Search

Your search keyword '"Cornblath, DR"' showing total 295 results
Start Over Author "Cornblath, DR"
295 results on '"Cornblath, DR"'

1. Original research: Second IVIg course in Guillain-Barré syndrome with poor prognosis: the non-randomised ISID study

Author

Verboon, C, van den Berg, B, Cornblath, Dr, Venema, E, Gorson, Kc, Lunn, Mp, Lingsma, H, Van den Bergh, P, Harbo, T, Bateman, K, Pereon, Y, Sindrup, Sh, Kusunoki, S, Miller, J, Islam, Z, Hartung, Hp, Chavada, G, Jacobs, Bc, Hughes, Rac, van Doorn PA, Ajroud-Driss S, IGOS Consortium., Antonini, G, Attarian, S, Barroso, Fa, Benedetti, L, Bertorini, Te, Brannagan, Th, Briani, C, Bhavaraju-Sanka, R, Butterworth, S, Casasnovas, C, Cavaletti, G, Chen, S, Claeys, Kg, Cosgrove, Js, Davidson, A, Dardiotis, E, Dornonville de la Cour, C, Faber, Cg, Feasby, Te, Fujioka, T, Galassi, G, Gilchrist, Jm, Goyal, Na, Granit, V, Gutiérrez-Gutiérrez, G, Hadden, Rdm, Holt, Jkl, Htut, M, Jericó Pascual, I, Karafiath, S, Katzberg, Hd, Kiers, L, Kieseier, Bc, Kimpinski, K, Kuwabara, S, Kwan, Jy, Ladha, Ss, Lawson, V, Lehmann, H, Manji, H, Marfia, Ga, Márquez Infante, C, Mattiazzi, Mg, Mcdermott, Cj, Monges, Ms, Morís de la Tassa, G, Nascimbene, C, Nobile Orazio, E, Nowak, Rj, Osei-Bonsu, M, Pardo Fernandez, J, Querol Gutierrez, L, Reisin, R, Rinaldi, S, Rojas-Marcos, I, Rudnicki, Sa, Schenone, A, Sedano Tous MJ, Shahrizaila, N, Sheikh, K, Silvestri, Nj, Sommer, Cl, Varrato, Jd, Verschuuren, J, Vytopil, Mv, Zhou, L, Bella, Ir, Bunschoten, C, Bürmann, J, Busby, M, Chao, Cc, Conti, Me, Dalakas, Mc, Van Damme, P, Doets, A, van Dijk GW, Dimachkie, Mm, Doppler, K, Echaniz-Laguna, A, Eftimov, F, Fazio, R, Fokke, C, Fulgenzi, Ea, Garssen, Mpj, Gijsbers, Cj, Gilhuis, J, Grapperon, A, Hsieh, St, Illa, I, Islam, B, Jellema, K, Kaida, K, Kokubun, N, Kolb, N, van Koningsveld, R, van der Kooi AJ, Kuitwaard, K, Landschoff Lassen, L, Leonhard, Se, Mandarakas, M, Martinez Hernandez, E, Mohammad, Qd, Pulley, M, Rajabally, Ya, Reddel, Sw, van der Ree, T, Roodbol, J, Sachs, Gm, Samijn, Jpa, Santoro, L, Stein, B, Vermeij, Fh, Visser, Lh, Willison, Hj, Wirtz, P, Zivkovich, Sa., Neurology, Public Health, Immunology, and ANS - Neuroinfection & -inflammation

Subjects
Adult, Male, second IVIg course, Pediatrics, medicine.medical_specialty, Poor prognosis, Time Factors, Guillain-Barre Syndrome, Original research, Drug Administration Schedule, Disease course, Disability Evaluation, 03 medical and health sciences, 0302 clinical medicine, hemic and lymphatic diseases, Clinical endpoint, Humans, Immunologic Factors, Medicine, In patient, Aged, treatment, Guillain-Barre syndrome, business.industry, Immunoglobulins, Intravenous, Middle Aged, poor prognosis, Prognosis, Guillain-Barré syndrome, medicine.disease, Psychiatry and Mental health, Treatment Outcome, Immunoglobulin G, 030220 oncology & carcinogenesis, Female, Surgery, Observational study, Neurology (clinical), business, 030217 neurology & neurosurgery
Abstract

ObjectiveTo compare disease course in patients with Guillain-Barré syndrome (GBS) with a poor prognosis who were treated with one or with two intravenous immunoglobulin (IVIg) courses.MethodsFrom the International GBS Outcome Study, we selected patients whose modified Erasmus GBS Outcome Score at week 1 predicted a poor prognosis. We compared those treated with one IVIg course to those treated with two IVIg courses. The primary endpoint, the GBS disability scale at 4 weeks, was assessed with multivariable ordinal regression.ResultsOf 237 eligible patients, 199 patients received a single IVIg course. Twenty patients received an ‘early’ second IVIg course (1–2 weeks after start of the first IVIg course) and 18 patients a ‘late’ second IVIg course (2–4 weeks after start of IVIg). At baseline and 1 week, those receiving two IVIg courses were more disabled than those receiving one course. Compared with the one course group, the adjusted OR for a better GBS disability score at 4 weeks was 0.70 (95%CI 0.16 to 3.04) for the early group and 0.66 (95%CI 0.18 to 2.50) for the late group. The secondary endpoints were not in favour of a second IVIg course.ConclusionsThis observational study did not show better outcomes after a second IVIg course in GBS with poor prognosis. The study was limited by small numbers and baseline imbalances. Lack of improvement was likely an incentive to start a second IVIg course. A prospective randomised trial is needed to evaluate whether a second IVIg course improves outcome in GBS.

Published
2019

2. Diretrizes Baseadas em Evidências Diagnóstico e manejo da Síndrome de Guillain–Barré em dez etapas

Author

Leonhard, SE, Mandarakas, MR, Gondim, FAA, Bateman, K, Ferreira, MLB, Cornblath, DR, Van Doorn, PA, Dourado, ME, Hughes, RAC, Islam, B, Kusunoki, S, Pardo, CA, Reisin, R, Sejvar, JJ, Shahrizaila, N, Soares, C, Umapathi, T, Wang, Y, Yiu, EM, Willison, HJ, Jacobs, BC, Leonhard, SE, Mandarakas, MR, Gondim, FAA, Bateman, K, Ferreira, MLB, Cornblath, DR, Van Doorn, PA, Dourado, ME, Hughes, RAC, Islam, B, Kusunoki, S, Pardo, CA, Reisin, R, Sejvar, JJ, Shahrizaila, N, Soares, C, Umapathi, T, Wang, Y, Yiu, EM, Willison, HJ, and Jacobs, BC

Abstract

A síndrome de Guillain–Barré (SGB) é uma doença imunomediada rara, mas potencialmente fatal, dos nervos periféricos e das raízes nervosas, que é geralmente desencadeada por infecções. A incidência da SGB pode, portanto, aumentar durante surtos de doenças infecciosas, como foi observado durante a epidemia do vírus Zika em 2013 na Polinésia Francesa e em 2015 na América Latina. O diagnóstico e manejo da SGB podem ser complicados visto que sua apresentação clínica e o curso da doença são heterogêneos e não existem atualmente diretrizes clínicas internacionais disponíveis. Para auxiliar os médicos, especialmente em um cenário de surto, desenvolvemos uma diretriz globalmente aplicável para o diagnóstico e manejo da SGB. A diretriz se baseia no consenso de especialistas e na literatura atual e tem uma estrutura de dez etapas para facilitar seu uso na prática clínica. Primeiro fornecemos uma introdução aos critérios diagnósticos, às variantes clínicas e aos diagnósticos diferenciais da SGB. A seguir, as dez etapas abrangem o reconhecimento e o diagnóstico precoces da SGB, a internação na unidade de terapia intensiva, a indicação e seleção do tratamento, o monitoramento e tratamento da progressão da doença, o prognóstico do curso e resultado clínico e o manejo das complicações e sequelas.

Published
2021

3. Guillain-Barré Syndrome Outbreak in Peru 2019 Associated With Campylobacter jejuni Infection

Author

Ramos, AP, Leonhard, Sonja, Halstead, SK, Cuba, MA, Castañeda, CC, Dioses, JA, Tipismana, MA, Abanto, JT, Llanos, A, Gourlay, D, Grogl, M, Ramos, M, Rojas, JD, Meza, R, Puiu, D, Sherman, RM, Salzberg, SL, Simner, PJ, Willison, HJ, Jacobs, B.C., Cornblath, DR, Umeres, HF, Pardo, CA, Ramos, AP, Leonhard, Sonja, Halstead, SK, Cuba, MA, Castañeda, CC, Dioses, JA, Tipismana, MA, Abanto, JT, Llanos, A, Gourlay, D, Grogl, M, Ramos, M, Rojas, JD, Meza, R, Puiu, D, Sherman, RM, Salzberg, SL, Simner, PJ, Willison, HJ, Jacobs, B.C., Cornblath, DR, Umeres, HF, and Pardo, CA

Abstract

OBJECTIVE: To identify the clinical phenotypes and infectious triggers in the 2019 Peruvian Guillain-Barré syndrome (GBS) outbreak. METHODS: We prospectively collected clinical and neurophysiologic data of patients with GBS admitted to a tertiary hospital in Lima, Peru, between May and August 2019. Molecular, immunologic, and microbiological methods were used to identify causative infectious agents. Sera from 41 controls were compared with cases for antibodies to Campylobacter jejuni and gangliosides. Genomic analysis was performed on 4 C jejuni isolates. RESULTS: The 49 included patients had a median age of 44 years (interquartile range [IQR] 30-54 years), and 28 (57%) were male. Thirty-two (65%) had symptoms of a preceding infection: 24 (49%) diarrhea and 13 (27%) upper respiratory tract infection. The median time between infectious to neurologic symptoms was 3 days (IQR 2-9 days). Eighty percent had a pure motor form of GBS, 21 (43%) had the axonal electrophysiologic subtype, and 18% the demyelinating subtype. Evidence of recent C jejuni infection was found in 28/43 (65%). No evidence of recent arbovirus infection was found. Twenty-three cases vs 11 controls (OR 3.3, confidence interval [CI] 95% 1.2-9.2, p < 0.01) had IgM and/or IgA antibodies against C jejuni. Anti-GM1:phosphatidylserine and/or anti-GT1a:GM1 heteromeric complex antibodies were strongly positive in cases (92.9% sensitivity and 68.3% specificity). Genomic analysis showed that the C jejuni strains were closely related and had the Asn51 polymorphism at cstII gene. CONCLUSIONS: Our study indicates that the 2019 Peruvian GBS outbreak was associated with C jejuni infection and that the C jejuni strains linked to GBS circulate widely in different parts of the world.

Published
2021

4. Placebo effect in chronic inflammatory demyelinating polyneuropathy: ThePATHstudy and a systematic review

Author

Lewis, RA, Cornblath, DR, Hartung, H-P, Sobue, G, Lawo, J-P, Mielke, O, Durn, BL, Bril, V, Merkies, ISJ, Bassett, P, Cleasby, A, van Schaik, IN, Lewis, RA, Cornblath, DR, Hartung, H-P, Sobue, G, Lawo, J-P, Mielke, O, Durn, BL, Bril, V, Merkies, ISJ, Bassett, P, Cleasby, A, and van Schaik, IN

Abstract

The Polyneuropathy And Treatment with Hizentra (PATH) study required subjects with chronic inflammatory demyelinating polyneuropathy (CIDP) to show dependency on immunoglobulin G (IgG) and then be restabilized on IgG before being randomized to placebo or one of two doses of subcutaneous immunoglobulin (SCIG). Nineteen of the 51 subjects (37%) randomized to placebo did not relapse over the next 24 weeks. This article explores the reasons for this effect. A post-hoc analysis of the PATH placebo group was undertaken. A literature search identified other placebo-controlled CIDP trials for review and comparison. In PATH, subjects randomized to placebo who did not relapse were significantly older, had more severe disease, and took longer to deteriorate in the IgG dependency period compared with those who relapsed. Published trials in CIDP, whose primary endpoint was stability or deterioration, had a mean non-deterioration (placebo effect) of 43%, while trials with a primary endpoint of improvement had a placebo response of only 11%. Placebo is an important variable in the design of CIDP trials. Trials designed to show clinical improvement will have a significantly lower effect of this phenomenon than those designed to show stability or deterioration.

Published
2020

6. Efficacy and safety of IVIG in CIDP: Combined data of the PRIMA and PATH studies

Author

Merkies, ISJ, van Schaik, IN, Leger, J-M, Bril, V, van Geloven, N, Hartung, H-P, Lewis, RA, Sobue, G, Lawo, J-P, Durn, BL, Cornblath, DR, De Bleecker, JL, Sommer, C, Robberecht, W, Saarela, M, Kamienowski, J, Stelmasiak, Z, Tackenberg, B, Mielke, O, Sabet, A, George, K, Roberts, L, Carne, R, Blum, S, Henderson, R, Van Damme, P, Demeestere, J, Larue, S, D'Amour, C, Kunc, P, Valis, M, Sussova, J, Kalous, T, Talab, R, Bednar, M, Toomsoo, T, Rubanovits, I, Gross-Paju, K, Sorro, U, Auranen, M, Pouget, J, Attarian, S, Le Masson, G, Wielanek-Bachelet, A, Desnuelle, C, Delmont, E, Clavelou, P, Aufauvre, D, Schmidt, J, Zschuentzsch, J, Kramer, D, Hoffmann, O, Goerlitz, C, Haas, J, Chatzopoulos, M, Yoon, R, Gold, R, Berlit, P, Jaspert-Grehl, A, Liebetanz, D, Kutschenko, A, Stangel, M, Trebst, C, Baum, P, Bergh, F, Klehmet, J, Meisel, A, Klostermann, F, Oechtering, J, Lehmann, H, Schroeter, M, Hagenacker, T, Mueller, D, Sperfeld, A, Bethke, F, Drory, V, Algom, A, Yarnitsky, D, Murinson, B, Di Muzio, A, Ciccocioppo, F, Sorbi, S, Mata, S, Schenone, A, Grandis, M, Lauria, G, Cazzato, D, Antonini, G, Morino, S, Cocito, D, Zibetti, M, Yokota, T, Ohkubo, T, Kanda, T, Kawai, M, Kaida, K, Onoue, H, Kuwabara, S, Mori, M, Iijima, M, Ohyama, K, Baba, M, Tomiyama, M, Nishiyama, K, Akutsu, T, Yokoyama, K, Kanai, K, Eftimov, F, Notermans, NC, Visser, N, Faber, C, Hoeijmakers, J, Rejdak, K, Chyrchel-Paszkiewicz, U, Casanovas Pons, C, Antonia, M, Gamez, J, Salvado, M, Marquez Infante, C, Benitez, S, Lunn, M, Morrow, J, Gosal, D, Lavin, T, Melamed, I, Testori, A, Ajroud-Driss, S, Menichella, D, Simpson, Lai, EC-H, Dimachkie, M, Barohn, RJ, Beydoun, S, Johl, H, Lange, D, Shtilbans, A, Muley, S, Ladha, S, Freimer, M, Kissel, J, Latov, N, Chin, R, Ubogu, E, Mumfrey, S, Rao, T, MacDonald, P, Sharma, K, Gonzalez, G, Allen, J, Walk, D, Hobson-Webb, L, Gable, K, Franques, J, Morales, RJ, Nguento, A, Schrey, C, Zwolinska, G, Merkies, ISJ, van Schaik, IN, Leger, J-M, Bril, V, van Geloven, N, Hartung, H-P, Lewis, RA, Sobue, G, Lawo, J-P, Durn, BL, Cornblath, DR, De Bleecker, JL, Sommer, C, Robberecht, W, Saarela, M, Kamienowski, J, Stelmasiak, Z, Tackenberg, B, Mielke, O, Sabet, A, George, K, Roberts, L, Carne, R, Blum, S, Henderson, R, Van Damme, P, Demeestere, J, Larue, S, D'Amour, C, Kunc, P, Valis, M, Sussova, J, Kalous, T, Talab, R, Bednar, M, Toomsoo, T, Rubanovits, I, Gross-Paju, K, Sorro, U, Auranen, M, Pouget, J, Attarian, S, Le Masson, G, Wielanek-Bachelet, A, Desnuelle, C, Delmont, E, Clavelou, P, Aufauvre, D, Schmidt, J, Zschuentzsch, J, Kramer, D, Hoffmann, O, Goerlitz, C, Haas, J, Chatzopoulos, M, Yoon, R, Gold, R, Berlit, P, Jaspert-Grehl, A, Liebetanz, D, Kutschenko, A, Stangel, M, Trebst, C, Baum, P, Bergh, F, Klehmet, J, Meisel, A, Klostermann, F, Oechtering, J, Lehmann, H, Schroeter, M, Hagenacker, T, Mueller, D, Sperfeld, A, Bethke, F, Drory, V, Algom, A, Yarnitsky, D, Murinson, B, Di Muzio, A, Ciccocioppo, F, Sorbi, S, Mata, S, Schenone, A, Grandis, M, Lauria, G, Cazzato, D, Antonini, G, Morino, S, Cocito, D, Zibetti, M, Yokota, T, Ohkubo, T, Kanda, T, Kawai, M, Kaida, K, Onoue, H, Kuwabara, S, Mori, M, Iijima, M, Ohyama, K, Baba, M, Tomiyama, M, Nishiyama, K, Akutsu, T, Yokoyama, K, Kanai, K, Eftimov, F, Notermans, NC, Visser, N, Faber, C, Hoeijmakers, J, Rejdak, K, Chyrchel-Paszkiewicz, U, Casanovas Pons, C, Antonia, M, Gamez, J, Salvado, M, Marquez Infante, C, Benitez, S, Lunn, M, Morrow, J, Gosal, D, Lavin, T, Melamed, I, Testori, A, Ajroud-Driss, S, Menichella, D, Simpson, Lai, EC-H, Dimachkie, M, Barohn, RJ, Beydoun, S, Johl, H, Lange, D, Shtilbans, A, Muley, S, Ladha, S, Freimer, M, Kissel, J, Latov, N, Chin, R, Ubogu, E, Mumfrey, S, Rao, T, MacDonald, P, Sharma, K, Gonzalez, G, Allen, J, Walk, D, Hobson-Webb, L, Gable, K, Franques, J, Morales, RJ, Nguento, A, Schrey, C, and Zwolinska, G

Abstract

Intravenous immunoglobulin (IVIG) is a potential therapy for chronic inflammatory demyelinating polyneuropathy (CIDP). To investigate the efficacy and safety of the IVIG IgPro10 (Privigen) for treatment of CIDP, results from Privigen Impact on Mobility and Autonomy (PRIMA), a prospective, open-label, single-arm study of IVIG in immunoglobulin (Ig)-naïve or IVIG pre-treated subjects (NCT01184846, n = 28) and Polyneuropathy And Treatment with Hizentra (PATH), a double-blind, randomized study including an open-label, single-arm IVIG phase in IVIG pre-treated subjects (NCT01545076, IVIG restabilization phase n = 207) were analyzed separately and together (n = 235). Efficacy assessments included change in adjusted inflammatory neuropathy cause and treatment (INCAT) score, grip strength and Medical Research Council (MRC) sum score. Adverse drug reactions (ADRs) and ADRs/infusion were recorded. Adjusted INCAT response rate was 60.7% in all PRIMA subjects at Week 25 (76.9% in IVIG pre-treated subjects) and 72.9% in PATH. In the pooled cohort (n = 235), INCAT response rate was 71.5%; median time to INCAT improvement was 4.3 weeks. No clear demographic differences were noticed between early (responding before Week 7, n = 148) and late responders (n = 21). In the pooled cohort, median change from baseline to last observation was -1.0 (interquartile range -2.0; 0.0) point for INCAT score; +8.0 (0.0; 20.0) kPa for maximum grip strength; +3.0 (1.0; 7.0) points for MRC sum score. In the pooled cohort, 271 ADRs were reported in 105 subjects (44.7%), a rate of 0.144 ADRs per infusion. This analysis confirms the efficacy and safety of IgPro10, a recently FDA-approved IVIG for CIDP, in a population of mainly pre-treated subjects with CIDP [Correction added on 14 March 2019 after first online publication: the INCAT response rate has been corrected.].

Published
2019

7. Diagnosis and management of Guillain-Barre syndrome in ten steps

Author

Leonhard, SE, Mandarakas, MR, Gondim, FAA, Bateman, K, Ferreira, MLB, Cornblath, DR, van Doorn, PA, Dourado, ME, Hughes, RAC, Islam, B, Kusunoki, S, Pardo, CA, Reisin, R, Sejvar, JJ, Shahrizaila, N, Soares, C, Umapathi, T, Wang, Y, Yiu, EM, Willison, HJ, Jacobs, BC, Leonhard, SE, Mandarakas, MR, Gondim, FAA, Bateman, K, Ferreira, MLB, Cornblath, DR, van Doorn, PA, Dourado, ME, Hughes, RAC, Islam, B, Kusunoki, S, Pardo, CA, Reisin, R, Sejvar, JJ, Shahrizaila, N, Soares, C, Umapathi, T, Wang, Y, Yiu, EM, Willison, HJ, and Jacobs, BC

Abstract

Guillain-Barré syndrome (GBS) is a rare, but potentially fatal, immune-mediated disease of the peripheral nerves and nerve roots that is usually triggered by infections. The incidence of GBS can therefore increase during outbreaks of infectious diseases, as was seen during the Zika virus epidemics in 2013 in French Polynesia and 2015 in Latin America. Diagnosis and management of GBS can be complicated as its clinical presentation and disease course are heterogeneous, and no international clinical guidelines are currently available. To support clinicians, especially in the context of an outbreak, we have developed a globally applicable guideline for the diagnosis and management of GBS. The guideline is based on current literature and expert consensus, and has a ten-step structure to facilitate its use in clinical practice. We first provide an introduction to the diagnostic criteria, clinical variants and differential diagnoses of GBS. The ten steps then cover early recognition and diagnosis of GBS, admission to the intensive care unit, treatment indication and selection, monitoring and treatment of disease progression, prediction of clinical course and outcome, and management of complications and sequelae.

Published
2019

8. Restabilization treatment after intravenous immunoglobulin withdrawal in chronic inflammatory demyelinating polyneuropathy: Results from the pre-randomization phase of the Polyneuropathy And Treatment with Hizentra study

Author

Mielke, O, Bril, V, Cornblath, DR, Lawo, J-P, van Geloven, N, Hartung, H-P, Lewis, RA, Merkies, ISJ, Sobue, G, Durn, B, Shebl, A, van Schaik, IN, Sabet, A, George, K, Roberts, L, Carne, R, Blum, S, Henderson, R, Van Damme, P, Demeestere, J, Larue, S, D'Amour, C, Kunc, P, Valis, M, Sussova, J, Kalous, T, Talab, R, Bednar, M, Toomsoo, T, Rubanovits, I, Gross-Paju, K, Sorro, U, Saarela, M, Auranen, M, Pouget, J, Attarian, S, Le Masson, G, Wielanek-Bachelet, A, Desnuelle, C, Delmont, E, Clavelou, P, Aufauvre, D, Schmidt, J, Zschuentzsch, J, Sommer, C, Kramer, D, Hoffmann, O, Goerlitz, C, Haas, J, Chatzopoulos, M, Yoon, R, Gold, R, Berlit, P, Jaspert-Grehl, A, Liebetanz, D, Kutschenko, A, Stangel, M, Trebst, C, Baum, P, Bergh, F, Klehmet, J, Meisel, A, Klostermann, F, Oechtering, J, Lehmann, H, Schroeter, M, Hagenacker, T, Mueller, D, Sperfeld, A, Bethke, F, Drory, V, Algom, A, Yarnitsky, D, Murinson, B, Di Muzio, A, Ciccocioppo, F, Sorbi, S, Mata, S, Schenone, A, Grandis, M, Lauria, G, Cazzato, D, Antonini, G, Morino, S, Cocito, D, Zibetti, M, Yokota, T, Ohkubo, T, Kanda, T, Kawai, M, Kaida, K, Onoue, H, Kuwabara, S, Mori, M, Iijima, M, Ohyama, K, Baba, M, Tomiyama, M, Nishiyama, K, Akutsu, T, Yokoyama, K, Kanai, K, Eftimov, F, Notermans, NC, Visser, N, Faber, C, Hoeijmakers, J, Rejdak, K, Chyrchel-Paszkiewicz, U, Casanovas Pons, C, Antonia, M, Gamez, J, Salvado, M, Marquez Infante, C, Benitez, S, Lunn, M, Morrow, J, Gosal, D, Lavin, T, Melamed, I, Testori, A, Ajroud-Driss, S, Menichella, D, Simpson, E, Lai, EC-H, Dimachkie, M, Barohn, RJ, Beydoun, S, Johl, H, Lange, D, Shtilbans, A, Muley, S, Ladha, S, Freimer, M, Kissel, J, Latov, N, Chin, R, Ubogu, E, Mumfrey, S, Rao, T, MacDonald, P, Sharma, K, Gonzalez, G, Allen, J, Walk, D, Hobson-Webb, L, Gable, K, Mielke, O, Bril, V, Cornblath, DR, Lawo, J-P, van Geloven, N, Hartung, H-P, Lewis, RA, Merkies, ISJ, Sobue, G, Durn, B, Shebl, A, van Schaik, IN, Sabet, A, George, K, Roberts, L, Carne, R, Blum, S, Henderson, R, Van Damme, P, Demeestere, J, Larue, S, D'Amour, C, Kunc, P, Valis, M, Sussova, J, Kalous, T, Talab, R, Bednar, M, Toomsoo, T, Rubanovits, I, Gross-Paju, K, Sorro, U, Saarela, M, Auranen, M, Pouget, J, Attarian, S, Le Masson, G, Wielanek-Bachelet, A, Desnuelle, C, Delmont, E, Clavelou, P, Aufauvre, D, Schmidt, J, Zschuentzsch, J, Sommer, C, Kramer, D, Hoffmann, O, Goerlitz, C, Haas, J, Chatzopoulos, M, Yoon, R, Gold, R, Berlit, P, Jaspert-Grehl, A, Liebetanz, D, Kutschenko, A, Stangel, M, Trebst, C, Baum, P, Bergh, F, Klehmet, J, Meisel, A, Klostermann, F, Oechtering, J, Lehmann, H, Schroeter, M, Hagenacker, T, Mueller, D, Sperfeld, A, Bethke, F, Drory, V, Algom, A, Yarnitsky, D, Murinson, B, Di Muzio, A, Ciccocioppo, F, Sorbi, S, Mata, S, Schenone, A, Grandis, M, Lauria, G, Cazzato, D, Antonini, G, Morino, S, Cocito, D, Zibetti, M, Yokota, T, Ohkubo, T, Kanda, T, Kawai, M, Kaida, K, Onoue, H, Kuwabara, S, Mori, M, Iijima, M, Ohyama, K, Baba, M, Tomiyama, M, Nishiyama, K, Akutsu, T, Yokoyama, K, Kanai, K, Eftimov, F, Notermans, NC, Visser, N, Faber, C, Hoeijmakers, J, Rejdak, K, Chyrchel-Paszkiewicz, U, Casanovas Pons, C, Antonia, M, Gamez, J, Salvado, M, Marquez Infante, C, Benitez, S, Lunn, M, Morrow, J, Gosal, D, Lavin, T, Melamed, I, Testori, A, Ajroud-Driss, S, Menichella, D, Simpson, E, Lai, EC-H, Dimachkie, M, Barohn, RJ, Beydoun, S, Johl, H, Lange, D, Shtilbans, A, Muley, S, Ladha, S, Freimer, M, Kissel, J, Latov, N, Chin, R, Ubogu, E, Mumfrey, S, Rao, T, MacDonald, P, Sharma, K, Gonzalez, G, Allen, J, Walk, D, Hobson-Webb, L, and Gable, K

Abstract

In patients with chronic inflammatory demyelinating polyneuropathy (CIDP), intravenous immunoglobulin (IVIG) is recommended to be periodically reduced to assess the need for ongoing therapy. However, little is known about the effectiveness of restabilization with IVIG in patients who worsen after IVIG withdrawal. In the Polyneuropathy And Treatment with Hizentra (PATH) study, the pre-randomization period included sudden stopping of IVIG followed by 12 weeks of observation. Those deteriorating were then restabilized with IVIG. Of 245 subjects who stopped IVIG, 28 did not show signs of clinical deterioration within 12 weeks. Two hundred and seven received IVIG restabilization with an induction dose of 2 g/kg bodyweight (bw) IgPro10 (Privigen, CSL Behring, King of Prussia, Pennsylvania) and maintenance doses of 1 g/kg bw every 3 weeks for up to 13 weeks. Signs of clinical improvement were seen in almost all (n = 188; 91%) subjects. During IVIG restabilization, 35 subjects either did not show CIDP stability (n = 21, analyzed as n = 22 as an additional subject was randomized in error) or withdrew for other reasons (n = 14). Of the 22 subjects who did not achieve clinical stability, follow-up information in 16 subjects after an additional 4 weeks was obtained. Nine subjects were reported to have improved, leaving a maximum of 27 subjects (13%) who either showed no signs of clinical improvement during the restabilization phase and 4 weeks post-study or withdrew for other reasons. In conclusion, sudden IVIG withdrawal was effective in detecting ongoing immunoglobulin G dependency with a small risk for subjects not returning to their baseline 17 weeks after withdrawal.

Published
2019

9. Diagnosis and management of Guillain-Barre syndrome in ten steps

Author

Leonhard, Sonja, Mandarakas, Melissa, Gondim, FAA, Bateman, K, Ferreira, MLB, Cornblath, DR, van Doorn, Pieter, Dourado, ME, Hughes, RAC, Islam, Badrul, Kusunoki, S, Pardo, CA, Reisin, R, Sejvar, JJ, Shahrizaila, N, Soares, C, Umapathi, T, Wang, YZ, Yiu, EM, Willison, HJ, Jacobs, B.C., Leonhard, Sonja, Mandarakas, Melissa, Gondim, FAA, Bateman, K, Ferreira, MLB, Cornblath, DR, van Doorn, Pieter, Dourado, ME, Hughes, RAC, Islam, Badrul, Kusunoki, S, Pardo, CA, Reisin, R, Sejvar, JJ, Shahrizaila, N, Soares, C, Umapathi, T, Wang, YZ, Yiu, EM, Willison, HJ, and Jacobs, B.C.

Published
2019

10. Patients' and physicians' interpretation of chemotherapy-induced peripheral neurotoxicity

Author

Cavaletti, G, Cornblath, Dr, Merkies, Isj, Postma, Tj, Rossi, E, Alberti, P, Bruna, J, Argyriou, Aa, Briani, C, Velasco, R, Kalofonos, Hp, Psimaras, D, Ricard, D, Pace, A, Faber, Cg, Lalisang, Ri, Brandsma, D, Koeppen, S, Kerrigan, S, Schenone, A, Grisold, W, Mazzeo, A, Padua, Luca, Dorsey, Sg, Penas-Prado, M, Valsecchi, Mg, Padua L (ORCID:0000-0003-2570-9326), Cavaletti, G, Cornblath, Dr, Merkies, Isj, Postma, Tj, Rossi, E, Alberti, P, Bruna, J, Argyriou, Aa, Briani, C, Velasco, R, Kalofonos, Hp, Psimaras, D, Ricard, D, Pace, A, Faber, Cg, Lalisang, Ri, Brandsma, D, Koeppen, S, Kerrigan, S, Schenone, A, Grisold, W, Mazzeo, A, Padua, Luca, Dorsey, Sg, Penas-Prado, M, Valsecchi, Mg, and Padua L (ORCID:0000-0003-2570-9326)

Abstract

To test if and how chemotherapy-induced peripheral neurotoxicity (CIPN) is perceived differently by patients and physicians, making assessment and interpretation challenging. We performed a secondary analysis of the CI-PeriNomS study which included 281 patients with stable CIPN. We tested: (a) the association between patients' perception of activity limitation in performing eight common tasks and neurological impairment and (b) how the responses to questions related to these daily activities are interpreted by the treating oncologist. To achieve this, we compared patients' perception of their activity limitation with neurological assessment and the oncologists' blind interpretation. Distribution of the scores attributed by oncologists to each daily life maximum limitation ("impossible") generated three groups: Group 1 included limitations oncologists attributed mainly to motor impairment; Group 2 ones mainly attributed to sensory impairment and Group 3 ones with uncertain motor and sensory impairment. Only a subset of questions showed a significant trend between severity in subjective limitation, reported by patients, and neurological impairment. In Group 1, neurological examination confirmed motor impairment in only 51%-65% of patients; 76%-78% of them also had vibration perception impairment. In Group 2, sensory impairment ranged from 84% to 100%; some degree of motor impairment occurred in 43%-56% of them. In Group 3 strength reduction was observed in 49%-50% and sensory perception was altered in up to 82%. Interpretation provided by the panel of experienced oncologists was inconsistent with the neurological impairment. These observations highlight the need of a core set of outcome measures for future CIPN trials.

Published
2019

11. Regional variation of Guillain-Barré syndrome

Author

Doets, Alex Y., Verboon, Christine, Van Den Berg, Bianca, Harbo, Thomas, Cornblath, David R., Willison, Hugh J., Islam, Zhahirul, Attarian, Shahram, Barroso, Fabio A., Bateman, Kathleen, Benedetti, Luana, Van Den Bergh, Peter, Casasnovas, Carlos, Cavaletti, Guido, Chavada, Govindsinh, Claeys, Kristl G., Dardiotis, Efthimios, Davidson, Amy, Van Doorn, Pieter A., Feasby, Tom E., Galassi, Giuliana, Gorson, Kenneth C., Hartung, Hans-Peter, Hsieh, Sung-Tsang, Hughes, Richard A. C., Illa, Isabel, Islam, Badrul, Kusunoki, Susumu, Kuwabara, Satoshi, Lehmann, Helmar C., Miller, James A. L., Mohammad, Quazi Deen, Monges, Soledad, Nobile Orazio, Eduardo, Pardo, Julio, Pereon, Yann, Rinaldi, Simon, Querol, Luis, Reddel, Stephen W., Reisin, Ricardo C., Shahrizaila, Nortina, Sindrup, Soren H., Waqar, Waheed, Jacobs, Bart C., Jacobs, Bc, Hughes, Rac, Cornblath, Dr, Gorson, Kc, Hartung, Hp, Kusunoki, S, van Doorn PA, Willison, Hj, van Woerkom, M, van den Berg, B, Verboon, C, Doets, Ay, Roodbol, J, Reisin, Rc, Reddel, Sw, Islam, Z, Islam, B, Mohammad, Qd, van den Bergh, P, Feasby, Te, Harbo, T, Péréon, Y, Lehmann, Hc, Dardiotis, E, Nobile-Orazio, E, Shahrizaila, N, Bateman, K, Illa, I, Querol, L, Hsieh, St, Chavada, G, Davidson, A, Addington, Jm, Ajroud-Driss, S, Andersen, H, Antonini, G, Ariatti, A, Attarian, S, Badrising, Ua, Barroso, Fa, Benedetti, L, Beronio, A, Bianco, M, Binda, D, Briani, C, Bunschoten, C, Bürmann, J, Bella, Ir, Bertorini, Te, Bhavaraju-Sanka, R, Brannagan, Th, Busby, M, Butterworth, S, Casasnovas, C, Cavaletti, G, Chao, Cc, Chetty, S, Claeys, Kg, Conti, Me, Cosgrove, Js, Dalakas, Mc, Derejko, Ma, Dimachkie, Mm, Doppler, K, Dornonville de la Cour, C, Echaniz-Laguna, A, Eftimov, F, Faber, Cg, Fazio, R, Fujioka, T, Fulgenzi, Ea, Galassi, G, Garcia-Sobrino, T, Garnero, M, Garssen, Mpj, Gijsbers, Cj, Gilchrist, Jm, Goldstein, Jm, Granit, V, Grapperon, A, Gutiérrez, G, Hadden, Rdm, Holbech, Jv, Holt, Jkl, Homedes Pedret, C, Htut, M, Jericó Pascual, I, Kaida, K, Karafiath, S, Katzberg, Hd, Kiers, L, Kieseier, Bc, Kimpinski, K, Kleyweg, Rp, Kokubun, N, Kolb, Na, Kuitwaard, K, Kuwabara, S, Kwan, Jy, Ladha, Ss, Landschoff Lassen, L, Lawson, V, Ledingham, D, Léon Cejas, L, Lucy, St, Lunn, Mpt, Magot, A, Manji, H, Marchesoni, C, Marfia, Ga, Márquez Infante, C, Martinez Hernandez, E, Mataluni, G, Mcdermott, Cj, Meekins, Gd, Miller, Jal, Monges, Ms, Montero, Mcj, Morís de la Tassa, G, Mozzoni, J, Nascimbene, C, Nowak, Rj, Orizaloa Balaguer, P, Osei-Bonsu, M, Lee Pan EB, Pardo, J, Pasnoor, M, Rajabally, Ya, Rinaldi, S, Ritter, C, Roberts, Rc, Rojas-Marcos, I, Rudnicki, Sa, Ruiz, M, Sachs, Gm, Samijn, Jpa, Santoro, L, Schenone, A, Schwindling, L, Sedano Tous MJ, Sekiguchi, Y, Sheikh, Ka, Silvestri, Nj, Sindrup, Sh, Sommer, Cl, Stein, B, Stino, Am, Spyropoulos, A, Srinivasan, J, Suzuki, H, Tankisi, H, Tigner, D, Twydell, Pt, van Damme, P, van der Kooi AJ, van Dijk GW, van der Ree, T, van Koningsveld, R, Varrato, Jd, Vermeij, Fh, Visser, Lh, Vytopil, Mv, Waheed, W, Wilken, M, Wilkerson, C, Wirtz, Pw, Yamagishi, Y, Zhou, L, Zivkovic, S., Doets, A, Verboon, C, van den Berg, B, Harbo, T, Cornblath, D, Willison, H, Islam, Z, Attarian, S, Barroso, F, Bateman, K, Benedetti, L, van den Bergh, P, Casasnovas, C, Cavaletti, G, Chavada, G, Claeys, K, Dardiotis, E, Davidson, A, van Doorn, P, Feasby, T, Galassi, G, Gorson, K, Hartung, H, Hsieh, S, Hughes, R, Illa, I, Islam, B, Kusunoki, S, Kuwabara, S, Lehmann, H, Miller, J, Mohammad, Q, Monges, S, Nobile Orazio, E, Pardo, J, Pereon, Y, Rinaldi, S, Querol, L, Reddel, S, Reisin, R, Shahrizaila, N, Sindrup, S, Waqar, W, Jacobs, B, Neurology, AII - Infectious diseases, AII - Inflammatory diseases, ANS - Neuroinfection & -inflammation, Immunology, UCL - SSS/IONS/NEUR - Clinical Neuroscience, and UCL - (SLuc) Service de neurologie

Subjects
Adult, Male, medicine.medical_specialty, Adolescent, clinical course, Guillain-Barre Syndrome, 03 medical and health sciences, Young Adult, 0302 clinical medicine, axonal degeneration, demyelination, outcome, polyradiculoneuropathy, Internal medicine, Severity of illness, medicine, Humans, 030212 general & internal medicine, Young adult, Child, Geographic difference, Aged, Aged, 80 and over, Guillain-Barre syndrome, Polyradiculoneuropathy, Overlap syndrome, Middle Aged, medicine.disease, Regional variation, Child, Preschool, neurology, Settore MED/26 - Neurologia, Female, Neurology (clinical), 030217 neurology & neurosurgery, Cohort study, polyradiculoneuropathy, demyelination, axonal degeneration, clinical course, outcome
Abstract

Guillain-Barré syndrome is a heterogeneous disorder regarding the clinical presentation, electrophysiological subtype and outcome. Previous single country reports indicate that Guillain-Barré syndrome may differ among regions, but no systematic comparative studies have been conducted. Comparative studies are required to identify factors determining disease susceptibility, variation and prognosis, and to improve diagnostic criteria. The International Guillain-Barré Syndrome Outcome Study is a prospective, observational cohort study including all patients within the diagnostic spectrum, aiming to describe the heterogeneity of Guillain-Barré syndrome worldwide. The current study was based on the first 1000 inclusions with a follow-up of at least 1 year and confirmed the variation in clinical presentation, course and outcome between patients. The full clinical spectrum of Guillain-Barré syndrome was observed in patients from all countries participating in the International Guillain-Barré Syndrome Outcome Study, but the frequency of variants differed between regions. We compared three regions based on geography, income and previous reports of Guillain-Barré syndrome subtypes: 'Europe/Americas', 'Asia' (without Bangladesh), and 'Bangladesh'. We excluded 75 (8%) patients because of alternative diagnoses, protocol violations, or missing data. The predominant clinical variant was sensorimotor in Europe/Americas (n = 387/562, 69%) and Asia (n = 27/63, 43%), and pure motor in Bangladesh (n = 74/107, 69%). Miller Fisher syndrome and Miller Fisher-Guillain-Barré overlap syndrome were more common in Asia (n = 14/63, 22%) than in the other two regions (Europe/Americas: n = 64/562, 11%; Bangladesh: n = 1/107, 1%) (P < 0.001). The predominant electrophysiological subtype was demyelinating in all regions (Europe/Americas: n = 312/573, 55%; Asia: n = 29/65, 45%; Bangladesh: n = 38/94, 40%). The axonal subtype occurred more often in Bangladesh (n = 34/94, 36%) than in Europe/Americas (n = 33/573, 6%) and other Asian countries (n = 4/65, 6%) (P < 0.001). In all regions, patients with the axonal subtype were younger, had fewer sensory deficits, and showed a trend towards poorer recovery compared to patients with the demyelinating subtype. The proportion of patients able to walk unaided after 1 year varied between Asia (n = 31/34, 91%), Europe/Americas (n = 334/404, 83%) and Bangladesh (n = 67/97, 69%) (P = 0.003). A similar variation was seen for mortality, being higher in Bangladesh (n = 19/114, 17%) than in Europe/Americas (n = 23/486, 5%) and Asia (n = 1/45, 2%) (P < 0.001). This study showed that factors related to geography have a major influence on clinical phenotype, disease severity, electrophysiological subtype, and outcome of Guillain-Barré syndrome.

Published
2018

12. Regional variation of Guillain-Barré syndrome

Author

Doets, A, Verboon, C, van den Berg, B, Harbo, T, Cornblath, D, Willison, H, Islam, Z, Attarian, S, Barroso, F, Bateman, K, Benedetti, L, van den Bergh, P, Casasnovas, C, Cavaletti, G, Chavada, G, Claeys, K, Dardiotis, E, Davidson, A, van Doorn, P, Feasby, T, Galassi, G, Gorson, K, Hartung, H, Hsieh, S, Hughes, R, Illa, I, Islam, B, Kusunoki, S, Kuwabara, S, Lehmann, H, Miller, J, Mohammad, Q, Monges, S, Nobile Orazio, E, Pardo, J, Pereon, Y, Rinaldi, S, Querol, L, Reddel, S, Reisin, R, Shahrizaila, N, Sindrup, S, Waqar, W, Jacobs, B, Doets,AY, Cornblath, DR, Willison, HJ, Barroso, FA, Claeys, KG, van Doorn, PA, Feasby, TE, Gorson, KC, Hartung, HP, Hsieh, ST, Hughes, RAC, Lehmann, HC, Miller, JAL, Mohammad, QD, Nobile Orazio ,E, Reddel, SW, Reisin, RC, Sindrup, SH, Jacobs, BC, Doets, A, Verboon, C, van den Berg, B, Harbo, T, Cornblath, D, Willison, H, Islam, Z, Attarian, S, Barroso, F, Bateman, K, Benedetti, L, van den Bergh, P, Casasnovas, C, Cavaletti, G, Chavada, G, Claeys, K, Dardiotis, E, Davidson, A, van Doorn, P, Feasby, T, Galassi, G, Gorson, K, Hartung, H, Hsieh, S, Hughes, R, Illa, I, Islam, B, Kusunoki, S, Kuwabara, S, Lehmann, H, Miller, J, Mohammad, Q, Monges, S, Nobile Orazio, E, Pardo, J, Pereon, Y, Rinaldi, S, Querol, L, Reddel, S, Reisin, R, Shahrizaila, N, Sindrup, S, Waqar, W, Jacobs, B, Doets,AY, Cornblath, DR, Willison, HJ, Barroso, FA, Claeys, KG, van Doorn, PA, Feasby, TE, Gorson, KC, Hartung, HP, Hsieh, ST, Hughes, RAC, Lehmann, HC, Miller, JAL, Mohammad, QD, Nobile Orazio ,E, Reddel, SW, Reisin, RC, Sindrup, SH, and Jacobs, BC

Abstract

Guillain-Barré syndrome is a heterogeneous disorder regarding the clinical presentation, electrophysiological subtype and outcome. Previous single country reports indicate that Guillain-Barré syndrome may differ among regions, but no systematic comparative studies have been conducted. Comparative studies are required to identify factors determining disease susceptibility, variation and prognosis, and to improve diagnostic criteria. The International Guillain-Barré Syndrome Outcome Study is a prospective, observational cohort study including all patients within the diagnostic spectrum, aiming to describe the heterogeneity of Guillain-Barré syndrome worldwide. The current study was based on the first 1000 inclusions with a follow-up of at least 1 year and confirmed the variation in clinical presentation, course and outcome between patients. The full clinical spectrum of Guillain-Barré syndrome was observed in patients from all countries participating in the International Guillain-Barré Syndrome Outcome Study, but the frequency of variants differed between regions. We compared three regions based on geography, income and previous reports of Guillain-Barré syndrome subtypes: 'Europe/Americas', 'Asia' (without Bangladesh), and 'Bangladesh'. We excluded 75 (8%) patients because of alternative diagnoses, protocol violations, or missing data. The predominant clinical variant was sensorimotor in Europe/Americas (n = 387/562, 69%) and Asia (n = 27/63, 43%), and pure motor in Bangladesh (n = 74/107, 69%). Miller Fisher syndrome and Miller Fisher-Guillain-Barré overlap syndrome were more common in Asia (n = 14/63, 22%) than in the other two regions (Europe/Americas: n = 64/562, 11%; Bangladesh: n = 1/107, 1%) (P < 0.001). The predominant electrophysiological subtype was demyelinating in all regions (Europe/Americas: n = 312/573, 55%; Asia: n = 29/65, 45%; Bangladesh: n = 38/94, 40%). The axonal subtype occurred more often in Bangladesh (n = 34/94, 36%) than in Europe/America

Published
2018

14. Protocol of a prospective observational cohort study on clinical and biological predictors of disease course and outcome in Guillain-Barré syndrome

Author

Jacobs, Bc, van den Berg, B, Verboon, C, Chavada, G, Cornblath, Dr, Gorson, Kc, Harbo, T, Hartung, Hp, Hughes, Rac, Kusunok, S, van Doorn PA, Willison, Hj, Briani, Chiara, the IGOS Consortium International Guillain Barré Syndrome Outcome Study, and Campagnolo, Marta

Subjects
treatment, diagnosis, Guillain-Barré syndrome, biomarkers, outcome, prognosis
Published
2017

15. International Guillain-Barre Syndrome Outcome Study: protocol of a prospective observational cohort study on clinical and biological predictors of disease course and outcome in Guillain-Barre syndrome

Author

Jacobs, BC, van den Berg, B, Verboon, C, Chavada, G, Cornblath, DR, Gorson, KC, Harbo, T, Hartung, HP, Hughes, RAC, Kusunoki, S, van Doorn, PA, Willison, HJ, Illa I., Querol L.A., and Zivkovic, Sasa A.

Subjects
treatment, diagnosis, outcome, biomarkers, Guillain-Barre syndrome, prognosis
Abstract

Guillain-Barre syndrome (GBS) is an acute polyradiculoneuropathy with a highly variable clinical presentation, course, and outcome. The factors that determine the clinical variation of GBS are poorly understood which complicates the care and treatment of individual patients. The protocol of the ongoing International GBS Outcome Study (IGOS), a prospective, observational, multicenter cohort study that aims to identify the clinical and biological determinants and predictors of disease onset, subtype, course and outcome of GBS is presented here. Patients fulfilling the diagnostic criteria for GBS, regardless of age, disease severity, variant forms, or treatment, can participate if included within 2 weeks after onset of weakness. Information about demography, preceding infections, clinical features, diagnostic findings, treatment, course, and outcome is collected. In addition, cerebrospinal fluid and serial blood samples for serum and DNA is collected at standard time points. The original aim was to include at least 1,000 patients with a follow-up of 1-3 years. Data are collected via a web-based data entry system and stored anonymously. IGOS started in May 2012 and by January 2017 included more than 1,400 participants from 143 active centers in 19 countries across 5 continents. The IGOS data/biobank is available for research projects conducted by expertise groups focusing on specific topics including epidemiology, diagnostic criteria, clinimetrics, electrophysiology, antecedent events, antibodies, genetics, prognostic modeling, treatment effects, and long-term outcome of GBS. The IGOS will help to standardize the international collection of data and biosamples for future research of GBS.

Published
2017

16. Correlation of the patient's reported outcome Inflammatory-RODS with an objective metric in immune-mediated neuropathies

Author

Draak, THP, Gorson, KC, Vanhoutte, EK, van Nes, SI, van Doorn, PA, Cornblath, DR, van den Berg, LH, Faber, CG, Merkies, ISJ, Querol, L., and de Visser, Marianne

Subjects
chronic inflammatory demyelinating polyneuropathy, peripheral neuropathy, patient reported outcome measure, IgM-monoclonal gammopathy of undetermined significance related polyneuropathy, Guillain-Barre syndrome
Abstract

Background and purposeThere is increasing interest in using patient-reported outcome measures (PROMs) in clinical studies to capture individual changes over time. However, PROMs have also been criticized because they are entirely subjective. Our objective was to examine the relationship between a subjective PROM and an objective outcome tool in patients with Guillain-Barre syndrome (GBS), chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) and gammopathy-related polyneuropathy (MGUSP). MethodsThe Inflammatory Rasch-built Overall Disability Scale (I-RODS (c), a multi-item scale that examines functionality) was completed by 137 patients with newly diagnosed (or relapsing) GBS (55), CIDP (59) and MGUSP (23) who were serially examined (GBS/CIDP, T0/T1/T3/T6/T12 months; MGUSP, T0/T3/T12). Possible association between the I-RODS findings and the vigorimeter scores, an objective linear instrument to assess grip strength, was examined. ResultsA significant correlating trend was found between the I-RODS and grip strength scores for the overall group and in each illness, independently. ConclusionThe objectivity of patients' subjective report on their functional state based on a strong correlation between the I-RODS and grip strength in patients with GBS, CIDP and MGUSP has been demonstrated. These findings provide further support to use the I-RODS and grip strength in future clinical studies in these conditions.

Published
2016

17. Does ability to walk reflect general functionality in inflammatory neuropathies?

Author

Draak, THP, Gorson, KC, Vanhoutte, EK, van Nes, SI, van Doorn, PA, Cornblath, DR, van den Berg, LH, Faber, CG, Merkies, ISJ, Querol, L., and van Schaik, Ivo N.

Subjects
outcome research, inflammatory neuropathies
Abstract

The ability to walk is considered a benchmark for good clinical recovery and prognosis, particularly in patients with Guillain-Barre syndrome (GBS) and chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). However, it has never been determined whether being able to walk represents general functionality. The purpose of this study was to examine whether the ability to walk outside independently reflects general functional improvement in patients with GBS, CIDP, and gammopathy-related neuropathy (MGUSP). A total of 137 patients with newly diagnosed (or relapsing) GBS (55), CIDP (59), and MGUSP (23) were serially examined (1-year). Predefined arbitrary cut-offs (so-called patients' Functional-Acceptable-Clinical-Thresholds [FACTs]) were taken at the 50th, 75th, and 90th percentile of the Inflammatory-Rasch-built-Overall-Disability-Scale (I-RODS (c)). We determined the proportion of patients able to walk outside independently that reached the postulated cut-offs. A mean total of 85%, 39%, and 12% of all patients able to walk reached 50th, 75th, and 90th percentile thresholds, respectively. These findings were not neuropathy type related. Our findings show that assessing only one construct of functionality (e.g., walking ability) does not reflect the full scope of daily/social functional deficits perceived by patients. The ability to walk shows a patient is doing better, but not necessarily doing well. The I-RODS (c) bypasses these limitations.

Published
2016

18. Rasch-Transformed Total Neuropathy Score clinical version (RT-TNSc©) in patients with chemotherapy-induced peripheral neuropathy

Author

Binda, D, Cavaletti, G, Cornblath, Dr, Merkies, Is, CI PeriNomS, Sg, Postma, Tj, Valsecchi, Mg, Galimberti, S, Rossi, E, Frigeni, B, Lanzani, F, Mattavelli, L, Piatti, Ml, Alberti, P, Bidoli, P, Cazzaniga, M, Cortinovis, D, Bruna, J, Velasco, R, Argyriou, Aa, Kalofonos, Hp, Psimaras, D, Ricard, D, Pace, A, Galiè, E, Briani, C, Lucchetta, M, Campagnolo, M, Dalla Torre, C, Faber, Cg, Vanhoutte, Ek, Bakkers, M, Brouwer, B, Boogerd, M, Lalisang, Ri, Boogerd, W, Brandsma, D, Koeppen, S, Grant, R, Storey, D, Kerrigan, S, Schenone, Angelo, Reni, L, Piras, B, Fabbri, S, Pessino, A, Padua, L, Granata, G, Leandri, M, Ghignotti, I, Plasmati, R, Pastorelli, F, Heimans, Jj, Eurelings, M, Meijer, Rj, Josef, Kf, Grisold, W, Lindeck Pozza, E, Mazzeo, A, Toscano, A, Russo, M, Tomasello, C, Altavilla, G, Penas Prado, M, Dominguez Gonzalez, C, Dorsey, S. g., RS: GROW - Oncology, RS: GROW - R3 - Innovative Cancer Diagnostics & Therapy, Binda, D, Cavaletti, G, Cornblath, D, Merkies, I, Valsecchi, M, and Bidoli, P

Subjects
Rasch analysi, Male, psychometrics, medicine.medical_specialty, peripheral neuropathy, Drug-Related Side Effects and Adverse Reactions, Psychometrics, psychometric, chemotherapy, Rasch analysis, total neuropathy score, Neurology (clinical), Neuroscience (all), Severity of Illness Index, Age Factors, Female, Humans, Peripheral Nervous System Diseases, Quality of Life, Retrospective Studies, Disability Evaluation, Medicine (all), Physical medicine and rehabilitation, Quality of life, Severity of illness, Medicine, Balance (ability), Rasch model, business.industry, General Neuroscience, Retrospective cohort study, medicine.disease, Peripheral neuropathy, Chemotherapy-induced peripheral neuropathy, Physical therapy, business
Abstract

Composite scales such as the Total Neuropathy Score clinical version (TNSc(©) ) have been widely used to measure neurological impairment in a standardized manner but they have been criticized due to their ordinal setting having no fixed unit. This study aims to improve impairment assessment in patients with chemotherapy-induced peripheral neuropathy (CIPN) by subjecting TNSc(©) records to Rasch analyses. In particular, we wanted to investigate the influence of factors affecting the use of the TNSc(©) in clinical practice. TNSc(©) has 7 domains (sensory, motor, autonomic, pin-prick, vibration, strength, and deep tendon reflexes [DTR]) each being scored 0-4. Data obtained in 281 patients with stable CIPN were subjected to Rasch analyses to determine the fit to the model. The TNSc(©) did not meet Rasch model's expectations primarily because of misfit statistics in autonomic and DTR domains. Removing these two, acceptable model fit and uni-dimensionality were obtained. However, disordered thresholds (vibration and strength) and item bias (mainly cultural) were still seen, but these findings were kept to balance the assessment range of the Rasch-Transformed TNSc(©) (RT-TNSc(©) ). Acceptable reliability findings were also obtained. A 5-domains RT-TNSc(©) may be a more proper assessment tool in patients with CIPN. Future studies are needed to examine its responsive properties.

Published
2015

19. Grip strength comparison in immune-mediated neuropathies: Vigorimeter vs. Jamar

Author

Draak, THP, Pruppers, MHJ, Nes, Sonja, Vanhoutte, EK, Bakkers, M (Mayienne), Gorson, KC, van der Pol, WL, Lewis, RA, Notermans, NC, Nobile-Orazio, E, Leger, JM, Van den Bergh, PYK, Lauria, G, Bril, V, Katzberg, H, Lunn, MPT, Pouget, J, van der Kooi, AJ, van den Berg, LH, van Doorn, Pieter, Cornblath, DR, Hahn, AF, Faber, CG, Merkies, ISJ (Ingemar), Amsterdam Neuroscience, Neurology, Amsterdam institute for Infection and Immunity, 05 Neurology and psychiatry, MUMC+: DA KG Polikliniek (9), RS: MHeNs - R1 - Cognitive Neuropsychiatry and Clinical Neuroscience, and Klinische Neurowetenschappen

Subjects
grip strength, Jamar dynamometer, responsiveness comparison, Vigorimeter
Abstract

The Jamar dynamometer and Vigorimeter have been used to assess grip strength in immune-mediated neuropathies, but have never been compared to each other. Therefore, we performed a comparison study between these two devices in patients with immune-mediated neuropathies. Grip strength data were collected in 102 cross-sectional stable and 163 longitudinal (new diagnoses or changing condition) patients with Guillain-Barré syndrome (GBS), chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), gammopathy-related polyneuropathy (MGUSP), and multifocal motor neuropathy (MMN). Stable patients were assessed twice (validity/reliability studies). Longitudinal patients were assessed 3-5 times during 1 year. Responsiveness comparison between the two tools was examined using combined anchor-/distribution-based minimum clinically important difference (MCID) techniques. Patients were asked to indicate their preference for the Jamar or Vigorimeter. Both tools correlated highly with each other (ρ = 0.86, p

Published
2015

20. Impairment measures versus inflammatory RODS in GBS and CIDP: a responsiveness comparison

Author

Vanhoutte, EK, Draak, THP, Gorson, KC, van Nes, SI, Hoeijmakers, JGJ, Van der Pol, WL, Notermans, NC, Lewis, RA, Nobile-Orazio, E, Leger, JM, Van den Bergh, PYK, Lauria, G, Bril, V, Katzberg, H, Lunn, MPT, Pouget, J, van der Kooi, AJ, Hahn, AF, van Doorn, PA, Cornblath, DR, van den Berg, LH, Faber, CG, Merkies, ISJ, Illa I., Querol, L., and PeriNomS Study Grp

Subjects
outcome measure, immune-mediated neuropathies, minimum clinically important difference, Rasch
Abstract

This study aimed to define responder' through the concept of minimum clinically important differences using the individually obtained standard errors (MCID-SE) and a heuristic external criterion' responsiveness method in patients with Guillain-Barre syndrome (GBS) and chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). One hundred and fourteen newly diagnosed or relapsing patients (GBS: 55, CIDP: 59) were serially examined (1-year follow-up). The inflammatory Rasch-built overall disability scale (I-RODS), Rasch-transformed MRC sum score (RT-MRC), and Rasch-transformed modified-INCAT-sensory scale (RT-mISS) were assessed. Being-a-responder was defined as having a MCID-SE cut-off 1.96. Also, the correlations between patients' scores on each scale and the EuroQoL health-status thermometer' (external criterion) were determined (higher correlation indicated better responsiveness). In both diseases, the SEs showed a characteristic U'-shaped dynamic pattern across each scales' continuum. The number of patients showing a meaningful change were higher for the I-RODS>RT-MRC>RT-mISS and were in GBS higher than CIDP patients. The MCID-SE concept using Rasch-transformed data demonstrated an individual pattern of being-a-responder' in patients with immune-mediated neuropathies, and the findings were validated by the external criterion responsiveness method. The I-RODS showed greater responsiveness compared with the MRC and INCAT-sensory scales, and its use is therefore recommended in future trials in GBS and CIDP.

Published
2015

21. Rasch-built Overall Disability Scale for Multifocal motor neuropathy (MMN-RODS(C))

Author

Vanhoutte, EK, Faber, CG, van Nes, SI, Cats, EA, Van der Pol, WL, Gorson, KC, van Doorn, PA, Cornblath, DR, van den Berg, LH, Merkies, ISJ, Illa I., Querol, L., and PeriNomS Study Grp

Subjects
MMN, outcome measure, multifocal motor neuropathy, Rasch-built disability scale
Abstract

Clinical trials in multifocal motor neuropathy (MMN) have often used ordinal-based measures that may not accurately capture changes. We aimed to construct a disability interval outcome measure specifically for MMN using the Rasch model and to examine its clinimetric properties. A total of 146 preliminary activity and participation items were assessed twice (reliability studies) in 96 clinically stable MMN patients. These patients also assessed the ordinal-based overall disability sum score (construct, sample-dependent validity). The final Rasch-built overall disability scale for MMN (MMN-RODS(C)) was serially applied in 26 patients with newly diagnosed or relapsing MMN, treated with intravenous immunoglobulin (IVIg) (1-year follow-up; responsiveness study). The magnitude of change for each patient was calculated using the minimum clinically important difference technique related to the individually obtained standard errors. A total of 121 items not fulfilling Rasch requirements were removed. The final 25-item MMN-RODS(C) fulfilled all Rasch model's expectations and showed acceptable reliability and validity including good discriminatory capacity. Most serially examined patients improved, but its magnitude was low, reflecting poor responsiveness. The constructed MMN-RODS(C) is a disease-specific, interval measure to detect activity limitations in patients with MMN and overcomes the shortcomings of ordinal scales. However, future clinimetric studies are needed to improve the MMN-RODS(C)'s responsiveness by longer observations and/or more rigorous treatment regimens.

Published
2015

22. Comparing the NIS vs. MRC and INCAT sensory scale through Rasch analyses

Author

Draak, THP, Vanhoutte, EK, van Nes, SI, Gorson, KC, Van der Pol, WL, Notermans, NC, Nobile-Orazio, E, Lewis, RA, Leger, JM, Van den Bergh, PYK, Lauria, G, Bril, V, Katzberg, H, Lunn, MPT, Pouget, J, van der Kooi, AJ, Hahn, AF, van den Berg, LH, van Doorn, PA, Cornblath, DR, Faber, CG, Merkies, ISJ, Illa I., Querol, L., and PeriNomS Study Grp

Subjects
mISS, MRC, responsiveness, NIS, Rasch
Abstract

We performed a comparison between Neuropathy Impairment Scale-sensory (NISs) vs. the modified Inflammatory Neuropathy Cause and Treatment sensory scale (mISS), and NIS-motor vs. the Medical Research Council sum score in patients with Guillain-Barre syndrome (GBS), chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), and IgM monoclonal gammopathy of undetermined significance-related polyneuropathy (MGUSP). The ordinal data were subjected to Rasch analyses, creating Rasch-transformed (RT)-intervals for all measures. Comparison between measures was based on validity/reliability with an emphasis on responsiveness (using the patient's level of change related to the individually obtained varying SE for minimum clinically important difference). Eighty stable patients (GBS: 30, CIDP: 30, and MGUSP: 20) were assessed twice (entry: two observers; 2-4weeks later: one observer), and 137 newly diagnosed or relapsing patients (GBS: 55, CIDP: 59, and IgM-MGUSP: 23) were serially examined with 12months follow-up. Data modifications were needed to improve model fit for all measures. The sensory and motor scales demonstrated approximately equal and acceptable validity and reliability scores. Responsiveness scores were poor but slightly higher in RT-mISS compared to RT-NISs. Responsiveness was equal for the RT-motor scales, but higher in GBS compared to CIDP; responsiveness was poor in patients with MGUSP, suggesting a longer duration of follow-up in the latter group of patients.

Published
2015

23. Modifying the Medical Research Council grading system through Rasch analyses

Author

Vanhoutte, Ek, Faber, Cg, Van Nes, Si, Jacobs, Bc, Van Doorn, Pa, Van Koningsveld, R, Cornblath, Dr, Van Der Kooi, Aj, Cats, Ea, Van Den Berg, Lh, Notermans, Nc, Van Der Pol, Wl, Hermans, Mc, Van Der Beek, Na, Gorson, Kc, Eurelings, M, Engelsman, J, Boot, H, Meijer, Rj, Lauria, G, Tennant, A, Merkies, Is, Barreira, Aa, Bennett, D, Van Den Bergh, Py, Bril, V, Devigili, G, Hadden, Rd, Hahn, Af, Hartung, Hp, Hughes, Ra, Illa, I, Katzberg, H, Léger, Jm, Lewis, Ra, Lunn, Mp, Nascimento, Oj, Nobile Orazio, E, Padua, Luca, Pouget, J, Reilly, Mm, Van Schaik, I, Smith, B, De Visser, M, Walk, D., Neurology, Immunology, MUMC+: DA KG Polikliniek (9), Klinische Neurowetenschappen, RS: MHeNs School for Mental Health and Neuroscience, ANS - Amsterdam Neuroscience, AII - Amsterdam institute for Infection and Immunity, and Faculteit der Geneeskunde

Subjects
Adult, Male, medicine.medical_specialty, Weakness, Biomedical Research, Adolescent, medicine.medical_treatment, Disease, neuromuscular disorders, Young Adult, Bias, Muscular Diseases, medicine, Humans, Muscle Strength, Occasional Paper, Child, Rasch, Rasch model, Rehabilitation, business.industry, manual muscle testing, Infant, Newborn, Infant, Polyradiculoneuropathy, Middle Aged, medicine.disease, Medical research, Settore MED/26 - NEUROLOGIA, MRC, PESQUISA (MÉTODOS), Child, Preschool, Cohort, Physical therapy, Female, Health Planning Councils, Neurology (clinical), medicine.symptom, business, Multifocal motor neuropathy
Abstract

The Medical Research Council grading system has served through decades for the evaluation of muscle strength and has been recognized as a cardinal feature of daily neurological, rehabilitation and general medicine examination of patients, despite being respectfully criticized due to the unequal width of its response options. No study has systematically examined, through modern psychometric approach, whether physicians are able to properly use the Medical Research Council grades. The objectives of this study were: (i) to investigate physicians' ability to discriminate among the Medical Research Council categories in patients with different neuromuscular disorders and with various degrees of weakness through thresholds examination using Rasch analysis as a modern psychometric method; (ii) to examine possible factors influencing physicians' ability to apply the Medical Research Council categories through differential item function analyses; and (iii) to examine whether the widely used Medical Research Council 12 muscles sum score in patients with Guillain-Barre syndrome and chronic inflammatory demyelinating polyradiculoneuropathy would meet Rasch model's expectations. A total of 1065 patients were included from nine cohorts with the following diseases: Guillain-Barre syndrome (n = 480); myotonic dystrophy type-1 (n = 169); chronic inflammatory demyelinating polyradiculoneuropathy (n = 139); limb-girdle muscular dystrophy (n = 105); multifocal motor neuropathy (n = 102); Pompe's disease (n = 62) and monoclonal gammopathy of undetermined related polyneuropathy (n = 8). Medical Research Council data of 72 muscles were collected. Rasch analyses were performed on Medical Research Council data for each cohort separately and after pooling data at the muscle level to increase category frequencies, and on the Medical Research Council sum score in patients with Guillain-Barre syndrome and chronic inflammatory demyelinating polyradiculoneuropathy. Disordered thresholds were demonstrated in 74-79% of the muscles examined, indicating physicians' inability to discriminate between most Medical Research Council categories. Factors such as physicians' experience or illness type did not influence these findings. Thresholds were restored after rescoring the Medical Research Council grades from six to four options (0, paralysis; 1, severe weakness; 2, slight weakness; 3, normal strength). The Medical Research Council sum score acceptably fulfilled Rasch model expectations after rescoring the response options and creating subsets to resolve local dependency and item bias on diagnosis. In conclusion, a modified, Rasch-built four response category Medical Research Council grading system is proposed, resolving clinicians' inability to differentiate among its original response categories and improving clinical applicability. A modified Medical Research Council sum score at the interval level is presented and is recommended for future studies in Guillain-Barre syndrome and chronic inflammatory demyelinating polyradiculoneuropathy.

Published
2012

24. Randomised controlled trial of methotrexate for chronic inflammatory demyelinating polyradiculoneuropathy (RMC trial): a pilot, multicentre study

Author

Mahdi Rogers, M, Rutterford, C, Hughes, Ra, Léger, Jm, Nobile Orazio, E, Van den Bergh, P, van Doorn, P, van Schaik IN, Hadden, Rd, Choy, E, Reilly, M, Winer, J, Evers, E, van Doorn PA, Créange, A, Gueguen, A, Uzenot, D, Behin, A, Nicolas, G, Pautot, V, Uncini, A, Manzoli, C, Lauria, G, Pareyson, D, Casellato, C, Sabatelli, M, Conte, A, Luigetti, M, Briani, Chiara, Lucchetta, Marta, Schenone, A, Benedetti, L, Fiorina, E, Brusse, E, van der Kooi AJ, Guiloff, Rj, Rakowicz, Wp, Lecky, Br, Dougan, Cf, Marshall, D, Davies, N, Busby, M, Lansbury, A, Overell, J, Willison, Hj, Rajabally, Ya, Kendall, B, Gow, D, Nixon, J, Kulkarni, O, Katifi, H, Hammans, S, Gibson, A, Mcdermott, C, Cornblath, Dr, Chaudry, V., Amsterdam institute for Infection and Immunity, Amsterdam Neuroscience, and Neurology

Subjects
Male, medicine.medical_specialty, Anti-Inflammatory Agents, Pilot Projects, Placebo, Placebo group, law.invention, Disability Evaluation, Folic Acid, Randomized controlled trial, Double-Blind Method, law, Adrenal Cortex Hormones, Internal medicine, Medicine, Humans, Adverse effect, Aged, Intention-to-treat analysis, business.industry, Immunoglobulins, Intravenous, Polyradiculoneuropathy, Odds ratio, Vitamins, Middle Aged, medicine.disease, Surgery, Methotrexate, Treatment Outcome, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating, Female, Neurology (clinical), business, Immunosuppressive Agents, medicine.drug
Abstract

BACKGROUND: Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) responds to treatment with corticosteroids, intravenous immunoglobulin, and plasma exchange. We aimed to test whether the standard immunosuppressive drug methotrexate was of use in treatment of CIDP. METHODS: In a pilot, multicentre, randomised, double-blind, controlled trial we compared oral methotrexate 7.5 mg weekly for 4 weeks, then 10 mg weekly for 4 weeks, and finally 15 mg weekly for 32 weeks (40 weeks' total treatment) with placebo in patients with CIDP requiring intravenous immunoglobulin or corticosteroids. After about 16 weeks, the dose of corticosteroids or intravenous immunoglobulin was decreased by 20% every 4 weeks if participants did not deteriorate. Primary outcome was a greater than 20% reduction in mean weekly dose in the last 4 weeks of the trial compared with the first 4 weeks. Secondary outcomes analysed separately at the mid-trial and final visits measured activity limitations and strength. Analyses were done by intention to treat. This study is registered as an International Standard Randomised Controlled Trial, number ISRCTN73774524. FINDINGS: 59 of the 60 enrolled participants completed the trial. 14 (52%) of 27 taking methotrexate and 14 (44%) of 32 taking placebo had a greater than 20% reduction in mean weekly dose of corticosteroids or intravenous immunoglobulin (adjusted odds ratio 1.21, 95% CI 0.40-3.70). There were no clinically and statistically significant differences in secondary outcomes. The one serious adverse event in the placebo group and the three in the methotrexate group were not thought to be related to treatment. INTERPRETATION: Oral methotrexate 15 mg weekly showed no significant benefit, but limitations in the trial design and the high rate of response in the placebo group meant that a treatment effect could not be excluded. This study can inform design of future trials in CIDP. FUNDING: The GBS/CIDP Foundation International

Published
2009

25. Improving fatigue assessment in immune-mediated neuropathies: the modified Rasch-built fatigue severity scale

Author

Van Nes, Si, Vanhoutte, Ek, Faber, Cg, Garssen, M, Van Doorn, Pa, Merkies, Is, Bennett, D, Van Den Berg, Lh, Van Den Bergh, Py, Cornblath, Dr, Dalakas, M, Devigili, G, Gorson, Kc, Hadden, Rd, Hahn, Af, Hartung, Hp, Hughes, Ra, Lauria, Giuseppe, Léger, Jm, Lewis, Ra, Lunn, Mp, Nobile Orazio, E, Notermans, Nc, Padua, Luca, Reilly, Mm, and Smith, B.

Subjects
Settore MED/26 - NEUROLOGIA, fatigue, Rash analysis, immune-mediated neuropathy, fatigue severity scale
Published
2009

26. Revising two-point discrimination assessment in normal aging and in patients with polyneuropathies

Author

Van Nes, Si, Faber, Cg, Hamers, Rm, Harschnitz, O, Bakkers, M, Hermans, Mc, Meijer, Rj, Van Doorn, Pa, Merkies, Is, Bennett, D, Van Den Berg, Lh, Van Den Bergh, Py, Cornblath, Dr, Dalakas, M, Gorson, Kc, Hadden, Rd, Hahn, Af, Hughes, Ra, Lauria, G, Léger, Jm, Lewis, Ra, Lunn, Pt, Nobile Orazio, E, Notermans, Nc, Padua, Luca, and Reilly, Mm

Subjects
Settore MED/26 - NEUROLOGIA, polyneuropathies
Published
2008

27. Physician-assessed and patient-reported outcome measures in chemotherapy-induced sensory peripheral neurotoxicity: two sides of the same coin

Author

Alberti, P, Rossi, E, Cornblath, D, Ingemar, S, Postma, T, Frigeni, B, Bruna, J, Velasco, R, Andreas, A, Kalofonos, H, Psimaras, D, Ricard, D, Pace, A, Galiè, E, Briani, C, Dalla Torre, C, Faber, C, Lalisang, R, Boogerd, W, Brandsma, D, Koeppen, S, Hense, J, Storey, D, Kerrigan, S, Schenone, A, Fabbri, S, Valsecchi, M, Cavaletti, G, Cortinovis, D, Cornblath, DR, Ingemar, SJM, Postma, TJ, Andreas, AA, Kalofonos, HP, Briani C, Faber, CG, Lalisang, RI, Valsecchi, MG, Cortinovis, DL, Alberti, P, Rossi, E, Cornblath, D, Ingemar, S, Postma, T, Frigeni, B, Bruna, J, Velasco, R, Andreas, A, Kalofonos, H, Psimaras, D, Ricard, D, Pace, A, Galiè, E, Briani, C, Dalla Torre, C, Faber, C, Lalisang, R, Boogerd, W, Brandsma, D, Koeppen, S, Hense, J, Storey, D, Kerrigan, S, Schenone, A, Fabbri, S, Valsecchi, M, Cavaletti, G, Cortinovis, D, Cornblath, DR, Ingemar, SJM, Postma, TJ, Andreas, AA, Kalofonos, HP, Briani C, Faber, CG, Lalisang, RI, Valsecchi, MG, and Cortinovis, DL

Abstract

Background: The different perception and assessment of chemotherapy-induced peripheral neurotoxicity (CIPN) between healthcare providers and patients has not yet been fully addressed, although these two approaches might eventually lead to inconsistent, possibly conflicting interpretation, especially regarding sensory impairment. Patients and methods: A cohort of 281 subjects with stable CIPN was evaluated with the National Cancer Institute- Common Toxicity Criteria (NCI-CTC v. 2.0) sensory scale, the clinical Total Neuropathy Score (TNSc©), the modified Inflammatory Neuropathy Cause and Treatment (INCAT) sensory sumscore (mISS) and the European Organization for Research and Treatment of Cancer CIPN specific self-report questionnaire (EORTC QOL-CIPN20). Results: Patients' probability estimates showed that the EORTC QLQ-CIPN20 sensory score was overall more highly related to the NCI-CTC sensory score. However, the vibration perception item of the TNSc had a higher probability to be scored 0 for EORTC QLQ-CIPN20 scores lower than 35, as vibration score 2 for EORTC QLQ-CIPN20 scores between 35 and 50 and as grade 3 or 4 for EORTC QLQ-CIPN20 scores higher than 50. The linear models showed a significant trend between each mISS item and increasing EORTC QLQ-CIPN20 sensory scores.

Published
2013

28. Rasch-built Overall Disability Scale for patients with chemotherapy-induced peripheral neuropathy (CIPN-R-ODS)

Author

Binda, D, Vanhoutte, E, Cavaletti, G, Cornblath, D, Postma, T, Frigeni, B, Alberti, P, Bruna, J, Velasco, R, Argyriou, A, Kalofonos, H, Psimaras, D, Ricard, D, Pace, A, Galiè, E, Briani, C, Dalla Torre, C, Lalisang, R, Boogerd, W, Brandsma, D, Koeppen, S, Hense, J, Storey, D, Kerrigan, S, Schenone, A, Fabbri, S, Rossi, E, Valsecchi, M, Faber, C, Merkies, I, Bidoli, P, Cortinovis, D, Vanhoutte, EK, Cornblath, DR, Postma, TJ, Argyriou, AA, Kalofonos, HP, Lalisang, RI, Valsecchi, MG, Faber, CG, Merkies, ISJ, Binda, D, Vanhoutte, E, Cavaletti, G, Cornblath, D, Postma, T, Frigeni, B, Alberti, P, Bruna, J, Velasco, R, Argyriou, A, Kalofonos, H, Psimaras, D, Ricard, D, Pace, A, Galiè, E, Briani, C, Dalla Torre, C, Lalisang, R, Boogerd, W, Brandsma, D, Koeppen, S, Hense, J, Storey, D, Kerrigan, S, Schenone, A, Fabbri, S, Rossi, E, Valsecchi, M, Faber, C, Merkies, I, Bidoli, P, Cortinovis, D, Vanhoutte, EK, Cornblath, DR, Postma, TJ, Argyriou, AA, Kalofonos, HP, Lalisang, RI, Valsecchi, MG, Faber, CG, and Merkies, ISJ

Abstract

Chemotherapy-induced peripheral neuropathy (CIPN) is a common neurological side-effect of cancer treatment and may lead to declines in patients' daily functioning and quality of life. To date, there are no modern clinimetrically well-evaluated outcome measures available to assess disability in CIPN patients. The objective of the study was to develop an interval-weighted scale to capture activity limitations and participation restrictions in CIPN patients using the Rasch methodology and to determine its validity and reliability properties. A preliminary Rasch-built Overall Disability Scale (pre-R-ODS) comprising 146 items was assessed twice (interval: 2-3weeks; test-retest reliability) in 281 CIPN patients with a stable clinical condition. The obtained data were subjected to Rasch analyses to determine whether model expectations would be met, and if necessarily, adaptations were made to obtain proper model fit (internal validity). External validity was obtained by correlating the CIPN-R-ODS with the National Cancer Institute-Common Toxicity Criteria (NCI-CTC) neuropathy scales and the Pain-Intensity Numeric-Rating-Scale (PI-NRS). The preliminary R-ODS did not meet Rasch model's expectations. Items displaying misfit statistics, disordered thresholds, item bias or local dependency were systematically removed. The final CIPN-R-ODS consisting of 28 items fulfilled all the model's expectations with proper validity and reliability, and was unidimensional. The final CIPN-R-ODS is a Rasch-built disease-specific, interval measure suitable to detect disability in CIPN patients and bypasses the shortcomings of classical test theory ordinal-based measures. Its use is recommended in future clinical trials in CIPN

Published
2013

29. CI-PERINOMS: chemotherapy-induced peripheral neuropathy outcome measures study

Author

Cavaletti, G, Cornblath, Dr, Postma, Tj, Merkies, I, Argyriou, A, Boogerd, W, Briani, C, Bruna, J, Faber, Cg, Grisold, W, Heimans, Jj, Kalofonos, H, Krarup Hansen, A, Koeppen, S, Lauria, G, Leandri, M, Mielke, S, Nobile Orazio, E, Pace, A, Padua, Luca, Plasmati, R, Psimaras, D, Ros, T, Schenone, A, Stragliotto, G, Galimberti, S, Valsecchi, Mg, Merkies, Is, Padua, Luca (ORCID:0000-0003-2570-9326), Cavaletti, G, Cornblath, Dr, Postma, Tj, Merkies, I, Argyriou, A, Boogerd, W, Briani, C, Bruna, J, Faber, Cg, Grisold, W, Heimans, Jj, Kalofonos, H, Krarup Hansen, A, Koeppen, S, Lauria, G, Leandri, M, Mielke, S, Nobile Orazio, E, Pace, A, Padua, Luca, Plasmati, R, Psimaras, D, Ros, T, Schenone, A, Stragliotto, G, Galimberti, S, Valsecchi, Mg, Merkies, Is, and Padua, Luca (ORCID:0000-0003-2570-9326)

Published
2009

31. Reply

Author

Corse, Am, primary, Chaudhry, V, additional, Kuncl, Rw, additional, Cornblath, Dr, additional, Crawford, T, additional, and Griffin, Jw, additional

Published
1996
Full Text
View/download PDF

43. Electrodiagnostic subtyping in Guillain-Barré syndrome patients in the International Guillain-Barré Outcome Study.

Author

Arends S, Drenthen J, de Koning L, van den Bergh P, Hadden RDM, Kuwabara S, Reisin RC, Shahrizaila N, Ajroud-Driss S, Antonini G, Attarian S, Balducci C, Bertorini T, Brannagan TH, Cavaletti G, Chao CC, Chavada G, Dillmann KU, Dimachkie MM, Galassi G, Gutiérrez-Gutiérrez G, Harbo T, Islam B, Islam Z, Katzberg H, Kusunoki S, Manganelli F, Miller JAL, Pardo J, Pereon Y, Rajabally YA, Sindrup S, Stettner M, Uncini A, Verhamme C, Vytopil M, Waheed W, Jacobs BC, and Cornblath DR

Subjects
Humans, Male, Female, Middle Aged, Adult, Amyotrophic Lateral Sclerosis diagnosis, Amyotrophic Lateral Sclerosis classification, Amyotrophic Lateral Sclerosis physiopathology, Aged, Cohort Studies, Guillain-Barre Syndrome diagnosis, Guillain-Barre Syndrome classification, Guillain-Barre Syndrome physiopathology, Neural Conduction physiology, Electrodiagnosis methods
Abstract

Background and Purpose: Various electrodiagnostic criteria have been developed in Guillain-Barré syndrome (GBS). Their performance in a broad representation of GBS patients has not been evaluated. Motor conduction data from the International GBS Outcome Study (IGOS) cohort were used to compare two widely used criterion sets and relate these to diagnostic amyotrophic lateral sclerosis criteria., Methods: From the first 1500 patients in IGOS, nerve conduction studies from 1137 (75.8%) were available for the current study. These patients were classified according to nerve conduction studies criteria proposed by Hadden and Rajabally., Results: Of the 1137 studies, 68.3% (N = 777) were classified identically according to criteria by Hadden and Rajabally: 111 (9.8%) axonal, 366 (32.2%) demyelinating, 195 (17.2%) equivocal, 35 (3.1%) inexcitable and 70 (6.2%) normal. Thus, 360 studies (31.7%) were classified differently. The areas of differences were as follows: 155 studies (13.6%) classified as demyelinating by Hadden and axonal by Rajabally; 122 studies (10.7%) classified as demyelinating by Hadden and equivocal by Rajabally; and 75 studies (6.6%) classified as equivocal by Hadden and axonal by Rajabally. Due to more strictly defined cutoffs fewer patients fulfilled demyelinating criteria by Rajabally than by Hadden, making more patients eligible for axonal or equivocal classification by Rajabally. In 234 (68.6%) axonal studies by Rajabally the revised El Escorial (amyotrophic lateral sclerosis) criteria were fulfilled; in axonal cases by Hadden this was 1.8%., Conclusions and Discussion: This study shows that electrodiagnosis in GBS is dependent on the criterion set utilized, both of which are based on expert opinion. Reappraisal of electrodiagnostic subtyping in GBS is warranted., (© 2024 The Authors. European Journal of Neurology published by John Wiley & Sons Ltd on behalf of European Academy of Neurology.)

Published
2024
Full Text
View/download PDF

44. Repurposing chemotherapy-induced peripheral neuropathy grading.

Author

Velasco R, Argyriou AA, Cornblath DR, Bruna P, Alberti P, Rossi E, Merkies ISJ, Psimaras D, Briani C, Lalisang RI, Schenone A, Cavaletti G, and Bruna J

Subjects
Humans, Male, Female, Middle Aged, Aged, Severity of Illness Index, Prospective Studies, Adult, Quality of Life, Neoplasms drug therapy, Peripheral Nervous System Diseases chemically induced, Peripheral Nervous System Diseases diagnosis, Antineoplastic Agents adverse effects
Abstract

Background and Purpose: Chemotherapy-induced peripheral neuropathy (CIPN) is perceived differently by patients and physicians, complicating its assessment. Current recommendations advocate combining clinical and patient-reported outcomes measures, but this approach can be challenging in patient care. This multicenter European study aims to bridge the gap between patients' perceptions and neurological impairments by aligning both perspectives to improve treatment decision-making., Methods: Data were pooled from two prospective studies of subjects (n = 372) with established CIPN. Patient and physician views regarding CIPN were assessed using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE), Total Neuropathy Scale-clinical version (TNSc) items, and the disease-specific quality of life - Chemotherapy-Induced Peripheral Neuropathy questionnaire (QLQ-CIPN20) from the European Organization for Research and Treatment of Cancer (EORTC). To identify inherent neurotoxic severity patterns, we employed hierarchical cluster analysis optimized with k-means clustering and internally validated by discriminant functional analysis., Results: Both NCI-CTCAE and TNSc demonstrated a significant difference in the distribution of severity grades in relation to QLQ-CIPN20 scores. However, a proportion of subjects with different neurotoxic severity grades exhibited overlapping QLQ-CIPN20 scores. We identified three distinct clusters classifying subjects as having severely impaired, intermediately impaired, and mildly impaired CIPN based on TNSc and QLQ-CIPN20 scores. No differences in demographics, cancer type distribution, or class of drug received were observed., Conclusions: Our results confirm the heterogeneity in CIPN perception between patients and physicians and identify three well-differentiated subgroups of patients delineated by degree of CIPN impairment based on scores derived from TNSc and QLQ-CIPN20. A more refined assessment of CIPN could potentially be achieved using the calculator tool derived from the cluster equations in this study. This tool, which facilitates individual patient classification, requires prospective validation., (© 2024 The Author(s). European Journal of Neurology published by John Wiley & Sons Ltd on behalf of European Academy of Neurology.)

Published
2024
Full Text
View/download PDF

45. European Academy of Neurology/Peripheral Nerve Society Guideline on diagnosis and treatment of Guillain-Barré syndrome.

Author

van Doorn PA, Van den Bergh PYK, Hadden RDM, Avau B, Vankrunkelsven P, Attarian S, Blomkwist-Markens PH, Cornblath DR, Goedee HS, Harbo T, Jacobs BC, Kusunoki S, Lehmann HC, Lewis RA, Lunn MP, Nobile-Orazio E, Querol L, Rajabally YA, Umapathi T, Topaloglu HA, and Willison HJ

Subjects
Humans, Immunoglobulins, Intravenous therapeutic use, Peripheral Nerves, Pain, Adrenal Cortex Hormones, Guillain-Barre Syndrome diagnosis, Guillain-Barre Syndrome therapy, Respiratory Insufficiency drug therapy
Abstract

Guillain-Barré syndrome (GBS) is an acute polyradiculoneuropathy. Symptoms may vary greatly in presentation and severity. Besides weakness and sensory disturbances, patients may have cranial nerve involvement, respiratory insufficiency, autonomic dysfunction and pain. To develop an evidence-based guideline for the diagnosis and treatment of GBS, using Grading of Recommendations, Assessment, Development and Evaluation (GRADE) methodology, a Task Force (TF) of the European Academy of Neurology (EAN) and the Peripheral Nerve Society (PNS) constructed 14 Population/Intervention/Comparison/Outcome questions (PICOs) covering diagnosis, treatment and prognosis of GBS, which guided the literature search. Data were extracted and summarised in GRADE Summaries of Findings (for treatment PICOs) or Evidence Tables (for diagnostic and prognostic PICOs). Statements were prepared according to GRADE Evidence-to-Decision (EtD) frameworks. For the six intervention PICOs, evidence-based recommendations are made. For other PICOs, good practice points (GPPs) are formulated. For diagnosis, the principal GPPs are: GBS is more likely if there is a history of recent diarrhoea or respiratory infection; CSF examination is valuable, particularly when the diagnosis is less certain; electrodiagnostic testing is advised to support the diagnosis; testing for anti-ganglioside antibodies is of limited clinical value in most patients with typical motor-sensory GBS, but anti-GQ1b antibody testing should be considered when Miller Fisher syndrome (MFS) is suspected; nodal-paranodal antibodies should be tested when autoimmune nodopathy is suspected; MRI or ultrasound imaging should be considered in atypical cases; and changing the diagnosis to acute-onset chronic inflammatory demyelinating polyradiculoneuropathy (A-CIDP) should be considered if progression continues after 8 weeks from onset, which occurs in around 5% of patients initially diagnosed with GBS. For treatment, the TF recommends intravenous immunoglobulin (IVIg) 0.4 g/kg for 5 days, in patients within 2 weeks (GPP also within 2-4 weeks) after onset of weakness if unable to walk unaided, or a course of plasma exchange (PE) 12-15 L in four to five exchanges over 1-2 weeks, in patients within 4 weeks after onset of weakness if unable to walk unaided. The TF recommends against a second IVIg course in GBS patients with a poor prognosis; recommends against using oral corticosteroids, and weakly recommends against using IV corticosteroids; does not recommend PE followed immediately by IVIg; weakly recommends gabapentinoids, tricyclic antidepressants or carbamazepine for treatment of pain; does not recommend a specific treatment for fatigue. To estimate the prognosis of individual patients, the TF advises using the modified Erasmus GBS outcome score (mEGOS) to assess outcome, and the modified Erasmus GBS Respiratory Insufficiency Score (mEGRIS) to assess the risk of requiring artificial ventilation. Based on the PICOs, available literature and additional discussions, we provide flow charts to assist making clinical decisions on diagnosis, treatment and the need for intensive care unit admission., (© 2023 The Authors. Journal of the Peripheral Nervous System published by Wiley Periodicals LLC on behalf of Peripheral Nerve Society.)

Published
2023
Full Text
View/download PDF

46. Safety and Tolerability of Intravenous Immunoglobulin in Chronic Inflammatory Demyelinating Polyneuropathy: Results of the ProCID Study.

Author

Cornblath DR, van Doorn PA, Hartung HP, Merkies ISJ, Katzberg HD, Hinterberger D, and Clodi E

Subjects
Humans, Headache chemically induced, Treatment Outcome, Immunoglobulins, Intravenous adverse effects, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating drug therapy, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating chemically induced
Abstract

Background and Aims: The ProCID study evaluated the efficacy and safety of three doses of a 10% liquid intravenous immunoglobulin (IVIg) preparation (panzyga ® ) in patients with chronic inflammatory demyelinating polyneuropathy (CIDP). This report describes the safety findings., Methods: Patients were randomised to receive a 2.0 g/kg induction dose followed by maintenance doses of either 0.5, 1.0 or 2.0 g/kg IVIg every 3 weeks over 24 weeks., Results: All 142 enrolled patients were included in the safety analyses. In total, 286 treatment-emergent adverse events (TEAEs) were reported in 89 patients, of which 173 (60.5%) were considered treatment-related. Most TEAEs were of mild severity. Eleven serious TEAEs were reported in 6 patients. Two serious TEAEs in one patient (headache and vomiting) were considered related to treatment, which resolved without study discontinuation. No treatment-related thrombotic events, haemolytic transfusion reactions or deaths occurred. One patient discontinued the study due to a TEAE (allergic dermatitis) probably related to IVIg. Headache was the only dose-dependent TEAE, with incidences ranging from 2.9 to 23.7%, the incidence of all other TEAEs was similar across treatment groups. Most TEAEs were associated with the induction dose infusion, and the rate of TEAEs decreased thereafter. The median (IQR) daily IVIg dose was 78 (64-90) g, and 94.4% of patients tolerated the maximal infusion rate of 0.12 ml/kg/min without pre-medication., Interpretation: Infusions of 10% IVIg at doses up to 2.0 g/kg with high infusion rates were safe and well tolerated in patients with CIDP., Clinical Trial Numbers: EudraCT 2015-005443-14, NCT02638207., (© 2023. The Author(s).)

Published
2023
Full Text
View/download PDF

47. Peripheral Nerve Safety of Nerve Growth Factor Inhibition by Tanezumab: Pooled Analyses of Phase III Clinical Studies in Over 5000 Patients with Osteoarthritis.

Author

Brown MT, Cornblath DR, Koltzenburg M, Gorson KC, Hickman A, Pixton GC, Gaitonde P, Viktrup L, and West CR

Subjects
Humans, Hypesthesia complications, Pain drug therapy, Peripheral Nerves, Treatment Outcome, Clinical Trials, Phase III as Topic, Randomized Controlled Trials as Topic, Osteoarthritis, Knee drug therapy, Paresthesia complications, Antibodies, Monoclonal, Humanized therapeutic use, Nerve Growth Factor antagonists & inhibitors
Abstract

Background: Tanezumab, a humanized anti-nerve growth factor antibody, was developed for the treatment of pain associated with osteoarthritis. Due to its mechanism of action, peripheral nerve safety was assessed in all clinical studies., Objectives: To summarize the neurological safety of intravenous (IV) and subcutaneous (SC) tanezumab versus placebo in patients with osteoarthritis., Methods: Data were pooled from 3389 patients across seven studies that investigated IV administration, and from 1840 patients across three studies that investigated SC administration. The treatment period of each study ranged from 16 to 24 weeks, and follow-up periods ranged from 8 to 24 weeks. Neurological safety evaluations focused on adverse events (AEs) of abnormal peripheral sensation (APS), neurologic examinations, and consultations., Results: Across datasets, the incidence of AEs of APS was higher in tanezumab groups versus placebo. Paresthesia and hypoesthesia were the most frequently reported AEs in tanezumab-treated patients, compared with placebo. In both datasets, most AEs were of mild severity, resolved, and rarely resulted in discontinuation. In all treatment groups in both IV and SC studies, over 90% of patients had no new or worsened neurological examination abnormalities at the last study visit. Across datasets, mononeuropathy was diagnosed more frequently in tanezumab groups compared with placebo. Polyneuropathy was diagnosed in ≤ 0.9% of patients in tanezumab and placebo groups., Conclusions: Tanezumab IV or SC had an increased incidence of AEs of APS, such as paresthesia and hypoesthesia, and diagnoses of mononeuropathy compared with placebo. However, tanezumab was not associated with generalized peripheral neuropathy., Gov Identifiers: NCT00733902, NCT00744471, NCT00830063, NCT00863304, NCT00863772, NCT01089725, NCT00985621, NCT02697773, and NCT02709486., (© 2023. The Author(s), under exclusive licence to Springer Nature Switzerland AG.)

Published
2023
Full Text
View/download PDF

48. CSF Findings in Relation to Clinical Characteristics, Subtype, and Disease Course in Patients With Guillain-Barré Syndrome.

Author

Al-Hakem H, Doets AY, Stino AM, Zivkovic SA, Andersen H, Willison HJ, Cornblath DR, Gorson KC, Islam Z, Mohammad QD, Sindrup SH, Kusunoki S, Davidson A, Casasnovas C, Bateman K, Miller JAL, van den Berg B, Verboon C, Roodbol J, Leonhard SE, Arends S, Luijten LWG, Benedetti L, Kuwabara S, Van den Bergh P, Monges S, Marfia GA, Shahrizaila N, Galassi G, Pereon Y, Bürmann J, Kuitwaard K, Kleyweg RP, Marchesoni C, Sedano Tous MJ, Querol L, Martín-Aguilar L, Wang Y, Nobile-Orazio E, Rinaldi S, Schenone A, Pardo J, Vermeij FH, Waheed W, Lehmann HC, Granit V, Stein B, Cavaletti G, Gutiérrez-Gutiérrez G, Barroso FA, Visser LH, Katzberg HD, Dardiotis E, Attarian S, van der Kooi AJ, Eftimov F, Wirtz PW, Samijn JPA, Gilhuis HJ, Hadden RDM, Holt JKL, Sheikh KA, Kolb N, Karafiath S, Vytopil M, Antonini G, Feasby TE, Faber C, Kramers H, Busby M, Roberts RC, Silvestri NJ, Fazio R, van Dijk GW, Garssen MPJ, Verschuuren J, Harbo T, and Jacobs BC

Subjects
Adult, Female, Humans, Male, Middle Aged, Cell Count, Cerebrospinal Fluid cytology, Cohort Studies, Disease Progression, Internationality, Miller Fisher Syndrome cerebrospinal fluid, Miller Fisher Syndrome diagnosis, Miller Fisher Syndrome pathology, Miller Fisher Syndrome physiopathology, Prognosis, Treatment Outcome, Guillain-Barre Syndrome cerebrospinal fluid, Guillain-Barre Syndrome diagnosis, Guillain-Barre Syndrome pathology, Guillain-Barre Syndrome physiopathology
Abstract

Background and Objectives: To investigate CSF findings in relation to clinical and electrodiagnostic subtypes, severity, and outcome of Guillain-Barré syndrome (GBS) based on 1,500 patients in the International GBS Outcome Study., Methods: Albuminocytologic dissociation (ACD) was defined as an increased protein level (>0.45 g/L) in the absence of elevated white cell count (<50 cells/μL). We excluded 124 (8%) patients because of other diagnoses, protocol violation, or insufficient data. The CSF was examined in 1,231 patients (89%)., Results: In 846 (70%) patients, CSF examination showed ACD, which increased with time from weakness onset: ≤4 days 57%, >4 days 84%. High CSF protein levels were associated with a demyelinating subtype, proximal or global muscle weakness, and a reduced likelihood of being able to run at week 2 (odds ratio [OR] 0.42, 95% CI 0.25-0.70; p = 0.001) and week 4 (OR 0.44, 95% CI 0.27-0.72; p = 0.001). Patients with the Miller Fisher syndrome, distal predominant weakness, and normal or equivocal nerve conduction studies were more likely to have lower CSF protein levels. CSF cell count was <5 cells/μL in 1,005 patients (83%), 5-49 cells/μL in 200 patients (16%), and ≥50 cells/μL in 13 patients (1%)., Discussion: ACD is a common finding in GBS, but normal protein levels do not exclude this diagnosis. High CSF protein level is associated with an early severe disease course and a demyelinating subtype. Elevated CSF cell count, rarely ≥50 cells/μL, is compatible with GBS after a thorough exclusion of alternative diagnoses., Classification of Evidence: This study provides Class IV evidence that CSF ACD (defined by the Brighton Collaboration) is common in patients with GBS., (© 2023 American Academy of Neurology.)

Published
2023
Full Text
View/download PDF

49. IgM anti-MAG ± peripheral neuropathy (IMAGiNe) study protocol: An international, observational, prospective registry of patients with IgM M-protein peripheral neuropathies.

Author

Hamadeh T, van Doormaal PTC, Pruppers MHJ, van de Mortel JPM, Hoeijmakers JGJ, Cornblath DR, Vrancken AFJE, Faber CG, Notermans NC, and Merkies ISJ

Subjects
Humans, Immunoglobulin M, Myelin-Associated Glycoprotein, Biomarkers, Autoantibodies, Ataxia, Observational Studies as Topic, Peripheral Nervous System Diseases diagnosis, Peripheral Nervous System Diseases therapy
Abstract

Background: International consensus on IgM ± anti-MAG ± PNP (IgM PNP) is lacking. Despite increasing interest in clinical trials, validated disease-specific measures are needed to adequately capture limitations and changes over time. The IMAGiNe (IgM ± anti-myelin associated glycoprotein [MAG] peripheral neuropathy) study surges as an international collaboration to create a standardized registry of patients with IgM ± anti-MAG PNP. The consortium, which currently consists of 11 institutions from 7 countries, presents here the IMAGiNe study design and protocol., Aims: Functional outcome measures will be constructed at the level of impairment, as well as activity and participation. We aim to describe the natural history of the cohort, the role of anti-MAG antibodies, the presence of clinical subtypes, and potential biomarkers., Methods: The IMAGiNe study is a prospective, observational cohort study with a 3-year follow-up. At each assessment, researchers collect clinical data and subjects complete a list of preselected outcome measures. Among these, the "Pre-Rasch-built Overall Disability Scale (Pre-RODS)" questionnaire will be submitted to Rasch analysis to assess classic and modern clinimetric requirements., Results: The final measures will include the IgM-PNP-specific RODS and Ataxia Rating Scale (IgM-PNP-ARS). Descriptions of the disease course, clinical heterogeneity, treatment regimes, variations in laboratory values, and antibody titers will help reach consensus on diagnosis and follow-up strategies., Conclusion: The constructed interval scales will be cross-culturally valid and suitable for use in future clinical trials and daily practice. The ultimate goals are to improve functional individualized assessment, reach international consensus, and lay the foundations for successful designs in future studies., (© 2023 The Authors. Journal of the Peripheral Nervous System published by Wiley Periodicals LLC on behalf of Peripheral Nerve Society.)

Published
2023
Full Text
View/download PDF

50. Letter to the Editor re "Complete Foot Drop With Normal Electrodiagnostic Studies: Sunderland 'Zero' Ischemic Conduction Block of the Common Peroneal Nerve" by Peters and Colleagues.

Author

Cornblath DR, Kiernan MC, Kimura J, Koltzenburg M, Lewis RA, Parry GJ, Pollard JD, and Sumner AJ

Subjects
Humans, Neural Conduction, Peroneal Nerve, Peroneal Neuropathies diagnosis
Abstract

Competing Interests: Conflicts of interest and sources of funding: none declared.

Published
2022
Full Text
View/download PDF

Catalog

Books, media, physical & digital resources
See catalog results