Clinical outcomes of individualized busulfan-dosing in hematopoietic stem cell transplantation in Chinese children undergoing with therapeutic drug monitoring.
Bone Marrow Transplant
; 57(3): 473-478, 2022 03.
Article
en En
| MEDLINE
| ID: mdl-35039622
ABSTRACT
To identify relationships between busulfan (Bu) exposure and outcomes of a cohort pediatric patients receiving hematopoietic stem cell transplantation (HSCT), along with a targeted busulfan-based conditioning regimen. We retrospectively evaluated targeted busulfan concentrations in 53 pediatric patients (age 0.4-16 years) who received busulfan 4 times daily according to recommended weight-based doses in a single-center analysis between 2018 and 2020. In this trial, individual busulfan pharmacokinetics were performed following dose 5 of the conditioning regimen. Twenty four of 53 patients (45.3%) studies did not require dose adjustments. Equal number of patients (24/53) required one dose adjustments while two-dose adjustment applied for 5 of 53 (9.4%). Twenty-one percent of the patients exhibited ll-lV aGVHD. The incidence of veno-occlusive disease (VOD) was in 3.8% of the 53 patients, while incidence of hemorrhagic cystitis (II-III) reached to 9.7%. Engraftment was successful in 98% of the 53 patients with relapse in 2% of cases. The probability of overall survival and disease-free survival at day 100 was 96% and 94%, respectively. In conclusion, therapeutic drug monitoring (TDM) and individualization of Bu dosage are essential to improve the efficacy and safety of busulfan-based regimen in Chinese pediatric HSCT recipients.
Texto completo:
1
Colección:
01-internacional
Asunto principal:
Busulfano
/
Trasplante de Células Madre Hematopoyéticas
Tipo de estudio:
Observational_studies
Límite:
Adolescent
/
Child
/
Child, preschool
/
Humans
/
Infant
País/Región como asunto:
Asia
Idioma:
En
Revista:
Bone Marrow Transplant
Asunto de la revista:
TRANSPLANTE
Año:
2022
Tipo del documento:
Article
País de afiliación:
China