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.