Phenotypes Determined by Cluster Analysis and Their Survival in the Prospective European Scleroderma Trials and Research Cohort of Patients With Systemic Sclerosis

Objective: Systemic sclerosis (SSc) is a heterogeneous connective tissue disease that is typically subdivided into limited cutaneous SSc (lcSSc) and diffuse cutaneous SSc (dcSSc) depending on the extent of skin involvement. This subclassification may not capture the entire variability of clinical phenotypes. The European Scleroderma Trials and Research (EUSTAR) database includes data on a prospective cohort of SSc patients from 122 European referral centers. This study was undertaken to perform a cluster analysis of EUSTAR data to distinguish and characterize homogeneous phenotypes without any a priori assumptions, and to examine survival among the clusters obtained. / Methods: A total of 11,318 patients were registered in the EUSTAR database, and 6,927 were included in the study. Twenty‐four clinical and serologic variables were used for clustering. / Results: Clustering analyses provided a first delineation of 2 clusters showing moderate stability. In an exploratory attempt, we further characterized 6 homogeneous groups that differed with regard to their clinical features, autoantibody profile, and mortality. Some groups resembled usual dcSSc or lcSSc prototypes, but others exhibited unique features, such as a majority of lcSSc patients with a high rate of visceral damage and antitopoisomerase antibodies. Prognosis varied among groups and the presence of organ damage markedly impacted survival regardless of cutaneous involvement. / Conclusion: Our findings suggest that restricting subsets of SSc patients to only those based on cutaneous involvement may not capture the complete heterogeneity of the disease. Organ damage and antibody profile should be taken into consideration when individuating homogeneous groups of patients with a distinct prognosis

Balanced performance measurement in research hospitals: the participative case study of a haematology department

Background: The paper aims to review, design and implement a multidimensional performance measurement system for a public research hospital in order to address the complexity of its multifaceted stakeholder requirements and its double institutional aim of care and research. Method: The methodology relies on a participative case study performed by external researchers in close collaboration with the staff of an Italian research hospital. Results: The paper develops and applies a customized version of balanced scorecard based on a new set of performance measures. Our findings suggest that it can be considered an effective framework for measuring the research hospital performance, thanks to a combination of generalizable and context-specific factors. Conclusions: By showing how the balanced scorecard framework can be customized to research hospitals, the paper is especially of interest for complex healthcare organizations that are implementing management accounting practices. The paper contributes to the body of literature on the application of the balanced scorecard in healthcare through an examination of the challenges in designing and implementing this multidimensional performance tool. This is one of the first papers that show how the balanced scorecard model can be adapted to fit the specific requirements of public research hospitals