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64 Plasma KCE Reports 120 • In patients with IgM paraprotein-associated demyelinating neuropathy, IG produced short-term benefit, such as reduced disability and improved muscle strength. • In IgG and IgA paraproteinemic neuropathy, evidence on IG effectiveness is insufficient. Multiple sclerosis Multiple sclerosis (MS) is a disorder of the central nervous system with progressive deterioration of neurological functions caused by autoimmune, inflammatory and neurodegenerative mechanisms. The course of the disease is relapsing remitting (called RRMS) initially in 80-85% of all MS patients. A minority of patients (10-15%) have a chronic progressive course from the beginning, without typical relapses and remissions, termed primary progressive MS or PPMS. In most patients, the disease progresses to a stage with steady deterioration, known as secondary chronic progressive MS or SPMS. 84 , 85 A Cochrane systematic review included only 2 RCT out of the 10 RCT on RRMS cases that were eligible, due to methodological issues or studies being in progress, and did not perform any meta-analysis. 86 Another review was published in 2008 and performed a meta-analysis based on 4 RCT. 87 Nine additional RCT published after the last Cochrane review update (Jan 2003) have been retrieved. All findings are relatively consistent across studies and are presented below by type of MS. 1. RRMS: The two literature reviews have evaluated IG effectiveness compared to placebo in treating RRMS, and three additional RCT have been published. • The 2008 review included four RCT, involving 265 RRMS patients. 87 A meta-analysis of the 4 RCT showed a significant reduction in relapse rate, proportion of relapse-free patients and changes of disability scores in the groups treated with IG compared to placebo. 88,89, 90 A small RCT comparing two doses to placebo also found a significant reduction in groups treated with IG. 91 A small RCT assessing Magnetic Resonance Imaging (MRI) impact on 26 patients showed a significant reduction in MRI lesions. 90 • The 2003 Cochrane review was based on two of these RCT, involving 168 RRMS participants, and found a significant reduction in relapse rate and an increased time to first relapse during IG treatment. • One recent multi-national RCT, published after the two reviews and involving 127 patients from 30 European and US sites, showed no beneficial effect of IG at doses of 0.2 to 0.4g/kg as compared to placebo in RRMS patients after a year period. 92 Authors could not explain why these results contrast with other findings, except for an overly optimistic sample size calculation. • Two RCT involving 19 and 76 patients with MS and acute relapses found that IG combined to prednisolone was not more effective in the treatment of relapses than prednisolone alone. 93,94 2. PPMS: A small RCT published after the search period of the two reviews and involving 34 patients with PPMS showed that IG significantly reduced disease progression as measured by clinical indicators, compared to placebo. 84 3. SPMS: Three RCT, involving a total of 585 SPMS patients, consistently showed no significant effect of IG on disease progression, relapses or MRI lesions compared to placebo. 84,95,96, 97 No meta-analysis was performed as only one RCT was included in the 2008 review (though no exclusion criteria was stated). 4. Post-partum relapses: The risk of MS relapses is increased in the post-partum period and the other available therapies are not recommended during pregnancy or lactation. Several non-controlled trial or retrospective studies on IG treatment suggested that the exacerbation rate in the treated group
KCE Reports 120 Plasma 65 was lower than in a reference population. 98, 99 A multi-national RCT in 9 EU countries including Belgium, comparing two doses in the 6 months postpartum (but not to placebo), did not show significant differences in outcome between the two doses. 100 Despite the lack of RCT comparing IG to placebo, many authors conclude that IG can be considered as an optional treatment to reduce the increased rate of relapses in this period, because no alternative treatment with proven efficacy is available. 5. In patients with clinically isolated syndrome: An RCT evaluated the occurrence of a second attack in 91 MS cases after a first neurological event suggestive of demyelinating disease. Among patients in the IG group, the incidence of a second attack and disease activity as measured by brain MRI significantly reduced compared to placebo. 101 6. Dosage: all RCT but one used a dose ranging 0.15-0.4g/kg monthly. A single study used 2g/kg but did not yield higher efficacy rates. 90 A study comparing two dose treatment (0.2 and 0.4g/kg/month) for the treatment of RRMS could not show any significant differences between the effects of the two doses. 92 A systematic review searched clinical and cost effectiveness studies on immunomodulatory drugs for MS, but did not find any cost-effectiveness study including IG. 102 • In RRMS disease, IG has shown beneficial effects on disease progression and on magnetic resonance imaging (MRI) lesions (moderate). Combined to prednisolone, IG was not more effective in the treatment of relapses than prednisolone alone (high). The ideal dose and the effect on long term disability have not been well studied. IG is considered as one of the options for the treatment of RRMS. • In PPMS patients, IG delayed disease progression in one RCT (moderate). • In SPMS disease, IG had no significant effect on disease progression or MRI lesions (high). • IG reduced the increased rate of relapses in the post-partum period in open studies and is considered as an optional treatment (low). Lambert-Eaton myasthenic syndrome Lambert-Eaton myasthenic syndrome or LEMS is a rare autoimmune disorder of neuromuscular transmission, most commonly seen as a paraneoplasic syndrome (60% of patients). It is characterized by proximal muscle weakness, ocular symptoms, bulbar symptoms and autonomic dysfunction. LEMS often responds to steroids or other immunosuppressant. A Cochrane review published in 2005 included only 1 RCT assessing the use of IG in LEMS cases. 103 Significant improvement in limb muscle strength, vital capacity and drinking time was observed compared to placebo, but was short term and declined after 8 weeks. The review concluded that IG can be considered as an alternative treatment to steroids and immunosupressants for LEMS. A small RCT showed that IG improved functional capacities and muscle strength in Lambert-Eaton myasthenic syndrome on the short term
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Comment assurer l’autosuffisance
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Comment assurer l’autosuffisance
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KCE Reports 120B Plasma i PRÉFACE
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KCE Reports 120B Plasma iii MÉTHOD
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KCE Reports 120B Plasma v En ce qui
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KCE Reports 120B Plasma vii RECOMMA
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KCE Reports 120 Plasma 1 Scientific
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KCE Reports 120 Plasma 3 GLOSSARY A
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KCE Reports 120 Plasma 121 APPENDIX
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KCE reports 1. Efficacité et renta
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73. Financement du programme de soi
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