Is low-risk status a surrogate outcome in pulmonary arterial hypertension? An analysis of three randomised trials
Blette B, Moutchia J, Al-Naamani N, Ventetuolo C, Cheng C, Appleby D, Urbanowicz R, Fritz J, Mazurek J, Li F, Kawut S, Harhay M. Is low-risk status a surrogate outcome in pulmonary arterial hypertension? An analysis of three randomised trials. The Lancet Respiratory Medicine 2023, 11: 873-882. PMID: 37230098, PMCID: PMC10592525, DOI: 10.1016/s2213-2600(23)00155-8.Peer-Reviewed Original ResearchConceptsPulmonary arterial hypertensionPulmonary arterial hypertension trialsWorsening pulmonary arterial hypertensionFood and Drug AdministrationLow-risk statusClinical worseningLong-term outcomesRisk scoreArterial hypertensionPAH associated with connective tissue diseaseIdiopathic pulmonary arterial hypertensionPulmonary arterial hypertension treatmentSurrogate outcomesObservational study of outcomesLong-term follow-upDiscontinuation of study treatmentWHO functional classUS Food and Drug AdministrationMeta-analysisMeta-analysis of RCTsAll-cause deathConnective tissue diseaseEffects of therapyPredictive of outcomeTreatment effectsCausal Bayesian machine learning to assess treatment effect heterogeneity by dexamethasone dose for patients with COVID-19 and severe hypoxemia
Blette B, Granholm A, Li F, Shankar-Hari M, Lange T, Munch M, Møller M, Perner A, Harhay M. Causal Bayesian machine learning to assess treatment effect heterogeneity by dexamethasone dose for patients with COVID-19 and severe hypoxemia. Scientific Reports 2023, 13: 6570. PMID: 37085591, PMCID: PMC10120498, DOI: 10.1038/s41598-023-33425-3.Peer-Reviewed Original ResearchConceptsCritical COVID-19Better long-term outcomesCOVID-19Entire trial populationStandardized dosing protocolsMultiple patient characteristicsDose of dexamethasoneLong-term outcomesIL-6 inhibitorsDexamethasone doseSevere hypoxemiaMost patientsPatient characteristicsRespiratory supportDiabetes mellitusClinical outcomesDosing protocolTrial populationTreatment effect heterogeneityPatient featuresPatientsAdditional studiesDexamethasoneDoseMore evidence