Autologous hematopoietic stem cell transplantation improves long term survival – data from a national registry

Background: Current recommendations on the management of systemic sclerosis (SSc) suggest that autologous hematopoietic stem cell therapy (HSCT) can be a rescue therapy for patients with rapidly progressive SSc. Objectives: To assess safety and efficacy of HSCT for patients with SSc and to compare these with non-HSCT patient in a control cohort with adjusted risk factors. Methods: A retrospective analysis of data from the multicentric German network for systemic scleroderma (DNSS) with 5000 patients with SSc. Control groups consisted of all patients with diffuse cutaneous (dc)-SSc (group A) and an adjusted high risk cohort of male patients with Scl70-positive dc-SSc (group B). Results: Eighty SSc patients received an HSCT 4.1 ± 4.8 years after SSc diagnosis. Among them 86.3% had dc-SSc, 43.5% were males, and 71.3% were positive for Scl70 antibodies. The control group A showed a significant underrepresentation of these risk factors for mortality. When the survival of the control group B was compared with the HSCT group, a lower mortality of the latter was observed instead. Within 5 years after HSCT, we observed an improvement of the mRSS from 17.6 ± 11.5 to 11.0 ± 8.5, and an improvement of the DLCO from 54.6 ± 17.8% to 60.0 ± 18.5%. We did not see differences in transplant-related mortality between patients who received HSCT within 3 years after SSc diagnosis or later. Conclusion: Our analysis of real life data show that the distribution of risk factors for mortality is critical when HSCT cohorts are compared with non-HSCT control groups.