Parent Project Research Articles

Eplerenone for early cardiomyopathy in Duchenne muscular dystrophy: a randomised, double-blind, placebo-controlled trial

Cardiomyopathy is a leading cause of death in patients with Duchenne muscular dystrophy and myocardial damage precedes decline in left ventricular systolic function. We tested the efficacy of eplerenone on top of background therapy in patients with Duchenne muscular dystrophy with early myocardial disease. In this randomised, double-blind, placebo-controlled trial, boys from three centres in the USA aged 7 years or older with Duchenne muscular dystrophy, myocardial damage by late gadolinium enhancement cardiac MRI and preserved ejection fraction received either eplerenone 25 mg or placebo orally, every other day for the first month and once daily thereafter, in addition to background clinician-directed therapy with either angiotensin-converting enzyme inhibitors (ACEI) or angiotensin receptor blockers (ARB). Computer-generated randomisation was done centrally using block sizes of four and six, and only the study statistician and the investigational pharmacy had the preset randomisation assignments. The primary outcome was change in left ventricular circumferential strain (Ecc) at 12 months, a measure of contractile dysfunction. Safety was established through serial serum potassium levels and measurement of cystatin C, a non-creatinine measure of kidney function. This trial is registered with ClinicalTrials.gov, number NCT01521546. Between Jan 26, 2012, and July 3, 2013, 188 boys were screened and 42 were enrolled. 20 were randomly assigned to receive eplerenone and 22 to receive placebo, of whom 20 in the eplerenone group and 20 in the placebo group completed baseline, 6-month, and 12-month visits. After 12 months, decline in left ventricular circumferential strain was less in those who received eplerenone than in those who received placebo (median ΔEcc 1·0 [IQR 0·3-2·2] vs 2·2 [1·3-3·1]; p=0·020). Cystatin C concentrations remained normal in both groups, and all non-haemolysed blood samples showed normal potassium concentrations. One 23-year-old patient in the placebo group died of fat embolism, and another patient in the placebo group withdrew from the trial to address long-standing digestive issues. All other adverse events were mild: short-lived headaches coincident with seasonal allergies occurred in one patient given eplerenone, flushing occurred in one patient given placebo, and anxiety occurred in another patient given placebo. In boys with Duchenne muscular dystrophy and preserved ejection fraction, addition of eplerenone to background ACEI or ARB therapy attenuates the progressive decline in left ventricular systolic function. Early use of available drugs warrants consideration in this population at high risk of cardiac death, but further studies are needed to determine the effect of combination cardioprotective therapy on event-free survival in Duchenne muscular dystrophy. BallouSkies, Parent Project for Muscular Dystrophy, US National Center for Advancing Translational Sciences, and US National Institutes of Health.

Parental engagement projects eligible for share of £1m fund

Grants of up to £200,000 are available to support projects that seek to close the attainment gap in the early years and primary school through engaging parents in their children's learning

Avoir un enfant et accéder au DPN/DPI pour des femmes porteuses d’une mutation BRCA ? Malades et indemnes appréhendent la question différemment

Genetic tests in families with a mutation related to breast and ovarian cancers (BRCA1/2) are now offered to the persons before completion of their reproductive project. The aim of this qualitative study was to descriptively explore how the issues of reproduction are faced in women belonging to these families, and how the possible use of prenatal diagnostic (PND) and preimplantation genetic diagnosis (PGD) would be faced in a theoretical context. We conducted in-depth interviews, face to face, according to the so-called Grounded Theory approach. Twenty women with a BRCA genetic mutation participated in the study (age range: 31-57 years); 12 have had a breast and/or ovarian cancer. The knowledge of having the mutation did not modify the parental project; however prophylactic anexectomy was likely to alter it in some women. If the majority of women were in favor of PGD (n = 14), medical termination of pregnancy was a constraint towards the position in relation to PND. Besides ethical and moral arguments, the women's attitudes were constructed differently according to their own personal or familial experience of the disease. The women's perceptions of the cancer severity, risk and cure were organized according to this experience.

Graduate ethics education: A content analysis of syllabi.

Ethical practice of psychology is emphasized by American Psychological Association (APA) accreditation requirements. The current study is a content analysis of 53 ethics course syllabi from all APA accredited programs listed in the American Psychologist 2011 annual report. This article is a companion to Domenech Rodriguez et al. (2014, pp. 241–247) and contributes knowledge on the current state of graduate ethics education. Of the parent project respondents (n 364), 14% returned syllabi for the present study. General information (e.g., objectives, honor code, academic honesty, common policy, and classroom expectations), assignments, APA format, and teaching sources were coded. Coding of objectives were developed from McKeachie and Svinicki’s (2011) definition and de las Fuentes, Willmuth, and Yarrow’s (2005) conceptualization of specific objectives (awareness, skill, and knowledge). A little more than half (56.6%) of syllabi stated clear objectives, only 52.8% covered academic honesty, and 16 (30.2%) did not include a section to address accommodations for students with disabilities. Most syllabi used APA format to cite sources, however most sources used were 5 years or older. Overall, syllabi were quite varied in the information they included. Greater consistency in syllabus content would make future evaluations on the state of graduate ethics education more accurate. Recommendations for observational and self-report alternatives are also presented.

The Traveller Forum: An Advice-seeking Site Contributing to the Construction of Parental Social Actors and to On-line Parental Bond Creations

This paper studies how parental social actors and co-parental relationships are interactively constructed in traveller forums, analysing a corpus obtained from the London for Kids Trip Advisor forum, and carrying out a qualitative study of the interactants’ uses of kids in their choice of transitivity patterns. Starting from a Critical Discourse Analytical methodological framework and from corpus-driven research, I address the question of how participants are endowed with authority and legitimation in parenting matters while engaged in this advice-seeking site. The results point at the emergence of inter-subjective role creations of parenting social actors, of instances of co-parental intrusion or complicity, whose legitimation and investment with authority often falls back on cultural and in general stereotypical presuppositions. The study is of interest for the tourist industry in general. Offering rich, authentic and often unsolicited customer feedback, forums are recognised to provide a cost-effective method to assess the quality of tourist services and consequently improve travellers’ real demands. Through this research, the tourist industry may become aware of how parents construct and project themselves discursively as consumers of touristic services, and adapt these findings to their marketing interests..

G.P.302: Perceived barriers and facilitators of participation in clinical trials for Duchenne and Becker muscular dystrophy

Introduction: Industry, clinicians, advocacy, and families have a stake in the success of clinical trials (CTs). Understanding decision-makers' perceptions of CTs inform efforts to improve recruitment; this is particularly relevant for rare diseases like Duchenne/Becker muscular dystrophy (DBMD). Methods: The study team employed a community-based participatory research approach. US/Canadian parents of children with DBMD who never participated in a CT were recruited for an online survey through Parent Project Muscular Dystrophy and clinics. Participants answered questions about CT preferences and perceived barriers and facilitators. Results: Among the first 99 participants with children age 4–12, 65% wanted their child in a CT, 32% were unsure, and 3% did not. The most endorsed barriers were: possibility of receiving placebo (46% very or moderately true); insufficient information about risks (27%); the possibility of an unsuccessful trial (25%); child may not benefit (25%); and insufficient information about day-to-day requirements (25%). Parents unsure about CT participation endorsed barriers at notably different frequencies than those wanting participation for: insufficient risk information (55% vs. 13%) and child not benefiting (39% vs. 16%). Highly-endorsed facilitators of increased future CT interest were: guaranteed treatment access after CT (82%); having confidence CT would improve DBMD understanding (76%); agreeing with CT goals (75%); treating doctor's suggestion (72%); perceiving a good researcher reputation (71%). Conclusions: Many facilitators were highly rated for increasing interest in CT participation, supporting adaptive trial design and education and outreach. Parents unsure about participation may have more needs for CT information. The most-endorsed facilitator was treatment access after CT, which is very difficult to achieve. Sponsors should increase outreach through community forums and clinicians to clarify CT processes, personnel, and goals.

G.P.303: Influences on parental decision making about participation in Duchenne and Becker muscular dystrophy clinical trials

Introduction: Parents’ decisions about enrolling children in Duchenne/Becker muscular dystrophy (DBMD) clinical trials (CTs) may be influenced by hopes and expectations, which when unrealistic may challenge informed consent. We explore influences on decision-making. Methods: The study team employed a community-based participatory research approach. Parents of children in CTs in U.S. or Canada were recruited for an online survey through Parent Project Muscular Dystrophy and clinics. Participants viewed a series of benefit and worry statements and rated each statement’s influence on decision-making, and how much they expected and hoped or worried about each. Results: Among the first 61 participants, CT decision-making was influenced by potential altruistic and individual benefits: learning generalizable information (97% slightly to strongly agree), CT resulting in a drug that works (82%), leading to better future for other children (82%), improving quality-of-life for their child (82%), and parent doing everything to help their child (82%). CT worries most affecting decision-making were: child would be bothered by side effects (39%), child’s eligibility for a competing trial (36%), and child not liking the trial (29%). Expectation and hope ratings were highly correlated with ratings of factors that influenced decision-making ( r = 0.5–0.8, p

More Choices For Boys With DMD

Lately, staffers at the advocacy group Parent Project Muscular Dystrophy have been fielding a flurry of phone calls from parents asking for help in making a difficult decision for their children. Two similar treatments for a subset of patients with Duchenne muscular dystrophy, a rare and fatal degenerative disease that affects boys, are suddenly much closer to approval than the DMD community had expected. More clinical studies of the drugs—one each from the biotech firms Sarepta Therapeutics and Prosensa Therapeutics—are opening up, and there’s a real possibility that one or both of the treatments could be on the market next year. Deciding which treatment to try—a choice that PPMD, the advocacy group, can’t make—is agonizing. Parents worry that if they pick the wrong drug, the window for their son to benefit from treatment will close. Moreover, scant data exist to guide them. But it’s a choice nonetheless, and one that ...

Mothers’ Physical Illness in One- and Two-Parent Families

Parental physical illness has been associated with a variety of negative child outcomes but the mechanisms explaining this relationship are not fully understood. Additionally, few studies have explored what role, if any, marital status may play in this relationship. This study used prospective longitudinal survey data from 382 two-parent and 182 single-parent families in the Iowa Youth and Families and Single Parents Project to explore the relationship between maternal illness and parenting with depressed affect as a potential mediator. Multiple group structural equation modeling was used to investigate whether the relationships between these indicators were the same for single and married mothers. Analyses revealed associations between mothers’ illness and parenting for both single and married mothers, but mothers’ depressed affect played a meditational role for single mothers, which was not evident with married mothers. Both the empirical and clinical implications of these findings are discussed.

Ouverture de l’assistance médicale à la procréation avec sperme de donneur (AMPD) à des indications non médicales 1

In France as in other countries, more and more single women and lesbian couples wish to become mothers. To carry through their parenting project they may consult a physician in France and often go abroad in order to get Assisted Reproductive Technologies with donor sperm (ARTD). Should ARTD be available to those women in France? The physician has not to take the decision. In such situations ARTD has no medical indication or contraindication. This assisted procreation raises many questions on children development and well-being. The results of studies made in other countries are often reassuring but their methodologies do not allow any conclusion to be drawn and grey areas persist. Therefore it should be necessary to develop a research effort in the field as it recently started in France. Would ARTD access to women without a male partner be legalized, the law should respect the ethical principles of non-payment and anonymity associated with donation of all body components. In any case, it should also allow an efficient medical care to be performed to ensure under the best conditions the well-being of the children and their mothers.

200th ENMC International Workshop “European Reference Networks: Recommendations and Criteria in the Neuromuscular field”, 18–20 October 2013, Naarden, the Netherlands

200th ENMC International Workshop “European Reference Networks: Recommendations and Criteria in the Neuromuscular field”, 18–20 October 2013, Naarden, the Netherlands

Commitment across the Transition to Parenthood among Married and Cohabiting Couples.

Commitment has long been hypothesized to increase across the transition to parenthood, even though much research has found that relationship functioning declines during this period. We examined change in interpersonal commitment, measured as personal dedication and relationship confidence, and constraint commitment, measured as felt constraint, across the transition to parenthood. We tested for marital status differences in the change in commitment across the transition among three groups: cohabitation, marriage preceded by cohabitation, and direct marriage. Data came from the New Parents Project, a community sample of 173 married and cohabiting couples. Difference-in-difference estimates indicated that cohabiting fathers, in comparison to married fathers, dropped further in personal dedication and relationship confidence and increased more in felt constraint across the transition to parenthood. No significant differences across the transition were found between cohabiting and married mothers. Further research on the transition to parenthood among unmarried couples is suggested.

Zaangażowanie w rodzicielstwo na tle współczesnej rodziny

Parental experiences are integrated into a complex system of cultural, social and economic processes. Dichotomous view of reality in which the high valuation of family and parenthood in Poland follow in the wake of negative demographic indicators contradict presented assessments. So there is a question about causes of the current state and how the issue of functioning in parental roles relates to the postmodernism, for which affirmation of independence and lack of commitment is characteristic. This paper presents an attempt to define involvement in parenting and research project that came as a result of searching an answer to the question about the correlation between parental involvement and self-creation of adults. Because parenting expresses interpersonal dimension of the individual activity, its quality has two-subjective consequences, i.e. it affects both sides of the parent-child relationship. Therefore, the second aspect of reported studies concerns the correlation between parenting involvement and education, recognized in terms of parental attitudes. The results indicate the possibility to notice implications of parental involvement expressed in self-creation experience of adults and desirable condition of child development, as defined mainly by parental attitude of acceptance. Involved parents actively participate in the care and upbringing of children and such activity promotes a sense of self-improvement. The idea of self-creation as a result of the parental role contradicts the attitude defined as freedom from children (called childfree), according to which the care and education of children are the source of limited individual development.

Time 2 tlk 2nite: Use of Electronic Media by Adolescents during Family Meals and Associations with Demographic Characteristics, Family Characteristics, and Foods Served

We examined the frequency of adolescents' use of electronic media (ie, television/movie watching, text messaging, talking on the telephone, listening to music with headphones, and playing with hand-held games) at family meals and examined associations with demographic characteristics, rules about media use, family characteristics, and the types of foods served at meals using an observational, cross-sectional design. Data were drawn from two coordinated, population-based studies of adolescents (Project Eating Among Teens 2010) and their parents (Project Families and Eating Among Teens). Surveys were completed during 2009-2010. Frequent television/movie watching during family meals by youth was reported by 25.5% of parents. Multivariate logistic regression analyses indicated significantly higher odds of mealtime media use (P<0.05) for girls and older teens. In addition, higher odds of mealtime media use (P<0.05) were also seen among those whose parents had low education levels or were black or Asian; having parental rules about media use significantly reduced these odds. Frequent mealtime media use was significantly associated with lower scores on family communication (P<0.05) and scores indicating less importance placed on mealtimes (P<0.001). Furthermore, frequent mealtime media use was associated with lower odds of serving green salad, fruit, vegetables, 100% juice, and milk at meals, whereas higher odds were seen for serving sugar-sweetened beverages (P<0.05). The ubiquitous use of mealtime media by adolescents and differences by sex, race/ethnicity, age, and parental rules suggest that supporting parents in their efforts to initiate and follow-through on setting mealtime media use rules may be an important public health strategy.

Cognitive function after bariatric surgery: evidence for improvement 3 years after surgery

BackgroundBariatric surgery is associated with improved cognition, and it is possible that such improvements are found at extended follow-ups. We hypothesized that cognitive improvement would be maintained 3 years after bariatric surgery. MethodsFifty bariatric patients were recruited from the Longitudinal Assessment of Bariatric Surgery parent project. Participants completed a computerized cognitive test battery to assess cognitive function at 12 weeks, 12 months, 24 months, and 36 months after surgery. ResultsRepeated measures revealed main effects for attention, executive function, and memory. Attention improved up to 24 months and then slightly declined although it still fell within the average range at 36 months. Improvements in executive function reached their peak at 36 months after surgery. Short-term improvements in memory were maintained at 36 months. No main effect emerged for language. ConclusionsBariatric surgery may lead to lasting improvements in cognition. Prospective studies with extended follow-ups (eg, 10 years) should examine whether bariatric surgery can attenuate cognitive decline in severely obese patients.

La procréation médicalement assistée en France : une question d’égalité ?

The equality principle is not stated in French law. It is deduced from a series of rules that provide for an identical treatment of different situations. Applied to the gamete donation, it exploits the female body by placing it under a unique regime of sperm donation, entirely harmless, and ovum donation, significantly more dangerous. Only one requirement ensures the protection of donors: their written consent. This is why some people would like the legislator to validate the gestational surrogacy by presenting it as “gestation donation.” However, this qualification is totally inappropriate for a contractual approach. When applied to infertile heterosexual couples, which the law recognizes as sole receivers, the equality principle ignores the fact that the woman’s body is the sole tool employed in the medical acts required in human reproduction. The parental project promotes the notion of equality imposed on the couple. It allows the biological dimension of reproduction to be ignored by prioritizing consent to use anonymous gamete donation. This consent always takes the form of a genuine consent that, ignoring their unequal participation in the child’s conception, engages the man and the woman—equally—as intended parents. Reproductive services, practised outside the legal context, allow fertile people to conceive by using an anonymous sperm donation or gestational surrogacy in order to deprive the child of a father or a mother. This abuse sheds light on the inequality of situations that are fundamental to the quality of the mother or father. The equality principle does not lead to any kind of equality but, instead, establishes an indisputable mess in parentage due to reproductive services.

Filling the gaps

The aim of this paper is to demonstrate the functionality of the WebCorp Linguist’s Search Engine (WebCorpLSE) by comparing it with two other freely-available online data resources: Google and the Corpus of Contemporary American English (COCA). In particular, the paper looks at how WebCorpLSE can fill gaps in these sources. It begins by contextualising the WebCorpLSE project in relation to its parent project, WebCorp, and other ‘web as corpus’ initiatives. It then outlines the advantages of WebCorpLSE over Google search, before turning to a comparison of WebCorpLSE with COCA. Comparisons are made by matching extracts of textual and graphical output generated by each system. It is shown that WebCorpLSE provides information not available via the other search mechanisms, and that it can thus, in accordance with Mair’s (2007) argument for a combined approach to diachronic text study, usefully serve not only as a stand-alone tool, but as a complementary resource.

P.7.11 The clinic services resource survey on Duchenne Connect

DuchenneConnect (DC) is a self-report registry for Duchenne/Becker muscular dystrophy (DBMD). We present the Clinic Services Resource (Clinic Survey), a new addition to the registry in 2011. The goals of the survey include (1) providing families a resource that helps them understand the services offered by clinics caring for DBMD patients, and (2) providing clinics with data they can use to improve their clinical care and trial readiness. Anonymous results are publically available. The Clinic Survey includes data on clinical care in the US, reported by patients/families and clinic representatives. Survey participants are recruited through DC and Parent Project Muscular Dystrophy; participants do not have to be registered on DC. The survey includes ∼50 questions covering access, satisfaction, medical services, steroid use, movement, cardiac, pulmonary, support and learning/behavior. Questions are based on the care guidelines. Of registrants living in the US, 12% (207) have completed the survey. An additional 44 non-registrants have participated, for a total of 251 completed surveys as of March 2013. More than 60 clinical sites are represented, and the majority are specialty clinics. 20% of patients report never using steroids. Reasons for not taking steroids are explored. 4% report never having an echocardiogram and 23% report never having a pulmonary function test. When asked to compare their clinic to the recommended care, 41% feel it is “way better,” 17% “a little better,” 19% “exactly what is recommended,” 12% “a little below,” and 6% “way below.” The Clinic Survey provides a publically available and searchable resource that reports parent perspectives on clinics across the US. Clinic-specific and overall strengths and areas for improvement are emerging. We expect this resource to grow over time with increased participation, and we anticipate enhancement of the survey to better meet the needs of DBMD families.

Transforming Duchenne Care: Meeting 25–26 June 2012, Ft. Lauderdale, Florida, USA

Fifty-seven attendees representing 17 U.S. academic institutions, in addition to Parent Project Muscular Dystrophy (PPMD), the Centers for Disease Control and Prevention (CDC), Muscular Dystrophy Association (MDA), Cystic Fibrosis Foundation (CFF), National Institutes of Health (NIH), parents and industry, gathered to discuss the care of patients and families living with Duchenne/Becker muscular dystrophy (DBMD). The meeting, sponsored by Parent Project Muscular Dystrophy, was organized in response to clinical care discrepancies that exist between centers treating patients with Duchenne/Becker muscular dystrophy in North America. Currently, a number of care delivery models are in existence, each presenting unique administrative and financial challenges. Given the complexity of the disease, however, a multi-disciplinary care delivery model theoretically seems optimal. The purpose of this conference was to explore the unique clinical needs of the Duchenne/Becker patient and to investigate how centers could best align clinical programs to address these needs.

Cognitive function predicts 24-month weight loss success after bariatric surgery

BackgroundClinically significant cognitive impairment, particularly in attention/executive and memory function, is found in many patients undergoing bariatric surgery. These difficulties have previously been linked to decreased weight loss 12 months after surgery, but more protracted examination of this relationship has not yet been conducted. The present study prospectively examined the independent contribution of cognitive function to weight loss 24 months after bariatric surgery. Given the rapid rate of cognitive improvement observed after surgery, postoperative cognitive function (i.e., cognition 12 weeks after surgery, controlling for baseline cognition) was expected to predict lower body mass index (BMI) and higher percent total weight loss (%WL) at 24-month follow-up. MethodsData were collected by 3 sites of the Longitudinal Assessment of Bariatric Surgery (LABS) parent project. Fifty-seven individuals enrolled in the LABS project who were undergoing bariatric surgery completed cognitive evaluation at baseline, 12 weeks, and 24 months. BMI and %WL were calculated for 24-month postoperative follow-up. ResultsBetter cognitive function 12 weeks after surgery predicted higher %WL and lower BMI at 24 months, and specific domains of attention/executive and memory function were robustly related to decreased BMI and greater %WL at 24 months. ConclusionsResults show that cognitive performance shortly after bariatric surgery predicts greater long-term %WL and lower BMI 24 months after bariatric surgery. Further work is needed to clarify the degree to which this relationship is mediated by adherence to postoperative guidelines.