On-demand treatment of hereditary angioedema attacks: patient-reported utilization, barriers, and outcomes.

Background: Hereditary angioedema (HAE) is clinically characterized by recurrent attacks of cutaneous and submucosal swelling.

Objective: To investigate real-world timing, potential barriers, and impact of delaying on-demand treatment (OD) of HAE attacks METHODS: Patients with HAE (type I or II) aged ≥12 years with ≥1 treated (Treated Cohort) or untreated (Untreated Cohort) attack in the past 3 months were recruited by the US HAE Association. Respondents completed a 20-minute, self-reported, online survey about their last HAE attack.

The complexities of decision-making associated with on-demand treatment of hereditary angioedema (HAE) attacks.

Background: Hereditary angioedema (HAE) is characterized by debilitating attacks of tissue swelling in various locations. While guidelines recommend the importance of early on-demand treatment, recent data indicate that many patients delay or do not treat their attacks.

Objective: This survey aimed to investigate patient behavior and evaluate the key factors that drive on-demand treatment decision-making, as reported by those living with HAE.

Recognition, Description, and Variability of Spasticity in Individuals With Multiple Sclerosis and Potential Barriers to Clinician-Patient Dialogue: Results From SEEN-MSS, a Large-Scale, Self-Reported Survey.

Background: The experience with spasticity varies among individuals with multiple sclerosis and spasticity (MSS), as they may not recognize it as spasticity or have the language to describe their symptoms. This can lead to potential delays in diagnosis and treatment.

Methods: Symptoms and Emotions Exploration Needed in Multiple Sclerosis Spasticity was an online survey completed by 1177 individuals with MSS in 2021.

Caregiver-reported outcomes with real-world use of cannabidiol in Lennox-Gastaut syndrome and Dravet syndrome from the BECOME survey.

Purpose: Plant-derived highly purified cannabidiol (CBD) reduced the frequency of seizures associated with Lennox-Gastaut syndrome (LGS) and Dravet syndrome (DS) and improved the overall condition of patients in placebo-controlled phase 3 clinical trials. Anecdotal reports also suggest a positive effect on nonseizure outcomes. In this study, we aimed to identify, through a caregiver survey which nonseizure outcomes were most likely to change in these patients.

Lower mortality with andexanet alfa vs 4-factor prothrombin complex concentrate for factor Xa inhibitor-related major bleeding in a U.S. hospital-based observational study.

Background: Well-designed studies with sufficient sample size comparing andexanet alfa vs 4-factor prothrombin complex concentrate (4F-PCC) in routine clinical practice to evaluate clinical outcomes are limited.

Objectives: To compare in-hospital mortality in patients hospitalized with rivaroxaban- or apixaban-related major bleeding who were treated with andexanet alfa or 4F-PCC.

Methods: An observational cohort study (ClinicalTrials.

Hereditary Angioedema With Normal C1 Inhibitor: US Survey of Prevalence and Provider Practice Patterns.

Background: Hereditary angioedema (HAE) with normal C1-INH (HAE-nl-C1INH) is phenotypically similar to HAE resulting from C1-INH deficiency (HAE-C1INH). Confirmatory diagnostic tests for HAE-nl-C1INH are limited and few clinical study data exist regarding management of the condition. Therefore, survey studies may provide initial estimates of prevalence, diagnosis, and management patterns of this condition.

A Coala-T-Cannabis Survey Study of breast cancer patients' use of cannabis before, during, and after treatment.

Background: The goal of this study was to characterize cannabis use among patients with breast cancer, including their reasons for and timing of use, their sources of cannabis information and products, their satisfaction with the information found, their perceptions of its safety, and their dialogue about cannabis with their physicians.

Methods: United States-based members of the Breastcancer.org and Healthline.

Real-world management of oral factor Xa inhibitor-related bleeds with reversal or replacement agents including andexanet alfa and four-factor prothrombin complex concentrate: a multicenter study.

We describe the real-world utilization and outcomes associated with managing oral factor Xa inhibitor (FXai)-related major bleeds. Electronic records from 45 US hospitals were queried (ICD-10-CM billing codes D68.32, T45.

Barriers to timely diagnosis of interstitial lung disease in the real world: the INTENSITY survey.

Background: The diagnosis of idiopathic pulmonary fibrosis (IPF) and other interstitial lung diseases (ILD) presents significant clinical challenges. To gain insights regarding the diagnostic experience of patients with ILD and to identify potential barriers to a timely and accurate diagnosis, we developed an online questionnaire and conducted a national survey of adults with a self-reported diagnosis of ILD.

Methods: A pre-specified total of 600 subjects were recruited to participate in a 40-question online survey.