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Ann Rheum Dis 67:98-104 doi:10.1136/ard.2007.071464
  • Extended report

Long-term follow-up results after autologous haematopoietic stem cell transplantation for severe systemic sclerosis

  1. M C Vonk1,
  2. Z Marjanovic2,
  3. F H J van den Hoogen1,
  4. S Zohar3,
  5. A V M B Schattenberg4,
  6. W E Fibbe5,
  7. J Larghero6,
  8. E Gluckman7,
  9. F W M B Preijers4,
  10. A P J van Dijk10,
  11. J J Bax11,
  12. P Roblot12,
  13. P L C M van Riel1,
  14. J M van Laar8,
  15. D Farge9
  1. 1
    Department of Rheumatology, Radboud University Nijmegen Medical Centre, Nijmegen, The Netherlands
  2. 2
    Assistance Publique Hôpitaux de Paris, Hôpital Hôtel-Dieu, Département d’Hématologie, Paris, France
  3. 3
    Assistance Publique Hôpitaux de Paris, Hôpital Saint-Louis, Département d’Informatique Médicale et Biostatistiques, Paris, France
  4. 4
    Department of Hematology, Radboud University Nijmegen Medical Centre, Nijmegen, The Netherlands
  5. 5
    Department of Hematology, Leiden University Medical Centre, Leiden, The Netherlands
  6. 6
    Assistance Publique Hôpitaux de Paris, Hôpital Saint-Louis, Cell therapy Unit, Paris, France
  7. 7
    Assistance Publique Hôpitaux de Paris, Hôpital Saint-Louis, Service de Greffe de Moelle, Paris, France
  8. 8
    Department of Rheumatology, Leiden University Medical Centre, Leiden, The Netherlands
  9. 9
    Assistance Publique Hôpitaux de Paris, Hôpital Saint-Louis, Department of Internal Medicine and INSERM U 697 Paris, University Paris 7 Denis Diderot, France
  10. 10
    Heart-Lung Centre, Department of Cardiology, Radboud University Nijmegen Medical Centre, Nijmegen, The Netherlands
  11. 11
    Department of Cardiology, Leiden University Medical Centre, Leiden, The Netherlands
  12. 12
    Département de Médecine Interne, Hopital Universitaire La Miletrie, Poitiers, France
  1. Madelon C Vonk, Department of Rheumatology, Radboud University Nijmegen Medical Centre, P.O.Box 9101, 6500 HB Nijmegen, The Netherlands; M.Vonk{at}reuma.umcn.nl; Dominique Farge, Service de Médecine Interne et Unité INSERM U667, Hôpital Saint-Louis, 1 avenue Claude Vellefaux, 75 010 Paris France; dominique.farge-bancel{at}sls.ap-hop-paris.fr
  • Accepted 22 May 2007
  • Published Online First 25 May 2007

Abstract

Objective: Systemic sclerosis (SSc) is a generalised autoimmune disease, causing morbidity and a reduced life expectancy, especially in patients with rapidly progressive diffuse cutaneous SSc. As no proven treatment exists, autologous haematopoietic stem cell transplantation (HSCT) is employed as a new therapeutic strategy in patients with a poor prognosis. This study reports the effects on survival, skin and major organ function of HSCT in patients with severe diffuse cutaneous SSc.

Patients and methods: A total of 26 patients were evaluated. Peripheral blood stem cells were collected using cyclophosphamide (4 g/m2) and rHu G-CSF (5 to 10 μg/kg/day) and were reinfused after positive CD34+ selection. For conditioning, cyclophosphamide 200 mg/kg was used.

Results: After a median follow-up of 5.3 (1–7.5) years, 81% (n = 21/26) of the patients demonstrated a clinically beneficial response. The Kaplan–Meier estimated survival at 5 years was 96.2% (95% CI 89–100%) and at 7 years 84.8% (95% CI 70.2–100%) and event-free survival, defined as survival without mortality, relapse or progression of SSc, resulting in major organ dysfunction was 64.3% (95% CI 47.9–86%) at 5 years and 57.1% (95% CI 39.3–83%) at 7 years.

Conclusion: This study confirms that autologous HSCT in selected patients with severe diffuse cutaneous SSc results in sustained improvement of skin thickening and stabilisation of organ function up to 7 years after transplantation.

Footnotes

  • Funding: Supported by grants from: Délégation Régionale è la Recherche Clinique (DRRC), Assistance Publique- Hôpitaux de Paris (AP-HP); the French Ministry of Health (Programme Hospitalier de Recherche Clinique: PHRC 1997 AOM 97-030); the Etablissement Français des Greffes (2003); the Groupe Français de Recherche sur la Sclérodermie (GFRS); the Association Française contre la Sclérodermie; and the Dutch Arthritis Foundation, Amsterdam, The Netherlands.

  • Competing interests: None declared