HEALTH-RELATED QUALITY-OF-LIFE IN PATIENTS WITH RARE MOLD INFECTIONS: EQ-5D-5L RESULTS FROM A PHASE IIB TRIAL EVALUATING OLOROFIM IN ADULTS WITH INVASIVE FUNGAL INFECTIONS AND LIMITED OR NO TREATMENT OPTIONS
Author(s)
Andrej Spec, MD1, Johan Maertens, MD2, C. Orla Morrissey, PhD3, Monica Slavin, MD4, Belinda Lovelace, PharmD, MS, MJ5, Craig I. Coleman, PharmD6.
1Washington University in St Louis School of Medicine, St Louis, MO, USA, 2KU Leuven, Leuven, Belgium, 3Monash University, Melbourne, Australia, 4University of Melbourne, Melbourne, Australia, 5F2G, Inc., Princeton, NJ, USA, 6University of Connecticut, Storrs, CT, USA.
1Washington University in St Louis School of Medicine, St Louis, MO, USA, 2KU Leuven, Leuven, Belgium, 3Monash University, Melbourne, Australia, 4University of Melbourne, Melbourne, Australia, 5F2G, Inc., Princeton, NJ, USA, 6University of Connecticut, Storrs, CT, USA.
OBJECTIVES: Rare mold infections are associated with substantial morbidity and mortality. Olorofim is a novel antifungal with activity against difficult-to-treat rare molds (e.g., Lomentospora prolificans, Scedosporium spp.). The impact of olorofim on health-related quality-of-life (HRQoL) in patients with rare molds has not been evaluated.
METHODS: Study 32 was a prospective, multicenter, single-arm, phase IIb trial evaluating olorofim in adults with invasive fungal infections and limited or no treatment options (NCT03583164). This analysis evaluated changes in HRQoL, as measured by the EuroQol 5-Dimension 5-Level (EQ-5D-5L) instrument, among subjects with Lomentospora prolificans, Scedosporium spp., or other olorofim-susceptible rare-mold infections. EQ-5D-5L health index scores (HIS) were assessed at baseline, days 42, and 84. Analyses were restricted to subjects with observed baseline HIS. Longitudinal changes in HIS were analyzed using a repeated-measures linear mixed model with visit as a categorical fixed effect and Subject as a random intercept. Mean HIS and changes from baseline were reported with 95% confidence intervals (CIs). Responder analyses evaluated HIS improvements of ≥0.03, ≥0.05, and ≥0.07 points (common thresholds indicating a minimally important difference).
RESULTS: Forty-four subjects [Lomentospora prolificans (N=19), Scedosporium spp. (N=18), or other olorofim-susceptible rare molds (N= 7)] were included. The mean HIS increased from 0.547 at baseline to 0.611 at day 42 (N=35), corresponding to a mean change of 0.064 (95%CI, −0.022 to 0.150, p=0.144). At day 84, among 36 subjects with observed data, the mean HIS was 0.634, with a mean change from baseline of 0.087 (95% CI, 0.002 to 0.172; p=0.044). At day 42, HIS improvements of ≥0.03, ≥0.05, and ≥0.07 were observed in 57.6%, 51.5%, and 45.5% of subjects. At day 84, corresponding improvement rates were 57.6%, 57.6%, and 51.5%.
CONCLUSIONS: Subjects with rare mold infections receiving olorofim demonstrated statistically and clinically relevant improvements in HIS, with response rates >50% at day 84.
METHODS: Study 32 was a prospective, multicenter, single-arm, phase IIb trial evaluating olorofim in adults with invasive fungal infections and limited or no treatment options (NCT03583164). This analysis evaluated changes in HRQoL, as measured by the EuroQol 5-Dimension 5-Level (EQ-5D-5L) instrument, among subjects with Lomentospora prolificans, Scedosporium spp., or other olorofim-susceptible rare-mold infections. EQ-5D-5L health index scores (HIS) were assessed at baseline, days 42, and 84. Analyses were restricted to subjects with observed baseline HIS. Longitudinal changes in HIS were analyzed using a repeated-measures linear mixed model with visit as a categorical fixed effect and Subject as a random intercept. Mean HIS and changes from baseline were reported with 95% confidence intervals (CIs). Responder analyses evaluated HIS improvements of ≥0.03, ≥0.05, and ≥0.07 points (common thresholds indicating a minimally important difference).
RESULTS: Forty-four subjects [Lomentospora prolificans (N=19), Scedosporium spp. (N=18), or other olorofim-susceptible rare molds (N= 7)] were included. The mean HIS increased from 0.547 at baseline to 0.611 at day 42 (N=35), corresponding to a mean change of 0.064 (95%CI, −0.022 to 0.150, p=0.144). At day 84, among 36 subjects with observed data, the mean HIS was 0.634, with a mean change from baseline of 0.087 (95% CI, 0.002 to 0.172; p=0.044). At day 42, HIS improvements of ≥0.03, ≥0.05, and ≥0.07 were observed in 57.6%, 51.5%, and 45.5% of subjects. At day 84, corresponding improvement rates were 57.6%, 57.6%, and 51.5%.
CONCLUSIONS: Subjects with rare mold infections receiving olorofim demonstrated statistically and clinically relevant improvements in HIS, with response rates >50% at day 84.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
CO149
Topic
Clinical Outcomes, Economic Evaluation, Patient-Centered Research
Topic Subcategory
Clinical Outcomes Assessment
Disease
Infectious Disease (non-vaccine), No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases