Ivy Altomare, MD, an oncologist and professor at Duke University, answers some questions about current and ongoing clinical trials for myelofibrosis.

It’s a very active time for myelofibrosis research. A few years ago, the JAKARTA and JAKARTA2 trials reported spleen shrinkage and symptom improvement with the selective JAK2 inhibitor fedratinib.

More recently, the PERSIST trial has finished and resulted in the approval of Pacritinib for myelofibrosis. The SIMPLIFY trial showed encouraging results for the JAK1/JAK2 inhibitor momelotinib.

Dozens of ongoing clinical trials are looking at new targeted drugs, either alone or in combination with medications already approved for myelofibrosis. We will hopefully have more tools in our toolbox to treat this disease as ongoing research is completed.

Absolutely. Doctors have known about the importance of JAK2 inhibition to treat myelofibrosis ever since the approval of Jakafi (ruxolitinib) for treatment of myelofibrosis in 2011.

The JAK2 inhibitor Inrebic (fedratinib) was approved last year for intermediate-2 or high-risk myelofibrosis. We can now use it either upfront or after progression on Jakafi.

Pacritinib has been approved for use in treating myelofibrosis and thrombocytopenia. Because it doesn’t suppress bone marrow, we can use it in patients with very low platelet counts. This is a common finding in myelofibrosis patients that can limit treatment options.

The easiest and best way to participate in a trial is to ask your doctor. They can screen dozens of trials to see which would be most appropriate for your type and stage of disease. If the trial is not available at your doctor’s office, they can coordinate a referral to a center offering the trial.

Clinicaltrials.gov is a database maintained by the National Institutes of Health that lists all available clinical trials. It’s open for anyone to review and is easily searchable. However, it can be confusing to people without a medical background.

Patient advocacy groups are also excellent resources for a number of topics including clinical trials. Check out the MPN Education Foundation or MPN Advocacy & Education International.

Myelofibrosis management has come a long way in the last 10 years. Genomic analyses have helped to fine-tune our risk-scoring system. It helps doctors determine who will benefit most from a bone marrow transplant.

The list of effective myelofibrosis drugs is growing. These medications help patients live longer with fewer symptoms and better quality of life.

We still have a long way to go. Hopefully current and future research will bring us even more approved therapies and better treatment combinations to further improve outcomes for people with myelofibrosis.

Every medical treatment has risks and benefits. Clinical trials are no exception.

Clinical trials are very important. They’re the only way doctors can discover new and improved cancer treatments. Patients in trials receive the best management of care.

Risks are different for each individual study. They could include specific side effects of the investigated drug, lack of benefit of the therapy, and assignment to placebo.

You must sign an informed consent to enroll in a clinical trial. This is a lengthy process with the research team. Your healthcare team will thoroughly review and explain all risks and benefits to you.

It’s still unclear how we can truly impact disease progression. Long-term follow-up of pooled data from the COMFORT trials suggests that treatment with Jakafi may double overall survival compared with the best available therapy at the time.

This finding is somewhat controversial. It’s not clear if the survival benefit is from delayed progression or other benefits, such as improved nutrition after spleen shrinkage.

There’s been a good deal of research into this topic but nothing that’s been really determined. Many pathways are being looked into and much research is ongoing in this area.

The best chance for long-term disease control is a bone marrow transplant, also known as a stem cell transplant. It does seem to cure some patients. It’s difficult to predict with certainty.

Transplant is a high-risk, high-reward option. It’s only appropriate for certain patients who can withstand the rigors of the process. Your doctor can advise if a bone marrow transplant is an appropriate option for you and coordinate referral to an experienced transplant team for a consultation.

Ivy Altomare, MD, is an associate professor of medicine at Duke University and the assistant medical director of the Duke Cancer Network. She is an award-winning educator with a clinical focus on increasing awareness of and access to oncology and hematology clinical trials in rural communities.