Quality of Life Benefits Seen With Long-term Hemlibra Use in Trials

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by Vanda Pinto |

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Hemlibra and life quality

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Preventive treatment with Hemlibra (emicizumab) sustainably improved physical health-related quality of life and led to fewer missed workdays among people with severe hemophilia A without inhibitors, an analysis of pooled data from two clinical trials found.

According to researchers, these findings add to previous data showing that Hemlibra not only lowers the number of bleeds, but is also considered a more convenient treatment option by patients.

These life-quality findings were reported in the study, “The effect of emicizumab prophylaxis on long-term, self-reported physical health in persons with haemophilia A without factor VIII inhibitors in the HAVEN 3 and HAVEN 4 studies,” published in the journal Hemophilia.

Hemophilia A is an inherited bleeding disorder caused by the lack of a blood-clotting protein called factor VIII (FVIII). Administering the missing FVIII prophylactically, or as a preventive measure, is the standard treatment but one that can be burdensome, as the process is frequent and time-consuming.

Constant bleeding episodes, which may still occur despite prophylaxis, as well as treatment burden have a significant impact on patients’ health-related quality of life (HRQoL).

Hemlibra is an approved treatment for managing the symptoms of hemophilia A, administered as an under-the-skin injection, once weekly, every two, or every four weeks. (Originally developed by Roche’s subsidiary Chugai Pharmaceutical, it is now marketed in the U.S. by Genentech.)

The safety and efficacy of Hemlibra at preventing bleeding episodes in people with hemophilia A was previously shown in four Phase 3 trials: HAVEN 1 (NCT02622321), HAVEN 2 (NCT02795767), HAVEN 3 (NCT02847637), and HAVEN 4 (NCT03020160). In addition, most participants stated their preference for Hemlibra over their previous treatment regimen, possibly because of its convenience and lower overall burden.

Significant improvements in HRQoL in the HAVEN studies were also during 25 weeks of preventive Hemlibra use by teenage and adult patients with severe hemophilia A with or without FVIII inhibitors. Of note, inhibitors are neutralizing antibodies that lower the effectiveness of standard replacement therapies.

Now, an international team of researchers — assisted by scientists with Roche and Genentech — reported additional, extended data from a joint analysis of HAVEN 3 and 4 on preventive Hemlibra treatment and patients’ HRQoL. This analysis focused specifically on adults with hemophilia A without FVIII inhibitors, since they were the majority in both trials, who had remained on Hemlibra prophylaxis for up to 73 weeks, or nearly 1.5 years.

The team used the Haemophilia-Specific Quality of Life Questionnaire for Adults (Haem-A-QoL) to assess HRQoL. This test has a total of 46 items across 10 domains, with each domain given a score; a total score is then determined from all 10 domains. In general, lower scores indicate higher, or better, HRQoL.

The physical health domain reflects the impact of hemophilia on a person’s physical health status. A 10-point reduction in this domain is considered the limit for clinically meaningful improvements.

Health status was assessed using the EuroQoL 5-Dimensions 5-levels (EQ-5D-5L) test, and the impact of treatment on work was evaluated based on self-reported missed workdays.

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Data covering 176 adults from the HAVEN 3 and 4 studies, with a median age of 39 years, were in the analysis.

In the 24 weeks prior to starting with Hemlibra, 55% of these adults received episodic treatment, 45% prophylaxis, and 51%  experienced nine or more bleeds. Before enrolling in the study, 70% of patients had one or more target joints, or joints frequently affected by bleeding episodes.

The mean physical health Haem-A-QoL and total score improved after starting Hemlibra treatment. The analysis showed a mean 12-point reduction in physical health, and a mean 8.6-point reduction in the Haem-A-QoL total score from the study’s start to week 73.

In addition, 54% of patients experienced clinically meaningful improvement in the physical health domain, achieving score reductions greater than 10 points by week 73. Participants with a lower HRQoL before using Hemlibra, including those receiving episodic treatment or having nine or more bleeds and target joints before study entry, showed the highest improvements in physical health in the Haem-A-QoL.

With preventive use of Hemlibra, more of these adults reported never missing a work day by week 73 compared with the study’s start (90.9% vs. 75.0%). Again, this improvement was more evident in patients with a lower initial HRQoL.

“These findings add to the evidence suggesting that [Hemlibra] can provide an improved HRQoL in [severe hemophilia A patients] without FVIII inhibitors with less burdensome prophylaxis,” the researchers wrote.

However, no notable changes were seen on overall health status, as assessed by the EQ-5D-5L test.

“With [Hemlibra], clinically meaningful improvements were observed in [physical health Haem-A-QoL] scores in more than half of adult [hemophilia A patients] without FVIII inhibitors in HAVEN 3 and 4,” the team wrote, adding “these improvements are complementary to demonstrated efficacy of [Hemlibra] in bleed prevention.”