@article{a934860844a64118879bc81079745588,
title = "Long-term safety and efficacy of subcutaneous immunoglobulin IgPro20 in CIDP: PATH extension study",
abstract = "ObjectiveTo investigate the long-term safety and efficacy of weekly subcutaneous IgPro20 (Hizentra, CSL Behring) in chronic inflammatory demyelinating polyneuropathy (CIDP).MethodsIn a 48-week open-label prospective extension study to the PATH study, patients were initially started on 0.2 g/kg or on 0.4 g/kg weekly and - if clinically stable - switched to 0.2 g/kg weekly after 24 weeks. Upon CIDP relapse on the 0.2 g/kg dose, 0.4 g/kg was (re)initiated. CIDP relapse was defined as a deterioration by at least 1 point in the total adjusted Inflammatory Neuropathy Cause and Treatment score.ResultsEighty-two patients were enrolled. Sixty-two patients initially received 0.4 g/kg, 20 patients 0.2 g/kg weekly. Seventy-two received both doses during the study. Sixty-six patients (81%) completed the 48-week study duration. Overall relapse rates were 10% in 0.4 g/kg-treated patients and 48% in 0.2 g/kg-treated patients. After dose reduction from 0.4 to 0.2 g/kg, 51% (27/53) of patients relapsed, of whom 92% (24 of 26) improved after reinitiation of the 0.4 g/kg dose. Two-thirds of patients (19/28) who completed the PATH study without relapse remained relapse-free on the 0.2 g/kg dose after dose reduction in the extension study. Sixty-two patients had adverse events (AEs) (76%), of which most were mild or moderate with no related serious AEs.ConclusionsSubcutaneous treatment with IgPro20 provided long-term benefit at both 0.4 and 0.2 g/kg weekly doses with lower relapse rates on the higher dose. Long-term dosing should be individualized to find the most appropriate dose in a given patient.Classification of evidenceThis study provides Class IV evidence that for patients with CIDP, long-term treatment with SCIG beyond 24 weeks is safe and efficacious.",
author = "{Van Schaik}, {Ivo N.} and Orell Mielke and Vera Bril and {Van Geloven}, Nan and Hartung, {Hans Peter} and Lewis, {Richard A.} and Gen Sobue and Lawo, {John Philip} and Michaela Praus and Durn, {Billie L.} and Cornblath, {David R.} and Merkies, {Ingemar S.J.}",
note = "Funding Information: I.N. van Schaik chairs a steering committee for CSL Behring and received departmental honoraria for serving on scientific advisory boards for CSL Behring and Baxter. He received departmental research support from The Netherlands Organization for Scientific Research and from the Dutch Prinses Beatrix Fonds. All lecturing and consulting fees for INS were donated to the Stichting Klinische Neurologie, a local foundation that supports research in the field of neurologic disorders. He served on the editorial board of the Cochrane Neuromuscular Disease Group and is a member of the scientific board of the Kreuth III meeting on the optimal use of plasma-derived medicinal products, especially coagulation factors and normal immunoglobulins organized under the auspices of the European Directorate for the Quality of Medicines & HealthCare (EDQM). V. Bril is consultant to CSL Behring, Grifols, UCB, Bionevia, ArgenX, Shire, Alnylam, and Alexion. She serves on international scientific advisory boards of MGFA and the CIDP/GBS International Foundation and has received research support from CSL Behring, Grifols, Bionevia, Baxter, UCB, and ArgenX. N. van Geloven received departmental honoraria for serving at a scientific advisory board of CSL Behring. H.P. Hartung received fees for consulting and serving on steering committees or ad boards from Baxter, Bayer Healthcare, Biogen, CSL Behring, GeNeuro, Kedrion, LFB, MedImmune, Merck, Novartis, Octapharma, Receptos Celgene, Roche, Sanofi Genzyme, and Teva with approval by the Rector of Heinrich Heine University D{\"u}sseldorf. R. A. Lewis is chair of the Inflammatory Neuropathy Consortium (INC), a standing committee of the Peripheral Nerve Society, a member of the board of Directors of the Peripheral Nerve Society, and a member of the medical advisory board of the GBS-CIDP Foundation, MGF of America, and MGF of California. He is a paid consultant for CSL Behring, Novartis, Pharnext, Axelacare, Biotest, and NuFactor. G. Sobue served on the scientific advisory boards of the Kanae Science Foundation for the Promotion of Medical Science and the Takeda Foundation. He serves on a steering committee for CSL Behring. He received funding for travel and speaker honoraria from Mitsubishi Tanabe Pharma Co, Shionogi Co Ltd, Bristol-Myers Squibb, Sumitomo Dainippon Pharma Co Ltd, Novartis Pharma KK, Bayer Yakuhin Ltd, Pfizer Japan Inc, Boehringer Ingelheim Japan, Inc, Kissei Pharmaceutical Co Ltd, Janssen Pharmaceutical KK, Teijin Pharma Ltd, FP Pharmaceutical Co, Nihon Pharmaceutical Co Ltd, Japan Blood Products Organization, Kowa Pharmaceutical Co Ltd, Ono Pharmaceutical Co Ltd, and Eisai Co Ltd. He also received grants from the Ministry of Health, Labour and Welfare Japan, Japanese Ministry of Education, Culture, Sports, Science and Technology, and Japan Society for the Promotion of Science. J.P. Lawo and M. Praus are CSL employees and biostatisticians for the PATH study. O. Mielke is a CSL employee and program director for the PATH study. B.L. Durn is a CSL employee and clinical scientist for the PATH study. D.R. Cornblath is consultant for Annexon Biosciences, argenx BVBA, Biotest Pharmaceuticals, Inc., Cigna Health Management, Inc., CSL Behring, DP Clinical, Inc., Grifols S.A., Hansa Medical Inc., Merrimack Pharmaceuticals, Inc., Neurocrine Biosciences, Novartis Corp., Octapharma AG, Pharnext SAS, Seattle Genetics, Inc., Sun Pharmaceuticals, and Syntimmune. He is a member of Data Safety Monitoring Boards for Pfizer Inc., Axovant Sciences LTD., Ampio Pharmaceuticals, PledPharma, Momenta Pharma, and Sanofi; Technology Licensing for the Total Neuropathy Score to AstraZeneca Pharmaceuticals, LP, Calithera Biosciences, Genentech Inc, Neurocrine Biosciences, Merrimack Pharmaceuticals, Inc., and Seattle Genetics, Inc. He is a nonvoting member of the Board of Directors of The Peripheral Nerve Society. He is on the Global Medical Advisory Board of the GBS-CIDP Foundation International. He is Editor-in-Chief of the Journal of the Peripheral Nervous System. I.S.J. Merkies received funding for research from the Talecris Talents program, the GSB CIDP Foundation International, Princes Beatrix foundation, and from the European Union 7th Framework Programme (grant n°602273). Furthermore, a research foundation at the University of Maastricht received honoraria on behalf of him for participation in steering committees of the Talecris ICE Study, LFB, CSL Behring, Novartis, Grifols, and Octapharma. He serves on the editorial board of the Journal of Peripheral Nervous system and is a member of the Inflammatory Neuropathy Consortium (INC) and the Peripheral Nerve Society. Go to https://nn.neurology.org/content/6/5/e590/tab-article-info for full disclosures. Publisher Copyright: {\textcopyright} 2019 American Academy of Neurology.",
year = "2019",
month = sep,
day = "1",
doi = "10.1212/NXI.0000000000000590",
language = "English (US)",
volume = "6",
journal = "Neurology: Neuroimmunology and NeuroInflammation",
issn = "2332-7812",
publisher = "Lippincott Williams and Wilkins Ltd.",
number = "5",
}