Immunoglobulins for primary or secondary immunodeficiency or for immunomodulation in neurological autoimmune diseases: Insights from the prospective SIGNS registry

Aim: Outside the context of clinical trials, there is a lack of data on the management and long-term outcomes of patients with primary or secondary antibody deficiency (PID and SID), or those with neurological autoimmune diseases (AID) treated with immunoglobulins (IG). Methods: SIGNS is a large-scale prospective observational cohort study with up to 550 patients in currently 50 sites throughout Germany. It investigates treatment patterns, effectiveness, quality of life and long-term outcomes of IG administered for PID, SID or AID. The study identifier at ClinTrials.Gov is NCT01287689. Results: Of 306 patients currently included (1 Nov 2011), 108 had PID (49 children), 147 SID and 51 neurological AIDs. First symptoms had occurred 7.9 (PID children), 10.4 (PID adults), 5.5 (SID) and 7.2 years (AID) before entry, and disease duration since diagnosis was 4.8 (PID children), 7.0 (PID adults), 5.3 (SID) and 5.9 years (AID). Newly initiated IG therapy was reported in 45% (PID), 54% (SID) and 56% (AID). 13 different IG preparations were utilized at inclusion. In PID, only 25.0% of children and 24.1% of adults had no infection in the past 12 months; while 3 serious bacterial infections (1 meningitis, 2 pneumonia) were reported in children, and 1 in adults. With respect to AID, 21 patients had chronic inflammatory demyelinating polyneuropathy (CIDP), 7 had multifocal motor neuropathy (MMN), 11 multiple sclerosis (MS), 6 myasthenia gravis, 2 myositis, 4 other (no cases of Guillain Barré Syndrome). Quality of life of patients in all groups was reduced as assessed by ED-5D and SF-36. Conclusions: A broad spectrum of indications and IG preparations is documented in SIGNS. It is expected that the registry will contribute to optimization of therapy in this diverse patient population. © Springer-Verlag 2012.