Hematopoietic Cell Transplantation for Chronic Granulomatous Disease in Japan

Yanagimachi, Masakatsu; Kato, Koji; Iguchi, Akihiro; Sasaki, Koji; Kiyotani, Chikako; Koh, Katsuyoshi; Kitahara, Takashi; Sano, Hideki; Shigemura, Tomonari; Muramatsu, Hideki; Okada, Keiko; Inoue, Masami; Tabuchi, Ken; Nishimura, Toyoki; Mizukami, Tomoyuki; Nunoi, Hiroyuki; Imai, Kohsuke; Kobayashi, Masao; Morio, Tomohiro

doi:10.3389/fimmu.2020.01617

Cited by 16 publications

(9 citation statements)

References 25 publications

(44 reference statements)

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“…The patients were more commonly complicated with infection or respiratory impairment at HCT (Table S1), which may also pose a risk for infection, concerning poor engraftment in URCBT. URCBT for CGD patients is reported to have poor engraftment in studies from Japan [42] and European countries [43], which is consistent with our results. Because the time between diagnosis and HCT was relatively long and urgent HCT is considered rare, URCB may be used for these diseases only on limited occasions.…”

Section: Discussionsupporting

confidence: 92%

“…Third, a precise analysis of each disease was not performed. For further detailed analysis, we have already published retrospective studies for each IEI from Japan [42,46,47] and will also perform such studies for other diseases in the future on behalf of the Hereditary Disorder Working Group of the JSTCT, collaborating with the Primary Immunode ciency Database in Japan [48] and the TRUMP.…”

Section: Discussionmentioning

confidence: 99%

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

et al. 2022

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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...

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Section: Discussionsupporting

confidence: 92%

Section: Discussionmentioning

confidence: 99%

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

et al. 2022

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“…Granulocyte transfusions post-HCT were overall well-tolerated and did not appear to be associated with an increased risk of graft failure. Two of 15 (13%) patients who received granulocytes post-HCT only developed graft failure, which is comparable to the reported rate of graft failure following HCT for CGD [ 19 – 21 ]. Both patients were successfully re-transplanted, again with prophylactic granulocyte transfusions.…”

Section: Discussionsupporting

confidence: 69%

“…Notably, 3 of 10 (30%) patients who received granulocytes in preparation for HCT had primary graft failure. This is higher than the 12–16% rate of graft failure following HCT reported in recent large cohorts of CGD patients [ 19 – 21 ]. All three patients had anti-HLA antibodies present; however, the role of granulocyte transfusions in graft failure for 2 of the patients is unclear given that donor-specific antibodies were not detected.…”

Section: Discussionmentioning

confidence: 60%

Granulocyte Transfusions in Patients with Chronic Granulomatous Disease Undergoing Hematopoietic Cell Transplantation or Gene Therapy

et al. 2022

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Granulocyte transfusions are sometimes used as adjunctive therapy for the treatment of infection in patients with chronic granulomatous disease (CGD). However, granulocyte transfusions can be associated with a high rate of alloimmunization, and their role in CGD patients undergoing hematopoietic cell transplantation (HCT) or gene therapy (GT) is unknown. We identified 27 patients with CGD who received granulocyte transfusions pre- (within 6 months) and/or post-HCT or GT in a retrospective survey. Twelve patients received granulocyte transfusions as a bridge to cellular therapy. Six (50%) of these patients had a complete or partial response. However, six of 10 (60%) patients for whom testing was performed developed anti-HLA antibodies, and three of the patients also had severe immune-mediated cytopenia within the first 100 days post-HCT or GT. Fifteen patients received granulocyte transfusions post-HCT only. HLA antibodies were not checked for any of these 15 patients, but there were no cases of early immune-mediated cytopenia. Out of 25 patients who underwent HCT, there were 5 (20%) cases of primary graft failure. Three of the patients with primary graft failure had received granulocyte transfusions pre-HCT and were subsequently found to have anti-HLA antibodies. In this small cohort of patients with CGD, granulocyte transfusions pre-HCT or GT were associated with high rates of alloimmunization, primary graft failure, and early severe immune-mediated cytopenia post-HCT or GT. Granulocyte transfusions post-HCT do not appear to confer an increased risk of graft failure. Supplementary Information The online version contains supplementary material available at 10.1007/s10875-022-01261-1.

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“…However, the group of HCT-treated patients demonstrated excellent survival rates, although the risks and benefits still need to be assessed in individual patients. Based on the significant progress of patients with CGD treated with HCT, it is regarded as the only known curative treatment with an improved life expectancy owing to its improved implementation over time [55] [56][57][58].…”

Section: Haematopoietic Stem Cell Transplantation (Hsct/hct)mentioning

confidence: 99%

Chronic Granulomatous Disease (CGD): Pathogens and Management

Justiz-Vaillant,

Williams-Persad,

Arozarena-Fundora

et al. 2023

Preprint

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Chronic granulomatous disease (CGD) is a primary immunodeficiency caused by a defect in the phagocytic function of the innate immune system owing to mutations in genes encoding the five subunits of the nicotinamide adenine dinucleotide (NADPH) oxidase enzyme complex. This review aimed to provide a comprehensive approach to the pathogens associated with chronic granulomatous disease (CGD) and its management. The topics covered included common pathogens infecting CGD patients, granulomas, and CGD-related infectious diseases, including invasive aspergillosis, Mulch pneumonitis, and liver abscesses. In addition, we also covered the differential diagnosis of CGD and its management. Patients with CGD, often children, have recurrent life-threatening infections and may develop infectious or inflammatory complications. The most common microorganisms observed in the patients with CGD are Staphylococcus aureus, Aspergillus spp, Candida spp, Nocardia spp, Burkholderia spp, Serratia spp, and Salmonella spp. Triple-dose antimicrobial prophylaxis with empiric regimens is recommended. Therefore, therapeutics against this disease must be optimised, considering the lack of curative therapies and the long-term side effects of prophylactic regimens. Consequently, allogeneic haematopoietic stem cell transplantation from a human leukocyte antigen-identical donor has been proven to cure CGD.

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Hematopoietic Cell Transplantation for Chronic Granulomatous Disease in Japan

Cited by 16 publications

References 25 publications

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

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

Granulocyte Transfusions in Patients with Chronic Granulomatous Disease Undergoing Hematopoietic Cell Transplantation or Gene Therapy

Chronic Granulomatous Disease (CGD): Pathogens and Management

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