Study of the Week: Nucala’s Effectiveness in EGPA Confirmed in Real-World Study

Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest or importance and go more in-depth. In this story we will talk about the details of the study and explain why it’s important, who will be impacted, and more.

If you read our short form research stories and find yourself wanting to learn more, you’ve come to the right place.


This week’s study is…

Effectiveness and safety of mepolizumab in combination with corticosteroids in patients with eosinophilic granulomatosis with polyangiitis

We previously published about this research in a story titled “Nucala and Corticosteroids Can Increase EGPA Remission” which can be found here. The study was originally published in the research journal Arthritis Research and Therapy. You can view the full text of the study here.

The researchers were affiliated with the University of Occupational and Environmental Health in Japan.

What Happened?

In the clinical trial setting, mepolizumab (marketed as Nucala) has proven to be a safe and effective therapy for the treatment of eosinophilic granulomatosis with polyangiitis (EGPA). However, no study has been conducted that evaluated the impact of the treatment in real-world practice. Real-world practice refers to the treatment environment outside of the controlled setting of a clinical trial or study. 

The goal of this research was to investigate the impact of Nucala under real-world conditions. This study specifically utilized a 300 mg/month dose of the drug in patients with relapsed/refractory disease that was resistant to corticosteroid treatment. The patients were followed for one year. A total of 16 EGPA patients were treated as part of this study. The researchers gathered retroactive data for these patients from the year prior to beginning treatment with Nucala.

The primary endpoint was the remission rate at 12 months following administration, while secondary endpoints included vasculitis damage index (VDI), Birmingham vasculitis activity score (BVAS), changes in other corticosteroid/immunosuppressant use, eosinophil counts, retention rate, and frequency of adverse events. The treatment regimen produced a 75 percent remission rate at one year, and after treatment with Nucala, eosinophil count, BVAS, and corticosteroid use decreased. 

Some patients halted their concomitant steroid use altogether. The treatment retention rate (percentage of patients who continued treatment through the 12 month period) was 100 percent, with only three patients experiencing infections. 

About Eosinophilic Granulomatosis with Polyangiitis (EGPA)

Eosinophilic granulomatosis with polyangiitis (EGPA) is a form of ANCA-associated vasculitisThis autoimmune disease is characterized by inflammation impacting the small and mid-sized blood vessels; these patients have a history of airway allergic sensitivity. While a direct cause hasn’t been determined, a history of asthma-associated lung abnormalities, asthma, and allergy is the primary known risk factor. The disease occurs in three stages; a prodromal stage characterized by allergic rhinitis, asthma, and airway inflammation; an eosinophilic stage, in which the numbers of eosinophils are extremely high, leading to fever, gastrointestinal bleeding, night sweats, weight loss, cough, and abdominal pain; and vasculitic stage, in which the blood vessels become inflamed. This can lead to blood clots, arterial atrophy, abdominal pain/damage, and heart disease, which causes around half of all deaths linked to EGPA. While there is no cure, a variety of immune-suppressing drugs can control the symptoms. To learn more about EGPA, click here.

Why Does it Matter?

The results from this study have helped to confirm the benefits of Nucala as a treatment for EGPA. Compared to the prior treatment standards, the therapy is able to produce a greater rate of remission and reduction in disease activity. In addition, the confirmation that Nucala treatment can allow for a lower dose of corticosteroids to be used is also important. This is because corticosteroid use, particularly over a long period of time to manage a chronic condition like EGPA, can cause severe adverse effects. 

Nucala can also serve as an important supplementation to steroid use in patients whose disease is no longer responding well to corticosteroids on its own. The treatment was also able to improve symptoms soon after administration. While the impact of this study was somewhat limited by its use of pre-treated patients as the control group and small sample size, the scientists nevertheless concluded:

“[Nucala] therapy was sufficiently effective in controlling disease activity and prevented adverse events induced by [corticosteroids] and immunosuppressants.”

The researchers also emphasized that future studies should focus on the longer-term impacts of Nucala treatment to determine if further dose reduction or elimination of corticosteroids would be possible while avoiding symptom relapse.  

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