William R. Lumry

The 2025 WAO Guidelines for the classification, diagnosis, and treatment of hereditary angioedema, with consideration of worldwide disparities

Daniel Vázquez, Pedro Giavina-Bianchi, Darío Josviack, Allen P. Kaplan et autres

The 2025 World Allergy Organization (WAO) Guidelines for the Classification, Diagnosis, and Treatment of Hereditary Angioedema (HAE) with Consideration of Worldwide Disparities provide a comprehensive, evidence-informed, and globally applicable framework for the care of this rare and potentially life-threatening disorder. HAE is …

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Sebetralstat for on-demand treatment of hereditary angioedema: A pooled analysis of placebo-controlled clinical trials

Emel Aygören-Pürsün, Danny M. Cohn, Nancy Agmon-Levin, Aleena Banerji et autres

Background Clinical trial designs evaluating on-demand therapies for hereditary angioedema attacks have evolved in response to changes in treatment guidelines. Sebetralstat, an oral plasma kallikrein inhibitor, was evaluated in 2 randomized, placebo-controlled clinical trials, which instructed early treatment of attacks with no …

A Multicentric Clinical Study to Evaluate Pharmacokinetics, Efficacy, and Safety of Immune Globulin Subcutaneous 20% Weekly/Biweekly Dosing in Treatment-Experienced Patients and Loading/Weekly Maintenance Dosing in Treatment-Naïve Patients with Primary Immunodeficiency

William R. Lumry, Michael Ostaric Palumbo, Connie Hsu, Iftikhar Hussaın et autres

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% is approved to treat PI in North …

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Sebetralstat for On‐Demand Treatment of Mucosal Hereditary Angioedema Attacks in KONFIDENT‐S

Jonathan A. Bernstein, Emel Aygören‐Pürsün, Mauro Cancian, Danny M. Cohn et autres

BACKGROUND: Hereditary angioedema (HAE-C1INH) attacks involving mucosal tissue may progress rapidly and often lead to substantial morbidity. Severe laryngeal attacks can be fatal without prompt administration of on-demand treatment. This prespecified interim analysis evaluated the safety and effectiveness of sebetralstat in laryngeal …

us, de, it, nl, vn, mk, nz, jp, Afrique du Sud, pl, hu (code pays fourni par la source)

Barriers and promoters to adapting research findings to clinical care in hereditary angioedema in the United States: A qualitative study

Esther L. Langmack, Dana Ravyn, Rob Lowney, Beth Goodwin et autres

Treatment options for hereditary angioedema (HAE) have evolved due to discoveries in basic and clinical research. HAE clinical guidelines emphasize optimizing quality of life through attack prevention strategies that include long-term prophylaxis. We sought to identify barriers to and promoters of research …

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Long-Term Safety and Effectiveness of Sebetralstat: Interim Analysis of KONFIDENT-S Open-label Extension

John T. Anderson, Laurence Bouillet, Teresa Caballero, Mauro Cancian et autres

BACKGROUND: Poor compliance with hereditary angioedema guidelines for on-demand treatment is common due to challenges with parenteral administration. Sebetralstat, an oral plasma kallikrein inhibitor, demonstrated faster times to beginning of symptom relief, reduction in attack severity, and complete resolution than placebo in …

hu, fr, es, it, vn, jp, mk, us, ca, gb, at, nz, de, Afrique du Sud, bg, pl, nl (code pays fourni par la source)

Garadacimab in hereditary angioedema due to normal C1INH with F12/PLG mutations

Danny M. Cohn, Avner Reshef, Petra Staubach, William R. Lumry et autres

BACKGROUND: Hereditary angioedema (HAE) with normal C1 inhibitor (HAE-nC1INH), including HAE arising from F12 (HAE-FXII) or PLG (HAE-PLG) mutations, has a high unmet therapeutic need. OBJECTIVE: Our aim was to explore long-term prophylaxis with monthly subcutaneous garadacimab (anti-activated factor XII mAb) in …

nl, il, de, ch, us (code pays fourni par la source)

Donidalorsen Treatment of Hereditary Angioedema in Patients Previously on Long-Term Prophylaxis

Marc A. Riedl, Jonathan A. Bernstein, Joshua Jacobs, Timothy Craig et autres

BACKGROUND: Hereditary angioedema (HAE) is a rare, potentially life-threatening disorder characterized by episodes of tissue swelling. Donidalorsen, an investigational ligand-conjugated antisense oligonucleotide, reduces plasma prekallikrein production. OBJECTIVE: We report an interim analysis on safety, efficacy, quality of life (QoL), and treatment preference …

us, vn, it, nl (code pays fourni par la source)

Long-Term Real-World Outcomes in Patients with Hereditary Angioedema Receiving Lanadelumab for 3 or More Years

William R. Lumry, Mark A. Davis-Lorton, Daniel F. Soteres, Lucy Earl et autres

Introduction: Hereditary angioedema (HAE) is a rare genetic disorder characterized by unpredictable, painful swelling attacks that significantly impair patients' quality of life (QoL). Clinical trials of lanadelumab led to its approval for long-term prophylaxis in patients with HAE; however, real-world data on …

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Sustained Effectiveness, Tolerability, and Safety of Long-Term Prophylaxis with Lanadelumab in Hereditary Angioedema: The Prospective, Phase 4, Noninterventional EMPOWER Real-World Study

Jonathan A. Bernstein, Stephen Betschel, Paula Jane Busse, Aleena Banerji et autres

INTRODUCTION: Lanadelumab is approved for long-term prophylaxis of hereditary angioedema (HAE) attacks in patients aged ≥ 2 years in the USA and aged ≥ 12 years in Canada. The EMPOWER Study (NCT03845400) evaluated the real-world effectiveness and safety of lanadelumab in male …

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Factors contributing to non-compliance with on-demand treatment guidelines in hereditary angioedema

Stephen Betschel, Jonny Peter, William R. Lumry, Hilary Longhurst et autres

BACKGROUND: Hereditary angioedema (HAE) is a rare genetic disorder characterized by painful and potentially life-threatening tissue swelling due to a deficiency or dysfunction of the C1 esterase inhibitor protein. Despite the availability of comprehensive on-demand treatment guidelines, compliance to guideline recommendations remains …

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Évaluation d’une administration hebdomadaire versus bimensuelle de Xembify® chez des patients déjà substitués par immunoglobulines, et dose de charge et d’entretien chez des patients naïfs de traitement atteints de déficits immunitaires primitifs

Elsa Mondou, William R. Lumry, Manlio Palumbo, Chin‐Ying Stephen Hsu et autres

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