IDH2 (AG 221) Inhibitor in Patients With IDH2 Mutated Myelodysplastic Syndrome

Inclusion criteria

• . Myelodysplastic syndrome according to World Health Organization (WHO) classification including non-proliferative AML up to 29% of Bone marrow (BM) blast
• . Age ≥ 18 years
• . Belonging to one of the following categories:
• . higher risk MDS (IPSS int-2, high) without response to azacitidine (Complete response (CR),Partial Response (PR), stable disease with HI) after at least 6 cycles , or relapsing after a response but without overt progression (defined by at least doubling of marrow blasts, compared to pre azacitidine bone marrow, or AML progression beyond 30% blasts)
• . Untreated higher risk MDS (IPSS int-2, high) without life threatening cytopenia including absolute neutrophil count (ANC) \<500/mm3 or any recent severe infections and/or platelets below 30,000/mm3 and any bleeding symptom
• . Lower risk MDS with resistance or loss of response to a previous treatment with epoetin alpha/ beta (≥60000 U/w) or Darbopoetin (≥250 ug/w) given for at least 12 weeks and red blood cell (RBC) transfusion requirement at least 2 U/8 weeks in the previous 16 weeks.
• . Presence of IDH2 mutation in either blood or marrow prior to start of therapy
• . Normal renal function, defined by creatinine less than 1.5 times the upper limit of normal, creatinine clearance (Modification of diet in renal disease) (MDRD) ≥ 50 mL/min.

Exclusion criteria