Child Health | 4 years ago

Shining a Much-Needed Light on Sickle Cell Disease

During Sickle Cell Disease Awareness Month, we’re bringing attention to this chronic disease affecting thousands of children in the United States. See how Levine Children’s Hospital is stepping up with curative care.

If you don’t know much about sickle cell disease, you’re not alone.

“Sickle cell disease is probably the least known major disease in the United States,” says Daniel McMahon, MD, director of the sickle cell clinic at Levine Children’s Hospital. “There are 100,000 people in the United States with sickle cell disease, yet there is very little public awareness.”

September is National Sickle Cell Awareness Month, focusing attention on the need for treatment and research of this inherited blood disease that can cause chronic pain, stroke, organ damage or blindness.

Fortunately, there is a cure for sickle cell disease: blood and marrow transplant (BMT). A BMT replaces unhealthy blood-forming cells with healthy ones – either from a person’s own body or from a donor. This procedure is usually only performed after other courses of treatment have been exhausted.

As the only pediatric BMT unit in Charlotte, LCH is experienced in all types of BMT procedures, including those from donors whose cells are only “half-matched” to the patient. Our doctors have performed more than 10 BMTs for sickle cell patients in just the past two years.

“Many patients have come from as far south as Louisiana, and as far up north as New York, to come here for their transplants,” says Javier Oesterheld, MD, medical director for pediatric hematology, oncology, and blood and marrow transplantation.

A BMT isn’t always the best option for patients with sickle cell disease. For them, new medications can help manage their condition. A drug recently approved by the FDA called L-glutamine can be used to reduce pain and other complications.

Another promising advance, says Dr. McMahon, is gene therapy. “In the near future, we will be able to correct the genetic abnormality such that these individuals would no longer have sickle cell disease. Now, that research is in its infancy, but the early results are promising,” says Dr. McMahon.

Having practiced at Carolinas HealthCare System for 22 years, Dr. McMahon has seen treatments evolve. He’s also followed patients as young as two months old all the way until their late teens.

“We have a lot of children that I've known for their whole lives. I’ve seen some go through strokes, meningitis, serious infections, even require ICU care, and they’ve grown up and gone to college. One’s in medical school,” says Dr. McMahon. “It’s rewarding and truly inspiring.”