Original Article

Bone Marrow Transplantation (2006) 38, 421–426. doi:10.1038/sj.bmt.1705467; published online 7 August 2006

Cord Blood Stem Cell

Phase II study of unrelated cord blood transplantation for adults with high-risk hematologic malignancies

L Lekakis1, S Giralt1, D Couriel1, E J Shpall1, C Hosing1, I F Khouri1, P Anderlini1, M Korbling1, T Martin2, R E Champlin1 and M de Lima1

  1. 1Department of Blood and Marrow transplantation, UT MD Anderson Cancer Center, Houston, TX, USA
  2. 2Division of Hematology and Oncology, Bone Marrow Transplantation, University of California at San Francisco, San Francisco, CA, USA

Correspondence: Dr M de Lima, Department of Blood and Marrow Transplantation, MD Anderson Cancer Center, 1515 Holcombe Blvd, Unit 423, Houston, TX 77030-4009, USA. E-mail: mdelima@mdanderson.org

Received 16 May 2006; Revised 28 June 2006; Accepted 10 July 2006; Published online 7 August 2006.

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Abstract

Cell dose is a critical determinant of outcomes in unrelated cord blood (CB) transplantation. We investigated a strategy in which CB units should contain at least 2 times 107 total nucleated cells/kg of recipient weight, otherwise a second unit had to be added. We report the results of a study that was prematurely closed owing to toxicity. Patients with advanced hematologic malignancies without a human leukocyte antigen-matched sibling or unrelated donor were eligible. Conditioning regimen consisted of fludarabine and 12 Gy of total body irradiation (n=11), or melphalan (n=4), with antithymocyte globulin. Graft-versus-host disease prophylaxis was tacrolimus and methotrexate. Fifteen patients with acute leukemia (n=9), chronic myelogenous leukemia (n=2), multiple myeloma (n=2) and lymphoma (n=2) were treated; 60% had relapsed disease at transplantation. Three patients received double CB transplants. The 100-day and 1-year treatment-related mortality rates were 40 and 53%, respectively. Median time to neutrophil and platelet engraftment was 22 days (n=10) and 37 days (n=10), respectively. One patient had secondary graft failure and five patients failed to engraft. Two patients are alive and disease free; 4-year actuarial survival is 33 versus 0% for patients transplanted in remission versus in relapse. We concluded that disease status was the main determinant of treatment failure in this study.

Keywords:

cord blood transplantation, leukemia, phase II study

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