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Drs. Alexanian and Barlogie were given the opportunity to present concluding remarks on the role of high dose therapy/stem cell transplantation in multiple myeloma.

Concluding Remarks On High Dose Therapy/Stem Cell Transplantation:  Dr. Raymond Alexanian

Dr. Alexanian summarized his experience with tandem transplantation with an eye towards understanding the outstanding issues. The base included patients with the following characterisitics:

A total of 376 patients were studied, about two-thirds responsive to induction therapy.

For patients with responsive myeloma, there was only a modest survival advantage for high dose therapy versus patients with a partial response to standard therapy.

Patients with unresponsive myeloma were most likely to benefit from tranplant within one year of initial treatment, although the advantage did not persist beyond that first year.

People achieving complete remission had the longest survival irrespective of whether or not they had been transplanted.

The highest CR rate was achieved in those who had already had a partial response to treatment.

While there is apparent sutvival advantage for high dose therapy, the difference was not statistically significant for this group of patients.

Dr, Alexanian showed data that the benefit of the second transplant in terms of the incremental protein reduction was mlimited.

Dr. Alexanian concluded by saying that while there is major value in autologous transplantation for myeloma patients, the case for tandem transplants has yet to be made.

Concluding Remarks On High Dose Therapy/Stem Cell Transplantation:  Dr. Bart Barlogie

Dr. Barlogie focused on consolidating the response in trials designed to accelerate cure, emphasising the benefits of tandem transplats, particularly for patients with good risk profiles.

Dr. Barlogie reviewed the Little Rock Total Therapy I and II protocols for tandem transplant...

...and shared results from 550 patients who received the Total Therapy II regimen.

Patients with chromosal abnormailities had poorer overall and event-free survival.

Dr. Barlogie also presented a comparison of 231 patients from each regimen...

The patients in the Total Therapy II program had higher rates of complete or near complete remission. There were also a larger number of older patients successfully completing the regimen with lower transplat-related mortality.

Comparing the two groups of patients, the Total Therapy II patients had better overall survival.

Although the follow-up periods for Total Therapy II patients are still relatively short, there appears to be an advantage in both overall and event free survival for those patients without chromosomal abnormalities.

High LDH appears to be a risk factor for early relapse and transplant-related mortality.

Dr. Barlogie concluded by discussing how what has been learned about risk groups, defined by chromosomal abnormalities and LDH levels are shaping thinking about Total Therapy III.

Clearly there has been much good work done in advancing the state of the art in high dose therapy/stem cell transplantation. Yet, there are still many unknowns, given the difficulty of conducting prospective, randomized trials that provide clear answers to the questions about who, specifically should have a transplant, the timing of the transplant, the conditioning regimens, the potential role of second transplants, and the role of maintenance therapy.

Further complicating these questions is the good news that with new therapies/new agents coming onto the scene, the definition of "conventional therapy" will likely change, potentially raising the bar for transplant benefit.

Lots of good news here for the patient community!

Please note that the Unknown Patient is a patient, not a doctor and not a scientist. This summary represents a layman's view of what was said at the conference and should form a basis for raising awareness of issues that could be discussed with a qualified professional. In no way should anything contained in this report be taken as medical advice or form the basis for action without first consulting a qualified medical professional who is familiar with your specific medical situation.