A phase II trial of weekly nab-paclitaxel for progressive and symptomatic desmoid tumors

Area of Science:

• Oncology
• Medical Oncology
• Clinical Trials

Background:

• Desmoid fibromatosis (DF) is a rare mesenchymal neoplasm with aggressive potential.
• Symptomatic and progressing DF patients require novel systemic therapies.
• Current treatment options for DF have limitations, necessitating further research.

Purpose of the Study:

• To evaluate the efficacy and safety of a short course of weekly nab-paclitaxel in patients with symptomatic or progressing DF.
• To determine if nab-paclitaxel can achieve significant clinical and radiological responses.
• To assess progression-free survival (PFS) and toxicity profiles of this treatment regimen.

Main Methods:

• A multicenter, phase II clinical trial (NCT03275818) involving 40 eligible patients with symptomatic/progressing DF.
• Patients received three cycles of weekly nab-paclitaxel.
• Primary endpoint: composite of RECIST 1.1 overall response rate (ORR) and/or clinical response (≥2 points improvement in Brief Pain Inventory-Short Form [BPI-SF] worst pain).
• Secondary endpoints included toxicity, PFS, and pain variation.

Main Results:

• The study met its primary endpoint: 7 (20%) patients achieved RECIST partial response, and 31 (89%) experienced ≥2 points pain reduction.
• Nab-paclitaxel therapy was well-tolerated with manageable toxicity.
• Median 12- and 24-month PFS rates were 91% and 84%, respectively.
• Clinical progression PFS rates at 12 and 24 months were 85% and 74%.

Conclusions:

• A short course of nab-paclitaxel demonstrates significant activity and safety in patients with progressing/symptomatic DF.
• The treatment achieves rapid and sustained responses, including substantial pain relief.
• Nab-paclitaxel represents a promising therapeutic option for this challenging patient population.