AB0914-PARE INFORMATION PREFERENCES ABOUT TREATMENT OPTIONS IN DIFFUSE CUTANEOUS SYSTEMIC SCLEROSIS: A DELPHI CONSENSUS STUDY
Authors: J. Spierings, H. Nienhuis, E. Van Lieshout, J. M. Van Laar, A. Pieterse
Journal: Annals of the Rheumatic Diseases (2021)

Background: Diffuse cutaneous systemic sclerosis (dcSSc) has a high impact on quality of life and is associated with increased mortality. Several treatment options are available in dcSSc, including immunosuppressives, chemotherapy and hematopoietic stem cell transplantation. Shared decision making is key in choosing the appropriate treatment strategy. In earlier studies however, patients reported that the lack of accessible and reliable patient information about treatment options complicates decision making. In order to provide information that meets the needs of patients and addresses relevant benefits and harms, more insight in the preferences of patients is needed. Objectives: The aim of this study was to identify and prioritize aspects that should be addressed when informing patients with dcSSc about treatment options, from a patient’s perspective. Methods: A three-round Delphi study was conducted with a panel of patients with dcSSc. The first questionnaire was based on a systematic literature search regarding benefits and harms of the four main treatments in dcSSc: methotrexate, mycophenolate mofetil, cyclophosphamide, and stem cell transplantation. In the first round, patients were asked to indicate which information on the benefits and harms is essential for making a treatment decision; they could also add new items. In the second and third rounds, patients were asked to prioritize the items. After the third round, a live, online panel discussion was held in order to reach consensus. Consensus was defined as ≥75% agreement among panel members. Results: Of the 36 patients invited, 67% (n=24) completed the first, 69% (n=25) the second and 75% (n=27) the third round. In the third round, median age of participants was 51 years (IQR 18), median disease duration was 4 years (IQR 5), 52% was female. Most patients had completed either an intermediate education level (59%) or high level of education (30%). Patients were or had been treated with mycophenolate mofetil (67%), methotrexate (44%), cyclophosphamide (41%), autologous stem cell transplantation (26%), rituximab (4%). Seven percent of patients were treatment-naïve. After the third round, eight patients joined the online panel discussion to reach final consensus. The panel reached consensus on seven benefits (prolonged progression-free survival, improved quality of life, improved daily functioning, improved pulmonary function, improved skin thickness, improved mobility and reduced fatigue) and four adverse events (treatment-related mortality, viral infections, cardiac damage, increased risk of cancer) as key themes for the decision making process (Table 1). Conclusion: This study identified essential aspects of the four main treatment options for dcSSc. Our results can be used to develop effective patient information. Table 1. Agreement on benefits and harms that should be addressed Benefits Agreement Harms Agreement Improved pulmonary function 100% Viral infections 88% Improved skin thickness 100% Cardiac damage 88% Improved mobility 100% Increased risk of cancer 88% Prolonged progression-free survival 95%* Treatment-related mortality 79%* Improved quality of life 77%* Flare or relapse of disease 63% Improved daily functioning 77%* Temporary increase in fatigue 25% Reduced fatigue 75% Temporary negative impact on social activities 25% Improved hand function 25% Nausea 25% Improved mood 25% Other autoimmune disease 13% Improved cardiac function 13% Hormonal imbalance 13% Improved oesophageal function 7% Concentration difficulty 13% Diarrhea 13% Erectile dysfunction 13% Depression 13% Infertility 13% Hair loss 13% * Items on which consensus was reached in round 3 Disclosure of Interests: None declared