Abstract
Allogeneic hematopoietic cell transplantation (HCT) for children with nonmalignant disorders is challenged by potential drug-related toxicities and poor engraftment. This retrospective analysis expands on our single pediatric medical center experience with targeted busulfan, fludarabine, and intravenous (IV) alemtuzumab as a low-toxicity regimen to achieve sustained donor engraftment. Sixty-two patients received this regimen for their first HCT for a nonmalignant disorder between 2004 and 2018. Donors were matched sibling in 27%, 8/8 HLA allele-matched unrelated in 50%, and 7/8 HLA allele-mismatched in 23% (some of whom received additional immunoablation with thiotepa or clofarabine). Five patients experienced graft failure for a cumulative incidence of 8.4% (95% CI, 1 to 16%). In engrafted patients, the median donor chimerism in whole blood and CD3, CD14/15, and CD19 subsets at 1-year were 96%, 90%, 99%, and 99%, respectively. Only one patient received donor lymphocyte infusions (DLIs) for poor chimerism. Two patients died following disease progression despite 100% donor chimerism. The 3-year cumulative incidence of treatment-related mortality was 10% (95% CI, 2 to 17%). Overall survival and event-free-survival at 3-years were...Continue Reading
References
Jan 1, 1996·Cancer Chemotherapy and Pharmacology·L E RobertsonJ A Nelson
Aug 22, 2002·Bone Marrow Transplantation·Z HassanM Hassan
Oct 31, 2007·Bone Marrow Transplantation·C C Dvorak, M J Cowan
Jan 27, 2009·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Imke H BartelinkJaap Jan Boelens
Mar 24, 2010·Pediatric Clinics of North America·Frans J SmiersGuido Lucarelli
Apr 8, 2010·Blood·Adam J MeadKarl S Peggs
Sep 8, 2010·Bone Marrow Transplantation·J StyczynskiM S Cairo
Feb 18, 2011·Blood·Cindy NeunertUNKNOWN American Society of Hematology
Dec 14, 2011·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Ji Won LeeHyo Seop Ahn
May 23, 2012·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Jason LawBiljana Horn
Oct 29, 2013·Lancet·Tayfun GüngörUNKNOWN Inborn Errors Working Party of the European Society for Blood and Marrow Transplantation
May 31, 2014·Blood·Sonata JodeleBenjamin L Laskin
Apr 14, 2015·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Rebecca A MarshStella M Davies
May 31, 2015·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Jessica WardWilliam T Tse
Dec 9, 2015·Blood·Rebecca A MarshAlexandra H Filipovich
Oct 18, 2016·The Lancet. Haematology·Imke H BartelinkJaap Jan Boelens
Jan 10, 2017·Frontiers in Immunology·Raja Rajalingam
Jul 8, 2017·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Vijay IvaturiJanel Long-Boyle
Aug 2, 2017·Bone Marrow Transplantation·S CorbaciogluP Bader
Jun 7, 2018·Bone Marrow Transplantation·Helene M SchoemansUNKNOWN EBMT (European Society for Blood and Marrow Transplantation) Transplant Complications Working Party and the “EBMT−NIH (Natio
Jul 13, 2018·Blood·Carl E AllenMichael A Pulsipher
May 10, 2020·Blood Advances·Matthias FelberTayfun Güngör
Citations
Oct 18, 2020·Journal of Clinical Immunology·Sharat ChandraRebecca A Marsh
Mar 31, 2021·Transplantation and Cellular Therapy·Beth Apsel WingerJanel Long-Boyle
Jul 19, 2021·Bone Marrow Transplantation·Tami D JohnKathryn Leung
Aug 11, 2021·The Journal of Allergy and Clinical Immunology·Rebecca A MarshMary Eapen
Oct 17, 2021·Journal of Clinical Immunology·Alexandra Y KreinsClaire Booth
Dec 11, 2021·Hematology·Paulina HorveiSandhya Kharbanda