No abstract

Multiple endocrine neoplasia type 2 (MEN2) refers to the autosomal-dominant neuroendocrine tumour syndromes, MEN type 2A (MEN2A) and MEN type 2B (MEN2B). They are typified by the development of medullary thyroid cancer (MTC), phaeochromocytoma and parathyroid hyperplasia in MEN2A and MTC, phaeochromocytomas, ganglioneuromatosis and skeletal abnormalities in MEN2B. The aggressiveness of MTC is variable according to genotype, and although it is still the major cause of mortality in both conditions, prognosis has improved dramatically in those diagnosed and treated at a young age thanks to predictive genetic testing. Nevertheless, metastatic MTC, ganglioneuromatosis and a variety of other negative clinical and psychosocial impacts on quality of life and/or prognosis in MEN2 persist. In the absence, at the time of writing, of any large-scale research into quality of life specifically in MEN2, this review includes data from patient surveys and anonymised patient anecdotes from the records of the Association for Multiple Endocrine Neoplasia Disorders (AMEND), for whom the authors work. We recommend that these patients are cared for only in centres of expertise able to provide expert diagnosis, treatment and continuity of care, including psychological and transition support. Only in this way can the clinical advances of the last two and half decades be built upon further to ensure that the care of these complex, lifelong patients can be considered truly holistic.

Context Besides medullary thyroid carcinoma and other endocrinopathies, people with Multiple Endocrine Neoplasia Type 2 (MEN2) are at risk of gastrointestinal (GI) symptoms. Objective To investigate the impact of GI symptoms on the daily lives of patients with MEN2. Design An online survey was conducted among patients with MEN2 via the Association for Multiple Endocrine Neoplasia Disorders (AMEND). Methods The survey incorporated two validated questionnaires for the assessment of GI symptoms (SAGIS, PAC‐QoL). Participants There were 91 respondents, MEN2A (n = 57), MEN2B (n = 34). Results People in the MEN2A group reported a high level of GI symptoms, the most prevalent being abdominal pain 85% (n = 49), diarrhoea 85% (n = 49) and constipation 75% (n = 43) with one patient having a SAGIS score > 10/12 in the constipation domain. People in the MEN2B group reported constipation in 79% (n = 27) with one quarter of these scoring > 10/12 in the constipation domain. Other GI symptoms included diarrhoea 62% (n = 21), excessive gas and flatulence (79%), epigastric pain (59%) abdominal cramps (76%) and dysphagia (41%). The effect of constipation on quality of life was severe in all MEN2 patients as measured by PAC‐QOL and all patients reported dissatisfaction of with their current treatment for constipation. There was a trend towards higher severity of GI symptoms in MEN2B. Conclusions We report unmet needs of patients with MEN2 syndromes. The GI symptoms, especially constipation, had a severe impact on quality of life in people with MEN2. This suggests that there is room for improvement in the quality of care offered for these patients.

Aim: To examine the impact of different types of insulin pumps (CSII) on HbA1c lowering control in a large UK type 1 diabetes practice. Methods: Observational retrospective study of electronic database. We identified 597 adults (≥18 years) in our service who started on CSII between 2002 and 2017 with HbA1c data available at baseline and at 6 +/or 12 months after starting CSII. We classified by the starting pump model/manufacturer into traditional “tethered pumps” (Medtronic n=369; Roche n=79; Animas n=60) and “small pumps” (Omnipod n=79 and Cellnovo n=6). We examined change in HbA1c data (shown here in mmol/mol except where otherwise indicated) over the first year of CSII treatment. Results: In general, CSII improved HbA1c during the first 12 months (72±0.7 to 64±0.6 and 65±0.6 mmol/mol at baseline, 6 and 12 months). Despite similar starting HbA1c, those using tethered pumps achieved a significantly lower HbA1c compared with small pump users after 6 and 12 months CSII therapy. Conclusions: In real world data from a large pump service, we found significant differences in glycemic outcomes between different pumps. Those starting on tethered pumps had greater HbA1c lowering over first 12 months.HbA1c Outcomes for Tethered vs small PumpsPump typeInitial HbA1c range mol/mol (%)Baseline HbA1c6 months HbA1cp vs baseline12 months HbA1cp vs baselineTethered (n=509)All starting values71±0.763±0.6*<0.00165±0.7*<0.001Small (n=85)71±0.970±1.6NS69±1.6NSTethered (n=273)≥ 69 (85%)83±0.769±0.8**<0.00171±0.9*<0.001Small (n=43)83±2.377±2<0.0577±2.1<0.05Tethered (n=132)59-68 (7.5-8.4%)64±0.259±0.7**<0.00159±0.8*<0.001Small (n=28)64±0.566±1.4NS64±1.4NSData shown as mean±SEM. * p<0.05, ** p<0.001 for tethered vs small Disclosure S.D. De Soysa: None. J. Grey: None. K.H. Davenport: Advisory Panel; Self; Novo Nordisk Inc. E.M. Gurnell: Advisory Panel; Spouse/Partner; AstraZeneca, Pfizer Inc. S. Hartnell: Speaker's Bureau; Self; Medtronic, Roche Pharma. M. Evans: Advisory Panel; Self; Novo Nordisk A/S, Eli Lilly and Company, Cellnovo, Roche Pharma. Speaker's Bureau; Self; Abbott, Novo Nordisk A/S.

No abstract