Document Detail


Exercise capacity and haemodynamics in patients with sickle cell disease with pulmonary hypertension treated with bosentan: results of the ASSET studies.
MedLine Citation:
PMID:  20175775     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
Doppler-defined pulmonary hypertension (PH) in sickle cell disease (SCD) is associated with 40% mortality at 40 months. To assess the effect of bosentan in SCD-PH, two randomized, double-blind, placebo-controlled, 16-week studies were initiated. Safety concerns are particularly relevant in SCD due to comorbid conditions. ASSET-1 and -2 enrolled patients with pulmonary arterial hypertension (PAH) and pulmonary venous hypertension (PH), respectively. Haemodynamics and 6-min walk distance (6MWD) were obtained at baseline and week 16. The studies were terminated due to slow site initiation and patient enrolment (n = 26). Bosentan appeared to be well tolerated. Although sample sizes were limited, in ASSET-1 at baseline, 6MWD correlated with cardiac output (CO; P = 0.006) with non-significant inverse correlations between 6MWD and pulmonary vascular resistance (PVR; P = 0.07) and between 6MWD and right atrial pressure (P = 0.08). In ASSET-2 at baseline, there was a non-significant correlation between 6MWD and CO (P = 0.06). Due to limited sample sizes, efficacy endpoints were not analysed. However, in both studies, non-significant increases in CO were observed with bosentan compared to placebo. Similarly, non-significant decreases in PVR were observed with bosentan. Limited data in SCD-PH suggest that a low 6MWD predicts a low CO. Standard-dose bosentan appears to be well tolerated. Further investigation is warranted. Clinicaltrials.gov registration numbers NCT00310830, NCT00313196, NCT00360087.
Authors:
Robyn J Barst; Kamal K Mubarak; Roberto F Machado; Kenneth I Ataga; Raymond L Benza; Oswaldo Castro; Robert Naeije; Namita Sood; Paul S Swerdlow; Mariana Hildesheim; Mark T Gladwin;
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Publication Detail:
Type:  Journal Article; Multicenter Study; Randomized Controlled Trial; Research Support, Non-U.S. Gov't     Date:  2010-02-17
Journal Detail:
Title:  British journal of haematology     Volume:  149     ISSN:  1365-2141     ISO Abbreviation:  Br. J. Haematol.     Publication Date:  2010 May 
Date Detail:
Created Date:  2010-05-04     Completed Date:  2010-06-29     Revised Date:  2011-10-19    
Medline Journal Info:
Nlm Unique ID:  0372544     Medline TA:  Br J Haematol     Country:  England    
Other Details:
Languages:  eng     Pagination:  426-35     Citation Subset:  IM    
Affiliation:
Department of Pediatric Cardiology, Columbia University, New York, NY, USA. robyn.barst@gmail.com
Data Bank Information
Bank Name/Acc. No.:
ClinicalTrials.gov/NCT00310830;  NCT00313196;  NCT00360087
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MeSH Terms
Descriptor/Qualifier:
Adult
Anemia, Sickle Cell / complications*,  physiopathology
Antihypertensive Agents / adverse effects,  therapeutic use*
Double-Blind Method
Exercise Test / methods
Exercise Tolerance / drug effects*
Female
Hemodynamics / drug effects
Humans
Hypertension, Pulmonary / drug therapy*,  etiology,  physiopathology
Male
Middle Aged
Pulmonary Artery / physiopathology
Sulfonamides / adverse effects,  therapeutic use*
Vascular Resistance / drug effects
Grant Support
ID/Acronym/Agency:
Z01 CL001174-07/CL/CLC NIH HHS; ZIA HL005074-04/HL/NHLBI NIH HHS; ZIA HL005074-05/HL/NHLBI NIH HHS
Chemical
Reg. No./Substance:
0/Antihypertensive Agents; 0/Sulfonamides; 147536-97-8/bosentan
Investigator
Investigator/Affiliation:
R J Barst / ; K K Mubarak / ; K I Ataga / ; R L Benza / ; O Castro / ; R Naeije / ; G Simonneau / ; P S Swerdlow / ; M T Gladwin / ; M Hildesheim / ; F Galacteros / ; C H U Henri Mondor / ; G Coghlan / ; K I Ataga / ; R Benza / ; A Frost / ; E Klings / ; R F Machado / ; K K Mubarak / ; L Muñoz / ; E Berman-Rosenzweig / ; W Smith / ; N Sood / ; M Telen /
Comments/Corrections

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