How Texas Children’s Hospital’s Rare Tumor Program Aids Pediatric Oncologists Around the Country

For Physicians

All pediatric cancers are rare, but some are so rare that discerning a precise diagnosis from hundreds of possibilities requires particular expertise. With no standard of care, data in the literature, or clinical trials available, it is challenging to unlock the keys to such rare cancers. Enter Texas Children’s Cancer Center’s Rare Tumors Program

The program, led by Rajkumar Venkatramani, MD, was one of the first pediatric rare tumor programs in the country and remains the only one in the southwest United States. The team specializes in first providing a precise diagnosis when a patient presents with a rare tumor. 

Rajkumar Venkatramani, MD, MS, MBA

“We work closely with our genomics team to sequence the tumor to identify genetic changes that may more precisely diagnose the tumor,” Dr. Venkatramani said. “Because we’re dedicated to just rare tumors, we have the time to focus on understanding their genetic basis and identifying novel treatment targets.” 

The Texas Children’s Cancer Center program sees about 75-100 children with rare tumor diagnoses each year. Due to the large number of rare tumors that we see and our dedicated rare tumor research program, we have been able to develop standardized treatment regimens for multiple childhood rare tumors—regimens that didn’t previously exist. What’s more, patients and their oncologists need not live in Houston to benefit from the team support of our rare tumor program, which is also offered virtually. 

“Each month, we host an online Rare Tumor Board where physicians present patients with difficult-to-treat cancers for consultation with experts in the field,” Dr. Venkatramani said. “I do more than 60 consults with oncologists from around the country each year, providing suggested treatment protocols so that patients can receive state-of-the-art treatment by their local care team. Additionally, we have a repository of presentations for many rare tumor diagnoses that we make readily available to physicians from around the world.” 

Filling the need for data  

In addition to working directly with physicians, the team also collects and provides data for national tumor-specific registries. However, for many rare tumors, such dedicated registries did not exist. To fill this gap, the Texas Children’s Rare Tumors Program has established registries for many rare tumors, including clear cell sarcoma of the kidney, esthesioneuroblastoma, nasopharyngeal carcinoma, neuroendocrine tumors, salivary gland tumors, BCOR mutated sarcomas, and melanomas. 

“The goal of the registries is to learn more about the biology of rare tumors so that we can develop more successful therapies, minimize treatment side effects, and achieve better long-term outcomes,” Dr. Venkatramani said. “Any patient in the country can enroll in the registry over the phone.” 

These dedicated registries collect information on treatment approaches, and obtain and bank tumor and saliva samples for ongoing and future research. To date, the program has enrolled 250 patients in its registries and tissue bank—all of which will help in the identification of future treatment strategies. Members of our team are currently working with the National Institutes of Health/National Cancer Institute to design registries based on the Texas Children’s Hospital model. 

Bringing research findings to practice 

The program’s research has brought practical applications to patients in need. The team was the first to treat pediatric neuroendocrine tumors with Lutathera® in 2019, a drug approved for adults and currently in clinical trials for use in children. Similarly, the team achieved favorable responses treating central giant cell granuloma with denosumab, a drug that had not previously been studied in children. The program’s researchers also found a way to reduce the need for surgical procedures in children with recurrent respiratory papillomatosis (a condition in which benign tumors repeatedly grow in the respiratory tract and heretofore required surgical removal) by treating them with bevacizumab, a drug typically used for colon and other cancers in adults.  

“Not surprisingly, what we find is that children need to be treated much differently than adults,” Dr. Venkatramani said. “Although some pediatric rare cancers may be more advanced than adult tumors, children tend to tolerate treatment better than adults, resulting in better outcomes.” 

For second opinion consultations about the care and management of individual patients or to participate in an upcoming Rare Tumors Board, call 832-822-0673 - or learn more about the Rare Tumors Program online. 

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