. Author manuscript; available in PMC: 2014 Aug 7.

Published in final edited form as: Curr Opin Hematol. 2014 Mar;21(2):123–130. doi: 10.1097/MOH.0000000000000016

Table 1.

Selected novel agents under active investigation for patients with myelodysplastic syndromes

Agent	Patient population	Response
Clofarabine [51]	Higher-risk MDS (n = 58)	ORR: 29–41% Median OS with response: 13.4 months Median OS: 7.4 months
Oral azacitidine [58]	MDS, CMML, AML (n = 41)	ORR (previously-treated): 35% (6/17) ORR (treatment-naive): 73% (11/15)
Rigosertib [59]	MDS (n =60) HMA failure (n =39)	31% (16/51) ≥ 50% blast decrease Median OS with response: 11 months
Sapcitabine [60]	Phase 1 refractory AML/MDS (n = 47)	ORR: 28%
Erlotinib [61]	HMA failure higher-risk MDS (n = 35)	ORR (evaluable): 19% (5/26) Median OS with response: 16.8 months Median OS: 6.8 months
Dasatinib [62]	HMA failure higher-risk MDS, CMML, AML (n = 18)	ORR: 16.7%

AML, acute myeloid leukemia; CMML, chronic myelomonocytic leukemia; HMA, hypomethylating agents; MDS, myelodysplastic syndromes; ORR, overall response rate; OS, overall survival.