wysocki, M., Zalas‐Wiącek, P., Zaucha‐Prażmo, A., Kowalczyk, J.R., Goździk, J., and Styczyński, J.

Subjects
*HEMATOPOIETIC stem cell transplantation, *STEM cell transplantation, *BACTERIAL diseases, *URINARY tract infections, *MULTIDRUG-resistant tuberculosis
Abstract

Aims: Multidrug‐resistant (MDR) bacteria are an emerging cause of morbidity and mortality after haematopoietic stem cell transplantation (HSCT). The aim of the study was to analyse the incidence, clinical characteristics and survival from bacterial infections (BI) caused by MDR pathogens in paediatric HSCT recipients. Methods and Results: Among 971 transplanted patients, BI were found in 416 children between the years 2012 and 2017. Overall, there were 883 bacterial episodes, which includes 85·8% after allo‐HSCT and 14·2% after auto‐HSCT. MDR strains were responsible for half of the total number of bacterial episodes. Over 50% of MDR pathogens were Enterobacteriaceae causing mainly gut infections or urinary tract infections. Conclusions: Regarding HSCT type, we did not find differences in the profile of MDR BI between allo‐ and auto‐HSCT recipients. However, survival in MDR and non‐MDR infections was comparable. Significance and Impact of the Study: The large sample size enables unique analysis and makes our data more applicable to other paediatric HSCT centres. In the absence of local epidemiological data, presented clinical characteristics of MDR‐caused infections may be used to optimize the prophylactic strategies, early identification of infectious complications of MDR aetiology and thus promptly initiate adequate antibiotic therapy and further improve patients' outcome. [ABSTRACT FROM AUTHOR]

Published
2020
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63. 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, 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, Bader, P, Dalle, JH, Macklon, KT, de Heredia Rubio, CD, 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, Bader, P, Dalle, JH, Macklon, KT, and de Heredia Rubio, CD

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

Published
2017

66. Incidence of Infectious Complications in Children With Acute Lymphoblastic Leukemia Treated With Hematopoietic Stem Cell Transplantation

Author

Zaucha-Prażmo, A., primary, Kowalczyk, J.R., additional, Drabko, K., additional, Czyżewski, K., additional, Goździk, J., additional, Zając-Spychała, O., additional, Wachowiak, J., additional, Frączkiewicz, J., additional, Gorczyńska, E., additional, Kałwak, K., additional, and Styczyński, J., additional

Published
2017
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67. Diagnosis and severity criteria for sinusoidal obstruction syndrome/veno-occlusive disease in pediatric patients: a new classification from the European society for blood and marrow transplantation

Author

Corbacioglu, S, primary, Carreras, E, additional, Ansari, M, additional, Balduzzi, A, additional, Cesaro, S, additional, Dalle, J-H, additional, Dignan, F, additional, Gibson, B, additional, Guengoer, T, additional, Gruhn, B, additional, Lankester, A, additional, Locatelli, F, additional, Pagliuca, A, additional, Peters, C, additional, Richardson, P G, additional, Schulz, A S, additional, Sedlacek, P, additional, Stein, J, additional, Sykora, K-W, additional, Toporski, J, additional, Trigoso, E, additional, Vetteranta, K, additional, Wachowiak, J, additional, Wallhult, E, additional, Wynn, R, additional, Yaniv, I, additional, Yesilipek, A, additional, Mohty, M, additional, and Bader, P, additional

Published
2017
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69. Treosulfan-based conditioning regimens for allogeneic HSCT in children with acute lymphoblastic leukaemia

Author

Boztug, H., Zecca, M., Sykora, K.W., Veys, P., Lankester, A., Slatter, M., Skinner, R., Wachowiak, J., Potschger, U., Glogova, E., Peters, C., and EBMT Paediat Dis Working Party

Subjects
Diarrhea, Male, Oncology, medicine.medical_specialty, Transplantation Conditioning, Adolescent, Treosulfan, Vomiting, medicine.medical_treatment, Graft vs Host Disease, Hematopoietic stem cell transplantation, Disease-Free Survival, hemic and lymphatic diseases, Internal medicine, Antineoplastic Combined Chemotherapy Protocols, medicine, Humans, Transplantation, Homologous, Child, Busulfan, Retrospective Studies, Stomatitis, Dose-Response Relationship, Drug, Toxicity, business.industry, Remission Induction, Hematopoietic Stem Cell Transplantation, Stem cell transplantation, Infant, Retrospective cohort study, Paediatrics, Hematology, General Medicine, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Total body irradiation, Surgery, Transplantation, Treatment Outcome, surgical procedures, operative, Child, Preschool, Multivariate Analysis, Female, business, ALL, medicine.drug
Abstract

Standard myeloablative conditioning regimens for children with acute lymphoblastic leukaemia are based on total body irradiation (TBI). However, TBI causes profound short-term and long-term side effects, provoking the necessity for alternative regimens. Treosulfan combines a potent immunosuppressive and antileukaemic effect with myeloablative activity and low toxicity profile. We retrospectively studied toxicity and outcome of 71 paediatric patients with acute lymphoblastic leukaemia (ALL) undergoing haematopoietic stem cell transplantation (HSCT) following treosulfan-based conditioning aiming to identify risk factors for treatment failure and dose-depending outcome differences. Early regimen-related toxicity was low. No case of veno-occlusive disease was reported. There was no association of toxicity with age or number of HSCT. Event-free survival (EFS) of infants was significantly better compared to older children. Overall survival (OS) at 3 years was 51 % and not significantly influenced by number of HSCT (first HSCT 54 %, ≥second HSCT 44 %, p = 0.71). In multivariate analysis, OS and EFS were significantly worse for patients transplanted without complete remission (p = 0.04 and 0.004). Treatment-related mortality was low at 14 %. We conclude that treosulfan-based conditioning is a safe and efficacious approach for paediatric ALL.

Published
2015

73. Bacterial infections in pediatric hematopoietic stem cell transplantation recipients: incidence, epidemiology, and spectrum of pathogens: report of the Polish Pediatric Group for Hematopoietic Stem Cell Transplantation

Author

Zając‐Spychała, O., primary, Wachowiak, J., additional, Pieczonka, A., additional, Siewiera, K., additional, Frączkiewicz, J., additional, Kałwak, K., additional, Gorczyńska, E., additional, Chybicka, A., additional, Czyżewski, K., additional, Jachna‐Sawicka, K., additional, Wysocki, M., additional, Klepacka, J., additional, Goździk, J., additional, Zaucha‐Prażmo, A., additional, Kowalczyk, J.R., additional, and Styczyński, J., additional

Published
2016
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74. Recommendations on hematopoietic stem cell transplantation for inherited bone marrow failure syndromes

Author

de Latour, R. Peffault, Peters, C., Gibsons, B., Strahm, B., Lankester, A., de Heredia, C. D., Longoni, D., Fioredda, F., Locatelli, F., Yaniv, I., Wachowiak, J., Donadieu, J., Lawitschka, A., Bierings, M., Wlodarski, M., Corbacioglu, S., Bonanomi, S., Samarasinghe, S., Leblanc, T., Dufour, C., Dalle, J-H, Pediat Working Party PDWP, European Grp Blood Marrow, de Latour, R. Peffault, Peters, C., Gibsons, B., Strahm, B., Lankester, A., de Heredia, C. D., Longoni, D., Fioredda, F., Locatelli, F., Yaniv, I., Wachowiak, J., Donadieu, J., Lawitschka, A., Bierings, M., Wlodarski, M., Corbacioglu, S., Bonanomi, S., Samarasinghe, S., Leblanc, T., Dufour, C., Dalle, J-H, Pediat Working Party PDWP, and European Grp Blood Marrow

Published
2015

75. Paediatric reduced intensity conditioning : analysis of centre strategies on regimens and definitions by the EBMT Paediatric Diseases and Complications and Quality of Life WP

Author

Lawitschka, A., Faraci, M., Yaniv, I., Veys, P., Bader, P., Wachowiak, J., Socie, G., Aljurf, M. D., Arat, M., Boelens, J. J., Duarte, R., Tichelli, A., Peters, C., Lawitschka, A., Faraci, M., Yaniv, I., Veys, P., Bader, P., Wachowiak, J., Socie, G., Aljurf, M. D., Arat, M., Boelens, J. J., Duarte, R., Tichelli, A., and Peters, C.

Published
2015

76. Recommendations on hematopoietic stem cell transplantation for inherited bone marrow failure syndromes

Author

Haematologie, Child Health, Regenerative Medicine and Stem Cells, de Latour, R. Peffault, Peters, C., Gibsons, B., Strahm, B., Lankester, A., de Heredia, C. D., Longoni, D., Fioredda, F., Locatelli, F., Yaniv, I., Wachowiak, J., Donadieu, J., Lawitschka, A., Bierings, M., Wlodarski, M., Corbacioglu, S., Bonanomi, S., Samarasinghe, S., Leblanc, T., Dufour, C., Dalle, J-H, Pediat Working Party PDWP, European Grp Blood Marrow, Haematologie, Child Health, Regenerative Medicine and Stem Cells, de Latour, R. Peffault, Peters, C., Gibsons, B., Strahm, B., Lankester, A., de Heredia, C. D., Longoni, D., Fioredda, F., Locatelli, F., Yaniv, I., Wachowiak, J., Donadieu, J., Lawitschka, A., Bierings, M., Wlodarski, M., Corbacioglu, S., Bonanomi, S., Samarasinghe, S., Leblanc, T., Dufour, C., Dalle, J-H, Pediat Working Party PDWP, and European Grp Blood Marrow

Published
2015

77. Paediatric reduced intensity conditioning: analysis of centre strategies on regimens and definitions by the EBMT Paediatric Diseases and Complications and Quality of Life WP

Author

Child Health, Infection & Immunity, SCT onderzoek, Lawitschka, A., Faraci, M., Yaniv, I., Veys, P., Bader, P., Wachowiak, J., Socie, G., Aljurf, M. D., Arat, M., Boelens, J. J., Duarte, R., Tichelli, A., Peters, C., Child Health, Infection & Immunity, SCT onderzoek, Lawitschka, A., Faraci, M., Yaniv, I., Veys, P., Bader, P., Wachowiak, J., Socie, G., Aljurf, M. D., Arat, M., Boelens, J. J., Duarte, R., Tichelli, A., and Peters, C.

Published
2015

78. Recommendations on hematopoietic stem cell transplantation for inherited bone marrow failure syndromes

Author

Peffault De Latour, R., Peters, C., Gibson, B., Strahm, B., Lankester, A., De Heredia, C. D., Longoni, D., Fioredda, F., Locatelli, Franco, Yaniv, I., Wachowiak, J., Donadieu, J., Lawitschka, A., Bierings, M., Wlodarski, M., Corbacioglu, S., Bonanomi, S., Samarasinghe, S., Leblanc, T., Dufour, C., Dalle, J. -H., Locatelli F. (ORCID:0000-0002-7976-3654), Peffault De Latour, R., Peters, C., Gibson, B., Strahm, B., Lankester, A., De Heredia, C. D., Longoni, D., Fioredda, F., Locatelli, Franco, Yaniv, I., Wachowiak, J., Donadieu, J., Lawitschka, A., Bierings, M., Wlodarski, M., Corbacioglu, S., Bonanomi, S., Samarasinghe, S., Leblanc, T., Dufour, C., Dalle, J. -H., and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) offers the potential to cure patients with an inherited bone marrow failure syndrome (IBMFS). However, the procedure involves the risk of treatment-related mortality and may be associated with significant early and late morbidity. For these reasons, the benefits should be carefully weighed against the risks. IBMFS are rare, whereas case reports and small series in the literature illustrate highly heterogeneous practices in terms of indications for HSCT, timing, stem cell source and conditioning regimens. A consensus meeting was therefore held in Vienna in September 2012 on behalf of the European Group for Blood and Marrow Transplantation to discuss HSCT in the setting of IBMFS. This report summarizes the recommendations from this expert panel, including indications for HSCT, timing, stem cell source and conditioning regimen.

Published
2015

79. Is FLT3 Interal Tandem Duplication an Unfavorable Risk Factor for High Risk Children with Acute Myeloid Leukemia?- Polish Experience

Author

Pawińska-Wąsikowska, Katarzyna, Książek, Teofila, Wieczorek, Aleksandra, Matysiak, M., Fic-Sikorska, B., Adamkiewicz-Drożyńska, Elżbieta, Maciejka-Kapuscinska, L., Chybicka, A., Potocka, K., Wachowiak, J., Skalska-Sadowska, J., Kowalczyk, J., Wojcik, B., Wysocki, M., Koltan, S., Krawczuk-Rybak, Maryna, Muszynska-Roslan, K., Mlynarski, W., Stolarska, M., Urasinski, T., Kamienska, E., Szczepanski, T., Tomaszewska, R., Sobol-Milejska, G., Mizia-Malarz, A., Karolczyk, G., Pohorecka, J., Wieczorek, M., Karpińska-Derda, Irena, and Balwierz, Walentyna

Subjects
FLT3 Internal Tandem Duplication, Oncology, medicine.medical_specialty, business.industry, Myeloid leukemia, hemic and immune systems, treatment result, acute myeloid leukemia, high risk group, body regions, children, hemic and lymphatic diseases, Internal medicine, embryonic structures, FLT3/ITD mutation, Medicine, Risk factor, business, psychological phenomena and processes
Abstract

According to the AML-BFM 2004 Interim, a treatment protocol used in Poland since 2005, presence of FLT3 internal tandem duplication (FLT3/ITD) qualifies a patient with acute myeloid leukemia (AML) to a high-risk group (HRG). The present study was aimed to identify the prevalence of FLT3/ITD in children with AML in Poland and to evaluate its prognostic significance in the HRG patients. Out of 291 children with de novo AML treated in 14 Polish centers between January 2006 and December 2012, samples from 174 patients were available for FLT3/ITD analysis. Among study patients 108 children (61.7%) were qualified to HRG. Genomic DNA samples from bone marrow were tested for identification of FLT3/ITD mutation by PCR amplification of exon 14 and 15 of FLT3 gene. Clinical features and treatment outcome in patients with and without FLT3/ITD were analyzed in the study. The FLT3/ITD was found in 14 (12.9%) of 108 HRG children. There were no significant differences between children with and without FLT3/ITD in age and FAB distribution. The white blood cells count in peripheral blood at diagnosis was significantly higher (p

Published
2013

80. Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood?

Author

Fernandes, Jf, Rocha, V, Labopin, M, Neven, B, Moshous, D, Gennery, Ar, Friedrich, W, Porta, F, Diaz de Heredia, C, Wall, D, Bertrand, Y, Veys, P, Slatter, M, Schulz, A, Chan, Kw, Grimley, M, Ayas, M, Gungor, T, Ebell, W, Bonfim, C, Kalwak, K, Taupin, P, Blanche, S, Gaspar, Hb, Landais, P, Fischer, A, Gluckman, E, Cavazzana Calvo, M, Eurocord, Inborn Errors Working Party of European Group for Blood, Marrow Transplantation: Ahmed, A, Auiti, A, Biffi, A, Cant, A, Fasth, A, Gennery, A, Hassan, A, Lankester, A, O'Mera, A, Plabani, A, Rovelli, A, Salmon, A, Scarselli, A, Thrasher, A, Van Royen, A, Villa, A, Wawer, A, Wahadneh, A, Worth, A, Belohradsky, B, Wolska, B, Gaspar, B, Bonfirm, C, Booth, C, Klein, C, Messina, C, Peters, C, Steward, C, Lindemans, C, Schuetz, C, de Heredia Rubio CD, Bensoussan, D, Gleadow, D, Lilic, D, Gambineri, Eleonora, Smith, E, Aerts, F, Caracseghi, F, Roberts, G, Davies, G, Al Mousa, H, Jossanc, H, Ozsahim, H, Hirsch, I, Meyts, I, Tezcan, I, Mueller, I, Andresc, I, Boelens, J, Fernandes, J, Folloni, J, Keuhl, J, Reichenbach, J, Stary, J, Wachowiak, J, Xu Bayford, J, Cunha, Jm, Ehlert, J, Rao, K, Sykora, K, Andais, L, Brown, L, Dal Cortivo, L, Griffith, L, Notarangelo, L, Abinun, M, Albert, M, Bierings, M, Bouchet, M, Cavazzana, M, Hirschfield, M, Cowan, M, Hoenig, M, Loubser, M, Roncarolo, M, Sauer, M, Schneider, M, Verstegen, M, Schroeder, M, Essink, M, Yesilipek, M, Entz Werle, N, Mahlaoui, N, Schlautmann, N, Taylor, N, Vanroyen, N, Walffraat, N, Sanal, O, Amrolia, P, Bordigoni, P, De Coppi, P, Frange, P, Orchard, P, Sedlacek, P, Shaw, P, Stephensky, P, Bacchetta, R, Bredius, R, Formankova, R, Gale, R, Seger, R, Wynn, R, Corbacioglu, S, Ehl, S, Hacein Bey, S, Hambleton, S, Mohsen, S, Mueller, S, Pai, Sy, Espanol, T, Flood, T, Guengoer, T, Bordon, V, Ormoor, V, Pashano, V, Courteille, V, Czogala, W, Qasim, W, Camci, Y, and Nademi, Z.

Subjects
Male, medicine.medical_specialty, Transplantation Conditioning, medicine.medical_treatment, Immunology, Graft vs Host Disease, Kaplan-Meier Estimate, Cord Blood Stem Cell Transplantation, Hematopoietic stem cell transplantation, Biochemistry, Gastroenterology, SCID HSCT, Internal medicine, medicine, Humans, Proportional Hazards Models, Retrospective Studies, Preparative Regimen, Severe combined immunodeficiency, business.industry, Umbilical Cord Blood Transplantation, Incidence, Hematopoietic Stem Cell Transplantation, Infant, Newborn, Infant, Cell Biology, Hematology, medicine.disease, Surgery, Transplantation, Treatment Outcome, Child, Preschool, Histocompatibility, Cord blood, Female, Severe Combined Immunodeficiency, business
Abstract

Pediatric patients with SCID constitute medical emergencies. In the absence of an HLA-identical hematopoietic stem cell (HSC) donor, mismatched related-donor transplantation (MMRDT) or unrelated-donor umbilical cord blood transplantation (UCBT) are valuable treatment options. To help transplantation centers choose the best treatment option, we retrospectively compared outcomes after 175 MMRDTs and 74 UCBTs in patients with SCID or Omenn syndrome. Median follow-up time was 83 months and 58 months for UCBT and MMRDT, respectively. Most UCB recipients received a myeloablative conditioning regimen; most MMRDT recipients did not. UCB recipients presented a higher frequency of complete donor chimerism (P = .04) and faster total lymphocyte count recovery (P = .04) without any statistically significance with the preparative regimen they received. The MMRDT and UCBT groups did not differ in terms of T-cell engraftment, CD4+ and CD3+ cell recoveries, while Ig replacement therapy was discontinued sooner after UCBT (adjusted P = .02). There was a trend toward a greater incidence of grades II-IV acute GVHD (P = .06) and more chronic GVHD (P = .03) after UCBT. The estimated 5-year overall survival rates were 62% ± 4% after MMRDT and 57% ± 6% after UCBT. For children with SCID and no HLA-identical sibling donor, both UCBT and MMRDT represent available HSC sources for transplantation with quite similar outcomes.

Published
2012

83. Ilościowe oddziaływanie aktywujących receptorów KIR z cząsteczkami HLA dawcy w transplantacjach krwiotwórczych komórek macierzystych u chorych z nowotworami

Author

Nowak, J., primary, Kościńska, K., additional, Mika-Witkowska, R., additional, Rogatko-Koroś, M., additional, Jaskuła, E., additional, Mordak-Domagała, M., additional, Lange, J., additional, Gronkowska, A., additional, Jędrzejczak, W.W., additional, Kyrcz-Krzemień, S., additional, Markiewicz, M., additional, Dzierżak-Mietła, M., additional, Tomaszewska, A., additional, Nasiłowska-Adamska, B., additional, Szczepiński, A., additional, Hałaburda, K., additional, Hellmann, A., additional, Czyż, A., additional, Gil, L., additional, Komarnicki, M., additional, Wachowiak, J., additional, Barańska, M., additional, Kowalczyk, J., additional, Drabko, K., additional, Goździk, J., additional, Wysoczańska, B., additional, Bogunia-Kubik, K., additional, Graczyk-Pol, E., additional, Witkowska, A., additional, Marosz-Rudnicka, A., additional, Nestorowicz, K., additional, Dziopa, J., additional, Szlendak, U., additional, Warzocha, K., additional, and Lange, A., additional

Published
2015
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87. Korzystniejsze wyniki przeszczepienia szpiku w zespołach mielodysplastycznych i młodzieńczej białaczce mielomonocytowej u dzieci przeszczepianych przed 6. miesiącem od rozpoznania – analiza retrospektywna Polskiej Grupy Pediatrycznej

Author

Ussowicz, M., primary, Musiał, J., additional, Drabko, K., additional, Zaucha-Prażmo, A., additional, PIeczonka, A., additional, Wachowiak, J., additional, Styczyński, J., additional, Gorczyńska, E., additional, Kałwak, K., additional, Gil, L., additional, and Chybicka, A., additional

Published
2015
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88. „License to kill” – hamujące receptory KIR mogą zwiększać zdolność komórek NK do niszczenia komórek nowotworu w odpowiednim środowisku HLA klasy I u biorców przeszczepu krwiotwórczych komórek macierzystych

Author

Nowak, J., primary, Kościńska, K., additional, Mika-Witkowska, R., additional, Rogatko-Koroś, M., additional, Jaskuła, E., additional, Mordak-Domagała, M., additional, Lange, J., additional, Gronkowska, A., additional, Jędrzejczak, W.W., additional, Kyrcz-Krzemień, S., additional, Markiewicz, M., additional, Dzierżak-Mietła, M., additional, Tomaszewska, A., additional, Nasiłowska-Adamska, B., additional, Szczepiński, A., additional, Hałaburda, K., additional, Hellmann, A., additional, Komarnicki, M., additional, Gil, L., additional, Czyż, A., additional, Wachowiak, J., additional, Barańska, M., additional, Kowalczyk, J., additional, Drabko, K., additional, Goździk, J., additional, Bogunia-Kubik, K., additional, Wysoczańska, B., additional, Graczyk-Pol, E., additional, Witkowska, A., additional, Marosz-Rudnicka, A., additional, Nestorowicz, K., additional, Dziopa, J., additional, Szlendak, U., additional, Warzocha, K., additional, and Lange, A., additional

Published
2015
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89. Wyniki leczenia młodzieży z ostrą białaczką szpikową wg protokołu AML-BFM 2004 Interim w ośrodkach Polskiej Grupy Pediatrycznej ds Leczenia Białaczek i Chłoniaków

Author

Balwierz, W., primary, Czogała, M., additional, Pawińska-Wąsikowska, K., additional, Badowska, W., additional, Balcerska, A., additional, Chybicka, A., additional, Kamieńska, E., additional, Karpińska-Derda, I., additional, Kołtan, S., additional, Kowalczyk, J., additional, Krawczuk-Rybak, M., additional, Maciejka-Kembłowska, L., additional, Matysiak, M., additional, Mizia-Malarz, A., additional, Młynarski, W., additional, Muszyńska-Rosłan, K., additional, Pohorecka, J., additional, Potocka, K., additional, Sikorska–Fic, B., additional, Skalska-Sadowska, J., additional, Sobol-Milejska, G., additional, Stolarska, M., additional, Szczepański, T., additional, Tomaszewska, R., additional, Urasiński, T., additional, Wachowiak, J., additional, Wiśniewska-Ślusarz, H., additional, and Wysocki, M., additional

Published
2015
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90. Long-term outcome following hematopoietic stem cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and the European Group for Blood and Marrow Transplantation

Author

Ozsahin, H, CAVAZZANA CALVO, M, Notarangelo, Luigi Daniele, Schulz, A, Thrasher, Aj, Mazzolari, Evelina, Slatter, Ma, LE DEIST, F, Blanche, S, Veys, P, Fasth, A, Bredius, R, Sedlacek, P, Wulfraat, N, Ortega, J, Heilmann, C, O'Meara, A, Wachowiak, J, Kalwak, K, MATTHES MARTIN, S, Gungor, T, Ikinciogullari, A, Landais, P, Cant, Aj, Friederich, W, and Fisher, A.

Published
2007

93. Influence of Age on Treatment Results in Children and Adolescence with Hodgkin's Lymphoma Polish Experience

Author

Balwierz, W, primary, Klekawka, T, additional, Moryl-Bujakowska, A, additional, Matysiak, M, additional, Malinowska, I, additional, Chybicka, A, additional, Chaber, R, additional, Szczepanski, T, additional, Janik-Moszant, A, additional, Wachowiak, J, additional, Wziatek, A, additional, Kowalczyk, J, additional, Mitura-Lesiuk, M, additional, Adamkiewicz-Drozynska, E, additional, Stachowicz-Stencel, T, additional, Wysocki, M, additional, Koltan, A, additional, Krawczuk-Rybak, M, additional, Muszynska-Roslan, K, additional, Mlynarski, W, additional, Stolarska, M, additional, Sobol, G, additional, Wieczorek, M, additional, Piatek, T, additional, Karolczyk, G, additional, Dadela-Urbanek, A, additional, Urasinski, T, additional, Kamienska, E, additional, and Dzikowska, K, additional

Published
2014
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94. Marrow versus peripheral blood for geno-identical allogeneic stem cell transplantation in acute myelocytic leukemia: Influence of dose and stem cell source shows better outcome with rich marrow

Author

Gorin N.C., Labopin M., Rocha V., Arcese W., Beksac M., Gluckman E., Ringden O., Ruutu T., Reiffers J., Bandini G., Falda M., Zikos P., Willemze R., Frassoni F., Abecasis M., Abráhamová J., Afanassiev B.V., Aglietta M., Alabdulaaly A., Aleinikova O., Paolo Alessandrino E., Al Shemmari S.H., Amadori D., Amadori S., Amos T., Andolina M., Andreesen R., Angelucci E., Anhuf J., Arnold R., Arpaci F., Attal M., Azevedo W., Azim H.A., Baccarani M., Bacigalupo A., Barbui T., Bargetzi M., Barnard D.L., Bartsch H.H., Baruchel A., Battista C., Bay J.-O., Bayik M., Bazarbachi A., Beguin Y., López J.L.B., Benedek I., Benedetti F., Bengala C., Berrebi A., Besalduch J., Biesma D., Biron P., Björkholm M., Blaise D., Blesing N.E., Boasson M., Bobev D., Boccadoro M., Bolaman Z., Boogaerts M.A., Bordessoule D., Bosi A., Aida B.S., Bourhis J.H., Bourikas G., Bowen D.T., Bregni M., Bries G., Brinch L., Brittain D., Bron D., Brune M., Bullorsky E.O., Bunjes D., Burdach S., Burnett A.K., Buzyn A., Caballero D., Cagirgan S., Cahn J.-Y., Canepa C.O., Cao A., Carella A.M., Carrera F.D., Carret A.-S., Cascinu S., Castel V., Caswell M., Cavanna L., Cetto G.L., Chapuis B., Chasty R., Chen Y.-C., Chisesi T., Chopra R., Chybicka A., Clark R.E., Colombat P., Colovic M.D., Constenla-Figueiras M., Contreras M., Contu L., Cordonnier C., Cornelissen J.J., Cornish J., Coser P., Costa N., Coze C., Craddock C., Crown J., Culligan D.J., Danova M., Darbyshire P.J., Davies J.M., de Bock R., de Pablos Gallego J.M., De Prijck B., de Revel T., De Rossi G., De Souza C.A., Deb G., Degos L., Demuynck H., Dervenoulas I., Di Bartolomeo P., Di Renzo N., Diaz M.A., Diehl V., Diez-Martin J.L., Dincer S., Giorgio D., Dmoszynska A., Doelken G., Peter P.D., Dulley F., Easow J., Ebell W., Efremidis A., Ehninger G., Eichler H., Eimermacher H., Enno A., Errazquin L., Aguado J.E., Everaus H., Fagioli F., Fanin R., Fassas A., Fasth A., Faulkner L.B., Fauser A.A., Feldman L., Feremans W., Ferhanoglu B., Fernández M.N., Fernández-Ranada J.M., Ferrant A., Ferrara F., Finke J., Fischer A., Fischer J., Fitzsimons T., Floristan F., Forjaz de Lacerda J.M.F., Fossati-Bellani F., Fosser V., Franklin I., Freund M., Frickhofen N., Gabbas A., Gadner H., Gallamini A., Galvin M.C., López J.G., García-Conde J., Gaska T., Gastl G., Gedikoglu G., Ghavamzadeh A., Gianni A., Gibson B.E., Gil J.L., Gilleece M.H., Gisselbrecht C., Glass B., Gmür J., Göbel U., Goldman J.M., Goldstone A.H., San Miguel J.D.G., González-López M.-A., Grafakos S., Gramatzki M., Grañena A., Gratecos N., Gratwohl A., Greinix H.T., Gugliotta L., Guilhot F., Guimaraes J.E., Gülbas Z., Gulyuz O., Gurman G., Gutierrez M.M., Haas R., Hamladji R.-M., Hamon M.D., Hansen N.E., Harhalakis N., Harousseau J.L., Hartenstein R., Hartmann C.O., Hausmaninger H., Haznedar R., Heit W., Hellmann A., Herrmann R.P., Hertenstein B., Hess U., Hinterberger W., Ho A.D., Hoelzer D., Holowiecki J., Horst H.-A., Hossfeld D.K., Huebsch L., Hunter A.E., Iacopino P., Iannitto E., Indrák K., Iriondo A., Izzi T., Jackson G.L., Jacobs P., Jacobsen N., Janvier M., Jebavy L., Joensuu H., Joerg S., Jones F.G.C., Jouet J.P., Joyner M.V., Juliusson G., Jürgens H., Kalayoglu-Besisik S., Kalman N., Kalmanti M., Kansoy S., Kansu E., Kanz L., Karianakis G., Kernéis Y., Khalifeh O., Khomenko V., Kienast J., Killick S., Kirchner H.H., Klingebiel T., Knauf W., Koenigsmann M., Koistinen P., Koivunen E., Kolb H.-J., Kolbe K., Koller E., Komarnicki M., Koscielniak E., Kovacsovics T., Kowalczyk J.R., Koza V., Kozak T., Kugler J., Kuliczkowski K., Kvaloy S., Labar B., Laciura P., Palacios J.J.L., Lakota J., Lambertenghi D.G., Lange A., Lanza F., Isasti R.L., Lauria F., Le Moine F., Leblond V., Lelli G., Lenhoff S., Leon L.A., Leoncini-Franscini L., Leone G., Leoni P., Levis A., Leyvraz S., Liberati M., Link H., Linkesch W., Liso V., Lisukov I.A., Littlewood T., Ljungman P., Locatelli F., Losonczy H., Lotz J.-P., Ludwig H., Lukac J., Lutz D., Macchia P., Madrigal A., Maiolino A., Majolino I., Eloy-García J.M., Malesevic M., Mandelli F., Marc A., Marcus R., Marianska B., Markuljak I., Marsh J.C.W., Martelli M.F., Marti Tutusaus J.M., Martin S., Martin M., Martinelli G., Martínez-Rubio A.M., Martoni A., Maschan A., Maschmeyer G., Masszi T., Mazza P., McCann S., Meier C.R., Messina C., Mettivier V., Metzner B., Michallet M., Michieli M., Michon J., Milligan D.W., Milone J.H., Giuseppe G.M., Minigo H., Mistrik M., Moicean A.D., Monfardini S., Montserrat E., Moraleda Jimenez J.M., Morales-Lazaro A., Morandi S., Morra E., Mufti G.J., Musso M., Nagler A., Nalli G., Naparstek E., Narni F., Nenadov-Beck M., Neubauer A., Newland A.C., Niederwieser D., Niethammer D., Noens L.A., Nousiainen T., Novik A., Novitzky N., Occhini D., Odriozolas J., Ojanguren J.M., O’meara A., Onat H., Orchard K., Ortega J.J., Osieka R., Ossenkoppele G.J., Othman B., Ovali E., Ozcebe O.I., Ozerkan K., Ozturk A., Papatryfonos A., Parker J.E., Pastore M., Patrone F., Patton N., Pejin D., Peñarrubia M.J., Equiza E.P., Peschel C., Pession A., Pigaditou A., Pignon B., Pihkala U., Pimentel P., Pitini V., Podoltseva E., Pogliani E.M., Anna A.P., Porta F., Potter M., Powles R., Prentice G.H., Pretnar J., Ptushkin V., Quarta G., Reiter A., Remes K., Reykdal S., Santasusana J.M.R., Rifón J., Rio B., Rizzoli V., Robak T., Robinson A.J., Rodeghiero F., Rodríguez Fernández J.M., Rombos Y., Romeril K.R., Rosenmayr A., Rossi J.F., Rosti G., Rotoli B., Rowe J.M., Russell N.H., Ryzhak O., Rzepecki P., Saglio G., Salwender H., Samonigg H., Santoro A., Sanz M.A., Sayer H.G., Scanni A., Schaafsma M.R., Schaefer U.W., Schanz U., Schattenberg A., Schey S.A.M., Schlimok G., Schmoll H.-J., Schots R., Schouten H., Schwarer A.P., Schwerdtfeger R., Scimè R., Segel E., Seger R., Selleslag D., Serban M., Shamaa S., Shaw P.J., Siegert W., Siena S., Sierra J., Simonsson B., Singer C.R.J., Sirchia G., Skotnicki A.B., Slavin S., Snowden J., Sotto J.J., Tanyeli A., Tedeschi L., Tidefelt U., Tissot J.-D., Tobler A., Tomas J.F., Torres J.P., Torres G.A., Touraine J.-L., Trneny M., Uderzo C., Unal E., Unal A., Undar L., Urban C., Van den Berg H., van Marwijk K.M., Vellenga E., Venturini M., Verdonck L.F., Veys P., Vilardell J., Vinante O., Visani G., Vitek A., Vivancos P., Volpe E., Vora A., Vorlicek J., Vowels M., Vujic D., Wachowiak J., Wagner T., Wahlin A., Walewski J., Wandt H., Weissinger F., Wijermans P.W., Wiktor-Jedrzejczak W., Will A.M., Woell E., Wörmann B., Yaniv I., Yesilipek M.A., Yilmaz U., Yong A., Zachée P., Zambelli A., Zander A.R., Zintl F., Zoumbos N.C., Çukurova Üniversitesi, Maltepe Üniversitesi, and Ege Üniversitesi

Subjects
Myeloid, Male, Pathology, Time Factors, Graft vs Host Disease, Biochemistry, Gastroenterology, Blood cell, Bone Marrow, Child, Bone Marrow Transplantation, Leukemia, Remission Induction, Hematology, Middle Aged, Prognosis, Leukemia, Myeloid, Acute, ComputingMilieux_MANAGEMENTOFCOMPUTINGANDINFORMATIONSYSTEMS, medicine.anatomical_structure, Treatment Outcome, Child, Preschool, Female, Stem cell, InformationSystems_MISCELLANEOUS, Homologous, Adult, medicine.medical_specialty, Adolescent, Immunology, Acute, Disease-Free Survival, Internal medicine, medicine, Transplantation, Homologous, Humans, Preschool, Aged, Transplantation, business.industry, ComputerSystemsOrganization_COMPUTER-COMMUNICATIONNETWORKS, Cell Biology, medicine.disease, Peripheral blood, Histocompatibility, Multivariate Analysis, Stem Cell Transplantation, ComputingMethodologies_PATTERNRECOGNITION, Myelocytic leukemia, Bone marrow, business, Settore MED/15 - Malattie del Sangue
Abstract

PubMed ID: 12829583, Several studies have compared bone marrow (BM) and peripheral blood (PB) as stem cell sources in patients receiving allografts, but the cell doses infused have not been considered, especially for BM. Using the ALWP/EBMT registry, we retrospectively studied 881 adult patients with acute myelocytic leukemia (AML), who received a non-T-depleted allogeneic BM (n = 515) or mobilized PB (n = 366) standard transplant, in first remission (CR1), from an HLA-identical sibling, over a 5-year period from January 1994. The BM cell dose ranged from 0.17 to 29 × 10 8 /kg with a median of 2.7 × 10 8 /kg. The PB cell dose ranged from 0.02 to 77 × 10 8 /kg with a median of 9.3 × 10 8 /kg. The median dose for patients receiving BM (2.7 × 10 8 /kg) gave the greatest discrimination. In multivariate analyses, high-dose BM compared to PB was associated with lower transplant-related mortality (RR = 0.61; 95% CI, 0.39-0.98; P = .04), better leukemia-free survival (RR = 0.65; 95% CI, 0.46-0.91; P = .013), and better overall survival (RR = 0.64; 95% CI, 0.44-0. 92; P = .016). The present study in patients with AML receiving allografts in first remission indicates a better outcome with BM as compared to PB, when the dose of BM infused is rich. © 2003 by The American Society of Hematology.

Published
2003

95. Marrow versus peripheral blood for geno-identical allogeneic stem cell transplantation in acute myelocytic leukemia: Influence of dose and stem cell source shows better outcome with rich marrow

Author

Gorin, N.C. Labopin, M. Rocha, V. Arcese, W. Beksac, M. Gluckman, E. Ringden, O. Ruutu, T. Reiffers, J. Bandini, G. Falda, M. Zikos, P. Willemze, R. Frassoni, F. Abecasis, M. Abráhamová, J. Afanassiev, B.V. Aglietta, M. Alabdulaaly, A. Aleinikova, O. Paolo Alessandrino, E. Al Shemmari, S.H. Amadori, D. Amadori, S. Amos, T. Andolina, M. Andreesen, R. Angelucci, E. Anhuf, J. Arnold, R. Arpaci, F. Attal, M. Azevedo, W. Azim, H.A. Baccarani, M. Bacigalupo, A. Barbui, T. Bargetzi, M. Barnard, D.L. Bartsch, H.H. Baruchel, A. Battista, C. Bay, J.-O. Bayik, M. Bazarbachi, A. Beguin, Y. López, J.L.B. Benedek, I. Benedetti, F. Bengala, C. Berrebi, A. Besalduch, J. Biesma, D. Biron, P. Björkholm, M. Blaise, D. Blesing, N.E. Boasson, M. Bobev, D. Boccadoro, M. Bolaman, Z. Boogaerts, M.A. Bordessoule, D. Bosi, A. Aida, B.S. Bourhis, J.H. Bourikas, G. Bowen, D.T. Bregni, M. Bries, G. Brinch, L. Brittain, D. Bron, D. Brune, M. Bullorsky, E.O. Bunjes, D. Burdach, S. Burnett, A.K. Buzyn, A. Caballero, D. Cagirgan, S. Cahn, J.-Y. Canepa, C.O. Cao, A. Carella, A.M. Carrera, F.D. Carret, A.-S. Cascinu, S. Castel, V. Caswell, M. Cavanna, L. Cetto, G.L. Chapuis, B. Chasty, R. Chen, Y.-C. Chisesi, T. Chopra, R. Chybicka, A. Clark, R.E. Colombat, P. Colovic, M.D. Constenla-Figueiras, M. Contreras, M. Contu, L. Cordonnier, C. Cornelissen, J.J. Cornish, J. Coser, P. Costa, N. Coze, C. Craddock, C. Crown, J. Culligan, D.J. Danova, M. Darbyshire, P.J. Davies, J.M. de Bock, R. de Pablos Gallego, J.M. De Prijck, B. de Revel, T. De Rossi, G. De Souza, C.A. Deb, G. Degos, L. Demuynck, H. Dervenoulas, I. Di Bartolomeo, P. Di Renzo, N. Diaz, M.A. Diehl, V. Diez-Martin, J.L. Dincer, S. Giorgio, D. Dmoszynska, A. Doelken, G. Peter, P.D. Dulley, F. Easow, J. Ebell, W. Efremidis, A. Ehninger, G. Eichler, H. Eimermacher, H. Enno, A. Errazquin, L. Aguado, J.E. Everaus, H. Fagioli, F. Fanin, R. Fassas, A. Fasth, A. Faulkner, L.B. Fauser, A.A. Feldman, L. Feremans, W. Ferhanoglu, B. Fernández, M.N. Fernández-Ranada, J.M. Ferrant, A. Ferrara, F. Finke, J. Fischer, A. Fischer, J. Fitzsimons, T. Floristan, F. Forjaz de Lacerda, J.M.F. Fossati-Bellani, F. Fosser, V. Franklin, I. Freund, M. Frickhofen, N. Gabbas, A. Gadner, H. Gallamini, A. Galvin, M.C. López, J.G. García-Conde, J. Gaska, T. Gastl, G. Gedikoglu, G. Ghavamzadeh, A. Gianni, A. Gibson, B.E. Gil, J.L. Gilleece, M.H. Gisselbrecht, C. Glass, B. Gmür, J. Göbel, U. Goldman, J.M. Goldstone, A.H. San Miguel, J.D.G. González-López, M.-A. Grafakos, S. Gramatzki, M. Grañena, A. Gratecos, N. Gratwohl, A. Greinix, H.T. Gugliotta, L. Guilhot, F. Guimaraes, J.E. Gülbas, Z. Gulyuz, O. Gurman, G. Gutierrez, M.M. Haas, R. Hamladji, R.-M. Hamon, M.D. Hansen, N.E. Harhalakis, N. Harousseau, J.L. Hartenstein, R. Hartmann, C.O. Hausmaninger, H. Haznedar, R. Heit, W. Hellmann, A. Herrmann, R.P. Hertenstein, B. Hess, U. Hinterberger, W. Ho, A.D. Hoelzer, D. Holowiecki, J. Horst, H.-A. Hossfeld, D.K. Huebsch, L. Hunter, A.E. Iacopino, P. Iannitto, E. Indrák, K. Iriondo, A. Izzi, T. Jackson, G.L. Jacobs, P. Jacobsen, N. Janvier, M. Jebavy, L. Joensuu, H. Joerg, S. Jones, F.G.C. Jouet, J.P. Joyner, M.V. Juliusson, G. Jürgens, H. Kalayoglu-Besisik, S. Kalman, N. Kalmanti, M. Kansoy, S. Kansu, E. Kanz, L. Karianakis, G. Kernéis, Y. Khalifeh, O. Khomenko, V. Kienast, J. Killick, S. Kirchner, H.H. Klingebiel, T. Knauf, W. Koenigsmann, M. Koistinen, P. Koivunen, E. Kolb, H.-J. Kolbe, K. Koller, E. Komarnicki, M. Koscielniak, E. Kovacsovics, T. Kowalczyk, J.R. Koza, V. Kozak, T. Kugler, J. Kuliczkowski, K. Kvaloy, S. Labar, B. Laciura, P. Palacios, J.J.L. Lakota, J. Lambertenghi, D.G. Lange, A. Lanza, F. Isasti, R.L. Lauria, F. Le Moine, F. Leblond, V. Lelli, G. Lenhoff, S. Leon, L.A. Leoncini-Franscini, L. Leone, G. Leoni, P. Levis, A. Leyvraz, S. Liberati, M. Link, H. Linkesch, W. Liso, V. Lisukov, I.A. Littlewood, T. Ljungman, P. Locatelli, F. Losonczy, H. Lotz, J.-P. Ludwig, H. Lukac, J. Lutz, D. Macchia, P. Madrigal, A. Maiolino, A. Majolino, I. Eloy-García, J.M. Malesevic, M. Mandelli, F. Marc, A. Marcus, R. Marianska, B. Markuljak, I. Marsh, J.C.W. Martelli, M.F. Marti Tutusaus, J.M. Martin, S. Martin, M. Martinelli, G. Martínez-Rubio, A.M. Martoni, A. Maschan, A. Maschmeyer, G. Masszi, T. Mazza, P. McCann, S. Meier, C.R. Messina, C. Mettivier, V. Metzner, B. Michallet, M. Michieli, M. Michon, J. Milligan, D.W. Milone, J.H. Giuseppe, G.M. Minigo, H. Mistrik, M. Moicean, A.D. Monfardini, S. Montserrat, E. Moraleda Jimenez, J.M. Morales-Lazaro, A. Morandi, S. Morra, E. Mufti, G.J. Musso, M. Nagler, A. Nalli, G. Naparstek, E. Narni, F. Nenadov-Beck, M. Neubauer, A. Newland, A.C. Niederwieser, D. Niethammer, D. Noens, L.A. Nousiainen, T. Novik, A. Novitzky, N. Occhini, D. Odriozolas, J. Ojanguren, J.M. O’meara, A. Onat, H. Orchard, K. Ortega, J.J. Osieka, R. Ossenkoppele, G.J. Othman, B. Ovali, E. Ozcebe, O.I. Ozerkan, K. Ozturk, A. Papatryfonos, A. Parker, J.E. Pastore, M. Patrone, F. Patton, N. Pejin, D. Peñarrubia, M.J. Equiza, E.P. Peschel, C. Pession, A. Pigaditou, A. Pignon, B. Pihkala, U. Pimentel, P. Pitini, V. Podoltseva, E. Pogliani, E.M. Anna, A.P. Porta, F. Potter, M. Powles, R. Prentice, G.H. Pretnar, J. Ptushkin, V. Quarta, G. Reiter, A. Remes, K. Reykdal, S. Santasusana, J.M.R. Rifón, J. Rio, B. Rizzoli, V. Robak, T. Robinson, A.J. Rodeghiero, F. Rodríguez Fernández, J.M. Rombos, Y. Romeril, K.R. Rosenmayr, A. Rossi, J.F. Rosti, G. Rotoli, B. Rowe, J.M. Russell, N.H. Ryzhak, O. Rzepecki, P. Saglio, G. Salwender, H. Samonigg, H. Santoro, A. Sanz, M.A. Sayer, H.G. Scanni, A. Schaafsma, M.R. Schaefer, U.W. Schanz, U. Schattenberg, A. Schey, S.A.M. Schlimok, G. Schmoll, H.-J. Schots, R. Schouten, H. Schwarer, A.P. Schwerdtfeger, R. Scimè, R. Segel, E. Seger, R. Selleslag, D. Serban, M. Shamaa, S. Shaw, P.J. Siegert, W. Siena, S. Sierra, J. Simonsson, B. Singer, C.R.J. Sirchia, G. Skotnicki, A.B. Slavin, S. Snowden, J. Sotto, J.J. Tanyeli, A. Tedeschi, L. Tidefelt, U. Tissot, J.-D. Tobler, A. Tomas, J.F. Torres, J.P. Torres, G.A. Touraine, J.-L. Trneny, M. Uderzo, C. Unal, E. Unal, A. Undar, L. Urban, C. Van den Berg, H. van Marwijk, K.M. Vellenga, E. Venturini, M. Verdonck, L.F. Veys, P. Vilardell, J. Vinante, O. Visani, G. Vitek, A. Vivancos, P. Volpe, E. Vora, A. Vorlicek, J. Vowels, M. Vujic, D. Wachowiak, J. Wagner, T. Wahlin, A. Walewski, J. Wandt, H. Weissinger, F. Wijermans, P.W. Wiktor-Jedrzejczak, W. Will, A.M. Woell, E. Wörmann, B. Yaniv, I. Yesilipek, M.A. Yilmaz, U. Yong, A. Zachée, P. Zambelli, A. Zander, A.R. Zintl, F. Zoumbos, N.C. The Acute Leukemia Working Party (ALWP) of the European Cooperative Group for Blood Marrow Transplantation (EBMT)

Abstract

Several studies have compared bone marrow (BM) and peripheral blood (PB) as stem cell sources in patients receiving allografts, but the cell doses infused have not been considered, especially for BM. Using the ALWP/EBMT registry, we retrospectively studied 881 adult patients with acute myelocytic leukemia (AML), who received a non-T-depleted allogeneic BM (n = 515) or mobilized PB (n = 366) standard transplant, in first remission (CR1), from an HLA-identical sibling, over a 5-year period from January 1994. The BM cell dose ranged from 0.17 to 29 × 108/kg with a median of 2.7 × 108/kg. The PB cell dose ranged from 0.02 to 77 × 10 8/kg with a median of 9.3 × 108/kg. The median dose for patients receiving BM (2.7 × 108/kg) gave the greatest discrimination. In multivariate analyses, high-dose BM compared to PB was associated with lower transplant-related mortality (RR = 0.61; 95% CI, 0.39-0.98; P = .04), better leukemia-free survival (RR = 0.65; 95% CI, 0.46-0.91; P = .013), and better overall survival (RR = 0.64; 95% CI, 0.44-0. 92; P = .016). The present study in patients with AML receiving allografts in first remission indicates a better outcome with BM as compared to PB, when the dose of BM infused is rich. © 2003 by The American Society of Hematology.

Published
2003

96. In vitro activity of oxazaphosphorines in childhood acute leukemia: preliminary report

Author

Styczynski, J., Wysocki, M., Debski, R., Balwierz, W., Rokicka-Milewska, R., Michał Matysiak, Balcerska, A., Kowalczyk, J., Wachowiak, J., Sonta-Jakimczyk, D., and Chybicka, A.

Subjects
Male, Adolescent, leukemia, Infant, In Vitro Techniques, Precursor Cell Lymphoblastic Leukemia-Lymphoma, sensitivity, General Biochemistry, Genetics and Molecular Biology, resistance, Leukemia, Myeloid, Acute, Glucose, Child, Preschool, Tumor Cells, Cultured, glufosfamide, Humans, Female, Phosphoramide Mustards, Ifosfamide, ALL, Child, Cyclophosphamide
Abstract

Glufosfamide (beta-D-glucosyl-ifosfamide mustard) is a new agent for cancer chemotherapy. Its pharmacology is similar to commonly used oxazaphosphorines, but it does not require activation by hepatic cytochrome P-450 and preclinically demonstrates lower nephrotoxicity and myelosuppression than ifosfamide. The aim of the study was a comparison of the drug resistance profiles of glufosfamide and other oxazaphosphorines in childhood acute leukemias. Leukemic cells, taken from children with ALL on diagnosis (n = 41), ALL on relapse (n = 12) and AML on diagnosis (n = 13) were analyzed by means of the MTT assay. The following drugs were tested: glufosfamide (GLU), 4-HOO-ifosfamide (IFO), 4-HOO-cyclophosphamide (CYC) and mafosfamide cyclohexylamine salt (MAF). In the group of initial ALL samples median cytotoxicity values for GLU, IFO, CYC and MAF were 15.5, 33.8, 15.7 and 7.8 microM, respectively. In comparison with initial ALL samples, the relative resistance for GLU and IFO in relapsed ALL samples was 1.9 (p = 0.049) and 1.3 (ns), and in initial AML samples 31 (p < 0.001) and 5 (p = 0.001), respectively. All oxazaphosphorines presented highly significant cross-resistance. Glufosfamide presented high activity against lymphoblasts both on diagnosis and on relapse.

Published
2002

97. [Preliminary results of BFM 96 protocol in treatment of childhood ALL relapse in the experience of the Polish Paediatric Leukaemia /Lymphoma Study Group]

Author

Chybicka, A., Boguslawska-Jaworska, J., Gorczynska, E., Armata, J., Balcerska, A., Balwierz, W., Bubala, H., Filiks-Litwin, B., Kolecki, P., Kowalczyk, J., Lukowska, K., Michał Matysiak, Rokicka-Milewska, R., Rola-Kurc, E., Stencel, D., Sonta-Jakimczyk, D., Strojny, W., Wachowiak, J., Wieczorek, M., Wysocki, M., and Zelenay, E.

Subjects
Male, Adolescent, Daunorubicin, Remission Induction, Infant, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Survival Analysis, Treatment Outcome, Risk Factors, Vincristine, Child, Preschool, Antineoplastic Combined Chemotherapy Protocols, Asparaginase, Humans, Prednisone, Female, Poland, Neoplasm Recurrence, Local, Child, Bone Marrow Transplantation, Follow-Up Studies
Abstract

Between 1998 and 1999, 36 children aged from 3 months to 18 years (10 girls and 26 boys) with first relapse of acute lymphoblastic leukaemia were included in the study. The children were treated according to the BFM 96 relapse protocol. There were 24 cases with early (including 9 children with very early) and 12 cases with late relapse ( BM-20, local extra BM-6, combined 10). The overall second complete remission (CR) rate was 83,33%. The probability of overall EFS after 2 years was 73,3%. The results obtained with BFM 96 chemotherapy in children with first late relapse are acceptable. For children with early relapses, megachemotherapy with BMT in second remission should be used.

Published
2002

98. Risk of complications during hematopoietic stem cell collection in pediatric sibling donors: a prospective European Group for Blood and Marrow Transplantation Pediatric Diseases Working Party study

Author

Styczynski, J, Balduzzi, A, Gil, L, Labopin, M, Hamladji, R, Marktel, S, Yesilipek M., A, Fagioli, F, Ehlert, K, Matulova, M, Dalle, J, Wachowiak, J, Miano, M, Messina, C, Diaz Miguel, A, Vermylen, C, Eyrich, M, Badell, I, Dreger, P, Gozdzik, J, Hutt, D, Rascon, J, Dini, G, Peters, C, Styczynski Jan, Balduzzi A, Gil Lidia, Labopin Myriam, Hamladji Rose-Marie, Marktel Sarah, Yesilipek M. Akif, Fagioli Franca, Ehlert Karoline, Matulova Martina, Dalle Jean-Hugues, Wachowiak Jacek, Miano Maurizio, Messina Chiara, Diaz Miguel Angel, Vermylen Christiane, Eyrich Matthias, Badell Isabel, Dreger Peter, Gozdzik Jolanta, Hutt Daphna, Rascon Jelena, Dini Giorgio, Peters Christina, Styczynski, J, Balduzzi, A, Gil, L, Labopin, M, Hamladji, R, Marktel, S, Yesilipek M., A, Fagioli, F, Ehlert, K, Matulova, M, Dalle, J, Wachowiak, J, Miano, M, Messina, C, Diaz Miguel, A, Vermylen, C, Eyrich, M, Badell, I, Dreger, P, Gozdzik, J, Hutt, D, Rascon, J, Dini, G, Peters, C, Styczynski Jan, Balduzzi A, Gil Lidia, Labopin Myriam, Hamladji Rose-Marie, Marktel Sarah, Yesilipek M. Akif, Fagioli Franca, Ehlert Karoline, Matulova Martina, Dalle Jean-Hugues, Wachowiak Jacek, Miano Maurizio, Messina Chiara, Diaz Miguel Angel, Vermylen Christiane, Eyrich Matthias, Badell Isabel, Dreger Peter, Gozdzik Jolanta, Hutt Daphna, Rascon Jelena, Dini Giorgio, and Peters Christina

Abstract

We investigated prospectively factors influencing the safety of hematopoietic stem cell (HSC) collection in 453 pediatric donors. The children in the study donated either BM or peripheral blood stem cells (PBSCs) according to center policy. A large variability in approach to donor issues was observed between the participating centers. Significant differences were observed between BM and PBSC donors regarding pain, blood allotransfusion, duration of hospital stay, and iron supplementation; however, differences between the groups undergoing BM vs PBSC donation preclude direct risk comparisons between the 2 procedures. The most common adverse event was pain, reported mainly by older children after BM harvest, but also observed after central venous catheter (CVC) placement for PBSC collection. With regard to severe adverse events, one patient (0.7%) developed a pneumothorax with hydrothorax after CVC placement for PBSC collection. The risk of allotransfusion after BM harvest was associated with a donor age of < 4 years and a BM harvest volume of > 20 mL/kg. Children < 4 years were at higher risk than older children for allotransfusion after BM harvest and there was a higher risk of complications from CVC placement before apheresis. We conclude that PBSC and BM collection are safe procedures in children. (Blood. 2012;119(12):2935-2942), We investigated prospectively factors influencing the safety of hematopoietic stem cell (HSC) collection in 453 pediatric donors. The children in the study donated either BM or peripheral blood stem cells (PBSCs) according to center policy. A large variability in approach to donor issues was observed between the participating centers. Significant differences were observed between BM and PBSC donors regarding pain, blood allotransfusion, duration of hospital stay, and iron supplementation; however, differences between the groups undergoing BM vs PBSC donation preclude direct risk comparisons between the 2 procedures. The most common adverse event was pain, reported mainly by older children after BM harvest, but also observed after central venous catheter (CVC) placement for PBSC collection. With regard to severe adverse events, one patient (0.7%) developed a pneumothorax with hydrothorax after CVC placement for PBSC collection. The risk of allotransfusion after BM harvest was associated with a donor age of < 4 years and a BM harvest volume of > 20 mL/kg. Children < 4 years were at higher risk than older children for allotransfusion after BM harvest and there was a higher risk of complications from CVC placement before apheresis.We conclude that PBSC and BM collection are safe procedures in children. © 2012 by The American Society of Hematology.

Published
2012

99. Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation

Author

Ozsahin, H., Cavazzana-Calvo, M., Notarangelo, L.D., Schulz, A., Thrasher, A.J., Mazzolari, E., Slatter, M.A., Le, Deist F., Blanche, S., Veys, P., Fasth, A., Bredius, R., Sedlacek, P., Wulffraat, N., Ortega, J., Heilmann, C., O'Meara, A., Wachowiak, J., Kalwak, K., Matthes-Martin, S., Gungor, T., Ikinciogullari, A., Landais, P., Cant, A.J., Friedrich, W., Fischer, A., Ozsahin, H., Cavazzana-Calvo, M., Notarangelo, L.D., Schulz, A., Thrasher, A.J., Mazzolari, E., Slatter, M.A., Le, Deist F., Blanche, S., Veys, P., Fasth, A., Bredius, R., Sedlacek, P., Wulffraat, N., Ortega, J., Heilmann, C., O'Meara, A., Wachowiak, J., Kalwak, K., Matthes-Martin, S., Gungor, T., Ikinciogullari, A., Landais, P., Cant, A.J., Friedrich, W., and Fischer, A.

Abstract

Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiency with microthrombocytopenia, eczema, recurrent infections, autoimmune disorders, and malignancies that are life-threatening in the majority of patients. In this long-term, retrospective, multicenter study, we analyzed events that occurred in 96 WAS patients who received transplants between 1979 and 2001 who survived at least 2 years following hematopoietic stem-cell transplantation (HSCT). Events included chronic graft-versus-host disease (cGVHD), autoimmunity, infections, and sequelae of before or after HSCT complications. Three patients (3%) died 2.1 to 21 years following HSCT. Overall 7-year event-free survival rate was 75%. It was lower in recipients of mismatched related donors, also in relation with an older age at HSCT and disease severity. The most striking finding was the observation of cGVHD-independent autoimmunity in 20% of patients strongly associated with a mixed/split chimerism status (P < .001), suggesting that residual-host lymphocytes can mediate autoimmune disease despite the coexistence of donor lymphocytes. Infectious complications (6%) related to splenectomy were also significant and may warrant a more restrictive approach to performing splenectomy in WAS patients. Overall, this study provides the basis for a prospective, standardized, and more in-depth detailed analysis of chimerism and events in long-term follow-up of WAS patients who receive transplants to design better-adapted therapeutic strategies Udgivelsesdato: 2008/1/1

Published
2008

100. Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation

Author

Ozsahin, H, Cavazzana-Calvo, M, Notarangelo, L D, Schulz, A, Thrasher, A J, Mazzolari, E, Slatter, M A, Le Deist, F, Blanche, S, Veys, P, Fasth, A, Bredius, R, Sedlacek, P, Wulffraat, N, Ortega, J, Heilmann, C, O'Meara, A, Wachowiak, J, Kalwak, K, Matthes-Martin, S, Güngör, Tayfun; https://orcid.org/0000-0002-3261-1186, Ikinciogullari, A, Landais, P, Cant, A J, Friedrich, W, Fischer, A, Ozsahin, H, Cavazzana-Calvo, M, Notarangelo, L D, Schulz, A, Thrasher, A J, Mazzolari, E, Slatter, M A, Le Deist, F, Blanche, S, Veys, P, Fasth, A, Bredius, R, Sedlacek, P, Wulffraat, N, Ortega, J, Heilmann, C, O'Meara, A, Wachowiak, J, Kalwak, K, Matthes-Martin, S, Güngör, Tayfun; https://orcid.org/0000-0002-3261-1186, Ikinciogullari, A, Landais, P, Cant, A J, Friedrich, W, and Fischer, A

Abstract

Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiency with microthrombocytopenia, eczema, recurrent infections, autoimmune disorders, and malignancies that are life-threatening in the majority of patients. In this long-term, retrospective, multicenter study, we analyzed events that occurred in 96 WAS patients who received transplants between 1979 and 2001 who survived at least 2 years following hematopoietic stem-cell transplantation (HSCT). Events included chronic graft-versus-host disease (cGVHD), autoimmunity, infections, and sequelae of before or after HSCT complications. Three patients (3%) died 2.1 to 21 years following HSCT. Overall 7-year event-free survival rate was 75%. It was lower in recipients of mismatched related donors, also in relation with an older age at HSCT and disease severity. The most striking finding was the observation of cGVHD-independent autoimmunity in 20% of patients strongly associated with a mixed/split chimerism status (P < .001), suggesting that residual-host lymphocytes can mediate autoimmune disease despite the coexistence of donor lymphocytes. Infectious complications (6%) related to splenectomy were also significant and may warrant a more restrictive approach to performing splenectomy in WAS patients. Overall, this study provides the basis for a prospective, standardized, and more in-depth detailed analysis of chimerism and events in long-term follow-up of WAS patients who receive transplants to design better-adapted therapeutic strategies.

Published
2008

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