Elicitation of Patient Preferences in Scleroderma May Inform Future RCT Design and Enrollment

Elderly Man Talking To Doctor About Test Results
Elderly Man Talking To Doctor About Test Results
Researchers explored the impact of elicitation of patient preferences in informing design, enrollment, and modifiable aspects of treatment in future randomized controlled trials.

Using a patient-engaged approach to preference elicitation for different features of autologous hematopoietic stem cell transplant (AHSCT), predictors of decisions among patients with scleroderma to participate in randomized controlled trials (RCTs) can be determined, according to findings from a survey published in Trials.

The researchers sought to evaluate the effect of elicitation of patient preferences on RCT design and how changing modifiable features of treatments or trial design can impact study enrollment.

An online discrete choice experiment (DCE) survey and focus groups were used to collect information about preferences pf patients with scleroderma for AHSCT treatment interventions. The survey included 4 main sections: demographic information, AHSCT treatment information, the DCE component, and questions about patient health. Study participants were asked to select “AHSCT treatment A,” “AHSCT treatment B,” or a fixed “no AHSCT treatment” alternative.

A mixed-logit model was used to estimate preferences for 7 different attributes of treatment — effectiveness, immediate risk, long-term risk, composition of care team, experience of care team, cost, and travel distance — and predict patient participation in RCTs.

The “Scleroderma: Cyclophosphamide or Transplantation (SCOT)” trial is an example of a published study that evaluated AHSCT in patients with scleroderma and experienced difficulty with patient recruitment.

A total of 278 patients with scleroderma responded to the survey. The majority of the respondents identified as women (88%); were aged 40 years and older (90%); were White (74%); and resided in Canada (45%), the US (28%), or France (17%). Overall, 54% of the cohort had diffuse scleroderma, the main diagnosis for which AHSCT is currently indicated; 44% had limited scleroderma; and 3% reported having other types of scleroderma. Disease duration among the participants was between 0 and 54 years (mean, 13.9±9.9 years); 51% of the respondents reported that their age at diagnosis was between 40 and 59 years. Disease duration was significantly longer among those with limited scleroderma compared with those with diffuse scleroderma (16.0 vs 12.3 years, respectively; P =.002).

Results of the study showed that all AHSCT treatment attributes significantly affected patient preferences. Effectiveness of treatment and risk for development of late complications were associated with the highest contributions to patients’ choices; modifiable factors of distance to treatment center and cost also affected patients’ preferences.

Predicted enrollment rates calibrated with those from the recent SCOT trial (33%), suggesting that offering a treatment that is closer in distance to home, at a lower cost to the patient, and with holistic, multidisciplinary care might increase patient participation to 51%.

The researchers concluded, “Knowledge regarding concerns and the trade-offs people are willing to make can inform clinical study design, improving recruitment rates and potential uptake of the treatment of interest.”

Disclosure: Some of the study authors have declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of authors’ disclosures. 


Aguiar M, Laba T-L. Munro S, et al. Co-production of randomized clinical trials with patients: a case study in autologous hematopoietic stem cell transplant for patients with scleroderma. Trials. 2021;22(1):611. doi:10.1186/s13063-021-05575-0

This article originally appeared on Rheumatology Advisor