A Survey of Patient-Relevant Outcomes in Pediatric Craniopharyngioma: Focus on Hypothalamic Obesity

Abstract

ContextIndividuals treated for pediatric craniopharyngioma, a rare, grade 1 brain tumor, frequently develop hypothalamic obesity, a complication often recalcitrant to intervention. Although hypothalamic obesity is known to adversely impact quality of life, less is known about how caregivers and patients experience this condition.ObjectiveOur goal was to examine the approaches that families take towards weight management and the impact on social function in individuals with craniopharyngioma and obesity. Individuals with craniopharyngioma without obesity were included as a comparison.Subjects and MethodsAdult caregivers of children <18y with craniopharyngioma completed a web-based survey posted by a patient advocacy organization between February and July 2020. Questions related to the child’s diagnosis, medications, lifestyle modifications, and social function along with research priorities. Descriptive statistics were generated. Linear regression was used to assess the independent effects of obesity and other covariates on social function.ResultsOf 106 respondents, 60 (57%) reported their child had obesity at the time of survey completion. In contrast, only 6 (5.7%) had obesity prior to craniopharyngioma diagnosis. A majority (92%) of those with obesity had tried limiting calories or carbohydrates; 31% and 69% found these helpful, respectively. Thirty-eight percent had tried weight loss medications (stimulants, metformin, GLP1R-agonists, and topiramate) and 48% found at least one helpful. Both stimulant and anti-depressant use were reported more frequently with obesity. An index (T-score) reflecting social function was lower in the cohort than a population reference, 41 (SD 11) vs. 50 (SD 10), p<0.001. In a linear model, both older age and obesity were independently associated with greater social impairment. Ninety-four percent of respondents caring for a child with obesity (and 79% of all respondents) identified “improving treatments and prevention for hypothalamic obesity” as a key research priority.ConclusionsOnly a minority of individuals with hypothalamic obesity had trialed medication, even though many reported that lifestyle modification was inadequate. Furthermore, social function was significantly impaired overall in survivors compared to a reference cohort, and even more so in individuals with obesity. These findings highlight the opportunity to improve social functioning as an additional potential benefit of improved treatments for hypothalamic obesity.

A Pilot Randomized Clinical Trial of Intranasal Oxytocin to Promote Weight Loss in Individuals With Hypothalamic Obesity

“…Another set of outcomes that may be sensitive to OXT are related to socioemotional and cognitive function. Individuals and families frequently report concerns related to these domains [ 64 ], and in this study, a substantial number of participants and caregivers reported impairment in family function, similar to a previous report in other survivors of childhood brain tumors [ 42 ]. Although not directly related to metabolism per se, interventions that mitigate anxiety or increase social engagement could promote the success of weight loss efforts in hypothalamic obesity, for example, by decreasing barriers to participating in group-based physical activity.…”

Retrospective dataset and survey analyses identify gaps in data collection for craniopharyngioma and priorities of patients and families affected by the disease

“…Our work demonstrates likely under-reporting of both frequency and treatment of functional and QoL outcomes. For example, only 5% of eligible patients in our cohort reported treatment for thyroid deficiency, 47% of patients underwent treatment for growth hormone deficiency and 61% of patients receiving treatment for hypogonadism [25] . Sleep disturbances, too, were self-reported in a significant proportion of patients but only 35% reported treatment for a respective sleep disorder.…”

“…Separate work has shown the significant neuroendocrine complications faced by patients: hypothyroidism, growth hormone deficiency, hypogonadism, diabetes insipidus, adrenal insufficiency, as well as obesity and obesity-related health problems [25] . The concomitant increase in conservative approaches at recurrence could possibly arise from increased concerns for impact on QoL [ 26 , 27 ].…”

Caregiver Burden and Its Relationship to Health-Related Quality of Life in Craniopharyngioma Survivors

“…Hypothalamic dysfunction is associated with an abysmal prognosis ( 16 ) and is the strongest predictor of lower QOL in CP survivors ( 7 , 17 ). Hypothalamic dysfunction results in impaired regulation of temperature, thirst, hunger, sleep/wake cycles, social interactions, mood ( 18 , 19 ), metabolism, and energy ( 20 , 21 ) and causes hypothalamic obesity (HO) in about half of all CP survivors ( 22 , 23 ). No effective treatments are available to address HO or other symptoms of hypothalamic dysfunction ( 24 ).…”