Sickle Cell Disease: Treatment Goals and Guidelines

Neil B. Minkoff, MD: I’m going to use that as a segue. Now we have either the patient who was found at birth, at screening, or those other cohorts, a smaller cohort of patients, where you’re either picking it up in that first year of life or at age 6 to 10 they start to develop chronic pain, and these other symptoms you described. At this point, we’re going to start to talk about what do we do about that. We’re going to start therapy, so let’s start with the goals of therapy and then work backward from there. What are you trying to accomplish as your primary goal, and then are there secondary goals?

Ahmar U. Zaidi, MD: Absolutely. The primary goal for me as a physician is to minimize the long-term effect of having a sickle erythrocyte in your body to prevent the long-term organ damage that’s accumulating over a life span. The frontline idea there for me as a pediatrician is to prevent stroke. That for me is the most important piece in the care of these patients. Should we talk a little bit about how we get to the point where we’re trying to prevent stroke?

Neil B. Minkoff, MD: Sure, absolutely. That would move to the idea of getting to that point of preventing stroke. Before we get to that, what are the other secondary goals? You mentioned chronic pain, infection.

Ahmar U. Zaidi, MD: Reduction of pain, of course, is always something that is going to be an issue in all of these patients and preventing those emergency department admissions, keeping patients out of the emergency department, keeping them out of the hospital, helping to reduce their burden on their quality of life with chronic pain that they’re dealing with at home. Acute pain episodes are tremendously disruptive and unpredictable to patients. It interferes with their ability to go to school. It interferes with their ability to maintain jobs. It’s a huge burden that they have to carry. We of course also want to minimize the infectious risk that patients are at, and fever becomes a really big issue in the pediatric population where every child who has fever requires intravenous antibiotics and blood cultures to be monitored, which adds a tremendous amount of burden to the families.

Neil B. Minkoff, MD: The question that I would then ask you is about the use of guidelines and how well followed they are.

Ahmar U. Zaidi, MD: The best guideline I would say that we have is the 2014 NHLBI [National Heart, Lung, and Blood Institute] guidelines. And there are certain groups that are working on new guidelines with colleagues, sickle cell colleagues, around the country. But the 2014 NHLBI guidelines have a very strong emphasis on preventive care, preventive ways to reduce the burden that this disease has on individuals.