Fludarabine-based conditioning for allogeneic transplantation in adults with sickle cell disease

Bone Marrow Transplantation
Koen van BesienR Hoffman


Although allogeneic transplantation can be curative for patients with sickle cell disease, the toxicity of conditioning regimens has precluded its use in adults with significant end-organ damage. Newer conditioning regimens have been developed that are less toxic and that may broaden the applicability of allogeneic transplantation in this disorder. We report two adults with end-stage sickle cell disease, who underwent allogeneic transplantation from an HLA-identical sibling donor after conditioning with fludarabine/melphalan and ATG. Both patients had been extensively transfused and one had multiple RBC antibodies. One of the patients also had end-stage renal disease, and was dialysis dependent. Engraftment occurred promptly in both patients. Both achieved 100% donor chimerism and both were free of pain crises after transplant. The first patient died of a respiratory failure related to chronic graft-versus-host disease (GVHD) on day 335 after transplantation. The second patient developed severe gastro-intestinal GVHD and TTP and died on day 147 after transplantation. Conditioning with fludarabine/melphalan and ATG followed by allogeneic stem cell transplantation resulted in prompt and reliable engraftment in adults with end-sta...Continue Reading


Jun 9, 1994·The New England Journal of Medicine·O S PlattP P Klug
Dec 16, 1995·BMJ : British Medical Journal·A LeeG Serjeant
Aug 8, 1996·The New England Journal of Medicine·O S Platt, E C Guinan
Aug 8, 1996·The New England Journal of Medicine·Mark C WaltersK M Sullivan
Dec 1, 1996·American Journal of Kidney Diseases : the Official Journal of the National Kidney Foundation·S M WallT Madden
Aug 28, 1999·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·P A McSweeney, R Storb


Jun 24, 2003·Blood Reviews·Christiane Vermylen
May 2, 2002·Journal of Hematotherapy & Stem Cell Research·F Baron, Y Beguin
Feb 27, 2001·Current Opinion in Oncology·C C Hoppe, Mark C Walters
Oct 9, 2002·Journal of Pediatric Hematology/oncology·Robert IannoneJames F Casella
May 4, 2012·Cold Spring Harbor Perspectives in Medicine·Guido LucarelliJavid Gaziev
Oct 3, 2002·Annual Review of Medicine·Martin H Steinberg, Carlo Brugnara
Dec 20, 2007·Pediatric Hematology and Oncology·Lakshmanan Krishnamurti
Jan 26, 2007·Expert Opinion on Biological Therapy·Lakshmanan Krishnamurti
Dec 1, 2001·Expert Opinion on Biological Therapy·V Bhatia, D L Porter
Dec 4, 2003·Expert Opinion on Biological Therapy·Robert C Atkins, Mark C Walters
May 7, 2005·European Journal of Haematology·Jan T KielsteinJörg Radermacher
Mar 16, 2004·British Journal of Haematology·Franco Locatelli, Piero De Stefano
Mar 5, 2003·British Journal of Haematology·Persis J AmroliaSally C Davies
Mar 18, 2003·The Journal of Clinical Investigation·Christopher E D LammingCatherine M Verfaillie
Jun 4, 2015·Bone Marrow Transplantation·N Or-Geva, Y Reisner
Oct 6, 2016·Pediatric Research·Emily Riehm Meier, Angeli Rampersad
Aug 30, 2005·Blood Cells, Molecules & Diseases·Robert E RichardC Anthony Blau
Dec 11, 2007·Current Problems in Pediatric and Adolescent Health Care·Lakshmanan KrishnamurtiJakub Tolar
Dec 3, 2014·Hematology/oncology Clinics of North America·Courtney D FitzhughMatthew M Hsieh
Oct 2, 2013·Therapeutic Advances in Hematology·Shalini Shenoy
Dec 2, 2016·The Cochrane Database of Systematic Reviews·Vanitha A JagannathAkshay Sharma
Sep 25, 2004·Pediatric Blood & Cancer·Robert IannoneAllen R Chen
May 1, 2012·Pediatric Blood & Cancer·Franco Locatelli, Daria Pagliara
Oct 19, 2001·Bone Marrow Transplantation·Koen van BesienE Kodish
Oct 21, 2006·Current Opinion in Hematology·Claudia R MorrisMark C Walters
May 22, 2008·European Journal of Immunology·David PolchertAmelia Bartholomew
Jun 29, 2011·British Journal of Haematology·Jorge Mansilla-SotoMichel Sadelain
Jul 13, 2005·Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology·Koen van BesienW Stock
Nov 26, 2002·Hematology·Mark C WaltersElliott Vichinsky
May 20, 2016·The Cochrane Database of Systematic Reviews·Chioma OringanjeOluseyi Oniyangi
Jul 4, 2020·The Cochrane Database of Systematic Reviews·Chioma OringanjeOluseyi Oniyangi
Feb 2, 2013·European Journal of Haematology·Susanne Matthes-MartinMilen Minkov
May 8, 2018·Bone Marrow Transplantation·Hafiz Muhammad AslamShahrukh K Hashmi
Oct 5, 2019·Journal of Clinical Medicine·Santosh L Saraf, Damiano Rondelli
Sep 12, 2017·Expert Review of Hematology·Djamila L GhafuriDeva Sharma

Related Concepts

Anemia, Sickle Cell
Graft Survival
Graft-vs-Host Disease
Immunosuppressive Agents
Transplantation, Homologous
Fatal Outcome
Transplantation Chimera

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