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An analysis of 2 phase 3 trials supporting the use of pegcetacoplan (PEG) in the treatment of paroxysmal nocturnal hemoglobinuria (PNH) assessed the benefits of this treatment compared with eculizumab (ECU) and supportive care measures.

A study using a large Korean health database found that people with a rare hair loss disorder called primary cicatricial alopecia were more likely to also have additional health problems like diabetes and high blood pressure compared with people who had other hair loss conditions or no hair loss at all.

Investigators explored the utility of unplanned resections, also called reresection, among patients who have soft tissue sarcomas.

Amy Shapiro, MD, medical director, Indiana Hemophilia and Thrombosis Center, discusses the clinical trials that influenced plasminogen's (Ryplazim; Kedrion Biopharma) FDA approval, as well as details valuable information for the treatment of patients with plasminogen deficiency.

Amit Singal, MD, medical director of the Liver Tumor Program at UT Southwestern Medical Center, discussed how the etiologies of hepatocellular carcinoma (HCC) and underlying liver function impact treatment approach.

Cardiovascular disease (CVD) risk was reduced by 1.2% in the intention-to-treat analysis and 5% in the per protocol analysis for adults aged 75 and older.

A retrospective study of over 100 pediatric and adolescent patients showed that providing virtual training for insulin pump use was both safe and effective.

However, investigators said there is some evidence suggesting attention-deficit/hyperactivity disorder may be more common in certain age groups of children with hemophilia.

In the final part of our interview with Binod Dhakal, MD, lead investigator of the CARTITUDE-4 study, he looks to the future of earlier treatment for relapsed/refractory multiple myeloma.

Patients who took the weekly glucagon-like peptide 1 (GLP-1) receptor agonist were 21% less likely to experience major kidney disease events and 29% less likely to die due to cardiovascular causes.

Newly published findings from the pivotal phase 3 BE HEARD I and BE HEARD II trials demonstrated that bimekizumab was well tolerated and produced clinically meaningful responses in patients with moderate to severe hidradenitis suppurativa.

A retrospective analysis found glucocorticoid treatment to reduce comorbidities in adults with Duchenne muscular dystrophy (DMD) and assessed the relationship between anthropometric measures and respiratory function and functional abilities.

The data refute the rapid-onset gender dysphoria hypothesis by showing that the initially higher depressive symptoms reported by youths transitioning from cisgender to transgender or gender diverse were not significant after accounting for exposure to LGBT violence, the researchers wrote.

Although an aerobic cooldown indeed lowers blood glucose levels in adults with type 1 diabetes after fasted exercise, by itself, the impact it has on hyperglycemia is insignificant.

From 2015 to 2020, the number of patients opting for valve-in-valve transcatheter aortic valve replacement (ViV-TAVR) over redo surgical aortic valve replacement (SAVR) rose significantly, from 35.3% to 62.5%.

A 68-year-old man was diagnosed with ocular myasthenia gravis after presenting with double-vision and photophobia.

Investigators continue to seek more information on how to optimally integrate treatment with tyrosine kinase inhibitors (TKIs) into an established concurrent chemoradiotherapy regimen.

A new report finds secondary immunodeficiency disease (SID) also increases the risk of mortality in patients with B-cell malignancies.

Vamorolone (Agamree) is a first-in-class corticosteroid to treat Duchenne muscular dystrophy (DMD) in patients 2 years and older.

The FDA approved the first 2 biosimilars referencing aflibercept (Eylea). However, patent litigation has left it unclear when the biosimilars will come to market.

Patients taking semaglutide for a year saw significant improvements in exercise capacity and lost 2.9% more body weight compared with patients taking placebo.

Amy Shapiro, MD, medical director, Indiana Hemophilia and Thrombosis Center, continues her discussion on plasminogen deficiency type 1 by highlighting the recent FDA approval of plasminogen (Ryplazim; Kedrion Biopharma), the first treatment specifically indicated for this disorder.

Fatima Rodriguez, MD, MPH, from Stanford University, and Matthew DeCamp, MD, PhD, from the University of Colorado, joined Michael Howell, MD, MPH, of Google, on the stage at ATS 2024 to discuss artificial intelligence (AI) in health care.

Data from real-world and clinical-trial settings on frontline monotherapy treatment with the KRAS inhibitor sotorasib both show similar progression-free survivals and a high likelihood that the treatment’s efficacy is not affected with dose reduction.

A recent study found that screening for hepatocellular carcinoma (HCC) is associated with reduced mortality, highlighting the need to promote HCC screening for patients with known risk factors.

The findings add to recent research on the growing utilization, expenditure, and prices of Duchenne muscular dystrophy (DMD) therapies in the current landscape, an area health care policy could potentially address.

The FDA has approved tarlatamab (Imdelltra; Amgen), a novel bispecific T-cell engager, to treat patients with extensive-stage small cell lung cancer (SCLC).