Multi-parametric MRI of kidney disease progression for autosomal recessive polycystic kidney disease: mouse model and initial patient results.

2021

157-162

2021 01

1530-0447

<p><strong>BACKGROUND: </strong>Autosomal recessive polycystic kidney disease (ARPKD) is a rare but potentially lethal genetic disorder typically characterized by diffuse renal microcysts. Clinical trials for patients with ARPKD are not currently possible due to the absence of sensitive measures of ARPKD kidney disease progression and/or therapeutic efficacy.</p>

<p><strong>METHODS: </strong>In this study, animal and human magnetic resonance imaging (MRI) scanners were used to obtain quantitative kidney T1 and T2 relaxation time maps for both excised kidneys from bpk and wild-type (WT) mice as well as for a pediatric patient with ARPKD and a healthy adult volunteer.</p>

<p><strong>RESULTS: </strong>Mean kidney T1 and T2 relaxation times showed significant increases with age (p &lt; 0.05) as well as significant increases in comparison to WT mice (p &lt; 2 × 10). Significant or nearly significant linear correlations were observed for mean kidney T1 (p = 0.030) and T2 (p = 0.054) as a function of total kidney volume, respectively. Initial magnetic resonance fingerprinting assessments in a patient with ARPKD showed visible increases in both kidney T1 and T2 in comparison to the healthy volunteer.</p>

<p><strong>CONCLUSIONS: </strong>These preclinical and initial clinical MRI studies suggest that renal T1 and T2 relaxometry may provide an additional outcome measure to assess cystic kidney disease progression in patients with ARPKD.</p>

<p><strong>IMPACT: </strong>A major roadblock for implementing clinical trials in patients with ARPKD is the absence of sensitive measures of ARPKD kidney disease progression and/or therapeutic efficacy. A clinical need exists to develop a safe and sensitive measure for kidney disease progression, and eventually therapeutic efficacy, for patients with ARPKD. Mean kidney T1 and T2 MRI relaxation times showed significant increases with age (p &lt; 0.05) as well as significant increases in comparison to WT mice (p &lt; 2 ×10), indicating that T1 and T2 may provide sensitive assessments of cystic changes associated with progressive ARPKD kidney disease. This preclinical and initial clinical study suggests that MRI-based kidney T1 and T2 mapping could be used as a non-invasive assessment of ARPKD kidney disease progression. These non-invasive, quantitative MRI techniques could eventually be used as an outcome measure for clinical trials evaluating novel therapeutics aimed at limiting or preventing ARPKD kidney disease progression.</p>

10.1038/s41390-020-0883-9

Pediatr Res

32283547

Why Is the Electronic Health Record So Challenging for Research and Clinical Care?

2021

2021 Jul 19

2511-705X

<p><strong>BACKGROUND: </strong> The electronic health record (EHR) has become increasingly ubiquitous. At the same time, health professionals have been turning to this resource for access to data that is needed for the delivery of health care and for clinical research. There is little doubt that the EHR has made both of these functions easier than earlier days when we relied on paper-based clinical records. Coupled with modern database and data warehouse systems, high-speed networks, and the ability to share clinical data with others are large number of challenges that arguably limit the optimal use of the EHR OBJECTIVES:  Our goal was to provide an exhaustive reference for those who use the EHR in clinical and research contexts, but also for health information systems professionals as they design, implement, and maintain EHR systems.</p>

<p><strong>METHODS: </strong> This study includes a panel of 24 biomedical informatics researchers, information technology professionals, and clinicians, all of whom have extensive experience in design, implementation, and maintenance of EHR systems, or in using the EHR as clinicians or researchers. All members of the panel are affiliated with Penn Medicine at the University of Pennsylvania and have experience with a variety of different EHR platforms and systems and how they have evolved over time.</p>

<p><strong>RESULTS: </strong> Each of the authors has shared their knowledge and experience in using the EHR in a suite of 20 short essays, each representing a specific challenge and classified according to a functional hierarchy of interlocking facets such as usability and usefulness, data quality, standards, governance, data integration, clinical care, and clinical research.</p>

<p><strong>CONCLUSION: </strong> We provide here a set of perspectives on the challenges posed by the EHR to clinical and research users.</p>

10.1055/s-0041-1731784

Methods Inf Med

34282602