- The addition of pracinostat to a hypomethylating agent (HMA) failed to improve outcomes in patients with myelodysplastic syndromes (MDSs) who did not respond to prior HMA monotherapy.
Why this matters
- Only 30%-40% of patients respond to HMAs.
- Phase 2 study to investigate addition of pracinostat to an HMA in patients with MDS who failed to respond to prior HMA monotherapy.
- Patients were assigned to 2 groups:
- Group 1 (n=39): patients with primary/secondary HMA failure.
- Group 2 (n=6): patients who achieved stable disease but not response with prior HMA monotherapy.
- Median patient age: 74 (range, 53-86) years.
- Funding: MEI Pharma.
- Among all patients:
- 2% achieved complete remission (CR).
- 16% achieved CR in bone marrow.
- 40% achieved stable disease.
- Median OS:
- Group 1: 5.7 (95% CI, 3.2-8.8) months.
- Group 2: 5.6 (95% CI, 0.6-not available) months.
- The most common grade ≥3 adverse events included: thrombocytopenia (33%), neutropenia (31%), anemia (29%), and febrile neutropenia (27%).
- 27% discontinued study treatment at a median of 1 (range, 1-6) month.
- The value of the findings is limited by insufficient exposure to the study drug.