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 microg/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.
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 microg/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.
Authors: Vasiliki Kalliopi K Bournia; Panayiotis G Vlachoyiannopoulos; Carlo Selmi; Haralampos M Moutsopoulos; M Eric Gershwin Journal: Clin Rev Allergy Immunol Date: 2009-06 Impact factor: 8.667
Authors: J Baraut; E I Grigore; F Jean-Louis; S H Khelifa; C Durand; F Verrecchia; D Farge; L Michel Journal: Bone Marrow Transplant Date: 2013-12-23 Impact factor: 5.483
Authors: Keith M Sullivan; Navneet S Majhail; Christopher Bredeson; Paul A Carpenter; Soumya Chatterjee; Leslie J Crofford; George E Georges; Richard A Nash; Marcelo C Pasquini; Stefanie Sarantopoulos; Jan Storek; Bipin Savani; E William St Clair Journal: Biol Blood Marrow Transplant Date: 2018-06-25 Impact factor: 5.742
Authors: Dominique Farge; Myriam Labopin; Alan Tyndall; Athanasios Fassas; Gian Luigi Mancardi; Jaap Van Laar; Jian Ouyang; Tomas Kozak; John Moore; Ina Kötter; Virginie Chesnel; Alberto Marmont; Alois Gratwohl; Riccardo Saccardi Journal: Haematologica Date: 2009-09-22 Impact factor: 9.941