RT Journal Article
T1 A phase II trial of weekly nab-paclitaxel for progressive and symptomatic desmoid tumors.
A1 Martin-Broto, Javier
A1 Redondo, Andres
A1 Moura, David S
A1 Valverde, Claudia
A1 Morales, Jose Manuel
A1 Lopez-Pousa, Antonio
A1 Martinez-Trufero, Javier
A1 Gutierrez, Antonio
A1 Díaz-Beveridge, Roberto
A1 Luna, Pablo
A1 Martinez-Marin, Virginia
A1 Marcilla, David
A1 Arribas, Ivan
A1 Ledesma, Patricio
A1 Lopez-Martin, Jose Antonio
A1 Di Lernia, Davide
A1 Zamora, Jorge
A1 Hindi, Nadia
AB 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.
YR 2022
FD 2022-10-21
LK http://hdl.handle.net/10668/19548
UL http://hdl.handle.net/10668/19548
LA en
DS RISalud
RD Apr 11, 2025