Randomised clinical trial: First-line infliximab biosimilar is cost-effective compared to conventional treatment in paediatric Crohn's disease.

BACKGROUND: Data on cost-effectiveness of first-line infliximab in paediatric patients with Crohn's disease are limited. Since biologics are increasingly prescribed and accompanied by high costs, this knowledge gap needs to be addressed. AIM: To investigate the cost-effectiveness of first-line infliximab compared to conventional treatment in children with moderate-to-severe Crohn's disease. METHODS: We included patients from the Top-down Infliximab Study in Kids with Crohn's disease randomised controlled trial. Children with newly diagnosed moderate-to-severe Crohn's disease were treated with azathioprine maintenance and either five induction infliximab (biosimilar) infusions or conventional induction treatment (exclusive enteral nutrition or corticosteroids). Direct healthcare consumption and costs were obtained per patient until week 104. This included data on outpatient hospital visits, hospital admissions, drug costs, endoscopies and surgeries. The primary health outcome was the odds ratio of being in clinical remission (weighted paediatric Crohn's disease activity index<12.5) during 104 weeks. RESULTS: We included 89 patients (44 in the first-line infliximab group and 45 in the conventional treatment group). Mean direct healthcare costs per patient were €36,784 for first-line infliximab treatment and €36,874 for conventional treatment over 2 years (p = 0.981). The odds ratio of first-line infliximab versus conventional treatment to be in clinical remission over 104 weeks was 1.56 (95%CI 1.03-2.35, p = 0.036). CONCLUSIONS: First-line infliximab treatment resulted in higher odds of being in clinical remission without being more expensive, making it the dominant strategy over conventional treatment in the first 2 years after diagnosis in children with moderate-to-severe Crohn's disease. TRIAL REGISTRATION NUMBER: NCT02517684.

Doxapram versus placebo in preterm newborns: a study protocol for an international double blinded multicentre randomized controlled trial (DOXA-trial).

BACKGROUND: Apnoea of prematurity (AOP) is one of the most common diagnoses among preterm infants. AOP often leads to hypoxemia and bradycardia which are associated with an increased risk of death or disability. In addition to caffeine therapy and non-invasive respiratory support, doxapram might be used to reduce hypoxemic episodes and the need for invasive mechanical ventilation in preterm infants, thereby possibly improving their long-term outcome. However, high-quality trials on doxapram are lacking. The DOXA-trial therefore aims to investigate the safety and efficacy of doxapram compared to placebo in reducing the composite outcome of death or severe disability at 18 to 24 months corrected age. METHODS: The DOXA-trial is a double blinded, multicentre, randomized, placebo-controlled trial conducted in the Netherlands, Belgium and Canada. A total of 396 preterm infants with a gestational age below 29 weeks, suffering from AOP unresponsive to non-invasive respiratory support and caffeine will be randomized to receive doxapram therapy or placebo. The primary outcome is death or severe disability, defined as cognitive delay, cerebral palsy, severe hearing loss, or bilateral blindness, at 18-24 months corrected age. Secondary outcomes are short-term neonatal morbidity, including duration of mechanical ventilation, bronchopulmonary dysplasia and necrotising enterocolitis, hospital mortality, adverse effects, pharmacokinetics and cost-effectiveness. Analysis will be on an intention-to-treat principle. DISCUSSION: Doxapram has the potential to improve neonatal outcomes by improving respiration, but the safety concerns need to be weighed against the potential risks of invasive mechanical ventilation. It is unknown if the use of doxapram improves the long-term outcome. This forms the clinical equipoise of the current trial. This international, multicentre trial will provide the needed high-quality evidence on the efficacy and safety of doxapram in the treatment of AOP in preterm infants. TRIAL REGISTRATION: ClinicalTrials.gov NCT04430790 and EUDRACT 2019-003666-41. Prospectively registered on respectively June and January 2020.

Music to prevent deliriUm during neuroSurgerY (MUSYC): a single-centre, prospective randomised controlled trial.

OBJECTIVES: Delirium is a serious complication following neurosurgical procedures. We hypothesise that the beneficial effect of music on a combination of delirium-eliciting factors might reduce delirium incidence following neurosurgery and subsequently improve clinical outcomes. DESIGN: Prospective randomised controlled trial. SETTING: Single centre, conducted at the neurosurgical department of the Erasmus Medical Center, Rotterdam, the Netherlands. PARTICIPANTS: Adult patients undergoing craniotomy were eligible. INTERVENTIONS: Patients in the intervention group received preferred recorded music before, during and after the operation until day 3 after surgery. Patients in the control group were treated according to standard of clinical care. PRIMARY AND SECONDARY OUTCOME MEASURES: Primary outcome was presence or absence of postoperative delirium within the first 5 postoperative days measured with the Delirium Observation Screening Scale (DOSS) and, in case of a daily mean score of 3 or higher, a psychiatric evaluation with the latest Diagnostic and Statistical Manual of Mental Disorders (DSM-5) criteria. Secondary outcomes included anxiety, heart rate variability (HRV), depth of anaesthesia, delirium severity and duration, postoperative complications, length of stay and location of discharge. RESULTS: We enrolled 189 patients (music=95, control=94) from July 2020 through September 2021. Delirium, as assessed by the DOSS, was less common in the music (n=11, 11.6%) than in the control group (n=21, 22.3%, OR:0.49, p=0.048). However, after DSM-5 confirmation, differences in delirium were not significant (4.2% vs 7.4%, OR:0.47, p=0.342). Moreover, music increased the HRV (root mean square of successive differences between normal heartbeats, p=0.012). All other secondary outcomes were not different between groups. CONCLUSION: Our results support the efficacy of music in reducing the incidence of delirium after craniotomy, as found with DOSS but not after DSM-5 confirmation, substantiated by the effect of music on preoperative autonomic tone. Delirium screening tools should be validated and the long-term implications should be evaluated after craniotomy. TRIAL REGISTRATION NUMBER: Trialregister.nl: NL8503 and ClinicalTrials.gov: NCT04649450.

COllaborative Neonatal Network for the first European CPAM Trial (CONNECT): a study protocol for a randomised controlled trial.

INTRODUCTION: Consensus is lacking on the optimal management of asymptomatic congenital pulmonary airway malformation (CPAM). For future studies, the CONNECT consortium (the COllaborative Neonatal Network for the first European CPAM Trial)-an international collaboration of specialised caregivers-has established consensus on a core outcome set of outcome parameters concerning respiratory insufficiency, surgical complications, mass effect and multifocal disease. These outcome parameters have been incorporated in the CONNECT trial, a randomised controlled trial which, in order to develop evidence-based practice, aims to compare conservative and surgical management of patients with an asymptomatic CPAM. METHODS AND ANALYSIS: Children are eligible for inclusion after the CPAM diagnosis has been confirmed on postnatal chest CT scan and they remain asymptomatic. On inclusion, children are randomised to receive either conservative or surgical management. Subsequently, children in both groups are enrolled into a standardised, 5-year follow-up programme with three visits, including a repeat chest CT scan at 2.5 years and a standardised exercise tolerance test at 5 years.The primary outcome is exercise tolerance at age 5 years, measured according to the Bruce treadmill protocol. Secondary outcome measures are molecular genetic diagnostics, validated questionnaires-on parental anxiety, quality of life and healthcare consumption-, repeated imaging and pulmonary morbidity during follow-up, as well as surgical complications and histopathology. This trial aims to end the continuous debate surrounding the optimal management of asymptomatic CPAM. ETHICS AND DISSEMINATION: This study is being conducted in accordance with the Declaration of Helsinki. The Medical Ethics Review Board of Erasmus University Medical Centre Rotterdam, The Netherlands, has approved this protocol (MEC-2022-0441). Results will be disseminated through peer-reviewed scientific journals and conference presentations. TRIAL REGISTRATION NUMBER: NCT05701514.

Effect of music on clinical outcome after hip fracture operations (MCHOPIN): study protocol of a multicentre randomised controlled trial.

BACKGROUND: Patients undergoing proximal femur fracture surgery are at high risk of postoperative complications, with postoperative delirium occurring in 25%-40% of patients. Delirium has profound effects on patient outcome and recovery, the patient's family, caregivers and medical costs. Perioperative music has a beneficial effect on eliciting modifiable risk factors of delirium. Therefore, the aim of this trial was to evaluate the effect of perioperative recorded music on postoperative delirium in patients with proximal femur fracture undergoing surgery. METHODS AND ANALYSIS: The Music on Clinical Outcome after Hip Fracture Operations study is an investigator-initiated, multicentre, randomised controlled, open-label, clinical trial. Five hundred and eight patients with proximal femur fracture meeting eligibility criteria will be randomised to the music intervention or control group with concealed allocation in a 1:1 ratio, stratified by hospital site. The perioperative music intervention consists of preselected lists totalling 30 hours of music, allowing participants to choose their preferred music from these lists (classical, jazz and blues, pop and Dutch). The primary outcome measure is postoperative delirium rate. Secondary outcome measures include pain, anxiety, medication requirement, postoperative complications, hospital length of stay and 30-day mortality. A 90-day follow-up will be performed in order to assess nursing home length of stay, readmission rate and functional ability to perform daily living activities. Furthermore, the cost and cost-effectiveness of the music intervention will be assessed. Data will be analysed according to an intention-to-treat principle. ETHICS AND DISSEMINATION: The study protocol has been approved by the Medical Research Ethics Committee Erasmus MC on 8 October 2018 (MEC-2018-110, NL64721.078.18). The trial will be carried out following the Declaration of Helsinki principles, Good Clinical Practice guidelines and Dutch Medical Research Involving Human Subjects Act. Research data will be reported following Consolidated Standards of Reporting Trials guidelines and study results will be published in a peer-reviewed journal. TRIAL REGISTRATION NUMBER: NTR7036.

Music to prevent deliriUm during neuroSurgerY (MUSYC) Clinical trial: a study protocol for a randomised controlled trial.

INTRODUCTION: Delirium is a neurocognitive disorder characterised by an acute and temporary decline of mental status affecting attention, awareness, cognition, language and visuospatial ability. The underlying pathophysiology is driven by neuroinflammation and cellular oxidative stress.Delirium is a serious complication following neurosurgical procedures with a reported incidence varying between 4% and 44% and has been associated with increased length of hospital stay, increased amount of reoperations, increased costs and mortality.Perioperative music has been reported to reduce preoperative anxiety, postoperative pain and opioid usage, and attenuates stress response caused by surgery. We hypothesize that this beneficial effect of music on a combination of delirium eliciting factors might reduce delirium incidence following neurosurgery and subsequently improve clinical outcomes. METHODS: This protocol concerns a single-centred prospective randomised controlled trial with 6 months follow-up. All adult patients undergoing a craniotomy at the Erasmus Medical Center in Rotterdam are eligible. The music group will receive recorded music through an overear headphone before, during and after surgery until postoperative day 3. Patients can choose from music playlists, offered based on music importance questionnaires administered at baseline. The control group will receive standard of clinical careDelirium is assessed by the Delirium Observation Scale and confirmed by a delirium-expert psychiatrist according to the DSM-5 criteria. Risk factors correlated with the onset of delirium, such as cognitive function at baseline, preoperative anxiety, perioperative medication use, depth of anaesthesia and postoperative pain, and delirium-related health outcomes such as length of stay, daily function, quality of life (ie, EQ-5D, EORTC questionnaires), costs and cost-effectiveness are collected. ETHICS AND DISSEMINATION: This study is being conducted in accordance with the Declaration of Helsinki. The Medical Ethics Review Board of Erasmus University Medical Center Rotterdam, The Netherlands, approved this protocol. Results will be disseminated via peer-reviewed scientific journals and conference presentations. TRIAL REGISTRATION NUMBERS: NL8503 and NCT04649450.

Interventions with Music in PECTus excavatum treatment (IMPECT trial): a study protocol for a randomised controlled trial investigating the clinical effects of perioperative music interventions.

INTRODUCTION: Pectus excavatum repair is associated with substantial postoperative pain, despite the use of epidural analgesia and other analgesic regimens. Perioperative recorded music interventions have been shown to alleviate pain and anxiety in adults, but evidence for children and adolescents is still lacking. This study protocol describes a randomised controlled trial that evaluates the effects of recorded music interventions on postoperative pain relief in children and adolescents after pectus excavatum repair. METHODS: A multicentre randomised controlled trial was set up comparing the effects of perioperative recorded music interventions in addition to standard care with those of standard care only in patients undergoing a Nuss procedure for pectus excavatum repair. One hundred and seventy subjects (12-18 years of age) will be included in three centres in the Netherlands. Patient inclusion has started in November 2018, and is ongoing. The primary outcome is self-reported perceived pain measured on the visual analogue scale. Secondary outcomes are anxiety level, analgesics consumption, vital parameters such as heart rate, blood pressure and respiratory rate, length of hospital stay, postoperative complications, quality of life and cost-effectiveness. ETHICS AND DISSEMINATION: This study is being conducted in accordance with the Declaration of Helsinki. The Medical Ethics Review Board of Erasmus University Medical Centre Rotterdam, The Netherlands, has approved this protocol. Results will be disseminated via peer-reviewed scientific journals and conference presentations. TRIAL REGISTRATION NUMBER: NL6863.

Association between children's emotional/behavioral problems before adenotonsillectomy and postoperative pain scores at home.

BACKGROUND: Children undergoing adenotonsillectomy are at risk of severe postoperative pain and sleep problems. Little is known about the specific child risk factors for these problems. AIMS: The aim of this study was to assess the occurrence of postoperative pain, sleep problems, and medication adherence, and assess the influence of internalizing and externalizing problems on postoperative pain. METHODS: This prospective cohort study included 160 children, aged 1.5-5 years undergoing day-care adenotonsillectomy. Parents rated their child's pain with the Parents' Postoperative Pain Measure and their child's sleep problems with Vernon's Post Hospital Behavioral Questionnaire during the first 3 days and at day 10 postoperatively. Emotional/behavioral problems (ie, internalizing and externalizing behaviors) during the past 2 months were assessed using the Child Behavior Checklist. Regression analysis was used to assess whether children's pain intensity at home was associated with internalizing/externalizing problems, after controlling for age, preoperative child state anxiety, parental state anxiety, parental need for information, and socioeconomic status. RESULTS: Applying a threshold of ≥6 on the Parents' Postoperative Pain Measure, the incidence of moderate to severe pain was 57.6% at day 1, 53.5% at day 2, 35.4% at day 3, and 4.8% at day 10. During the first three postoperative nights, 37.1% of the children woke up. Internalizing problems (ß = 0.343; P = 0.001) and parental need for information (ß = 0.207; P = 0.011) were independently associated with higher pain scores at home during the first 3 days (R2 = 0.225). CONCLUSION: Following adenotonsillectomy, children often experienced moderate to severe pain and sleep problems during the first 3 days at home. Preoperative internalizing problems and parental need for information were independently associated with increased pain at home. Screening for these problems can help to identify vulnerable children and adapt the perioperative analgesic strategy accordingly (which includes preparation, information, and prescription of pain analgesics).

Changes in sensory processing after anesthesia in toddlers.

BACKGROUND: Anesthesia and surgery may influence toddlers' sensory processing and consequently postoperative adjustment and behavior. This is the first study to: 1) test pre- to postoperative changes in sensory processing after pediatric anesthesia using the validated Infant/Toddler-Sensory Profile for 7-36 months (ITSP7-36); 2) identify putative predictors of these changes. METHODS: This prospective cohort study included 70 healthy boys (ASA I & II), aged 18-30 months, who underwent circumcision for religious reasons. Exclusion: boys with prior surgery and known developmental delay. PRIMARY OUTCOME: changes in sensory processing from the day of admission to day 14 postoperatively. The accompanying parent completed the ITSP7-36. Putative predictors: 1) child's preoperative emotional/behavioral problems; 2) child's state anxiety at induction; 3) postoperative pain at home. All children received standardized anesthesia and pain management. RESULTS: For 45 boys, assessments were completed at both time points. Significant changes in sensory processing (mean ITSP7-36 scores) were found on: low registration (47.5 to 49.8; P=0.015), sensory sensitivity (45.2 to 48.0; P=0.011), sensation avoiding (48.2 to 51.3; P=0.010), low threshold (93.4 to 99.4; P=0.007), auditory processing (39.3 to 43.3; P=0.000) and tactile processing (53.9 to 58.4; P=0.002). Higher scores on emotional/behavioral problems predicted changes on sensory processing. CONCLUSIONS: Sensory processing of these toddlers had changed after anesthesia. Children with more pre-existent emotional/behavioral problems are more vulnerable to these changes.

A Visual Analog Scale to assess anxiety in children during anesthesia induction (VAS-I): Results supporting its validity in a sample of day care surgery patients.

BACKGROUND: The modified Yale Preoperative Anxiety Scale is widely used to assess children's anxiety during induction of anesthesia, but requires training and its administration is time-consuming. A Visual Analog Scale, in contrast, requires no training, is easy-to-use and quickly completed. AIM: The aim of this study was to evaluate a Visual Analog Scale as a tool to assess anxiety during induction of anesthesia and to determine cut-offs to distinguish between anxious and nonanxious children. METHODS: Four hundred and one children (1.5-16 years) scheduled for daytime surgery were included. Children's anxiety during induction was rated by parents and anesthesiologists on a Visual Analog Scale and by a trained observer on the modified Yale Preoperative Anxiety Scale. Psychometric properties assessed were: (i) concurrent validity (correlations between parents' and anesthesiologists' Visual Analog Scale and modified Yale Preoperative Anxiety Scale scores); (ii) construct validity (differences between subgroups according to the children's age and the parents' anxiety as assessed by the State-Trait Anxiety Inventory); (iii) cross-informant agreement using Bland-Altman analysis; (iv) cut-offs to distinguish between anxious and nonanxious children (reference: modified Yale Preoperative Anxiety Scale ≥30). RESULTS: Correlations between parents' and anesthesiologists' Visual Analog Scale and modified Yale Preoperative Anxiety Scale scores were strong (0.68 and 0.73, respectively). Visual Analog Scale scores were higher for children ≤5 years compared to children aged ≥6. Visual Analog Scale scores of children of high-anxious parents were higher than those of low-anxious parents. The mean difference between parents' and anesthesiologists' Visual Analog Scale scores was 3.6, with 95% limits of agreement (-56.1 to 63.3). To classify anxious children, cut-offs for parents (≥37 mm) and anesthesiologists (≥30 mm) were established. CONCLUSIONS: The present data provide preliminary data for the validity of a Visual Analog Scale to assess children's anxiety during induction.

Burden of surgical congenital anomalies in Kenya: a population-based study.

BACKGROUND: Congenital malformations are a significant component of the global burden of disease among children, accounting for 25 million disability-adjusted life years (DALYs) worldwide. Unfortunately, efforts to estimate the burden of pediatric surgical disease in Africa are limited by the absence of population-based data. The objective of this study was to estimate both the prevalence and the disease burden of several common congenital surgical malformations among children in Kenya. METHODS: Community volunteers randomly surveyed households at sites across Kenya. Caretakers were asked to identify on a photographic portfolio, several congenital malformations present among the children in their household, including club foot, hypospadias, hydrocephalus, spina bifida/encephalocele, cleft lip, bladder exstrophy and imperforate anus. DALYs were then calculated based on life expectancy tables and published and estimated disability weights for the conditions encountered. RESULTS: The caregivers of 5559 children (54% female) were surveyed in 1909 households, 56% of which were rural, 31% suburban and 12% urban. The overall prevalence of congenital malformations was 6.3 per 1000 children, amounting to 54-120 DALYs per 1000 children, depending on the life tables used. The most prevalent condition in the survey was club foot, whereas spina bifida had the highest burden of disease. DISCUSSION: This study is the first to document the prevalence of selected surgical congenital malformations among children in Kenya and the burden of disease associated with them. The results will serve to inform strategies aimed at reducing the unmet burden of surgical disease in resource-limited regions.

Impact of infantile short bowel syndrome on long-term health-related quality of life: a cross-sectional study.

BACKGROUND/PURPOSE: Studies on the long-term effects of short bowel syndrome (SBS) on the quality of life are scarce. Therefore, we determined health-related quality of life (HRQoL) in children and adolescents with a history of infantile SBS compared with that of same-aged controls drawn from the general population. METHODS: Cross-sectional assessment (2005-2007) of HRQoL in children with a history of infantile SBS treated in their first year of life, born between 1987 and 2002, using generic HRQoL measures was performed. Children aged 5 to 18 years and their parents filled out the Pediatric Quality of Life Inventory. Differences in scores between subjects and controls were analyzed using independent sample t tests. RESULTS: Thirty-one children with a history of SBS (19 girls and 12 boys; mean age, 11.8 ± 4.2 years) participated, giving an overall response rate of 70%. The children and their parents reported significantly lower HRQoL than 275 healthy age-matched children and their parents (P < .05). CONCLUSIONS: Children with a history of infantile SBS have a lower HRQoL than healthy age-matched controls. This difference lasts beyond childhood and way beyond the period of intestinal rehabilitation early in life. These findings highlight the need for targeted interventions to address these dimensions of impaired HRQoL.

Late vs early ostomy closure for necrotizing enterocolitis: analysis of adhesion formation, resource consumption, and costs.

BACKGROUND: Surgeons prefer to close ostomies at least 6 weeks after the primary operation because of the anticipated postoperative abdominal adhesions. Limited data support this habit. Our aim was to evaluate adhesion formation-together with an analysis of resource consumption and costs-in patients with necrotizing enterocolitis who underwent early closure (EC), compared with a group of patients who underwent late closure (LC). METHODS: Chart reviews and cost analyses were performed on all patients with necrotizing enterocolitis undergoing ostomy closure from 1997 to 2009. Operative reports were independently scored for adhesions by 2 surgeons. RESULTS: Thirteen patients underwent EC (median, 39 days; range, 32-40), whereas 62 patients underwent LC (median, 94 days; range, 54-150). Adhesion formation in the EC group (10/13 patients, or 77%) was not significantly different (P = 1.000) from the LC group (47/59 patients, or 80%). No differences were found in the costs of hospital stay, surgical interventions, and outpatient clinic visits. CONCLUSIONS: Ostomy closure within 6 weeks of the initial procedure was not associated with more adhesions or with changes in direct medical costs. Therefore, after stabilization of the patient, ostomy closure can be considered within 6 weeks during the same admission as the initial laparotomy.

Patient safety culture in a Dutch pediatric surgical intensive care unit: an evaluation using the Safety Attitudes Questionnaire.

OBJECTIVE: Nowadays, the belief is widespread that a safety culture is crucial to achieving patient safety, yet there has been virtually no analysis of the safety culture in pediatric hospital settings so far. Our aim was to measure the safety climate in our unit, compare it with benchmarking data, and identify potential deficiencies. DESIGN: Prospective longitudinal survey study at two points in time. SETTING: Pediatric surgical intensive care unit at a Dutch university hospital. SUBJECTS: All unit personnel. INTERVENTIONS: To measure the safety climate, the Safety Attitudes Questionnaire was administered to physicians, nurses, nursing assistants, pharmacists, technicians, and ward clerks in both May 2006 and May 2007. This questionnaire assesses caregiver attitudes through use of the six following scales: teamwork climate, job satisfaction, perceptions of management, safety climate, working conditions, and stress recognition. Earlier research showed that the Safety Attitudes Questionnaire has good psychometric properties and produced benchmarking data that can be used to evaluate strengths and weaknesses in a given clinical unit against peers. MEASUREMENTS AND MAIN RESULTS: The response rates for the Safety Attitudes Questionnaire were 85% (May 2006) and 74% (May 2007). There were mixed findings regarding the difference between physicians and nurses: on three scales (i.e., teamwork climate, safety climate, and stress recognition), physicians scored better than nurses at both points in time. On another two scales (i.e., perceptions of management and working conditions), nurses consistently had higher mean scale scores. Probably due to the small number of physicians, only some of these differences between physicians and nurses reached the level of statistical significance. Compared to benchmarking data, scores on perceptions of management were higher than expected (p < .01), whereas scores on stress recognition were low (p < .001). The scores on the other scales were somewhat above (job satisfaction), close to (teamwork climate, safety climate), or somewhat below (working conditions) what was expected on the basis of benchmarking data, but no persistent significant differences were observed on these scales. CONCLUSIONS: Although on most domains the safety culture in our unit was good when compared to benchmark data, there is still room for improvement. This requires us to continue working on interventions intended to improve the safety culture, including crew resource management training, safety briefings, and senior executive walk rounds. More research is needed into the impact of creating a safety culture on patient outcomes.

Interdisciplinary management of infantile short bowel syndrome: resource consumption, growth, and nutrition.

BACKGROUND/PURPOSE: To date, there are hardly any data on the treatment costs of infantile short bowel syndrome (SBS), despite growing interest in evidence-based and cost-effective medicine. Therefore, the aim of the study was to evaluate resource consumption and costs, next to studying nutritional and growth outcomes, in children with SBS who were treated by an interdisciplinary short bowel team. METHODS: Data were collected for 10 children with infantile SBS (

Role of health technology assessment in shaping the benefits package in The Netherlands.

In many countries of the Western world, the role of health technology assessment (HTA) in funding decisions of medical technologies is increasing. HTAs are expected to support decision-makers in delineating the collectively funded benefits package. To maximize their potential, it is essential that assessments are valid, reliable and timely, and that it is transparent how information provided in assessments is used in decision-making. Against this background, this article aims to review the current state of affairs regarding the use of HTA in the area of medical specialist care in The Netherlands and to evaluate strengths and weaknesses of the HTA-based system for priority setting. The reason to do so was the introduction of a new hospital financing system in The Netherlands, which allowed for expansion of the HTA system that already existed for pharmaceuticals to medical specialist care. A comprehensive account of the HTA system for medical specialist care was created using the so-called Hutton framework, followed by an exploration of its strengths and weaknesses. An important lesson to be learned from the early Dutch experiences with HTA in the area of medical specialist care is that the nature and complexity of health technologies in this area create practical problems regarding the amount and quality of available data needed to make the HTA-based system work. This hampers an unambiguous interpretation of assessment data and thus calls for stronger requirements regarding transparency and stakeholder participation. Future work focusing on the role of HTA in funding decisions is needed to provide insights in best practices for HTA systems in circumstances where a delicate balance needs to be achieved between promoting innovation, supporting effective and timely decision-making and preventing the coverage of technologies that represent a waste of resources.

Cost-effectiveness of neonatal surgery: first greeted with scepticism, now increasingly accepted.

Mortality rates in neonatal surgery have dropped markedly, illustrating the enormous progress made. Yet, new questions have arisen. To mention one, health care budgets have tightened. It follows that the effects of medical interventions should be weighted against their costs. As evidence was particularly sparse, we set out to analyse cost-effectiveness of neonatal surgery. The purpose of this article is to summarise our findings and to review recent studies. Moreover, this article explains the relevance of cost-effectiveness analysis and explores how cost-effectiveness interacts with other determinants of health care priority setting. Our research revealed that treatments for two common diagnostic categories in neonatal surgery (congenital anorectal malformations and congenital diaphragmatic hernia) produce good cost-effectiveness. Other groups also published cost-effectiveness studies in the field of neonatal surgery, although their number is still small. Contemporaneously, the economic aspects of health care have captured the interest of policy makers. Importantly, this is not to say that there are no other factors playing a role in priority setting, foremost among which are ethical questions and arguments of equity. This article concludes that, according to present evidence, neonatal surgery yields good value for money and contributes to equity in health.

The cost-effectiveness of treatment for congenital diaphragmatic hernia.

BACKGROUND/PURPOSE: The cost-effectiveness of medical interventions is becoming an important issue for decision makers. Until recently, evidence of the cost-effectiveness of neonatal surgery was largely lacking. The authors analyzed the cost-effectiveness of neonatal surgery and subsequent treatment for congenital diaphragmatic hernia (CDH). METHODS: Both costs incurred inside and outside the health care sector (eg, out-of-pocket expenses and productivity losses) were included. Quality-adjusted life years (QALYs) were measured using the EuroQol EQ-5D questionnaire. Descriptive quality-of-life data were collected using a disease-specific questionnaire. Both costs and effects basically were measured in a life-time setting. RESULTS: Total costs of treatment average euro 42,658, mainly consisting of costs of the initial hospitalization. Productivity losses in both the patients and their caregivers appear to be minor. Treated CDH patients, even adults, suffer from respiratory difficulties and stomach aches. According to the EQ-5D, however, their quality of life does not differ from the general population, suggesting that these symptoms barely affect overall quality of life. Treatment results in a gain of 17.5 QALYs. Costs per QALY amount to euro 2,434. CONCLUSIONS: Treatment for CDH has favorable cost-effectiveness. Considering the growing importance of cost-effective medicine, these are important and encouraging results. Health economics outlines the inevitability of making choices that directly affect patient care and places relative values on different health care programs. The results of this study provide convincing evidence that treatment for CDH is indeed cost effective.