Abstract Introduction The European Haemophilia Consortium (EHC) Think Tank Workstream on Access Equity aims to identify and address the key challenges to ensuring access equity to healthcare services for people with rare diseases. In this context, access refers to the ability to benefit from any given aspect of treatment and care; equity refers to being ‘fair and impartial’ in providing access. Identifying key challenges At the first virtual workshop of the Access Equity Workstream, participants representing a wide range of stakeholders, including healthcare providers, patient groups, research, and industry, shared their perspectives to identify the key challenges to achieving access equity. It was agreed to prioritise three challenges: 1. Patient journey and pathways; 2. Behavioural change, mindsets and incentives; 3. Budget and resources. The Iceberg Model was used to identify the factors (events, patterns, structures, and mental models) which should be prioritised for future discussions about potential interventions. Summary From an access equity viewpoint, the key events in the patient journey and pathways that need to be addressed are access to diagnosis, screening and treatment, taking account of the considerable national, regional and local variation in the availability of specialist expertise, genetic testing and counselling. There will be debate about sharing limited budgets to treat small numbers of people with rare disorders with gene therapy, compared to treating much larger numbers of patients with common diseases with cheaper drugs. In terms of behavioural change, mindset and incentives, there is a misalignment of personal, collective, organisational and national needs. Among providers, the ‘savings mindset’ is counterproductive for access equity, and short-termism may result in a restricted allocation of funding for innovative and preventive therapies. Industry mindset is largely transactional instead of outcomes-based and the patient community tends to focus on specific diseases instead of the wider implications for the provision of healthcare services and for society. Budgets and resources are key to access equity; reduced/delayed access to expensive new drugs for rare diseases such as haemophilia is a major issue. Cost-effectiveness prioritisation tools used for health technology assessments (HTAs) do not take into account data from small populations, leading to cost-effectiveness ratios that exceed accepted thresholds, and treatments restricted to subgroups of patients within an approved indication. Benchmarking international pricing, service centralisation and cross-border cooperation for delivery of gene therapy need to be addressed if access equity is to be achieved.
{"title":"Access equity: key questions and challenges - A report from the 1st workshop of the European Haemophilia Consortium (EHC) Think Tank Workstream on Access Equity","authors":"Naja Skouw-Rasmussen, L. Savini","doi":"10.2478/jhp-2023-0011","DOIUrl":"https://doi.org/10.2478/jhp-2023-0011","url":null,"abstract":"Abstract Introduction The European Haemophilia Consortium (EHC) Think Tank Workstream on Access Equity aims to identify and address the key challenges to ensuring access equity to healthcare services for people with rare diseases. In this context, access refers to the ability to benefit from any given aspect of treatment and care; equity refers to being ‘fair and impartial’ in providing access. Identifying key challenges At the first virtual workshop of the Access Equity Workstream, participants representing a wide range of stakeholders, including healthcare providers, patient groups, research, and industry, shared their perspectives to identify the key challenges to achieving access equity. It was agreed to prioritise three challenges: 1. Patient journey and pathways; 2. Behavioural change, mindsets and incentives; 3. Budget and resources. The Iceberg Model was used to identify the factors (events, patterns, structures, and mental models) which should be prioritised for future discussions about potential interventions. Summary From an access equity viewpoint, the key events in the patient journey and pathways that need to be addressed are access to diagnosis, screening and treatment, taking account of the considerable national, regional and local variation in the availability of specialist expertise, genetic testing and counselling. There will be debate about sharing limited budgets to treat small numbers of people with rare disorders with gene therapy, compared to treating much larger numbers of patients with common diseases with cheaper drugs. In terms of behavioural change, mindset and incentives, there is a misalignment of personal, collective, organisational and national needs. Among providers, the ‘savings mindset’ is counterproductive for access equity, and short-termism may result in a restricted allocation of funding for innovative and preventive therapies. Industry mindset is largely transactional instead of outcomes-based and the patient community tends to focus on specific diseases instead of the wider implications for the provision of healthcare services and for society. Budgets and resources are key to access equity; reduced/delayed access to expensive new drugs for rare diseases such as haemophilia is a major issue. Cost-effectiveness prioritisation tools used for health technology assessments (HTAs) do not take into account data from small populations, leading to cost-effectiveness ratios that exceed accepted thresholds, and treatments restricted to subgroups of patients within an approved indication. Benchmarking international pricing, service centralisation and cross-border cooperation for delivery of gene therapy need to be addressed if access equity is to be achieved.","PeriodicalId":372940,"journal":{"name":"The Journal of Haemophilia Practice","volume":"10 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"122270733","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Bhabani Sankar Dhal, Anupama Dutta, A. Das, Dhruba Borpatragohain, A. Sharma, Arnav Kashyap
Abstract Background Despite the availability of factor replacement therapy, including prophylaxis, to treat and prevent bleeding, haemophilic arthropathy continues to be the most common complication of haemophilia and significantly impairs the quality of life in people with haemophilia (PwH). Regular periodic assessment of joint status in PwH is essential to identify early arthropathic changes and prevent the development or progression of haemophilic arthropathy. Kinematic and kinetic assessment are preferable and MRI is the gold standard for diagnosing haemophilic arthropathy, but availability is limited in developing countries. HJHS and ultrasound in conjunction with HEAD-US have been shown to effective in assessing changes indicating arthropathy. Aim This study is designed to identify which radiological assessment tool(s) works best when integrated with clinical assessment tools to examine joint health in PwH, with the aim of establishing a cheap, effective, multimodal approach to joint assessment in clinical practice in low resource settings which can then help to guide treatment. Methods A cross-sectional observational study was carried out among PwH attending a clinic in the department of medicine at Assam Medical College and Hospital, Northeast India, over a one-year time period. The elbow, knee and ankle joints of participants were assessed clinically and functionally using HJHS and FISH and radiologically by Pettersson (X-ray) and HEAD-US (ultrasound) scoring. The resulting data was analysed statistically to ascertain correlation between these parameters. Results Sixty-seven PwH were enrolled in the study, with a mean age of 21.69±8.24 years (median 21 years); 38 (57.72%) had severe factor deficiency. The majority (71.70%) presented with joint bleeds; 40 (59.70%) had the knee as a target joint, followed by the elbow (11; 16.41%) and ankle (6; 8.95%). Mean ABR (annual bleed rate), AJBR (annual joint bleed rate) and ATJBR (annual target joint bleed rate) values had a positive correlation with increased severity of factor deficiency (p <0.001). FISH score increased with factor activity level; HJHS, HEAD-US and Pettersson scores decreased with factor activity level. Significant positive correlation was established between Pettersson score, age and ABR. The degree of association was greatest between HJHS and HEAD-US at 60.62%; between HJHS and Pettersson it was 57.74%. Statistically significant negative correlation was established between FISH and HEAD-US. Conclusion A combined multimodal approach using the HJHS, FISH and HEAD-US scoring systems can provide a cheap, quick, more reliable and accurate option for assessing haemophilic joints, with minimal inter and intra observer reliability. This can support the optimisation of management of joint damage in PwH in resource-constrained settings.
{"title":"Clinical and radiological assessment of joints in people with haemophilia in Assam, Northeast India: a cross-sectional study","authors":"Bhabani Sankar Dhal, Anupama Dutta, A. Das, Dhruba Borpatragohain, A. Sharma, Arnav Kashyap","doi":"10.2478/jhp-2023-0002","DOIUrl":"https://doi.org/10.2478/jhp-2023-0002","url":null,"abstract":"Abstract Background Despite the availability of factor replacement therapy, including prophylaxis, to treat and prevent bleeding, haemophilic arthropathy continues to be the most common complication of haemophilia and significantly impairs the quality of life in people with haemophilia (PwH). Regular periodic assessment of joint status in PwH is essential to identify early arthropathic changes and prevent the development or progression of haemophilic arthropathy. Kinematic and kinetic assessment are preferable and MRI is the gold standard for diagnosing haemophilic arthropathy, but availability is limited in developing countries. HJHS and ultrasound in conjunction with HEAD-US have been shown to effective in assessing changes indicating arthropathy. Aim This study is designed to identify which radiological assessment tool(s) works best when integrated with clinical assessment tools to examine joint health in PwH, with the aim of establishing a cheap, effective, multimodal approach to joint assessment in clinical practice in low resource settings which can then help to guide treatment. Methods A cross-sectional observational study was carried out among PwH attending a clinic in the department of medicine at Assam Medical College and Hospital, Northeast India, over a one-year time period. The elbow, knee and ankle joints of participants were assessed clinically and functionally using HJHS and FISH and radiologically by Pettersson (X-ray) and HEAD-US (ultrasound) scoring. The resulting data was analysed statistically to ascertain correlation between these parameters. Results Sixty-seven PwH were enrolled in the study, with a mean age of 21.69±8.24 years (median 21 years); 38 (57.72%) had severe factor deficiency. The majority (71.70%) presented with joint bleeds; 40 (59.70%) had the knee as a target joint, followed by the elbow (11; 16.41%) and ankle (6; 8.95%). Mean ABR (annual bleed rate), AJBR (annual joint bleed rate) and ATJBR (annual target joint bleed rate) values had a positive correlation with increased severity of factor deficiency (p <0.001). FISH score increased with factor activity level; HJHS, HEAD-US and Pettersson scores decreased with factor activity level. Significant positive correlation was established between Pettersson score, age and ABR. The degree of association was greatest between HJHS and HEAD-US at 60.62%; between HJHS and Pettersson it was 57.74%. Statistically significant negative correlation was established between FISH and HEAD-US. Conclusion A combined multimodal approach using the HJHS, FISH and HEAD-US scoring systems can provide a cheap, quick, more reliable and accurate option for assessing haemophilic joints, with minimal inter and intra observer reliability. This can support the optimisation of management of joint damage in PwH in resource-constrained settings.","PeriodicalId":372940,"journal":{"name":"The Journal of Haemophilia Practice","volume":"26 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"122168659","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Abstract Now nearing retirement, Regina, who was involved in establishing a haemophilia programme for children and young adults in the United States, reflects on the therapeutic relationship between haemophilia nurses and their patients. Having been involved in haemophilia nursing in the 1980s, Regina’s practice has been influenced by her experience of caring for young patients who were infected with HIV/ AIDS following treatment with contaminated blood products. She considers compassion and emotion in nurses’ relationships with their patients and highlights the key role of contact with colleagues and peer support, particularly during difficult times. While trust is an essential part of the therapeutic relationship and is valued by both the patient and the nurse, Regina reflects on how this can result in dependence. She points to the importance of setting clear boundaries and how this ensures that the patient establishes trust with the wider team or system delivering their care, not just an individual nurse. In her roles as clinical manager, haemophilia centre director and nurse coordinator, this is now part of the advice she shares with her own team. Despite a sense of caution about new therapies as a result of her experience during the 1980s, Regina has been involved in gene therapy trials and feels that the future looks positive for people with haemophilia.
{"title":"Living, Caring, Learning – Reflections on the therapeutic relationship in haemophilia care","authors":"Reginald O. Butler","doi":"10.2478/jhp-2023-0005","DOIUrl":"https://doi.org/10.2478/jhp-2023-0005","url":null,"abstract":"Abstract Now nearing retirement, Regina, who was involved in establishing a haemophilia programme for children and young adults in the United States, reflects on the therapeutic relationship between haemophilia nurses and their patients. Having been involved in haemophilia nursing in the 1980s, Regina’s practice has been influenced by her experience of caring for young patients who were infected with HIV/ AIDS following treatment with contaminated blood products. She considers compassion and emotion in nurses’ relationships with their patients and highlights the key role of contact with colleagues and peer support, particularly during difficult times. While trust is an essential part of the therapeutic relationship and is valued by both the patient and the nurse, Regina reflects on how this can result in dependence. She points to the importance of setting clear boundaries and how this ensures that the patient establishes trust with the wider team or system delivering their care, not just an individual nurse. In her roles as clinical manager, haemophilia centre director and nurse coordinator, this is now part of the advice she shares with her own team. Despite a sense of caution about new therapies as a result of her experience during the 1980s, Regina has been involved in gene therapy trials and feels that the future looks positive for people with haemophilia.","PeriodicalId":372940,"journal":{"name":"The Journal of Haemophilia Practice","volume":"6 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"114896253","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Abstract Background The patient voice is an important consideration in the availability and choice of pharmaceuticals – however, how to capture this complex area and apply it formally within regulation, health technology assessment and reimbursement remains subject to ongoing debate. Patient preference studies such as discrete choice experiments (DCEs) are being utilised more frequently in healthcare and it is anticipated that patient preference data will be incorporated more frequently into regulatory submissions moving forward. Aim The aim of this review is to provide an overview of DCEs conducted within haemophilia to date and to consider the key issues in response to a rapidly evolving therapeutic pathway. Methods A systematic literature search was undertaken via Ovid MEDLINE and EMBASE CLASSIC + EMBASE. Abstracts were uploaded and analysed via Rayyan systematic review software. Results: Of 478 records identified from the database searches, 12 full text journal articles met the inclusion criteria with a date range from 2005–2021. There have been two published studies exploring haemophilia patient preferences in relation to gene therapy: one DCE and one utilising a threshold technique. Surveyed audiences included physicians, patients, pharmacists, healthcare professionals and caregivers. 50% of the included studies (n=6) were exclusively conducted in the US, whilst 3 recruited participants across multiple countries. The sample size varied considerably between studies with the total sample size ranging from 30 participants to 505 participants. For the studies involving patients and their caregivers, the mean patient age range was 8.2–41.4 years. There was diversity in (a) the scale of the qualitative work undertaken to support the DCEs, (b) the undertaking of pilots, and (c) how extensively these elements were reported in the included studies. There is a notable trend towards using an online web-based format, with 3 out of 4 DCEs since 2019 utilising this approach. The number of attributes observed per DCE ranged from 5–12 with a median of 6 attributes from the included studies. The number of levels per attribute was relatively consistent (range 2–5) with 2–3 (n=4) and 2–4 levels (n=4) being utilised most frequently. Conclusion Patient preferences and the methods for capturing these are likely to be subject to ongoing debate as the haemophilia care pathway evolves to offer more therapeutic options with a range of risks and benefits. Whilst techniques such as DCE are effective at quantifying patient preferences, they tell us little about the reasons driving these decisions and the likelihood that they will change in response to temporal or external factors. DCEs could be particularly useful for estimating the uptake of new products and assessing potential budget impact. Accelerated and reformed regulatory processes are likely to increase demand for patient preference studies. There is therefore an increased requirement to ensure that patient adv
{"title":"Discrete choice experiments: An overview of experience to date in haemophilia","authors":"J. Spoors, A. Miners, J. Cairns","doi":"10.2478/jhp-2022-0006","DOIUrl":"https://doi.org/10.2478/jhp-2022-0006","url":null,"abstract":"Abstract Background The patient voice is an important consideration in the availability and choice of pharmaceuticals – however, how to capture this complex area and apply it formally within regulation, health technology assessment and reimbursement remains subject to ongoing debate. Patient preference studies such as discrete choice experiments (DCEs) are being utilised more frequently in healthcare and it is anticipated that patient preference data will be incorporated more frequently into regulatory submissions moving forward. Aim The aim of this review is to provide an overview of DCEs conducted within haemophilia to date and to consider the key issues in response to a rapidly evolving therapeutic pathway. Methods A systematic literature search was undertaken via Ovid MEDLINE and EMBASE CLASSIC + EMBASE. Abstracts were uploaded and analysed via Rayyan systematic review software. Results: Of 478 records identified from the database searches, 12 full text journal articles met the inclusion criteria with a date range from 2005–2021. There have been two published studies exploring haemophilia patient preferences in relation to gene therapy: one DCE and one utilising a threshold technique. Surveyed audiences included physicians, patients, pharmacists, healthcare professionals and caregivers. 50% of the included studies (n=6) were exclusively conducted in the US, whilst 3 recruited participants across multiple countries. The sample size varied considerably between studies with the total sample size ranging from 30 participants to 505 participants. For the studies involving patients and their caregivers, the mean patient age range was 8.2–41.4 years. There was diversity in (a) the scale of the qualitative work undertaken to support the DCEs, (b) the undertaking of pilots, and (c) how extensively these elements were reported in the included studies. There is a notable trend towards using an online web-based format, with 3 out of 4 DCEs since 2019 utilising this approach. The number of attributes observed per DCE ranged from 5–12 with a median of 6 attributes from the included studies. The number of levels per attribute was relatively consistent (range 2–5) with 2–3 (n=4) and 2–4 levels (n=4) being utilised most frequently. Conclusion Patient preferences and the methods for capturing these are likely to be subject to ongoing debate as the haemophilia care pathway evolves to offer more therapeutic options with a range of risks and benefits. Whilst techniques such as DCE are effective at quantifying patient preferences, they tell us little about the reasons driving these decisions and the likelihood that they will change in response to temporal or external factors. DCEs could be particularly useful for estimating the uptake of new products and assessing potential budget impact. Accelerated and reformed regulatory processes are likely to increase demand for patient preference studies. There is therefore an increased requirement to ensure that patient adv","PeriodicalId":372940,"journal":{"name":"The Journal of Haemophilia Practice","volume":"52 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2022-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"130525000","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Abstract Introduction Patient agency refers to the abilities and capabilities of patients to act, contribute, influence and make decisions about their healthcare. It depends on both the willingness of patients to participate and the constraints imposed by healthcare providers, services and systems. To determine the factors affecting patient agency, especially for patients with chronic, rare diseases such as haemophilia requiring lifelong care, it is important to consider the patterns, structures, and mental models that define the ecosystem that patients are a part of, irrespective of their level of engagement. Identifying key challenges At the first workshop of the EHC Think Tank Workstream on Patient Agency in December 2021, participants identified five key themes for in-depth discussion relevant to patient agency: the concept of shared decision-making (SDM), patient empowerment, the spectrum of engagement, cultural change and health literacy. The Iceberg Model was used to unpack challenges by identifying composite factors on four levels: events, patterns, structures and mental models. Summary Across the five themes, four common perceived challenges stand out: uneven relationships between patients and healthcare professionals, services and systems; paternalism and hierarchical cultures; failure to recognise problems; conservatism and resistance to change. Despite some progress towards patient empowerment, a ‘glass ceiling’ prevents patients from driving transformation and taking leadership roles in strategy, policymaking and governance. Patient engagement is fluid and those who could benefit most are least likely to engage. Health literacy is perceived as the problem of the patient, not the system, and patients rather than healthcare providers are typically expected to adapt. Preliminary suggestions for addressing these challenges include behavioural communication training for patients and healthcare professionals, a learning system for empowered patient and family care, and a level playing field for stakeholders to interact equally, leading to mutual acceptance and respect.
{"title":"Patient agency: key questions and challenges – A report from the 1st workshop of the EHC Think Tank Workstream on Patient Agency","authors":"A. Bok, D. Noone, Naja Skouw-Rasmussen","doi":"10.2478/jhp-2022-0004","DOIUrl":"https://doi.org/10.2478/jhp-2022-0004","url":null,"abstract":"Abstract Introduction Patient agency refers to the abilities and capabilities of patients to act, contribute, influence and make decisions about their healthcare. It depends on both the willingness of patients to participate and the constraints imposed by healthcare providers, services and systems. To determine the factors affecting patient agency, especially for patients with chronic, rare diseases such as haemophilia requiring lifelong care, it is important to consider the patterns, structures, and mental models that define the ecosystem that patients are a part of, irrespective of their level of engagement. Identifying key challenges At the first workshop of the EHC Think Tank Workstream on Patient Agency in December 2021, participants identified five key themes for in-depth discussion relevant to patient agency: the concept of shared decision-making (SDM), patient empowerment, the spectrum of engagement, cultural change and health literacy. The Iceberg Model was used to unpack challenges by identifying composite factors on four levels: events, patterns, structures and mental models. Summary Across the five themes, four common perceived challenges stand out: uneven relationships between patients and healthcare professionals, services and systems; paternalism and hierarchical cultures; failure to recognise problems; conservatism and resistance to change. Despite some progress towards patient empowerment, a ‘glass ceiling’ prevents patients from driving transformation and taking leadership roles in strategy, policymaking and governance. Patient engagement is fluid and those who could benefit most are least likely to engage. Health literacy is perceived as the problem of the patient, not the system, and patients rather than healthcare providers are typically expected to adapt. Preliminary suggestions for addressing these challenges include behavioural communication training for patients and healthcare professionals, a learning system for empowered patient and family care, and a level playing field for stakeholders to interact equally, leading to mutual acceptance and respect.","PeriodicalId":372940,"journal":{"name":"The Journal of Haemophilia Practice","volume":"101 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2022-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"124129805","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"The Second European Conference on Women and Bleeding Disorders, Basel, Switzerland, 10–12 May 2022","authors":"Naja Skouw-Rasmussen, Evelyn Grimberg, D. Noone","doi":"10.2478/jhp-2022-0014","DOIUrl":"https://doi.org/10.2478/jhp-2022-0014","url":null,"abstract":"","PeriodicalId":372940,"journal":{"name":"The Journal of Haemophilia Practice","volume":"65 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2022-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"117074702","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Abstract Many of the experiences described by women 20 years ago remain prevalent today, and healthcare provision needs to change to offer better treatment and support to women in the bleeding disorder community. These were the key findings of the recently published Cinderella study, which explored the lived experience of women who bleed due to a diagnosed bleeding disorder in order to improve understanding of their unmet needs. Following a systematic literature review to establish what previous research had been carried out in women with bleeding disorders, the Cinderella study undertook an online survey and in-depth discussions (focus groups and one-to-one interviews) to further explore the lived experiences of women haemophilia carriers (WHC), women with a diagnosed bleeding disorder (WBD) and women with immune thrombocytopenia (WITP). The study demonstrated the significant impact of bleeding disorders on the daily lives of survey respondents and the challenges of accessing specialist support, particularly for WHCs. Themes discussed included difficulty obtaining a diagnosis, lack of awareness amongst and poor communication from HCPs, stress and anxiety, coping strategies and sources of support.
{"title":"The Cinderella Study: women's lived experience of bleeding disorders – CSL Behring Symposium","authors":"D. Pollard, K. Khair","doi":"10.2478/jhp-2022-0018","DOIUrl":"https://doi.org/10.2478/jhp-2022-0018","url":null,"abstract":"Abstract Many of the experiences described by women 20 years ago remain prevalent today, and healthcare provision needs to change to offer better treatment and support to women in the bleeding disorder community. These were the key findings of the recently published Cinderella study, which explored the lived experience of women who bleed due to a diagnosed bleeding disorder in order to improve understanding of their unmet needs. Following a systematic literature review to establish what previous research had been carried out in women with bleeding disorders, the Cinderella study undertook an online survey and in-depth discussions (focus groups and one-to-one interviews) to further explore the lived experiences of women haemophilia carriers (WHC), women with a diagnosed bleeding disorder (WBD) and women with immune thrombocytopenia (WITP). The study demonstrated the significant impact of bleeding disorders on the daily lives of survey respondents and the challenges of accessing specialist support, particularly for WHCs. Themes discussed included difficulty obtaining a diagnosis, lack of awareness amongst and poor communication from HCPs, stress and anxiety, coping strategies and sources of support.","PeriodicalId":372940,"journal":{"name":"The Journal of Haemophilia Practice","volume":"28 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2022-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"129445828","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Jon Ulrich Hansen, Cléa Percier, Henrik Damgaard-Rasmussen, K. Palmer
Abstract Introduction People with haemophilia (PwH) require frequent infusions with plasma-derived or recombinant coagulation factors to help prevent and treat acute bleeding episodes. This ‘replacement therapy’ can be administered at home by PwH or their caregivers using a drug reconstitution device. MixPro® and Mix2Vial® are two such devices. Aims/Objectives To compare the experiences and evaluate the preferences of PwH and caregivers using two reconstitution devices: MixPro® and Mix2Vial®. Methods Qualitative interviews were conducted between the 22 June and 4 August 2021 with male PwH or caregivers of PwH. Participants were asked questions about the reconstitution devices they have used in general, followed by questions relating specifically to the MixPro® or Mix2Vial® devices. Demonstration devices were provided to all participants during the interview. Results In total, 105 participants (71 PwH and 34 caregivers) were interviewed in the USA, Italy, UK, and Japan. PwH had a mean age of 29 years (3–69 years). Overall, participants reported the number of parts, speed of reconstitution, and ease of use to be the largest unmet needs with reconstitution devices. Regarding the device features, low contamination risk was ranked as most important (importance score: 15.1) for all countries except Italy, where portability of the device was most important (11.7 for portability vs. 10.6 for low contamination risk). When MixPro® and Mix2Vial® features were independently evaluated, MixPro® outperformed Mix2Vial® across 17 of the 18 features; both devices were rated equally for low contamination risk. When asked which device performed best on each feature, MixPro® was chosen by the majority of participants (74%). MixPro® was associated with words such as quick (54%), user-friendly (47%), and easy (46%), while Mix2Vial® was associated with the words easy (33%), safe (32%), and awkward (30%). Participants felt MixPro® would make a positive difference to their lives citing reasons such as saving time, ease of portability, and general confidence in using the system. Relatively few participants thought Mix2Vial® would make a positive difference to their lives, with some noting it was not much different than the previous generation of devices. Conclusion In this study, MixPro® was preferred over Mix2Vial® as a reconstitution device for PwH and caregivers of PwH.
{"title":"Comparative evaluation and preference of MixPro® versus Mix2Vial® reconstitution devices among people with haemophilia and caregivers","authors":"Jon Ulrich Hansen, Cléa Percier, Henrik Damgaard-Rasmussen, K. Palmer","doi":"10.2478/jhp-2022-0012","DOIUrl":"https://doi.org/10.2478/jhp-2022-0012","url":null,"abstract":"Abstract Introduction People with haemophilia (PwH) require frequent infusions with plasma-derived or recombinant coagulation factors to help prevent and treat acute bleeding episodes. This ‘replacement therapy’ can be administered at home by PwH or their caregivers using a drug reconstitution device. MixPro® and Mix2Vial® are two such devices. Aims/Objectives To compare the experiences and evaluate the preferences of PwH and caregivers using two reconstitution devices: MixPro® and Mix2Vial®. Methods Qualitative interviews were conducted between the 22 June and 4 August 2021 with male PwH or caregivers of PwH. Participants were asked questions about the reconstitution devices they have used in general, followed by questions relating specifically to the MixPro® or Mix2Vial® devices. Demonstration devices were provided to all participants during the interview. Results In total, 105 participants (71 PwH and 34 caregivers) were interviewed in the USA, Italy, UK, and Japan. PwH had a mean age of 29 years (3–69 years). Overall, participants reported the number of parts, speed of reconstitution, and ease of use to be the largest unmet needs with reconstitution devices. Regarding the device features, low contamination risk was ranked as most important (importance score: 15.1) for all countries except Italy, where portability of the device was most important (11.7 for portability vs. 10.6 for low contamination risk). When MixPro® and Mix2Vial® features were independently evaluated, MixPro® outperformed Mix2Vial® across 17 of the 18 features; both devices were rated equally for low contamination risk. When asked which device performed best on each feature, MixPro® was chosen by the majority of participants (74%). MixPro® was associated with words such as quick (54%), user-friendly (47%), and easy (46%), while Mix2Vial® was associated with the words easy (33%), safe (32%), and awkward (30%). Participants felt MixPro® would make a positive difference to their lives citing reasons such as saving time, ease of portability, and general confidence in using the system. Relatively few participants thought Mix2Vial® would make a positive difference to their lives, with some noting it was not much different than the previous generation of devices. Conclusion In this study, MixPro® was preferred over Mix2Vial® as a reconstitution device for PwH and caregivers of PwH.","PeriodicalId":372940,"journal":{"name":"The Journal of Haemophilia Practice","volume":"34 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2022-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"124539961","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
P. Kasirye, Agnes Kisakye, A. Nakalyango, Atwiine Barnabas
Abstract Haemophilia knowledge and care are largely missing in much of sub-Saharan Africa and there is a need for concerted efforts to ensure access to care services by affected persons. Haemophilia Foundation Uganda, supported by the global haemophilia community and working with the Uganda Ministry of Health, has set up eight satellite haemophilia treatment centres (HTCs) as part of a wider initiative to raise awareness and improve haemophilia care. Setting up the HTCs has involved a six-step process involving stakeholders in government, healthcare and the community, and ranging from securing initial support to an ongoing follow-up programme of mentorship and training. Over 1,700 healthcare professionals have been trained and 186 patients have been registered at these peripheral facilities over the past five years. This is helping to improve access to care, but there are still shortcomings around diagnostic capacity, available healthcare personnel, and facilities to procure recombinant factor products. We will continue and further our advocacy for budgetary inclusion of haemophilia at political and facility levels. We also propose a continued strengthening of the haemophilia care teams through mentorship, networking, and mobilisation for diagnostic support at large public hospitals.
{"title":"Processes and experiences of satellite haemophilia clinic set-ups in Uganda – a short report","authors":"P. Kasirye, Agnes Kisakye, A. Nakalyango, Atwiine Barnabas","doi":"10.2478/jhp-2022-0009","DOIUrl":"https://doi.org/10.2478/jhp-2022-0009","url":null,"abstract":"Abstract Haemophilia knowledge and care are largely missing in much of sub-Saharan Africa and there is a need for concerted efforts to ensure access to care services by affected persons. Haemophilia Foundation Uganda, supported by the global haemophilia community and working with the Uganda Ministry of Health, has set up eight satellite haemophilia treatment centres (HTCs) as part of a wider initiative to raise awareness and improve haemophilia care. Setting up the HTCs has involved a six-step process involving stakeholders in government, healthcare and the community, and ranging from securing initial support to an ongoing follow-up programme of mentorship and training. Over 1,700 healthcare professionals have been trained and 186 patients have been registered at these peripheral facilities over the past five years. This is helping to improve access to care, but there are still shortcomings around diagnostic capacity, available healthcare personnel, and facilities to procure recombinant factor products. We will continue and further our advocacy for budgetary inclusion of haemophilia at political and facility levels. We also propose a continued strengthening of the haemophilia care teams through mentorship, networking, and mobilisation for diagnostic support at large public hospitals.","PeriodicalId":372940,"journal":{"name":"The Journal of Haemophilia Practice","volume":"21 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2022-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"116901246","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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