Abstract

Purpose

We aim to describe the characteristics of patients with childhood‐onset craniopharyngioma and to analyze factors that impair quality of life (QoL) in this population.

Methods

Multicenter national study including patients treated between 2008 and 2022, from 2 to 25 years of age diagnosed with craniopharyngioma. QoL was assessed once during patient’s follow‐up by age‐adapted versions of Pediatric Quality of Life Inventory (PedsQL) questionnaire.

Results

Sixty‐six patients were included. Median age at diagnosis was 5 years (interquartile range [IQR]: 3–8), while median follow‐up was 7.4 years (IQR: 2.8–9.7). Most craniopharyngioma were suprasellar (93.9%), and 59.7% had hypothalamic involvement (HI). All patients underwent surgery, 44.4% received radiotherapy, and 23.6% intracystic therapy. Most frequent long‐term complications were visual deficit (72.7%) and endocrine impairment (94.5%). Patients exhibited hypothyroidism requiring hormone replacement (92.4%), hypocortisolism (80.3%), diabetes insipidus (86.4%), and/or growth hormone therapy (50%). When parents evaluated QoL, PedsQL median score was 53.8 points out of 100 (IQR: 41–71.6). Higher scores were noted when patients assessed their own QoL (median score 64.8 [IQR: 57.3–81.8]), observing statistically significant differences (p = .019). QoL was impaired by repeated surgeries (r = −.44; p = .014), HI (median score 51.5 [IQR: 39–63.8] vs. 76.4 [59–84.8]; p = .001), radiotherapy (median score 51.9 [IQR: 38.1–61.3] vs. 63.8 [IQR: 49–82.5]; p = .02) and longer follow‐up (r = −.3; p = .01).

Conclusion

In our study, most patients had significant comorbidities and low overall QoL scores, which was mainly affected by repeated surgery, radiation, and hypothalamic involvement. This reflects the need for further research and intensified studies of systemic therapy/alternate strategies to broaden the standard‐of‐care options, so that treatment‐related sequalae can be avoided.