Does Emicizumab Change Routine Clinical Practice?
With the increasing number of patients with hemophilia A being treated with emicizumab, the question arises whether its use in any way changes established clinical practice. A team of American authors sought answers based on a survey conducted among the treating physicians of these patients. The findings were presented at the 13th Annual EAHAD Congress, which took place at the beginning of February in The Hague, Netherlands.
Survey
An online survey was conducted in the USA, which took approximately 30 minutes to complete. Fifty hematologists from a total of 20 centers, where both adult and pediatric patients with hemophilia A (HA) with and without FVIII inhibitors are treated, participated. At least 2 years of experience with administering emicizumab and managing at least 3 patients per month under emicizumab therapy were required.
Findings
The majority of treated patients had severe HA, most did not have an inhibitor, and were previously treated with other drugs. The most commonly cited reasons for initiating emicizumab therapy were the presence of an inhibitor in the patients and the significant burden associated with previous treatments (16% of respondents).
When asked whether care for HA patients treated with emicizumab differed from other hemophiliacs, 58% responded negatively. However, doctors also reported less frequent testing of FVIII activity and inhibitor presence in patients receiving emicizumab.
Only 34% of respondents reported that they had changed their recommendations for managing breakthrough bleeding in clinical practice. Most suggested that patients contact their hemophilia center in case of difficulties. Almost half (42%) recommended that their patients keep 3-4 doses of FVIII concentrate or bypass agent at home to manage breakthrough bleeding. Most educated their patients on what to do if a dose of emicizumab is missed.
40% of physicians reported approximately the same and 50% reported better or significantly better adherence to prescribed treatment after switching to emicizumab.
According to 84% of respondents, the level of physical activity in HA patients remained the same or increased after switching to emicizumab. 48% recommended gradually increasing physical activity after switching to emicizumab.
More than half of the participating specialists were already treating HA patients on emicizumab with immune tolerance therapy (22%) or were considering it for their patients (38%). Among those who initiated immune tolerance, most reported using lower doses of FVIII and shortening the duration of immune tolerance compared to previous experiences.
22% (11/50) of respondents reported that their patients underwent surgical procedures while on emicizumab therapy. Ten of these eleven physicians administered prophylaxis with another hemostatic agent before the procedure due to concerns about high bleeding risk.
Conclusion
Most of the surveyed specialists stated that the standard of care for hemophiliacs treated with emicizumab remains unchanged. However, with the increasing number of physicians administering emicizumab to their patients, it is necessary to pay attention to the signals of changing clinical practice.
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Source: Owens W. E., Patel A. M., Poulos K. et al. A survey of hematologists in The United States to understand disease management of patients with hemophilia A (PwHA) treated with emicizumab (Emi). Poster 205. 13th annual congress of European Association for Haemophilia and Allied Disorders, The Hague, February 5-7, 2020.
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