Peer-reviewed article
RARE-17. Multi-institutional craniopharyngioma cohort highlights need for more comprehensive data collection on comorbidities and quality of life
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Summary
Written by AI, not yet AICS's view · claude-haiku-4-5-20251001This is a medical research paper about craniopharyngioma, a rare brain tumor in children. It reports on treatment outcomes and survival rates from a multi-institutional database, finding that combined surgery and radiation therapy led to higher recurrence rates than surgery alone. The authors note that data on quality of life and long-term complications were poorly recorded across institutions.
Key points
- 52% of the 75 children with confirmed craniopharyngioma experienced at least one progression or recurrence
- Children who received both surgery and radiation had significantly higher recurrence rates (84%) compared to surgery alone (32%)
- Time to recurrence was shorter for children who had partial rather than near-total tumor resection
- Comorbidity data were severely underreported, with only 27-32% of institutions documenting visual, neuroendocrine, or neurologic complications
- The authors recommend that future studies systematically collect quality of life outcomes alongside treatment and survival data
Limitations
This is a clinical outcomes study focused on tumor recurrence and treatment patterns, not child well-being or social outcomes. It does not evaluate an intervention to improve children's quality of life or well-being, only documents existing treatment practices and their consequences.
Abstract
Abstract BACKGROUND: Pediatric craniopharyngioma is associated with long-term survival, but tumor- and therapy-related complications often negatively impact quality of life (QoL). Standard treatments include resection and radiation, but institutional practices vary and recurrence rates remain high. In this review, we utilized a cohort from the Children’s Brain Tumor Network (CBTN) to evaluate outcomes for craniopharyngioma. METHODS: CBTN provides clinical and genomic data for pediatric patients diagnosed with primary central nervous system tumors across 25+ institutions. We collected data for 124 patients, ages 0-21, diagnosed with craniopharyngioma between 2012-2020. Variables collected included treatment, recurrence/progression, and comorbidities. RESULTS: Excluding patients without confirmed pathologic diagnosis (n=10) or follow-up data (n=39), 75 patients remained. For initial treatment, most (n=46, 61%) received surgery alone (9 partial, 33 near-total resection). Twenty-six (35%) underwent both surgery and radiation, with 9 receiving both therapies upfront and 17 receiving radiation at progression/recurrence. Four (5%) patients received chemotherapy. Over half of the cohort (n=39, 52%) had at least one progression/recurrence, and four died (5%). Significantly higher rates of progression/recurrence (84% vs. 32%, p=4.0e-5) were identified in patients that had surgery and radiation, compared to surgery alone. Time to recurrence, progression, or death was shorter for the surgery and radiation group (HR=4.1, p<1.0e-4), and for those that underwent partial versus near-total resection (HR=2.7, p=0.1.2e-2). Comorbidities were likely underreported, based on low rates of visual (32%), neuroendocrine (27%), and neurologic (28%) deficits at diagnosis, and 29 patients (39%) with unspecified medical history. CONCLUSIONS: CBTN provides a robust repository of information on treatment and survival of craniopharyngioma patients. However, we found a paucity of data on associated comorbidities and QoL outcomes. We advocate that future datasets and clinical trials routinely collect functional outcomes alongside therapy and survival data, particularly in craniopharyngioma where long-term survival is balanced with future QoL.