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

Abstract

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 immunode ciency (non-SCID) in Japan. MethodsData from the Japanese national database (Transplant Registry Uni ed Management Program, TRUMP) for 567 patients with non-SCID IEI, who underwent their rst 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 su cient 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.Hematopoietic cell transplantation (HCT) was rstly performed for a patient with severe combined immunode ciency (SCID) in 1968 [3]. Since then, HCT has been widely applied as a curative therapy for patients with IEI, especially for those with severe defects or dysregulation in cellular immunity. Unrelated cord blood (URCB) is commonly used in Japan and accounted for 33% of all allogenic HCTs during the period from 2009 to 2018 [4]. We previously performed a nationwide survey in Japan involving 88 patients with IEI who underwent unrelated cord blood transplantation (URCBT) and demonstrated an overall survival (OS) of 69% over 5 years [5]. However, no study has covered all HCTs for patients with IEI in Japan. Recently, we conducted a retrospective analysis of HCT for SCID in Japan. In this study, we conducted a nationwide retrospective analysis of HCT for patients with non-SCID IEI to provide an overview of the status and outcomes of HCTs and to develop strategies for HCT in Japan. Methods Data CollectionThis study was approved by the Institutional Ethics Committee of Tokyo Medical and Dental University. The participants (and/or their guardians) provided written informed consents and were registered in the Transplant Registry Uni ed Management Program (TRUMP), an electronic database of all HCTs performed in Japan established by the Japanese Society for Transplantation and Cellular Therapy (JSTCT) [6]. The patients with non-SCID IEI who underwent their r...

Oral Mucositis and Nutritional Status in Children Who Underwent Hematopoietic Cell Transplantation: A Comparison Between Nonmalignant and Malignant Primary Diseases

“…The 3-year OS rates in the current study were 73.0% for the NMD group and 73.4% for the MD group. These rates align with OS estimates from multicenter studies on HSCT and children with immunodeficiencies in Brazil [33], Colombia [34], and Japan [35]. For MD the OS appears to be higher compared to previous study conducted in Brazil [36] and to rates reported in other countries [37].…”

Allogeneic Hematopoietic Stem Cell Transplantation Activity in Inborn Errors of Immunity in Russian Federation

“…The OS in IEI after HSCT varies between countries; recent reports demonstrate lower rates of survival in low-income countries and higher rates in high-income countries. Thus, the reported OS is 0.61 in India [ 20 ], 0.63 in Colombia [ 21 ], 0.71 in Brazil [ 22 ], 0.67–0.74 in Japan, [ 23 , 24 ], 0.83 in the UK [ 8 ], and least 0.92 Germany [ 7 ]. In Russia, the OS in all IEI patients who received HSCT is 0.74, and since 2012, the OS has improved from 0.54 to 0.76.…”