Phase I/II trial shows potential safety and efficacy of venetoclax with FLAMSA-Treosulfan in high-risk patients.
Key Points
This research aims to assess the safety and preliminary efficacy of combining venetoclax with the FLAMSA-Treosulfan regimen in high-risk myeloid malignancies.
Conducted a Phase I/II trial with a classical 3+3 design.
Enrolled patients with high-risk MDS, CMML, or low blast count AML who received escalating doses of Venetoclax.
Administered FLAMSA treatment followed by high-dose Treosulfan, followed by alloHSCT with GvHD prophylaxis.
All nine patients achieved neutrophil and platelet recovery after a median of 14 and 16 days, respectively.
No dose limiting toxicity was observed, and patients remained alive, with 8 in complete remission.
Acute GvHD occurred in 8 patients, while 3 patients experienced relapse with 2 achieving CR after salvage therapy.