Systematic Review of Motor Function Scales and Patient-Reported Outcomes in Spinal Muscular Atrophy.

ABSTRACT: Spinal muscular atrophy is a heterogeneous disease that results in loss of motor function. In an evolving treatment landscape, establishing the suitability and limitations of existing motor function scales and patient-reported outcomes used to monitor patients with this disease is important. A systematic review was conducted to examine utility of motor function scales and patient-reported outcomes in evaluating patients with spinal muscular atrophy. Published literature was reviewed up to June 2021 with no start date restriction. Of the reports screened, 122 were deemed appropriate for inclusion and are discussed in this review (including 24 validation studies for motor function scales or patient-reported outcomes). Fifteen motor function scales and patient-reported outcomes were identified to be commonly used (≥5 studies), of which 11 had available validation assessments. Each instrument has its strengths and limitations. It is imperative that the patient population (e.g., age, mobility), goals of treatment, and outcomes or endpoints of interest be considered when selecting the appropriate motor function scales and patient-reported outcomes for clinical studies.

Selective dorsal rhizotomy: an illustrated review of operative techniques.

OBJECTIVE: Selective dorsal rhizotomy (SDR) is a procedure primarily performed to improve function in a subset of children with limitations related to spasticity. There is substantial variability in operative techniques among centers and surgeons. Here, the authors provide a technical review of operative approaches for SDR. METHODS: Ovid MEDLINE, Embase, and PubMed databases were queried in accordance with PRISMA guidelines. All studies included described a novel surgical technique. The technical nuances of each approach were extracted, including extent of exposure, bone removal, and selection of appropriate nerve roots. The operative approach preferred at the authors' institution (the "2 × 3 exposure") is also detailed. RESULTS: Five full-text papers were identified from a total of 380 articles. Operative approaches to SDR varied significantly with regard to level of exposure, extent of laminectomy, and identification of nerve roots. The largest exposure involved a multilevel laminectomy, while the smallest exposure involved a keyhole interlaminar approach. At the Hospital for Sick Children, the authors utilize a two-level laminoplasty at the level of the conus medullaris. The benefits and disadvantages of the spectrum of techniques are discussed, and illustrative figures are provided. CONCLUSIONS: Surgical approaches to SDR vary considerably and are detailed and illustrated in this review as a guide for neurosurgeons. Future studies should address the long-term impact of these techniques on functional outcomes and complications such as spinal deformity.

Intrathecal baclofen versus selective dorsal rhizotomy for children with cerebral palsy who are nonambulant: a systematic review.

OBJECTIVE: Cerebral palsy (CP) is the most common childhood physical disability. Historically, children with hypertonia who are nonambulatory (Gross Motor Function Classification System [GMFCS] level IV or V) were considered candidates for intrathecal baclofen (ITB) therapy to facilitate care and mitigate discomfort. Selective dorsal rhizotomy (SDR) was often reserved for ambulant children to improve gait. Recently, case series have suggested SDR as an alternative to ITB in selected children functioning at GMFCS level IV/V. The objective for this study was to systematically review the evidence for ITB and SDR in GMFCS level IV or V children. METHODS: Medline, Embase, Web of Science, and Cochrane databases were systematically searched. Articles were screened using the following inclusion criteria: 1) peer-reviewed articles reporting outcomes after SDR or ITB; 2) outcomes reported using a quantifiable scale or standardized outcome measure; 3) patients were < 19 years old at the time of operation; 4) patients had a diagnosis of CP; 5) patients were GMFCS level IV/V or results were reported based on GMFCS status and included some GMFCS level IV/V patients; 6) article and/or abstract in English; and 7) primary indication for surgery was hypertonia. Included studies were assessed with the Risk of Bias in Non-Randomized Studies - of Interventions (ROBINS-I) tool. RESULTS: Twenty-seven studies met inclusion criteria. The most commonly reported outcomes were spasticity (on the Mean Ashworth Scale) and gross motor function (using the Gross Motor Function Measure), although other outcomes including frequency of orthopedic procedures and complications were also reported. There is evidence from case series that suggests that both ITB and SDR can lower spasticity and improve gross motor function in this nonambulatory population. Complication rates are decidedly higher after ITB due in part to the ongoing risk of device-related complications. The heterogeneity among study design, patient selection, outcome selection, and follow-up periods was extremely high, preventing meta-analysis. There are no comparative studies, and meaningful health-related quality of life outcomes such as care and comfort are lacking. This review is limited by the high risk of bias among included studies. Studies of SDR or ITB that did not clearly describe patients as being GMFCS level IV/V or nonambulatory were excluded. CONCLUSIONS: There is a lack of evidence comparing the outcomes of ITB and SDR in the nonambulatory CP population. This could be overcome with standardized prospective studies using more robust methodology and relevant outcome measures.