Short Term Health-Related Quality of Life Change in Adults with Sickle Cell Disease
Authors: Kanter Julie , Lanzkron Sophie , Saif Ur Rehman Sana , Little Jane A. , Ullman Megan M. , Curtis Randall , Crook Nicole , Roberts Jonathan C. , Robertson Derek , Wu Joanne , Nichol Michael B. 
Published: December 2024

Introduction: Adults with sickle cell disease (SCD) suffer from periodic episodes of severe acute pain, chronic pain, fatigue, acute complications, end-organ damage and early mortality, which negatively impact health-related quality of life (HRQoL). We assessed HRQoL change within a 12-month period.
Methods: We analyzed data from 49 adults aged ≥18 years with a diagnosis of SCD who completed both baseline and 12-month follow-up surveys from four U.S. NASCC recognized SCD centers participating in the Globin Research Network for Data and Discovery (GRNDaD) registry. We collected data on patient socio-demographics, fatigue, chronic pain, and HRQoL measured by EQ-5D-5L and ASCQ-Me via patient survey, including baseline (enrollment) and 12-month follow-up. A medical history checklist (MHC) score summed 9 SCD complications and treatment history. We also collected an 8-item Patient Health Questionnaire (PHQ-8) for depression and 7-item Generalized Anxiety Disorder (GAD-7) questionnaire at follow-up. Clinical characteristics of SCD phenotype and hemoglobin were obtained from GRNDaD. Paired T-tests were used to assess changes in the HRQoL scores between baseline and follow-up. We used the Paretian classification of health change (PCHC) to judge overall health improvement or worsening in EQ-5D profiles. We explored the variables associated with changes in health states.
Results: Mean (standard deviation) age was 39.9 (14.8) years, 67.4% of the sample was female, and 79.6% had hemoglobin (Hb) SS disease. Over 12 months, four domains (Sleep impact, emotion impact, social functioning impact, and pain impact) of ASCQ-Me scores (higher score represents better health), pain episode frequency, and pain episode severity scores (a lower score represents better health) were slightly increased, and the scores difference between baseline and follow-up ranged from 0.09 for sleep impact to 1.40 for pain impact; the stiffness impact score decreased 1.14 point. However, these changes in ASCQ-Me scores were not statistically significant (P&gt;0.05). There were no statistically significant changes in mean hemoglobin, fatigue score, or number of acute care visits (emergency, hospitalizations, and infusions center). Mean EQ visual analogue scale (VAS) was 69.2 at baseline and 72.3 at follow-up (both lower than the U.S. aged 35-44 general population mean of 81.8). Mean EQ index score was 0.71, and was unchanged at 12-month follow-up. These scores were lower than the U.S. 35-44 age population norm of 0.85. The follow-up mean EQ index score was higher in participants with emergency room or infusion center visits (0.62±0.27 vs. 0.76±0.20, p=0.06) than those without the visits. EQ-5D PCHC classified the sample into three groups: 1) 44.9% with improvement or no change; 2) 28.6% with worsening health states; and 3) 26.5% had mixed results (some profiles included both improved and worsening health states). More individuals who used disease modifying therapy (DMT) (31.3% vs. 14.3%, p=0.20), or had infusion center visits (35.0% vs. 19.2%, p=0.19) experienced improved health states over the 12-month period than those without. Individuals who had emergency room visits (20.0% vs. 30.8%, p=0.88), hospitalizations (20.7% vs. 35.3%, p=0.92), anxiety (20.0% vs. 26.8%, p=0.80), or depression (0% vs. 31.6%, p=0.07) were less likely to experience improved health states.
Conclusions: In this adult SCD sample with 12-month follow-up observations, HRQoL scores were not significantly changed. EQ-5D profile analyses indicated that individuals on DMT may have improved health states over a 12-month period. Larger studies are needed to further evaluate the EQ-5D PCHC tool and to better understand the association with better HRQoL scores and infusion center visits. Evaluating health states and HRQoL and the sensitivity of these tools over time could be useful clinic tools to assess response to therapies.