Prospective development of a patient-reported outcomes instrument for desmoid tumors or aggressive fibromatosis.

ABSTRACT:

Background

Desmoid tumors (or aggressive fibromatosis) are locally infiltrative connective-tissue tumors that can arise in any anatomic location; they can be asymptomatic, or they can result in pain, deformity, swelling, and loss of mobility and/or threaten visceral organs with bowel perforation, hydronephrosis, neurovascular damage, and other complications. Existing clinical trial endpoints such as the Response Evaluation Criteria in Solid Tumors (version 1.1) and progression-free survival are inadequate in capturing treatment efficacy. This study was designed to develop a novel clinical trial endpoint by capturing patient-reported outcomes (PROs).

Methods

Following best practices in qualitative methodology, this study used concept elicitation (CE) interviews to explore des

SUBMITTER: Gounder MM 

PROVIDER: S-EPMC6980244 | biostudies-literature | 2020 Feb

REPOSITORIES: biostudies-literature