ASH Annual Meeting and Exposition

ASH Annual Meeting and Exposition

December 23, 2019
2 min read

Hemophilia A patients ‘highly satisfied’ after switching to emicizumab

You've successfully added to your alerts. You will receive an email when new content is published.

Click Here to Manage Email Alerts

We were unable to process your request. Please try again later. If you continue to have this issue please contact

ORLANDO — Patients with hemophilia A who had higher annual bleeding rates and poor adherence to infusion-based prophylaxis were more likely to switch treatment to emicizumab and were “highly satisfied with this switch,” according to researchers at the ASH Annual Meeting and Exposition.

Emicizumab (Hemlibra, Genentech) is a bispecific humanized monoclonal antibody that is administered via weekly subcutaneous injections. It is approved for routine prophylaxis to prevent or reduce the frequency of bleeding episodes among adults and children with hemophilia A.

“As the first in a new class of substitutive, non-factor hemophilia A therapy, acceptance of emicizumab by patients with hemophilia is unclear,” Hanny Al-Samkari, MD, and Stacy E. Croteau, MD, MMS, of Harvard Medical School, wrote. “The impact of emicizumab on treatment adherence, pursuit of athletic activity, psychosocial connection to the hemophilia patient community and patient satisfaction have not been assessed. Additionally, reasons for choosing to initiate or not initiate emicizumab in patients who have been offered it have not been evaluated.”

To address these questions, Al-Samkari and Croteau conducted a nationwide survey of patients with hemophilia A who were offered bleeding prophylaxis. The voluntary, anonymous survey was electronically distributed through hemophilia treatment centers and the National Hemophilia Foundation.

Overall, 126 patients (67 adults and 59 parents of children) responded to the survey. Most respondents (89%) were familiar with emicizumab, and 43% of patients had received the agent. Less than 20% of patients were receiving inhibitors.

Patients who used emicizumab had higher median annualized bleeding rates before emicizumab use compared with patients who never received emicizumab, regardless of inhibitor status (5 bleeds per year vs. 2 bleeds per year). Further, 76.7% of patients who used emicizumab reported good or excellent adherence to infusion-based prophylaxis before emicizumab use vs. 96.8% of patients who never received emicizumab.

Using a 5-point Likert scale, the researchers showed that patients not receiving inhibitors believed that subcutaneous administration (score of 4.33) and the ability to receive one dose or less per week (score of 4.24) were just as important or even more so than the safety (score of 4.27) and effectiveness (score of 4.15) of emicizumab. In contrast, patients receiving inhibitors were less concerned with subcutaneous administration (score of 3.82) and viewed effectiveness (score of 4.27) and safety (score of 4.18) as the most important reasons for initiating emicizumab.

Most patients were highly satisfied with their decision to start emicizumab, giving it a rating of 4.42 or higher. They also reported that emicizumab had a positive impact on their quality of life (4.42 among non-inhibitor patients; 4.55 among inhibitor patients) and they have fewer bleeding episodes than before receiving the agent (4.18 among non-inhibitor patients; 4.55 among inhibitor patients).


Sixty-four survey respondents said they decided not to switch to emicizumab. The primary reasons for declining emicizumab use included safety concerns (score of 4.38), unknowns with a new treatment (score of 4.22) and lack of laboratory monitoring (score of 4.10).

“Effectiveness was not a major concern,” the researchers wrote.

Sixty percent of patients who did not switch to emicizumab discussed the agent with their physicians. Among them, 90% reported that they wanted to wait for more information on its use. The remaining 10% could not start emicizumab because their insurance denied coverage.

One-third of patients who did not discuss emicizumab with their physician “felt they lacked the information to decide whether to use it,” according to the researchers. – by Stephanie Viguers


Al-Samkari H, Croteau SE. Abstract 3395. Presented at: ASH Annual Meeting and Exposition; Dec. 7-10, 2019; Orlando.

Disclosures: Al-Samkari reports receiving research funding from Agios, Amgen and Dova, and being a consultant for Agios, Dova and Moderna. Croteau reports receiving honoraria from Bayer, CSL Behring, Genentech, Novo Nordisk, Octapharma, and Shire; receiving research funding from Novo Nordisk, Pfizer and Spark Therapeutics, and being a consultant for Bayer, CSL Behring, Genentech, Novo Nordisk and Shire.