Jennell White, Ph.D., Xiufeng Gao, M.D., Aliya Zaidi, Ph.D., Kerry Bellemare, MPH, Patrick Hines, M.D., Ph.D.
Key Findings
- Purpose
To evaluate whether a biologicmarker — flow adhesion of whole blood to P-selectin (FA-WB-PSEL) — correlates with clinical outcomes inpatients with sickle cell disease treated with crizanlizumab. - Population (Model)
Retrospective real-world dataset including 994 patients (5,507 samples), with a small treated subgroup (n = 22) receiving crizanlizumab; only 3 patients met criteria for longitudinal analysis over one year. - Headline Result
The study observed variability inbiologic response to crizanlizumab, with distinct responder profiles (“complete,” “incomplete,” “non-,” and “adverse”) based on FA-WB-PSEL levels; in the small longitudinal cohort (n = 3), reductions in FA-WB-PSEL were observed alongside changes in clinical utilization metrics. - Why It Matters
Crizanlizumab targets P-selectin–mediated adhesion, a central mechanism in vaso-occlusion. These findings suggest that biologicresponse to therapy may differ across patients and that biomarker-based monitoring could help contextualize treatment response. - Evidence Gaps & Limitations
Very small longitudinal sample (n = 3) and retrospective design limit interpretability. Findings are exploratory and do not establish causal relationships between biomarker changes and clinical outcomes. Larger prospective studies are required.
Source: Journal of Sickle Cell Disease- “Comparative Assessment of Biologic (Flow Adhesion of Whole Blood to P-selectin) and Clinical Response to Crizanlizumab in a Real-world Clinical Setting”
Regulatory & Guideline Watch
The American Society of Hematology includes P-selectin inhibition (e.g., crizanlizumab) among disease-modifying options for reducing vaso-occlusive crises, withrecommendations tailored to patient characteristics and prior therapy. Evidence supporting biomarker-guided treatment selection remains preliminary and is not yet incorporated into guideline-directed care.