المؤلفون: Bart C. Jacobs, Pieter A. van Doorn, Peter Van den Bergh, Carina Bunschoten, David R. Cornblath

المساهمون: UCL - SSS/IONS/NEUR - Clinical Neuroscience, UCL - (SLuc) Service de neurologie, Neurology, Immunology

المصدر: The Lancet. Neurology, Vol. 18, no. 8, p. 784-794 (2019)
Lancet Neurology, 18(8), 784-794. Lancet Publishing Group

مصطلحات موضوعية: 0301 basic medicine, medicine.medical_specialty, Future studies, Nerve ultrasound, Disease, Subcutaneous immunoglobulin, 03 medical and health sciences, 0302 clinical medicine, Adrenal Cortex Hormones, Immunoglobulin g4, medicine, Humans, Intensive care medicine, Plasma Exchange, Treatment regimen, business.industry, Immunoglobulins, Intravenous, Polyradiculoneuropathy, medicine.disease, Treatment Outcome, 030104 developmental biology, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating, Neurology (clinical), business, 030217 neurology & neurosurgery, After treatment

الوصف: Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is a rare and heterogeneous but treatable immune-mediated neuropathy. Nerve conduction studies are considered essential for a definite diagnosis, but poor performance and misinterpretation of the results frequently leads to misdiagnosis. Nerve ultrasound and MRI could be helpful in diagnosis. Whereas typical CIDP is relatively easy to diagnose, atypical variants with distinct phenotypes can be a diagnostic challenge. Intravenous or subcutaneous immunoglobulin, corticosteroids, and plasma exchange are effective treatments, but maintenance treatments are often required for years, and treatment regimens require careful and regular adjustments to avoid undertreatment or overtreatment. Patients who do not improve, or insufficiently improve after treatment, might have specific characteristics related to a distinct disease mechanism caused by immunoglobulin G4 antibodies to nodal or paranodal proteins, and could require alternative treatments. Future studies should focus on curative and individualised treatment regimens to improve the patient's condition and to prevent further nerve damage.

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c03562102041e48b98e68f5555bc0d84Test
https://doi.org/10.1016/s1474-4422Test(19)30144-9

المؤلفون: James Miller, Richard A. C. Hughes, Peter Van den Bergh, Gwendal Le Masson, Masahiro Iijima, Annie Dionne, Jérôme De Seze, Timothy Day, Norman Latov, Ewa Motta, David R. Cornblath, Alain Maertens de Noordhout, Ingemar S. J. Merkies, S. Larue, Jens Ejbye Schmidt, Jean-Marc Léger, Stanley Iyadurai, Hans-Peter Hartung, Carolyn Marie Ervin, Anthony A. Amato, Rup Tandan, Judith Spies, Krzysztof Selmaj, William Camu, Michel Melanson, Vivian E. Drory, Masahiro Mori, Eduardo Nobile-Orazio, Waldemar Fryze, Martin Merschhemke, Marinos C. Dalakas, Masayuki Baba, Martin M. Brown, James Holt, John Kelemen, Antonio Guerrero Sola, Thomas H. Brannagan, Jean Pouget, Victoria Lawson, Tomoko Okamoto, Philip Van Damme, Susumu Kusunoki, Khema Sharma, Joab Chapman, Mark Gudesblatt, Carlos Casasnovas, Vasilios K Kimiskidis, Kourosh Rezania, Gen Sobue, Leslie Roberts, Isabel Illa, Angela Genge, Rami Massie, Ivo N. van Schaik, Raffaella Fazio, Catharina G. Faber, Francesca Gallia, Michael P. Lunn, Catherine Agoropoulou

المساهمون: Centre d'Immunologie et de Maladies Infectieuses (CIMI), Université Pierre et Marie Curie - Paris 6 (UPMC)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), Montreal Neurological Institute and Hospital, McGill University = Université McGill [Montréal, Canada], Centre Hospitalier Régional Universitaire [Montpellier] (CHRU Montpellier), Université de Montpellier (UM), RS: MHeNs - R1 - Cognitive Neuropsychiatry and Clinical Neuroscience, McGill University

المصدر: The Lancet Neurology
The Lancet Neurology, Elsevier, 2018, 17 (8), pp.689--698. ⟨10.1016/S1474-4422(18)30202-3⟩
Lancet Neurology, 17(8), 689-698. Elsevier Science

مصطلحات موضوعية: FTY720, Male, [SDV]Life Sciences [q-bio], law.invention, Disability Evaluation, Electrocardiography, 0302 clinical medicine, Randomized controlled trial, law, Adrenal Cortex Hormones, Clinical endpoint, Medicine, Data monitoring committee, 030212 general & internal medicine, Chronic Inflammatory Demyelinating, Hand Strength, Middle Aged, Fingolimod, 3. Good health, LYMPHOCYTE, Treatment Outcome, Administration, GRIP STRENGTH, Female, Intravenous, Immunosuppressive Agents, medicine.drug, Oral, Adult, medicine.medical_specialty, Polyradiculoneuropathy, Immunoglobulins, CIDP, Placebo, 03 medical and health sciences, Double-Blind Method, Internal medicine, Fingolimod Hydrochloride, Humans, Aged, Proportional Hazards Models, NEUROPATHIES, business.industry, Interim analysis, Discontinuation, Neurology (clinical), RELAPSING MULTIPLE-SCLEROSIS, business, 030217 neurology & neurosurgery, Follow-Up Studies

الوصف: International audience; BACKGROUND: Fingolimod is approved for the treatment of relapsing-remitting multiple sclerosis and was effective in experimental autoimmune neuritis in rats, a possible model for chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). We aimed to evaluate the efficacy of fingolimod in delaying disability progression in patients with CIDP who withdrew from currently effective treatments (intravenous immunoglobulin [IVIg] or corticosteroids). METHODS: This double-blind, multicentre, randomised, placebo-controlled, parallel-group, event-driven study was done at 48 neurology centres in Australia, Canada, Israel, Japan, the USA, and nine countries in Europe. Participants with CIDP who were receiving IVIg or corticosteroids were randomly assigned (1:1) to once-daily oral fingolimod 0\textperiodcentered5 mg or placebo. Owing to the event-driven design, treatment duration was flexible and could be up to 4\textperiodcentered5 years. Randomisation was done with an automated interactive voice response-web response system and was stratified by Inflammatory Neuropathy Cause and Treatment (INCAT) disability scale scores. Previous IVIg treatment was discontinued after one final course ending the day before the first dose of fingolimod or placebo was given, whereas corticosteroids were tapered off over 8 weeks after randomisation. The primary endpoint was time to first confirmed worsening (>=1 point increase on the adjusted INCAT disability scale score versus baseline) and was assessed in the full analysis set, which consisted of all patients who underwent randomisation and had at least one efficacy assessment for the primary analysis. The survival distribution functions of time to first worsening were estimated within each treatment group according to the Kaplan-Meier survival distribution function and compared with a stratified log-rank test. The trial is registered with ClinicalTrials.gov, number NCT01625182. FINDINGS: Of 106 participants randomly assigned between Jan 24, 2013, and March 10, 2016, 54 received fingolimod (41 who had been receiving IVIg and 13 who had been receiving corticosteroids) and 52 received placebo (41 who had been receiving IVIg and 11 who had been receiving corticosteroids). The trial ended for futility as recommended by an independent data monitoring committee after an interim analysis when 44 confirmed worsening events had occurred. At the end of the study, the survival estimate of the proportion of participants free from confirmed worsening was not significantly different between the fingolimod group (42%, 95% CI 23-60) and the placebo group (43%, 28-59; p=0\textperiodcentered91). Adverse events occurred in 41 (76%) participants receiving fingolimod and 44 (85%) on placebo, and serious adverse events occurred in nine (17%) and four (8%) patients, respectively. The most common adverse events with fingolimod were headache (12 [22%] patients), hypertension (ten [19%]), and extremity pain (seven [13%]). Adverse events leading to study discontinuation occurred in seven (13%) participants on fingolimod and none on placebo. INTERPRETATION: Fingolimod 0\textperiodcentered5 mg once-daily was not better than placebo for the treatment of CIDP. Future trial designs should take account of the possibility that if IVIg is stopped abruptly, some patients might relapse soon afterwards whereas others might remain in remission. FUNDING: Novartis Pharma.

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::22492e836639116d2d45267972e919e9Test
https://hal.archives-ouvertes.fr/hal-02317675Test

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: Yusuf A. Rajabally, Peter Van den Bergh

المصدر: Presse medicale (Paris, France : 1983). 42(6 Pt 2)

مصطلحات موضوعية: medicine.medical_specialty, Nerve biopsy, biology, medicine.diagnostic_test, business.industry, Prevalence, Disease Management, Peripheral Nervous System Diseases, Polyradiculoneuropathy, General Medicine, Disease, Gold standard (test), medicine.disease, Gastroenterology, Pathophysiology, Pathogenesis, Treatment Outcome, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating, Internal medicine, biology.protein, Medicine, Humans, Immunotherapy, Antibody, business

الوصف: Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is the most common autoimmune neuropathy. The diagnosis depends on the clinical presentation with a progressive or relapsing course over at least 2 months and electrophysiological evidence of primary demyelination. Whereas typical CIDP is quite easily recognizable because virtually no other neuropathies present with both distal and proximal motor and sensory deficit, atypical CIDP, focal and multifocal variants in particular, may represent a difficult diagnostic challenge. CIDP very likely is an underdiagnosed condition as suggested also by a positive correlation between prevalence rates and sensitivity of electrophysiological criteria. Since no 'gold standard' diagnostic marker exists, electrophysiological criteria have been optimized to be at the same time as sensitive and as specific as possible. Additional supportive laboratory features, such as increased spinal fluid protein, MRI abnormalities of nerve segments, and in selected cases nerve biopsy lead to the correct diagnosis in the large majority of the cases. Objective clinical improvement following immune therapy is also a useful parameter to confirm the diagnosis. The pathogenesis and pathophysiology of CIDP remain poorly understood, but the available evidence for an inflammatory origin is quite convincing. Steroids, intravenous immunoglobulin (IVIG), and plasma exchange (PE) have been proven to be effective treatments. IVIG usually leads to rapid improvement, which is useful in severely disabled patients. Repeat treatment over regular time intervals for many years is often necessary. The effect of steroids is slower and the side-effect profile may be problematic, but they may induce disease remission more frequently than IVIG. An important and as of yet uncompletely resolved issue is the evaluation of long-term outcome to determine whether the disease is still active and responsive to treatment.

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::24adb898fff4325bd3aa1b3c2de19723Test
https://pubmed.ncbi.nlm.nih.gov/23623582Test

المؤلفون: Giancarlo, Comi, Luisa, Roveri, Antony, Swan, Hugh, Willison, Martin, Bojar, Isabel, Illa, Clementine, Karageorgiou, Eduardo, Nobile-Orazio, Peter, van den Bergh, Tony, Swan, Richard, Hughes, Jaques, Aubry, Nicole, Baumann, Robert, Hadden, Michael, Lunn, Martin, Knapp, Jean-Marc, Léger, Pierre, Bouche, Radim, Mazanec, Nicoletta, Meucci, Frans, van der Meché, Klaus, Toyka

مصطلحات موضوعية: Male, medicine.medical_specialty, Paraproteinemias, Polyradiculoneuropathy, Placebo, law.invention, Route of administration, Grip strength, Disability Evaluation, Randomized controlled trial, Double-Blind Method, law, hemic and lymphatic diseases, Internal medicine, medicine, Humans, Intravenous immunoglobulin, Paraproteinaemic demyelinating neuropathy, Adverse effect, Aged, Cross-Over Studies, business.industry, Immunoglobulins, Intravenous, medicine.disease, Crossover study, Surgery, Clinical trial, Peripheral neuropathy, Treatment Outcome, Neurology, Immunoglobulin M, Female, Settore MED/26 - Neurologia, Neurology (clinical), business

الوصف: This multicentre randomised double blind crossover trial tested the short term efficacy of intravenous immunoglobulin (IVIg) 2.0 g/kg given over 24 or 48 hours in patients with paraproteinaemic demyelinating neuropathy (PDN). Twenty-two patients were randomised and completed the trial. After 2 weeks, the overall disability grade decreased during both IVIg treatment and placebo but neither change was significant nor was the mean difference between the treatment effects. After 4 weeks the overall disability decreased by a mean of 0.55 [0.67] grades during the IVIg period (p = 0.001) while it was substantially unmodified during the placebo period. The mean difference between the treatment effects was significant (p = 0.05). Overall during the IVIg period 10 patients improved and 11 were stable and one got worse. During the placebo period 4 patients improved, 4 deteriorated and 14 were stable. Many secondary outcome measures, including Rankin scale, time to walk 10 metres, grip strength, sensory symptoms score were significantly better during IVIg treatment. Two serious adverse events occurred during the trial, both during placebo treatment. In conclusion the trial showed some short-term benefit of IVIg in about half of the patients confirming previous observation.

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4b3eed6b37dce47e06de45a49e87447dTest
http://hdl.handle.net/2434/15224Test