Patient_Reported_Outcome_Measures_in_Rheumatic_Diseases

301

Conclusion

SSc is a chronic connective tissue disease with many possible manifestations. The
effects of SSc are often debilitating and tend to worsen with time, leading to func-
tional, emotional, and social impairment. PROs are valuable tools to assess SSc
patients, as they provide patients with the opportunity to concisely and quantita-
tively express their disease experience, which is different from physiologic param-
eters and measurements performed by a healthcare professional. Thus, this method
of measuring disease aspects and symptoms goes beyond standard clinical assess-
ments and is complementary to other outcome measurements. Due to the heteroge-
neous nature of SSc, there are consequentially many tools that can be used. These
PROs may be either general (used in many diseases) or specifi c to SSc. Further, the
tools may assess overall HRQoL or functional status, or they may be specifi c to
certain disease manifestations, such as pain or dyspnea. One of the fi rst and most
commonly used assessments of functional status is the HAQ-DI. Although used
extensively in SSc, it was originally designed for RA. On the other hand, tools such
as the SHAQ, SBI, and UKFS were developed to measure functional status and
HRQoL in SSc. Symptom-specifi c PROs designed for SSc include the MHISS and
UCLA SCTC GIT 2.0 to measure mouth disability and gastrointestinal involve-
ment, respectively. Many other PROs have been studied for use in SSc.
To effectively improve treatment for patients, it is important to understand what
outcomes are meaningful to them. Therefore, in research trials and sometimes in
clinical care, utilizing PROs allows us to determine which treatments make the
greatest positive difference in patients’ lives, or conversely, which are most detri-
mental to their well-being. Additional research is required to refi ne SSc PROs and
investigate the validity of some measures within SSc. Due to the rarity and unique-
ness of SSc, newer tools will be tested in SSc such as computer-based question-
naires that allow patients to skip areas that are irrelevant to their HRQoL such as use
of the PROMIS questionnaire.

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11 PROMs for Systemic Sclerosis (Scleroderma)