ESPEYB17 3. Thyroid Clinical trials for thyroid disease (3 abstracts)
3.11. Thyroid hormone and folinic acid in young children with Down syndrome: the phase 3 ACTHYF trial
Mircher C , Sacco S , Bouis C , Gallard J , Pichot A , Le Galloudec E , Cieuta , Marey I , Greiner-Mahler O , Dorison N , Gambarini A , Stora S , Durand S , Polak M , Baruchel A , Schlumberger E , Dewailly J , Azar-Kolakez A , Guéant-Rodriguez RM , Guéant JL , Borderie D , Bonnefont-Rousselot D , Blondiaux E , Ravel A & Sturtz FG
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 lacking. Folic acid as well as thyroxine are both crucial for normal neurodevelopment in utero and postnatally. Previously, two randomized controlled studies suggested beneficial effects of either folic acid especially in children treated with levothyroxine in an age group of 3–30 months [1] and levothyroxine alone started in the neonatal period [2], while a short-term cross-over study did not observe effects of levothyroxine on cognitive function in DS patients [3].
This new, important, well designed clinical trial with four highly homogenous patient groups showed no effect on any of the analyzed subclasses (locomotor, social skills, hearing and language, hand-eye coordination, general performance) of global development – neither of folic acid or levothyroxine alone nor in combination. In summary, neither folic acid nor levothyroxine if TSH is below 7 mU/l is recommended to improve neurologic development in DS infants, although these therapies are widely used since years without evidence of effect. Non-pharmacological approaches might open new avenues for research. One suggested by the authors could be prevention of sleep apnea.
Reference:
1. Blehaut H, Mircher C, Ravel A, Conte M, de Portzamparc V, Poret G, de Kermadec FH, Rethore MO, Sturtz FG. Effect of leucovorin (folinic acid) on the developmental quotient of children with Down’s syndrome (trisomy 21) and influence of thyroid status. PLoS One. 2010;5:e8394.
2. van Trotsenburg AS, Vulsma T, van Rozenburg-Marres SL, van Baar AL, Ridder JC, Heymans HS, Tijssen JG, de Vijlder JJ. The effect of thyroxine treatment started in the neonatal period on development and growth of two-year-old Down syndrome children: a randomized clinical trial. J Clin Endocrinol Metab. 2005;90:3304–3311.
3. Tirosh E, Taub Y, Scher A, Jaffe M, Hochberg Z. Short-term efficacy of thyroid hormone supplementation for patients with Down syndrome and low-borderline thyroid function. Am J Ment Retard. 1989;93:652–656.
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ESPE Yearbook of Paediatric Endocrinology
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