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

Prior treatment

• Patients with a history of prior administration of eltrombopag, romiplostim, or other TPO-RA
• Therapy-related MDS per WHO classification revised 4th edition
• Currently receiving treatments for MDS (e.g., HMA, cyclosporine A (CsA) or lenalidomide). Supportive treatment with erythropoiesis-stimulating agents (ESAs) or erythroid mutation agent 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

Other illness

• Known history of IPSS-R high or very high risk MDS

Other

• 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
• 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.

• 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