Treatment of Neurological Autoimmune Diseases with Immunoglobulins: First Insights from the Prospective SIGNS Registry

Springer Science and Business Media LLC - Tập 33 - Trang 67-71 - 2012
Martin Stangel1,2, Ulrich Baumann3, Michael Borte4, Maria Fasshauer4, Manfred Hensel5, Dörte Huscher6, Wilhelm Kirch7, David Pittrow7, Marcel Reiser8, Ralf Gold9
1Department of Neurology, Hannover Medical School, Hannover, Germany
2Medizinische Hochschule Hannover, Klinik für Neurologie, Hannover, Germany
3Paediatric Pulmonology, Allergy and Neonatology, Hannover Medical School, Hannover, Germany
4Paediatric Rheumatology, Immunology and Infectiology, Hospital St. Georg, Leipzig, Germany
5Mannheimer Onkologie Praxis, Mannheim, Germany
6Epidemiology, German Rheumatism Research Centre, Berlin, and Rheumatology, Charité, Berlin, Germany
7Institute for Clinical Pharmacology, Medical Faculty, Technical University Dresden, Dresden, Germany
8PIOH–Praxis Internistische Onkologie, Hämatologie, Köln, Germany
9Department for Neurology, St. Josef-Hospital, Ruhr University Bochum, Bochum, Germany

Tóm tắt

Several immunoglobulin (IG) preparations have been approved for the immunomodulatory treatment of the neurological autoimmune diseases (AID) Guillain-Barré syndrome (GBS), chronic inflammatory demyelinating polyneuropathy (CIDP), and multifocal motor neuropathy (MMN). Although efficacy has been proven in randomised clinical trials, long-term outcome data on drug utilization, effectiveness, tolerability, health related quality of life, and economic variables are lacking. In the prospective, observational internet-based SIGNS registry, patients of all age groups are eligible if they have received or are scheduled for IG therapy for neurological AID or primary or severe secondary immunodeficiency. Of the 306 patients currently included in the database (1 November 2011), 51 have neurological AID (27 males; mean age 56 ± 15 years): 21 CIDP, 7 MMN, 11 multiple sclerosis (MS), 6 myasthenia gravis, 2 myositis, 4 others (no cases of GBS). Mean duration of disease since first symptoms was 7.8 years, and disease duration since diagnosis was 5.9 years. Eight different IG preparations have been reported as current therapy. According to SF-36, patients’ quality of life is substantially impaired. Present data indicate some off-label use of IG (e.g. in MS) in patients with neurological AID. Quality of life in these patients is substantially compromised. Increasing patient numbers and extended follow-up periods will provide data on treatment concepts and disease development in AID patients.

Tài liệu tham khảo

Elovaara I, Apostolski S, van Doorn P, Gilhus NE, Hietaharju A, Honkaniemi J, van Schaik IN, Scolding N, Soelberg Sorensen P, Udd B. FNS guidelines for the use of intravenous immunoglobulin in treatment of neurological diseases: EFNS task force on the use of intravenous immunoglobulin in treatment of neurological diseases. Eur J Neurol. 2008;15:893–908.

Stangel M, Gold R. Administration of intravenous immunoglobulins in neurology. An evidence-based consensus: update 2010 [Article in German]. Nervenarzt. 2011;82:415–30.

Stangel M. New advances in the treatment of neurological diseases using high dose intravenous immunoglobulins. Ther Adv Neurol Disord. 2008;1:52–61.

Gold R, Stangel M, Dalakas MC. Drug insight: the use of intravenous immunoglobulin in neurology–therapeutic considerations and practical issues. Nat Clin Pract Neurol. 2007;3:36–44.

Jacob S, Rajabally YA. Current proposed mechanisms of action of intravenous immunoglobulins in inflammatory neuropathies. Curr Neuropharmacol. 2009;7:337–42.

Harbo T, Andersen H, Hess A, Hansen K, Sindrup SH, Jakobsen J. Subcutaneous versus intravenous immunoglobulin in multifocal motor neuropathy: a randomized, single-blinded cross-over trial. Eur J Neurol. 2009;16:631–8.

Kirch W, Stangel M, Pittrow D, Baumann U, Fasshauer M, Huscher D, Hensel M, Reiser M, Gold R, Borte M. Immunoglobulins for primary or secondary immunodeficiency or for immunomodulation in neurological autoimmune diseases: insights from the prospective SIGNS registry. J Public Health. 2012;20:289–96.

Ware Jr JE, Sherbourne CD. The MOS 36-item short-form health survey (SF-36). I. Conceptual framework and item selection. Med Care. 1992;30:473–83.

Greiner W, Claes C, Busschbach JJ, von der Schulenburg JM. Validating the EQ-5D with time trade off for the German population. Eur J Health Econ. 2005;6:124–30.

World Health Organisation (WHO). WHO-5 interpretation. Internet: http://www.cure4you.dk/354/index.php?m=2&a=e2&ei=4151. Accessed on 12 November 2011.

Fahrenberg J, Myrtek M, Schumacher J, Brähler E. Fragebogen zur Lebenszufriedenheit (FLZ). Handanweisung. Göttingen: Hogrefe Verlag für Psychologie; 2000.

Anonymous. SF-36. US population norm values (1998). Internet: http://www.sf-36.org/research/sf98norms.pdf. Accessed on 11 November 2011.

Schwarz S, Knorr C, Geiger H, Flachenecker P. Complementary and alternative medicine for multiple sclerosis. Mult Scler. 2008;14:1113–9.

Hughes RA, Donofrio P, Bril V, Dalakas MC, Deng C, Hanna K, Hartung HP, Latov N, Merkies IS, van Doorn PA. Intravenous immune globulin (10 % caprylate-chromatography purified) for the treatment of chronic inflammatory demyelinating polyradiculoneuropathy (ICE study): a randomised placebo-controlled trial. Lancet Neurol. 2008;7:136–44.

Hughes RA. Intravenous immunoglobulin for chronic inflammatory demyelinating polyradiculoneuropathy: the ICE trial. Expert Rev Neurother. 2009;9:789–95.

Merkies IS, van Nes SI, Hanna K, Hughes RA, Deng C. Confirming the efficacy of intravenous immunoglobulin in CIDP through minimum clinically important differences: shifting from statistical significance to clinical relevance. J Neurol Neurosurg Psychiatry. 2010;81:1194–9.

Merkies IS, Bril V, Dalakas MC, Deng C, Donofrio P, Hanna K, Hartung HP, Hughes RA, Latov N, van Doorn PA. Health-related quality-of-life improvements in CIDP with immune globulin IV 10 %: the ICE Study. Neurology. 2009;72:1337–44.

van der Pol WL, Cats EA, van den Berg LH. Intravenous immunoglobulin treatment in multifocal motor neuropathy. J Clin Immunol. 2010;30 Suppl 1:S79–83.

Cats EA, van der Pol WL, Piepers S, Notermans NC, van den Berg-Vos RM, van den Berg LH. New liquid intravenous immunoglobulin (10 % IVIg) for treatment of multifocal motor neuropathy: a prospective study of efficacy, safety and tolerability. J Neurol. 2008;255:1598–9.

European Medicines Agency (EMA). Kiovig. Human normal immunoglobulin (IVIg). Summary of opinion (Document EMA/CHMP/495548/2011). Internet: http://www.ema.europa.eu/docs/en_GB/document_library/Summary_of_opinion/human/000628/WC500107997.pdf. Accessed on 31 October 2011.

Morfeld M, Bullinger M, Nantke J, Brahler E. The version 2.0 of the SF-36 health survey: results of a population-representative study. Soz Präventivmed. 2005;50:292–300.

Bellach B, Ellert U, Radoschewski M. Der SF-36 im Bundesgesundheitssurvey - Erste Ergebnisse und neue Fragen. Bundesgesundheitsbl Gesundheitsforsch Gesundheitsschutz. 2000;43:210–6.

Kirch W, Gold R, Hensel M, Fasshauer M, Pittrow D, Huscher D, Reiser M, Stangel M, Baumann U, Borte M. Assessment of immunoglobulins in a long-term non-interventional study (SIGNS Study). Rationale, design, and methods. Med Klin (Munich). 2010;105:647–51.

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Tập 33

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