Hip Displacement After Triradiate Closure in Ambulatory Cerebral Palsy: Who Needs Continued Surveillance?

BACKGROUND: Hip surveillance in cerebral palsy (CP) is an accepted practice with evidence-based guidelines implemented. For the skeletally immature with open triradiate cartilage (TRC), recommendations for radiographic surveillance stemmed from population-based studies. For nonambulatory CP, progression of hip displacement after skeletal maturity has been reported; less is known for ambulatory CP. We aimed to determine the prevalence and risk factors associated with progressive hip displacement after TRC closure, a proxy for skeletal maturity, for ambulatory CP. METHODS: This is a retrospective cohort study of patients with ambulatory CP (Gross Motor Function Classification System I-III), with unilateral or bilateral involvement, hypertonic motor type, regular hip surveillance (≥3 radiographs after age 10 yr, 1 before TRC closure, ≥1 after age 16 yr), and 2-year follow-up post-TRC closure. The primary outcome was migration percentage (MP). Other variables included previous preventative/reconstructive surgery, topographic pattern, sex, scoliosis, epilepsy, and ventriculoperitoneal shunt. An "unsuccessful hip" was defined by MP ≥30%, MP progression ≥10%, and/or requiring reconstructive surgery after TRC closure. Statistical analyses included chi-square and multivariate Cox regression. Kaplan-Meier survivorship curves were also determined. Receiver operating characteristic analysis was used to determine the MP threshold for progression to an "unsuccessful hip" after TRC closure. RESULTS: Seventy-six patients (39.5% female) met the inclusion criteria, mean follow-up 4.7±2.1 years after TRC closure. Sixteen (21.1%) patients had an unsuccessful hip outcome. By chi-square analysis, diplegia (P=0.002) and epilepsy (P=0.04) were risk factors for an unsuccessful hip. By multivariate analysis, only first MP after TRC closure (P<0.001) was a significant risk factor for progression to an unsuccessful hip; MP ≥28% being the determined threshold (receiver operating characteristic curve analysis, area under curve: 0.845, P<0.02). CONCLUSIONS: The risk of MP progression after skeletal maturity is relatively high (21%), similar to nonambulatory CP. Annual hip surveillance radiographs after TRC closure should continue for Gross Motor Function Classification System I-III with an MP ≥28% after TRC closure, especially for bilateral CP and epilepsy. LEVEL OF EVIDENCE: III.

Development and validation of a stakeholder-driven, self-contained electronic informed consent platform for trio-based genomic research studies.

Increasingly long and complex informed consents have yielded studies demonstrating comparatively low participant comprehension and satisfaction with traditional face-to-face approaches. In parallel, interest in electronic consents for clinical and research genomics has steadily increased, yet limited data are available for trio-based genomic discovery studies. We describe the design, development, implementation, and validation of an electronic iConsent application for trio-based genomic research deployed to support genomic studies of cerebral palsy. iConsent development incorporated stakeholder perspectives including researchers, patient advocates, institutional review board members, and genomic data-sharing considerations. The iConsent platform integrated principles derived from prior electronic consenting research and elements of multimedia learning theory. Participant comprehension was assessed in an interactive teachback format. The iConsent application achieved nine of ten proposed desiderata for effective patient-focused electronic consenting for genomic research. Overall, participants demonstrated high comprehension and retention of key human subjects' considerations. Enrollees reported high levels of satisfaction with the iConsent, and we found that participant comprehension, iConsent clarity, privacy protections, and study goal explanations were associated with overall satisfaction. Although opportunities exist to optimize iConsent, we show that such an approach is feasible, can satisfy multiple stakeholder requirements, and can realize high participant satisfaction and comprehension while increasing study reach.

Scoliosis Development in Spinal Muscular Atrophy: The Influences of Genetic Severity, Functional Level, and Disease-Modifying Treatments.

BACKGROUND: Spinal muscular atrophy (SMA) is caused by abnormalities of the survival motor neuron (SMN) 1 gene, leading to deficiency in SMN protein and loss of spinal cord alpha motor neurons. Newer disease-modifying agents (DMA) targeting the involved genes, including nusinersen and gene replacement therapies, have improved gross motor and respiratory function, but their impact on scoliosis development has not been established. This study aimed to determine risk factors for scoliosis development in SMA, specifically genetic severity and DMA use. METHODS: In this retrospective cohort study, children with SMA and minimum 2-year follow-up were included. The primary outcome was the prevalence of clinically relevant scoliosis. Secondary outcomes included SMA type, SMN2 copy number, Hammersmith Functional Motor Scale (HFMS), ambulatory status [functional mobility scale at 50m (FMS50)], DMA use, and hip displacement as risk factors. Univariate/multivariate logistic regression analyses were performed to identify dependent/independent risk factors. RESULTS: One hundred sixty-five patients (51% female) with SMA types I-III met the inclusion criteria, with total follow-up of 9.8 years. The prevalence of scoliosis was 79%; age of onset 7.9 years. The major curve angle for the entire cohort at first assessment and final follow-up was 37 degrees (SD: 27 degrees) and 62 degrees (SD: 31 degrees) (P<0.0001), respectively. Significant risk factors for scoliosis by univariate analysis were SMA type (I/II, P=0.02), HFMS (>23, P<0.001), nonambulatory status (FMS50=1, P<0.0001), DMA treatment (P=0.02), and hip displacement (P<0.0001). Multivariate analysis revealed that HFMS >23 (P=0.02) and DMA (P=0.05) treatment were independent (protective) risk factors. CONCLUSIONS: The development of scoliosis in SMA is high, with risk factors associated with proxy measures of disease severity, including SMA type, nonambulatory status, hip displacement, and most notably, gross motor function (by HFMS). DMA use and HFMS >23 were associated with a decreased risk of scoliosis development. Identified risk factors can be used in the development of surveillance programs for early detection of scoliosis in SMA. LEVEL OF EVIDENCE: Level III.

Prevalence and Treatment of Surgical Complications Following Proximal Femoral Osteotomies in Children with Cerebral Palsy: An Analysis of 1085 Hips.

Purpose: Proximal femoral osteotomy (PFO) is a reconstructive surgical option used to improve hip containment or correct internal hip rotation gait in children with cerebral palsy (CP). A few reports describe the risk of surgical complications after PFO. The purpose of this study was to determine the risk factors associated with adverse postoperative surgical outcomes in pediatric patients with CP following PFO and to report the treatment of complications. Methods: Following institutional review board approval, 1085 (1003 primary and 82 secondary) PFO procedures were retrospectively reviewed in 563 children with CP with at least 1 year of follow-up after final surgery over an 18-year follow-up period. Demographic characteristics, motor type, gross motor function classification system (GMFCS) level, medical comorbidities, feeding tube status, seizure history, intervention type, and prevalence of PFO-related surgical complications and associated treatments were evaluated. Multivariate regression analysis was performed to determine risk factors for all surgical complications. Results: During a 5.8-year (± 3.8 years) follow-up, at least 1 surgical complication was identified in 143 (13.1%) hips in 121 (21.5%) patients after PFO in children with CP. Of these complications, the most common was heterotopic ossification (65 [6%] of hips); most of which were asymptomatic and required no treatment. Other complications included 25 (2%) nonunions, 21 (2%) deep or superficial infections, 13 (1%) delayed unions, 12 (1%) peri-implant fractures, and 7 early implant failures (0.64). The rate of revision surgery due to these complications was 13.1% (6.8% of hips), of which 41% (30 revision surgeries) were for the treatment of nonunion. Regarding the development of delayed union or nonunion, dystonia, GMFCS level IV/V, and seizure history were identified as risk factors by multivariate analysis. Conclusions: The prevalence of surgical complications after PFO was 13.1% with 6.8% of hips requiring revision surgery. Dystonia, seizure history, and nonambulatory status were the strongest predictors for the need for revision surgery after PFO. These data can be used to help counsel patients and families regarding the risks associated with PFO for children with CP.Level of proof: IV; retrospective study.

The impact of hamstring lengthening on stance knee flexion at skeletal maturity in ambulatory cerebral palsy.

This study reports the long-term outcomes of hamstring lengthening to treat flexed knee gait in children with ambulatory cerebral palsy (CP) after skeletal maturity. This retrospective longitudinal observational study used instrumented gait analysis (GA) <8 and >15 years old in children with bilateral CP. The primary variable was knee flexion in stance phase. Eighty children (160 limbs) were included; 49% were male, 51% female. Mean age at first GA was 6.0 (SD: 1.2) years and 19.6 (SD: 4.5) years at final GA. Mean follow-up was 13.7 (SD: 4.7) years. Children were classified as Gross Motor Function Classification System I-8, II-46 and III-26. Average Gross Motor Function Measure Dimension D was 72% (SD: 20%). Hamstring lengthenings occurred once in 82, twice in 54 and three times in 10 limbs. From initial to final GA, average knee flexion in stance was unchanged, 27.8° (SD: 14.8°) to final 27.0° (SD: 11.2°; P = 0.54). Knee flexion at foot contact was 39.6° (SD: 13.0°), improving to final GA of 30.7° (SD: 10.6°; P < 0.001). Initial gait deviation index was 65.8 (SD: 31.9), improving to final 78.9 (SD: 28.2; P < 0.001). Older age, males and concomitant plantar flexor lengthening predicted change toward more flexed knee gait. Hamstring lengthening did not lead to back-kneeing gait at maturity while maintaining childhood stance phase knee flexion. A subgroup still developed significant flexed knee gait posture and may have benefited from more aggressive treatment options. This outcome may also be impacted by diverse functional levels, etiologies and treatments of flexed knee gait.

Changes in foot posture evaluated with dynamic pedobarography over the course of childhood in ambulatory youth with cerebral palsy.

Purpose: Foot deformities are prevalent in children with cerebral palsy, but there is limited research on the progression of foot posture during growth. Our study aimed to evaluate the change in dynamic foot posture in children with cerebral palsy. Methods: Children with cerebral palsy, aged 17-40 months, were recruited to participate in this Institutional Review Board-approved prospective longitudinal study by having serial foot posture evaluations. The coronal plane index and foot segmental impulses were measured with dynamic pedobarography. Data were compared between children stratified by Gross Motor Function Classification System level and typically developing children using serial Welch's t-tests across time with Holm correction for multiple comparisons. Results: In total, 33 children (54 limbs) were included in the analysis (21 bilateral and 12 unilateral; Gross Motor Function Classification System: I-13, II-14, III-4, IV-2. Children completed 16.9 (± 4.4) evaluations (initial age 2.9 (± 0.7) and final age 18.6 (± 1.7) years)). Early valgus foot posture normalizes in children at Gross Motor Function Classification System levels I/II and persists in children at levels III/IV who do not have foot surgery. For most young children, foot posture development is variable. Conclusion: Foot posture in young children with cerebral palsy begins in valgus and tends to normalize in youth who walk without an assistive device. Conservative management of foot deformity is recommended in early childhood. Level of evidence: Level II, prognostic study.

Self-reported Health-related Quality of Life in Adolescents With Cerebral Palsy.

BACKGROUND: Happiness, comfort, and motor function contribute to satisfaction with life for individuals with cerebral palsy (CP). Evidence-based medical care can improve motor function and physical health of youth with CP. Less is known about medical care and its relationship to health-related quality of life (HRQOL) in adolescents and young adults with CP. This study aimed to describe HRQOL among adolescents with CP to examine differences between adolescent (self) and parent (proxy) reports of HRQOL and to explore associations of pain, age, and gross motor function with HRQOL. METHODS: This is a retrospective study including adolescents with CP classified as Gross Motor Function Classification System levels I to V, ages 11 to 20 years, reading ≥ a fourth-grade level, and who completed the self-reported Pediatric Outcomes Data Collection Instrument (PODCI). Parents completed the PODCI concurrently or within 12 months and scores were compared. In addition, self-reported scores were compared between age bands, across Gross Motor Function Classification System levels, with typically developing youth (TDY), and between youth with/without pain. RESULTS: PODCI scores from 102 adolescents [59 males; 15.0 (SD: 2.6) years old] were examined. Scores from 50 adolescents and parents were matched. Mean self-reported scores were significantly higher than mean parent-reported scores in 4 domains: upper extremity and physical function ( P =0.018), sports and physical function ( P =0.005), happiness ( P =0.023), and global functioning ( P =0.018). All domains, except Happiness, were significantly < TDY ( P <0.01). The presence of pain was associated with lower scores in all domains ( P <0.05). CONCLUSION: Examining HRQOL with the PODCI revealed significant limitations in physical function and higher pain in adolescents with CP compared with TDY. Self- and parent-reported PODCI results should be considered separately. Adolescents report higher HRQOL compared with parent proxy. Recognizing and validating the perspectives of youth and their parents presents an opportunity for providers to discuss different points of view with families. Such engagement can help promote self-efficacy in youth with CP as they transition to the responsibility of guiding their own care in adulthood. LEVEL OF EVIDENCE: III, Retrospective comparative study.