Hematopoietic Cell Transplantation for Inborn Errors of Immunity Other than Severe Combined Immunodeficiency in Japan: Retrospective Analysis for 1985–2016

Purpose Hematopoietic cell transplantation (HCT) is a curative therapy for most patients with inborn errors of immunity (IEI). We conducted a nationwide study on HCT for patients with IEI other than severe combined immunodeficiency (non-SCID) in Japan. Methods Data from the Japanese national databas...

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Bibliographic Details
Published in:Journal of clinical immunology 2022-04, Vol.42 (3), p.529-545
Main Authors: Miyamoto, Satoshi, Umeda, Katsutsugu, Kurata, Mio, Yanagimachi, Masakatsu, Iguchi, Akihiro, Sasahara, Yoji, Okada, Keiko, Koike, Takashi, Tanoshima, Reo, Ishimura, Masataka, Yamada, Masafumi, Sato, Maho, Takahashi, Yoshiyuki, Kajiwara, Michiko, Kawaguchi, Hiroshi, Inoue, Masami, Hashii, Yoshiko, Yabe, Hiromasa, Kato, Koji, Atsuta, Yoshiko, Imai, Kohsuke, Morio, Tomohiro
Format: Article
Language:English
Subjects:
Biomedical and Life Sciences
Biomedicine
Bone marrow
Cord blood
Engraftment
Graft vs Host Disease - etiology
Hematopoietic Stem Cell Transplantation - adverse effects
Humans
Immune system
Immunity
Immunology
Infectious Diseases
Internal Medicine
Japan - epidemiology
Leukocytes (neutrophilic)
Medical Microbiology
Original Article
Radiation
Retrospective Studies
Severe combined immunodeficiency
Severe Combined Immunodeficiency - diagnosis
Severe Combined Immunodeficiency - epidemiology
Severe Combined Immunodeficiency - therapy
Stem cell transplantation
Transplantation
Transplantation Conditioning - adverse effects
Transplants & implants
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Summary:Purpose Hematopoietic cell transplantation (HCT) is a curative therapy for most patients with inborn errors of immunity (IEI). We conducted a nationwide study on HCT for patients with IEI other than severe combined immunodeficiency (non-SCID) in Japan. Methods Data from the Japanese national database (Transplant Registry Unified Management Program, TRUMP) for 566 patients with non-SCID IEI, who underwent their first HCT between 1985 and 2016, were retrospectively analyzed. Results The 10-year overall survival (OS) and event-free survival (EFS) were 74% and 64%, respectively. The 10-year OS for HCT from unrelated bone marrow (URBM), accounting for 39% of HCTs, was comparable to that for HCT from matched sibling donor (MSD), 79% and 81%, respectively. HCT from unrelated cord blood (URCB), accounting for 28% of HCTs, was also common, with a 10-year OS of 69% but less robust engraftment. The intensity of conditioning was not associated with OS or neutrophil recovery; however, myeloablative conditioning was more frequently associated with infection-related death. Patients who received myeloablative irradiation showed poor OS. Multivariate analyses revealed that HCT in 1985–1995 (hazard ratio [HR], 2.0; P  = 0.03), URCB (HR, 2.0; P  = 0.01), and related donor other than MSD (ORD) (HR, 2.9; P  
ISSN:0271-9142
1573-2592
DOI:10.1007/s10875-021-01199-w