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- Tobias, Deirdre K, et al.
(author)
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Second international consensus report on gaps and opportunities for the clinical translation of precision diabetes medicine
- 2023
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In: Nature Medicine. - 1546-170X. ; 29:10, s. 2438-2457
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Research review (peer-reviewed)abstract
- Precision medicine is part of the logical evolution of contemporary evidence-based medicine that seeks to reduce errors and optimize outcomes when making medical decisions and health recommendations. Diabetes affects hundreds of millions of people worldwide, many of whom will develop life-threatening complications and die prematurely. Precision medicine can potentially address this enormous problem by accounting for heterogeneity in the etiology, clinical presentation and pathogenesis of common forms of diabetes and risks of complications. This second international consensus report on precision diabetes medicine summarizes the findings from a systematic evidence review across the key pillars of precision medicine (prevention, diagnosis, treatment, prognosis) in four recognized forms of diabetes (monogenic, gestational, type 1, type 2). These reviews address key questions about the translation of precision medicine research into practice. Although not complete, owing to the vast literature on this topic, they revealed opportunities for the immediate or near-term clinical implementation of precision diabetes medicine; furthermore, we expose important gaps in knowledge, focusing on the need to obtain new clinically relevant evidence. Gaps include the need for common standards for clinical readiness, including consideration of cost-effectiveness, health equity, predictive accuracy, liability and accessibility. Key milestones are outlined for the broad clinical implementation of precision diabetes medicine.
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- Van der Elst, Kristien, et al.
(author)
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One in five patients with rapidly and persistently controlled early rheumatoid arthritis report poor well-being after 1 year of treatment.
- 2020
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In: RMD open. - : BMJ. - 2056-5933. ; 6:1
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Journal article (peer-reviewed)abstract
- To identify and characterise a subgroup of patients with early rheumatoid arthritis (RA) reporting not feeling well 1 year after treatment initiation despite achieving optimal disease control according to current treatment standards.This observational study included participants of the Care in early RA trial with a rapid and sustained response (DAS28CRP<2.6) from week 16 until year 1 after starting the first RA treatment. Feeling well was assessed at year 1, using five patient-reported outcomes (PROs): pain, fatigue, physical functioning, RA-related quality of life and sleep quality. K-means clustering assigned patients to a cluster based on these PROs. Cohen's d effect size estimated cluster differences at treatment initiation and week 16, for the five clustering PROs, coping behaviour, illness perceptions and social support.Analyses revealed three clusters. Of 140 patients, 77.9% were assigned to the 'concordant to disease activity' cluster, 9.3% to the 'dominant fatigue' cluster and 12.9% to the 'dominant pain and fatigue' cluster. Large differences in pain and fatigue reporting were found at week 16 when comparing the 'concordant' with the 'dominant pain and fatigue' or the 'dominant fatigue' cluster. Small differences in reporting were found for the other PROs. Illness perceptions and coping style also differed in the 'concordant' cluster.Although most patients reported PRO scores in concordance with their well-controlled disease activity, one in five persistent treatment responders reported not feeling well at year 1. These patients reported higher pain and fatigue, and different illness perceptions and coping strategies early in the disease course.
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- Van der Elst, Kristien, et al.
(author)
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Patient reported outcome data from the Care in Early Rheumatoid Arthritis trial: Opportunities for broadening the scope of treating to target.
- 2019
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In: Arthritis care & research. - : Wiley. - 2151-4658 .- 2151-464X. ; 71:12, s. 1566-1575
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Journal article (peer-reviewed)abstract
- Treating early, intensively and to target leads to rapid disease control, preventing joint damage and loss of function in early rheumatoid arthritis (RA). We report the effect of such approach on patient-reported outcomes and explore the contribution of rapid and persistent disease control on wellbeing after 1 year of treatment.This study is part of the Care in early RA trial, a prospective, 2-year, investigator-initiated, randomized controlled trial rooted in daily practice and implementing the treat-to-target principle. SF-36 and IPQ-R data were collected prospectively. We defined 4 clinical response profiles based on speed and consistency of the treatment response within the first year, defined as DAS28CRP<2.6. Linear regression analyses including these response profiles and treatment type were constructed to predict SF-36 dimensions vitality, social functioning, role emotional, mental health, and IPQ-R illness perception subscales consequences, treatment control and illness coherence at year 1.333 patients were available for the main analyses, including 140 early persistent responders. Variation in each of the psychosocial outcomes at year 1 was explained mostly by their baseline values, followed by the clinical response profiles. Patients with an early persistent response reported significantly higher vitality, more positive beliefs about disease consequences and treatment effect. Treatment type did not matter.Rapid and persistent disease control and not treatment type was associated with favorable patient reported health and illness perceptions at year 1, but baseline psychosocial variables mattered most. Our data indicate opportunities to broaden the scope of the treat-to-target principle in early RA. This article is protected by copyright. All rights reserved.
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