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Unrelated cord blood transplantation for severe congenital neutropenia: Report of two cases with very different transplant courses
Author(s) -
Markel Melissa K.,
Haut Paul R.,
Renbarger Jamie A.,
Robertson Kent A.,
Scott Goebel W.
Publication year - 2008
Publication title -
pediatric transplantation
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.457
H-Index - 69
eISSN - 1399-3046
pISSN - 1397-3142
DOI - 10.1111/j.1399-3046.2008.00951.x
Subject(s) - medicine , neutropenia , stem cell , transplantation , leukemia , surgery , congenital neutropenia , umbilical cord , myelodysplastic syndromes , gastroenterology , chemotherapy , bone marrow , immunology , genetics , biology
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.