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Allogeneic Peripheral Blood Stem-Cell Compared With Bone Marrow Transplantation in the Management of Hematologic Malignancies: An Individual Patient Data Meta-Analysis of Nine Randomized Trials

From the Department of Interdisciplinary Oncology, University of South Florida, H. Lee Moffitt Cancer Center & Research Institute, Tampa, FL.

Address reprint requests to Benjamin Djulbegovic, MD, PhD, on behalf of Stem Cell Trialists' Collaborative Group, Department of Interdisciplinary Oncology, University of South Florida, SRB 4, Floor 4, Room 24031 (Room W 31), 12902 Magnolia Dr, Tampa, FL 33612; e-mail: djulbebm{at}moffitt.usf.edu

PURPOSE: Considerable uncertainty exists regarding relative effects of allogeneic peripheral blood stem cells transplantation (PBSCT) versus bone marrow transplantation (BMT) on outcomes of patients with hematologic malignancies.

PATIENTS AND METHODS: To provide the totality of research evidence related to the effects of PBSCT versus BMT, we conducted an individual-patient data meta-analysis using data from nine randomized trials enrolling 1,111 adult patients.

RESULTS: Compared with BMT, PBSCT led to faster neutrophil (odds ratio [OR] = 0.31; 95% CI, 0.25 to 0.38; P < .00001) and platelet engraftment (OR = 0.52; 95% CI, 0.44 to 0.61; P < .00001). PBSCT was associated with a significant increase in the development of grade 3-4 acute graft-versus-host disease (GVHD; OR = 1.39; 95% CI, 1.03 to 1.88) and extensive (47% v 31% at 3 years; OR = 1.89; 95% CI, 1.47 to 2.42; P < .000001) and overall chronic GVHD (68% v 52% at 3 years; OR = 1.92; 95% CI, 1.47 to 2.49; P < .000001), but not grade 2-4 acute GVHD (54% v 53%; P = .49). PBSCT was associated with a decrease in relapse (21% v 27% at 3 years; OR = 0.71; 95% CI, 0.54 to 0.93; P = .01) in both late-stage–(33% v 51% at 3 years; OR = 0.59; 95% CI, 0.38 to 0.93; P = .02) and early-stage–disease patients (16% v 20% at 3 years; OR = 0.69; 95% CI, 0.49 to 0.98; P = .04). Nonrelapse mortality was not different between groups. Overall and disease-free survival were only statistically significantly improved in patients with late-stage disease (overall survival: 46% v 31% at 3 years; OR = 0.64; 95% CI, 0.46 to 0.90; P = .01; disease-free survival: 41% v 27% at 3 years; OR = 0.63 95% CI, 0.45 to 0.87; P = .01).

CONCLUSION: PBSCT is associated with a decreased relapse rate in hematologic malignancies and improvement in overall and disease-free survival in patients with late-stage disease. PBSCT is also associated with a significant risk of extensive chronic GVHD.

Supported by the NIH/NHLBI grant No. 1R01HL71650-01 (PI: Dr Djulbegovic), and in part by The Jose Carreras Foundation Against Leukemia, NCI CA18029, CA18221 (Dr Bensinger), the Swiss National Research Foundation (Dr Gratwohl), and the French Ministry of Health (Programme Hospitalier de Recherche Clinique 1996); and a grant from the Ligue Nationale de Lutte Contre le Cancer (Dr Blaise).

Authors' disclosures of potential conflicts of interest are found at the end of this article.

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