Relationships between psoriatic arthritis composite measures of disease activity with patient-reported outcomes in phase 3 studies of tofacitinib

Abstract

Background In psoriatic arthritis (PsA), further understanding of the relationships between clinical measures and patient-reported outcomes (PROs) is needed. This post hoc analysis evaluated associations between minimal disease activity (MDA) as a continuous outcome (termed ScoreMDA) or Psoriatic Arthritis Disease Activity Score (PASDAS) with selected PROs not included in the composite measures. Methods Data from two phase 3 studies of tofacitinib in PsA (OPAL Broaden [NCT01877668; N = 422]; OPAL Beyond [NCT01882439; N = 394]) were included. MDA (binary outcome) was defined as meeting ≥5/7 criteria. For ScoreMDA, each criterion was assigned a value (1 = true; 0 = false; score range, 0–7; scores ≥5 indicated MDA). For PASDAS (score range, 0–10), higher scores indicated worse disease activity. PROs analyzed included Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F), Patient’s Assessment of Arthritis Pain visual analog scale (Pain VAS), and EuroQoL-Five Dimensions-Three Level Health Questionnaire visual analog scale (EQ-5D-3L VAS) and utility index. Relationships were evaluated using repeated measures regression models. Results Similar, approximately linear relationships were confirmed between PASDAS or ScoreMDA and PROs in both studies. In OPAL Broaden and OPAL Beyond, a one-point difference in PASDAS was associated with clinically relevant differences in PROs, including EQ-5D-3L VAS (− 6.7 mm, − 6.9 mm), Pain VAS (9.9 mm, 10.7 mm), and FACIT-F (− 2.8, − 3.3). A one-point difference in ScoreMDA was associated with clinically relevant differences in PROs, including EQ-5D-3L VAS (5.0 mm, 5.5 mm) and FACIT-F (1.9, 2.7) in OPAL Broaden and OPAL Beyond, respectively. Conclusions Linear associations between PASDAS or ScoreMDA and PROs provide interpretable and quantifiable metrics between composite clinical measures and PROs, highlighting the importance of these measures in understanding the relevance of treat-to-target goals in PsA. Trial registration ClinicalTrials.gov, NCT01877668. Registered on June 12, 2013. ClinicalTrials.gov, NCT01882439. Registered on June 18, 2013

Evaluation of the Disease Activity index for PSoriatic Arthritis (DAPSA) with a quick quantitative C reactive protein assay (Q-DAPSA) in patients with psoriatic arthritis: a prospective multicentre cross-sectional study

“…3 Another well-validated composite index is the Psoriatic Arthritis Disease Activity Score (PASDAS), 6 which has shown a close relationship with various patient reported outcome (PRO) measures. 7 The DAPSA is a continuous composite index focusing on joint involvement in PsA. 8 9 It encompasses swollen joint count (SJC) out of 66 joints, tender joint count out of 68 joints, patient global assessment on a visual analogue scale (VAS) ranging from 0 to 10 cm, patient pain assessment on a VAS ranging from 0 to 10 cm and C reactive protein (CRP) in mg/dL.…”

“… 3 Another well-validated composite index is the Psoriatic Arthritis Disease Activity Score (PASDAS), 6 which has shown a close relationship with various patient reported outcome (PRO) measures. 7 …”

The state of the art—psoriatic arthritis outcome assessment in clinical trials and daily practice

“…Despite these theoretical limitations, analysis of clinical trial data supports the robust validity of the MDA as a treatment target and its correlation with other remission thresholds. [32][33][34][35][36] While other instruments with multiple binary thresholds (ACR20/50/70 or DAPSA50/75/85) overcome some of the limitations of a single binary threshold and perform well as a response measure, variability in treatment baselines results in these responder indices being inappropriate as a treatment target.…”

“…Recent post-hoc analysis of the OPAL-Broaden and OPAL-Beyond cohort utilised the ScoreMDA, which converts the MDA into a linear scale (range 0-7) based the number of components in which the threshold for MDA is achieved. 35 In essence, it is an indicator of the breadth and depth of remission achieved in some of the key domains of psoriatic disease. While it was found to be linearly associated with multiple patient-reported outcome measures in one study, prospective longitudinal assessments in clinical trials and observational studies are needed to understand its value in these settings given the absence domains such as psoriatic nail and axial disease.…”

Impact of tofacitinib on patient-reported outcomes in patients with psoriatic arthritis. Data from the real clinical practice

“…Нами продемонстрировано улучшение PROs, сопровождавшееся снижением активности ПсА по DAPSA. Это совпадает с результатами субанализа 2 РПКИ III фазы, изучавших применение ТОФА при ПсА, -OPAL Broaden (n=422) и OPAL Beyond (n = 394), -в которых была выявлена взаимосвязь между достижением минимальной активности ПсА, индексом активности ПсА (PASDAS, Psoriatic Arthritis Disease Activity Score) и такими PROs про как утомляемость по FACIT-F, боль, EQ-5D-3L и HAQ [29].…”