MDS: Treatments

It is important that patients with MDS be treated by a specialist with experience treating this condition, given its rarity and complexity.

Treatment for MDS depends upon whether a patient has symptoms or not, and whether the disease is progressing. For those with a lower risk of progress, our goal is reduce symptoms, reduce the number of infections, and increase the patient’s quality of life.

For those with a higher risk of progression, our immediate goal is to increase life expectancy and find novels ways to halt the disease, or to treat it, if and when it develops into AML.

Using the most sophisticated techniques, physicians are now able to track molecular and genetic changes in the bone marrow over time.

The Columbia MDS Program is led by Dr. Azra Raza, the world’s foremost expert on this disease, who has studied this condition for over 30 years. Columbia offers the most comprehensive, multidisciplinary approach to MDS and the highest level of patient care. It has also been designated a national Center of Excellence by the Myelodysplastic Syndromes Foundation and offers the following approaches to this condition:

Supportive Care

Low-risk patients with MDS are closely monitored for symptoms, with periodic checks of their blood counts. Supportive care may include any or all of the following: Transfusion if the patient develops low red blood cells or platelets.  Antibiotics to fight infections related to a drop in white cell counts.  An injection of growth factors to help the bone marrow manufacture more healthy white and red blood cells. 

Drug Therapy

Currently three drugs have been approved by the FDA for the treatment of MDS.

  • Azacitibine (Vidaza) This form of chemotherapy, known as a “demethylating” agent, is believed to improve the bone marrow’s ability to make more healthy blood cells.  It can also lower the number of abnormal cells produced in the marrow.
  • Decitadine (Dacogen) This is another chemotherapy that works in a similar way as azacitidine.
  • Lenalidomide (Revlimid) This drug modifies the immune system and is used to treat a form of MDS associated with a genetic mutation—the loss of part of chromosome 5, known as del(5q). Lenolidamide is a similar to the drug thalidomide but has fewer side effects.  

Allogeneic Stem Cell Transplantaion

Some patients with MDS may benefit from a stem cell transplant. In allogeneic (al-o-gen-A-ic) transplantation, stem cells harvested from a compatible donor ("allo" means other).  

Genetic Sequencing

This is a new therapeutic option available only at Columbia. A highly sophisiticated genetic sequencing test, developed by Dr. Azra Raza, allows physicians to track the way MDS is evolving in the individual patient and to provide therapies that address these changes over time.  This advance means that treatments can be planned, in sequence, to keep pace with the disease, and changes made in the regimen before the patient develops drug resistance.

Investigational Therapies

The Columbia MDS Program offers patients the opportunity to participate in studies that shed light on their particular form of MDS.  Our ongoing program of clinical trials allows us to administer highly individualized therapies and to offer experimental treatments to high-risk patients.