Sustained response to subcutaneous immunoglobulins in chronic ataxic neuropathy with anti-disialosyl IgM antibodies (CANDA): report of two cases and review of the literature

Marastoni, Damiano; Africa, L.M.; Peretti, Alessandro; Bocci, Silvia; Insana, L; Ferrari, Sergio; Ginanneschi, Federica; Zanette, Giampietro; Fabrizi, Gian Maria; Giannini, Fabio

doi:10.1007/s00415-020-09843-y

Cited by 7 publications

(9 citation statements)

References 44 publications

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“…There was no visible association between the clinical and biological responses. Table 3 shows the biological response in these patients in the systematic review [1,[6][7][8][9][10][11]. Regarding the electrophysiologic pattern of CANOMAD, there was no association with the clinical response to rituximab therapy.…”

Section: Study Outcomesmentioning

confidence: 99%

“…Those with stable disease had the longest disease duration, at 8.5 years. Table 1 shows the outcomes of this systematic review [ 1 , 6 - 11 ].…”

Section: Reviewmentioning

confidence: 99%

“…There was no visible association between the clinical and biological responses. Table 3 shows the biological response in these patients in the systematic review [ 1 , 6 - 11 ].…”

Section: Reviewmentioning

confidence: 99%

“…Those with stable disease had the longest disease duration, at 8.5 years. Table 1 shows the outcomes of this systematic review [1,[6][7][8][9][10][11]. The biological response was evaluated in 27 patients based on the decrease in antibody titers.…”

Section: Study Outcomesmentioning

confidence: 99%

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Efficacy of Rituximab in CANOMAD: A Systematic Review

Aguirre,

Vivanco,

Ortiz

et al. 2023

Cureus

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CANOMAD, characterized by chronic ataxic neuropathy, ophthalmoplegia, immunoglobulin M (IgM) paraprotein, cold agglutinins, and disialosyl antibodies, encompasses a clinical, radiological, and laboratory diagnosis. CANOMAD is a rare condition, with fewer than 100 cases reported in the literature. The understanding and diagnosis of the disease have improved in the last few years, but the treatment of CANOMAD is mainly unknown, and there is not a clear consensus about it. We conducted a systematic review regarding the efficacy of rituximab in CANOMAD's treatment to investigate the clinical and biological response of CANOMAD in patients treated with rituximab. We used the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) and Meta-Analyses of Observational Studies in Epidemiology (MOOSE) reporting guidelines for this systematic review. To analyze the bias of the study, we used the Joanna Briggs Institute's (JBI) Critical Appraisal Checklist to analyze the bias of the case reports, and we used the Risk of Bias in Non-Randomized Studies of Interventions (ROBINS-I) tool for the observational studies. We only included case reports, case series, and observational studies written in English with patients formally diagnosed with CANOMAD and treated with rituximab. We excluded systematic reviews, literature reviews, and meta-analyses. We investigated the clinical and biological responses of the patients to rituximab. The clinical response was classified as complete recovery (CR), partial response (PR), stable disease (SD), and non-response (NR). We gathered 34 patients. The literature uses a modified Rankin score to define complete improvement (CR), partial response (PR), stable disease (SD), and progression. Clinically, there were three patients with CR, five with PR, 15 with SD, and 11 with progression. The biological response was assessed by measuring the decrease in antibody titers in 27 patients. Among those, six patients had CR, 12 had PR, eight had SD, and one had progression. Among 15 patients with neurological evaluation, 10 had ocular symptoms, and two presented with bulbar symptoms. Seven of the ten patients with ocular symptoms had SD, two had PR, and one had progression. Only 14 patients had a report of demyelinating features. Three had an axonal pattern, six had a demyelinating pattern, and five had a mixed pattern. Among patients with an axonal pattern, three had an SD. Among patients with a demyelinating pattern, three had a PR, two had an SD, and one had progression. Among patients with a mixed pattern, four had SD, and one had progression. We concluded that patients with CR have a shorter disease duration than patients with PR, SD, or progression. In addition, patients with CR had longer follow-ups than the other groups, suggesting that being treated early with rituximab improves the clinical outcome and has a sustained effect. There were no differences in the frequency of ocular and bulbar symptoms among patients with CANOMAD. The axonal patt...

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Section: Study Outcomesmentioning

confidence: 99%

“…Those with stable disease had the longest disease duration, at 8.5 years. Table 1 shows the outcomes of this systematic review [ 1 , 6 - 11 ].…”

Section: Reviewmentioning

confidence: 99%

“…There was no visible association between the clinical and biological responses. Table 3 shows the biological response in these patients in the systematic review [ 1 , 6 - 11 ].…”

Section: Reviewmentioning

confidence: 99%

Section: Study Outcomesmentioning

confidence: 99%

See 2 more Smart Citations

Efficacy of Rituximab in CANOMAD: A Systematic Review

Aguirre,

Vivanco,

Ortiz

et al. 2023

Cureus

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“…56 In a report of two cases on CANDA, IVIg achieved a favorable response, albeit with significant wearing-off fluctuations; treatment with subcutaneous immunoglobulins was an effective alternative, leading to a clinical response for at least 2 years (Table 3). 59 2.4 | IgM-type paraproteinemia-associated neuropathy without anti-nerve antibodies…”

Section: Treatmentmentioning

confidence: 99%

Paraproteinemia‐associated neuropathy with or without anti‐myelin‐associated glycoprotein antibody

Nakajima

2024

Clinical & Exp Neuroim

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Paraproteinemia‐associated neuropathy (PAN) develops with paraproteinemia and is mainly caused by the monoclonal proliferation of mature B cells, especially monoclonal gammopathy of undetermined significance (MGUS). PAN tends to increase with age, and in a super‐aging society, its frequency is expected to increase. Among paraproteinemias, the immunoglobulin (Ig)G type is most common, but the frequency of PAN is reported to be higher for the IgM type. IgG/IgA‐type PAN includes MGUS, plasma cell myeloma, and polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy and skin changes syndrome. IgM‐type PAN includes anti‐myelin‐associated glycoprotein antibody‐associated neuropathy, chronic ataxic neuropathy with IgM antibody that recognizes disialosyl ganglioside and IgM‐type PAN without anti‐nerve antibodies. Light chain‐type PAN includes immunoglobulin‐related amyloidosis. PAN has the characteristics of a blood disease, as well as an immune‐mediated disease, and is treated by immunotherapy. As an example, anti‐myelin‐associated glycoprotein antibody‐associated neuropathy is treated with rituximab, plasmapheresis and intravenous immunoglobulin therapy, but their effectiveness has not been established. As novel treatments, lenalidomide, Bruton tyrosine kinase inhibitors, B‐cell lymphoma 2 inhibitors and mimetic human natural killer‐1 epitope polymers have been investigated. By analyzing the human natural killer‐1‐related sugar chain structure as an antigen, the selective removal of pathological antibodies might be a new therapeutic target.

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Subcutaneous immunoglobulins (SCIG) for chronic inflammatory demyelinating polyneuropathy (CIDP): A comprehensive systematic review of clinical studies and meta-analysis

Ramzi,

Maya,

Balousha

et al. 2024

Neurol Sci

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Background Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) presents significant treatment challenges due to its chronic nature, varied clinical presentations, and rarity. Subcutaneous immunoglobulin (SCIG) has emerged as a maintenance therapy, offering potential advantages in administration and patient experience over the previously recognized intravenous immunoglobulin (IVIG). Methods: We included all clinical studies involving CIDP patients treated with SCIG from eleven databases up to March 2024. Results 50 clinical studies were included in the systematic review, with 22 involved in the meta-analysis. These studies offer clinical data on around 1400 CIDP patients. Almost all studies considered SCIG a maintenance therapy, with the majority of results suggesting it as a viable substitute that may offer comparable or enhanced advantages. Studies covered aspects such as efficacy, safety, quality of life, practicality, economic evaluation, and patient preference. Meta-analysis showed SCIG significantly improved muscle strength and sensory function, had fewer and milder side effects, reduced relapse rates, and received a strong preference. Conclusions Findings suggest that SCIG for CIDP maintenance not only provides a more feasible alternative, with economic evaluations showing considerable cost reductions over time, and patient preference for SCIG being pronounced, but may also deliver comparable or superior health outcomes. Ongoing research lines on formulations, techniques, and direct comparative studies are critical to further illuminate, enhance, and expand SCIG's role in treatment.

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Sustained response to subcutaneous immunoglobulins in chronic ataxic neuropathy with anti-disialosyl IgM antibodies (CANDA): report of two cases and review of the literature

Cited by 7 publications

References 44 publications

Efficacy of Rituximab in CANOMAD: A Systematic Review

Efficacy of Rituximab in CANOMAD: A Systematic Review

Paraproteinemia‐associated neuropathy with or without anti‐myelin‐associated glycoprotein antibody

Subcutaneous immunoglobulins (SCIG) for chronic inflammatory demyelinating polyneuropathy (CIDP): A comprehensive systematic review of clinical studies and meta-analysis

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