J Clin Res Pediatr Endocrinol. 2021; 13(2): 146-151 | DOI: 10.4274/jcrpe.galenos.2020.2020.0007

Growth Hormone Treatment and Papilledema: A Prospective Pilot Study

Nieves Martín-Begué¹, Eduard Mogas², Charlotte Wolley Dod¹, Silvia Alarcón¹, María Clemente³, Ana Fábregas², Diego Yeste^3
1Hospital Universitari Vall d’Hebron, Department of Paediatric Ophthalmology, Barcelona, Spain
²Hospital Universitari Vall d’Hebron, Department of Paediatric Endocrinology, Barcelona, Spain
³Hospital Universitari Vall d’Hebron, Department of Paediatric Endocrinology, Barcelona, Spain & Universitat Autònoma de Barcelona (UAB), Barcelona, Spain & Centro de Investigación Biomédica en Red: Enfermedades Raras (CIBERER), Madrid, Spain

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.

Keywords: Growth hormone treatment, pseudotumor cerebri syndrome, idiopathic intracranial hypertension, papilledema, risk factors

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