The necessity for rigour in rare disease study design
Mené sur 72 patients atteints de tumeurs desmoïdes évolutives (âge médian : 40 ans ; durée médiane de suivi : 23,4 mois), cet essai multicentrique randomisé de phase II évalue l’efficacité, du point de vue de la survie sans progression à 6 mois, et la toxicité du pazopanib par rapport à une chimiothérapie standard combinant méthotrexate et vinblastine
In The Lancet Oncology, Maud Toulmonde and colleagues report the results from the phase 2 DESMOPAZ trial. The study findings suggest that pazopanib at a daily dose of 800 mg could be considered a valid treatment option for patients who have progressive growth of a desmoid tumour. The study was described as a non-comparative randomised trial. The treatment population was randomly assigned to receive pazopanib or a chemotherapy combination of methotrexate and vinblastine at a ratio of 2:1. Desmoid tumour, also called aggressive fibromatosis, is a rare disease that does not have metastatic potential, but can be invasive and lead to substantial morbidity. Unfortunately, desmoid tumour can recur even after a complete resection. Common reasons for systemic therapy include multiple recurrences, unresectable tumours, and treatment of a rapidly growing intra-abdominal tumour. A hallmark of this tumour is its unpredictability.
The Lancet Oncology , commentaire, 2018