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

Blood
Elie HaddadLuigi D Notarangelo

Abstract

The Primary Immune Deficiency Treatment Consortium (PIDTC) performed a retrospective analysis of 662 patients with severe combined immunodeficiency (SCID) who received a hematopoietic cell transplantation (HCT) as first-line treatment between 1982 and 2012 in 33 North American institutions. Overall survival was higher after HCT from matched-sibling donors (MSDs). Among recipients of non-MSD HCT, multivariate analysis showed that the SCID genotype strongly influenced survival and immune reconstitution. Overall survival was similar for patients with RAG, IL2RG, or JAK3 defects and was significantly better compared with patients with ADA or DCLRE1C mutations. Patients with RAG or DCLRE1C mutations had poorer immune reconstitution than other genotypes. Although survival did not correlate with the type of conditioning regimen, recipients of reduced-intensity or myeloablative conditioning had a lower incidence of treatment failure and better T- and B-cell reconstitution, but a higher risk for graft-versus-host disease, compared with those receiving no conditioning or immunosuppression only. Infection-free status and younger age at HCT were associated with improved survival. Typical SCID, leaky SCID, and Omenn syndrome had similar out...Continue Reading

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References

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Citations

Jul 27, 2018·Journal of Clinical Immunology·Ottavia M DelmonteLuigi D Notarangelo
Apr 18, 2019·The New England Journal of Medicine·Ewelina MamcarzBrian P Sorrentino
May 10, 2019·Expert Review of Clinical Immunology·Reem ElfekyPaul Veys
Oct 27, 2018·Blood·Andrew R Gennery
Sep 20, 2019·Expert Review of Clinical Immunology·Adeeb A BulkhiJolan E Walter
Dec 12, 2019·The Journal of Experimental Medicine·Alain Fischer, Salima Hacein-Bey-Abina
Oct 1, 2019·Current Opinion in Pediatrics·Maria Fernanda Villavicencio, Luis Alberto Pedroza
Mar 30, 2019·Journal of Clinical Immunology·Aydan IkinciogullariUNKNOWN Turkish Pediatric Bone Marrow Transplantation Sub Group (TPBMT-SG)
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Nov 19, 2019·Frontiers in Pediatrics·Andrew R GenneryArjan Lankester
Apr 9, 2020·Frontiers in Immunology·Christin DealManish J Butte
Oct 3, 2020·Journal of Clinical Immunology·Morna J DorseyJennifer Heimall
Feb 26, 2019·F1000Research·Andrew Gennery
Aug 24, 2019·Frontiers in Pediatrics·Riccardo CastagnoliLuigi Daniele Notarangelo
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Jul 11, 2020·Current Allergy and Asthma Reports·Mary A Slatter, Andrew R Gennery
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Apr 7, 2019·Bone Marrow Transplantation·Rafael F DuarteUNKNOWN European Society for Blood and Marrow Transplantation (EBMT)
Aug 9, 2019·World Journal of Transplantation·Tatyana Gavrilova
Dec 6, 2019·Frontiers in Pediatrics·Elie Haddad, Manfred Hoenig
Apr 1, 2020·F1000Research·Giorgia Bucciol, Isabelle Meyts
Dec 16, 2019·Journal of Clinical Immunology·Noa Greenberg-KushnirTali Stauber
Sep 20, 2020·The Journal of Allergy and Clinical Immunology. in Practice·Gabriela MaronEwelina Mamcarz
May 11, 2020·The Journal of Allergy and Clinical Immunology·Maria Carmina CastielloLuigi D Notarangelo
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Dec 18, 2020·Frontiers in Immunology·Anna VillaMaria Carmina Castiello
Feb 9, 2021·The Journal of Allergy and Clinical Immunology·Robert Currier, Jennifer M Puck

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