Background: The management of MEN1 in CYP<19 years is challenging due to its rarity, and diverse presentations of its component tumours to several adult and paediatric medical and surgical specialists. There is little high quality evidence for treatment recommendations.
Aim: To ensure age- and tumour-specific paediatric and adult teams are involved in co-ordinated discussions to improve high quality care and hence survival and reduce long term morbidity; to improve and expedite diagnosis (including complex screening of familial cases), acute decision making and peri-operative care.
Methods: Having recognised these challenges, a multi-disciplinary guideline development group (GDG) was convened in conjunction with the Childrens Cancer and Leukaemia Group (CCLG), the British Society for Paediatric Endocrinology and Diabetes (BSPED) and the Royal College of Paediatrics and Child Health (RCPCH). Clinical questions were formulated based on a PICO (Population, Intervention, Comparison, Outcome) format by the multidisciplinary GDG to guide systematic searches via MEDLINE and EMBASE databases using OVID. The guideline objectives and clinical questions were reviewed by previously identified stakeholders (including patients with MEN1, and support groups) to ensure no relevant areas had been omitted. The systematic literature search identified 327 articles reviewed using the GRADE approach, of which 245 were excluded and 82 incorporated into the evidence-base for recommendations. Where recommendations could not be made, a two-stage international Delphi consensus process was conducted.
Results: Eighteen clinical questions were identified, producing 13 recommendations largely based on low quality evidence. Twenty-four further recommendations achieved >70% agreement via the Delphi consensus process. Important recommendations for the care of CYP with MEN1 include:
1. This is provided in an age appropriate tertiary setting (linked to a paediatric or Teenage Young Adult (TYA) CCLG oncology centre) by a designated endocrinologist with MEN1 expertise.
2. Investigation and treatment (including decisions about the timing and extent of surgical intervention) occurs at an appropriately constituted organ specific MDT that includes adult MEN1/NET MDT members.
3. Surgery in patients under 16 years of age is performed by the Paediatric tertiary Centres designated surgical team with a nominated surgeon from the adult organ specific MDT.
4. All are entered into a lifelong national outcomes registry to inform and improve future management.
Conclusions: These CCLG, BSPED and RCPCH-endorsed guidelines developed according to AGREE II principals provide the first evidence- and consensus-based national recommendations for the management of CYP with MEN 1 aimed at achieving better consistency in quality of care and improving long-term quality of survival.
27 - 29 Sep 2018
European Society for Paediatric Endocrinology