- Ixazomib reduced 6-month historical treatment failure rate for chronic graft vs host disease (cGVHD).
Why this matters
- Patients with advanced cGVHD have limited treatment options for improved outcomes.
- Phase 2, single-arm trial at 6 institutions involving 50 adults with cGVHD.
- 16% moderate, 84% severe cGVHD.
- 80% classic, 20% overlap cGVHD.
- 52% had ≥4 affected organs.
- 78% had ≥3 previous lines of systemic therapy.
- All had failure of ≥1 previous line of systemic immune suppressive (IS) therapy.
- Patients received 4 mg oral ixazomib on days 1, 8, and 15 of 28-day cycles for 6 cycles (or more for responders).
- 26 patients completed 6 months of planned therapy with dose reductions primarily due to thrombocytopenia, fatigue, diarrhea, and infection; all continued some IS at 12 months.
- 6-month treatment failure rate (death, relapse, or additional IS therapy) was 28% vs historical 44% (P=.01).
- Overall response rate was 34% at 6 months.
- 6- and 12-month survival was 92% and 90%, respectively.
- 38% experienced severe adverse events, with 2 of 5 deaths potentially treatment-related.