Summary Given its anti‐angiogenic activity, lenalidomide may have a role in the treatment of POEMS ( P eripheral neuropathy, O rganomegaly, E ndocrinopathy, M onoclonal plasma cell disorder and S kin changes) syndrome. This prospective, open‐label, pilot study evaluated the combination of lenalidomide + dexamethasone ( RD ) in 18 POEMS syndrome patients (13 pre‐treated, 5 newly‐diagnosed but ineligible for high‐dose therapy). Treatment consisted of six cycles of lenalidomide (25 mg/day for 21 days followed by 7 days rest) plus dexamethasone (40 mg/once a week). Patients responding after six cycles continued treatment until progression or unbearable toxicity. The primary endpoint was the proportion of patients with either neurological or clinical improvement. The RD combination was considered as deserving further evaluation if 9 of the first 15 patients responded. Ten responses were observed among the first 15 enrolled patients, meeting the primary endpoint. Fifteen of 18 patients (83%) completed six RD cycles: 13 (72%) patients responded and nine had both clinical and neurological improvement. Among the 15 patients who completed the six RD cycles, four were still on treatment after a 25‐month follow‐up. At 39 months of follow‐up, all patients were alive with a 3‐year progression‐free survival of 59%. No patient discontinued RD for toxicity. Overall, the RD regimen showed a high incidence of prolonged symptoms improvement and was well tolerated in most POEMS patients.
Objective: To compare the efficacy and safety of six-month therapy with intravenous immunoglobulin (IVIg) or methylprednisolone (IVMP) in chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). Background IVIg and steroid are similarly effective as initial treatment in CIDP but little is known on their relative risk-benefit profile over the long period. Design/Methods: Forty-six patients fulfilling EFNS/PNS criteria for definite CIDP were randomly allocated in a 1:1 proportion to receive IVIg (IgVena, Kedrion SpA) (2g/kg) + IVMP-placebo or IVMP (2 g) + IVIg-placebo over four days, monthly for six months. Patients were then followed for six months to assess relapses. The primary endpoint was the difference in the number of patients suspending IVIg or IVMP for intolerance or inefficacy. Secondary end-points included the difference in the improvement in assessment scales after 15 days, 2 months and 6 months of therapy, and the difference in the proportion of patients worsening after therapy discontinuation. Results: Forty-five patients (24 on IVIg and 21 on IVMP) were evaluated. One was excluded for inappropriate inclusion. More patients suspended IVMP (10/21, 52.5%) than IVIg (3/24, 12.5%) (p= 0,0085) for unresponsiveness or intolerance to therapy. At the end of the 6-month therapy, both groups had similarly improved in the Rankin, ONLS, Rotterdam and SF-36 quality of life scale and the IVIg group also in the MRC and sensory score and time to walk 10 meters. After therapy discontinuation more patients on IVIg (8/21, 38.1%) than IVMP (0/10) worsened and required further therapy. By 12 months, a similar proportion of patients remained improved without therapy in the IVIg (13/24, 54.1%) and IVMP (10/21, 47.6%) group. Conclusions: More patients on IVMP than on IVIg suspended therapy during the six months of therapy because of inefficacy or adverse events. After therapy discontinuation, more patients treated with IVIg than with IVMP worsened and had to resume therapy. Supported by: Kedrion SpA, Italy. Disclosure: Dr. Nobile-Orazio has received personal compensation for activities with CSL Behring. Dr. Cocito has nothing to disclose. Dr. Jann has nothing to disclose. Dr. Uncini has nothing to disclose. Dr. Beghi has received personal compensation for activities with UCB Pharma as a speaker. Dr. Beghi has received personal compensation in an editorial capacity for Epilepsia. Dr. Antonini has nothing to disclose. Dr. Fazio has nothing to disclose. Dr. Gallia has nothing to disclose. Dr. Schenone has nothing to disclose. Dr. Francia has nothing to disclose. Dr. Pareyson has nothing to disclose. Dr. Santoro has nothing to disclose. Dr. Tamburin has nothing to disclose. Dr. Macchia has received personal compensation for activities with Kedrion as an employee. Dr. Guarneri has received personal compensation for activities with Kedrion Biopharmaceutical an employee. Dr. Cavaletti has nothing to disclose. Dr. Giannini has nothing to disclose. Dr. Sabatelli has nothing to disclose.
OBJECTIVE. To compare the frequency and time to relapse after discontinuing 6-months therapy with intravenous immunoglobulin (IVIg) or methylprednisolone (IVMP) in patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). BACKGROUND. We recently reported that 6-month therapy with IVIg was more frequently effective or tolerated than methylprednisolone (IVMP) in patients with CIDP. More patients however worsened and required therapy in the six-month following IVIg than IVMP discontinuation. We extended the follow-up to compare the proportion of patients who eventually relapsed after therapy discontinuation. METHODS. By march 2013, data were available from 41 out of the 45 patients completing the trial with a median follow-up after therapy discontinuation of 42 months (range 1-60) Three patients had retired during the original study and one failed to respond to both therapies. No patient received a diagnosis alternative to CIDP during the follow-up. RESULTS. Twenty-eight of the 32 patients treated with IVIg as primary or secondary therapy after failing to respond to IVMP improved after therapy (87.5%) as compared to 13 of the 24 patients treated with IVMP as primary or secondary therapy (54.2%). After a median follow-up of 42 months (range 1-57), 24 out of 28 patients responsive to IVIg (85.7%) worsened after therapy discontinuation. The same occurred to 10 out of 13 patients (76.9%) treated with IVMP (p: 0.659) after a median follow-up of 43 months (range 7-60). Worsening occurred 1-24 months (median 4.5) after IVIg discontinuation and 1-31 months (median 14) after IVMP discontinuation (p: 0.0126). Kaplan-Meier survival curves of the between groups time to relapse censored at 1, 2 and 3 years of follow-up reported log-rank p-values of 0.0139, 0.0272 and 0.0278. CONCLUSIONS. A similarly high proportion of patients treated with IVIg or IVMP eventually relapse after therapy discontinuation but the median time to relapse is significantly longer after IVMP than IVIg. These results may influence the initial choice of treatment in CIDP.
This record contains data related to article “Sensitivity and specificity of a commercial ELISA test for anti-MAG antibodies in patients with neuropathy". Abstract For the diagnosis of anti-MAG polyneuropathy the commercial ELISA manufacturer currently recommends a cut-off of 1000 Bühlmann Titer Units (BTU). We analyzed sera from 80 anti-MAG neuropathy patients and 383 controls (with other neuropathies or healthy controls) to assess the ELISA sensitivity and specificity at different thresholds. A better combination of sensitivity/specificity was found at a threshold >1500 BTU than at >1000 BTU. The best value of specificity was obtained at threshold >7000 BTU. There was a diagnostic grey area between 1500 and 7000 BTU in which the clinical phenotypes as well as electrophysiological studies need to be carefully assessed particularly to differentiate CIDP and anti-MAG neuropathy.
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