SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery

Author:

Haddad Elie1ORCID,Logan Brent R.2,Griffith Linda M.3,Buckley Rebecca H.4,Parrott Roberta E.4,Prockop Susan E.5,Small Trudy N.5,Chaisson Jessica5,Dvorak Christopher C.6,Murnane Megan6,Kapoor Neena7,Abdel-Azim Hisham7,Hanson Imelda C.8,Martinez Caridad9,Bleesing Jack J. H.10,Chandra Sharat10,Smith Angela R.11,Cavanaugh Matthew E.12,Jyonouchi Soma13,Sullivan Kathleen E.13,Burroughs Lauri1415,Skoda-Smith Suzanne15,Haight Ann E.16,Tumlin Audrey G.16,Quigg Troy C.17,Taylor Candace17,Dávila Saldaña Blachy J.18,Keller Michael D.18,Seroogy Christine M.19,Desantes Kenneth B.19,Petrovic Aleksandra20,Leiding Jennifer W.2021,Shyr David C.22,Decaluwe Hélène1,Teira Pierre1,Gillio Alfred P.23,Knutsen Alan P.24,Moore Theodore B.25,Kletzel Morris26,Craddock John A.27,Aquino Victor28,Davis Jeffrey H.29,Yu Lolie C.30,Cuvelier Geoffrey D. E.31,Bednarski Jeffrey J.32,Goldman Frederick D.33,Kang Elizabeth M.34,Shereck Evan35,Porteus Matthew H.36,Connelly James A.37,Fleisher Thomas A.38,Malech Harry L.34,Shearer William T.8,Szabolcs Paul39,Thakar Monica S.40,Vander Lugt Mark T.41,Heimall Jennifer13,Yin Ziyan2,Pulsipher Michael A.7,Pai Sung-Yun12,Kohn Donald B.25,Puck Jennifer M.6,Cowan Morton J.6,O'Reilly Richard J.5,Notarangelo Luigi D.42

Affiliation:

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

2. Division of Biostatistics, Medical College of Wisconsin, Milwaukee, WI,

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

4. Duke University Medical Center, Durham, NC;

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

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

7. Blood and Marrow Transplant Program, Division of Hematology, Oncology and Blood and Marrow Transplantation, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA;

8. Immunology, Allergy and Rheumatology and

9. Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston, TX;

10. Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH;

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

12. Hematology-Oncology, Boston Children's Hospital, Boston, MA;

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

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

15. Seattle Children’s Hospital, Seattle, WA;

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

17. Texas Transplant Institute, Methodist Children's Hospital, San Antonio, TX;

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

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

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

21. Division of Allergy and Immunology, Department of Pediatrics, University of South Florida, St. Petersburg, FL;

22. Department of Pediatrics, Primary Children’s Hospital, University of Utah, Salt Lake City, UT;

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

24. Pediatric Allergy and Immunology, Saint Louis University, Cardinal Glennon Children's Medical Center, St. Louis, MO;

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

26. 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;

27. Children’s Hospital of Colorado, University of Colorado School of Medicine, Aurora, CO;

28. Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX;

29. Pediatrics, British Columbia Children's Hospital, Vancouver, BC, Canada;

30. Division of Hematology/Oncology and Hematopoietic Stem Cell Transplantation, The Center for Cancer and Blood Disorders, Children's Hospital/Louisiana State University Medical Center, New Orleans, LA;

31. Manitoba Blood and Marrow Transplant Program, CancerCare Manitoba, University of Manitoba, Winnipeg, MB, Canada;

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

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

34. Genetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD;

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

36. Pediatric Stem Cell Transplantation, Stanford University, Stanford, CA;

37. Vanderbilt University Medical Center, Nashville, TN;

38. Department of Laboratory Medicine, Clinical Center, National Institutes of Health, Bethesda, MD;

39. Children's Hospital of Pittsburgh, Pittsburgh, PA;

40. Pediatric Blood and Marrow Transplant Program, Division of Hematology, Oncology, and Blood Marrow Transplantation, Medical College of Wisconsin, Milwaukee, WI;

41. Pediatric Hematology/Oncology, University of Michigan, Ann Arbor, MI; and

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

Abstract

Key Points The genetic cause of SCID impacts on survival and immune reconstitution and should be considered in tailoring HCT for individual patients. Total and naive CD4+ cell counts in SCID patients 6 and 12 months post-HCT predict long-term survival and sustained immune reconstitution.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

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