To improve patient outcomes in systemic sclerosis (scleroderma), optimization of hematopoietic stem cell therapy is necessary, and will be investigated in the UPSIDE study. This was concluded by Julia Spierings who defended her PhD thesis on October 12 in Utrecht.
Despite improvements in health care and better understanding of pathophysiological processes, patients with systemic sclerosis (SSc) have an increased mortality and high morbidity. Management of SSc can be challenging due to its heterogeneous nature, multi-organ involvement and limited therapeutic options. In this thesis Julia Spierings (Department of Rheumatology and Clinical Immunology, UMC Utrecht) zoomed in on the organization of health care and autologous hematopoietic stem cell transplantation (SCT) as possible goals to improve outcomes.
Research in 650 Dutch SSc patients showed that they are generally satisfied with their care. The most important quality indicators according to patients are good interaction between patient and doctor and structural multidisciplinary collaboration. A multidisciplinary study among doctors, nurses and paramedics was performed to identify areas for improvement. Recommendations were formulated in five domains (shared care and multidisciplinary collaboration, exchange of medical data, education, patient empowerment and non-drug care).
SCT is increasingly used in patients with rapidly progressive diffuse disease. SCT leads to greatly improved long-term outcomes, however, treatment-related mortality is relatively high. In a qualitative study in which Spierings studied the decision-making process for SCT or alternative treatment, it emerged that many factors influence patients’ decision making. It also emerged that patients are strongly guided by their doctor in their choice of treatment. Patients indicated that much depends on the choice and that they trust their doctor, but that there is also insufficient clear information available that provides a good overview of the various treatment options. A study of the experiences of patients during and after autologous stem cell transplantation shows that SCT has a major physical and psychosocial impact and that post-treatment counseling can be more proactive.
Further research into the optimal use of autologous SCT and subsequent management is necessary to improve outcomes. The international UPSIDE (Upfront autologous hematopoietic Stem cell transplantation versus Immunosuppressive medication in early DiffusE cutaneous systemic sclerosis) study will provide more information about optimal timing of SCT.
Julia Spierings (1985, Breda) defended her PhD thesis on October 12, 2020 at Utrecht University. The title of her thesis was “Optimizing outcomes of patients with systemic sclerosis”. Supervisor was prof. dr. Jaap van Laar (Department of Rheumatology and Clinical Immunology, UMC Utrecht) and co-supervisor was dr. Madelon Vonk (Department of Rheumatic Diseases, Radboudumc, Nijmegen). As of July 2020 Julia Spierings works as a honorary clinical research fellow and the Royal Free Hospital-University College London, UK.
