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 Journal Article


Authors: Hochberg, J.; Zahler, S.; Geyer, M. B.; Chen, N.; Krajewski, J.; Harrison, L.; Militano, O.; Ozkaynak, M. F.; Cheerva, A. C.; Talano, J.; Moore, T. B.; Gillio, A. P.; Walters, M. C.; Baxter-Lowe, L. A.; Hamby, C.; Cairo, M. S.
Article Title: 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
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/m2), cytarabine 1000 mg/m2, 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. © 2018, Macmillan Publishers Limited, part of Springer Nature.
Journal Title: Bone Marrow Transplantation
Volume: 54
Issue: 2
ISSN: 0268-3369
Publisher: Nature Publishing Group  
Date Published: 2019-02-01
Start Page: 226
End Page: 235
Language: English
DOI: 10.1038/s41409-018-0247-9
PROVIDER: scopus
PUBMED: 29899571
DOI/URL:
Notes: Article -- Export Date: 1 March 2019 -- Source: Scopus
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MSK Authors
  1. Mark Blaine Geyer
    14 Geyer