Contemporary trials of medications to treat hereditary angioedema (HAE) attacks define efficacy endpoints using Patient Global Impression of Severity (PGI-S) and Patient Global Impression of Change (PGI-C) scales; these differ from the visual analog scale (VAS) and treatment effect questionnaire (TEQ) used in legacy recombinant human C1 esterase inhibitor (rhC1-INH) studies. This post hoc analysis aimed to evaluate pooled rhC1-INH trial data, mapped to contemporary HAE clinical trial endpoints and designs. Pooling data from three rhC1-INH trials for acute HAE attacks (NCT00225147, NCT01188564, NCT00262301), this analysis applied results from a bookmarking study to convert TEQ and VAS measurements to PGI-S and PGI-C scales. Kaplan–Meier estimates were calculated for time to complete resolution (TTCR; defined as “none” on PGI-S) and time to onset of symptom relief (TOSR; defined as “a little better” on PGI-C at ≥ 2 consecutive time points within 12 h). Log-rank tests were used to determine differences between treatment groups. Across trials, 48 and 60 patients received rhC1-INH 50 U/kg and placebo, respectively. The most common attack locations were peripheral (47.9
BACKGROUND:Although current treatment options for acute exacerbations of antihistamine refractory chronic spontaneous urticaria (CSU) are generally considered safe and effective, patients have breakthrough symptoms necessitating treatment. Epinephrine injections were once used for the treatment for acute urticaria or acute exacerbations of chronic urticaria or angioedema, but their use has declined. The development of an intranasal epinephrine spray (ARS-2) offers a needle-free alternative for the treatment of CSU exacerbations. OBJECTIVE:To assess the efficacy and safety of ARS-2 for the treatment of exacerbations of CSU. METHODS:This was a phase 2, single-dose, randomized, placebo-controlled crossover efficacy study in which adult patients (n = 21) experiencing an acute flare or exacerbation of urticaria symptoms were treated with ARS-2 (1 or 2 mg) or placebo. Urticaria symptoms and severity were assessed based on both patient-reported and investigator-rated assessments. RESULTS:Relative to placebo, both 1- and 2-mg doses of ARS-2 resulted in lower patient-reported hive and pruritus scores (P < .05) and a lower investigator-reported extent of urticaria and erythema scores (P < .05). Additionally, a greater percentage of patients receiving ARS-2 were considered by investigators to have been effectively treated. Patient-reported satisfaction scores were also significantly higher for both doses of ARS-2 relative to placebo. Only minor adverse events were reported. CONCLUSION:ARS-2 may offer a safe and effective treatment option for urticaria exacerbations.
Intravenous immunoglobulin (IVIG) therapy is a well-documented and effective treatment for primary immunodeficiencies (PI). Subcutaneous immunoglobulins (SCIG) have emerged as an effective alternative for some patients that offers additional flexibility. Currently, caprylate/chromatography purified IGSC (human) 20
