Many angioedema patients see relief with Xolair in new real-world study
Benefits seen whether or not patients also had hives alongside swelling
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Treatment with Xolair (omalizumab), an approved therapy for allergic asthma and chronic spontaneous urticaria (CSU), was associated with strong symptom control in people with mast cell–mediated angioedema, according to a large real-world study.
Benefits were observed regardless of whether patients experienced wheals, itchy skin bumps commonly known as hives, alongside swelling, the main symptom of angioedema, the researchers noted.
“This first large, long-term, multicenter real-world study demonstrates the high effectiveness and safety of [Xolair] in mast cell-mediated angioedema,” the researchers wrote. “[Xolair]’s performance was independent of the presence of subordinary wheals, providing strong support for its clinical use in this patient population.”
The study, “Performance of omalizumab in patients with mast cell-mediated angioedema with and without wheals,” was published in The Journal of Allergy and Clinical Immunology: In Practice.
Study explores Xolair use in mast cell angioedema
Angioedema occurs when fluid builds up beneath the skin or mucous membranes, leading to swelling in areas such as the face, lips, or throat. In mast cell–mediated angioedema, swelling is triggered by mast cell activation, which plays a role in allergic and inflammatory responses.
This type of angioedema is considered a subtype of CSU, a condition marked by recurring hives with or without swelling. It is typically managed according to urticaria treatment guidelines.
Xolair, jointly marketed by Novartis and Genentech, is approved in the U.S. and Europe for the treatment of allergic asthma and CSU. Given as a subcutaneous (under-the-skin) injection, it targets immunoglobulin E (IgE), a protein involved in immune responses, including allergic reactions.
For people with mast cell–mediated angioedema who do not respond to treatment with high-dose antihistamines, Xolair is recommended as an add-on treatment. While its effectiveness and safety have been well established in CSU, where hives are the main symptom, less is known about its use in patients whose main manifestation is angioedema.
“To date, the performance of [Xolair] in patients with predominantly angioedema has only been reported in case series and small [group] studies,” the researchers wrote.
To address this gap, an international team led by researchers in the Netherlands conducted a real-world study involving 148 people with mast cell–mediated angioedema treated with Xolair across 14 specialized urticaria centers between June 2014 and February 2022.
Real-world study looks at outcomes in 148 patients
Participants, most of them women (74%) with a mean age of 45, were classified as having angioedema alone (45%) or angioedema with hives as a secondary feature (55%). At the start of treatment, disease control was poor in both groups, based on scores from the Urticaria Control Test, a measure of symptom control.
Among participants with available data, most (80%) received standard-dose Xolair, while 20% were treated with higher-than-standard doses.
By the end of treatment, 77% of participants achieved a good or complete response, 14% had a partial response, and 9% showed little or no response. Response rates were similar in participants with angioedema alone and those with wheals (78% vs. 76%), indicating that the presence of hives did not affect treatment effectiveness.
During follow-up, 41% of participants discontinued treatment — most often due to well-controlled disease (63%) — while fewer stopped due to ineffectiveness (18%) or side effects (5%). Nearly half (48%) remained on treatment, and 11% were lost to follow-up.
Statistical analysis of drug survival, an estimate of how long patients stay on treatment based on how well it works and how safe it is, further supported these findings. The probability of remaining on treatment due to well-controlled disease was 82% at one year, 67% at two years, and 58% at five years, with similar outcomes in patients with and without wheals.
Longer disease linked to slower path to symptom control
Further analysis showed that patients whose disease lasted more than two years before starting treatment tended to remain on therapy longer before achieving disease control.
“This possibly reflects a lower chance of spontaneous remission in patients with longstanding disease, thus requiring prolonged treatment,” the researchers wrote.
In contrast, a fast response — defined as good disease control within four weeks and observed in nearly half (48%) of participants — was associated with a shorter time to treatment discontinuation.
Among participants who stopped treatment due to well-controlled disease, more than half (53%) restarted therapy after about five months. All those with available data responded again after restarting treatment.
“This is the first study to present multinational, real-world data on the performance of [Xolair] in a large [group] of patients with isolated and predominantly [mast cell–mediated angioedema] over an 8-year study period,” the researchers wrote. “The high effectiveness and safety were supported by the finding that most patients discontinued [Xolair] due to well-controlled disease, while ineffectiveness and side effects were minor reasons for discontinuation.”
They also noted that “all outcomes were independent of the presence of subordinary wheals” and that the findings support Xolair use in “the management of mast cell–mediated angioedema as part of CSU.”
