第一临床医学院知识库

目的：评价CAG方案在急性髓细胞白血病（AML）及骨髓增生异常综合征-难治性贫血伴原始细胞过多（MDS-RAEB）患者的临床有效性和安全性。方法：回顾性分析2008年1月～2017年7月就诊于兰州大学第一医院，采用CAG方案化疗的131例AML及MDS-RAEB患者。所有患者均给与CAG方案，评估化疗后患者完全缓解（CR）率及总体有效（OR）率，分析疾病类型、年龄、疾病状态、染色体核型对患者CR率、OR率的影响；观察化疗相关不良反应并评估其严重程度。缓解后继续给与原方案、IA(E)、DA(E)、HA与中大剂量Ara-C方案进行交替巩固治疗6-8个疗程，并对患者总生存期进行长期随访。

结果：1. 一般特征：131例患者中，男性66例，女性37例，中位年龄53（6～82）岁。AML患者103例，其中初治73例，复发/难治30例，继发于MDS 5例，MDS-RAEB 患者23例。2. 近期疗效： AML患者103例，其中初治73例，复发/难治30例；初治患者CR及OR率分别为56.2%、64.4%；复发/难治患者CR及OR率分别为53.3%、63.3%；初治与复发/难治AML患者CR及OR率无明显差异（p>0.05）。AML患者中年龄＜60岁69例，CR及OR率分别为56.5%、63.8%；年龄≥60岁患者39例，CR及OR率分别为53.8%、61.4%。年龄≥60岁与＜60岁患者CR及OR率无明显差（p>0.05）。AML-M4、AML-M5患者CR率低于其它类型AML患者，但差异无统计学意义（p>0.05）。MDS-RAEB 23例，其中初治患者21例，CR及OR率分别为47.6%、61.9%；复发难治患者2例，无缓解病例。76例患者进行染色体核型分析，预后良好及中等组患者CR及OR率明显高于预后不良组，差异具有统计学意义（p<0.05）。3. 不良反应：主要的血液学不良反应为骨髓抑制，其中有65（49.6%）例患者发生III-IV度骨髓抑制；非血液学不良反应中，发生感染患者46（35.1%）例，少数患者出现轻度胃肠道反应及肝肾功能损伤，早期死亡率为3.8%。4. 预后及随访：28例患者失访，103例患者中，MST为15（0～96）个月。AML患者总OS率明显高于MDS-RAEB患者，差异具有统计学意义（p<0.05）。

结论  1. CAG方案对初治、复发/难治AML及MDS-RAEB患者CR率均较高，疗效较好，且年龄≥60岁与＜60岁患者CR及OR率无明显差异，老年患者可耐受；2. CAG方案对初治及复发/难治AML患者CR及OR率无明显差异， CAG方案有克服细胞耐药的作用；3. CAG方案对于AML-M4/AML-M5患者，其CR率低于其它类型AML；4. CAG方案对染色体核型预后良好及中等患者的CR及OR率，明显高于预后不良组；5. CAG方案化疗相关毒副作用低，主要血液学不良反应为骨髓抑制，非血液学不良反应轻微，耐受性好，心脏毒性及早期死亡率较低。

Purpose：This study aims to assessing the clinical effectiveness and safety of CAG regimen in acute myelogenous leukemia (AML) and myelodysplastic syndrome-refractory anemia with excess blasts (MDS-RAEB).Methods：In this study, acute myeloid leukemia and myelodysplastic syndrome -refractory anemia with excess blasts in first hospital of Lanzhou university from January 2008 to July 2017 were collected and a total of 131 patients treated with CAG regimen were enrolled. All patients were cured with CAG regimen. The complete remission (CR) rate and overall response (OR) rate of patient after CAG regimen are evaluated and the different of CR and OR rate between disease types, age, disease condition and karyotype are compared. The adverse reaction of chemotherapy is observed and its severity assessed. In addition, the regimen of CAG, IA (E), DA (E) HA and medium/large dose of Ara-c was used to consolidate treatment for 6-8 courses after remission, and long-term follow-up was conducted for the total survival of patients.

Results：1. Clinical features: Among the 131 patients, 66 are male and 37 are female, and the median age was 53(6～82) years old. There were 103 cases of AML patients, including 73 cases of primary and 30 cases of recurrence/refractory, 5 cases of MDS-AML, and 23 cases of MDS-RAEB.2. Curative effect: There are 103 patients with AML and 73 cases of which are primary and 30 cases are recurrence/refractory. The CR and OR rate are 56.2%, 64.4% and 53.3%, 63.3% respectively. There is no significant difference between CR and OR rate in primary and recurrence/refractory AML. 39 patients are aged ≥ 60, and the CR and OR rate are 53.8% and 64.1% respectively. The CR and OR rate are 56.5% and 63.8% of 69 patients aged < 60 years old. There was also no significant difference in CR and OR rates between aged ≥ 60 and < 60 years old (p>0.05). In addition，among the 103 patients with AML, The CR rate is lower in patients with AML-M4/AML-M5 than other types, but the difference has not statistically significant (p>0.05). There are 23 patients with MDS-RAEB 21 cases of which are primary and 2 cases are recurrence/refractory. In the primary patients, the CR and OR rates are 47.6% and 61.9% respectively. Patients with recurrence/ refractory had no remission. Among the 76 patients with chromosomal examination, the CR and the OR rate of the patients with good and moderate prognosis is significantly higher than those with poor prognosis, and the difference has statistically significant (p<0.05).2. Adverse reactions: The main adverse reaction of hematology is bone marrow suppression. There are 65(49.6%) patients with III-IV bone marrow suppression. According to the WHO toxicity criteria, 46（35.1%）patients were infected. A few patients have mild gastrointestinal reactions and slight damage of liver and kidney,. Only 5(3.8%) patients died during the treatment.3. Prognosis and follow-up: Among the 131 patients, 28 patients lost visit, the median total survival was 15(0～96) months. The OS rate of AML patients is significantly higher than that in MDS-RAEB patients, and the difference have statistically significant (p<0.05).

Conclusions： 1. The CAG regimen has good efficacy for patients with primary, recurrence/refractory AML and MDS-RAEB, and there was no significant difference in CR and OR rate between age ≥ 60 and < 60 years old, suggesting that the elderly patients could be tolerated. 2. There is no significant difference between the CR and OR rate in primary and refractory/recurrence AML patients, considering that the CAG regimen may be overcomes cell resistance. 3. The CR rate of patients with AML-M4/AML-M5 is lower than that of other AML patients. 4. The CR and OR rates of patients with poor and moderate prognosis are significantly higher than those with poor prognosis of chromosome karyotypes. 5. The CAG regimen has low toxicity and good tolerance, the non-hematologic adverse reactions mild, cardiac toxicity and early mortality is low.