Table 2.
Treatment response on secondary outcome measures after IVIg treatment expressed in median change scoresa as calculated by the non-parametric Wilcoxon signed rank test
| Outcome measure | Baseline, median (IQR) | End, median (IQR) | Δ Base-end, median (IQR) | P-value |
|---|---|---|---|---|
| Core set measures | ||||
| PhGA | 3.8 (3.2–4.0) | 2.3 (1.0–4.0) | −1.3 (−2.0 to −0.1) | < 0.01 |
| PaGA | 6.1 (5.3–7.6) | 4.6 (2.0–6.6) | −2.7 (−4.1 to −0.7) | 0.03 |
| MMTb | 211 (185–225) | 227 (191–241) | 10 (−7.0 to 29) | 0.12 |
| HAQ | 2.0 (1.5–2.5) | 1.6 (0.8–2.1) | −0.6 (−1.1 to 0.0) | 0.03 |
| sCK activity, U/l | 1199 (179–6500) | 196 (83–3877) | −103 (−3066 to −12) | < 0.01 |
| Extramuscular disease activity | 2.2 (0.6–3.0) | 1.0 (0.3–2.3) | −0.3 (−1.2 to 0.0) | 0.04 |
| Exploratory outcome measures of muscle function | ||||
| Rasch MRC-SS | 27 (24–28) | 28 (24–30) | 0.0 (−2.0 to 2.0) | 0.78 |
| Dynamometric muscle strengthc | ||||
| Deltoid muscles | 52 (41–72) | 53 (30–88) | 3.0 (−16 to 14) | 0.78 |
| Biceps muscles | 77 (46–95) | 73 (52–124) | 4.5 (−18 to 32) | 0.28 |
| Psoas muscles | 141 (124–162) | 174 (128–209) | 28 (−16 to 61) | 0.10 |
| ALSSS-SW | 9.0 (8.0–10) | 9.0 (8.0–10) | 0.0 (−1.0 to 1.0) | 0.40 |
| Exploratory outcome measures of disability | ||||
| mRS | 3.0 (3.0–4.0) | 3.0 (2.0–4.0) | 0.0 (−1.0 to 0.0) | 0.06 |
| Exploratory outcome measures of quality of life | ||||
| EQ-5D-5L | 40 (35–55) | 60 (40–70) | 15 (−5.0 to 30) | 0.03 |
The median change score was calculated as the 50th percentile of all individual differences between baseline and outcome assessment.
MMT according to Kendall.
Dynamometric muscle strength expressed as mean of three measurements per side by a hand-held Citec dynamometer in Newton. Of note, dynamometric muscle strength of the psoas muscles from one patient with DM was not available at baseline. ALSSS-SW: amyotrophic lateral sclerosis severity scale swallowing; EMA: extramuscular disease activity; EQ-5D-5L: EuroQol Group Health Questionnaire; IQR: interquartile range; MMT: manual muscle testing; mRS: modified Rankin Scale; PaGA: patient global activity; PhGA: physician global activity; Rasch MRC-SS: Rasch modified Medical Research Sum Score; sCK: serum creatine kinase.