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Pdf Summary
The article "Selexipag Use in Clinical Practice—Mind the Gap" by R. James White, MD, PhD, and Daniel J. Lachant, DO, discusses the complexities clinicians face when prescribing selexipag for pulmonary arterial hypertension (PAH) due to limited evidence-based guidance. For common medical issues, large-scale clinical trials offer robust data guiding treatment choices; however, rare diseases like PAH often rely on studies with fewer participants, rendering subgroup analyses less conclusive.<br /><br />For PAH, multiple therapies exist, but the lack of compelling evidence makes choosing a specific drug challenging. Experts can offer nuanced guidance, and the Delphi method has been used for this purpose. The article references a study by McLaughlin et al., which used the RAND/UCLA approach to assess the appropriateness of using oral selexipag or treprostinil for specific PAH clinical scenarios. Experts evaluated case vignettes considering risk factors like right heart function and hospitalization history, assigning appropriateness scores. Vignettes got the consensus if the median score was 6 or more, with minimal dissent.<br /><br />Although expert guidance is helpful, challenges remain in evaluating selexipag's efficacy in practice, as clinical trials show modest hemodynamic improvements without significant functional benefits. Questions on dose-response relationship and adverse effects management remain unanswered.<br /><br />The field is evolving rapidly. The recent Freedom-EV study highlighted improvements with oral treprostinil, which might make treatment benefit determination easier. Future consensus efforts could focus on improvement criteria and opinion on dosing.<br /><br />The article underlines the need for clinicians to "mind the gap" in their knowledge, seeking expert consensus while remaining vigilant to advances and gaps in evidence for optimal PAH therapy, particularly when using selexipag.
Keywords
Selexipag
Pulmonary Arterial Hypertension
PAH
Clinical Practice
Evidence-Based Guidance
Delphi Method
RAND/UCLA Approach
Expert Consensus
Dose-Response Relationship
Adverse Effects Management
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