Moulard Odile, Mehta Jyotsna, Fryzek Jon, Olivares Robert, Iqbal Usman, Mesa Ruben A
Oncology-Global Evidence and Value Development, Medical Affairs, Chilly-Mazarin, France, USA.
Eur J Haematol. 2014 Apr;92(4):289-97. doi: 10.1111/ejh.12256. Epub 2014 Feb 3.
Primary myelofibrosis (PMF), essential thrombocythemia (ET), and polycythemia vera (PV) are BCR ABL-negative myeloproliferative neoplasms (MPN). Published epidemiology data are scarce, and multiple sources are needed to assess the disease burden.
We assembled the most recent information available on the incidence and prevalence of myelofibrosis (MF), ET, and PV by conducting a structured and exhaustive literature review of the published peer-reviewed literature in EMBASE and by reviewing online documentation from disease registries and relevant health registries in European countries. The search was restricted to human studies written in English or French and published between January 1, 2000, and December 6, 2012.
Eleven articles identified from EMBASE, three online hematology or oncology registries, and two Web-based databases or reports were used to summarize epidemiological estimates for MF, PV, and ET. The incidence rate of MF ranged from 0.1 per 100,000 per year to 1 per 100,000 per year. Among the various registries, the incidence of PV ranged from 0.4 per 100,000 per year to 2.8 per 100,000 per year, while the literature estimated the range of PV incidence to be 0.68 per 100,000 to 2.6 per 100,000 per year. The estimated incidence of ET was between 0.38 per 100,000 per year and 1.7 per 100,000 per year. While a few studies reported on the MPNs' prevalences, it is difficult to compare them as various types of prevalence were calculated (point prevalence vs. period prevalence) and standardization was made according to different populations (e.g., the world population and the European population).
There is a wide variation in both prevalence and incidence estimates observed across European data sources. Carefully designed studies, with standardized definitions of MPNs and complete ascertainment of patients including both primary and secondary MFs, should be conducted so that estimates of the population aimed to receive novel treatments for these neoplasms are better understood assist public health planning and provide valuable information about the burden of illness to policy makers, funding agencies, resource planners, healthcare insurers, and pharmaceutical manufacturers.
原发性骨髓纤维化（PMF）、真性红细胞增多症（ET）和真性红细胞增多症（PV）是BCR ABL阴性骨髓增殖性肿瘤（MPN）。已发表的流行病学数据稀缺，需要多个来源来评估疾病负担。
我们通过对EMBASE上已发表的同行评审文献进行结构化和详尽的文献综述，并查阅欧洲国家疾病登记处和相关健康登记处的在线文档，收集了有关骨髓纤维化（MF）、ET和PV发病率和患病率的最新信息。搜索仅限于2000年1月1日至2012年12月6日期间以英文或法文撰写并发表的人体研究。
从EMBASE、三个在线血液学或肿瘤学登记处以及两个基于网络的数据库或报告中确定的11篇文章用于总结MF、PV和ET的流行病学估计。MF的发病率范围为每年每10万人0.1例至每年每10万人1例。在各个登记处中，PV的发病率范围为每年每10万人0.4例至每年每10万人2.8例，而文献估计PV发病率范围为每年每10万人0.68例至每年每10万人2.6例。ET的估计发病率在每年每10万人0.38例至每年每10万人1.7例之间。虽然有一些研究报告了MPN的患病率，但由于计算了不同类型的患病率（点患病率与期间患病率）并根据不同人群（例如世界人口和欧洲人口）进行了标准化，因此难以进行比较。
在欧洲各数据源中观察到的患病率和发病率估计值存在很大差异。应开展精心设计的研究，对MPN进行标准化定义，并全面确定包括原发性和继发性MF在内的患者，以便更好地了解旨在接受这些肿瘤新治疗的人群估计数，协助公共卫生规划，并为政策制定者、资助机构、资源规划者、医疗保险公司和制药制造商提供有关疾病负担的宝贵信息。
