Clinical

A 72-year-old patient with Burkitt lymphoma was successfully treated with nivolumab after standard-of-care chemotherapy failed.

In a longitudinal cohort study, researchers identified 17 seemingly novel variants of the PDE6A retinitis pigmentosa gene, suggesting it may be amenable to gene therapy.

Although renal replacement therapy is a clinically meaningful end point, trials must include numerous patients and follow them for long periods of time, said Kunihiro Matsushita, MD, an associate professor in the Department of Epidemiology and Division of Cardiology at Johns Hopkins University.

This story was updated in February 2022 to show that this original story, published in October 2020, is out of date, and adds new information.

Initial preliminary efficacy and safety data are expected sometime next year, stated Aditya Bardia, MBBS, MPH, breast medical oncologist, Massachusetts General Hospital, Harvard Medical School.

Early treatment of infants with spinal muscular atrophy (SMA) can drastically improve motor function for patients, explained Jill Jarecki, PhD, chief scientific officer at Cure SMA.

Andre Goy, MD, chairman, director, and chief of the Division of Lymphoma at John Theurer Cancer Center in Hackensack, NJ, discussed the potential benefits of using CAR T-cell therapies as a second-line treatment.

Two abstracts presented at a recent multiple sclerosis meeting examined depression in pregnancy and pregnancy outcomes in ozanimod trials.

Their study outlines several treatments being investigated for the treatment of patients with myelofibrosis and other myeloproliferative neoplasms, generally after they have become resistant or intolerant to Janus kinase inhibition.

Finerenone slowed chronic kidney disease (CKD) progression by 18% and cut cardiovascular (CV) events by 14%, according to phase 3 trial results.

Despite the benefits of switching to a different mode of action in the second line, almost half of patients with rheumatoid arthritis were cycled to a second tumor necrosis factor inhibitor.

In her presentation, Aimee Tharaldson, PhD, senior clinical pharmacist of emerging therapeutics at Express Scripts, discussed the upcoming pipeline of specialty drug approvals and their potential impact on the market.

A new study is underscoring the importance of diagnostic testing for patients with spinal muscular atrophy (SMA) to ensure early diagnosis of the disease.

Heart failure with reduced ejection fraction (HFrEF) is when the heart’s left ventricle can only pump out 40% or less of the blood it contains, resulting in less oxygen-rich blood being disseminated to the body than it actually needs.

Vadadustat is an investigational drug belonging to a class known as hypoxia-inducible prolyl hydroxylase inhibitors (HIF-PHIs) for the treatment of anemia in chronic kidney disease.

The investigational drug finerenone met its primary endpoint in FIDELIO-DKD of slowing progression of chronic kidney disease (CKD) in patients with type 2 diabetes and CKD, said Rajiv Agarwal, MD, MS, FASN, a professor of medicine at Indiana University School of Medicine and a staff physician at the Veterans Affairs Medical Center in Indianapolis, Indiana.

Chimeric antigen receptor (CAR) T-cell therapy does not always lead to a durable response, and we are trying to figure out why, noted Michael R. Green, PhD, University of Texas MD Anderson Cancer Center.

Experts presenting the American Society of Nephrology’s Kidney Week session entitled “From Kids to Adults: Chronic Kidney Disease-Mineral and Bone Disorder (CKD-MBD) Across the Life Course,” outlined the detrimental effects of childhood CKD and other kidney diseases on long-term health.

It is still unclear why patients who should respond to anti–interleukin-5 therapies do not, noted Anne Reihman, MD, Division of Pulmonary Sciences and Critical Care Medicine, University of Colorado.

In a session focused on sodium-glucose cotransporter 2 (SGLT2) inhibitors presented at the American Society of Nephrology’s (ASN) Kidney Week, experts highlighted the benefits of using this drug class in patients with kidney disease and outlined future steps for integration of care.

Recent guidelines have renewed the focus on treating severe asthma. During 2 panels at CHEST 2020, experts discussed key updates, limitations of the guidelines, and more.

Solriamfetol was found to be efficacious and safe in the treatment of excessive daytime sleepiness in patients with narcolepsy and obstructive sleep apnea, according to study findings.

Shared decision-making is very important when it comes to choosing biologics for patients, emphasized Nicola Hanania, MD, MS, pulmonary critical care physician and director, Airways Clinical Research Center, Baylor College of Medicine, Houston, Texas.

Investigators may have found a minimally invasive way to predict which patients with multiple myeloma are about to experience a relapse.

Challenges remain, in light of new guidelines, when making treatment decisions for patients with severe asthma, noted Megan Althoff, MD, PhD, second year fellow, University of Colorado, Division of Pulmonary Sciences and Critical Care Medicine.

T-cell prolymphocytic leukemia is rare and shares many features with other conditions. A new review article helps explain some of the tactics physicians can use to correctly identify it.

A recent review in Current Diabetes Reports delves into the utility of sodium-glucose co-transporter-2 (SGLT2) inhibitors for patients with heart failure currently on other medications.

Chronic cough is frustrating for patients and their families, especially during the pandemic, explained Krishna M. Sundar, MD, FCCP, clinical professor, Pulmonary Division, Department of Medicine, and medical director, Sleep-Wake Center, University of Utah.

Anti–vascular endothelial growth factor (VEGF) treatment was associated with preserved useful visual acuity in almost 20% of patients with neovascular age-related macular degeneration (nAMD).

We are still working to figure out why low levels of vitamin D are associated with worse outcomes in critical illness, noted Todd W. Rice, MD, FCCP, associate professor of medicine at Vanderbilt University Medical Center in Nashville, Tennessee.