Great strides in managing sickle cell disease

In this series, Ahmed Hassan, Accordant General Manager and Vice President, speaks with members of our Medical Advisory Board about managing complex conditions and driving better outcomes for payors and plan members. Here he speaks with Biree Andemariam, MD, Professor of Medicine, University of Connecticut Health, and Medical Director, New England Sickle Cell Institute.

Around 100,000 Americans have sickle cell disease (SCD), which is an inherited blood condition.¹ For those who have SCD and reach the age of 45, their total lifetime health care costs are estimated to be $1 million. Annual costs for health care for those with SCD range from more than $10,000 for children to more than $30,000 for adults.² Increasing provider understanding of how to manage patient pain and avert complications are key tools in lowering costs and improving care.³

How did you become involved with working with SCD? What inspires you to keep focusing on SCD?

Early on in my career as a physician, I noticed a trend when I was providing hematology and oncology consults in the hospital. I kept seeing the same young adults being admitted into the hospital for long periods of time because of pain related to SCD.

As I got to know these patients better, at least part of the reason that they were admitted so frequently is that they weren't being followed longitudinally once discharged and they weren't being started on medications that could help prevent these pain crises. They weren't being monitored for possible complications associated with SCD. I decided I needed to be able to help this population of patients suffering. I started an adult sickle cell center at my institution in 2009.

According to the Centers for Disease Control, the mortality rate for children with SCD has fallen in recent years within the United States. What changes have happened in the United States to lower the mortality rate and help people with SCD to live longer?

We now screen every child born in America for SCD regardless of race, ethnicity, or family history. We identify those with SCD at birth. This is important because infection from pneumococcus was the leading cause of death in children with SCD. Now when newborns are identified as having SCD, they are put on prophylactic penicillin twice a day every day until the age of five – which confers a significant survival benefit in children with SCD.

More than 99 percent of children born today with SCD are expected to survive into adulthood. What are the challenges now in taking care of an adult population?

A 2010 study looked at the Dallas SCD newborn cohort and found at the time of this publication a 99 percent survival rate into adulthood. But they also found the highest rate of death occurred when the patient was transferred from pediatric care to adult care, with the first year of transfer carrying the highest risk. What this demonstrates is that we — the medical community — do a poor job of transitioning children with SCD over to adult care.

Some issues around access to care include finding a willing and knowledgeable physician, but also transportation limitations. Some individuals with SCD live very far from centers of excellence or specialty care. Then there are often competing psychosocial stressors in this population, particularly in adults with SCD, which is a chronic, debilitating and progressively worsening disease.

As young adults transition out of pediatric care, they often begin to assume the responsibility of managing their condition on their own for the first time. What would you say are the top positive indicators that determine the outlook for how successful they are?

It starts with assessing how well-informed that young adult is about their disease. Do they know their exact sickle-cell genotype? Do they know the names and doses and frequency of their medications, and understand why they’re taking them? Do they know the name of their physicians, how to get to their offices, and how to make and reschedule appointments? Do they know the name of their pharmacy, and how to fill and refill a prescription? In my experience, patients who can do those things around the time of transfer usually have the greatest success because those skills translate into good disease self-management.

As you may know, CVS Health offers a care management solution with Accordant that provides additional support for those managing chronic conditions through the help of a dedicated nurse. What effect do you think this added support could have on a person living with SCD?

The more support we can provide a patient outside of the doctor's office, the better. Many patients living with chronic conditions, including SCD, grapple with impediments and barriers within their home environment that make it hard to self-manage their disease. That's where I think assistance from a nurse who can be in touch with the patient is key. The nurse can help identify barriers and solutions to those barriers, whether it be transportation challenges, understanding when and how to use medications, or reinforcing treatment plans that have been outlined by the physician. All of these things are really useful in supporting the patient and helping them be successful in managing their condition.

What makes you optimistic about advancements for treating SCD? How does gene therapy play a role?

We’ve come a long way in a short time, with a second FDA-approved drug in 2017, then a third and a fourth in 2019 — plus dozens of novel treatments that are in clinical trials right now. I anticipate that many new drugs will be approved in the coming years, and physicians will be able to finally tailor their treatment recommendations to the individual patient in front of them.

Gene therapy is also very exciting because we are talking about something that's a potential cure. We know that SCD can be cured by an allogeneic* bone marrow transplantation. In the case of gene therapy, it is an autologous bone marrow transplant from the person back to themselves. Once those cells are treated, either by gene editing or gene addition, they are then infused back into the patient. As they return to the bone marrow, they grow and replicate. We hope they produce healthy bone marrow that doesn't allow the making of sickle red blood cells.

Gene therapy is still just in clinical trials, but there are several strategies that look very promising. And the fact that we are making strides in both the drug pipeline and gene therapy gives us a lot of hope for the future of these patients.

Patients can access help with a dedicated nurse and other care management solutions with Accordant for support of their chronic conditions, including SCD. CVS Health helps to manage difficult conditions to lower health care costs and improve member experiences.

*Allogeneic meaning that the bone marrow is obtained from a well-matched donor.

The source for data in this document is CVS Health Enterprise Analytics, unless otherwise noted.

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