Welcome to the Future of Sickle Cell Care at Texas Children’s Hospital
Sickle cell disease (SCD) is a group of blood disorders that disproportionately affect Black children and families. For years, children with these conditions had very few treatment options, but recent advancements are transforming care. With one of the largest pediatric sickle cell programs in the United States, Texas Children’s Hospital is at the forefront of groundbreaking treatments and research aimed at improving the lives of children affected by this disease.
Dr. Venée Tubman, Co-Director of the Sickle Cell and Thalassemia Program at Texas Children’s, emphasized the program’s commitment to providing high-quality, equitable, and evidence-based care to every child and young adult.
“We see about 1,300 kids from birth to age 22 across our five campuses,” said Dr. Tubman. “Our goal is to not only provide the best care, but also to continue expanding knowledge through research.”
Better treatments are here
In the past, there weren’t many ways to treat SCD. However, recent advancements in medication and therapies have changed the landscape of treatment options. Dr. Tubman mentioned that medications like L-glutamine, crizanlizumab, and voxelotor have been approved in the last five years. Additionally, two new cell-based gene therapies were recently approved by the FDA for patients aged 12 years and older.
Gene therapy is a revolutionary approach in SCD care, offering new options for patients without a matching donor for stem cell transplants. Texas Children’s Hospital has already seen success in treating thalassemia patients with gene therapy, marking a new era of personalized treatment for blood disorders.
Dr. Tubman highlighted the importance of staying informed about these new developments, noting that the landscape of SCD treatment has evolved significantly in recent years.
More than medicine
Aside from cutting-edge treatments, Texas Children’s Hospital provides a comprehensive range of support services to address the various complications of the disease. Their team consists of experts from various fields, ensuring that patients receive holistic care encompassing medical, social, emotional, and financial support.
Dr. Tubman emphasized that SCD impacts more than just the blood, affecting multiple aspects of health. The hospital works closely with specialists to provide tailored care to address each patient’s unique needs.
Advancing research for the future
Dr. Tubman and her team are deeply involved in research to enhance the understanding of SCD biology and improve treatment outcomes. Collaborations with engineers have led to the development of new diagnostic tools, particularly beneficial in regions with limited access to newborn screening.
One area of research focuses on finding safe, effective alternatives to opioids for pain management in SCD patients. Exploring options like ketamine offers new pathways for pain relief, reducing the side effects associated with prolonged opioid use.
Texas Children’s commitment to global health is evident through the Global Hematology Oncology Pediatric Excellence (HOPE) program. This initiative aims to enhance treatment capacity for children with blood disorders in sub-Saharan Africa, where resources are limited.
A time of hope
Despite the challenges of managing SCD, Dr. Tubman remains hopeful about the future of treatment and care for patients. She highlighted the progress made in extending the lifespan and improving the quality of life for children with SCD, emphasizing the dedication of the hospital’s team in advancing these efforts.
If your child has been diagnosed with SCD or thalassemia, take action now. Contact the Texas Children’s team at 1-800-226-2379 to explore the innovative treatments and resources available.
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