Outcome of second allogenic stem cell transplantation in pediatric patients with non-malignant hematological and immune deficiency disorders

Pediatr Blood Cancer. 2011 Feb;56(2):289-93. doi: 10.1002/pbc.22804. Epub 2010 Oct 22.

Abstract

Background: Second stem cell transplantation (SCT) is usually associated with high morbidity and mortality and the data on its outcome in pediatric patients with non-malignant disorders are scarce.

Patients and methods: We present 30 children with non-malignant conditions who underwent second SCT at our institution for graft failure after the first SCT; 20 had a non-malignant hematological disorder and 10 had an immune deficiency disorder. Median age at the second SCT was 6.1 years (range, 0.4-13 years) and median time from the first SCT to the second SCT was 6.2 months (range, 1.2-96 months).

Results: Twenty patients (70%) engrafted; severe acute GVHD developed in four patients (13%), and chronic GVHD developed in two patients of those at risk (10%). Thirteen deaths occurred and nine were considered treatment related. The 5-year overall (OS) and event free survival (EFS) for all patients were 53% and 47% respectively. The interval between the two transplants seemed to affect the outcome; patients who had the second SCT ≥ 6 months from the first SCT had better survival; the 5-year OS for the two groups (<6 months and ≥ 6 months) respectively were 30% and 74% (P = 0.004), and the 5-year EFS were 27% and 66% (P = 0.004). The underlying disease did not affect the outcome nor did the use of radiation in the conditioning regimen for the second SCT.

Conclusions: Second SCT for graft failure should be considered for children with non-malignant hematological and immune deficiency disorders.

MeSH terms

  • Adolescent
  • Child
  • Child, Preschool
  • Disease-Free Survival
  • Female
  • Graft vs Host Disease / etiology
  • Graft vs Host Disease / surgery
  • Hematologic Diseases / mortality
  • Hematologic Diseases / surgery*
  • Hematopoietic Stem Cell Transplantation* / adverse effects
  • Humans
  • Immunologic Deficiency Syndromes / mortality
  • Immunologic Deficiency Syndromes / surgery*
  • Infant
  • Kaplan-Meier Estimate
  • Male
  • Reoperation
  • Transplantation, Homologous
  • Treatment Failure