Expert Q&A: 2021 World Sickle Cell Day

Sickle cell disease is an inherited blood disorder that impacts the red blood cells that carry oxygen in the body. These cells take on a sickle or crescent shape that tends to clog small blood vessels and block blood flow to the tissues and organs.

In recognition of World Sickle Cell Day on June 19, Dr. Andrew Campbell, Lisa Thaniel and Dr. Deepika Darbari of the Children’s National Comprehensive Sickle Cell Disease Program sat down with us to discuss sickle cell disease, its impact on kids and how Children’s National is leading the charge to help those diagnosed with this disorder.

How are children with sickle cell disease and their families affected by this blood disorder?

Andrew Campbell, MD, Director, Children’s National Comprehensive Sickle Cell Disease Program: Sickle cell disease can be devastating to the health and well-being of children. It’s the most common genetic disease diagnosed by newborn screening in the United States. Daily treatments for babies with sickle cell disease start by 2 months. As babies age, pain becomes the hallmark of this disease. Recurrent, unprovoked pain “crises” in the bones of patients can last several days at a time. The pain frequency and severity generally increase as a child with sickle cell disease grows older.

Acute complications from sickle cell disease can lead to difficulty breathing and hospitalization. Some children even have strokes at a young age. Sickle cell can cause ongoing damage to multiple organs throughout the body as patients enter their teens and twenties.

Lisa Thaniel, DSW, LICSW, LCSW-C, Clinical Social Worker: Children and adolescents with sickle cell disease may experience numerous complications that lead to frequent emergency room visits and hospitalizations. These impact family life, school attendance and social activities. Our patients also face a greater risk for neurologic complications that can cause cognitive impairment and lead to poor academic achievement.

Many children with sickle cell disease are at increased risk for depression and anxiety. Their parents can also experience high levels of stress. This can affect a family’s financial security and interpersonal relationships. Socioeconomic and psychosocial programs are crucial for building resilience in these patients and families.

How is the Children’s National Comprehensive Sickle Cell Disease Program leading care in our region?

Deepika Darbari, MD, Hematologist: We care for one of the largest populations of children and young adults with sickle cell disease in the United States. Children come to us as soon as newborn screening detects sickle cell disease. Each patient has a core team that typically includes physicians, nurse practitioners, psychologists and social workers who work very closely together. A patient’s core team also coordinates with different subspecialities, including physical therapy, bone health, orthopedics, pulmonology, neurology, pain and others. Our goal is to pay close attention to all aspects of our patients’ needs to provide them with the best, most comprehensive care possible.

Our Infant Sickle Cell Program and Sickle Cell Adolescent Transition Program ensure that each patient and their family have the resources they need to navigate this disease from birth all the way to adulthood. We also work with families to provide access to newly approved therapies and promising clinical trials that may not yet be available outside of a research study.

AC: We’re also a leader in bone marrow transplants for sickle cell disease patients. This procedure can cure sickle cell disease instead of simply treating its symptoms. More children receive bone marrow transplants for sickle cell disease at Children’s National than anywhere else in our region. It’s also one of the leaders in pediatric clinical trials for sickle cell disease bone marrow transplantation in the country.

We’ve launched several new, innovative bone marrow transplant studies that show a lot of promise for improving outcomes and care. One such study is testing a new method that uses less chemotherapy prior to a transplant. This type of approach could prevent the negative side effects of chemo and get kids back to doing the things they love faster. Most recently, we are the only program in our area where children can receive gene therapy as a curative option sickle cell disease.

What is the global impact of sickle cell disease?

AC: Sickle cell disease is a global condition. Outside of the U.S., the burden of sickle cell primarily lies among countries like Ghana, Nigeria and Congo in Sub-Saharan Africa. However, sickle cell exists in very high numbers in South America, Central America, the Caribbean, Middle East and India. In fact, India has the third highest population of cases in the world.

Our sickle cell patients reflect that same diversity. We see kids from all those regions. Being able to help children and study sickle cell disease from so many different populations provides much needed insights. This, in addition to our research collaborations in Europe and Africa, gives us a better understanding of the disease that can have an impact worldwide. So, we’re not just a leader in sickle cell disease regionally — we’re a leader internationally.

LT: Many of our patient families emigrated from various countries around the world so that their children can have access to the best care possible here at Children’s National. We care for children from Nigeria, Ghana, Sierra Leone, El Salvador, Afghanistan and Iraq, among so many others.

Children’s National Hospital is a diverse community that employs more than 3,000 healthcare providers from around the world. As a result, our providers can meet the unique social, cultural and linguistic needs of our patients and families. Families value their ability to obtain the best care from providers who reflect the cultural and ethnic diversity of our community.

What treatments are available to help children manage the pain caused by sickle cell disease?

DD: As Dr. Campbell said, pain is the most common complication experienced. Episodes of acute pain can start as early as 4 to 6 months of age and occur throughout life. Standard treatment for sickle cell pain includes hydration, as well as non-steroidal anti-inflammatory drugs (NSAIDs) and opioid pain medications. Opioids are a mainstay therapy for acute pain. However, their use, especially long-term, can have many side effects. Research into preventing chronic pain in our patients is an integral part of our work. This includes a multitude of non-pharmacologic approaches.

LT: While most studies show that opioids provide short-term relief, their numerous side effects include constipation, enhanced sensitivity to pain (hyperalgesia) and risk of dependence and addiction. Opioids also are associated with numerous co-occurring psychiatric disorders like depression and anxiety.

Our Integrative Clinic for Sickle Cell Disease offers non-pharmacological therapies that help our patients manage the stress and pain that comes with sickle cell disease. Patients in this one-of-a-kind clinic have access to acupuncture, mindfulness, healing touch, massage therapy and aroma therapy along with standard sickle cell treatments. We’ve seen that this multidisciplinary, comprehensive approach can be really effective.

How is Children’s National advancing care for kids with sickle cell disease?

AC: Children’s National is a leader in clinical trials for children with sickle cell disease around the world. These trials allow our patients to have access to cutting-edge treatments that aren’t otherwise available. For example, we’re one of just a handful of sites globally that’s doing gene therapy trials for sickle cell disease. We’re using techniques like stem cell transplants to cure patients of their disease. These patients are now doing things — everyday things many of us take for granted — that they could never do before. Every child cured of sickle cell disease is a lifetime of pain prevented.

There are even more breakthroughs on the horizon. The support of our community can provide the resources needed to fuel further innovation. We are looking for philanthropic partners who can help bring hope and healing to kids with sickle cell disease in D.C., across the U.S., and around the world. Every gift, of any size, helps children.

To learn how you can support sickle cell disease care and research at Children’s National, please contact Bethany Metzroth at [email protected] or 301-565-8520.

Make a Difference

Your charitable donation changes children’s lives. Support exceptional health care and discoveries that offer hope, healing and brighter futures.