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Review
. 2024 Jun 7:16:617-628.
doi: 10.2147/CMAR.S362694. eCollection 2024.

Capturing Patient Voice to Improve Outcomes That Matter to Patients with Desmoid Tumor

Bernd Kasper  1 , Mrinal Gounder  2   3 , Lynne Hernandez  4 , Christina Baumgarten  5 , Ravin Ratan  6
Affiliations
Review

Capturing Patient Voice to Improve Outcomes That Matter to Patients with Desmoid Tumor

Bernd Kasper et al. Cancer Manag Res. .

Abstract

Desmoid tumors (DT) are rare, intermediate-grade sarcomas characterized by locally aggressive growths that commonly occur intra-abdominally, in the abdominal wall, or in the extremities. Desmoid tumors are 2-3-fold more common in females than males, with most patients aged <40 years at diagnosis. Clinical course of DT is highly variable but rarely fatal, with median overall survival >80% at 20 years. However, patient morbidity and DT symptom burden can be high. DT significantly reduce patient quality of life, imposing substantial physical, emotional, and social burdens. Pain, fatigue, and insomnia are common symptoms; disfigurement, mobility restrictions, and, rarely, the need for amputation may also result. Despite its limited impact on survival, patients with DT may have anxiety and depression levels commensurate with those associated with malignant sarcomas. Thus, DT impose an array of significant, long-term morbidities on a young patient population. In order to evaluate the impact of these morbidities, patient-reported outcome (PRO) tools are used, which assess outcomes of importance to patients that extend beyond traditional oncology endpoints. General or oncology-related PROs can be used; although currently, the only DT-specific, validated PRO measure is the GOunder/Desmoid Tumor Research Foundation DEsmoid Symptom/Impact Scale (GODDESS©), consisting of an 11-item DT Symptom Scale (DTSS) and a 17-item DT Impact Scale (DTIS). DTSS and DTIS were secondary endpoints in DeFi, a randomized phase 3 trial of nirogacestat; blinded, pooled data from DeFi were used to validate GODDESS reliability and responsiveness as a PRO measure in DT. Another DT-specific PRO measure, the Desmoid-Type Fibromatosis Quality of Life (DTF-QoL) questionnaire, has been developed but not validated. As novel DT therapies continue to be developed, incorporating DT-specific PRO measures into clinical trials will be key to capturing patient voice, improving outcomes of importance to this unique patient population, and assisting patients and providers in selecting optimal treatment.

Keywords: GODDESS; PRO; fibromatosis; patient-reported outcomes; quality of life; rare disease.

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Conflict of interest statement

Bernd Kasper: Consultant for Ayala, Bayer, Boehringer Ingelheim, GSK, Roche, and SpringWorks Therapeutics, Inc.; received honoraria from Bayer, GSK, and PharmaMar; research funding from Ayala, Cogent, PharmaMar, Rain Therapeutics, and SpringWorks Therapeutics, Inc. Mrinal Gounder: Personal honoraria/advisory boards and/or associated research paid to institution for Aadi, Ayala, Bayer, Boehringer Ingelheim, Daiichi, Epizyme, Karyopharm, Regeneron, Rain, SpringWorks Therapeutics, Inc., Tracon, and TYME; Other: Guidepoint, GLG, Third Bridge, and Flatiron Health; CME Honoraria: Medscape, More Health, Physicians Education Resource, MJ LifeSciences, and touchIME; Royalties: Wolters Kluwer, patents with MSKCC (GODDESS PRO), and uncompensated research with Foundation Medicine; Grants: Food and Drug Administration (R01 FD005105) and the National Cancer Institute, National Institutes of Health (P30CA008748)—core grant (CCSG shared resources and core facility). Lynne Hernandez: Executive Director of The Desmoid Tumor Research Foundation, which has received grants from SpringWorks Therapeutics, Inc., and Ayala Pharmaceuticals. Christina Baumgarten: Sarcoma Patients Advocacy Global Network received grants and sponsorship from SpringWorks Therapeutics, Inc. Ravin Ratan: Consultant for SpringWorks Therapeutics, Inc., Inhibrx, and Bayer. Research funding from SpringWorks Therapeutics, Inc., Ayala Pharmaceuticals, and C4 Therapeutics. The authors report no other conflicts of interest in this work.

Figures

Figure 1
Figure 1
Mean EORTC QLQ-C30 scores in patients with DT (n=102) versus healthy controls (n=102). (A) Global health and functional scales, with higher scores representing higher functioning. (B) Symptom scales, with higher scores representing higher patient symptom burden. *Indicates P = 0.0002, and **Indicates P < 0.0001.
Figure 2
Figure 2
GOunder/Desmoid Tumor Research Foundation DEsmoid Symptom/Impact Scale (GODDESS©) PRO Measure. aAdministered only to those reporting intra-abdominal tumor location in item 8. b11-point scale where 0 indicates “none” and 10 indicates “as bad as you can imagine”, with a 24-hour recall period. c5-point Likert scale ranging from “none of the time” to “all of the time”. d11-point scale where 0 indicates “none” and 10 indicates “as bad as you can imagine”, with a 7-day recall period.
Figure 3
Figure 3
Desmoid Tumor Fibromatosis Quality of Life (DTF-QoL) questionnaire. Patients are asked to score items on a Likert scale ranging from 1 (not at all) to 4 (very much).
See this image and copyright information in PMC

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