Adult patients
Among adult patients (≥ 18 years old; n = 11), the mean (range) IWQOL-Lite total score at active treatment baseline was 74.9 (59.0–97.0), indicating moderate HRQOL impairment (Table At the patient level, 8 of 11 (72.7%) adults experienced HRQOL impairment at active treatment baseline (mean [range], 68.8 [59.0–78.0]). Of these, 4 adults reported severe HRQOL impairment, and 4 reported moderate HRQOL impairment. Of adults with HRQOL impairment at active treatment baseline, 5 of 8 (62.5%) experienced clinically meaningful improvement in IWQOL-Lite and improved their health status level of impairment after 52 weeks of treatment; notably, 2 patients reporting moderate impairment at baseline improved to non-impaired status at the end of the study. The 3 remaining patients preserved their HRQOL, demonstrating improvements in total IWQOL-Lite score that did not meet the threshold for being clinically meaningful. Mean (range) change in total IWQOL-Lite scores in those with impairment at baseline (n = 8) was + 14.5 (1.0–28.0). All adults without clinically relevant HRQOL impairment at active treatment baseline (n = 3) either preserved their non-impaired status (n = 2) or meaningfully improved (n = 1) their HRQOL based on IWQOL-Lite scores. Mean (range) change in total IWQOL-Lite scores in those without impairment at baseline (n = 3) was + 5.3 (2.0–9.0).