Effectiveness of omalizumab in adolescent and adult patients with chronic idiopathic/spontaneous urticaria: a systematic review of ‘real-world’ evidence

ABSTRACT

Introduction: Chronic idiopathic/spontaneous urticaria (CIU/CSU) is a dermatological condition characterized by itchy wheals and/or angioedema of continuous or intermittent duration of ≥6 weeks with a high burden of disease and impact on quality of life. Omalizumab is a recombinant humanized monoclonal antibody that inhibits the binding of IgE to high affinity receptors, and is approved for the CIU/CSU indication. The objective of this systematic review was to evaluate and synthesize the evidence on the real-world effectiveness of omalizumab in CIU/CSU in daily clinical practice.

Areas covered: This review of 84 observational effectiveness studies covers treatments (dosing, medication use), clinical outcomes (treatment response, disease activity, quality of life), and safety.

Expert opinion: The clinical outcomes observed across studies underscore the real-world effectiveness of omalizumab in the management of CIU/CSU. Continued treatment may assist patients showing an initial response to achieve a complete treatment response. Response rates are aligned with observed changes in disease activity, symptom experience, and quality of life, and this across subtypes of CIU/CSU. The positive therapeutic profile is complemented by a positive safety profile. The real-world evidence summarized here points convincingly at the high degree of effectiveness of omalizumab in the treatment of CIU/CSU in daily clinical practice.

KEYWORDS:

• Chronic idiopathic urticaria
• chronic spontaneous urticaria
• omalizumab
• effectiveness
• real-world evidence
• systematic review

Article highlights

• As there is no cure for chronic idiopathic/spontaneous urticaria (CIU/CSU), treatment is focused on improving clinical outcomes – from disease control and symptom management to quality of life. Omalizumab is a recombinant humanized monoclonal anti-IgE antibody approved initially for the treatment of severe allergic asthma, but since 2014 also for CIU/CSU in adults and adolescents 12 years of age and older who remain symptomatic despite treatment with non-sedating anti-histamines.

• A systematic review was conducted of 84 case reports, case series, and retrospective and prospective observational studies showing a trajectory from early exploration of the use of omalizumab in CIU/CSU to larger studies of treatment patterns and outcomes in daily clinical practice.

• There is strong and sustained real-world evidence that a broad range of CIU/CSU patients respond to treatment with omalizumab in terms of reductions in disease activity and improvement in disease control; translating into significant improvements in quality of life.

• The most commonly prescribed initiation and titration dose is 300mg, though about one-third of patients are started on 150mg. The most common frequency of administration is Q4W, though titrated dosing regimens may vary.

• The safety profile in daily clinical practice is in line with results of phase III trials and the product label.

• Most real-world studies reviewed here were case reports, case series, and retrospective observational studies. There is a continued need for prospective real-world effectiveness and safety studies.

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Declaration of interest

J. Bernstein received consultancy fees from Novartis Pharmaceuticals Corporation for work on this study. He is affiliated with Bernstein Clinical Research Center, LLC which was under contract with Novartis Pharmaceuticals Corporation as a PI to conduct clinical research outside of the submitted work. He has received speaker fees from Novartis Pharmaceuticals Corporation as well as consulting fees from Genentech. He is an author on the Joint Task Force for Practice Parameters for Urticaria guideline and the GALEN international guideline for urticaria. A. Kavati is an employee and stockholder of Novartis Pharmaceuticals Corporation. M. Tharp received consultancy fees from Novartis Pharmaceuticals Corporation for work on this study. B. Ortiz is an employee and stockholder of Novartis Pharmaceuticals Corporation. I. Abraham, K. MacDonald and K. Denhaerynck are affiliated with Matrix45. By company policy, employees are prohibited from owning equity in client organizations (except through mutual funds or other independently administered collective investment instruments) or contracting independently with client organizations. Matrix45 provides services similar to those described in this article to other biopharmaceutical companies on a non-exclusivity basis. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed. A reviewer on this manuscript has disclosed working with all pharmaceutical companies involved in the field of urticaria and have ordered that the worldwide guidelines on urticaria. A reviewer on this manuscript disclosed having been speaker for Novartis and FAES Farma.

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Additional information

Funding

This systematic review was sponsored by Novartis.