Unrelated cord blood transplantation for severe congenital neutropenia: Report of two cases with very different transplant courses

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9 Scopus citations


SCN is characterized by neutropenia, life-threatening infections, and progression to myelodysplastic syndrome/acute myelogenous leukemia. The only curative option is SCT, but few reports using UCB as a stem cell source exist. Here, we report two SCN patients transplanted with UCB. Patient 1 was transplanted at seven yr of age due to increasingly large injections of G-CSF (>100 μg/kg/day) and the risk of developing leukemia. He engrafted promptly and is clinically well and immune reconstituted >2 yr post-transplant. Patient 2 underwent UCB SCT at nine months of age for recurrent severe infections, despite high doses of G-CSF. He rejected his first graft, having 100% host cells on day +35, and immediately underwent a second UCB SCT. He engrafted but experienced late graft rejection six months after the second transplant. He received a third UCB SCT following a more immunosuppressive conditioning regimen. His course was complicated by HHV-6 viremia and gut GVHD, but he is now clinically well and has 99% donor engraftment >20 months post-transplant. We conclude that UCB is an acceptable stem cell source for SCN patients, but conditioning must be adequately immunosuppressive to ensure engraftment in patients without prior chemotherapy.

Original languageEnglish (US)
Pages (from-to)896-901
Number of pages6
JournalPediatric Transplantation
Issue number8
StatePublished - Dec 1 2008



  • Conditioning
  • Engraftment
  • Kostmann's syndrome
  • Severe congenital neutropenia
  • Umbilical cord blood

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Transplantation

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