Alexanian R, Weber D, Delasalle K, Cabanillas F, Dimopoulos M
University of Texas M.D. Anderson Cancer Center, Houston 77030-4009, USA.
Semin Oncol. 2003 Apr;30(2):206-10. doi: 10.1053/sonc.2003.50051.
A study was undertaken to evaluate the frequency and natural history of disease in patients with asymptomatic Waldenstrom's macroglobulinemia (WM). Among 132 consecutive, newly diagnosed patients with monoclonal IgM, 82 (27%) had symptomatic WM indicated by anemia, lymphadenopathy, or splenomegaly. Thirty-one patients had similar clinical features but were asymptomatic and followed without therapy until disease progression. There were 19 patients with monoclonal gammopathy of undetermined significance of IgM type (MGUS). In comparison to overt WM, patients with asymptomatic WM had significantly higher hemoglobin (Hgb) level (median, 12.1 v 9.7 g/dL), lower serum beta(2)-microglobulin (B(2)M) level (median, 2.4 v 3.4 mg/L), and similar IgM peaks (median, 2.2 and 1.8 g/dL). The IgM component was 3.6 g/dL or less in all patients with asymptomatic disease. For asymptomatic WM, median time to disease progression was 6.9 years with rare morbidity. Prognostic factors for early progression were Hgb <11.5 g/dL, B(2)M >or= 3.0 mg/L, and IgM peak >3.0 g/dL. Combinations of these variables defined three risk groups for progression with markedly different median times to progression of >5 years, 2 years, and 0.5 year, respectively. Response rate and survival after institution of treatment were similar to those of patients treated promptly for overt disease. We conclude that, among patients with WM, 27% were asymptomatic with slow disease progression before the need for chemotherapy. Since disease outcomes after treatment were similar to those of patients treated at diagnosis, patients with asymptomatic disease should be identified and followed without treatment for as long as risks of complications remain low.
一项研究旨在评估无症状华氏巨球蛋白血症（WM）患者的疾病发生频率和自然病程。在132例连续新诊断的单克隆IgM患者中，82例（27%）有以贫血、淋巴结病或脾肿大为特征的有症状WM。31例患者有相似的临床特征但无症状，在疾病进展前未接受治疗而进行随访。有19例IgM型意义未明的单克隆丙种球蛋白病（MGUS）患者。与显性WM相比，无症状WM患者的血红蛋白（Hgb）水平显著更高（中位数，12.1对9.7 g/dL），血清β2微球蛋白（B2M）水平更低（中位数，2.4对3.4 mg/L），IgM峰值相似（中位数，2.2和1.8 g/dL）。所有无症状疾病患者的IgM成分均为3.6 g/dL或更低。对于无症状WM，疾病进展的中位时间为6.9年，发病率很低。早期进展的预后因素为Hgb<11.5 g/dL、B2M≥3.0 mg/L和IgM峰值>3.0 g/dL。这些变量的组合定义了三个进展风险组，其进展的中位时间分别显著不同，>5年、2年和0.5年。开始治疗后的缓解率和生存率与显性疾病患者及时治疗后的相似。我们得出结论，在WM患者中，27%无症状，在需要化疗之前疾病进展缓慢。由于治疗后的疾病转归与诊断时治疗的患者相似，应识别无症状疾病患者，并在并发症风险仍然较低时不进行治疗而进行随访。
