February 17, 2020

When providers are evaluating how well they care for patients, they look to something called a risk-adjusted score to steer them in the right direction. Accurate risk scores are contingent upon solid patient data, which is abundant in the adult population but scarce when you are looking at children, especially children with complex conditions.

“Children are not little adults,” said Trudy Leidich, vice president of quality and medical staff services. “The course of care for a cardiac patient in pediatrics is very different than a cardiac patient in adults. We need to understand those differences so that we can better design the future of care for these kids.”

That’s why Texas Children’s, along with Children’s National Hospital, have invested in Configo Health Inc., a health care data and analytics company specializing in pediatrics.

Configo Health is addressing the lack of credible benchmarked data accessible both to administrators and clinicians working in pediatrics, and to patients and their families. Using both machine learning and more traditional retrospective analytics, Configo Health delivers actionable insight with near real-time relevance to improve patient safety and achieve better outcomes for children.

By creating a dataset that will cover the breadth of pediatric care, Configo Health will provide public ratings organizations with the option to use risk-adjusted performance data to assess the quality of care delivered by hospitals across the country more effectively.

“Today’s six month lag for benchmarked pediatric outcomes data just isn’t acceptable,” said Texas Children’s System Chief Quality Officer Dr. Eric Williams. “Configo’s move to near real-time insight will be a game changer for driving improvement. Quicker access to this validated data means better decisions can be made at the bedside leading to improved outcomes for the patients and families we serve.”

Configo’s analytic tools will integrate multiple pediatric data sets to accurately establish standardized risk scores, allowing providers to internally and externally compare themselves and push for better patient outcomes. Providers at Texas Children’s and Children’s National will be able to critically look at patients’ health outcomes, their risk scores, and understand why they had the outcome they did.

“If you look at just pure administrative data, it’s not going to capture the outcomes that you need to see in that complex patient population,” Leidich said. “We’re leveling the playing field.”

Connecting various data sources and generating a standardized pediatric risk score will also help children’s hospitals better compare their performance to each other.

“Having a risk-adjusted database allows for children’s hospitals that are big and complex to understand what we really are improving on and if we are outperforming,” Leidich said. “We’ll know there’s a value to the care we give.”

Experts in extracting administrative data from pediatric hospitals, Configo Health provides HIPAA-compliant processes, secure data storage facilities, data cleansing and sophisticated analytical processing. By analyzing and evaluating data, organizations can reduce serious safety events, reduce mortality rates, optimize length of stay and reduce readmissions.

“We are delighted to announce Texas Children’s Hospital and Children’s National Hospital, two of the top children’s hospitals in the nation, as our founding investors,” Configo Health CEO Craig Manson said. “These relationships go well beyond capital and will allow us to develop solutions that truly meet the needs of pediatric hospitals and their patients and families.”

February 10, 2020

Texas Children’s and Baylor College of Medicine pediatric ophthalmologist Dr. Evelyn Paysse and Baylor College of Medicine refractive surgery specialist Dr. Mitchell Weikert have spent nearly two decades giving children with certain eye conditions hope of better vision.

The work they are doing focuses on children with amblyopia, a condition that if left untreated can lead to permanent vision loss.

“Amblyopia is a condition where the vision, in either one eye or both eyes, does not develop normally,” Weikert said. “If one eye has a lot more need for glasses, the brain may ignore that eye, and focus on the eye that’s creating a clearer image. If that happens, and it’s not discovered, or isn’t treated optimally, a child may never develop the vision they could have had in that eye and might end up with what we call a lazy eye, or amblyopia.”

The solution Paysse and Weikert are offering to patients who have not had success with traditional therapies such as wearing glasses or contacts, and patching the better-seeing eye, is a procedure called photorefractive keratectomy or PRK.

“Photo refractive keratectomy is a surface ablation of the cornea using an excimer laser,” Paysse said. “The laser shaves away, or somewhat sculpts, the cornea, taking away a certain amount of it to reduce the refractive error.”

The desired outcome of PRK is that the child’s refractive disorder will be improved or corrected and that their brain will then be able to better develop the vision in the weaker eye. Thus far, the majority of the more than 180 patients Paysse and Weikert have operated on have had excellent results.

“We find that the patients respond better if they are treated at a younger age,” Paysse said. “The reason is that amblyopia is a time-sensitive problem that you really must treat before the patient is visually mature in the brain.”

One patient who had success with PRK is 8-year-old Lila McLaughlin. Lila was born three months early with a myriad of vision problems that eventually led her and her mother, Laura, to Paysse, who performed PRK surgery on Lila just before her second birthday. The surgery, and a subsequent procedure, changed Lila’s life.

“She went into surgery and came back with what I say was a new set of eyes,” said Lila’s mother, Laura McLaughlin said.

Prior to PRK surgery, Lila’s vision was deteriorating quickly causing the toddler to become legally blind in one of her eyes. After undergoing PRK, Lila could see almost perfectly.

“This surgery has given her a healthy outcome,” Laura McLaughlin said. “Lila has blossomed into a phenomenal, fun-loving, feisty firecracker of a third grader.”

Lila herself is extremely thankful for the care she received from Paysse and others at Texas Children’s who have participated in her care.

“She’s a really great doctor,” Lila said. “She successfully helped me see way better than I used to.”

Currently, PRK surgery is not approved by the Federal Drug Administration in children. All the patients Paysse and Weikert have treated are part of a study led by the Institutional Review Board of Baylor College of Medicine, a consortium of committees that checks doctors’ protocols making sure they are designed safely.

January 21, 2020

For the second consecutive year, Transplant Services at Texas Children’s was named the top pediatric transplant center in the US, performing a remarkable 106 solid organ transplants in 2019. That figure includes the highest volume of pediatric liver and lung transplants in the United States.

For the year, we performed:

  • 36 liver transplants
  • 14 lung transplants
  • 27 kidney transplants
  • 29 heart transplants

In addition to top notch volumes, the transplant team’s one-year survival data, as reported on the January 2020 Scientific Registry of Transplant Recipients (SRTR) outcomes report, was better than the expectation in each of our four programs.

“Our numbers and outcomes data are a true testament to the magnitude and quality of our program,” said Dr. John Goss, medical director of Transplant Services. “I am extremely proud of and thankful for our team and their commitment to being the absolute best at what they do.”

Goss added that he also is appreciative of our donors whose generous and selfless decisions make transplants possible. “Without our donors, many of our patients would run out of options,” he said.

Transplant Services provides a comprehensive, multidisciplinary approach to care through all aspects of the transplant process, from initial referral to hospitalization and outpatient management. Our team of experts includes physicians and surgical advanced practice providers, transplant coordinators, pediatric ventricular assist device coordinators, perfusionists, child life specialists, dietitians, social workers, financial counselors, pharmacists, inpatient and outpatient nursing and support staff, Perioperative Services, physical and occupational therapists, Radiology, Pathology, our LifeGift partners, and many others.

Since its inception, Transplant Services has grown at a steady pace and is now one of the largest pediatric transplant programs in the nation with a total of more than 1,800 solid organ transplants performed. Our depth of skill and service enables us to offer world-class care for patients, from newborns to young adults, in need of heart, kidney, liver and lung transplants. That expertise has allowed us to successfully treat some cases that other national and international programs might consider untreatable.

Members of the Transplant Team are involved in academic research as well. That research is presented at many national and international conferences, and contributes to advancing the practice of pediatric transplant as a whole.

Transplant Services is accredited by the United Network for Organ Sharing (UNOS) and is fully accredited by the Centers for Medicare and Medicaid Services (CMS).

“Congratulations to everyone on our Transplant Services team,” said Texas Children’s Surgeon-in-Chief Dr. Larry Hollier. “Everyone on this team does an amazing job at providing excellent care and support for our transplant patients and families.”

Learn more about Transplant Services at Texas Children’s.

December 23, 2019

In 2019, Texas Children’s reached many milestones including the continued expansion of our services into Austin and the one-year anniversary of the Lester and Sue Smith Legacy Tower, our new home for heart, intensive care and surgery. In this Year in Review video, we celebrate Texas Children’s One Amazing Team and the system wide accomplishments we achieved together in 2019. View more

September 26, 2019

The Sickle Cell Program at Texas Children’s Hematology Center is the largest in Texas, serving more than 1,000 children a year. The clinic offers the latest treatments for the inherited red blood cell disorder and conducts some of the top research in the field.

The program’s multidisciplinary staff is made up of board-certified pediatric hematologists, hematology-trained nurse practitioners and physician assistants, research staff, as well as social worker and child life specialists. A couple of providers suffer from sickle cell themselves, giving them a unique perspective in caring for their patients.

Texas Children’s Sickle Cell Program Hematologist Dr. Titilope Fasipe and Nurse Practitioner Precious Uwaezuoke both have sickle cell disease. In honor of National Sickle Cell Disease Awareness Month, we talked to them about why they chose to dedicate their profession to caring for children with sickle cell disease and whether living with the disease has allowed them to better connect with patients and their family members.

How has sickle cell affected your life?

Uwaezuoke: My journey with sickle cell has been a tough one. I was in and out of the hospital multiple times when I was younger and there were occasions when I would get depressed. Thanks to family and staff here at Texas Children’s Sickle Cell Program, I was encouraged to stay the course. Sickle cell flares still occur, but I’m able to manage my disease by living a healthy lifestyle and hydroxyurea therapy.

Fasipe: I too suffered from bouts of depression during my early years of battling sickle cell disease. One of my lowest points was when my cousin died from the disease at just 17 years old. Childhood deaths related to sickle cell are not uncommon in Nigeria, which is where I’m from and where screenings and treatments are limited. Because of this, I try not to take life for granted and believe in treasuring all moments, big and small. Like Precious, hydroxyurea has also made a positive difference in my life.

Why did you decide to get into the medical field and help people like yourself?

Uwaezuoke: The treatment I received and the people I met at Texas Children’s inspired me to go into the medical field and help people like myself. I will never forget a nurse who told me to not let sickle cell take over my life; that I could and would be able to do more than just deal with my disease. The connection she made with me during that hard stay in the hospital is part of what made me pursue what is now my passion.

Fasipe: My cousin’s death and my parents’ encouragement motivated me to make a difference in the world and to do work that would help prevent any more deaths like hers. I chose pediatric hematology because I wanted to reach sickle cell patients and their families before they defined how their lives are going to be; and teach them that life can still be beautiful with this disease.

How has having sickle cell helped you connect with patients and families?

Uwaezuoke: A lot of people are devastated when they find out their child has sickle cell disease. Some think it’s a death sentence. The minute I tell them I have the disease and have managed to live a productive, well-balanced life, they break down in tears. They are overjoyed to meet someone older who is living with and managing their disease.

Fasipe: I think many of the families think I can read their minds! For the parents, I acknowledge the guilt and worry they have; and for the children, I acknowledge their pain and fears. After discussing the challenges, we then talk about how we can work together to best manage their disease. The bravery I have seen in our patients has inspired me to become a community sickle cell advocate. I have participated in several Houston sickle cell programs, and served on the first Sickle Cell Advisory Committee of the Texas Department of State Health Services. Precious and I also help coordinate the Texas Children’s Hematology Center’s annual Sickle Cell Education and Research Day, which was recently held at the Houston Museum of Natural Science.

How much progress is being made in finding better treatments cures, for sickle cell disease?

Uwaezuoke: I’m very optimistic. Funding for sickle cell research continues to grow as well as the number of people trying to find better ways to help people with the disease. People who have sickle cell are living longer, especially here in the United States where newborns are screened. All of these things are a step in the right direction.

Fasipe: We are learning more about this disease every day. There are currently several disease-modifying treatments in various stages of the research pipeline; some with successful clinical trials. Gene therapy trials have also shown promise to be a more universal cure than bone marrow transplant, which is only available to a few patients. I am encouraged by how far things have come since I was a child. And, I believe things will only get better.

To learn more about The Sickle Cell Program at Texas Children’s Hematology Center, click here.

September 17, 2019

As one of the best pediatric cancer centers in the nation, experts at Texas Children’s Cancer Center are on the forefront of cutting-edge basic, translational and clinical research. The center’s world-renowned faculty have pioneered many of the now standard protocols for treating and curing children with cancer.

Scientists and clinical experts at the center are conducting more than 250 clinical trials – more than the majority of pediatric cancer centers in the nation. In addition, the center’s Developmental Therapeutics Program and its Cell and Gene Therapy Program have been leaders in the study of novel agents and immunotherapies, respectively, for the treatment of childhood cancer.

“Our physicians employ evidence-based guidelines and the most advanced clinical trials in the delivery of oncology care,” said Cancer Center Director Dr. Susan Blaney. “Our vast expertise in the development of new therapeutics means our patients have access to the most advanced and innovative therapies.”

Dr. Andras Heczey, one of the Cancer Center’s physician scientists and a member of the newly-formed Sky High Immunotherapy Center, is working with colleagues in his laboratory to develop novel treatments for children with solid tumors. His work is a prime example of what the Cancer Center is doing to advance treatments and ultimately find a cure for a disease that remains the leading cause of non-accidental death in children.

In honor of National Childhood Cancer Awareness Month, we interviewed Heczey about his research and why he thinks such work is so important to enhanced patient care.

What is the focus of your research?
My research focuses on redirecting the normal immune system to attack cancer cells. T cells are a type of white blood cell that participate in the immune response. I am using genetically engineered T cells to treat liver tumors in children and Natural Killer T (NK-T) cells to treat neuroblastoma, a childhood tumor that most commonly involves the adrenal gland.

How exactly does this work?
We take patients’ white blood cells from a simple blood draw, modify them in the test tube to recognize and kill tumor cells, and then re-infuse these genetically engineered (modified) cells into the patient’s blood stream. The idea is that we train the immune system to kill the cancer cells. In patients with neuroblastoma, we modify Natural Killer T cells to express a protein called chimeric antigen receptor (CAR for short) that targets GD2, a molecule on tumor cells but not on normal tissues. When the Natural Killer T cells arrive at the neuroblastoma tissue, they recognize the GD2 molecule and kill the cancer cells. In liver cancer patients, we are using a similar strategy using T cells that express CAR targeting a molecule called Glypican-3, which is primarily expresses on liver cancer cells.

Have you used these treatments in children at Texas Children’s?
Last summer, we started our first human clinical study in children who have relapsed or have refractory high-risk neuroblastoma. So far, three children have been treated. The results we have observed thus far are very promising. We also have started our first human clinical study in children with relapsed or refractory liver cancer. The first patient was recently treated and we are eagerly waiting for the results.

What else are you studying in your laboratory?
In addition to developing strategies to kill the tumor cell, we are also closely studying the tumor environment. We know that when genetically engineered cells enter the tumor, the cancer cells and other cells within the tumor will fight back. We don’t really know how this happens. We have some ideas, but we and others in the field are closely evaluating what happens in human tumors after the engineered cells are infused. This is really important because we want to make sure our cell therapies are effective and that the responses are sustained and the patients are cured. Ultimately, we’ll also need to develop techniques to engineer the cells inside the body. Right now, we have to grow and test the cells before infusing them into the patient. This typically takes four to six weeks, depending on the product and how many cells needed to be manufactured. A patient cannot always wait that long. In the future, we envision strategies where a patient’s immune cells are engineered inside their body to quickly redirect them against the growing tumor cells.

Are you encouraged by what you are finding?
Yes, I am very encouraged. We are getting closer each and every day to finding a cure for some forms of childhood cancer.

Click here to view a preview of the next installment of “This is Cancer: Reflections from our patients.” This installment focuses on Tajah, who was diagnosed with osteosarcoma after visiting Texas Children’s Emergency Center with intense knee pain. The 13-year-old had to have her knee and femur removed. Surgeons replaced them with a metal extendable one, which will prevent her from undergoing several additional surgeries as she grows. The “This is Cancer” series documents the journeys of several families receiving care at Texas Children’s Cancer and Hematology Center. Their stories illustrate in intimate detail what they’re experiencing and how to better support them. Click here to learn more.

September 16, 2019

The Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital recently hosted the first-ever family conference for EBF3-HADD (Hypotonia, ataxia and delayed development) syndrome (HADDS). Medical residents, genetics counseling students and research scientists joined more than 20 families from across North America that attended in person and 13 families that participated via live-streaming services from countries around the world – including Ireland and Australia – making it a truly international event.

The conference was organized by the EBF3-HADDS Foundation, a nonprofit organization created in 2018 by families to promote awareness, research and support for this genetic syndrome. The foundation was co-founded by Ashley LeMaire and her husband, Mark. After one of their children was diagnosed with HADDS in 2016, the LeMaires started a Facebook group for HADDS families. In just two years, that group has grown into an international community and was the impetus behind the foundation’s creation.

“Our HADDS community is a motivated and talented group of families dedicated to supporting research, education and advocacy efforts for HADDS patients, and we support each other on this journey,” said LeMaire, who is a clinical neuropsychologist at the Menninger Clinic, assistant professor of Psychiatry & Behavioral Sciences at Baylor College of Medicine, and also board member of the EBF3-HADDS Foundation. “There is still much to learn about HADDS, but when you have such a dedicated team of physicians and researchers collaborating with families to learn about the condition and provide needed support, it fosters so much hope for our families.”

During the conference, guests attended presentations on a variety of HADDS-related topics given by NRI researchers Drs. Hsiao-Tuan Chao and Michael Wangler; pediatric urologist Dr. Irina Stanasel, a former Texas Children’s fellow; Texas Children’s genetic counselor Pilar Magoulas; and Geraldine Bliss, research director of the Phelan-McDermid Foundation. Additionally, Chao and Wangler offered clinical evaluations for patients, and attendees were also able to tour research labs and facilities at the NRI, where one of the first genetic discoveries for HADDS was made in 2016, and the first lab (Chao’s) devoted to understanding the biology of this disorder.

A search for answers

If you’ve never heard of HADDS, there’s a good reason. The rare genetic disorder, caused by a mutation in the EBF3 gene, was only discovered in 2016 by Chao and Wangler when they were training in NRI investigator Dr. Hugo Bellen’s lab, in collaboration with colleagues at the Undiagnosed Diseases Network (UDN) and New York University Langone Health.

The UDN had been stumped by a case in which a child exhibited symptoms including impaired speech and cognition, low muscle tone, balance and gait issues, reduced ability to feel physical pain, and an inability to show facial emotional expressions. Though earlier DNA sequencing had yielded a few candidate genes, there wasn’t a significant patient cohort or research to help determine which gene was responsible for the symptoms.

Researchers selected EBF3 as the most likely candidate gene. Chao then used fruit flies to mimic mutations to better understand EBF3’s role, and within a few months three patients were found who presented with similar symptoms and similar mutations in the EBF3 gene.

As result of those efforts, more than 200 patients with HADDS have been identified to date.

“The gene discovery of EBF3 illustrates the ‘Power of One’ in medicine and biomedical research, how a single patient with an undiagnosed disorder – a ‘medical mystery’ – can lead to the discovery of a gene responsible for a previously unknown disorder,” Chao said. “This becomes the starting point to develop the diagnostic tests and therapies that can transform the lives of many patients and their families.”

Since 2017 Chao and Wangler have offered monthly clinical evaluations for HADDS patients at Texas Children’s and have now seen the largest number of such patients at any single institution worldwide. They are also enrolling patients in a study to better understand the condition and to help translate research into potential clinical interventions in the future.

The conference was a testament to the power of teamwork and collaboration in research, and also shows how dedicated parents and volunteers from across the globe can work together to build a community of support.