Hope for Babies with Sickle Cell Disease

    It’s like slamming a car door on your hand—then multiply that by 10.

    Imagine your 8-month-old baby suffering a pain crisis like that one. Why? Because of sickle cell disease.

    You’ve heard of sickle cell disease, and its most severe type, sickle cell anemia. About 70,000 people in the United States have various forms of sickle cell disease. One out of 400 African-American babies is born with it. But labeling it as a purely African-American disease is a mistake; Middle Eastern and Caucasian people can carry and inherit the genes that cause sickle cell disease. Doctors are also seeing rising numbers of the disease in the booming U.S. Hispanic population. However, many non-sufferers don’t realize how devastating sickle cell disease can be. It can cause pain crises, infections, lethal pneumonia and organ damage. And the devastation starts in infancy.

    When babies are born with sickle cell disease, they are protected for about six months by fetal hemoglobin. However, as the level of fetal hemoglobin drops during the first year of life, the disease starts its damaging effects. A medicine called hydroxyurea can keep hemoglobin levels elevated in older children and adults.

    St. Jude researchers wanted to know: Will the drug work on babies too? Several years ago, they created a study to find the answer.

    “We wanted to try to apply this to younger children and see if the treatment that worked for adults and older patients would work just the same for the babies,” says Jane Hankins, MD, St. Jude Hematology-Oncology. “Could we prevent pain crises? Could we prevent pneumonia? Could we prevent organ damage?

    “Pneumonia is dramatic in sickle cell disease because it progresses so fast,” she continues. When people without sickle cell disease get pneumonia, they usually just take antibiotics and stay home. But in individuals with sickle cell disease, pneumonia often progresses from one lung to the next.

    “It can take both lungs very quickly,” Hankins says. “I’ve had many patients who quickly went from having a little bit of cough to needing oxygen and a few hours later to being intubated in the ICU.”

    The St. Jude feasibility study called Hydroxyurea Safety and Organ Toxicity (HUSOFT) was designed to see if babies could tolerate taking hydroxyurea. Clinicians weren’t sure the babies would take the drug, even in a liquid preparation. But the results were encouraging.

    DaSean Hill was one of those babies. Today, he proudly prepares to tie his belt around the waist of his taekwondo uniform. He studies martial arts five days a week, and he loves it. This straight-A student is a shining example of how hydroxyurea’s protective effects can lead to a normal, healthy life. The boy started in the HUSOFT study when he was only a year old; in the nine years since, he has never fallen victim to the terrible pain that can come with sickle cell disease.

    “DaSean has never had a crisis,” asserts his mother, Tashonda Crenshaw. “And he’s on a normal growth path. I honestly feel this medicine has kept him from developing these problems.”

    Initially, Tashonda was hesitant to put her baby in the HUSOFT study.

    “He’s my only child; as a mother, you are frightened to think of giving your baby anything that hasn’t been thoroughly tested already for years and years,” she says. “But he’s done so well. And he’s never missed a day of school. Not one.”

    Results of the HUSOFT project led researchers to conduct a multicenter, long-term study of babies and hydroxyurea, named BABY HUG.

    “The results of the HUSOFT trial showed that infants with sickle cell anemia not only tolerated hydroxyurea, but could benefit from it just as older children and adults do,” says Winfred Wang, MD, director of the St. Jude Comprehensive Sickle Cell Center. “In addition, these infants may have had less organ damage and better growth than untreated patients.”

    Hydroxyurea is an affordable medication that could help babies with sickle cell disease in lower-income countries around the world. The drug is already given free of cost by the Brazilian government to adults and children older than 3. The St. Jude study may eventually lead to infants in Brazil and in other countries receiving hydroxyurea as well.

    Hankins feels that could certainly be the case. “If the BABY HUG study shows that hydroxyurea prevents damage to the organs—the spleen, the brain (strokes or other lesions), the kidneys—then that’s going to revolutionize the way we treat patients with sickle cell anemia,” she says. “From a very early age, everybody would get hydroxyurea and keep taking it.”

    And that could quite possibly lead to a world in which all babies with sickle cell disease have a much better chance at growing up to lead normal, pain-free lives. What better way for St. Jude researchers to give babies a HUG that lasts?

    Reprinted from Promise magazine, spring 2006

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