Abstract
Treatment options for patients with lower risk non-del(5q) myelodysplastic syndromes (MDS) who fail erythroid stimulating agents are restricted to one of the hypomethylating drugs with an expected response rate of ~50%. Ezatiostat HCl, an agent with the potential for producing multi-lineage responses in this population is currently in clinical investigation phase. This case report describes a 77 year old male who received less than two cycles of therapy with ezatiostat HCl which had to be aborted due to intolerable side effects, but which produced a sustained normalization of all three blood counts. This trilineage response has now lasted for more than a year. Interestingly, the patient began with a del(5q) abnormality and responded briefly to lenalidomide. Upon relapse of the anemia, a bone marrow showed the disappearance of the del(5q) but the appearance of a new clonal abnormality t(2;3). Given that the patient had a complete cytogenetic response to a truncated exposure to lenalidomide followed by a trilineage response to an even briefer course of ezatiostat HCl suggests a potential role for ezatiostat HCl in del(5q) patients who relapse following lenalidomide.
Highlights
Myelodysplastic syndromes (MDS) are a group of bone marrow stem cell disorders which generally affect the elderly and present with the paradox of a variable cytopenia with a cellular marrow
Allogeneic stem cell transplant offers the only potential for cure to these patients; because of the advanced age, this is an option for only a select few
We report this unique case, as the patient received therapy with oral ezatiostat HCl tablets for less than 2 cycles, but has shown an impressive hematologic response that has lasted for over a year
Summary
Myelodysplastic syndromes (MDS) are a group of bone marrow stem cell disorders which generally affect the elderly (median age of 70 years) and present with the paradox of a variable cytopenia with a cellular marrow. * Correspondence: araza@aptiumoncology.com 2 St. Vincent's Comprehensive Cancer Center, 325 West 15th Street, New York, New York 10011 USA Full list of author information is available at the end of the article del(5q) and lower risk disease while the two hypomethylating agents (azacytidine and decitabine) are approved for all categories. With the exception of del(5q) patients, the response rate is approximately 50%, highlighting the need for clinical trials of new agents.
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