A multicenter Phase II trial, MDS-002, showed that Revlimid ( Lenalidomide ) is active in a larger group of patients with low and intermediate-1 risk myelodysplastic syndromes ( MDS ), who lack a distinct cytogenetic abnormality.
"This data is consistent with the results from our prior trial MDS-001, and combined with the findings of the MDS-003 trial, it demonstrates the potential of Revlimid for treating a broad range of patients with MDS," said Alan List, the study's lead investigator. " I am delighted that the primary endpoint of this trial was achieved. In many patients, the response was accompanied by an unprecedented normalization of the bone marrow both histologically and cytogenetically."
The analysis was based on clinical data available as of March 31, 2005.
This trial will continue and be updated on an ongoing basis.
List, at H. Lee Moffitt Cancer Center, Tampa, reported that 215 patients entered the study, of whom, 166 were documented to have low to intermediate-1 risk myelodysplastic syndromes.
Of the patients who entered the study the median age was 72 years ( range, 27-94 ).
Forty-six patients remain on study.
On a per- protocol analysis ( eligible basis ), eighty-four patients ( 51% ) responded to treatment.
Fifty-four patients ( 33% ) became transfusion independent as defined by a more stringent modification of the MDS International Working Group ( IWG ) Response Criteria, requiring eight consecutive weeks without a blood transfusion and a 1g/dl or greater rise in hemoglobin.
Thirty patients ( 18% ) achieved a minor response, defined as a 50% or greater decrease in blood transfusion requirement.
The median duration of transfusion-independence was 41 weeks.
In addition, the median baseline hemoglobin level was 8.0 grams per deciliter. With Revlimid treatment, the median increase in hemoglobin was 3.2 grams per deciliter.
Twenty patients were evaluable for cytogenetic response. Of these nine patients ( 45% ) experienced cytogenetic remission.
Based on an intent-to-treat analysis, which accounts for all patients registered in MDS-002 as opposed to only patients meeting the specific inclusion criteria on a per-protocol basis, ninety-four patients ( 44% ) responded to treatment.
Fifty-eight patients ( 27% ) became transfusion independent, defined according to International Working Group ( IWG ) as eight weeks without blood transfusion.
Thirty-six patients ( 17% ) achieved a minor response, defined as a greater than 50% decrease in transfusion dependence according to IWG criteria.
The median duration of transfusion-independence was 43 weeks. The median increase in hemoglobin (Hgb) was 3.3 grams per deciliter.
The most common Grade three or higher adverse events were neutropenia ( 24% ) and thrombocytopenia ( 19% ).
The most common non-hematologic adverse events for all National Cancer Institute Common Toxicity Criteria grades included rash ( 22% ), pruritus ( 21% ), constipation ( 14% ), diarrhea ( 13% ), and fatigue ( 12% ).
In the MDS-002 trial, twenty ( 9.3% ) patients died; two ( 0.9% ) infectious patient deaths were suspected by the local investigator to be possibly drug related.
Revlimid is a member of a new class of novel immunomodulatory drugs, or ImiDs.
Source: 10th Congress of the European Hematology Association ( EHA ), 2005