Table I.

Summary of important studies using single-agent clofarabine in the treatment of adult patients with AML and MDS.

Reference	Phase of study	Number of patients	Patient characteristics	Median age (years)	Treatment schema	ORR (%)	CR (%)	Remarks
Kantarjian et al. (2003) [18]	II	39	Refractory AML and MDS	54	Clofarabine induction	56	38	ORR=87% for patients with AML with prolonged first CR
Krawczyk et al. (2010) [26]	II	8	Frontline therapy AML	67.5	Clofarabine induction ± consolidation	61	50	Induction mortality = 17%
Faderl et al. (2010) [28]	II	32	High risk MDS	70	Three doses of oral clofarabine	43	25	Low dose as efficacious, less toxic
Faderl et al. (2012) [27]	II	58	High risk MDS	68	Low vs. high dose clofarabine	36	26	Median OS = 7.4 months; low dose as efficacious, less toxic
Burnett et al. (2010) [44]	Two consecutive phase II	106	Frontline elderly AML	71	Reduced dose clofarabine induction and consolidation	48	32
Kantarjian et al. (2010) [45]	II	112	Frontline elderly AML	71	Reduced dose clofarabine induction and consolidation	46	38	Median OS = 42 weeks
Foran et al. (2003) [24]	II	40	Relapsed/refractory AML	55.6	Clofarabine induction and consolidation	5	0

AML, acute myeloid leukemia; MDS, myelodysplastic syndrome; ORR, overall response rate; CR, complete remission; OS, overall survival.