The National Heart, Lung, and Blood Institute (NHLBI) is strongly committed to supporting research aimed at preventing and treating heart, lung, blood, and sleep diseases and conditions.
NHLBI-supported research has led to a better understanding of sickle cell disease (SCD) and its complications. Research has led to advances in the treatment of SCD and improvements in the quality of life.
The NHLBI continues to support research aimed at learning more about SCD and how to prevent and treat complications. For example, NHLBI-supported research includes studies that examine:
- Stroke prevention in high-risk children with SCD
- Long-term study on the effectiveness of hydroxyurea in children with SCD (BABY HUG Follow-Up Study)
- Treatment of SCD crises with regadenoson (children and adults)
- Lower-intensity bone marrow transplantation for SCD
- Treatment of leg ulcers in SCD
Much of this research depends on the willingness of volunteers to take part in clinical research and trials.
For example, a clinical research study might need to collect information about you or your child’s diagnosis, medications, problems, and or screening tests. You might be asked if you would allow such information to be entered into a large database that includes information about many other people with SCD. The information would be confidential and carefully protected.
When data are collected this way, researchers can learn a lot about SCD, what complications commonly occur, and how good the treatments are.
Clinical trials involve testing new ways to prevent, diagnose, or treat different diseases and conditions. For example, new treatments for a disease or condition (such as medicines, medical devices, surgeries, or procedures) are tested in volunteers who have the illness. Testing shows whether a treatment is safe and effective in volunteers before it is made available for widespread use.
By taking part in a clinical trial, you or your child can gain access to new treatments before they are widely available. You also will have the support of a team of health care providers who will monitor your health closely. Even if you do not directly benefit from the results of a clinical trial, the information gathered may help others and add to scientific knowledge.
If you volunteer for a clinical trial, the research will be explained to you in detail. You will learn about treatments and tests that you may receive, and their benefits and risks. You will be given as much time as needed to ask questions about the research. This process is called informed consent.
If you agree to take part in clinical research or a clinical trial, you will be asked to sign an informed consent form. This form is not a contract. You have the right to leave the study at any time, for any reason. Also, you have the right to learn about new risks or findings that emerge during the research or trial.
For more information about SCD clinical research and trials, talk with your doctor. You can visit the following websites to learn more about clinical research and to search for clinical trials:
- http://patientrecruitment.nhlbi.nih.gov/BloodDiseases.aspx (Scroll down for SCD trials)
- Children and Clinical Studies
- Clinical Trials (Health Topics)
- Current Research (ClinicalTrials.gov)
- NHLBI Clinical Trials
- NIH Clinical Research Trials and You (National Institutes of Health)
Living With and Managing Sickle Cell Disease (Nicholas)09/01/2011
Living With and Managing Sickle Cell Disease (Tiffany)05/26/2011