What is sickle cell disease?
- Sickle cell disease is a genetic condition that affects the body’s red blood cells. It occurs when a child receives two sickle cell genes—one from each parent. In someone living with this disease, the red blood cells become hard and sticky and look like a C-shaped farm tool called a “sickle”.
- Sickle cell disease affects an approximately 100,000 Americans. And, globally, four million people are affected by the disease.
- It’s been more than a century since Dr. James B. Herrick became the first person to document the existence of sickle cell disease (SCD).
- A cure is within our reach. At the department of Health and Human Services we are committed to extending the lives of patients with SCD by 10 years within 10 years.
How does this disease impact ethnic and racial minorities?
- While the disease is most common among African Americans, other racial and ethnic groups are affected, including Latinos and people of Middle Eastern, Indian, Asian and Mediterranean backgrounds.
- According the Centers for Disease and Prevention, sickle cell disease occurs among 1 out of every 365 Black or African American births and 1 out of every Hispanic-American births.
- Sub-Saharan Africa has the greatest burden of disease, with more than 300,000 babies born with the disease each year. Unfortunately, 240,000 will die before their 5th SCD is now a primary global cause of infant mortality.
What treatments are available for people living with sickle cell disease?
- In the last century there were only two drugs to treat SCD. Today, there are nearly 40 therapies being tested under clinical trial to treat manage pain symptoms that patients are likely experiencing.
- Just 1 in 4 patients in the U.S. with SCD are receiving hydroxyurea, which helps reduce pain crisis and is the standard of care for this disease.
- HHS is starting a new program to help improve the care and treatment of people living with sickle cell disease. The new HHS SCD Training and Mentoring Program for Primary Care Providers (STAMP) program will train primary care providers on the basics of SCD care.
- We have to ensure that patients with sickle cell disease don’t become collateral damage in the fight against the opioid epidemic.
- During a pain crisis they are often subject to discrimination, which ignores the complex nature and mechanism of acute and chronic sickle cell pain. We are committed to protecting their access to the appropriate and safe use of opioids.
What happens when a child living with sickle cell disease becomes an adult?
- Transition can be a difficult and overwhelming time for teens with SCD and their families because it can also coincide with major life changes and milestones that young adults will face such as attending college, finding a job, or moving to a new location.
What is being done to find a cure for all?
- HHS committed to extending the lives of American with SCD by 10 years, within 10 years.
- Through a collaborative new effort called the Cure Sickle Cell (CureSC) Initiative, researchers in academia, government and industry—with the critical help of patients, their caregivers and patient advocacy groups—are now working together to accelerate the development and testing of the most promising genetic-based curative therapies
“Text Tom” questions answered by CAPT David Wong, Office of the Assistant Secretary (OASH) Office of Minority Health.
Is it true that a man in Alabama has been cured of sickle cell. It was on the news?
Indeed, many people have been cured of sickle cell disease. The gentleman from Mobile, Alabama was reportedly part of a clinical trial in gene therapy. Others have been cured by bone marrow transplants.
What’s the criteria for finding out if you’re a bone marrow match for someone with sickle cell
Almost 1 in 5 people with sickle cell disease have a healthy, fully-matched sibling donor. Siblings can be your donor even if they have sickle cell trait.
What is the life span of a person with sickle cell?
Many people live into their 40s or about two decades shorter than average. Among our main goals is to extend life span and improve quality of life.
How close is beta thalamissimia to sickle cell?
Beta thalamissimia is another inherited blood disorder. There is a type of sickle cell disease that has one copy of the sickle gene and one copy of the beta thalamissimia, which can lead to similar symptoms that you see in other types of sickle cell disease.
Can a person develop the sickle cell trait without a prior diagnoses, or is sickle cell absolutely passed on genetically?
Sickle cell is absolutely passed on genetically. However, people with the trait might not find out until later in life.
Can a biracial child get sickle cell?
Yes. Sickle cell disease is most common among African-Americans, but other racial and ethnic groups are affected, including Latinos and people of Middle Eastern, Indian, Asian and Mediterranean backgrounds.
Will the cure, if and when it comes, be affordable?
The National Institutes of Health, the National, Heart, Lung, and Blood Institute (NHLBI) has initiated the Cure Sickle Cell Initiative to accelerate gene therapies to cure the disease. We’re on track to have a cure in the next 5 to 10 years. We have to do everything we can to make it widely accessible and affordable to all who need it.
How do we navigate the emergency room when needing pain meds since doctors no longer want to give opioids? My son has been released without being given adequate medication. When he is having a crisis, he cannot make it to his “assigned” hospital. It is very frustrating to be caught in the middle of these new opioid regulations. Additionally, are there stronger non opioid meds that can be used and where can I find the list of new 40 drugs being used for sickle cell?
We agree that this is very frustrating. We’ve heard similar stories from other sickle cell patients and we continue to work with doctors to stress that sickle cell patients need their medications and need to have access to them. At HHS we are committed to making sure that SCD patients and cancer patients, for example, have access to the medications they need and are not swept up in the opioid crisis.
A few years ago, the FDA approved the first new sickle cell drug in 20 years. The drug, Endari, reduces pain and other complications caused by SCD, with other therapeutics moving through the pipeline. The American Society of Hematology is developing a Sickle Cell Clinical Trials Network to help track the status of these therapeutics. A community engagement workshop relating to this was held about a week ago.
You say stay hydrated – how does that affect dialysis patients?
It’s still important for everyone to stay hydrated, whether we’re under dialysis treatment or not. It’s also important to keep talking to your healthcare providers about your care and nutrition.
Why is the cold(er) weather/climate painful for those with sickle cell or the trait? Any tips?
Not everyone has the same trigger, but temperature changes can trigger pain crises. It’s important to minimize exposure to cold weather, bundle up, and consider your commuting patterns.
Is it true that the sickle cell gene makes you immune to malaria?
Yes, if you have sickle cell trait, there is some protective advantage against malaria. As a result, the frequencies of sickle cell carriers are high in malaria-endemic areas.
I have the sickle cell trait. When I’m dehydrated, I get really, really, really bad leg cramps. Is that what a crisis is like for people with sickle cell disease?
Sickle cell trait usually has no symptoms. But with extreme exertion or dehydration, someone with sickle cell trait can have these symptoms. With sickle cell disease these symptoms would be more frequent, more severe and affect different parts of the body.
Get more information about sickle cell disease HERE.
Admiral Brett Giroir is the 16th United States Assistant Secretary for Health in the Department of Health and Human Services. He serves as the Secretary’s principal public health and science advisor. He oversees Office of the Surgeon General and the U.S. Public Health Service Commissioned Corps, as well as key public health and science offices that focus on transforming the current “sick-care system” into a “health-promoting system.”
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