The use of low-dose prophylaxis may be more beneficial than episodic treatment for the management of hemophilia, according to a recent study published in Vox Sanguinis.
The review found that low-dose prophylaxis led to a reduced annualized bleeding rate, as well as improvement in joint function, pain, and health-related quality of life compared to episodic treatment for patients with hemophilia. Low-dose prophylaxis did not improve arthropathy already caused by earlier bleeding events, however.
Also, based on the results, treatment should be individualized and based on pharmacokinetic and/or clinical parameters. Protocols with individualized dose escalations had better results than fixed low-dose prophylaxis and standard-dose protocols.
Individualized low-dose prophylaxis protocols were also found to be more cost-effective than standard dose protocols which the authors highlighted for developing and resource-limited countries seeking to implement treatments for patients with hemophilia. Most patients are managed with on-demand treatment in developing countries due to the high cost of clotting factors, the authors said.
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“The benefits of [low-dose prophylaxis] over [episodic treatment] in improving the standard of living of hemophilia patients in developing countries with almost similar cost are evident,” the authors said. “It is unreasonable to deny the benefits of prophylaxis to 70%-80% of the world hemophilia population.”
The authors did point out that while low-dose prophylaxis was more desirable than episodic treatment, it was still insufficient to fully treat hemophilia. Previous research has shown that patients with factor levels above 1 IU/dl had fewer bleeding episodes and only rarely had arthropathy leading to a prophylactic goal of trough levels above this threshold.
Several of the studies analyzed in the review article found conflicting results with patients regularly below this level having no difference in hemarthrosis and bleeding rate to patients above the threshold. The authors highlighted the need for further long-term research into necessary trough levels to prevent spontaneous joint bleeds and subclinical bleeding as well as better standardized outcome parameters.
A total of 19 studies were included in the review article analysis and featured studies comparing episodic treatment and low-dose prophylaxis in the same and different patient cohorts as well as long-term analysis of patients on low-dose prophylaxis and patients following individualized treatment protocols.
Shetty S, Bansal S, Kshirsagar S, Rangarajan S, Hajirnis K, Phadke V. Low‐dose prophylaxis and its impact on the health of haemophilia patients. Vox Sang. Published online April 18, 2022. doi:10.1111/vox.13278