Hematology · 3 h ago
Phase 2 study evaluates three-drug regimen for BPDCN
A phase 2 study enrolled 27 patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN) to receive tagraxofusp, azacitidine, and venetoclax. Composite complete remission rates were 88% in previously untreated patients and 64% in those with relapsed or refractory disease.
- Phase 2 study included 27 patients with BPDCN.
- Composite remission: 88% untreated; 64% relapsed or refractory.
- Capillary leak syndrome occurred in 15%, mostly grade 2.
- Small, non-comparative study; confirmatory evidence is needed.
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an aggressive malignancy with high CD123 expression. In this phase 2 study, 27 patients received 28-day cycles of tagraxofusp, azacitidine, and venetoclax: 16 had previously untreated disease and 11 had relapsed or refractory disease. The primary reported response measure was composite complete remission, including complete remission, remission with incomplete count recovery, and clinical remission.
Composite complete remission rates were 88% in the untreated cohort and 64% in the relapsed or refractory cohort. Median duration of response was not reached in the untreated group and was 7.2 months in the relapsed or refractory group. In the untreated cohort, two-year overall and progression-free survival were 65% and 53%, respectively; median overall survival in the relapsed or refractory cohort was 8.4 months. Capillary leak syndrome occurred in 15% of patients, mostly at grade 2. Allogeneic stem cell transplantation in remission followed in 63% of untreated and 55% of relapsed or refractory patients.
These results indicate activity in both groups, but the study was small and phase 2; the abstract does not establish comparative benefit or a new standard of care. The trial was registered as NCT03113643.
In practice
For patients with BPDCN, this phase 2 study provides evidence that tagraxofusp, azacitidine, and venetoclax can produce remissions in both previously untreated and relapsed or refractory disease. The reported composite remission rates were 88% and 64%, respectively, and capillary leak syndrome occurred in 15%, mostly at grade 2. These results may inform specialist discussion of treatment options, but the 27-patient, non-comparative study does not establish superiority or justify treating the regimen as a new standard.
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Blood: Tagraxofusp, azacitidine, and venetoclax for blastic plasmacytoid dendritic cell neoplasm ↗This is an automated AI-condensed summary that has not yet been reviewed by an editor. Always consult the full item at the original source.