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New risk score predicts person’s chance that transplant will work to treat sickle cell disease

Children who have matched sibling donors are helped most

Age1 - 48 years old
Number of Participants1,425
Research GoalImprove Survival

What is sickle cell disease? 

BMT sometimes cures sickle cell disease People with sickle cell disease have a gene that makes C-shaped red blood cells, instead of round cells. The C-shaped cells get stuck in small blood vessels and cause pain, strokes and other problems. 

BMT sometimes cures sickle cell disease

Before BMT, a person’s own blood and marrow cells are wiped out by medicines. BMT replaces these cells with healthy cells donated by another person. When BMT works, the healthy cells stay and the person is cured of sickle cell disease. 

However, sometimes the healthy cells don’t grow like they should in the bone marrow, and the person isn’t cured. 

Who should get a transplant for sickle cell disease? 
Researchers studied the medical records of about 1,400 people who got BMT for sickle cell disease. Any of these 3 things raised the likelihood that BMT would work to cure the sickle cell disease: 
  • Patient was aged 12 or younger during transplant 
  • Donor was the patient’s brother or sister (people will sickle cell trait can still donate for someone with sickle cell disease) 
  • Donor was well matched (had similar genes) to the patient 

Using complex math, doctors developed a risk score to predict who would be healthiest after BMT. The risk score can help people with sickle cell disease and their families make treatment decisions. 

For example, researchers found BMT was most helpful for children who got BMT when they were aged 12 or younger if their disease was severe enough to need a BMT and whose donor was a matched brother or sister. Three years after BMT, 92% (9 out of 10) of these children were alive and had healthy cells. 

Ask your doctor 

  • What is my risk score? 
  • What are the potential benefits and harms of BMT and other treatments?