Systematic review of omalizumab for refractory clonal and non-clonal mast cell activation syndrome.

Patients with mast cell activation syndrome (MCAS) can be refractory to standard antimediator therapy. Alternative treatment options to reduce disease burden and improve quality of life are needed. To compile the evidence that supports the use of omalizumab for patients with refractory MCAS.

Interplay between on-demand treatment trials for hereditary angioedema and treatment guidelines.

Over the past two decades, guidelines for the on-demand treatment of hereditary angioedema (HAE) attacks have undergone significant evolution. Early treatment guidelines, such as the Canadian 2003 International Consensus Algorithm, often gated on-demand treatment by attack location and/or severity. Pivotal trials for on-demand injectable treatments (plasma-derived C1 esterase inhibitor [C1INH], icatibant, ecallantide [US only], recombinant C1INH), which were approved in the US and EU between 2008-2014, were designed accordingly.

Stereotactic Body Radiotherapy vs Sorafenib Alone in Hepatocellular Carcinoma: The NRG Oncology/RTOG 1112 Phase 3 Randomized Clinical Trial.

Importance: Most patients with locally advanced hepatocellular carcinoma (HCC) recur within the liver following systemic therapy.

Objective: To determine whether stereotactic body radiation therapy (SBRT) improves outcomes in patients with locally advanced HCC compared with sorafenib alone.

Design, Setting, And Participants: This multicenter phase 3 randomized clinical trial randomized patients with HCC 1:1 to sorafenib or SBRT followed by sorafenib, stratified by performance status, liver function, degree of metastases, and macrovascular invasion.

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.