Reduced intensity allogeneic umbilical cord blood transplantation in children and adolescent recipients with malignant and non-malignant diseases

Bone Marrow Transplant. 2007 Oct;40(7):621-31. doi: 10.1038/sj.bmt.1705785. Epub 2007 Jul 30.

Abstract

There is a significant amount of morbidity and mortality following myeloablative umbilical cord blood transplantation (UCBT). Reduced intensity (RI) conditioning offers an alternative to myeloablative conditioning before UCBT. We investigated RI-UCBT in 21 children and adolescents with malignant (n=14), and non-malignant diseases (n=7). RI conditioning consisted of fludarabine (150-180 mg/m2) with either busulfan (< or = 8 mg/kg)+rabbit antithymocyte globulin (R-ATG; n=16) or cyclophosphamide+R-ATG+/-etoposide (n=5). Human leukocyte antigen match: 4/6 (n=13), 5/6 (n=5) and 6/6 (n=3). The median total nucleated cell and CD34+ cell dose per kilogram were 3.58 x 10(7) and 2.54 x 10(5), respectively. The median time for neutrophil and platelet engraftment was 17.5 and 52 days, respectively. There were six primary graft failures (chronic myelogenous leukemia (CML), beta-thalassemia, hemophagocytic lymphohistiocytosis (HLH) and myelodysplastic syndrome (MDS)). The probability of developing grade II to grade IV acute graft-versus-host disease (GVHD) and chronic GVHD was 28.6 and 16.7%, respectively. Incidence of transplant-related mortality (TRM) was 14%. The 5 years overall survival (OS) in all patients was 59.8%. The 5 years OS for patients with average versus poor-risk malignancy was 77.8 versus 22.2% (P=0.03). RI-UCBT may result in graft failure in specific high-risk chemo-naïve patients (CML, beta-thalassemia, HLH and MDS), but in more heavily pretreated pediatric and adolescent recipients results in rapid engraftment and may be associated with decreased severe GVHD and TRM.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Antigens, CD34 / analysis
  • Child
  • Child, Preschool
  • Cord Blood Stem Cell Transplantation / methods*
  • Fetal Blood / cytology*
  • Graft vs Host Disease / prevention & control
  • Granulocyte-Macrophage Colony-Stimulating Factor / therapeutic use
  • Humans
  • Immunologic Factors / therapeutic use
  • Living Donors
  • Myelodysplastic Syndromes / mortality
  • Myelodysplastic Syndromes / therapy
  • Neoplasms / mortality
  • Neoplasms / therapy*
  • Patient Selection
  • Recombinant Proteins
  • Survival Analysis
  • Transplantation Chimera
  • Transplantation Conditioning
  • Treatment Failure
  • Treatment Outcome
  • beta-Thalassemia / mortality
  • beta-Thalassemia / therapy

Substances

  • Antigens, CD34
  • Immunologic Factors
  • Recombinant Proteins
  • sargramostim
  • Granulocyte-Macrophage Colony-Stimulating Factor