Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT Inborn Errors Working Party analysis-Reference-Cited by-同舟云学术

Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT Inborn Errors Working Party analysis

Published:2022-03-31 Issue:13 Volume:139 Page:2066-2079
ISSN:0006-4971
Container-title:Blood
language:en
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Author:

Albert Michael H.¹^ORCID,Slatter Mary A.²³,Gennery Andrew R.²³,Güngör Tayfun⁴^ORCID,Bakunina Katerina⁵,Markovitch Benyamin⁵,Hazelaar Sheree⁵,Sirait Tiarlan⁵,Courteille Virginie⁶,Aiuti Alessandro⁷⁸,Aleinikova Olga V.⁹^ORCID,Balashov Dmitry¹⁰,Bernardo Maria Ester⁷⁸,Bodova Ivana¹¹¹²,Bruno Benedicte¹³,Cavazzana Marina¹⁴¹⁵^ORCID,Chiesa Robert¹⁶,Fischer Alain¹⁵¹⁷,Hauck Fabian¹^ORCID,Ifversen Marianne¹⁸,Kałwak Krzysztof¹⁹^ORCID,Klein Christoph¹,Kulagin Alexander²⁰^ORCID,Kupesiz Alphan²¹,Kuskonmaz Baris²²^ORCID,Lindemans Caroline A.²³²⁴,Locatelli Franco²⁵,Lum Su Han²,Maschan Alexey¹⁰^ORCID,Meisel Roland²⁶^ORCID,Moshous Despina¹⁵²⁷,Porta Fulvio²⁸,Sauer Martin G.²⁹,Sedlacek Petr³⁰,Schulz Ansgar³¹,Suarez Felipe¹⁵³²^ORCID,Vallée Tanja C.¹,Winiarski Jacek H.³³,Zecca Marco³⁴^ORCID,Neven Bénédicte¹⁵²⁷,Veys Paul¹⁶³⁵,Lankester Arjan C.³⁶

Affiliation:

1. Department of Pediatrics, Dr. von Hauner Children’s Hospital, University Hospital, Ludwig-Maximilians-Universität München, Munich, Germany;

2. Paediatric Stem Cell Transplant Unit, Great North Children’s Hospital, Newcastle upon Tyne, United Kingdom;

3. Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, United Kingdom;

4. Hematology/Oncology/Immunology, Gene Therapy, and Stem Cell Transplantation, University Children’s Hospital Zurich–Eleonore Foundation & Children’s Research Center, Zürich, Switzerland;

5. Statistical and Study Unit, EBMT office, Leiden, The Netherlands;

6. CEREDIH, SCETIDE, Necker-Enfants Malades University Hospital, Assistance Publique Hôpitaux de Paris, Paris, France;

7. Pediatric Immunohematology, IRCCS Ospedale San Raffaele, Milan, Italy;

8. Vita Salute San Raffaele University, Milan, Italy;

9. Scientific Department, Belarusian Research Center for Pediatric Oncology, Hematology and Immunology, Borovlyany, Minsk Region, Belarus;

10. Department of Hematopoietic Stem Cell Transplantation, Dmitriy Rogachev National Center for Pediatric Hematology, Oncology, and Immunology, Moscow, Russian Federation;

11. Bone Marrow Transplantation Unit, National Institute of Children’s Diseases, Bratislava, Slovakia;

12. Department of Pediatric Hematology and Oncology, Comenius University, Bratislava, Slovakia;

13. Pediatric Hematology, CHU Lille, Lille, France;

14. Biotherapy Department and Clinical Investigation Center, Assistance Publique Hôpitaux de Paris, Paris, France;

15. IMAGINE Institute, Université de Paris, Sorbonne Paris Cité, Paris, France;

16. Bone Marrow Transplant Department, Great Ormond Street Hospital For Sick Children, London, England;

17. Necker-Enfants Malades University Hospital, Assistance Publique Hôpitaux de Paris, Paris, France;

18. Department for Children and Adolescent Medicine, Rigshospitalet, Copenhagen University Hospital, Copenhagen, Denmark;

19. Clinical Department of Paediatric Bone Marrow Transplantation, Oncology and Haematology, Wroclaw Medical University, Wroclaw, Poland;

20. RM Gorbacheva Research Institute, Pavlov University, St. Petersburg, Russian Federation;

21. Pediatric Hematology Oncology, Akdeniz University School of Medicine, Antalya, Turkey;

22. Department of Pediatrics, BMT Unit, Hacettepe University, Faculty of Medicine, Ankara, Turkey;

23. Princess Máxima Center, Utrecht, The Netherlands;

24. Department of Pediatric Hematoloy/Oncology, Cell and Gene Therapy, University Medical Center, University Utrecht, Utrecht, The Netherlands;

25. Department of Pediatric Hematoloy/Oncology, Cell and Gene Therapy, Bambino Gesù Children’s Hospital, Sapienza University, Rome, Italy;

26. Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology and Clinical Immunology, Medical Faculty, Heinrich-Heine-University, Düsseldorf, Germany;

27. Pediatric Immunology, Hematology and Rheumatology Department, Necker-Enfants Malades University Hospital, Assistance Publique Hôpitaux de Paris, Paris, France;

28. Pediatric Oncohaematology and BMT, Children’s Hospital, Brescia, Italy;

29. Pediatric Hematology and Oncology, MHH, Hannover, Germany;

30. Department of Pediatric Hematology and Oncology, University Hospital Motol, Prague, Czech Republic;

31. Department of Pediatrics, University Medical Center Ulm, Ulm, Germany;

32. Adult Hematology, Necker-Enfants Malades University Hospital, Assistance Publique Hôpitaux de Paris, Paris, France;

33. Department of Pediatrics, Karolinska University Hospital Huddinge, Clintec, Karolinska Institutet, Stockholm, Sweden;

34. Pediatric Hematology/Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy;

35. University College London, GOS Institute of Child Health, London, England; and

36. Department of Pediatrics, Pediatric Stem Cell Transplantation Program, Willem-Alexander Children’s Hospital, Leiden University Medical Center, The Netherlands

Abstract

Abstract 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.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Link

https://ashpublications.org/blood/article-pdf/139/13/2066/1884227/bloodbld2021014687.pdf

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