학술논문
Hyaluronidase‐facilitated subcutaneous immunoglobulin 10% as maintenance therapy for chronic inflammatory demyelinating polyradiculoneuropathy: The ADVANCE‐CIDP 1 randomized controlled trial.
Document Type
Article
Author
Bril, Vera; Hadden, Robert D. M.; Brannagan, Thomas H.; Bar, Michal; Chroni, Elisabeth; Rejdak, Konrad; Rivero, Alberto; Andersen, Henning; Latov, Norman; Levine, Todd; Pasnoor, Mamatha; Sacconi, Sabrina; Souayah, Nizar; Anderson‐Smits, Colin; Duff, Kim; Greco, Erin; Hasan, Shabbir; Li, Zhaoyang; Yel, Leman; Ay, Hakan
Source
Subject
*THERAPEUTIC use of immunoglobulins
*DRUG efficacy
*IMMUNOGLOBULINS
*INTRAVENOUS therapy
*CONFIDENCE intervals
*TREATMENT duration
*HEALTH outcome assessment
*HEALTH surveys
*RANDOMIZED controlled trials
*PLACEBOS
*TREATMENT effectiveness
*GUILLAIN-Barre syndrome
*GLYCOSIDASES
*BLIND experiment
*QUESTIONNAIRES
*DESCRIPTIVE statistics
*RESEARCH funding
*SUBCUTANEOUS infusions
*STATISTICAL sampling
*PATIENT safety
*EVALUATION
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Language
ISSN
1085-9489
Abstract
Background and Aims: ADVANCE‐CIDP 1 evaluated facilitated subcutaneous immunoglobulin (fSCIG; human immunoglobulin G 10% with recombinant human hyaluronidase) efficacy and safety in preventing chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) relapse. Methods: ADVANCE‐CIDP 1 was a phase 3, double‐blind, placebo‐controlled trial conducted at 54 sites in 21 countries. Eligible adults had definite or probable CIDP and adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability scores of 0–7 (inclusive), and received stable intravenous immunoglobulin (IVIG) for ≥12 weeks before screening. After stopping IVIG, patients were randomized 1:1 to fSCIG 10% or placebo for 6 months or until relapse/discontinuation. fSCIG 10% was administered at the same dose (or matching placebo volume) and interval as pre‐randomization IVIG. The primary outcome was patient proportion experiencing CIDP relapse (≥1‐point increase in adjusted INCAT score from pre‐subcutaneous treatment baseline) in the modified intention‐to‐treat population. Secondary outcomes included time to relapse and safety endpoints. Results: Overall, 132 patients (mean age 54.4 years, 56.1% male) received fSCIG 10% (n = 62) or placebo (n = 70). CIDP relapse was reduced with fSCIG 10% versus placebo (n = 6 [9.7%; 95% confidence interval 4.5%, 19.6%] vs n = 22 [31.4%; 21.8%, 43.0%], respectively; absolute difference: −21.8% [−34.5%, −7.9%], p =.0045). Relapse probability was higher with placebo versus fSCIG 10% over time (p =.002). Adverse events (AEs) were more frequent with fSCIG 10% (79.0% of patients) than placebo (57.1%), but severe (1.6% vs 8.6%) and serious AEs (3.2% vs 7.1%) were less common. Interpretation: fSCIG 10% more effectively prevented CIDP relapse than placebo, supporting its potential use as maintenance CIDP treatment. [ABSTRACT FROM AUTHOR]