Background Autologous haematopoietic stem cell transplantation (HSCT) improves survival in systemic sclerosis (SSc) with poor prognosis, but is hampered by treatment-related mortality (TRM).
Objective To evaluate event-free survival (EFS), TRM, response to treatment, disease progression and patient characteristics associated with events.
Methods All patients treated with HSCT for SSc in The Netherlands until 2017 (n=92) were included. Data on skin involvement (modified Rodnan skin score (mRSS), pulmonary function (forced vital capacity (FVC) and diffusion capacity of the lungs for carbon monoxide (DLCO)), extent of interstitial lung disease on high-resolution CT using Goh scores and left ventricular ejection fraction (LVEF) were collected at baseline, 1, 2 and 5 years. Occurrence of events, defined as death or major organ failure, were collected until 2019. As control, a comparison between patients treated with cyclophosphamide (CYC) and patients with HSCT who participated in the Autologous Stem Cell Transplantation International Scleroderma (ASTIS) trial was performed.
Results Median follow-up was 4.6 years. EFS estimates at 5, 10 and 15 years were 78%, 76% and 66%, respectively. Twenty deaths occurred. Mean FVC, DLCO, mRSS and Goh scores all improved significantly. Disease progression occurred in 22 patients. Frequency of TRM decreased over time and occurred more often in males. Events were independently associated with male sex, LVEF <50% and older age. In ASTIS, patients treated with HSCT (n=23) 7 events occurred versus 13 in the CYC group (n=22).
Conclusion Our data confirm long-term efficacy of HSCT in improving survival, skin and lung involvement in SSc. Male sex, lower LVEF and older age at baseline were identified as risk factors for events.
- systemic sclerosis
- autoimmune diseases
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Handling editor Josef S Smolen
Contributors SvB and JKdV-B contributed equally and share first authorship. All authors contributed to drafting and reviewing the manuscript and approved the final version for publication. CE and MV had access to all data and analyses. Study conception and design: MV, SvB, CE, JKdV-B and MB. Acquisition of data, analysis and interpretation of data: all authors.
Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.
Competing interests None declared.
Patient and public involvement Patients and/or the public were not involved in the design, or conduct, or reporting, or dissemination plans of this research.
Patient consent for publication Not required.
Ethics approval The current study has been approved by the medical ethics committee CMO region Arnhem-Nijmegen, Nijmegen, the Netherlands NL-2016-2915. Due to the retrospective nature of this study, informed consent was not deemed required. Informed consent was obtained from all alive patients not lost to follow-up.
Provenance and peer review Not commissioned; externally peer reviewed.
Data availability statement Data are available upon reasonable request. All data are available on request to Investigators whose proposed use of the data has been approved by an independent review committee identified for this purpose and for meta-analysis, as well as the study protocol and analysis plan.