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What is PAH?

Treatment guidelines: goal-oriented

Treatment guidelines recommend that patients with PAH receive initial monotherapy.1-3 The choice of initial therapy depends on a variety of factors, including disease severity (see section: Assessing the severity of PAH), route of administration, side effect profiles, approval and reimbursement status, and preferences of patients and clinicians.

Once treatment is initiated, patients should be monitored regularly and their response to therapy assessed using a range of clinical, exercise haemodynamic and echocardiographic parameters. Guidelines have identified factors associated with prognosis which should be used to monitor treatment response, including functional class, six-minute walk distance, the presence of pericardial effusion on echocardiography, and haemodynamic parameters such as right atrial pressure.2,3,4

Treatment guidelines recommend that, if the patient shows an inadequate clinical response to monotherapy, combination therapy using a combination of PAH-specific therapies from different classes (i.e. prostacyclin/prostacyclin analogues, endothelin receptor antagonists, phosphodiesterase-5 inhibitors) should be considered.1–3 The type of combination therapy chosen by the healthcare professional will depend on a variety of factors, including approval and reimbursement status and preferences of patients and clinicians.

A goal-orientated strategy whereby treatment goals are set based on achieving or maintaining particular thresholds or values in parameters known to be prognostically relevant can be used to assess response.3,4 While this strategy is discussed in international guidelines,5 European guidelines currently give the most detailed advice as to the use of this strategy.3

According to European guidelines, predetermined goals based on parameters that have been shown to be associated with better prognosis (see section: Assessing the severity of PAH) should be set for the patient, and regular follow-up visits are recommended (every 3–6 months, or 3–4 months after the initiation of, or a change to, treatment) in order to ascertain progress towards the treatment goals. Based on these parameters, a patient's condition can be categorised as:3

  • Stable and satisfactory: a patient fulfilling the majority of goals or targets
  • Stable but not satisfactory: a patient who, although stable, has not achieved the status that the patient and treating physician would consider desirable and some of the treatment goals that have been shown to be associated with a better prognosis have not been met
  • Unstable and deteriorating: a patient not meeting goals or targets, and exhibiting a number of parameters associated with a worse prognosis

Patients in the ‘stable but not satisfactory' or ‘unstable and deteriorating' categories require re-evaluation and consideration for escalation of treatment.

The management of patients with PAH is complex. Given these complexities, referral of patients with PAH to an expert centre is recommended wherever possible.1-3



  1. Barst RJ, Gibbs JS, Ghofrani HA, et al. Updated evidence-based treatment algorithm in pulmonary arterial hypertension. J Am Coll Cardiol 2009;54:S78–84.
  2. Badesch DB, Abman SH, Simonneau G, et al. Medical therapy for pulmonary arterial hypertension: updated ACCP evidence-based clinical practice guidelines. Chest 2007;131:1917–28.
  3. Galiè N, Hoeper MM, Humbert M, et al. Guidelines for the diagnosis and treatment of pulmonary hypertension. Eur Heart J 2009;30:2493–537.
  4. McLaughlin VV, Archer SL, Badesch DB, et al; ACCF/AHA. ACCF/AHA 2009 expert consensus document on pulmonary hypertension: a report of the American College of Cardiology Foundation Task Force on Expert Consensus Documents and the American Heart Association: developed in collaboration with the American College of Chest Physicians, American Thoracic Society, Inc., and the Pulmonary Hypertension Association. Circulation 2009;119:2250–94.
  5. Badesch DB, Champion HC, Sanchez MAG et al. Diagnosis and assessment of pulmonary arterial hypertension. J Am Coll Cardiol 2009;54:S55–66.