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

Abstract

PurposeHematopoietic 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.MethodsData from the Japanese national database (Transplant Registry Unified Management Program, TRUMP) for 567 patients with non-SCID IEI, who underwent their first HCT between 1985­ and 2016, were retrospectively analyzed.ResultsThe 10-year overall survival (OS) and event-free survival (EFS) was 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), being 79% and 81%, respectively. HCT from unrelated cord blood (URCB), accounting for 27% 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, hematologic recovery, or retransplantation incidence. Multivariate analyses of data on those receiving HCT during the 2006–2016 period revealed that respiratory impairment at HCT was associated with poor OS (hazard ratio [HR], 2.3; P = 0.01) and that URCB (HR, 2.7; P = 0.003) and related donor other than MSD (HR, 2.7; P = 0.02) were associated with poor EFS.ConclusionsWe present the 1985–2016 status of HCT for non-SCID IEI in Japan with sufficient statistical power, highlighting the potential of URBM as an alternative donor and the substantial applicability of URCB. Detailed evaluation is needed for optimizing the HCT strategy for each IEI.