Hematopoietic Cell Transplantation for Severe Combined Immunodeficiency Patients: a Japanese Retrospective Study.
J Clin Immunol
; 41(8): 1865-1877, 2021 11.
Article
em En
| MEDLINE
| ID: mdl-34448087
ABSTRACT
PURPOSE:
Hematopoietic 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.METHODS:
A 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).RESULTS:
The 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).CONCLUSION:
We 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.Palavras-chave
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Imunodeficiência Combinada Severa
/
Transplante de Células-Tronco Hematopoéticas
/
Transplante de Células-Tronco de Sangue do Cordão Umbilical
Tipo de estudo:
Etiology_studies
/
Observational_studies
/
Prognostic_studies
Limite:
Adolescent
/
Child
/
Child, preschool
/
Female
/
Humans
/
Infant
/
Male
País como assunto:
Asia
Idioma:
En
Ano de publicação:
2021
Tipo de documento:
Article