Real-World Treatment Patterns in Sickle Cell Disease (SCD) in Europe, Africa, Eastern Mediterranean Region (EMR), and India: A Systematic Literature Review (SLR)

Speaker(s)

Filonenko A1, Ronnebaum S2, Zuchinali P3, Wirz R4, Tedesco Barcelos G4, Ho M5
1Pfizer, Berlin, BE, Germany, 2Evidera, Inc., Bethesda, MD, USA, 3Evidera, Inc, Montreal, QC, Canada, 4Pfizer AG, Zurich, ZH, Switzerland, 5Pfizer, San Francisco, CA, USA

Presentation Documents

OBJECTIVES: To identify the real-world pharmacologic treatment patterns in patients with SCD in Europe, Africa, EMR, and India.

METHODS: A SLR was conducted in Embase/MEDLINE to identify observational studies published from January 2008-January 2024 describing the use of hydroxyurea (HU), iron chelators, opioids, and other pharmacological treatments for SCD (PROSPERO registration CRD42023487289).

RESULTS: From 845 unique citations, 63 primary studies that described over 75,000 patients from Africa (12 studies), Europe (30 studies), EMR (17 studies), India (2 studies), and multi-region (2 studies) were included.

Estimates for HU utilization, the current standard of care SCD treatment, varied from 0.3%-100% of patients (46 studies), without clear geographic patterns, and only 40% of studies reported >50% of patients using HU. Perceived lack of benefit, adverse events, cost, access, and clinical complications/contraindications were cited as reasons for poor adherence or discontinuation. Iron chelation was used by 2% (Saudi Arabia, 1 study), 4% (Germany, 1 study), and 22%-26% (Turkey, 2 studies) of patients. Among over 28,000 patients followed ≥1 year (France, Germany, Ghana, Italy, Saudi Arabia, UK, 8 studies), opioid use ranged from 9%-75%. Among 367 patients with reported acute chest syndrome or pain crises, opioids were prescribed to 33% to 100% of patients (Europe, 4 studies; multi-region, 1 study).

Few identified studies described longitudinal treatment patterns or utilization of recently approved drugs (e.g., voxelotor, crizanlizumab). Relatively little information was available from Africa, despite high regional SCD prevalence, compared to Europe and EMR.

The variation in patient populations and research methods may limit quantitative synthesis and direct comparison of treatment utilization across the studies.

CONCLUSIONS: SCD is a severe genetic condition requiring lifelong, tailored treatments. Existing evidence indicates substantial care gaps across geographies, underscoring the need for robust longitudinal studies on treatment patterns.

Code

HSD3

Topic

Patient-Centered Research, Study Approaches

Topic Subcategory

Adherence, Persistence, & Compliance, Literature Review & Synthesis

Disease

No Additional Disease & Conditions/Specialized Treatment Areas, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)