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Jonathan Alicea is an assistant editor for HCPLive. He graduated from Princeton University with a degree with English and minors in Linguistics and Theater. He spends his free time writing plays, playing PlayStation, enjoying the company of his 2 pugs, and navigating a right-handed world as a lefty. You can email him at email@example.com.
A sickle cell expert reflects on the state of care and access for sickle cell communities.
Today is World Sickle Cell Day, an opportunity for healthcare providers, advocates, and patients to reflect on and raise awareness for the state of care for sickle cell disease.
While scientific and therapeutic advancements have been made for these patients, there is still so much work that needs to be done, notes Kim Smith-Whitley, MD, executive vice president and head of research and development at Global Blood Therapeutics. The issues that sickle cell communities consistently face, as exacerbated by their physical and socioeconomic vulnerabilities, are particular and nuanced.
In an interview with HCPLive®, Smith-Whitley spoke on the importance of World Sickle Cell Day, what it means for affected communities, the therapeutic advancements, and how healthcare providers can continue seeking educational opportunities to better care for their patients.
HCPLive: What do you consider to be the importance of World Sickle Cell Day for you and the healthcare community?
Smith-Whitley: As I'm sure you're aware, sickle cell has been often overlooked, and communities have been impacted by healthcare disparities and social inequities. And although, as scientists, we took a deep dive into sickle cell disease to understand the genetic variation that actually led to this condition, but science kind of abandoned sickle cell patients in the process.
Despite the first person being described with sickle cell in 1910, we still do not have good access to high quality care in 2021.
So, I think that the spirit of World Sickle Cell Day— when it was sponsored by the United Nations in 2008—was to increase awareness of sickle cell. But I believe this was not only to increase awareness in general, but to increase awareness of the journey of those individuals living with sickle cell disease—to emphasize their experience, to emphasize the way that they have to deal with the stigma and the way that they have had limited access to therapies.
We've been limited with a number of disease modifying therapies for sickle cell disease, only reaching 4 in November of 2019. So, when you compare that to other inherited rare diseases, it really is quite low in number, and that's a discrepancy that we need to impact.
For me, the spirit of World Sickle Cell Day is to raise awareness of those healthcare disparities and to emphasize the impact of the social inequities. Part of this spirit is to talk about the stigma that individuals with sickle cell disease have to overcome, particularly as individuals who are drug seeking for their pain episodes, and raise their voice. This is all to allow us to understand the experiences of those both nationally and globally with sickle cell.
You said there are few disease modifying therapies for patients with sickle cell. Can you just provide an overview of where we are now and what you see as hope and potential for the future?
One of the striking things to me is that, although sickle cell has been in the US since 1910, we really are not doing more than just supportive care for the majority of these patients. We use medications to treat the pain, but we don't have good therapies that treat the underlying cause of that acute pain.
And so, for individuals living with sickle cell disease, and really until the 1990s, we didn't have specific disease modifying therapies. We would use transfusion therapy to try to prevent complications or to lessen the severity of complications once they occurred.
We had antibiotics that we could use to decrease infections, which was very important to increasing survival in childhood and was the basis for newborn screening. But we really didn't have therapies that could prevent some of the complications of sickle cell disease—until hydroxyurea therapy.
And then we had an agent L-glutamine (Endari) that came along about 20 years later. This helped address the antioxidant stress associated with red cell health.
And finally, in November of 2019, we had two new therapeutic agents available for us: crizanlizumab (Adakveo) and voxelotor (Oxbryta). One of the wonderful things about these two agents is that these drugs were developed specifically for sickle cell disease.
When we had L-glutamine and hydroxyurea, we were kind of borrowing from other processes in order to apply them to sickle cell disease. But these two agents were directed specifically at sickle cell from the start.
And one of the things that was, I think, very beneficial about these two agents is that they came at a time where we were having almost a slump in awareness about sickle cell disease.
Crizanlizumab was used to decrease pain in individuals with sickle cell disease. It is a piece of lectin inhibitor that you get once a month through an IV in order to decrease pain episodes.
And then we have voxelotor, which is an agent that prevents sickle hemoglobin polymerization and improves red cell health. Subsequently, hemoglobin levels rise and the early destruction of red cells or hemolysis decreases. And I think that individuals have a sense of improved health when they're taking x-rays.
These agents then came along, and then the pandemic hit. Now, individuals with sickle cell disease are probably less likely to hear about these agents because I'm sure that their medical teams were focused on trying to communicate the complications of COVID-19 infection, trying to educate them on how to lower their exposure.
One of the things that is distressing to me after having come through the pandemic is a lack of sense of urgency about getting these disease modifying therapies to individuals with sickle cell.
I think that there is a false sense of confidence in the sickle cell provider community and thinking that our patients are doing okay. Because they may be on hydroxyurea, they may not have frequent pain episodes—but again, that is not preventing progression of chronic organ damage.
We really need to take a step back and look at what we need to do to get voxelotor in the hands of individuals with sickle cell disease. We should do our best to improve red cell health and to limit, hopefully, what I believe will be chronic organ damage in this patients.
What is your advice to healthcare providers who are looking to find more opportunities to educate themselves and others on sickle cell disease, its related complications, and the therapeutic options?
I think you should always listen to your patients and their families first. Talk to your patients about what they're experiencing and educate your patients about what's available to them.
When it comes to discussions around disease modifying therapies, this is an opportunity to bring individuals living with sickle cell disease into the decision making process. This is respectful and compassionate care, giving individuals the information that they need to make a decision alongside you.
And so, you should be educating yourself as a health care provider about what these options are. They should be going to conferences. Global Blood Therapeutics recently has been at EHA and at the FSCDR conferences. We are very prominent in the ASH meeting as well.
Educate yourselves by going to conferences, accessing information online, and learning more information about Oxbryta and the benefits that it can provide for individuals with sickle cell disease.
I think that that is a challenge they take up on World Sickle Cell Day, which is designed to bring awareness to sickle cell and awareness to the patient experience. And as a health care provider, I think that part of that is to try to find ways to increase a trusting relationship with your patients, to give them all of the options necessary in order to have a healthier life.
And so, I think that individuals who care for people with sickle cell disease, whether or not they are a healthcare provider, or a caregiver, can do patients with sickle cell a service by educating them about the options for disease modifying therapies.