Dr. Garcia on Future Expectations of Trials and Therapies in Myelofibrosis

Jacqueline S. Garcia, MD, physician, Dana-Farber Cancer Institute, assistant professor of Medicine, Harvard Medical School, discusses the future expectations of trials and therapies to improve outcomes in myelofibrosis.

Within the treatment landscape of myelofibrosis, many clinical trials and therapies primarily focused on improving and managing the symptoms of the disease, Garcia says. Because myelofibrosis is a chronic condition, patients suffer quite a bit from their symptoms, which can include splenomegaly, disease burden, fever, fatigue, body aches, pain, depression, and more. However, there remains an unmet need to develop therapies that can modify the disease, rather than only improve symptoms.

For example, treatment with ruxolitinib (Jakafi) can reduce the symptoms; however, the underlying disease remains, and damage is still being done to the bone marrow, Garcia explains. As a result, bone marrow fibrosis reduction is not observed on a common basis, and current treatments for symptom management have not translated into a survival benefit.

As new drugs and therapies are developed and tested, particularly in combination therapy, the hope is to see increased evidence of disease modification. Garcia and colleagues published a review of clinical trials in myelofibrosis in Cancer that detailed asks and expectations that could help investigators research regimens that can improve outcomes for patients with myelofibrosis now that there are more active drugs that could benefit this patient population.

If achieving transfusion independence, bone marrow fibrosis reduction, and mutant allele reduction can correlate with a survival benefit, these would serve as meaningful end points for clinical trials, Garcia notes. The review also concluded that clinical trials tend to take place in the heavily pretreated setting, and the greatest impact of disease-modifying treatment could be observed when treatment is initiated early.

It is hoped that newer drugs will expand the treatment portfolio and positively impact patients over time, Garcia concludes.