On Alhemo, patients without inhibitors report better life quality
71% of patients preferred Alhemo over their previous hemophilia treatment
Prophylactic, or preventive, treatment with Alhemo (concizumab) is associated with better quality of life and a lower treatment burden for people with hemophilia A or B without inhibitors, according to patient-reported data from a Phase 3 trial.
The ongoing study, called explorer8 (NCT04082429), is investigating the efficacy and safety of Alhemo, when given via once-daily subcutaneous (under-the-skin) injections, in people with hemophilia A or B without inhibitors.
Patient-reported outcome results “can provide further insights into the potential benefits of [Alhemo] through patients’ perspectives on [health-related quality of life] and treatment experience,” researchers wrote.
The study, “Concizumab prophylaxis in people with hemophilia A or B without inhibitors: patient-reported outcome results from the phase 3 explorer8 study,” was published in Research and Practice in Thrombosis and Haemostasis.
Hemophilia is mainly caused by genetic mutations that lead to a deficiency or dysfunction of specific proteins needed for the blood to clot, which place patients at an increased risk of excessive and prolonged bleeding. Hemophilia A is caused by a deficiency in a clotting protein known as factor VIII (FVIII), while hemophilia B is caused by a deficiency in factor IX (FIX).
Inhibitors can reduce efficacy of replacement therapies
The standard treatment for hemophilia is replacement therapy, which involves supplying a version of the missing clotting factor to patients. However, some patients develop neutralizing antibodies, or inhibitors, against clotting factors and this may reduce a treatment’s efficacy.
Alhemo is an antibody-based therapy that is designed to promote blood clotting through a mechanism that doesn’t rely on FVIII or FIX, allowing it to be used to treat hemophilia patients with inhibitors. The therapy is approved in the U.S. and other countries as a daily prophylaxis to prevent or reduce the frequency of bleeds in people, 12 and older, with hemophilia A or B and inhibitors. It is given by a subcutaneous injection.
In the explorer8 trial, Alhemo’s safety and efficacy were assessed in 148 men and boys, 12 and older, with hemophilia A or B without inhibitors. Patients were randomly assigned to receive either daily prophylaxis with Alhemo (127 patients) for at least 32 weeks (about eight months) or no prophylaxis (21 patients).
The trial met its main goal, with Alhemo reducing the annualized bleeding rates for treated spontaneous and traumatic bleeding episodes by about 80%.
In the published study, researchers evaluated data from patient-reported outcomes assessed over the course of explorer8, including changes in health-related quality of life, treatment burden, and treatment preferences.
Health-related quality of life was assessed using the 36-item short-form health survey and the Haemophilia Quality of Life Questionnaire for Adults.
Patients on Alhemo prophylaxis reported better scores on health survey
According to the health survey, patients on Alhemo prophylaxis reported generally higher (better) mean scores across all domains after six months compared with patients who didn’t receive treatment.
Similar results were seen for the life-quality questionnaire, also favoring Alhemo prophylaxis over no prophylaxis.
The researchers also used the Patient Global Impression of Severity and the Patient Global Impression of Change (PGI-C) questionnaires to assess the level and changes in patients’ physical function.
Overall, a higher proportion of patients on Alhemo described their level of physical function as very good (27.5% vs. 7.7%) or good (46.1% vs. 38.5%), compared with those not receiving prophylaxis. Similar results were seen for the PGI-C questionnaire.
Treatment burden was assessed using the Hemophilia Treatment Experience Measure, with results demonstrating that scores decreased for all patients, indicating a reduction in treatment burden after about six months. However, scores in the domains of “interference” and “treatment bother” increased among patients who were not on prophylaxis.
As for treatment preference, most patients (70.9%) treated with Alhemo said they preferred it, while 10.9% had no preference, and 1.6% preferred their previous treatment. The most common reasons why patients preferred Alhemo included requiring less time to administer (61.1%), having fewer bleeds (33.3%), being less painful to inject (28.9%), and easier to remember to inject (28.9%).
“Data collected in this study provided additional support for [Alhemo] prophylaxis compared with no prophylaxis as a treatment option for patients with [hemophilia A or B],” the researchers wrote.
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