Objectives: There is less clinical data on multiple myeloma (MM) in China, and the aim of this study was to collect and analyze the clinical data of newly diagnosed multiple myeloma (NDMM) patients in Hunan Province during 1 year, to understand the real clinical features and treatment outcome for Hunan Province patients with MM, and to strengthen the understanding of the standardized diagnosis process and treatment plan of MM.
Methods: The clinical data of 529 patients with NDMM in 12 large-scale general hospitals in Hunan Province from January 1 to December 31, 2019 were collected and analyzed, including baseline data, treatment regimens, duration of treatment, and adverse reactions. The clinical characteristics, treatment, and safety of patients were analyzed by SPSS 21.0.
Results: Among the 529 NDMM patients, the age was 33-90 (median 64) years and the male-female ratio was 1.38꞉1. The clinical features ranged from high to low were as follows: Bone pain (77.7%), anemia (66.8%), renal insufficiency (40.6%), hypercalcemia (15.1%). Typing: IgG 46.5%, IgA 24.6%, IgD 2.6%, IgM 0.8%, light chain 15.7%, double clone 3.0%, no secretion 0.6%, absence 6.2%. Staging: Durie-Salmon stage I, II, and III were 4.5%, 10.6%, 77.3%, respectively, and 40 cases (7.6%) missed this data. International Staging System (ISS) stage I, II, and III were 10.4%, 24.4%, and 47.6%, respectively, and 93 cases (17.6%) were missing. Revised International Staging System (R-ISS) stage I, II, and III were 5.5%, 27.0%, 23.1%, respectively, and 235 cases (44.4%) missed this data. Among the 98 NDMM patients in the Third Xiangya Hospital, Central South University, Durie-Salmon (DS) stage missing 2.0%, ISS stage missing 12.3%, and R-ISS stage missing 12.3%.Treatment: Among the 529 patients,475 received treatment, the rate of treatment was 89.8%; 67.4% of the patients were able to complete four courses of chemotherapy at induction phase, 90.3% of the patients received proteasome inhibitor based combination chemotherapy regimen more than once, 67.2% received immunomodulator based regimen more than once, and 59.8% of the patients received proteasome inhibitor and immunomodulator based combination chemotherapy regimen more than once. Curative: Overall response rate (ORR) and high quality response rate (HQR) of the 4-course group were better than those of the 2-course group (ORR: 85% vs 65%, P=0.006; HQR: 68.3% vs 24.0%, P<0.001). The HQR of the standard chemotherapy group was better than that of the non-standard chemotherapy group (65.1% vs 48.2%, P=0.035). Adverse reactions during treatment included hematologic toxicity (17.5%), peripheral neuropathy (24.8%), gastrointestinal adverse events (23.8%), pulmonary infection (25.9%), herpes zoster (4.6%), and venous thrombotic events (1.7%).
Conclusions: In 2019, the missed diagnosis rate of MM patients was high, the medium age of diagnosis was older, and the accuracy of patient diagnosis was not high. There is a great difference among medical centers, especially in the stage and risk stratified, nearly half of NDMM patients are not diagnosed with R-ISS stage; the lack of cytogenetic data needs to be supplemented by follow-up studies. A high proportion of patients with NDMM present with bone pain and anemia.Patients received treatment have higher use of chemotherapy regimens containing proteasome inhibitors and/or immunomodulators, but there is a significant gap among different medical centers, and standardized treatment needs to be strengthened. The safety during chemotherapy is controllable.
目的: 国内多发性骨髓瘤(multiple myeloma，MM)临床大数据较少，本研究旨在分析湖南省多中心1年期间新诊断多发性骨髓瘤(newly diagnosed multiple myeloma，NDMM)患者的临床资料，了解湖南省MM患者真实的临床特征和诊疗情况，加强对MM规范化诊疗流程和诊疗方案的认识。方法: 收集湖南省12家大型综合性医院2019年1月1日至12月31日收治的共529例NDMM患者的临床资料，包括基线资料、治疗方案、疗程和不良反应等。采用SPSS 21.0统计软件分析患者的临床特征、治疗情况及安全性。结果: 529例NDMM患者确诊年龄33~90(中位数64)岁，男女比为1.38꞉1；临床特征占比从高到低依次为骨痛(77.7%)、贫血(66.8%)、肾功能不全(40.6%)、高钙血症(15.1%)。分型：IgG型46.5%，IgA型24.6%，IgD型2.6%，IgM型0.8%，轻链型15.7%，双克隆型3.0%，不分泌0.6%，缺失6.2%。DS(Durie-Salmon)分期I期、II期、III期分别为4.5%、10.6%、77.3%，缺失40例(7.6%)；国际分期系统(International Staging System，ISS)分期I期、II、III期分别为10.4%、24.4%、47.6%，缺失93例(17.6%)；修订的国际分期系统(Revised International Staging System，R-ISS)分期I期、II、III期分别为5.5%、27.0%、23.1%，缺失235例(44.4%)，其中中南大学湘雅三医院单中心98例NDMM患者中DS分期缺失2.0%，ISS分期缺失12.3%，R-ISS分期缺失12.3%。治疗情况：529例患者中475例接受治疗，接受治疗率为89.8%；67.4%的患者能规范完成4个疗程诱导阶段的化学治疗(以下简称化疗)，其中90.3%的患者接受1次以上以蛋白酶体抑制剂为基础的化疗方案，67.2%接受1次以上以免疫调节剂为基础的化疗方案，59.8%的患者接受1次以上含蛋白酶体抑制剂与免疫调节剂的联合化疗方案。疗效：4个疗程组总缓解率(overall response rate，ORR)及高质量缓解率(high quality response rate，HQR)均优于2个疗程组(ORR为85% vs 65%，P=0.006；HQR为68.3% vs 24.0%，P<0.001)，规范化治疗组HQR优于非规范化治疗组(65.1% vs 48.2%，P=0.035)。治疗期间出现的不良反应：血液学毒性17.5%、外周神经病变24.8%、胃肠道不良反应23.8%、肺部感染25.9%、带状疱疹4.6%、静脉血栓1.7%。结论: 2019年期间湖南省NDMM的漏诊率较高，中位确诊年龄较大；诊断精准度不高，特别是在分期及危险分层上各医疗中心差异较大，将近一半的NDMM患者诊断时没有作出R-ISS分期；细胞遗传学数据缺失，需在后续研究中跟进。NDMM患者就诊时骨痛、贫血症状占比较高；接受治疗的患者中蛋白酶体抑制剂和/或免疫调节剂化疗方案使用率较高，但各医疗中心差距明显，规范化治疗尚待加强；化疗期间的安全性总体可控。.
Keywords: multicenter study; multiple myeloma; real world study.