What can patients tell us in Sjögren's syndrome?

Abstract

In Sjögren's Syndrome (SS), clinical heterogeneity and discordance between disease activity measures and patient experience are key obstacles to effective therapeutic development. Patient reported outcome measures (PROMs) are useful tools for understanding the unmet needs from the patients' perspective and therefore they are key for the development of patient centric healthcare systems. Initial concern about the subjectivity of PROMs has given way to methodological rigour and clear guidance for the development of PROMs. To date, several studies of patient stratification using PROMs have identified similar symptom-based subgroups. There is evidence to suggest that these subgroups may represent different disease endotypes with differing responses to therapeutic interventions. Stratified medicine approaches, alongside sensitive outcome measures, have the potential to improve our understanding of SS pathobiology and therapeutic development. The inclusion of PROMs is important for the success of such approaches. In this review we discuss the opportunities of using PROMs in understanding the pathogenesis of and therapeutic development for SS.

Keywords: Sjögren’s; composite endpoints; electronic patient-reported outcomes; endotypes; heterogeneity; newcastle Sjögren’s stratification tool; patient-reported outcomes; stratified medicine.

Figure 1

A) ARACNE Clinical network reconstruction for a cross-sectional dataset of 624 patient reported outcome measures and objective clinical and laboratory observations in SS. Edges between nodes represent shared information between the nodes. B) Sub-network of the ESSDAI subdomains showing the connections between them. Percentages in brackets represent the proportion of non-zero scores for each domain. Abbreviations: fVAS, Fatigue Visual Analogue Scale; BMI, Body Mass Index; Clin-ESSDAI, Clinical ESSDAI; CK, Creatin Kinase; Other_cancers, Current / Historical Cancer (not lymphoma); Lymphoma, Current / Historical Lymphoma; EQ-5D-D, EQ-5D- Depression; EQ-5D-M, EQ-5D- Mobility; EQ-5D-PD, EQ-5D- Pain/Discomfort; EQ-5D-SC, EQ-5D- Self Care; EQ-5D-UA, EQ-5D- Usual Activities; ESR, Erythrocyte Sendimentation Rate; ESSPRI, EULAR Sjögren’s Syndrome Patient Reported Index; ESSDAI, European League Against Rheumatism (EULAR) Sjögren’s Syndrome Disease Activity Index; Articular, ESSDAI- Articular Domain; Biological, ESSDAI- Biological Domain; CNS, ESSDAI- Central Nervous System Domain; Constitutional, ESSDAI- Constitutional Domain; Cutaneous, ESSDAI- Cutaneous Domain; Glandular, ESSDAI- Glandular Domain; Haematological, ESSDAI- Haematological Domain; Lymphadenopathy, ESSDAI- Lymphadenopathy Domain; Muscular, ESSDAI- Muscular Domain; PNS, ESSDAI- Peripheral Nervous System Domain; Renal, ESSDAI- Renal Domain; Respiratory, ESSDAI- Respiratory Domain; EULAR-SS, EULAR Sicca Scale; EULAR-SS-Ocular, EULAR Sicca Scale- Ocular; EULAR-SS-Oral, EULAR Sicca Scale- Oral; EQ-5D-TTO, EuroQual 5 Dimensions (EQ-5D) - Time Trade Off; HAD-A, Hospital Anxiety Depression Scale- Anxiety; HAD-D, Hospital Anxiety Depression Scale-Depression; OSF, Oral Salivary Flow; Plt, Platelet Count; PROf-Mental, Profile of Fatigue (PROf) - Mental Fatigue; PROf-Physical, PROf- Physical Fatigue; PROf-C, PROf- Concentration; PROf-F, PROf- Forget; PROf-HGG, PROf- Hard to Get Going; PROf-LE, PROf- Lacking Energy; PROf-NR, PROf- Need Rest; PROf-W, PROf- Weak; Schirmers_L, Schirmer’s Left Eye; Schirmers_R, Schirmer’s Right Eye; SBP, Systolic Blood Pressure; DBP, Diastolic Blood Pressure; WCC, White Cell Count.