Clinical

New data show the stark contrast between untreated infants with type 1 spinal muscular atrophy (SMA) and those who received risdiplam.

In this case report, a 50-year-old female patient, with a family history of lung cancer, received a diagnosis of multiple myeloma (MM) following presentation with an apical lung mass.

Still, investigators say there are gaps in spinal muscular atrophy (SMA) screening protocol that need to be filled.

New real-world evidence on non–small cell lung cancer (NSCLC) aligns with the results of a 2017 clinical trial.

Analysis of more than 16 million visits for hypertension care suggests that a large fraction of face-to-face care is low value and could be provided differently or potentially eliminated.

Panelists discuss treatment for heart failure in the presence of comorbid disease impacting payer considerations.

Ryan Haumschild, PharmD, MS, MBA, leads a conversation regarding value statements and their alignment with cost-effective care.

Dr Sonia T. Oskouei focuses on automatic substitution in both the pharmacy setting and in patients’ health plans.

Mariam Alboustani, PharmD, discusses the one published example of a switching study and the implications of interchangeability for pharmacists, as well as payers.

Both the ZUMA-7 trial for axicabtagene ciloleucel—approved for second-line treatment earlier this year—and the TRANSFORM trial for lisocabtagene maraleucel showed significant improvement over standard of care for large B cell lymphoma (LBCL), while the BELINDA trial for tisagenlecleucel did not.

The recommendations follow the World Health Organization’s recent update to its classifications of central nervous system (CNS) tumors to now include a structure for pediatric patients with CNS tumors.

The findings, coming from the phase 3 SEQUOIA study, found that the next-generation, selective Bruton tyrosine kinase inhibitor had superior efficacy versus chemoimmunotherapy and demonstrated an acceptable safety profile among 600 older patients with comorbidities.

Compared with sensor-augmented pump therapy, closed-loop insulin delivery increased glucose time in range and reduced hypoglycemia episodes but detracted from sleep quality.

Previous studies have produced heterogeneous results on next-generation flow (NGF)-based minimal residual disease (MRD) detection, despite improvements to the method’s reproducibility and sensitivity, prompting the present authors to investigate its clinical utility.

The KidneyIntelX test would affect primary care physician (PCP) decision-making, and PCPs would use the results of KidneyIntelX more than albuminuria and estimated glomerular filtration rate when making decisions about diabetic kidney disease management.

The combination is approved to treat hepatocellular carcinoma (HCC), but this case report suggests it can also work for combined HCC and cholangiocarcinoma (CHC).

Smaller lumen sizes in women resulted in worse respiratory morbidity and mortality in chronic obstructive pulmonary disease (COPD), according to a recent study.

Despite the known potential benefits of newborn screening, the exact long-term clinical benefits and cost-effectiveness in large cohorts remain uncertain due to a lack of large-scale longitudinal research.

Current standard modalities for detecting and quantifying monoclonal immunoglobin in patients with cold agglutinin disease lack adequate sensitivity. Heavy chain/light chain assay may be more effective.

Kanwal Raghav, MD, MBBS, discusses appropriate candidates for HER2 testing and current utilization strategies for biomarker testing in mCRC.

Testing prioritization and frequency are highlighted in Dr Ciombor’s final insights for this HER2 biomarker focus.

Investigators say classification based on magnetic resonance imaging can predict a patient’s risk of recurrence for intrahepatic cholangiocarcinoma (iCCA).

After years of dependence on systemic corticosteroids, ruxolitinib treatment was effective for an adolescent patient with graft-versus-host disease (GVHD) who was initially thought to have eosinophilic gastroenteritis.

This retrospective study and prospective follow-up provide insight into predictors of thrombotic episodes in patients with autoimmune hemolytic anemia (AIHA) and indicators that anticoagulant prophylaxis may be beneficial to certain patients.

Drs Kaddis and Oskouei define interchangeable biosimilars, their FDA requirements, and switching studies.