Utility valuation of health states for haemophilia and related complications in Europe and in the United States.

INTRODUCTION: There is currently a paucity of health utility data describing the consequences of haemophilia and related complications. AIM: To quantify the impact of distinct stages of severity of haemophilia and disease-related complications on health-related quality of life, expressed as health utilities in Europe and the United States. METHODS: Nine health state descriptions were developed based on literature review and interviews with haematologists and haemophilia patients. Three descriptions characterized the impact of mild, moderate and severe haemophilia without inhibitors. Six descriptions characterized disease-related complications added to the moderate haemophilia description (arthroscopic synovectomy, prosthetic joint replacement, chronic pain, spontaneous bleed, traumatic bleed and end-stage joint disease). Time trade-off (TTO) interviews were conducted with 100 adults from the general public in the UK, France, Germany, Italy, Sweden and the United States. Mean TTO-derived utility values were expressed on a scale from 0 (death) to 1 (full health). RESULTS: Utility values obtained for the health states corresponding to mild (0.73-0.86), moderate (0.68-0.76) and severe (0.64-0.71) haemophilia followed the increase in severity. The addition of a complication to the "moderate" state leads to a decrease in the associated utility value. The most severe disutility (0.23-0.36) across all countries was associated with the burden of end-stage joint disease. CONCLUSIONS: This study underlines the value that the French, Italian, German, Swedish, United States and UK populations ascribe to the avoidance of disease progression in haemophilia without inhibitors. Improved treatment options hold a potential for important benefits to haemophilia patients.

Test-retest properties of the Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire and its constituent domains.

BACKGROUND: The Patient Reported Outcomes, Burdens and Experiences (PROBE) study aims to develop and validate questionnaire for assessing health status in patients with haemophilia and participants without bleeding disorders. OBJECTIVE: To investigate the test-retest properties of the PROBE questionnaire. METHODS: The PROBE questionnaire covers four domains and is comprised of 29 questions. People with haemophilia (PWH) and participants without bleeding disorder were invited to participate in this study. All participants were asked to complete the PROBE questionnaire three times (paper-based survey on two consecutive days: T1 and T2 and then a web-based version: T3). Test-retest properties and percentage agreement were analysed. RESULTS: A total of 63 participants were enrolled in this study with a median age of 50 (range: 17-76) years. Of these, 30 (47.6%) were PWH. On the questions common to PWH and participants without bleeding disorder, Kappa coefficients ranged from 0.69 to 1.00, indicating substantial to almost perfect agreement (T1 vs T2). For haemophilia-related questions (T1 vs T2), Kappa coefficients ranged from 0.5 to 1.0. Of these, 5 of 11 items were in perfect agreement (Kappa = 1.0). The web-based questionnaire (T3) showed substantial to almost perfect agreement with the paper version (T1 test-retest properties were comparable between PWH and individuals without a bleeding disorder). CONCLUSIONS: The results suggest that PROBE is a reliable tool to assess patient-reported outcomes for PWH and benchmark data in participants without bleeding disorder. The web-based questionnaire and the standard paper-based version can be used interchangeably.

Use of telehealth in the delivery of comprehensive care for patients with haemophilia and other inherited bleeding disorders.

Advances in technology such as telemedicine (TM) have made access to cost-effective, quality health care feasible for remote patients. TM is especially well suited for patients with chronic disorders such as haemophilia and related haemostatic disorders that benefit not only from more frequent interaction with care providers at a specialized haemophilia treatment center but also from consultations with other specialists. Telehealth refers to a broader application of TM and includes non-clinical services such as education, provider training, administrative meetings etc. Collaboration with the local primary care provider for management and implementation is key for successful and sustainable TM. This review article provides an overview of types of telemedicine, technical aspects, its benefits and challenges and focuses on the applicability of this technology to persons with bleeding and other blood disorders. Examples of TM strategies, process flow of TM clinic and experiences at the authors haemophilia treatment center (HTC) setting are shared. In addition, mobile health (mHealth) and electronic health (eHealth), both a part of telehealth, and their applications are briefly described. Clearly, widespread adoption of this technology will not only enhance care of patients but will enable more people, especially in underserved areas, to receive specialty care.

Bullying in medically fragile youth: a review of risks, protective factors, and recommendations for medical providers.

Bullying is a common child and adolescent phenomenon that has concurrent and long-term implications for victims' psychological, psychosomatic, social, and academic functioning. Youth with chronic illnesses are at increased risk for being bullied, but few studies have evaluated specific risk and protective factors for medically fragile youth. Despite recommendations by the American Academy of Pediatrics and the Society for Adolescent Medicine that pediatric health care providers should contribute to bullying prevention and intervention efforts, researchers also have yet to identify the best ways for providers to intervene with medically fragile youth. In this article, the authors review risk and protective factors for bullying among healthy samples. Then, the authors specifically address the ways in which these risk and protective factors are likely to apply to children with fragile medical conditions, and they provide summaries of extant bullying research for selected examples of medically fragile pediatric populations. Finally, the authors present recommendations for intervening with medically fragile youth and suggest several areas in which additional research is needed.

The experience of girls and young women with inherited bleeding disorders.

Haemophilia carriers and women with inherited bleeding disorders (IBD) experience menorrhagia, bleed following dentistry, surgery, injury or childbirth. Symptoms are easily treated leading to full and active lives. Nevertheless, some girls and women suffer with abnormal bleeding for many years before diagnosis. We explored the experiences of girls and young women (aged 9-34 years) with IBD by means of focus groups which consisted of moderated discussion addressing specific aspects of bleeding, management and coping strategies. Subsequently, these issues were explored further though a paper-based questionnaire distributed via five specialist haemophilia centres in the UK. The study suggested that young women with IBD who are managed at haemophilia centres receive appropriate care and feel well supported. Although the clinic-based literature available to these women is "fit for purpose", it does not fully address the perceived needs specifically regarding sex, menorrhagia, conception and childbirth, the Pill, tattoos/piercings and so on, leading many to turn to other information sources. Most of those who responded to our survey are confident in their lives, able to manage their IBD and take pragmatic views towards the inherited nature of their condition. But there is a substantial subgroup of women who experience stigmatization, isolation and bullying and express concerns relating to fertility and conception. Overall, this cohort would benefit from opportunities for mutual support. This could be via Internet-based social networking and may be of particular value to those who are unable to seek help from traditional medical services due to religious or other cultural barriers.

Dental health and oral health-related quality of life in children with congenital bleeding disorders.

The purpose of this study was to investigate the dental and some other aspects of oral health status of young patients with congenital bleeding disorders (CBD) and the impact of these on their quality of life (OHR-QoL) compared with controls. DMFS-dmfs (Decayed, Missed, Filled Tooth surfaces in permanent and primary teeth) scores, Simplified oral hygiene index, occurance of hypoplasia of first permanent molars, Temporomandibular joint dysfunction and occlusion of 46 CBD patients at the age range of 2-15 years and 46 of other children as control were compared, and the impact of their oral health situation on quality of life was also investigated. Data were analysed by chi-square, t-test and Pearson correlation. Patients were significantly more caries-free with less decayed teeth in primary-permanent dentition (P = 0.03, t = -2.17).The mean scores of OHR-QoL of CBD patients and controls were not significantly different. Oral Bleeding was the significant variable in relation to 'oral health-related quality of life' in CBD groups (Pearson correlation, r = -0.56, P = 0.000). OHR-QoL in the control group was related to dmfs score (r = -0.392, P = 0.011) and male gender (r = -0.329, P = 0.026). Congenital bleeding disorder CBD patients were found to have a better dental health situation in primary dentition compared with controls; however, their 'oral health-related quality of life' was similar. Oral bleeding was the only significant factor related to OHR-QoL in CBD. It shows an overall importance of development of comprehensive care centres for CBD as the main cause of this achievement.

The long-term psychological management of women and girls with inherited bleeding disorders.

A woman with an inherited bleeding disorder faces two main challenges: managing her symptoms medically and integrating her condition into her daily life. Health professionals have an obligation to support young girls and women affected with these disorders as they negotiate the life-cycle transition of their condition. This support should include helping women to integrate their diagnosis into a new sense of self. The psychological effects of menorrhagia can also be addressed by working with key family members such as a young patient's mother or a woman's partner to prevent the experience of body shame. Couples require support to sustain their sexual relationship and to develop communal coping strategies as they face the challenges that menorrhagia brings to their relationship. These may include both fertility and pregnancy issues. Maintaining contact with the medical team can benefit women approaching the menopause by preparing them psychologically for the change and its associated symptoms.

How to deal with medical and social aspects of bleeding disorders--preparing women and the family in developing countries.

There is a considerable number of women with inherited bleeding disorders in Iran. von Willebrand disease, Glanzman thrombasthenia and factor XIII deficiency are the most common coagulation disorders. The main cause of this high rate of coagulation disorders is attributed to a high rate of consanguineous marriages in Iran. Medical care continues to improve for individuals affected with coagulation disorders in Iran. However, these disorders continue to have a significant impact on the affected Iranian women. As a result of the hereditary nature of these disorders, the impact extends to the psychosocial dimension of the lives of the women. Therefore it is recommended that women with coagulation disorders are provided with psychological and social support along with coagulation therapy.

The burden of HCV treatment in patients with inherited bleeding disorders.

Many patients with inherited bleeding disorders are infected with hepatitis C virus (HCV). Antiviral treatment, consisting of pegylated interferon and ribavirin, has many side-effects. The aim of the study was to prospectively assess the occurrence and course of side-effects and changes in health-related quality of life (HRQoL) during antiviral treatment in patients with inherited bleeding disorders and chronic HCV. Forty-seven patients were followed during antiviral treatment. Side-effects of treatment were recorded, and the Beck Depression Inventory and the RAND-36 HRQoL questionnaire were administered at regular intervals. Frequently reported side-effects were fatigue (100%), headache (94%), pruritus and skin rash (94%), concentration problems (89%), decreased appetite (89%), fever, irritability and hair loss (all 85%). Many side-effects disappeared soon after end of treatment, but 4 weeks after cessation fatigue, concentration problems and sleeping problems were still present in more than 30% of patients. Dose reduction was necessary in 21 patients (45%), mostly because of decreasing weight or haemoglobin levels. Two patients stopped treatment prematurely because of side-effects. Depression was present in 28 patients (60%). HRQoL decreased significantly during treatment in all RAND-36 domains, and increased again within 4 weeks after treatment. Major side-effects were similar in patients with successful (n = 31, 66%) and unsuccessful antiviral treatment. In patients with inherited bleeding disorders and chronic HCV, antiviral treatment has many, but mostly transient side-effects and a significant impact on quality of life. Careful follow-up and management of side-effects will ensure optimal compliance and treatment results.

Occurrence, course and risk factors of depression during antiviral treatment for chronic hepatitis C in patients with inherited bleeding disorders: a prospective study.

Treatment of hepatitis C virus (HCV) consists of pegylated interferon (IFN)-alpha and ribavirin for 24 or 48 weeks. An important side-effect of IFN-alpha is depression. The occurrence, course and risk factors of depression during antiviral treatment were studied prospectively in HCV patients with inherited bleeding disorders. The Beck Depression Inventory, indicating no, mild, moderate or severe depression, was administered to 47 patients before starting therapy, after 4, 12, 24 and 48 weeks of treatment, and 4 weeks after cessation of therapy. At baseline, five patients (11%) had mild depression. Depression worsened during treatment in three of these patients. In all five patients, (mild) depression persisted 4 weeks after treatment. Of the remaining 42 patients, 23 (55%) developed depression during treatment (14 mild, eight moderate and one severe), mostly (78%) during the first 12 weeks. Four weeks after cessation of treatment, three of 23 patients still had mild depression. The only independent risk factor for development of depression was a history of depression or other psychiatric problems (odds ratio 9.7). For patients with inherited bleeding disorders, depression is a significant, mostly transient, problem during HCV treatment. We recommend close monitoring of patients, especially those with previous psychiatric problems, to ensure adequate detection and treatment of depression during antiviral therapy.

Health-related hardiness in individuals with chronic illnesses.

The aims of this study were to (a) describe health stressors, health-related hardiness, perception of illness impact, self-perception of health status, and psychosocial adjustment to illness in individuals living with an inherited bleeding disorder; (b) determine relationships between health stressors, health-related hardiness, perception of illness impact, self-perception of health status, and psychosocial adjustment to illness; and (c) determine if perception of illness impact had a direct and/or mediating effect on the relationship between health stressors, health-related hardiness, and self-perception of health status and psychosocial adjustment to illness. A cross-sectional survey design was used. Sixty participants composed the sample. Participants completed five questionnaires. Higher health stressors were associated with higher perception of illness impact, lower perception of health status, and poorer psychosocial adjustment to illness. Individuals with higher perception of illness impact were associated with lower self-perception of health status and poorer psychosocial adjustment to illness.

Assessing quality of life in individuals with hereditary blood coagulation disorders.

The aim of this study was to test the reliability and validity of the SF-36 questionnaire among the patients with hereditary blood coagulation disorders, to compare their quality of life (QoL) to that of healthy controls, and to identify the dimensions of life the patients consider most important. Results showed that the SF-36 questionnaire had good internal consistency reliability and construct and known group validity in individuals with hereditary blood coagulation disorders. Leisure activities/hobbies, availability of work/ study, followed by relationships with other people, own health and relationships with family/relatives appeared most frequently across the patients' and controls' priority ranks. The areas affected most by the disease were financial security, own health and relationships with family/relatives. A comparison of standardized scale scores suggests that blood coagulation disorders are diseases with a predominantly physical impact. Patients with blood coagulation disorders had health-related quality of life that was lower in most domains compared to healthy controls. However, when a wider concept of QoL was applied no differences between the patients' and controls' perceived QoL could be noted.