Longitudinal Bone Mineralization Assessment in Children Treated With Long-Term Parenteral Nutrition for Severe Intestinal Failure.

BACKGROUND: Metabolic bone disease is common in children receiving home parenteral nutrition (HPN) for intestinal failure (IF). Long-term evolution of bone mass in pediatric IF is poorly documented. The aims of this study were (1) to determine the prevalence of low bone mass (LBM) in children receiving HPN for IF, (2) to evaluate the evolution of total bone mineral content (TBMC) during HPN with dual-energy x-ray absorptiometry (DXA), and (3) to identify related factors. METHODS: All children referred in our HPN center from 2004 to 2014 were eligible. Inclusion criteria were HPN dependence due to noninflammatory IF, at least 2 TBMC assessments, and HPN duration of at least 2 years at last DXA. TBMC was expressed in z score for ideal weight for height (WFH). LBM was defined by a TBMC WFH z score ≤-2 standard deviations (SD). RESULTS: A total of 175 DXAs for 31 children were performed, mean of 5.6 ± 2.9 assessments per child. The median time between first and last DXA recorded was 6.2 years (0.7-16.6). At the first DXA, 14 children (45%) had a LBM. TBMC increased by +0.1 ± 0.04 SD per year of HPN (P = .012). The risk of LBM decreased with an odds ratio of 0.9 per year of HPN (95% confidence interval, 0.92-0.99; P = .018). Lean mass z score and calcium parenteral intakes were related to the TBMC improvement. CONCLUSION: LBM is common in pediatric IF, but bone status could improve during HPN in these children.

Impact of the Early Start Denver Model on the cognitive level of children with autism spectrum disorder: study protocol for a randomised controlled trial using a two-stage Zelen design.

INTRODUCTION: Early intervention for autism spectrum disorder (ASD) in the European French-speaking countries is heterogeneous and poorly evaluated to date. Early intervention units applying the Early Start Denver Model (ESDM) for toddlers and young children with ASD have been created in France and Belgium to improve this situation. It is essential to evaluate this intervention for the political decision-making process regarding ASD interventions in European French-speaking countries. We will evaluate the effectiveness of 12 hours per week ESDM intervention on the cognitive level of children with ASD, over a 2-year period. METHODS AND ANALYSIS: The study will be a multicentre, randomised controlled trial, using a two-stage Zelen design. Children aged 15-36 months, diagnosed with ASD and with a developmental quotient (DQ) of 30 or above on the Mullen Scale of Early Learning (MSEL) will be included. We will use a stratified minimisation randomisation at a ratio 1:2 in favour of the control group. The sample size required is 180 children (120 in the control and 60 in the intervention group). The experimental group will receive 12 hours per week ESDM by trained therapists 10 hours per week in the centre and 2 hours in the toddlers' natural environment (alternatively by the therapist and the parent). The control group will receive care available in the community. The primary outcome will be the change in cognitive level measured with the DQ of the MSEL scored at 2 years. Secondary outcomes will include change in autism symptoms, behavioural adaptation, communicative and productive language level, sensory profile and parents' quality of life. The primary analysis will use the intention-to-treat principle. An economic evaluation will be performed. DISSEMINATION: Findings from the study will be disseminated through peer reviewed publications and meetings. TRIAL REGISTRATION NUMBER: NCT02608333 (clinicaltrials.gov); Pre-results.

[The emergence of patient safety issues in France]. / L'émergence de la question de la sécurité des patients en France.

INTRODUCTION: The objective of this study was to elucidate the conditions of emergence of patient safety issues in the public debate and the limits to implementation of patient safety in the current health system. METHOD: A narrative review of the international literature was conducted by searching PubMed, Cairn and Persée databases. RESULTS: The database search retrieved 2,206 documents, 48 of which were included in the study. The theme of patient safety has spread worldwide, but emerged late in France. The delayed emergence of patient safety in France is essentially related to the euphemistic approach to the problem of patient safety, the difficulty of adopting systematic reasoning, the lack of human resources management levers and the ambiguous position of patients in relation to patient safety.

Distraction osteogenesis versus fibula free flap for mandibular reconstruction after gunshot injury: socioeconomic and technical comparisons.

BACKGROUND: Since the birth of maxillofacial surgery, ballistic injuries are a real challenge for surgeons. For more than 20 years, fibula free flap (FFF) became the criterion standard for mandibular reconstructions. But FFF is not always the perfect answer for mandibular reconstruction, and a technique named distraction osteogenesis (DO) was raised. The purposes of this study are to estimate the financial cost and reattempt for the patient each techniques in our experience to determine the best-choice criteria for mandibular reconstruction after gunshot injury. METHODS: We performed a retrospective study over the last 15 years, including 15 patients with a mandibular ballistic injury. Ten patients were treated with FFF, and 5 with DO. We evaluated the complications and morbidity encountered with each technique. We also decided to estimate the cost of different rehabilitations, including the cost of the device and hospitalization. RESULTS: In our study, the global cost of the DO protocol appears as not more expensive than the FFF one. Postoperative complications encountered during the FFF protocols were related to donor-site morbidity. The DO patient had pseudoarthrosis, mucosa irritation, or local infection. DISCUSSION: Our study demonstrated both the economic and technical interest of DO compared with the FFF for mandibular reconstruction. Thus, nowadays, DO appears as an alternative to the FFF for mandibular reconstruction, the main decisional criterion being the evaluation of the tissues dilapidated during the initial traumatism, but social environment of the patient shall also be considered.

Crude versus case-mix-adjusted control charts for safety monitoring in thyroid surgery.

INTRODUCTION: Patient-safety monitoring based on health-outcome indicators can lead to misinterpretation of changes in case mix. This study aimed to compare the detection of indicator variations between crude and case-mix-adjusted control charts using data from thyroid surgeries. METHODS: The study population included each patient who underwent thyroid surgery in a teaching hospital from January 2006 to May 2008. Patient safety was monitored according to two indicators, which are immediately recognisable postoperative complications: recurrent laryngeal nerve palsy and hypocalcaemia. Each indicator was plotted monthly on a p-control chart using exact limits. The weighted κ statistic was calculated to measure the agreement between crude and case-mix-adjusted control charts. RESULTS: We evaluated the outcomes of 1405 thyroidectomies. The overall proportions of immediate recurrent laryngeal nerve palsy and hypocalcaemia were 7.4% and 20.5%, respectively. The proportion of agreement in the detection of indicator variations between the crude and case-mix-adjusted p-charts was 95% (95% CI 85% to 99%). The strength of the agreement was κ = 0.76 (95% CI 0.54 to 0.98). The single special cause of variation that occurred was only detected by the case-mix-adjusted p-chart. CONCLUSIONS: There was good agreement in the detection of indicator variations between crude and case-mix-adjusted p-charts. The joint use of crude and adjusted charts seems to be a reasonable approach to increase the accuracy of interpretation of variations in outcome indicators.

Coagulation assessment by rotation thrombelastometry in normal pregnancy.

We analysed changes in coagulation during normal pregnancy with a novel point-of-care device based on thrombelastometry (ROTEM). We compared the results obtained with those of standard coagulation tests in 104 patients: 20 non-pregnant women (controls) and 84 women in the first (T1, n = 17), second (T2, n = 9) and third (T3, n = 58) trimesters of pregnancy. We measured the clotting time (CT), the maximum clot firmness (MCF), the early clot amplitude at 5 and 15 minutes (CA(5), CA(15)) and the clot lysis index (CLI(30)) with four tests containing specific reagents. (a) The INTEM test involving ellagic acid activated the intrinsic pathway and (b) the EXTEM test using tissue factor triggered the extrinsic pathway; (c) The FIBTEM test based on a platelet inhibitor (cytochalasin D) evaluated the contribution of fibrinogen to clot formation and (d) the APTEM test was similar to the EXTEM but was based on inhibition in vitro of fibrinolysis by aprotinin. CT and CLI(30) were not significantly modified during pregnancy whereas MCF, CA(5) and CA(15) (INTEM, EXTEM, FIBTEM) increased significantly between the second and third trimesters (e.g. median [interquartile range]: MCF-FIBTEM, 13 [11-16] mm vs. 19 [17-23] mm, respectively, in controls and T3, p < 0.001). EXTEM values were not significantly different from those measured with APTEM. There were significant correlations between the results obtained with ROTEM and those from standard coagulation tests. ROTEM analysis showed a marked increase in coagulability during normal pregnancy. ROTEM values may serve as the basis for future studies in pregnant women.

Evolution of costs of care for cystic fibrosis patients after clinical guidelines implementation in a French network.

OBJECTIVES: The aim of this study was to evaluate how advances in CF management in France between 2000 and 2003 impacted CF-related costs. METHODS: The analysis of direct medical costs was done in 2000 and 2003 from the perspective of the French national healthcare insurance system. The patients, 65 in 2000 and 64 in 2003, were followed-up in one pediatric and one adult CF reference center (CFRC). We quantified and valued CF-related home and hospital care costs. RESULTS: We found an average cost of euro16474/patient/year in 2000, and euro22725 in 2003 (based on the 2003 euro value). Hospital care increased from 15% of the total cost in 2000 to 22% in 2003. Medications accounted for 45% of the total cost for the two periods, with an average cost of euro7229/patient/year in 2000 and euro10336 in 2003. Home intravenous antibiotic therapy accounted for 20% of the total cost for the two periods. CONCLUSIONS: We highlighted an increase in CF care costs between 2000 and 2003, which might be related to the changes in practice patterns that followed guidelines implementation, such as the use of new medications (dornase alpha and tobramycin) and more frequent follow-up in the CFRC.

Cost of home and hospital care for patients with cystic fibrosis followed up in two reference medical centers in France.

OBJECTIVES: In France, new guidelines for clinical practices concerning cystic fibrosis came out in 2002, underscoring the need for early and intensive management of this disease. Because no recent health economic studies on cystic fibrosis in France were available, we conducted a cost-analysis study before the new guidelines were put into practice, with a view to a later study on the medical and economic impact of these guidelines. METHODS: A cost-analysis study was performed of the inpatient and outpatient costs of patients with cystic fibrosis for the 2000-2001 period. The various direct costs were estimated on a sample of sixty-five adult and pediatric patients managed for cystic fibrosis in two reference medical centers. Data were obtained from medical records, and questionnaires were filled out by the patients. Analysis was made from the perspective of the French healthcare system. RESULTS: We studied sixty-five patients, 54 percent male patients and 72 percent children under 18 years of age. The total cost of cystic fibrosis care totaled 16,189 euros per year and per patient. Outpatient costs accounted for 88 percent of the total cost versus 12 percent for inpatient costs; medication costs were the highest with 21 percent of the total cost for home intravenous antibiotic treatments and 49 percent of the total cost for chronic medications. CONCLUSIONS: The results show that outpatient costs were higher than inpatient costs, which could be related to the importance granted to home health care in France, notably for intravenous antibiotic treatments given for pulmonary complications.

Cost analysis of human islet transplantation for the treatment of type 1 diabetes in the Swiss-French Consortium GRAGIL.

OBJECTIVE: To evaluate the cost of islet transplantation in type 1 diabetic patients with a functional renal graft in a multicenter network. RESEARCH DESIGN AND METHODS: The study involved nine diabetic patients transplanted in the Swiss-French Groupe Rhône-Alpes, Rhin et Geneve pour la transplantation d'Ilots Langerhans (GRAGIL) consortium between March 1999 and June 2000. The direct medical costs were estimated from Social Security's perspective from the inclusion of the patient to 1 year after transplantation. All cost components were computed separately and included evaluation, screening and candidacy, organ retrieval, islet processing, pancreas and islet transportation, hospitalization for transplantation, follow-up, medications (immunosuppressive, antidiabetic, and adjuvant drugs), and adverse events requiring hospitalization. RESULTS: During the study period, 56 pancreata were processed and 14 islet preparations were transplanted. The average cost of an islet transplantation (procedure and 1-year follow-up) was 77,745 euro (French rate, year 2000). The four main cost components were islet preparation (30% of the total cost), adverse events (24%), drugs (14%), and hospitalization (13%). CONCLUSIONS: Overall costs of islet transplantation are slightly higher than those of pancreas transplantation. The cell isolation process is a critical point; a reduction in overall cost will require more efficient ways of isolating high yields of viable islets. Costs generated by shipments within the GRAGIL network did not represent an economic burden. It can be expected that the costs will decrease with growing experience and improving technology.

Cost-effectiveness analysis of gestational diabetes mellitus screening in France.

OBJECTIVE: To compare three strategies for gestational diabetes screening (i) screening of high-risk pregnant women with the 50 g oral glucose tolerance test (OGTT); (ii) screening of all pregnant women with the 50 g OGTT; (iii) screening of all pregnant women according to the 75 g OGTT. STUDY DESIGN: Cost-effectiveness analysis. The outcome measures, i.e. macrosomia, prematurity, perinatal mortality, hypertensive disorders rates were estimated from published studies and the costs from a prospective study involving 120 pregnant women. RESULTS: Compared to the first strategy, the cost to obtain one unit of additional effectiveness with the second screening strategy, was up to 1.1 times more expensive, and with the third strategy was up to 3.7 times more expensive. CONCLUSION: The costs per case prevented reflect a favourable cost-effectiveness ratio (CER) for screening of high-risk pregnant women by 50 g oral glucose test.