Role of Placenta Growth Factor in Sickle Acute Chest Syndrome

NCT ID: NCT00448370

Last Updated: 2017-12-21

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 136 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2007-03-31

Brief Summary

The purpose of this research study is to find out whether Placenta Growth Factor (PlGF) and related tests can predict the development of acute chest syndrome (ACS) in patients with sickle cell disease (SCD) during a period where patients are well and during admission to the hospital for an acute sickle event to see if these measures can predict the development of ACS. Understanding events precipitating ACS may lead to preventative and interventional therapies which will improve patient outcomes and quality of life.

Detailed Description

The proposed research is a clinical study designed to test the hypothesis that PlGF levels in blood at baseline in patients with SCD will correlate with leukotriene (LT) levels and will be reflective of the degree of airway obstruction; and that acute elevations in PlGF levels will occur during an acute sickle event and precede clinical and radiological recognition of ACS. This is a biological study that does not fall into the criteria of a phase I-IV trial.

Measurements will be done at two stages. First, patients who are admitted to the hospital with an acute sickle event will have a daily evaluation for the first 4 days of the admission or up until the patient has an ACS event. Measurements of inpatient spirometry (at primary site only) and impulse oscillometry will be performed daily. Measurements on 35 ACS events with ACS developing on the 3rd/4th day of admission will be collected. Measurements on patients developing ACS will be compared to those who do not develop ACS will allow for earlier prediction of ACS. Finally, patients who have an ACS or admission for an acute sickling event will be evaluated again after 3-4 weeks in order to get a baseline measurement. One hundred baseline measurements will be made. Twenty of these baseline patients will also have one or two additional baseline evaluations at subsequent clinic visits to evaluate the overall non-event baseline distribution and intra-subject baseline variability. Patients will have measurements repeated if admitted for an acute event greater than one year since the last baseline measurement . These measurements will be used to see if there is any prognostic information for a subsequent acute or ACS event. Some patients may only have baseline data collected.

Conditions

Anemia, Sickle Cell

Keywords

Anemia, sickle cell

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Eligibility Criteria

Inclusion Criteria

1. Patients must have a diagnosis of SCD (HbSS, HbSC or HbS beta/thal) by hemoglobin electrophoresis or gene mapping. Very few patients with HbSC or HbS beta/thal would go onto develop ACS, but they will serve as important controls.

2. Patients must be ≥5.5 years of age and ≤30 years of age.

3. Patients must be able to comply with pulmonary function tests.

Exclusion Criteria

1. Patients taking anti-leukotriene medications such as montelukast (Singulair®) or zileuton (Zyflo®) 30 days prior to enrollment will be ineligible, although patients with SCD and asthma who are not on leukotriene inhibitors will be eligible.

2. Patients with known congenital heart disease or with congenital lung abnormality/disease that will affect the pulmonary function testing and methacholine challenge testing will be ineligible (examples include patients with cyanotic heart disease, immotile cilia syndrome, cystic fibrosis, diaphragmatic hernia).

3. Any patient with neurologic abnormalities, stroke, developmental delay, or other medical condition that would preclude cooperation with pulmonary function testing or compliance with protocol procedures will be ineligible.

4. Patients over 30 years of age will be ineligible.

5. Patients who are pregnant or nursing.

6. Patients on chronic transfusions will be ineligible.

• If patients come off transfusion therapy, they will be eligible 3 months following the last transfusion.
• Patients receiving simple transfusions will be eligible, but baseline measurements will be made 3 months following the simple transfusion.

• Minimum Eligible Age: 66 Months
• Maximum Eligible Age: 30 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Heart, Lung, and Blood Institute (NHLBI)

NIH

Sponsor Role: collaborator

Children's Hospital Medical Center, Cincinnati

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Karen Kalinyak, MD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital Medical Center, Cincinnati

Punam Malik, MD

Role: STUDY_CHAIR

Children's Hospital Medical Center, Cincinnati

Locations

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Countries

United States

Related Links

http://cincinnatichildrens.org

Cincinnati Children's Hospital Medical Center

http://www.cincinnatichildrens.org/svc/alpha/b/blood/programs/sickle-cell

Cincinnati Comprehensive Sickle Cell Center

Other Identifiers

R01

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

CCHMC 06-09-57

Identifier Type: -

Identifier Source: org_study_id