Validation of the SF-36 in patients with endometriosis.

OBJECTIVES: Endometriosis presents with significant pain as the most common symptom. Generic health measures can allow comparisons across diseases or populations. However, the Medical Outcomes Study Short Form 36 (SF-36) has not been validated for this disease. The goal of this study was to validate the SF-36 (version 2) for endometriosis. METHODS: Using data from two clinical trials (N = 252 and 198) of treatment for endometriosis, a full complement of psychometric analyses was performed. Additional instruments included a pain visual analog scale (VAS); a physician-completed questionnaire based on patient interview (modified Biberoglu and Behrman--B&B); clinical global impression of change (CGI-C); and patient satisfaction with treatment. RESULTS: Bodily pain (BP) and the Physical Component Summary Score (PCS) were correlated with the pain VAS at baseline and over time and the B&B at baseline and end of study. In addition, those who had the greatest change in BP and PCS also reported the greatest change on CGI-C and patient satisfaction with treatment. Other subscales showed smaller, but significant, correlations with change in the pain VAS, CGI-C, and patient satisfaction with treatment. CONCLUSIONS: The SF-36--particularly BP and the PCS--appears to be a valid and responsive measure for endometriosis and its treatment.

Potential reduction in neural tube defects associated with use of Metafolin-fortified oral contraceptives in the United States.

OBJECTIVE: The objective of the study was to estimate the potential reduction of neural tube defects (NTDs) through the use of Metafolin-fortified oral contraceptives (OCs) in the United States. STUDY DESIGN: A population-based decision analytic model was developed to estimate the benefits of increased red blood cell (RBC) folate levels through the use of Metafolin-fortified OCs on NTD risk during pregnancy. We modeled women who began the year taking Metafolin-fortified or traditional OCs. Folate levels were derived from the National Health and Nutrition Examination Survey and clinical trial data. NTD risk was estimated by applying a published risk equation to respective RBC folate levels. RESULTS: The number of predicted NTD cases declined by 23.7% to 31.4%, depending on median baseline folate levels in women taking a fortified OC compared with taking a traditional OC. CONCLUSION: Metafolin-fortified OCs have the potential to reduce the number of folate-dependent NTDs among current and recent OC users.

Economic burden of neural tube defects and impact of prevention with folic acid: a literature review.

Neural tube defects (NTDs) are the second most common group of serious birth defects. Although folic acid has been shown to reduce effectively the risk of NTDs and measures have been taken to increase the awareness, knowledge, and consumption of folic acid, the full potential of folic acid to reduce the risk of NTDs has not been realized in most countries. To understand the economic burden of NTDs and the economic impact of preventing NTDs with folic acid, a systematic review was performed on relevant studies. A total of 14 cost of illness studies and 10 economic evaluations on prevention of NTDs with folic acid were identified. Consistent findings were reported across all of the cost of illness studies. The lifetime direct medical cost for patients with NTDs is significant, with the majority of cost being for inpatient care, for treatment at initial diagnosis in childhood, and for comorbidities in adult life. The lifetime indirect cost for patients with spina bifida is even greater due to increased morbidity and premature mortality. Caregiver time costs are also significant. The results from the economic evaluations demonstrate that folic acid fortification in food and preconception folic acid consumption are cost-effective ways to reduce the incidence and prevalence of NTDs. This review highlights the significant cost burden that NTDs pose to healthcare systems, various healthcare payers, and society and concludes that the benefits of prevention of NTDs with folic acid far outweigh the cost. Further intervention with folic acid is justified in countries where the full potential of folic acid to reduce the risk of NTDs has not been realized.

Defining a minimal clinically important difference for endometriosis-associated pelvic pain measured on a visual analog scale: analyses of two placebo-controlled, randomized trials.

BACKGROUND: When comparing active treatments, a non-inferiority (or one-sided equivalence) study design is often used. This design requires the definition of a non-inferiority margin, the threshold value of clinical relevance. In recent studies, a non-inferiority margin of 15 mm has been used for the change in endometriosis-associated pelvic pain (EAPP) on a visual analog scale (VAS). However, this value was derived from other chronic painful conditions and its validation in EAPP was lacking. METHODS: Data were analyzed from two placebo-controlled studies of active treatments in endometriosis, including 281 patients with laparoscopically-confirmed endometriosis and moderate-to-severe EAPP. Patients recorded EAPP on a VAS at baseline and the end of treatment. Patients also assessed their satisfaction with treatment on a modified Clinical Global Impression scale. Changes in VAS score were compared with patients' self-assessments to derive an empirically validated non-inferiority margin. This anchor-based value was compared to a non-inferiority margin derived using the conventional half standard deviation rule for minimal clinically important difference (MCID) in patient-reported outcomes. RESULTS: Anchor-based and distribution-based MCIDs were-7.8 mm and-8.6 mm, respectively. CONCLUSIONS: An empirically validated non-inferiority margin of 10 mm for EAPP measured on a VAS is appropriate to compare treatments in endometriosis.

Health-related quality of life is compromised in individuals with spina bifida: results from qualitative and quantitative studies.

Spina bifida (SB) accounts for approximately 90% of total neural tube defects. According to the degree of SB severity, the range of associated symptoms and complications varies greatly. Given the complexity and diversity of these complications, individuals with SB might suffer from livelong impairment. This review presents an overview of the impact of SB on patients' health-related quality of life (HRQoL) and explores results from published quantitative and qualitative studies regarding the HRQoL impact of SB on patients, as well as comparing results of existing studies to national norms. A literature search using three electronic databases PUBMED, PsycINFO, and Embase was performed to identify relevant studies dating from January 1976 to November 2010. To satisfy the initial inclusion criteria, articles had to contain studies that were specific to HRQoL in patients with SB. Findings highlight that HRQoL is significantly impaired in patients with SB. Making sure that the public is aware of the disease and how to minimise the risk of NTD, such as SB (e.g., through adequate folate levels at time of conception, etc.) is essential to ensure that fewer individuals face the burden of NTDs in the future.

A review of the social, psychological, and economic burdens experienced by people with spina bifida and their caregivers.

INTRODUCTION: Despite measures to reduce the incidence of neural tube defects (NTDs), the rate of decline has not been as dramatic as expected. At least 300,000 newborns worldwide are known to be affected by NTDs each year. This comprehensive literature review summarizes the human and economic burden of NTDs to patients and caregivers, with particular focus on spina bifida (SB). METHODS: PubMed, PsycINFO, and Embase were searched for studies from January 1976 to November 2010 that included clinical terms, such as NTD, and at least one patient-reported outcome or cost term. A conceptual model was also developed. RESULTS: Areas of peoples' lives affected by SB included physical and role functioning, activities of daily living, bodily pain, vitality, emotional functioning, mental health, self-esteem, self-image, social functioning, relationships, and sexual functioning. Areas of caregivers' lives affected included activities of daily living, work impact, time consumption, parental responsibilities (including responsibilities to other children), confidence, feelings and emotions, mental health, stress, social impact, psychological adjustment, relationships (with SB child, siblings, other family members), social support, coping strategies, and termination decisions. Cost burdens on patients and caregivers also include out-of-pocket costs, lost wages, or household production due to increased morbidity and mortality, transportation and other nonmedical costs. CONCLUSIONS: This review highlights the need to provide care and support to individuals with SB and their caregivers. Results also emphasize the importance of effective long-term public health campaigns and/or newer strategies to prevent NTDs, such as SB.

The impact of spina bifida on caregivers.

INTRODUCTION: Neural tube defects (NTDs) are the second most common birth defects. Spina bifida (SB) and anencephaly make up approximately 90% of total NTDs. Given the number of infants born with an NTD each year, anyone who provides unpaid care for the child (especially caregivers) is affected. This literature review explores the humanistic burden on caregivers of people with SB, specifically myelomeningocele. METHODS: A search using PubMed, PsycINFO, and Embase was performed to find studies from 1976 to 2010. Interpretative phenomenological analysis was performed on qualitative data and relevant extracts from the data were collated to form master themes. RESULTS: A total of 168 abstracts met the inclusion and exclusion criteria. Of these, 25 articles related to caregivers of individuals with SB. Four master themes emerged: initial diagnosis, living with an individual with SB, social support, and coping. Different aspects of caregivers' lives were found to be affected by caring for a child with SB, including activities of daily living, work impact, time consumption, parental responsibilities, confidence, feelings and emotions, mental health, stress, social impact, psychological adjustment, and relationships. CONCLUSION: NTDs, such as SB, present a multitude of issues to caregivers. Issues that affect caregivers of individuals with SB must be addressed in order to reduce the considerable burden that SB places on the caregiver. Continued and enhanced support from health services and patient advocacy groups is needed. For example, providing additional information, support, and empathy can help parents prepare themselves for dealing with the needs of a child with SB over their lifetime.