In a randomized trial of patients with sickle cell disease recently published in New England Journal of Medicine, the P-selectin inhibitor crizanlizumab resulted in a significantly lower rate of disease-related pain crises than placebo. Hydroxyurea is currently the only drug that modifies the natural history of the disease, but it doesn’t prevent pain crises.
The phase 2 trial assigned 198 patients with sickle cell disease in a 1:1:1 ratio to receive low-dose crizanlizumab (2.5 mg/kg of body weight), high-dose crizanlizumab (5.0 mg/kg), or placebo administered intravenously 14 times over 52 weeks. Approximately 40% of patients in each group were receiving concomitant hydroxyurea.
Anita Slomski. Crizanlizumab Prevents Sickle Cell Pain Crises. JAMA. 2017;317(8):798. doi:10.1001/jama.2017.0355