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New protocol may improve rare pediatric brain cancer survival
New standard treatment developed for choroid plexus tumors.
41st Annual Meeting of the International Society of Pediatric Oncology
In young patients with choroid plexus tumors — rare brain tumors — a new standard treatment protocol may improve survival by nearly twofold, according to findings from a recent study presented at the 41st Annual Meeting of the International Society of Pediatric Oncology held in Sao Paulo, Brazil.
Johannes Wolff, MD, professor in the Children’s Cancer Hospital and professor of biomathematics at The University of Texas M.D. Anderson Cancer Center, presented the findings.
Typically, most physicians see patients with choroid plexus tumors only about once a year. Choroid plexus tumors are rare and affect approximately 1,500 children each year worldwide and are seen more often in infants. Physicians often do not have relevant literature or they do not know enough about the disease to treat these patients, according to Wolff.
At the meeting, Wolff revealed that the protocol, which consists of three chemotherapy agents, carboplatinum, etoposide and cyclophosamide, in combination with radiation, had projected survival rates of 93% at one year, 82% at five years and 78% at eight years.
“This SIOP 2000 study started 10 years ago and has grown to include more than 100 institutions from more than 20 countries,” Wolff said in a press release. “With the data we have, we can tell which patients are prone to do better and which ones have a poor prognosis,” he said.
Standard protocol established for rare disease
For the study, researchers used information from an international registry, an algorithm from a standard clinical recommendation and clinical research comparing treatment protocols. “A combination of those three elements together in one single project is uncommon, but necessary because the disease is rare and there needs to be a comprehensive approach for treatment,” Wolff told HemOnc Today.
The new protocol takes into account the different tumor malignancy grades, including those assigned by WHO, and assists physicians in determining a plan of action for a patient, whether it is close follow-up, immediate treatment or surgery.
One of the researchers' findings contradicted historical research. Historically, data indicated there was a significant advantage to complete surgical resection; however, findings from the SIOP 2000 study showed patients who received an intense chemotherapy protocol had similar outcomes to those who received surgical treatment.
“We think the better outcomes had to do with the fact that physicians will prolong chemotherapy treatment if there is residual tumor,” Wolff said in a press release. “If we can prove this hypothesis, this would be an argument for extending treatment in the future.”
The next step is to begin a study that will investigate the four-armed chemotherapy protocol and explore the possibility of adding another chemotherapy agent to further improve survival rates, according to Wolff.
“After 10 years of running this protocol, an update was necessary; we put together the data and our knowledge, and made decisions about changes for all patients [with choroid plexus tumors],” Wolff said. “In Sao Paulo, Brazil, at the SIOP Meeting last week, we made the decision that we have finally planned enough for the next protocol and it is time to open it.”
