By Dr. Kevin Williams (Chief Medical Officer, Pfizer Rare Disease Unit)
The “Ask Dr. Kevin” series is brought to you by Pfizer Rare Disease in collaboration with the National Newspaper Publishers Association (NNPA) to increase understanding of sickle cell disease.
With National Sickle Cell Awareness Month just next month, I’ve been thinking a lot about the challenges that people with sickle cell disease (SCD) face and what can be done to make a meaningful change in their care.
Evolution of the science behind SCD has advanced in recent years, resulting in a new understanding of the disease. However, this knowledge is not commonplace among individuals living with SCD and their healthcare professionals. As with any disease, better understanding and awareness of the condition is the foundation for improvement in appropriate and timely care. Recently, the Sickle Cell Disease Council For CHANGE (CFC) has set their sights on raising awareness of the expanded science behind the disease among patients, their families, and healthcare professionals.
The CFC, a group convened by Pfizer and inclusive of more than 20 experts, advocates, and patients working to improve care for SCD patients, is looking at this issue as part of a recently released report called “Improving Vaso-occlusive Crisis (VOC) Management.” In the report, the CFC explains how the understanding of SCD has evolved through the years, and outlines recommendations for how to get this information into the hands of those who can use it—healthcare professionals, emergency department staff, and people living with SCD—to make a positive change and improve patient care.
Here is an overview of what the CFC found, which is outlined in the report:
The Expanded Science Behind the Disease
Let’s start at the beginning. A common (and somewhat outdated) view of SCD is that complications from the disease are caused by a simple “plumbing malfunction.” In other words, it is only the sickled red blood cells that clump together and cause blockages in blood vessels that lead to pain and organ damage.
Now it has been understood that the underlying science is more complex than this.
Recent breakthroughs in the understanding of SCD have shown in fact that the vaso-occlusion (or blocking of minute blood vessels involving multiple pathways and different types of blood cells), is a key factor underlying all of the different manifestations of the disease.
In SCD, these rigid, crescent-shaped cells have difficulty passing through small blood vessels and irritate the internal lining of these vessels. This causes the lining of blood vessels to become “sticky,” and as a result, not only the red blood cells, but also white blood cells and platelets clump together. This can further contribute to obstructions in blood flow and inflammation. These events can occur even when there are no outward signs or symptoms, ultimately causing several of the complications seen with SCD.
Other factors that contribute to vaso-occlusion may include stress, the thickness of a person’s blood, as well as infection or conditions that commonly trigger a painful crisis.
The most common symptom of vaso-occlusion is the pain crises (or vaso-occlusive crisis – VOC). VOC is the cardinal manifestation of SCD and is often thought of synonymously with SCD.
In addition, there is an increasing awareness of another SCD complication—one that is often overlooked: chronic pain. VOCs are sudden, intense episodes of pain, while chronic pain, especially for older adolescents and adults, is defined as ongoing pain that has been regularly present over a period of the past six months.
What makes it more complicated? People with SCD can experience VOCs while also suffering from chronic pain. And while VOCs are routinely treated with opioid medications, chronic pain should be managed separately and with a different approach. Ongoing, long-term use of opioids can lead to drug tolerance, which causes people with SCD to require higher doses. However, according to the report, considering alternative pain relief options when appropriate, along with regular reassessment of medication use and dose, may be helpful in minimizing this issue.
Getting the Word Out
It is great news that the understanding of the science of SCD has evolved, but it is important to make sure that the right people, especially patients and their doctors, are aware that SCD is far more complex than being just a disease of red blood cells in order to make a meaningful difference.
Armed with this information, individuals living with SCD and their healthcare professionals better understand and explain the varied symptoms an individual can have. They can make more informed treatment decisions for their SCD patients, and people with SCD can be empowered to advocate for better care. Additionally, this new understanding of SCD among both healthcare professionals and patients can lead to better communication and more positive interactions in the future.
The time for change is now, and the CFC is working to get the word out about the urgency around improved care for SCD. Learn more about the Council here and read their first report here.
