MDS: add-on pracinostat fails to improve outcomes in HMA nonresponders

  • Yalniz FF & al.
  • Br J Haematol
  • 29 Aug 2019

  • curated by David Reilly
  • Univadis Clinical Summaries
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Takeaway

  • 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. 

Study design

  • 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.

Key results

  • 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.

Limitations

  • The value of the findings is limited by insufficient exposure to the study drug.