RESUMO
OBJECTIVE: In prior open-label studies, eteplirsen, a phosphorodiamidate morpholino oligomer, enabled dystrophin production in Duchenne muscular dystrophy (DMD) with genetic mutations amenable to skipping exon 51. The present study used a double-blind placebo-controlled protocol to test eteplirsen's ability to induce dystrophin production and improve distance walked on the 6-minute walk test (6MWT). METHODS: DMD boys aged 7 to 13 years, with confirmed deletions correctable by skipping exon 51 and ability to walk 200 to 400 m on 6 MWT, were randomized to weekly intravenous infusions of 30 or 50 mg/kg/wk eteplirsen or placebo for 24 weeks (n = 4/group). Placebo patients switched to 30 or 50 mg/kg eteplirsen (n=2/group) at week 25; treatment was open label thereafter. All patients had muscle biopsies at baseline and week 48. Efficacy included dystrophin-positive fibers and distance walked on the 6MWT. RESULTS: At week 24, the 30 mg/kg eteplirsen patients were biopsied, and percentage of dystrophin-positive fibers was increased to 23% of normal; no increases were detected in placebo-treated patients (p≤0.002). Even greater increases occurred at week 48 (52% and 43% in the 30 and 50 mg/kg cohorts, respectively), suggesting that dystrophin increases with longer treatment. Restoration of functional dystrophin was confirmed by detection of sarcoglycans and neuronal nitric oxide synthase at the sarcolemma. Ambulation-evaluable eteplirsen-treated patients experienced a 67.3 m benefit compared to placebo/delayed patients (p≤0.001). INTERPRETATION: Eteplirsen restored dystrophin in the 30 and 50 mg/kg/wk cohorts, and in subsequently treated, placebo-controlled subjects. Duration, more than dose, accounted for dystrophin production, also resulting in ambulation stability. No severe adverse events were encountered.
Assuntos
Músculo Esquelético/patologia , Distrofia Muscular de Duchenne/tratamento farmacológico , Oligonucleotídeos/uso terapêutico , Adolescente , Criança , Método Duplo-Cego , Distrofina/genética , Humanos , Masculino , Morfolinos , Distrofia Muscular de Duchenne/genética , Distrofia Muscular de Duchenne/patologia , Mutação , Resultado do TratamentoRESUMO
BACKGROUND: Forearm fractures are common skeletal injuries in childhood and can usually be treated nonoperatively with closed reduction and casting. Trends toward increasing operative treatment of these fractures have emerged. We aim to demonstrate the safety and efficacy of cast wedging for treatment of pediatric forearm fractures. METHODS: We performed a prospective chart review of patients with forearm fractures, including distal radius (DR) fractures, treated with cast wedging at a single large pediatric hospital from June 2011 to September 2012. Inclusion criteria specified open distal radial physis, closed injury, loss of acceptable reduction, and availability of clinical and radiographic data from injury to cast removal. Exclusion criteria included pathologic fractures, neurovascular injury, fracture dislocations, open fractures, and closed DR physis. Reductions were performed and patients followed according to standard protocol at our institution, including placement into long-arm casts, initial follow-up visit within 5 to 10 days postinjury, and weekly visits for 2 weeks thereafter. If alignment were deemed unacceptable within 3 weeks of injury, cast wedging was utilized. Radiographic measurements of alignment included both radius and ulna on the injury film, postreduction, prewedge, postwedge, and final films. Radiographic technique was standardized, with repeatability testing demonstrating a precision of ±2 degrees. RESULTS: Over 15 months, our hospital treated 2124 forearm or DR fractures with closed reduction and casting. There were 60 fractures treated either with percutaneous fixation (36) or open treatment (24). A total of 79 forearm or DR fractures were treated with cast wedging secondary to loss of reduction, of which 70 patients had complete clinical and radiographic data. Average age was 8.4 years (range, 3 to 14 y), with 25 females and 45 males. Significant improvement in angulation for both-bone forearm fracture from prewedge to final films was seen in 69 children, with no major complications. One patient failed wedging and required surgical reduction and fixation. CONCLUSIONS: Cast wedging is a simple, safe, noninvasive, and effective method for treatment of excessive angulation in pediatric forearm fractures. LEVEL OF EVIDENCE: Level IV.
Assuntos
Moldes Cirúrgicos/estatística & dados numéricos , Pediatria/tendências , Fraturas do Rádio/diagnóstico por imagem , Fraturas da Ulna/diagnóstico por imagem , Adolescente , Moldes Cirúrgicos/normas , Criança , Pré-Escolar , Feminino , Seguimentos , Traumatismos do Antebraço/diagnóstico por imagem , Traumatismos do Antebraço/cirurgia , Humanos , Masculino , Estudos Prospectivos , Radiografia , Fraturas do Rádio/cirurgia , Resultado do Tratamento , Fraturas da Ulna/cirurgiaRESUMO
PURPOSE OF THE STUDY: To evaluate the rate of infection after minimally invasive procedures on a consecutive series of pediatric orthopaedic patients. We hypothesized that the use of preoperative antibiotics for minimally invasive pediatric orthopaedic procedures does not significantly reduce the incidence of surgical site infection requiring surgical debridement within 30 days of the primary procedure. METHODS: We retrospectively reviewed 2330 patients having undergone minimally invasive orthopaedic procedures at our institution between March 2008 and November 2010. Knee arthroscopy, closed reduction with percutaneous fixation, soft tissue releases, excision of bony or soft-tissue masses, and removal of hardware constituted the vast majority of included procedures. Two groups, based on whether prophylactic antibiotics were administered before surgery, were created and the incidence of a repeat procedure required for deep infection was recorded. Statistical analysis was performed to determine significance, if any, between the 2 groups. RESULTS: Chart review of the 2330 patients identified 1087 as having received preoperative antibiotics, whereas the remaining 1243 patients did not receive antibiotics before surgery. Only 1 patient out of the 1243 cases in which antibiotics were not given required additional surgery within 30 days of the primary procedure due to a complicated surgical site infection (an incidence of 0.0008%). No patients in the antibiotic group developed a postoperative infection within 30 days requiring a return to the operating room for management. Our data revealed no significant increase in the incidence of complicated infection requiring additional procedures when antibiotics were not administered before surgery. DISCUSSION: Though prophylactic antibiotics have been shown to confer numerous benefits for patients undergoing relatively major operations, their use in cases of minimally invasive and/or percutaneous orthopaedic surgery is not well defined. Our data suggest that the use of prophylactic antibiotics may not be indicated for many less invasive procedures when performed in a low-risk pediatric population. Future studies are warranted to help establish evidence-based guidelines regarding the routine use of prophylactic antibiotics in this specific population, hopefully resulting in improved cost-effectiveness and safety while slowing the emergence of new drug-resistant organisms. LEVEL OF EVIDENCE: Level III, retrospective comparative.
Assuntos
Antibacterianos/administração & dosagem , Procedimentos Ortopédicos/métodos , Cuidados Pré-Operatórios/métodos , Infecção da Ferida Cirúrgica/prevenção & controle , Antibacterianos/uso terapêutico , Antibioticoprofilaxia/métodos , Pré-Escolar , Desbridamento/métodos , Feminino , Seguimentos , Humanos , Incidência , Masculino , Procedimentos Cirúrgicos Minimamente Invasivos/métodos , Estudos Retrospectivos , Infecção da Ferida Cirúrgica/epidemiologia , Fatores de TempoRESUMO
BACKGROUND: Deep wound infection in patients that have had a posterior spinal fusion for scoliosis is a major complication in pediatric spine surgery. PURPOSE: To explore characteristics of pediatric patients who had a posterior spinal fusion with segmental spinal instrumentation and bone graft and subsequently developed deep spinal wound infections that were treated with wound vacuum-assisted closure (V.A.C.) therapy METHOD: Retrospective Chart Review. Characteristics of patients' age, gender, comorbid illnesses, identified bacteria, antibiotics, time on V.A.C. device, infection recurrence, any instrumentation removal, and additional surgical intervention was collected. FINDINGS: There were 249 patients who had spinal fusions from December 2002 through January 2006, and 11 developed an infection after their spinal fusion and had the use of the V.A.C. device. Those who developed infection within 1 year of their fusion had instrument retention. Wound closure occurred in all cases that used V.A.C. therapy.
Assuntos
Escoliose/cirurgia , Coluna Vertebral/patologia , Infecção da Ferida Cirúrgica/terapia , Vácuo , Adolescente , Criança , Educação Continuada , Feminino , Humanos , Masculino , Auditoria Médica , Estudos RetrospectivosRESUMO
OBJECTIVE: Over the past decade, our institution has instituted three different scheduling models in an attempt to care for pediatric trauma at our Level I Trauma Center. This has been in response to a number of factors, including a limited number of physicians covering the call schedule, increasing competition for operating room (OR) time after hours (pediatric surgery, urology, neurosurgery), an attempt to fully utilize OR time during the daytime, fully staffed hours, and optimizing patients' timeliness to surgery. We examined the three on-call systems in place at our institution to determine whether a more flexible approach to pediatric trauma call resulted in delays in treatment. METHODS: We retrospectively reviewed patient records for three distinct 1-year periods with three different surgical call schedules: (i) a traditional call schedule in which the call physician was responsible for patients who presented to our emergency room; (ii) a half-day trauma block OR reserved the morning following call; and (iii) a full-day trauma block. Variables included date of injury, time of admission, admission diagnosis, cause of injury, and OR procedure and start time. RESULTS: We reviewed 951 cases over the entire study, 268 during the traditional call schedule, 282 during the half-call block and 401 over the time period of the full-day block. Mechanisms of injury were similar among the three groups, with falls and motor vehicle accidents being the leading causes. The average delay time was 17:40 for the traditional call group, 15:10 for the half-block call group, and 15:09 for the full-day block group. Our findings suggest that there was a high incidence of cases performed on weekdays after peak staffing hours with a traditional call model (59%). In contrast, half-day and full-day block models saw only 4% and 1% of the cases performed after peak staffing hours, respectively. There was a statistically significant difference in the number of patients admitted to the OR among the three groups (χ(2) = 488.8449, P < 0.0001). The number of patients seen during Monday through Friday was also statistically significant among the three groups (χ(2) = 382.0576, P < 0.0001). CONCLUSIONS: The institution of more flexible and physician-directed half-call and full-day blocks did result in delays in treatment. However, it also has demonstrated benefits to patients in reducing the number of operative cases performed after weekday peak staffing hours; helped our institution better manage its staffing and financial resources; and provided the treating surgeon flexibility in determining the timing of operative care.
Assuntos
Serviços de Saúde da Criança/organização & administração , Modelos Organizacionais , Ortopedia/organização & administração , Centros de Traumatologia/organização & administração , Ferimentos e Lesões/cirurgia , Criança , Humanos , Ohio , Salas Cirúrgicas/organização & administração , Duração da Cirurgia , Procedimentos Ortopédicos , Admissão e Escalonamento de Pessoal/organização & administração , Estudos RetrospectivosRESUMO
PURPOSE: Controversy exists regarding approach to treatment of pediatric patients with fibrous dysplasia. METHODS: We retrospectively reviewed medical records of seven patients who were treated at our institution for fibrous dysplasia by intramedullary rod fixation without bisphosphonate supplementation. RESULTS: Seven patients with a total of ten fibrous dysplasia lesion sites surgically treated by intramedullary rod fixation were included. Of these ten lesion sites, eight demonstrated pathologic fracture at the time of fixation. Complete fracture healing was observed in all eight sites, with no incidence of recurrent pathologic fractures examined radiographically. There were no major infections or neurologic deficits, and lesions appeared to stabilize. CONCLUSIONS: In this series, intramedullary rod fixation proved to be successful in treatment of acute pathologic fracture and incompletely healed fibrous dysplasia lesions. We observed partial resolution of fibrous dysplasia lesions at all ten sites without significant long-term complications. Following treatment, there were no refractures. LEVEL OF EVIDENCE: Level IV, case series.
Assuntos
Artroplastia de Quadril , Desigualdade de Membros Inferiores/etiologia , Artroplastia de Quadril/efeitos adversos , Artroplastia de Quadril/métodos , Colo do Fêmur/cirurgia , Humanos , Desigualdade de Membros Inferiores/diagnóstico por imagem , Desigualdade de Membros Inferiores/prevenção & controle , Osteoartrite do Quadril/cirurgia , Osteotomia , Complicações Pós-Operatórias/diagnóstico por imagem , Complicações Pós-Operatórias/prevenção & controle , Radiografia , Resultado do TratamentoRESUMO
The treatment of foot problems in the adolescent should be nonsurgical whenever possible. Surgical treatment should be considered when medical treatment does not relieve pain. Surgical corrections to prevent problems in adulthood may cause complications and lead to an undesirable outcome. This article highlights treatment for overriding fifth toe, juvenile bunion, Morton toe, Freiberg infraction, stress fractures of metatarsals, accessory navicular, tarsal coalition, plantar fascitis, pes cavus, and pes planus. Significant deformities generally require treatment before ambulation, and innocent deformities usually resolve spontaneously.