Document Detail

Patients with pulmonary arterial hypertension in clinical trials: who are they?
MedLine Citation:
PMID:  18625752     Owner:  NLM     Status:  MEDLINE    
Results from clinical trials serve as the basis for approval of therapies by regulatory agencies as well as for treatment decisions by clinicians. But these findings are relevant only to patients who are similar to the ones enrolled into the trials. This is germane to clinical trials on pulmonary arterial hypertension (PAH) because the disease is uncommon but highly heterogeneous and results can easily be misapplied. The characteristics of patients entering the trials are largely determined by inclusion/exclusion criteria, with the result that most participants have idiopathic, connective tissue disease- and congenital heart disease-related PAH. Earlier trials enrolled patients mainly with New York Heart Association functional class III and IV disease and with severe pulmonary hemodynamic abnormalities. Because it has been the major outcome variable in most of the trials, eligibility is also dependent on six-minute-walk distance, ensuring that patients are moderately but not too severely functionally impaired, thereby maximizing the likelihood of detecting a favorable response to therapy. More recent trials have enrolled less ill patients, with more patients with New York Heart Association functional class II disease, less severe hemodynamic abnormalities, and more stability over time. This reflects, in part, ethical concerns about enrolling sicker patients into placebo-controlled trials. Trials have mainly enrolled white females in their 40s and 50s and have consistently excluded non-WHO group 1 forms of pulmonary hypertension. These characteristics must be carefully considered when applying the findings of pulmonary hypertension trials in clinical practice.
Nicholas S Hill; Ioana R Preston; Kari E Roberts
Publication Detail:
Type:  Journal Article; Research Support, Non-U.S. Gov't; Review    
Journal Detail:
Title:  Proceedings of the American Thoracic Society     Volume:  5     ISSN:  1546-3222     ISO Abbreviation:  Proc Am Thorac Soc     Publication Date:  2008 Jul 
Date Detail:
Created Date:  2008-07-15     Completed Date:  2008-11-18     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  101203596     Medline TA:  Proc Am Thorac Soc     Country:  United States    
Other Details:
Languages:  eng     Pagination:  603-9     Citation Subset:  IM    
Division of Pulmonary, Critical Care, and Sleep Medicine, Tufts Medical Center, 800 Washington Street, #257, Boston, MA 02111, USA.
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MeSH Terms
Antihypertensive Agents / therapeutic use*
Clinical Trials as Topic / methods*
Hypertension, Pulmonary / drug therapy*
Patient Selection*
Reg. No./Substance:
0/Antihypertensive Agents

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