Patient_Reported_Outcome_Measures_in_Rheumatic_Diseases

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elbows and knees, but may also affect the face and neck. In dcSSc, skin fi brosis is
found both distally and proximally and represents the more severe form of SSc, with
a more progressive course and earlier internal organ manifestations [ 10 – 13 ].
People with SSc experience many additional problems that can markedly impair
their quality of life and general health [ 3 , 14 , 15 ]. Patients frequently experience
pain that is multifactorial in nature, occurring as a result of, but not limited to, fi n-
gertip ulcers, Raynaud’s phenomenon, skin infl ammation, calcinosis, infl ammatory
arthritis, and muscle tenderness [ 16 , 17 ]. A sometimes overlooked but common
symptom of SSc is pruritus, which negatively impacts quality of life through several
physical and psychosocial mechanisms, such as by affecting sleep cycles [ 18 , 19 ].
The gastrointestinal tract can be a source of signifi cant impairment as it becomes
fi brotic, causing dysmotility and an inappropriately patent lower esophageal sphinc-
ter, resulting in gastroesophageal refl ux disorder (GERD), dysphagia, choking,
cramps, bloating, early satiety, gastric dumping, incontinence, diarrhea, and consti-
pation [ 8 , 16 ]. The lungs can be involved with interstitial lung disease (ILD) and
pulmonary arterial hypertension (PAH), both of which can be lethal, and can cause
symptoms of dyspnea and fatigue [ 8 , 16 ]. ILD can be associated with cough and
occasionally chest pain. There may be pleural or pericardial effusions, which can
also cause chest pain. The kidneys may be affected with scleroderma renal crisis
(SRC). There are several cardiac features in SSc including cardiomyopathy, arrhyth-
mia, and constrictive pericarditis, further complicating the already complex sys-
temic picture of SSc [ 16 ].
Personal, social, and work life are not only disrupted by the aggressive and wide-
spread nature of SSc and its physically destructive complications, but also by other
features including fatigue, diffi culty sleeping, depressive symptoms, fear of progres-
sion and dying, body image issues, work disability, and sexual dysfunction that fre-
quently occurs [ 8 , 20 – 25 ]. In fact, the rate of work disability in SSc is higher than in
rheumatoid arthritis [ 7 ]. Research by Bassel et al. demonstrated that fatigue was the
most common symptom (89 %) SSc patients experienced, often with a moderate- to-
severe impact on daily activities (72 %) [ 8 ]. Other notable symptoms in terms of
frequency and impact were Raynaud’s phenomenon, hand stiffness, joint pain, sleep-
ing diffi culties, poor hand function, and pain [ 8 ]. SSc impacts self-esteem by causing
visible disfi gurement on socially important areas of skin, such as the hands (e.g.,
digital ulcers or loss, contractures, and swollen fi ngers) and face (e.g., tight skin,
telangiectasia, thin lips, loss of subcutaneous tissue, and increased wrinkles around
the mouth as SSc regresses). No cure exists for SSc, and many features of SSc are
physically prominent and worsen with time, further enhancing fears regarding their
disease [ 24 , 25 ]. See Fig. 11.1 for a visual depiction of some features of SSc.
Some symptoms can be managed to reduce their impact on patients; however, at
present there is a paucity of proven effective disease-modifying agents and often a
lack of recommendations for psychosocial management. Thus, SSc treatment typi-
cally requires a combination of organ-based symptomatic treatment and potentially
disease-modifying agents. These treatments have possible side effects and risk to the
patient and result in complex regimens of medications and frequent follow-up. By
determining which treatments SSc patients fi nd effective, high-quality instruments
for measuring patient-reported outcomes (PROs) can help enrich and expand opti-


R.E. Pellar et al.
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