Background/Purpose: Patient reported outcomes (PROs)
are becoming increasingly important in the care of patients with chronic
disease. Involving patients in their own care significantly improves health
care outcomes, health care utilization and patient satisfaction. Robust PROs
have not been well defined for patients with juvenile dermatomyositis (JDM). We
sought to assess the importance of different PROs to patients with JDM and
their families to help guide development of these measures and care discussions.

Methods: A survey was developed by members of the Childhood
Arthritis and Rheumatology Research Alliance JDM Quality Measures Workgroup
through a consensus process and was distributed to approximately 2100 email
addresses of families of patients with JDM through the Cure JM foundation.  Data
were abstracted in a standardized database for analysis, including
demographics, myositis characteristics, functional disability, and rating of
importance of a variety of common PROs.

Results: Overall, 194 relatives of patients with
inflammatory myositis responded to the survey. Nearly all patients (187, or 97.4%)
were diagnosed with JDM, while 5 (2.6%) were diagnosed with juvenile
polymyositis. Of the respondents, 168 (93%) were parents and the remainder were
grandparents and other relatives. The majority of patients were female (140, or
73%). There was a wide range of age at diagnosis (1 to 16 years), with average
time to diagnosis 6.7 months (range 1 to over 40). Most patients (166, or 86%)
were Caucasian/white, 21 (11%) were Hispanic and 15 (8%) were African American.

The PROs that families of patients with JDM rated as having
the highest importance (weighted average over 4.5 out of 5) included overall
quality of life (173, or 89% reporting this among the most important factors),
timely diagnosis (171, or 89%), access to a rheumatologist (166, or 86%),
normal function and strength (155, or 80.3%), ability to perform self-care
(146, or 75%), resolution of pain (145, or 75%), discontinuation of steroids
(143, or 74%), resolution of fatigue (135, or 70%), and medication tolerance
(135, or 70%). Conversely, access to a dermatologist and physical therapy, work
attendance, minimizing hospital visits, medication counseling and monitoring of
medication toxicity, discontinuation of medications, normalization of labs,
resolution of rash, and school attendance were not rated as highly (Figure 1).

Families of patients diagnosed within the past 2 years
tended to rate all of the PROs as more important compared to those with
diagnoses over 2 years ago, as did families of patients with high current functional
impact from the disease, compared to those with low current functional impact.

Conclusion: Patients and their families with JDM find
certain PROs more meaningful and relevant than others, which should be taken
into account when establishing quality metrics and caring for patients with JDM.