ISSN 1662-4009 (online)

Previous issue | Volume 21 | ESPEYB21

Yearbook of Paediatric Endocrinology 2024

15. Editors’ Choice

New Treatments

ey0021.15-1 | New Treatments | ESPEYB21

15.1. Semaglutide in early type 1 diabetes

P Dandona , A Chaudhuri , H. Ghanim

In Brief: This research letter describes a recent case series of 10 patients aged 21–39 years who started taking Semaglutide within 3 months of their diagnosis of antibody-positive type 1 diabetes (T1D), in addition to standard basal and prandial insulin. All 10 patients stopped prandial insulin within 3 months of Semaglutide, and 7 patients also stopped basal insulin by 6 months. Mean HbA1c fell from 11.7% at diagnosis to 5.9% at 6 months and 5.7% at 12 months. Mean fast...

ey0021.15-2 | New Treatments | ESPEYB21

15.2. A phase 3, randomized, controlled trial of resmetirom in NASH with liver fibrosis

SA Harrison , P Bedossa , CD Guy , JM Schattenberg , R Loomba , R Taub , D Labriola , SE Moussa , GW Neff , ME Rinella , QM Anstee , MF Abdelmalek , Z Younossi , SJ Baum , S Francque , MR Charlton , PN Newsome , N Lanthier , I Schiefke , A Mangia , JM Pericas , R Patil , AJ Sanyal , M Noureddin , MB Bansal , N Alkhouri , L Castera , M Rudraraju , V Ratziu , the MAESTRO-NASH Investigators.

In Brief This phase 3 trial including 966 adults with biopsy-confirmed non-alcoholic steatohepatitis (NASH) with fibrosis were randomly assigned (1:1:1 ratio) to once-daily resmetirom 80 mg or 100 mg or placebo. Resmetirom, both 80 and 100 mg daily, were superior to placebo with respect to NASH resolution and improvement in liver fibrosis.NASH resolution occurred more frequently in patients on 80-mg (25.9%) and 100-mg resmetirom (29.9%) than on placebo (...

ey0021.15-3 | New Treatments | ESPEYB21

15.3. Triple-hormone-receptor agonist retatrutide for obesity - a phase 2 trial

AM Jastreboff , LM Kaplan , JP Frias , Q Wu , Y Du , S Gurbuz , T Coskun , A Haupt , Z Milicevic , ML Hartman , for the Retatrutide Phase 2 Obesity Trial Investigators.

In Brief: This phase 2 trial randomized 338 adults with overweight or obesity to once-weekly subcutaneous Retatrutide (LY3437943), a ‘triple agonist’ for glucose-dependent insulinotropic polypeptide (GIP), glucagon-like peptide 1 (GLP1), and glucagon receptors, or placebo. Mean % change in body weight at 24 weeks was −7.2%, −12.9%, −17.3% and −17.5% in the 1-mg, 4-mg, 8-mg and 12-mg Retatrutide groups, compared with −1.6% in the placebo ...

ey0021.15-4 | New Treatments | ESPEYB21

15.4. Anti-insulin receptor antibody for malignant insulinoma and refractory hypoglycemia

S Osataphan , M Vamvini , ED Rosen , L Pei , N Erlikh , G Singh , P Dhorajiya , JA Parker , JM Dreyfuss , A Rattani , ME. Patti

In Brief: This case report describes the successful use of RZ358, a novel human anti–insulin receptor monoclonal antibody therapy, in a patient with severe, refractory hypoglycaemia due to metastatic insulinoma.The tumour and resulting severe hypoglycaemia had been resistant to treatments with octreotide, lutetium Lu 177 dotatate radionuclide, diazoxide, everolimus, dexamethasone, pasireotide, and glucagon – and the patient, a 55-year old man w...