Steen V D, Medsger T A
Georgetown University, Washington, DC 20007-2197, USA.
Arthritis Rheum. 2000 Nov;43(11):2437-44. doi: 10.1002/1529-0131(200011)43:11<2437::AID-ANR10>3.0.CO;2-U.
To determine the natural history and timing of severe involvement of the kidney, heart, lung, gastrointestinal (GI) tract, and skin in patients with systemic sclerosis (SSc) and diffuse cutaneous involvement.
This study used the Pittsburgh Scleroderma Databank and included patients with diffuse scleroderma who were seen between January 1, 1972 and December 31, 1995. Patients had frequent follow-ups, and a 95% accountability for these patients was maintained. Severe organ involvement was defined as the presence of any of the following: 1) in the kidney, scleroderma "renal crisis"; 2) in the heart, cardiomyopathy, symptomatic pericarditis, or an arrhythmia requiring treatment; 3) in the lung, pulmonary fibrosis on chest radiograph and a forced vital capacity of <55% of predicted; 4) in the GI tract, malabsorption, repeated episodes of pseudoobstruction, or severe problems requiring hyperalimentation; and 5) in the skin, a modified Rodnan skin score >40. The timing from disease onset to survival for each case of severe organ involvement was determined.
Of the 953 patients with diffuse scleroderma, kidney involvement developed in 177 (19%), heart involvement in 143 (15%), lung involvement in 151 (16%), GI tract involvement in 74 (8%), and skin involvement in 233 (24%). Severe skin and kidney involvement occurred during the first 3 years in 70% of those who ever developed these problems throughout a mean of 10 years of followup. Severe heart, lung, and GI tract involvement developed during the first 3 years in 45-55% of those who were ever affected. The survival of patients with severe organ involvement was poor. The 9-year cumulative survival rate of all patients with severe organ involvement was 38%, compared with 72% in patients without such involvement (P < 0.0001).
This study demonstrates that severe organ involvement in SSc patients with diffuse scleroderma most often occurs early in the course of the disease. Survival for patients with severe organ involvement is markedly reduced. Patients should therefore be monitored very closely during the first 3 years of disease for signs and symptoms that may signal the subsequent development of severe organ damage. Potential disease-modifying therapies must be initiated early to modify the natural history of SSc and to improve survival. Patients who survive the first few years without developing severe organ involvement are less likely to develop such life-threatening involvement later in the disease course.
确定系统性硬化症（SSc）弥漫性皮肤受累患者肾脏、心脏、肺、胃肠道（GI）和皮肤严重受累的自然病程及发生时间。
本研究使用匹兹堡硬皮病数据库，纳入1972年1月1日至1995年12月31日期间就诊的弥漫性硬皮病患者。患者接受频繁随访，并对这些患者保持95%的随访率。严重器官受累定义为出现以下任何一种情况：1）在肾脏，硬皮病“肾危象”；2）在心脏，心肌病、有症状的心包炎或需要治疗的心律失常；3）在肺，胸部X线片显示肺纤维化且用力肺活量<预测值的55%；4）在胃肠道，吸收不良、反复出现假性肠梗阻发作或需要胃肠外营养的严重问题；5）在皮肤，改良Rodnan皮肤评分>40。确定每例严重器官受累患者从疾病发作到生存的时间。
在953例弥漫性硬皮病患者中，177例（19%）出现肾脏受累，143例（15%）出现心脏受累，151例（16%）出现肺受累，74例（8%）出现胃肠道受累，233例（24%）出现皮肤受累
