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A Symptoms and Signs
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Most frequently, the disease makes its appearance in the skin, although visceral involvement may precede the cutaneous features Polyarthralgia and Raynaud s phenomenon (present in 90% of patients) are early manifestations Subcutaneous edema, fever, and malaise are common With time the skin becomes thickened and hidebound, with loss of normal folds Telangiectasia, pigmentation, and depigmentation are characteristic Ulceration about the fingertips and subcutaneous calcification are seen Dysphagia due to esophageal dysfunction is common and results from abnormalities in motility and later from fibrosis Fibrosis and atrophy of the gastrointestinal tract cause hypomotility, and malabsorption results from bacterial overgrowth Large-mouthed diverticuli occur in the jejunum, ileum, and colon Diffuse pulmonary fibrosis and
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Arthritis & Musculoskeletal Disorders
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acid once sold as an over-the-counter remedy for insomnia and premenstrual symptoms (before removal from the market) Since banning of L-tryptophan sales, the eosinophilia-myalgia syndrome has essentially disappeared
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Treatment
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Treatment of systemic sclerosis is symptomatic and supportive and focuses on the prominent organ system involved There is no medication that is known to alter the underlying disease process However, interventions for management of specific organ manifestations of this disease have improved substantially Severe Raynaud s syndrome may respond to calcium channel blockers, eg, long-acting nifedipine, 30 120 mg/d orally, or to losartan, 50 mg/d orally Intravenous epoprostenol, a prostacyclin analog that causes vasodilation and platelet inhibition, is moderately effective in healing digital ulcers (Intravenous iloprost is also effective but not available in the United States) Patients with esophageal disease should take medications in liquid or crushed form Esophageal reflux can be reduced and the risk of scarring diminished by avoidance of late-night meals and elevation of the head of the bed In addition, proton pump inhibitors (eg, omeprazole, 20 40 mg/ d orally), the only drugs that achieve near-complete inhibition of gastric acid production, are remarkably effective for refractory esophagitis Patients with delayed gastric emptying maintain their weight better if they eat small, frequent meals and remain upright for at least 2 hours after eating Octreotide, a somatostatin analog, helps some patients with bacterial overgrowth and pseudoobstruction Malabsorption due to bacterial overgrowth also responds to antibiotics, eg, tetracycline, 500 mg four times orally daily, often prescribed cyclically The hypertensive crises associated with systemic sclerosis renal crisis must be treated early and aggressively (in the hospital) with angiotensin-converting enzyme inhibitors, eg, captopril, initiated at 25 mg every 6 hours and titrated up as needed to a maximum of 100 mg every 6 hours Prednisone has little or no role in the treatment of scleroderma Cyclophosphamide, a drug with many important side effects, improves dyspnea and pulmonary function tests modestly in patients with severe interstitial lung disease Bosentan, an endothelin receptor antagonist, improves exercise capacity and cardiopulmonary hemodynamics in patients with pulmonary hypertension and helps prevent digital ulceration The 9-year survival rate in scleroderma averages approximately 40% The prognosis tends to be worse in those with diffuse scleroderma, in blacks, in males, and in older patients In most cases, death results from renal, cardiac, or pulmonary failure Those persons in whom severe internal organ involvement does not develop in the first 3 years have a substantially better prognosis, with 72% surviving at least 9 years Breast and lung cancer may be more common in patients with scleroderma
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Baroni SS et al Stimulatory autoantibodies to the PDGF receptor in systemic sclerosis N Engl J Med 2006 Jun 22;354(25): 2667 76 [PMID: 16790699] Denton CP et al Bosentan treatment for pulmonary arterial hypertension related to connective tissue disease: a subgroup analysis of the pivotal clinical trials and their open-label extensions Ann Rheum Dis 2006 Oct;65(10):1336 40 [PMID: 16793845]
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Ioannidis JP et al Mortality in systemic sclerosis: an international meta-analysis of individual patient data Am J Med 2005 Jan;118(1):2 10 [PMID: 15639201] Korn JH et al Digital ulcers in systemic sclerosis: prevention by treatment with bosentan, an oral endothelin receptor antagonist Arthritis Rheum 2004 Dec;50(12):3985 93 [PMID: 15593188] Tashkin DP et al; Scleroderma Lung Study Research Group Cyclophosphamide versus placebo in scleroderma lung disease N Engl J Med 2006 Jun 22;354(25):2655 66 [PMID: 16790698]
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IDIOPATHIC INFLAMMATORY MYOPATHIES (POLYMYOSITIS & DERMATOMYOSITIS)
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