Nucala helps patients with rare form of ANCA-associated vasculitis

EGPA patients report ‘quick’ health-related improvements, study finds

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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Adults with active eosinophilic granulomatosis with polyangiitis (EGPA), a rare type of ANCA-associated vasculitis (AAV), report improvements in health-related quality of life as early as one week after starting treatment with Nucala (mepolizumab).

These are the findings of a multicenter European study that evaluated the effects of Nucala on two patient-reported tests: the AAV patient-reported outcome (AAV-PRO) questionnaire and the patient global assessment (PtGA).

These findings suggest that questionnaires designed to capture feedback from patients may provide “valuable insights into treatment response beyond traditional disease activity measures,” researchers wrote.

The study, “Impact of mepolizumab on the AAV-PRO questionnaire in eosinophilic granulomatosis with polyangiitis: data from a European multicentre study,” was published in the journal Rheumatology.

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Like other AAV types, EGPA is marked by inflamed small blood vessels, which can lead to damage in multiple organs. Clumps of eosinophils, a type of immune cell, help drive the damaging inflammation to small blood vessels that’s seen in EGPA.

GlaxoSmithKline’s Nucala is approved in the U.S. as an add-on to standard glucocorticoids in adults with EGPA. In the European Union, it can be used to treat adults and children 6 years and older with relapsing or treatment-resistant disease. It works by blocking a signaling molecule called IL-5, which recruits eosinophils to inflamed tissues.

Data from clinical trials and real-world studies have shown that Nucala treatment helps patients achieve disease remission while reducing the need for glucocorticoids, which may have serious side effects when taken for long periods of time or at high doses. However, it’s unclear how patients perceive their improvement.

To learn more, a team of researchers in France, Germany, Italy, and Spain, working as part of the European EGPA Study Group, assessed PtGA and AAV-PRO scores in EGPA patients at the study’s start (baseline) and after about a week, two weeks, a month, three months, and six months of Nucala treatment.

PtGA asks patients to rate how active they believe their EGPA was in the past week on a scale from 0-100. AAV-PRO includes 29 items across six domains: organ-specific symptoms, systemic (whole-body) symptoms, treatment side effects, social and emotional impact, concerns about the future, and physical function. A score from 0-100 is calculated for each domain. In both measures, higher scores indicate more active disease.

The study included 70 people with active EGPA, median age 56 years, who started Nucala treatment between January 2021 and June 2022. Nucala was approved in the European Union for EGPA in late 2021. Most patients had relapsing or treatment-resistant disease.

About two-thirds (67.1%) received 100 mg of Nucala every four weeks, while the remaining 32.9% were given the approved dose of 300 mg once a month. Patients on the low dose had more active disease, but less long-term damage compared with those on the high dose.

The median PtGA score was 60 at baseline, and it showed a significant reduction as early as after the first week of treatment, with the score progressively dropping up to six months, when the median score was 10.

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Patients had significantly lower AAV-PRO score after six months

AAV-PRO data showed that, at the start of the study, concerns about the future scored the highest (median of 35), followed by organ-specific symptoms (30) and social and emotional impact (27). Domains that scored lowest were systemic symptoms (18.8) and treatment side effects (15).

After about two weeks of Nucala treatment, patients reported significantly lower median scores in all AAV-PRO domains, except treatment side effects, which showed a significant reduction after a month of treatment.

By six months, participants had significantly lower AAV-PRO scores in all domains — particularly in organ symptoms and physical function — indicating sustained improvements.

“The psychosocial implications of the disease and its treatment significantly influence the patients’ perception of symptom burden, as reflected by relatively high baseline concerns about the future and social and emotional impact scores and their less pronounced improvement,” the researchers wrote.

Higher values in the Birmingham Vasculitis Activity Score (BVAS), indicating more active disease, were significantly linked to higher scores in all AAV-PRO domains, except for treatment side effects. This suggests feedback from patients provides “valuable insights into several aspects of their health-related quality of life,” the researchers wrote.

The psychosocial implications of the disease and its treatment significantly influence the patients’ perception of symptom burden, as reflected by relatively high baseline concerns about the future and social and emotional impact scores and their less pronounced improvement.

Women reported significantly more severe systemic symptoms, treatment side effects, social and emotional impact of the disease, and concerns about the future. Patients on higher doses of glucocorticoids reported more concerns about the future. Higher blood levels of C-reactive protein, an inflammation marker, were significantly linked to more severe systemic symptoms.

After six months, 78.6% of patients achieved complete remission, and nearly one-third (31.4%) stopped glucocorticoids due to sustained remission. During this period, there was a significant reduction in BVAS and daily glucocorticoid dose.

While achieving remission at six months didn’t change AAV-PRO scores, stopping glucocorticoids was associated with a trend toward reduced side effects and concerns about the future.

The findings show Nucala “was associated with a significant and quick improvements in [health-related quality of life], with positive implications for … clinical, functional, and psychosocial components,” the researchers wrote. “Integrating the AAV-PRO questionnaire into daily practice may improve care quality and foster patient engagement (toward] a precision-medicine approach.”