Hydroxyurea Underused in Sickle Cell Pain Crises

Ricki Lewis, PhD

April 29, 2015

Hydroxyurea is used in less than 25% of patients with sickle cell anemia (SCA) who could benefit from it, according to results of a study published in the April 28 issue of JAMA.

In 2014, the National Heart, Lung, and Blood Institute issued recommendations to treat all adults with SCA who experience at least three moderate to severe pain crises a year with hydroxyurea. Despite evidence that the drug reduces pain crises, hospitalizations, and blood transfusions, hydroxyurea is reportedly underused.

To track the use of hydroxyurea, Nicolas Stettler, MD, from the Lewin Group, Falls Church, Virginia, and colleagues analyzed a nationwide sample of commercial health and pharmacy claims from the Optum Normative Health Informatics database. They extracted information on patients with SCA who had three or more hospitalizations or emergency department visits or both within a year from January 2009 to June 2013. To be included in the study, patient visits needed to include diagnosis codes for one of the five most frequent diagnosis codes for SCA (acute chest syndrome, limb pain, chest pain, unspecified pain, other chest pain).

The study defined treatment with hydroxyurea as filling at least one prescription during the 3, 6, or 12 months of continued enrollment after the third hospitalization or ED visit.

Of 2086 people older than 18 years with probable SCA, 677 met the criteria and 570 had at least 3 months of insurance coverage after the third event. Among those 570 patients, 86 (15.1%; 95% confidence interval [CI], 12.3% - 18.3%) received hydroxyurea within the 3 months. Over time, uptake increased, to 18.2% (95% CI, 15.0% - 21.8%) at 6 months and 22.7% (95% CI, 18.9% - 27.0%) at 12 months (P = .002 for trend).

The researchers hypothesize that several factors might contribute to the low use of this inexpensive and effective treatment, including clinician unfamiliarity with hydroxyurea, fear of adverse events, and absence of shared decision-making.

The study may have underestimated the magnitude of the hydroxyurea gap in three ways: the study population was privately insured and did not include patients who could manage their pain crises at home. The study also did not consider, "the broadened criteria described in new guidelines for the use of hydroxyurea, such as patients who have daily pain that affects their quality of life."

The investigators conclude that to address the gap in hydroxyurea use, "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."

The researchers have disclosed no relevant financial relationships.

JAMA. 2015;313:1671-1672. Extract

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