No Difference in Event-Free Survival With Stem Cell Transplantation Using Matched Unrelated vs Sibling Donor Grafts in Children With ALL


Key Points

  • No difference in event-free survival was observed for matched unrelated donor vs sibling donor grafts.
  • No difference in event-free survival was observed for matched unrelated donor grafts matched at 9 vs 10 HLA loci.

In a European trial (Berlin-Frankfurt-Muenster study group trial ALL-SCT-BFM 2003) reported in the Journal of Clinical Oncology, Peters et al found no difference in event-free survival with stem cell transplantation using matched unrelated vs sibling donors in pediatric patients with high-risk acute lymphoblastic leukemia (ALL).

Study Details

In the trial, after conditioning with total-body irradiation and etoposide, 411 patients received grafts from HLA-genoidentical siblings (n = 105) or HLA-matched unrelated donors (n = 306) who were identified and matched by high-resolution allelic typing and were compatible in ≥ 9 of 10 HLA loci. Treatment occurred between September 2003 and September 2011. The primary endpoint was event-free survival.

Event-Free Survival

There was no difference in 4-year event-free survival between the matched unrelated donor group and the sibling donor group (67% vs 71%, P = .405). There were no differences in 4-year overall survival (73% vs 79%, P = .230) or 4-year incidence of relapse (22% vs 24%, P = .732). Four-year nonrelapse mortality was greater in the matched unrelated donor group (10% vs 3%, P = .017).

There were no differences in event-free survival, overall survival, or nonrelapse mortality between patients in the matched unrelated donor group with 9 vs 10 matched HLA loci and no differences according to use of peripheral blood stem cells or bone marrow.

Graft-vs-Host Disease and Engraftment

No between-group differences in incidence or severity of acute graft-vs-host disease were found, with extensive chronic graft-vs-host disease occurring more frequently in the matched sibling donor group. No differences in event-free survival were found for patients with vs without limited or extensive chronic graft-vs-host disease. Engraftment occurred earlier after bone marrow transplantation from siblings (median time to neutrophil engraftment = 22 vs 17 days, 30-day cumulative incidence = 44% vs 76%, P < .001), with the sibling group having lower rates of infection (39% vs 16%, P < .001) and pulmonary complications (eg, hypoxia in 18% vs 9%, P = .026).

The investigators concluded: “Outcome among high-risk pediatric patients with ALL after hematopoietic stem-cell transplantation was not affected by donor type. Standardized myeloablative conditioning produced a low incidence of treatment-related mortality and effective control of leukemia.”

Christina Peters, MD, of St. Anna Children’s Hospital, Vienna, is the corresponding author for the Journal of Clinical Oncology article.

The study was supported by the Children’s Cancer Research Institute, Vienna, Deutsche Krebshilfe, Deutsche Knochenmarkspenderdatei, Orphan Pharmaceuticals,  Amomed Pharma,  Fresenius Biotech, Gilead Sciences, and Medac. For full disclosures of the study authors, visit

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.