The Bruton tyrosine kinase inhibitor ibrutinib improves anti-MAG antibody polyneuropathy

Francesca Castellani, Andrea Visentin, Marta Campagnolo, Alessandro Salvalaggio, Mario Cacciavillani, Cinzia Candiotto, Roberta Bertorelle, Livio Trentin, Chiara Briani

Research output: Contribution to journalArticlepeer-review


OBJECTIVE: To assess whether neuropathy with anti-myelin-associated glycoprotein (MAG) antibody may improve after treatment with ibrutinib, an oral inhibitor of Bruton tyrosine kinase, we prospectively treated with ibrutinib a cohort of 3 patients with anti-MAG neuropathy and Waldenström macroglobulinemia (WM).

METHODS: All 3 patients underwent bone marrow biopsy showing WM, with MYD88 L265P mutated and CXCR4S338X wild type, and were started on ibrutinib 420 mg/die. Patients were assessed at baseline, at 3-6-9 months, and at 12 months in 2 patients with a longer follow-up, using Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Score, INCAT sensory sum score, and Medical Research Council sum score. The modified International Cooperative Ataxia Rating Scale was performed in 2 patients, whereas it was not used in the patient with Parkinson disease as a major comorbidity. Responders were considered the patients improving by at least one point in 2 clinical scales.

RESULTS: All the patients reported an early and subjective benefit, consistent with the objective improvement, especially of the sensory symptoms as shown by clinical scales. Treatment was well tolerated.

CONCLUSION: These preliminary data point to a possible efficacy of ibrutinib in anti-MAG antibody neuropathy, which is the most common disabling paraproteinemic neuropathy, where active treatment is eagerly needed.

CLASSIFICATION OF EVIDENCE: This study provides Class IV evidence that for patients with anti-MAG antibody neuropathy, ibrutinib improves neuropathy symptoms.

Original languageEnglish
Article numbere720
Number of pages6
JournalNeurology(R) neuroimmunology & neuroinflammation
Issue number4
Publication statusPublished - Jul 2020


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