【摘要】：目的:系統(tǒng)性輕鏈型淀粉樣變性(systemic immunoglobulin light chain amyloidosis,AL)是臨床上最常見,也是預(yù)后最差的一種淀粉樣變性�；熀妥泽w干細(xì)胞移植是該病的主要治療手段,國(guó)外小樣本研究表明沙利度胺聯(lián)合地塞米松(thalidomide combined dexamethasone,TD)化療方案對(duì)復(fù)發(fā)和難治性AL患者有一定的療效,但國(guó)內(nèi)外尚缺乏TD方案對(duì)初治AL患者的療效和毒副反應(yīng)的報(bào)道。本研究旨在通過回顧性分析AL患者的臨床和預(yù)后資料,探討TD方案對(duì)初治AL的療效和安全性。方法:回顧性分析2009年4月至2014年12月在南京總醫(yī)院確診并采用TD方案作為初治方案的63例AL型淀粉樣變性患者的臨床及隨訪資料。所有患者均采用TD方案治療4個(gè)療程及以上,沙利度胺起始治療劑量為50mg/晚,1周后未見不良反應(yīng)則加量至100mg/晚,以此類推,最高劑量為200mg/晚,地塞米松起始治療劑量為40mg/周,28天為1個(gè)治療周期。應(yīng)用Kaplan-Meier法計(jì)算患者生存時(shí)間,組間對(duì)比采用log-rank檢驗(yàn),利用COX風(fēng)險(xiǎn)比例模型分析預(yù)后危險(xiǎn)因素。結(jié)果:本研究共納入患者63例,男女比例為1.52:1,中位年齡為57歲,中位治療周期為19個(gè)(4-78個(gè))。38例患者(60.3%)心臟受累,38例患者(60.3%)兩個(gè)或以上的器官受累。37例患者取得血液學(xué)緩解,總體血液學(xué)緩解率為58.7%,中位到達(dá)緩解的時(shí)間為4個(gè)月,其中18例患者(28.5%)達(dá)到完全緩解(complete response,CR),中位到達(dá)緩解的時(shí)間為3個(gè)月,10例患者(15.9%)達(dá)到非常好的部分緩解(very good partial response,VGPR),中位到達(dá)緩解的時(shí)間為5.5個(gè)月,9例患者(14.2%)達(dá)到部分緩解(partial response,PR),中位到達(dá)緩解的時(shí)間為5個(gè)月。23例患者(36.5%)獲得器官緩解,腎臟緩解和心臟緩解分別為23例(36.5%)和10例(15.9%),中位到達(dá)緩解的時(shí)間分別為8.5和18.8個(gè)月。有3例患者病情復(fù)發(fā),中位復(fù)發(fā)時(shí)間為7個(gè)月(3-19個(gè)月)�；颊叩闹形浑S訪時(shí)間為28個(gè)月,2年生存率為78.5%,K-M曲線估計(jì)的中位生存時(shí)間尚未達(dá)到。達(dá)到血液學(xué)緩解的患者相比未達(dá)到血液學(xué)緩解的患者預(yù)后更好(3年生存率87.5%vs.36.3%,P0.01),心臟受累的患者相比無心臟受累的患者預(yù)后更差(3年生存率52.4%vs.87%,P0.05)。常見不良反應(yīng)包括外周神經(jīng)病變(23.8%)、胃腸道反應(yīng)(17.5%)、乏力(11/1%)、皮疹(6.3%)等。9例患者因不良反應(yīng)和治療無效停藥,12例患者沙利度胺減量,22例患者地塞米松減量。結(jié)論:TD方案對(duì)初治的AL型淀粉樣變性患者有一定的療效,并具有較好的耐受性,可以作為不適合行大劑量馬法蘭聯(lián)合自體外周血干細(xì)胞移植(high-dose melphalan/autologous peripheral blood stem cell transplantation,HDM/ASCT)及硼替佐米聯(lián)合地塞米松(bortezomib combined dexamethasone,BD)方案化療患者的備選方案,其長(zhǎng)期療效有待進(jìn)一步觀察。
[Abstract]:Objective: systemic light chain amyloidosis (systemic immunoglobulin light chain amyloidosis,AL) is the most common and poor prognosis in clinic. Chemotherapy and autologous stem cell transplantation are the main treatments for the disease. Small sample studies abroad have shown that thalidomide combined with dexamethasone (thalidomide combined dexamethasone,TD) chemotherapy regimen has certain efficacy in patients with recurrent and refractory AL, but there is still a lack of reports on the efficacy and side effects of TD regimen in patients with newly diagnosed AL. The purpose of this study was to analyze the clinical and prognostic data of AL patients and to explore the efficacy and safety of TD regimen in the treatment of newly diagnosed AL. Methods: from April 2009 to December 2014, the clinical and follow-up data of 63 patients with AL type amyloidosis diagnosed in Nanjing General Hospital and treated with TD regimen were analyzed retrospectively. the clinical data of 63 patients with AL type amyloidosis diagnosed in Nanjing General Hospital from April 2009 to December 2014 were analyzed retrospectively. All patients were treated with TD regimen for 4 courses or more. The initial dose of thalidomide was late for 50mg/, but no adverse reaction was found until the late 100mg/ after 1 week, and so on, the highest dose was 200mg/ late, the initial dose of dexamethasone was 40mg/ week, and 28 days was a treatment cycle. Kaplan-Meier method was used to calculate the survival time of the patients, log-rank test was used to compare the groups, and COX risk ratio model was used to analyze the prognostic risk factors. Results: a total of 63 patients were enrolled in this study, the male to female ratio was 1.52 鈮，

本文編號(hào)：2499753