Endari significantly reduced blood transfusion requirements for Sickle cell patients
Plastic bags with blood in container
Post-hoc analyses of the Endari (L–glutamine) phase 3 clinical study in SCD presented at ASH showed a significant reduction of transfusion requirements for Sickle cell patients compared to placebo group. Patients that were assigned to Endari required approximately 43% fewer units of red blood cells. Results showed that there was a significant difference in the number of units of RBCs transfused in the Endari treatment arm than in the placebo arm; 2.86 units per patient-year in the Endari group vs. 5.38 units per patient-year in the placebo group.
Endari (L–glutamine) was approved by FDA in July 2017 to treat SCD patients, age 5 and older. This decision made Endari the first approved treatment for children with sickle cell disease and the first new treatment for adults with the disease in almost 20 years.
About Sickle Cell Disease (SCD):
Sickle cell disease is a group of inherited and lifelong health conditions that affect the red blood cells. The most serious condition is called sickle cell anemia. It is common among African and Caribbean family background population. In the United States, it affects between 70,000 to 100,000 Americans.
Patients that suffer from sickle cell disease produce abnormal red blood cells that have a sickled shape, that’s why it is called sickle cell disease, the abnormal shape of the red blood cells make them unhealthy leading to different complications as a result of blood vessels blockage.
