Xin-xin Cao, MD1*, Shuhua Yi, MD2*, Zhongxing Jiang, MD3*, Jingsong He, MD4*, Wei Yang5*, Juan Du Jr., MD6*, Chunyan Sun, MD, PhD7*, Yu Wu, MD8, Chen Wenming, MD9*, Xiao-jun Liu10*, Bingzong Li, MD, PhD11, Chunrui Li, MD, PhD12, Wei Sang13*, Qin-hua Liu14*, Xiaoxia Chu15*, Li Fei16, Ou Bai17*, Min Mao18*, Rong Fu, PhD19*, Wei WANG, MD20*, Lihong Liu, MD21*, Luqun Wang, MD, PhD22*, Yujun Dong, MD23*, Jun Luo, DO24*, Zhenling Li25*, Yongqiang Wei26*, Qi-ke Zhang27*, Jing Liu28*, Kaiyang Ding29*, Liang Zou30*, Bi-yun Chen31*, Luoming Hua, PhD32*, Hongmei Jing33*, Juan He, PhD34*, Liang Wang35*, Lugui Qiu, MD36 and Jian Li, MD1
1Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China
2lymphoma and myeloma center, National Clinical Research Center for Blood Diseases, State Key Laboratory of Experimental Hematology, Blood Diseases Hospital & Institute of Hematology, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China
3Department of Hematology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, China
4Department of Hematology, Bone Marrow Transplantation Center, The First Affiliated Hospital of Zhejiang University School of Medicine, Zhejiang, China
5Shengjing Hospital of China Medical University, Shengjing, China
6Department of Hematology, Myeloma & Lymphoma Center, Shanghai Changzheng Hospital, The Second Military Medical University, Shanghai, China
7Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Institute of Hematology, Wuhan, China
8Department of Hematology and Hematology Research Laboratory, West China Hospital, Sichuan University, Chengdu, China
9Department of Hematology, Beijing Chao-Yang Hospital of Capital Medical University, Beijing, China
10Department of Hematology, The Second Hospital of Hebei Medical University, Hebei, China
11Department of Hematology, the Second Affiliated Hospital of Soochow University, Suzhou, Jiangsu, China
12Department of Hematology, Tongji Hospital, Tongji Medical College of Huazhong University of Science and Technology, Wuhan, China
13Department of Hematology, The Affiliated Hospital of Xuzhou Medical University, Xuzhou, China
14Department of Hematology, The First Affiliated Hospital of Anhui Medical University, Anhui, China
15Department of Hematology, Yantai Yuhuangding Hospital, Yantai, China
16the First Affiliated Hospital of Nanchang University, Nanchang, Jiangxi Province, China
17Department of Hematology, The First Affiliated Hopital of Jilin University, Jilin, China
18Department of Hematology, Xinjiang Uiger Municipal People’s Hospital, Xinjiang, China
19Tianjin Medical University General Hospital, Tianjin, China
20Department of Hematology, The Affiliated Hospital of Qingdao University, QING DAO, CHN
21The Fourth Hospital of Hebei Medical University, Shijiazhuang, China
22Department of Hematology, Qilu Hospital of Shandong University, Jinan, Shandong, China
23Department of Hematology, Peking University First Hospital, Beijing, China
24Department of Hematology, The First Affiliated Hospital of Guangxi Medical University, Guangxi, China
25Department of Hematology, China-Japan Friendship Hospital, Beijing, CHN
26Department of Hematology, Nanfang Hospital, Southern Medical University, Guangzhou, China
27Department of Hematology, Gansu Provincial Hospital, Gansu, China
28Department of Hematology, The Third Xiangya Hospital of Central South University, Hunan, China
29The First Affiliated Hospital of University of Science and Technology of China, Hefei, China
30Department of Hematology, Wuhan No.1 Hospital, Wuhan, China
31Department of Hematology, Fujian Provincial Hospital, Fujian, China
32The Affiliated Hospital of Hebei University, Baoding, China
33Peking University Third Hospital, Beijing, China
34Department of Hematology, The First Hospital of China Medical University, Shenyang, CHN
35Department of Hematology, Zhujiang Hospital of Southern Medical University, Guangdong, China
36State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China
Backgroud: Waldenström macroglobulinemia (WM) is an uncommon indolent B cell non-Hodgkin lymphoma, which has heterogeneous clinical presentations and indications for treatment. Mostly the choice of first-line therapy is based on the individual patient’s characteristic and indications for treatment. In China, previous studies on WM are mostly from single-center with small sample size, limiting the information available on treatment and outcome patterns. To address this knowledge gap, we present data from an analysis based on a nationwide multicenter registry with 17-years follow-up. Our study focuses on the clinical presentation, first-line therapies, as well as outcome and prognosis of WM in China.
Methods: Patients diagnosed with WM between January 2003 and December 2019 at 35 academic hospitals in China, which have been entered in the database of the China Waldenström macroglobulinemia Registration (CWMG), were included in this retrospective study. Data including baseline clinical features, symptoms requiring treatment, treatment and survival were collected. The overall survival (OS) was defined as the duration from the diagnosis of WM to the date of death or last follow-up.
Results: Overall 1141 patients were enrolled, 829 patients were male (72.7%), with a male-to-female ratio of 2.7:1. The median age at diagnosis was 64 years (range, 29–89 years), which 472 patients (41.4%) were older than 65 years, and 126 patients (11.0%) were older than 75 years. The patients’ family histories included 6 WM and 4 other lymphoproliferative disorders. Symptoms leading to treatment initiation including anemia in 828 patients (72.6%), organomegaly in 441 patients (38.7%), thrombocytopenia in 302 (26.5%), neutropenia in 246 (21.6%), constitutional symptoms in 203 (17.8%), Bing-Neel syndrome in 13 (1.1%), IgM-related symptoms in including secondary amyloidosis in 32 (2.8%), secondary autoimmune hemolysis in 25 (2.2%), peripheral neuropathy in 23 (2.0%), secondary cold agglutinin disease in 21 (1.8%), secondary cryoglobulinemia in 11 (1.0%). At the time of diagnosis, 1125 patients had full information for IPSS-WM risk stratification. Among them, 194 patients (17.2%) were classified as low risk, 436 patients (38.8%) were intermediate risk, and 495 patients (44.0%) were high risk. Overall, 734 patients had documented treatment information. 75 patients (10.2%) received monotherapy, 264 (36.0%) received chemoimmunotherapy, and 395 (53.8%) receive other combination regimens (Figure 1). The most frequently used monotherapy was chlorambucil (3.1%), followed by ibrutinib (2.9%) and rituximab (2.5%). Rituximab, cyclophosphamide and dexamethasone or prednisone (DRC or RCP) were the most frequently used chemoimmunotherapy (10.8%). Followed by rituximab plus cyclophosphasmide, vincristine/vincristine and prednisone/prednisolone (R-COP) (6.8%), R-COP plus doxorubicin/epirubicin (R-CHOP) (6.1%), rituximab plus fludarabine, cyclophosphamide (R-FC) (4.5%), rituximab plus bortezomib based regimen (3.5%). Other combination regimens including bortezomib based regimen (18.6%), FC (10.6%), CHOP (9.3%), immunomodulatory drug based regimen (5.7%), chlorambucil plus prednisone (4.4%). After a median of 23 months (range 1-201 months) follow-up, 123 patients died. The estimated 5-year OS was 74.9%. Median OS were similar among patients who received monotherapy, chemoimmunotherapy or other combination regimens. To evaluate the prognostic factors of OS using multivariate Cox regression model, age > 65 years old (P＝0.011, HR 0.622, 95% CI 0.431-0.898), platelet < 100×109/L (P＝0.006, HR 0.570, 95% CI 0.381-0.853), serum albumin <3.5 g/dl (P＝0.020, HR 0.582, 95% CI 0.369-0.918), β-2 microglobulin concentration ≥4 mg/L (P＝0.019, HR 0.630, 95% CI 0.429-0.926), LDH≥250 IU/L (P＝0.016, HR 0.538, 95% CI 0.326-0.890) and secondary amyloidosis (P<0.001, HR 0.277, 95% CI 0.137-0.562) at baseline had significantly shorter OS .
Conclusion: Frontline treatment choices of WM are wide heterogeneity due to various clinical presentations and the rarity of the disease. Old age, low platelet, low albumin, high β-2 microglobulin, high LDH and secondary amyloidosis indicate worse prognosis in WM. These findings may provide guidance for management of WM and better prognostic stratification of risk-adapted treatment strategies.
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