Naddei, Roberta (2022) Clinical outcome measures in juvenile idiopathic arthritis: toward a new disease activity score based on the parent/patient’s perspective. [Tesi di dottorato]

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The regular assessment of disease activity through validated and reliable tools is of utmost importance in the management of patients with juvenile idiopathic arthritis (JIA). The treatment target and the therapeutic plan should be based on shared decisions between the parent/patient and the physician. Patient/parent reported outcome measures (PCROs) provide a direct insight on the parent’s and child’s perceptions of disease course and effectiveness of therapeutic interventions. The incorporation of PCROs in the routine assessment of children with JIA may lead to more efficient and effective clinical care, by enforcing concordance with physician’s choices. The identification of valid and reliable PCROs could be crucial to remotely monitor disease activity when in-face evaluation is not possible, as happened during Coronavirus Disease 2019 pandemic and reported in the chapter 2 of this thesis. Against this scenario, our research was aimed to develop a new tool, solely based on PCROs, for the assessment of disease activity by parent/patient. First, to identify the items suitable to be included into this new tool, we analyzed the measurement properties of following 4 PCROs for disease activity: 1) parent’s or child’s assessment of overall disease activity on a 21-numbered circle visual analogue scale (VAS) (0 = no disease activity; 10 = maximum disease activity; 2) parent’s or child’s assessment of pain on a 21-numbered circle VAS (0 = no pain; 10 = maximum pain); 3) parent’s or child’s assessment of the activity of joint disease; 4) morning stiffness duration, scored on a 10-point scale as follows: absent (score = 0); less than 15 minutes (score = 2); 15-30 minutes (score 4); 30 minutes-1 hour (score = 6); 1-2 hours (score = 8); > 2 hours (score =10). We provided evidence of the validity and reliability of these four PCROs, showing that they could be appropriate instruments to remotely monitor the disease activity of JIA, as reported in chapter 4. On the other hand, we showed that the parent/patient global assessment of wellbeing might be an imperfect indicator of disease activity in patients with JIA, because it can be affected by several other factor irrespective of disease activity, such as persistent pain, functional ability, treatment burden, medications’ side effects and psychosocial aspects (chapter 3). Therefore, we decided to rule out this PCRO as a component of the new PCROs-based score for JIA activity. Finally, in chapter 5, we proposed the parent/child Juvenile Arthritis Disease Activity Score (par/childJADAS) as a new disease activity tool for JIA, solely based on PCROs. The parJADAS and childJADAS are calculated as the simple linear sum of the scores of their 4 components, which are the 4 above-mentioned PCROs. The parJADAS and the childJADAS exhibited very good measurement properties, possessing good construct validity, discriminant and predictive ability, internal consistency, and responsiveness to change. A regular home-completion of parJADAS and the childJADAS through electronic devices could be used for the remote assessment of the disease activity and telehealth, leading to an improvement of the quality of care of patients with JIA.

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