Abstract
Allogeneic hematopoietic SCT (HSCT) has been used as treatment for single patients with autoimmune diseases (AD). To summarise currently available information, we analyzed all patients who underwent allogeneic HSCT for AD and who reported to the European Group for Blood and Marrow Transplantation (EBMT) database. Thirty-five patients receiving 38 allogeneic transplantations for various hematological and non-hematological AD were identified. Four patients had had an allogeneic HSCT for a conventional hematological indication in the past. Fifty-five per cent of the transplantation procedures led to a complete clinical response of the refractory AD and 23% to at least a partial response. The median duration of response at the last follow-up was 70.7 (15.2–130) months. Three patients relapsed at a median of 12.3 months after HSCT. Treatment-related mortality at 2 years was 22.1% (95% CI: 7.3–36.9%). Two deaths were caused by progression of AD. The probability of survival at 2 years was 70%. No single factor predicting the outcome could be identified. The retrospective nature of this study and the heterogeneous, partly incomplete data are its limitations. However, allogeneic HSCT can induce remission in patients suffering from refractory AD. These data provide the basis for carefully conducted prospective trials.
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Acknowledgements
We thank Dr Martin Stern for help with statistics and for discussion of the paper. The cooperation of all participating teams and their staff (listed in the Appendix 1), the EBMT Co-ordination office; Barcelona (F McDonald, E McGrath, SM Jones and EJ Mac Hale), Paris (V Chesnel, C Kenzey, C Durand and NC Gorin), London (C Ruiz de Elvira, S Hewerdine, S de Souza and N Fortin-Robertson), the Austrian Registry (H Greinix, B Lindner), the Czech Registry (K Benesova, M Trnkova), the French Registry SFGM (D Blaise, C Raffoux and Z Chir), the German Registry (H Ottinger, K Fuchs, C Müller, S Allgaier and A Müller), the Italian Registry (A Bacigalupo, R Oneto and B Bruno), the Dutch Registry (A Schattenberg, A v Biezen, M Sneets and R Brand), the Spanish Registry (J Rifon, A Cedillo, and J López), the Swiss Registry (U Schanz, H Baldomero and E Buhrfeind), the Turkish Registry (G Gurman, M Arat, F Arpaci and M Ertem) and the British Registry (C Craddock, J Cornish, K Towlson and M Wilson) is greatly appreciated. This study was supported in part by the European Leukemia Net LSH-2002-2.2.0-3, by a grant from the Swiss National Research Foundation, 3200B0-118176, the Swiss Cancer League, the Regional Cancer League and the Horton Foundation. EBMT is supported by grants from the corporate members: Amgen Europe GmbH, F Hoffmann-La Roche Ltd, Gilead Sciences UK, Miltenyl Biotec GmbH, Schering-Plough International Inc., Celegene International SARL, Genzyme, ViroPharma Europe, Chugai Sanofi-Aventis SNC, Fresenius Biotech GmbH, Gambro BCT, Bayer Schering Pharma AG, Therakos, Bristol Myers Squibb, Novartis, Pharmacon, Cephalon, Pierre Fabre Médicament, GE Healthcare, Alexion Europe, Pfizer, Biosafe SA, Merck Sharp and Dohme.
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Appendix 1
Appendix 1
Additionally contributing EBMT centers
Hôpital Necker, Unité d'Immunologie et d'Hématologie and INSERM U 29, Paris, France.
Leiden University Hospital, BMT Centre Leiden, Leiden, The Netherlands.
University Medical Centre, Department of Haematology, Utrecht, The Netherlands.
Hotel Dieu, Department de Hematologie, Nantes, France.
Division of Stem Cell Transplantation and Immunotherapy, University Hospital Schleswig-Holstein, Kiel, Germany.
Hadassah University Hospital, Department of Bone Marrow Transplantation, Jerusalem, Israel.
Clinica di Oncoematologia Pediatrica, Dipartimento di Pediatria, Padova, Italy.
Charité-CVK, University Medicine Berlin, Pediatric Bone Marrow Transplant Service, Berlin, Germany.
Klinik/Poliklinik für Kinderheilkunde, Pädiatrische Hämatologie/Onkologie, Münster, Germany.
Unit of Hematology and Bone Marrow Transplantation, San Giovanni Rotondo, Italy.
Postgraduate Medical School, Department of Pediatrics/BMT Unit, Miskolc, Hungary.
CHRU, Service des Maladies du Sang, Angers, France.
University Hospital Gent, Haematology and Bloodbank, Gent, Belgium.
University Hospital of Palermo, Department of Hematology and Bone Marrow Transplantation, Palermo, Italy.
St László Hospital, Bone Marrow Transplantation Unit, Budapest, Hungary.
IRCCS Policlinico San Matteo, Pediatric Hematology–Oncology, Pavia, Italy.
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Daikeler, T., Hügle, T., Farge, D. et al. Allogeneic hematopoietic SCT for patients with autoimmune diseases. Bone Marrow Transplant 44, 27–33 (2009). https://doi.org/10.1038/bmt.2008.424
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DOI: https://doi.org/10.1038/bmt.2008.424
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