Hydroxyurea Treatment Makes Economic Sense in Kids With SCA

Lara C. Pullen, PhD

September 03, 2013

Hydroxyurea treatment of young children with sickle cell anemia results in a significantly lower overall medical care cost when compared with placebo. The total estimated annual cost for the hydroxyurea group was $11,072 per patient compared with $13,962 per patient in the placebo group (P = .038). This savings is likely to increase as the children age, further justifying widespread use of hydroxyurea in this patient population.

Winifred C. Wang, MD, from St. Jude's Children's Research Hospital in Memphis, Tennessee, and colleagues present the economic analysis of the Hydroxyurea to Prevent Organ Damage in Children With Sickle Cell Anemia (BABY HUG) trial data in an article published online September 2 in Pediatrics. They identified substantial cost savings associated with the use of hydroxyurea treatment even in very young children who were not selected for disease severity.

"You get benefit and you decrease cost. How beautiful is that?" exclaimed Michael A. Bender, MD, PhD, pediatric hematology/oncology specialist at Seattle Children's in Washington, in a telephone interview with Medscape Medical News.

"I would say [all patients with sickle cell anemia] should be on the drug. I am a hydroxyurea zealot," emphasized Matthew Heeney, MD, clinical director, Dana-Farber/Boston Children Cancer and Blood Disorder Center in Massachusetts, also in a telephone interview with Medscape Medical News.

Dr. Wang and colleagues estimated costs using the 2009 MarketScan Multi-state Medicaid Database for children (age 1 - 3 years) with sickle cell disease who participated in the BABY HUG trial. Outpatient expenses were calculated on the basis of the treatment schedules for 1- to 3-year-olds in the BABY HUG trial and a standard treatment schedule for this patient population. During the study, 232 children in the hydroxyurea group were hospitalized, as were 324 children in the placebo group. Median length of stay was similar in the 2 groups.

"They have done a pretty good job at trying to estimate the true costs," explained Dr. Heeney. He went on to describe the difference in the cost between the 2 groups as "striking."

Although the article was purely an economic analysis, it reinforces that treatment with hydroxyurea not only is helpful for patients but also helps conserve healthcare dollars. Hydroxyurea is available as a generic and is a relatively inexpensive medication. Therefore, outpatient treatment translates into considerable savings when compared with the high cost of increased hospitalizations in the placebo group.

Although many physicians have long believed the hydroxyurea treatment results in medical cost savings, both Dr. Bender and Dr. Heeney expressed pleasure in seeing a well-designed study that produced data documenting this belief.

The need for the study rests on the fact that treatment with hydroxyurea requires increased clinic visits. These visits may be daunting to patients and parents and be perceived as expensive. The results from the study point out that the inconvenience and cost of increased outpatient visits are more than offset by a decrease in the pain and cost of hospitalizations.

"This gives us another arrow in our quiver to really try and spread the use of this drug for this disease," elaborated Dr. Heeney.

The authors, Dr. Bender, and Dr. Heeney have disclosed no relevant financial relationships.

Pediatrics. Published online September 2, 2013. Abstract


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