医学
骨髓增生异常综合症
生活质量(医疗保健)
癸他滨
随机对照试验
重症监护医学
临床试验
阿扎胞苷
疾病
贫血
梅德林
儿科
物理疗法
内科学
DNA甲基化
法学
化学
护理部
基因表达
基因
骨髓
生物化学
政治学
作者
Giovanni Caocci,Giorgio La Nasa,Fabio Efficace
标识
DOI:10.1586/17474086.2.1.69
摘要
Health-related quality of life (HRQOL), symptom burden and other types of patient-reported outcomes have acquired an important role in clinical research, as they can provide precious information on the patient's perspective of disease symptoms and treatment-related effects. HRQOL in patients with myelodysplastic syndromes (MDSs) may be compromised for several reasons, including severe anemia, the frequent occurrence of infections and the need for blood transfusions. Many MDS patients are elderly patients who might present with comorbidities from the time of diagnosis. Our investigation started with a systematic search of the literature in which prospective studies were identified and evaluated according to a predefined coding scheme. Both HRQOL outcomes and traditional clinical reported outcomes were systematically analyzed. Overall, we found nine prospective studies, four of which evaluated HRQOL in a randomized controlled trial setting and, interestingly, all these studies were published after the year 2001, possibly reflecting a recent interest in HRQOL research in MDS patients. While methodological drawbacks were identified, mainly in terms of small sample size and amount of missing data, HROQL assessment has been shown to be feasible in MDS patients and there are excellent examples of how this approach can provide additional key outcomes. A good example is the important evidence emerging from two recent randomized controlled trials that HRQOL benefits are obtained with azacitidine and decitabine compared with supportive care. In view of the enormous potential of patient-reported outcome assessment in providing valuable outcomes in support of clinical decision-making, its implementation in future studies of MDS patients is strongly recommended.
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