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By Elizabeth Jane Walker
The results of the Baby HUG study are music to the ears of Emmanuel and Pamela Madu and to other parents of young children with sickle cell anemia.
When Elechi Madu practices the violin, the 6-year-old produces the requisite squeals and squeaks of a beginning musician. But to Emmanuel and Pamela Madu, those halting melodies are sweeter than any symphony. The Madu home—once resounding with Elechi’s cries of pain—now reverberates with music, laughter and the little girl’s excited chatter about soccer and gymnastics.
The transformation occurred as a result of treatment Elechi received at St. Jude Children’s Research Hospital.
In December 2004, Emmanuel and Pamela were devastated to learn that their beautiful, newborn daughter had the most severe form of sickle cell disease. “I don’t think there’s anything as scary as knowing that your child has something that you cannot fix,” Pamela says. “It was an overwhelming, indescribable feeling.”
Elechi is one of about 100,000 Americans with the disorder. Individuals with this inherited disease have red blood cells that may be shaped like crescents instead of discs. Instead of moving smoothly through the circulatory system, the sickle-shaped cells clog blood vessels, triggering episodes of extreme pain and causing other dangerous complications that range from organ damage and stroke to a pneumonia-like illness known as acute chest syndrome.
“Within a year-and-a-half, Elechi had at least six bouts of acute chest syndrome,” Pamela recalls. “It seemed like she had pneumonia back to back to back. She would get a blood transfusion and stay in the hospital for days. She was tired and exhausted all the time.”
When Elechi was 3 years old, doctors at St. Jude prescribed a drug called hydroxyurea, a cherry-flavored liquid that Elechi swallows once each day. The results have been stunning.
“Now she is full of energy,” Pamela says. ‘She went from being hospitalized many times in a year to having only one hospitalization in the last 2½ years. Hydroxyurea has been the answer to our prayers.”
St. Jude researchers have been studying the effects of hydroxyurea for about 16 years, according to Winfred Wang, MD, of Hematology. Soon after the U.S. Food and Drug Administration approved the drug for use in adults with severe sickle cell disease, Wang and his colleagues began to investigate the possibility of its use for children. In 2002, their HUSOFT study proved the feasibility of giving liquid hydroxyurea to infants and indicated that the drug might help preserve spleen function.
As a result of the study, the National Institutes of Health began a project to study the use of hydroxyurea in young children. Wang was principal investigator of the six-year study, deemed Baby HUG, which involved 193 children at 13 centers across the country. Children were randomly assigned to receive daily doses of either hydroxyurea or a placebo for two years. Neither the families nor their clinicians knew which children were receiving the drug and which were receiving the placebo.
St. Jude Pediatric Nurse Practitioner Lynn Wynn organized the study coordinators nationwide, while Lane Faughnan, RN, served as the local Baby HUG coordinator. Wang attributes Wynn and Faughnan with helping families understand the importance of the research.
“Our patients and their families were diligent about their involvement in the study,” Wang says. “The study was demanding, because they had to be seen every couple of weeks at the beginning and every four weeks thereafter for two years. Some participants experienced some sickle-cell–related problems; we suspected and later learned that most of those individuals were on the placebo. But even those families stuck with the study instead of dropping out. Every patient at St. Jude remained in the study for the entire two years.”
Pamela says the education and personal attention she received at St. Jude helped her cope with the stresses of having a child with severe sickle cell anemia.
“My husband and I learned so much from the people at St. Jude,” she says. “It was like Biology 101 all over again. They taught me step by step everything I needed to know to take care of Elechi. I would email Lane Faughnan in the middle of the night, and she would write me back immediately. My child wasn’t a number to them—they genuinely cared about Elechi and our family.”
The couple’s experiences at St. Jude inspired Pamela and her husband to make career changes. Emmanuel is pursuing a doctorate in cancer research while Pamela completes the prerequisites for nursing school.
As a result of Baby HUG, researchers discovered that hydroxyurea reduces the incidence of pain crises, acute chest syndrome and other symptoms, as well as the number of days children spend in the hospital. Wang and his colleagues published their findings in the medical journal The Lancet this summer.
“We found that it’s effective, beneficial and reasonably safe to give hydroxyurea to young children with sickle cell anemia,” he says. “We think the drug should ideally be prescribed for all sickle cell anemia patients. It has the potential to dramatically improve the quality of life for an entire generation of patients with this disease.”
Hydroxyurea works by inhibiting DNA production. Created as an anti-cancer drug decades ago, the drug is relatively inexpensive—costing about $1 a day—and easy to administer, which makes it a viable option for both children and adults with sickle cell anemia.
All St. Jude Baby HUG participants have agreed to participate in a follow-up study that should continue through 2016. Children in this study are offered the opportunity to take hydroxyurea daily. Participants who received hydroxyurea in Baby HUG received relatively low doses of the drug during that study. The current research project will address possible long-term benefits of continuing treatment with higher doses.
As part of the study, Elechi continues to make regular visits to St. Jude, where she delights the medical staff with her energy and intelligence.
A budding gymnast and soccer player, Elechi frequently emulates her older brother, Chinua. When Chinua learned to read, she quickly followed suit; when he took up the violin, she became interested in music, as well. Although she has not yet begun first grade, Elechi’s reading skills now approach a third-grade academic level. Her musical repertoire includes such standards as “Twinkle, Twinkle, Little Star” and a special song that she composed for her daddy’s birthday.
Keeping up with Elechi is both exhausting and exhilarating, but Pamela says she wouldn’t change a thing.
“I honestly don’t know where her energy comes from,” she says, laughing. “The change is so phenomenal. Now she’s growing, she’s active and she’s doing great. It’s such a blessing to our entire family.”
Promise magazine, Autumn 2011