TABLE 2.
Study | Active treatment | Primary endpoint measure | Sample size | Dose | Duration of study/phase | Placebo arm response | Active arm response | ||
---|---|---|---|---|---|---|---|---|---|
Total | Placebo | Active | |||||||
CIDP studies with an improvement endpoint | |||||||||
Hughes et al 14 (ICE phase 1) | Gamunex (IVIG) | Total adjusted INCAT | 117 | 58 | 59 | 1 g/kg every 3 weeks | 24 weeks | 21% improved | 54% improved |
Mendell et al 17 | Gammar IV (IVIG) | AMS | 53 | 23 | 30 | 1 g/kg every 3 weeks | 6 weeks | 9% improved | 38% improved |
Hadden et al 10 | Interferon beta 1a | Improvement in any 3 of 8 clinical measures a | 10 | Crossover study | 3 MIU 3x per week 2 weeks and 6 MIU for 10 weeks | 12 weeks | 20% improved | 10% improved | |
Hahn et al 12 | IVIG | NDS, CG, and grip strength | 30 | Crossover study | 0.4 g/kg bw over 5 days | 9 weeks | 17% improved | 63% improved | |
Hahn et al 11 |
Plasma exchange | NDS, CG, andgrip strength | 18 | Crossover study | 10 exchanges over 4 weeks | 9 weeks | 0% improved | 80% improved | |
Vermeulen et al 21 | IVIG | Rankin scale and MRC sum score | 28 | 13 | 15 | 0.4 g/kg bw over 5 days | 3 weeks | 23% improved | 27% improved |
van Doorn et al 20 | IVIG | Rankin scale | 7 | Crossover study | 0.4 g/kg bw over 5 days | Unclear | 0% improved | 100% improved | |
Dyck et al 8 | Plasma exchange | NDS | 29 | 14 | 15 | Twice a week | 3 weeks | 0% improved | 33% improved |
Dyck et al 9 | Plasma exchange | NDS | 14 | 7 | 7 | Twice a week | 3 weeks | 43% improved | 57% improved |
CIDP studies with a stability vs deterioration endpoint | |||||||||
Hughes et al 13 (FORCIDP) | Fingolimod | Total adjusted INCAT | 106 | 52 | 54 | 0.5 mg daily | 3 years | 43% non‐deterioration | 42% non‐deterioration |
van Schaik et al 4 (PATH) | Hizentra (SCIG) | Total adjusted INCAT | 172 | 51 b | 115 c | 0.2 or 0.4 g/kg weekly | 24 weeks | 37% non‐deterioration | 61%‐67% non‐deterioration |
Markvardsen et al 16 | Subcuvia (SCIG) | IKS | 30 | 15 | 14 | 1:1 previous IVIG dose | 12 weeks | 33% non‐deterioration | 79% non‐deterioration |
RMC et al 18 (RMC group) |
Methotrexate | Reduction in mean weekly dose d | 59 | 32 | 27 | 7.5‐15 mg weekly | 32 weeks | 44% maintained | 52% maintained |
Hughes et al 14 (ICE phase 2) |
Gamunex (IVIG) | Total adjusted INCAT | 57 | 26 | 31 | 1 g/kg every 3 weeks | 24 weeks | 58% non‐deterioration | 87% non‐deterioration |
Abbreviations: AMS, average muscle score; CG, functional clinical grade; IKS, isokinetic strength; INCAT, inflammatory neuropathy cause and treatment; IVIG, intravenous immunoglobulin; MRC, Medical Research Council; NDS, neurological disability scale; SCIG, subcutaneous immunoglobulin.
Clinical measures included: timed 10‐m walk, ambulation index, expanded Medical Research Council (MRC) sum score, nine‐hole peg test time, functional independence measure, Hammersmith motor ability, a new Guy's neurological disability scale, and the EuroQoL quality of life scale.
20% reduction in mean weekly dose of IVIG or corticosteroids in last 4 weeks compared with first 4 weeks.
Of the 172 subjects randomized in the original paper, six from the placebo group were removed from our analysis due to either withdrawing without relapse (n = 4) or erroneously entering the restabilization phase (n = 2). This results in n = 51 for the placebo arm.
Combined SCIG‐treatment groups (0.2 g/kg, n = 57 and 0.4 g/kg, n = 58).