ArticleJournal of clinical immunology2021
Hematopoietic Cell Transplantation for Severe Combined Immunodeficiency Patients: a Japanese Retrospective Study.
Article in Journal of clinical immunology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers, 2 of them syntheses that pooled it.
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Who cites it
20 citing papers in PubMed, 2 syntheses or guidelines pooled it, 35 citations in OpenAlex.
- Newborn screening for severe combined immunodeficiency in the NHS newborn blood spot screening programme: a systematic review.Health technology assessment (Winchester, England) · 2026Pooled it
- Late effects after allogeneic hematopoietic stem cell transplantation in patients with primary immunodeficiency.Frontiers in immunology · 2026Pooled it
- Clinical characteristics and risk factors associated with ICU-acquired infections in sepsis: A retrospective cohort study.Frontiers in cellular and infection microbiology · 2022Trial
- Nationwide TREC/KREC-based newborn screening and clinical outcomes in Japan.Journal of human immunity · 2026Article
- Allogeneic Hematopoietic Cell Transplantation for Congenital Athymia: A Nationwide Retrospective Study in Japan.Journal of clinical immunology · 2026Article
- Outcomes following matched sibling donor transplantation for severe combined immunodeficiency: a report from the PIDTC.Blood advances · 2026Article
- Newborn Screening Followed By Early Treatment is Essential to Improve Survival in SCID.Journal of clinical immunology · 2025Article
- Survival After Hematopoietic Stem Cell Transplantation in Severe Combined Immunodeficiency (SCID): A Worldwide Review of the Prognostic Variables.Clinical reviews in allergy & immunology · 2024Review
- Reducing Mortality and Morbidity in Children with Severe Combined Immunodeficiency in Switzerland: the Role of Newborn Screening.Journal of clinical immunology · 2024Article
- Perspectives in newborn screening for SCID in Japan. Case report: newborn screening identified X-linked severe combined immunodeficiency with a novelFrontiers in immunology · 2024Article
- Unmasking inborn errors of immunity: identifying the red flags of immune dysregulation.Frontiers in immunology · 2024Review
- Allogeneic Hematopoietic Stem Cell Transplantation Activity in Inborn Errors of Immunity in Russian Federation.Journal of clinical immunology · 2023Article
- Initial manifestations in Patients with Inborn Errors of Immunity Based on Onset Age: a Study from a Nationwide Survey in Japan.Journal of clinical immunology · 2023Article
- Clinical and Genetic Characterization of Patients with Artemis Deficiency in Japan.Journal of clinical immunology · 2023Article
- Unresolved issues in allogeneic hematopoietic cell transplantation for non-malignant diseases.International journal of hematology · 2022Review
- Conditioning regimens for inborn errors of immunity: current perspectives and future strategies.International journal of hematology · 2022Review
- Hematopoietic Cell Transplantation for Inborn Errors of Immunity Other than Severe Combined Immunodeficiency in Japan: Retrospective Analysis for 1985-2016.Journal of clinical immunology · 2022Article
- Population pharmacokinetics and initial dose optimization of tacrolimus in children with severe combined immunodeficiency undergoing hematopoietic stem cell transplantation.Frontiers in pharmacology · 2022Article
- Endocrinopathies in Inborn Errors of Immunity.Frontiers in immunology · 2021Review
- Centralized rapid genetic diagnosis of combined immunodeficiency in Japan.Pediatrics international : official journal of the Japan Pediatric SocietyArticle
Corrections and comments
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Authors and funding
21 authors at 15 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
purposeHematopoietic cell transplantation (HCT) is a curative therapy for patients with severe combined immunodeficiency (SCID). Here, we conducted a nationwide study to assess the outcome of SCID patients after HCT in Japan.
methodsA cohort of 181 SCID patients undergoing their first allogeneic HCT in 1974-2016 was studied by using the Japanese national database (Transplant Registry Unified Management Program, TRUMP).
resultsThe 10-year overall survival (OS) of the patients who received HCT in 2006-2016 was 67%. Umbilical cord blood (UCB) transplantation was performed in 81 patients (45%). The outcomes of HCT from HLA-matched UCB (n = 21) and matched sibling donors (n = 22) were comparable, including 10-year OS (91% vs. 91%), neutrophil recovery (cumulative incidence at 30 days, 89% vs. 100%), and platelet recovery (cumulative incidence at 60 days, 89% vs. 100%). Multivariate analysis of the patients who received HCT in 2006-2016 demonstrated that the following factors were associated with poor OS: bacterial or fungal infection at HCT (hazard ratio (HR): 3.8, P = 0.006), cytomegalovirus infection prior to HCT (HR: 9.4, P = 0.03), ≥ 4 months of age at HCT (HR: 25.5, P = 0.009), and mismatched UCB (HR: 19.8, P = 0.01).
conclusionWe showed the potential of HLA-matched UCB as a donor source with higher priority for SCID patients. We also demonstrated that early age at HCT without active infection is critical for a better prognosis, highlighting the importance of newborn screening for SCID.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.