Recent Advances in Hematopoietic Stem Cell Transplantation for Childhood Acute Lymphoblastic Leukemia

Jude, Veronica H.; Chan, Ka Wah

doi:10.1007/s11899-010-0050-1

Cited by 4 publications

(1 citation statement)

References 45 publications

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“…Treatment outcomes in pediatric acute lymphoblastic leukemia (ALL) have dramatically improved, but some highrisk patients still suffer from poor outcomes. Hematopoietic stem cell transplantation (HSCT) can be a curative treatment option for these high-risk or relapsed patients [1][2][3][4][5]. The usual conditioning regimens for pediatric ALL include total body irradiation (TBI) [6][7][8], but TBI often causes serious sequelae, such as growth impairment, endocrinologic and metabolic problem, and secondary malignancies [9,10].…”

Section: Introductionmentioning

confidence: 99%

Favorable Outcome of Hematopoietic Stem Cell Transplantation Using a Targeted Once-Daily Intravenous Busulfan–Fludarabine–Etoposide Regimen in Pediatric and Infant Acute Lymphoblastic Leukemia Patients

Lee

Kang

Kim

et al. 2015

Biology of Blood and Marrow Transplantation

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Conditioning regimens for pediatric acute lymphoblastic leukemia (ALL) usually include total body irradiation (TBI), but TBI may result in serious sequelae. Busulfan and cyclophosphamide have been used as an alternative to TBI. Etoposide also has been widely used to enhance antileukemic effect. However, toxicities have been reported in some studies using busulfan, cyclophosphamide, and etoposide regimen. A reduced toxicity myeloablative regimen using busulfan and fludarabine showed promising results. Also, therapeutic drug monitoring (TDM) and administration of targeted doses of busulfan have been recommended to improve the outcome of hematopoietic stem cell transplantation (HSCT). In this study, we evaluated the outcome of HSCT using a targeted once-daily i.v. busulfan-fludarabine-etoposide (BuFluVP) regimen in pediatric and infant ALL. Busulfan (age ≥ 1 year, 120 mg/m(2); age < 1 year, 80 mg/m(2)) was administered once daily as the first dose on day -8, and a targeted dose of busulfan was used according to the TDM results on days -7 to -5. Forty-four patients were evaluated. Donor-type neutrophil engraftment was achieved in all patients. Veno-occlusive disease occurred in 7 patients (15.9%), but all patients were successfully treated. Cumulative incidence of treatment-related mortality and relapse were 9.1% and 9.9%, respectively. One-year overall survival and event-free survival rates of all patients were 86.2% and 83.8%, respectively. Twelve patients (27.3%) were infants at diagnosis, and their 1-year overall survival rate was 83.3%. Our study demonstrated that HSCT using a targeted once-daily i.v. BuFluVP regimen showed favorable outcomes and could be an option for HSCT in pediatric and infant ALL.

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