‘Vigilant surveillance’ detects asymptomatic tumors in children predisposed to cancer
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Key takeaways:
- When analyzing across all imaging modalities, researchers noted high sensitivity and specificity.
- Imaging generated few false-positive or false-negative reports.
Standardized surveillance enabled early tumor detection for a variety of cancer predisposition syndromes, allowing for surgical resection and treatment of children and young adults with cancer, study findings in JAMA Oncology showed.
The results, when analyzed across all imaging modalities, also generated few false-positive and false-negative findings, according to researchers.
“I was very surprised by some of the findings and pleased by others,” Kim E. Nichols, MD, director of the division of cancer predisposition at St. Jude Children’s Research Hospital, told Healio.
“I was surprised because 1 in 10 patients being monitored in our study developed a new tumor that was detected through surveillance in only 3 years of surveillance — this is a very short time frame. Further, almost one-third of these new tumors were detected on the very first scans,” she said.
“I was pleased because many of these new tumors were localized and effectively treated,” Nichols added. “These data tell us that providers need to start surveillance as soon as they learn that a child has an underlying genetic predisposition, and they should not wait until that child has finished therapy for a prior cancer.”
Background, methodology
Previous research has indicated that many pediatric patients with cancer have an underlying cancer predisposition syndrome, with surveillance recommended to detect new tumors at the most curable stages.
However, limited data exist on both the effectiveness and outcomes of surveillance for children and young adults with cancer predisposition syndrome.
Blake and colleagues conducted a cohort study reviewing surveillance outcomes for 274 children and young adults (mean age, 8 years; 52.6% female) from birth to 23 years of age with a clinical and/or molecular cancer predisposition syndrome diagnosis from Jan. 1, 2009, to Sept. 31, 2021.
Researchers monitored patients using standard surveillance regimens for their respective cancer predisposition syndrome at a specialty pediatric oncology center. They excluded patients with hereditary retinoblastoma and bone marrow failure syndromes.
Surveillance outcomes evaluated the incidence, spectrum and clinical course of newly detected tumors, with surveillance modalities being classified for accuracy and assessed for common strengths and weaknesses.
Results, next steps
During the study period, researchers detected 35 asymptomatic tumors in 27 patients through surveillance (9.9% of the cohort), with five symptomatic tumors being detected in five patients (1.8% of the cohort) outside of surveillance, two of whom also had tumors detected through surveillance.
Researchers reported that first surveillance imaging detected 10 of the 35 asymptomatic tumors (28.5%). They noted more malignant solid and brain tumors identified through surveillance to be localized (20 of 24; 83.3%) than similar tumors detected before cancer predisposition syndrome diagnosis (71 of 125; 56.8%).
Of the 24 tumors detected through surveillance that received surgical resection, 17 (70.8%) had completely negative margins.
Upon analyzing across all imaging modalities, researchers found high sensitivity (96.4%), specificity (99.6%), positive predictive value (94.3%) and negative predictive value (99.6%) of surveillance, with few false-positive (6; 0.4%) or false-negative (5; 0.3%) findings.
Researchers noted several limitations, including the study not being randomized with a control arm consisting of patients who did not receive surveillance. Additionally, initial analysis relied heavily on reviewing reports and not imaging due to the high number of investigations conducted, and a 3-year data collection timespan that, if done longer, could have increased the number of tumors detected.
The findings, researchers said, indicate a need for standardized surveillance among children and young adults in to detect potential tumors earlier.
“There are several remaining questions for the field,” Nichols told Healio.
“We looked across a very heterogeneous cohort of children who had many different cancer predisposing conditions, and for some of these conditions, we only had a limited number of patients to study,” she added. “As each condition has its own unique tumor risks, it is not possible for us to comment on the effectiveness of the specific protocols designed for each condition. We will need larger cohorts to address this question.”
Nichols also highlighted that several study participants developed new tumors between their scheduled surveillance assessments.
“We still have more work to do to refine current protocols to determine the optimal timing and types of surveillance modalities to use so that we can catch all new tumors at their earliest and most curable stages,” she said.
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Blake A, et al. JAMA Oncol. 2024;doi:10.1001/jamaoncol.2024.1878.
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