This is an open-label study designed to evaluate the safety and efficacy of the selective inhibitor of nuclear export (SINE) compound, Selinexor given orally to patients with transfusion-dependent, EPO-refractory lower-risk MDS (Low risk and Intermediate-1 as defined by IPSS).
This is a single-arm, open-label, study designed to evaluate the safety and efficacy of the selective inhibitor of nuclear export (SINE) compound, Selinexor given orally to patients with transfusion-dependent, EPO-refractory lower-risk MDS (Low risk and Intermediate-1 as defined by IPSS). Patients will be dosed at the clinic on clinic visit days and received Selinexor for dosing at home on additional days. Patients will be evaluated for disease response according to the 2006 IWG response criteria for MDS. This includes evaluation for altering the natural history of disease, cytogenic response hematologic improvement, and quality of life.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
60 mg on Day 1 of each week for a 4 week cycle, given for ≥6 cycles.
Hematologic improvement (based on the 2006 International Working Group (IWG) response criteria)
Hematologic improvement in erythroid, platelet, and neutrophil counts, evaluated based on the 2006 International Working Group (IWG) response criteria for MDS.
Time frame: 6 months
Overall response rate
Overall response is defined as complete remission (CR) or partial remission (PR).
Time frame: 6 months
Quality of Life (QOL-E) MDS Questionnaire)
Evaluate quality of life using the Quality of Life-E (QOL-E) MDS Questionnaire
Time frame: 6 months
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