Sickle cell disease (SCD) is a serious, chronic, genetic disease that leads to the production of abnormal hemoglobin and is marked by many complications, including recurrent episodes of severe pain. Despite 100 years of SCD being a recognized clinical entity in Western medical literature, hydroxyurea (HU) is currently the only approved disease-modifying medication available for the treatment of SCD.
HU has been shown to be efficacious in the treatment of SCD [1
]. Studies also suggest that treatment with HU may reduce the costs of health care for SCD, improve health outcomes, and decrease early mortality [2
]. A recent independent panel convened by the National Heart, Lung, and Blood Institute concluded that the benefits of HU therapy for SCD outweigh its known risks [11
Unfortunately, despite its potential benefits, the clinical literature suggests that HU is underutilized as a treatment for SCD. Although multiple studies have examined provider-level barriers to HU use, evidence on SCD patient beliefs and attitudes about HU is limited [15
]. Lack of patient knowledge about HU, misperceptions about its side effects, and poor health literacy are hypothesized as patient-level barriers [17
]. Hankins et al.
found that the perceived safety and efficacy of therapies were shown to be the two most important factors affecting treatment preference among parents and their children with SCD [16
]. The aim of our study was to address the gap in current knowledge regarding SCD patient attitudes and beliefs about HU by examining surveys administered to adults with SCD who were taking part in a larger study assessing their experiences with health care.
Ninety-four adult patients (age 18 years and older) participated in our study and provided data about their HU utilization and attitudes. The distribution of HU use was as follows: 39% never users, 24% former users, and 36% current users. describes the characteristics of our sample by HU use. Compared with those with current or former use, the never users had fewer sickle cell-related comorbidities and were less likely to report being on disability. When combined, those with current or former use experienced more hospital visits in the prior 12-month period than the never users (mean visits = 3.7 vs. 2.0, P = 0.054).
Patient Characteristics by Self-Reported Hydroxyurea Use
Seventy percent of current HU users reported some level of improvement from the drug (“average” or “very much”), and 80% reported “not at all” or “not much” trouble from side effects. Approximately, 83% of the former users reported taking HU for 1 year or less, with 57% taking HU for less than 6 months. The most commonly reported reasons given for stopping HU were their “doctor's recommendation” (39%) and “not liking the way it made me feel” (35%). Thirty-six percent of the never users reported that their doctors had previously suggested that they take HU. Fifty percent of the never users reported receiving no information about HU from any source. Eighty-five percent of the never users thought that they would receive no improvement if they were to take HU.
Thirty-three respondents (eight never users, nine former users, and 16 current users) described their concerns about HU in an open-ended manner. We labeled these concerns as representing three general categories (): no perceived benefit, lack of knowledge, and side effects. Examples of specific concerns raised in each category are as follows:
Number of Adult Sickle Cell Patients Describing Concerns About Hydroxyurea (n = 33)