Individualized Therapy in Sickle Cell Disease


Ify Osunkwo, MD, MPH: I want to segue to a different topic, looking at currently available treatments. Sickle cell disease has been around for a long time. There initially had been only 1 FDA-approved drug, and that’s hydroxyurea. Over the past 5 years we’ve had several other drugs come to market. I’m going to talk Dr Kanter. What are the goals of treatment for sickle cell disease? How do you determine what the treatment plan should be for your patient when they show up in your clinic?

Julie Kanter, MD: I always tell my residents and students, you must always meet the patient where they are. I always start with trying to get to know my patients a little: what are you interested in, what are your goals? If they’re 18 or 19 years old, maybe they finished high school. Do you want to go to college? It’s important to understand what their personal goals are as well as their specific goals related to their sickle cell disease.

Sickle cell is involved so much in their lives that it’s important to know both sides. Then from there we can talk about how to best prevent some of the complications. Always try to remind patients that pain is the hardest thing to deal with and that we know that that’s a big problem. There are other things too. We have to make sure they’re getting their eyes checked regularly, because blindness is a terrible complication. Also, getting hearts, lungs, and kidneys checked.

We really talk about the goals of preventing organ dysfunction. We make sure that we do appropriate screening and use therapies that can help reduce damage, then we also make sure that we reduce things that cause pain. While some crises are completely unpredictable, we also know that people can induce a crisis by perhaps stressing themselves out so much that they forget to drink water and they forget to eat. It would never, ever be intentional if we help them learn how to do better stress-relieving activities, stay hydrated, and avoid things like alcohol that can also dehydrate them.

Ify Osunkwo, MD, MPH: The goal is to have a treatment plan for each patient that is individualized, specific to their needs, their goals, and what their clinical situation is.

Transcript edited for clarity.

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