A bibliometric analysis of medication studies in congenital heart disease

Background: Randomized controlled trials represent robust foundational material for evidence-based clinical practice in cardiology; however, such trials appear to be scarce in congenital heart disease (CHD). No bibliometric analysis has formally quantified nor characterized this gap in the literature, nor examined the impact of existing medication studies in CHD patients. Objectives: This investigation aimed to evaluate and characterize existing medication studies in congenital heart disease. Methods: PubMed, Embase, Web of Science, Cochrane Library, Emcare and Academic Search Premier were searched (Jan 2000-May 2024) for CHD drug studies. Trials focusing on genetic conditions associated with cardiovascular abnormalities (e.g. Marfan syndrome) and pulmonary hypertension not associated with congenital heart disease were excluded.PROSPERO ID: CRD420251073438. Results: A total of 160 CHD medication studies involving 59,208 participants were included, spanning a study period of 24.5 years (2000-2024). Sex was reported for 20,092 participants; 10,514 (52.3%) were women. Only 29.4% (n = 47) of trials were RCTs. Time-series breakpoint analysis showed an inflection in annual publication output in 2013 (p = 0.009), with growth before and contraction thereafter. Pulmonary-hypertension drugs accounted for 58.1% of publications. Collectively, industry-funded trials were over twice as likely to be RC type trials (56.7% vs 23.1% in non-industry funded studies). Conclusions: Contemporary CHD pharmacotherapy research is limited in spectrum/demographics and appears to have been plateauing globally since 2013. Many studies are single-center, pulmonary-hypertension-focused and not placebo-controlled. There is a need to develop a broader disease evidence-base for medication interventions in CHD.