Summary:
To determine the results of allogeneic hematopoietic stem cell (HSC) transplantation for chronic myelogenous leukemia (CML) at various stages of the disease in children, a retrospective analysis was carried out on the outcome of transplants performed on 76 children and teenagers with CML between 1982 and 1998. In all, 60 patients were transplanted from a matched sibling donor (MSD) and 16 from a matched unrelated donor (MUD). There was a higher incidence of acute graft-versus-host disease after MUD transplantation (P<10−3). The main cause of death was transplant-related toxicity in both groups. In MSD recipients, the probability of relapse at 5 years for patients transplanted in the first chronic phase was lower than in patients transplanted in the advanced phase (relative risk (rr)=5.90; 95% confidence interval (CI), 1.85–18.82, P<0.01). The estimated 5-year event-free survival (EFS) rate was higher after MSD vs MUD transplantation (61% (95% CI, 48–73%) vs 27% (95% CI, 4–49%), rr=0.25, P<10−3). In children transplanted from MSD, the 5-year EFS was higher when transplantation was performed in the first chronic phase vs the advanced phases (73% (95% CI, 59–87%) vs 32% (95% CI, 10–54%), P<10−3). Disease status at transplantation was the unique factor influencing survival in patients undergoing transplantation from MSD with a better outcome for those transplanted in the first chronic phase. Allogeneic HSC offers a possibility of curing childhood CML with a significant advantage for patients transplanted in chronic phase using a human leukocyte antigen-identical sibling donor.
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Appendix 1
Appendix 1
Participating centers and center directors:
Hopital Saint Louis, Paris (Pr Gluckmann, Dr Esperou); Hopital Pellegrin, Pessac (Pr Reiffers); Centre Hospitalier Universitaire, Grenoble (Dr Garban); Hopital Intercommunal, Créteil (Dr Kuentz); Institut Paoli Calmette, Marseille (Pr Blaise); Centre Hospitalier Universitaire, Besançon (Pr Cahn, Dr Plouvier); Hopital Edouard Herriot, Lyon (Pr Michallet); Hopital d'enfants, Vandoeuvre (Pr Bordigoni); Centre Hospitalier Universitaire, Nantes (Pr Milpied); Institut Gustave Roussy, Villejuif (Dr Bourhis); Hopital Necker, Paris (Pr Fisher); Hopital Robert Debré, Paris (Pr Vilmer); Hopital La Timone, Marseille (Pr Michel); Centre Hospitalier Universitaire, Dijon (Pr Caillot); Hopital Charle Nicolle, Rouen (Pr Vannier); Hopital Jean Bernard, Poitiers (Pr Guilhot); Hopital Calmette, Lille (Pr Jouet); Centre Hospitalier Universitaire, Clermont Ferrand (Pr Demeocq); CHU Strabourg (Dr Lioure); Centre Hospitalier Universitaire, Saint-Etienne (Pr Guyotat); Centre Hospitalier Universitaire, Toulouse (Pr Attal, Dr Rubie); Hopital Pitié Salpétrière, Paris (Pr Vernant).
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Millot, F., Esperou, H., Bordigoni, P. et al. Allogeneic bone marrow transplantation for chronic myeloid leukemia in childhood: a report from the Société Française de Greffe de Moelle et de Thérapie Cellulaire (SFGM-TC). Bone Marrow Transplant 32, 993–999 (2003). https://doi.org/10.1038/sj.bmt.1704255
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DOI: https://doi.org/10.1038/sj.bmt.1704255
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