Sherif Badawy, MD from Ann & Robert H. Lurie Children’s Hospital of Chicago speaks about A systematic review of quality of life in sickle cell disease and thalassemia after stem cell transplant or gene therapy.
Link to Article:
https://pubmed.ncbi.nlm.nih.gov/33496753/
Summary
Patients with sickle cell disease (SCD) and thalassemia face a variety of complications during their lives, which result in impairment in various aspects of health-related quality of life (HRQOL). Curative therapies for patients with SCD and thalassemia, such as hematopoietic stem cell transplant (HSCT) and gene therapy, are gaining popularity; however, the impact of these treatments on different HRQOL domains is unknown. Our aim was to assess the most current data on the impact of HSCT and gene therapy on HRQOL in patients with SCD and thalassemia. A thorough search of medical literature databases was carried out. A total of 16 studies involving 517 participants (thalassemia, n = 416; SCD, n = 101) met inclusion criteria (thalassemia, n = 9; SCD, n = 6; both, n = 1). In most HRQOL domains, HSCT was correlated with small to significant positive effects (Cohen’s d; mean = 0.47; median = 0.37; range, 0.27-2.05). HSCT was frequently linked to significantly positive effects in the physical and emotional HRQOL domains in thalassemia patients (median d = 0.79 and d = 0.57, respectively). In SCD, HSCT was linked to significant improvements in all HRQOL domains. New evidence suggests that gene therapy for thalassemia and SCD improves HRQOL outcomes across several domains. In 13 studies, the evidence standard was moderate (81 percent ). In patients with SCD and thalassemia, HSCT has a positive influence on many HRQOL domains; however, further longitudinal studies are needed to determine the long-term effects of these effects. It’s important to report HRQOL results from ongoing gene therapy or gene-editing trials in SCD and thalassemia to better understand the therapies’ benefits.