Shemesh Eyal, Mitchell Jeffrey, Neighbors Katie, Feist Susan, Hawkins Andre, Brown Amanda, Wanrong Yin, Anand Ravinder, Stuber Margaret L, Annunziato Rachel A
Icahn School of Medicine at Mount Sinai, New York, NY, USA.
The Emmes Corporation, Rockville, MD, USA.
Pediatr Transplant. 2017 Dec;21(8). doi: 10.1111/petr.13067. Epub 2017 Oct 6.
Medication adherence is an important determinant of transplant outcomes. Attempts to investigate adherence are frequently undermined by selection bias: It is very hard to recruit and retain non-adherent patients in research efforts. This manuscript presents recruitment strategies and results from the MALT (Medication Adherence in children who had a Liver Transplant) multisite prospective cohort study. MALT sites recruited 400 pediatric liver transplant patients who agreed to be followed for 2 years. The primary purpose was to determine whether a marker of adherence, the Medication Level Variability Index (MLVI), predicts rejection outcomes. The present manuscript describes methods used in MALT to ensure that a representative sample was recruited, and presents detailed recruitment results. MALT sites were able to recruit a nationally representative sample, as determined by a comparison between the MALT cohort and a national sample of transplant recipients. Strategies that helped ensure that the sample was representative included monitoring of the outcome measure in comparison with a national sample, drastically limiting patient burden, and specific recruitment methods. We discuss the importance of a representative sample in adherence research and recommend that future efforts to study adherence pay special attention to sample characteristics.
药物依从性是移植结果的一个重要决定因素。对依从性的研究常常受到选择偏倚的影响：在研究中招募并留住不依从的患者非常困难。本文介绍了MALT（肝移植儿童的药物依从性）多中心前瞻性队列研究的招募策略和结果。MALT研究点招募了400名同意接受为期2年随访的小儿肝移植患者。主要目的是确定依从性标志物——药物水平变异指数（MLVI）是否能预测排斥反应结果。本文描述了MALT研究中用于确保招募到具有代表性样本的方法，并展示了详细的招募结果。通过将MALT队列与全国移植受者样本进行比较发现，MALT研究点能够招募到具有全国代表性的样本。有助于确保样本具有代表性的策略包括与全国样本比较监测结果指标、大幅减轻患者负担以及采用特定的招募方法。我们讨论了具有代表性的样本在依从性研究中的重要性，并建议未来研究依从性的工作应特别关注样本特征。
