Category: News

Pain Narratives in Children with Sickle Cell Disease (SCD)

This study aims to assess wellbeing (i.e., pain, anxiety, distress) in children and young adults who have, and have not, undergone a transplant. We are recruiting individuals with SCD ages 8-25, and their parent/caregiver from across Canada to partake. This study has been designed to be completed online to ensure convenience and ease for all …

Advancing Treatments and Cures for Sickle Cell Disease

The NHLBI launched the Cure Sickle Cell Initiative in 2018 to support the development of gene-based cures for sickle cell disease (SCD) that will work for all patients, including those ineligible for bone marrow transplants. Researchers involved in the Cure Sickle Cell Initiative helped develop an accurate, affordable, and easy-to-use device for point-of-care diagnosis of SCD in …

CDC’s Sickle Cell Data Collection Program Expands to Nine States

The Centers for Disease Control and Prevention’s (CDC) Sickle Cell Data Collection program gathers health information from multiple sources to determine how many people live with the disease and monitor changes related to their health over time.  In 2020, CDC funded the expansion of the program from two to nine states to collect essential data …

MARAC Statement: Temporary Suspension of Clinical Trials

March 1, 2021 – The Sickle Cell Disease Association of America’s Medical and Research Advisory Committee (MARAC) is aware of the announcement on February 16, regarding the temporary suspension of bluebird bio clinical trials of LentiGlobin Gene Therapy for Sickle Cell Disease and the pause of all commercial use of bluebird bio European gene therapy. …

A systematic review of quality of life in sickle cell disease and thalassemia after stem cell transplant or gene therapy

Abstract Patients with sickle cell disease (SCD) and thalassemia experience several complications across their lifespan that lead to impairment in different health-related quality of life (HRQOL) domains. There is increasing interest in curative therapies for patients with SCD and thalassemia, including hematopoietic stem cell transplant (HSCT) and gene therapy; however, the effect of these therapies …

Influence of Nutrition on Disease Severity and Health-related Quality of Life in Adults with Sickle Cell Disease: A Prospective Study

Abstract Background & aims: Sickle cell disease (SCD), a genetic disorder resulting from the presence of a mutated hemoglobin S (HbS), has a worldwide distribution and causes significant morbidity and mortality in children and adults. Few studies addressed the determinants of SCD severity in adults; therefore, we investigated the impact of nutrition on the outcome of …

Transfusion and Cellular Therapy in Pediatric Sickle Cell Disease

Abstract Red blood cell (RBC) transfusion is critical in managing acute and chronic complications of sickle cell disease. Alloimmunization and iron overload remain significant complications of transfusion therapy and are minimized with prophylactic Rh and K antigen RBC matching and iron chelation. Matched sibling donor hematopoietic stem cell transplant (HSCT) is a curative therapeutic option. …

The role of hematopoietic stem cell transplantation for sickle cell disease in the era of targeted disease-modifying therapies and gene editing

Abstract Sickle cell disease is one of the most common, life-threatening, non-communicable diseases in the world and a major public health problem. Following the implementation of simple preventive and therapeutic modalities, infant mortality has almost been abolished in high-income countries, but only a small amount of progress has been made in improving survival in adulthood. …