Abstract
Outcomes remain poor for refractory severe aplastic anemia (SAA) patients. Alternative donor transplantation may be considered, but results from previous studies are not encouraging. We conducted a prospective nationwide phase 2 study to assess unrelated cord blood (CB) transplantation (CBT) efficacy and safety in refractory SAA patients (Aplastic Anemia and Cord Blood Transplantation protocol). To demonstrate a significant difference in 1-year survival from 20% (null hypothesis) to 50% (alternative hypothesis), we needed to include 25 transplanted patients and therefore included 26 (median age, 16 years). Eligibility criteria required 1 or 2 unrelated CB units, containing separately or together >4 × 107 frozen nucleated cells (NCs) per kilogram of recipient body weight. Conditioning regimen comprised fludarabine (FLU), cyclophosphamide (CY), antithymocyte globulin (ATG), and 2-Gy total body irradiation (TBI). With a median follow-up of 38.8 months, engraftment occurred in 23 patients (88%); cumulative incidences of grade II-IV acute and chronic graft-versus-host disease were 45.8% and 36%, respectively. Twenty-three patients were alive at 1 year, with an 88.5% overall survival (OS) rate, differing significantly from the expect...Continue Reading
References
Oct 1, 1974·Transplantation·H GlucksbergE D Thomas
Aug 1, 1980·The American Journal of Medicine·H M ShulmanE D Thomas
Jul 1, 1999·The New England Journal of Medicine·G SociéJ P Klein
Dec 12, 2002·Bone Marrow Transplantation·S SaavedraM A Sanz
Dec 17, 2003·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Gwynn D LongNelson J Chao
Aug 30, 2008·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Ayami YoshimiUNKNOWN Japan Cord Blood Bank Network (JCBBN)
Oct 20, 2009·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Anne H BlaesClaudio G Brunstein
Jun 22, 2010·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Regis Peffault de LatourVanderson Rocha
Sep 23, 2010·JAMA : the Journal of the American Medical Association·Phillip ScheinbergNeal S Young
Apr 5, 2011·Bone Marrow Transplantation·A DominiettoA Bacigalupo
Apr 21, 2012·Blood·Phillip Scheinberg, Neal S Young
Jul 6, 2012·The New England Journal of Medicine·Matthew J OlnesCynthia E Dunbar
Dec 19, 2013·Blood·Ronan DesmondCynthia E Dunbar
Dec 4, 2016·Hematology·Régis Peffault de Latour
Jun 14, 2017·Stem Cell Investigation·Sophie ServaisYves Beguin
Nov 25, 2017·Haematologica·Etienne LenglineRegis Peffault de Latour
Citations
Jul 18, 2019·Expert Review of Hematology·Filomena Pierri, Carlo Dufour
Mar 21, 2020·Pediatric Transplantation·François SevrinBenedicte Bruno
Feb 28, 2020·Journal of Human Genetics·M De RyckeW Verpoest
Oct 31, 2019·Bone Marrow Transplantation·Shengnan DuJiahua Ding
Dec 7, 2019·Hematology·Annalisa Ruggeri
Aug 5, 2020·European Journal of Haematology·Jean-Sébastien ClaveauJean Roy
Oct 26, 2018·The New England Journal of Medicine·Neal S Young
Nov 9, 2018·Pediatric Transplantation·Georgia AvgerinouKitra Vasiliki
Dec 18, 2019·Bone Marrow Transplantation·Pedro H PrataUNKNOWN SAA WP of the EBMT
Aug 15, 2019·British Journal of Haematology·Maria GabelliRobert Chiesa
Jul 12, 2020·Bone Marrow Transplantation·P E DebureauxR Peffault de Latour
Oct 1, 2020·Expert Review of Hematology·Andrea BacigalupoLivio Pagano
Oct 17, 2019·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Shinichi KakoUNKNOWN Adult Aplastic Anemia Working Group of the Japanese Society for Hematopoietic Cell Transplantation
Oct 7, 2018·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Tetsuro OchiHideo Harigae
Aug 1, 2020·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Ioannis PolitikosUNKNOWN American Society for Transplantation and Cellular Therapy Cord Blood Special Interest Group
Apr 11, 2021·Transplantation and Cellular Therapy·Leland MethenyUNKNOWN American Society for Transplantation and Cellular Therapy Cord Blood Special Interest Group
Apr 21, 2021·Transplantation and Cellular Therapy·Kirsten M WilliamsStefanie Sarantopoulos
Nov 15, 2020·Blood Reviews·Raheel IftikharParvez Ahmed
May 17, 2021·British Journal of Haematology·Richard W ChildsGeorg Aue
May 17, 2021·British Journal of Haematology·Didier Blaise, Bipin N Savani
Jun 30, 2021·The Journal of Organic Chemistry·Xiaoxian LiYunfei Du
Aug 19, 2021·Best Practice & Research. Clinical Haematology·Andrea Bacigalupo, Giulia Benintende
Aug 22, 2021·Transplantation Proceedings·Qiurong LiuDepei Wu
Sep 22, 2021·Annals of Hematology·Nobuhiro HiramotoUNKNOWN Adult Aplastic Anemia Working Group of the Japanese Society for Hematopoietic Cell Transplantation
Oct 17, 2021·Clinical Transplantation·Fan LinLanping Xu
Nov 2, 2021·Stem Cells Translational Medicine·Xiaoyu ZhuZimin Sun
Sep 20, 2020·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Leonardo Javier ArcuriCarmem Bonfim