Abstract
BackgroundSystemic sclerosis (SSc) patients often need immunosuppressive medication (IS) for disease control. If SSc is progressive despite IS, autologous hematopoietic stem cell transplantation (aHSCT) is a treatment option for selected SSc patients. aHSCT is effective with good available evidence, but not all patients achieve a treatment-free remission after aHSCT. Thus far, data about the need of IS after aHSCT in SSc is not published. The aim of this study was to investigate the use of IS after aHSCT, its efficacy, and the occurrence of severe adverse events (SAEs).MethodsTwenty-seven patients with SSc who had undergone aHSCT were included in this single-center retrospective cohort study. Clinical data, including IS, SAEs, and lung function data, were collected.ResultsSixteen of 27 (59.3%) patients received IS after aHSCT. Methotrexate, rituximab, mycophenolate, cyclophosphamide, and hydroxychloroquine were most commonly used. The main reason for starting IS was SSc progress. Nine patients received rituximab after aHSCT and showed an improvement in modified Rodnan skin score and a stabilization of lung function 2 years after rituximab. SAEs in patients with IS after aHSCT (50.0%) were not more common than in patients without IS (54.6%). SAEs were mostly due to SSc progress, secondary autoimmune diseases, or infections. Two deaths after aHSCT were transplantation related and three during long-term follow-up due to pulmonary arterial hypertension.ConclusionDisease progression and secondary autoimmune diseases may necessitate IS after aHSCT in SSc. Rituximab seems to be an efficacious treatment option in this setting. Long-term data on the safety of aHSCT is reassuring.
Highlights
Systemic sclerosis (SSc) patients often need immunosuppressive medication (IS) for disease control
48.1% were female, with a median age of 47.2 years and a median disease duration of 25.0 months before Autologous hematopoietic stem cell transplantation (aHSCT); 93.3% were positive for anti-nuclear antibodies (ANA), and 74.1% were positive for Scl-70 antibodies; 88.9% had a diffuse cutaneous form with a mean modified Rodnan skin score of 23.0; and 37.0% had ever smoked
Cardiac involvement was present in 44.4%; 25.9% had a history of pulmonary arterial hypertension (PAH), which was well controlled at the beginning of mobilization
Summary
Systemic sclerosis (SSc) patients often need immunosuppressive medication (IS) for disease control. Autologous hematopoietic stem cell transplantation (aHSCT) has the best evidence for effective treatment of SSc with the three randomized controlled trials ASSIST [11], ASTIS [12], and SCOT [13]. These showed that aHSCT is superior to intravenous cyclophosphamide regarding skin and lung involvement, quality of life, and overall survival. No data or guidelines are published about the need and use of IS after aHSCT in SSc. the aim of the present study was to describe the indication for IS and the prevalence of the use of IS after aHSCT in SSc and to record the occurrence of severe adverse events (SAEs)
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