Effectiveness of Combined Rituximab and Chlorambucil or Cyclophosphamide Treatment in Anti-MAG Neuropathy
Vincent Brochard1, Marion Malphettes2, Marie Scuccimarra1, Christophe Carreau1, Claire Fieschi3, Antoine Guéguen1
1Neurologie, Hôpital Fondation A. de Rothschild, 2Immunopathologie Clinique, Hôpital Saint-Louis, APHP, 3Immunopathologie Clinique, Hôpital Saint-Louis, APHP; Institut Universitaire d'Hématologie-IHU, Université Paris Cité
Objective:
To evaluate effectiveness and management of bitherapy with Rituximab and Chlorambucil or Cyclophosphamide in patient with anti-MAG neuropathy
Background:

The anti-MAG IgM antibody plays a pathogenic role by depositing within the myelin sheath. Randomized Controlled Trials using Rituximab in anti-MAG neuropathy have failed to demonstrate significant benefits, suggesting that greater efficacy in reducing IgM monoclonal levels is needed, as achieved through combined therapy in the IELSG-19 trial.

Design/Methods:

We conducted a retrospective cohort study, describing the clinical, electrophysiological, and biological characteristics at diagnosis, during the 2 years after combined treatment and throughout long-term follow up. Various clinical outcomes were defined in order to analyzed treatment effectiveness. 

Results:

Fifteen patients with anti-MAG neuropathy were followed in our cohort. The median follow-up time was 3.1 years [2.3 -13.9] with a long-term follow-up data available for 10 patients (76.9%). The median age at the onset was 62 years [42 -82]. Eleven patients (73.3%) received biotherapy combining Rituximab with Chlorambucil while 2 patients (13.3%) received Rituximab with Cyclophosphamide.  During the first 2 years of follow-up after treatment, 7/10 patients (70%) showed clinical improvement, 2/10 experienced deterioration, and 1 remained stable. Over long-term follow-up, 3/10 patients remained stable, 5/10 showed neurological deterioration due in 3/10 cases to slow axonal loss in lower limbs and in 2/10 patients to relapse of neuropathy and hematologic disease leading to retreatment with positive outcome. One patient was retreated due to lack of hematological response. Two patients (15.4%) died, one from cancer within the first year after diagnosis and another from a pulmonary infection related to a long-standing lung condition.

Conclusions:
Our results suggest that Rituximab combination with either Chlorambucil or Cyclophosphamide may be a viable treatment option for anti-MAG neuropathy. Additionally, we defined clinical out-comes that may aid in guiding follow-up management and treatment or retreatment decisions.
10.1212/WNL.0000000000211238
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