Percutaneous cecostomy: 25-year two institution experience.

BACKGROUND: Reports of technical success, adverse events, and long-term outcome of percutaneous cecostomy in children are limited. OBJECTIVE: To characterize technical success, 30-day severe adverse events, and long-term outcome of percutaneous cecostomy at two centers. MATERIALS AND METHODS: A retrospective review of hospital course and long-term follow-up (through May 2022) of percutaneous cecostomy tubes placed May 1997 to August 2011 at two children's hospitals was used. Outcomes assessed included technical success (defined as successful tube placement into the colon allowing antegrade colonic enemas), length of stay, 30-day severe adverse events, surgery consults, surgical repair, VP shunt infection, ongoing flushes, tube removal, duration between maintenance tube exchanges, and deaths. RESULTS: A total of 215 procedures were performed in 208 patients (90 institution A, 125 institution B). Tubes were placed for neurogenic bowel (72.1%, n = 155) and functional constipation (27.9%, n = 60). Technical success was 98.1% (211/215) and did not differ between centers (p = 0.74). Surgical repair was required for bowel leakage in 5.1% (11/215) and VP shunt infection was managed in 2.1% (2/95). Compared to functional constipation, patients with neurogenic bowel had higher % tube remaining (65.3% [96/147] versus 25.9% [15/58], p < 0.001) and higher ongoing flushes at follow-up (42.2% [62/147] versus 12.1% [7/58], p < 0.001). Tube removal for dissatisfaction occurred in 15.6% [32/205] and did not differ between groups (p = 0.98). Eight deaths due to co-morbidity occurred after a median of 7.4 years (IQR 9.3) of tube access. CONCLUSION: Percutaneous cecostomy is technically successful in the vast majority of patients and provided durable access in most. Bowel leakage and VP shunt infection are uncommon, severe adverse events.

Generation of Terawatt Attosecond Pulses from Relativistic Transition Radiation.

When a femtosecond duration and hundreds of kiloampere peak current electron beam traverses the vacuum and high-density plasma interface, a new process, that we call relativistic transition radiation (RTR), generates an intense ∼100 as pulse containing ∼1 terawatt power of coherent vacuum ultraviolet (VUV) radiation accompanied by several smaller femtosecond duration satellite pulses. This pulse inherits the radial polarization of the incident beam field and has a ring intensity distribution. This RTR is emitted when the beam density is comparable to the plasma density and the spot size much larger than the plasma skin depth. Physically, it arises from the return current or backward relativistic motion of electrons starting just inside the plasma that Doppler up shifts the emitted photons. The number of RTR pulses is determined by the number of groups of plasma electrons that originate at different depths within the first plasma wake period and emit coherently before phase mixing.

Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion.

In a previous limb-girdle muscular dystrophy type 2D (LGMD2D) clinical trial, robust alpha-sarcoglycan gene expression was confirmed following intramuscular gene (SGCA) transfer. This paved the way for first-in-human isolated limb infusion (ILI) gene transfer trial to the lower limbs. Delivery of scAAVrh74.tMCK.hSGCA via an intravascular route through the femoral artery predicted improved ambulation. This method was initially chosen to avoid safety concerns required for large systemic vascular delivery viral loads. ILI methods were adopted from the extensive chemotherapy experience for treatment of malignancies confined to the extremities. Six LGMD2D subjects were enrolled in a dose-ascending open-label clinical trial. Safety of the procedure was initially assessed in the single limb of a non-ambulant affected adult at a dose of 1 × 1012 vg/kg. Subsequently, ambulatory children (aged 8-13 years) were enrolled and dosed bilaterally with either 1 × 1012 vg/kg/limb or 3 × 1012 vg/kg/limb. The six-minute walk test (6MWT) served as the primary clinical outcome; secondary outcomes included muscle strength (maximum voluntary isometric force testing) and SGCA expression at 6 months. All ambulatory participants except one had pre- and post-treatment muscle biopsies. All four subjects biopsied had confirmed SGCA gene delivery by immunofluorescence, Western blot analysis (14-25% of normal), and vector genome copies (5.4 × 103-7.7 × 104 vg/µg). Muscle strength in the knee extensors (assessed by force generation in kilograms) showed improvement in two subjects that correlated with an increase in fiber diameter post gene delivery. Six-minute walk times decreased or remained the same. Vascular delivery of AAVrh74.tMCK.hSGCA was effective at producing SGCA protein at low doses that correlated with vector copies and local functional improvement restricted to targeted muscles. Future trials will focus on systemic administration to enable targeting of proximal muscles to maximize clinical benefit.

Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes.

Sporadic inclusion body myositis, a variant of inflammatory myopathy, has features distinct from polymyositis/dermatomyositis. The disease affects men more than women, most commonly after age 50. Clinical features include weakness of the quadriceps, finger flexors, ankle dorsiflexors, and dysphagia. The distribution of weakness is similar to Becker muscular dystrophy, where we previously reported improvement following intramuscular injection of an isoform of follistatin (FS344) by AAV1. For this clinical trial, rAAV1.CMV.huFS344, 6 × 1011 vg/kg, was delivered to the quadriceps muscles of both legs of six sporadic inclusion body myositis subjects. The primary outcome for this trial was distance traveled for the 6-min walk test. The protocol included an exercise regimen for each participant. Performance, annualized to a median 1-year change, improved +56.0 m/year for treated subjects compared to a decline of -25.8 m/year (p = 0.01) in untreated subjects (n = 8), matched for age, gender, and baseline measures. Four of the six treated subjects showed increases ranging from 58-153 m, whereas two were minimally improved (5-23 m). Treatment effects included decreased fibrosis and improved regeneration. These findings show promise for follistatin gene therapy for mild to moderately affected, ambulatory sporadic inclusion body myositis patients. More advanced disease with discernible muscle loss poses challenges.

A comparison of pediatric gastrostomy tube placement techniques.

PURPOSE: This study aimed to compare different techniques for placement of gastrostomy tubes in a pediatric population. STUDY DESIGN: A retrospective review was performed for patients less than 18 years of age who underwent gastrostomy tube placement at a single academic children's hospital between 2010 and 2012. Techniques for gastrostomy placement included Open Stamm, percutaneous endoscopic gastrostomy (PEG), fluoroscopy guided, laparoscopic, and laparoscopic assisted PEG. Pre-operative characteristics of patients and post-operative outcomes were compared between techniques. RESULTS: Most patients underwent an Open Stamm (43 %) or PEG (39 %). There were significant differences between groups with respect to primary diagnoses, prior surgeries, and ASA classification. Major complications were rare, with less than 3 % requiring reoperation within 30 days; however, minor complications and returns to the emergency department were common. Unintentional tube dislodgements occurred in 22 % of all patients, with Open Stamm technique identified as an independent predictor of unintentional dislodgement (p < 0.0001). CONCLUSIONS: Although conclusions from this retrospective analysis are limited due to heterogeneity between groups, open Stamm gastrostomy placement in children was associated with increased negative outcomes including unintentional tube dislodgements, returns to the emergency department, and need for reoperation within 30 days. Prospective analysis of the various techniques is needed to confirm that minimally invasive techniques for gastrostomy tube placement are associated with a less complicated post-operative course.

A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy.

Becker muscular dystrophy (BMD) is a variant of dystrophin deficiency resulting from DMD gene mutations. Phenotype is variable with loss of ambulation in late teenage or late mid-life years. There is currently no treatment for this condition. In this BMD proof-of-principle clinical trial, a potent myostatin antagonist, follistatin (FS), was used to inhibit the myostatin pathway. Extensive preclinical studies, using adeno-associated virus (AAV) to deliver follistatin, demonstrated an increase in strength. For this trial, we used the alternatively spliced FS344 to avoid potential binding to off target sites. AAV1.CMV.FS344 was delivered to six BMD patients by direct bilateral intramuscular quadriceps injections. Cohort 1 included three subjects receiving 3 × 10(11) vg/kg/leg. The distance walked on the 6MWT was the primary outcome measure. Patients 01 and 02 improved 58 meters (m) and 125 m, respectively. Patient 03 showed no change. In Cohort 2, Patients 05 and 06 received 6 × 10(11) vg/kg/leg with improved 6MWT by 108 m and 29 m, whereas, Patient 04 showed no improvement. No adverse effects were encountered. Histological changes corroborated benefit showing reduced endomysial fibrosis, reduced central nucleation, more normal fiber size distribution with muscle hypertrophy, especially at high dose. The results are encouraging for treatment of dystrophin-deficient muscle diseases.

Development of a pediatric-specific clinical probability tool for diagnosis of venous thromboembolism: a feasibility study.

BACKGROUND: Pediatric venous thromboembolism (VTE) is an increasingly common, difficult to diagnose problem. Clinical probability tools (CPT) for adults estimate VTE likelihood, but are not available for children. We hypothesized that a pediatric-specific CPT is feasible. METHODS: Radiology reports were utilized to identify children imaged for suspected VTE. Relevant signs, symptoms, and comorbidity variables, identified from published literature, were extracted from corresponding medical records. Variables associated with pediatric VTE were incorporated into a multivariate logistic regression to create a pilot CPT which was confirmed on a separate cohort. RESULTS: A total of 389 subjects meeting inclusion criteria were identified: 91 with VTE and 298 without. Univariate analysis revealed male gender (odds ratio (OR) = 2.96; P < 0.001), asymmetric extremity (OR = 1.76; P = 0.033), central venous catheter utilization and/or dysfunction (OR = 2.51; P < 0.001), and cancer (OR = 2.35; P = 0.014) as VTE predictive variables. Documentation of an alternate diagnosis was inversely related to VTE (OR = 0.42; P = 0.004). Receiver operating characteristic analysis of the derived CPT demonstrated reasonable ability to discriminate VTE probability in the training cohort (area under the curve (AUC) = 0.73; P < 0.001) and moderate discrimination in a separate validation cohort of 149 children (AUC = 0.64; P = 0.011). CONCLUSION: A pediatric-specific VTE CPT is feasible, would facilitate early diagnosis, and could lead to improved outcomes.

Percutaneous transcervical thoracic duct embolization for treatment of a cervical lymphocele following anterior spinal fusion: a case report.

Thoracic duct injury is an uncommon complication of neck dissection and cervical spinal surgery that is associated with significant morbidity. The authors describe an unusual case of thoracic duct injury during anterior spinal fusion resulting in a large prevertebral lymphocele presenting with dysphagia, respiratory distress, and chyloptysis. Surgical closure of the lymphocele was unsuccessful, and percutaneous drainage and sclerotherapy was performed. A large thoracic duct branch communicating with the lymphocele became evident during sclerotherapy, and embolization of the duct was performed via a percutaneous transcervical approach. Symptoms immediately resolved, and the patient remained asymptomatic at 6-month follow-up.

Quality improvement guidelines for pediatric abscess and fluid drainage.

Image-guided drainage of abscesses and fluid collections is a valuable tool in the treatment of pediatric patients. It may obviate surgery or optimize the child's clinical condition for subsequent surgery. Compared with adults, several differences exist in terms of etiology, risks (especially radiation exposure), preprocedural imaging and planning, technical considerations, support issues such as sedation, and complications. Knowledge of these differences is important in the planning and treatment of these patients. In addition, a quality improvement plan can be used to assess practice performance.

Safety of pediatric percutaneous liver biopsy performed by interventional radiologists.

OBJECTIVE: National data suggest that pediatric percutaneous liver biopsy is increasingly being performed by interventional radiologists rather than pediatric gastroenterologists. The objective of the present report is to describe the safety and effectiveness of percutaneous liver biopsy performed by interventional radiologists in a large cohort of children and to compare the results with the existing literature on biopsies performed by pediatric gastroenterologists. PATIENTS AND METHODS: The medical records of 249 children undergoing ultrasound-guided percutaneous liver biopsy by interventional radiologists were reviewed for adverse events and success of obtaining tissue. Two hundred ninety-four biopsies were reviewed. RESULTS: There were no deaths. There were 2 instances of a 2-g or greater drop in hemoglobin following biopsy, neither of which was associated with clinical signs of hemorrhage. A small, asymptomatic pneumothorax quickly resolved without treatment. One patient developed Klebsiella sepsis 48  hours after biopsy. In all but 1 case, an adequate sample size was obtained. This low incidence of adverse events compares favorably with existing published reports of morbidity and mortality following percutaneous liver biopsy performed by pediatric gastroenterologists. CONCLUSIONS: Ultrasound-guided percutaneous liver biopsy performed by experienced pediatric interventional radiologists in a children's hospital setting is as safe and effective as biopsy performed by pediatric gastroenterologists.

Infection in pediatric interventional radiology.

Interventional radiology in children involves nearly every aspect of infectious disease. Diagnosis, treatment, prophylaxis and disease transmission in infectious disease are a daily part of pediatric interventional radiology practice. This article will discuss each of these aspects of infection with respect to interventional radiology.

Interventions in the chest in children.

The scope for image-guided intervention in the chest is very wide and encompasses procedures in the heart, pulmonary and systemic vasculature, lungs and pleural cavities, airway, and esophagus. This review describes the most important procedures that are usually performed by radiologists. Percutaneous drainage is now the most common method of treating both empyemas and lung abscesses in children. Although most lung biopsies are carried out by other means, percutaneous biopsy and localization are important alternatives for the diagnosis of focal lung lesions. Esophageal strictures are common in children and are usually best treated by balloon dilatation. The use of retrievable or biodegradable stents has recently been introduced for refractory esophageal strictures. Similarly, balloon dilatation and stenting are now increasingly used in children with stenosis or extrinsic compression of the trachea or bronchi.

Biopsy and drainage techniques in children.

Drainage and biopsy are mainstay procedures in pediatric interventional radiology. As in the adult population, percutaneous biopsy and fluid collection drainage can be performed almost anywhere in the body, in almost all organ systems, and for myriad indications. However, there are some technique differences in children. Radiation protection is paramount, requiring alterations in imaging and guidance. Children have unique sedation and anesthetic requirements, and smaller patients provide both advantages and disadvantages that require/allow for alteration of the procedural techniques. This article will focus on these differences and describe specific techniques applicable to pediatric patients.

Percutaneous treatment of lymphatic malformations.

OBJECTIVE: The aim of this study is to investigate a new treatment regimen for macrocystic and microcystic lymphatic malformations (LM) of the head and neck. STUDY DESIGN: The study represents a retrospective review of outcomes from new percutaneous treatments for lymphatic malformations. SUBJECTS AND METHODS: Thirty-one patients (age range, 2 days to 51 years of age) underwent percutaneous treatment for LM of the head and neck from 2001 to 2007. The LM involved the orbit, ear, parotid gland, face, and neck. Twenty-seven patients underwent primary treatment of LM; 4 were treated for recurrence after operative resection. Macrocysts (>or=1 cm) were treated with dual-drug chemoablation (sequential intracystic sodium tetradecyl sulfate and ethanol); doxycycline was used for microcysts. Macrocysts and microcysts were treated after complete cyst aspiration with sonographic guidance. Fifty-four macrocysts and 125 microcysts were treated. The goal of treatment was complete cyst ablation documented by sonography or MR imaging. RESULTS: Mean number of treatments was 1.7 per patient; mean number of treatments for macrocysts was 1.1; mean treatments for microcysts was 1.7. Ablation efficacy was 179 of 179 (100%) cysts. Effective cyst ablation achieved effective clinical control with resolution of the external mass appearance. Treatments included massive head and neck mixed LM and cysts surrounding the facial nerve and brachial plexus. Infection occurred in 2 (6%) of 31 patients. No patient experienced postprocedural pain, skin necrosis, neuropathy, skin retraction, or myoglobinuria. CONCLUSION: Percutaneous sclerotherapy provides effective treatment for macrocystic and microcystic LM as primary treatment or for recurrence after surgical resection.

Interventional radiology for paediatric trauma.

Paediatric interventional radiology plays a cornerstone role in the management of paediatric trauma. In the acute setting, interventional radiology techniques allow minimally invasive control of haemorrhage or re-establishment of blood flow. Percutaneous stenting and drainage can allow disruptions in urinary or biliary systems to heal without the need for further surgery. Interventional radiology techniques also have a significant role in treating delayed complications of trauma, including embolization of arterial pseudoaneurysms and pulmonary embolism prophylaxis in individuals immobilized due to the trauma or its operative treatment.