Reduced intensity haemopoietic stem-cell transplantation for treatment of non-malignant diseases in children

Lancet
David A JacobsohnMorris Kletzel

Abstract

Transplantation of allogeneic haemopoietic stem cells can cure several non-malignant disorders in children. Transplantation with reduced intensity preparation might achieve the same goals but with less toxicity. We undertook a pilot study to determine engraftment rates, kinetics of engraftment, toxicity, and acute graft-versus-host disease (GVHD) associated with a uniform reduced intensity haemopoietic stem-cell transplant (HSCT) regimen for children with non-malignant diseases. We studied 13 paediatric patients with non-malignant disorders who underwent reduced intensity HSCT at Children's Memorial Hospital from January, 2000, to February, 2004. Stem-cell sources included unrelated donor, matched-sibling peripheral blood stem cells, and unrelated cord blood. A uniform preparative regimen was used, consisting of fludarabine, busulfan, and anti-thymocyte globulin. Major endpoints were engraftment, transplant-related mortality at day 100, short-term toxicities, and incidence of acute GVHD. 72% of evaluable patients achieved full donor engraftment. There was rapid reconstitution of platelets (median 13.5 days) and neutrophils (median 18 days). Short-term toxicities were minimal, as seen by a median length of hospital stay of 7 day...Continue Reading

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Citations

Jul 24, 2008·Bone Marrow Transplantation·I YanivUNKNOWN EBMT Paediatric Working Party
Oct 16, 2007·Bone Marrow Transplantation·D A Jacobsohn
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Aug 28, 2009·British Journal of Haematology·Shinsuke TakagiShuichi Taniguchi
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May 31, 2015·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Jessica WardWilliam T Tse
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Dec 21, 2012·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Prakash SatwaniMitchell S Cairo

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