Global Treatment Satisfaction Levels and Treatment Patterns from the International Sickle Cell World Assessment Survey (SWAY): Hydroxyurea (HU) Versus No HU

Background: SWAY was a cross-sectional survey that assessed the global impact and treatment of sickle cell disease (SCD) (James et al. ASH 2019). SCD puts patients at risk of multiple complications driven by vaso-occlusion and hemolytic anemia. Vaso-occlusive crises (VOCs) are the hallmark of SCD and can require healthcare attention. VOC frequency may be reduced by HU (Charache et al. N Engl J Med 1995). Aims: We assessed self-reported symptoms and quality of life (QoL) indicators for patients who reported using HU at the time of SWAY versus patients who did not, and we collected data on all treatments reported by SCD patients, by geographical region. Data were also collected regarding historical patient-reported use of HU prior to SWAY but these are not included here. Methods: SWAY was completed between April and October 2019 by SCD patients from 16 countries across 6 regions. A limitation is that Asia and South America were represented by single countries (India and Brazil, respectively). SWAY was completed by proxy (parent/guardian/caregiver) for patients aged 6-11 years and could be optionally self-completed by patients aged ≥12 years. Opinions were captured using a 1-7 Likert scale for some questions (5-7 indicated high satisfaction/impact/agreement). SWAY did not assess treatment outcomes. Results: Of 2145 patients, 652 (30%) reported receiving HU at the time of SWAY (56% female; 50% aged 6-25 years); 1493 patients reported not receiving HU at the time of SWAY (51% female; 59% aged 6-25 years). The number of patients reporting HU use varied regionally (Table). Patients who reported using HU also reported a lower VOC burden than patients who did not report using HU at the time of SWAY (median: 3 vs 4 VOCs in the 12 months before SWAY, respectively). However, considering other symptoms commonly experienced in the month prior to SWAY, a greater proportion of patients who reported using HU experienced these symptoms than patients who did not report using HU, except for headache and poor appetite, which were experienced by a lower proportion of patients who reported HU use (Figure). Similar proportions of patients reported that SCD had a high impact (Likert scale 5-7) on emotional wellbeing (61% [reported HU use at time of SWAY] vs 59% [did not report HU use at time of SWAY]) and daily activities (39% vs 40%, respectively). Overall, when including dietary supplements, the most common treatment reported at the time of SWAY in all regions except the Middle