This is the first of our series about various diseases and health conditions that we are calling, “Get to Know.” We’ll provide some facts you may not have known about rare and not-so-rare diseases. And you’ll also get to hear from patients with the conditions and their caregivers explaining about the disease.
Our first is, Get to Know Sickle Cell Disease.
Those of us at Rare Patient Voice have gone to many Sickle Cell Disease (SCD) patient events over the years and have met many patients and caregivers of this painful and misunderstood disease. Sickle Cell Disease is an inherited blood cell disorder. The patient doesn’t have enough healthy red blood cells to get adequate oxygen to all parts of the body. In an SCD patient, the red blood cells look “C” shaped (that is where the name Sickle Cell comes from). They can get stuck in the small blood vessels. This causes great pain and can also cause other medical problems such as infection and stroke.
There haven’t been new treatments for SCD for many years. The good news is that several companies are now working on new treatments that should be coming out over the next few years. That is why we’ve been having so many surveys on SCD recently – companies want to learn what patients and caregivers are going through.
There is an interesting reason why SCD exists. For those that are carriers (called sickle cell trait), it offers protection against malaria. That is why it affects those whose ancestors lived in areas where malaria was common, such as Africa. We tend to think of most sufferers of SCD as being of African descent, and many are, but it also affects those from Central and South America, the Middle East, Asia and the Mediterranean area.
One of the unfortunate things about SCD is that patients, when they are having a severe pain crisis, may need to go to the emergency room and need treatment for pain. We’ve heard from patients who weren’t treated well, as they might appear to just be wanting opioids for an addiction. The more people know about this difficult disease, the better, so that the patients are treated with the respect they deserve.
Those of you who are patients or caregivers with SCD, let us know your thoughts. Please tell us how you would describe Sickle Cell Disease. And if you’d like to make a video about it, we’ll help you do that!