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Use of outcome measures in pulmonary hypertension clinical trials.

Publication ,  Journal Article
Parikh, KS; Rajagopal, S; Arges, K; Ahmad, T; Sivak, J; Kaul, P; Shah, SH; Tapson, V; Velazquez, EJ; Douglas, PS; Samad, Z
Published in: Am Heart J
September 2015

OBJECTIVES: To evaluate the use of surrogate measures in pulmonary hypertension (PH) clinical trials and how it relates to clinical practice. BACKGROUND: Studies of pulmonary arterial hypertension (PAH) employ a variety of surrogate measures in addition to clinical events because of a small patient population, participant burden, and costs. The use of these measures in PH drug trials is poorly defined. METHODS: We searched PubMed/MEDLINE/Embase for randomized or prospective cohort PAH clinical treatment trials from 1985 to 2013. Extracted data included intervention, trial duration, study design, patient characteristics, and primary and secondary outcome measures. To compare with clinical practice, we assessed the use of surrogate measures in a clinical sample of patients on PH medications at Duke University Medical Center between 2003 and 2014. RESULTS: Between 1985 and 2013, 126 PAH trials were identified and analyzed. Surrogate measures served as primary endpoints in 119 trials (94.0%). Inclusion of invasive hemodynamics decreased over time (78.6%, 75.0%, 52.2%; P for trend = .02), while functional testing (7.1%, 60.0%, 81.5%; P for trend < .0001) and functional status or quality of life (0%, 47.6%, 62.8%; P for trend < .0001) increased in PAH trials over the same time periods. Echocardiography data were reported as a primary or secondary outcome in 32 trials (25.4%) with increased use from 1985-1994 to 1995-2004 (7.1% vs 35.0%, P = .04), but the trend did not continue to 2005-2013 (25.0%). In comparison, among 450 patients on PAH therapies at our institution between 2003 and 2013, clinical assessments regularly incorporated serial echocardiography and 6-minute walk distance tests (92% and 95% of patients, respectively) and repeat measurement of invasive hemodynamics (46% of patients). CONCLUSIONS: The majority of PAH trials have utilized surrogate measures as primary endpoints. The use of these surrogate endpoints has evolved significantly over time with increasing use of patient-centered endpoints and decreasing or stable use of imaging and invasive measures. In contrast, imaging and invasive measures are commonly used in contemporary clinical practice. Further research is needed to validate and standardize currently used measures.

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Published In

Am Heart J

DOI

EISSN

1097-6744

Publication Date

September 2015

Volume

170

Issue

3

Start / End Page

419 / 29.e3

Location

United States

Related Subject Headings

  • Outcome Assessment, Health Care
  • Hypertension, Pulmonary
  • Humans
  • Hemodynamics
  • Clinical Trials as Topic
  • Cardiovascular System & Hematology
  • 3201 Cardiovascular medicine and haematology
  • 1117 Public Health and Health Services
  • 1102 Cardiorespiratory Medicine and Haematology
 

Citation

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Parikh, K. S., Rajagopal, S., Arges, K., Ahmad, T., Sivak, J., Kaul, P., … Samad, Z. (2015). Use of outcome measures in pulmonary hypertension clinical trials. Am Heart J, 170(3), 419-29.e3. https://doi.org/10.1016/j.ahj.2015.06.010
Parikh, Kishan S., Sudarshan Rajagopal, Kristine Arges, Tariq Ahmad, Joseph Sivak, Prashant Kaul, Svati H. Shah, et al. “Use of outcome measures in pulmonary hypertension clinical trials.Am Heart J 170, no. 3 (September 2015): 419-29.e3. https://doi.org/10.1016/j.ahj.2015.06.010.
Parikh KS, Rajagopal S, Arges K, Ahmad T, Sivak J, Kaul P, et al. Use of outcome measures in pulmonary hypertension clinical trials. Am Heart J. 2015 Sep;170(3):419-29.e3.
Parikh, Kishan S., et al. “Use of outcome measures in pulmonary hypertension clinical trials.Am Heart J, vol. 170, no. 3, Sept. 2015, pp. 419-29.e3. Pubmed, doi:10.1016/j.ahj.2015.06.010.
Parikh KS, Rajagopal S, Arges K, Ahmad T, Sivak J, Kaul P, Shah SH, Tapson V, Velazquez EJ, Douglas PS, Samad Z. Use of outcome measures in pulmonary hypertension clinical trials. Am Heart J. 2015 Sep;170(3):419–29.e3.
Journal cover image

Published In

Am Heart J

DOI

EISSN

1097-6744

Publication Date

September 2015

Volume

170

Issue

3

Start / End Page

419 / 29.e3

Location

United States

Related Subject Headings

  • Outcome Assessment, Health Care
  • Hypertension, Pulmonary
  • Humans
  • Hemodynamics
  • Clinical Trials as Topic
  • Cardiovascular System & Hematology
  • 3201 Cardiovascular medicine and haematology
  • 1117 Public Health and Health Services
  • 1102 Cardiorespiratory Medicine and Haematology