Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report-Reference-Cited by-同舟云学术

Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report

Published:2020-06-04 Issue:23 Volume:135 Page:2094-2105
ISSN:0006-4971
Container-title:Blood
language:en
Short-container-title:

Author:

Burroughs Lauri M.¹²,Petrovic Aleksandra¹²,Brazauskas Ruta³,Liu Xuerong³,Griffith Linda M.⁴,Ochs Hans D.²,Bleesing Jack J.⁵,Edwards Stephanie⁵,Dvorak Christopher C.⁶^ORCID,Chaudhury Sonali⁷,Prockop Susan E.⁸,Quinones Ralph⁹,Goldman Frederick D.¹⁰^ORCID,Quigg Troy C.¹¹,Chandrakasan Shanmuganathan¹²,Smith Angela R.¹³,Parikh Suhag¹⁴^ORCID,Dávila Saldaña Blachy J.¹⁵^ORCID,Thakar Monica S.¹⁶,Phelan Rachel¹⁶,Shenoy Shalini¹⁷,Forbes Lisa R.¹⁸^ORCID,Martinez Caridad¹⁹,Chellapandian Deepak²⁰,Shereck Evan²¹,Miller Holly K.²²,Kapoor Neena²³,Barnum Jessie L.²⁴,Chong Hey²⁴,Shyr David C.²⁵,Chen Karin²⁶^ORCID,Abu-Arja Rolla²⁷,Shah Ami J.²⁸,Weinacht Katja G.²⁸,Moore Theodore B.²⁹,Joshi Avni³⁰,DeSantes Kenneth B.³¹,Gillio Alfred P.³²,Cuvelier Geoffrey D. E.³³,Keller Michael D.³⁴³⁵,Rozmus Jacob³⁶,Torgerson Troy²,Pulsipher Michael A.²³^ORCID,Haddad Elie³⁷^ORCID,Sullivan Kathleen E.³⁸,Logan Brent R.³,Kohn Donald B.²⁹^ORCID,Puck Jennifer M.⁶,Notarangelo Luigi D.³⁹^ORCID,Pai Sung-Yun⁴⁰⁴¹^ORCID,Rawlings David J.²,Cowan Morton J.⁶^ORCID

Affiliation:

1. Fred Hutchinson Cancer Research Center, Seattle, WA;

2. Department of Pediatrics, University of Washington–Seattle Children’s Hospital, Seattle, WA;

3. Division of Biostatistics, Medical College of Wisconsin, Milwaukee, WI;

4. Division of Allergy, Immunology, and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD;

5. Division of Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics, Cincinnati Children’s Hospital Medical Center, University of Cincinnati, Cincinnati, OH;

6. Pediatric Allergy, Immunology, and Blood and Marrow Transplant Division, University of California, San Francisco Benioff Children’s Hospital, San Francisco, CA;

7. Division of Hematology, Oncology, and Stem Cell Transplantation, Ann & Robert H. Lurie Children’s Hospital of Chicago–Northwestern University Feinberg School of Medicine, Chicago, IL;

8. Bone Marrow Transplant Service, Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY;

9. Pediatric Bone Marrow Transplant (BMT) and Cellular Therapy Section, Department of Pediatrics, The University of Colorado School of Medicine, Aurora, CO;

10. Department of Pediatrics, The University of Alabama at Birmingham, Birmingham, AL;

11. Texas Transplant Institute, Methodist Children’s Hospital, San Antonio, TX;

12. Aflac Cancer and Blood Disorders Center, Emory/Children’s Healthcare of Atlanta, Atlanta, GA;

13. Division of Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN;

14. Duke University Medical Center, Durham, NC;

15. Division of Blood and Marrow Transplantation, Children's National Hospital–George Washington University School of Medicine and Health Sciences, Washington, DC;

16. Center for Blood and Marrow Transplant Research–Division of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation, Medical College of Wisconsin, Milwaukee, WI;

17. Department of Pediatrics, Washington University School of Medicine, St. Louis, MO;

18. Department of Pediatrics, Baylor College of Medicine Section of Immunology, Allergy, and Retrovirology, Texas Children’s Hospital, Baylor, TX;

19. Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children’s Hospital Cancer Center, Baylor, TX;

20. Blood and Marrow Transplant, Johns Hopkins All Children's Hospital, St. Petersburg, FL;

21. Division of Pediatric Hematology/Oncology, Oregon Health and Science University, Portland, OR;

22. Phoenix Children’s Hospital, Phoenix, AZ;

23. Transplantation and Cellular Therapy Program, Cancer and Blood Disease Institute, Children’s Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA;

24. UPMC Children's Hospital of Pittsburgh, Pittsburgh, PA;

25. Division of Pediatric Hematology/Oncology, Primary Children’s Hospital, University of Utah School of Medicine, Salt Lake City, UT;

26. Division of Allergy and Immunology, Department of Pediatrics, University of Utah School of Medicine, Salt Lake City, UT;

27. Nationwide Children’s Hospital, Columbus, OH;

28. Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford School of Medicine Pediatric Stem Cell Transplantation, Stanford University, Stanford, CA;

29. Department of Pediatrics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA;

30. Mayo Clinic Children’s Center, Rochester, MN;

31. American Family Children's Hospital, University of Wisconsin, Madison, WI;

32. Institute for Pediatric Cancer and Blood Disorders, Hackensack University Medical Center, Hackensack, NJ;

33. Cancer Care Manitoba, University of Manitoba, Winnipeg, MB, Canada;

34. Division of Allergy & Immunology, Children’s National Hospital, Washington, DC.;

35. GW Cancer Center, George Washington University, Washington, DC.;

36. Children’s & Women’s Health Centre of British Columbia, Vancouver, BC, Canada;

37. Pediatric Immunology and Rheumatology Division, CHU Sainte-Justine, Department of Pediatrics, University of Montreal, Montreal, QC, Canada;

38. Allergy and Immunology, The Children's Hospital of Philadelphia, Philadelphia, PA;

39. Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD;

40. Division of Hematology-Oncology, Boston Children's Hospital, Boston, MA; and

41. Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA

Abstract

Abstract Wiskott-Aldrich syndrome (WAS) is an X-linked disease caused by mutations in the WAS gene, leading to thrombocytopenia, eczema, recurrent infections, autoimmune disease, and malignancy. Hematopoietic cell transplantation (HCT) is the primary curative approach, with the goal of correcting the underlying immunodeficiency and thrombocytopenia. HCT outcomes have improved over time, particularly for patients with HLA-matched sibling and unrelated donors. We report the outcomes of 129 patients with WAS who underwent HCT at 29 Primary Immune Deficiency Treatment Consortium centers from 2005 through 2015. Median age at HCT was 1.2 years. Most patients (65%) received myeloablative busulfan-based conditioning. With a median follow-up of 4.5 years, the 5-year overall survival (OS) was 91%. Superior 5-year OS was observed in patients <5 vs ≥5 years of age at the time of HCT (94% vs 66%; overall P = .0008). OS was excellent regardless of donor type, even in cord blood recipients (90%). Conditioning intensity did not affect OS, but was associated with donor T-cell and myeloid engraftment after HCT. Specifically, patients who received fludarabine/melphalan-based reduced-intensity regimens were more likely to have donor myeloid chimerism <50% early after HCT. In addition, higher platelet counts were observed among recipients who achieved full (>95%) vs low-level (5%-49%) donor myeloid engraftment. In summary, HCT outcomes for WAS have improved since 2005, compared with prior reports. HCT at a younger age continues to be associated with superior outcomes supporting the recommendation for early HCT. High-level donor myeloid engraftment is important for platelet reconstitution after either myeloablative or busulfan-containing reduced intensity conditioning. (This trial was registered at www.clinicaltrials.gov as #NCT02064933.)

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Link

http://ashpublications.org/blood/article-pdf/135/23/2094/1743290/bloodbld2019002939.pdf

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