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The Desmoid Tumor Research Foundation

October 2022

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

Author Information

Martin-Broto J (1, 2, 3, 4), Redondo A (5), Moura DS (6), Valverde C (7), Morales JM (8), Lopez-Pousa A (9), Martinez-Trufero J (10), Gutierrez A (11), Díaz-Beveridge R (12), Luna P (13), Martinez-Marin V (5), Marcilla D (14), Arribas I (15), Ledesma P (16), Lopez-Martin JA (17), Di Lernia D (6), Zamora J (6), Hindi N (6, 18, 19, 20).

(1) Health Research Institute-Fundación Jiménez Díaz University Hospital, Universidad Autónoma de Madrid (IIS-FJD, UAM), 28040, Madrid, Spain. jmartin@atbsarc.org. (2) Medical Oncology Department, Fundación Jimenez Diaz University Hospital, 28040, Madrid, Spain. jmartin@atbsarc.org. (3) General de Villalba University Hospital, 28400, Madrid, Spain. jmartin@atbsarc.org. (4) Autonomous University of Madrid, 28049, Madrid, Spain. jmartin@atbsarc.org. (5) Department of Medical Oncology, Hospital Universitario La Paz, 28046, Madrid, Spain. (6) Health Research Institute-Fundación Jiménez Díaz University Hospital, Universidad Autónoma de Madrid (IIS-FJD, UAM), 28040, Madrid, Spain. (7) Department of Medical Oncology, Vall d'Hebron University Hospital, 08035, Barcelona, Spain. (8) Radiology Department, Virgen del Rocio University Hospital, 41013, Sevilla, Spain. (9) Medical Oncology Department, Sant Pau Hospital, 08025, Barcelona, Spain. (10) Medical Oncology Department, Miguel Servet University Hospital, 50009, Zaragoza, Spain. (11) Hematology Department, University Hospital Son Espases, 07120, Mallorca, Spain. (12) Medical Oncology Department, Hospital Universitari i Politècnic La Fe, 46026, Valencia, Spain. (13) Medical Oncology Department, Son Espases University Hospital, 07120, Mallorca, Spain. (14) Pathology Department, Virgen del Rocio University Hospital, 41013, Sevilla, Spain. (15) Universitat de València, 46010, Valencia, Spain. (16) Sofpromed Investigacion Clinica SLU, 07009, Mallorca, Spain. (17) Medical Oncology Department, 12 de Octubre University Hospital, 28041, Madrid, Spain. (18) Medical Oncology Department, Fundación Jimenez Diaz University Hospital, 28040, Madrid, Spain. (19) General de Villalba University Hospital, 28400, Madrid, Spain. (20) Autonomous University of Madrid, 28049, Madrid, Spain.

Publication

Nat Commun

Summary

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.

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