Azacytadine Trial

Drug Name:
Azacytadine

Drug Info:
Azacytadine is a subcutaneously administered drug, from the class of drugs known as hypomethylating agents.. The drug is made by Pharmion.

Why might this drug help?
Azacytadine is an approved drug for the bone marrow disease myelodysplastic syndrome (MDS). In patients with MDS (which share many features with patients with MF) the drug has been shown to improve low blood counts and delay the change of the disease to acute leukemia. We are hopeful based on the biology, and history of MF patients responding to MDS drugs that we will see a similar benefit in MF patients.

Who is eligible (partial list)?

  • Adult (>18 years) Patients with myelofibrosis (primary as well as post PV/ET MF) (biopsy confirmed)
  • Patients need to be symptomatic from their disease
    • Anemia (hemoglobin <10g/dL or needing transfusions) or
    • Enlarged spleen causing symptoms
  • Blood counts (because this drug might lower blood counts patients need a minimum of:
    • Neutrophils > 1 x 109/L
    • Platelets > 50 x 109/L
    • Nearly normal kidney and liver functions (please check with contact person below)
  • Patients need to be off all other MPD treatment (hydrea, steroids, aranesp, erythropoietin, anagrelide, etc) for at least 14 days before enrolling.
  • No prior thalidomide or lenalidomide
  • No severe other current illnesses, significant neuropathy, or cancers.
  • Please check with the contact person below for full details.

How do I take this drug?
This drug is given as a shot under the skin for 5 days in a row once a month

What is involved with being on the trial?
You will need go to Mayo Clinic for an exam, blood tests, and a baseline bone marrow biopsy. You will need to seen monthly. The first dose each month of the drug is given at Mayo, the subsequent 4 doses may be given byu your local hematologist-oncologist if they agree. How long you remain on the treatment depends on how well you respond, or if you have any side effects but will be usually at least 6 months. The drug is provided free by the company, but the rest of the expenses are processed in the usual way.

Where is it being offered / Contact Info?
Mayo Clinic Rochester
Dr. Ruben Mesa
mesa.ruben@mayo.edu

For Questions Contact: Ian Sweet | Webmaster: Digital Attic, LLC.
www.MPDInfo.org is a resource of the CMPD Education Foundation.