Mycophenolate Facilitates Improvement in Outcome Measures in Treatment Resistant Chronic Inflammatory Demyelinating...

Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is a potentially disabling autoimmune neuropathy. Approximately 20% of patients are refractory to first-line treatments necessitating second-line options with limited evidence and potential adverse effects. This study evaluates the efficacy of mycophenolate mofetil (MMF) in refractory CIDP.

A retrospective case-control study of MMF plus IVIg ± corticosteroids (MMF cohort, n = 11) versus IVIg only treatment (comparator cohort, n = 33) for CIDP in a single institution is reported. IVIg dosing, day care unit (DCU) utilization, disease specific outcome measures, and adverse events were recorded before and 1 year after MMF initiation.

Median Medical Research Council Sum Score (MRC-SS) and Inflammatory Rasch-built Overall Disability Scale logit score (I-RODS) were lower, and DCU utilization was greater, in the MMF cohort than the comparator cohort prior to the introduction of MMF, representing this refractory patient group. Clinical outcomes improved with MMF: median MRC-SS improved from 58 to 68.5 (p = 0.012) and median I-RODS improved from 47 to 78 (p value = 0.028). There was a fiscally meaningful reduction in median IVIg dose and a reduction in DCU utilization from 2.89 days/month to 1.65 days/month (p value = 0.042). Cost savings averaged £38,432 per patient/year. MMF was well tolerated at 1 to 1.5 mg twice daily.

MMF is a well-tolerated and potentially effective adjunctive medication in CIDP patients with suboptimal response to first-line therapies. In refractory patients, MMF addition was associated with improvements in outcome measures and meaningful cost reductions. Further trials are required for confirmation.