García-Muñoz Ana María, Victoria-Montesinos Desirée, Cerdá Begoña, Ballester Pura, de Velasco Eloisa María, Zafrilla Pilar
Faculty of Pharmacy and Nutrition, Universidad Católica San Antonio de Murcia (UCAM), Campus de los Jerónimos, 30107 Murcia, Spain.
Healthcare (Basel). 2023 May 31;11(11):1609. doi: 10.3390/healthcare11111609.
The visibility of Rare Diseases is a new challenge for society. These diseases are numerous, heterogeneous in nature and distribution, characterized by a high mortality rate but low prevalence, and usually presenting a severe evolution. Adherence to medication studies in rare diseases are uncommon, due to treatment scarcity.
The main purpose of this study is to do a meta-analysis, evaluating the level of adherence to medication in the most prevalent rare diseases.
This work is a systematic review, and meta-analysis was registered in the International Prospective Register of Systematic Reviews (PROSPERO) (Registration number: CRD42022372843) and conducted according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) statement. Adherence to treatment in this systematic review and meta-analysis was collected from all studies included, based on the crude numerators and denominators reported, using either the Morisky Medication Adherence Scale 4 or -8.
A total of 54 records were identified through database searches, or after screening relevant manuscripts' references. Finally, 18 studies were included in this systematic review and meta-analysis. A total of 1559 participants (54.18% women) aged less than 84 years old were included. Twelve studies used the MMAS-8. In 8 of them, they established the level of adherence to treatment in three categories (low, medium, and high), with the mean prevalence in each of them being 41.4%, 30.4%, and 28.2%, respectively.
The results observed in adherence to treatment in patients with rare diseases show great variability, due to the different aspects involved in the greater or lesser applicability of the medication.
罕见病的可见性是社会面临的新挑战。这些疾病数量众多，在性质和分布上具有异质性，其特点是死亡率高但患病率低，且通常呈现严重的病情发展。由于治疗方法稀缺，针对罕见病的药物依从性研究并不常见。
本研究的主要目的是进行一项荟萃分析，评估最常见的罕见病的药物依从性水平。
本研究为系统评价，并在国际前瞻性系统评价注册库（PROSPERO）中进行了注册（注册号：CRD42022372843），并根据系统评价和荟萃分析的首选报告项目（PRISMA）声明进行。在本系统评价和荟萃分析中，基于所报告的原始分子和分母，使用Morisky药物依从性量表4或8，从纳入的所有研究中收集治疗依从性数据。
通过数据库检索或筛选相关手稿参考文献后，共识别出54条记录。最终，18项研究纳入了本系统评价和荟萃分析。共纳入1559名年龄小于84岁的参与者（54.18%为女性）。12项研究使用了MMAS-8。其中8项研究将治疗依从性水平分为三类（低、中、高），每类的平均患病率分别为41.4%、30.4%和28.2%。
在罕见病患者中观察到的治疗依从性结果显示出很大的变异性，这是由于药物适用性或多或少所涉及的不同方面所致。
