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Outcome of Second Allogeneic HSCT for Patients with Inborn Errors of Immunity: Retrospective Study of 20 Years’ Experience

Lookup NU author(s): Dr Christo TsilifisORCiD, Dr Su Han Lum, Professor Mary Slatter, Professor Sophie HambletonORCiD, Dr Stephen Owens, Dr Eleri Williams, Dr Terence Flood, Professor Andrew GenneryORCiD, Dr Zohreh Nademi


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© 2023, The Author(s), under exclusive licence to Springer Science+Business Media, LLC, part of Springer Nature.A significant complication of HSCT is graft failure, although few studies focus on this problem in patients with inborn errors of immunity (IE). We explored outcome of second HSCT for IEI by a retrospective, single-centre study between 2002 and 2022. Four hundred ninety-three patients underwent allogeneic HSCT for severe combined immunodeficiency (SCID; n = 113, 22.9%) or non-SCID IEI (n = 380, 77.1%). Thirty patients (6.0%) required second HSCT. Unconditioned infusion or no serotherapy at first HSCT was more common in patients who required second transplant. Median interval between first and second HSCT was 0.97 years (range: 0.19–8.60 years); a different donor was selected for second HSCT in 24/30 (80.0%) patients. Conditioning regimens for second HSCT were predominately treosulfan-based (with thiotepa: n = 18, 60.0%; without, n = 6, 20.0%). Patients received grafts from peripheral blood stem cell (n = 25, 83.3%) or bone marrow (n = 5, 16.7%) with median stem cell dose 9.5 × 106 CD34 + cells/kilogram (range: 1.4–32.3). Median follow-up was 1.92 years (0.22–16.0). Overall survival was 80.8% and event-free survival was 64.7%. Four patients died, two of early-transplant related complications, and two of late sepsis post-second HSCT. Three patients required third HSCT; all are alive with 100% donor chimerism. Cumulative incidence of acute graft-versus-host disease was 28.4%, (all grade I–II). Viral reactivation was seen in 13/30 (43.3%) patients, including HHV6 (n = 6), CMV (n = 4), and adenovirus (n = 2). At latest follow-up, 25/26 surviving patients have donor chimerism ≥ 90% and 16/25 (64.0%) have discontinued immunoglobulin replacement. Second HSCT offers IEI patients with graft failure curative treatment with good overall survival and immunological recovery.

Publication metadata

Author(s): Mehta P, Tsilifis C, Lum SH, Slatter MA, Hambleton S, Owens S, Williams E, Flood T, Gennery AR, Nademi Z

Publication type: Article

Publication status: Published

Journal: Journal of Clinical Immunology

Year: 2023

Volume: 43

Pages: 1812–1826

Online publication date: 14/07/2023

Acceptance date: 06/07/2023

ISSN (print): 0271-9142

ISSN (electronic): 1573-2592

Publisher: Springer


DOI: 10.1007/s10875-023-01549-w

PubMed id: 37452206


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