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. 2022 Sep;31(9):2681-2694.
doi: 10.1007/s11136-022-03132-z. Epub 2022 Apr 21.

An evaluation of patient-reported outcomes in sickle cell disease within a conceptual model

Marsha J Treadwell  1 , Swapandeep Mushiana  2 , Sherif M Badawy  3   4 , Liliana Preiss  5 , Allison A King  6 , Barbara Kroner  5 , Yumei Chen  7 , Jeffrey Glassberg  8 , Victor Gordeuk  9 , Nirmish Shah  10 , Angela Snyder  11 , Theodore Wun  12 ; Sickle Cell Disease Implementation Consortium (SCDIC)
Affiliations

An evaluation of patient-reported outcomes in sickle cell disease within a conceptual model

Marsha J Treadwell et al. Qual Life Res. 2022 Sep.

Abstract

Purpose: To examine the relations between patient-reported outcomes (PROs) within a conceptual model for adults with sickle cell disease (SCD) ages 18 - 45 years enrolled in the multi-site Sickle Cell Disease Implementation Consortium (SCDIC) registry. We hypothesized that patient and SCD-related factors, particularly pain, and barriers to care would independently contribute to functioning as measured using PRO domains.

Methods: Participants (N = 2054) completed a 48-item survey including socio-demographics and PRO measures, e.g., social functioning, pain impact, emotional distress, and cognitive functioning. Participants reported on lifetime SCD complications, pain episode frequency and severity, and barriers to healthcare.

Results: Higher pain frequency was associated with higher odds of worse outcomes in all PRO domains, controlling for age, gender and site (OR range 1.02-1.10, 95% CI range [1.004-1.12]). Reported history of treatment for depression was associated with 5 of 7 PRO measures (OR range 1.58-3.28 95% CI range [1.18-4.32]). Fewer individual barriers to care and fewer SCD complications were associated with better outcomes in the emotion domain (OR range 0.46-0.64, 95% CI range [0.34-0.86]).

Conclusions: Study results highlight the importance of the biopsychosocial model to enhance understanding of the needs of this complex population, and to design multi-dimensional approaches for providing more effective interventions to improve outcomes.

Keywords: Implementation science; Models—biopsychosocial; Patient-reported outcome measures; Sickle cell disease.

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Conflict of interest statement

Drs. Treadwell, Mushiana, Badawy, Preiss, King, Kroner, Glassberg, Gordeuk, Snyder, Wun and Ms. Chen have none to declare. Dr. Shah receives research funding from Global Blood Therapeutics, is a consultant for Novartis, Emmaus Medical and Forma Therapeutics and a speaker for Global Blood Therapeutics and Novartis.

Figures

Fig. 1
Fig. 1
Conceptual model for inter-relations of patient-reported outcomes (PROs) in sickle cell disease (SCD). The model includes the inter-relations of four PRO groups (emotional distress, social functioning, pain impact, and fatigue, sleep and cognitive functioning) with health behaviors (acute healthcare utilization and preventive care), SCD complications (number of complications and pain frequency/severity), SCD-related factors (genotype, hydroxyurea, chronic transfusion history), patient related factors (education, employment, income, marital status, diabetes and depression) and barriers to care (systemic and individual). All inter-relations are adjusted for age and gender identity
See this image and copyright information in PMC

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