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1. Electrodiagnosis of Guillain-Barre syndrome in the International GBS Outcome Study: Differences in methods and reference values

Author

Arends, S, Drenthen, J, van den Bergh, P, Franssen, H, Hadden, R, Islam, B, Kuwabara, S, Reisin, R, Shahrizaila, N, Amino, H, Antonini, G, Attarian, S, Balducci, C, Barroso, F, Bertorini, T, Binda, D, Brannagan, T, Buermann, J, Casasnovas, C, Cavaletti, G, Chao, C, Dimachkie, M, Fulgenzi, E, Galassi, G, Gutierrez Gutierrez, G, Harbo, T, Hartung, H, Hsieh, S, Kiers, L, Lehmann, H, Manganelli, F, Marfia, G, Mataluni, G, Pardo, J, Pereon, Y, Rajabally, Y, Santoro, L, Sekiguchi, Y, Stein, B, Stettner, M, Uncini, A, Verboon, C, Verhamme, C, Vytopil, M, Waheed, W, Wang, M, Zivkovic, S, Jacobs, B, Cornblath, D, Arends S., Drenthen J., van den Bergh P., Franssen H., Hadden R. D. M., Islam B., Kuwabara S., Reisin R. C., Shahrizaila N., Amino H., Antonini G., Attarian S., Balducci C., Barroso F., Bertorini T., Binda D., Brannagan T. H., Buermann J., Casasnovas C., Cavaletti G., Chao C. -C., Dimachkie M. M., Fulgenzi E. A., Galassi G., Gutierrez Gutierrez G., Harbo T., Hartung H. -P., Hsieh S. -T., Kiers L., Lehmann H. C., Manganelli F., Marfia G. A., Mataluni G., Pardo J., Pereon Y., Rajabally Y. A., Santoro L., Sekiguchi Y., Stein B., Stettner M., Uncini A., Verboon C., Verhamme C., Vytopil M., Waheed W., Wang M., Zivkovic S., Jacobs B. C., Cornblath D. R., Arends, S, Drenthen, J, van den Bergh, P, Franssen, H, Hadden, R, Islam, B, Kuwabara, S, Reisin, R, Shahrizaila, N, Amino, H, Antonini, G, Attarian, S, Balducci, C, Barroso, F, Bertorini, T, Binda, D, Brannagan, T, Buermann, J, Casasnovas, C, Cavaletti, G, Chao, C, Dimachkie, M, Fulgenzi, E, Galassi, G, Gutierrez Gutierrez, G, Harbo, T, Hartung, H, Hsieh, S, Kiers, L, Lehmann, H, Manganelli, F, Marfia, G, Mataluni, G, Pardo, J, Pereon, Y, Rajabally, Y, Santoro, L, Sekiguchi, Y, Stein, B, Stettner, M, Uncini, A, Verboon, C, Verhamme, C, Vytopil, M, Waheed, W, Wang, M, Zivkovic, S, Jacobs, B, Cornblath, D, Arends S., Drenthen J., van den Bergh P., Franssen H., Hadden R. D. M., Islam B., Kuwabara S., Reisin R. C., Shahrizaila N., Amino H., Antonini G., Attarian S., Balducci C., Barroso F., Bertorini T., Binda D., Brannagan T. H., Buermann J., Casasnovas C., Cavaletti G., Chao C. -C., Dimachkie M. M., Fulgenzi E. A., Galassi G., Gutierrez Gutierrez G., Harbo T., Hartung H. -P., Hsieh S. -T., Kiers L., Lehmann H. C., Manganelli F., Marfia G. A., Mataluni G., Pardo J., Pereon Y., Rajabally Y. A., Santoro L., Sekiguchi Y., Stein B., Stettner M., Uncini A., Verboon C., Verhamme C., Vytopil M., Waheed W., Wang M., Zivkovic S., Jacobs B. C., and Cornblath D. R.

Abstract

Objective: To describe the heterogeneity of electrodiagnostic (EDx) studies in Guillain-Barré syndrome (GBS) patients collected as part of the International GBS Outcome Study (IGOS). Methods: Prospectively collected clinical and EDx data were available in 957 IGOS patients from 115 centers. Only the first EDx study was included in the current analysis. Results: Median timing of the EDx study was 7 days (interquartile range 4–11) from symptom onset. Methodology varied between centers, countries and regions. Reference values from the responding 103 centers were derived locally in 49%, from publications in 37% and from a combination of these in the remaining 15%. Amplitude measurement in the EDx studies (baseline-to-peak or peak-to-peak) differed from the way this was done in the reference values, in 22% of motor and 39% of sensory conduction. There was marked variability in both motor and sensory reference values, although only a few outliers accounted for this. Conclusions: Our study showed extensive variation in the clinical practice of EDx in GBS patients among IGOS centers across the regions. Significance: Besides EDx variation in GBS patients participating in IGOS, this diversity is likely to be present in other neuromuscular disorders and centers. This underlines the need for standardization of EDx in future multinational GBS studies.

Published
2022

3. Efficacy of botulinum toxin type a in the targeted treatment of sleep bruxism: a double-blind, randomised, placebo-controlled, cross-over study

Author

Cruse, B, Dharmadasa, T, White, E, Hollis, C, Evans, A, Sharmin, S, Kalincik, T, Kiers, L, Cruse, B, Dharmadasa, T, White, E, Hollis, C, Evans, A, Sharmin, S, Kalincik, T, and Kiers, L

Abstract

BACKGROUND: Intramuscular injections of botulinum toxin A (BTX-A) have been used in the treatment of sleep bruxism (SB) however controlled trials are limited and the optimal injection strategy and dose is not known. METHODS: This double-blind, randomised, placebo-controlled, cross-over study evaluated the efficacy and safety of BTX-A in participants with SB. Average bruxism events per hour of sleep (Bruxism Index, BI) was calculated using surface electromyography. Participants with BI >5 were included and randomised by order of injection (active or placebo with the opposite 20 weeks later) and into one of three differing treatment groups: bilateral masseter (60 units(U)), bilateral masseter and temporalis (90U) and bilateral masseter, temporalis and medial pterygoid muscles (120U). Change in BI and subjective measures of headache, pain, and bruxism at 4 and 12 weeks was calculated following intervention, and differences between treatment groups analysed. RESULTS: 41 participants were recruited, 35 randomised and data from 22 participants (14 female) were analysed. BI was significantly lower at 4 weeks after active treatment when compared with placebo (mean=-1.66, p=0.003), not sustained at 12 weeks. The difference was greater with higher doses injected and among those with greater baseline BI. There was no difference in subjective measures at any time point. Five participants injected had mild, transient side effects. DISCUSSION: Targeted BTX-A injection is a safe and effective treatment for SB. A greater benefit may be achieved by administering BTX-A into more muscles and at higher total doses and among those with higher baseline BI. TRIAL REGISTRATION NUMBER: ACTRN12618001430224.

Published
2022

4. Current treatment practice of Guillain-Barré syndrome

Author

Verboon, C, Doets, A, Galassi, G, Davidson, A, Waheed, W, Pereon, Y, Shahrizaila, N, Kusunoki, S, Lehmann, H, Harbo, T, Monges, S, Van Den Bergh, P, Willison, H, Cornblath, D, Jacobs, B, Hughes, R, Gorson, K, Hartung, H, Van Doorn, P, Van den Berg, B, Roodbol, J, Van Woerkom, M, Reisin, R, Reddel, S, Islam, Z, Islam, B, Mohammad, Q, Feasby, T, Dardiotis, E, Nobile-Orazio, E, Bateman, K, Illa, I, Querol, L, Hsieh, S, Chavada, G, Addington, J, Ajroud-Driss, S, Andersen, H, Antonini, G, Ariatti, A, Attarian, S, Badrising, U, Barroso, F, Benedetti, L, Beronio, A, Bianco, M, Binda, D, Briani, C, Bunschoten, C, Burmann, J, Bella, I, Bertorini, T, Bhavaraju-Sanka, R, Brannagan, T, Busby, M, Butterworth, S, Casasnovas, C, Cavaletti, G, Chao, C, Chen, S, Chetty, S, Claeys, K, Conti, M, Cosgrove, J, Dalakas, M, Demichelis, C, Derejko, M, Dillmann, U, Dimachkie, M, Doppler, K, Dornonville de la Cour, C, Echaniz-Laguna, A, Eftimov, F, Faber, C, Fazio, R, Fokke, C, Fujioka, T, Fulgenzi, E, Garcia-Sobrino, T, Garssen, M, Georgios, H, Gijsbers, C, Gilchrist, J, Gilhuis, J, Giorli, E, Goldstein, J, Goyal, N, Granit, V, Grapperon, A, Gutierrez, G, Hadden, R, Holbech, J, Holt, J, Pedret, C, Htut, M, Jellema, K, Pascual, I, Jimeno-Montero, M, Kaida, K, Karafiath, S, Katzberg, H, Kiers, L, Kieseier, B, Kimpinski, K, Kleyweg, R, Kokubun, N, Kolb, N, Kuitwaard, K, Kuwabara, S, Kwan, J, Ladha, S, Lassen, L, Lawson, V, Ledingham, D, Lucy, S, Lunn, M, Magot, A, Manji, H, Marchesoni, C, Marfia, G, Infante, C, Hernandez, E, Mataluni, G, Mattiazi, M, Mcdermott, C, Meekins, G, Miller, J, Moris de la Tassa, G, Physiotherapist, J, Nascimbene, C, Nowak, R, Balaguer, P, Osei-Bonsu, M, Pan, E, Pardal, A, Pardo, J, Pasnoor, M, Pulley, M, Rajabally, Y, Rinaldi, S, Ritter, C, Roberts, R, Rojas-Marcos, I, Rudnicki, S, Ruiz, M, Sachs, G, Samijn, J, Santoro, L, Savransky, A, Schenone, A, Schwindling, L, Tous, M, Sekiguchi, Y, Sheikh, K, Silvestri, N, Sindrup, S, Sommer, C, Stein, B, Stino, A, Spyropoulos, A, Srinivasan, J, Styliani, R, Suzuki, H, Tankisi, H, Tigner, D, Twydell, P, Van Damme, P, Van der Kooi, A, Van Dijk, G, Van der Ree, T, Van Koningsveld, R, Valzania, F, Varrato, J, Vermeij, F, Verschuuren, J, Visser, L, Vytopil, M, Wilken, M, Wilkerson, C, Wirtz, P, Yamagishi, Y, Zhou, L, Zivkovic, S, Verboon C., Doets A. Y., Galassi G., Davidson A., Waheed W., Pereon Y., Shahrizaila N., Kusunoki S., Lehmann H. C., Harbo T., Monges S., Van Den Bergh P., Willison H. J., Cornblath D. R., Jacobs B. C., Hughes R. A. C., Gorson K. C., Hartung H. P., Van Doorn P. A., Van den Berg B., Roodbol J., Van Woerkom M., Reisin R. C., Reddel S. W., Islam Z., Islam B., Mohammad Q. D., Feasby T. E., Dardiotis E., Nobile-Orazio E., Bateman K., Illa I., Querol L., Hsieh S. T., Chavada G., Addington J. M., Ajroud-Driss S., Andersen H., Antonini G., Ariatti A., Attarian S., Badrising U. A., Barroso F. A., Benedetti L., Beronio A., Bianco M., Binda D., Briani C., Bunschoten C., Burmann J., Bella I. R., Bertorini T. E., Bhavaraju-Sanka R., Brannagan T. H., Busby M., Butterworth S., Casasnovas C., Cavaletti G., Chao C. C., Chen S., Chetty S., Claeys K. G., Conti M. E., Cosgrove J. S., Dalakas MC., Demichelis C., Derejko M. A., Dillmann U., Dimachkie M. M., Doppler K., Dornonville de la Cour C., Echaniz-Laguna A., Eftimov F., Faber C. G., Fazio R., Fokke C., Fujioka T., Fulgenzi E. A., Garcia-Sobrino T., Garssen M. P. J., Georgios H. M., Gijsbers C. J., Gilchrist J. M., Gilhuis J., Giorli E., Goldstein J. M., Goyal N. A., Granit V., Grapperon A., Gutierrez G., Hadden R. D. M., Holbech J. V., Holt J. K. L., Pedret C. H., Htut M., Jellema K., Pascual I. J., Jimeno-Montero M. C., Kaida K., Karafiath S., Katzberg H. D., Kiers L., Kieseier B. C., Kimpinski K., Kleyweg R. P., Kokubun N., Kolb N. A., Kuitwaard K., Kuwabara S., Kwan J. Y., Ladha S. S., Lassen L. L., Lawson V., Ledingham D., Lucy S. T., Lunn M. P. T., Magot A., Manji H., Marchesoni C., Marfia G. A., Infante C. M., Hernandez E. M., Mataluni G., Mattiazi M., McDermott C. J., Meekins G. D., Miller J. A. L., Moris de la Tassa G., Physiotherapist J. M., Nascimbene C., Nowak R. J., Balaguer P. O., Osei-Bonsu M., Pan E. B. L., Pardal A. M., Pardo J., Pasnoor M., Pulley M., Rajabally Y. A., Rinaldi S., Ritter C., Roberts R. C., Rojas-Marcos I., Rudnicki S. A., Ruiz M., Sachs G. M., Samijn J. P. A., Santoro L., Savransky A., Schenone A., Schwindling L., Tous M. J. S., Sekiguchi Y., Sheikh K. A., Silvestri N. J., Sindrup S. H., Sommer C. L., Stein B., Stino A. M., Spyropoulos A., Srinivasan J., Styliani R., Suzuki H., Tankisi H., Tigner D., Twydell P., Van Damme P., Van der Kooi A. J., Van Dijk G. W., Van der Ree T., Van Koningsveld R., Valzania F., Varrato J. D., Vermeij F. H., Verschuuren J., Visser L. H., Vytopil M. V., Wilken M., Wilkerson C., Wirtz P. W., Yamagishi Y., Zhou L., Zivkovic S. A., Verboon, C, Doets, A, Galassi, G, Davidson, A, Waheed, W, Pereon, Y, Shahrizaila, N, Kusunoki, S, Lehmann, H, Harbo, T, Monges, S, Van Den Bergh, P, Willison, H, Cornblath, D, Jacobs, B, Hughes, R, Gorson, K, Hartung, H, Van Doorn, P, Van den Berg, B, Roodbol, J, Van Woerkom, M, Reisin, R, Reddel, S, Islam, Z, Islam, B, Mohammad, Q, Feasby, T, Dardiotis, E, Nobile-Orazio, E, Bateman, K, Illa, I, Querol, L, Hsieh, S, Chavada, G, Addington, J, Ajroud-Driss, S, Andersen, H, Antonini, G, Ariatti, A, Attarian, S, Badrising, U, Barroso, F, Benedetti, L, Beronio, A, Bianco, M, Binda, D, Briani, C, Bunschoten, C, Burmann, J, Bella, I, Bertorini, T, Bhavaraju-Sanka, R, Brannagan, T, Busby, M, Butterworth, S, Casasnovas, C, Cavaletti, G, Chao, C, Chen, S, Chetty, S, Claeys, K, Conti, M, Cosgrove, J, Dalakas, M, Demichelis, C, Derejko, M, Dillmann, U, Dimachkie, M, Doppler, K, Dornonville de la Cour, C, Echaniz-Laguna, A, Eftimov, F, Faber, C, Fazio, R, Fokke, C, Fujioka, T, Fulgenzi, E, Garcia-Sobrino, T, Garssen, M, Georgios, H, Gijsbers, C, Gilchrist, J, Gilhuis, J, Giorli, E, Goldstein, J, Goyal, N, Granit, V, Grapperon, A, Gutierrez, G, Hadden, R, Holbech, J, Holt, J, Pedret, C, Htut, M, Jellema, K, Pascual, I, Jimeno-Montero, M, Kaida, K, Karafiath, S, Katzberg, H, Kiers, L, Kieseier, B, Kimpinski, K, Kleyweg, R, Kokubun, N, Kolb, N, Kuitwaard, K, Kuwabara, S, Kwan, J, Ladha, S, Lassen, L, Lawson, V, Ledingham, D, Lucy, S, Lunn, M, Magot, A, Manji, H, Marchesoni, C, Marfia, G, Infante, C, Hernandez, E, Mataluni, G, Mattiazi, M, Mcdermott, C, Meekins, G, Miller, J, Moris de la Tassa, G, Physiotherapist, J, Nascimbene, C, Nowak, R, Balaguer, P, Osei-Bonsu, M, Pan, E, Pardal, A, Pardo, J, Pasnoor, M, Pulley, M, Rajabally, Y, Rinaldi, S, Ritter, C, Roberts, R, Rojas-Marcos, I, Rudnicki, S, Ruiz, M, Sachs, G, Samijn, J, Santoro, L, Savransky, A, Schenone, A, Schwindling, L, Tous, M, Sekiguchi, Y, Sheikh, K, Silvestri, N, Sindrup, S, Sommer, C, Stein, B, Stino, A, Spyropoulos, A, Srinivasan, J, Styliani, R, Suzuki, H, Tankisi, H, Tigner, D, Twydell, P, Van Damme, P, Van der Kooi, A, Van Dijk, G, Van der Ree, T, Van Koningsveld, R, Valzania, F, Varrato, J, Vermeij, F, Verschuuren, J, Visser, L, Vytopil, M, Wilken, M, Wilkerson, C, Wirtz, P, Yamagishi, Y, Zhou, L, Zivkovic, S, Verboon C., Doets A. Y., Galassi G., Davidson A., Waheed W., Pereon Y., Shahrizaila N., Kusunoki S., Lehmann H. C., Harbo T., Monges S., Van Den Bergh P., Willison H. J., Cornblath D. R., Jacobs B. C., Hughes R. A. C., Gorson K. C., Hartung H. P., Van Doorn P. A., Van den Berg B., Roodbol J., Van Woerkom M., Reisin R. C., Reddel S. W., Islam Z., Islam B., Mohammad Q. D., Feasby T. E., Dardiotis E., Nobile-Orazio E., Bateman K., Illa I., Querol L., Hsieh S. T., Chavada G., Addington J. M., Ajroud-Driss S., Andersen H., Antonini G., Ariatti A., Attarian S., Badrising U. A., Barroso F. A., Benedetti L., Beronio A., Bianco M., Binda D., Briani C., Bunschoten C., Burmann J., Bella I. R., Bertorini T. E., Bhavaraju-Sanka R., Brannagan T. H., Busby M., Butterworth S., Casasnovas C., Cavaletti G., Chao C. C., Chen S., Chetty S., Claeys K. G., Conti M. E., Cosgrove J. S., Dalakas MC., Demichelis C., Derejko M. A., Dillmann U., Dimachkie M. M., Doppler K., Dornonville de la Cour C., Echaniz-Laguna A., Eftimov F., Faber C. G., Fazio R., Fokke C., Fujioka T., Fulgenzi E. A., Garcia-Sobrino T., Garssen M. P. J., Georgios H. M., Gijsbers C. J., Gilchrist J. M., Gilhuis J., Giorli E., Goldstein J. M., Goyal N. A., Granit V., Grapperon A., Gutierrez G., Hadden R. D. M., Holbech J. V., Holt J. K. L., Pedret C. H., Htut M., Jellema K., Pascual I. J., Jimeno-Montero M. C., Kaida K., Karafiath S., Katzberg H. D., Kiers L., Kieseier B. C., Kimpinski K., Kleyweg R. P., Kokubun N., Kolb N. A., Kuitwaard K., Kuwabara S., Kwan J. Y., Ladha S. S., Lassen L. L., Lawson V., Ledingham D., Lucy S. T., Lunn M. P. T., Magot A., Manji H., Marchesoni C., Marfia G. A., Infante C. M., Hernandez E. M., Mataluni G., Mattiazi M., McDermott C. J., Meekins G. D., Miller J. A. L., Moris de la Tassa G., Physiotherapist J. M., Nascimbene C., Nowak R. J., Balaguer P. O., Osei-Bonsu M., Pan E. B. L., Pardal A. M., Pardo J., Pasnoor M., Pulley M., Rajabally Y. A., Rinaldi S., Ritter C., Roberts R. C., Rojas-Marcos I., Rudnicki S. A., Ruiz M., Sachs G. M., Samijn J. P. A., Santoro L., Savransky A., Schenone A., Schwindling L., Tous M. J. S., Sekiguchi Y., Sheikh K. A., Silvestri N. J., Sindrup S. H., Sommer C. L., Stein B., Stino A. M., Spyropoulos A., Srinivasan J., Styliani R., Suzuki H., Tankisi H., Tigner D., Twydell P., Van Damme P., Van der Kooi A. J., Van Dijk G. W., Van der Ree T., Van Koningsveld R., Valzania F., Varrato J. D., Vermeij F. H., Verschuuren J., Visser L. H., Vytopil M. V., Wilken M., Wilkerson C., Wirtz P. W., Yamagishi Y., Zhou L., and Zivkovic S. A.

Abstract

ObjectiveTo define the current treatment practice of Guillain-Barré syndrome (GBS).MethodsThe study was based on prospective observational data from the first 1,300 patients included in the International GBS Outcome Study. We described the treatment practice of GBS in general, and for (1) severe forms (unable to walk independently), (2) no recovery after initial treatment, (3) treatment-related fluctuations, (4) mild forms (able to walk independently), and (5) variant forms including Miller Fisher syndrome, taking patient characteristics and hospital type into account.ResultsWe excluded 88 (7%) patients because of missing data, protocol violation, or alternative diagnosis. Patients from Bangladesh (n = 189, 15%) were described separately because 83% were not treated. IV immunoglobulin (IVIg), plasma exchange (PE), or other immunotherapy was provided in 941 (92%) of the remaining 1,023 patients, including patients with severe GBS (724/743, 97%), mild GBS (126/168, 75%), Miller Fisher syndrome (53/70, 76%), and other variants (33/40, 83%). Of 235 (32%) patients who did not improve after their initial treatment, 82 (35%) received a second immune modulatory treatment. A treatment-related fluctuation was observed in 53 (5%) of 1,023 patients, of whom 36 (68%) were re-treated with IVIg or PE.ConclusionsIn current practice, patients with mild and variant forms of GBS, or with treatment-related fluctuations and treatment failures, are frequently treated, even in absence of trial data to support this choice. The variability in treatment practice can be explained in part by the lack of evidence and guidelines for effective treatment in these situations.

Published
2019

5. Lesson study als afstudeeronderzoek op de pabo

Author

Gijssel, M.A.R. (Martine), Klaver, L.T. (Lida), Kiers, L. (Lilian), Lubbers, M.J.C. (Mariët), and Lenferink, M.L.G. (Marian)

Subjects
Onderzoekend vermogen, praktijkonderzoek, artikel
Abstract

Eerder gepubliceerd in Tijdschrift voor Lerarenopleiders, 2021, nr.2 Studenten van de lerarenopleiding basisonderwijs (pabo) voeren in het laatste jaar van hun studie doorgaans zelfstandig een praktijkonderzoek uit, dat getoetst wordt aan de hand van een verslag. Lerarenopleiders van Hogeschool Saxion en vanuit het werkveld vroegen zich af of met een andere insteek van het afstudeeronderzoek de onderzoekende houding van studenten en de aansluiting bij de onderwijspraktijk beter gerealiseerd zouden kunnen worden. Daarom is een nieuwe variant ontwikkeld en beproefd: Lesson Study als afstudeeronderzoek. Studenten formuleerden in groepjes op basis van een analyse van de schoolcontext een onderzoeksvraag en ontwikkelden met behulp van de theorie vervolgens samen een les. Deze les werd uitgevoerd, geobserveerd en nabesproken door de studenten, waarbij het denken en leren van leerlingen centraal stond. We onderzochten in hoeverre ‘Lesson Study als afstudeeronderzoek’ geschikt is om het onderzoekend vermogen van pabo-studenten te stimuleren, en welke aanpassingen in het ontwerp nodig zijn voor implementatie. Zes deeltijdstudenten en twee voltijdstudenten hebben deelgenomen (twee groepen van vier studenten). De groepen werden begeleid door twee docenten. Door middel van een vragenlijst, interviews en docentenlogboeken is antwoord verkregen op de onderzoeksvragen. Op basis van de resultaten concluderen we dat de onderzoekende houding van studenten in dit afstudeeronderzoek ruim voldoende is gestimuleerd en de onderzoeksvaardigheden zijn bevorderd. Met name het praktijkgerichte karakter van de Lesson Study en de samenwerking tussen studenten worden als positief ervaren door studenten en opleiders. Wanneer een aantal kleine aanpassingen wordt doorgevoerd ten aanzien van de ondersteuning, planning en informatieoverdracht en de vorm en eisen die gesteld worden aan het eindproduct goed worden (her)overwogen, kan deze vorm van afstuderen met succes worden geïmplementeerd.

Published
2021

6. 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

7. Intravenous immunoglobulin treatment for mild Guillain-Barré syndrome. An international observational study

Author

Verboon, C., Harbo, T., Cornblath, D. R., Hughes, R. A. C., Van Doorn, P. A., Lunn, M. P., Gorson, K. C., Barroso, F., Kuwabara, S., Galassi, G., Lehmann, H. C., Kusunoki, S., Reisin, R. C., Binda, D., Cavaletti, G., Andersen, Jacobs B. C. H., PhD (Aarhus University Hospital, Aarhus, Denmark), Attarian, S., PhD (CHU Timone, Marseille, France), Badrising, U. A., PhD (Leiden University Medical Centre, Leiden, The, Netherlands), Bateman, K., PhD (Groote Schuur Hospital, Cape, Town, South-Africa), Benedetti, L., PhD (Ospedale Sant’ Andrea La Spezia, Spezia, La, Italy), van den Berg, B., MD (Franciscus Gasthuis, Rotterdam, Van den Bergh, P., Luc, PhD (University Clinic St., Leuven, Belgium), Bertorini, T. E., MD (The University of Tennessee Health Science Center (UTHSC), Memphis, USA), Bhavaraju-Sanka, R., MD (University Hospital/ University of Texas Health Science Center, San Antonio Texas, USA), Bianco (Milan University, M., Humanitas Clinicala and Research Institute Milan, Briani, C., MD (University of Padova, Padova, Italy), Bürmann, J., MD (Universitätsklinikum des Saarlandes, Homburg, Germany), Casasnovas, C., Ciberer, PhD (Bellvitge University Hospital - IDIBELL Neurometabolic Diseases Group., Barcelona, Spain), Chao, C. C., PhD (National Taiwan University Hospital, Taipei, Taiwan), Chavada, G., PhD (Glasgow University, Glasgow, UK), Claeys, K. G., University Hospitals Leuven, PhD (1., Leuven, Belgium, KU Leuven, 2., Cosgrove, J. S., MD (Leeds General Infirmary, Leeds, UK), Dalakas, M. C., Thomas Jefferson University, MD (1., Philadelphia, Usa, National and Kapodistrian University of Athens, 2., Athens, Greece), Davidson, A., MD (University of Glasgow, van Dijk, G. W., MD (Canisius Wilhelmina Hospital, Nijmegen, Dardiotis, E., MD (University of Thessaly, Hospital of Larissa, Larissa, Greece), Derejko, M., MD (Odense University Hospital, Odense, Denmark), Dimachkie, M. M., MD (University of Kansas Medical Center, Kansas, City, Dornonville de la Cour, C., MD (National Hospital Copenhagen, Copenhagen, Denmark), Echaniz-Laguna, A., MD (Bicêtre University Hospital, Paris, France), Eftimov, F., PhD (Amsterdam University Medical Centre, Amsterdam, Faber, C. G., PhD (Maastricht University Medical Centre, Maastricht, Fazio, R., MD (Scientific Institute San Raffaele, Milan, Italy), Fulgenzi, J. Fehmi (University of Oxford E. A., MD (Hospital Cesar Milstein Buenos Aires, Buenos, Aires, Argentina), García-Sobrino, T., MD (Hospital Clínico de Santiago, Santiago de Compostela (A Coruña), Spain), Gijsbers, C. J., MD (Vlietland Hospital, Schiedam, Granit, V., MD (Montefiore Medical, Center, New, York, Grisanti, S., MD (Ospedale Sant’ Andrea La Spezia, Gutiérrez-Gutiérrez, G., MD (Hospital Universitario Infanta Sofia, San, Sebastian, Holbech, J. V., PhD (Odense University Hospital, Holt, J. K. L., Phd, FRCP (The Walton Centre, Liverpool, UK), Homedes, C., Ciberer, MD (Bellvitge University Hospital - IDIBELL Neurometabolic Diseases Group., Islam, B., PhD (International Centre for Diarrhoeal Disease Research, Bangladesh, (icddr, Dhaka, b), Bangladesh), Islam, Z., Jahan, I., PhD candidate (International Centre for Diarrhoeal Disease Research, Jericó Pascual, I., PhD (Complejo Hospitalario de Navarra, Pamplona, Spain), Karafiath, S., MD (University of Utah School of Medicine, Salt Lake City, Kerkhoff, H., PhD (Albert Schweitzer Hospital, Dordrecht, Kimpinski, K., MD (University Hospital, Lhsc, London-Ontario, Canada), Kohler, A., MD (Instituto de Investigaciones Neurológicas Raúl Carrea, Fleni, Kolb, N., MD (University of Vermont, Burlington, Vt, Kuitwaard, K., Albert Schweitzer Hospital, PhD (1., Erasmus MC, 2., Kuwahara, M., PhD (Kindai University, Osaka, Japan), Ladha, S. S., MD (Barrow Neurology Clinics, Phoenix, Arizona, Lee Pan, E., MBChB (Groote Schuur Hospital, Marfia, G. A., MD (Neurological Clinic, Policlinico Tor Vergata, Rome, Italy), Magot, A., MD (Reference Centre for NMD, Nantes University Hospital, France), Márquez Infante, C., MD (Hospital Universitario Virgen del Rocio, Seville, Spain), Martín-Aguilar, L., MD (Hospital de la Santa Creu, i Sant Pau, Universitat Autònoma de Barcelona, Martinez Hernandez, E., MD (Institut d’Investigacions Biomèdiques August Pi, i Sunyer (IDIBAPS), Hospital, Clinic, Mataluni, G., PhD (Neurological Clinic, Meekins, G., MD (University of Minnesota, Miller, J. A. L., PhD (Royal Victoria Infirmary, Newcastle, UK), Monges, M. S., Garrahan, MD (Hospital de Pediatría J. P., Nobile Orazio, E., PhD (Milan University, Pardal, A., MD (Hospital Britanico, Pardo Fernandez (Hospital Clínico de Santiago, J., Péréon, Y., PhD (Reference Centre for NMD, Pulley, M., MD (University of Florida, Jacksonville, USA), Querol Gutierrez, L., PhD (Hospital de la Santa Creu, i Sant Pau, Reddel, S. W., PhD (Concord Repatriation General Hospital, Sydney, Australia), van der Ree, T., (Westfriesgasthuis, Md, Hoorn, Rinaldi, S., Mbchb, Samijn, PhD (University of Oxford J. P. A., MD (Maasstad Hospital, Samukawa, M., Santoro, L., PhD (University Federico II, Napels, Italy), Savransky, A., Garrahan, PhD (Hospital de Pediatría J. P., Schwindling, L., Sedano Tous, M. J., MD (Hospital Universitario Marques de Valdecilla, Santander, Cantabria, Sekiguchi, Y., PhD (Chiba University, Chiba, Japan), Shahrizaila, N., MD (Neurology Unit, Department of Medicine, Faculty of Medicine, University of Malaya, Malaya), Silvestri, N. J., Sindrup, MD (Buffalo Jacobs School of Medicine S., Sommer, C. L., MD (Universitätsklinikum Würzburg, Würzburg, Germany), Spyropoulos (Royal Victoria Infirmary, A., Stein, B., Joseph’s Regional Medical Center, MD (St., Paterson, USA), Tan, C. Y., MRCP (Neurology Unit, Tankisi, H., Vermeij, F., Vytopil, M. V., Wirtz, PhD (Tufts University School of Medicine Lahey Hospital P. W., Phd, (HagaZiekenhuis, The, Hague, Waheed, W., MD (University of Vermont Medical Center, Burlington, Addington, USA). Other collaborators were:J. M., MD (University of Virginia, Charlottesville, USA), Ajroud-Driss, S., MD (Northwestern University Feinberg, Chicago, USA), Antonini, G., MD (Mental Health and Sensory Organs (NESMOS), Sapienza, University, Sant’Andrea, Hospital, Bella, I. R., MD (University of Mass Medical School, Worcester, USA), Brannagan, T. H., MD (Columbia University, New York City, Bunschoten, C., PhD candidate (Erasmus University Medical Centre, Busby, M., Bradford, UK), Butterworth, S., MD (Pinderfields Hospital, Wakefield, UK), Conti, M. E., MD (University Hospital Clinicas, Chen, S., Phd, (Rutgers, Robert Wood Johnson University Hospital, New, Brunswick, Doets, A., Feasby, T. E., MD (University of Calgary, Calgary, Canada), Fokke, C., MD (Gelre Hospital, Zutphen and Apeldoorn, Fujioka, T., MD (Toho University Medical Center, Tokyo, Japan), Garssen, M. P. J., PhD (Jeroen Bosch Hospital, Hertogenbosch, ’S, Gilchrist, J. M., MD (Soulthern Illinois University School of Medicine, Springfield, USA), Gilhuis, J., PhD (Reinier de Graaf Gasthuis, Delft, Goldstein, J. M., MD (Yale University School of Medicine, New, Haven, Goyal, N. A., MD (University of California, Irvine, USA), Hadden, R. D. M., PhD (King’s College Hospital, London, UK), Hsieh, S. T., Htut, M., George’s Hospital, MD (St., Illa, I., Jellema, K., PhD (Haaglanden Medisch Centrum, Kaida, K., PhD (National Defense Medical College, Saitama, Japan), Katzberg, H. D., MD (University of Toronto, Toronto, Canada), Kiers, L., MD (University of Melbourne, Royal Melbourne Hospital, Parkville, Australia), Kokubun, N., MD (Dokkyo Medical University, Tochigi, Japan), van Koningsveld, R., PhD (Elkerliek Hospital, Helmond and Deurne, van der Kooi, A. J., Kwan, J. Y., MD (University of Maryland School of Medicine, Baltimore, USA), Landschoff Lassen, L., MD (Glostrup Hospital, Glostrup, Denmark), Lawson, V., MD (Wexner Medical Center at The Ohio State University, Columbus, USA), Leonhard, S. E., Mandarakas, M., PhD (Erasmus University Medical Centre, Manji, H., FRCP (Ipswich Hospital, Ipswich, UK), Mattiazzi, M. G., MD (Hospital Militar Central, Mcdermott, C. J., MD (Royal Hallamshire Hospital, Nihr, Clinical, Sheffield, UK), Mohammad, Q. D., PhD (National Institute of Neurosciences and Hospital, Dhaka, Bangladesh), Morís de la Tassa, G., MD (Hospital UniversitarioCentral de Asturias, Asturias, Spain), Nascimbene, C., PhD (Luigi Sacco Hospital, Niks, E. H., Nowak, R. J., Osei-Bonsu, M., PhD (James Cook University Hospital, Middlesbrough, UK), Pascuzzi, R. M., MD (University of Indiana School of Medicine, Indianapolis, USA), Roberts, R. C., MD (Addenbrooke’s Hospital Cambridge, Cambridge, UK), Rojas-Marcos, I., MD (Hospital Univesitario Reina Sofia, Cordoba, Spain), Roodbol, J., Rudnicki, S. A., MD (University of Arkansas, Fayetteville, USA), Sachs, G. M., MD (University of Rhode Island, Providence, USA), Schenone, A., Department of Neurosciences, PhD (1., Rehabilitation, Ophthalmology, Genetics and Maternal and Infantile Sciences (DINOGMI), University of Genova, Genova, IRCCS Policlinico San Martino, Italy 2., Genova, Italy), Sheikh, K., PhD (The University of Texas Health Science Center at Houston, Houston, USA), Twydell, P., DO (Spectrum Health System, Grand, Rapids, Van Damme, P., PhD (University Hospital Leuven, Varrato, J. D., DO (Lehigh Valley Health Network, Allentown, USA), Visser, L. H., PhD (Elisabeth-TweeSteden Hospital, Tilburg and Waalwijk, Willison, H. J., PhD (University of Glasgow, van Woerkom (Erasmus MC, M., Zhou, L., PhD (Icahn School, Verboon, C, Harbo, T, Cornblath, D, Hughes, R, Van Doorn, P, Lunn, M, Gorson, K, Barroso, F, Kuwabara, S, Galassi, G, Lehmann, H, Kusunoki, S, Reisin, R, Binda, D, Cavaletti, G, Jacobs, B, consortium, IGOS, consortium, GOS, Neurosurgery, Neurology, and Immunology

Subjects
Adult, Male, medicine.medical_specialty, intravenous immunoglobulins, DIAGNOSIS, Guillain-Barre Syndrome, Settore MED/26, DISEASE, Disease course, Disability Evaluation, 03 medical and health sciences, 0302 clinical medicine, SDG 3 - Good Health and Well-being, hemic and lymphatic diseases, Internal medicine, Clinical endpoint, medicine, Humans, In patient, guillain-barré syndrome, 030212 general & internal medicine, NEUROPATHIES, biology, Guillain-Barre syndrome, business.industry, Guillain-Barré syndrome (GBS), treatment, course, Confounding, Immunoglobulins, Intravenous, Middle Aged, medicine.disease, Confidence interval, Psychiatry and Mental health, Treatment Outcome, biology.protein, Female, Surgery, Observational study, Neurology (clinical), Antibody, business, 030217 neurology & neurosurgery
Abstract

ObjectiveTo compare the disease course in patients with mild Guillain-Barré syndrome (GBS) who were treated with intravenous immunoglobulin (IVIg) or supportive care only.MethodsWe selected patients from the prospective observational International GBS Outcome Study (IGOS) who were able to walk independently at study entry (mild GBS), treated with one IVIg course or supportive care. The primary endpoint was the GBS disability score four weeks after study entry, assessed by multivariable ordinal regression analysis.ResultsOf 188 eligible patients, 148 (79%) were treated with IVIg and 40 (21%) with supportive care. The IVIg group was more disabled at baseline. IVIg treatment was not associated with lower GBS disability scores at 4 weeks (adjusted OR (aOR) 1.62, 95% CI 0.63 to 4.13). Nearly all secondary endpoints showed no benefit from IVIg, although the time to regain full muscle strength was shorter (28 vs 56 days, p=0.03) and reported pain at 26 weeks was lower (n=26/121, 22% vs n=12/30, 40%, p=0.04) in the IVIg treated patients. In the subanalysis with persistent mild GBS in the first 2 weeks, the aOR for a lower GBS disability score at 4 weeks was 2.32 (95% CI 0.76 to 7.13). At 1 year, 40% of all patients had residual symptoms.ConclusionIn patients with mild GBS, one course of IVIg did not improve the overall disease course. The certainty of this conclusion is limited by confounding factors, selection bias and wide confidence limits. Residual symptoms were often present after one year, indicating the need for better treatments in mild GBS.

Published
2021

9. Oromandibular parafunction in chronic graft-versus-host disease: novel association and treatment approach

Author

Joshi, P, Kiers, L, Evans, A, Ritchie, D, Szer, J, Cruse, B, Joshi, P, Kiers, L, Evans, A, Ritchie, D, Szer, J, and Cruse, B

Abstract

Chronic graft-versus-host disease (cGVHD) complicating allogeneic haemopoietic stem cell transplantation rarely involves the nervous system; oromandibular parafunction has not been previously reported. We describe five patients with cGVHD, presenting with bruxism, limitation of mouth opening, jaw locking, pain and masseter hypertrophy. Pathophysiological mechanisms are discussed. Targeted botulinum toxin injections were an effective treatment with minimal side-effects.

Published
2021

10. International Guillain-Barré Syndrome Outcome Study

Author

Jacobs, B, van den Berg, B, Verboon, C, Chavada, G, Cornblath, D, Gorson, K, Harbo, T, Hartung, H, Hughes, R, Kusunoki, S, van Doorn, P, Willison, H, Consortium, I, van Woerkom, M, Roodbol, J, Reisin, R, Reddel, S, Islam, Z, Islam, B, Mohammad, Q, van den Bergh, P, Feasby, T, Wang, Y, Péréon, Y, Lehmann, H, Dardiotis, E, Nobile Orazio, E, Shahrizaila, N, Bateman, K, Illa, I, Querol, L, Hsieh, S, Davidson, A, Addington, J, Ajroud Driss, S, Andersen, H, Antonini, G, Attarian, S, Badrising, U, Barroso, F, Benedetti, L, Beronio, A, Bianco, M, Binda, D, Briani, C, Bürmann, J, Bella, I, Bertorini, T, Bhavaraju Sanka, R, Brannagan, T, Busby, M, Butterworth, S, Campagnolo, M, Casasnovas, C, Cavaletti, G, Chao, C, Chen, S, Chetty, S, Claeys, K, Cohen, J, Conti, M, Cosgrove, J, Dalakas, M, Dimachkie, M, Dillmann, U, Domínguez González, C, Doppler, K, Dornonville de la Cour, C, Echaniz Laguna, A, Eftimov, F, Faber, C, Fazio, R, Fokke, C, Fujioka, T, Fulgenzi, E, Galassi, G, Garcia, T, Garnero, M, Garssen, M, Gijsbers, C, Gilchrist, J, Gilhuis, H, Goldstein, J, Goyal, N, Granit, V, Grapperon, A, Gutiérrez Gutiérrez, G, Gutmann, L, Hadden, R, Holbech, J, Holt, J, Homedes Pedret, C, Htut, M, Jellema, K, Jericó Pascual, I, Kaida, K, Karafiath, S, Katzberg, H, Kiers, L, Kieseier, B, Kimpinski, K, Kleyweg, R, Kokubun, N, Kolb, N, Kuitwaard, K, Kuwabara, S, Kwan, J, Ladha, S, Landschoff Lassen, L, Lawson, V, Ledingham, D, Léon Cejas, L, Luciano, C, Lucy, S, Lunn, M, Magot, A, Manji, H, Marchesoni, C, Marfia, Ga, Márquez Infante, C, Martinez Hernandez, E, Mataluni, G, Mattiazi, M, Mcdermott, C, Meekins, G, Miller, J, Monges, M, Montero, M, Morís de la Tassa, G, Nascimbene, C, Neumann, C, Nowak, R, Orizaola Balaguer, P, Osei Bonsu, M, Pan, E, Pardo Fernandez, J, Pasnoor, M, Pulley, M, Rajabally, Y, Rinaldi, S, Ritter, C, Roberts, R, Rojas Marcos, I, Rudnicki, S, Sachs, G, Samijn, J, Santoro, L, Saperstein, D, Savransky, A, Schneider, H, Schenone, A, Sedano Tous, M, Sekiguchi, Y, Sheikh, K, Silvestri, N, Sindrup, S, Sommer, C, Stein, B, Stino, A, Spyropoulos, A, Srinivasan, J, Suzuki, H, Taylor, S, Tankisi, H, Tigner, D, Twydell, P, Valzania, F, van Damme, P, van der Kooi, A, van Dijk, G, van der Ree, T, van Koningsveld, R, Varrato, J, Vermeij, F, Verschuuren, J, Visser, L, Vytopil, M, Waheed, W, Wilken, M, Wilkerson, C, Wirtz, P, Yamagishi, Y, Yiu, E, Zhou, L, Zivkovic, S, Immunology, Neurology, Jacobs, B, van den Berg, B, Verboon, C, Chavada, G, Cornblath, D, Gorson, K, Harbo, T, Hartung, H, Hughes, R, Kusunoki, S, van Doorn, P, Willison, H, van Woerkom, M, Roodbol, J, Reisin, R, Reddel, S, Islam, Z, Islam, B, Mohammad, Q, van den Bergh, P, Feasby, T, Wang, Y, Pã©rã©on, Y, Lehmann, H, Dardiotis, E, Nobile Orazio, E, Shahrizaila, N, Bateman, K, Illa, I, Querol, L, Hsieh, S, Davidson, A, Addington, J, Ajroud Driss, S, Andersen, H, Antonini, G, Attarian, S, Badrising, U, Barroso, F, Benedetti, L, Beronio, A, Bianco, M, Binda, D, Briani, C, Bã¼rmann, J, Bella, I, Bertorini, T, Bhavaraju Sanka, R, Brannagan, T, Busby, M, Butterworth, S, Campagnolo, M, Casasnovas, C, Cavaletti, G, Chao, C, Chen, S, Chetty, S, Claeys, K, Cohen, J, Conti, M, Cosgrove, J, Dalakas, M, Dimachkie, M, Dillmann, U, Domínguez González, C, Doppler, K, Dornonville de la Cour, C, Echaniz Laguna, A, Eftimov, F, Faber, C, Fazio, R, Fokke, C, Fujioka, T, Fulgenzi, E, Galassi, G, Garcia, T, Garnero, M, Garssen, M, Gijsbers, C, Gilchrist, J, Gilhuis, H, Goldstein, J, Goyal, N, Granit, V, Grapperon, A, Gutiérrez Gutiérrez, G, Gutmann, L, Hadden, R, Holbech, J, Holt, J, Homedes Pedret, C, Htut, M, Jellema, K, Jericó Pascual, I, Kaida, K, Karafiath, S, Katzberg, H, Kiers, L, Kieseier, B, Kimpinski, K, Kleyweg, R, Kokubun, N, Kolb, N, Kuitwaard, K, Kuwabara, S, Kwan, J, Ladha, S, Landschoff Lassen, L, Lawson, V, Ledingham, D, Léon Cejas, L, Luciano, C, Lucy, S, Lunn, M, Magot, A, Manji, H, Marchesoni, C, Marfia, G, Márquez Infante, C, Martinez Hernandez, E, Mataluni, G, Mattiazi, M, Mcdermott, C, Meekins, G, Miller, J, Monges, M, Montero, M, Morís de la Tassa, G, Nascimbene, C, Neumann, C, Nowak, R, Orizaola Balaguer, P, Osei Bonsu, M, Pan, E, Pardo Fernandez, J, Pasnoor, M, Pulley, M, Rajabally, Y, Rinaldi, S, Ritter, C, Roberts, R, Rojas Marcos, I, Rudnicki, S, Sachs, G, Samijn, J, Santoro, L, Saperstein, D, Savransky, A, Schneider, H, Schenone, A, Sedano Tous, M, Sekiguchi, Y, Sheikh, K, Silvestri, N, Sindrup, S, Sommer, C, Stein, B, Stino, A, Spyropoulos, A, Srinivasan, J, Suzuki, H, Taylor, S, Tankisi, H, Tigner, D, Twydell, P, Valzania, F, van Damme, P, van der Kooi, A, van Dijk, G, van der Ree, T, van Koningsveld, R, Varrato, J, Vermeij, F, Verschuuren, J, Visser, L, Vytopil, M, Waheed, W, Wilken, M, Wilkerson, C, Wirtz, P, Yamagishi, Y, Yiu, E, Zhou, L, Zivkovic, S, Rehabilitation medicine, Internal medicine, and ANS - Neuroinfection & -inflammation

Subjects
Male, Pediatrics, PROGNOSIS, diagnosis, International Cooperation, Guillain-Barré syndrome, biomarkers, outcome, prognosis, treatment, Guillain-Barre syndrome, Guillain-Barré syndrome, Neuroscience (all), Neurology (clinical), Cohort Studies, 0302 clinical medicine, Epidemiology, Outcome Assessment, Health Care, INFECTION, CRITERIA, 030212 general & internal medicine, General Neuroscience, Biobank, Observational Studies as Topic, diagnosi, Disease Progression, biomarker, Female, Settore MED/26 - Neurologia, medicine.symptom, prognosi, Cohort study, medicine.medical_specialty, Weakness, Guillain-Barre Syndrome, CLASSIFICATION, VALIDATION, 03 medical and health sciences, medicine, Humans, INTRAVENOUS IMMUNOGLOBULIN, Protocol (science), business.industry, Polyradiculoneuropathy, medicine.disease, ANTIBODIES, Observational study, business, COLLECTION, 030217 neurology & neurosurgery
Abstract

Guillain-Barré 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

11. Current treatment practice of Guillain-Barré syndrome

Author

Verboon C., Doets A. Y., Galassi G., Davidson A., Waheed W., Pereon Y., Shahrizaila N., Kusunoki S., Lehmann H. C., Harbo T., Monges S., Van Den Bergh P., Willison H. J., Cornblath D. R., Jacobs B. C., Hughes R. A. C., Gorson K. C., Hartung H. P., Van Doorn P. A., Van den Berg B., Roodbol J., Van Woerkom M., Reisin R. C., Reddel S. W., Islam Z., Islam B., Mohammad Q. D., Feasby T. E., Dardiotis E., Nobile-Orazio E., Bateman K., Illa I., Querol L., Hsieh S. T., Chavada G., Addington J. M., Ajroud-Driss S., Andersen H., Antonini G., Ariatti A., Attarian S., Badrising U. A., Barroso F. A., Benedetti L., Beronio A., Bianco M., Binda D., Briani C., Bunschoten C., Burmann J., Bella I. R., Bertorini T. E., Bhavaraju-Sanka R., Brannagan T. H., Busby M., Butterworth S., Casasnovas C., Cavaletti G., Chao C. C., Chen S., Chetty S., Claeys K. G., Conti M. E., Cosgrove J. S., Dalakas MC., Demichelis C., Derejko M. A., Dillmann U., Dimachkie M. M., Doppler K., Dornonville de la Cour C., Echaniz-Laguna A., Eftimov F., Faber C. G., Fazio R., Fokke C., Fujioka T., Fulgenzi E. A., Garcia-Sobrino T., Garssen M. P. J., Georgios H. M., Gijsbers C. J., Gilchrist J. M., Gilhuis J., Giorli E., Goldstein J. M., Goyal N. A., Granit V., Grapperon A., Gutierrez G., Hadden R. D. M., Holbech J. V., Holt J. K. L., Pedret C. H., Htut M., Jellema K., Pascual I. J., Jimeno-Montero M. C., Kaida K., Karafiath S., Katzberg H. D., Kiers L., Kieseier B. C., Kimpinski K., Kleyweg R. P., Kokubun N., Kolb N. A., Kuitwaard K., Kuwabara S., Kwan J. Y., Ladha S. S., Lassen L. L., Lawson V., Ledingham D., Lucy S. T., Lunn M. P. T., Magot A., Manji H., Marchesoni C., Marfia G. A., Infante C. M., Hernandez E. M., Mataluni G., Mattiazi M., McDermott C. J., Meekins G. D., Miller J. A. L., Moris de la Tassa G., Physiotherapist J. M., Nascimbene C., Nowak R. J., Balaguer P. O., Osei-Bonsu M., Pan E. B. L., Pardal A. M., Pardo J., Pasnoor M., Pulley M., Rajabally Y. A., Rinaldi S., Ritter C., Roberts R. C., Rojas-Marcos I., Rudnicki S. A., Ruiz M., Sachs G. M., Samijn J. P. A., Santoro L., Savransky A., Schenone A., Schwindling L., Tous M. J. S., Sekiguchi Y., Sheikh K. A., Silvestri N. J., Sindrup S. H., Sommer C. L., Stein B., Stino A. M., Spyropoulos A., Srinivasan J., Styliani R., Suzuki H., Tankisi H., Tigner D., Twydell P., Van Damme P., Van der Kooi A. J., Van Dijk G. W., Van der Ree T., Van Koningsveld R., Valzania F., Varrato J. D., Vermeij F. H., Verschuuren J., Visser L. H., Vytopil M. V., Wilken M., Wilkerson C., Wirtz P. W., Yamagishi Y., Zhou L., Zivkovic S. A., Neurology, AII - Infectious diseases, AII - Inflammatory diseases, ANS - Neuroinfection & -inflammation, Immunology, Erasmus MC other, UCL - SSS/IONS/NEUR - Clinical Neuroscience, UCL - (SLuc) Service de neurologie, Verboon, C, Doets, A, Galassi, G, Davidson, A, Waheed, W, Pereon, Y, Shahrizaila, N, Kusunoki, S, Lehmann, H, Harbo, T, Monges, S, Van Den Bergh, P, Willison, H, Cornblath, D, Jacobs, B, Hughes, R, Gorson, K, Hartung, H, Van Doorn, P, Van den Berg, B, Roodbol, J, Van Woerkom, M, Reisin, R, Reddel, S, Islam, Z, Islam, B, Mohammad, Q, Feasby, T, Dardiotis, E, Nobile-Orazio, E, Bateman, K, Illa, I, Querol, L, Hsieh, S, Chavada, G, Addington, J, Ajroud-Driss, S, Andersen, H, Antonini, G, Ariatti, A, Attarian, S, Badrising, U, Barroso, F, Benedetti, L, Beronio, A, Bianco, M, Binda, D, Briani, C, Bunschoten, C, Burmann, J, Bella, I, Bertorini, T, Bhavaraju-Sanka, R, Brannagan, T, Busby, M, Butterworth, S, Casasnovas, C, Cavaletti, G, Chao, C, Chen, S, Chetty, S, Claeys, K, Conti, M, Cosgrove, J, Dalakas, M, Demichelis, C, Derejko, M, Dillmann, U, Dimachkie, M, Doppler, K, Dornonville de la Cour, C, Echaniz-Laguna, A, Eftimov, F, Faber, C, Fazio, R, Fokke, C, Fujioka, T, Fulgenzi, E, Garcia-Sobrino, T, Garssen, M, Georgios, H, Gijsbers, C, Gilchrist, J, Gilhuis, J, Giorli, E, Goldstein, J, Goyal, N, Granit, V, Grapperon, A, Gutierrez, G, Hadden, R, Holbech, J, Holt, J, Pedret, C, Htut, M, Jellema, K, Pascual, I, Jimeno-Montero, M, Kaida, K, Karafiath, S, Katzberg, H, Kiers, L, Kieseier, B, Kimpinski, K, Kleyweg, R, Kokubun, N, Kolb, N, Kuitwaard, K, Kuwabara, S, Kwan, J, Ladha, S, Lassen, L, Lawson, V, Ledingham, D, Lucy, S, Lunn, M, Magot, A, Manji, H, Marchesoni, C, Marfia, G, Infante, C, Hernandez, E, Mataluni, G, Mattiazi, M, Mcdermott, C, Meekins, G, Miller, J, Moris de la Tassa, G, Physiotherapist, J, Nascimbene, C, Nowak, R, Balaguer, P, Osei-Bonsu, M, Pan, E, Pardal, A, Pardo, J, Pasnoor, M, Pulley, M, Rajabally, Y, Rinaldi, S, Ritter, C, Roberts, R, Rojas-Marcos, I, Rudnicki, S, Ruiz, M, Sachs, G, Samijn, J, Santoro, L, Savransky, A, Schenone, A, Schwindling, L, Tous, M, Sekiguchi, Y, Sheikh, K, Silvestri, N, Sindrup, S, Sommer, C, Stein, B, Stino, A, Spyropoulos, A, Srinivasan, J, Styliani, R, Suzuki, H, Tankisi, H, Tigner, D, Twydell, P, Van Damme, P, Van der Kooi, A, Van Dijk, G, Van der Ree, T, Van Koningsveld, R, Valzania, F, Varrato, J, Vermeij, F, Verschuuren, J, Visser, L, Vytopil, M, Wilken, M, Wilkerson, C, Wirtz, P, Yamagishi, Y, Zhou, L, and Zivkovic, S

Subjects
Adult, medicine.medical_specialty, Adolescent, Patient characteristics, PLASMA-EXCHANGE, 030204 cardiovascular system & hematology, Guillain-Barre Syndrome, Settore MED/26, Severity of Illness Index, 03 medical and health sciences, 0302 clinical medicine, Internal medicine, Severity of illness, Medicine, Effective treatment, INTRAVENOUS IMMUNOGLOBULIN, Humans, Prospective Studies, Child, Child, Preschool, Treatment Outcome, Prospective cohort study, Preschool, Guillain-Barre syndrome, business.industry, medicine.disease, RANDOMIZED-TRIAL, Prospective Studie, Hospital treatment, Treatment practice, Observational study, Neurology (clinical), business, 030217 neurology & neurosurgery, Human
Abstract

ObjectiveTo define the current treatment practice of Guillain-Barré syndrome (GBS).MethodsThe study was based on prospective observational data from the first 1,300 patients included in the International GBS Outcome Study. We described the treatment practice of GBS in general, and for (1) severe forms (unable to walk independently), (2) no recovery after initial treatment, (3) treatment-related fluctuations, (4) mild forms (able to walk independently), and (5) variant forms including Miller Fisher syndrome, taking patient characteristics and hospital type into account.ResultsWe excluded 88 (7%) patients because of missing data, protocol violation, or alternative diagnosis. Patients from Bangladesh (n = 189, 15%) were described separately because 83% were not treated. IV immunoglobulin (IVIg), plasma exchange (PE), or other immunotherapy was provided in 941 (92%) of the remaining 1,023 patients, including patients with severe GBS (724/743, 97%), mild GBS (126/168, 75%), Miller Fisher syndrome (53/70, 76%), and other variants (33/40, 83%). Of 235 (32%) patients who did not improve after their initial treatment, 82 (35%) received a second immune modulatory treatment. A treatment-related fluctuation was observed in 53 (5%) of 1,023 patients, of whom 36 (68%) were re-treated with IVIg or PE.ConclusionsIn current practice, patients with mild and variant forms of GBS, or with treatment-related fluctuations and treatment failures, are frequently treated, even in absence of trial data to support this choice. The variability in treatment practice can be explained in part by the lack of evidence and guidelines for effective treatment in these situations.

Published
2019

12. Second IVIg course in Guillain-Barré syndrome with poor prognosis. The non-randomised ISID study

Author

Verboon, C., Van Den Berg, B., Cornblath, D. R., Venema, E., Gorson, K. C., Lunn, M. P., Lingsma, H., Van Den Bergh, P., Harbo, T., Bateman, K., Pereon, Y., Sindrup, So. H., Kusunoki, S., Miller, J., Islam, Z., Hartung, H. -P., Chavada, G., Jacobs, B. C., Hughes, R. A. C., Addington, Van Doorn P. A. J. M., MD (University of Virginia, Charlottesville, USA), on October 7, S. Consortia. Protected by copyright., Downloaded from 8 Verboon C, 2019 at Uppsala Universitet BIBSAM http://jnnp. bmj. com/ J Neurol Neurosurg Psychiatry: first published as 10. 1136/jnnp-2019-321496 on 5 October 2019., J Neurol Neurosurg Psychiatry 2019, et al., 1136/jnnp-2019-321496 Neuromuscular Ajroud-Driss, 0:1–9. doi:10., MD (Northwestern University Feinberg, Chicago, USA), Antonini, G., MD (Mental Health and Sensory Organs (NESMOS), Sapienza, University, Sant’Andrea, Hospital, Rome, Italy), Attarian, S., PhD (CHU Timone, Marseille, France), Barroso, F. A., MD (Instituto de Investigaciones Neurológicas Raúl Carrea, Fleni, Buenos, Aires, Argentina), Benedetti, L., PhD (Ospedale Sant’ Andrea La Spezia, Spezia, La, Italy), Bertorini, T. E., MD (The University of Tennessee Health Science Center (UTHSC), Memphis, USA), Brannagan, T. H., MD (Columbia University, New York City, USA), Briani, C., MD (University of Padova, Padova, Italy), Bhavaraju-Sanka, R., MD (University Hospital/University of Texas Health Science Center, San Antonio Texas, Butterworth, S., MD (Pinderfields Hospital, Wakefield, UK), Casasnovas, C., Ciberer, PhD (Bellvitge University Hospital – IDIBELL Neurometabolic Diseases Group., Barcelona, Spain), Cavaletti, G., MD (University Milano-Bicocca, Monza, Italy), Chen, S., Phd, (Rutgers, Robert Wood Johnson University Hospital, New, Brunswick, Claeys, K. G., University Hospitals Leuven, PhD (1., Leuven, Belgium, KU Leuven, 2., Leuven, Belgium), Cosgrove, J. S., MD (Leeds General Infirmary, Leeds, UK), Davidson, A., MD (University of Glasgow, Glasgow, UK), Dardiotis, E., MD (University of Thessaly, Hospital of Larissa, Larissa, Greece), Dornonville de la Cour, C., MD (National Hospital Copenhagen, Copenhagen, Denmark), Faber, C. G., PhD (Maastricht University Medical Centre, Maastricht, The, Netherlands), Feasby, T. E., MD (University of Calgary, Calgary, Canada), Fujioka, T., MD (Toho University Medical Center, Tokyo, Japan), Galassi, G., MD (University Hospital of Modena, Modena, Italy), Gilchrist, J. M., MD (Soulthern Illinois University School of Medicine, Springfield, USA), Goyal, N. A., MD (University of California, Irvine, USA), Granit, V., MD (Montefiore Medical, Center, New, York, Gutiérrez-Gutiérrez, G., MD (Hospital Universitario Infanta Sofia, San, Sebastian, Spain), Hadden, R. D. M., PhD (King’s College Hospital, London, UK), Holt, J. K. L., Phd, FRCP (The Walton Centre, Liverpool, UK), Htut, M., George’s Hospital, MD (St., Jericó Pascual, I., PhD (Complejo Hospitalario de Navarra, Pamplona, Spain), Karafiath, S., MD (University of Utah School of Medicine, Salt Lake City, Katzberg, H. D., MD (University of Toronto, Toronto, Canada), Kiers, L., MD (University of Melbourne, Royal Melbourne Hospital, Parkville, Australia), Kieseier, B. C., MD (Heinrich Heine University, Düsseldorf, Germany), Kimpinski, K., MD (University Hospital, Lhsc, London-Ontario, Canada), Kuwabara, S., PhD (Chiba University, Chiba, Japan), Kwan, J. Y., MD (University of Maryland School of Medicine, Baltimore, USA), Ladha, S. S., MD (Barrow Neurology Clinics, Phoenix, Arizona, Lawson, V., MD (Wexner Medical Center at The Ohio State University, Columbus, USA), Lehmann, H., PhD (University Hospital of Cologne, Universitätsklinikum, Köln, Cologne, Germany), Manji, H., FRCP (Ipswich Hospital, Ipswich, UK), Marfia, G. A., MD (Neurological Clinic, Policlinico Tor Vergata, Márquez Infante, C., MD (Hospital Universitario Virgen del Rocio, Seville, Spain), Mattiazzi, M. G., MD (Hospital Militar Central, Mcdermott, C. J., MD (Royal Hallamshire Hospital, Nihr, Clinical, Sheffield, UK), Monges, M. S., Garrahan, MD (Hospital de Pediatría J. P., Morís de la Tassa, G., MD (Hospital Universitario Central de Asturias, Asturias, Spain), Nascimbene, C., PhD (Luigi Sacco Hospital, Milan, Italy), Nobile Orazio, E., PhD (Milan University, Humanitas Clinicala and Research Institute Milan, Nowak, R. J., MD (Yale University School of Medicine, New, Haven, Osei-Bonsu (James Cook University Hospital, M., Middlesbrough, UK), Pardo Fernandez (Hospital Clínico de Santiago, J., Santiago de Compostela (A Coruña), Querol Gutierrez, L., PhD (Hospital de la Santa Creu, i Sant Pau, Universitat Autònoma de Barcelona, Reisin (Hospital Britanico, R., Rinaldi, S., Mbchb, Roberts, PhD (University of Oxford R. C., MD (Addenbrooke’s Hospital Cambridge, Cambridge, UK), Rojas-Marcos, I., MD (Hospital Univesitario Reina Sofia, Cordoba, Spain), Rudnicki, S. A., MD (University of Arkansas, Fayetteville, USA), Schenone, A., Department of Neurosciences, PhD (1., Rehabilitation, Ophthalmology, Genetics and Maternal and Infantile Sciences (DINOGMI), University of Genova, Genova, IRCCS Policlinico San Martino, Italy 2., Genova, Italy), Sedano Tous, M. J., MD (Hospital Universitario Marques de Valdecilla, Santander, Cantabria, Shahrizaila, N., MD (Neurology Unit, Department of Medicine, Faculty of Medicine, University of Malaya, Malaya), Sheikh, K., PhD (The University of Texas Health Science Center at Houston, Houston, USA), Silvestri, N. J., MD (Buffalo General Medical Center, Buffalo, Ny, Sommer, C. L., MD (Universitätsklinikum Würzburg, Würzburg, Germany), Varrato, J. D., DO (Lehigh Valley Health Network, Allentown, USA), Verschuuren, J., PhD (Leiden University Medical Centre, Leiden, Vytopil, M. V., Waheed, PhD (Tufts University School of Medicine Lahey Hospital W., MD (University of Vermont Medical Center, Burlington, USA), Zhou, L., PhD (Icahn School of Medicine at Mount Sinai, Badrising, USA). Other collaborators were:U. A., Bella, I. R., MD (University of Mass Medical School, Worcester, USA), Bunschoten, C., PhD candidate (Erasmus University Medical Centre, Rotterdam, Bürmann, J., Universitätsklinikum des Saarlandes, Homburg, Germany), Busby, M., Bradford, UK), Chao, C. C., PhD (National Taiwan University Hospital, Taipei, Taiwan), Conti, M. E., MD (University Hospital Clinicas, Dalakas, M. C., Thomas Jefferson University, MD (1., Philadelphia, Usa, National and Kapodistrian University of Athens, 2., Athens, Greece), Van Damme, P., PhD (University Hospital Leuven, Doets, A., van Dijk, G. W., MD (Canisius Wilhelmina Hospital, Nijmegen, Dimachkie, M. M., MD (University of Kansas Medical Center, Kansas, City, Doppler, K., Echaniz-Laguna, A., MD (Hopital de Hautepierre, Strasbourgh, France), Eftimov, F., PhD (Amsterdam University Medical Centre, Amsterdam, Fazio, R., MD (Scientific Institute San Raffaele, Fokke, C., MD (Gelre Hospital, Zutphen and Apeldoorn, Fulgenzi, E. A., MD (Hospital Cesar Milstein Buenos Aires, Garssen, M. P. J., PhD (Jeroen Bosch Hospital, Hertogenbosch, ’S, Zaltbommel and Drunen, Gijsbers, C. J., MD (Vlietland Hospital, Schiedam, Gilhuis, J., PhD (Reinier de Graaf Gasthuis, Delft, Grapperon, A., MD (CHU Timone, Hsieh, S. T., Illa, I., Islam, B., PhD (International Centre for Diarrhoeal Disease Research, Bangladesh, (icddr, Dhaka, b), Bangladesh), Jellema, K., PhD (Haaglanden Medisch Centrum, The, Hague, Kaida, K., PhD (National Defense Medical College, Saitama, Japan), Kokubun, N., MD (Dokkyo Medical University, Tochigi, Japan), Kolb, N., MD (University of Vermont, Burlington, Vt, van Koningsveld, R., PhD (Elkerliek Hospital, Helmond and Deurne, van der Kooi, A. J., Kuitwaard, K., PhD (Albert Schweitzer Hospital, Dordrecht, Landschoff Lassen, L., MD (Glostrup Hospital, Glostrup, Denmark), Leonhard, S. E., Mandarakas, M., PhD (Erasmus University Medical Centre, Martinez Hernandez, E., MD (Institut d’Investigacions Biomèdiques August Pi, i Sunyer (IDIBAPS), Hospital, Clinic, Mohammad, Q. D., PhD (National Institute of Neurosciences and Hospital, Dhaka, Bangladesh), Pulley, M., MD (University of Florida, Jacksonville, USA), Rajabally, Y. A., PhD (Queen Elizabeth Hospital, Birmingham, UK), Reddel, S. W., PhD (Concord Repatriation General Hospital, Sydney, Australia), van der Ree, T., (Westfriesgasthuis, Md, Hoorn, Roodbol, J., Sachs, G. M., MD (University of Rhode Island, Providence, USA), Samijn, J. P. A., PhD (Maasstad Hospital, Santoro, L., PhD (University Federico II, Napels, Italy), Stein, B., Joseph’s Regional Medical Center, MD (St., Paterson, USA), Vermeij, F. H., MD (Franciscus Gasthuis, Visser, L. H., PhD (Elisabeth-TweeSteden Hospital, Tilburg and Waalwijk, Willison, H. J., PhD (University of Glasgow, Wirtz, P., Phd, (HagaZiekenhuis, Zivkovich, S. A., PhD (University of Pittsburgh Medical Center, and Pittsburgh, USA).

Subjects
treatment, disability evaluation, drug administration schedule, adult, guillain-barré syndrome, poor prognosis, second ivig course, aged, female, guillain-barre syndrome, humans, immunoglobulin g, immunoglobulins, intravenous, immunologic factors, male, middle aged, prognosis, time factors, treatment outcome
Published
2020

13. Neurology of Fabry disease

Author

Low, M., Nicholls, K., Tubridy, N., Hand, P., Velakoulis, D., Kiers, L., Mitchell, P., and Becker, G.

Published
2007

15. Predicting Outcome in Guillain-Barré Syndrome

Author

Doets, Alex Y., Lingsma, Hester F., Walgaard, Christa, Islam, Badrul, Papri, Nowshin, Davidson, Amy, Yamagishi, Yuko, Kusunoki, Susumu, Dimachkie, Mazen M., Waheed, Waqar, Kolb, Noah, Islam, Zhahirul, Mohammad, Quazi Deen, Harbo, Thomas, Sindrup, Soren H., Chavada, Govindsinh, Willison, Hugh J., Casasnovas, Carlos, Bateman, Kathleen, Miller, James A.L., van den Berg, Bianca, Verboon, Christine, Roodbol, Joyce, Leonhard, Sonja E., Benedetti, Luana, Kuwabara, Satoshi, Van den Bergh, Peter, Monges, Soledad, Marfia, Girolama A., Shahrizaila, Nortina, Galassi, Giuliana, Péréon, Yann, Bürmann, Jan, Kuitwaard, Krista, Kleyweg, Ruud P., Marchesoni, Cintia, Sedano Tous, María J., Querol, Luis, Illa, Isabel, Wang, Yuzhong, Nobile-Orazio, Eduardo, Rinaldi, Simon, Schenone, Angelo, Pardo, Julio, Vermeij, Frederique H., Lehmann, Helmar C., Granit, Volkan, Cavaletti, Guido, Gutiérrez-Gutiérrez, Gerardo, Barroso, Fabio A., Visser, Leo H., Katzberg, Hans D., Dardiotis, Efthimios, Attarian, Shahram, van der Kooi, Anneke J., Eftimov, Filip, Wirtz, Paul W., Samijn, Johnny P.A., Gilhuis, H. Jacobus, Hadden, Robert D.M., Holt, James K.L., Sheikh, Kazim A., Karafiath, Summer, Vytopil, Michal, Antonini, Giovanni, Feasby, Thomas E., Faber, Catharina G., Gijsbers, Cees J., Busby, Mark, Roberts, Rhys C., Silvestri, Nicholas J., Fazio, Raffaella, van Dijk, Gert W., Garssen, Marcel P.J., Straathof, Chiara S.M., Gorson, Kenneth C., Jacobs, Bart C., Hughes, R.A.C., Cornblath, D.R., Hartung, H.P., van Doorn, P.A., de Koning, L.C., van Woerkom, M., Mandarakas, M., MPhty, BHIthSci(Hons), Reisin, R.C., Reddel, S.W., Ripellino, P., Hsieh, S.T., Addington, J.M., Ajroud-Driss, S., Andersen, H., Badrising, U.A., Bella, I.R., Bertorini, T.E., Bhavaraju-Sanka, R., Bianco, M., Brannagan, T.H., Briani, Chiara, Butterworth, S., Chao, C.C., Chen, S., Claeys, K.G., Conti, M.E., Cosgrove, J.S., Dalakas, M.C., Dornonville de la Cour, C., Echaniz-Laguna, A., Fehmi, J., Fokke, C., Fujioka, T., Fulgenzi, E.A., García-Sobrino, T., Gilchrist, J.M., Goldstein, J.M., Goyal, N.A., Grisanti, S.G., Gutman, L., Holbech, J.V., Homedes, C., Htut, M., Jellema, K., Pascual, I. Jericó, JimenoMontero, M.C., Kaida, K., Khoshnoodi, M., Kiers, L., Kimpinski, K., Köhler, A.A., Kokubun, N., Kuwahara, M., Kwan, J.Y., Ladha, S.S., Lassen, L. Landschoff, Lawson, V., Pan, E.B. Lee, Cejas, L. Léon, Lunn, M.P.T., Magot, A., Manji, H., Infante, C. Márquez, Martín-Aguilar, L., Hernandez, E. Martinez, Mataluni, G., Mattiazzi, M.G., McDermott, C.J., Meekins, G.D., Morís de la Tassa, G., Nascimbene, C., Nowak, R.J., Osei-Bonsu, M., Pascuzzi, R.M., Prada, V., Rojas-Marcos, I., Rudnicki, S.A., Sachs, G.M., Samukawa, M., Santoro, L., Savransky, A.G., Schwindling, L., Sekiguchi, Y., Sommer, C.L., Spyropoulos, A., Stein, B., Stino, A.M., Tan, C.Y., Tankisi, H., Twydell, P.T., van Damme, P., van der Ree, T., van Koningsveld, R., Varrato, J.D., Xing, C., Zhou, L., and Zivkovic, S.

Published
2022
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17. 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

18. Diagnostic and cost utility of whole exome sequencing in peripheral neuropathy

Author

Walsh, M, Bell, KM, Chong, B, Creed, E, Brett, GR, Pope, K, Thorne, NP, Sadedin, S, Georgeson, P, Phelan, DG, Day, T, Taylor, JA, Sexton, A, Lockhart, PJ, Kiers, L, Fahey, M, Macciocca, I, Gaff, CL, Oshlack, A, Yiu, EM, James, PA, Stark, Z, Ryan, MM, and Melbourne Genomics Health Alliance

Subjects
1103 Clinical Sciences, 1109 Neurosciences
Abstract

OBJECTIVE: To explore the diagnostic utility and cost effectiveness of whole exome sequencing (WES) in a cohort of individuals with peripheral neuropathy. METHODS: Singleton WES was performed in individuals recruited though one pediatric and one adult tertiary center between February 2014 and December 2015. Initial analysis was restricted to a virtual panel of 55 genes associated with peripheral neuropathies. Patients with uninformative results underwent expanded analysis of the WES data. Data on the cost of prior investigations and assessments performed for diagnostic purposes in each patient was collected. RESULTS: Fifty patients with a peripheral neuropathy were recruited (median age 18 years; range 2-68 years). The median time from initial presentation to study enrollment was 6 years 9 months (range 2 months-62 years), and the average cost of prior investigations and assessments for diagnostic purposes AU$4013 per patient. Eleven individuals received a diagnosis from the virtual panel. Eight individuals received a diagnosis following expanded analysis of the WES data, increasing the overall diagnostic yield to 38%. Two additional individuals were diagnosed with pathogenic copy number variants through SNP microarray. CONCLUSIONS: This study provides evidence that WES has a high diagnostic utility and is cost effective in patients with a peripheral neuropathy. Expanded analysis of WES data significantly improves the diagnostic yield in patients in whom a diagnosis is not found on the initial targeted analysis. This is primarily due to diagnosis of conditions caused by newly discovered genes and the resolution of complex and atypical phenotypes.

Published
2017

19. Multifocal Motor Neuropathy Presenting as Pseudodystonia

Author

Garg, N, Heard, RNS, Kiers, L, Gerraty, R, Yiannikas, C, Garg, N, Heard, RNS, Kiers, L, Gerraty, R, and Yiannikas, C

Abstract

Multifocal motor neuropathy (MMN) is an immune-mediated neuropathy. Wasting and weakness typically dominate the clinical presentation. We describe four cases presenting with prominent cramping resembling a primary movement disorder. All cases had features of focal motor conduction block on neurophysiological studies. The involuntary movements resolved in all four patients following treatment with intravenous immunoglobulin. The presented cases highlight an unusual presentation of MMN and emphasize that peripheral nerve pathology can present with movement disorders mimicking central nervous system disease. Furthermore, the movement disorder appears particularly sensitive to standard therapy.

Published
2017

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

Author

Jacobs, B, van den Berg, B, Verboon, C, Chavada, G, Cornblath, D, Gorson, K, Harbo, T, Hartung, H, Hughes, R, Kusunoki, S, van Doorn, P, Willison, H, van Woerkom, M, Roodbol, J, Reisin, R, Reddel, S, Islam, Z, Islam, B, Mohammad, Q, van den Bergh, P, Feasby, T, Wang, Y, Pã©rã©on, Y, Lehmann, H, Dardiotis, E, Nobile Orazio, E, Shahrizaila, N, Bateman, K, Illa, I, Querol, L, Hsieh, S, Davidson, A, Addington, J, Ajroud Driss, S, Andersen, H, Antonini, G, Attarian, S, Badrising, U, Barroso, F, Benedetti, L, Beronio, A, Bianco, M, Binda, D, Briani, C, Bã1⁄4rmann, J, Bella, I, Bertorini, T, Bhavaraju Sanka, R, Brannagan, T, Busby, M, Butterworth, S, Campagnolo, M, Casasnovas, C, Cavaletti, G, Chao, C, Chen, S, Chetty, S, Claeys, K, Cohen, J, Conti, M, Cosgrove, J, Dalakas, M, Dimachkie, M, Dillmann, U, Domínguez González, C, Doppler, K, Dornonville de la Cour, C, Echaniz Laguna, A, Eftimov, F, Faber, C, Fazio, R, Fokke, C, Fujioka, T, Fulgenzi, E, Galassi, G, Garcia, T, Garnero, M, Garssen, M, Gijsbers, C, Gilchrist, J, Gilhuis, H, Goldstein, J, Goyal, N, Granit, V, Grapperon, A, Gutiérrez Gutiérrez, G, Gutmann, L, Hadden, R, Holbech, J, Holt, J, Homedes Pedret, C, Htut, M, Jellema, K, JericÃ3 Pascual, I, Kaida, K, Karafiath, S, Katzberg, H, Kiers, L, Kieseier, B, Kimpinski, K, Kleyweg, R, Kokubun, N, Kolb, N, Kuitwaard, K, Kuwabara, S, Kwan, J, Ladha, S, Landschoff Lassen, L, Lawson, V, Ledingham, D, Léon Cejas, L, Luciano, C, Lucy, S, Lunn, M, Magot, A, Manji, H, Marchesoni, C, Marfia, G, Márquez Infante, C, Martinez Hernandez, E, Mataluni, G, Mattiazi, M, Mcdermott, C, Meekins, G, Miller, J, Monges, M, Montero, M, Morís de la Tassa, G, Nascimbene, C, Neumann, C, Nowak, R, Orizaola Balaguer, P, Osei Bonsu, M, Pan, E, Pardo Fernandez, J, Pasnoor, M, Pulley, M, Rajabally, Y, Rinaldi, S, Ritter, C, Roberts, R, Rojas Marcos, I, Rudnicki, S, Sachs, G, Samijn, J, Santoro, L, Saperstein, D, Savransky, A, Schneider, H, Schenone, A, Sedano Tous, M, Sekiguchi, Y, Sheikh, K, Silvestri, N, Sindrup, S, Sommer, C, Stein, B, Stino, A, Spyropoulos, A, Srinivasan, J, Suzuki, H, Taylor, S, Tankisi, H, Tigner, D, Twydell, P, Valzania, F, van Damme, P, van der Kooi, A, van Dijk, G, van der Ree, T, van Koningsveld, R, Varrato, J, Vermeij, F, Verschuuren, J, Visser, L, Vytopil, M, Waheed, W, Wilken, M, Wilkerson, C, Wirtz, P, Yamagishi, Y, Yiu, E, Zhou, L, Zivkovic, S, Zivkovic, S., BINDA, DAVIDE, CAVALETTI, GUIDO ANGELO, Jacobs, B, van den Berg, B, Verboon, C, Chavada, G, Cornblath, D, Gorson, K, Harbo, T, Hartung, H, Hughes, R, Kusunoki, S, van Doorn, P, Willison, H, van Woerkom, M, Roodbol, J, Reisin, R, Reddel, S, Islam, Z, Islam, B, Mohammad, Q, van den Bergh, P, Feasby, T, Wang, Y, Pã©rã©on, Y, Lehmann, H, Dardiotis, E, Nobile Orazio, E, Shahrizaila, N, Bateman, K, Illa, I, Querol, L, Hsieh, S, Davidson, A, Addington, J, Ajroud Driss, S, Andersen, H, Antonini, G, Attarian, S, Badrising, U, Barroso, F, Benedetti, L, Beronio, A, Bianco, M, Binda, D, Briani, C, Bã1⁄4rmann, J, Bella, I, Bertorini, T, Bhavaraju Sanka, R, Brannagan, T, Busby, M, Butterworth, S, Campagnolo, M, Casasnovas, C, Cavaletti, G, Chao, C, Chen, S, Chetty, S, Claeys, K, Cohen, J, Conti, M, Cosgrove, J, Dalakas, M, Dimachkie, M, Dillmann, U, Domínguez González, C, Doppler, K, Dornonville de la Cour, C, Echaniz Laguna, A, Eftimov, F, Faber, C, Fazio, R, Fokke, C, Fujioka, T, Fulgenzi, E, Galassi, G, Garcia, T, Garnero, M, Garssen, M, Gijsbers, C, Gilchrist, J, Gilhuis, H, Goldstein, J, Goyal, N, Granit, V, Grapperon, A, Gutiérrez Gutiérrez, G, Gutmann, L, Hadden, R, Holbech, J, Holt, J, Homedes Pedret, C, Htut, M, Jellema, K, JericÃ3 Pascual, I, Kaida, K, Karafiath, S, Katzberg, H, Kiers, L, Kieseier, B, Kimpinski, K, Kleyweg, R, Kokubun, N, Kolb, N, Kuitwaard, K, Kuwabara, S, Kwan, J, Ladha, S, Landschoff Lassen, L, Lawson, V, Ledingham, D, Léon Cejas, L, Luciano, C, Lucy, S, Lunn, M, Magot, A, Manji, H, Marchesoni, C, Marfia, G, Márquez Infante, C, Martinez Hernandez, E, Mataluni, G, Mattiazi, M, Mcdermott, C, Meekins, G, Miller, J, Monges, M, Montero, M, Morís de la Tassa, G, Nascimbene, C, Neumann, C, Nowak, R, Orizaola Balaguer, P, Osei Bonsu, M, Pan, E, Pardo Fernandez, J, Pasnoor, M, Pulley, M, Rajabally, Y, Rinaldi, S, Ritter, C, Roberts, R, Rojas Marcos, I, Rudnicki, S, Sachs, G, Samijn, J, Santoro, L, Saperstein, D, Savransky, A, Schneider, H, Schenone, A, Sedano Tous, M, Sekiguchi, Y, Sheikh, K, Silvestri, N, Sindrup, S, Sommer, C, Stein, B, Stino, A, Spyropoulos, A, Srinivasan, J, Suzuki, H, Taylor, S, Tankisi, H, Tigner, D, Twydell, P, Valzania, F, van Damme, P, van der Kooi, A, van Dijk, G, van der Ree, T, van Koningsveld, R, Varrato, J, Vermeij, F, Verschuuren, J, Visser, L, Vytopil, M, Waheed, W, Wilken, M, Wilkerson, C, Wirtz, P, Yamagishi, Y, Yiu, E, Zhou, L, Zivkovic, S, Zivkovic, S., BINDA, DAVIDE, and CAVALETTI, GUIDO ANGELO

Abstract

Guillain-Barré 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

21. Immunomodulation of inflammatory leukocyte markers during intravenous immunoglobulin treatment associated with clinical efficacy in chronic inflammatory demyelinating polyradiculoneuropathy

Author

Dyer, WB, Tan, JCG, Day, T, Kiers, L, Kiernan, MC, Yiannikas, C, Reddel, S, Ng, K, Mondy, P, Dennington, PM, Dean, MM, Trist, HM, dos Remedios, C, Hogarth, PM, Vucic, S, Irving, DO, Dyer, WB, Tan, JCG, Day, T, Kiers, L, Kiernan, MC, Yiannikas, C, Reddel, S, Ng, K, Mondy, P, Dennington, PM, Dean, MM, Trist, HM, dos Remedios, C, Hogarth, PM, Vucic, S, and Irving, DO

Abstract

OBJECTIVE: The objective of the study was to profile leukocyte markers modulated during intravenous immunoglobulin (IVIg) treatment, and to identify markers and immune pathways associated with clinical efficacy of IVIg for chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) with potential for monitoring treatment efficacy. METHODS: Response to IVIg treatment in newly diagnosed IVIg-naïve and established IVIg-experienced patients was assessed by changes in expression of inflammatory leukocyte markers by flow cytometry. The adjusted INCAT disability and Medical Research Council sum scores defined clinical response. RESULTS: Intravenous immunoglobulin modulated immunopathogenic pathways associated with inflammatory disease in CIDP. Leukocyte markers of clinical efficacy included reduced CD185+ follicular helper T cells, increased regulatory markers (CD23 and CD72) on B cells, and reduction in the circulating inflammatory CD16+ myeloid dendritic cell (mDC) population and concomitant increase in CD62L and CD195 defining a less inflammatory lymphoid homing mDC phenotype. A decline in inflammatory CD16+ dendritic cells was associated with clinical improvement or stability, and correlated with magnitude of improvement in neurological assessment scores, but did not predict relapse. IVIg also induced a nonspecific improvement in regulatory and reduced inflammatory markers not associated with clinical response. CONCLUSIONS: Clinically effective IVIg modulated inflammatory and regulatory pathways associated with ongoing control or resolution of CIDP disease. Some of these markers have potential for monitoring outcome.

Published
2016

23. Analysis of HLA-DRB3 alleles and supertypical genotypes in the MHC Class II region in sporadic inclusion body myositis

Author

Rojana-udomsart, A., Mitrpant, C., James, I., Witt, C., Needham, M., Day, T., Kiers, L., Corbett, A., Martinez, P., Wilton, S.D., Mastaglia, F.L., Rojana-udomsart, A., Mitrpant, C., James, I., Witt, C., Needham, M., Day, T., Kiers, L., Corbett, A., Martinez, P., Wilton, S.D., and Mastaglia, F.L.

Abstract

We compared the carriage frequencies of HLA-DRB3 and its major alleles and of HLA-DRB4 and HLA-DRB5 in an Australian sIBM cohort and a population control group who had previously been genotyped for the HLA-DRB1*03:01 risk allele. There was a strong disease association with the carriage of the DRB3*01:01 allele which was accounted for by its linkage disequilibrium with DRB1*03:01. The carriage of HLA-DRB4 was found to be strongly protective and abrogated the risk effect of HLA-DRB1*03:01. The findings indicate that haplotypic combinations of alleles at the HLA-DRB1 and secondary HLA-DRB loci have important risk modifying effects in sIBM.

Published
2013

24. G.P.63 High-resolution analysis of HLA-DRB1 alleles and diplotypes in an Australian inclusion body myositis cohort

Author

Rojana-udomsart, A., Castley, A., James, I., Needham, M., Scott, A., Day, T., Kiers, L., Corbett, A., Sue, C., Martinez, P., Christiansen, F., Mastaglia, F.L., Rojana-udomsart, A., Castley, A., James, I., Needham, M., Scott, A., Day, T., Kiers, L., Corbett, A., Sue, C., Martinez, P., Christiansen, F., and Mastaglia, F.L.

Abstract

In Caucasians, susceptibility to sporadic inclusion body myositis (s-IBM) is associated with the HLA-DR3 serotype and the HLA-DRB1∗0301 allele which encodes it, and with the extended 8.1 MHC ancestral haplotype. Most previous HLA studies in s-IBM have used serological typing or low-resolution (2-digit) genotyping to identify disease-associated alleles, and the contribution of alleles other than HLA-DRB1∗0301 and of the complementary in trans allele at the HLA-DRB1 locus has received little attention. In the present study we performed high-resolution (4-digit) HLA-DRB1 genotyping in an Australian cohort of 105 s-IBM patients and 189 controls from the Busselton Population Study and analysed risk-associated alleles and effects on age-at-onset (AAO) of the disease. Case–control logistic regression analysis showed that, apart from the strongly associated HLA-DRB1∗0301 allele (OR 13.6), HLA-DRB1∗0101 (OR 3.65) and HLA-DRB1∗1301 (OR 3.31) are independent risk alleles. A number of other alleles, HLA-DRB1∗0401, ∗0404, ∗0701, ∗0901, ∗1101 and ∗1501 were reduced in frequency in s-IBM cases and may be protective. We evaluated the influence of allele combinations on disease risk and found that the strongest estimated risk is with the HLA-DRB1∗0301/∗0101 diplotype, while other allele combinations have diminishing risk effects. HLA-DRB1∗0301 was associated with an AAO 4.3 years earlier than other alleles and DRB1∗1301 with an AAO 4.5 years later, despite it being a risk factor. The findings indicate that the influence of HLA-DRB1 in s-IBM is complex, influencing both disease risk and AAO, and that allelic interactions at the HLA-DRB1 locus contribute to disease susceptibility.

Published
2012

25. High-resolution HLA-DRB1 genotyping in an Australian inclusion body myositis (s-IBM) cohort: An analysis of disease-associated alleles and diplotypes

Author

Rojana-udomsart, A., James, I., Castley, A., Needham, M., Scott, A., Day, T., Kiers, L., Corbett, A., Sue, C., Witt, C., Martinez, P., Christiansen, F., Mastaglia, F., Rojana-udomsart, A., James, I., Castley, A., Needham, M., Scott, A., Day, T., Kiers, L., Corbett, A., Sue, C., Witt, C., Martinez, P., Christiansen, F., and Mastaglia, F.

Abstract

We performed high-resolution (4-digit) HLA-DRB1 genotyping in an Australian cohort of 105s-IBM patients and 189 controls. Our findings showed that whilst the strongest association was with the HLA-DRB1*03:01 allele and the HLA-DRB1*03:01/*01:01 diplotype, HLA-DRB1*01:01 and HLA-DRB1*13:01 are also risk alleles. A number of other alleles, HLA-DRB1*04:01, *04:04, *07:01, *09:01, *11:01 and *15:01, as well as the HLA-DRB1*03:01/*04:01 and HLA-DRB1*03:01/*07:01 diplotypes were reduced in s-IBM cases and may be protective. The HLA-DRB1*03:01 and HLA-DRB1*13:01 alleles also appear to have an influence on the age at onset of the disease and severity of muscle weakness. Our findings indicate that the influence of HLA-DRB1 in s-IBM is complex and that epistatic interactions at the HLA-DRB1 locus contribute both to disease susceptibility and to the clinical phenotype.

Published
2012

26. G.P.16.09 Epistatic interactions between DRB1 alleles influence susceptibility and clinical phenotype in sporadic inclusion body myositis (sIBM)

Author

Mastaglia, F.L., Needham, M., Scott, A., James, I., Day, T., Kiers, L., Corbett, A., Witt, C., Garlepp, M., Allcock, R., Laing, N., Christiansen, F., Mastaglia, F.L., Needham, M., Scott, A., James, I., Day, T., Kiers, L., Corbett, A., Witt, C., Garlepp, M., Allcock, R., Laing, N., and Christiansen, F.

Abstract

Background: Susceptibility to sIBM is strongly associated with HLA-DRB1*03 and the 8.1 MHC ancestral haplotype but little is known about the effect of allelic interactions at the DRB1 locus or disease-modifying effects of HLA alleles. Objectives: To determine whether DRB1 allele interactions influence susceptibility to sIBM and disease phenotype. Patients & Methods: HLA-DRB1 genotyping was carried out in 80 biopsy-proven cases of sIBM using high-resolution DNA sequencing in 55 cases and serological typing in 25 cases. Allele frequencies were compared with those in a group of 190 healthy population controls. All patients had manual muscle testing using an expanded 10-point MRC scale as well as documentation of historical aspects of their disease. Results: The strong positive association with HLA-DRB1*03 was confirmed (OR 9.6) while negative associations were found with HLA-DRB1∗04 and DRB1∗07. In addition, amongst those carrying the HLA-DRB1∗03 allele DRB1∗03/∗01 heterozygotes were over-represented in the sIBM group (p < 0.003). DRB1∗03/∗04 was the most significantly under-represented combination (p < 0.008) and remained so when those with DRB1∗03/∗01 were excluded (p = 0.03). Age at onset (AAO) was not associated with any individual allele but the mean AAO was earlier in the HLA-DRB1∗03/∗01 heterozygotes (55.8 years vs 62.3 years, p = 0.006) who also had more severe quadriceps weakness than the rest of the cohort (average MRC grade 3.57 vs 5.63, p = 0.003). Conclusions: The findings indicate that the HLA-DRB1*03 allele interacts with other alleles at the DRB1 locus to influence susceptibility to sIBM as well as AAO of the disease and disease severity.

Published
2009

27. Sporadic inclusion body myositis: HLA-DRB1 allele interactions influence disease risk and clinical phenotype

Author

Mastaglia, F.L., Needham, M., Scott, A., James, I., Zilko, P., Day, T., Kiers, L., Corbett, A., Witt, C.S., Allcock, R., Laing, N., Garlepp, M., Christiansen, F.T., Mastaglia, F.L., Needham, M., Scott, A., James, I., Zilko, P., Day, T., Kiers, L., Corbett, A., Witt, C.S., Allcock, R., Laing, N., Garlepp, M., and Christiansen, F.T.

Abstract

Susceptibility to sIBM is strongly associated with the HLA-DRB1*03 allele and the 8.1 MHC ancestral haplotype (HLA-A1, B8, DRB1*03) but little is known about the effects of allelic interactions at the DRB1 locus or disease-modifying effects of HLA alleles. HLA-A, B and DRB1 genotyping was performed in 80 Australian sIBM cases and the frequencies of different alleles and allele combinations were compared with those in a group of 190 healthy controls. Genotype-phenotype correlations were also investigated. Amongst carriers of the HLA-DRB1*03 allele, DRB1*03/*01 heterozygotes were over-represented in the sIBM group (p < 0.003) while. DRB1*03/*04 heterozygotes were under-represented (p < 0.008). The mean age-at-onset (AAO) was 6.5 years earlier in DRB1*03/*01 heterozygotes who also had more severe quadriceps muscle weakness than the rest of the cohort. The findings indicate that interactions between the HLA-DRB1*03 allele and other alleles at the DRB1 locus can influence disease susceptibility and the clinical phenotype in sIBM.

Published
2009

29. G.P.5.06 HLA alleles and MHC haplotypes in sporadic inclusion body myositis: Frequencies and phenotypic correlations

Author

Needham, M., Scott, A., Christiansen, F., James, I., Corbett, A., Day, T., Kiers, L., Laing, N., Allcock, R., Mastaglia, F.L., Needham, M., Scott, A., Christiansen, F., James, I., Corbett, A., Day, T., Kiers, L., Laing, N., Allcock, R., and Mastaglia, F.L.

Abstract

Background. Sporadic inclusion body myositis (sIBM) is strongly associated with HLA-DR3 and the MHC 8.1 ancestral haplotype (AH) in Caucasians, but less is known about other HLA associations or whether HLA haplotypes modify disease expression. Objective. To determine the frequency of HLA alleles and haplotypes in a cohort of 80 Australian Caucasian cases of biopsy-proven sIBM and correlations with phenotypic features. Methods. Patients were recruited from neuromuscular clinics in Perth, Melbourne and Sydney. All were examined by one or more of the authors and fulfilled the criteria for definite or probable sIBM [1] and [2]. Typing for Class I and II HLA alleles was performed by high resolution sequence-based genotyping, and assignment of ancestral haplotypes (AHs) was predicted from the presence of specific HLA-B and DRB1 alleles. The frequencies of alleles and AHs were compared with a control group of 190 healthy Caucasian West Australians. Results. The strong association with HLA-DR3 (p < 0.001), HLA-B8 (p < 0.001) and the 8.1AH (p < 0.001) was confirmed. Significant positive associations were also found with HLA-DR1 (p < 0.001) and the 7.2AH (p = 0.026), as well as with HLA-B55 (p = 0.018), the 52.1AH (p = 0.052) and 62.3AH (p = 0.023). HLA-DR1 (p = 0.06) and B27 (p = 0.04) were associated with an earlier age-of-onset. HLA-DR3 was associated with a lower quadriceps muscle strength (p = 0.04) and B27 and B49 with higher quadriceps strength even when allowing for treatment as an independent variable. Conclusions. The findings demonstrate novel HLA alleles and MHC haplotypes associated with susceptibility to sIBM in a Caucasian population, and that certain HLA alleles have a disease-modifying effect.

Published
2008

33. Mitochondrial DNA variants in inclusion body myositis

Author

Kok, C.C., Boyt, A., Gaudieri, S., Martins, R., Askanas, V., Dalakas, M., Kiers, L., Mastaglia, F., Garleep, M., Kok, C.C., Boyt, A., Gaudieri, S., Martins, R., Askanas, V., Dalakas, M., Kiers, L., Mastaglia, F., and Garleep, M.

Abstract

Mitochondrial DNA variants have been shown to be associated with many diseases. Mutations at mitochondrial DNA nucleotide positions 3192, 3196, 3397 and 4336 have been described in association with late-onset Alzheimer's disease. The pathological similarities between inclusion body myositis and Alzheimer's disease prompted an analysis of the relationship between the reported mutations and sporadic inclusion body myositis. The 4336G variant was not significantly increased in patients with inclusion body myositis or Alzheimer's disease when compared to controls. None of the patients with inclusion body myositis carried mutations at nucleotide positions 3192, 3196 and 3397. A transition at nucleotide position 4580 was detected in some patients with inclusion body myositis and Alzheimer's disease but was not significantly higher in frequency when compared to controls. Phylogenetic analysis showed that the 4336G and 4580A variants clustered together in their respective group. A group of patients with inclusion body myositis also clustered together on a separate branch of the phylogenetic tree. Closer investigation of this group revealed a common polymorphism at nucleotide position 16311. The frequency of the 16311C variant was higher in inclusion body myositis than in Alzheimer's disease and controls, although when only caucasian patients were considered the increased frequency was not statistically significant. Further studies will be required to determine whether this variant plays a role in the pathogenesis of inclusion body myositis.

Published
2000

34. Mapping of a candidate region for susceptibility to inclusion body myositis in the human major histocompatibility complex

Author

Kok, C.C., Croager, E.J., Witt, C.S., Kiers, L., Mastaglia, F.L., Abraham, L.J., Garlepp, M.J., Kok, C.C., Croager, E.J., Witt, C.S., Kiers, L., Mastaglia, F.L., Abraham, L.J., and Garlepp, M.J.

Abstract

Inclusion body myositis (IBM) is a form of idiopathic inflammatory myopathy of unknown aetiology. A strong association with HLA class II (HLA-DR3) suggested a role for genes in the human major histocompatibility complex (MHC) in the predisposition to this disease. In this study, we have taken advantage of the ancestral haplotype (AH) concept and historical recombinations to map for a possible susceptibility gene(s) in the MHC. We performed detailed typing of three MHC-related HSP70 genes and defined allelic combinations in the context of MHC AH. We also modified existing methods to give a simple and accurate method for typing two TNF microsatellites. Using the HSP70 and TNF markers and HLA-DR, –B, and C4 typing of our patients with IBM, we defined a potential site for the MHC-associated susceptibility gene(s) in the region between HLA-DR and C4.

Published
1999

35. Effects of routine hyperventilation on PCO2 and PO2 in normal subjects: implications for EEG interpretations.

Author

Achenbach-Ng, J, Siao, T C, Mavroudakis, Nicolas, Chiappa, Keith, Kiers, L, Achenbach-Ng, J, Siao, T C, Mavroudakis, Nicolas, Chiappa, Keith, and Kiers, L

Abstract

There are few data in the EEG literature describing the time course of hyperventilation-(HV) induced changes in blood gases, despite this being a routine activating procedure. We studied changes in blood gases and EEG in nine normal adult subjects before, during, and after HV. The mean PCO2 fell 18 mm Hg from the baseline during HV and recovered in 7 min. The mean PO2 rose 7 mmHg during HV and fell to 25 mm Hg below baseline 5 min after HV. The PCO2 recovery period is longer than is usually assumed in clinical EEG. The PO2 fall to a nadir at 5 min after the end of HV suggests that close attention should be paid to this period, as is confirmed by the re-buildup seen in moyamoya disease. Despite uniform changes in blood gases, the EEG median power frequency change showed marked variability; on average, it dropped by 1 Hz during HV and returned to baseline within 2 min of resumption of normal respiration. The EEG root-mean-square power showed a 200% increase during HV and also had returned to normal within 2 min., Journal Article, info:eu-repo/semantics/published

Published
1994

46. Reply

Author

KIERS, L., primary, DAVIS, S., additional, and EBELING, P., additional

Published
1990
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