Subscribe to RSS
DOI: 10.1055/a-0965-7485
Aktuelle Therapiekonzepte und Ergebnisse für Kinder mit Spinaler Muskelatrophie und Wirbelsäulendeformität
Current Therapy Concepts and Results for Children with Spinal Muscular Atrophy and Spinal Deformity![](https://www.thieme-connect.de/media/physmed/202003/lookinside/thumbnails/2019-05-0376_10-1055-a-0965-7485-1.jpg)
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 kontrolliertKey words
spinal muscular atrophy (SMA) - scoliosis - pelvic obliquity - surgical technique - magnetically controlledPublication History
Received: 15 May 2019
Accepted: 28 June 2019
Article published online:
16 June 2020
© Georg Thieme Verlag KG
Stuttgart · New York
-
Literatur
- 1 Canavese F, Rousset M, Le Gledic B. et al. Surgical advances in the treatment of neuromuscular scoliosis. World J Orthop 2014; 5: 124-133
- 2 Chandran S, McCarthy J, Noonan K. et al. Early treatment of scoliosis with growing rods in children with severe spinal muscular atrophy: a preliminary report. J Pediatr Orthop 2011; 31: 450-454
- 3 Fujak A, Raab W, Schuh A. et al. Natural course of scoliosis in proximal spinal muscular atrophy type II and IIIa: descriptive clinical study with retrospective data collection of 126 patients. BMC Musculoskelet Disord 2013; 14: 283
- 4 Putzier M, Groß C, Zahn RK. et al. Characteristics of neuromuscular scoliosis. Orthopade 2016; 45: 500-508
- 5 Sucato DJ. Spine deformity in spinal muscular atrophy. J Bone Joint Surg Am 2007; 89 (Suppl. 01) 148-154
- 6 Chua K, Tan CY, Chen Z. et al. Long-term Follow-up of Pulmonary Function and Scoliosis in Patients With Duchenne’s Muscular Dystrophy and Spinal Muscular Atrophy. J Pediatr Orthop 2016; 36: 63-69
- 7 Akbarnia BA, Marks DS, Boachie-Adjei O. et al. Dual growing rod technique for the treatment of progressive early-onset scoliosis: a multicenter study. Spine 2005; 30: S46-S57
- 8 Campbell RM, Smith MD, Mayes TC. et al. The characteristics of thoracic insufficiency syndrome associated with fused ribs and congenital scoliosis. J Bone Joint Surg Am 2003; 85-A: 399-408
- 9 Skaggs DL, Akbarnia BA. Chest Wall and Spine Deformity Study Group . et al. Growing Spine Study Group, Pediatric Orthopaedic Society of North America, Scoliosis Research Society Growing Spine Study Committee. A classification of growth friendly spine implants. J Pediatr Orthop 2014; 34: 260-274
- 10 Plaass C, Hasler CC, Heininger U. et al. Bacterial colonization of VEPTR implants under repeated expansions in children with severe early onset spinal deformities. Eur Spine J 2016; 25: 549-556
- 11 Wagner L, Braunschweig L, Eiffert H. et al. Detection of Bacteria Colonizing Titanium Spinal Implants in Children. Surg Infect (Larchmt) 2018; 19: 71-77
- 12 Groenefeld B, Hell AK. Ossifications after vertical expandable prosthetic titanium rib treatment in children with thoracic insufficiency syndrome and scoliosis. Spine 2013; 38: E819-E823
- 13 Sankar WN, Skaggs DL, Yazici M. et al. Lengthening of dual growing rods and the law of diminishing returns. Spine 2011; 36: 806-809
- 14 Cheung KM-C, Cheung JP-Y, Samartzis D. et al. Magnetically controlled growing rods for severe spinal curvature in young children: a prospective case series. Lancet 2012; 379: 1967-1974
- 15 Hell AK, Groenefeld K, Tsaknakis K. et al. Combining Bilateral Magnetically Controlled Implants Inserted Parallel to the Spine With Rib to Pelvis Fixation: Surgical Technique and Early Results. Clin Spine Surg 2018; 31: 239-246
- 16 Lorenz HM, Braunschweig L, Badwan B et al. High Correlation Between Achieved and Expected Distraction Using Magnetically Controlled Growth Rods (MCGR) With Rib to Pelvis Fixation in Pediatric Spine Deformity. J Pediatr Orthop 2018
- 17 Lorenz HM, Kühnle I, Edler J. et al. Intrathecal Nursinersen Therapy in Children with Spinal Muscular Atrophy and Spinal Deformities. Klin Padiatr 2018; 230: 231-233
- 18 Strauss KA, Carson VJ, Brigatti KW. et al. Preliminary Safety and Tolerability of a Novel Subcutaneous Intrathecal Catheter System for Repeated Outpatient Dosing of Nusinersen to Children and Adults With Spinal Muscular Atrophy. J Pediatr Orthop 2018; 38: e610-e617
- 19 Lorenz HM, Badwan B, Hecker MM. et al. Magnetically Controlled Devices Parallel to the Spine in Children with Spinal Muscular Atrophy. JB JS Open Access 2017; 2: e0036