Objectives

To compare survival rates of patients with hematologic cancers that received transplantation of granulocyte colony stimulating factor (G-CSF) mobilized peripheral blood stem cells (PBSC) versus marrow from HLA-compatible unrelated donors.

Background

Many studies of allogeneic marrow transplantation have shown that a higher dose of marrow cells correlates with a stronger hematopoietic engraftment and lower mortality from infectious complications. Peripheral blood stem cells (PBSC) collected after mobilization with granulocyte colony stimulating factor (G-CSF) contain a large number of CD34-positive progenitors and total cells than bone marrow. These observations led to the hypothesis that transplantation of PBSC would lead to lower mortality compared to transplantation of marrow. However, some studies have also shown increased risk of acute and chronic graft-versus-host disease (GVHD), as compared to bone marrow.

Participants

Between March 2004 and September 2009, 551 subjects were enrolled at 48 centers. Eligible subjects were less than 66 years of age and were planning to undergo transplantation for acute leukemia, myelodysplasia, chronic myeloid or myelomonocytic leukemia, or myelofibrosis.

Design

Subjects were randomly assigned in a 1:1 ratio to peripheral-blood stem-cell or bone marrow transplantation, stratified according to transplantation center and disease risk. Donors and recipients were DNA typed to ensure matching for 5 or 6 of the HLA-A, B, and DRB1 antigens. Peripheral-blood stem-cell donors were prescribed filgrastim or lenograstim at a dose of 10 μg per kilogram per day for 5 days and underwent apheresis. Recipients received one of four conditioning regimens and one of two GVHD prophylaxis regimens that were specified prior to randomization. Supportive care and treatment for GVHD were provided according to institutional standards. Subjects were followed in the study for 3 years.

The primary end point was 2-year survival as assessed by means of an intention-to-treat analysis. Secondary end points included post-transplantation incidences of neutrophil and platelet engraftment, graft failure, acute and chronic GVHD, relapse, and infections.

Conclusions

There was no detected significant survival differences between peripheral-blood stem-cell and bone marrow transplantation from unrelated donors. Further analysis indicated that peripheral-blood stem cells may reduce the risk of graft failure, whereas bone marrow may reduce the risk of chronic GVHD.

N Engl J Med. 2012 Oct 18;367(16):1487-96.

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