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

Madelon C. Vonk ^1*, Zora Marjanovic ², Frank H.J. van den Hoogen ¹, Sarah Zohar ³, Anton V.M.B. Schattenberg ¹, Willem E. Fibbe ⁴, Jerome Larghero ⁵, Eliane Gluckman ⁵, Jacob M. van Laar ⁴ and Dominique Farge ⁵

¹ Radboud University Nijmegen Medical Centre, Netherlands
² Hôpital Hôtel-Dieu, Paris, France
³ Hôpital Saint-Louis, Paris, France
⁴ Leiden University Medical Centre, Netherlands
⁵ Hôpital Saint-Louis, Paris, Netherlands

^* To whom correspondence should be addressed. E-mail: m.vonk{at}reuma.umcn.nl.

Accepted 22 May 2007

Abstract

Systemic sclerosis (SSc) is a generalised autoimmune disease, causing morbidity and a reduced life expectancy, especially in patients with rapidly progressive diffuse cutaneous SSc. Since no proven treatment exists, autologous haematopoietic stem cell transplantation (HSCT) is employed as a new therapeutic strategy in patients with a poor prognosis. Objective: This study reports the effects on survival, skin and major organ function of HSCT in patients with severe diffuse cutaneous SSc. Patients and methods: Twenty-six patients were evaluated. Peripheral blood stem cells were collected using cyclophosphamide (4 g/m2) and rHu G-CSF (5 to 10 mg/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 seven years after transplantation.

Keywords: Autologous haematopoietic stem cell transplantation, modified Rodnan skin score, peripheral blood stem cells, systemic sclerosis

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