Author(s): ,
Martin Besser
Addenbrooke's Hospital,Cambridge,United Kingdom
Miranda Bailey
Novartis Pharmaceuticals Corporation,East Hanover,United States
Stephen Brown
Cornerstone Research Group Inc.,Miamisburg,United States
Melissa Thompson
Cornerstone Research Group Inc.,Miamisburg,United States
EHA Library. Bailey M. Jun 15, 2019; 267124; PS1507
Miranda Bailey
Miranda Bailey

Abstract: PS1507

Type: Poster Presentation

Presentation during EHA24: On Saturday, June 15, 2019 from 17:30 - 19:00

Location: Poster area

Sickle cell disease (SCD) is a global health concern. Each year as many as 200,000 infants are born with this disease worldwide. Despite improvements in outcomes over the last few decades, patient life expectancy remains 10 to 30 years shorter than that of the healthy population. A major cause of morbidity among patients with SCD is the occurrence of intense, recurrent, acute, severely painful episodes, known as vaso-occlusive crisis (VOCs). As patients age and SCD progresses, an increased frequency of VOCs and other disease-related complications is correlated with an increased risk of morbidity and mortality.

The aim of the current analysis was to determine whether VOC frequency is associated with a decrease in the health-related quality of life (HRQoL) and associated utility of patients with SCD.

LEGACY was a prospective, multicenter, observational study that documented the outcomes of 181 adult patients with SCD living in the United States. Patients were assessed at baseline and every six months for 3 years, on several health outcomes, including the number of VOCs experienced and patient health as assessed by the Short Form-36 (SF-36) Health Survey. For the cohort of patients aged 18 years and older, the total number of VOCs experienced over the previous 12 months was determined for each administration of the SF-36. Patients were categorized into three groups:


  •          0 VOCs in the previous 12 months
  •          1 to 2 VOCs in the previous 12 months
  •          3 or more (3+) VOCs in the previous 12 months


The SF-36 scores for individual questionnaires were then mapped to the EuroQoL five-dimension (EQ-5D) questionnaire, which is a standardized instrument that can be used to derive a single utility value.  The mapping was done using the random effects GLS mapping algorithm published by Rowan and colleagues (2009). Mean EQ-5D scores were compared between groups using the Student’s T-test. Differences between the groups on the SF-36 component scores and SF-36 domains were also assessed.

Patients with 3+ VOC events in the previous 12 months had mapped mean EQ-5D scores that tended to be lower (15%) than those of patients who had no VOC events (see Figure 1).  Patients who had 3+ VOC events also showed decreases in both SF-36 Physical Component Scores and Mental Component Scores compared to patients with no VOC events. 

To our knowledge, this is the first analysis to examine the relationship between VOC frequency and HRQoL in patients with SCD. The results of this analysis indicate that patients who have a higher number of VOC events experience a worse HRQoL compared to patients with no events.  Additional research is required to understand the drivers of this difference in HRQoL.

Session topic: 35. Quality of life, palliative & supportive care, ethics and health economics

Keyword(s): Quality of life, Sickle cell disease

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