المؤلفون: Michael Benatar, Bernhard Greve, Peter Kiessling, Melissa Brock, Henning Andersen, John Vissing, Peter Van den Bergh, Franz Woltering, Laura Griffin, Vera Bril

المصدر: Neurology.

مصطلحات موضوعية: medicine.medical_specialty, business.industry, Placebo, medicine.disease, Gastroenterology, Myasthenia gravis, law.invention, Clinical trial, 03 medical and health sciences, 0302 clinical medicine, Randomized controlled trial, law, Multicenter trial, Internal medicine, Clinical endpoint, Medicine, 030212 general & internal medicine, Neurology (clinical), business, Generalized myasthenia, Adverse effect, 030217 neurology & neurosurgery

الوصف: ObjectiveTo explore the clinical efficacy and safety of subcutaneous (SC) rozanolixizumab, an anti-neonatal Fc receptor humanized monoclonal antibody, in patients with generalized myasthenia gravis (gMG).MethodsIn this phase 2a, randomized, double-blind, placebo-controlled, 2-period, multicenter trial (NCT03052751), patients were randomized (1:1) in period 1 (days 1–29) to 3 once-weekly (Q1W) SC infusions of rozanolixizumab 7 mg/kg or placebo. In period 2 (days 29–43), patients were re-randomized to either rozanolixizumab 7 mg/kg or 4 mg/kg (3 Q1W SC infusions), followed by an observation period (days 44–99). Primary endpoint: change from baseline to day 29 in Quantitative Myasthenia Gravis (QMG) score. Secondary endpoints: change from baseline to day 29 in MG-Activities of Daily Living (MG-ADL) and MG-Composite (MGC) scores; safety.ResultsForty-three patients were randomized (rozanolixizumab: 21 patients; placebo: 22 [period 1]). Least squares (LS) mean change from baseline to day 29 for rozanolixizumab vs placebo: QMG (LS mean: −1.8 vs −1.2; difference: −0.7; 95% UCL: 0.8; p = 0.221; not statistically significant), MG-ADL (LS mean: −1.8 vs −0.4; difference: −1.4; 95% UCL: −0.4), and MGC (LS mean: −3.1 vs −1.2; difference: −1.8; 95% UCL: 0.4) scores. Efficacy measures continued to improve with rozanolixizumab 7 mg/kg in period 2. The most common adverse event in period 1 was headache (rozanolixizumab: 57%; placebo: 14%).ConclusionWhile change from baseline in QMG was not statistically significant, the data overall suggest rozanolixizumab may provide clinical benefit in patients with gMG and was generally well tolerated. Phase 3 evaluation is ongoing (NCT03971422).Classification of evidenceThis study provides class I evidence that for patients with gMG, rozanolixizumab is well tolerated, but did not significantly improve QMG score.

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::587f79858e5ceb05d888b31a4529dbd2Test
https://doi.org/10.1212/wnl.0000000000011108Test

المؤلفون: Hans D. Katzberg, Jean Marc Léger, Kenneth C. Gorson, Eduardo Nobile-Orazio, Catharina G. Faber, Pieter A. van Doorn, Jean Pouget, Giuseppe Lauria, Ingemar S. J. Merkies, Sonja I. van Nes, Angelika F. Hahn, Anneke J. van der Kooi, W. Ludo van der Pol, Leonard H. van den Berg, Nicolette C. Notermans, Peter Van den Bergh, David R. Cornblath, Vera Bril, Els K. Vanhoutte, Thomas H P Draak, Michael P. Lunn

المساهمون: Neurology, MUMC+: DA KG Polikliniek (9), RS: MHeNs - R1 - Cognitive Neuropsychiatry and Clinical Neuroscience, Klinische Neurowetenschappen, ANS - Amsterdam Neuroscience

المصدر: Neurology, 83(23), 2124-2132. Lippincott Williams & Wilkins
Neurology, 83(23), 2124-2132. LIPPINCOTT WILLIAMS & WILKINS
Neurology, 83(23), 2124-2132. Lippincott Williams and Wilkins

مصطلحات موضوعية: Adult, Male, medicine.medical_specialty, Time Factors, Guillain-Barre Syndrome, Severity of Illness Index, New diagnosis, Polyneuropathies, Internal medicine, Gammopathy, medicine, Humans, Aged, Aged, 80 and over, Rasch model, business.industry, Minimal clinically important difference, Polyradiculoneuropathy, Middle Aged, medicine.disease, Standard error, Immunoglobulin M, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating, Physical therapy, Female, Neurology (clinical), sense organs, business, Inflammatory neuropathy, Polyneuropathy

الوصف: Objectives: We performed responsiveness comparison between the patient-reported Inflammatory Rasch-built Overall Disability Scale (I-RODS) and the widely used clinician-reported Inflammatory Neuropathy Cause and Treatment-Overall Neuropathy Limitation Scale (INCAT-ONLS) in patients with Guillain-Barre syndrome (GBS), chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), and immunoglobulin M-monoclonal gammopathy of undetermined significance related polyneuropathy (IgM-MGUSP). Methods: One hundred thirty-seven patients (GBS: 55, CIDP: 59, IgM-MGUSP: 23) with a new diagnosis or clinical relapse assessed both scales. Patients with GBS/CIDP were examined at 0, 1, 3, 6, and 12 months; patients with IgM-MGUSP at 0, 3, and 12. We subjected all data to Rasch analyses, and calculated for each patient the magnitude of change on both scales using the minimal clinically important difference (MCID) related to the individual standard errors (SEs). A responder was defined as having anMCID-SE >= 1.96. Individual scores on both measures were correlated with the EuroQoL thermometer (heuristic responsiveness). Results: The I-RODS showed a significantly higher proportion of meaningful improvement compared with the INCAT-ONLS findings in GBS/CIDP. For IgM-MGUSP, the lack of responsiveness during the 1-year study did not allow a clear separation. Heuristic responsiveness was consistently higher with the I-RODS. Conclusion: The I-RODS more often captures clinically meaningful changes over time, with a greater magnitude of change, compared with the INCAT-ONLS disability scale in patients with GBS and CIDP. The I-RODS offers promise for being a more sensitive measure and its use is therefore suggested in future trials involving patients with GBS and CIDP.

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b408ec3f92c81a65378b6cc3c9fbbc0fTest
https://doi.org/10.1212/wnl.0000000000001044Test

المؤلفون: Nicol C. Voermans, Thierry Kuntzer, Michel Delforge, Marie-Christiane Vekemans, Olivier Benveniste, Monique C. Minnema, Peter Van den Bergh, Véronique Leblond, Norma B. Romero, Jan Novy, Thomas Pabst, Françoise Bouhour, Henk M. Lokhorst, Bruno Eymard, Wouter Meersseman, Baziel G.M. van Engelen, Martin Lammens

المصدر: Neurology
Neurology, 83, 23, pp. 2133-9
Voermans, Nicol C; Benveniste, Olivier; Minnema, Monique C; Lokhorst, Henk; Lammens, Martin; Meersseman, Wouter; Delforge, Michel; Kuntzer, Thierry; Novy, Jan; Pabst, Thomas; Bouhour, Françoise; Romero, Norma; Leblond, Veronique; Bergh, Peter van den; Vekemans, Marie-Christiane; Engelen, Baziel G van; Eymard, Bruno (2014). Sporadic late-onset nemaline myopathy with MGUS: long-term follow-up after melphalan and SCT. Neurology, 83(23), pp. 2133-2139. Lippincott Williams & Wilkins 10.1212/WNL.0000000000001047 <http://dx.doi.org/10.1212/WNL.0000000000001047Test>
ResearcherID
Europe PubMed Central
Neurology, 83, 2133-9

مصطلحات موضوعية: Melphalan, Adult, Male, medicine.medical_specialty, Paraproteinemias, Late onset, 610 Medicine & health, Myopathies, Nemaline, Gastroenterology, Transplantation, Autologous, Autologous stem-cell transplantation, Nemaline myopathy, immune system diseases, Internal medicine, hemic and lymphatic diseases, Medicine, Humans, Age of Onset, Myopathy, Retrospective Studies, business.industry, Muscle weakness, medicine.disease, Disorders of movement Donders Center for Medical Neuroscience [Radboudumc 3], Hematologic Response, 3. Good health, Surgery, Treatment Outcome, Female, Neurology (clinical), Human medicine, medicine.symptom, business, Case series, medicine.drug, Follow-Up Studies, Stem Cell Transplantation

الوصف: Item does not contain fulltext OBJECTIVE: Sporadic late-onset nemaline myopathy (SLONM) is a rare, late-onset myopathy that progresses subacutely. If associated with a monoclonal gammopathy of unknown significance (MGUS), the outcome is unfavorable: the majority of these patients die within 1 to 5 years of respiratory failure. This study aims to qualitatively assess the long-term treatment effect of high-dose melphalan (HDM) followed by autologous stem cell transplantation (SCT) in a series of 8 patients with SLONM-MGUS. METHODS: We performed a retrospective case series study (n = 8) on the long-term (1-8 years) treatment effect of HDM followed by autologous SCT (HDM-SCT) on survival, muscle strength, and functional capacities. RESULTS: Seven patients showed a lasting moderate-good clinical response, 2 of them after the second HDM-SCT. All of them had a complete, a very good partial, or a partial hematologic response. One patient showed no clinical or hematologic response and died. CONCLUSIONS: This case series shows the positive effect of HDM-SCT in this rare disorder. Factors that may portend an unfavorable outcome are a long disease course before the hematologic treatment and a poor hematologic response. Age at onset, level and type of M protein (kappa vs lambda), and severity of muscle weakness were not associated with a specific outcome. CLASSIFICATION OF EVIDENCE: This study provides Class IV evidence that for patients with SLONM-MGUS, HDM-SCT increases the probability of survival and functional improvement.

وصف الملف: pdf; application/pdf

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::8519e93627827a67a1c17d2569a0441dTest
https://hdl.handle.net/10067/1221760151162165141Test

المؤلفون: Jean-Louis Thonnard, Christine Detrembleur, Peter Van den Bergh

المصدر: Neurology. 49(1)

مصطلحات موضوعية: Male, medicine.medical_specialty, Hand function, Time Factors, medicine.diagnostic_test, business.industry, Neural Conduction, Motor control, Sensory system, Sensory loss, Chronic inflammatory demyelinating polyneuropathy, Electromyography, Middle Aged, medicine.disease, Hand, Surgery, Grip strength, Anesthesia, Sensation, Task Performance and Analysis, medicine, Humans, Neurology (clinical), business, Demyelinating Diseases

الوصف: A 60-year-old man presented with progressive large fiber sensory loss in the right first three fingers and, to a lesser extent, in both fourth and fifth fingers. Electrophysiologic studies were characteristic of chronic sensory demyelinating polyneuropathy, a variant of chronic inflammatory demyelinating polyneuropathy. Plasma exchange was unsuccessful, but intravenous immunoglobulin (IVIG) led to complete recovery of sensation for 2 months, although neurophysiologic abnormalities persisted. A battery of noninvasive tests to measure hand grip strength, tactile sensation at the fingertips, and motor control of prehension during precision grip revealed marked abnormalities in the right hand before IVIG. One month after IVIG, all test results had normalized, but they returned to pretreatment levels after 3 months. Functional evaluation of the hand may be a sensitive method to objectively quantify loss of and changes in cutaneous mechanoreceptor function of the fingers in large fiber sensory neuropathy.

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::37037f062868e154e2c2c3f0b1ea56f3Test
https://pubmed.ncbi.nlm.nih.gov/9222200Test