A prolonged-release form of anagrelide may be effective and safe against essential thrombocythemia, according to a study recently published in the British Journal of Haematology.
Anagrelide is a standard therapy for essential thrombocythemia, with efficacy at relatively low doses and adverse effects associated with higher doses. A prolonged-release form of the drug could be efficacious while limiting adverse effects.
This multicenter, double blind phase 3 trial compared a prolonged-release form of anagrelide with an immediate-release anagrelide formulation for treatment of essential thrombocythemia. A total of 106 patients were randomly assigned to either treatment group at an approximately 1:1 ratio.
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Primary endpoint was mean platelet count during the maintenance treatment period. Safety and tolerability were also examined.
Initial mean platelet levels were similar between the 2 treatment groups, with measurements of 822 x 109/L (95% CI, 707-936 x 109/L) for the prolonged-release group and 797 x 109/L (95% CI, 708-883 x 109/L) for the immediate-release cohort.
Both formulations reduced mean platelet levels (P <.0001 for noninferiority). During the maintenance phase, for the prolonged-release cohort, the adjusted mean platelet level was 281 x 109/L (95% CI, 254-311 x 109/L), compared with an adjusted mean platelet level of 305 x 109/L (95% CI, 276-337 x 109/L) for the immediate-release cohort.
Cardiac adverse events (AEs) were primarily palpitations and tachycardia and were slightly less numerous in the prolonged-release group, though this difference was not significant.
Gastrointestinal AEs were more common in the prolonged-release cohort (P =.048) and represented the only class of AEs that showed a significant difference between groups.
There were 18 serious AEs among 10 patients in the prolonged-release group, and 1 fatality considered unrelated to the study treatment. One serious AE in this group, a case of acute pancreatitis, was considered to be treatment-related.
One patient in the immediate-release group had 2 serious AEs deemed unrelated to the study treatment. AEs led to discontinuations in 3 patients from the prolonged-release group and 4 patients from the immediate-release group.
The study authors considered the prolonged-release formulation of anagrelide to be safe and effective against thrombocythemia and noted that it enabled more convenient administration than the immediate-release form.
Disclosure: This study was sponsored by AOP Orphan Pharmaceuticals. Please refer to the original study for a full list of disclosures.
Reference
- Gisslinger H, Buxhofer-Ausch V, Hodisch J, et al. A phase III randomized, multicentre, double blind, active controlled trial to compare the efficacy and safety of two different anagrelide formulations in patients with essential thrombocythaemia – the TEAM-ET 2.0 trial [published online March 28, 2019]. Br J Haematol. doi:10.1111/bjh.15824
