Omalizumab Seems Safe, Effective for Real-World Chronic Idiopathic Urticaria

By Marilynn Larkin

November 27, 2018

NEW YORK (Reuters Health) - In real-world patients with chronic idiopathic urticaria (CIU), omalizumab meets or exceeds the safety and efficacy results seen in clinical trials, researchers say.

Dr. Christopher Lee of Boston College in Chestnut Hill and colleagues conducted a meta-analysis of 67 observational studies and abstracts published from 2006-2018 that included at least one outcome in common with the other studies at a concurrent time of omalizumab use.

The studies, which were included in the group's previous systematic review, included 19 of patients with CIU or chronic spontaneous urticaria (CSU; another name for CIU); two with chronic autoimmune urticaria; five with chronic urticaria not otherwise specified (CU NOS); 38 with CIU, CSU, or CU NOS with comorbidities; and three with mixed samples of patients with urticaria.

As reported online November 14 in JAMA Dermatology, omalizumab use was associated with a 25.6-point reduction (indicating improvement) in weekly Urticaria Activity Scores (UAS7) across 15 studies involving 294 patients.

Although there was "substantive" heterogeneity in score improvement across the studies, according to the authors, "omalizumab was equally effective in reducing UAS7 scores across study subgroups."

Similarly, omalizumab was associated with a 4.7-point reduction in overall UAS among 1,158 patients; an average complete response rate of 72.2% in the same number of patients; an average partial response rate of 17.8% in 908 patients; and an average adverse event rate of any severity of 4% across 47 studies including 1,314 patients.

"Treatment effectiveness should be expected to vary in clinical practice," Dr. Lee told Reuters Health by email. "Since several factors were not reported consistently across studies...future work may reveal that dosing and/or duration of therapy may play a role in response similar to what was observed in clinical trials, or that effectiveness may vary between patients with and without angioedema or between patients with and without inducible urticarias."

"What would be most helpful in synthesizing real-world evidence on the effectiveness of therapies used in the management of CIU," he added, "is consistency in the reporting of dosing and duration of the study drug and other therapies, as well as harmonization of definitions of key outcomes like complete response that is used inconsistently across trials and across real-world studies."

Dr. Adam Friedman, Director of Translational Research, Department of Dermatology at George Washington School of Medicine and Health Sciences in Washington, DC, said by email, "This is an important study because...success is often understated in clinical trials as the lofty primary outcomes, the measures by which the FDA will judge and approve/deny a drug, are set to an extremely high bar, and treatment 'failures' could in fact be clinical successes by real-world standards."

"Safety can also be obscured in these sorts of trials, in either direction, so having real-world data is of the utmost importance," he told Reuters Health.

"I think (omalizumab) is a great treatment," he said. "It just requires the right office/set up, as this medication needs to be stirred for 30-40 minutes prior to use - the liquid is very thick and therefore impossible to inject without this preparation - and patients must wait around up to two hours after treatment, given the small risk of anaphylaxis."

"That said," he concluded, "it is a game changer for patients that have difficult-to-treat and disabling disease."

Dr. Holly Kanavy, Director of Dermatopharmacology, Montefiore Medical Center and Assistant Professor, Albert Einstein College of Medicine, in New York City said in an email to Reuters Health, "I do find that my clinical experience is quite similar to the finding of this paper. (Omalizumab) has become our most effective treatment option for CIU. While it does not work for all patients, it has helped many of my patients suffering with the condition."

The study was funded by Novartis. Two coauthors are employees and stockholders and two have received consulting fees from the company.


JAMA Dermatol 2018.