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. 2025 Aug;72(2):250-257.
doi: 10.1002/mus.28436. Epub 2025 May 13.

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

Shamim Miah  1 , Nicole Japzon  1 , Amar Elsaddig  1 , Shiwen Koay  1 , Laura Compton  1 , Michael P Lunn  1   2 , Aisling S Carr  1   2
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Mycophenolate Facilitates Improvement in Outcome Measures in Treatment Resistant Chronic Inflammatory Demyelinating Polyradiculoneuropathy

Shamim Miah et al. Muscle Nerve. 2025 Aug.

Abstract

Introduction/aims: 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.

Methods: 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.

Results: 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.

Discussion: 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.

Keywords: chronic inflammatory demyelinating polyradiculoneuropathy; intravenous immunoglobulin; mycophenolate mofetil; outcome measures; treatment‐refractory.

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References

    1. A. L. Oaklander, M. P. T. Lunn, R. A. Hughes, et al., “Treatments for Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP): An Overview of Systematic Reviews,” Cochrane Database of Systematic Reviews 2017, no. 1 (2017): CD010369, https://doi.org/10.1002/14651858.CD010369.pub2.
    1. P. P. Choudhary and R. A. C. Hughes, “Long‐Term Treatment of Chronic Inflammatory Demyelinating Polyradiculoneuropathy With Plasma Exchange or Intravenous Immunoglobulin,” Quarterly Journal of Medicine 88, no. 7 (1995): 493–502.
    1. S. R. M. Bus, M. C. Broers, I. M. Lucke, et al., “Clinical Outcome of CIDP One Year After Start of Treatment: A Prospective Cohort Study,” Journal of Neurology 269, no. 2 (2022): 945–955.
    1. M. P. T. Lunn, H. Manji, P. P. Choudhary, R. A. C. Hughes, and P. K. Thomas, “Chronic Inflammatory Demyelinating Polyradiculoneuropathy: A Prevalence Study in South East England,” Journal of Neurology, Neurosurgery, and Psychiatry 66, no. 5 (1999): 677–680.
    1. C. Bunschoten, P. H. Blomkwist‐Markens, A. Horemans, P. A. van Doorn, and B. C. Jacobs, “Clinical Factors, Diagnostic Delay, and Residual Deficits in Chronic Inflammatory Demyelinating Polyradiculoneuropathy,” Journal of the Peripheral Nervous System 24, no. 3 (2019): 253–259.

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