HABIT efficacy and sustainability trial, a multi-center randomized controlled trial to improve hydroxyurea adherence in youth with sickle cell disease: a study protocol

Abstract

Background Hydroxyurea (HU) is recommended as standard practice for youth with sickle cell disease (SCD). Yet, despite its efficacy, HU adherence in adolescents and young adults is often poor. Poor medication adherence increases disease burden, healthcare cost and widens health disparities. Adolescence is a critical time to improve adherence through improved chronic disease self-management. This study aims to test the efficacy of an intervention delivered to youth/parent dyads by community health workers (CHWs), augmented by tailored text messages on HU adherence (primary outcome). Secondary outcomes are intervention sustainability, youth health-related quality of life, self-management responsibility concordance, acute hospital use and self-reported disease symptoms. Methods Hydroxyurea Adherence for Personal Best in Sickle Cell Disease, “HABIT,” is a 12 month multi-center randomized controlled trial. One hundred four youth, 10 to 18 years of age prescribed HU who meet eligibility criteria, enrolled with their parent as dyads, will be randomized 1:1 to either the HABIT intervention or to usual clinical care plus education handouts. All subjects will complete clinic visits at months 0, 2, 4, 6 (efficacy component), 9 and 12 (sustainability component) for assessment of HbF biomarker, other hematologic parameters, and to complete questionnaires. In addition, dyads assigned to the HABIT intervention will work with CHWs to identify a daily habit (e.g., brushing teeth) on which to build a HU adherence habit. Tailored daily text message reminders to support the habit will be developed by the dyad in collaboration with the CHWs and sent to parent and youth. At the 6 month visit, the intervention will end and the sustainability portion of the trial will begin. All data analyses will be based on intention to treat with all randomized subjects included in the analyses. Discussion Prior retrospective studies demonstrate that a majority of adolescents are poorly adherent to HU. If efficacious, the HABIT intervention has the potential to improve the lives of youth with SCD. Trial registration Clinicaltrials.gov NCT03462511. Registered March 6, 2018, last updated July 26, 2019.

Real‐Life experience with hydroxyurea in patients with sickle cell disease: Results from the prospective ESCORT‐HU cohort study

“…[3][4][5] Although the clinical efficacy of HU in preventing pain has been demonstrated, [6][7][8] and its ability to increase life expectancy suggested, 9,10 many publications continue to report low/poor adherence to HU, 11,12 associated with a poor quality of life and school absenteeism. [13][14][15] Reluctance to use HU may be partially related to fears about potential side effects of this medication, especially in terms of fertility and carcinogenicity. 16 As malignancies are rare events, safety assessment requires very large-scale studies covering long periods of follow-up.…”

Feasibility of establishing a network of community health workers to support care of people with sickle cell disease in Kumasi, Ghana

“…There are some ongoing projects exploring CHWs for SCD. Smaldone et al (2019) shared a protocol for a study aiming at testing the efficacy of an intervention delivered to youth/parent dyads by CHWs, augmented by tailored text messages on medication adherence. In their feasibility study, CHWs worked with people and families for improved understanding of SCD and medication adherence, identified and addressed barriers to medication adherence, built youthparent disease self-management partnerships, and identified a cue on which a medication habit could be built (Green et al 2017).…”

Mental health assessment of youth with sickle cell disease and their primary caregivers during the COVID‐19 pandemic

“…Fifty dyads of youth with SCD ages 10–18 years and their primary caregivers enrolled in our multi‐site randomized, controlled trial, HABIT (“Hydroxyurea Adherence for Personal Best in Sickle Cell Treatment”) aimed at improving youth's adherence to hydroxyurea therapy, based on their flagging fetal hemoglobin levels. 19 The HABIT trial was conducted from 2018 through 2021 at four pediatric SCD centers located in New York City and Philadelphia. Trial enrollment occurred between May 2018 and December 2020, with 89% enrolled prior to onset of the COVID‐19 pandemic.…”