Marc A. Riedl
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View article: Long-Term Safety and Effectiveness of Sebetralstat: Interim Analysis of KONFIDENT-S Open-label Extension
Long-Term Safety and Effectiveness of Sebetralstat: Interim Analysis of KONFIDENT-S Open-label Extension Open
Oral sebetralstat enabled compliance with treatment guidelines. No new safety signals were observed, and effectiveness for repeated attacks was consistent with the KONFIDENT trial results.
View article: Donidalorsen Treatment of Hereditary Angioedema in Patients Previously on Long-Term Prophylaxis
Donidalorsen Treatment of Hereditary Angioedema in Patients Previously on Long-Term Prophylaxis Open
Donidalorsen was well tolerated, decreased HAE attack rate, and improved QoL and disease control. Most patients preferred donidalorsen over their prior treatment. Further analyses are planned at week 52.
View article: Patient‐Reported Outcomes in the Phase III OASIS‐HAE Study of Donidalorsen for Hereditary Angioedema
Patient‐Reported Outcomes in the Phase III OASIS‐HAE Study of Donidalorsen for Hereditary Angioedema Open
Background Hereditary angioedema (HAE) is a rare disease characterized by unpredictable, frequently severe swelling that negatively impacts patients' quality of life (QoL). In the phase III OASIS‐HAE study (NCT05139810), donidalorsen reduc…
View article: Global frequency, diagnosis, and treatment of hereditary angioedema with normal C1 inhibitor
Global frequency, diagnosis, and treatment of hereditary angioedema with normal C1 inhibitor Open
HAE-nC1INH may be more prevalent than previously reported. Importantly, our findings revealed varying diagnostic and treatment approaches. Validated, accessible diagnostic biomarkers and clinical outcomes derived from rigorous clinical tri…
View article: Health care providers’ experiences with genetic testing in patients at risk for hereditary angioedema
Health care providers’ experiences with genetic testing in patients at risk for hereditary angioedema Open
The study found that HCPs consider genetic testing during a diagnostic workup for HAE; however, many lack confidence in ordering and/or interpreting results. The findings of this study suggest a need for (1) HCP education on the process of…
View article: Hereditary angioedema
Hereditary angioedema Open
Barriers to HAE diagnosis and effective treatment persist among US patients from underrepresented racial and ethnic groups.
View article: Interplay between on-demand treatment trials for hereditary angioedema and treatment guidelines
Interplay between on-demand treatment trials for hereditary angioedema and treatment guidelines Open
Over the past 2 decades, guidelines for the on-demand treatment of hereditary angioedema attacks have undergone significant evolution. Early treatment guidelines, such as the Canadian 2003 International Consensus Algorithm, often gated on-…
View article: Clinical burden, treatment, and disease control in patients with chronic spontaneous urticaria
Clinical burden, treatment, and disease control in patients with chronic spontaneous urticaria Open
Of patients with CSU who were prescribed treatment, more than 50% experienced uncontrolled CSU, which was associated with increased HCRU and health care costs.
View article: The Chronic Angioedema Registry (CARE): Rationale, Methods and Implementation
The Chronic Angioedema Registry (CARE): Rationale, Methods and Implementation Open
International audience
View article: Oral Sebetralstat for On-Demand Treatment of Hereditary Angioedema Attacks
Oral Sebetralstat for On-Demand Treatment of Hereditary Angioedema Attacks Open
Oral sebetralstat provided faster times to the beginning of symptom relief, reduction in attack severity, and complete attack resolution than placebo. (Funded by KalVista Pharmaceuticals; KONFIDENT ClinicalTrials.gov number, NCT05259917; E…
View article: COVID-19 and Hereditary Angioedema (HAE): Incidence, Outcomes and Mechanistic Implications
COVID-19 and Hereditary Angioedema (HAE): Incidence, Outcomes and Mechanistic Implications Open
Background: HAE patients have been postulated to be at increased risk for COVID-19 infection due to inherent dysregulation of the plasma kallikrein-kinin system. Only limited data has been available to explore this hypothesis. The aim of t…
View article: Clinical Progress in Hepatic Targeting for Novel Prophylactic Therapies in Hereditary Angioedema
Clinical Progress in Hepatic Targeting for Novel Prophylactic Therapies in Hereditary Angioedema Open
Hereditary angioedema (HAE) is typically caused by a deficiency of the protease inhibitor C1 inhibitor (C1INH). The absence of C1INH activity on plasma kallikrein and factor XIIa leads to overproduction of the vasoactive peptide bradykinin…
View article: Patient outcomes associated with subcutaneous C1INH prophylaxis for hereditary angioedema: a retrospective analysis
Patient outcomes associated with subcutaneous C1INH prophylaxis for hereditary angioedema: a retrospective analysis Open
Background Real-world data on subcutaneous C1INH (C1INH[SC]) usage and patient-level impacts on hereditary angioedema (HAE)-related outcomes and quality of life (QoL) are both lacking and challenging to generate using conventional study me…
View article: Refractory Status Epilepticus Associated With a Pathogenic Variant in TNFRSF13B
Refractory Status Epilepticus Associated With a Pathogenic Variant in TNFRSF13B Open
Febrile infection-related epilepsy syndrome (FIRES) is a rare epileptic syndrome characterized by new-onset refractory status epilepticus preceded by a febrile illness. Limited literature exists regarding the relationship between primary i…
View article: Evaluation of patient‐reported outcome measures for on‐demand treatment of hereditary angioedema attacks and design of KONFIDENT, a phase 3 trial of sebetralstat
Evaluation of patient‐reported outcome measures for on‐demand treatment of hereditary angioedema attacks and design of KONFIDENT, a phase 3 trial of sebetralstat Open
Background Hereditary angioedema (HAE) with C1‐inhibitor deficiency (HAE‐C1‐INH) is characterized by recurrent, debilitating episodes of swelling. Sebetralstat, an investigational oral plasma kallikrein inhibitor, demonstrated promising ef…
View article: Healthcare utilization of patients with hereditary angioedema treated with lanadelumab and subcutaneous C1-inhibitor concentrate
Healthcare utilization of patients with hereditary angioedema treated with lanadelumab and subcutaneous C1-inhibitor concentrate Open
Background: New hereditary angioedema (HAE) treatments have become available in recent years for the treatment of HAE due to C1-inhibitor (C1-INH) deficiency, including two subcutaneous (SC) options: a monoclonal antibody (lanadelumab) and…
View article: Long-term lanadelumab treatment improves health-related quality of life in patients with hereditary angioedema
Long-term lanadelumab treatment improves health-related quality of life in patients with hereditary angioedema Open
ClinicalTrials.gov Identifiers: NCT02586805 (HELP Study) and NCT02741596 (HELP open-label extension).
View article: Hereditary Angioedema: Impact of COVID-19 pandemic stress upon disease related morbidity and well-being
Hereditary Angioedema: Impact of COVID-19 pandemic stress upon disease related morbidity and well-being Open
Background: Individuals with hereditary angioedema (HAE) experience stress-related sequelae, including enhanced disease morbidity and reduced quality of life. The pervasive societal strain that surround the coronavirus disease 2019 (COVID-…