Dr. Mascarenhas on the Impact of Ruxolitinib on the Myelofibrosis Treatment Paradigm

John O. Mascarenhas, associate professor of medicine, hematology, and medical oncology at the Icahn School of Medicine at Mount Sinai; director of the Adult Leukemia Program; and leader of Clinical Investigation within the Myeloproliferative Disorders Program at Mount Sinai; and a member of the Tisch Cancer Institute, discusses the impact of ruxolitinib (Jakafi) in the myelofibrosis treatment paradigm.

In 2011, ruxolitinib was approved by the FDA for myelofibrosis, and in 2014, it was approved for polycythemia vera. Prior to ruxolitinib, there weren’t any effective treatments available to address symptom burden and splenomegaly, which are 2 important aspects of the disease process, explains Mascarenhas.

With the commercial availability of ruxolitinib, quality of life and outcomes of patients with myelofibrosis changed significantly. Reports have shown that patients feel better, have less spleen-related complaints, and they are able to eat more. A reversal of cachexia has also been observed, and thus, patients are living longer than they would in the absence of the drug, says Mascarenhas.

Unfortunately, the great benefits of ruxolitinib come with a cost, adds Mascarenhas. For example, similar to some other cancer treatments, there is an element of myelosuppression. Investigators have also discovered that there is some degree of infectious risk, such as herpes zoster, urinary tract infections, or pneumonia. However, for most patients, the many benefits of the drug outweigh the risks, says Mascarenhas.

The median time of discontinuation with the agent is approximately 3 years, and 85% of patients discontinue treatment in 5 years. Ruxolitinib marks a huge advancement in the myelofibrosis treatment paradigm, according to Mescarenhas, but some unmet needs remain.