Search

Chronic complications of sickle cell disease take an increasing role in the management of patients due to their morbidity and mortality impact. The prevalence of chronic organ damages increases as the age of patients followed in France. Few organs seem unaffected by the disease. The natural history of chronic complication is highly variable from one patient to another, and the distribution of those manifestations throughout life, is different depending on their nature and pathophysiology. Thus we can, for example, distinguish SS patients presenting a "hyperhemolytic" phenotype associated with dense red blood cells that have a high risk of vascular complications including kidney disease, pulmonary hypertension, leg ulcers and priapism, from SC patients with high hemoglobin levels, who have a higher risk of retinopathy, osteonecrosis and sensory syndrome, probably related to hyperviscosity. Dependent on the age, we could also oppose cerebral vasculopathy responsible of ischemic stroke since childhood, and kidney damage which effects are visible more gradually with aging. Sickle cell disease is one of the most systemic pathologies contrasting with its monogenic characteristic and its inter- and intra-individual variability. A better understanding of pathophysiological mechanisms responsible for those complications is necessary to develop new preventive and therapeutic approaches.

Clinical trials are the foundation of therapeutic evaluation. Their interpretation and usefulness depend on their compliance with strict methodological rules. Departments of internal medicine admit patients with many different diseases, a variety that makes it difficult to organize and conduct clinical trials in their standard form. This article reviews the rules of clinical trials that are essential to their validity and reliability. The prospective randomized double-blinded clinical trial is the most scientifically rigorous method for assessing treatment. Rare diseases and those involving several specialties can and should be performed in accordance with the standard methodological rules, despite their particular aspects. This article considers ways to improve the participation of departments of internal medicine to future clinical trials.