musculoskeletal and connective tissue disorders
Systemic Sclerosis
Treatment of Systemic Sclerosis
Treatment directed at symptoms and dysfunctional organs No drug significantly influences the natural course of systemic sclerosis overall, but various drugs are of value in treating specific symptoms or organ systems. Corticosteroids may be helpful if there is overt myositis or mixed connective tissue disease but may predispose to renal crisis and thus are used only if necessary. mofetil is effective for the treatment of scleroderma-associated interstitial lung disease ( Treatment references ) and has been considered standard of care. mofetil ( Treatment references ). (150 mg orally every 12 hours) has been shown to slow the rate of lung function decline in scleroderma-associated interstitial lung disease and can be added to immunosupression in patients with progressive disease despite adequate immunosuppressive treatment ( Treatment references ). Epoprostenol(prostacyclin) andbosentanare used for pulmonary hypertension. Successful lung transplantation has been reported. Calcium channel blockers, such as long-acting oralnifedipine(eg, 30 to 120 mg a day), may help Raynaud syndrome but may worsen gastric reflux.Bosentan,sildenafil,tadalafil, andvardenafilare other alternatives for severe Raynaud syndrome. Patients should dress warmly, wear mittens, and keep their head warm. IV infusions of prostaglandin E1 (alprostadil) orepoprostenolor sympathetic blockers can be used for severe digital ischemia. Reflux esophagitis is relieved by frequent small feedings, high-dose proton pump inhibitors, sleeping with the head of the bed elevated, and not lying supine within 3 hours of the last meal. Esophageal strictures may require periodic dilation; gastroesophageal reflux may possibly require gastroplasty. Oralciprofloxacin500 mg 2 times a day or the combination ofmetronidazole(500 mg 3 times a day) andtrimethoprim/sulfamethoxazole double strength (1 tablet 2 times a day) for 7 to 10 days or another broad-spectrum antibiotic can suppress overgrowth of intestinal flora and may alleviate symptoms of small intestine bacterial overgrowth, such as bloating, flatulence, and diarrhea. Physical therapy may help preserve muscle strength but is ineffective in preventing joint contractures. No treatment has clear benefit in treating calcinosis. However,sodium thiosulfateinjected into the lesion can be used, as well as surgical excision of large lesions that affect joint function or are cosmetically distressing for the patient. For acute renal crisis, a medical emergency, prompt treatment with an angiotensin-converting enzyme inhibitor can dramatically prolong survival. The mortality rate of renal crisis remains high, but the crisis is often reversible if treatment is prompt. Dialysis may be necessary, temporarily or long-term. Renal transplantation is a feasible option in patients who develop end-stage renal disease. Recent evidence showed that Hematopoietic Stem Cell Transplantation in early generalized systemic sclerosis increases survival after the first year more than does IV 1.Tashkin DP, Roth MD, Clements PJ, et al:Mycophenolatemofetil versus oralcyclophosphamidein scleroderma-related interstitial lung disease (SLS II): A randomised controlled, double-blind, parallel group trial.Lancet Respir Med4(9):708–719, 2016. doi: 10.1016/S2213-2600(16)30152-7 2.Roofeh D, Lin CJF, Goldin J, et al.Tocilizumabprevents progression of early systemic sclerosis-associated interstitial lung disease.Arthritis Rheumatol73(7):1301-1310, 2021. doi:10.1002/art.41668 3.Distler O, Highland KB, Gahlemann M, et al:Nintedanibfor systemic sclerosis-associated interstitial lung disease.N Engl J Med380(26):2518-2528, 2019. doi:10.1056/NEJMoa1903076 4.van Laar JM, Farge D, Sont JK, et al: Autologous hematopoietic stem cell transplantation vs intravenous pulsecyclophosphamidein diffuse cutaneous systemic sclerosis: A randomized clinical trial.JAMA311(24):2490–2498, 2014. doi: 10.1001/jama.2014.6368 