Treatments for myelodysplasia

Last Modified: August 1, 2004


Dear OncoLink "Ask The Experts,"
I am struggling with secondary myleodysplasia, resulting from being given CHOP (cyclophosphamide, doxorubicin, vincristine, and prednisone ), for non- Hodgkin's lymphoma. Doctors say that my prognosis is poor because secondary MDS is fairly intractable to treatment. I do have a sibling donor match for a mini stem cell transplant. Any thoughts on the matter would be appreciated. 


Selina M. Luger, MD, Director of the Leukemia Program and Assistant Professor of Medicine at the University of Pennsylvania, responds:

You are describing the dilemma that exists with myelodysplasia syndrome (MDS). It is a disease that results in cytopenias (low blood counts) and has a variable risk of progression to leukemia. Over the last decade there has been a scoring system developed in MDS, based on the number of low blood counts, the number of immature cells in the bone marrow, and the cytogenetics of the bone marrow cells. Patients with a low score can live for years with their disease, without the risk of developing leukemia and patients with a high score are at risk for life threatening complications and progression to acute leukemia.

Although allogeneic bone marrow transplantation remains the only known "curative" treatment for MDS, its place in the treatment of MDS is unclear. The patients who do best after a transplant (younger patient with low risk disease) are also the patients who do best without a transplant. Also, the older age group of patients with MDS makes most patients ineligible for a standard allogeneic bone marrow transplant.

Recently the use of mini-transplants has opened up the range of patients eligible for transplant. It appears that these procedures are better tolerated and result in responses in a significant number of patients. But the duration of the response (i.e. how long patients are in remission) is unknown, since the procedure is relatively new. Also, the optimal regimen for mini-transplant is not clearly defined.

At this point, given that you have the fortune of having an HLA identical sibling, it is reasonable to consider a mini-transplant in your situation, if your physician feels it is appropriate. It would be great if you could go on a clinical trial for treatment.

As you noted, there are not many other treatment options available for patients with MDS, other than supportive care with transfusions and growth factors, if they are working.