Unrelated cord blood transplantation in patients with idiopathic refractory severe aplastic anemia: a nationwide phase 2 study.

Blood
Regis Peffault de LatourFrancophone Society of Bone Marrow Transplantation and Cellular Therapy

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

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References

Aug 1, 1980·The American Journal of Medicine·H M ShulmanE D Thomas
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
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 4, 2016·Hematology·Régis Peffault de Latour

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Citations

Jul 18, 2019·Expert Review of Hematology·Filomena Pierri, Carlo Dufour
Feb 28, 2020·Journal of Human Genetics·M De RyckeW Verpoest
Dec 7, 2019·Hematology·Annalisa Ruggeri
Oct 26, 2018·The New England Journal of Medicine·Neal S Young
Aug 15, 2019·British Journal of Haematology·Maria GabelliRobert Chiesa
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
May 17, 2021·British Journal of Haematology·Didier Blaise, Bipin N Savani
Aug 19, 2021·Best Practice & Research. Clinical Haematology·Andrea Bacigalupo, Giulia Benintende
Sep 22, 2021·Annals of Hematology·Nobuhiro HiramotoUNKNOWN Adult Aplastic Anemia Working Group of the Japanese Society for Hematopoietic Cell Transplantation
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

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