Emerging drugs in randomized controlled trials for sickle cell disease: are we on the brink of a new era in research and treatment?

This article offers an overview of the key studies of new therapeutic options for SCD. We searched the PubMed database and Cochrane Database of Systemic Reviews for agents in early phase clinic trials and preclinical development.Expert Opinion: Although knowledge of SCD has progressed, patient survival and quality of life must be improved. Phase II and III clinical trials investigating pathophysiology-based novel agents show promising results in the clinical management of SCD acute events. The design of long-term clinical studies is necessary to fully understand the clinical impact of these new therapeutics on the natural history of the disease. Furthermore, the building of global collaborations will enhance the clinical management of SCD and the design of primary outcomes of future clinical trials. PMID: 31847604 [PubMed - as supplied by publisher]
Source: Expert Opinion on Investigational Drugs - Category: Drugs & Pharmacology Tags: Expert Opin Investig Drugs Source Type: research