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● Dynamic sickling assay (DSA) provides a simple, robust method for assessing responses to Hb differences and monitoring both pharmacodynamic SCD therapies and curative therapies.
● As HbA and HbF percentages increase in transfused SCD and SCT samples, improvements in DSA sickling parameters are observed. Additionally, comparative data analysis from patients pre- and post-hematopoietic stem cell transplant (HSCT) also show improvement in sickling parameters.
Kevin H.M. Kuo, MD, FRCPC, MSc1,2,3, Sylvia Titi Singer, MD4, Gershwin Theophilus Blyden, MD, PhD5*, Lanetta Bronté-Hall, MD, MPH, MSPH6, Aidan Faller, BS7*, Abdullah Kutlar, MD8, Frans A. Kuypers, PhD9*, Aliya U. Zaidi, PhD10*, Patrick C. Hines, MD, PhD10, Niren Patel, MBBS11*, Sandra Larkin, MS12*, Hanny Al-Samkari, MD13, Sujit Sheth, MD14, Seyed Mehdi Nouraie, MD15* and Susan Perrine, MD16
● Treatment with PB-04, an oral fetal globin stimulant, shows normalization of RBC health as assessed by flow adhesion of whole blood to VCAM (FA-WB-VCAM) over a 26-week treatment duration
Gianna G. Valenti , Meera Chitlur, Patrick Hines, Aliya Zaidi, Rasa Borhan, Kelsey Cinciarelli , Alexander K. Glaros
● Elevated baseline flow adhesion indices of whole blood to P-selectin may predict individuals likely to benefit from crizanlizumab, while normal adhesive indices at baseline may predict non-responders or even adverse effects
Olufunke Y. Martin, MD; Rasa Borhan, Aliya U. Zaidi, PhD; Deepika S. Darbari, MD; Patrick C. Hines, MD, PhD; Andrew D. Campbell, MD
● Understanding changes in objective RBC biomarkers from baseline to acute VOC in SCD patients with or without chronic pain
- Validation of patient treatment with a hemoglobin-modifying agent like GBT021601, results in normalization of RBC health as assessed by VCAM-1 adhesion, as early as Day 15 and sustainable up to Day 43.
