Esperoct Stabilizes/Improves Health-related Quality of Life of Patients With Severe Hemophilia A, Study Suggests

Esperoct Stabilizes/Improves Health-related Quality of Life of Patients With Severe Hemophilia A, Study Suggests

Treatment with Esperoct led to stabilization and/or improvement of health-related quality of life (HRQoL) in patients with severe hemophilia A from all age groups, a study suggests.

The study, “Health-related quality-of-life and treatment satisfaction of individuals with hemophilia A treated with turoctocog alfa pegol (N8-GP): a new recombinant extended half-life FVIII,” was published in Patient Preference and Adherence.

Hemophilia is a genetic blood disorder that affects the body’s ability to make blood clots to prevent excessive bleeding. In hemophilia A, this inability of the blood to clot is caused by the lack of a specific clotting protein, called factor VIII (FVIII).

Current treatments for hemophilia A are based on providing the missing FVIII to patients as a prophylaxis, or preventive measure, to avoid spontaneous bleeding episodes.

Esperoct (N8-GP, turoctocog alfa pegol) — Novo Nordisk‘s human recombinant (lab-made) extended half-life factor VIII — was recently approved by the U.S. Food and Drug Administration (FDA) as a prophylactic therapy to reduce the frequency of bleeds and for on-demand use to control spontaneous bleeding episodes in children and adults with hemophilia A.

In two previous Phase 3 clinical trials — Pathfinder2 (NCT01480180) and Pathfinder5 (NCT01731600) — treatment with Esperoct led to reductions in the frequency of bleeding episodes without significant adverse side effects in patients with severe hemophilia A.

“It was hypothesized that there would be improvements in patients’ HRQoL and increased treatment satisfaction (TS), as N8-GP [Esperoct] extends the duration of coverage, which results in less bleeds, and requires fewer injections,” the investigators wrote.

In this study, the researchers gathered and summarized the findings from Pathfinder2 and Pathfinder5 to determine changes in patients’ HRQoL and TS after treatment with Esperoct.

The patients’ HRQoL was measured using the Haemo-QoL questionnaire (for children and their parents) and the Haem-A-QoL questionnaire (for adult patients). TS was assessed using the Hemophilia Satisfaction (Hemo-Sat) questionnaire.

All questionnaires were scored from zero to 100, with lower scores indicating better HRQoL and higher TS. Negative changes in scores from baseline to the end of both trials indicated that the patients’ HRQoL and TS improved with Esperoct.

The study analyzed data from 14 children from 4 to 7 years old; 21 children from 8 to 11; 10 adolescents from 13 to 16; and 163 adults with severe hemophilia A who participated in Pathfinder2 or Pathfinder5.

Pooled data revealed negative changes in Haemo-QoL scores reported by children and adolescents (-14.0 for children 4 to 7; -3.6 for children 8 and 11; and -0.1 for adolescents 13 and 16), and their parents (-11.5 for those with children 4 and 7; -8.6 for parents of children 8 and 11; and -4.0 for parents of adolescents 13 and 16).

In addition, negative mean changes in Haem-A-QoL were also reported by adult patients (-3.1 for those receiving on-demand treatment, and -2.3 for those receiving Esperoct prophylactically).

These negative mean changes indicated that the scores were lower, and therefore, that the health-related quality of life for these patients had improved.

Finally, analyses showed that most parents of children and adolescents with hemophilia A, as well as adult patients, had high levels of TS at the end of both trials.

“Treatment with N8-GP resulted in a good disease-specific HRQoL of children, adolescents, and adults with severe hemophilia A. While most patients entered their respective trials with a good disease-specific HRQoL, the HRQoL of patients was either maintained or further improved when treated with N8-GP. Adults and parents of children and adolescents further reported high levels of treatment satisfaction with N8-GP,” the researchers wrote.

Joana is currently completing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. She also holds a BSc in Biology and an MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that make up the lining of blood vessels — found in the umbilical cord of newborns.
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Joana is currently completing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. She also holds a BSc in Biology and an MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that make up the lining of blood vessels — found in the umbilical cord of newborns.
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