Meet Leslie ...
Leslie is a STAR now cured of sickle cell disease after a bone marrow transplant, but her journey to this cure through transplant has not been easy. Her story powerfully illustrates why more research is needed to improve transplant for sickle cell disease so that more children can be safely cured of this devastating disease.
As a young child Leslie unfortunately suffered many of the common complications of sickle cell disease. She was hospitalized for severe pain affecting multiple bones in her body and needed continuous morphine. She also had acute chest syndrome—a severe pneumonia that affects people with sickle cell disease and can be fatal.
“I was always on edge, I never knew when I might start having a pain crisis so I was always extra careful.”
When she was 8, an ultrasound of the blood vessels around her brain revealed that Leslie was at very high risk of having a stroke, one of the worst complications of sickle cell disease. To prevent this, she needed monthly blood transfusions. Fortunately, Leslie did not suffer a stroke while receiving transfusions every month, but over time a dangerous amount of iron bulit up in her body from these transfusions. MRI scans also showed abnormal narrowing in the blood vessels around her brain so she remained at high risk for a stroke. After seven years of monthly transfusions, Leslie and her family were interested in other options.
“I was interested in a transplant because the monthly transfusions were becoming more and more difficult.”
Leslie did not have a sibling who could be a bone marrow donor, but through the National Marrow Donor Program "Be The Match" she found a bone marrow donor match!
Leslie's transplant hospitalization began well—she had no major problems with her conditioning regimen (the chemotherapy medications given pre-transplant) or the infusion of her donor’s stem cell cells. However, two weeks after the stem cell infusion she suddenly lost the ability to talk and had a seizure. She had posterior reversible encephalopathy syndrome, a rare complication associated with transplant. Fortunately she recovered completely and was able to leave the hospital about a week later.
At home Leslie took multiple medications and came to the transplant clinic frequently. Testing confirmed that the transplant had cured her sickle cell disease-- all of her blood cells were now from her donor. Unfortunately she developed another transplant complication, graft versus host disease (GVHD)-- a serious problem in which Leslie’s new immune system from her donor attacks her own body. GVHD can cause rashes, joint tightness, mouth sores, as well as damage to internal organs. GVHD is more common in patients who receive transplants from donors who are HLA-mismatched and older.
Now over 2 years after her transplant Leslie is cured of sickle cell disease and attending college, but she is still battling GVHD. Leslie’s experience is not unique. Unfortunately, patients and families must undergo many hardships during and after a blood and marrow transplant.
“I would describe my entire transplant experience as a rollercoaster ride. There have been a lot of ups and downs through this entire journey. For me the most difficult part of my experience was being out of school for so long because I was really involved with my school.”
Research is key. Only through research will we be able to make transplant safer and available to more patients with sickle cell disease.
“People should care about research on transplant for sickle cell disease because right now a transplant is the only cure. We need more research to ensure the success with transplants.”
The mission of STAR is to help fund and lead this important research. Help support this much-needed research so children with sickle cell disease can be cured with fewer after effects.