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Patient-Reported Experiences in Voxelotor-Treated Children and Adults with Sickle Cell Disease: A Semistructured
Clark Brown1, Modupe Idowu2, Richard Drachtman3
1Aflac Cancer & Blood Disorders Center of Children's Healthcare of Atlanta and Department of Pediatrics, Emory University, Atlanta, GA, USA.
Insights
Patients with sickle cell disease (SCD) reported improved symptoms like pain crises and fatigue after treatment with voxelotor. Voxelotor also enhanced their self-reported quality of life, offering significant benefits for SCD management.
Area of Science:
- Hematology
- Pharmacology
- Patient-Reported Outcomes
Background:
- Voxelotor is a novel sickle hemoglobin-polymerization inhibitor approved for sickle cell disease (SCD) treatment.
- Regulatory approvals in the US, UAE, and EU highlight its growing clinical significance.
- Understanding patient experiences is crucial for optimizing SCD care.
Purpose of the Study:
- To explore and understand the lived experiences of patients with SCD undergoing voxelotor treatment.
- To gather insights into symptomatic changes and health-related quality of life (HRQoL) from the patient perspective.
Main Methods:
- Semistructured interviews were conducted with adult, adolescent, and pediatric patients with SCD and their caregivers in the US.
- Thematic analysis was employed to analyze interview transcripts, focusing on patient-reported outcomes.
- Concept saturation was assessed across different patient cohorts.
Main Results:
- A majority of patients reported notable improvements in SCD symptoms, including reduced pain crises, jaundice, and fatigue.
- Nearly all participants experienced enhanced self-reported health-related quality of life while on voxelotor treatment.
Conclusions:
- This study underscores the positive symptomatic benefits and improved HRQoL associated with voxelotor treatment in SCD patients.
- Findings provide valuable patient and caregiver perspectives to inform future research on SCD-specific outcomes.
Objective:
Voxelotor is a first-in-class sickle hemoglobin-polymerization inhibitor that was approved in 2019 by the US Food and Drug Administration for treatment of patients with sickle cell disease (SCD) aged ≥12 years; in 2021, the approval was extended to children with SCD aged 4 to 11 years. Additionally, both the Ministry of Health and Prevention for the United Arab Emirates and the European Commission granted marketing authorization for voxelotor in September 2021 and February 2022, respectively, for treatment of SCD in adults and pediatric patients aged ≥12 years. Thus, additional information on the patient experience with voxelotor would be useful for patients, caregivers, and healthcare professionals alike. The purpose of this study was to conduct semistructured interviews in an effort to understand the experiences and perspectives of voxelotor-treated patients with SCD.
Methods:
One-time semistructured interviews with adults, adolescents, and children with SCD and their primary caregivers were conducted in the United States. Twenty-three adults and adolescents were recruited across 4 clinical sites, and 10 children-caregiver dyads were recruited from a single site. The interview was designed to elicit patient perspectives on symptomatic changes with voxelotor and the impact of treatment on patients' perceived health-related quality of life. Individual interview transcripts were analyzed using a thematic analytic approach, and concept saturation was assessed in both cohorts.
Results:
Most patients reported improvements in their SCD symptoms with voxelotor treatment, specifically regarding pain crises, jaundice, and fatigue. Almost all patients experienced improvements in self-reported health-related quality of life with voxelotor treatment.
Conclusions:
This study provides patient and caregiver perspectives on the symptomatic benefits of voxelotor treatment. These findings not only highlight the benefits of voxelotor treatment in improving symptoms and increasing health-related quality of life across the entire SCD population but also can inform further research on SCD-specific patient-reported outcomes.
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