A novel point-of-care assay demonstrated sensitivity to monitor coagulation factor replacement therapy in patients with severe hemophilia A, with and without inhibitors, according to recent study findings.
The ClotChip assay is a novel factor-replacement therapy monitoring tool that uses a dielectric microsensor to assess whole-blood coagulation in patients with severe hemophilia A.
Debnath Maji of Case Western Reserve University, Cleveland, and colleagues evaluated the novel assay in attempt to address the unmet need of reliable monitoring of coagulation factor–replacement therapy. While global haemostasis assays are available to measure the missing function, these tools require expert interpretation, are technically challenging to operate, and may be unavailable at the bedside, they wrote in.
They evaluated the analytical capabilities of the assay in 6 patients with inhibitors and 12 patients without inhibitors. A total of 50 healthy controls were included in the study.
The team collected whole blood samples pre- and postcoagulation factor–replacement therapy. Measurements were also conducted on the thrombin generation assay (TGA), thromboelastography (TEG), and rotational thromboelastometry (ROTEM) global assays for comparison.
After analysis, the researchers found that ClotChip T-peak parameters showed a significant reduction for samples obtained post–factor replacement therapy versus pretherapy among patients with and without inhibitors (P = .028 and P = .0001, respectively).
Additionally, ClotChip T-peak values exhibited strong correlations with factor VIII clotting activity, clotting time parameters of ROTEM, and peak thrombin and endogenous thrombin potential of TGA.
“Taken together, these data suggest that the ClotChip T-peak parameter is sensitive to detection and correction of coagulopathy in children with haemophilia with and without inhibitors, as indicated by T-peak reaching reference‐range values after coagulation factor replacement therapy,” the researchers wrote.
One key limitation of the study was the small sample size, which may limit the generalizability of the findings.
The novel assay may be an appropriate tool for “real-world” decision making around the use of coagulation factor replacement therapy in patients with severe hemophilia A, they added.
The study was funded by the National Institutes of Health, the American Heart Association, and a Veterans Affairs Research Center of Excellence at Case Western Reserve University. The authors reported financial affiliations with Bayer, Bioverativ, Shire, and XaTek.
SOURCE: Maji D et al. Haemophilia. 2019 Jul 7. .