ESPE Abstracts (2015) 84 FC11.6

Long-term Outcome of Patients Treated for Paediatric Cushing's Disease

Galina Yordanovaa, Martin Leeb, Farhad Afsharc, Ian Sabinc, Ghasan Alusid, Nicholas Plowmane, Jane Evansonf, Mattew Matsonf, Ashley Grossmang,h, Scott Akkerg, John Monsong, Wiliam Drakeg, Martin Savageh & Helen Storrh

aMU of Varna, UMHAT St Marina, Varna, Bulgaria; bThe Royal London Hospital, London, UK; cDepartment of Neurosurgery, St Bartholomew’s Hospital, London, UK; dDepartment of Otolaryngology, St Bartholomew’s Hospital, London, UK; eDepartment of Radiotherapy, St Bartholomews Hospital, London, UK; fDepartment of Radiology, St Bartholomew’s Hospital, London, UK; gDepartment of Endocrinology, St Bartholomew’s Hospital, London, UK; hCentre for Endocrinology, Wiliam Harvey Research Institute, Barts and The London School of Medicine and Dentistry, Queen Mary University of London, London, UK

Background: Due to the rarity of Paediatric Cushing’s disease (CD) there is limited data on the long-term consequences of treatment.

Objective and hypotheses: We assessed recurrence, anterior pituitary function and psychiatric disorders in a group of paediatric CD patients treated in a single centre.

Method: Retrospective review of 20 patients with CD, mean age 11.75 years (5.74–17.8), managed in our centre between 1986 and 2010. Mean follow-up from the first surgical treatment was 10.5 years (5.01–27.2). ‘Cure’ was defined as undetectable postoperative 09:00 cortisol level (<50 nmol/l) and ‘cure’ after radiotherapy (RT) by mean serum cortisol on 5-point day curve of <150 nmol/l and midnight sleeping cortisol <50 nmol/l.

Results: 14 patients were ‘cured’ by transsphenoidal surgery (TSS) and five were ‘cured’ following TSS+radiotherapy (RT). One patient underwent bilateral adrenalectomy (BA) following failed TSS. CD recurrence was seen in 3 (15%) patients: two after TSS (2 years after TSS) and one following TSS+RT (2 years post RT). The former two patients were treated with TSS+RT successfully. The latter was treated by BA. One BA patient developed Nelson’s syndrome requiring RT 0.5 years post surgery. GHD was documented in 11 patients (55%) (seven following TSS and four after TSS+RT) and three (15%) had long-term GHD. 15 patients were treated with hGH, nine have reached final height (FH) on treatment. Gonadotropin deficiency causing delayed or slow pubertal development was diagnosed in six patients (30%), only one needed treatment post-pubertally. There were no reproductive problems. two (10%) patients had TSH deficiency. Two (10%) had psychiatric problems and two (10%) have poor memory and concentration after treatment.

Conclusion: Pituitary deficiencies occurred in 60% patients after treatment for CD but long term deficiencies were less common (25% patients). Relapse occurred in 15% of patients after apparent ‘cure’ of CD and emphasises the importance of continued surveillance.