Dr Mesa and Dr Mikhael Discuss Latest MPN Research 2017
A Solid Summary on the Latest MPN Research in 2017
Drs. Joseph R. Mikhael and Ruben A. Mesa analyze and discuss pertinent clinical studies and late breaking updates pertaining to the care of patients with chronic myelogenous leukemia (CML) and myeloproliferative neoplasms (MPN) in this Best of the Day discussion from the annual hematology meeting held in San Diego.
by David Wallace
Dr. Mesa begins discussion of MPN’s at 6 minutes, 23 seconds into the video. Good news to hear discussion of Pegasys as “upfront therapy for PV and ET” while looking at the long term impact at 5 years or 10 years as important benchmarks. Molecular responses (immunologic remission) seen with Pegasys and Ropeginterferon (taken every 2 weeks)….no real surprise there. Perhaps not an indefinite therapy, but one that may lead to remission during a multi-year time frame. I had an in depth interview with Dr Kiladjian at ASH 2016 regarding patient outcomes after Interferon therapy discontinuation.
Dr. Mesa went on to say “Ruxolitinib continues to remain our only available therapy, 5 year data looks good regarding survival and deep response on bone marrow fibrosis.” There were discussions of various combination studies, as well as updates on Pacritinib and Momelitinib. Overall, an engaging review of current MPN therapies with a vision of things to come.
A Gaping Hole in U.S. Research on MPN Combo Therapies (RUXOPeg)
I find it rather disappointing there are no formal U.S. studies underway on the Pegasys and Ruxolitinib combo. A search of clinicaltrials.gov does not reveal any formal clinical trials or research studies being conducted in the U.S. I know first hand, this combo therapy has produced exciting results for PV and MF patients. On the other hand, I’m thankful our European and Scandinavian counterparts are studying the impact of Ruxolitinib and Peg-interferon Alpha-2A in patients with Primary Myelofibrosis (French Innovative Leukemia Organisation, 2016) and Polycythemia Vera (the Danish study, 2015).
Click here for the recently announced clinical trial.
At ASH 2015, I attended the presentation “Safety and Efficacy of Combination Therapy of Interferon-Alpha2 + JAK1-2 Inhibitor in the Philadelphia Negative Chronic Myeloproliferative Neoplasms. Preliminary results from the Danish Combo Trial.” The early results from that presentation were very encouraging. Click here for the presentation slides, start with the first one and click the right arrow to progress through the 8 slides.
I expect updated results on that study later this year. One downside to this combo therapy is it’s VERY costly, in the range of $15,000 – $20,000/month. Most insurance plans will cover Jakafi (it is FDA approved for MF and PV), although there may be sizable copay, there a numerous foundations that will assist with the copay, click here for a list. While Pegasys is off-label, many leading hematologists are familiar with its efficacy in treating MPNs, so a prescription should not be too difficult to come by (a letter of need may be required) for your insurance coverage approval.
Now the tricky part – Treatment with this combo therapy should be closely monitored by an MPN specialist (one who is progressive, pioneering and open minded) and only after a patient has failed to improve using Pegasys (which is a slow acting drug) or Jakafi (Ruxolitinib) alone. Both of these drugs are being used to treat MF and PV. 15 out of 20 PV patients saw Complete clinical hematologic response in the Danish study.
In conclusion, I sincerely hope leading MPN specialists in the United States get approval to conduct clinical trials on this innovative, yet overlooked, combo or will pioneer this therapy with patients whose MF or PV are not responding to traditional treatment.
