Safety and efficacy of physician-supervised self-managed C1 inhibitor replacement therapy.

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.

Cardiopulmonary arrest in a patient with delayed diagnosis of immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome.

We present the case of a 21-year-old male patient with a history of autoimmune nephritis, peripheral eosinophilia, eosinophilic esophagitis, and enteropathy who developed subacute worsening cardiomyopathy with systolic dysfunction. Diagnostic studies revealed a one-codon deletion in the FoxP3 gene, which led to the diagnosis of immune dysregulation polyendocrinopathy, enteropathy X-linked syndrome. Unfortunately, this patient suffered from cardiopulmonary arrest with resulting anoxic encephalopathy before diagnosis confirmation. Here, we discuss the key issues surrounding the diagnostic and therapeutic approaches to this patient's condition.

The role of monoclonal antibodies in the treatment of severe asthma.

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.

Biologic modulators in allergic and autoinflammatory diseases.

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.