Percutaneous core needle biopsy of neuroblastoma in the pediatric population: what have we learned in the last decade.

INTRODUCTION: Historically, neuroblastoma has been diagnosed by surgical open biopsy (SB). In recent decades, core needle biopsy (CNB) has replaced surgical biopsy due to its safe and adequate method of obtaining tissue diagnosis. AIM: Our study aimed to assess the effectiveness of CNB in obtaining tissue diagnosis for neuroblastoma and evaluate its safety profile in terms of post-operative complications, in comparison to SB. METHODS: A retrospective cohort study, including all patients younger than 18 years who were diagnosed with neuroblastoma from 2012 until 2022 in a single tertiary medical center. Patients' demographics, tumor size and location, pathological results, and clinical outcomes were collected. RESULTS: 79 patients were included in our study: 35 biopsies were obtained using image-guided CNB and 44 using SB. Patients' and tumor characteristics including age, gender, tumor volume, and stage were similar in both groups. The biopsy adequacy rate in the CNB group was 91% and 3 patients in this group underwent repeated biopsy. The safety profile in the CNB group was similar to the SB group. CONCLUSIONS: CNB is a safe method and should be considered the first choice for obtaining tissue diagnosis when feasible due to its high adequacy in terms of tumor histopathological features.

Muscle microRNAs in the cerebrospinal fluid predict clinical response to nusinersen therapy in type II and type III spinal muscular atrophy patients.

BACKGROUND AND PURPOSE: The antisense oligonucleotide nusinersen (Spinraza) regulates splicing of the survival motor neuron 2 (SMN2) messenger RNA to increase SMN protein expression. Nusinersen has improved ventilator-free survival and motor function outcomes in infantile onset forms of spinal muscular atrophy (SMA), treated early in the course of the disease. However, the response in later onset forms of SMA is highly variable and dependent on symptom severity and disease duration at treatment initiation. Therefore, we aimed to identify novel noninvasive biomarkers that could predict the response to nusinersen in type II and III SMA patients. METHODS: Thirty-four SMA patients were included. We applied next generation sequencing to identify microRNAs in the cerebrospinal fluid (CSF) as candidate biomarkers predicting response to nusinersen. Hammersmith Functional Motor Scale Expanded (HFMSE) was conducted at baseline and 6 months after initiation of nusinersen therapy to assess motor function. Patients changing by ≥3 or ≤0 points in the HFMSE total score were considered to be responders or nonresponders, respectively. RESULTS: Lower baseline levels of two muscle microRNAs (miR-206 and miR-133a-3p), alone or in combination, predicted the clinical response to nusinersen after 6 months of therapy. Moreover, miR-206 levels were inversely correlated with the HFMSE score. CONCLUSIONS: Lower miR-206 and miR-133a-3p in the CSF predict more robust clinical response to nusinersen treatment in later onset SMA patients. These novel findings have high clinical relevance for identifying early treatment response to nusinersen in later onset SMA patients and call for testing the ability of miRNAs to predict more sustained long-term benefit.

Transthoracic intracardiac lines-A double edged sword.

OBJECTIVE: Routine use of central venous access is needed in children undergoing open heart surgery for pressure monitoring and inotrope infusion. We sought to evaluate the efficiency and safety of routine use of transthoracic intracardiac lines (ICLs) in patients undergoing cardiac surgery and to compare them to patients who have been previously treated with traditional central venous lines (non-ICLs). METHODS: Retrospective review of charts of all patients who underwent cardiac surgery and had an ICL inserted in the operating room. Case control matching was done with similar patient in which ICL was not inserted. Patients characteristics, diagnosis, operative, and intensive care data were collected for each patient and analyzed. RESULTS: A total number of 376 patient records were reviewed (198 ICL patients and 178 non-ICL patients). Umbilical line and non-ICL durations were longer in the non-ICL group. ICL duration was the longest of all lines, averaging 12.87 ± 10.82 days. The necessity for multiple line insertions (˃2 insertions) was significantly higher in the non-ICL group, with a relative risk ratio of 3.24 (95% confidence interval: 1.617-6.428). There was no statistical difference of infections rate and line complications between the two groups. CONCLUSION: ICLs are safe in infants undergoing cardiac surgery and can be kept in place for a long period of time with a low rate of line complications and infection. Routine use of ICLs reduces the number of central venous catheter placement in this complex patient population.

Clinical Features and Comparison of Kingella and Non-Kingella Endocarditis in Children, Israel.

Kingella spp. have emerged as an important cause of invasive pediatric diseases. Data on Kingella infective endocarditis (KIE) in children are scarce. We compared the clinical features of pediatric KIE cases with those of Streptococcus species IE (StIE) and Staphylococcus aureus IE (SaIE). A total of 60 patients were included in the study. Throughout the study period, a rise in incidence of KIE was noted. KIE patients were significantly younger than those with StIE and SaIE, were predominately boys, and had higher temperature at admission, history of oral aphthae before IE diagnosis, and higher lymphocyte count (p<0.05). Pediatric KIE exhibits unique features compared with StIE and SaIE. Therefore, in young healthy children <36 months of age, especially boys, with or without a congenital heart defect, with a recent history of oral aphthae, and experiencing signs and symptoms compatible with endocarditis, Kingella should be suspected as the causative pathogen.

Fetal-TAPSE for Surveillance of Cardiac Function in Growth-Restricted Fetuses With a Portosystemic Shunt.

OBJECTIVES: Congenital portosystemic shunt (CPSS) in a growth-restricted fetus may lead to cardiac overload and ultimately hemodynamic imbalance. The aim of the study was to describe the application of tricuspid annular plane systolic excursion (TAPSE) for surveillance of cardiac function in growth-restricted fetuses diagnosed with CPSS. METHODS: The study group consisted of 7 fetuses with growth restriction diagnosed with CPSS between 2018 and 2020. Patients were followed longitudinally every 2 weeks. Sonographic fetal-TAPSE (f-TAPSE) was performed every 2 weeks. At each visit, the following parameters were recorded: estimated fetal weight, biophysical profile, nonstress test, Doppler flow indices, and fetal cardiothoracic index. Postnatal laboratory and imaging tests were retrieved from the medical files. RESULTS: Mean gestational age at diagnosis of CPSS was 32 + 1 weeks. Cardiomegaly was observed in all cases. All portosystemic shunts were classified as intrahepatic. Values of f-TAPSE were above the 95th percentile in 6/7 fetuses at presentation and throughout follow-up. Gestational age at delivery ranged between 36 + 5 and 38 + 5 weeks. Postnatally, spontaneous closure of the shunt was noted in 2 infants. Transient hyperammonemia was diagnosed in 2 neonates, with no signs of the characteristic complication. CONCLUSIONS: In growth-restricted fetuses diagnosed concomitantly with CPSS, f-TAPSE offers a practical sonographic tool for assessment of cardiac function and may serve as an additional clinical marker for follow-up. The appearance of cardiomegaly in growth-restricted fetuses should prompt a dedicated sonographic evaluation of the fetal portal system.

Mid-term results of vascular ring surgery.

BACKGROUND: Vascular rings cause respiratory symptoms in children. Treatment consists of surgical division; however, data regarding mid-term results are scarce. The purpose of this study was to evaluate clinical results of vascular ring surgery. METHODS: Retrospective chart review of consecutive patients who underwent vascular ring surgery. Mid-term follow-up consisted of clinic visits and telephone questionnaire over a 1-year period sampling at five points in time. RESULTS: Follow-up of 85 patients who underwent vascular rings surgery revealed significant symptomatic improvement within 6 months. In total, 50% were symptomatic to some degree at their last documented clinic visit complaining of stridor (36.8%), cough (34.2%), wheezing (10.5%), dyspnoea on exertion (10.5%), or recurrent respiratory infections (23.7%). By telephone questionnaires, 83% described a significant improvement in respiratory symptoms, 63.4% had some residual symptoms, 36.6% stridor, 38.8% chronic cough, 32.4% bronchodilator use,18.3% had at least one episode of pneumonia per year, 31% of children had dyspnoea or respiratory limitation, and 12.7% while doing physical activities. We found no association between the age at surgery or anatomic variant to the presence of symptoms at mid-term follow-up. CONCLUSIONS: Surgical division of vascular rings results in significant clinical improvement within 1 year; nevertheless, some patients remain symptomatic to some degree. We found no association between the age at surgery or anatomic variant to the presence of symptoms at mid-term follow-up. Further evaluation is warranted to assess the nature of residual symptoms and explore whether anatomical causes can be identified leading to surgical modifications.

Urgent Surgical Treatment of Aortic Endocarditis in Infants and Children.

Infective endocarditis (IE) in the pediatric population can present as a life-threatening condition. Optimal timing for surgical intervention should consider surgical risks versus the risk of neurologic complications. We herein report our experience with this group of critically ill children. Retrospective analysis of patient charts of all patients who underwent urgent surgical treatment of aortic IE from 1994 to 2014 was performed. Nine patients with acute storming IE of the aortic valve or the ascending aorta were urgently operated (eight normal heart, one congenital aortic stenosis), age ranged from 8 weeks to 4.2 years. Causative organisms were Staphylococcus aureus (2), Staphylococcus coagulase negative (1), Kingella kingae (2), Streptococcus pneumoniae (2), or culture negative (2). Presenting symptoms other than hemodynamic instability were neurologic decompensation (5) coronary embolization (1) and cardiogenic shock due to scalded skin syndrome (1). CT and MRI demonstrated significant brain infarcts in four patients. Operations performed were the Ross operation (7) and ascending aortic reconstruction (2). There were no operative deaths. At a median follow-up of 6 years (range 2-196 months), all patients are alive and well. Re-intervention included homograft replacement (2) and transcatheter Melody valve implantation (1). At their last follow-up, the neo-aortic valve was functional in all patients with minimal regurgitation and all had full resolution of the neurological deficits. Urgent surgical treatment for aortic valve IE in infants is challenging. Although surgery is complex and pre-disposing conditions such as sepsis, neurologic and cardiac decompensations are prevalent, operative results are excellent and gradual and significant neurologic improvement was noted over time.

Doxorubicin-Eluting Intra-Arterial Therapy for Pediatric Extra-Abdominal Desmoid Fibromatoses: A Promising Approach for a Perplexing Disease.

Systemic doxorubicin is effective for desmoid fibromatosis (DF), but its use is limited by dose-dependent cardiotoxicity. A protocol of selective intra-arterial doxorubicin drug-eluting embolization (DEE) was designed to maximize target tissue efficacy of doxorubicin, while minimizing systemic exposure. Four children with recurrent or refractory DF were treated between 2014 and 2017. Tumor volumes were reduced by 54%-97% over a follow-up interval of 6-32 months. A single patient experienced transient lower extremity paresthesia (Common Terminology Criteria for Adverse Events grade I). Further investigation is needed to better establish these promising results for doxorubicin DEE in DF treatment.

Neonatal Cardiac Surgery in the New Era: Lessons learned from 1000 Consecutive Cases.

BACKGROUND: neonatal cardiac surgery has evolved over the last 50 years with a large percentage of the patients achieving complete physiological repair in the neonatal period. The remaining patients achieve staged palliation with an increasing amount of success. OBJECTIVES: To report our experience with 1000 neonatal cardiac surgical procedures performed in the last 10 years. METHODS: We conducted a retrospective analysis of surgical outcome in all neonatal patients who underwent cardiac surgery between January 2007 and July 2016 at Schneider Children's Medical Center of Israel. RESULTS: A total of 1003 neonates aged < 30 days underwent surgery for congenital heart defects at our center. Neonatal surgery accounted for 22.5% of all cardiac surgeries. Neonatal operative mortality was 7.3%, Operative mortality for individual lesions were: simple aortic coarctation (CoA) (198 patients, 2.5%), CoA with hypoplastic arch (24, 4%), CoA with ventricular septal defect (VSD) (84, 2.3%), transposition of the great arteries (TGA, simple and complex, 185, 6.3%), TGA with VSD (37, 0%), truncus arteriosus (26, 3.8%), interrupted aortic arch (25, 4%), Norwood Sano (71, 19.7%), neonatal tetralogy of Fallot (41, 0%), and shunt (131 patients, 12%). CONCLUSIONS: Neonatal surgical capabilities have improved substantially over the last decades. Excellent results can be expected for lesions that can be repaired to create biventricular circulation. Improved results can be attributed in part to the evolution of surgical strategies and assistive technologies, but essential is the collaborative effort of surgeons, cardiologists, anesthesiologists, and intensive care specialists acting as a cohesive team whose performance far exceeds the sum of its individual members' contributions.

The Amplatzer Duct Occluder II Additional Sizes device for transcatheter PDA closure: initial experience.

OBJECTIVES: To present our initial experience with the Amplatzer Duct Occluder II Additional Sizes (ADOIIAS) device. BACKGROUND: The ADOIIAS is a modified PDA closure device of various lengths and widths with small disks to avoid flow disturbance in the pulmonary arteries and descending aorta. PATIENT POPULATION: All patients who underwent attempted closure with an ADOIIAS device at our institution CATHETERIZATION: Following aortography the PDA was closed using a 4-5Fr delivery system from the pulmonary or aortic side with an ADOIIAS. Aortography to confirm position and leak was performed before and after device release. Echocardiography was performed before discharge the following day. RESULTS: Between June 2011 and December 2012, 60 patients [33 female], median age 3.3 yrs [0.6-15.8 years], and weight 14.5 kg [4-79] underwent attempted PDA closure with an ADOIIAS device. 56/60 [93.3%] ADOIIAS devices were successfully deployed [52 aortic side]. In 55/56 [98.2%], the PDA was closed on follow up echocardiogram usually on the next day. In one case, there was a small residual PDA of no hemodynamic significance on follow up. In four cases, the ADOIIAS was unstable, including one device embolization which was retrieved with no sequelae, and the PDA was closed in all cases with an alternative device in the same procedure. The mean ratio of ADOIIAS height:PDA pulmonary diameter was 2.4 ± 0.5:1. CONCLUSIONS: We report a large single-center experience with the new ADOIIAS device which proved to be versatile, safe, effective, and easy to use in the appropriate ductal anatomy.

Percutaneous intraperitoneal catheters in neonates following open heart surgery.

OBJECTIVE: Ascitic fluid in the peritoneal cavity may severely impair respiratory and renal function following neonatal heart surgery. It has been our practice to liberally insert percutaneous peritoneal catheters (PPCs) in order to manage fluid balance and thereby improve ventilatory function. We herein report our experience with PPC. METHODS: Retrospective analysis of charts of all surviving neonates that underwent PPC insertion from January 2007 through March 2010. Charts were reviewed for demographic and clinical variables from the preoperative, operative, and postoperative periods. RESULTS: A total of 1268 patients underwent surgery, 292 (23%) were neonates. 17 (5.8%) patients required PPC. Mean age and weight were 16 days and 3.1 kg, respectively. Mean amount drained upon insertion was 55 ± 46 ml. Catheters were maintained for a mean of 5 days and drained an average of 201 ml on the first postinsertion day. Ventilatory settings did not change significantly prior to and postcatheter insertion (respiratory rate [29 ± 3.8 vs 28.7 ± 3.9; P = .93], inspiratory pressures [26.3 ± 3.6 vs 26.1 ± 3.3 cm H2O; P = .34], and fraction of inspired oxygen [0.66 ± 0.21 vs 0.63 ± 0.18; P = .53]). Carbon dioxide values decreased significantly (43.2 ± 9.7 vs 37 ± 4.9 mm Hg; P = .01), and PO2 values increased (78 ± 69 vs 104 ± 57 mm Hg; P = .05). CONCLUSIONS: The PPC insertion can be easily performed at the bedside with minimal complications. Fluid balance management is facilitated, and ventilation is improved. The PPC insertion is a valuable addition to the armamentarium of the physician treating neonates in the intensive care unit after complex congenital heart surgery.

Efficacy of bronchoscopic closure of a bronchopleural fistula with amplatzer devices: long-term follow-up.

BACKGROUND: The development of a bronchopleural fistula (BPF) is associated with high rates of morbidity and mortality. We have developed a minimally invasive method of bronchoscopic BPF closure using Amplatzer devices (AD) and Amplatzer vascular plugs (AVP), with excellent short-term results. OBJECTIVES: The aim of the present report was to explore the long-term outcome of patients treated by Amplatzer occluders and the durability of this novel modality of BPF treatment. METHODS: A total of 31 central BPF in 31 patients (mean age 66.8 years, range 19-91) were sealed under moderate sedation bronchoscopically by either AD (n = 19) or AVP (n = 12). The average follow-up period was 17.6 months (range 1-68 months). RESULTS: The main etiology for BPF was surgery (n = 24), pneumonectomy (n = 14) or lobectomy/segmentectomy (n = 10). The underlying disease was either primary (n = 19) or metastatic (n = 2) lung cancer. The immediate success rate was 96% as symptoms related to BPF disappeared in 30 of the 31 patients. Short-term (<30 days) mortality was 13% (4 patients). At follow-up, 14 patients (45%) are still alive. Out of 12 patients with late mortality, in 5 patients (41%) the death was directly related to cancer relapse, and no patient died due to BPF recurrence. CONCLUSION: Endobronchial closure of BPF using both types of Amplatzer occluders (AD and AVP) is a minimally invasive effective modality of treatment with high safety profile and satisfactory long-term outcome considering the poor prognosis in this particular group of patients.

Extensive Hydrocelectomy Using the Harmonic Scalpel Supersedes Classic Techniques.

OBJECTIVE: To present our technique using the harmonic scalpel for scrotal, extensive hydrocelectomy in adolescents and its advantages, compared with the historical techniques. Scrotal hydrocelectomy is an old-timer procedure in-which the Lord plication, the Jaboulay and the bottleneck techniques are the standard of care, up to date. A cumbersome, unpleasant scrotal dressing is commonly used to try to mitigate postoperative edema and hematoma.1 The harmonic scalpel denaturates proteins by ultrasonic vibrations, and provides advantages over conventional electrocautery with respect to bleeding, drain volume, postoperative pain, and the return to normal activity.2-5 MATERIALS: Adolescents with a large hydrocele, and no inguinal hernia were selected for this operation. All tissue layers except the skin were divided by harmonic scalpel Focus (Eticon). RESULTS: Between 2017 and 2023, 43 adolescents at a median age of 14.3years (range 12-18years), were operated with this technique, using the harmonic scalpel, by a single surgeon. Median operative time was 23 minutes (range 18-35 minutes). No scrotal dressing was used. One patient had a postoperative seroma that was drained spontaneously. CONCLUSION: The described technique is simple and time-saving, with no postoperative major edema nor hematoma in this series. The only disadvantage is the higher cost of the harmonic scalpel, that may be offset by a short time of recovery. Following our experience, we no longer use other techniques for this surgery.

Three-Patch Reconstruction of Hourglass Supravalvar Pulmonary Artery Stenosis Mid-Term Results.

BACKGROUND: Some patients with pulmonary stenosis present with a complex multilevel disease that involves the valve, the leaflets, and the sinotubular junction (STJ) forming an hourglass appearance. We herein report the mid-term results of our experience with the reconstruction of the supravalvar narrowing using three pericardial patches. MATERIAL AND METHODS: Retrospective analysis of patient charts and echocardiography studies of patients who underwent three-patch reconstruction of the pulmonary valve (PV) from 2013 to 2022. After PV transection distal to STJ, vertical incisions into the sinuses were performed, and leaflets were trimmed and thinned. The three sinuses were augmented using three pericardial patches. RESULTS: Nineteen patients underwent repair of hourglass supravalvar pulmonary stenosis. Mean weight at surgery was 9.4 kg (median 7.2, range 4.7-35); 16 patients underwent previous catheterization with unsuccessful balloon dilatation of the PV (13 pts.). Preoperative aortic/pulmonary annulus ratio was 1.02 (median 1, range 0.89-1.25). After surgery, gradients across the PV were significantly reduced (94 ± 26 vs 29 ± 9 mm Hg, P = .02). Postoperatively, 14 patients had mild or no pulmonary insufficiency (PI) and five had mild to moderate PI. At a mean follow-up of 71 months (median 78 months, range 8-137), gradients continued to decrease (29 ± 9 vs 15 ± 5 mm Hg, P < .001). CONCLUSION: The three-patch technique for the repair of supravalvar pulmonary stenosis is simple, reproducible, and achieves excellent and long-standing relief of the right ventricular outflow tract gradient.

Preliminary experience using the transcatheter mitral valve leaflet repair procedure.

BACKGROUND: Mitral regurgitation (MR) causes increased morbidity and mortality in heart failure patients and is often associated with augmented surgical risk. OBJECTIVES: To assess the preliminary results oftranscatheter mitral valve leaflet repair (TMLR) in a single academic center. METHODS: Data were collected prospectively in the cardiology department of Rabin Medical Center in 2012. Ten consecutive patients (age 69.3 +/- 15.9 years, ejection fraction 36.5 +/- 9.4) who were poor surgical candidates with severe functional MR underwent general anesthesia, followed by trans-septal puncture and a TMLR procedure using the MitraClip device. RESULTS: All 10 patients were considered to have severe functional MR prior to TMLR treatment and were all symptomatic; the mean New York Heart Association (NYHA) class was 3.4 +/- 0.5. The MR severity was 4 +/- 0. There were no immediate complications or failures of the procedure. One patient died on day 5 due to massive gastrointestinal bleeding. Immediately following TMLR all 10 patients showed a profound MR reduction to a mean severity grade of 1.6 +/- 0.6. At one month after the procedure, NYHA had decreased to an average of 1.7 +/- 1.0 and was at least grade 2 in all but one patient. After 6 months the MR remained < or = 2 in six of eight patients, with a NYHA average of 1.4 +/- 0.5. CONCLUSIONS: The MitraClip procedure was shown to be relatively safe, providing significant clinical benefit to a relatively sick population with severe MR. It is therefore an important alternative to surgery in these high risk patients.

Amplatzer Occluders for Effective Nonsurgical Management of Bronchopleural Fistulae.

BACKGROUND: To assess the safety and efficacy of bronchopleural fistulae closure with Amplatzer occluder devices (AGA Medical, Golden Valley, MN) through our experience of over 14 years. METHODS: Retrospective data review of patients from Rabin Medical Center who underwent Amplatzer occluder device placement between March 2007 and September 2021 for bronchopleural fistulae closure. RESULTS: In total, 72 patients had 83 Amplatzer occluder devices implanted for bronchopleural fistulae closure. The median age was 65.5 (interquartile range 56.0-72.3) years. The primary diseases were lung malignancy (48 [66.7%]) and thoracic infection (9 [12.5%]). Bronchopleural fistulae developed mainly following pneumonectomy (40.3%) and lobectomy (33.3%), with a median time from surgery to Amplatzer placement of 3.9 (interquartile range 1.4-16.4) months. We encountered no procedural or immediate postprocedural complications or deaths. Six months after Amplatzer insertion, there were 7 (8.4%) Amplatzer removals and 11 (15.3%) fistula-related deaths. CONCLUSIONS: Amplatzer occluders are a safe modality for nonsurgical bronchopleural fistulae management with ease of placement under moderate sedation and flexible bronchoscopy with good short- and long-term effectivity.

The Amplatzer duct occluder (ADOII) and Piccolo devices for patent ductus arteriosus closure: a large single institution series.

Purpose: Evaluate Piccolo and ADOII devices for transcatheter patent ductus arteriosus (PDA) closure. Piccolo has smaller retention discs reducing risk of flow disturbance but residual leak and embolization risk may increase. Methods: Retrospective review of all patients undergoing PDA closure with an Amplatzer device between January 2008 and April 2022 in our institution. Data from the procedure and 6 months follow-up were collected. Results: 762 patients, median age 2.6 years (range 0-46.7) years and median weight 13 kg (range 3.5-92) were referred for PDA closure. Overall, 758 (99.5%) had successful implantation: 296 (38.8%) with ADOII, 418 (54.8%) with Piccolo, and 44 (5.8%) with AVPII. The ADOII patients were smaller than the Piccolo patients (15.8 vs. 20.5 kg, p < 0.001) and with larger PDA diameters (2.3 vs. 1.9 mm, p < 0.001). Mean device diameter was similar for both groups. Closure rate at follow-up was similar for all devices ADOII 295/296 (99.6%), Piccolo 417/418 (99.7%), and AVPII 44/44 (100%). Four intraprocedural embolizations occurred during the study time period: two ADOII and two Piccolo. Following retrieval the PDA was closed with an AVPII in two cases, ADOI in one case and with surgery in the fourth case. Mild stenosis of the left pulmonary artery (LPA) occurred in three patients with ADOII devices (1%) and one patient with Piccolo device (0.2%). Severe LPA stenosis occurred in one patient with ADOII (0.3%) and one with AVPII device (2.2%). Conclusions: ADOII and Piccolo are safe and effective for PDA closure with a tendency to less LPA stenosis with Piccolo. There were no cases of aortic coarctation related to a PDA device in this study.

Selective Intra-Arterial Doxorubicin Eluting Microsphere Embolization for Desmoid Fibromatosis: A Combined Prospective and Retrospective Study.

Desmoid fibromatoses (DFs) are locally aggressive tumors composed of monoclonal fibroblasts within an abundant extracellular matrix. Systemic doxorubicin treatment is effective, but toxic. We investigated arterial doxorubicin eluting embolization (DEE), an approach characterized by high drug concentrations in the tumor alongside limited systemic drug exposure. The primary and secondary endpoints were radiological response using MRI and RECIST 1.1, respectively. The study included 24 patients (median age, 24; interquartile range, 16-34 years). Data were collected prospectively for 9 patients and retrospectively for 15 patients. The most frequent tumor locations were chest/abdomen wall and neck/shoulder/axilla (29% each). Of 24 patients, 7 (24%) were treatment naïve, and 17 (71%) had received one or two prior treatments. Patients underwent a median of two treatments (range, 1-4), with a median of 49 mg (range, 8-75) doxorubicin/treatment. Efficacy outcomes were available for 23 patients. With a median follow-up of 8 months (interquartile range, 3-13), median tumor volumes decreased by 59% (interquartile range, 40-71%) and T2 signal intensity decreased by 36% (interquartile range, 19-55%). Of 23 patients, 9 (39%), 12 (52%), and 2 (9%) had a partial response, stable disease, and progressive disease, respectively. DEE was safe and well tolerated, with one reported grade 3-4 adverse event (cord injury). In conclusion, DEE was safe and achieved rapid clinical/volumetric responses in DFs.

Complex Catheter-Based Structural Heart Reconstruction in a Patient With Tricuspid Atresia and Björk Palliative Conduit.

Björk conduit failure is a common reason for reintervention after a Björk modification of the Fontan procedure. We describe a first performed in human percutaneous procedure for the treatment of a failing Björk circuit in an adult with congenital heart disease and complex anatomic features. (Level of Difficulty: Advanced.).

Extracorporeal Membrane Oxygenation as a Rescue Therapy for Postoperative Diastolic Dysfunction and Refractory Chylothorax.

This is the first published case, as far as we know, of a term neonate with refractory chylothorax secondary to diastolic dysfunction in the cardiac postoperative period, where extracorporeal membrane oxygenation (ECMO) was used to improve the physiologic derangements, thus allowing resolution of the chylous effusion. The infant was prenatally diagnosed with d-transposition of the great arteries. He was started on prostaglandin infusion and underwent balloon atrial septostomy followed by arterial switch operation. After surgery, he developed anasarca and high-volume chylothorax that did not respond to medical management and fasting. Cardiac catheterization demonstrated severe diastolic dysfunction and pulmonary hypertension. On postoperative day 19, he was placed on veno-arterial (VA) ECMO and had gradual regression of the chylothorax and edema. After 13 days on ECMO support, he was decannulated with small, self-limiting, reaccumulation of chylous effusion. He was discharged home on postoperative day 57, and has since been thriving with no evidence of reaccumulation of the chylous effusion. In summary, VA ECMO support could be considered as a rescue modality for patients with uncontrollable refractory high-volume chylous effusion, after other treatment options have been pursued.