July 2016, Vol 7, No 6

Hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome, is a rare and life-threatening liver condition that is characterized by rapid weight gain, ascites, painful hepatomegaly, and jaundice. It is often observed in patients after allogeneic or autologous hematopoietic stem-cell transplantation (HSCT), and has also been reported during the treatment of Wilms tumor, rhabdomyosarcoma associated with actinomycin D, and acute lymphoblastic leukemia.

New recommendations issued by the US Preventive Services Task Force (USPSTF) focus on increasing colorectal cancer screening, which, according to the task force, is “a substantially underused preventive health strategy.” Previous USPSTF guidelines recommended specific screening approaches, including colonoscopy, fecal occult blood testing, or sigmoidoscopy, for adults aged 50 through 75 years.

The multitarget stool DNA test, a noninvasive screening tool for colorectal cancer (CRC), demonstrated potential for identifying cancer and advanced adenomas in community-based individuals who had previously not followed national screening recommendations, reported Mark Prince, MD, MBA, Director of Gastroenterology, USMD Health System, Arlington, TX, at the 2016 American Association for Cancer Research annual meeting.

The multitargeted RAF inhibitor BGB-283 demonstrated activity in several types of advanced solid tumors associated with different mutations in the RAF family of genes, results of a preliminary clinical trial showed.

Holding what was essentially a one-person debate, Michael B. Atkins, MD, Deputy Director of the Georgetown-Lombardi Comprehensive Cancer Center, Washington, DC, described strong cases for immunotherapy or molecularly targeted therapy as initial treatment for patients with advanced BRAF mutation–positive melanoma.

When combined with other clinically relevant parameters, a novel protein biomarker called IsoPSA can improve selection of patients with prostate cancer for biopsy. IsoPSA holds promise for improved diagnostic accuracy, said Eric A. Klein, MD, Chairman, Glickman Urological and Kidney Institute, Cleveland Clinic, OH, at the 2016 American Urological Association annual meeting.

A new article by a New York surgical team confirms that there is a survival benefit from an emerging therapy for difficult-to-treat soft-tissue tumors, but that it is not without a steep price for some patients.

On April 25, 2016, an FDA advisory committee voted not to recommend the approval of eteplirsen, an experimental drug that targets one of many genetic mutations causing Duchenne muscular dystrophy (DMD), a deadly degenerative disease that has no cure. After agreeing to study the real-world effects of eteplir­sen, the FDA advisory committee rejected findings that patients who have been taking eteplirsen since 2011 were still able to walk because the clinical data did not meet the FDA requirements for a well-controlled study.

In the United States, the standard of care for locally advanced bladder cancer after radical cystectomy is to “consider” adjuvant chemotherapy and ad­juvant radiation. Results of a 3-arm randomized clinical trial showed that adjuvant radiation therapy alone or combined with chemotherapy (ie, chemoradiotherapy) did not significantly improve disease-free survival compared with adjuvant chemotherapy alone. However, the findings hint at benefits for chemoradiotherapy that should be studied further. Brian Baumann, MD, a radiation oncology resident at the University of Pennsylvania, Philadelphia, presented the findings at the 2016 Genitourinary Cancers Symposium.