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Background: When availability and/or affordability of anti-hemophilic factor concentrates are limited, optimal prophylaxis regimens in severe hemophilia A (HA) remain to be determined. In selected situations, low-dose daily prophylaxis (LDDP) may be an effective and economical option. The goal of our study was to evaluate if subjects on a LDDP regimen could achieve adherence and good clinical outcome. Methods: Seventeen subjects (age between 15.2 and 28.4) on LDDP suffering from severe/moderate HA were followed prospectively for 2 to 3 years as part of a health-related quality of life (HRQoL) study. Bleeding and treatments data were collected using electronic diaries and validated every three months. The SF-36 questionnaire was administered at the beginning of the study and then every 6 months until the end of the study. Results: The subjects (mean age 22.0, median 21.9, standard deviation 4.06), were all from a single centre and on LDDP for at least 12 months as part of their routine care before entering the study. Fifteen subjects were prescribed a daily dose of 500 IU factor VIII (FVIII) and 2 subjects received 1000 IU FVIII per day, resulting into a median dose of 7.1 IU/kg/day (ranging from 4 to 13 IU/kg/day) and of 2591 IU/kg/year. Median adherence (the percentage of the prescribed daily dose received) was 84 % (mean 80 %, range 57 % to 94 %) throughout the study. Seventy-six bleeds in the 6 index joints and 51 other types of bleeds were observed throughout the study. The median annualized bleeding rate in joints (ABRjoints) was 0.7 and the median annualized bleeding rate for all bleeds (ABRall) was 1.6. The Physical Component and Mental Component Summary scores of SF-36, and the Hemophilia Joint Health Score were not significantly different over the course of the study (respective medians of 49.8, 52.4 and 16.0 at entry; vs. 52.5, 51.5 and 16.0 upon exit). Conclusions: This prospective longitudinal study in youth and young adults shows that LDDP may be associated with low ABRs, adequate adherence and HRQoL comparable to previously reported. [ABSTRACT FROM AUTHOR]

Background: Aboriginal children experience challenges to their health and well-being, yet also have unique strengths. It has been difficult to accurately assess their health outcomes due to the lack of culturally relevant measures. The Aboriginal Children's Health and Well-Being Measure (ACHWM) was developed to address this gap. This paper describes the validity of the new measure.Methods: We recruited First Nations children from one First Nation reserve in Canada. Participants were asked to complete the ACHWM independently using a computer tablet. Participants also completed the PedsQL. The ACHWM total score and 4 Quadrant scores were expected to have a moderate correlation of between 0.4 and 0.6 with the parallel PedsQL total score, domains (scale scores), and summary scores.Results: Paired ACHWM and PedsQL scores were available for 48 participants. They had a mean age of 14.6 (range of 7 to 19) years and 60.4 % were girls. The Pearson's correlation between the total ACHWM score and a total PedsQL aggregate score was 0.52 (p = 0.0001). The correlations with the Physical Health Summary Scores and the Psychosocial Health Summary Scores were slightly lower range (r = 0.35 p = 0.016; and r = 0.51 p = 0.0002 respectively) and approached the expected range. The ACHWM Quadrant scores were moderately correlated with the parallel PedsQL domains ranging from r = 0.45 to r = 0.64 (p ≤ 0.001). The Spiritual Quadrant of the ACHWM did not have a parallel domain in the PedsQL.Conclusions: These results establish the validity of the ACHWM. The children gave this measure an Ojibway name, Aaniish Naa Gegii, meaning "how are you?". This measure is now ready for implementation, and will contribute to a better understanding of the health of Aboriginal children. [ABSTRACT FROM AUTHOR]

Background: There are emerging opportunities to improve the health of Aboriginal children and youth. The Aboriginal Children's Health and Well-being Measure (ACHWM) was developed to enable Aboriginal communities to obtain group-level data from the perspectives of their children 8 to 18 years of age. The survey was developed in collaboration with children, based on the Medicine Wheel framework. The purpose of this study was to ensure that children and youth interpreted the ACHWM questions consistently and accurately and to establish the face validity of the survey. Methods: Children and parents/caregivers from the Wikwemikong Unceded Indian Reserve (Canada) participated in a detailed interview process as they completed the ACHWM, in 2012. Each participant worked through their thought process verbally, to enable the interviewer to identify questions that were misinterpreted or inconsistently interpreted. Questions were revised based on feedback from the participants, and reviewed with new participants until a stable version was established. The resulting version was reviewed by health care providers and community members to further ensure cultural relevance and face validity within the community. Results: A total of 18 interviews, with 9 children and 9 caregivers, were required to achieve a stable version of the survey. The children ranged in age from 8 to 18 years. Revisions were required for 19 questions. Most of these revisions were minor linguistic changes. In addition, 6 questions were deleted due to consistent problems and 4 questions were created to address gaps identified during the process. Community members confirmed the appropriateness of the measure for their community and communicated their pride in their youth's role in the development of this survey. Conclusions: The result was a 58-question version of the ACHWM that was consistently interpreted and culturally appropriate, and had face validity confirmed by experts from the community, children and their parents/caregivers. The ACHWM is ready to be assessed for relevance to other Aboriginal communities. [ABSTRACT FROM AUTHOR]

Background Immune thrombocytopenia (ITP) is a childhood disorder that is often life-altering for children and their parents. Health related quality of life (HRQL) has never been chronologically monitored in children with ITP. We initiated a prospective study to assess HRQL from diagnosis to six months and define factors that influence this outcome in children with ITP. Methods 73 children with acute ITP aged from 2 to 18 years were prospectively enrolled in the study. According to the presence of bleeding, they were or were not given a 4-day course of corticosteroid treatment. The PedsQL™ 4.0 Generic Core Scale was completed by children and parents upon their inclusion in the study and 6 months after diagnosis. Results Over the six month period, quality of life improved in terms of their global, physical and psychosocial well-being for 54.5 %, 35.6 % and 36.2 % of patients respectively. This improvement is clinically relevant compared to scores at diagnosis, corresponding at least to a minimal clinically important difference (MCID). Factors such as sex, age, platelet count, bleeding scores, bone marrow aspiration and persistence of ITP at 6 months were not significantly associated with HRQL scores. However, preceding viral infection was identified to have an impact on HRQL. Conclusions This first longitudinal study assessing HRQL in children with ITP reveals a global improvement in PedSQL™ 4.0. However, these results should be considered with caution since our data also confirm that self-report HRQL scores are not influenced by any analyzed biologic or clinical parameters. Others tools, such as Kids' ITP Tools, would probably be required to assess the HRQL of this population. [ABSTRACT FROM AUTHOR]

Background: Quality of life (QoL) is increasingly recognized as an important outcome measure in clinical trials. The Canadian Hemophilia Outcomes-Kids Life Assessment Tool (CHO-KLAT) shows promise for use in China. Objective: To adapt the CHO-KLAT version 2.0 for use in clinical trials in China. Methods: Forward and back translations of the CHO-KLAT2.0 were completed in 2008. Between October 2009 and June 2010, a series of 3 focus groups were held with 20 boys and 31 parents in rural and urban China to elicit additional concepts, important to their QoL, for the Chinese CHO-KLAT2.0. All of the items identified by boys and parents were reviewed by a group of experts, resulting in a Chinese version of the CHO-KLAT2.0. This version underwent a detailed cognitive debriefing process between October 2010 and June 2011. Thirteen patient-parent pairs participated in this cognitive debriefing process until a stable and clearly understood Chinese version of the CHO-KLAT2.0 was obtained. Results: The initial back translation of the Chinese CHO-KLAT2.0 was slightly discrepant from the original English version on 12 items. These were all successfully adjudicated. The focus groups identified 9 new items that formed an add-on Socio-Economic Context (SEC) module for China. Linguistic improvements were made after the 2nd, 5th, 7th and 13th cognitive debriefings pairs and affected a total of 18 items. The result was a 35 item CHO-KLAT2.0 and a SEC module in Simplified Chinese, both of which have good content validity. Conclusion: This detailed process proved to be extremely valuable in ensuring the items were accurately interpreted by Chinese boys with hemophilia ages ≤18 years. The need for the additional SEC module highlighted the different context that currently exists in China with regard to hemophilia care as compared to many Western countries, and will be important in tracking progress within both rural and urban China over time. Changes based on the cognitive debriefings suggest that expert verbatim translation alone is not sufficient. The Chinese version of the CHO-KLAT2.0 is well understood by boys with hemophilia in China. Next steps will be to test its construct validity and reliability in boys with hemophilia in China. [ABSTRACT FROM AUTHOR]

Background: Despite the multitude of health challenges faced by adults living with HIV, we know of no HIV-specific instrument developed for the purpose of describing the health-related consequences of HIV, a concept known as disability. In a previous phase of research, adults living with HIV conceptualized disability as symptoms/impairments, difficulties carrying out day-to-day activities, challenges to social inclusion, and uncertainty that may fluctuate on a daily basis and over the course of living with HIV. In this paper, we describe the extent to which existing HIV-specific health-status instruments capture the experience of disability for adults living with HIV. Methods: We searched databases from 1980 to 2006 for English language, HIV-specific, self-reported questionnaires consisting of at least two items that were tested for reliability and validity. We then conducted a content analysis to assess how well existing questionnaires describe disability as defined by the Episodic Disability Framework, a framework that conceptualizes this experience from the perspective of adults living with HIV. We matched items of the instruments with categories of the framework to evaluate the extent to which the instruments capture major dimensions of disability in the framework. Results: We reviewed 4274 abstracts, of which 30 instruments met the inclusion criteria and were retrieved. Of the four major dimensions of disability, symptoms/impairments were included in all 30 instruments, difficulties with day-to-day activities in 16, challenges to social inclusion in 16, and uncertainty in 9. Seven instruments contained at least 1 item from all 4 dimensions of disability (breadth) however, the comprehensiveness with which the dimensions were represented (depth) varied among the instruments. Conclusions: In general, symptoms/impairments and difficulties carrying out day-to-day activities were the disability dimensions characterized in greatest depth while uncertainty and challenges to social inclusion were less well represented. Although none of the instruments described the full breadth and depth of disability as conceptualized by the Episodic Disability Framework, they provide a foundation from which to build a measure of disability for adults living with HIV. [ABSTRACT FROM AUTHOR]

Background: Quality of life (QoL) is increasingly recognized as an important outcome measure in clinical trials. The Canadian Hemophilia Outcomes-Kids Life Assessment Tool (CHO-KLAT) shows promise for use in China., Objective: To adapt the CHO-KLAT version 2.0 for use in clinical trials in China., Methods: Forward and back translations of the CHO-KLAT2.0 were completed in 2008. Between October 2009 and June 2010, a series of 3 focus groups were held with 20 boys and 31 parents in rural and urban China to elicit additional concepts, important to their QoL, for the Chinese CHO-KLAT2.0. All of the items identified by boys and parents were reviewed by a group of experts, resulting in a Chinese version of the CHO-KLAT2.0. This version underwent a detailed cognitive debriefing process between October 2010 and June 2011. Thirteen patient-parent pairs participated in this cognitive debriefing process until a stable and clearly understood Chinese version of the CHO-KLAT2.0 was obtained., Results: The initial back translation of the Chinese CHO-KLAT2.0 was slightly discrepant from the original English version on 12 items. These were all successfully adjudicated. The focus groups identified 9 new items that formed an add-on Socio-Economic Context (SEC) module for China. Linguistic improvements were made after the 2nd, 5th, 7th and 13th cognitive debriefings pairs and affected a total of 18 items. The result was a 35 item CHO-KLAT2.0 and a SEC module in Simplified Chinese, both of which have good content validity., Conclusion: This detailed process proved to be extremely valuable in ensuring the items were accurately interpreted by Chinese boys with hemophilia ages ≤18 years. The need for the additional SEC module highlighted the different context that currently exists in China with regard to hemophilia care as compared to many Western countries, and will be important in tracking progress within both rural and urban China over time. Changes based on the cognitive debriefings suggest that expert verbatim translation alone is not sufficient. The Chinese version of the CHO-KLAT2.0 is well understood by boys with hemophilia in China. Next steps will be to test its construct validity and reliability in boys with hemophilia in China.

Background: Despite the multitude of health challenges faced by adults living with HIV, we know of no HIV-specific instrument developed for the purpose of describing the health-related consequences of HIV, a concept known as disability. In a previous phase of research, adults living with HIV conceptualized disability as symptoms/impairments, difficulties carrying out day-to-day activities, challenges to social inclusion, and uncertainty that may fluctuate on a daily basis and over the course of living with HIV. In this paper, we describe the extent to which existing HIV-specific health-status instruments capture the experience of disability for adults living with HIV., Methods: We searched databases from 1980 to 2006 for English language, HIV-specific, self-reported questionnaires consisting of at least two items that were tested for reliability and validity. We then conducted a content analysis to assess how well existing questionnaires describe disability as defined by the Episodic Disability Framework, a framework that conceptualizes this experience from the perspective of adults living with HIV. We matched items of the instruments with categories of the framework to evaluate the extent to which the instruments capture major dimensions of disability in the framework., Results: We reviewed 4274 abstracts, of which 30 instruments met the inclusion criteria and were retrieved. Of the four major dimensions of disability, symptoms/impairments were included in all 30 instruments, difficulties with day-to-day activities in 16, challenges to social inclusion in 16, and uncertainty in 9. Seven instruments contained at least 1 item from all 4 dimensions of disability (breadth) however, the comprehensiveness with which the dimensions were represented (depth) varied among the instruments., Conclusions: In general, symptoms/impairments and difficulties carrying out day-to-day activities were the disability dimensions characterized in greatest depth while uncertainty and challenges to social inclusion were less well represented. Although none of the instruments described the full breadth and depth of disability as conceptualized by the Episodic Disability Framework, they provide a foundation from which to build a measure of disability for adults living with HIV.

Background: Disease-specific quality of life (QoL) measures have enhanced the capacity of outcome measures to evaluate subtle changes and differences between groups. However, when the specific disease is rare, the cohort of patients is small and international collaboration is often necessary to accomplish meaningful research. As many of the QoL measures have been developed in North American English, they require translation to ensure their usefulness in a multi-cultural and/or international society. Published guidelines provide formal methods to achieve cross-culturally comparable versions of a QoL tool. However, these guidelines describe a rigorous process that is not always feasible, particularly in rare disease groups. The objective of this manuscript is to describe the process that was developed to achieve accurate cross-cultural translations of a disease-specific QoL measure, to overcome the challenges of a small sample size, i.e. children with a rare disorder., Procedure: A measurement study was conducted in the United Kingdom (UK), France, Germany and Uruguay, during which the validated measure was translated into the languages of the respective countries., Results: This is a report of a modified, child-centric, cross-cultural translation and adaptation process in which culturally appropriate and methodologically valid translations of a disease-specific QoL measure, the Kids' ITP Tools (KIT), were performed in children with immune thrombocytopenic purpura (ITP). The KIT was translated from North American English into UK English, French, German, and Spanish., Conclusion: This study was a successful international collaboration. The modified process through which culturally appropriate and methodologically valid translations of QoL measures may be achieved in a pediatric population with a relatively rare disorder is reported.

Background: Since the advent of combination antiretroviral therapy, in developed countries HIV increasingly is perceived as a long-term illness. Individuals may experience health-related consequences of HIV and its associated treatments, a concept that may be termed disability. To date, a comprehensive framework for understanding the health-related consequences experienced by people living with HIV has not been developed. The purpose of this research was to develop a conceptual framework of disability from the perspective of adults living with HIV., Methods: We conducted four focus groups and 15 face-to-face interviews with 38 adults living with HIV. We asked participants to describe their health-related challenges, their physical, social and psychological areas of life affected, and impact on their overall health. We analyzed data using grounded theory techniques. We also conducted two validity check focus groups with seven returning participants., Results: Disability was conceptualized by participants as multi-dimensional and episodic characterized by unpredictable periods of wellness and illness. The Episodic Disability Framework consisted of three main components: a) dimensions of disability that included symptoms and impairments, difficulties carrying out day-to-day activities, challenges to social inclusion, and uncertainty that may fluctuate on a daily basis and over the course of living with HIV, b) contextual factors that included extrinsic factors (social support and stigma) and intrinsic factors (living strategies and personal attributes) that may exacerbate or alleviate disability, and c) triggers that initiate momentous or major episodes of disability such as receiving an HIV diagnosis, starting or changing medications, experiencing a serious illness, and suffering a loss of others., Conclusion: The Episodic Disability Framework considers the variable nature of disability, acknowledges uncertainty as a key component, describes contextual factors that influence experiences of disability, and considers life events that may initiate a major or momentous episode. This framework presents a new way to conceptualize disability based on the experience of living with HIV.