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Allergy, Asthma and Immunology
Oral Sebetralstat for On-Demand Treatment of Hereditary Angioedema Attacks
por iva...@gmail.com May 31, 2024



Marc A. Riedl, M.D., Henriette Farkas, M.D., Ph.D., D.Sc., Emel Aygören-Pürsün, M.D. et al. NEJM Published May 31, 2024 DOI: 10.1056/NEJMoa2314192

Abstract

BACKGROUND

Approved on-demand treatments for hereditary angioedema attacks need to be administered parenterally, a route of administration that is associated with delays in treatment or withholding of therapy.

METHODS

In this phase 3, double-blind, three-way crossover trial, we randomly assigned participants at least 12 years of age with type 1 or type 2 hereditary angioedema to take up to two oral doses of sebetralstat (300 mg or 600 mg) or placebo for an angioedema attack. The primary end point, assessed in a time-to-event analysis, was the beginning of symptom relief, defined as a rating of “a little better” on the Patient Global Impression of Change scale (ratings range from “much worse” to “much better”) at two or more consecutive time points within 12 hours after the first administration of the trial agent. Key secondary end points, assessed in a time-to-event analysis, were a reduction in attack severity (an improved rating on the Patient Global Impression of Severity [PGI-S] scale, with ratings ranging from “none” to “very severe”) at two or more consecutive time points within 12 hours and complete attack resolution (a rating of “none” on the PGI-S scale) within 24 hours.

RESULTS

Primary and Key Secondary End Points.
A total of 136 participants were assigned to one of six trial sequences, with 110 treating 264 attacks. The time to the beginning of symptom relief with the 300-mg dose and the 600-mg dose was faster than with placebo (P<0.001 and P=0.001 for the two comparisons, respectively), with median times of 1.61 hours (interquartile range, 0.78 to 7.04), 1.79 hours (1.02 to 3.79), and 6.72 hours (1.34 to >12), respectively. The time to reduction in the attack severity with the 300-mg dose and the 600-mg dose was faster than with placebo (P=0.004 and P=0.003), with median times of 9.27 hours (interquartile range, 1.53 to >12), 7.75 hours (2.19 to >12), and more than 12 hours (6.23 to >12). The time to complete resolution was faster with the 300-mg and 600-mg doses than with placebo (P=0.002 and P<0.001)...

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Efficacy and Safety of Donidalorsen for Hereditary Angioedema
por iva...@gmail.com May 31, 2024


Marc A. RiedlM.D.Raffi TachdjianM.D., M.P.H.William R. LumryM.D., et al. NEJM Published May 31, 2024 DOI: 10.1056/NEJMoa2402478

Abstract

BACKGROUND

Hereditary angioedema is a rare disorder characterized by episodic, potentially life-threatening swelling caused by kallikrein–kinin dysregulation. Long-term prophylaxis can stabilize this system. Donidalorsen, an antisense oligonucleotide, specifically reduces prekallikrein expression.

METHODS

In this phase 3, double-blind, randomized trial, we assigned patients with hereditary angioedema to receive donidalorsen (80 mg subcutaneously) or placebo once every 4 or 8 weeks. The primary end point was the time-normalized number of investigator-confirmed hereditary angioedema attacks per 4 weeks (attack rate) from week 1 to week 25.

RESULTS

Patients with a Reduction in Hereditary Angioedema Attacks.
A total of 90 patients received donidalorsen every 4 weeks (45 patients), donidalorsen every 8 weeks (23 patients), or placebo (22 patients). The least-squares mean time-normalized attack rate was 0.44 (95% CI, 0.27 to 0.73) in the 4-week group, 1.02 (95% CI, 0.65 to 1.59) in the 8-week group, and 2.26 (95% CI, 1.66 to 3.09) in the placebo group...

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Rhinomanometry: A Comprehensive Review of Its Applications and Advancements in Rhinology Practice
por iva...@gmail.com May 31, 2024


Patil N, Jain S (May 30, 2024) Rhinomanometry: A Comprehensive Review of Its Applications and Advancements in Rhinology Practice. Cureus 16(5): e61370. doi:10.7759/cureus.61370


Abstract

Emerging trends in rhinomanometry research
Rhinomanometry is a pivotal diagnostic technique in rhinology, providing a quantitative assessment of nasal airflow and resistance. This review comprehensively examines the historical development, principles and clinical applications of rhinomanometry, emphasising its role in diagnosing nasal obstructions, preoperative evaluations and monitoring therapeutic outcomes. Recent advancements, including the integration with imaging technologies and the application of artificial intelligence (AI), have significantly enhanced the accuracy and utility of rhinomanometry...

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