| Titre : | Drug treatment for spinal muscular atrophy types II and III |
| Revue : | The Cochrane database of systematic reviews, 1 |
| Auteurs : | Wadman RI ; van der Pol WL ; Bosboom WM ; Asselman FL ; van den Berg LH ; Iannaccone ST ; Vrancken AF |
| Type de document : | Article |
| Editeur : | England, 01/2020 |
| Langues: | Anglais |
| Mots-clés : | amyotrophie spinale ; amyotrophie spinale proximale (type II) ; amyotrophie spinale proximale liée à SMN1 ; amyotrophie spinale proximale type 3 ; article de synthèse ; maladie du motoneurone ; maladie neuromusculaire ; prise en charge thérapeutique |
| Résumé : | MAIN RESULTS: The review authors found 10 randomised, placebo-controlled trials of treatments for SMA types II and III for inclusion in this review, with 717 participants. We added four of the trials at this update. The trials investigated creatine (55 participants), gabapentin (84 participants), hydroxyurea (57 participants), nusinersen (126 participants), olesoxime (165 participants), phenylbutyrate (107 participants), somatotropin (20 participants), thyrotropin-releasing hormone (TRH) (nine participants), valproic acid (33 participants), and combination therapy with valproic acid and acetyl-L-carnitine (ALC) (61 participants). Treatment duration was from three to 24 months. None of the studies investigated the same treatment and none was completely free of bias. All studies had adequate blinding, sequence generation and reporting of primary outcomes. Based on moderate-certainty evidence, intrathecal nusinersen improved motor function (disability) in children with SMA type II, with a 3.7-point improvement in the nusinersen group on the Hammersmith Functional Motor Scale Expanded (HFMSE; range of possible scores 0 to 66), compared to a 1.9-point decline on the HFMSE in the sham procedure group (P |
| Pubmed / DOI : |
Pubmed : 32006461  / DOI : 10.1002/14651858.CD006282.pub5
|
| N° Profil MNM : | 2020021 |
| En ligne : | http://www.ncbi.nlm.nih.gov/pubmed/32006461 |
| Voir aussi : |
MYOBASE
Portail documentaire sur les maladies neuromusculaires
Se connecter
Accueil
Sélection de la langue
Adresse
MYOBASEcontact