Table 2Inclusion and exclusion criteria

Study CharacteristicInclusion CriteriaExclusion Criteria
Population
  • KQ 1: Patients with suspected pulmonary arterial hypertension (PAH) or asymptomatic patients at high risk for PAH (e.g., patients with a collagen vascular disorder such as scleroderma)
  • KQs 2 and 3: Patients with PAH
KQ 1: Patients have neither (1) a condition associated with a high risk of undiagnosed PAH (e.g., a collagen vascular disorder) nor (2) signs or symptoms suspicious for PAH
KQ 2 and KQ 3: No patients have PAH
Interventions
  • KQ 1 (screening): Echocardiography plus biomarkers including natriuretic peptides (e.g., atrial natriuretic peptide, brain natriuretic peptide), endothelin-1, uric acid, troponin T, nitric oxide, asymmetric dimethylarginine, cyclic guanosine monophosphate, D-dimer, and serotonin
  • KQ 2 (management):
    • Biomarkers plus clinical assessment (e.g., history, physical exam, functional status)
    • Echocardiography plus biomarkers plus clinical assessment
  • KQ 3 (pharmacotherapies):
Study does not include a comparison of echocardiography or biomarkers for screening, diagnosis, or management of PAH, or does not include a comparison of monotherapy with combination therapy for PAH
Comparators
  • KQ 1: Echocardiography vs. echocardiography plus biomarkers
  • KQ 2:
    • Echocardiography vs. biomarkers (direct comparison)
    • Echocardiography vs. echocardiography plus biomarkers (direct comparison)
    • Echocardiography vs. clinical assessment (indirect comparison)
    • Biomarkers vs. clinical assessment (indirect comparison)
  • KQ 3:
    • One pharmacotherapy vs. another pharmacotherapy
    • Monotherapy vs. combination therapy
Study does not include a comparison of echocardiography or biomarkers for screening, diagnosis, or management of PAH, or does not include a comparison of monotherapy with combination therapy for PAH
Outcomes
  • KQ 1: Test-associated outcomes: Diagnostic accuracy efficacy (sensitivity, specificity, positive predictive value/negative predictive value); verification by right heart catheterization for test positive patients was required (incomplete verification of test negative patients was allowed)
  • KQ 2: Efficacy outcomes:
    • Diagnostic thinking efficacy and therapeutic efficacy (clinician judgment about diagnosis/prognosis, choice of treatment)
    • Patient outcome efficacy for intermediate-term outcomes (hemodynamic parameters, dyspnea, and 6-minute walk) and long-term outcomes (functional class, quality of life, right heart failure, and mortality)
  • KQ 3: Effectiveness of pharmacotherapies:
    • Intermediate-term outcomes such as hemodynamic parameters, dyspnea, and 6-minute walk
    • Long-term outcomes such as functional class, quality of life, right heart failure or right ventricular dysfunction, and mortality
No primary or secondary outcomes of interest are reported
Outcomes (safety)
  • KQs 1 and 2: Adverse effects of echocardiography and biomarkers, such as bleeding, bruising, infection, and transient ischemic attack
  • KQ 3: Adverse effects of pharmacotherapies (liver function abnormalities, headache, flushing, cough, epistaxis, dyspepsia, diarrhea, peripheral edema, nausea, nasal congestion, dizziness, syncope, hypoxia, increased international normalized ratio or prothrombin time) and parenteral therapy (line infection, site pain, abrupt catheter occlusion)
None
TimingIntermediate-term (≤120 days) and long-term (>120 days)None
Setting
  • Inpatient and outpatient
  • Specialty (pulmonary, cardiology, rheumatology) and primary care
None
Study design
  • Randomized controlled trial, prospective or retrospective observational study, or registry
  • Original data (or related methodology paper of an included article) for any of the screening or diagnostic tests listed in the KQs, or original data with intermediate-term or long-term outcomes associated with monotherapy or combination therapy for PAH
  • Relevant systematic review or meta-analysis (used for background only)
  • All sample sizes
Not a clinical study (e.g., editorial, nonsystematic review, letter to the editor, case series)
Publications
  • English-language only
  • Peer-reviewed article
  • KQs 1 and 2: Published January 1, 1995, to present
  • KQ 3: Published January 1, 1990, to present
Given the high volume of literature available in English-language publications (including the majority of known important studies), non-English articles are excludedb

KQ = Key Question; PAH = pulmonary arterial hypertension

From: Methods

Cover of Pulmonary Arterial Hypertension: Screening, Management, and Treatment
Pulmonary Arterial Hypertension: Screening, Management, and Treatment [Internet].
Comparative Effectiveness Reviews, No. 117.
McCrory DC, Coeytaux RR, Schmit KM, et al.

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