MPN News from the 2017 American Society of Hematology Annual Meeting

December 13, 2017 

MPNRF will share relevant publications from ASH's Annual Meeting, which wrapped up Monday, starting with the information below. We will also be partnering with Patient Power to host a webinar on Thursday December 14th 1:00 PM Central (11:00 AM Pacific; 2:00 PM Eastern). Register here to watch the program live, or view later

What's old is new again
Claire Harrison presented an analysis of Wernicke’s Encephalopathy (WE) in MPN patients to see if the incidences of WE reported among those on the defunct JAKARTA trial testing Fedratinib are higher than the norm for MPN patients. Fedratinib was discontinued by the drug company Sanofi in 2013 after the FDA put the drug on clinical hold because of instances of WE in the study population. However, some patients had received benefit and there were doubts about whether WE was related to the drug or not. Dr. Harrison presented that WE seen in the JAKARTA trial participants was on par with what is to be expected on MPN patients generally. With the recent news about the investment in Fedratinib, this could suggest renewed hope for those patients who did well on the drug. 

In this category is also Ropeginterferon (Ropeg), a new formulation of pegylated interferon (commonly known as Pegasys and used off-label by MPN patients). Hans Gisslinger presented data from a randomized trial comparing Hydroxyurea to Ropeg, stating that patients on Ropeg experienced "high and durable hematologic response and symptom improvement", with the drug. They further describe the excellent safety and tolerability profile of Ropeg and mention the drug's ability to reduce the JAK2 allele burden, which makes it truly novel among other drugs that have been tried in MPNs.

Novel Therapies
Representing an entirely new angle to new therapies for MPN is LCL-161, an oral smac mimetic which is being tested now in high risk MF patients. Naveen Pemmaraju was interviewed by Patient Power at the beginning of their ASH wrap up, describing this drug and how smac mimetics work. Idasanutlin was shown to have activity in Polycythemia Vera ( and Sotatercept, both alone and in combination with Ruxolitinib shows anemia benefit for people with Myelofibrosis (

Diagnostic and Prognostic Update
Drs. Tefferi and Vannuchi presented an updated DIPPS  (Dynamic International Prognostic Scoring System) for Myelofibrosis. This new "Revised" DIPPS-plus” incorporates clinical, cytogenetic and molecular information, in order to provide a comprehensive prognostic tool for facilitating treatment decision-making in PMF. RDIPSS-plus was most effective in extracting patients with better than expected outcome, from specific DIPSS-plus risk categories. This is important, especially for doctors who don't see as many MF patients as the specialists in major cancer centers who nevertheless must make decisions about how best to manage their patients treatment plan. 



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