Sickle Cell Disease

After years of hydroxyurea treatment for pediatric patients with sickle cell disease, nearly 30% of children experienced a significant decrease in fetal hemoglobin levels, resulting in an increase in clinical complictions. By stimulating the expression of fetal hemoglobin (HbF), hydroxyurea helps patients with sickle cell disease (SCD). Improved clinical outcomes are correlated with higher HbF...

GBT021601, a potent sickle hemoglobin polymerization inhibitor, reduces abnormal angiogenesis and ineffective erythropoiesis, which may help sickle cell disease patients benefit from cell-based therapy, according to the results of a preliminary study. Damage from hypoxia results in abnormal blood vessel formation in sickle cell disease (SCD), which affects bone marrow and red blood cell production....

A new study suggests the importance of functional blood tests in identifying the success of sickle cell anemia using stem cell transplantation or autologous gene therapy. For those diagnosed with sickle cell disease (SCD), allogeneic hematopoietic stem cell transplantation (HCT) and ex vivo autologous gene therapies (GT) are among the most promising treatment approaches. However,...

Crizanlizumab was effective in reducing the number of priapic events in sickle cell disease patients and was both safe and well-tolerated among study participants. Priapism, defined as a painful, sustained erection of the penis, is observed in 35% of adult male sickle cell disease (SCD) patients. Priapism is thought to be mediated by vaso-occlusion in...

The influence of hydroxyurea on fertility is complex, and cryopreservation appears to be a good method for mitigating any deleterious effects, as indicated by the birth of at least one infant in a recent study cohort after using cryopreserved semen. Many men with sickle cell disease experience reproductive health issues. Pubertal delay is linked to...

In sickle cell anemia, red blood cells have a predominant role in the enhancement of phagocytic activity by mast cells, but in polycythemia vera, both cell types contribute to this phenomenon Stress erythropoiesis (SE) occurs when the physiological process of erythropoiesis is insufficient to fulfill the required production of functional red blood cells (RBCs), resulting...

Study shows 33 percent have nonproliferative retinopathy and 6 percent have proliferative retinopathy For pediatric patients with sickle cell disease (SCD), ophthalmologic complications include nonproliferative retinopathy (NPR) and proliferative retinopathy (PR), which occur in 33 and 6 percent, respectively, according to a study presented at the annual meeting of the American Academy of Ophthalmology, held...

Sickle cell disease is known to be associated with worse pregnancy outcomes and higher maternal mortality. However, a new study shows that racial disparities significantly contribute to adverse pregnancy outcomes in Black women. Sickle cell disease (SCD) is more likely to affect people of African descent. Additionally, it is known that SCD significantly increases the...

Medically reviewed by Dr. Samuel Sarmiento, M.D., MPH on Oct. 4, 2023 A new study finds that Black patients have a lower life expectancy when living with sickle cell disease compared to Non-Blacks. This is despite being insured by Medicare and Medicaid, and might point to healthcare disparities requiring further investigation. Sickle Cell Disease (SCD) affects about...