mecasermin
Sources réglementaires consultées
Indications approuvées
- Traitement prolongé du retard de croissance chez les enfants et adolescents de 2 à 18 ans ayant un déficit primaire sévère confirmé en IGF-1.
Contre-indications
Absolues
- Tumeur active ou suspectée, ou affection/antécédent augmentant le risque tumoral ; hypersensibilité ; ne pas utiliser chez le nouveau-né ou le prématuré en raison de l’alcool benzylique.
Mises en garde cliniques
- Mise en garde majeure · Risque d’hypoglycémie similaire à celui de l’insuline. Administrer autour d’une prise alimentaire, surveiller la glycémie au début et éviter les activités à risque pendant 2 à 3 heures ; disposer de glucagon en cas d’antécédent d’hypoglycémie sévère. — CIMA/AEMPS, ficha técnica 07402001
- Mise en garde majeure · Il existe un risque de tumeurs bénignes et malignes ; ne pas utiliser hors indication ni dépasser la dose. Arrêter définitivement si une tumeur apparaît. — CIMA/AEMPS, ficha técnica 07402001
- Mise en garde majeure · Réaliser un fond d’œil avant le traitement puis périodiquement en raison du risque d’hypertension intracrânienne. Surveiller l’hypertrophie amygdalienne/adénoïdienne, les ronflements, l’apnée et les otites dus à la croissance du tissu lymphoïde. — CIMA/AEMPS, ficha técnica 07402001
- Mise en garde majeure · Évaluer toute boiterie ou douleur de hanche/genou à la recherche d’une épiphysiolyse de la tête fémorale ou d’une ostéonécrose, et surveiller la progression d’une scoliose pendant la croissance rapide. — CIMA/AEMPS, ficha técnica 07402001
Interactions médicamenteuses
- SévèreInsuline ou autres médicaments hypoglycémiants
Mécanisme: Les effets hypoglycémiants peuvent s’additionner.
Recommandation: Une réduction du traitement hypoglycémiant peut être nécessaire ; surveiller étroitement la glycémie.
CIMA/AEMPS, ficha técnica 07402001https://cima.aemps.es/cima/dochtml/ft/07402001/FT_07402001.html
Effets indésirables
Communs (≥1%)
Hypoglycémie · Céphalées · Vomissements · Hypertrophie au site d’injection · Otite moyenne
Rares mais graves
Tumeur bénigne ou maligne · Hypertension intracrânienne · Anaphylaxie ou angio-œdème · Convulsion hypoglycémique
Grossesse et allaitement
Exiger un test de grossesse négatif et une contraception efficace. Ne doit pas être utilisé pendant la grossesse et l’allaitement n’est pas recommandé.
Bibliographie récente (PubMed)
Mecasermin, a recombinant analogue of insulin‑like growth factor 1 (IGF‑1), is under investigation as a potential therapy for Rett syndrome (RTT), a neurodevelopmental disorder resulting from mutations in the MECP2 gene. In this systematic review, we assessed the impact of mecasermin on the full spectrum of RTT severity by screening relevant clinical studies identified through MeSH‑based database queries. Evidence indicates that IGF‑1 administration may help preserve social engagement and cognitive function in RTT, although autonomic control and behavioral outcomes have been inconsistent, and electroencephalographic alterations display considerable heterogeneity. Moreover, transcriptomic analyses have uncovered discrete gene‑expression signatures in molecularly defined patient subgroups, suggesting that genetic background modulates therapeutic response. These findings highlight the promise of mecasermin for ameliorating specific RTT features while underscoring the necessity of larger, rigorously designed trials to refine treatment regimens and implement stratified, patient‑specific approaches. CADTH recommends that Increlex should be reimbursed by public drug plans for the treatment of treatment of growth failure in children and adolescents from 2 to 18 years with confirmed severe primary insulin-like growth factor-1 deficiency (SPIGFD) if certain conditions are met. WHICH PATIENTS ARE ELIGIBLE FOR COVERAGE? Increlex should only be covered to treat patients who are at least 2 years of age with confirmed diagnosis of SPIGFD and in whom epiphyseal growth plates have not yet closed. WHAT ARE THE CONDITIONS FOR REIMBURSEMENT? Increlex should only be reimbursed if prescribed by a pediatric endocrinologist, if it is not prescribed in combination with recombinant growth hormone treatment, and the price of Increlex is reduced. WHY DID CADTH MAKE THIS RECOMMENDATION? Evidence from 1 clinical trial demonstrated that Increlex increases height velocity in children with open epi
Autism spectrum disorder (ASD), a heterogeneous group of neurodevelopmental disorders, is characterized by social impairment and repetitive and stereotypic behaviors. Because of the lack of approved laboratory diagnostic markers and effective therapeutic medications, it is one of the most challenging diseases. Therefore, it is urgent to explore potential diagnosis markers or therapeutic targets. Insulin-like growth factor 1 (IGF-1) is a neurotrophic growth factor that enhances brain development. IGF-1 levels in body fluids are lower in preschool children with ASD than in typically developing children, which may serve as a potential diagnostic marker. In various ASD models associated with genetic or environmental exposure, IGF-1 treatment can improve core symptoms or pathological changes, including neuronal development, neural cell survival, balance of synaptic excitation and inhibition, neuroimmunology, and oxidative stress status. In March 2023 an IGF-1 derivative was approved as the first drug for treating Rett syndrome, an ASD-related neurodevelopmental disorder, to improve fundamental symptoms such as social communication. Thus, in this review, we present accumulating evidence of altered IGF-1 levels in ASD patients and the possible mechanisms, as well as evidence that IGF-1 treatment improves the pathophysiology in various ASD models. IGF-1 has the potential to be an early diagnosis marker and an effective therapeutic for ASD.