Study of Efficacy and Safety of Eltrombopag in Lower-risk MDS Patients With Platelet Transfusion Dependence

Inclusion Criteria:

Patients diagnosed with MDS according to the WHO classification revised 4th edition by investigator assessment with one of the following prognostic risk categories, based on the International

Prognostic Scoring System (IPSS-R):

very low (0-1.5)
low (2-3)
intermediate risks (3.5-4.5) All following criteria for prognostic variables per IPSS-R should be met.
Bone marrow blast < 5% (per both investigator's assessment and central review)

Cytogenetic very good, good or intermediate risk corresponding to IPSS-R

Platelet transfusion dependence
Refractory, intolerant to, or ineligible for MDS treatments
Eastern Cooperative Oncology Group (ECOG) Performance status (PS) 0 or 1

Exclusion Criteria:

Patients with a history of prior administration of eltrombopag, romiplostim, or other TPO-RA
Therapy-related MDS per WHO classification revised 4th edition
MDS/myeloproliferative neoplasms including chronic myelomonocytic leukaemia per the WHO classification revised 4th edition
MDS with excess blasts (EB) per WHO classification revised 4th edition
Known history of IPSS-R high or very high risk MDS
Currently receiving treatments for MDS (e.g., HMA, cyclosporine A (CsA) or lenalidomide). Supportive treatment with erythropoiesis-stimulating agents (ESAs) in anemic patients or granulocyte-colony stimulating factor (G-CSF) in patients with severe neutropenia and recurrent infections is allowed if at stable dosage for 3 months prior to screening and continued at the same dosing/schedule until the optimal dose of eltrombopag has been established.
Patients scheduled for hematopoietic stem cell transplantation
Bone marrow fibrosis that leads to an inability to aspirate adequate bone marrow sample
Known thrombophilic risk factors (except in cases where potential benefits of participating in the study outweighed potential risks of thromboembolic events(TEE), as determined by the investigator)

Other protocol-defined inclusion/exclusion criteria may apply.