The present role of bone marrow and stem cell transplantation in the therapy of children with acute leukemia

Ann N Y Acad Sci. 1997 Sep 17:824:38-64. doi: 10.1111/j.1749-6632.1997.tb46208.x.


Hematopoetic stem cell transplantation (SCT) often represents a unique opportunity for curing children with leukemia. Nevertheless, selecting the patient who could really benefit from this procedure remains a controversial issue. The current consensus is as follows: About 20% of children with ALL can be defined as high-risk patients by criteria such as t(9;22), t(4;11), no complete remission at day 42, poor prednisone response, and T-immunophenotype or pre-pre B-ALL, myeloid markers or more than 100,000 white blood cells/microliter. This high-risk group is eligible for alloBMT in first remission, provided a family-matched donor is available. At relapse the majority of patients will benefit from alloBMT, and alternative donor sources can be considered in high-risk patients. Only early alloBMT relapses (up to 6 months after end of initial therapy) are sure candidates, whereas late relapses, especially extramedullary sites, may equally benefit from an intensive conventional relapse treatment. However, any alloBMT relapse beyond second remission should be transplanted with allogeneic stem cells (bone marrow or peripheral stem cells). In particular, family mismatched donors or matched unrelated donors may be acceptable in high-risk cases beyond first remission. In contrast, ASCR in ALL seems not to be superior to conventional therapy. In AML the standard-risk patient, defined by criteria such as FAB M1/M2-Auer rods positive, all FAB M3, and FAB M4, is not a candidate for SCT in first remission. Patients presenting other criteria or more than 5% of blasts in the bone marrow at day 15 are at high risk in first remission and should be considered for allo BMT if a family matched donor is available. ASCR in first remission AML remains a controversial issue. In contrast, in second remission alloBMT as well as ASCR are superior to conventional chemotherapy.

Publication types

  • Review

MeSH terms

  • Acute Disease
  • Bone Marrow Transplantation*
  • Child
  • Clinical Trials as Topic
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Infant
  • Leukemia, Myeloid / therapy*
  • Multicenter Studies as Topic
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma / therapy*
  • Prognosis