Measuring disease activity and predicting response to intravenous immunoglobulin in chronic inflammatory demyelinating polyneuropathy

Khoo, Anthony; Frasca, Joseph; Schultz, David

doi:10.1186/s40364-019-0154-2

Cited by 13 publications

(9 citation statements)

References 53 publications

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“…As previous studies have shown, NCS results, including possible axonal changes (no or very low amplitude), do not reflect neither the disease severity nor response to treatment (27,28). Features of the inflammation of nerve roots seen in MRI are observed more often in adults, ∼60%, than in children, 38% (3).…”

Section: Discussionmentioning

confidence: 98%

Pediatric CIDP: Diagnosis and Management. A Single-Center Experience

et al. 2021

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Background: Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare acquired polyneuropathy that especially among youngest children should be differentiated with hereditary neuropathies. Even though upon diagnosis treatment options are similar in children and adults, diagnostic challenges are faced in the pediatric population.Methods: We conducted a retrospective analysis of clinical symptoms, nerve conduction study results, modes of treatment, and final outcome in 37 children aged 3.5–17 years with a final diagnosis of CIDP (18 girls, 19 boys). We established three groups of patients based on age at onset of CIDP: 0–4, 4–13, and 13–18 years. Follow-up ranged from 10 to 222 months.Results: In our analysis, 19/37 patients (51.4%) had an atypical presentation: distal variant of CIDP in 12/37 patients (32.4%) and pure motor variant of CIDP in 5/37 patients (13.5%), and one patient had a pure sensory variant (1/37, 2.7%). Furthermore, 3/37 patients (8.1%) had additional concurring symptoms, including involuntary movements of face muscles (1/37, 2.7%) or hand tremor (2/37, 5.4%). During the follow-up, 23/37 patients (62.2%) received intravenous immunoglobulin (IVIg); 22/37 patients (59.5%) received steroids, 6/37 patients (16.2%) received IVIg and steroids, and 12/37 patients (32.4%) received immunosuppressive drugs, mostly azathioprine, but also methotrexate and rituximab. One patient was treated with plasmapheresis. Complete remission was achieved in 19/37 patients (51.4%) with CIDP in its typical form. Remission with residual symptoms or minimal deficit was observed in 4/37 patients (10.8%), whereas 14/37 patients (37.8%) remain on treatment with gradual improvement.Conclusion: Childhood CIDP may occur in its typical form, but even ~50% of children can present as an atypical variant including distal, pure motor, or pure sensory. Most children have a good prognosis; however, many of them may require long-term treatment. This highlights the importance of an early diagnosis and treatment for childhood CIDP.

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Section: Discussionmentioning

confidence: 98%

Pediatric CIDP: Diagnosis and Management. A Single-Center Experience

et al. 2021

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“… 6–8 To this date, however, reliable prognostic serum-based biomarkers applicable to the general population of CIDP patients are not yet available. 9 …”

Section: Introductionmentioning

confidence: 99%

Prognostic value of neurofilament light chain in chronic inflammatory demyelinating polyneuropathy

Godelaine

Schaepdryver

Bossuyt

et al. 2021

Brain Communications

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Chronic inflammatory demyelinating polyneuropathy is a neuroinflammatory disorder with considerable variation in clinical phenotype, disease progression and therapy response among patients. Recently, paranodal antibodies associated with poor response to intravenous immunoglobulin therapy and more aggressive disease course have been described in small subsets of patients, but reliable serum-based prognostic biomarkers are not yet available for the general population. In current retrospective longitudinal study, we utilized logistic regression models to investigate the associations of serum neurofilament light chain levels with 1-year disease progression and therapy response during follow-up in chronic inflammatory demyelinating polyneuropathy. 1-year disease progression was defined as a decrease of four or more points (the minimal clinically important difference) on an 80-point Medical Research Council sum-score scale one year after sampling. Patients who, compared to treatment received at time of sampling, required therapy switch during follow-up due to insufficient effect were classified as non-responders. Serum neurofilament light chain was measured by electrochemiluminescence assay in clinical residual serum samples of 76 patients diagnosed with probable (13 patients) or definite (63 patients) chronic inflammatory demyelinating polyneuropathy according to European Federation of Neurological Societies/Peripheral Nerve Society diagnostic criteria. 11 (15%) patients were female, and mean (standard deviation) cohort age was 61.5 (11.7) years. In both univariate and multivariable (including demographics) models, elevated serum neurofilament light chain harbored increased odds for 1-year disease progression (respectively odds ratio (OR), 1.049; 95% confidence interval (95%CI), 1.022—1.084 and OR, 1.097; 95%CI, 1.045—1.169; both P = 0.001). Patients with levels above the median cohort neurofilament light chain level (28.3 pg/mL) had largely increased odds of 1-year disease progression (univariate: OR, 5.597; 95%CI, 1.590—26.457; P = 0.01; multivariable: OR, 6.572; 95%CI, 1.495—39.702; P = 0.02) and of insufficient treatment response (univariate: OR, 4.800; 95%CI, 1.622—16.442; P = 0.007; multivariable: OR, 6.441; 95%CI, 1.749—29.357; P = 0.009). In a combined approach analysis, patients with levels above median cohort serum neurofilament light chain level reported strongly increased odds of demonstrating 1-year disease progression and/or therapy non-response during follow-up (univariate: OR, 6.337; 95%CI, 2.276—19.469; P<0.001; multivariable: OR, 10.138; 95%CI, 2.801—46.404; P = 0.001). These results show that in various logistic regression models, serum neurofilament light chain was associated with both 1-year disease progression and therapy response during follow-up in chronic inflammatory demyelinating polyneuropathy. Hence, our findings warrant further prospective research regarding the value of neurofilament light chain as potential prognostic biomarker in chronic inflammatory demyelinating polyneuropathy.

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“…Second, the ethnical differences and genetic backgrounds between Italy and China may represent reasons for the different rates of disease progression. Finally, several studies have shown that different treatment initiation times and treatment regimens may also lead to different results [26][27][28] . The uneven distribution of medical resources and limited therapeutic levels in China lead to the late start of treatment for patients, and most patients are treated with hormones; only a few patients have received a single course of IVIg (intravenous immunoglobulin).…”

Section: Discussionmentioning

confidence: 99%

The comparison of MRN, electrophysiology and progression among typical CIDP and atypical CIDP subtypes

Feng

Zheng

et al. 2020

Sci Rep

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We aimed to compare the electrophysiology and magnetic resonance neurography (MRN) results of chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) subtypes and to explore the progression from atypical CIDP to typical CIDP. We collected the medical records of 45 CIDP patients to analyse the rate of progression from atypical CIDP to typical CIDP subtypes. The cerebrospinal fluid (CSF) protein (p = 0.024) and overall disability sum score (ODSS) (p = 0.000) differed among patients with typical CIDP, distal acquired demyelinating symmetric neuropathy (DADS) and Lewis-Sumner syndrome (LSS). The compound motor action potential (CMAP) of typical CIDP was lower than that of the other subtypes (p = 0.016, p = 0.022 and p = 0.012). The cross-sectional area (CSA) of nerve roots in typical CIDP was significantly thicker than that of nerve roots in DADS and LSS. There were fewer DADS and LSS patients who progressed to typical CIDP than those who progressed to pure motor and pure sensory CIDP (p = 0.000), and the progression from pure motor to typical CIDP required a significantly longer time than the progression from pure sensory to typical CIDP (p = 0.007). Typical CIDP was more severe than the other subtypes not only in terms of clinical and electrophysiology factors but also in terms of MRN factors.

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Measuring disease activity and predicting response to intravenous immunoglobulin in chronic inflammatory demyelinating polyneuropathy

Cited by 13 publications

References 53 publications

Pediatric CIDP: Diagnosis and Management. A Single-Center Experience

Pediatric CIDP: Diagnosis and Management. A Single-Center Experience

Prognostic value of neurofilament light chain in chronic inflammatory demyelinating polyneuropathy

The comparison of MRN, electrophysiology and progression among typical CIDP and atypical CIDP subtypes

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