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口服治疗新时代慢性髓性白血病患者生存的决定因素:来自英国基于人群的患者队列的研究结果

Determinants of survival in patients with chronic myeloid leukaemia treated in the new era of oral therapy: findings from a UK population-based patient cohort.

作者信息

Smith A G, Painter D, Howell D A, Evans P, Smith G, Patmore R, Jack A, Roman E

机构信息

Department of Health Sciences, University of York, York, UK.

出版信息

BMJ Open. 2014 Jan 15;4(1):e004266. doi: 10.1136/bmjopen-2013-004266.

Abstract

OBJECTIVES

To examine contemporary survival patterns in the general population of patients diagnosed with chronic myeloid leukaemia (CML), and to identify patient groups with less than optimal outcomes.

DESIGN

Prospective population-based cohort.

SETTING

The UK's Haematological Malignancy Research Network (catchment population 3.6 million, with >2000 new haematological malignancies diagnosed annually).

PARTICIPANTS

All patients newly diagnosed with CML, from September 2004 to August 2011 and followed up to 31 March 2013.

MAIN OUTCOME MEASURE

Incidence and survival.

RESULTS

With a median diagnostic age of 59 years, the CML age standardised (European) incidence was 0.9/100 000 (95% CIs 0.8 to 0.9), 5-year overall survival was 78.9% (72.3 to 84.0) and 5-year relative survival 88.6% (81.0 to 93.3). The efficacy of treatment across all ages was clearly demonstrated; the relative survival curves for those under 60 and over 60 years being closely aligned. Survival findings were similar for men and women, but varied with deprivation; the age and sex adjusted HR being 3.43 (1.89 to 6.22) for deprivation categories 4-5 (less affluent) versus 1-3 (more affluent). None of these differences were attributable to the biological features of the disease.

CONCLUSIONS

When therapy is freely provided, population-based survival for CML is similar to that reported in clinical trials, and age loses its prognostic significance. However, although most of the patients with CML now experience close to normal lifespans, those living in more deprived areas tend to have poorer outcomes, despite receiving the same clinical care. A significant improvement in overall population outcomes could be achieved if these socioeconomic differences, which may reflect the treatment compliance, could be eliminated.

摘要

目的

研究慢性髓系白血病(CML)确诊患者总体人群的当代生存模式,并确定预后欠佳的患者群体。

设计

基于人群的前瞻性队列研究。

背景

英国血液恶性肿瘤研究网络(覆盖人群360万,每年有超过2000例新发血液恶性肿瘤确诊)。

参与者

2004年9月至2011年8月新确诊为CML且随访至2013年3月31日的所有患者。

主要观察指标

发病率和生存率。

结果

CML诊断年龄中位数为59岁,年龄标准化(欧洲)发病率为0.9/10万(95%可信区间0.8至0.9),5年总生存率为78.9%(72.3至84.0),5年相对生存率为88.6%(81.0至93.3)。各年龄段治疗效果均得到明确证明;60岁以下和60岁以上患者的相对生存曲线紧密一致。男性和女性的生存结果相似,但因贫困程度而异;4 - 5类(较贫困)与1 - 3类(较富裕)相比,年龄和性别校正后的风险比为3.43(1.89至6.22)。这些差异均与疾病的生物学特征无关。

结论

当免费提供治疗时,CML的基于人群的生存率与临床试验报告的相似,年龄失去其预后意义。然而,尽管现在大多数CML患者的寿命接近正常,但生活在较贫困地区的患者尽管接受相同的临床护理,预后往往较差。如果能够消除这些可能反映治疗依从性的社会经济差异,总体人群的预后可能会有显著改善。

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