The Clinton Herald, Clinton, Iowa

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Food & Health

January 15, 2014

Is there any treatment for severe sickle cell anemia?

DEAR DR. ROACH: My 39-year-old beloved niece just died of sickle cell anemia. Her life involved countless hospitalizations and blood transfusions every month to five weeks. She leaves behind a beautiful 5-year-old son to be raised by his father. On the day of my niece’s funeral, this son suffered a stroke. Luckily, he survived and appears to be doing well. Tests showed that he’d had an earlier stroke, which was undetected at the time. He’s only 5!

Words cannot describe the anguish of my family. Please advise what progress has been made in sickle cell research. I am somewhat familiar with bone marrow transplant; however, my niece could not benefit from it because of her stroke history — first stroke at age 9, second at 35, fatal at 39. She suffered first from the shutdown of her liver, followed by that last stroke. I believe it to be likely that her son may not benefit from a bone marrow transplant because of his strokes. — J.G.

ANSWER: I am very sorry to hear of your niece. I trained in the South Side of Chicago, and cared for far too many people with this devastating disease.

Sickle cell is one of the best-understood diseases there is. We know the gene that causes it, the specific amino acid in the specific protein that causes it, and how that changes a normal red blood cell into one with a sickle shape, blocking blood vessels and leading to irreversible death of tissues, especially in the brain (stroke), but also in the kidney, bones, spleen, heart and many others. But that understanding doesn’t mean we can treat it as well as we’d like.

You have identified the only known cure for sickle cell disease: transplantation of the cells that make red blood cells into the bone marrow. Genetic cures may be on the horizon, but as yet they aren’t available, and bone marrow transplant is limited — as you rightly point out, only some people are right for the procedure.

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