Cord Blood Transplant in Leukemia With Minimal Residual Disease Interview with:

Dr. Filippo Milano, MD, PhD Assistant Member, Clinical Research Division Associate Director Cord Blood Transplantation Cord Blood Program Assistant Professor, University of Washington Fred Hutchinson Cancer Research Center

Dr. Filippo Milano

Dr. Filippo Milano, MD, PhD
Assistant Member, Clinical Research Division
Associate Director Cord Blood Transplantation
Cord Blood Program
Assistant Professor, University of Washington
Fred Hutchinson Cancer Research Center What is the background for this study?

Response: When first introduced, cord blood (CB) graft was used only as a last resort when no suitable conventional donor could be identified, largely due to the limiting cell doses available in a cord blood graft. A CB graft, however, is attractive due to the increased level of HLA disparity that can be tolerated, without increased risk of graft versus host disease, allowing nearly all patients to find such a donor.

The main intent of the study was to evaluate whether or not, at our Institution, cord blood SHOULD STILL BE considered only AS an alternative DONOR or IF instead outcomes were comparable to those obtained with more “conventional” types of transplants from matched and mismatched unrelated donors. What are the main findings?

Response: Our study showed:
1. Overall survival after cord blood transplantation (CBT) was outstanding and comparable to the one observed after matched unrelated transplants with suggestion of better survival when compared to patients receiving mismatched unrelated transplants.
2. THE RISK OF POST TRANSPLANT DISEASE RELAPSE WAS LOWEST IN CORD BLOOD TRANSPLANT RECIPIENTS. THIS WAS MOST PRONOUNCED IN PATIENTS WITH residual disease still measurable before transplantation, IN WHOM RELAPSE was significantly lower in patients receiving cord blood transplant.

Therefore for patients who are at high risk of post-transplant relapse, the ability to rapidly identify and procure a  cord blood donor may offer the advantage of a shorter time to transplantation. Furthermore CBT offers a unique platform considering that the CB units are readily available and there is no risk for the donor differently than for transplants using different stem cell sources. What should readers take away from your report?

Response: It is a retrospective study and as such conclusions need to be drawn with caution. However our study shows that  cord blood transplant is associated with very positive outcomes and that maybe we should not consider it only as an alternative in case no other donors are available. Indeed our results suggest that for patients at high risk of relapse it might be considered as a first choice. What recommendations do you have for future research as a result of this study?

Response: Our study still confirms that   cord blood transplant is associated with delayed hematopoietic recovery which represent a big obstacle in the CBT setting. Novel strategies, including ex-vivo stem cell expansion, are now becoming part of the standard clinical approach and preliminary results are extremely encouraging. The hope is to keep improving CBT outcomes over the 70% survival rate that has been reported in the study. Ideally our study should promote a better investigation between different stem cell sources currently used in the allogeneic transplant setting.

A randomized study is the only way that differences between   cord blood transplant and matched and mismatched unrelated transplants can be definitively assessed. Our hope is to plan such study in the near future Is there anything else you would like to add?

Response: I would like to highlight once more how important is the contribution and all the support we receive by the clinical care team at the SCCA, SEATTLE CHILDRENS HOSPITAL and at UWMC. Their expertise and care is outstanding and CONTRIBUTES SIGNIFICANTLY TO THE great outcomes for patients treated at our center. Furthermore all the staff working in the CB team ARE MOTIVATED BY THE DESIRE TO MAKE A DIFFERENCE IN THE LIVES OF OUR PATIENTS. THEIR PASSION IS UNPARALLELED and the success of this study has to be shared with all of them and with all the patients who AGREES to be part of our clinical trials. Thank you for your contribution to the community.


Cord-Blood Transplantation in Patients with Minimal Residual Disease

Filippo Milano, M.D., Ph.D., Ted Gooley, Ph.D., Brent Wood, M.D., Ann Woolfrey, M.D., Mary E. Flowers, M.D., Kristine Doney, M.D., Robert Witherspoon, M.D., Marco Mielcarek, M.D., Joachim H. Deeg, M.D., Mohamed Sorror, M.D., Ann Dahlberg, M.D., Brenda M. Sandmaier, M.D., Rachel Salit, M.D., Effie Petersdorf, M.D., Frederick R. Appelbaum, M.D., and Colleen Delaney, M.D.
N Engl J Med 2016; 375:944-953
September 8, 2016 DOI: 10.1056/NEJMoa1602074

Not intended as medical advice.
Please consult your health care provider regarding your specific medical condition and questions.

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Last Updated on September 8, 2016 by Marie Benz MD FAAD