Virginie Nerich

LOW EXPOSURE TO SUNLIGHT IS A RISK FACTOR FOR CROHN?S DISEASE

Aliment Pharmacol Ther 2011; 33: 940–945

Bevacizumab efficacy in metastatic colorectal cancer is dependent on primary tumor resection.

AbstractPurposeBevacizumab plus fluoropyrimidine-based chemotherapy is standard treatment for first-line and second-line metastatic colorectal cancer (mCRC). However, to date, there is no current biomarker predictive for the benefit of bevacizumab use for these patients. Preclinical data suggest that the presence of the primary tumor could be involved in less efficient antitumor activity of antiangiogenic agents, but no clinical data currently support this hypothesis.MethodsnWe performed a retrospective analysis of factors associated with overall survival (OS) in a study cohort of 409 mCRC patients. Univariate and multivariate Cox proportional hazard regression models were used to assess the influence of primary tumor resection and bevacizumab use on OS. We evaluated associations linking bevacizumab use and OS among patients who previously underwent or did not undergo primary tumor resection. Results were externally validated in a second independent cohort of 328 mCRC patients.ResultsIn the study cohort, bevacizumab use and resection of the primary tumor were associated with improved OS. However, subgroup analyses indicate that bevacizumab did not influence survival of patients bearing a primary colorectal tumor (hazard ratio (HR) 0.98, 95xa0% confidence interval (CI) 0.60–1.61, log-rank test Pxa0=xa00.6). By contrast, the survival benefit of bevacizumab was restricted to patients who previously underwent primary tumor resection (HR 0.71, 95xa0% CI 0.55–0.92, Pxa0=xa00.009). Similar results were observed in the validation cohort.ConclusionsAddition of bevacizumab to chemotherapy is associated with improvement of OS only in patients with primary tumor resection. These data support the rationale to validate prospectively the influence of primary tumor resection on bevacizumab antitumor effect in synchronous mCRC.

Cost-of-illness study of severe haemophilia A and B in five French haemophilia treatment centres

Objective The aim of this study was to assess the consumption of anti-haemophilic drugs by adults and children with severe haemophilia A or B (residual activity of FVIII or FIX ≤2%) and to quantify the average direct medical costs. Method A retrospective multicentre cost-of-illness study from the perspective of French national health insurance system. The costs include only the use of clotting factors. Main outcome measure Consumption was expressed in UI/kg/year and costs in euros/kg/year. Results From January 1, 2001 to December 31, 2002, data from 81 adults and 30 children with severe haemophilia A (nxa0=xa092) or B (nxa0=xa019) and included in the “SNH” were collected and analysed. A coagulation factor inhibitor was present in 10 patients (9%). Four of them were high responders. Mean age and body weight were respectively 28xa0±xa017xa0years and 58xa0±xa024xa0kg. Except for one adult patient, all (99%) had outpatient treatment, 44 patients (40%) were hospitalized and treated by recombinant or/and plasma-derived FVIII or FIX or/and rFVIIa. Overall median annual consumption of anti-haemophilic drugs per patient was estimated at 1,333xa0UI/kg, with a median cost-of-illness of 1,156xa0euros/kg. Patients with severe haemophilia B consumed more than patients with severe haemophilia A, though not significantly (Pxa0=xa00.096), with a median of 2,167 vs. 1,100xa0UI/kg/year and a median cost of 1,760 vs. 917xa0euros/kg/year (Pxa0=xa00.13). Children consumed respectively more than adults (Pxa0=xa00.008), with a median of 3,204 vs. 1,106xa0UI/kg/year and a median cost of 2,614 vs. 913xa0euros/kg/year (Pxa0=xa00.012). The median cost for patients with an inhibitor was 3,291xa0euros/kg/year, approximately threefold higher than that of patients without an inhibitor (926xa0euros/kg/year) (Pxa0=xa00.022). Conclusion It suggests a higher consumption and cost of anti-haemophilic drugs among children when compared to adults. Haemophilia B patients did not consume significantly more than haemophilia A patients, whereas the consumption and cost for patients with or without inhibitors differed significantly.

Quality assessment of economic evaluation studies in pediatric surgery: A systematic review

PURPOSEnTo assess economic evaluation studies (EES) in pediatric surgery and to identify potential factors associated with high-quality studies.nnnMETHODSnA systematic review of the literature using PubMed and Cochrane databases was conducted to identify EES in pediatric surgery published between 1 June 1993 and 30 June 2013. Assessment criteria are derived from the Drummond checklist. A high quality study was defined as a Drummond score ≥7. Logistic regression analysis was used to determine factors associated with high quality studies.nnnRESULTSn119 studies were included. 43.7% (n=52) of studies were full EES. Cost-effectiveness analysis was the most frequent (61.5%) type of full EES. Only 31.6% of studies had a Drummond score ≥7 and 73% of these were full EES. The factors associated with high quality were identification of costs (OR: 14.08; 95% CI: 3.38-100; p<0.001), estimation of utility value (OR: 8.13; 95% CI: 2.02-43.47; p=0.005) and study funding (OR: 3.50; 95% CI: 1.27-10.10; p=0.02).nnnCONCLUSIONnThis review shows that the number and the quality of EES are low despite the increasing number of studies published in recent years. In the current context of budget constraints, our results should encourage pediatric surgeons to focus more on EES.

Factors Influencing Overall Survival for Patients With Metastatic Clear-Cell Renal-Cell Carcinoma in Daily Practice

Purpose To describe factors associated with overall survival (OS) among patients with metastatic clear‐cell renal‐cell carcinoma (mccRCC) in regard to evolution of systemic therapies. Patients and Methods Two hundred twenty‐four consecutive patients with histologically confirmed mccRCC who received targeted therapy on first‐line treatment between January 2007 and March 2015 were included. The primary end point was OS for metastatic first‐line or second‐line treatment. An analysis of prognostic factors of long survival was performed using a 2‐step approach: univariate, then multivariate analysis. Results Median OS [95% confidence interval] was 19.4 months [16.1‐24.9]. Three prognostic factors were identified in first‐line treatment: Memorial Sloan Kettering Cancer Center (MSKCC) favorable and intermediate risks (hazard ratio [95% confidence interval] = 0.362 [0.207‐0.630] and 0.561 [0.393‐0.801], respectively, P = 4.10−4), metastasectomy (0.667 [0.468‐0.951], P = .03), and lack of lymph node metastasis (0.715 [0.513‐0.994], P = .049). In second‐line treatment, median OS [95% confidence interval] was 11.0 months [8.9‐14.4] for 167 patients. Three different prognostic factors predicted long survival: toxicity for first‐line treatment discontinuation (HR [95% confidence interval] = 0.298 [0.180‐0.493], P < 10−4), duration of disease control in first‐line therapy (0.961 [0.942‐0.979], P = 2.10−4), and MSKCC favorable and intermediate risks (0.461 [0.252‐0.843] and 0.936 [0.607‐1.443], respectively, P = .02). Conclusion These real‐life data confirm the positive impact of targeted therapy in the mccRCC setting. Moreover, it emphasizes the importance of considering many factors in order to better estimate prognosis in patient pretreated with systemic therapy. Micro‐Abstract This retrospective study aimed to describe, in daily practice, factors associated with overall survival among 224 patients with metastatic clear‐cell renal‐cell carcinoma. Different prognostic factors were identified in metastatic first‐line and second‐line treatment. These real‐life data confirm the positive impact of targeted therapy and underline the importance of considering many factors to better estimate patient prognosis.

Prévalence et prise en charge de la douleur chez les patients présentant un cancer métastatique en Franche-Comté

INTRODUCTIONnPain management is a major public health problem, especially in oncology. In order to assess professional practice, the IRFC-FC conducted a survey amongst patients with metastatic osteophilic solid tumor in Franche-Comté. The aims were to assess the pain prevalence, and its characteristics, its management and its impact on patients quality of life in patients in pain.nnnMETHODSnAn observational, prospective and multicenter survey was conducted using a self-report questionnaire. Patients with metastatic breast or prostate cancer managed in 5 day-hospitals of the IRFC-FC over a period of three months were included.nnnRESULTSnTwo hundred thirty-three questionnaires were analyzed. Pain prevalence rate was 48.5%. Three quarters of patients in pain had chronic background pain, moderate to severe, with or without breakthrough pain. Considering their pain intensity and their analgesic therapy, 42.0% of patients seem to have an inadequate treatment. Eighty-five percent of treated patients reported to be compliant and felt that their pain was well managed despite a strong impact on their quality of life.nnnCONCLUSIONnThe setting of a specific clinical pathway is essential to secure the standardized, optimal and efficient management of patients in pain. The assessment of patient satisfaction and quality of life must be integrated in clinical practice to identify patients in pain for which the treatment is inappropriate.

Efficacité, tolérance et coût de l’éribuline chez des patientes présentant un cancer du sein métastatique

Eribulin gained its approval in March 2011 for the treatment of patients with locally advanced or metastatic breast cancer (MBC) whose disease has progressed despite anthracycline and taxane-containing regimens. This study retrospectively assessed the efficacy, safety and cost of this treatment for all patients with MBC treated by eribulin in Franche-Comté. Ninety-four patients received eribulin between July 2006 and October 2013. The median age was 62 years (35-83). Median overall survival was 10.3 months [95% CI: 7.6 to 17.9]. Median progression-free-survival was 3.8 months [95% CI: 2.9 to 5.0]. Clinical benefit was obtained in 55% evaluable patients [95% CI: 43.1 to 66.9] by RECIST criteria. Most common grade 3-4 adverse events (AEs) were neutropenia (38%), asthenia (10%) and peripheral neuropathy (7%). Median cost of the treatment was 9767xa0€ per patient (6344-17,517). This analysis found similar results to the EMBRACE study despite less selected population. A medico-economic evaluation cost-utility type would assess the effectiveness of this strategy compared to standard treatments.

Evaluation of efficacy and efficiency of a pragmatic intervention by a social worker to support informal caregivers of elderly patients (The ICE Study): study protocol for a randomized controlled trial

BackgroundMedical progress and the lifestyle modification have prolonged life expectancy, despite the development of chronic diseases. Support and care for older subjects are often provided by a network of informal caregivers composed of family, friends and neighbors, who are essential in helping older persons to continue living at home. It has been shown that the extent and diversity of informal tasks may jeopardize the physical, mental and social wellbeing of caregivers.Methods/designThe aim of the Informal Carers of Elderly cohort is to define, through a longitudinal study, profiles of caregivers of older patients with a diagnosis of one of the following diseases: cancer (breast, prostate, colorectal), neurodegenerative diseases (Parkinson’s disease, Alzheimer’s disease and similar diseases), neurovascular diseases (stroke), sensory diseases (age-related macular degeneration (AMD)) and heart disease (heart failure). Patients must be at least 60xa0years old and living in the region of Burgundy-Franche-Comte (France). By following the different phases of the caregiving relationship from the announcement of the diagnosis, it will be possible to assess the quality of life of caregivers, coping strategies, levels of anxiety and depression, social support and the extent of their burden. We will also evaluate the efficacy and efficiency of the implementation of a pragmatic intervention by a social worker to help informal caregivers, through a randomized interventional trial nested in the cohort. Qualitative approaches aimed at studying the caregiver/patient relationship, and situations leading to breakdown of the caregiver relationship will be also undertaken.DiscussionThrough an analytical and longitudinal definition of profiles of informal caregivers, this study will gather detailed information on their life courses and their health trajectory by identifying consequences associated with the concept of their role as carers. In addition, the randomized interventional trial will explore the relevance of the implementation of a supportive intervention by a social worker to help caregivers. These data will help to identify strategies that could be used to improve the existing sources of aid and to propose new approaches to help caregivers. This study will provide the opportunity to identify the most relevant means of support adapted to caregivers, and provide an impulse for new health care policies.Trial registrationClinicalTrials.gov Identifier: NCT02626377. Retrospectively registered on 9 December 2015. Protocol date/version: 23 October 2014/version 2.

Adjuvant hormonal therapy for early breast cancer: an epidemiologic study of medication adherence

PurposeThe aim of this study was to determine the prevalence of adherence to adjuvant hormonal therapy (AHT) and to identify risk factors for medication non-adherence in clinical practice in patients with early-stage hormone receptor (HR)-positive breast cancer (BC) previously treated with chemotherapy.MethodsWe carried out a cross-sectional, observational, prospective, and multicenter survey based on a structured self-report postal questionnaire (35 items investigating six areas). A sample of 474 patients was drawn from 676 patients potentially eligible. The structured and validated Morisky Medication Adherence Scale-4 items was used for measuring medication adherence. An analysis of risk factors for non-adherence to AHT was performed using a two-step approach: univariate, then multivariate analysis.ResultsA total of 280 patients out of the 428 analyzed patients participated in the survey, yielding a response rate of 65.4% [60.9–69.9]. The prevalence of adherence to AHT was estimated at 68.6% [63.1–74.0], corresponding to a high level of adherence. Three risk factors for non-adherence to AHT were identified:xa0>xa02 medications to treat comorbidities (p-valuexa0=xa00.003), age less than 65xa0years (p-valuexa0=xa00.008), and patient management in a university hospital setting (p-valuexa0=xa00.014).ConclusionsNon-adherence is a common, complex, and multidimensional healthcare problem. This better understanding and knowledge of risk factors will allow healthcare providers (such as oncologists, general practitioners, pharmacists) to more easily identify patients at risk for non-adherence and help them provide appropriate information about AHT and its management, thus improving medication adherence in their patients.

A systematic review of economic evaluation in pancreatic ductal adenocarcinoma

OBJECTIVESnThe economic evaluation (EE) of healthcare interventions has become a necessity. However, high quality needs to be ensured in order to achieve validated results and help making informed decisions. Thus, the objective of the present study was to systematically identify and review published pancreatic ductal adenocarcinoma-related EEs and to assess their quality.nnnMETHODSnSystematic literature research was conducted in PubMed and Cochrane to identify published EEs between 2000 and 2015. The quality of each selected EE was assessed by two independent reviewers, using the Drummonds checklist.nnnRESULTSnOur systematic review was based on 32xa0EEs and showed a wide variety of methodological approaches, including different perspectives, time horizon, and cost effectiveness analyses. Nearly two-thirds of EEs are full EEs (nxa0=xa021), and about one-third of EEs had a Drummond score ≥7, synonymous with high quality. Close to 50% of full EEs had a Drummond score ≥7, whereas all of partial EEs had a Drummond score <7 (nxa0=xa011).nnnCONCLUSIONSnOver the past 15 years, a lot of interest has been evinced over the EE of pancreatic ductal adenocarcinoma (PDAC)xa0and its direct impact on therapeutic advances in PDAC. To provide a framework for health care decision-making, to facilitate transferability and to lend credibility to health EEs, their quality must be improved. For the last 4 years, a tendency towards a quality improvement of these studies has been observed, probably coupled with a context of rational decision-making in health care, a better and wider spread of recommendations and thus, medical practitioners full endorsement.