Phosphodiesterase Type-5 Inhibitor Therapy in Sickle Cell People With Pulmonary Hypertension

NCT03572036 | Completed

• 2 other identifiers: 99991810918-H-N109
• Study Type: observational
• Target: 38
• Locations: 1 country
• Sites: 1

Brief Summary

Background: Sickle cell disease (SCD) is a common inherited blood disorder. Many people with SCD are at risk to get pulmonary hypertension (PH). PH means that the blood pressure in the blood vessels to the lungs is high, and is a serious disease and. Very few studies have looked at the success of treatments for PH in people with SCD. Researchers want to learn more about treating PH with a type of drug called phosphodiesterase type 5 inhibitors (PDE5-I). They will look at the records of people who have already joined other studies. Objective: To identify people who already joined NIH SCD protocols whose medical records should be reviewed. The review will look at the description of SCD patients with PH who have already taken PDE5-I and the outcomes for these people. Eligibility: Adults ages 18 and older with SCD and PH. They must have joined certain NIH studies and taken PDE5-I therapy for at least 16 weeks. Design: This study is a review of medical records. Researchers will collect data from databases of existing studies. They will identify people in those studies who have SCD and PH and took the study drug for at least 16 weeks. Researchers will review the full medical records of those people. From that review, researchers will find participants who meet the inclusion criteria. They will extract data from those records. Researchers will analyze the data. This includes results from heart and lung tests, imaging, and walking tests. It will also include results of a procedure called right heart catheterization. Demographic data and lab data will also be collected. Researchers will remove identifying information from the data, then share it in a database.

Conditions

Pulmonary Hypertension; Sickle Cell Disease

Keywords

Outcomes Assessment; Sickle Cell Disease

Outcome Measures

Primary Outcomes (1)

• Perform multivariable regression analyses for factors associated with morbidity and mortality

  We hypothesize that there are some subjects, for example those with RHC-defined PH, with particular characteristics who may benefit from improved morbidity and mortality with the use of long- term PDE5-I therapy.

  At the end of data analysis

Study Arms (1)

Arm 1

aged 18 and older, participated in 001-H-0088 and 04-H-0161 and 1) a diagnosis of SCD 2) a diagnosis of PH 3) prescribed and/ or reported taking PDE5-I therapy for a duration of \>16 weeks.

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Subjects aged 18 and older, participated in 001-H-0088 and 04-H-0161 and 1) a diagnosis of SCD 2) a diagnosis of PH 3) prescribed and/ or reported taking PDE5-I therapy for a duration of \>16 weeks.

You may qualify if:

• Subjects aged 18 and older, participated in 001-H-0088 and 04-H-0161
• a diagnosis of SCD
• a diagnosis of PH by right heart catheterization
• prescribed and/ or reported taking PDE5-I therapy for a duration of greater than or equal to 16 weeks. (We will not be able to use medication dispensation records alone to verify whether subjects were compliant as not all medications were dispensed directly from the NIH pharmacy.)

Sponsors & Collaborators

• National Heart, Lung, and Blood Institute (NHLBI): lead

Study Sites (1)

National Heart, Lung and Blood Institute (NHLBI)

Bethesda, Maryland, 20892, United States

Location

MeSH Terms

Conditions

Hypertension, Pulmonary; Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Hypertension; Vascular Diseases; Cardiovascular Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Swee Lay Thein, M.D.

  National Heart, Lung, and Blood Institute (NHLBI)

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 27, 2018
• First Posted: June 28, 2018
• Study Start: June 20, 2018
• Primary Completion: April 16, 2021
• Study Completion: April 16, 2021
• Last Updated: April 19, 2021

Record last verified: 2021-04

Locations

US (1)