April 28, 2015
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Hydroxyurea may be underused for patients with sickle cell anemia

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Less than a quarter of patients with sickle cell anemia and pain crises received hydroxyurea within 1 year of a third hospital or emergency department visit, despite the safety and availability of the therapy, according to study results.

“The recommendation from the 2014 NHLBI guidelines to treat all adults with sickle cell anemia and 3 or more moderate to severe pain crises within 1 year with hydroxyurea was rated strong based on high-quality evidence reviewed in 2008,” Nicolas Stettler, MD, MSCE, of The Lewin Group in Falls Church, Virginia, and colleagues wrote. “Despite benefits in reducing pain crises, hospitalizations, blood transfusions and possibly mortality, it is thought that hydroxyurea is underused, although the extent of its use is unknown.”

Stettler and colleagues evaluated data from the Optum Normative Health Informatics database, a nationwide sample of commercial health and pharmacy claims. The researchers selected patients from the database if they had three or more hospitalizations, ED visits, or both within 12 months that included at least one of the most frequent codes used for patients with sickle cell anemia and pain crises.

The researchers identified 2,086 adults with probable sickle cell anemia. The median age of the cohort was 39 years (range, 28-52) and 59% (n = 1,231) were women.

Of the patients, 677 had three or more pain-related hospitalizations or ED visits within 12 months and 570 had at least 3 months of coverage after the third event.

Researchers then evaluated treatment use, which was defined as filling at least one hydroxyurea prescription 3, 6 or 12 months after the third event.

Overall, 15.1% (95% CI, 12.3-18.3) of patients received treatment with hydroxyurea within 3 months of the third hospital or ED visit. The proportion of patients who received hydroxyurea increased to 18.2% (95% CI, 15-21.8) at 6 months and 22.7% (95% CI, 18.9-27) at 12 months (P = .002 for trend).

“Despite evidence demonstrating the benefits of hydroxyurea in patients with sickle cell anemia and frequent pain crises, this analysis suggests that more than 3 of 4 patients who might benefit were not treated with this safe and inexpensive drug,” Stettler and colleagues wrote. “Several barriers to treatment have been identified, including fear of adverse events, lack of clinical training and failure to engage in shared decision making. Our estimate reflects the combined effect of all barriers to treatment, regardless of source.”

Still, the researchers acknowledged that these data are limited by the reliance on ICD-9 codes to identify patients, and that patients with pain crises who were not hospitalized at least three times were not included in the analysis.

“Our data do not include the large uninsured or publicly insured population who may have more limited access to health care or awareness to treatment options,” the researchers wrote. “Therefore, these findings may not be representative of the entire U.S. population with sickle cell anemia and may be a conservative estimate of the hydroxyurea gap. To address this gap, it may be necessary to enhance patient outreach and clinician training and develop health care quality measures aimed at increasing the use of hydroxyurea for all patients who would benefit.” – by Cameron Kelsall

Disclosure: The researchers report no relevant financial disclosures.