Clinical trial update in MDS

Chair: P. Fenaux.

Update on MDS trials presented by E. Hellström-Lindberg for Nordic Group.

1. ATG + CsA for low risk MDS. No difference in survival between treatment v supportive therapies. A number of elderly patients were admitted to ICU .
2. Epo + G-CSF. No negative impact on survival. Good response rate in sideroblastic anaemia. Median survival in responders 29 months. Will compare with Pavia group.
3. Darbopoietin + RBC transfusion v transfusion. 66% response rate to Darbopoietin. Practical problems with transfusion in some patients. QOL and performance currently being evaluated.
4. Maintenance Azacytadine in high risk MDS, MDS/AML who are in CR after chemotherapy. Neutropenia was a problem but did not result in loss of patients from trial. Best response seen in patients with good cytogenetics.
5. A study with Lenalidomide in high risk MDS is planned.

Update from EORTC. P Muus.

1. Currently investigating Infliximab in low and intermeidate risk MDS.
2. Randomising auto v allograft for MDS in CR after chemo. Compare results with MD Anderson. Overall survival was the same with chemo only v allo or autograft. Again cytogenetics provided best prognostic information. CRIANT study. Chemo followed by randomization to auto or HDAC. CR 57%. 4 year survival 28%. No difference in two groups but numbers in final randomization were very low. Currently investigating Myelotarg added toinduction regimens.

Update from German group. A Ganser.

1. Very large registry in Dusseldorf. Detailed data on diagnostic information and follow up on 2000 patients.
2. Most patients over 70 years.
3. 5q- seen as the only abnormality in 50% and combined with other abnormalities in 50%.
4. Looking at methylation patterns in CD34+ cells from MDS and normal with a view to targeted therapy.
5. ATG + CsA v support for low risk MDS. Results awaited.
6. Lenalidomide trial: difficulty with Regulators has delayed start.
7. Trials with Azacytadine v support and Decitabine v support are being designed.

Update of French group. P Fenaux.

1. Currently investigating Lenalidomide in two doses v placebo (Cellgene sponsored).
2. Darbopoietin+- G-CSF. 69% response at 12 weeks. Better than Epo. Median duration of response 38 weeks.
3. Epo beta +- ATRA. Response reflects serum Epo levels. ATRA may have a role in patients failing to respond to Epo. Limited response in WBC and platelets.
4. Arsenic Trioxide. 26% response rate in low risk group and 17% in high risk group. ATRA may be added to other drugs in future trials.
5. Maintenance Aza gave same result as Nordic group.
6. Current trial: Lenalidomide in 5q-.
7. Current trial:Bortezomib + LDAC in high and int risk MDS. Also ATO + LDAC.