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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 Transplant 2019 02;54(2):226-235



Pubmed ID




Scopus ID

2-s2.0-85048460040   5 Citations


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 40a??-a??52a??mg/m2), cytarabine 1000a??mg/m2, and 1200a??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, Pa??=a??0.011) and umbilical cord blood source (RR 2.17, CI95: 1.33-4.15, Pa??=a??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.

Author List

Hochberg J, Zahler S, Geyer MB, Chen N, Krajewski J, Harrison L, Militano O, Ozkaynak MF, Cheerva AC, Talano J, Moore TB, Gillio AP, Walters MC, Baxter-Lowe LA, Hamby C, Cairo MS


Julie-An M. Talano MD Professor in the Pediatrics department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Acute Disease
Antineoplastic Combined Chemotherapy Protocols
Child, Preschool
Hematopoietic Stem Cell Transplantation
Leukemia, Myeloid, Acute
Myeloablative Agonists
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Transplantation Conditioning
Transplantation, Homologous
Treatment Outcome
Whole-Body Irradiation
Young Adult