Hydroxyurea still not used enough, properly in children with sickle cell disease

51st ASH Annual Meeting

Despite it being the standard of care for treating vasocclusive disease associated with sickle cell disease, there are still barriers to widespread use of hydroxyurea among patients and pediatric hematologist/oncologists, according to data presented at the 51st ASH Annual Meeting.

“Our survey suggested there is a substantial variation in the utilization of hydroxyurea in children and we show that many providers are recommending the therapy for complications other than pain despite relative evidence showing its efficacy for these complications,” Amanda M. Brandow, DO, MS, assistant professor of pediatrics at the Medical College of Wisconsin Children’s Research Institute of the Children’s Hospital of Wisconsin, said during a press conference. “Provider and patient barriers do exist and need to be addressed.”

Brandow and colleagues conducted a survey to examine the practice patterns of hydroxyurea use and to identify possible barriers of its use in children with sickle cell disease.

A survey including questions on provider demographics, characteristics of sickle cell disease, criteria for hydroxyurea prescription and barriers to utilization of hydroxyurea was sent to members of the American Society of Pediatric Hematology/Oncology; 350 surveys were returned of which 63% of physicians stated they care for children with sickle cell disease.

Spearman correlation was used to evaluate factors associated with hydroxyurea utilization.

Provider utilization

The median number of years respondents had been in practice was 12; 50% of respondents were women and 84% practiced in an urban teaching hospital.

Of the respondents who had more than 100 patients with sickle cell disease in their practice (50%); 43% stated these patients make up more than 20% of their practice.

Despite the fact that 90% of respondents stated hydroxyurea was effective or very effective for pain prevention, only 8% of respondents had 50% to 90% of their patients with sickle cell disease on hydroxyurea.

Fifty-four percent of respondents had 10% to 30% of their patients with sickle cell disease on hydroxyurea. Ten percent of respondents had less than 10% of patients on hydroxyurea.

Respondents said that apprehension about future reproductive issues were one reason they did not prescribe hydroxyurea to children.

In some cases, hydroxyurea was not prescribed due to patient refusal. Twenty-six percents of respondents said that more than 20% of patients refused hydroxyurea for:

• Fear of cancer (51%).
• Other adverse effects (62%).
• Did not want to take medication (49%).
• Did not think it would work (17%).
• Required lab monitoring (28%).

For more information:

• Brandow AM. #242. Presented at: 51st ASH Annual Meeting and Exposition; Dec. 4-8, 2009; New Orleans.

This study is a useful demonstration of the well known, but previously undocumented, underutilization of hydroxyurea in children, as well as some of the underlying physician and patient reasons for the problem. For example, the drug is often refused due to fears of leukemogenesis and infertility, which have not been borne out in studies in humans.

- Peter Newburger, MD
HemOnc Today Editorial Board member

More ASH Meeting Highlights>>