Current Clinical Trials

With the exception of the use of lenalidomide for low risk patients with abnormalities of chromosome 5, there are no clinical trials informing the appropriate selection of current therapies for patients with specific subtypes of myelodysplastic syndrome. Patients who have ceased to respond or did not respond to one therapy are frequently offered another from the therapies described in the previous sections. There are currently no data evaluating the success of switching from one azacytosine analogue to the other in the case of nonresponse. Patients who have responded, as in the CALGB-8421 ¹, CALGB-8921 ², and CALGB-9221 ³ trials, to an azacytosine nucleoside and relapse off-therapy may respond to the reinstitution of the nucleoside.[1] Relapsed patients should be considered for enrollment in clinical trials. In patients previously treated with growth factors, there are studies that have shown responses to non–growth factor approaches.

Standard treatment options:

• Myeloablative allogeneic stem cell transplantation.
• Erythropoeitic growth factors, in patients with endogenous erythropoietin levels less than 500 u/mL.
• 5-azacitidine or decitabine.
• Lenalidomide for patients with deletions of chromosome 5q31.[2,3]
• Antithymocyte globulin.
• Supportive care.

Treatment options under clinical evaluation:

• Nonmyeloablative stem cell transplantation.
• Farnesyl transferase inhibitors (tipifarnib and lonafarnib).
• Combination therapies.
• Thrombopoietic agents.

Check for U.S. clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with previously treated myelodysplastic syndromes ⁴. The list of clinical trials can be further narrowed by location, drug, intervention, and other criteria.

General information about clinical trials is also available from the NCI Web site ⁵.

References

1. Silverman LR, McKenzie DR, Peterson BL, et al.: Further analysis of trials with azacitidine in patients with myelodysplastic syndrome: studies 8421, 8921, and 9221 by the Cancer and Leukemia Group B. J Clin Oncol 24 (24): 3895-903, 2006.  [PUBMED Abstract]
   
   
2. List A, Kurtin S, Roe DJ, et al.: Efficacy of lenalidomide in myelodysplastic syndromes. N Engl J Med 352 (6): 549-57, 2005.  [PUBMED Abstract]
   
   
3. List A, Dewald G, Bennett J, et al.: Lenalidomide in the myelodysplastic syndrome with chromosome 5q deletion. N Engl J Med 355 (14): 1456-65, 2006.  [PUBMED Abstract]