Clinical Trial: Preventing Acute Chest Syndrome by Transfusion Feasibility Study

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Preventing Acute Chest Syndrome by Transfusion Feasibility Study( PROACTIVE Feasibility Study)

Brief Summary: Acute chest syndrome (ACS) is similar to severe pneumonia and is a common cause of hospitalizations for people with sickle cell disease (SCD). Blood transfusions are one treatment option for ACS. High levels of an enzyme called secretory phospholipase A2 (sPLA2) may be present in people before they develop ACS. This study will determine how well sPLA2 levels can predict the onset of ACS and whether identifying high sPLA2 levels allows enough time to prevent ACS with blood transfusions. Results from this study will help to determine the feasibility of conducting a larger study that would further examine the use of sPLA2 levels and blood transfusions to prevent ACS in people with SCD.

Detailed Summary:

SCD is an inherited blood disorder, and symptoms include anemia, infections, organ damage, and intense episodes of pain, which are called "sickle cell crises." ACS, characterized by fever, respiratory distress, and lung tissue damage, is the second most common cause of hospitalization and the leading cause of death among people with SCD. Most people with SCD will experience at least one episode of ACS, and repeated episodes can result in progressive lung disease. ACS can appear suddenly and often requires immediate hospitalization and treatment, which can include blood transfusions. People with elevated blood levels of sPLA2 may be at risk for developing ACS, and this enzyme is often detectable before the onset of ACS symptoms. The purpose of this study is to examine the use of sPLA2 as a predictor of ACS and to determine whether subsequent blood transfusions can be administered early enough to prevent the onset of ACS in people with SCD who are at risk for ACS. Study researchers will also assess the feasibility of conducting a larger study that would further examine the effectiveness of using sPLA2 levels and blood transfusions to prevent ACS.

This study will involve two parts. In the first part of the study, participants with SCD who are admitted to the hospital with an acute sickle cell pain event will be randomly assigned to receive either a single blood transfusion or standard care for ACS and no blood transfusion. All participants will be closely monitored while in the hospital for the development of ACS, and study researchers will review participants' medical records. All participants will undergo daily blood collections, which will include testing for sPLA2 levels, and at least two chest x-rays. Twenty-eight days after hospital discharge, all participants will attend a follow-up study visit for blood collection, again to determine sPLA2 levels.

Sponsor: New England Research Institutes

Current Primary Outcome: Acute Chest Syndrome [ Time Frame: Chest x-rays (CXR) were ordered for trial eligibility, as a result of clinical indications, or at discharge or 72 hours if no prior CXR. ]

Original Primary Outcome: Assess feasibility of conducting larger trial in terms of elapsed time between eligibility, randomization, transfusion, Assess number of CXR, number of patients who develop ACS and predictive value of elevated sPLA2 for predictive ACS. [ Time Frame: 13 months ]

Current Secondary Outcome:

Original Secondary Outcome: Assess timely completion of all 3 sPLA2 for subjects with persistently low sPLA2 levels, % of screened subjects who are deemed eligible for randomization, % randomized, child/adult ratio, timing and results of sPLA2 samples. [ Time Frame: 13 months ]

Information By: New England Research Institutes

Dates:
Date Received: July 31, 2009
Date Started: July 2009
Date Completion:
Last Updated: April 16, 2013
Last Verified: April 2013