
As the feasibility of phase 1 clinical trials for prenatal spinal muscular atrophy (SMA) therapies is explored, patient and parent input on prenatal testing and possibly treatment is a valuable tool for guiding research discussions.


As the feasibility of phase 1 clinical trials for prenatal spinal muscular atrophy (SMA) therapies is explored, patient and parent input on prenatal testing and possibly treatment is a valuable tool for guiding research discussions.



A number of psychologic interventions were assessed to see if they could have an effect on patient confidence in biosimilars to treat psoriasis.

Policy observers and industry stakeholders debate the possible spillover effects of Medicare drug pricing reform in the employer-based insurance market; a California federal judge rules Walgreens could be held liable for not investigating suspicious orders of opioids in San Francisco; a blood test performed the day of a traumatic brain injury (TBI) can predict which patients may die and who may survive with a disability.

Ide-cel is a B-cell maturation antigen (BCMA)-directed CAR T-cell therapy, which uses the process of genetically modifying a patient’s T cells and infusing them back into the patient to attack the cancer.

Facilitating access to treatments for diffuse large B-cell lymphoma (DLBCL) remains a top issue.

Authors explained how multiple myeloma emerges from earlier clinical phases and the tumor’s microenvironment in the early course of the disease plays a critical role in the survival of myeloma cells.


Results looking at acceptance of minimal residual disease (MRD) status found that 60% of participants “would change at least one decision based on an MRD result,” and 54% would use both MRD status and disease risk to make decisions.

National Institutes of Health (NIH) researchers found a new form of macular dystrophy, which is a cause of central vision loss.

Data presented at ASCO show that after 6 years, patients with advanced Hodgkin lymphoma who were treated with a combination of brentuximab vedotin and chemotherapy had a sharply reduced risk of death and a reduced risk of a secondary cancer.

Findings from the phase 3 SHINE trial could make ibrutinib the standard treatment option for older patients with mantle cell lymphoma (MCL), who often cannot tolerate chemotherapy or transplant strategies, according to study authors.

Encouraging outcomes in recent studies have the authors of a recent review calling for more research on the effect of cannabinoids on sleep disorders.

Angel Qin, MD, clinical assistant professor of medicine at the University of Michigan, discusses the need for more clinical trials in small cell lung cancer and for more patients to qualify for trials.

Authors wrote that their analysis was the first to combine incidence and mortality of overall BTC, and its anatomic subtypes, into a single study

Ryan Haumschild PharmD, MS, MBA, director of Pharmacy Services at Emory Healthcare and Winship Cancer Institute, spoke with AJMC® about health care resource utilization and clinical decision support in the care of patients with small cell lung cancer (SCLC).

Cholangiocarcinoma (CCA), or bile duct cancer, is classified by its location, with intrahepatic CCA (iCCA) referring to those cancers that form in bile ducts within the liver.

Editor’s Note: After this issue of Evidence-Based Oncology™ went to press, Bristol Myers Squibb Foundation announced the $100 million, 5-year initiative will now be called the Robert A. Winn Diversity in Clinical Trials Award Program. The program will also receive a $14 million donation from Gilead Sciences, Inc., over the next 4 years.

Researchers presented results for ZB131, a monoclonal antibody that would target cancer-specific plectin (CSP); experiments with mice showed this may be present in more than 80% of the most common types of cholangiocarcinoma.

Traditional cardiovascular (CV) risk prediction models may not apply accurately to patients with HIV who may develop the cardiovascular disease younger than usual, said Jorge Plutzky, MD, director of the Vascular Disease Prevention Program and director of Preventive Cardiology at Brigham and Women’s Hospital and associate professor of medicine at Harvard Medical School.

The study by the Veneto Institute of Oncology (IOV) in Padua, Italy, examined 286 patients for genomic mutations in cholangiocarcinoma as well as survival status.

Teprotumumab has shown great efficacy in treating thyroid eye disease in both clinical trials and real-world settings, but more data are needed on adverse events, said Shoaib Ugradar, MD, UCLA Stein Eye Center Santa Monica.

A study from Japan identifies markers for a presurgical staging system in intrahepatic cholangiocarcinoma, with results on par with an existing postsurgical staging system.

As more data are gathered on teprotumumab for use in thyroid eye disease (TED), we’ll have a better understanding of which subgroups respond better or worse to the treatment, explained Robert G. Fante, MD, FACS, president of the American Society of Ophthalmic Plastic & Reconstructive Surgery and facial plastic surgeon and cosmetic surgeon, Fante Eye & Face Centre in Denver, Colorado.