Consensus statement of the Hungarian Clinical Neurogenic Society about the therapy of adult SMA patients

• Published in: Ideggyógyászati szemle Vol. 74; no. 3-4; pp. 79 - 86
• Main Authors: Boczán, Judit, Klivényi, Péter, Kálmán, Bernadette, Széll, Márta, Karcagi, Veronika, Zádori, Dénes, Molnár, Judit Mária
• Format: Journal Article
• Language: Hungarian
• Published: Hungary 30.03.2021
• Subjects: Adult; Child; Consensus; Humans; Hungary; Muscular Atrophy, Spinal - genetics; Muscular Atrophy, Spinal - therapy; Prospective Studies; Retrospective Studies; Hungary; spinal muscular atrophy; risdiplam; therapy; spinal muscular atrophy (SMA); onasemnogene abeparvovec-xioi
• ISSN: 0019-1442
• DOI: 10.18071/isz.74.0079

Background - Spinal muscular atrophy (SMA) is an autosomal recessive, progressive neuromuscular disorder resulting in a loss of lower motoneurons. Recently, new disease-modifying treatments (two drugs for splicing modification of SMN2 and one for SMN1 gene replacement) have become available. Purpose - The new drugs change the progression of SMA with neonatal and childhood onset. Increasing amount of data are available about the effects of these drugs in adult patients with SMA. In this article, we summarize the available data of new SMA therapies in adult patients. Methods - Members of the Executive Committee of the Hungarian Clinical Neurogenetic Society surveyed the literature for palliative treatments, randomized controlled trials, and retrospective and prospective studies using disease modifying therapies in adult patients with SMA. Patients - We evaluated the outcomes of studies focused on treatments of adult patients mainly with SMA II and III. In this paper, we present our consensus statement in nine points covering palliative care, technical, medical and safety considerations, patient selection, and long-term monitoring of adult patients with SMA. This consensus statement aims to support the most efficient management of adult patients with SMA, and provides information about treatment efficacy and safety to be considered during personalized therapy. It also highlights open questions needed to be answered in future. Using this recommendation in clinical practice can result in optimization of therapy.