Outcome of treosulfan-based reduced-toxicity conditioning regimens for HSCT in high-risk patients with primary immune deficiencies.
Pediatr Transplant
; 22(7): e13266, 2018 11.
Article
em En
| MEDLINE
| ID: mdl-29992714
ABSTRACT
INTRODUCTION:
HSCT is the curative therapeutic option in PIDs. Due to the increase in survival rates, reduced-toxicity conditioning regimens with treosulfan have become another alternative. The purpose of this retrospective study was to analyze the outcome of treosulfan-based conditioning before HSCT for patients with PID.METHOD:
A total of 15 patients that received a treosulfan-based conditioning regimen for HSCT were recruited. Type of diagnosis, donor and stem cell source, pretransplant organ damage, infections, engraftment, chimerism, and transplant-related toxicities were analyzed.RESULTS:
At a median follow-up time of 32 months, the overall survival was 86.7%. Following HSCT, 14 of 15 patients had engraftment, with 86.7% of the cohort having full-donor chimerism. The most common toxicity was seen on the skin (53.3%). Acute GVHD and chronic GVHD were documented in 53% and 20% of the study population, respectively. Although the cohort consisted of patients with pretransplant liver damage, SOS manifestations were documented in 20%.CONCLUSION:
Treosulfan-based conditioning regimens before HSCT are associated with lower toxicity compared to myeloablative regimens, are safe, and have high engraftment rates with full-donor chimerism in patients having PID, regardless of the specified genetic diagnosis and donor type.Palavras-chave
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Base de dados:
MEDLINE
Assunto principal:
Bussulfano
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Transplante de Células-Tronco Hematopoéticas
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Condicionamento Pré-Transplante
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Síndromes de Imunodeficiência
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Imunossupressores
Idioma:
En
Ano de publicação:
2018
Tipo de documento:
Article
País de afiliação:
Turquia