PURPOSE: Sickle cell disease (SCD) is an inherited blood disorder that affects the hemoglobin protein of red blood cells and has a significant impact on morbidity, mortality, and quality of life. Hydroxyurea has been FDA approved since 1998 as a disease-modifying therapy for SCD. However, hydroxyurea has not been optimally utilized for those with SCD. The purpose of this study was to evaluate reasons for hydroxyurea use, from the perspectives of providers, adults with SCD, and parents/caregivers of children with SCD, as well as perceived barriers to its use. We examined indications and reasons for being “on hydroxyurea,” defined by patients as currently taking hydroxyurea, and reported on pain frequency, perceptions of barriers, hydroxyurea adherence, and health care access for patients with SCD who were either on and not on hydroxyurea.
METHODS: We conducted a cross sectional analysis of data collected within the Pacific Sickle Cell Regional Collaborative (PSCRC), a consortium of nine western U.S. states. Individuals were eligible for this study if they 1) had a confirmed diagnosis of SCD, 2) were followed at one of the PSCRC sites, and 3) were eligible for hydroxyurea therapy. Parents/caregivers of children with SCD less than 18 years and adults with SCD 18 years and older completed a brief survey about hydroxyurea use, indications, side effects, pain frequency, number of hospital and emergency department (ED) admissions per year, and individual and family perceptions of barriers to hydroxyurea use. Participants completed a follow-up survey annually, but we reported only on baseline data. Data collection occurred between February 2016 and May 2018.
RESULTS: Individuals with SCD (n = 413) included 1) children (n=178; 6.7 ± 3.4 years), 2) adolescents (n=66; 15.0 ± 1.4 years), 3) young adults (n=57; 21.4 ± 2.6 years), and 4) adults (n=112; 39.2 ± 10.6 years). The majority were predominantly female (51.6%), African American (93.2%), and had HgbSS (74.1%) genotype. The majority of children (65.2%), adolescents (62.1%), and young adults (54.4%) were on hydroxyurea; fewer adults (39.3%) were on hydroxyurea. The majority with HgbSS (65.5%) were adherent to hydroxyurea. There was no significant difference in hospitalizations for pain, ED visits, and pain severity in the previous 12 months between individuals who were and were not on hydroxyurea, and between individuals who were and were not adherent to hydroxyurea. For those with a current prescription for hydroxyurea, the majority (66.5%) were receiving hydroxyurea for recurrent pain episodes or acute chest syndrome (19.9%). Hydroxyurea was discontinued because of patient/family preference (34.5%), chronic transfusions (31.1%), and side effects (24.1%). Patients prescribed hydroxyurea for empiric use (n=21) had fewer hospitalizations for pain, ED visits, and severe pain interfering with daily activities. The major barriers to hydroxyurea use, from the perspective of individuals with SCD or their caregivers, were 1) forgetting to take the medicine (19.4%), 2) worried about side effects (16.4%), and 3) lack of knowledge about hydroxyurea (13.6%). Fewer young adults (49.1%) and adults (50.0%) had primary care providers than children (78.1%) and adolescents (65.2%).
CONCLUSIONS: Barriers to hydroxyurea use persist with emerging solutions to alleviate these barriers. For this sample, while hydroxyurea prescription rates by sickle cell specialists were similar to what has been seen in some other studies, neither hydroxyurea use nor adherence were associated with decreased frequency of hospitalizations for pain, ED visits, and severe acute pain episodes in the previous 12 months. Future studies need to evaluate hydroxyurea prescription patterns, duration on hydroxyurea, and adherence to hydroxyurea. Healthcare providers are recommended to prescribe hydroxyurea for eligible individuals who may benefit from it, such as those HgbSS or HgbS-β0 thalassemia genotype, and prescribe for empiric use to minimize complications. Provider and patient education about hydroxyurea could reduce common barriers experienced by individuals with SCD. It is important to customize educational resources to specific concerns for different age groups. Individuals 18 years and older with SCD have been documented with more ED visits and hospitalizations due to pain, most likely because they did not have a primary care provider and an adult hematologist with expertise in SCD. Future studies need to evaluate whether primary care providers who receive SCD education may promote hydroxyurea use and adherence. Dedicating time and resources for shared decision making between providers and patients/families can address concerns about hydroxyurea and increase patient/family confidence when deciding about hydroxyurea. As more disease-modifying therapies become available for individuals with SCD, strategies for shared decision making facilitate standardization and optimize the use of hydroxyurea and emerging therapies.
| Identifer | oai:union.ndltd.org:bu.edu/oai:open.bu.edu:2144/43355 |
| Date | 11 November 2021 |
| Creators | Du, Lisa |
| Contributors | Trinkaus-Randall, Vickery E., Treadwell, Marsha J. |
| Source Sets | Boston University |
| Language | en_US |
| Detected Language | English |
| Type | Thesis/Dissertation |
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