NHLBI IN THE PRESS

Physicians publish guidelines for sickle cell disease transplant therapy

A patient speaks with her doctor in a medical office, while wearing a mask.

Eight clinical recommendations are now available to answer questions about a common sickle cell disease therapy, which transfers blood-forming stem cells from a matched donor to help someone living with sickle cell disease produce healthy red blood cells. The clinical guidelines appear in Blood Advances, a journal published by the American Society of Hematology (ASH).  
 
The first time 
hematopoietic stem cell transplantation occurred was almost 40 years ago. Since then, research has steadily advanced. However, while eight in 10 patients who have this type of transplant have success, questions remain. Should patients at higher risk for neurological complications, like stroke, receive a stem cell transplant? What about patients who have frequent chest pain? And when should a patient have a transplant? 

Hematologists and researchers, including experts from NHLBI, collaborated to answer these and other questions based on the best available evidence. They also outline ways to advance sickle cell disease research, such as by creating patient registries and conducting randomized controlled trials, to help physicians and patients make informed treatment decisions.