Long-term follow-up results after autologous haematopoietic stem cell transplantation for severe systemic sclerosis
- M C Vonk1,
- Z Marjanovic2,
- F H J van den Hoogen1,
- S Zohar3,
- A V M B Schattenberg4,
- W E Fibbe5,
- J Larghero6,
- E Gluckman7,
- F W M B Preijers4,
- A P J van Dijk10,
- J J Bax11,
- P Roblot12,
- P L C M van Riel1,
- J M van Laar8,
- D Farge9
- 1 Department of Rheumatology, Radboud University Nijmegen Medical Centre, Nijmegen, The Netherlands
- 2 Assistance Publique Hôpitaux de Paris, Hôpital Hôtel-Dieu, Département d’Hématologie, Paris, France
- 3 Assistance Publique Hôpitaux de Paris, Hôpital Saint-Louis, Département d’Informatique Médicale et Biostatistiques, Paris, France
- 4 Department of Hematology, Radboud University Nijmegen Medical Centre, Nijmegen, The Netherlands
- 5 Department of Hematology, Leiden University Medical Centre, Leiden, The Netherlands
- 6 Assistance Publique Hôpitaux de Paris, Hôpital Saint-Louis, Cell therapy Unit, Paris, France
- 7 Assistance Publique Hôpitaux de Paris, Hôpital Saint-Louis, Service de Greffe de Moelle, Paris, France
- 8 Department of Rheumatology, Leiden University Medical Centre, Leiden, The Netherlands
- 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 Heart-Lung Centre, Department of Cardiology, Radboud University Nijmegen Medical Centre, Nijmegen, The Netherlands
- 11 Department of Cardiology, Leiden University Medical Centre, Leiden, The Netherlands
- 12 Département de Médecine Interne, Hopital Universitaire La Miletrie, Poitiers, France
- Madelon C Vonk, Department of Rheumatology, Radboud University Nijmegen Medical Centre, P.O.Box 9101, 6500 HB Nijmegen, The Netherlands; ; Dominique Farge, Service de Médecine Interne et Unité INSERM U667, Hôpital Saint-Louis, 1 avenue Claude Vellefaux, 75 010 Paris France;
- Accepted 22 May 2007
- Published Online First 25 May 2007
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.
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