358
markers can be misleading [ 6 ]. Reliable and comparable outcomes are required to
balance the benefi ts and adverse events of long-term steroids therapy and to evalu-
ate the use of corticosteroids sparing agents.
Patient reported outcomes (PROs), defi ned as outcomes that are completed by
patients, have been increasingly recognized as important measures over the past few
years. They incorporate the patient’s perspective of the disease, capturing the impact
of the disease in patients’ lives, and they perform well in assessing disease activity
in patients with PMR [ 7 ]. Different PROs, such as pain, morning stiffness, or physi-
cal function, have been proposed as recommended outcome measures to be used in
practice and clinical trials [ 8 ]. In addition, most remission or fl are defi nitions include
at least one self-reported variable from the medical history [ 9 ].
This review summarizes the use of PROs in clinical trials of patients with PMR
and the inclusion of PROs in diagnostic criteria or the evaluation of disease activity.
In addition, data are presented concerning the performance of a multidimensional
health assessment questionnaire (MDHAQ), only including PROs, to document
improvement over time in patients with PMR seen in routine care.
Literature Review of PROs Used in Trials of PMR
A systematic review was performed in PubMed (up to April 2015) to obtain all
published articles reporting any type of PROs in PMR [ 10 ]. Of 118 publications
identifi ed by the literature search, 20 met the selection criteria: 10 randomized con-
trolled trials, 8 prospective cohorts, 1 case control study, and 1 pilot observational
study. Patients included were typical for PMR populations, with a mean age between
62.5 and 76.6.
Pain was the most frequently reported domain, described as an outcome in the
majority of the studies (90 %). Of these, 61 % used a visual analogue scale (VAS)
to evaluate pain with no defi ned stem anchors, and the remainder used different
grades or presence versus absence of pain. In some studies, pain was evaluated as
an outcome to compare treatment groups [ 11 – 16 ], or to evaluate disease activity
differences according to gender [ 17 ], or as a potential predictor of vertebral frac-
tures [ 18 ].
Morning stiffness was recorded in 17 (85 %), with no consistency about how this
was defi ned or collected. It was most frequently evaluated by morning stiffness
duration in minutes without any grades (53 %); some studies graded morning stiff-
ness from 0 to 3 or 4, and 2 studies only evaluated the presence or absence of morn-
ing stiffness. Function was only reported in 25 % of the studies, more frequently in
cohorts than in randomized controlled trials (RCTs). The most frequent tool for this
domain was the Health Assessment Questionnaire (HAQ) [ 19 ], and the modifi ed
HAQ (MHAQ), which is a modifi ed shorter version of the original HAQ [ 20 ]. Both
are self-reported questionnaires developed initially for rheumatoid arthritis (RA)
I. Castrejon