Clinical outcomes of individualized busulfan-dosing in hematopoietic stem cell transplantation in Chinese children undergoing with therapeutic drug monitoring

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 pati...

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Published in:Bone marrow transplantation (Basingstoke) 2022-03, Vol.57 (3), p.473-478
Main Authors: Shao, Duan-Fang, Li, Jun-Hui, Hu, Tao, Zhang, Zhao-Xia, Zhang, Lei, Li, Juan-Juan, Cao, Jing, Feng, Shun-Qiao, Tang, Rui-Hong, Zhong, Di-Xiao, Song, Ze-Liang, Yue, Mei, Hu, Meng-Ze, Xuan, Li-Tian, Zhai, Meng-Na, Zhang, Hai-Feng, Wang, Xiang-Yan, Shi, Xiao-Dong, Liu, Rong
Format: Article
Language:English
Subjects:
Adolescent
Busulfan
Busulfan - adverse effects
Child
Child, Preschool
China
Conditioning
Dosage
Drug dosages
Drug Monitoring
Hematopoietic Stem Cell Transplantation
Hematopoietic stem cells
Hemorrhage
Hemorrhagic cystitis
Humans
Infant
Monitoring
Patients
Pediatrics
Pharmacokinetics
Retrospective Studies
Stem cell transplantation
Stem cells
Survival
Telemedicine
Therapeutic drug monitoring
Transplantation
Transplantation Conditioning - adverse effects
Veno-occlusive disease
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Summary: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.
ISSN:0268-3369
1476-5365
DOI:10.1038/s41409-021-01545-x