Experts in Minimally Invasive Surgery are Outperformed by Trained Novices on Suturing Skills.

INTRODUCTION: Learning minimally invasive suturing can be challenging, creating a barrier to further implementation, especially with the development of easier methods. Nevertheless, mastering intracorporeal knot tying is crucial when alternative techniques prove inadequate. Therefore, the minimally invasive surgery (MIS) suturing skills of MIS experts are compared with a group of novices during their learning curve on a simulator. METHODS: The novice participants repeatedly performed the intracorporeal suturing task on the EoSim MIS simulator (up to a maximum of 20 repetitions). The experts (>50 MIS procedures and advanced MIS experience) completed the same task once. The first and last exercises of the novices and the expert tasks were all blindly recorded and assessed by two independent assessors using the Laparoscopic Suturing Competency Assessment Tool (LS-CAT). Additionally, objective assessment parameters, "time" and "distance", using instrument tracking, were collected. The scores of the experts were then compared with the novices. RESULTS: At the end of the training, novices significantly outperformed the experts on both the expert assessment (LS-CAT: 16.8 versus 26.8, P = 0.001) and objective parameters (median time: 190 s versus 161 s, P < 0.001; median distance: 6.1 m versus 3.6 m, P < 0.001). Although the experts showed slightly better performance than the novices during their first task, the difference was not significant on the expert assessment (LS-CAT experts 16.8, novices 20.5, P = 0.057). CONCLUSIONS: Our findings underscore the significance of continued MIS suturing training for both residents and surgeons. In this study, trained novices demonstrated a significant outperformance of experts on both quantitative and qualitative outcome parameters within a simulated setting.

The feasibility and benefit of unsupervised at-home training of minimally invasive surgical skills.

BACKGROUND: Simulation-based training may be used to acquire MIS skills. While mostly done in a simulation center, it is proposed that this training can be undertaken at-home as well. The aim of this study is to evaluate whether unsupervised at-home training and assessment of MIS skills is feasible and results in increased MIS skills. METHODS: Medical doctors and senior medical students were tested on their innate abilities by performing a pre-test on a take-home simulator. Henceforth, they followed a two-week interval training practicing two advanced MIS skills (an interrupted suture with knot tying task and a precise peg transfer task) and subsequently performed a post-test. Both tests and all training moments were performed at home. Performance was measured using motion analysis software (SurgTrac) and by expert-assessment and self-assessment using a competency assessment tool for MIS suturing (LS-CAT). RESULTS: A total of 38 participants enrolled in the study. Participants improved significantly between the pre-test and the post-test for both tasks. They were faster (632 s vs. 213 s, p < 0.001) and more efficient (distance of instrument tips: 9.8 m vs. 3.4 m, p = 0.001) in the suturing task. Total LS-CAT scores, rated by an expert, improved significantly with a decrease from 36 at the pre-test to 20 at the post-test (p < 0.001) and showed a strong correlation with self-assessment scores (R 0.771, p < 0.001). The precise peg transfer task was completed faster (300 s vs. 163 s, p < 0.001) and more efficient as well (14.8 m vs. 5.7 m, p = 0.005). Additionally, they placed more rings correctly (7 vs. 12, p = 0.010). CONCLUSION: Unsupervised at-home training and assessment of MIS skills is feasible and resulted in an evident increase in skills. Especially in times of less exposure in the clinical setting and less education on training locations this can aid in improving MIS skills.

Health-related quality of life in children with congenital vascular malformations.

A cross-sectional study was performed to evaluate health-related quality of life (HRQOL) in children with congenital vascular malformations (CVM) and to investigate factors associated with an impaired HRQOL. Children (2-17 years) with CVMs who visited the HECOVAN expertise center between 2016-2018 were included. The PedsQL 4.0 Generic Core Scales were used and a score ≥ 1.0 SD below the normative mean was defined as an impaired HRQOL. Factors associated with impairment were investigated using univariate and multivariate logistic regression analysis. The median overall HRQOL was 84.8/100 (n = 207; 41% boys, 59% girls; self-reported IQR 73.9-92.4 and parent-reported IQR 71.4-92.4). Patients aged 13-17 years reported significantly worse physical functioning than those aged 8-12 years (median 84.4, IQR 71.1-93.8 versus median 90.6, IQR 81.3-96.9; p = 0.02). Parents reported a significantly lower overall HRQOL than their children (median 80.4, IQR 70.7-90.8 versus median 85.9, IQR 76.1-92.4; p = 0.001). HRQOL was impaired in 25% of patients. Impairment occurred significantly more often in lower extremity CVMs (38%, p = 0.01) and multifocal CVMs (47%, p = 0.01) compared to CVMs in the head/neck region (13%). Other associated factors included invasive management (31% versus 14%; p = 0.01), age at first treatment ≤ 5 years (48% versus 25%; p = 0.02) and ongoing treatment (38% versus 18%; p = 0.004). After correction for other factors, significance remained for lower extremity CVMs and ongoing invasive treatment. CONCLUSIONS: Overall median HRQOL was reasonable and not significantly different from the norm sample. Parental ratings were significantly lower than their children's ratings. A quarter of the patients had an impaired HRQOL, which seemed to worsen with age. Independently associated factors included a lower extremity CVM and invasive management. WHAT IS KNOWN: • Congenital vascular malformations could affect health-related quality of life (HRQOL). • Studies on pediatric patients are limited and either very small or in combination with adult patient series. WHAT IS NEW: • This study raises awareness of an impaired HRQOL in 25% of pediatric patients with congenital vascular malformations. • Associated factors included a lower extremity CVM and invasive management.

Effective low-dose sirolimus regimen for kaposiform haemangioendothelioma with Kasabach-Merritt phenomenon in young infants.

AIMS: Management of kaposiform haemangioendotheliomas (KHE) with Kasabach-Merritt phenomenon is challenging in young infants who are subjected to developmental pharmacokinetic changes. Sirolimus, sometimes combined with corticosteroids, can be used as an effective treatment of KHE. Simultaneously, toxicities such as interstitial pneumonitis related to the use of sirolimus may be fatal. As infants have a very low CYP3-enzyme expression at birth, which rises during ageing, we hypothesize that a reduced metabolization of sirolimus might lead to high sirolimus serum levels and low dose may be sufficient without the side effects. METHODS: A case series of 5 infants with kaposiform haemangioendothelioma with Kasabach-Merritt phenomenon was analysed retrospectively. All infants were treated with sirolimus 0.2 mg/m2 every 24 or 48 hours according to their age. Prednisone was added to the therapy for additional effect in 4 patients. RESULTS: In all patients, low dose of sirolimus led to therapeutic sirolimus levels (4-6 ng/mL). All infants (aged 4 days-7 months) had a complete haematological response, without serious adverse events. In all patients, the Kasabach-Merritt phenomenon resolved, the coagulation profile normalized and tumour size reduction was seen. CONCLUSION: Low-dose sirolimus treatment is safe for infants with kaposiform haemangioendothelioma and Kasabach-Merritt phenomenon. It is essential to realize that during the first months of life, metabolism is still developing and enzymes necessary to metabolise drugs like sirolimus still have to mature. To avoid toxic levels, the sirolimus dosage should be based on age and the associated pharmacological developments.

The effect of continuous at-home training of minimally invasive surgical skills on skill retention.

BACKGROUND: Skill deterioration of minimally invasive surgical (MIS) skills may be prevented by continuous training. The aim of this study is to evaluate whether unsupervised continuous at-home training of MIS skills results in better skill retention compared to no training. METHODS: Medical doctors followed a two-week interval training for two MIS tasks (precise peg transfer and interrupted suture with knot tying), ending with a baseline test. They were randomly assigned to the no-practice group or continuous-practice group. The latter practiced unsupervised at home every two weeks during the study period. Skill retention was measured after three and six months on both tasks by the total time needed, distance traveled by instruments and LS-CAT score (8 best possible score and > 40 worst score). RESULTS: A total of 38 participants were included. No significant differences in performance were found at pre-test or baseline. At six months the no-practice group needed more time for the suturing task (309 s vs. 196 s at baseline, p = 0.010) and the LS-CAT score was significantly worse (30 vs. 20 at baseline, p < 0.0001). The continuous-practice group performed the suturing task significantly better than the no-practice group at both three and six months (17 vs. 25, p < 0.001 and 17 vs. 30, p < 0.001) and faster as well (p = 0.034 and p = 0.001). CONCLUSION: This study shows a skill decay after only a few months of non-use and shows better skill retention after continuous unsupervised at-home practice of MIS skills. This indicates an added value of regular at-home practice of surgical skills.

Lymphatic malformations in children: treatment outcomes of sclerotherapy in a large cohort.

This retrospective study examines the outcomes of sclerotherapy in children with (veno)lymphatic malformations who received sclerotherapy between 2011 and 2016 (116 children, 234 procedures). Complication severity was classified using the Society of Interventional Radiology classification. Clinical response was rated on a scale of 0 (no change) to 3 (good improvement). The sclerosants used were bleomycin (n = 132; 56%), lauromacrogol (n = 42; 18%), doxycycline (n = 15; 6%), ethanol (n = 12; 5%), or a combination (n = 33; 14%). Four major and 25 minor complications occurred without significant differences between the agents. The median response rate per procedure was 2-some improvement-for all sclerosants. However, in pure LMs (67%), bleomycin and a combination of agents resulted in the best clinical response. On patient level, all had some or good clinical response. Mixed macrocystic and microcystic lesions showed a significantly lower clinical response (median 2 versus 3; p = 0.023 and p = 0.036, respectively) and required significantly more procedures (median 2 versus 1; p = 0.043 and p = 0.044, respectively) compared with lesions with one component.Conclusion: Sclerotherapy for (V)LMs in children is safe and effective. Bleomycin is the most frequently used agent in this clinic and seemed most effective for pure LMs. Mixed macrocystic and microcystic lesions are most difficult to treat effectively. What is Known: • A variety of agents can be used for sclerotherapy of lymphatic malformations in children. • Macrocystic lesions have favorable outcomes compared with microcystic and mixed lesions. What is New: • Bleomycin and a combination of agents seem to be most effective to treat lymphatic malformations in children. • Mixed macrocystic and microcystic lesions are more difficult to treat effectively compared with lesions with either one of these components.

Treatment Outcomes of Embolization for Peripheral Arteriovenous Malformations.

PURPOSE: To evaluate treatment outcomes of embolization for peripheral arteriovenous malformations (AVMs) in a tertiary referral center where ethanol is the primary agent of choice. METHODS: A retrospective study was performed of 93 patients (median age, 31 years; range, 2-66 years) with peripheral AVMs treated with embolization (n = 442; median, 2 per patient; range, 1-82) between January 2010 and July 2016. Ethanol was used in most cases (n = 428; 97%). AVMs were classified as type I (n = 3), type II (n = 57), type IIIa (n = 5), type IIIb (n = 15), and type IV (n = 13) according to the Yakes classification system. Effectiveness of embolization was based on AVM devascularization on angiography: 100% (total), 90%-99% (near-total), 70%-90% (substantial), 30%-70% (partial), and 0%-30% (failure). Complications were graded according to the Society of Interventional Radiology classification. RESULTS: In 69% of patients, 70%-100% devascularization was achieved. Total and near-total occlusion of the nidus were more often achieved in AVMs of types I and IIIa (both 100%) than in AVMs of types II, IIIb, and IV (56%, 67%, and 39%, respectively; P = .019). A total of 109 complications were identified: 101 minor (22.9%) and 8 major (1.8%). Major complications included wounds (n = 5), false aneurysm (n = 1), finger contracture (n = 1), and severe pain (n = 1) requiring therapy. The patient complication risk was significantly affected by the number of procedures (relative risk = 2.0; P < .001). Age, AVM location, and angioarchitecture type did not significantly affect complication risk. CONCLUSIONS: AVM embolization resulted in 70%-100% devascularization in 69% of patients, with few major complications. This study indicates that the type of AVM angioarchitecture affects the number of procedures needed and the achievability of AVM devascularization.

Comprehensive molecular and clinicopathological analysis of vascular malformations: A study of 319 cases.

Vascular malformations are part of overgrowth syndromes characterized by somatic mosaic mutations or rarely by germline mutations. Due to their similarities and diversity, clinicopathological classification can be challenging. A comprehensive targeted Next Generation Sequencing screen using Unique Molecular Identifiers with a technical sensitivity of 1% mutant alleles was performed for frequently mutated positions in ≥21 genes on 319 formalin-fixed paraffin-embedded samples. In 132 out of 319 cases pathogenic mosaic mutations were detected affecting genes previously linked to vascular malformations e.g. PIK3CA (n=80), TEK (TIE2) (n=11), AKT1 (n=1), GNAQ (n=7), GNA11 (n=4), IDH1 (n=3), KRAS (n=9), and NRAS (n=1). Six cases harbored a combination of mutations in PIK3CA and in GNA11 (n=2), GNAQ (n=2), or IDH1 (n=2). Aberrations in PTEN and RASA1 with a variant allele frequency approaching 50% suggestive of germline origin were identified in six out of 102 cases tested; four contained a potential second hit at a lower allele frequency. Ninety-one of the total 142 pathogenic mutations were present at a variant allele frequency <10% illustrating the importance of sensitive molecular analysis. Clinicopathological characteristics showed a broad spectrum and overlap when correlated with molecular data. Sensitive screening of recurrently mutated genes in vascular malformations may help to confirm the diagnosis and reveals potential therapeutic options with a significant contribution of PIK3CA/mTOR and RAS-MAPK pathway mutations. The co-existence of two activating pathogenic mutations in parallel pathways illustrates potential treatment challenges and underlines the importance of multigene testing. Detected germline mutations have major clinical impact.

Self-assessment, and not continuous training, improves basic open suturing skills.

BACKGROUND: To develop and maintain suturing skills, clinical exposure is important. When clinical exposure cannot be guaranteed, an adequate training schedule for suturing skills is required. This study evaluates the effect of continuous training, 'reflection before practice' and self-assessment on basic open suturing skills. METHODS: Medical students performed four basic suturing tasks on a simulation set up before ('pre-test') and after their surgical rotation ('after-test'). Participants were divided in three groups; the 'clinical exposure group' (n = 44) had clinical exposure during their rotation only, the 'continuous training group' (n = 16) completed a suturing interval training during their rotation and the 'self-assessment group' (n = 16) also completed a suturing interval training, but with the use of reflection before practice and self-assessment. Parameters measured by a tracking system during the suturing tasks and a calculated 'composite score' were compared between groups and test-moments. RESULTS: A significantly better composite score was found at the after-test compared to the pre-test for all groups for all basic suturing tasks (0.001 ≤ p ≤ 0.049). The self-assessment group scored better at the pre-test than the other two groups for all tasks, except for 'knot tying by hand' (0.004 ≤ p ≤ 0.063). However, this group did not score better at the after-test for all tasks, compared to the other two groups. This resulted in a smaller delta of time ('transcutaneous suture', p = 0.013), distance ('Donati suture' and 'intracutaneous suture', 0.005 ≤ p ≤ 0.009) or composite score (all tasks, except for knot tying by hand, 0.007 ≤ p ≤ 0.061) in the self-assessment group. CONCLUSION: Reflection before practice and self-assessment during continuous training of basic open suturing tasks, may improve surgical skills at the start of the learning curve.

Alcohol's impact on fine motor skills: Insights from minimally invasive surgical simulation.

Background: Alcohol misuse among medical professionals poses a significant concern, and there is a lack of clarity in (inter)national guidelines regarding alcohol use during work. Moreover, there exists an insufficient body of research on the specific impact of alcohol on fine motor skills within the medical sector, specifically surgery. This study aims to investigate the impact of alcohol on fine motor skills in a minimally invasive surgical setting. Methods: A cross-sectional study was conducted at Lowland Science on August 19th, 20th, and 21st, 2022, during the Lowlands music festival in Biddinghuizen, the Netherlands. Participants were divided into five groups based on measured alcohol consumption. Exclusion criteria included drug use, prior surgical experience, being underage, and previous participation. The main outcomes were the number of correctly transferred rings during the PEG transfer task and the number of errors. Blood alcohol concentration (BAC) was measured using a breathalyser. Results: A total of 1056 participants were included in the study. The results indicated an inverse relationship between BAC levels and surgical performance, with higher alcohol levels associated with a decrease in performance (p = 0.023). However, there was no significant difference in the number of errors among the five groups (p = 0.597). The group with the highest alcohol consumption (BAC >0.08 %) exhibited significantly worse performance compared to the group with a BAC of 0.0 % (p = 0.002). Conclusion: This study uncovers a negative impact of increased alcohol intake on fine motor skills in a minimally invasive surgery simulation exercise. While there was no effect on the occurrence of errors. Professional medical organizations should reconsider and explicate their position on alcohol use in (surgical) healthcare.

Congenital Vascular Malformations in Children: From Historical Perspective to a Multidisciplinary Approach in the Modern Era-A Comprehensive Review.

Congenital vascular malformations (CVMs) are the result of an aberrant development during embryogenesis. Although these lesions are present at birth, they are not always visible yet. Once symptomatic, patients suffer from pain, bleeding, ulcers, infections or lymphatic leakage, depending on the subtype of vessels involved. Treatment includes conservative management, surgery, sclerotherapy, embolization and pharmacological therapy. The clinical presentation varies widely and treatment can be challenging due to the rarity of the disease and potential difficulties of treatment. This review gives an overview of the historical developments in diagnosis and classification and exposes the key elements of innovations in the past decades on the identification of genetic mutations and personalized treatment. These advances in the field and a multidisciplinary approach are highly valuable in the optimization of clinical care aimed at both curing or stabilizing the CVM and pursuing physical and psychosocial wellbeing.

Concurrent validity of objective assessment by finger tracking for open surgical suturing training.

BACKGROUND: The aim of this study is to evaluate whether the parameters 'time' and 'distance', measured by SurgTrac, correlate with the assessment of the same skills by blinded experts. METHODS: Basic open suturing tasks were executed by medical students. SurgTrac software measured objective parameters by tracking fingers. The executed tasks were recorded by a tablet and additionally assessed by a blinded expert with a Competency Assessment Tool-form (CAT-form). A Pearson's correlation was used to investigate the correlation between the parameters and the outcomes of the expert assessment. RESULTS: A strong correlation between the measured parameters of SurgTrac and the expert-assessment was found for knot tying by hand (r â€‹= â€‹-0.703) and vertical mattress suture (r â€‹= â€‹-0.644) and a moderate correlation for transcutaneous suture (r â€‹= â€‹-0.555) and intracutaneous suture (r â€‹= â€‹-0.451). CONCLUSION: The use of finger tracking by SurgTrac showed a good concurrent validity for the basic open suturing tasks knot tying by hand, transcutaneous suture and vertical mattress suture.

Assessment of Minimally Invasive Suturing Skills: Is Instrument Tracking an Accurate Prediction?

Introduction: Minimally invasive surgery (MIS) suturing demands advanced surgical skills. Therefore, it is important these skills are adequately trained and assessed. Assessment and feedback can consist of judgments and scores of expert observers or objective parameters using instrument tracking. The aim of this study was to determine to what extent objective parameters correspond to expert assessment. Methods: Participants performed an intracorporeal suturing task on the EoSim simulator repeatedly (maximum 20 repetitions) during training. The best discriminating parameters, which previously shown construct validation, were combined into a composite score, using regression analysis. All videos were blinded and assessed by 2 independent reviewers using the validated laparoscopic suturing competency assessment tool (LS-CAT). These scores were compared with the composite score. Results: A 100 videos of 16 trainees, during separate points on their learning curve, and 8 experts were used. The parameters "time" and "distance" were statistically significantly correlated with all LS-CAT domains. The composite score (calculated from "time" and "distance") showed improvement between the first and the last knot (57% versus 94%, P < .001). Also the LS-CAT score improved (28 versus 17, P < .001). However, the correlation of the composite score with the LS-CAT score was weak (R: 0.351), with an accuracy of 55/100 when pooling the outcomes based on inadequate, adequate, or good performance. Conclusion: Instrument tracking parameters (using Surgtrac) could give an indication of the skill level, however, it missed important elements, essential for reliable assessment. Therefore, expert assessment remains superior to determine the skill level in MIS suturing skills.

Evaluating the Use of a Take-Home Minimally Invasive Surgery Box Training for At-Home Training Sessions Before and During the COVID Pandemic.

Introduction: Acquiring and retaining minimally invasive surgery (MIS) skills may be aided by using MIS take-home box simulators. This study aims to evaluate the use of a take-home MIS box trainer for training sessions at-home. Methods: Trainees who previously bought the LaparoscopyBoxx (between 2016 and 2020) were asked to complete an online questionnaire. The first part was on their demographic data, and the second part was on their use and opinion on the box trainer (on a 5-point Likert scale). Results: A total of 39 participants (9 surgeons and 30 trainees) completed the questionnaire (response rate 40%). Overall, participants had a positive opinion on the use of the box trainer and would recommend it to others for training (mean 4.2). Basic tasks, such as needle trail and ring transfer, were practiced most often and were regarded most relevant (mean 4.1-4.3). They regarded practicing on the stimulator of added value to practicing in the clinic (mean 4.2), were more confident during clinical procedures (mean 3.9), and felt that their skills had improved (mean 4.1). Of quarter of the participants who bought the MIS box trainer during the COVID-19 pandemic reported an increased use during times of less clinical exposure. Conclusion: Surgeons and trainees have a positive opinion on using a take-home MIS box trainer for at-home training for general as well as for pediatric MIS skills. Especially during periods of less exposure in the clinical setting and less hands-on courses, such as during the COVID-19 pandemic, at-home training may be a valuable addition.

Objective assessment for open surgical suturing training by finger tracking can discriminate novices from experts.

It is difficult, time consuming and expensive to assess manual skills in open surgery. The aim of this study is to investigate the construct validity of a low-cost, easily accessible tracking technique for basic open suturing tasks. Medical master students, surgical residents, and surgeons at the Radboud University Medical Center were recruited between September 2020 until September 2021. The participants were divided, according to experience, in a novice group (≤10 sutures performed) and an expert group (>50 sutures performed). For objective tracking, a tablet with SurgTrac software was used, which tracked a blue and a red tag placed on respectively their left and right index finger. The participants executed four basic tasks on a suturing model: 1) knot tying by hand, 2) transcutaneous suturing with an instrument knot, 3) 'Donati' (vertical mattress suture) with an instrument knot and 4) continuous intracutaneous suturing without a knot. In total 76 participants were included: 57 novices and 19 experts. All four tasks showed significant differences between the novice group and expert group for the parameters time (p<0.001), distance (p<0.001 for Task 1, 2 and 3 and p=0.034 for Task 4) and smoothness (p<0.001). Additionally, Task 3 showed a significant difference for the parameter handedness (p=0.006) and Task 4 for speed (p=0.033). Tracking index finger movements using SurgTrac software on a tablet while executing basic open suturing skills on a simulator shows excellent construct validity for time, distance and motion smoothness in all four suturing tasks.

Magnitude and relevance of change in health-related quality of life in patients with vascular malformations treated with sirolimus.

Introduction: Vascular malformations are rare congenital anomalies of the vascular system, which can involve the capillaries, veins, arteries, lymphatics, or a combination of vessel types. Patients with vascular malformations experience an impaired health-related quality of life (HRQoL) because of their symptoms (e.g., pain, swelling, and bleeding) and psychosocial distress. Sirolimus is an effective drug used in the medical treatment of these patients; however, relatively little is known about the effect of sirolimus on specific changes in the HRQoL domains and its magnitude. Methods: The magnitude of change (effect size) following intervention is more informative to clinical practitioners than statistically significant but clinically unimportant changes; therefore, this study aimed to examine the magnitude and meaningfulness of change in the HRQoL of children and adults with vascular malformations following sirolimus treatment using low target levels. Results: In total, 50 patients with vascular malformations (19 children, 31 adults) were included in this study. These patients experienced a lower HRQoL than the general population, with the adults reporting a significantly lower score in almost all domains. A 6-month sirolimus treatment improved the HRQoL in 29 patients, including 77.8% of the children (Pediatric Quality of Life Inventory score [PedsQL]) and 57.7% of the adults (Short Form 36 [SF-36]). The effect sizes of sirolimus for each SF-36/PedsQL domain ranged from 0.19 to 1.02. The clinically relevant moderate magnitude of changes was seen in the domains of the children's reports: "Physical functioning" and "Social functioning" and in the domains of the parent reports: "Social functioning," "School functioning," and "Psychosocial." A high-magnitude change was seen in the domains "Emotional functioning" and "Psychosocial" in the children's reports and "Physical functioning" in the parent reports. In addition, the moderate magnitude of changes was also seen in the adults SF-36: in all domains except for "Role limitations-physical problems," "Role limitations-emotional problems," and "General health perception." Conclusion: We believe this is the first study showing the magnitude of change in HRQoL after sirolimus treatment in patients with vascular malformations. Before treatment, these patients experienced an impaired HRQoL compared with the general Dutch population. A 6-month sirolimus treatment with low target levels led to moderate-to-high clinically relevant changes in multiple domains, which significantly improved the HRQoL. Clinical trial registration: https://clinicaltrials.gov/ct2/show/NCT03987152?cond=Vascular+Malformations&cntry=NL&city=Nijmegen&draw=2&rank=1, identifier: NCT03987152.

Clinical differences in sirolimus treatment with low target levels between children and adults with vascular malformations - A nationwide trial.

The clinical presentation of patients with slow-flow vascular malformations is very heterogeneous. High clinical burden and subsequent reduced health-related quality of life is something they have in common. There is an unmet medical need for these patients for whom regular treatments like surgery and embolization are either insufficient or technically impossible. Sirolimus has been reported to be effective and overall well-tolerated in most patients. However, the main limitation of sirolimus is the reported high toxicity, especially when target levels of 10-15 ng/mL are being used. We report the results of a phase IIB single-arm open-label clinical trial consisting of 68 (67 in the challenge phase and 68 in the rechallenge phase) evaluable patients (children n = 33 and adults n = 35) demonstrating that treatment with low sirolimus target levels (4-10 ng/mL) is effective in 79.1% of the patients. When sirolimus treatment was stopped, the majority of patients experienced a recurrence of symptoms, supporting prolonged or even lifelong treatment requirement. Adults experienced a higher baseline pain score compared with children, having an estimated marginal mean of 6.2 versus 4.1, p < 0.05; however, they showed a similar decrease to children. Furthermore, the pediatric population experienced less often a sirolimus-related grade I-IV adverse event (35.9% vs. 64.1%, p > 0.05) compared with adults. Additionally, response rates were higher in children compared with adults (93.8% vs. 65.7%, p < 0.05), and children responded faster (28 vs. 91 days, p < 0.05). These results suggest benefits of sirolimus in patients with slow-flow vascular malformations and support its initiation as young as possible.

Lymphatic malformations adjacent to the airway in neonates: Risk factors for outcome.

PURPOSE: To evaluate imaging, treatment, and outcomes in neonates with a lymphatic malformation (LM) adjacent to the airway and to evaluate risk factors that can predict outcome. METHODS: A retrospective case series was conducted of ten patients treated between January 2011 and July 2019. The main outcome measures included airway compromise and clinical response to sclerotherapy ± surgery. Categorical data were compared using the Fisher's exact test. RESULTS: Ex-utero intrapartum therapy was performed in four cases, among whom one died due to sepsis. All patients underwent schlerotherapy, with surgical debulking in two. Four patients showed a good clinical response, and five started experimental systemic treatment. Patients with bilateral disease and patients with an LM with >180° tracheal surrounding were significantly at risk for airway compromise (bilateral: n = 6/6 versus n = 0/4, p = 0.005; >180°: n = 5/5 versus n = 1/5, p = 0.048). The need for LM treatment in the neonatal period was indicative of a poor clinical response ('non-responders' 5/6, 'responders' 0/4; p = 0.048). CONCLUSIONS: This study indicates that bilateral disease and >180° tracheal surrounding are risk factors for airway compromise in neonates with an LM adjacent to the airway. In addition, the need for early treatment seems to be indicative of a poor clinical response.

Take-Home Laparoscopy Simulators in Pediatric Surgery: Is More Expensive Better?

Background: To increase complex minimally invasive skills (MIS), frequent training outside the clinical setting is of uttermost importance. This study compares two low-cost pediatric MIS simulators, which can easily be used preclinically. Materials and Methods: The LaparoscopyBoxx is a portable simulator without a tracking system, with costs ranging from €90 to €315. The EoSim simulator has a built-in camera and tracking system and costs range from €780 to €1800. During several pediatric surgical conferences and workshops (January 2017-December 2018), participants were asked to use both simulators. Afterward, they completed a questionnaire regarding their opinion on realism and didactic value, scored on a five-point Likert scale. Results: A total of 50 participants (24 experts and 25 target group, one unknown) evaluated one or both simulators. Both simulators scored well on the questionnaire. The LaparoscopyBoxx scored significantly better regarding the "on screen representation of the instrument actions" (mean 4.2 versus 3.5, P = .001), "training tool for pediatric surgery" (mean 4.4 versus 3.9, P = .005), and "appealing take-home simulator" (mean 4.6 versus 4.0, P = .002). Conclusion: The simulators tested in this study were both regarded an appealing take-home simulator. The LaparoscopyBoxx scored significantly better than the EoSim, even though this is a low budget simulator without tracking capabilities.