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Autologous stem cell transplantation in the treatment of systemic sclerosis: report from the EBMT/EULAR Registry
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  1. D Farge18,
  2. J Passweg11,
  3. J M van Laar1,
  4. Z Marjanovic18,
  5. C Besenthal2,
  6. J Finke2,
  7. H H Peter2,
  8. F C Breedveld1,
  9. W E Fibbe1,
  10. C Black14,
  11. C Denton14,
  12. I Koetter19,
  13. F Locatelli15,
  14. A Martini10,
  15. A V N Schattenberg22,
  16. F van den Hoogen22,
  17. L van de Putte22,
  18. F Lanza17,
  19. R Arnold4,
  20. P A Bacon21,
  21. S Bingham20,
  22. F Ciceri16,
  23. B Didier9,
  24. J L Diez-Martin7,
  25. P Emery20,
  26. W Feremans5,
  27. B Hertenstein12,
  28. F Hiepe4,
  29. R Luosujärvi23,
  30. A Leon Lara6,
  31. A Marmont13,
  32. A M Martinez8,
  33. H Pascual Cascon3,
  34. C Bocelli-Tyndall11,
  35. E Gluckman18,
  36. A Gratwohl11,
  37. A Tyndall11
  1. 1Academish Ziekenhuis, Leiden, The Netherlands
  2. 2Albert Ludwig Universität, Freiburg, Germany
  3. 3Carlos Haya Hospital, Malaga, Spain
  4. 4Charité Hospital, Berlin, Germany
  5. 5Hôpital Erasme, Brussels, Belgium
  6. 6Hospital del SAS, Cadiz Jerez de la Frontera, Spain
  7. 7Hospital G.U. Gregorio Maranon, Madrid, Spain
  8. 8Hospital Infantil La Paz, Madrid, Spain
  9. 9Institut Paoli Calmettes, Marseille, France
  10. 10IRCCS G. Gaslini, Genova, Italy
  11. 11Kantonsspital Basel, Switzerland
  12. 12Medizinische Hochschule, Hannover, Germany
  13. 13Ospedale San Martino, Genova, Italy
  14. 14Royal Free Hospital, London, UK
  15. 15San Matteo University Hospital, Pavia, Italy
  16. 16San Raffaele Hospital, Milano, Italy
  17. 17St Anna University Hospital, Ferrara, Italy
  18. 18St Louis Hospital, Paris, France
  19. 19Universitätsklinikum Tübingen, Germany
  20. 20University Hospital, Leeds, UK
  21. 21University Hospital, Birmingham, UK
  22. 22University Hospital, Nijmegen, The Netherlands
  23. 23University Hospital, Kuopio, Finland
  1. Correspondence to:
    Professor D Farge
    Service de Médecine Interne, hôpital Saint-Louis, 1 avenue Claude Vellefaux 75010 Paris, France; dominique.farge-bancelchu-stlouis.fr

Abstract

Objective: To analyse the durability of the responses after haematopoietic stem cell transplantation (HSCT) for severe systemic sclerosis (SSc) and determine whether the high transplant related mortality (TRM) improved with experience. This EBMT/EULAR report describes the longer outcome of patients originally described in addition to newly recruited cases.

Methods: Only patients with SSc, treated by HSCT in European phase I–II studies from 1996 up to 2002, with more than 6 months of follow up were included. Transplant regimens were according to the international consensus statements. Repeated evaluations analysed complete, partial, or non-response and the probability of disease progression and survival after HSCT (Kaplan-Meier).

Results: Given as median (range). Among 57 patients aged 40 (9.1–68.7) years the skin scores improved at 6 (n = 37 patients), 12 (n = 30), 24 (n = 19), and 36 (n = 10) months after HSCT (p<0.005). After 22.9 (4.5–81.1) months, partial (n = 32) or complete response (n = 14) was seen in 92% and non-response in 8% (n = 4) of 50 observed cases. 35% of the patients with initial partial (n = 13/32) or complete response (n = 3/14) relapsed within 10 (2.2–48.7) months after HSCT. The TRM was 8.7% (n = 5/57). Deaths related to progression accounted for 14% (n = 8/57) of the 23% (n = 13/57) total mortality rate. At 5 years, progression probability was 48% (95% CI 28 to 68) and the projected survival was 72% (95% CI 59 to 75).

Conclusion: This EBMT/EULAR report showed that response in two thirds of the patients after HSCT was durable with an acceptable TRM. Based on these results prospective, randomised trials are proceeding.

  • AD, autoimmune disease
  • ATG, antithymocyte globulins
  • EBMT, European Group for Blood and Marrow Transplantation
  • G-CSF, granulocyte-colony stimulating factor
  • HSCT, haematopoietic stem cell transplantation
  • LVEF, left ventricular ejection fraction
  • PAP, pulmonary artery pressure
  • PBSC, peripheral blood stem cells
  • SSc, systemic sclerosis
  • TBI, total body irradiation
  • Tlco, carbon monoxide transfer factor
  • TLI, total lymphoid irradiation
  • TRM, transplant related mortality
  • VC, vital capacity
  • systemic sclerosis
  • autoimmune diseases
  • autologous bone marrow transplantation
  • haematopoietic stem cell transplantation
  • Rodnan skin score

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