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.
Venée Tubman, MD, MMSc, 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. Hydroxyurea was one of the only medications available, and stem cell transplant, while a potential cure, was only an option for the very few kids who had a donor available. But that’s been changing over the past few years.
“Now we have medications like L-glutamine, crizanlizumab and voxelotor, some of which were only approved within the last five years,” said Dr. Tubman. “And in December, two new treatments were approved by the FDA as the first cell-based gene therapies for sickle cell disease for patients aged 12 years and older.”
Gene therapy represents a new frontier in SCD care, enabling patients to be their own donor. This means that kids who previously lacked a matching donor for stem cell transplants now have another option. Texas Children’s has already successfully treated two thalassemia patients with gene therapy, signaling a new era of personalized treatment for blood disorders.
Dr. Tubman underscored the importance of staying informed about these new developments.
“Many parents know of sickle cell from when they were young, and their experience may be much different from what we can do today,” she said. “We didn’t have these drugs when many of them were growing up. Every child’s experience is different, and the treatments available now can really make a difference.”
More than medicine
In addition to cutting-edge treatments, Texas Children’s offers a range of support services designed to address the various complications of the disease. The program’s team consists of board-certified pediatric hematologists, pediatric pulmonologists, hematology-trained nurse practitioners and physician assistants, psychologists, social workers and patient navigators to help families cope not only with the disease’s medical challenges, but also with the social, emotional and financial issues that often arise as a result. The team also offers outpatient blood transfusions, pain treatment programs and specialized clinics, such as a multidisciplinary sickle cell pulmonary clinic.
“Sickle cell doesn’t just affect the blood,” said Dr. Tubman. “It can affect everything from vision to kidney function to learning. That’s why we work closely with specialists in every field to ensure our patients get the comprehensive care they need.”
Advancing research for the future
Dr. Tubman and her team are deeply involved in research to better understand the biology of SCD and improve treatment. In collaboration with engineers, they’re also developing new diagnostic tools, particularly for use in regions where newborn screening isn’t yet widely available.
One area of research is finding safe, effective ways to treat pain. Dr. Tubman recently studied using ketamine as an alternative to opioids in treating sickle cell pain. The side effects of repeat or prolonged opioid use, such as constipation and tolerance (lessening effects over time), can be significant. Exploring non-opioid options like ketamine offers additional pathways for managing pain.
Texas Children’s commitment to global health is another crucial aspect of its research efforts. Through the Global Hematology Oncology Pediatric Excellence (HOPE) program, the hospital is building capacity to treat children with sickle cell and other blood disorders in sub-Saharan Africa, where most sickle cell patients live. This international collaboration aims to dramatically improve the lives of children there, who would otherwise have few treatment options.
A time of hope
Despite the challenges of managing SCD, Dr. Tubman is enthusiastic about the progress that’s been made and optimistic about the future.
“This is a time of great hope,” she said. “We have a lot of work yet to do, but we’ve also had a lot of success in extending quantity of life for children with SCD. Our physicians and scientists are dedicated to pushing those gains into adulthood and improving the quality of children’s lives.”
If your child has been diagnosed with SCD or thalassemia, don’t wait. Call the Texas Children’s team at 1-800-226-2379 to learn more about the innovative treatments and resources available.