Presenter: Alvaro Moreno-Aspitia Presenter's Affiliation: Mayo Clinic, Rochester, MN Type of Session: Scientific
Bone Marrow specimens in patients with MDS have increased levels of the following cytokines: TNF-á, TGF-â, IL-1, and IFN-ã.
Bone Marrow specimens also demonstrate increased neovascularization.
For these reasons, thalidomide seems to be a good option for the treatment of MDS.
This trial was undertaken to assess the efficacy and toxicity of thalidomide in adult MDS patients
Materials and Methods
Because of the heterogenous nature of this disease, patients were stratified into two groups based on the International Prognostic Scoring System (IPSS): favorable (FG; IPSS 0-1.0) and unfavorable (UG; IPSS 1.5-3.5).
A total of 73 patients were accrued in 10 months
Starting dose of thalidomide was 200mg/day with dose escalation of 50mg/week to a maximum goal of 1000mg/day.
At least half of patients in each group reached a max dose of 300mg/day.
Median Follow-up was 4 months.
There were no CR's
There was only 1 PR in the UG
4 patients had a major hematologic improvement (HI-maj). 1 in the FG and 3 in the UG.
3 patients had a minor HI (HI-min). 1 in the FG and 2 in the UG.
Most patients had stable disease (34 in FG and 17 in UG)
Only 10 patients failed therapy. (4 in FG and 6 in UG)
Only 44% of patients were able to complete three or more cycles of therapy and most patients ended therapy early due to toxicity.
For both groups grade 3 or higher toxicity occurred in approximately 90% of patients.
The current multi-center study in unselected patients with MDS demonstrates limited therapeutic efficacy of thalidomide alone. There was significant toxicity of this dose of thalidomide in this population of patients. Responses were limited to patients with intermediate IPSS and with less than 10% blasts in the marrow.
Perhaps future studies should be directed at lower dose thalidomide for a longer duration which may be better tolerated and thus more beneficial for patients with MDS. The current study dosing does not show any significant benefit for the use of Thalidomide in MDS.
Oncolink's ASH Coverage made possible by an unrestricted Educational Grant from Ortho Biotech.