Transcript for Vital Signs Telebriefing: Children with sickle cell anemia are at risk for stroke and other complications

Narrator (00:00):

Welcome and thank you for standing by. At this time, I’d like to inform all participants that today’s call is being recorded. If you have any objections, you may disconnect at this time. All lines have been placed in a listen only mode until the question answer session of today’s call. At that time, if you would like to ask a question, please press star, followed by one. Please make sure your phone is unmuted and record your name clearly when prompted. I would now like to turn the call over to Mr. Benjamin Hayes. Haynes, thank you sir. You may begin.

Ben Haynes (00:34):

Thank you Missy, and thank you all for joining us today as we release a new CDC Vital Signs. We are joined by Dr. Debra Houry, CDCs Acting Principal, Deputy Director, and Dr. Laura Schieve, Associate Director for Science in the Division of Blood Disorders at CDCs National Center on Birth Defects and Developmental Disabilities, and she is also the first author of this Vital Science Report. Before we get started, I want to remind everyone that this briefing is in embargoed until 1:00 PM when Vital Signs goes live on the CDC website. And now I’ll turn the call over to Dr. Houry.

Dr. Houry (01:08):

Good afternoon everyone, and thank you for joining us today. CDCs work on Covid and Monkeypox has not kept us from our unwavering commitment to equitably protect the health, safety, and security of all Americans against all threats to public health. Indeed, this month’s CDC Vital Signs report focuses on sickle cell disease, a blood disorder that affects approximately a hundred thousand people in this country. CDCs vital signs reports spotlight serious health threats like sickle cell disease, as well as the science-based actions that can be taken to curb these significant health issues. Sickle cell anemia is the most severe form of sickle cell disease. It primarily affects Black and American African-American individuals in the United States and is associated with severe complications such as stroke or kidney issues, and it reduces life expectancy. Today’s vital signs report focuses on the youngest of those with sickle cell anemia, children and teenagers.

Dr. Houry (02:21):

During my career as an emergency department physician, I vividly recall those long, tough nights with these young sickle cell warriors and seeing children and teens suffering from the severe pain that often accompanies sickle cell anemia was heartbreaking. Preventing complications of this disease requires strategies to reduce the impact of racism and disparities in healthcare. Healthcare providers can educate themselves, their colleagues and their institutions about the specialized needs of people with sickle cell anemia, including how racism inhibits optimal care. For example, despite their extensive healthcare needs, many people with sickle cell anemia have difficulty accessing appropriate care and report, feeling stigmatized and having their symptoms dismissed when they do seek care. Providers can advocate for and listen to their patients to better understand these needs. Comprehensive data on the healthcare needs and experiences of the population of people with sickle cell anemia are needed to fully address these healthcare gaps.

Dr. Houry (03:33):

And fiscal year 2021, CDC received a dedicated funding line for the Sickle Cell Data Collection program. Currently active in 11 states, which served 36% of those with sickle cell disease, the program collects health information about people with sickle cell disease to study long-term trends and diagnosis treatment and healthcare access for people with the disease. The data has been used to guide decisions on where new sickle cell disease clinics are needed to educate healthcare providers to develop state healthcare policies and to guide new research initiatives. We will expand to more states serving more of those affected by this disease as resources become available. Now, I will turn it over to Dr. Schieve who will highlight the findings of today’s Vital Signs report.

Dr. Schieve (04:28):

Thank you, Dr. Houry. Today’s Vital Signs highlights the disheartening fact that many young people are not receiving potentially life-saving screenings and treatment for this disease. Sickle cell disease, sickle cell anemia, the most common form of sickle cell disease, is a leading cause of stroke in children and teens. Yet as this report points out, less than half of children two to 16 years of age who were enrolled in Medicaid in 2019 were receiving their recommended annual screening to assess their risk of having a stroke, and that screening is known as Transcranial Doppler, ultrasound, or TCD screening. The Vital Signs report also notes that only two and five children age two to nine years of age and only about half of children and teens age 10 to 16 years of age we’re receiving treatment with a medication called Hydroxyurea. With sickle cell anemia, a child’s red blood cells get sticky.

Dr. Schieve (05:33):

They become rigid and crescent or sickle shaped, and these sickle shaped cells can get stuck in the small blood vessels and they can block the blood flow and thus lead to serious um, complications. Hydroxyurea is a medication that can prevent this from happening. It helps keep the red blood cells healthy and round, and that can prevent many complications, including the severe pain crises that these children often experience, which often necessitates hospitalization. Hydroxyurea has been used for many years in adults, and in 2014 the medication was recommended for use in children by an expert panel. But despite this, the low number, despite this, the number of children and teens receiving hydroxyurea is still far too low. So the bottom line is this, severe pain and complications associated with sickle cell anemia are preventable and much can be done to reduce pain and suffering for our youngest patients with this disease. Now I’ll turn it back over to Dr. Houry.

Dr. Houry (06:41):

Thank you, Dr. Schieve. This Vital Signs report identifies many of the healthcare barriers facing children and teens with sickle cell anemia. All of US healthcare providers, parents and other caregivers, community organizations and governmental agencies have a role to play in reducing the barriers to care for children and teens with this devastating disease. For example, healthcare providers can implement quality care, strategize strategies to maximize transcranial doppler screening and hydroxyurea use. Patients and families can learn about the importance of annual screening and the benefits and safety of hydroxyurea and community organizations can help connect patients and families with resources and tools to improve their understanding of these life-saving measures. I will now open it up for questions.

Ben Haynes (07:40):

Thank you Dr. Houry, and thank you Dr. Schieve. Missy, we are ready to open up for questions.

Narrator (07:46):

Yes, sir. If you would like to ask a question over the phone, please press star followed by one. Please make sure your phone is unmuted and record your name clearly when prompted. If you wish to withdraw your question, you can press star two. Please allow a moment for questions to come in. Thank you. Our first question comes from Joyce. Your line is open.

Joyce  (08:28):

Yeah, thanks for taking my question. Um, uh, can you talk about the percent of, uh, children, uh, with sickle cell that do end up, uh, having a stroke? And also are there any, uh, concerns with hydroxyurea such as side effects that might make, uh, doctors less likely to recommend it or parents, uh, concerned?

Dr. Schieve (08:56):

Um, so in terms of your first question on stroke, before the use of TCD screening, which detects, um, children who are at increased risk of stroke and then those children can be given transfusion therapy, which markedly will reduce that risk. There was, um, there were several large studies that, um, documented that by the age of 20, 11% of children with sickle cell anemia would develop a symptomatic stroke. And it is, um, expected that the number of strokes might have even been higher because there can be many silent strokes, um, which can cause brain injury even though there are no signs or symptoms. Um, we do know that, um, since TCD screening and chronic transfusion therapy have been recommended and implemented, there has been some evidence that hospitalizations for stroke among children, um, with sickle cell anemia has decreased. But as this study shows, there’s a lot of work to be done as half of the children and teens who are recommended and eligible to get TCD screening, um, are still not getting they’re recommended screening. Um, in terms of hydroxyurea, um, there have been concerns about this treatment, um, in terms of, um, the potential for cancer, but there are now many, many years of study of this, um, medication and there has never been evidence, uh, showing that it causes cancer, especially as the dosage used to treat sickle cell anemia. Also, um, it’s important to note that even though the, the, uh, medication is has been more recently been recommended for use in children, it has been safely used in adults with sickle cell anemia since the 1980s.

Dr. Houry (10:55):

And and this is Dr. Houry, I would just add that these data are both promising and disheartening. When I look at them, I look at, we do see that individuals who have had acute chest syndrome, which is a severe complication of this disease that you know, almost half of the young children and about 65 of the older percent, the older children were on hydroxyurea. But when you look at it from the other side, this means almost half and almost 35% weren’t. And this is a severe complication. So these are children who should be receiving this medication. So as we’re looking at healthcare disparities, we have to question why are these gaps and ha what can we do to further lessen them so that we don’t have these children with these acute complications? As Dr. Schieve said, you know, sickle cell anemia has severe long-term life consequences and complications. We need to focus as providers and community members on how we can reduce those consequences through some of these screening and treatments.

Joyce  (11:55):

Thank you.

Narrator (12:00):

Thank you. Our next question comes from Jason, your line is open.

Jason (12:06):

Yeah. Thank you so much for, uh, all the time. Um, I guess two things here. One, I mean it seems like, uh, like usage is the primary, um, indicator of whether or not people are then getting, um, are well sort of at I suppose prior, um, sort of complications and then being a primary indicator of whether or not like looking, screening and then treatment. Do you talk a bit about what might be going on for people who are, um, suffering complications and then not getting, um, say, um, this drug or recommendations for routine screening? And then can you also talk a bit about any efforts besides reinforcing guidelines or making sure that, you know, people are getting these kinds of, um, interventions before they have already interacted with healthcare system many times because of complications? Thank you.

Dr. Schieve (13:01):

Um, so in terms of, um, I I’m sorry, I forgot the first part of the question.

Jason (13:08):

Uh, sorry. Um, that’s my fault for, uh, uh, being so long winded. Uh, basically what might be going on for people who are, who are sort of being hospitalized or suffering sickle cell, uh, complications, but are then not being sort of really recommended for these screenings or really, um, being, uh, given this drug.

Dr. Schieve (13:29):

Sure. So in this, in this report, we were not able to directly examine the reasons the individuals did not receive their screening or were not using hydroxyurea. But there have been, um, many studies previously that have looked at barriers to healthcare, particularly for these types of healthcare for the screening. Um, one of the, the big barriers patients face our logistical challenges. So for TCD screenings, there can be limited availability of appointments and there can be a distance between where the patient lives or where they receive their usual healthcare and where the specialized center that performs the TCD screening is. Um,

Dr. Schieve (14:13):

And, and, and then, um, for, um, hydroxyurea use, um, when, when individuals take this medication, they need to be monitored through laboratory tests to make sure that they’re getting the right amount of the drug. And so that can be challenging for, um, patients as well. In terms of providers, there are also some barriers to care. For example, care coordination is difficult. Providers have reported they often don’t get timely information back from, um, TCD screening programs in order to keep up with who, um, which, um, children, um, are due for their annual TCD screening. Um, then we also know that there’s just a general lack of healthcare providers with expertise in sickle cell disease and that, um, when patients do seek care and often they are seeking care in emergency departments, there’s particularly a lack of knowledge about their disease and patients often report experiencing, um, discrimination, having their symptoms dismissed and feeling like they’re not being taken seriously.

Dr. Schieve (15:24):

So all of these are, um, uh, you know, both the global and the specific barriers and challenges that need to be faced. In terms of recommendations and what people can do. Um, in terms of providers, hematologists and other healthcare leaders can work with policy makers and advocates, um, to try to bring attention to racism. Um, and the impacts that this has had on sickle cell funding research policy decisions, healthcare systems can develop formal reporting systems to document and respond to racist behavior and to empower people with sickle cell anemia to safely report their concerns about prejudice or inequity. And there needs to be more healthcare education that, um, specifically focuses on the treatment of minority groups and particularly those with sickle cell disease. Um, uh, in terms of the specific challenges, um, there have been some, um, hopeful studies of quality care initiatives that have used electronic healthcare records to try to better, um, implement tracking within clinics. And then, um, patients and families can learn about the importance about getting a TCD screen. The importance of using hydroxyurea and community-based organizations can really be critical in developing patient and provider resources and connecting patients and families with these resources and tools.

Dr. Houry (17:01):

And this is Dr. Houry, I would just give, you know, an example of my time in the ER. So I would see a patient come in with an acute pain crisis and I would manage that pain crisis. I wasn’t thinking about hydroxyurea. My goal was to manage the acute complication of the patient, um, in front of me. But what we need to make sure is that these electronic health records, as Dr. Schieve said, are communicating with each other so that when the patient is discharged, if they aren’t on hydroxyurea, the provider considers starting it at that time or linking back with a hematologist or the primary care provider that all have access to those records and this is where, um, prompts and electronic healthcare records and tracking can be really helpful to make sure that when we do have these evidence-based recommendations, that they are being followed. Because we know that in a system with many different providers, there are opportunities to miss this and so the more that we can make sure these prompts tracking systems and coordination are in place, this can help reduce this.

Ben Haynes (18:04):

Next question, please.

Narrator (18:07):

I’m not showing any more questions at this time, but again, if you would like to ask a question, please press star followed by one. Please make sure your phone is unmuted and record your name and your affiliation when prompted. Again, that is star followed by one. Please allow a moment for questions to come in.

Ben Haynes (18:53):

Missy, if there are no further questions, I’d like to thank everybody for joining us, uh, particularly Dr. Houry and Dr. Schieve for taking the time. If you have follow-up questions, please call the main media line at 404- 639-3286 or you can email media@cdc.gov. Thank you, this will conclude our call.

Narrator (19:13):

Thank you. That does conclude today’s conference. You may disconnect at this time. And thank you for joining.

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