G.P.256: The German patient registry for inclusion body myositis

Abstract

Although inclusion body myositis (IBM) is the most common acquired myopathy in patients aged over 50 years, it is a very rare disease with an estimated prevalence between 4.5 and 9.5/million rising up to 50/million for individuals over 50 years with high variability in different populations and ethnic groups. Patients suffer from slowly progressive muscle weakness and pharyngeal impairment resulting in severe disability. No causative treatment is currently available but new therapeutic strategies are investigated and innovative clinical trials are considered for the next years. A German patient registry for IBM is being established to overcome the bottlenecks of data fragmentation, lack of harmonization and lack of trial readiness for this disease. We aim to include harmonized datasets of preferably all German patients diagnosed with IBM based on standardized clinical and histopathological findings (revised Hilton–Jones criteria). A harmonized dataset including clinical data and muscle biopsy reports is recorded using a dual patient and professional online report system ( www.ibm-register.de ). National legislation, data protection laws and ethical recommendations are strictly followed in this process. Approval of the project by the Munich University Ethics Board is awaited within 2014. The registry will start upon IRB approval and aims at collecting high-quality, curated and regularly updated longitudinal data of the majority of German IBM patients in the mid- and long-term. In addition, the registry may help to address research questions like prevalence, natural history and disease characterization. The IBM registry will provide a unique opportunity not only to effectively recruit German patients for international clinical trials and to support the translation of new therapies from “bench to bedside”, but also to improve patient care by assessing standards of diagnosis and care.