Patient_Reported_Outcome_Measures_in_Rheumatic_Diseases

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(for parents’ proxy reports) or even better than (for children’s self-reports) that of
healthy children. The poorer PsH seen in healthy children concerned predominantly
the adolescent age group. This observation was attributed to the fact that most JIA
patients currently seen in tertiary care pediatric rheumatology centers have well-
controlled disease with little or no disease activity or disability. Children with
chronic arthritis suffer in the active phase of their disease a considerable burden of
symptoms, namely pain and stiffness, which affects many aspects of their lives. For
these children, disease improvement represents a key priority. It is, therefore, con-
ceivable that resolution of symptoms leads to a marked improvement in their mental
and social health.


Compliance to Therapy

The fi rst drug-specifi c and disease- specifi c questionnaire for the measurement of
treatment tolerance in JIA was developed by Bulatovic and coworkers in 2011 [ 70 ].
They designed and validated a new questionnaire for methotrexate-related gastroin-
testinal and behavioral symptoms. Methotrexate is the fi rst-choice disease-
modifying antirheumatic drug for the treatment of JIA. Gastrointestinal adverse
effects, which include nausea, abdominal pain, vomiting, or diarrhea, are quite com-
mon during methotrexate treatment. The Methotrexate Intolerance Severity Score
(MISS) consists of 12 questions, assessing abdominal pain, nausea, and vomiting
after or before (anticipatory) methotrexate intake and when thinking of methotrex-
ate (associative). Furthermore, it assesses behavioral complaints associated with
methotrexate intake, such as crying, restlessness, irritability, and refusal to take the
drug. The score ranges between 0 and 36, and subjects with a score of ≥6, including
at least 1 anticipatory, associative or behavioral symptom, were defi ned as metho-
trexate intolerant.


Multidimensional Tools and Composite Scores

The heterogeneous and multidimensional nature of JIA implies that numerous dis-
ease domains should be evaluated simultaneously to appraise the full extent of the
illness [ 71 ]. In this respect, there are several PCROs not addressed by conventional
instruments, such as evaluation of morning stiffness and overall level of disease
activity, rating of disease status and course, proxy- or self-assessment of joint
involvement and extra-articular symptoms, description of side effects of medica-
tions, assessment of therapeutic compliance, and satisfaction with the outcome of
the illness, which may provide valuable insights into the infl uence of the disease and
its treatment on a child’s health.
A multidimensional questionnaire for the assessment of children with JIA in stan-
dard clinical care that incorporates most parent/child-reported outcomes has been


A. Consolaro et al.
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