Aktuelle Therapiekonzepte und Ergebnisse für Kinder mit Spinaler Muskelatrophie und Wirbelsäulendeformität

Zusammenfassung

Hintergrund Kinder mit Spinaler Muskelatrophie (SMA) Typ I und II entwickeln fast immer eine progrediente Wirbelsäulendeformität, welche nach Einführung der intrathekalen Injektionstherapie mit Nusinersen durch die damit verbundenen Schwierigkeiten bei der Punktion in den letzten Monaten in den Fokus gerückt sind.

Ziel der Arbeit Die vorliegende Untersuchung untersucht die Ergebnisse einer neuartigen operativen Therapie zur Wirbelsäulenkorrektur bei SMA Kindern im Vergleich zu unbehandelten SMA Jugendlichen.

Methoden In einer prospektiven Studie wurden 30 SMA Kinder (Gruppe I) mit von extern auszufahrenden magnetischen Implantaten versorgt, welche parallel zur Wirbelsäule implantiert wurden. Im Wachstumsverlauf wurden die Implantate ambulant alle drei Monate distrahiert. Diesem Kollektiv wurden 15 SMA Jugendliche (Gruppe II) mit unbehandelten Deformitäten gegenübergestellt.

Ergebnisse Gruppe I war bei Implantation durchschnittlich 7,6 Jahre alt und wurde 41 Monate nachuntersucht. Der Skoliosekrümmungswinkel konnte von 62 auf 25° (59%; p<0,001) und der Beckenschiefstand von 15 auf 4° (73%; p<0,001) reduziert werden. Im Gegensatz dazu hatte Gruppe II (Alter 13,7 Jahre) eine Skoliose von 101° und einen Beckenschiefstand von 22°. In Gruppe I konnten die erzielten Korrekturen im Untersuchungszeitraum gehalten werden.

Schlussfolgerung Bei SMA Kindern konnte durch die vorgestellte Operationstechnik eine signifikante Skoliosereduktion und Verbesserung des Beckenschiefstandes erreichen und im Verlauf gehalten werden im Vergleich zu unbehandelten Jugendlichen. Gerade auch in Hinblick auf die intrathekale Injektionstherapie mit Nusinersen zur SMA Behandlung ist die Notwendigkeit einer Wirbelsäule ohne starke Deformierung gegeben.

Abstract

Purpose Children with Spinal Muscular Atrophy (SMA) type I and II almost always develop progressive spinal deformities, which have become the focus of attention after the introduction of intrathecal injection therapy with nusinersen due to the associated difficulties with puncture in recent months.

Aim of the study The present study reports on the results of a new surgical treatment for spine correction in SMA children compared to untreated SMA adolescents.

Method In a prospective study, 30 SMA children (group I) were treated with externally controlled magnetic devices implanted parallel to the spine. The implants were extended in an outpatient setting every 3 months. Group I was compared to 15 SMA adolescents (group II) with untreated spinal deformities.

Results Group I was on average 7.6 years old at implantation and followed up for 41 months. The scoliotic curve angle could be reduced from 62 to 25° (59%, p<0.001) and the pelvic obliquity from 15 to 4° (73%, p<0.001). In contrast, group II (age 13.7 years) had a scoliosis of 101° and a pelvic obliquity of 22°. In group I, surgical corrections could be maintained during the investigation period.

Conclusion SMA children with spinal deformity were able to achieve a significant reduction of scoliosis and a significant improvement of the pelvic obliquity by the presented surgical technique. As expected, the compared untreated group II showed massive spinal deformity. Especially with regard to the intrathecal injection therapy with nusinersen for the treatment of SMA, the need for a spinal column without severe deformation is given.

Schlüsselwörter

Spinale Muskelatrophie (SMA) - Skoliose - Beckenschiefstand - Operationstechnik - magnetisch kontrolliert

Key words

spinal muscular atrophy (SMA) - scoliosis - pelvic obliquity - surgical technique - magnetically controlled

Publication History

Received: 15 May 2019

Accepted: 28 June 2019

Article published online:
16 June 2020

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