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Affecting about 70,000 to 100,000 Americans, sickle cell anemia (also called sickle cell disease) is the most common kind of inherited blood disorder. However, sickle cell anemia is underrecognized, and many people may be unfamiliar with this condition.
In light of September being National Sickle Cell Awareness Month, we talked with Susan Kelly, MD, a pediatric stem cell transplant physician at AdventHealth for Children to better understand the facts about sickle cell anemia.
1. Sickle Cell Anemia Is Present at Birth
Sickle cell anemia is an inherited, genetic blood disorder. “In this disease, the red blood cells are misshapen when stressed and get stuck going through small blood vessels, which causes stroke, damage to many organs, including kidneys and lungs, and severe pain,” Dr. Kelly says.
Today, all newborns are screened for sickle cell anemia, which empowers families to get started with treatment before the debilitating side effects of this condition occur. “When children are born, they’re not yet symptomatic because they still have protective fetal blood. As they grow, the symptoms appear. The damage to organs increases over time, so the best patients to treat are the youngest ones who are not yet as affected by the disease.”
2. Sickle Cell Anemia Is Severely Debilitating
Sickle cell anemia is a life of chronic pain and frequent hospitalizations. “I wish people understood how serious it is and how debilitating it is for people to have this disease; how difficult it is to live with severe pain, the risk of a stroke and your organs being damaged by your own blood, every day,” Dr. Kelly says.
Most patients need frequent blood transfusions as well, but even this treatment can be complicated and painful. “If you’re getting blood transfusions once a month, you can eventually develop antibodies limiting your ability to receive blood,” Dr. Kelly points out. “With chronic blood transfusions, the iron in the blood can damage your heart, lungs, liver and other organs over time.”
3. The Life Expectancy of Sickle Cell Anemia Is 40 Years Old
Even with medical intervention, people who have sickle cell anemia may not live to see middle age.
“During childhood, up to half of children who have sickle cell disease will have had a silent or clear stroke,” Dr. Kelly explains, which is a serious event that can be life-threatening. “Despite having the best current medical care in America, the life expectancy for sickle cell anemia is just over 40 years old, which is unacceptable.”
Fortunately, there is a cure for sickle cell anemia, and the sooner children receive it, the healthier they’ll be, and they can have a normal life expectancy, too.
4. Sickle Cell Anemia Can Be Cured by a Bone Marrow Transplant
Unlike treating cancer, no medicine cures sickle cell disease. “Medication management with drugs like hydroxyurea can improve the quality of life for patients by creating fetal blood that can’t take on that misshapen form, but it can’t cure the disease,” Dr. Kelly says.
Fortunately, though, sickle cell anemia can be completely cured through a bone marrow transplant. “Until recently, bone marrow transplant was viewed as being reserved for only the sickest patients, but now, if you have a sibling who’s a matched donor, you have about a 95% chance of surviving the transplant and being cured of sickle cell disease.”
A bone marrow transplant is not a temporary solution to sickle cell disease. “Bone marrow transplant is an actual cure,” Dr. Kelly explains. “The patient with sickle cell disease gets bone marrow from a brother or sister who’s a match, and the transplant then creates new, healthy blood in their body. Even siblings with sickle cell trait can be a donor.”
“It’s a long and difficult process, but it prevents any further complications from sickle cell and takes away the pain and risk of stroke — it’s a true cure,” Dr. Kelly says.
5. New, Half-Matched or Haploidentical Bone Marrow Transplants Expand Donor Options
“Full siblings have a 1-in-4 chance of being a match for bone marrow transplants, whereas anyone else has a very small chance of being a perfect match,” Dr. Kelly says. Needing a brother or sister who is a full match can be limiting for people, but a new type of bone marrow transplant is changing that fact.
“In the past, when we would screen families, maybe 1-in-12 or 14 kids would have a sibling who was also a match, who didn’t also have sickle cell disease and able to be their donor,” Dr. Kelly says. “Today, there’s a new, very promising type of transplant called a haploidentical transplant, where we use a half-matched donor, whether they’re a sibling, parent or other close relative who’s only half a match.”
This new type of transplant gives donor options to people who may not have options before, and the success rate is quickly catching up to what it is for full siblings. Recent studies show successful outcomes in more than 90% of patients.
“This is such an exciting breakthrough because almost all people have a half-match donor. Either a parent or sibling, or even sometimes a first cousin, aunt or uncle could be a half a match.”
“We have the possibility to cure children with sickle cell disease, to allow them to live healthy, happy lives with a normal life expectancy through bone marrow transplant either from a match-related donor or a haploidentical donor — it’s truly amazing,” Dr. Kelly says.
The most recent two patients that Dr. Kelly performed a haploidentical transplant for received bone marrow from their mother. “She gave birth to them, and now she’s their bone marrow donor as well. She gave life to them twice.”
6. Treatment Is Now Less Risky Than Simply Living With Sickle Cell Anemia
Through continued clinical trials and research, bone marrow transplants are now a safer option than living with sickle cell anemia.
“Bone marrow transplant is a true cure. It’s not a bandage, and it’s not just helping, it cures sickle cell anemia,” Dr. Kelly says. “Our treatments are so sophisticated we can keep the vast majority of patients safe during the treatment.”
“It’s actually safer than having the sickle cell disease. A child is more likely to die of sickle cell disease in a 5-to-10-year time span than from their transplant.”
Dedicated to Curing Kids With Sickle Cell Anemia
At AdventHealth for Children, kids are being cured of sickle cell anemia and going on to lead healthy, fulfilling lives — and it takes a team to make that possible.
“In our stem cell transplant program, we have a whole team of people who dedicate every moment of their day to helping children who need lifesaving transplants,” Dr. Kelly says.
“We treat each patient like they are part of our family, and every person on our team is so dedicated to these children and their families and is willing to go so far above and beyond for them, every day.”
To learn more about pediatric bone marrow transplants or get started with treatment for your child, we’re here to walk this path with you. Learn more about AdventHealth for Children or reach out to us today.