Rose McNulty

Despite excellent prognoses in low-risk patients with essential thrombocytopenia (ET) and polycythemia vera (PV), knowledge gaps remain and novel, more tolerable therapies require reevaluation of treatment algorithms.

A review of current research and drug development in motor neuron disorders such as amyotrophic lateral sclerosis and spinal muscular atrophy shows progress in the identification of treatment pathways and therapies.

In patients with BRAF-mutated melanoma, achieving a complete response and circulating tumor DNA negativity are promising prognostic markers.

A review of instruments used to assess upper-limb function in patients with neuromuscular diseases found that more tools are needed to address specific diseases.

A recent review of viral gene therapy in pediatric neurological diseases included positive outcomes in patients with spinal muscular atrophy (SMA).

Combination CDK4/6 and MEK inhibitor treatment reduced tumor growth in mouse models of melanoma with acquired resistance to BRAF and MEK inhibition.

The largest known prospective study of patients treated with trametinib plus dabrafenib for unresectable advanced melanoma with BRAF-V600 mutation confirmed the combination’s clinical activity.

Recent research identified spinal muscular atrophy (SMA)–specific growth percentiles, aiming to improve clinical management of the nutritional aspects of the disease.

A review of current data identified areas of progress in Bruton tyrosine kinase (BTK) inhibition for patients with chronic lymphocytic leukemia (CLL).

A pair of surveys found that there are knowledge and practice gaps preventing earlier diagnoses of spinal muscular atrophy (SMA) in infants and toddlers.

A study of patients given first-line metastatic melanoma treatment showed that utilization rates were highest in patients using ipilimumab-containing therapies.

An epidemiological investigation of spinal muscular atrophy (SMA) in Japan provided clarity on the genetic condition’s prevalence and incidence in the country and highlighted the importance of newborn screening for SMA

AJMC®TV interviews let you catch up on what’s new and important about changes in healthcare, with insights from key decision makers—from the clinician, to the health plan leader, to the regulator. When every minute in your day matters, AJMC®TV interviews keep you informed. Access the video clips at ajmc.com/interviews. These interviews have been edited lightly for clarity.

Kathryn Gold, MD, medical oncologist and associate professor of medicine at UC San Diego, who is a member of the NCCN guidelines panel for small-cell lung cancer, gives insight into the guidelines and the importance of frequent updates.

At the 2021 Cholangiocarcinoma Foundation Annual Conference, experts discussed clinical trials in cholangiocarcinoma and the ways in which clinical trials overall can be made more available to patients.

At the 2021 Cholangiocarcinoma Foundation Annual Conference, experts discussed guidelines and real-world data for the management of urgent symptoms in patients with cholangiocarcinoma who have biliary stents or drains.

Discussions at the start of the 2021 Annual Cholangiocarcinoma Foundation Conference centered on challenges and advances made during the COVID-19 pandemic, and the potential of liver transplantation in cholangiocarcinoma.

The approval marks the first B-cell maturation antigen-directed genetically modified autologous chimeric antigen receptor T-cell therapy for patients with relapsed or refractory multiple myeloma.

The sensitivity and specificity of liquid biopsy might be improved through selective venous sampling according to an article in press in The Journal of Vascular and Interventional Radiology.

The study was originally undertaken to convince MD Anderson’s providers that publishing survey results online would help their reputations.

At the National Comprehensive Cancer Network Annual Conference, Arlene O. Siefker-Radtke, MD, discussed immune checkpoint inhibitors and their potential in urothelial bladder cancer.

Al B. Benson, MD, FACP, FASCO, professor of medicine and director for Cooperative Groups at the Robert H. Lurie Comprehensive Cancer Center, discusses the potential for neoadjuvant intervention in hepatobiliary cancers.

A keynote session at the NCCN Virtual Annual Conference brought a panel of patient advocates and experts together for a timely and necessary discussion about strategies to reduce racial disparities in oncology.

During a session at the National Comprehensive Cancer Network (NCCN) annual virtual conference, the payer perspective and physician experiences with PRO systems in both the community and academic medical center settings took center stage.

A recently published 20-year study suggests that results of the Oncotype DX Genomic Prostate Score are associated with the risk of long-term outcomes in patients with localized prostate cancer.

Amye J. Tevaarwerk, MD, director of the Survivorship Program at the University of Wisconsin Carbone Cancer Center, discussed the impact that cancer diagnosis and treatment can have on survivors when it comes to work.

A new cost-effectiveness analysis suggests quality-adjusted life year gain and cost reduction for patients treated with axicabtagene ciloleucel versus tisagenlecleucel.

The FDA will reassess 6 indications for immune checkpoint inhibitors that were granted accelerated approval but failed to prove clinical benefit in confirmatory trials.

An experimental safety switch incorporated into chimeric antigen receptor (CAR) T-cell therapy reduced the severity of the treatment’s side effects in an early-phase trial.