Background: C1 inhibitor (C1INH) has recently been approved in the USA for the treatment of acute attacks in hereditary angioedema (HAE) patients. The literature suggests that treatment with C1INH is most effective when administered early in an attack. Home infusion of C1INH allows for the earliest possible intervention since patients can initiate therapy at the first sign of symptoms. Methods: We performed an observational, prospective study on 39 subjects with HAE utilizing two groups of patients: one receiving on-demand C1INH replacement therapy in a medical facility and the other self-managing on-demand C1INH replacement therapy in the home setting under the supervision of a treating physician. All subjects completed online questionnaires weekly for 8 weeks. Results: There were statistically significant decreases in attack duration (p < 0.0001), pain medication use (p < 0.0001) and graded attack severity (p < 0.005) in the subjects who received C1INH in the home setting versus the clinic-based group. Attack frequency was similar between the groups. The home group experienced more frequent injection-related side effects; however, the clinic group noted more severe adverse events from C1INH. Conclusion: Physician-supervised self-managed C1INH replacement therapy is a safe and effective treatment for patients with HAE with potential benefits in diminishing attack duration and attack severity.
Purpose of review The advent of molecular techniques has resulted in the ability to tailor medications to specific protein targets. This review will emphasize several biological therapies, specifically directed toward cytokine receptors and inhibitors, and their role in the treatment of atopic and autoinflammatory diseases. Recent findings Translational research and the identification of the molecular pathophysiology of diseases have led to more targeted treatment approaches. The biologic modulators, encompassing monoclonal antibodies as cytokine inhibitors, receptor blocking antibodies and new fusion receptors are now being applied to diseases beyond their original application. Summary The expanded use of biological therapies has experienced success in the treatment of numerous disorders, especially in subsets of patients with disease that has been refractory to conventional therapies.
Hereditary angioedema (HAE) is an autosomal dominant disorder clinically characterized by recurrent episodes of angioedema. Until late-2008, HAE therapy in the United States was largely limited to antifibrinolytic agents or attenuated androgens. Although these drugs decrease the number and severity of angioedema attacks, they are associated with significant dose-related adverse effects. Recent advances have dramatically changed the management of HAE. As a result, we are embarking on a new era of treatment for this condition that includes effective on-demand treatment of attacks as well as effective prophylactic treatment. Herein we discuss the various treatment options for C1-inhibitor deficiency, focusing on new developments and literature published over the past year, as well as the additional patient considerations that should be addressed when determining the most appropriate patient-specific treatment plan.
A number of therapeutic agents are available for the treatment of asthma, including inhaled corticosteroids, long-and short-acting beta-agonists, leukotriene-modifying agents, long-and short-acting anticholinergic agents, chromones, theophylline, allergen immunotherapy, and oral corticosteroid therapy. All available therapies, despite their proven efficacy, are purely symptomatic including the topical steroids. This issue has led to the development of several biologic agents to aid in asthma management and to potentially alter the course of the disease by interfering with specific aspects of inflammation which may modify remodeling in the airways. Monoclonal antibodies have offered a class of therapeutic agents that enhance treatment options for patients with moderate-to-severe persistent asthma. As such, this article provides an overview of present and future monoclonal antibody therapies for the treatment of patients with severe asthma.
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