Health-related quality of life and economic impacts in adults with sickle cell disease with recurrent vaso-occlusive crises: findings from a prospective longitudinal real-world survey.

purposeCharacterize health-related quality of life (HRQoL) and economic impacts associated with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs).

methodsA prospective longitudinal real-world study was performed with adults with SCD with recurrent VOCs in the US and Europe (France, Germany, Italy, and the UK). Evidence of recurrent VOCs was self-reported (≥ 2 VOCs per year in each of the 2 years before enrolment). Study outcomes were reported at baseline, months 3, and 6 and included self-reported demographics, clinical characteristics, patient-reported outcome measures (EQ-5D-5 L VAS, FACT-G, FACIT-F, 11-point numerical rating scale [NRS] of pain, ASCQ-Me, WPAI), and bespoke questions on financial burden, stigma/prejudice, and time burden associated with SCD.

resultsParticipants (N = 142) reported an annual mean (standard deviation) of 5.9 (4.8) VOCs requiring hospitalization and 7.5 (7.7) VOCs requiring home management in the past 24 months. Participants reported fatigue and HRQoL impairment, demonstrated by significantly lower baseline EQ-5D-5 L utility and VAS, FACT-G, and FACIT-F scores compared to published information about the US general population. Participants also reported increased pain, stiffness, and emotional impacts compared to a reference sample of adults with SCD on the ASCQ-Me, and most (60%) reported moderate to extreme pain/discomfort on the NRS of pain. Out-of-pocket expenses represented a moderate-to-major burden for 61.9% of participants, with employed participants missing a mean of 9.8 h of work in the last 7 days.

conclusionParticipants reported significant impairments to their HRQoL and financial constraints associated with SCD with recurrent VOCs, indicating humanistic and economic burden and unmet needs in this population.