Hem A patients in France report high satisfaction with Hemlibra

Patients report better quality of life, and reduced pain and bleeding after one year

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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Hemophilia A patients at a single center in France reported high rates of satisfaction with Hemlibra (emicizumab) after a year of real-world use, a study has found.

Patients reported improvements in health and life quality, as well as reductions in pain and bleeding with the therapy, but problems with chronic pain persisted for some patients.

“Overall, all patients were satisfied with emicizumab [Hemlibra] therapy,” researchers wrote.

The study, “Assessment of the clinical perception, quality of life and satisfaction of patients with severe congenital haemophilia A without inhibitor after 1 year of emicizumab therapy,” was published in the journal Haemophilia

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Hemlibra is an antibody-based therapy designed to mimic the actions of factor VIII (FVIII), an essential blood clotting protein that hemophilia A patients lack. In doing so, it works to restore the blood clotting cascade, preventing or reducing the frequency of bleeds.

The therapy is currently approved in both the U.S. and Europe for patients diagnosed with hemophilia A with or without inhibitors, a type of neutralizing antibody against the FVIII protein that can lower the effectiveness of standard replacement therapies.

Hemlibra can be given at home as an under-the-skin injection, with maintenance dosing typically done once every week to once every four weeks. That dosing schedule is thought to offer convenience and quality-of-life benefits compared with other preventive treatments, which may require into-the-vein infusions at a healthcare facility multiple times per week.

At the Haemophilia Treatment Centre of Cochin Hospital in France, patients starting on Hemlibra participate in a patient education program in which they consult with a pharmacist at the start of treatment, two months later, and again after a year. At this final meeting, pharmacists discuss the patients’ quality of life and satisfaction.

In this study, researchers reported findings from that program, with a focus on the evolution of patient perceptions about health, quality of life, and overall satisfaction.

The analysis included 38 adult men with hemophilia A who started treatment between June 2020 and March 2021. Patients had a mean age of 45.5 years, and none had FVIII inhibitors.

Health parameters, including general health, chronic pain, spontaneous bleeding, post-traumatic bleeding, and ability to identify bleeds, were assessed at the first consultation and again at the third meeting about a year later.

Quality of life was also evaluated with the EQ-5D-3L questionnaire, which encompasses five domains: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression.

Overall, all patients were satisfied with emicizumab [Hemlibra] therapy.

Patients report fewer bleeds, reduced chronic pain, and better health

Patients reported significant reductions in both types of bleeds (spontaneous and post-traumatic), as well as declines in chronic pain and improved general health after one year of treatment with Hemlibra. They also reported they had generally retained their ability to identify bleeds, with no significant change after treatment.

On a scale of 1 to 100, with 100 indicating best possible health, patients reported their health to be at 69.6.

Still, a persistent chronic pain issue was identified in the EQ-5D-3L. Most patients (73.7%) reported some pain problems, while 13.1% reported extreme problems. Moreover, 28 patients (73.7%) continued to use painkillers.

Other quality-of-life outcomes demonstrated benefits of Hemlibra treatment, with most patients reporting independence in their daily lives (89.5%) and comfort with usual activities (71.1%).

Moreover, nearly half (44.7%) said they were not having any mobility problems. Some patients were able to resume activities they had previously stopped (39.5%), while others started new ones (21.1%).

Among the 12 people who used physical therapy, half thought the need had decreased since starting treatment with Hemlibra, while one-third required more sessions than they previously had.

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While 60.5% of participants weren’t having issues with anxiety, 15 people reported some or extreme problems.

Despite several perceived improvements, patients indicated that Hemlibra “came too late to really improve the deleterious effects that had occurred because of haemophilia,” the researchers wrote, noting that they believed starting treatment in childhood or adolescence was likely to have a greater effect on problematic symptoms like pain and mobility problems.

In terms of medication management, about half of the patients (52.6%) forgot at least one Hemlibra dose, but all knew how to react. Side effects mainly involved infusion site reactions, including redness, skin rash, or local pain.

A total of 23 people required FVIII replacement infusions through the year due to suspected bleeds (18 people) or as a preventive measure due to scheduled surgeries/procedures (six people).

In general, patients rated their satisfaction with Hemlibra as a 9.1 out of 10, with none indicating that they wished to go back to a previous treatment. Satisfaction with the Hemlibra administration kit was rated as a 3.2 out of 6.

Altogether, the findings suggest high satisfaction with real-world Hemlibra use, which was associated with perceived health and life quality gains for patients. Larger and longer studies are warranted to extend these findings, the team noted.