Medication Monitor

Generic Name (Trade Name—Company)
November 18, 2019


First targeted therapy approved for patients with painful complication of sickle cell disease

FDA approved crizanlizumab-tmca, a treatment for patients ages 16 years and older to reduce the frequency of vaso-occlusive crisis, a common and painful complication of sickle cell disease that occurs when blood circulation is obstructed by sickled red blood cells.

Approval was based on the results of a randomized clinical trial enrolling 198 patients with sickle cell disease with a history of vaso-occlusive crisis. Patients received either crizanlizumab-tmca or a placebo. The patients treated with crizanlizumab-tmca experienced fewer health care visits for vaso-occlusive crisis annually (median annual rate of 1.63 visits), compared with patients who received a placebo (median annual rate of 2.98 visits). In addition, 36% of patients who received crizanlizumab-tmca did not experience vaso-occlusive crisis during the study, and it delayed the time that patients first experienced vaso-occlusive crisis after starting treatment from 1.4 months to 4.1 months.

Common adverse effects for patients taking crizanlizumab-tmca were back pain, nausea, fever, and arthralgia. Health professionals are advised to monitor patients for infusion-related reactions and to discontinue crizanlizumab-tmca for severe reactions. Patients who receive crizanlizumab-tmca should be monitored for interference with automated platelet counts or platelet clumping (platelet counts reported may be much lower than the actual count in the blood). Health professionals are advised to run tests as soon as possible or use citrate tubes.