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40岁之前确诊的年轻多发性骨髓瘤患者的流行病学概况:法国的经验。

Epidemiological landscape of young patients with multiple myeloma diagnosed before 40 years of age: the French experience.

作者信息

Caulier Alexis, Roussel Murielle, Morel Pierre, Lombion Naelle, Branco Benoît, Galtier Jean, Hulin Cyrille, Perrot Aurore, Richez Valentine, Michaud Anne-Victoire, Touzeau Cyrille, Doyen Chantal, Mariette Clara, Caillot Denis, Harel Stéphanie, Lenain Pascal, Ivanoff Sarah, Fontan Jean, Stoppa Anne-Marie, Manier Salomon, Garderet Laurent, Leleu Xavier, Marolleau Jean-Pierre, Arnulf Bertrand, Avet-Loiseau Hervé, Royer Bruno

机构信息

Hematology, CHU Amiens, Amiens, France.

Hematology, CHU Limoges, Limoges, France.

出版信息

Blood. 2021 Dec 23;138(25):2686-2695. doi: 10.1182/blood.2021011285.

Abstract

Multiple myeloma (MM) is rare in young patients, especially before age 40 years at diagnosis, representing <2% of all patients with MM. Little is known about the disease characteristics and prognosis of these patients. In this study, we examined 214 patients diagnosed with MM at age ≤40 years over 15 years, in the era of modern treatments. Among them, 189 patients had symptomatic MM. Disease characteristics were similar to older patients: 35% had anemia, 17% had renal impairment, and 13% had hypercalcemia. The staging was ISS-1 in 52.4%, ISS-2 in 27.5%, and ISS-3 in 20.1%. Overall, 18% of patients had high-risk cytogenetics [del 17p and/or t(4;14)]. Ninety percent of patients received intensive chemotherapy followed by autologous stem cell transplant, and 25% of patients had allogeneic stem cell transplant predominantly at time of relapse. The median follow-up was 76 months, the estimated median overall survival was 14.5 years, and the median progression free-survival was 41 months. In multivariate analysis, bone lesions (hazard ratio [HR], 3.95; P = .01), high ISS score (HR, 2.14; P = .03), and high-risk cytogenetics (HR, 4.54; P < .0001) were significant risk factors for poor outcomes. Among predefined time-dependent covariables, onset of progression (HR, 13.2; P < .0001) significantly shortened overall survival. At 5 years, relative survival compared with same age- and sex-matched individuals was 83.5%, and estimated standardized mortality ratio was 69.9 (95% confidence interval, 52.7-91.1), confirming that MM dramatically shortens the survival of young patients despite an extended survival after diagnosis.

摘要

多发性骨髓瘤(MM)在年轻患者中较为罕见,尤其是诊断时年龄小于40岁的患者,占所有MM患者的比例不到2%。对于这些患者的疾病特征和预后知之甚少。在本研究中,我们调查了在现代治疗时代15年间诊断为MM且年龄≤40岁的214例患者。其中,189例患者患有症状性MM。疾病特征与老年患者相似:35%有贫血,17%有肾功能损害,13%有高钙血症。国际分期系统(ISS)-1期占52.4%,ISS-2期占27.5%,ISS-3期占20.1%。总体而言,18%的患者有高危细胞遗传学异常[17p缺失和/或t(4;14)]。90%的患者接受了强化化疗,随后进行自体干细胞移植,25%的患者主要在复发时进行异基因干细胞移植。中位随访时间为76个月,估计中位总生存期为14.5年,中位无进展生存期为41个月。多变量分析显示,骨病变(风险比[HR],3.95;P = 0.01)、高ISS评分(HR,2.14;P = 0.03)和高危细胞遗传学异常(HR,4.54;P < 0.0001)是预后不良的显著危险因素。在预先定义的时间依赖性协变量中,疾病进展的发生(HR,13.2;P < 0.0001)显著缩短了总生存期。5年时,与年龄和性别匹配的个体相比,相对生存率为83.5%,估计标准化死亡比为69.9(95%置信区间,52.7 - 91.1),证实尽管诊断后生存期有所延长,但MM仍显著缩短了年轻患者的生存期。

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