Subcutaneous immunoglobulin for maintenance treatment in chronic inflammatory demyelinating polyneuropathy (PATH) : a randomised, double-blind, placebo-controlled, phase 3 trial
| العنوان: | Subcutaneous immunoglobulin for maintenance treatment in chronic inflammatory demyelinating polyneuropathy (PATH) : a randomised, double-blind, placebo-controlled, phase 3 trial |
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| المؤلفون: | Michael Schroeter, Mazen M. Dimachkie, J. Zschuentssch, Takuya Ohkubo, Kenichi Kaida, M. Bednar, M. Tomiyama, J. Sussova, D. Mueller, E. Chi-Ho Lai, Nicolette C. Notermans, Toomas Toomsoo, C. D'Amour, J. Haas, B. Murinson, Masahiro Mori, Richard A. Lewis, Masayuki Baba, Anne D. Sperfeld, Vivian E. Drory, Hans-Peter Hartung, J. Demeestere, Satoshi Kuwabara, Leslie Roberts, S. Mumfrey, David Gosal, Katrin Gross-Paju, M. Zibetti, Martin Vališ, Filip Eftimov, David Yarnitsky, D. Aufauvre, G. Le Masson, Takashi Kanda, Lisa D. Hobson-Webb, I. Melamed, Alexander Shtilbans, Inna Rubanovits, P. MacDonald, Janneke G. J. Hoeijmakers, Vera Bril, Ericka Simpson, Orell Mielke, Michaela Praus, Martin Stangel, Masahiro Iijima, Richard J. Barohn, Robert D. Henderson, P. Baum, Mari Auranen, David Walk, Said R. Beydoun, A. Jaspert-Grehl, Alessandro Testori, Giovanni Antonini, Ingemar S. J. Merkies, Sabrina Matà, A. Di Muzio, Ivo N. van Schaik, T. Kalous, Josep Gamez, Juliane Klehmet, Dario Cocito, Angelo Schenone, R. Carne, P. Kunc, Dale J. Lange, Miriam Freimer, S. Muley, Norman Latov, T. Rao, Jens Ejbye Schmidt, Jasper M. Morrow, Ari Breiner, C. Marquez Infante, C. G. Faber, U. Chyrchel-Paszkiewicz, Anne-Cécile Wielanek-Bachelet, Russell L. Chin, John-Philip Lawo, I. N. van Schaik, C. Goerlitz, M. Chatzopoulos, Tim Hagenacker, Claudia Sommer, H. Johl, D. Kramer, Stefania Morino, R. Yoon, Daniela M. Menichella, M. Alberti Aguiló, K. Nishiyama, Daniele Cazzato, F. Bethke, Helmar C. Lehmann, Konrad Rejdak, T. Lavin, Kazumasa Yokoyama, Olaf Hoffmann, M. Kawai, C. Casanovas Pons, Sandro Sorbi, Takanori Yokota, Nora A. Visser, R. Talab, Eroboghene E. Ubogu, Florian Then Bergh, Stefan Blum, Ginna Gonzalez, J. Oechtering, David R. Cornblath, F. Ciccocioppo, A. Sabet, Fabian Klostermann, Nan van Geloven, K. George, A. Kutschenko, S. Benitez Rivero, Karissa L. Gable, Michael P. Lunn, Senda Ajroud-Driss, Shahram Attarian, Marina Grandis, P. Van Damme, C. Trebst, Jeffrey A. Allen, A. Algom, H. Onoue, D. Liebetanz, Billie L. Durn, Maria Salvado Figueras, Jean Pouget, Emilien Delmont, Khema Sharma, Gen Sobue, K. Ohyama, John T. Kissel, K. Kanai, Tsugio Akutsu, Pierre Clavelou, Andreas Meisel, Giuseppe Lauria, M. Saarela, S. Larue, R. Gold, U. Sorro, Shafeeq Ladha, Claude Desnuelle, P. Berlit |
| المساهمون: | Neurologian yksikkö, Clinicum, HUS Neurocenter, ANS - Neuroinfection & -inflammation, AII - Inflammatory diseases, Other departments, Neurology, AII - Amsterdam institute for Infection and Immunity, Hagenacker, Tim (Beitragende*r), RS: MHeNs - R1 - Cognitive Neuropsychiatry and Clinical Neuroscience |
| المصدر: | Lancet Neurology, 17(1), 35. Lancet Publishing Group The Lancet Neurology Lancet neurology, 17(1), 35-46. Lancet Publishing Group The Lancet Neurology, 17(1), 35-46 Lancet Neurology, 17(1), 35-46. Elsevier Science |
| سنة النشر: | 2018 |
| مصطلحات موضوعية: | 0301 basic medicine, Male, SATISFACTION, Clinical Trial, Phase III, Medizin, Chronic inflammatory demyelinating polyneuropathy, THERAPY, 3124 Neurology and psychiatry, law.invention, MUSCLE STRENGTH, 0302 clinical medicine, Randomized controlled trial, Quality of life, law, QUALITY-OF-LIFE, Chronic Inflammatory Demyelinating, Subcutaneous, Absolute risk reduction, IGG SELF-INFUSIONS, Middle Aged, Clinical Trial, 3. Good health, Randomized Controlled Trial, POLYRADICULONEUROPATHY, Female, aged, double-blind method, female, humans, immunoglobulins, immunologic factors, injections, subcutaneous, male, middle aged, polyradiculoneuropathy, chronic inflammatory demyelinating, outcome assessment (health care), neurology (clinical), medicine.medical_specialty, Injections, Subcutaneous, Clinical Neurology, Immunoglobulins, CIDP, Placebo, Injections, 03 medical and health sciences, Outcome Assessment (Health Care), Phase III, Double-Blind Method, Internal medicine, medicine, Journal Article, Humans, Immunologic Factors, HOME, Adverse effect, Aged, business.industry, ICE, 3112 Neurosciences, Polyradiculoneuropathy, medicine.disease, Surgery, Clinical trial, 030104 developmental biology, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating, PRIMARY ANTIBODY DEFICIENCIES, Neurology (clinical), business, 030217 neurology & neurosurgery |
| الوصف: | Mika Saarela työryhmän jäsenenä. Background Approximately two-thirds of patients with chronic inflammatory demyelinating polyneuropathy (CIDP) need long-term intravenous immunoglobulin. Subcutaneous immunoglobulin (SCIg) is an alternative option for immunoglobulin delivery, but has not previously been investigated in a large trial of CIDP. The PATH study compared relapse rates in patients given SCIg versus placebo. Methods Between March 12, 2012, and Sept 20, 2016, we studied patients from 69 neuromuscular centres in North America, Europe, Israel, Australia, and Japan. Adults with definite or probable CIDP who responded to intravenous immunoglobulin treatment were eligible. We randomly allocated participants to 0.2 g/kg or 0.4 g/kg of a 20% SCIg solution (IgPro20) weekly versus placebo (2% human albumin solution) for maintenance treatment for 24 weeks. We did randomisation in a 1: 1:1 ratio with an interactive voice and web response system with a block size of six, stratified by region (Japan or non-Japan). The primary outcome was the proportion of patients with a CIDP relapse or who were withdrawn for any other reason during 24 weeks of treatment. Patients, caregivers, and study personnel, including those assessing outcomes, were masked to treatment assignment. Analyses were done in the intention-to-treat and per-protocol sets. This trial is registered with ClinicalTrials. gov, number NCT01545076. Findings In this randomised, double-blind, placebo-controlled trial, we randomly allocated 172 patients: 57 (33%) to the placebo group, 57 (33%) to the low-dose group, and 58 (34%) to the high-dose group. In the intention-to-treat set, 36 (63% [95% CI 50-74]) patients on placebo, 22 (39% [27-52]) on low-dose SCIg, and 19 (33% [22-46]) on high-dose SCIg had a relapse or were withdrawn from the study for other reasons (p=0.0007). Absolute risk reductions were 25% (95% CI 6-41) for low-dose versus placebo (p=0.007), 30% (12-46) for high-dose versus placebo (p=0.001), and 6% (-11 to 23) for high-dose versus low-dose (p=0.32). Causally related adverse events occurred in 47 (27%) patients (ten [18%] in the placebo group, 17 [30%] in the low-dose group, and 20 [34%] in the high-dose group). Six (3%) patients had 11 serious adverse events: one (2%) patient in the placebo group, three (5%) in the low-dose group, and two (3%) in the high-dose group; only one (an acute allergic skin reaction in the low-dose group) was assessed to be causally related. Interpretation This study, which is to our knowledge, the largest trial of CIDP to date and the first to study two administrations of immunoglobulins and two doses, showed that both doses of SCIg IgPro20 were efficacious and well tolerated, suggesting that SCIg can be used as a maintenance treatment for CIDP. |
| وصف الملف: | image/pdf; application/pdf |
| اللغة: | English |
| تدمد: | 1474-4422 |
| الوصول الحر: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f23ad596dcdfe17d2212d3b57623ed37Test https://dspace.library.uu.nl/handle/1874/363886Test |
| حقوق: | OPEN |
| رقم الانضمام: | edsair.doi.dedup.....f23ad596dcdfe17d2212d3b57623ed37 |
| قاعدة البيانات: | OpenAIRE |
| تدمد: | 14744422 |
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