Hematopoietic stem-cell transplantation using unrelated cord-blood versus matched sibling marrow in pediatric bone marrow failure syndrome

One center's experience

Peter H. Shaw, Paul Haut, Marie Olszewski, Morris Kletzel

Research output: Contribution to journalArticle

29 Citations (Scopus)

Abstract

Hematopoietic stem-cell transplantation (HSCT) is an effective mode of therapy in pediatrics for the treatment of both malignant and non-malignant disorders. We compared the course of children transplanted with unrelated umbilical cord blood (UCB) to those transplanted with allogeneic sibling bone marrow (BM) for bone marrow failure syndromes. Thirteen patients with a median age of 6.3 years were transplanted for the following diseases between April 1992 and November 1997: myelodysplastic syndromes, aplastic anemia, Diamond-Blackfan anemia, myelofibrosis, paroxysmal nocturnal hemoglobinuria, osteopetrosis and dyskeratosis congenita. The stem cell source was BM in ten patients and UCB in three. We retrospectively examined the conditioning regimens, stem cell source and dose, days to engraftment, survival and complication rate to see whether there was a significant advantage in using one source over the other. The median time to an absolute neutrophil count > 500 per μL was 25 days for UCB patients and 16 days for BM patients. The median time to a platelet count > 20 000 per μL was 55 days for UCB patients and 22 days for BM patients. The 100-day mortality was 66% in UCB patients and 20% in BM patients. The overall mortality rates were 66% and 40%, respectively. Three patients died prior to engraftment. Seven patients (54%) were still alive as of May 1999 with a median follow-up of 1574 days post- transplant. The patients transplanted with BM had faster engraftment and lower rates of graft-versus-host disease, 100-day mortality and overall mortality. HLA-matched sibling BM is preferred as a source but transplantation using unrelated UCB is still an option in treating pediatric bone marrow failure syndromes.

Original languageEnglish (US)
Pages (from-to)315-321
Number of pages7
JournalPediatric Transplantation
Volume3
Issue number4
DOIs
StatePublished - Nov 1999
Externally publishedYes

Fingerprint

Hematopoietic Stem Cell Transplantation
Fetal Blood
Siblings
Bone Marrow
Pediatrics
Mortality
Bone Marrow failure syndromes
Stem Cells
Dyskeratosis Congenita
Diamond-Blackfan Anemia
Osteopetrosis
Paroxysmal Hemoglobinuria
Primary Myelofibrosis
Aplastic Anemia
Myelodysplastic Syndromes
Graft vs Host Disease
Platelet Count
Neutrophils
Survival Rate
Transplantation

Keywords

  • Cord blood
  • Marrow failure syndromes
  • Transplantation

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Transplantation

Cite this

Hematopoietic stem-cell transplantation using unrelated cord-blood versus matched sibling marrow in pediatric bone marrow failure syndrome : One center's experience. / Shaw, Peter H.; Haut, Paul; Olszewski, Marie; Kletzel, Morris.

In: Pediatric Transplantation, Vol. 3, No. 4, 11.1999, p. 315-321.

Research output: Contribution to journalArticle

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