Multicentre versus single centre approach to rare diseases: The model of systemic light chain amyloidosis
Multicentre versus single centre approach to rare diseases: The model of systemic light chain amyloidosis
复制标题
DOI：
10.1080/13506120500107055
复制
发表时间：
2005-06-01
影响因子：
5.5
通讯作者：
Gertz, MA
中科院分区：
文献类型：
--
作者：
Palladini, G;Kyle, RA;Gertz, MA
背景。早期诊断和支持治疗对原发性系统性淀粉样变性（AL）非常重要。1986年，意大利建立了一个全国网络，以提高医疗专业人员的认识，实现早期诊断，并提供当地合格的护理。我们比较了在梅奥诊所转诊中心观察到的AL患者群体与通过意大利网络招募的AL患者群体。所有1988年至1998年间在梅奥诊所或意大利淀粉样变性研究小组的任何中心诊断为AL的患者均被纳入研究。意大利患者的中位生存期为30个月，而Mayo队列为12个月（P < 0.001）。梅奥诊所患者年龄较大（66.4 vs 60.1, P < 0.001）。在意大利队列中，显性肾脏受累更为频繁（49.3%比27.8%,P < 0.001），而在梅奥组中，更多的患者有显性心脏淀粉样变性（37.4%比27.8%,P = 0.03）。意大利患者更有可能累及肾脏而不累及心脏（43.7%比19.6%,P < 0.001）。在意大利患者中，78.9%的患者接受了烷基化剂治疗，而在梅奥队列中，这一比例为60.9% （P < 0.001）。多因素分析显示，年龄、运动状态、主要心脏受累、>= 2个受累器官和烷基化药物治疗独立预测生存率。本研究表明，不同中心的AL患者群体根据其权责发生制可能存在显著差异，这可能对治疗试验产生影响。
Background. Early diagnosis and supportive therapy are important in primary systemic amyloidosis (AL). In 1986, a national network was started in Italy to increase the awareness of medical professionals, achieve early diagnoses, and provide locally qualified care. We compared the AL patient population observed at a referral center, Mayo Clinic, with that recruited through the Italian network.Methods. All the patients diagnosed with AL between 1988 and 1998 at Mayo Clinic or in any of the centers of the Italian Amyloidosis Study Group were included.Findings. The median survival of Italian patients was 30 months versus 12 months in the Mayo cohort (P < 0.001). Mayo Clinic patients were older (66.4 vs. 60.1 y, P < 0.001). In the Italian cohort, dominant kidney involvement was more frequent (49.3% vs. 27.8%, P < 0.001), while in the Mayo group more patients had dominant cardiac amyloidosis (37.4% vs. 27.8%, P = 0.03). Italian patients were more likely to have kidney involvement without heart involvement (43.7% vs. 19.6%, P < 0.001). Among Italian patients, 78.9% received alkylating agents versus 60.9% in the Mayo cohort (P < 0.001). The multivariate analysis showed that age, performance status, dominant heart involvement, >= 2 organs involved, and treatment with alkylating agents independently predicted survival. This study shows that AL patient populations may differ significantly between centers according to their accrual systems, with possible impact on treatment trials.