Clinical

Promising topline results from a phase 3B study show the efficacy and quality of life (QOL) improvements of iptacopan (Fabhalta) as a twice-daily oral monotherapy for adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who transitioned from anti-C5 therapies.

Data presented at the 2024 American Heart Association (AHA) Scientific Sessions showed that continuous treatment with acoramidis (Attruby; BridgeBio Pharma) significantly reduced the risk of all-cause mortality (ACM) and cardiovascular-related hospitalizations (CVH) in patients with transthyretin amyloid cardiomyopathy (ATTR-CM).

Top coverage from the European Respiratory Society (ERS) Congress 2024 spanned various topics, including artificial intelligence–powered robots transforming respiratory care and late-breaking findings on an epilepsy drug showing promise for treating obstructive sleep apnea.

While race-aware models showed superior statistical calibration across racial and ethnic groups, their benefits in clinical decision-making were less pronounced than expected.

Gene therapy partially restores visual processing in the geniculostriate pathway of patients with Leber congenital amaurosis type 2 while maintaining compensatory activity in the retinotectal pathway.

The 6-year mandatory Increasing Organ Transplant Access Model aims to boost kidney transplants and address disparities by incentivizing hospitals, enhancing care coordination, and measuring transplant outcome performance.

Meaningful change thresholds for the EORTC Quality of Life Questionnaire in chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) are: −11/+11 for symptom burden, −16/+16 for physical condition/fatigue, and −16/+13 for worries/fears.

Investigators were seeking clarification on optimal sequencing of the immune checkpoint inhibitor atezolizumab following treatment failure and disease progression after receipt of nivolumab and pembrolizumab.

COVID-19 vaccination significantly reduced anxiety and depression in patients with idiopathic pulmonary fibrosis (IPF), improving their mental health one month after the first dose.

The findings show that increases in serum glial fibrillary acidic protein throughout B-cell depletion therapy are associated with disability worsening despite not relapsing—known as progression independent of relapse activity.

A decade after the discovery of CALR, the results are the first for a possible therapy for CALR-mutated MPNs to reach the clinic.

November is Diabetic Eye Disease Awareness Month. Diabetes is the leading cause of preventable blindness in the US. Here is a look at recent research related to diabetic eye diseases.

The European Commission has approved mirvetuximab soravtansine for select patients with pretreated folate receptor–alpha (FRα)-positive, platinum-resistant ovarian cancer.

Investigators said patients experienced adverse effects in line with those expected for myeloablative conditioning with busulfan following treatment with betibeglogene autotemcel (beti-cel; Zynteglo; bluebird bio).

The single-center report showed the therapy had a favorable safety profile and manageable side effects in children with relapsed or refractory (R/R) acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS).

A study in Japan found that antifibrotic treatment rates for patients with idiopathic pulmonary fibrosis (IPF) varied by medical facility, with higher treatment rates observed in referral hospitals than in general hospitals or clinics.

For some patients with myasthenia gravis (MG), activity and sleep were associated with reported fatigue and symptom levels.

Social isolation significantly increases the risk of mortality in patients with chronic obstructive pulmonary disease (COPD), highlighting the need for targeted interventions to address this issue.

Patients with locally advanced hepatocellular carcinoma (HCC) who achieve a complete response after locoregional therapy and immunotherapy may be able to follow a watch-and-wait protocol.

Despite challenges such as high attrition rates and adverse events, the trial highlights the value of established therapies in improving outcomes, particularly in regions with limited access to chimeric antigen receptor T cells and bispecific antibodies.

If approved, the combination regimen would be the first FDA-approved treatment for adults with recurrent KRAS mutant low-grade serous ovarian cancer (LGSOC).

This multiyear follow-up of more than 3300 patients with relapsed/refractory chronic lymphocytic leukemia (R/R CLL) who received ibrutinib—the longest study of its kind—confirms the agent’s efficacy as a salvage treatment but reveals new information about its impact in different subpopulations with varying clinical characteristics.

With approval, acoramaidis (Attruby; BridgeBio Pharma) becomes the first agent with a label specifying near-complete stabilization of transthyretin (TTR).

Adults with more unfavorable health-related social needs, such as unemployment or food insecurity, had a higher prevalence of low cardiovascular health, highlighting the importance of addressing social determinants to improve population health.

The increase in the global burden of interstitial lung disease (ILD) and pulmonary sarcoidosis had the greatest impact on countries with a high sociodemographic index.

For over 20 years, the Movember Foundation has used its mustache-growing campaign to raise awareness and funds for men's health issues, namely mental health and prostate and testicular cancers.

A presentation at the National Community Pharmacists Association Annual Meeting and Exposition focused on the latest on COVID-19 vaccination.

Initiating palliative care more than 3 months before death improves end-of-life outcomes for patients with ovarian cancer, highlighting the need for earlier interventions.

A new study aimed at exploring the relationship between obesity and multiple sclerosis severity using genetic data finds that higher BMI and other obesity measures were associated with increased disability progression in patients with MS.