Table III.

Summary of important studies using clofarabine-containing regimen prior to allogeneic stem cell transplant in patients with AML and high risk MDS.

Study design	Number of patients	Regimen	Baseline disease status	Median age	Outcome
Phase II	7	As a pre-AHSCT conditioning regimen: clofarabine 40 mg/m 2 IV on days −6 to −2; cytarabine 1 g/m 2 IV on days −6 to −2; 1 mg/kg of antithymocyte globulin (ATG) on day −4 and 2.5 mg/kg of ATG on days −3 and −2	Three patients with MDS, four with AML	54	3/7 expired on days 15, 26 and 32; ¾ relapsed with a median time to progression of 152 days
Phase I	15	Busulfan 0.8 mg/kg every 6 h IV on days −6 to −3 and clofarabine 30–60 mg/m 2 per day on days −6 to −2	Relapsed/refractory AML or ALL	48	1-year EFS and OS rates were 53% and 60%, respectively
Retrospective	17	As a cytoreductive regimen prior to AHSCT: 30–40 mg/m2 daily for 5 days with plans to initiate conditioning during the nadir	Relapsed/refractory AML	58	One-year PFS and OS rates were 25 and 38%, respectively. Two patients were alive in remission at 18 and 52 months
Retrospective	27	AHSCT after treatment with clofarabine and ara-C for 5 days and reduced intensity conditioning (4 Gy total body irradiation/cyclophosphamide/ATG)	High-risk, relapsed or refractory AML or MDS	58	The 2-year OS and RFS rates were 56% and 52%, respectively; seven patients relapsed within a median of 5.7 months

AML, acute myeloid leukemia; MDS, myelodysplastic syndrome; AHSCT, allogeneic hematopoietic stem cell transplant; ALL, acute lymphoblastic leukemia; EFS, event-free survival; OS, overall survival; PFS, progression-free survival; RFS, relapse-free survival.