Change from baseline in QoL and functional capacity at month 9 in patients with Mayo stage IV AL amyloidosis
| End points . | Patients with Mayo stage IV AL amyloidosis (n = 77) . | |||
|---|---|---|---|---|
| Birtamimab + SOC (n = 38) . | Placebo + SOC (n = 39) . | Group difference . | P . | |
| SF-36v2 PCS | ||||
| Baseline score, mean (SD) | 33.61 (8.753) | 33.75 (9.972) | NA | |
| Change from baseline at month 9, LS mean (SE)∗ | −0.75 (1.749) | −5.40 (1.597) | +4.65 (2.325)† | .046 |
| 6MWT distance | ||||
| Baseline distance (meters), mean (SD) | 336.10 (101.722) | 322.63 (100.484) | NA | |
| Change from baseline at month 9 (meters), LS mean (SE)∗ | 15.22 (20.010) | −21.15 (20.632) | +36.37 (26.310)† | .022‡ |
| End points . | Patients with Mayo stage IV AL amyloidosis (n = 77) . | |||
|---|---|---|---|---|
| Birtamimab + SOC (n = 38) . | Placebo + SOC (n = 39) . | Group difference . | P . | |
| SF-36v2 PCS | ||||
| Baseline score, mean (SD) | 33.61 (8.753) | 33.75 (9.972) | NA | |
| Change from baseline at month 9, LS mean (SE)∗ | −0.75 (1.749) | −5.40 (1.597) | +4.65 (2.325)† | .046 |
| 6MWT distance | ||||
| Baseline distance (meters), mean (SD) | 336.10 (101.722) | 322.63 (100.484) | NA | |
| Change from baseline at month 9 (meters), LS mean (SE)∗ | 15.22 (20.010) | −21.15 (20.632) | +36.37 (26.310)† | .022‡ |
ANCOVA, analysis of covariance; IWRS, interactive web response system; LS, least squares; MMRM, mixed-effect model for repeated measures; SD, standard deviation; SE, standard error.
Estimates of the LS mean and SE for each treatment group were estimated using an MMRM methodology, including fixed effects for treatment group, categorical time point (all postbaseline visits), treatment group by visit interaction, IWRS stratification factors (renal stage: I, II/III; baseline 6MWT distance: <300 m, ≥300 m), the associated baseline value as a covariate, and a compound symmetry covariance structure to model the within-patient errors.
Group difference favors birtamimab.
P value from rank ANCOVA; before analysis, patients were ranked from worst to best following the 7-step algorithm.