Haematologica (Jan 2020)

Long-term event-free survival, chimerism and fertility outcomes in 234 patients with sickle-cell anemia younger than 30 years after myeloablative conditioning and matched-sibling transplantation in France

  • Françoise Bernaudin,
  • Jean-Hugues Dalle,
  • Dominique Bories,
  • Regis Peffault de Latour,
  • Marie Robin,
  • Yves Bertrand,
  • Corinne Pondarre,
  • Jean-Pierre Vannier,
  • Benedicte Neven,
  • Mathieu Kuentz,
  • Sébastien Maury,
  • Patrick Lutz,
  • Catherine Paillard,
  • Karima Yakouben,
  • Isabelle Thuret,
  • Claire Galambrun,
  • Nathalie Dhedin,
  • Charlotte Jubert,
  • Pierre Rohrlich,
  • Jacques-Olivier Bay,
  • Felipe Suarez,
  • Nicole Raus,
  • Jean-Paul Vernant,
  • Eliane Gluckman,
  • Catherine Poirot,
  • Gérard Socié,
  • for the Société Française de Greffe de Moelle et de Thérapie Cellulaire

DOI
https://doi.org/10.3324/haematol.2018.213207
Journal volume & issue
Vol. 105, no. 1

Abstract

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Allogeneic stem cell transplantation remains the only curative treatment for sickle cell anemia (SCA), but the place of myeloablative conditioning in the procedure remains to be defined. The aim of the present study was to analyze long-term outcomes, including chimerism, SCA-related events and biological data (hemoglobin, reticulocytes, HbS%), and fertility in a French series of 234 SCA patients under 30 years of age who, from 1988 to 2012, received a matched-sibling-donor stem cell transplantation following standardized myeloablative conditioning [busulfan, cyclophosphamide and rabbit antithymocyte globulin (ATG)]. Since the first report of the series (1988-2004), 151 new consecutive patients with SCA have been similarly transplanted. Considering death, non-engraftment or rejection (donor cells 15 years (hazard ratio=4.37; P=0.002) and lower (5-15 vs. 20 mg/kg) ATG dose (hazard ratio=4.55; P=0.001). At one year, 44% of patients had mixed chimerism (5-95% donor cells), but those prepared with ATG had no graft rejection. No events related to SCA occurred in patients with mixed chimerism, even those with 15-20% donor cells, but hemolytic anemia stigmata were observed with donor cells