骨髓增生异常综合征的负担:流行病学和人文方面的文献综述。

IF 3.5 2区 医学 Q1 MEDICINE, GENERAL & INTERNAL
Shitong Xie, Jiajun Yan, Preston Tse, Brittany Humphries, Feng Xie
{"title":"骨髓增生异常综合征的负担:流行病学和人文方面的文献综述。","authors":"Shitong Xie,&nbsp;Jiajun Yan,&nbsp;Preston Tse,&nbsp;Brittany Humphries,&nbsp;Feng Xie","doi":"10.1111/jebm.70065","DOIUrl":null,"url":null,"abstract":"<p>Myelodysplastic syndromes (MDS) are a group of clonal neoplastic bone marrow disorders characterized by ineffective hematopoiesis and cytopenia [<span>1</span>]. Given the increasing incidence of MDS in aging populations and its chronic disease burden, understanding its epidemiological trends and humanistic burden is crucial. Despite several reviews that have examined epidemiological or humanistic burden of MD, existing literature is often limited in scope, focusing on specific subgroups, for example, higher-risk MDS, or single geographic regions. Furthermore, heterogeneous study methodologies have led to inconsistent estimates of MDS incidence, prevalence, and mortality, complicating comparisons across populations. The aim of this study was to conduct a scoping literature review to summarize both the epidemiological and humanistic burden of MDS.</p><p>A systematic search was conducted to understand the epidemiological, humanistic, and economic burden of MDS. The search was undertaken from inception to July 13, 2021 in Embase and Medline. A detailed search strategy is presented in Table S1. Bibliographic searches of systematic reviews identified during the screening were conducted to validate the search strategy and identify any additional publications that were not retrieved through the original search. Studies were included if they (1) included patients with MDS and assessed epidemiologic (i.e., prevalence, incidence or mortality) or humanistic (i.e., patient-reported outcome using either a generic or disease-specific instrument) outcomes; (2) had a sample of more than 100, and (3) were published in English. Systematic reviews were excluded, but if relevant, their reference lists were cross-referenced. Study selection involved two phases: an initial screening based on title and abstract followed by a full-text review. Both phases of screening were performed by two reviewers independently and in duplicate. Any discrepancies were solved by discussion between the two reviewers or consulting a third reviewer when necessary. A study mapping process was conducted to match publications reporting on the same study. For included studies, information on study and patient characteristics, and outcomes of interest were extracted by two reviewers independently using a standardized data extraction form. Discrepancies were reconciled by discussion between the two reviewers or in consultation with a third reviewer when necessary.</p><p>The study employed descriptive analyses to summarize the epidemiological and HRQoL outcomes in patients with MDS. The descriptive statistics included age-standardized incidence rate (ASIR), prevalence, and survival rates. Data were organized in Excel and visualized with R using distribution plots and trend graphs.</p><p>A total of 63 papers (56 studies) reported on the epidemiological burden of MDS and 57 papers (46 studies) on the HRQoL burden of MDS. The selection process is summarized in Figure S1. Details on studies reporting epidemiological and HRQoL outcomes are provided in Tables S2 and S3, respectively.</p><p>As shown in Table 1, among the 56 studies reporting epidemiological outcomes, 40 (71.4%) were database analyses, 11 (19.6%) retrospective cohort studies, and 5 (8.9%) prospective cohort studies. Studies were conducted across diverse regions: 30 (53%) studies in Europe, 13 (23%) in North America, 7 (13%) in Asia, and the rest in Oceania, South America, and Africa. Median age of patients at the diagnosis of MDS ranged from 62 to 79 years. The adult ASIR, reported in 16 studies for 12 countries, varied from 0.98 in Korea to 11.5 per 100,000 in Argentina (Figure S2a. ASIR was higher in males (1.0–7.9 per 100,000) than in females (0.5–6.2 per 100,000) (Figure S2b). Four studies showed ASIR rising with age, from 0.3–0.9 per 100,000 in those under 50 to 28.5–84.4 per 100,000 in patients over 80 (Figure S2c). France and the United Kingdom reported ASIRs of 0.03 and 0.04 per 100,000 for pediatric patients. Crude prevalence ranged from 6.2–12.0 per 100,000 in registries to 155 per 100,000 in claims data (Table S2). One-year survival ranged from 66%–69%, while 3-year and 5-year survival rates exhibited ranges of 42% to 45%, and 27% to 46%, respectively. Both 3-year and 5-year survival rates declined as age increased. Age-standardized mortality rates were 1.14–1.63 per 100,000, with crude mortality from 0.15 for very low-risk MDS to 6.44 for very high-risk disease.</p><p>Among the 46 studies reporting HRQoL outcomes (Table S3), 22 (48%) studies were conducted in Europe and North America, 9 (20%) studies were conducted in multiple countries or regions, and 2 studies were conducted in Turkey and Israel. Twenty-two studies (48%) were conducted among patients with MDS without risk stratifications, while 19 (41%) studies specifically focused on patients with low to intermediate risk of MDS, and 5 (11%) studies on higher risk of MDS. Twenty-four studies (52%) focused on HRQoL among patients with MDS, and 22 (48%) specifically assessed treatment-related HRQoL. The most frequently used cancer-specific HRQoL instruments were the European Organization for Research and Treatment of Cancer Quality of Life questionnaire-Core 30 (EORTC QLQ-C30) and Functional Assessment of Cancer Therapy—Anemia (FACT-AN), together accounting for 55% of the included studies (Figure S3). Another commonly used generic HRQoL instrument was the EQ-5D-3L, which was used in 19% of included studies. The global score of the EORTC QLQ-C30 was reported in 13 studies. As shown in Figure S4a, the global score ranged from 50.6 to 66.7 at baseline. For patients with lower-risk MDS, the score ranged from 52.2 to 62.3, and for those with higher-risk MDS, the range was 50.6 to 59.7. There were 11 studies reporting EORTC QLQ-C30 dimension scores (Figure S4b). Physical functioning and role functioning were identified as the most affected domains, and cognitive functioning and social functioning were the least affected. Fatigue, pain, and appetite loss were the most severe symptoms, with nausea/vomiting being the least severe. The FACT-AN total score related to MDS was assessed in five unique studies (Figure S5). The FACT-AN total score ranged from 111 to 132, suggesting a moderate level of quality of life for patients with MDS. Health utility was measured in six studies, using the EQ-5D-3L in all six studies and EORTC Quality of Life Utility Core 10 Dimensions (QLU-C10D) in one study (Figure S6) [<span>1-7</span>]. The range of the health utility values was 0.73 to 0.83 measured by the EQ-5D-3L, and 0.73 to 0.77 measured by the EORTC QLU-C10D.</p><p>This study summarizes the evidence landscape on the epidemiological and humanistic burden of MDS. As the average age of populations in countries around the world continues to rise, the burden of MDS is expected to increase. Not only does MDS have a high mortality rate, it exerts a substantial impact on patients’ humanistic burden, which is affected by both disease- and treatment-specific considerations. However, the evidence base, particularly relating to epidemiology, is heterogeneous and disease-specific measures evaluating humanistic burden are lacking.</p><p>Determining the incidence and prevalence of MDS is crucial for understanding disease burden and optimizing healthcare resource allocation [<span>8</span>]. We found that ASIR ranges from 0.98 to 11.5 per 100,000, with higher rates among older adults and males. In a previous review that used the Surveillance, Epidemiology, and End Results data (SEER) data, Zeidan et al. similarly reported rising ASIR with age and a male predominance (4.7–7.7 per 100,000 in males vs. 2.5–3.4 per 100,000 in females) [<span>9</span>]. Zeidan et al. also reported that Asians have the lowest ASIR compared with other ethnicities, especially white [<span>9</span>]. However, data stratified by race and from non-Western countries remain scarce. This is an important gap as racialized groups remain underrepresented in drug trials in MDS [<span>10</span>]. Overall, our results align with a previous review, which reported the crude global prevalence of MDS as ranging from 0.22 to 13.2 per 100,000 across age groups, genders, and ethnicities [<span>11</span>]. Variation in study design and population characteristics makes comparison of these estimates across studies challenging [<span>11</span>]. MDS Survival remains poor, with 5-year survival ranging from 27% in Australia to 46% in Korea, varying by patient characteristics (e.g., age, sex) and disease risk (e.g., IPSS-R classification).</p><p>The EORTC QLQ-C30 and FACT-AN were the most commonly used instruments to measure HRQoL among patients with MDS [<span>12, 13</span>]. These well-established, validated, instruments for cancer patients are sometimes used alongside generic measures of HRQoL, such as the EQ-5D. Given the disease- and treatment-related effects on patients’ HRQoL, MDS-specific instruments may be better suited to capture the humanistic burden experienced by this patient population. Efforts are underway to develop such tools, including the MD Anderson Symptom Inventory (MDASI) for MDS/AML, the Quality of Life in Myelodysplasia Scale (QUALMS) and the 29-item psychometric Quality of Life E (QOL-E). Generally speaking, disease-specific instruments are more sensitive to different aspects of HRQoL and can provide more nuanced insight into the impacts of a disease and/or treatment on patients. Future studies should aim to validate these new disease-specific instruments among patients with MDS to facilitate their incorporation into randomized trials and economic evaluations of new and existing therapies.</p><p>The results of this review are subject to several limitations. First, the studies included were heterogeneous in terms of their study design, patient profile, and treatments received. As a result, data were summarized by subgroup when possible and without adjustment for differences. Note this is an inherent limitation of the current body of evidence and not of the review itself. Second, this review focused on patient burden only. It has been reported that, in certain cases, the burden of MDS is even more severe for caregivers than patients. Particularly with respect to mental health. Future research should seek to summarize caregiver burden for a more comprehensive understanding of the humanistic burden of disease.</p><p>The authors declare no conflicts of interest.</p>","PeriodicalId":16090,"journal":{"name":"Journal of Evidence‐Based Medicine","volume":"18 3","pages":""},"PeriodicalIF":3.5000,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/jebm.70065","citationCount":"0","resultStr":"{\"title\":\"Burden of Myelodysplastic Syndromes: A Literature Review of Epidemiological and Humanistic Aspects\",\"authors\":\"Shitong Xie,&nbsp;Jiajun Yan,&nbsp;Preston Tse,&nbsp;Brittany Humphries,&nbsp;Feng Xie\",\"doi\":\"10.1111/jebm.70065\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p>Myelodysplastic syndromes (MDS) are a group of clonal neoplastic bone marrow disorders characterized by ineffective hematopoiesis and cytopenia [<span>1</span>]. Given the increasing incidence of MDS in aging populations and its chronic disease burden, understanding its epidemiological trends and humanistic burden is crucial. Despite several reviews that have examined epidemiological or humanistic burden of MD, existing literature is often limited in scope, focusing on specific subgroups, for example, higher-risk MDS, or single geographic regions. Furthermore, heterogeneous study methodologies have led to inconsistent estimates of MDS incidence, prevalence, and mortality, complicating comparisons across populations. The aim of this study was to conduct a scoping literature review to summarize both the epidemiological and humanistic burden of MDS.</p><p>A systematic search was conducted to understand the epidemiological, humanistic, and economic burden of MDS. The search was undertaken from inception to July 13, 2021 in Embase and Medline. A detailed search strategy is presented in Table S1. Bibliographic searches of systematic reviews identified during the screening were conducted to validate the search strategy and identify any additional publications that were not retrieved through the original search. Studies were included if they (1) included patients with MDS and assessed epidemiologic (i.e., prevalence, incidence or mortality) or humanistic (i.e., patient-reported outcome using either a generic or disease-specific instrument) outcomes; (2) had a sample of more than 100, and (3) were published in English. Systematic reviews were excluded, but if relevant, their reference lists were cross-referenced. Study selection involved two phases: an initial screening based on title and abstract followed by a full-text review. Both phases of screening were performed by two reviewers independently and in duplicate. Any discrepancies were solved by discussion between the two reviewers or consulting a third reviewer when necessary. A study mapping process was conducted to match publications reporting on the same study. For included studies, information on study and patient characteristics, and outcomes of interest were extracted by two reviewers independently using a standardized data extraction form. Discrepancies were reconciled by discussion between the two reviewers or in consultation with a third reviewer when necessary.</p><p>The study employed descriptive analyses to summarize the epidemiological and HRQoL outcomes in patients with MDS. The descriptive statistics included age-standardized incidence rate (ASIR), prevalence, and survival rates. Data were organized in Excel and visualized with R using distribution plots and trend graphs.</p><p>A total of 63 papers (56 studies) reported on the epidemiological burden of MDS and 57 papers (46 studies) on the HRQoL burden of MDS. The selection process is summarized in Figure S1. Details on studies reporting epidemiological and HRQoL outcomes are provided in Tables S2 and S3, respectively.</p><p>As shown in Table 1, among the 56 studies reporting epidemiological outcomes, 40 (71.4%) were database analyses, 11 (19.6%) retrospective cohort studies, and 5 (8.9%) prospective cohort studies. Studies were conducted across diverse regions: 30 (53%) studies in Europe, 13 (23%) in North America, 7 (13%) in Asia, and the rest in Oceania, South America, and Africa. Median age of patients at the diagnosis of MDS ranged from 62 to 79 years. The adult ASIR, reported in 16 studies for 12 countries, varied from 0.98 in Korea to 11.5 per 100,000 in Argentina (Figure S2a. ASIR was higher in males (1.0–7.9 per 100,000) than in females (0.5–6.2 per 100,000) (Figure S2b). Four studies showed ASIR rising with age, from 0.3–0.9 per 100,000 in those under 50 to 28.5–84.4 per 100,000 in patients over 80 (Figure S2c). France and the United Kingdom reported ASIRs of 0.03 and 0.04 per 100,000 for pediatric patients. Crude prevalence ranged from 6.2–12.0 per 100,000 in registries to 155 per 100,000 in claims data (Table S2). One-year survival ranged from 66%–69%, while 3-year and 5-year survival rates exhibited ranges of 42% to 45%, and 27% to 46%, respectively. Both 3-year and 5-year survival rates declined as age increased. Age-standardized mortality rates were 1.14–1.63 per 100,000, with crude mortality from 0.15 for very low-risk MDS to 6.44 for very high-risk disease.</p><p>Among the 46 studies reporting HRQoL outcomes (Table S3), 22 (48%) studies were conducted in Europe and North America, 9 (20%) studies were conducted in multiple countries or regions, and 2 studies were conducted in Turkey and Israel. Twenty-two studies (48%) were conducted among patients with MDS without risk stratifications, while 19 (41%) studies specifically focused on patients with low to intermediate risk of MDS, and 5 (11%) studies on higher risk of MDS. Twenty-four studies (52%) focused on HRQoL among patients with MDS, and 22 (48%) specifically assessed treatment-related HRQoL. The most frequently used cancer-specific HRQoL instruments were the European Organization for Research and Treatment of Cancer Quality of Life questionnaire-Core 30 (EORTC QLQ-C30) and Functional Assessment of Cancer Therapy—Anemia (FACT-AN), together accounting for 55% of the included studies (Figure S3). Another commonly used generic HRQoL instrument was the EQ-5D-3L, which was used in 19% of included studies. The global score of the EORTC QLQ-C30 was reported in 13 studies. As shown in Figure S4a, the global score ranged from 50.6 to 66.7 at baseline. For patients with lower-risk MDS, the score ranged from 52.2 to 62.3, and for those with higher-risk MDS, the range was 50.6 to 59.7. There were 11 studies reporting EORTC QLQ-C30 dimension scores (Figure S4b). Physical functioning and role functioning were identified as the most affected domains, and cognitive functioning and social functioning were the least affected. Fatigue, pain, and appetite loss were the most severe symptoms, with nausea/vomiting being the least severe. The FACT-AN total score related to MDS was assessed in five unique studies (Figure S5). The FACT-AN total score ranged from 111 to 132, suggesting a moderate level of quality of life for patients with MDS. Health utility was measured in six studies, using the EQ-5D-3L in all six studies and EORTC Quality of Life Utility Core 10 Dimensions (QLU-C10D) in one study (Figure S6) [<span>1-7</span>]. The range of the health utility values was 0.73 to 0.83 measured by the EQ-5D-3L, and 0.73 to 0.77 measured by the EORTC QLU-C10D.</p><p>This study summarizes the evidence landscape on the epidemiological and humanistic burden of MDS. As the average age of populations in countries around the world continues to rise, the burden of MDS is expected to increase. Not only does MDS have a high mortality rate, it exerts a substantial impact on patients’ humanistic burden, which is affected by both disease- and treatment-specific considerations. However, the evidence base, particularly relating to epidemiology, is heterogeneous and disease-specific measures evaluating humanistic burden are lacking.</p><p>Determining the incidence and prevalence of MDS is crucial for understanding disease burden and optimizing healthcare resource allocation [<span>8</span>]. We found that ASIR ranges from 0.98 to 11.5 per 100,000, with higher rates among older adults and males. In a previous review that used the Surveillance, Epidemiology, and End Results data (SEER) data, Zeidan et al. similarly reported rising ASIR with age and a male predominance (4.7–7.7 per 100,000 in males vs. 2.5–3.4 per 100,000 in females) [<span>9</span>]. Zeidan et al. also reported that Asians have the lowest ASIR compared with other ethnicities, especially white [<span>9</span>]. However, data stratified by race and from non-Western countries remain scarce. This is an important gap as racialized groups remain underrepresented in drug trials in MDS [<span>10</span>]. Overall, our results align with a previous review, which reported the crude global prevalence of MDS as ranging from 0.22 to 13.2 per 100,000 across age groups, genders, and ethnicities [<span>11</span>]. Variation in study design and population characteristics makes comparison of these estimates across studies challenging [<span>11</span>]. MDS Survival remains poor, with 5-year survival ranging from 27% in Australia to 46% in Korea, varying by patient characteristics (e.g., age, sex) and disease risk (e.g., IPSS-R classification).</p><p>The EORTC QLQ-C30 and FACT-AN were the most commonly used instruments to measure HRQoL among patients with MDS [<span>12, 13</span>]. These well-established, validated, instruments for cancer patients are sometimes used alongside generic measures of HRQoL, such as the EQ-5D. Given the disease- and treatment-related effects on patients’ HRQoL, MDS-specific instruments may be better suited to capture the humanistic burden experienced by this patient population. Efforts are underway to develop such tools, including the MD Anderson Symptom Inventory (MDASI) for MDS/AML, the Quality of Life in Myelodysplasia Scale (QUALMS) and the 29-item psychometric Quality of Life E (QOL-E). Generally speaking, disease-specific instruments are more sensitive to different aspects of HRQoL and can provide more nuanced insight into the impacts of a disease and/or treatment on patients. Future studies should aim to validate these new disease-specific instruments among patients with MDS to facilitate their incorporation into randomized trials and economic evaluations of new and existing therapies.</p><p>The results of this review are subject to several limitations. First, the studies included were heterogeneous in terms of their study design, patient profile, and treatments received. As a result, data were summarized by subgroup when possible and without adjustment for differences. Note this is an inherent limitation of the current body of evidence and not of the review itself. Second, this review focused on patient burden only. It has been reported that, in certain cases, the burden of MDS is even more severe for caregivers than patients. Particularly with respect to mental health. 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摘要

骨髓增生异常综合征(MDS)是一组以造血功能低下和细胞减少为特征的克隆性肿瘤骨髓疾病。鉴于MDS在老年人群中发病率的增加及其慢性疾病负担,了解其流行病学趋势和人文负担至关重要。尽管有几篇综述研究了MD的流行病学或人文负担,但现有文献的范围往往有限,主要集中在特定的亚群,例如高危MDS或单一地理区域。此外,异质性研究方法导致MDS发病率、患病率和死亡率的估计不一致,使人群间的比较复杂化。本研究的目的是进行大范围的文献综述,总结MDS的流行病学和人文负担。为了了解MDS的流行病学、人文和经济负担,进行了系统的检索。搜索从开始到2021年7月13日在Embase和Medline进行。表S1给出了详细的搜索策略。对筛选过程中确定的系统综述进行书目检索,以验证检索策略,并确定未通过原始检索检索到的任何其他出版物。纳入以下研究:(1)纳入MDS患者并评估流行病学(即患病率、发病率或死亡率)或人文病学(即使用通用或疾病特异性工具的患者报告结果)结果;(2)样本超过100人,(3)以英文出版。系统评价被排除在外,但如果相关,他们的参考文献列表被交叉引用。研究选择包括两个阶段:基于标题和摘要的初步筛选,然后是全文审查。两个阶段的筛选都是由两个独立的审稿人进行的,并且是重复的。任何差异均由两位审稿人讨论解决,必要时可咨询第三方审稿人。对同一研究的出版物进行了研究映射过程的匹配。对于纳入的研究,有关研究和患者特征的信息以及感兴趣的结果由两名评论者使用标准化数据提取表独立提取。通过两位审稿人之间的讨论或必要时与第三位审稿人协商来协调差异。本研究采用描述性分析对MDS患者的流行病学和HRQoL结果进行总结。描述性统计包括年龄标准化发病率(ASIR)、患病率和生存率。数据在Excel中组织,并使用R使用分布图和趋势图进行可视化。共有63篇论文(56项研究)报道MDS的流行病学负担,57篇论文(46项研究)报道MDS的HRQoL负担。选择过程如图S1所示。报告流行病学和HRQoL结果的研究详情分别见表S2和表S3。如表1所示,56项报告流行病学结果的研究中,数据库分析40项(71.4%),回顾性队列研究11项(19.6%),前瞻性队列研究5项(8.9%)。研究在不同地区进行:欧洲30项(53%)研究,北美13项(23%)研究,亚洲7项(13%)研究,其余在大洋洲、南美洲和非洲进行。诊断为MDS的患者中位年龄为62岁至79岁。12个国家的16项研究报告了成人ASIR,从韩国的0.98到阿根廷的11.5 / 100000不等(图S2a)。男性的ASIR(1.0-7.9 / 100000)高于女性(0.5-6.2 / 100000)(图S2b)。四项研究显示ASIR随年龄增长而上升,从50岁以下患者的0.3-0.9 / 10万增加到80岁以上患者的28.5-84.4 / 10万(图S2c)。法国和英国报告的儿科患者asir分别为0.03 / 100,000和0.04 / 100,000。粗略患病率从登记数据中的每10万人6.2-12.0人到索赔数据中的每10万人155人不等(表S2)。1年生存率为66%-69%,3年和5年生存率分别为42% - 45%和27% - 46%。3年和5年生存率均随年龄增长而下降。年龄标准化死亡率为1.14-1.63 / 10万,粗死亡率从极低风险MDS的0.15到极高风险疾病的6.44。在报告HRQoL结果的46项研究中(表S3), 22项(48%)研究在欧洲和北美进行,9项(20%)研究在多个国家或地区进行,2项研究在土耳其和以色列进行。22项研究(48%)在没有风险分层的MDS患者中进行,19项(41%)研究专门针对低至中度MDS风险患者,5项(11%)研究针对高风险MDS患者。 24项研究(52%)关注MDS患者的HRQoL, 22项研究(48%)专门评估治疗相关的HRQoL。最常用的癌症特异性HRQoL工具是欧洲癌症研究和治疗组织生活质量问卷- core 30 (EORTC QLQ-C30)和癌症治疗-贫血功能评估(FACT-AN),共占纳入研究的55%(图S3)。另一种常用的通用HRQoL仪器是EQ-5D-3L,在19%的纳入研究中使用。13项研究报告了EORTC QLQ-C30的整体评分。如图S4a所示,基线时的总体评分范围为50.6 - 66.7。对于低风险MDS患者,评分范围为52.2 - 62.3,对于高风险MDS患者,评分范围为50.6 - 59.7。有11项研究报告了EORTC QLQ-C30维度得分(图S4b)。身体功能和角色功能被认为是受影响最大的领域,认知功能和社会功能受到的影响最小。疲劳、疼痛和食欲减退是最严重的症状,恶心/呕吐是最不严重的。在五项独特的研究中评估了与MDS相关的FACT-AN总分(图S5)。FACT-AN总分在111 - 132之间,表明MDS患者的生活质量处于中等水平。在六项研究中测量了健康效用,在所有六项研究中使用EQ-5D-3L,在一项研究中使用EORTC生活质量效用核心10维度(qu - c10d)(图S6)[1-7]。EQ-5D-3L测量的健康效用值范围为0.73 ~ 0.83,EORTC qu - c10d测量的健康效用值范围为0.73 ~ 0.77。本研究总结了MDS的流行病学和人文负担的证据格局。随着世界各国人口平均年龄的不断上升,MDS的负担预计会增加。MDS不仅死亡率高,还会对患者的人文负担产生重大影响,这受到疾病和治疗特异性因素的影响。然而,证据基础,特别是与流行病学有关的证据基础是异质性的,并且缺乏评估人文负担的疾病特异性措施。确定MDS的发病率和患病率对于了解疾病负担和优化医疗资源配置至关重要[10]。我们发现ASIR在每10万人中0.98到11.5之间,老年人和男性的比例更高。在先前使用监测、流行病学和最终结果数据(SEER)数据的综述中,Zeidan等人类似地报告了ASIR随年龄和男性优势而上升(男性为4.7-7.7 / 10万,女性为2.5-3.4 / 10万)。Zeidan等人还报道,与其他种族相比,亚洲人的ASIR最低,尤其是白人。然而,按种族分层和来自非西方国家的数据仍然很少。这是一个重要的差距,因为种族化群体在MDS的药物试验中仍然代表性不足。总体而言,我们的研究结果与之前的一项综述一致,该综述报道了MDS的全球粗患病率,在年龄组、性别和种族中为0.22至13.2 / 100,000。研究设计和人群特征的变化使得这些估计在具有挑战性的研究中进行比较。MDS的生存率仍然很低,5年生存率从澳大利亚的27%到韩国的46%不等,因患者特征(如年龄、性别)和疾病风险(如IPSS-R分类)而异。EORTC QLQ-C30和FACT-AN是MDS患者最常用的HRQoL测量仪器[12,13]。这些完善的、经过验证的癌症患者仪器有时与一般的HRQoL测量方法(如EQ-5D)一起使用。鉴于疾病和治疗对患者HRQoL的相关影响,mds特异性仪器可能更适合于捕捉该患者群体所经历的人文负担。正在努力开发这样的工具,包括MDS/AML的MD安德森症状量表(MDASI),骨髓增生异常生活质量量表(QUALMS)和29项心理测量生活质量E (QOL-E)。一般来说,疾病特异性仪器对HRQoL的不同方面更敏感,可以更细致地了解疾病和/或治疗对患者的影响。未来的研究应致力于在MDS患者中验证这些新的疾病特异性仪器,以便将其纳入随机试验和新的和现有疗法的经济评估。本综述的结果受到一些限制。首先,纳入的研究在研究设计、患者概况和接受的治疗方面具有异质性。因此,数据尽可能按亚组汇总,不作差异调整。 请注意,这是当前证据体的固有限制,而不是审查本身的限制。其次,本综述仅关注患者负担。据报道,在某些情况下,MDS对护理人员的负担甚至比患者更严重。特别是在心理健康方面。未来的研究应寻求总结照顾者负担,以更全面地了解疾病的人文负担。作者声明无利益冲突。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

Burden of Myelodysplastic Syndromes: A Literature Review of Epidemiological and Humanistic Aspects

Burden of Myelodysplastic Syndromes: A Literature Review of Epidemiological and Humanistic Aspects

Myelodysplastic syndromes (MDS) are a group of clonal neoplastic bone marrow disorders characterized by ineffective hematopoiesis and cytopenia [1]. Given the increasing incidence of MDS in aging populations and its chronic disease burden, understanding its epidemiological trends and humanistic burden is crucial. Despite several reviews that have examined epidemiological or humanistic burden of MD, existing literature is often limited in scope, focusing on specific subgroups, for example, higher-risk MDS, or single geographic regions. Furthermore, heterogeneous study methodologies have led to inconsistent estimates of MDS incidence, prevalence, and mortality, complicating comparisons across populations. The aim of this study was to conduct a scoping literature review to summarize both the epidemiological and humanistic burden of MDS.

A systematic search was conducted to understand the epidemiological, humanistic, and economic burden of MDS. The search was undertaken from inception to July 13, 2021 in Embase and Medline. A detailed search strategy is presented in Table S1. Bibliographic searches of systematic reviews identified during the screening were conducted to validate the search strategy and identify any additional publications that were not retrieved through the original search. Studies were included if they (1) included patients with MDS and assessed epidemiologic (i.e., prevalence, incidence or mortality) or humanistic (i.e., patient-reported outcome using either a generic or disease-specific instrument) outcomes; (2) had a sample of more than 100, and (3) were published in English. Systematic reviews were excluded, but if relevant, their reference lists were cross-referenced. Study selection involved two phases: an initial screening based on title and abstract followed by a full-text review. Both phases of screening were performed by two reviewers independently and in duplicate. Any discrepancies were solved by discussion between the two reviewers or consulting a third reviewer when necessary. A study mapping process was conducted to match publications reporting on the same study. For included studies, information on study and patient characteristics, and outcomes of interest were extracted by two reviewers independently using a standardized data extraction form. Discrepancies were reconciled by discussion between the two reviewers or in consultation with a third reviewer when necessary.

The study employed descriptive analyses to summarize the epidemiological and HRQoL outcomes in patients with MDS. The descriptive statistics included age-standardized incidence rate (ASIR), prevalence, and survival rates. Data were organized in Excel and visualized with R using distribution plots and trend graphs.

A total of 63 papers (56 studies) reported on the epidemiological burden of MDS and 57 papers (46 studies) on the HRQoL burden of MDS. The selection process is summarized in Figure S1. Details on studies reporting epidemiological and HRQoL outcomes are provided in Tables S2 and S3, respectively.

As shown in Table 1, among the 56 studies reporting epidemiological outcomes, 40 (71.4%) were database analyses, 11 (19.6%) retrospective cohort studies, and 5 (8.9%) prospective cohort studies. Studies were conducted across diverse regions: 30 (53%) studies in Europe, 13 (23%) in North America, 7 (13%) in Asia, and the rest in Oceania, South America, and Africa. Median age of patients at the diagnosis of MDS ranged from 62 to 79 years. The adult ASIR, reported in 16 studies for 12 countries, varied from 0.98 in Korea to 11.5 per 100,000 in Argentina (Figure S2a. ASIR was higher in males (1.0–7.9 per 100,000) than in females (0.5–6.2 per 100,000) (Figure S2b). Four studies showed ASIR rising with age, from 0.3–0.9 per 100,000 in those under 50 to 28.5–84.4 per 100,000 in patients over 80 (Figure S2c). France and the United Kingdom reported ASIRs of 0.03 and 0.04 per 100,000 for pediatric patients. Crude prevalence ranged from 6.2–12.0 per 100,000 in registries to 155 per 100,000 in claims data (Table S2). One-year survival ranged from 66%–69%, while 3-year and 5-year survival rates exhibited ranges of 42% to 45%, and 27% to 46%, respectively. Both 3-year and 5-year survival rates declined as age increased. Age-standardized mortality rates were 1.14–1.63 per 100,000, with crude mortality from 0.15 for very low-risk MDS to 6.44 for very high-risk disease.

Among the 46 studies reporting HRQoL outcomes (Table S3), 22 (48%) studies were conducted in Europe and North America, 9 (20%) studies were conducted in multiple countries or regions, and 2 studies were conducted in Turkey and Israel. Twenty-two studies (48%) were conducted among patients with MDS without risk stratifications, while 19 (41%) studies specifically focused on patients with low to intermediate risk of MDS, and 5 (11%) studies on higher risk of MDS. Twenty-four studies (52%) focused on HRQoL among patients with MDS, and 22 (48%) specifically assessed treatment-related HRQoL. The most frequently used cancer-specific HRQoL instruments were the European Organization for Research and Treatment of Cancer Quality of Life questionnaire-Core 30 (EORTC QLQ-C30) and Functional Assessment of Cancer Therapy—Anemia (FACT-AN), together accounting for 55% of the included studies (Figure S3). Another commonly used generic HRQoL instrument was the EQ-5D-3L, which was used in 19% of included studies. The global score of the EORTC QLQ-C30 was reported in 13 studies. As shown in Figure S4a, the global score ranged from 50.6 to 66.7 at baseline. For patients with lower-risk MDS, the score ranged from 52.2 to 62.3, and for those with higher-risk MDS, the range was 50.6 to 59.7. There were 11 studies reporting EORTC QLQ-C30 dimension scores (Figure S4b). Physical functioning and role functioning were identified as the most affected domains, and cognitive functioning and social functioning were the least affected. Fatigue, pain, and appetite loss were the most severe symptoms, with nausea/vomiting being the least severe. The FACT-AN total score related to MDS was assessed in five unique studies (Figure S5). The FACT-AN total score ranged from 111 to 132, suggesting a moderate level of quality of life for patients with MDS. Health utility was measured in six studies, using the EQ-5D-3L in all six studies and EORTC Quality of Life Utility Core 10 Dimensions (QLU-C10D) in one study (Figure S6) [1-7]. The range of the health utility values was 0.73 to 0.83 measured by the EQ-5D-3L, and 0.73 to 0.77 measured by the EORTC QLU-C10D.

This study summarizes the evidence landscape on the epidemiological and humanistic burden of MDS. As the average age of populations in countries around the world continues to rise, the burden of MDS is expected to increase. Not only does MDS have a high mortality rate, it exerts a substantial impact on patients’ humanistic burden, which is affected by both disease- and treatment-specific considerations. However, the evidence base, particularly relating to epidemiology, is heterogeneous and disease-specific measures evaluating humanistic burden are lacking.

Determining the incidence and prevalence of MDS is crucial for understanding disease burden and optimizing healthcare resource allocation [8]. We found that ASIR ranges from 0.98 to 11.5 per 100,000, with higher rates among older adults and males. In a previous review that used the Surveillance, Epidemiology, and End Results data (SEER) data, Zeidan et al. similarly reported rising ASIR with age and a male predominance (4.7–7.7 per 100,000 in males vs. 2.5–3.4 per 100,000 in females) [9]. Zeidan et al. also reported that Asians have the lowest ASIR compared with other ethnicities, especially white [9]. However, data stratified by race and from non-Western countries remain scarce. This is an important gap as racialized groups remain underrepresented in drug trials in MDS [10]. Overall, our results align with a previous review, which reported the crude global prevalence of MDS as ranging from 0.22 to 13.2 per 100,000 across age groups, genders, and ethnicities [11]. Variation in study design and population characteristics makes comparison of these estimates across studies challenging [11]. MDS Survival remains poor, with 5-year survival ranging from 27% in Australia to 46% in Korea, varying by patient characteristics (e.g., age, sex) and disease risk (e.g., IPSS-R classification).

The EORTC QLQ-C30 and FACT-AN were the most commonly used instruments to measure HRQoL among patients with MDS [12, 13]. These well-established, validated, instruments for cancer patients are sometimes used alongside generic measures of HRQoL, such as the EQ-5D. Given the disease- and treatment-related effects on patients’ HRQoL, MDS-specific instruments may be better suited to capture the humanistic burden experienced by this patient population. Efforts are underway to develop such tools, including the MD Anderson Symptom Inventory (MDASI) for MDS/AML, the Quality of Life in Myelodysplasia Scale (QUALMS) and the 29-item psychometric Quality of Life E (QOL-E). Generally speaking, disease-specific instruments are more sensitive to different aspects of HRQoL and can provide more nuanced insight into the impacts of a disease and/or treatment on patients. Future studies should aim to validate these new disease-specific instruments among patients with MDS to facilitate their incorporation into randomized trials and economic evaluations of new and existing therapies.

The results of this review are subject to several limitations. First, the studies included were heterogeneous in terms of their study design, patient profile, and treatments received. As a result, data were summarized by subgroup when possible and without adjustment for differences. Note this is an inherent limitation of the current body of evidence and not of the review itself. Second, this review focused on patient burden only. It has been reported that, in certain cases, the burden of MDS is even more severe for caregivers than patients. Particularly with respect to mental health. Future research should seek to summarize caregiver burden for a more comprehensive understanding of the humanistic burden of disease.

The authors declare no conflicts of interest.

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来源期刊
Journal of Evidence‐Based Medicine
Journal of Evidence‐Based Medicine MEDICINE, GENERAL & INTERNAL-
CiteScore
11.20
自引率
1.40%
发文量
42
期刊介绍: The Journal of Evidence-Based Medicine (EMB) is an esteemed international healthcare and medical decision-making journal, dedicated to publishing groundbreaking research outcomes in evidence-based decision-making, research, practice, and education. Serving as the official English-language journal of the Cochrane China Centre and West China Hospital of Sichuan University, we eagerly welcome editorials, commentaries, and systematic reviews encompassing various topics such as clinical trials, policy, drug and patient safety, education, and knowledge translation.
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