Can personalized encouragement help people with sickle cell disease take hydroxyurea therapy regularly? / Lakshmanan Krishnamurti.

Format:

Book

Author/Creator:

Krishnamurti, Lakshmanan, author.

Language:

English

Subjects (All):

Sickle cell anemia.

Sickle cell anemia--Complications.

Physical Description:

1 online resource

Place of Publication:

Washington, DC : Patient-Centered Outcomes Research Institute (PCORI), 2020.

Summary:

BACKGROUND: Poor adherence to hydroxyurea (HU) therapy is a barrier to improving outcomes in patients with sickle cell disease (SCD). Understanding barriers and improving adherence to HU can improve outcomes for SCD and yield knowledge relevant to other chronic illnesses. OBJECTIVES: 1. To determine in a randomized controlled trial (RCT) if an individualized, structured combination of interventions to improve HU adherence is acceptable to patients with SCD2. To determine attitudes of patients and caregivers toward HU and acceptability and utility of interventions to improve medication adherence3. To determine the relationship of HU adherence in SCD with patient-related and health care use outcomes METHODS: Individuals receiving HU were randomly assigned to either standard of care or a combination of interventions that included the daily submission of a video documenting administration of the medication, with therapeutic alliance maintained through text, telephone, and in-person contact (called mobile directly observed therapy or Mobile-DOT). We used the Treatment Satisfaction Questionnaire for Medication (TSQM-9), the Adherence Starts with Knowledge (ASK-12), and an additional barriers survey to evaluate the barriers to HU adherence. Primary outcomes were medication possession ratio (MPR), the proportion of days on which patients submitted video, The MMAS consists of four items with a scoring scheme of "yes" = 0 and "no" = 1. The items are summed to give a range of scores from 0 to 4, and the proportion of patients with high self-reported medication adherence by Morisky Medication Adherence Scale (MMAS-4) less than 2. Secondary outcomes included the impact of the intervention on clinical, laboratory, and quality-of-life outcomes. Qualitative patient interviews examined attitudes toward HU and the acceptability and utility of adherence interventions. RESULTS: Surveys and qualitative interviews revealed low reported levels of barriers and moderately high levels of satisfaction with treatment and the intervention. We enrolled and randomly assigned 79 adults and 85 children. We found no statistically significant differences in demographics, clinical factors, adherence, patient-reported outcomes, and laboratory characteristics between the intervention and control arms at baseline. The rate of study discontinuation was 26%, including 28% in control and 23% in the intervention arm (P = .6) and 29% in adults and 22% in children (P = .40), with no significant difference in the characteristics of those who continued or discontinued the study. PRIMARY OUTCOMES: We observed no statistically significant differences in MPR and MMAS-4 in the intervention and control arms. Patients randomly assigned to the intervention arm submitted video documentation of medication intake on a mean of 34.8% of the study days. SECONDARY OUTCOMES: Overall we found no statistically significant differences between the intervention and control arms in clinical and laboratory characteristics, quality of life, and health care use. The mean corpuscular volume (MCV) of the red blood cells, a biomarker of response to HU therapy, in pediatric patients was higher in the intervention arm at all time points, but the increase with time was statistically significant only in the first 6 months after intervention; the change at 12 months was not statistically significant. We noted no similar change in MCV in adults. In qualitative interviews participants stated that the reminder texts, emails, and reminders helped them take the drug consistently but indicated that submitting the videos was cumbersome. In exploratory analyses, adult patients who reported MMAS-4 scores less than 2 (suggesting high self-reported medication adherence) were more likely to submit videos of medication administration; 50% of the participants submitted videos >25% of the days, and this group had higher MCV and fetal hemoglobin at baseline and showed significant improvement in MCV and patient-reported outcomes. CONCLUSIONS: Patient-reported Mobile-DOT was acceptable and useful, but it did not impact MPR, self-reported adherence, or clinical, laboratory, or patient-reported outcomes, compared with controls. LIMITATIONS: Significant rates of study discontinuation, low adherence with video submission, and missing data on MPR and patient-reported outcomes limit the interpretation of these data.

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