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

Abstract

Desmoid tumors; Phase II trial; Paclitaxel

Tumores desmoides; Ensayo de fase II; Paclitaxel

Tumors desmoides; Assaig de fase II; Paclitaxel

Desmoid fibromatosis (DF) are mesenchymal neoplasms, with potential aggressive course and relevant clinical impact. New systemic therapy modalities are needed in this symptomatic/progressive population. In this multicenter, phase II trial (NCT03275818), patients with symptomatic/progressing DF received three cycles of weekly nab-paclitaxel. Brief pain inventory short form (BPI-SF) was collected at baseline and in every visit. MRI was performed every 3 months. Primary composite endpoint was RECIST 1.1 overall response rate (ORR) and/or clinical response (improvement ≥ 2 points in BPI-SF). If 40% of patients achieved clinical/radiological response, further investigation would be warranted. Toxicity, progression-free survival (PFS), pattern of response and its correlation with clinical best response and BPI, variation of physical function, and analgesic consumption were secondary endpoints. The translational research reported was not a pre-specified secondary outcome. Forty eligible patients started therapy, being 35 radiologically and clinically evaluable. The study achieved its primary endpoint, as 7(20%) patients obtained RECIST partial response, whereas 31(89%) experienced pain reduction of ≥2 points in BPI-SF worst pain. Therapy was well tolerated. With a median follow-up of 30(14–44) months, median 12 and 24-months PFS rates were 91%(CI 95%, 82–100) and 84%(CI 95%, 71–97). For clinical progression, 12 and 24-months PFS rates were 85% (CI 95%, 73–97) and 74% (CI 95%, 58–90) respectively. Short course of nab-paclitaxel is active, safe and achieves quick and durable responses in progressing/symptomatic DF patients.

The authors would like to thank the Spanish group for Research on Sarcomas (GEIS) for supporting the study and BMS/ Celgene (study ID AX-CL-GEIS-007345) for providing drug supply. The sponsor (GEIS) has been in charge of study design, data collection, analysis and manuscript writing. The authors also thank the donors and the University Hospital Virgen del Rocío for participating in patient recruitment. The authors would like to thank Araceli Rodriguez Morales and Gabriela Golab for Data Management. David S. Moura is recipient of a Sara Borrell postdoctoral fellowship funded by the National Institute of Health Carlos III (ISCIII) (CD20/00155). The authors would also like to thank SELNET project. SELNET has received funding from the European Union’s Horizon 2020 research and innovation program under grant agreement No. 825806.