Subperiosteal delivery of transforming growth factor beta 1 and human growth hormone from mineralized PCL films.

The ability to locally deliver bioactive molecules to distinct regions of the skeleton may provide a novel means by which to improve fracture healing, treat neoplasms or infections, or modulate growth. In this study, we constructed single-sided mineral-coated poly-ε-caprolactone membranes capable of binding and releasing transforming growth factor beta 1 (TGF-ß1) and human growth hormone (hGH). After demonstrating biological activity in vitro and characterization of their release, these thin bioabsorbable membranes were surgically implanted using an immature rabbit model. Membranes were circumferentially wrapped under the periosteum, thus placed in direct contact with the proximal metaphysis to assess its bioactivity in vivo. The direct effects on the metaphyseal bone, bone marrow, and overlying periosteum were assessed using radiography and histology. Effects of membrane placement at the tibial growth plate were assessed via physeal heights, tibial growth rates (pulsed fluorochrome labeling), and tibial lengths. Subperiosteal placement of the mineralized membranes induced greater local chondrogenesis in the plain mineral and TGF-ß1 samples than the hGH. More exuberant and circumferential ossification was seen in the TGF-ß1 treated tibiae. The TGF-ß1 membranes also induced hypocellularity of the bone marrow with characteristics of gelatinous degeneration not seen in the other groups. While the proximal tibial growth plates were taller in the hGH treated than TGF-ß1, no differences in growth rates or overall tibial lengths were found. In conclusion, these data demonstrate the feasibility of using bioabsorbable mineral coated membranes to deliver biologically active compounds subperiosteally in a sustained fashion to affect cells at the insertion site, bone marrow, and even growth plate.

What is New in Pediatric Orthopaedic: Basic Science.

BACKGROUND: An understanding of musculoskeletal basic science underpins most advancements in the field of orthopaedic surgery. Knowledge of biomechanics, genetics, and molecular pathways is integral to the understanding of the pathophysiology of disease and guides novel treatment options to improve patient outcomes. The purpose of this review is to provide a comprehensive and current overview of musculoskeletal basic science relevant to pediatric orthopaedic surgery. METHODS: Comprehensive Pubmed database searches were performed for all English language articles published between January 2016 and November 2021 using the following search terms: basic science, pediatric orthopaedics, fracture, trauma, spine, scoliosis, DDH, hip dysplasia, Perthes, Legg-Calve-Perthes, clubfoot, and sports medicine. Inclusion criteria focused on basic science studies of pediatric orthopaedic conditions. Clinical studies or case reports were excluded. A total of 3855 articles were retrieved. After removing duplicates and those failing to meet our inclusion criteria, 49 articles were included in the final review. RESULTS: A total of 49 papers were selected for review based on the date of publication and updated findings. Findings are discussed in the subheadings below. Articles were then sorted into the following sub-disciplines of pediatric orthopaedics: spine, trauma, sports medicine, hip, and foot. CONCLUSIONS: With this review, we have identified many exciting developments in pediatric orthopaedic trauma, spine, hip, foot, and sports medicine that could potentially lead to changes in disease management and how we think of these processes. LEVEL OF EVIDENCE: Level V.

Hip Pain in Nonambulatory Children with Type-I or II Spinal Muscular Atrophy.

The purpose of the present study was to define the prevalence of hip pain in nonambulatory children with spinal muscular atrophy (SMA) (type I or II) treated with aggressive medical management, prior to widespread use of disease-modifying therapies (DMTs). Methods: A retrospective chart review (1993 to 2017) was performed on children diagnosed with SMA to identify subjective reports of hip pain and associated interventions, while radiographs were evaluated to assess hip instability and spinal deformity. Results: Seventy-two patients (33 with type I and 39 with type II) met the inclusion criteria. Hip pain was more frequent in type-II SMA (49% versus 12%; p = 0.001). Seventeen percent of the patients with 2 copies of the SMN2 (survival motor neuron 2) gene, 53% of patients with 3 copies, and 1 of the 2 patients with 4 copies reported hip pain. Nearly all patients had abnormal findings on hip radiographs made at the onset of pain or at the latest follow-up; however, no patient with type-I and 18% of those with type-II SMA had pain that was severe enough to undergo invasive intervention (p = 0.01). The intervention reduced the pain in most of those patients but completely eliminated it in only 1 patient. No significant differences were found with respect to the mean age at the onset of scoliosis, the mean age at the time of scoliosis surgery, or whether insertion of growing rods or posterior spine fusion was performed between those with and without hip pain requiring invasive treatment. Conclusions: This study is, to our knowledge, the largest investigation to date to assess hip pain among nonambulatory children with type-I or type-II SMA and suggests that symptoms rather than radiographs be utilized to direct care. These data will be crucial in assessing any effects that the new DMTs have on the natural history of hip pathology and pain in nonambulatory patients with SMA. Level of Evidence: Prognostic Level IV. See Instructions for Authors for a complete description of levels of evidence.

Novel Cast-saw Alarm System Reduces Blade-to-Skin Contact in a Pediatric Upper Extremity Model.

PURPOSE: To determine the effectiveness of a novel cast-saw alarm system in minimizing the number and duration of cast-saw blade-to-skin contacts. METHODS: Twenty orthopaedic residents removed a pair of long-arm casts applied to instrumented pediatric upper extremity models. The model and cast-saw were instrumented to detect blade to "skin" contact at a rate of 600 Hz. Each resident performed cast removal with and without the use of a cast-saw alarm, the order of which was randomized. Eleven additional "new" cast-saw users then removed pairs of casts, without and then with the cast alarm, to evaluate what effect the alarm would have on preventing blade-to-skin contact in users with no previous cast-saw experience. The number and duration of cast-saw touches were then evaluated. Statistical significance was determined paired 1-sided students t tests (number of touches). RESULTS: For the residents (n=20), the total number of blade-to-skin contacts was 233. One hundred eighty-one blade-to-skin contacts without the alarm and 52 with the alarm (71% reduction) (t(19)=-3.42, P=0.001), averaging 6.45 more blade-to-skin contacts per cast without the alarm. The median blade-to-skin contact duration was 0.166 seconds without the alarm and 0.087 seconds with the alarm. This was a 48% reduction in contact time (P=0.073). For the inexperienced users (n=11), the total number of blade-to-skin contacts was 356, 324 blade-to-skin contacts without the alarm and 32 with the alarm (90% reduction) (t(10)=-2.78, P=0.009), averaging 26.5 more blade-to-skin contacts without the alarm. The median blade-to-skin contact duration for the novice was 0.313 seconds without the alarm and 0.1 seconds with the alarm (68% reduction). Contact time was reduced in both groups but failed to reach statistical significance. However, alarm use significantly reduced the number of touches of >0.5 seconds duration (62 vs. 3) in the novice group, P=0.0176. Blade-to-skin contact of >0.5 seconds were felt to represent touches that were more likely to result in thermal injury to a living patient. CONCLUSION: Blade-to-skin contact can be reduced with the use of a cast-saw alarm. These effects appear most amplified in users with little prior cast-saw experience. LEVEL OF EVIDENCE: Not applicable.

Histological and radiographic evaluation of three common tendon transfer techniques in an un-ossified bone porcine model: implications for early anterior tibialis tendon transfers in children with clubfeet.

PURPOSE: To compare the histological healing and radiographic effects of tendons transferred to ossified or unossified bone using different tendon fixation techniques. METHODS: Nine new-born piglets underwent bilateral tendon transfers to either the ossified boney calcaneal body or unossified apophysis. The tendons were fixed using metallic suture anchors, sutures alone or a bone tunnel. At six weeks of age, calcanei were harvested, radiologically imaged and then prepared for histology. A semi-quantitative aggregated scoring system with values ranging from 0 (poor) to 15 (excellent), was used to grade healing at the surgical enthesis and the apophyseal ossification was graded by five independent reviewers in triplicate using a modified (1 to 4) validated scoring system. RESULTS: Histologically, the cartilaginous transfers utilizing the tunnel and suture techniques also demonstrated the best average aggregated scores of entheses healing rivalling that measured in transfers using the classic bone tunnel technique (clinical benchmark), whereas suture anchor fixation demonstrated the worst healing in both the ossified and unossified samples. All three transfer techniques caused at least minor alterations in apophyseal ossification, with the most significant changes observed in the metallic suture anchor cohort. The tunnel and suture techniques demonstrated similar and more mild abnormalities in ossification. CONCLUSION: Tendon transfers to unossified bone heal histologically as well as transfers classically performed through tunnels in bone. Suture fixation or tunnel techniques appear radiographically and histologically superior to suture anchors in our newborn porcine model.

A Prospective, Median 15-Year Comparison of Ponseti Casting and Surgical Treatment of Clubfoot.

BACKGROUND: In 2010, 2 authors of this current study reported the results of Ponseti treatment compared with primary posteromedial release (PMR) for congenital talipes equinovarus in a cohort of 51 prospective patients. This current study shows outcomes recorded at a median of 15 years after the original treatment. METHODS: Patient health records were available for all 51 patients at a median of 15 years (range, 13 to 17 years) following treatment of congenital talipes equinovarus with either the Ponseti method (25 patients [38 feet]) or PMR (26 patients [42 feet]). Thirty-eight of 51 patients could be contacted, and 33 patients (65%) participated in the clinical review, comprising patient-reported outcomes, clinical examination, 3-dimensional gait analysis, and plantar pressures. RESULTS: Sixteen (42%) of 38 Ponseti-treated feet and 20 (48%) of 42 PMR-treated feet had undergone a further surgical procedure. The PMR-treated feet were more likely to undergo osteotomies and intra-articular surgical procedures (15 feet) than the Ponseti-treated feet (5 feet) (p < 0.05). Of the 33 patients reviewed with multimodal assessment, the Ponseti group, compared with the PMR group, demonstrated better Dimeglio scores (5.8 compared with 7.0 points; p < 0.05), Disease Specific Instrument (80.7 compared with 65.6 points; p < 0.05), Functional Disability Inventory (1.1 compared with 5.1; p < 0.05), and American Academy of Orthopaedic Surgeons (AAOS) Foot and Ankle Outcomes Questionnaire scores (52.2 compared with 46.6 points; p < 0.05), as well as improved total sagittal ankle range of motion in gait and ankle plantar flexion range at toe-off. The PMR group with clinical hindfoot varus displayed higher pressures in the lateral midfoot and the forefoot. CONCLUSIONS: Although the numbers of repeat surgical interventions following Ponseti treatment and primary PMR were similar, the PMR-treated feet had greater numbers of osteotomies and intra-articular surgical procedures. Functional outcomes were improved at a median of 15 years for feet treated with the Ponseti method compared with feet treated with PMR, with advantages seen in the Ponseti group over several domains. This study provides the most comprehensive evaluation of outcomes close to skeletal maturity in prospective cohorts, reinforcing the Ponseti method as the initial treatment of choice for idiopathic clubfeet. LEVEL OF EVIDENCE: Therapeutic Level II. See Instructions for Authors for a complete description of levels of evidence.

Quality of Life Outcomes According to Differential Nusinersen Exposure in Pediatric Spinal Muscular Atrophy.

The purpose of this study was to explore early changes in patient and family caregiver report of quality of life and family impact during the transitional period of nusinersen use. Communication; family relationships; physical, emotional, social, and cognitive functioning; and daily activities were measured using Pediatric Quality of Life modules (Family Impact Modules and both Patient and Proxy Neuromuscular-Specific Reports) pre- and post-nusinersen exposure. A total of 35 patients with SMA (15 Type 1, 14 Type 2, and 6 Type 3) were grouped according to nusinersen exposure. When analyzed as a whole cross-sectional clinical population, no significant differences were found between the initial and final surveys. Nusinersen therapy was associated with improved communication and emotional functioning in subsets of the population, particularly for patients on maintenance therapy for longer duration. Several unexpected potentially negative findings including increases in family resources and trends towards increases in worry warrant further consideration. Further research is warranted to explore the impact of novel pharmaceuticals on quality of life for children with SMA longitudinally to optimize clinical and psychosocial outcomes.

Sagittal Plane Deformities in Children with SMA2 following Posterior Spinal Instrumentation.

This is a retrospective radiographic review to assess post-operative sagittal plane deformities in patients with Spinal Muscular Atrophy type 2 that had been treated with posterior spinal instrumentation. Thirty-two patients with a history of either spinal fusion (N = 20) or growing rods (N = 12) were identified with an average of 7.6 (2.1-16.6) years post-operative follow-up. Forty percent (13/32) of the patients were identified as having obvious "tucked chin" (N = 4), "tipped trunk" (N = 9), or both (N = 3). Sacral incidence was the only parameter that was statistically significant change between pre-operative or immediate post-operative measurements (66.9° vs. 55.2° p = 0.03). However, at final follow-up, the post-operative thoracic kyphosis had decreased over time in those that developed a subsequent sagittal deformity (24.2°) whereas it increased in those that did not (44.7°, p = 0.008). This decrease in thoracic kyphosis throughout the instrumented levels, resulted in a greater lordotic imbalance (30.4° vs. 5.6°, p = 0.001) throughout the instrumented levels in the group that developed the subsequent cervical or pelvic sagittal deformities. In conclusion, sagittal plane deformities commonly develop outside the instrumented levels in children with SMA type 2 following posterior spinal instrumentation and may be the result of lordotic imbalance that occurs through continued anterior growth following posterior instrumentation.

Keeping casts dry: A comparison of commercially available cast protectors using a mechanized short arm cast model.

BACKGROUND: Upper extremity fractures requiring cast immobilization are exceedingly common, especially in the pediatric population. Studies have shown improved outcomes when patients can participate in water-based activities while casted. However, waterproof cast material is not feasible in all clinical settings and wet cast complications remain a source of morbidity and expense. External cast protectors play an important role in preventing wet casts, but the efficacy of various commercially available brands during relevant water-based activity remains unknown. PURPOSES: To determine if there are differences in the rate and extent of moisture exposure for four commercially available cast protectors using a mechanized cast arm model and human volunteers. METHODS: A mechanized arm model was developed with four implanted humidity sensors. Cast protectors were applied over the arm, the model was submerged in water, and moved back and forth, simulating cast-wearers' motion. Data regarding humidity was recorded for successive 10-minute trials. Trials were analyzed using a mixed effects linear model to determine change in humidity over time. The top and bottom performing cast protectors were then applied to four adult volunteers prior to thirty minutes of swimming. Questionnaires regarding comfort and a qualitative assessment of cast wetness using a chemical color indicator were completed. RESULTS: 372 instances of sensor data from 96 10-minute trials was collected. The CVS, SealTight and Walgreens brands showed significant increases in humidity beginning at 10, 20 and 20 minutes, respectively. DryPro showed no significant increase in moisture level up to 50 minutes. In successive trials up to 120 minutes, DryPro showed only a 2% increase in moisture. In human subjects testing, 3/4 casts underneath CVS protectors had some degree of wetness-related color change that would require cast change as compared to 0/4 casts underneath DryPro protectors. CONCLUSIONS: Significant differences exist between commercially available cast protectors. Vacuum-sealed protectors performed best in both mechanical and human subject portions of this study and allowed minimal change in humidity for extended periods of sequential water immersion. Their cost is notably less than management of a wet cast. Lower-performing products may expose cast-wearers to an increased risk of wet cast complications.

Exosome-educated macrophages and exosomes differentially improve ligament healing.

Recently, our group used exosomes from mesenchymal stromal/stem cells (MSCs) to simulate an M2 macrophage phenotype, that is, exosome-educated macrophages (EEMs). These EEMs, when delivered in vivo, accelerated healing in a mouse Achilles tendon injury model. For the current study, we first tested the ability of EEMs to reproduce the beneficial healing effects in a different rodent model, that is, a rat medial collateral ligament (MCL) injury model. We hypothesized that treatment with EEMs would reduce inflammation and accelerate ligament healing, similar to our previous tendon results. Second, because of the translational advantages of a cell-free therapy, exosomes alone were also examined to promote MCL healing. We hypothesized that MSC-derived exosomes could also alter ligament healing to reduce scar formation. Similar to our previous Achilles tendon results, EEMs improved mechanical properties in the healing ligament and reduced inflammation, as indicated via a decreased endogenous M1/M2 macrophage ratio. We also showed that exosomes improved ligament remodeling as indicated by changes in collagen production and organization, and reduced scar formation but without improved mechanical behavior in healing tissue. Overall, our findings suggest EEMs and MSC-derived exosomes improve healing but via different mechanisms. EEMs and exosomes each have attractive characteristics as therapeutics. EEMs as a cell therapy are terminally differentiated and will not proliferate or differentiate. Alternatively, exosome therapy can be used as a cell free, shelf-stable therapeutic to deliver biologically active components. Results herein further support using EEMs and/or exosomes to improve ligament healing by modulating inflammation and promoting more advantageous tissue remodeling.

Effects of tourniquet use in paediatric lower leg surgery.

BACKGROUND: The purpose of this study is to evaluate the use of the tourniquet and its effect on post-operative pain in the paediatric population following lower leg procedures. METHODS: A retrospective study of paediatric patients (under the age of 18) undergoing inpatient orthopaedic procedure below the knee performed at a single academic institution between 1st December 2013 and 31st January 2019 was conducted. Primary outcome measures of total opioid consumption during hospital stay and pre-operative nerve block utilization were retrieved from the electronic medical record (EMR). Secondary outcome measures of blood loss, tourniquet time, procedure time and length of hospital stay were also retrieved. Student's t-tests were used to assess statistical significance between two sample means. RESULTS: The final analysis included 204 paediatric procedures, 118 of which used a tourniquet and 86 of which did not. Paediatric patients with a tourniquet had significantly more opioid consumption post-operatively in the form of weight-based morphine equivalents/length of stay (p = 0.01) compared to those who had no tourniquet. This held true for males (p = 0.049) and females (p = 0.04) respectively. We did not see an increase in wound complications or return trips to the operating room in the tourniquet cohort. All procedures included an osseous component except one procedure in the non-tourniquet group. CONCLUSION: Minimizing opioid consumption may be achieved by avoiding tourniquet use in paediatric patients with lower leg procedures. In non-anaemic paediatric patients, it is reasonably risk-free to perform these surgeries without the use of tourniquet to decrease opioid dependence in the post-operative period. LEVEL OF EVIDENCE: III.

Is There a Connection Between Attention Deficit Hyperactivity Disorder and Osteochondritis Dissecans?

Background: To determine if children with Osteochondritis Dessicans (OCD) lesions of the distal femur are more likely to have a co-morbid diagnosis of Attention Deficit/ Hyperactivity Disorder (ADHD) than age matched controls and to assess the impact of ADHD on OCD outcomes. Methods: A retrospective chart review of patients treated at a single tertiary care hospital between 2000-2012 was performed. Charts were reviewed for a diagnosis of OCD of the distal femur in all skeletally immature patients (males < 16 years and females < 14 years). These were then screened for a comorbid diagnosis of ADHD. Age-matched controls with anterior knee pain without OCD were then reviewed to determine if ADHD was more common in the OCD population. Treatment and outcomes of the OCD lesions were then compared in children with and without ADHD. Results: The prevalence of ADHD was 23% in patients with OCD lesions and was significantly greater than the 11% found in the anterior knee pain age-matched controls (p<0.05). The average grade of lesions at presentation was similar in both groups (2.2 ADHD vs 2.1 no ADHD) however, at final follow-up, the average OCD grade was significantly worse for children with ADHD (1.4 vs 0.7, p<0.004). Conclusion: There is a significantly higher prevalence of ADHD in children with OCD lesions compared with age-matched controls. This study suggests children that with osteochondritis dessicans and ADHD may not have as favorable treatment course as children without the hyperactivity disorder.Level of Evidence: III.

Cervical Kyphosis in Spinal Muscular Atrophy: A Case Report.

CASE: A 12-year-old boy with spinal muscular atrophy (SMA) Type II presented 5 years after undergoing spinal growing rod placement with cervical kyphosis at C2-3. He underwent anterior cervical discectomy and fusion but 6 years later developed significant kyphosis at the adjacent C3-4 level. CONCLUSION: We describe a rare adjacent segment kyphotic condition in a young man with SMA Type II. Clinicians should be cognizant of the risk of cervical kyphosis in adolescent patients with SMA.

KIAA1217: A novel candidate gene associated with isolated and syndromic vertebral malformations.

Vertebral malformations (VMs) are caused by alterations in somitogenesis and may occur in association with other congenital anomalies. The genetic etiology of most VMs remains unknown and their identification may facilitate the development of novel therapeutic and prevention strategies. Exome sequencing was performed on both the discovery cohort of nine unrelated probands from the USA with VMs and the replication cohort from China (Deciphering Disorders Involving Scoliosis & COmorbidities study). The discovery cohort was analyzed using the PhenoDB analysis tool. Heterozygous and homozygous, rare and functional variants were selected and evaluated for their ClinVar, HGMD, OMIM, GWAS, mouse model phenotypes, and other annotations to identify the best candidates. Genes with candidate variants in three or more probands were selected. The replication cohort was analyzed by another in-house developed pipeline. We identified rare heterozygous variants in KIAA1217 in four out of nine probands in the discovery cohort and in five out of 35 probands in the replication cohort. Collectively, we identified 11 KIAA1217 rare variants in 10 probands, three of which have not been described in gnomAD and one of which is a nonsense variant. We propose that genetic variations of KIAA1217 may contribute to the etiology of VMs.

Biomechanical Evaluation of a Minimally Invasive Fixation Method for Length Unstable Limb Injuries.

INFIX instrumentation has provided an alternative treatment option for anteriorly unstable pelvic injuries. In this study, we explore the biomechanical feasibility of using an INFIX construct in an unstable longbone model and present a unique clinical case of its use. The external fixation, locked plate and spinal implant constructs (n = 5 each) were applied to lengthunstable fracture models and tested under various loads. Analysis of variance and pairwise Ttests were performed with levels of significance adjusted by Bonferroni correction to account for multiple comparisons. The biomechanical stiffness of the INFIX was found to be intermediate between the other two constructs in axial loading and torsion and was equivalent to one of the other constructs in sagittal and lateral bending. It was never the most compliant construct in any testing mode. This study and case report demonstrate the biomechanical feasibility of using INFIX to treat limb injuries. (Journal of Surgical Orthopaedic Advances 29(1):1825, 2020).

Is prophylactic formal fusion with implant revision necessary in non-ambulatory children with spinal muscular atrophy and growing rods who are no longer lengthened?

STUDY DESIGN: Single center, retrospective chart review. OBJECTIVES: To determine if routine posterior spinal fusion (PSF) is unnecessary in non-ambulatory growing rod graduates with SMA. Most non-ambulatory children with SMA develop early-onset scoliosis (EOS). Posterior growing rods (GR) have been shown safe and effective in managing spinal deformities in these children. The best management of these children, once graduated from their GR, is currently unknown. In this study, we report the clinical results of managing these children without routine definitive fusion following a course of GR treatment. METHODS: A single-center, retrospective chart and radiographic review was performed on children with SMA treated with posterior distraction GR, with a two-year minimum follow-up since final lengthening. Electronic medical records and radiographs were reviewed for demographic variables, Cobb measurements, implant revisions, occult radiographic implant failure, symptomatic failure, and/or conversion to PSF. RESULT: 12 patients (2 type 1, 9 type 2, 1 type 1/2) met inclusion criteria. Mean age at growing rod insertion was 6.2 years of age (range 4.1-8.2) and age at final lengthening 10.3 years of age (range 9.3-11.9). The mean time between last lengthening and latest clinical or radiographic review was 5.5 (range 2.1-9.0) years. Average mean pre, post, final Cobb angles were 71°, 27° (p < 0.001), 25°. Following final lengthening, only one patient required hardware revision and conversion to definitive fusion in attempts to alleviate chronic hip pain, which was unsuccessful. One additional patient was found to have an occult rod failure that has not required treatment. CONCLUSION: While limited by sample size, this single-center cohort of non-ambulatory SMA patients with EOS treated with similar constructs suggests that routine, definitive fusion in SMA GR graduates may be unnecessary. LEVEL OF EVIDENCE: Level IV.

A Prospective, Crossover Survey Study of Child- and Proxy-Reported Quality of Life According to Spinal Muscular Atrophy Type and Medical Interventions.

BACKGROUND: Spinal muscular atrophy is an autosomal-recessive, progressive neuromuscular disease associated with extensive morbidity. Children with spinal muscular atrophy have potentially increased life spans due to improved nutrition, respiratory support, and novel pharmaceuticals. OBJECTIVES: To report on the quality of life and family experience for children with spinal muscular atrophy with attentiveness to patient- and proxy-concordance and to stratify quality of life reports by spinal muscular atrophy type and medical interventions. METHODS: A prospective, crossover survey study inclusive of 58 children (26 spinal muscular atrophy type I, 23 type II, 9 type III) and their family caregivers at a free-standing Midwestern children's hospital. Twenty-eight families completed the 25-item PedsQL 3.0 Neuromuscular Module. Forty-four participants completed the 36-item PedsQL Family Impact Module and 47 completed the Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD) questionnaire. RESULTS: The PedsQL Family Impact Module demonstrated significant differences between spinal muscular atrophy types I and II in functioning domains including physical, emotional, social, and family relations (P < .03). Child self-report and proxy report surveys demonstrated significant differences between spinal muscular atrophy types in the communication domains (P < .003). Children self-reported their quality of life higher than proxy report of child quality of life. Gastrostomy tube (P = .001) and ventilation support (P = .029) impacted proxy-reported quality of life perspectives, whereas nusinersen use did not. Spinal surgery was associated with improved parental quality of life and family impact (P < .03). CONCLUSIONS: The measurement and monitoring of quality of life for children with spinal muscular atrophy and their families represents an implementable priority for care teams.

Does an intrathecal baclofen pump impact scoliosis progression and complicate posterior spine fusion in patients with cerebral palsy?

STUDY DESIGN: Retrospective comparative study. OBJECTIVES: In patients with cerebral palsy (CP), we determine the impact of intrathecal baclofen pumps (ITBPs) on scoliosis curve progression before posterior spine fusion (PSF) and its effects on surgical outcome. BACKGROUND: Children with CP can have rapid scoliosis progression, and high rates of surgical complications can be encountered. It is unknown whether the presence of pre-existing ITBP results in more difficult surgery and higher complication rates in similarly affected children. METHODS: This is a single-center retrospective study of CP patients undergoing PSF over a 15-year period. Demographics, comorbidities, curve magnitudes, and surgical methods were compared between patients with ITBP and those without. Postoperative complications, length of intensive care unit/hospital stay, drain use and output volume, and need for further surgery were also compared. Curve progression analysis after ITBP placement was performed on a subgroup of patients with high-quality consistent radiographs. RESULTS: Nineteen patients with ITBP and 49 patients without ITBP met inclusion criteria. Age, comorbidities, number of levels fused, and fixation techniques during PSF were not significantly different between cohorts. ITBP patients were more likely to have PSF with osteotomy (p = 0.022). Increased intraoperative neurosurgical consultations were found for patients with ITBP (42.1% vs. 4.0%; p < 0.001). Median surgical time was 1.2 h greater in patients with ITBP (6.7 vs. 5.5 h, p = 0.039). There was no difference in hospital course and complications in patients with ITBP and those without ITBP. Thirty-one patients without ITB were compared with 15 ITBP patients for curve progression before PSF, demonstrating a mean rate of scoliosis progression of 9.6° ± 6.7°/year and 14.8° ± 9.1°/year (p = 0.0346), respectively. CONCLUSION: The presence of an ITBP appears to be associated with the increase in scoliosis progression; and these patients will likely have a more challenging spine fusion. Fortunately, the final outcome is not affected by ITBP. LEVEL OF EVIDENCE: Level III.

Peri-operative management of children with spinal muscular atrophy.

BACKGROUND AND AIMS: Current multi-disciplinary management of children with spinal muscular atrophy (SMA) often requires the surgical management of spinal deformities. We present the outcomes of our peri-operative experience around the time of their spinal surgery and share our neuromuscular perioperative protocol. METHODS: A single-centre retrospective chart review was performed to evaluate all children with SMA types I and II that underwent thoracolumbar spinal deformity correction (posterior spinal fusion or growing rod insertion) from 1990 to 2015. Electronic medical records were reviewed to assess pre-operative, intraoperative, and postoperative variables. T-tests, Wilcoxon Rank Sum, Fisher's Exact tests were performed as appropriate. RESULTS: Twelve SMA I and twenty-two SMA II patients were included. Type I patients tended to be smaller and had a higher percentage (36.4% vs 4.5%) of American Society of Anesthesiologists (ASA) class 4 patients. Preoperative total parenteral nutrition (TPN) was utilised in 75.0% of type I and 18.2% type II patients. A difficult intubation was experienced in around 25% of the patients (20.0% SMA I, 27.3% SMA II). Approximately two hours of anaesthetic time was required in addition to the actual surgical time in both types. The intensive care unit (ICU) length of stay averaged 6 (4.0-7.5) days for type I and 3 (3-5) days for type II (p = 0.144). Average post-operative length of stay was (8 (7-9) vs. 7 (6-8)) P = 1.0. CONCLUSION: Children with type I and II SMA have similar hospital courses. The surgical and anaesthesia team should consider perioperative TPN and NIPPV (non-invasive positive-pressure ventilation), anticipate difficult intubations, longer than usual anaesthetic times, and potentially longer ICU stays in both SMA type I and II.

Cervical Spine Injury Following Thoracic Spinal Fusion for Adolescent Idiopathic Scoliosis.

Spinal fusion for adolescent idiopathic scoliosis (AIS) can have many potential complications, including spinal cord injury. Most often, spinal cord injury occurs in the region of surgery due to direct mechanical trauma. Vascular compromise in this area may also occur due to a high degree of correction or excessive distraction of the spine. In these cases, the impairment of spinal cord function is often detected intraoperatively with spinal cord monitoring and confirmed in the immediate postoperative period. Injury to the spinal cord above the level of instrumentation is rare. We review the clinical history and outcome of a female adolescent who underwent posterior spinal fusion (PSF) for AIS and developed a cervical spine injury 12 hours postoperatively. The patient is a 13-year old female who underwent PSF for AIS from T1 to L1 for progressive scoliosis measuring over 53 degrees in her right thoracic curve. During surgery, she had modest correction with minimal blood loss and with normal intraoperative motor evoked and somatosensory evoked potentials. The immediate postoperative examination was neurologically intact. Twelve hours later, she developed weakness and tingling in her right upper extremity. Magnetic resonance imaging (MRI) of the cervical spine demonstrated myelomalacia on the right side of the spinal cord at the C5-7 levels. Cervical spine injuries are rare following lower-level fusions, however, these injuries can occur and it is important to be vigilant in monitoring patients for these symptoms. The exact mechanism is unknown and may include a combination of postoperative hypotension with altered vascular anatomy from cord stretch and abnormal cervical positioning.