Patient-reported outcomes with subcutaneous immunoglobulin in chronic inflammatory demyelinating polyneuropathy: the PATH study.

Autor: Hartung HP; Department of Neurology, UKD and Center for Neurology and Neuropsychiatry, LVR Klinikum, Medical Faculty, Heinrich Heine University, Düsseldorf, Germany., Mallick R; CSL Behring LLC, King of Prussia, PA, USA., Bril V; Ellen and Martin Prosserman Centre for Neuromuscular Diseases, Division of Neurology, Department of Medicine, University Health Network, University of Toronto, Toronto, Canada.; Institute for Research and Medical Consultations, Imam Abdulrahman Bin Faisal University, Dammam, Saudi Arabia., Lewis RA; Department of Neurology, Cedars-Sinai Medical Center, Los Angeles, CA, USA., Sobue G; Department of Neurology, Nagoya University Graduate School of Medicine, Nagoya, Japan., Lawo JP; CSL Behring, Marburg, Germany., Mielke O; CSL Behring, Marburg, Germany., Durn BL; CSL Behring LLC, King of Prussia, PA, USA., Cornblath DR; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD, USA., Merkies ISJ; Department of Neurology, Maastricht University Medical Center, Maastricht, The Netherlands.; Department of Neurology, St Elizabeth Hospital, Willemstad, Curacao., van Schaik IN; Department of Neurology, Amsterdam University Medical Centres, University of Amsterdam, Amsterdam, The Netherlands.; Spaarne Gasthuis, Haarlem, The Netherlands.
Jazyk: angličtina
Zdroj: European journal of neurology [Eur J Neurol] 2020 Jan; Vol. 27 (1), pp. 196-203. Date of Electronic Publication: 2019 Sep 23.
DOI: 10.1111/ene.14056
Abstrakt: Background and Purpose: Chronic inflammatory demyelinating polyneuropathy (CIDP) causes weakness which adversely impacts function and quality of life (QOL). CIDP often requires long-term management with intravenous or subcutaneous immunoglobulin. The Polyneuropathy and Treatment with Hizentra® (PATH) study showed that subcutaneous immunoglobulin (SCIG) was efficacious in CIDP maintenance. Here, patient-reported outcomes in patients on SCIG are assessed.
Methods: Subjects stabilized on intravenous immunoglobulin were randomly allocated to receive weekly 0.2 or 0.4 g/kg bodyweight of 20% SCIG (IgPro20) or placebo. Overall QOL/health status was assessed using the EuroQoL 5-Dimension (EQ-5D) health profile and visual analog scale, treatment satisfaction was assessed with the Treatment Satisfaction Questionnaire for Medicine (TSQM) and work-related impact was assessed with the Work Productivity and Activity Impairment Questionnaire for General Health (WPAI-GH). The EQ-5D health profile was assessed in terms of the percentage of subjects maintained or improved at week 25 of SCIG therapy on each of the EQ-5D domains versus baseline after intravenous immunoglobulin stabilization. TSQM and WPAI-GH were assessed by median score changes from baseline to week 25.
Results: In total, 172 subjects were randomized to placebo (n = 57), 0.2 g/kg IgPro20 (n = 57) and 0.4 g/kg IgPro20 (n = 58). Significantly higher proportions of IgPro20-treated subjects improved/maintained their health status on the EQ-5D usual activities dimension, and in additional dimensions (mobility and pain/discomfort) in sensitivity analyses. TSQM and WPAI-GH scores were more stable with IgPro20 treatment compared with placebo.
Conclusions: IgPro20 maintained or improved QOL in most subjects with CIDP, consistent with the PATH study findings that both IgPro20 doses were efficacious in maintaining CIDP.
(© 2019 CSL Behring. European Journal of Neurology published by John Wiley & Sons Ltd on behalf of European Academy of Neurology.)
Databáze: MEDLINE
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