Development of curative therapies for sickle cell disease
Yvette C. Tanhehco, Ghazala Nathu, Ljiljana V. Vasović
Abstract
Recent advances in managing Sickle Cell Disease (SCD) significantly improved patient survival and quality of life. Disease-modifying drug therapies such as hydroxyurea, L-glutamine, voxelotor, and crizanlizumab reduce pain crises and severe complications. Allogeneic hematopoietic stem cell transplantation using matched-sibling donors is currently the only standard curative option; however, only a small proportion of patients have such donors. Cord blood and haploidentical transplantation with a modified conditioning regimen have expanded the allogeneic donor pool, making the therapy available to more patients. Gene therapy is a promising cure that is currently undergoing clinical trials and different approaches have demonstrated efficacy. Multidisciplinary expertise is needed in developing the best treatment strategy for patients with SCD.