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Growth Hormone Treatment and Papilledema: A Prospective Pilot Study
Nieves Martín-Begué1, Eduard Mogas2, Charlotte Wolley Dod1
1Hospital Universitari Vall d’Hebron, Department of Paediatric Ophthalmology, Barcelona, Spain
Insights
Growth hormone (GH) treatment can cause pseudotumor cerebri syndrome (PTCS) in children. Children with GH deficiency and anatomical or genetic issues are at higher risk and require regular eye exams.
Area of Science:
- Pediatric Endocrinology
- Neuro-ophthalmology
Background:
- Growth hormone (GH) therapy is widely used in pediatric patients.
- Pseudotumor cerebri syndrome (PTCS) is a rare but serious complication that can occur in children.
Purpose of the Study:
- To determine the incidence of PTCS in children receiving GH treatment.
- To identify risk factors associated with PTCS development in this population.
Main Methods:
- A prospective pilot study was conducted on pediatric patients treated with recombinant human GH.
- Fundus examinations were performed before treatment initiation and at 3-4 months follow-up.
Main Results:
- Out of 289 patients, five (1.7%) developed papilledema, a sign of PTCS.
- All affected patients were asymptomatic and had underlying conditions like GH deficiency due to craniopharyngioma or genetic disorders.
Conclusions:
- GH treatment is a potential cause of PTCS in children.
- Children with GH deficiency, hypothalamic-pituitary anomalies, or genetic/chromosomal diseases are at increased risk.
- Systematic fundus screening is crucial for at-risk pediatric patients on GH therapy, regardless of symptoms.
Objective:
To investigate the incidence of pseudotumor cerebri syndrome (PTCS) in children treated with growth hormone (GH) in a paediatric hospital and to identify risk factors for this complication.
Methods:
Prospective pilot study of paediatric patients treated with recombinant human GH, prescribed by the Paediatric Endocrinology Department, between February 2013 and September 2017. In all these patients, a fundus examination was performed before starting treatment and 3-4 months later.
Results:
Two hundred and eighty-nine patients were included, of whom 244 (84.4%) had GH deficiency, 36 (12.5%) had short stature associated with small for gestational age, six (2.1%) had a mutation in the SHOX gene and three (1.0%) had Prader-Willi syndrome. Five (1.7%) developed papilledema, all were asymptomatic and had GH deficiency due to craniopharyngioma (n=1), polymalformative syndrome associated with hypothalamic-pituitary axis anomalies (n=2), a non-specified genetic disease with hippocampal inversion (n=1) and one with normal magnetic resonance imaging who had developed a primary PTCS years before.
Conclusion:
GH treatment is a cause of PTCS. In our series, at risk patients had GH deficiency and hypothalamic-pituitary anatomic anomalies or genetic or chromosomal diseases. Fundus examination should be systematically screened in all patients in this at-risk group, irrespective of the presence or not of symptoms.
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