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Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT Inborn Errors Working Party analysis
MH. Albert, MA. Slatter, AR. Gennery, T. Güngör, K. Bakunina, B. Markovitch, S. Hazelaar, T. Sirait, V. Courteille, A. Aiuti, OV. Aleinikova, D. Balashov, ME. Bernardo, I. Bodova, B. Bruno, M. Cavazzana, R. Chiesa, A. Fischer, F. Hauck, M....
Jazyk angličtina Země Spojené státy americké
Typ dokumentu časopisecké články, práce podpořená grantem
NLK
Free Medical Journals
od 1946 do Před 1 rokem
Freely Accessible Science Journals
od 1946 do Před 1 rokem
Open Access Digital Library
od 1946-01-01
Open Access Digital Library
od 1946-01-01
ROAD: Directory of Open Access Scholarly Resources
PubMed
35100336
DOI
10.1182/blood.2021014687
Knihovny.cz E-zdroje
- MeSH
- busulfan terapeutické užití MeSH
- dárci tkání MeSH
- lidé MeSH
- nemoc štěpu proti hostiteli * etiologie MeSH
- předškolní dítě MeSH
- příprava pacienta k transplantaci metody MeSH
- retrospektivní studie MeSH
- transplantace hematopoetických kmenových buněk * škodlivé účinky MeSH
- výsledek terapie MeSH
- Wiskottův-Aldrichův syndrom * terapie MeSH
- Check Tag
- lidé MeSH
- předškolní dítě MeSH
- Publikační typ
- časopisecké články MeSH
- práce podpořená grantem MeSH
Allogeneic hematopoietic stem cell transplantation (HSCT) is a potentially curative treatment for patients affected by Wiskott-Aldrich syndrome (WAS). Reported HSCT outcomes have improved over time with respect to overall survival, but some studies have identified older age and HSCT from alternative donors as risk factors predicting poorer outcome. We analyzed 197 patients undergoing transplant at European Society for Blood and Marrow Transplantation centers between 2006 and 2017 who received conditioning as recommended by the Inborn Errors Working Party (IEWP): either busulfan (n = 103) or treosulfan (n = 94) combined with fludarabine ± thiotepa. After a median follow-up post-HSCT of 44.9 months, 176 patients were alive, resulting in a 3-year overall survival of 88.7% and chronic graft-versus-host disease (GVHD)-free survival (events include death, graft failure, and severe chronic GVHD) of 81.7%. Overall survival and chronic GVHD-free survival were not significantly affected by conditioning regimen (busulfan- vs treosulfan-based), donor type (matched sibling donor/matched family donor vs matched unrelated donor/mismatched unrelated donor vs mismatched family donor), or period of HSCT (2006-2013 vs 2014-2017). Patients aged <5 years at HSCT had a significantly better overall survival. The overall cumulative incidences of grade III to IV acute GVHD and extensive/moderate/severe chronic GVHD were 6.6% and 2.1%, respectively. Patients receiving treosulfan-based conditioning had a higher incidence of graft failure and mixed donor chimerism and more frequently underwent secondary procedures (second HSCT, unconditioned stem cell boost, donor lymphocyte infusion, or splenectomy). In summary, HSCT for WAS with conditioning regimens currently recommended by IEWP results in excellent survival and low rates of GVHD, regardless of donor or stem cell source, but age ≥5 years remains a risk factor for overall survival.
Bone Marrow Transplant Department Great Ormond Street Hospital For Sick Children London England
Bone Marrow Transplantation Unit National Institute of Children's Diseases Bratislava Slovakia
Department of Pediatric Hematology and Oncology Comenius University Bratislava Slovakia
Department of Pediatric Hematology and Oncology University Hospital Motol Prague Czech Republic
Department of Pediatrics BMT Unit Hacettepe University Faculty of Medicine Ankara Turkey
Department of Pediatrics University Medical Center Ulm Ulm Germany
IMAGINE Institute Université de Paris Sorbonne Paris Cité Paris France
Necker Enfants Malades University Hospital Assistance Publique Hôpitaux de Paris Paris France
Pediatric Hematology and Oncology MHH Hannover Germany
Pediatric Hematology CHU Lille Lille France
Pediatric Hematology Oncology Akdeniz University School of Medicine Antalya Turkey
Pediatric Hematology Oncology Fondazione IRCCS Policlinico San Matteo Pavia Italy
Pediatric Immunohematology IRCCS Ospedale San Raffaele Milan Italy
Pediatric Oncohaematology and BMT Children's Hospital Brescia Italy
Princess Máxima Center Utrecht The Netherlands
RM Gorbacheva Research Institute Pavlov University St Petersburg Russian Federation
Statistical and Study Unit EBMT office Leiden The Netherlands
University College London GOS Institute of Child Health London England
Citace poskytuje Crossref.org
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