Haematopoietic stem cell transplantation for inborn errors of immunity: 25-year experience from University of Malaya Medical Centre, Malaysia.
J Paediatr Child Health
; 56(3): 379-383, 2020 Mar.
Article
em En
| MEDLINE
| ID: mdl-31479560
ABSTRACT
AIM:
Inborn errors of immunity (IEI) comprise a heterogeneous group of disorders of the immune system, most of which are curable by haematopoietic stem cell transplantation (HSCT). We present a 25-year audit of HSCT for IEI at a tertiary-level academic hospital in Malaysia.METHODS:
Review of medical records of all cases of IEI who underwent HSCT between January 1993 and December 2018 at our centre. Diagnoses, complications, HSCT protocols and outcome data were studied.RESULTS:
There were 20 patients (19 boys) with a median age at diagnosis of 11 months (range 2 months to 12 years). Eleven of 19 (58%) had malnutrition at presentation. Donor sources were variable 13 (65%) matched sibling donor (MSD), 4 (20%) human leukocyte antigen-haploidentical donor (HD) and 3 (15%) matched unrelated donor (MUD). Conditioning regimens were physician-dependent and adapted to each patient's clinical status. Grades III-IV acute graft-versus-host disease occurred in two of three cases who received MUD grafts, 50% in those who received HD, and 8% in the MSD group. Transplant-related mortality at day +100 was 5%. With a median follow-up of 7.5 years, 18 (90%) patients are alive and free of infections.CONCLUSION:
Outcome of HSCT for IEI in our centre is comparable with international reports. HSCT results using HD and MUD grafts are also good despite challenges from acute graft-versus-host disease, providing a feasible alternative for patients without matched donors.Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Transplante de Células-Tronco Hematopoéticas
/
Doença Enxerto-Hospedeiro
Tipo de estudo:
Guideline
Limite:
Humans
/
Male
País/Região como assunto:
Asia
Idioma:
En
Revista:
J Paediatr Child Health
Assunto da revista:
PEDIATRIA
Ano de publicação:
2020
Tipo de documento:
Article
País de afiliação:
Malásia