A phase II trial of weekly nab-paclitaxel for progressive and symptomatic desmoid tumors
Martin-Broto, Javier (Universidad Autónoma de Madrid)
Redondo, Andres (Hospital Universitario La Paz (Madrid))
Moura, David S. (Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz)
Valverde, Claudia (Vall d'Hebron Institut d'Oncologia)
Morales, Jose Manuel (Hospital Universitario Virgen del Rocío (Sevilla, Andalusia))
López Pousa, Antonio (Institut d'Investigació Biomèdica Sant Pau)
Martínez-Trufero, Javier (Hospital Universitario Miguel Servet. Servicio de Oncología Médica)
Gutierrez, Antonio (Hospital Universitario Son Espases. Servicio de Hematología)
Díaz-Beveridge, Roberto (Hospital Universitari i Politècnic La Fe (València))
Luna, Pablo (Hospital Universitario Son Espases. Servicio de Oncología Médica)
Martinez-Marin, Virginia (Hospital Universitario La Paz (Madrid))
Marcilla, David (Hospital Universitario Virgen del Rocío (Sevilla, Andalusia))
Arribas, Ivan (Universitat de València)
Ledesma, Patricio (Sofpromed Investigacion Clinica SLU)
Lopez-Martin, Jose A. (Hospital Universitario 12 de Octubre (Madrid))
Di Lernia, Davide (Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz)
Zamora, Jorge (Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz)
Hindi, Nadia (Universidad Autónoma de Madrid)

Fecha:	2022
Resumen:	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.
Ayudas:	Instituto de Salud Carlos III CD20/00155 European Commission. Horizon 2020 825806
Derechos:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, fins i tot amb finalitats comercials, sempre i quan es reconegui l'autoria de l'obra original.
Lengua:	Anglès
Documento:	Article ; recerca ; Versió publicada
Materia:	Albumins ; Fibromatosis, Aggressive ; Humans ; Paclitaxel ; Pain
Publicado en:	Nature communications, Vol. 13 Núm. 1 (december 2022) , p. 6278, ISSN 2041-1723

DOI: 10.1038/s41467-022-33975-6
PMID: 36271011

11 p, 765.0 KB

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