Secondary outcomes of scoliosis surgery in disease‐modifying treatment‐naïve patients with spinal muscular atrophy type 2 and nonambulant type 3

Author:

Brusa Chiara1ORCID,Baranello Giovanni1,Ridout Deborah2,de Graaf Julie1,Manzur Adnan Y3,Munot Pinki3,Sarkozy Anna1,Main Marion3,Milev Evelin1ORCID,Iodice Mario1,Ramsey Danielle1,Tucker Stewart4,Ember Tom4,Nadarajah Ramesh4,Muntoni Francesco1,Scoto Mariacristina1

Affiliation:

1. Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, & Great Ormond Street Hospital Trust London UK

2. Population, Policy and Practice Department University College London Great Ormond Street Institute of Child Health London UK

3. Dubowitz Neuromuscular Centre Great Ormond Street Hospital for Children London UK

4. Orthopaedic and Spinal Surgery Unit Great Ormond Street Hospital for Children London UK

Abstract

AbstractIntroduction/AimsAvailable studies on scoliosis surgery in spinal muscular atrophy (SMA) have focused on the primary outcome of the procedure—the correction of the curve—whereas research focusing on secondary outcomes is scarce. We aimed to investigate postsurgical changes in respiratory function, motor function, weight, pain, and satisfaction.MethodsWe retrospectively reviewed the clinical notes of 32 disease‐modifying treatment‐naïve patients (26 SMA2, 6 nonambulant SMA3). We also performed investigator‐developed phone interviews and conducted a focus group with families on postsurgical satisfaction.ResultsMean annual rate of forced vital capacity percent decline improved in SMA2: −3.2% postsurgery versus −6.9% presurgery (p < .001), with similar trajectories in SMA3. Gross motor functional scores (Hammersmith Functional Motor Scale) available in 12/32 dropped immediately after surgery: median loss of 6.5 points, with relatively spared upper limb function. Weight z‐scores postsurgery dropped in 16/32, requiring food supplements (5/16); one/16 lost >5% of total weight requiring gastrostomy. Postsurgical pain was frequently reported, especially hip pain (13/32). Overall, 10/10 patients/parents participating in the phone interview rated the procedure as very successful for posture and physical appearance. Nonetheless, 7/10 reported postsurgical pain, reduced mobility, and unmet care needs. The seven patients/parents attending the focus group highlighted lack of intensive physiotherapy programs, occupational therapy assistance, and psychological support as postsurgical unmet care needs.DiscussionThis study reports a positive impact of scoliosis surgery on respiratory function and overall satisfaction with posture and physical appearance. The observed negative impact on the other outcomes highlights the importance of multidisciplinary approaches to improve postoperative management.

Funder

Biogen

Publisher

Wiley

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