DETERMINING MEANINGFUL WITHIN-PATIENT CHANGE THRESHOLDS FOR GODDESS OUTCOMES IN THE PHASE 3 RINGSIDE PART B TRIAL
Author(s)
Shauna McManus, MPH1, Phoebe Wright, PharmD, MS2, Sam Parsons, MS, PhD1, Zsolt Hepp, PharmD, MS3, Furong Li, MS3, Mrinal Gounder, MD4.
1OPEN Health Group, New York, NY, USA, 2Immunome, Bothwell, WA, USA, 3Immunome, Bothell, WA, USA, 4Memorial Sloan Kettering Cancer Center, New York, NY, USA.
1OPEN Health Group, New York, NY, USA, 2Immunome, Bothwell, WA, USA, 3Immunome, Bothell, WA, USA, 4Memorial Sloan Kettering Cancer Center, New York, NY, USA.
OBJECTIVES: Desmoid tumors (DTs) are rare, aggressive fibroblastic tumors that grow into surrounding soft tissues and often recur locally, but don’t metastasize. DTs may impose substantial pain/functional burden. This analysis evaluated the scoring approach and meaningful within‑patient change (MWPC) thresholds for the Gounder/DTRF Desmoid Tumor Symptom/Impact Scale (GODDESS DTSS/DTIS) in the Phase 3 RINGSIDE Part B trial, focusing on the key secondary worst pain intensity (WPI) and exploratory GODDESS-based endpoints.
METHODS: Analyses used blinded, pooled data from all randomized subjects. GODDESS DTSS was captured via daily diaries over 7 days; weekly scores were derived as 7‑day averages. A missing‑data simulation evaluated the minimum number of diaries required to compute reliable weekly WPI scores. The Patient Global Impression of Change (PGI‑C) was evaluated for anchor suitability and used to estimate MWPC improvement thresholds for WPI and other GODDESS scores. Estimates were supported by empirical cumulative distribution/probability density functions (eCDFs/ePDFs).
RESULTS: Weekly WPI scores remained stable with up to four missing diaries. PGI‑C moderately correlated with WPI change, confirming anchor suitability. Consistent with anchor-based MWPC thresholds of WPI in other disease states, this analysis showed the median WPI change among participants reporting minimal improvement was -2.0, further supporting that a ≥2‑point reduction represents clinically meaningful within‑patient improvement in DT. PGI-C was also confirmed as a suitable anchor for all exploratory endpoints. Estimated MWPC thresholds were -1.23 for DTSS total symptom score (TSS), -1.16 for DTSS TSS‑5, -1.62 for DTSS pain, and -0.60 for DTIS physical functioning. eCDFs demonstrated clear separation between minimal improvement and non‑improvement groups at estimated thresholds.
CONCLUSIONS: The predefined requirement of ≥4/7 diaries per week to generate reliable weekly averages was supported. A ≥2‑point reduction in WPI and corresponding thresholds for additional GODDESS scores represent meaningful improvement. These results provide a robust framework for interpreting symptom/functioning changes and evaluating clinical meaningfulness in DT.
METHODS: Analyses used blinded, pooled data from all randomized subjects. GODDESS DTSS was captured via daily diaries over 7 days; weekly scores were derived as 7‑day averages. A missing‑data simulation evaluated the minimum number of diaries required to compute reliable weekly WPI scores. The Patient Global Impression of Change (PGI‑C) was evaluated for anchor suitability and used to estimate MWPC improvement thresholds for WPI and other GODDESS scores. Estimates were supported by empirical cumulative distribution/probability density functions (eCDFs/ePDFs).
RESULTS: Weekly WPI scores remained stable with up to four missing diaries. PGI‑C moderately correlated with WPI change, confirming anchor suitability. Consistent with anchor-based MWPC thresholds of WPI in other disease states, this analysis showed the median WPI change among participants reporting minimal improvement was -2.0, further supporting that a ≥2‑point reduction represents clinically meaningful within‑patient improvement in DT. PGI-C was also confirmed as a suitable anchor for all exploratory endpoints. Estimated MWPC thresholds were -1.23 for DTSS total symptom score (TSS), -1.16 for DTSS TSS‑5, -1.62 for DTSS pain, and -0.60 for DTIS physical functioning. eCDFs demonstrated clear separation between minimal improvement and non‑improvement groups at estimated thresholds.
CONCLUSIONS: The predefined requirement of ≥4/7 diaries per week to generate reliable weekly averages was supported. A ≥2‑point reduction in WPI and corresponding thresholds for additional GODDESS scores represent meaningful improvement. These results provide a robust framework for interpreting symptom/functioning changes and evaluating clinical meaningfulness in DT.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
PCR244
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
Oncology