Impact of Anemia & Hemolysis in Sickle Cell Disease - Episode 12
Experts in hematology address unmet needs and precautions patients can take to avoid complications of sickle cell disease.
Jeffrey D. Lebensburger, DO, MSPH: The first thing that our patients can do to prevent sickle cell complications is to be accepting of sickle cell disease-modifying therapies. It is important for our patients to appreciate that sickle cell disease is not just pain, but something that can impact your entire body and your quality of life. Being accepting of a sickle cell modifying therapy is vital. There’s a lot of work that we have to do as clinicians and health care providers to gain the trust of our patients because trust is the most important thing in our patient relationship, to build that trust and to get them to understand the importance that this is not just a disease of pain events, but that this is a multisystem and life-limiting disease. We strongly recommend a very healthy lifestyle. Many patients try to shy away from very strenuous exercise, which we agree with, but making sure that they have a healthy lifestyle has to include at least some moderate level of exercise. Being well hydrated is important for a patient with sickle cell disease and to maintain hydration even during sickle cell events.
Another thing that’s anecdotal, but very prevalent, is other healthy lifestyle things. For example, when patients are swimming, making sure they come out and get themselves warm. During the cold months, making sure that they’re very well dressed, so they don’t have the complications that can happen when they get themselves very cold. Or in the summer months when it’s very hot, maintaining that hydration despite being out in in the hot summer months is important.
High altitude also can put patients at risk for events. A very healthy lifestyle is important to our patients with sickle cell disease. That starts with sickle cell disease modifying therapy, but then doing other things in terms of eating right, exercising, and maintaining their body’s temperature are vital to protecting the lives of our patients.
John J. Strouse, MD, PhD: I did both combined pediatric and adult hematology training. For the first 14 years of my career, I was more pediatric-focused with just a toehold in adult sickle cell. For the last 5 years, I’ve been directing an adult sickle cell program, with a little bit of pediatric sickle cell disease in my practice. It’s quite clear that as people with sickle cell disease get older, we see disease progression, which includes developing more of a chronic pain phenotype that’s generally in adolescents and young adults. We see that developing, which leads to more daily impact of their pain, and more chronic pain that can really affect their quality of life and their ability to complete their daily activities, things like school, work, and family. We also see progressive organ dysfunction, such as elevations in creatinine, which are relatively uncommon in children, but become much more common in adults. We see more pulmonary hypertension. We see the long-term impact of anemia on the brain. All of that is progressive into adulthood. We see this progression in disease that affects multiple systems. We see orthopedic complications, such as avascular necrosis requiring joint replacement, which is relatively uncommon in children, but becomes more frequent decade by decade as people get older. We see more venous thromboembolism as people go from being adolescents, to young adults, and to older adults. One of the things that has been occurring in sickle cell disease is, with effective newborn screening and early preventive care, and better disease-modifying therapies, it’s rare that we see children with sickle cell disease die from complications of the disease. We have this increasing group of adults and older adults, where the progressive organ dysfunction from sickle cell disease is causing impact in their daily life, and that can be on physical functioning, cognitive functioning, or their activities of daily living.
This is what we think disease-modifying therapy helps with. We think that therapies that make their sickle cell disease more mild and help prevent acute complications will likely have positive impacts on some of the disease progression. It’s hard to study disease progression because it happens gradually, and it takes long studies and long-term follow-up. We don’t have as much evidence as I would like about the impact of disease-modifying therapies on chronic organ dysfunction, because of these challenges with studying. But we have little bits and pieces that being on hydroxyurea can lower your cerebral blood flow velocity. Maybe it helps prevent silent cerebral infarct, and it can certainly help prevent a second stroke after people have had one stroke. We have some information that being on hydroxyurea can lower the amount of albumin that is in people’s urine. Once people develop albuminuria, then putting them on hydroxyurea can decrease that. We know from other disease settings, such as diabetes, that if we decrease the amount of albumin in people’s urine, that that can decrease their progression of renal disease. We have these suggestions that hydroxyurea, and potentially some of the newer disease-modifying therapies, can prevent disease progression.
Sophie M. Lanzkron, MD, MHS: Lots of people have concern about dependency on opioids for people with sickle cell disease. Data suggest that the risk of developing dependency on opioids is less, or at most, the same as the general population. When you’re using opioids for any patient population, you have to pay attention to the risks of the misuse of opioids. Sadly, we don’t have great therapies to manage patients’ pain. At this point, we don’t have a medication that can stop a crisis once it has started. So, we have to rely on opioids to treat people who are presenting with acute pain. I see it as my job to keep an eye on my patients who are on opioid therapy, not the emergency department’s job. The primary sickle cell doctor and some pain management people should pay attention to how patients are using their opioids and whether the opioids are working. In the acute setting that is the primary therapy, but probably about 60% to 70% of adults with sickle cell disease develop chronic everyday pain. We don’t have great therapies to manage chronic everyday pain. We are often relying on chronic opioid therapy. We all have to be careful about our use of opioids. In patients who are functional, taking care of their families, going to work, who happen to take chronic everyday opioid therapy, the key is that they’re functional and able to do the things that they need to do despite the fact that they require chronic opioid therapy. The people we should be thinking about alternative therapies for are people who don’t respond to chronic opioid therapy and still have pain that’s a 7 out of 10 every day, who are in the hospital a lot, who aren’t able to go to work, which suggests that the chronic opioid therapy isn’t working for that population. You need a good team to think about alternatives to the use of chronic opioid therapy.
Transcript edited for clarity.