ISSN 1662-4009 (online)

ey0017.3-11 | Clinical trials for thyroid disease | ESPEYB17

3.11. Thyroid hormone and folinic acid in young children with Down syndrome: the phase 3 ACTHYF trial

C Mircher , S Sacco , C Bouis , J Gallard , A Pichot , E Le Galloudec , Cieuta , I Marey , O Greiner-Mahler , N Dorison , A Gambarini , S Stora , S Durand , M Polak , A Baruchel , E Schlumberger , J Dewailly , A Azar-Kolakez , RM Gueant-Rodriguez , JL Gueant , D Borderie , D Bonnefont-Rousselot , E Blondiaux , A Ravel , FG Sturtz

To read the full abstract: Genet Med. 2020;22:44–52.This single center, randomized, double-blind, placebo-controlled phase 3 study investigated the effects of levothyroxine, folic acid, or both in combination over 12 months on global development in 143 infants with Down syndrome (DS). Over the last decades, treatment of DS associated co-morbidities has improved their life-expectancy. However, a therapy to improve mental development is still...

ey0020.7-5 | Gonadal Function and Fertility Issues in Childhood Cancer Survivors | ESPEYB20

7.5. Ovarian function and spontaneous pregnancy after hematopoietic stem cell transplantation for leukemia before puberty: An L.E.A. cohort study

M Chabut , P Schneider , B Courbiere , P Saultier , Y Bertrand , MD Tabone , C Pochon , S Ducassou , C Paillard , V Gandemer , J Kanold , JH Dalle , M Poiree , G Plat , S Thouvenin , D Plantaz , N Sirvent , S Weinhard , J Berbis , A Baruchel , G Leverger , Z Hamidou , P Auquier , G Michel

Brief summary: This French retrospective observational study evaluated ovarian function, premature ovarian insufficiency (POI) and spontaneous pregnancy in 178 women who had undergone hematopoietic stem cell transplantation (HSCT) for leukemia before puberty; 116/178 had received total body irradiation (TBI) and 62 had received a busulfan-based conditioning regimen.Sixty percent of women needed pubertal induction; only 40% had spontaneous menarche and ha...