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

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8 Citations (Scopus)

Abstract

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
Pages (from-to)896-901
Number of pages6
JournalPediatric Transplantation
Volume12
Issue number8
DOIs
StatePublished - Dec 2008

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Fetal Blood
Transplantation
Transplants
Granulocyte Colony-Stimulating Factor
Immunosuppressive Agents
Stem Cells
Human Herpesvirus 6
Viremia
Myelodysplastic Syndromes
Graft Rejection
Infection
Neutropenia
Acute Myeloid Leukemia
Leukemia
Neutropenia, Severe Congenital, Autosomal Recessive 3
Tissue Donors
Drug Therapy
Injections

Keywords

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

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Transplantation

Cite this

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title = "Unrelated cord blood transplantation for severe congenital neutropenia: Report of two cases with very different transplant courses",
abstract = "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.",
keywords = "Conditioning, Engraftment, Kostmann's syndrome, Severe congenital neutropenia, Umbilical cord blood",
author = "Markel, {Melissa K.} and Paul Haut and Jamie Renbarger and Kent Robertson and W. Goebel",
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T1 - Unrelated cord blood transplantation for severe congenital neutropenia

T2 - Report of two cases with very different transplant courses

AU - Markel, Melissa K.

AU - Haut, Paul

AU - Renbarger, Jamie

AU - Robertson, Kent

AU - Goebel, W.

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AB - 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.

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