Anwendung von Immunglobulinen bei primären und sekundären Immundefekten und neurologischen Autoimmunerkrankungen

 

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Zusammenfassung

Humane Immunglobuline (IG, überwiegend IgG) werden zur Substitutionstherapie bei Patienten mit angeborenen (primären) Immundefekten (PID), sowie Patienten mit sekundären Immundefekten (SID), vor allem bedingt durch multiples Myelom oder chronisch lymphatische Leukämie angewendet. Ein weiteres Einsatzfeld besteht in der Immunmodulation bei neurologischen Autoimmunerkrankungen (NAID) wie Guillain-Barré-Syndrom, chronisch inflammatorisch demyelinisierende Polyneuropathie (CIDP) und multifokaler motorischer Neuropathie (MMN). Derzeit sind 16 IG-Präparate sind in Deutschland für die intravenöse oder subkutane Anwendung zugelassen. Aktuelle Daten zur Anwendung dieser Präparate werden derzeit in der SIGNS-Studie (Assessment of immunoglobulins in a long-term non-interventional study) in derzeit ca. 50 Studienzentren erfasst. In dieser nicht-interventionellen, prospektiven, offenen Versorgungs­forschungs­studie werden 550 Patienten mit bestehender oder neu initiierter Ig-Therapie hinsichtlich Therapiemuster (Präparatewahl, Dosierung etc.), Wirksamkeit (z. B. Infektrate bei PID und SID, Funktionalität bei NAID), Verträglichkeit, Lebensqualität und Therapiekosten über zwei Jahre beobachtet. Diese größte Studie ihrer Art soll zur Therapieoptimierung betroffener Patienten beitragen.

Abstract

Human immunoglobulins (IG, mostly IgG) are used as replacement therapy in patients with inherited primary immunodeficiencies, and in patients with secondary immuno­deficiencies often observed in multiple myeloma or chronic lymphocytic leukemia. Ig are also approved as immunomodulatory therapy in neurological autoimmune diseases (NAID) such as Guillain-Barré syndrome, chronic inflammatory demyelinating polyneuropathy (CIDP) and multifocal motor neuropathy (MMN). 16 different Ig preparations for intravenous and subcutaneous use are at the moment available in Germany. The SIGNS study (Assessment of immunoglobulins in a long-term non-interventional study) investigates the clinical use of these drugs under clinical practice conditions. In this non-interventional prospective open-label cohort study, 550 patients with new or maintenance Ig therapy are observed with respect to drug utilization, effectiveness, (i. e. number of infections in PID and SID, functionality in NAID), tolerability, quality of life and costs in approximately 50 sites throughout Germany (neurologists, pediatricians, oncologists, other) for at least two years. This largest study of its kind is expected to contribute to optimization of Ig therapy in the postmarketing setting.

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