Systemic Sclerosis: Autologous Hematopoietic Stem Cell Transplantation vs IV Pulse Cyclophosphamide

MedicalResearch.com Interview with:
ProfProf. Dr. Jacob M. van Laar Professor and Chair Dept of Rheumatology & Clinical Immunology University Medical Center Utrecht . Dr. Jacob M. van Laar
Professor and Chair
Dept of Rheumatology & Clinical Immunology
University Medical Center Utrecht

MedicalResearch: What are the main findings of the study?

Prof. van Laar: The results of the ASTIS-trial demonstrate that stem cell transplantation in selected patients with early, diffuse cutaneous systemic sclerosis, a rare, autoimmune connective tissue disease, prolongs long-term survival and improves clinical manifestations (skin, lung) and quality of life, when compared to monthly infusions with cyclophosphamide. The benefits must be weighed against the risks which include early treatment-related mortality (10% in the ASTIS-trial) and viral infections.

MedicalResearch: Were any of the findings unexpected?

Prof. van Laar: The beneficial effect of stem cell transplantation on survival was particularly evident in non-smoking patients. Also, stem cell transplantation resulted in a modest decline in kidney function which appeared transient. These unexpected findings merit further study.
MedicalResearch: What should clinicians and patients take away from your report?

Prof. van Laar: Stem cell transplantation may be considered a treatment option for a subgroup of systemic sclerosis patients with recent disease onset and clinical features of severe disease or rapidly progressive diseases with poor prognosis.

MedicalResearch: What recommendations do you have for future research as a result of this study?

Prof. van Laar: Further studies are needed to determine how to optimize patient selection and modification of the protocol to reduce the acute toxicity of the conditioning regimen without losing efficacy. In addition, further analysis of clinical and laboratory parameters of the ASTIS trial data may help identify factors which earlier predict non-responsiveness in the control arm.

Citation:

van Laar JM, Farge D, Sont JK, et al. Autologous Hematopoietic Stem Cell Transplantation vs Intravenous Pulse Cyclophosphamide in Diffuse Cutaneous Systemic Sclerosis: A Randomized Clinical Trial. JAMA. 2014;311(24):2490-2498. doi:10.1001/jama.2014.6368.