NYMC Faculty Publications
The Safety and Efficacy of Clofarabine in Combination With High-Dose Cytarabine and Total Body Irradiation Myeloablative Conditioning and Allogeneic Stem Cell Transplantation in Children, Adolescents, and Young Adults (CAYA) With Poor-Risk Acute Leukemia
DOI
10.1038/s41409-018-0247-9
Journal Title
Bone Marrow Transplantation
First Page
226
Last Page
235
Document Type
Article
Publication Date
2-1-2019
Department
Pediatrics
Second Department
Pathology, Microbiology and Immunology
Abstract
Acute leukemias in children with CR3, refractory relapse, or induction failure (IF) have a poor prognosis. Clofarabine has single agent activity in relapsed leukemia and synergy with cytarabine. We sought to determine the safety and overall survival in a Phase I/II trial of conditioning with clofarabine (doses 40 - 52 mg/m(2)), cytarabine 1000 mg/m(2), and 1200 cGy TBI followed by alloSCT in children, adolescents, and young adults with poor-risk leukemia. Thirty-seven patients; Age 12 years (1-22 years); ALL/AML: 34:3 (18 IF, 10 CR3, 13 refractory relapse); 15 related, 22 unrelated donors. Probabilities of neutrophil, platelet engraftment, acute GvHD, and chronic GvHD were 94%, 84%, 49%, and 30%, respectively. Probability of day 100 TRM was 8.1%. 2-year EFS (event free survival) and OS (overall survival) were 38.6% (CI95: 23-54%), and 41.3% (CI95: 25-57%). Multivariate analysis demonstrated overt disease at time of transplant (relative risk (RR) 3.65, CI95: 1.35-9.89, P = 0.011) and umbilical cord blood source (RR 2.17, CI95: 1.33-4.15, P = 0.019) to be predictors of worse EFS/OS. This novel myeloablative conditioning regimen followed by alloSCT is safe and well tolerated in CAYA with very poor-risk ALL or AML. Further investigation in CAYA with better risk ALL and AML undergoing alloSCT is warranted.
Recommended Citation
Hochberg, J., Zahler, S., Chen, N., Harrison, L., Ozkaynak, M., Hamby, C., & Cairo, M. (2019). The Safety and Efficacy of Clofarabine in Combination With High-Dose Cytarabine and Total Body Irradiation Myeloablative Conditioning and Allogeneic Stem Cell Transplantation in Children, Adolescents, and Young Adults (CAYA) With Poor-Risk Acute Leukemia. Bone Marrow Transplantation, 54 (2), 226-235. https://doi.org/10.1038/s41409-018-0247-9
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