A Phase I Study of Ruxolitinib and Decitabine in Patients with Myeloproliferative Neoplasms in Accelerated or Blast Phase

Full Title

Combination Therapy of Ruxolitinib and Decitabine in patients with Myeloproliferative Neoplasms in Accelerated and Blast Phase Disease


Myeloproliferative neoplasms (MPN) are a group of diseases of the bone marrow where too many cells are made. When MPN has entered an “accelerated” or “blast” phase, it means that between 10 and 19 percent of cells in the blood or bone marrow are immature cells called blasts. These cells are abnormal and don’t work well like normal cells. MPN in accelerated or blast phase is challenging to treat. MPN can also evolve into a disease called acute myeloid leukemia (AML) when the number of blast cells in the blood reaches 20 percent or greater.

The purpose of this study is to find the highest dose of a drug called ruxolitinib that can be give safely with another drug called decitabine in patients with MPN in accelerated or blast phase, or MPN which has progressed to AML. Ruxolitinib is already approved for treating a bone marrow disease called myelofibrosis. It works by inhibiting a protein called Jak that is abnormal in MPN. Ruxolitinib has been shown in prior studies to help some people with MPN achieve remission. Decitabine is a drug commonly used to treat acute leukemia.

Ruxolitinib is taken orally (by mouth), while decitabine is given intravenously (by vein).


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have MPN in accelerated or blast phase, including a diagnosis of acute myeloid leukemia (following a previous diagnosis of MPN), essential thrombocythemia, polycythemia vera, or primary myelofibrosis.
  • At least 4 weeks must pass between completion of prior therapies and entry into the study.
  • Patients must be able to walk and do routine activities for more than half of their normal waking hours.
  • This study is for patients age 18 and older.

For more information about this study and to inquire about eligibility, please contact Dr. Raajit Rampal at 212-639-2194.