Change from baseline EORTC QLQ-C30 subscales
| Subscale . | APPLY-PNH, iptacopan group (n = 62) Estimated average difference (95% CI)∗,† . | APPOINT-PNH (N = 40) Estimated average difference (95% CI)∗ . |
|---|---|---|
| Physical‡ | 14.7 (9.9-19.4) | 15.2 (12.7-17.7) |
| Role‡ | 17.4 (10.4-24.5) | 18.9 (15.1-22.7) |
| Fatigue§ | −16.8 (−23.0 to −10.7) | −21.9 (−25.6 to −18.2) |
| Pain§ | −7.4 (−13.7 to −1.2) | −11.4 (−14.4 to −8.5) |
| Dyspnea§ | −22.4 (−29.4 to −15.3) | −15.8 (−20.0 to −11.6) |
| Insomnia§ | −0.9 (−8.0 to 6.2) | −5.3 (−10.1 to −0.6) |
| Global health index‡ | 13.8 (9.0-18.7) | 24.4 (20.8-27.9) |
| Subscale . | APPLY-PNH, iptacopan group (n = 62) Estimated average difference (95% CI)∗,† . | APPOINT-PNH (N = 40) Estimated average difference (95% CI)∗ . |
|---|---|---|
| Physical‡ | 14.7 (9.9-19.4) | 15.2 (12.7-17.7) |
| Role‡ | 17.4 (10.4-24.5) | 18.9 (15.1-22.7) |
| Fatigue§ | −16.8 (−23.0 to −10.7) | −21.9 (−25.6 to −18.2) |
| Pain§ | −7.4 (−13.7 to −1.2) | −11.4 (−14.4 to −8.5) |
| Dyspnea§ | −22.4 (−29.4 to −15.3) | −15.8 (−20.0 to −11.6) |
| Insomnia§ | −0.9 (−8.0 to 6.2) | −5.3 (−10.1 to −0.6) |
| Global health index‡ | 13.8 (9.0-18.7) | 24.4 (20.8-27.9) |
ARMA, autoregressive moving average model.
The average is mean of the difference between least squares means at visits on days 126, 140, 154, and 168. The degrees of freedom follow the Rubin's rule of multiple imputation.24
Estimates are from a longitudinal model including baseline, visit, sex, age group, history of transfusions, and treatment group. The interactions are between visit and baseline and between visit and treatment group. Covariance structure is ARMA 2,3 with heteroscedastic variances.
Increases in the physical and role function subscales and the global health index indicate improvement.
Decreases in the fatigue, pain, dyspnea, and insomnia symptom subscales indicate improvements.