Treatment of inclusion-body myositis with IVIg

Abstract

We randomized 19 patients with inclusion-body myositis (IBM) to a double-blind, placebo-controlled, crossover study using monthly infusions of 2 g/kg intravenous immunoglobulin (IVIg) or placebo for 3 months. Patients crossed over to the alternate treatment after a washout period. We evaluated responses at baseline and at the end of each treatment period using expanded (0-10) MRC scales, the Maximum Voluntary Isometric Contraction (MVIC) method, symptom and disability scores, and quantitative swallowing studies. We calculated the differences in scores between IVIg and placebo from baseline to end of treatment. Of the 19 patients, 9 (mean age, 61.2 years; mean disease duration, 5.6 years) were randomized to IVIg and 10 (mean age, 66.1 years; mean disease duration, 7.4 years) to placebo. During IVIg the patients gained a mean of 4.2 (-16 to +39.8) MRC points, and during placebo lost 2.7 (-10 to +8) points (p < 0.1). These gains were not significant. Similar results were obtained with the MRC and MVIC scores when the patients crossed to the alternate treatment. Six patients had a functionally important improvement by more than 10 MRC points that declined when crossed over to placebo. Limb-by-limb analysis demonstrated that during IVIg the muscle strength in 39% of the lower extremity limbs significantly increased compared with placebo (p < 0.05), while a simultaneous decrease in 28% of other limbs was detected. The clinical importance of these minor gains is unclear. The duration of swallowing functions measured in seconds with ultrasound improved statistically in the IVIg-randomized patients (p < 0.05) compared with placebo. Although the study did not establish efficacy of IVIg, possibly because of the small sample size, the drug induced functionally important improvement in 6 (28%) of the 19 patients. Whether the modest gains noted in certain muscle groups justify the high cost of trying IVIg in IBM patients at a given stage of the disease remains unclear.

Immunotherapies for Immune-Mediated Myopathies: A Current Perspective

“…The first of these was a 6-month study in which 19 patients with IBM received either IVIG or placebo for 3 months. Although the overall results were not conclusive, swallowing function improved on IVIG and there were improvements in muscle strength, particularly in some lower limb muscle groups [50]. In keeping with these findings, a second crossover study in which 22 patients with IBM received either IVIG or placebo in combination with their previous immunosuppressant medications for periods of 6 months concluded that there were mild functional improvements and apparent disease stabilization in the majority of patients over the 12-month period of the study [52].…”

“…It has also been shown to be effective for the treatment of cases of drug-resistant PM, OS, and NAM, although its efficacy in these conditions has not been confirmed in a RCT [49]. The results of short-term (3-6-month) placebo-controlled trials of IVIG in IBM showed only minor functional benefits or temporary disease stabilization [50][51][52]; however, there have not been any longer-term studies and it is not known whether continued IVIG therapy would modify the course of the disease.…”

“…The use of corticosteroids and other immune therapies in IBM has been largely empirical and there have been few RCTs of immunosuppressive agents or other therapies [50][51][52].…”

“…There have been a number of studies to evaluate the efficacy of IVIG in IBM, including 3 short-term placebo-controlled crossover trials [50][51][52]. The first of these was a 6-month study in which 19 patients with IBM received either IVIG or placebo for 3 months.…”

Intravenøs IgG-behandling av nevromuskulære sykdommer

“…Kreatinkinasenivået i serum kan vaere normalt eller lett forhøyet, og elektromyografi kan vise denervasjon i tillegg til myopati. Mindre studier med intravenøs IgG-behandling, alene eller i kombinasjon med prednisolon, har ikke vist signifikant effekt, bortsett fra hos noen få pasienter som fikk forbedret svelgfunksjon (27)(28)(29).…”