Conditions: Myelodysplastic Syndrome (MDS)
Keywords: MDS bone marrow anemia cytopenia transfusion dependance EPO ESA erythropoietin LBH589 Myelodysplastic Syndromes hematopoietic improvement IPSS Low IPSS Int-1 HI-E HDAC Inhibitor HDAC-I DAC-I Deacetylase-Inhibitor Histone Deacetylase-Inhibitor red blood cell transfusions
Allocation: NON_RANDOMIZED | Intervention model: SINGLE_GROUP
Enrollment: 34
Healthy volunteers: no

Brief summary:
This study assessed the efficacy and safety of LBH589 as single agent and in combination with ESA in red blood cell transfusion-dependent Low and Int-1 MDS patients being either refractory to ESA or with a low probability of response. The study had a non-randomized core phase followed by a randomized phase.

Detailed description:

Arms:
arm 1: During the core phase, all participants received oral LBH589 40 mg (30 mg after a protocol amendment) for 4 months. During the randomization phase, participants with hematological improvement of the erythropoetic system (HI-E) and participants with stable disease, who were randomized to single agent LBH589, continued on single agent LBH589 40mg/30mg for an additional 4 months. arm 2: During the randomized phase, participants randomized to LBH589 + Epoetin Alfa (ESA) received oral LBH589 40mg/30mg + ESA 30000 international units (IU)/week injected subcutaneously for 4 months.

Interventions: intervention 1: LBH589 intervention 2: Epoetin Alfa
Intervention descriptions: intervention 1: LBH589 was supplied at dose strengths of 5 mg or 20 mg hard gelatin capsules. intervention 2: Epoetin alfa was supplied as 10000 IU/1 mL in a ready-to-use syringe.

Protocol document: