Abstract Introduction People with haemophilia (PwH) experience recurrent bleeds in weight-bearing joints. Optimal management for people with severe haemophilia involves prophylaxis with factor VIII or IX, which helps to reduce the risk of bleeds and joint damage. However, this is expensive and frequently not an option in economically developing countries, where on-demand treatment is more commonly used as bleeding occurs. PwH with moderate and mild haemophilia are also treated on demand. Pain from bleeds and arthropathy is common in PwH; it is recognised as a burden that impairs quality of life and can be challenging to manage. Aims This study aims to establish greater understanding of the experience of pain in PwH in different countries, the factors that influence this, and how pain is currently managed. Methods PwH attending haemophilia treatment centres (HTCs) completed an anonymous questionnaire about their experience of pain and pain-relief within the previous 28 days (up to 10 PwH per participating HTC). Results 209 PwH from 20 HTCs in 11 countries participated in the study. The median age was 36 (range 8–84); 181 (86.6%) had haemophilia A, 25 (12.0%) haemophilia B, and three (1.4%) did not know; 148 (70.8%) had severe haemophilia, 28 (13.4%) moderate, and 31 (14.8%) mild. Twenty-eight (13.4%) had an inhibitor. The majority (n=121; 57.9%) were on prophylaxis; 61 (29.2%) were treated on demand; 20 (9.6%) used a combination; 7 (3.3%) did not know. 154 PwH (73.9%) experienced a total of 1,945 days of pain with severity on a visual analogue scale reported as 4.5. The most commonly reported sites of pain were joints and muscles. There was no significant difference in pain incidence between countries. Children aged less than 16 years reported the lowest amount of pain, with reported pain increasing with age in older respondents. Simple analgesia such as paracetamol was used but participants reported that it did not relieve pain. Alternative pain-relief strategies including rest, physiotherapy, walking aids, alcohol or marijuana were also used with varying effect. Conclusions Pain is common among PwH and increases with age. Age and developmentally appropriate pain assessment should be a part of routine haemophilia care.
{"title":"A multi-country snapshot study of pain in people with haemophilia","authors":"G. Mulders, H. Thykjaer, K. Khair","doi":"10.17225/jhp00161","DOIUrl":"https://doi.org/10.17225/jhp00161","url":null,"abstract":"Abstract Introduction People with haemophilia (PwH) experience recurrent bleeds in weight-bearing joints. Optimal management for people with severe haemophilia involves prophylaxis with factor VIII or IX, which helps to reduce the risk of bleeds and joint damage. However, this is expensive and frequently not an option in economically developing countries, where on-demand treatment is more commonly used as bleeding occurs. PwH with moderate and mild haemophilia are also treated on demand. Pain from bleeds and arthropathy is common in PwH; it is recognised as a burden that impairs quality of life and can be challenging to manage. Aims This study aims to establish greater understanding of the experience of pain in PwH in different countries, the factors that influence this, and how pain is currently managed. Methods PwH attending haemophilia treatment centres (HTCs) completed an anonymous questionnaire about their experience of pain and pain-relief within the previous 28 days (up to 10 PwH per participating HTC). Results 209 PwH from 20 HTCs in 11 countries participated in the study. The median age was 36 (range 8–84); 181 (86.6%) had haemophilia A, 25 (12.0%) haemophilia B, and three (1.4%) did not know; 148 (70.8%) had severe haemophilia, 28 (13.4%) moderate, and 31 (14.8%) mild. Twenty-eight (13.4%) had an inhibitor. The majority (n=121; 57.9%) were on prophylaxis; 61 (29.2%) were treated on demand; 20 (9.6%) used a combination; 7 (3.3%) did not know. 154 PwH (73.9%) experienced a total of 1,945 days of pain with severity on a visual analogue scale reported as 4.5. The most commonly reported sites of pain were joints and muscles. There was no significant difference in pain incidence between countries. Children aged less than 16 years reported the lowest amount of pain, with reported pain increasing with age in older respondents. Simple analgesia such as paracetamol was used but participants reported that it did not relieve pain. Alternative pain-relief strategies including rest, physiotherapy, walking aids, alcohol or marijuana were also used with varying effect. Conclusions Pain is common among PwH and increases with age. Age and developmentally appropriate pain assessment should be a part of routine haemophilia care.","PeriodicalId":372940,"journal":{"name":"The Journal of Haemophilia Practice","volume":"4 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2020-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"125357316","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}
K. Strike, Anthony K. C. Chan, A. Iorio, M. Maly, P. Stratford, P. Solomon
Abstract Introduction Adherence to treatment recommendations in patients with chronic disease is complex and is influenced by numerous factors. Haemophilia is a chronic disease with reported levels of adherence ranging from 17–82%. Aim Based on the theoretical foundation of the World Health Organization Multidimensional Adherence Model, the objective of this study was to identify the best combination of the variables infusion frequency, annualised bleed rate, age, distance to haemophilia treatment centre (HTC) and Haemophilia Joint Health Score (HJHS), to predict adherence to treatment recommendations in patients with haemophilia A and B on home infusion prophylaxis in Canada. Methods A one-year retrospective cohort study investigated adherence to treatment recommendations using two measures: 1) subjective report via home infusion diaries, and 2) objective report of inventory ordered from Canadian Blood Services. Stepwise regression was performed for both measures. Results Eighty-seven patients with haemophilia A and B, median age 21 years, were included. Adherence for both measures was 81% and 93% respectively. The sample consisted largely of patients performing an infusion frequency of every other day (34%). Median scores on the HJHS was 10.5; annualised bleed rate was two. Distance to the HTC was 51km. Analysis of the objective measure weakly supported greater infusion frequency as a treatment-related factor for the prediction of lower adherence, however the strength of this relationship was not clinically relevant (R2=0.048). For the subjective measure, none of the explanatory variables were significant. Conclusion Adherence is a multifaceted construct. Despite the use of theory, most of the variance in adherence to treatment recommendations in this sample of patients with haemophilia remains unknown. Further research on other potential predictors of adherence, and possible variables and relationships within factors of the MAM is required.
{"title":"Predictors of treatment adherence in patients with chronic disease using the Multidimensional Adherence Model: unique considerations for patients with haemophilia","authors":"K. Strike, Anthony K. C. Chan, A. Iorio, M. Maly, P. Stratford, P. Solomon","doi":"10.17225/jhp00152","DOIUrl":"https://doi.org/10.17225/jhp00152","url":null,"abstract":"Abstract Introduction Adherence to treatment recommendations in patients with chronic disease is complex and is influenced by numerous factors. Haemophilia is a chronic disease with reported levels of adherence ranging from 17–82%. Aim Based on the theoretical foundation of the World Health Organization Multidimensional Adherence Model, the objective of this study was to identify the best combination of the variables infusion frequency, annualised bleed rate, age, distance to haemophilia treatment centre (HTC) and Haemophilia Joint Health Score (HJHS), to predict adherence to treatment recommendations in patients with haemophilia A and B on home infusion prophylaxis in Canada. Methods A one-year retrospective cohort study investigated adherence to treatment recommendations using two measures: 1) subjective report via home infusion diaries, and 2) objective report of inventory ordered from Canadian Blood Services. Stepwise regression was performed for both measures. Results Eighty-seven patients with haemophilia A and B, median age 21 years, were included. Adherence for both measures was 81% and 93% respectively. The sample consisted largely of patients performing an infusion frequency of every other day (34%). Median scores on the HJHS was 10.5; annualised bleed rate was two. Distance to the HTC was 51km. Analysis of the objective measure weakly supported greater infusion frequency as a treatment-related factor for the prediction of lower adherence, however the strength of this relationship was not clinically relevant (R2=0.048). For the subjective measure, none of the explanatory variables were significant. Conclusion Adherence is a multifaceted construct. Despite the use of theory, most of the variance in adherence to treatment recommendations in this sample of patients with haemophilia remains unknown. Further research on other potential predictors of adherence, and possible variables and relationships within factors of the MAM is required.","PeriodicalId":372940,"journal":{"name":"The Journal of Haemophilia Practice","volume":"49 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2020-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"121587177","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}
I t is fascinating for a person born with haemophilia almost 70 years ago to read the very insightful paper ‘What more can we ask for?: an ethnographic study of challenges and possibilities for people living with haemophilia’ by Hughes et al. in this issue of the Journal of Haemophilia Practice. While medical treatments have advanced miraculously over the last seven decades, from fresh frozen plasma to cryoprecipitate and a whole range of factor concentrates, to non-factor therapies and now to the cusp of gene therapy, the
{"title":"Uncertainty to normality using ethnographic and qualitative research: a personal view","authors":"D. Page","doi":"10.17225/jhp00170","DOIUrl":"https://doi.org/10.17225/jhp00170","url":null,"abstract":"I t is fascinating for a person born with haemophilia almost 70 years ago to read the very insightful paper ‘What more can we ask for?: an ethnographic study of challenges and possibilities for people living with haemophilia’ by Hughes et al. in this issue of the Journal of Haemophilia Practice. While medical treatments have advanced miraculously over the last seven decades, from fresh frozen plasma to cryoprecipitate and a whole range of factor concentrates, to non-factor therapies and now to the cusp of gene therapy, the","PeriodicalId":372940,"journal":{"name":"The Journal of Haemophilia Practice","volume":"39 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2020-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"116386759","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}
T. Hughes, Mikkel Brok-Kristensen, Yosha Gargeya, Anne Mette Worsøe Lottrup, Ask Bo Larsen, A. Torres-Ortuño, N. Mackett, J. Stevens
Abstract Background With the major advances in treatment of haemophilia in recent decades, people with haemophilia (PwH) are more protected in their daily lives than ever before. However, recent studies point to persisting or increasing patient experience of uncertainty. Aims The aim of this article is to further investigate findings related to how PwH understand and cope with uncertainty around their protection in their everyday life, one of the main themes identified in a large-scale ethnographic study of the everyday life of PwH, including beliefs and experiences related to their condition, their treatment, and their personal ways of managing the condition. Methods The study used ethnographic research methods. Five haemophilia experts provided historical and disease area context prior to the initiation of field research. During field research, study researchers collected data through 8–12 hours of participant observation, semi-structured interviews, written exercises, facilitated group dialogues, and on-site observations of the interactions of PwH with friends, family, and health care professionals (HCPs). Study researchers also conducted on-site observation at haemophilia treatment centres (HTCs) and interviewed HCPs. The study employed a multi-tiered grounded theory approach and combined data were analysed using techniques such as inductive and deductive analysis, cross-case analysis, challenge mapping, and clustering exercises. This article explores findings related to uncertainty and thus focuses on a subset of the data from the study. Results Fifty-one PwH in Italy, Germany, Spain, UK, and Ireland were interviewed and followed in their daily lives, and 18 HCPs were interviewed. Fifty-two per cent (n=26/50) of PwH in the study experience difficulties translating clinical understanding of protection into specific activities in everyday life. Many have developed their own mental models and care adaptations to navigate treatment uncertainy: these seldom match the medical community's view. These mental models of protection among PwH can cause distress and influence behaviour in a way that can limit possibilities, and/or increase risk. There is also a prevalent tension in the strategies PwH have for managing their protection in terms of day-to-day vs. long-term ambitions. Conclusions These findings on PwH's experience of treatment uncertainty suggest a need to develop tools and communication materials to help PwH better understand the protection provided by their treatment regimen and what that means practically for everyday life.
{"title":"Navigating uncertainty: an examination of how people with haemophilia understand and cope with uncertainty in protection in an ethnographic study","authors":"T. Hughes, Mikkel Brok-Kristensen, Yosha Gargeya, Anne Mette Worsøe Lottrup, Ask Bo Larsen, A. Torres-Ortuño, N. Mackett, J. Stevens","doi":"10.17225/jhp00168","DOIUrl":"https://doi.org/10.17225/jhp00168","url":null,"abstract":"Abstract Background With the major advances in treatment of haemophilia in recent decades, people with haemophilia (PwH) are more protected in their daily lives than ever before. However, recent studies point to persisting or increasing patient experience of uncertainty. Aims The aim of this article is to further investigate findings related to how PwH understand and cope with uncertainty around their protection in their everyday life, one of the main themes identified in a large-scale ethnographic study of the everyday life of PwH, including beliefs and experiences related to their condition, their treatment, and their personal ways of managing the condition. Methods The study used ethnographic research methods. Five haemophilia experts provided historical and disease area context prior to the initiation of field research. During field research, study researchers collected data through 8–12 hours of participant observation, semi-structured interviews, written exercises, facilitated group dialogues, and on-site observations of the interactions of PwH with friends, family, and health care professionals (HCPs). Study researchers also conducted on-site observation at haemophilia treatment centres (HTCs) and interviewed HCPs. The study employed a multi-tiered grounded theory approach and combined data were analysed using techniques such as inductive and deductive analysis, cross-case analysis, challenge mapping, and clustering exercises. This article explores findings related to uncertainty and thus focuses on a subset of the data from the study. Results Fifty-one PwH in Italy, Germany, Spain, UK, and Ireland were interviewed and followed in their daily lives, and 18 HCPs were interviewed. Fifty-two per cent (n=26/50) of PwH in the study experience difficulties translating clinical understanding of protection into specific activities in everyday life. Many have developed their own mental models and care adaptations to navigate treatment uncertainy: these seldom match the medical community's view. These mental models of protection among PwH can cause distress and influence behaviour in a way that can limit possibilities, and/or increase risk. There is also a prevalent tension in the strategies PwH have for managing their protection in terms of day-to-day vs. long-term ambitions. Conclusions These findings on PwH's experience of treatment uncertainty suggest a need to develop tools and communication materials to help PwH better understand the protection provided by their treatment regimen and what that means practically for everyday life.","PeriodicalId":372940,"journal":{"name":"The Journal of Haemophilia Practice","volume":"33 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2020-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"114534371","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 Factor VII deficiency is a rare inheritable bleeding disorder that can be challenging to manage. Blood activity levels do not correlate with bleeding risk, and prophylaxis is a more difficult treatment option than for people with haemophilia due to the short half-life of factor VII. Acute bleeding manifestations and long-term complications are similar to those associated with haemophilia. This case study illustrates the psychological and physical impact of severe factor VII deficiency on a woman with impaired mobility due to haemarthropathy who must retain her independence to provide care for her elderly parents. She self-manages her joint pain and bleeding risk, but her life is limited by the need to avoid injury and her reluctance to engage fully with health services.
{"title":"Fiona’s FVII footprint","authors":"G. Mulders, M. Tuinhout","doi":"10.17225/jhp00154","DOIUrl":"https://doi.org/10.17225/jhp00154","url":null,"abstract":"Abstract Factor VII deficiency is a rare inheritable bleeding disorder that can be challenging to manage. Blood activity levels do not correlate with bleeding risk, and prophylaxis is a more difficult treatment option than for people with haemophilia due to the short half-life of factor VII. Acute bleeding manifestations and long-term complications are similar to those associated with haemophilia. This case study illustrates the psychological and physical impact of severe factor VII deficiency on a woman with impaired mobility due to haemarthropathy who must retain her independence to provide care for her elderly parents. She self-manages her joint pain and bleeding risk, but her life is limited by the need to avoid injury and her reluctance to engage fully with health services.","PeriodicalId":372940,"journal":{"name":"The Journal of Haemophilia Practice","volume":"41 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2020-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"117278110","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}
G. Mulders, N. Uitslager, Sharon Alavian, A. Wareing, Kathi Stein Oldenburg
Abstract Introduction Switching between clotting factor products is becoming increasingly common as product choice increases and financial pressure grows to choose the most cost-effective options. Guidance on carrying out the switch recommends a complex and long process that may benefit from being defined in a protocol. Haemophilia nurses may be responsible for managing product switches; anecdotal evidence suggests that clinical practice is variable. Aim To explore the role of specialist nurses in switching between clotting factor products and their use of a protocol. Method Nurses attending the 2018 World Federation of Hemophilia Congress were surveyed about clinical practice at their treatment centre and use of a protocol for switching clotting factor products. Results Of 192 nurses attending the conference, 49 nurses returned completed questionnaires, 45 of which were included in the study after exclusions. Responses were exclusively from economically developed countries. Almost all respondents (96%) had direct experience of switching. Half of those who responded to a question about protocol-based switching reported that switches were based on a protocol. When authorship was reported, the protocol was written by haemophilia nurses in about half of cases. Practice about blood testing to determine individual pharmacokinetic parameters prior to the switch was variable, but most nurses (86%) reported screening for inhibitors prior to switching. Respondents agreed to share their protocols among their peers, although only four were received by the research team. Conclusions Clinical practice in switching between clotting factor products is variable. Some nurses are switching treatments for patients without the supported of a written protocol, whereas others are involved in writing and implementing protocols. Sharing protocols is a first step in helping to establish best practice.
{"title":"Do nurses have the switch factor?","authors":"G. Mulders, N. Uitslager, Sharon Alavian, A. Wareing, Kathi Stein Oldenburg","doi":"10.17225/jhp00162","DOIUrl":"https://doi.org/10.17225/jhp00162","url":null,"abstract":"Abstract Introduction Switching between clotting factor products is becoming increasingly common as product choice increases and financial pressure grows to choose the most cost-effective options. Guidance on carrying out the switch recommends a complex and long process that may benefit from being defined in a protocol. Haemophilia nurses may be responsible for managing product switches; anecdotal evidence suggests that clinical practice is variable. Aim To explore the role of specialist nurses in switching between clotting factor products and their use of a protocol. Method Nurses attending the 2018 World Federation of Hemophilia Congress were surveyed about clinical practice at their treatment centre and use of a protocol for switching clotting factor products. Results Of 192 nurses attending the conference, 49 nurses returned completed questionnaires, 45 of which were included in the study after exclusions. Responses were exclusively from economically developed countries. Almost all respondents (96%) had direct experience of switching. Half of those who responded to a question about protocol-based switching reported that switches were based on a protocol. When authorship was reported, the protocol was written by haemophilia nurses in about half of cases. Practice about blood testing to determine individual pharmacokinetic parameters prior to the switch was variable, but most nurses (86%) reported screening for inhibitors prior to switching. Respondents agreed to share their protocols among their peers, although only four were received by the research team. Conclusions Clinical practice in switching between clotting factor products is variable. Some nurses are switching treatments for patients without the supported of a written protocol, whereas others are involved in writing and implementing protocols. Sharing protocols is a first step in helping to establish best practice.","PeriodicalId":372940,"journal":{"name":"The Journal of Haemophilia Practice","volume":"99 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2020-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"134179802","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}
T he European Haemophilia Consortium’s (EHC) conference Women and Bleeding Disorders, held in May 2019, was the first such meeting dedicated entirely to discussing women’s experience of living with a bleeding disorder, the activities of national member organisations (NMOs), and diagnosis and medical management. The conference welcomed almost 150 delegates from nearly 30 countries, including representatives from the European Association for Haemophilia and Allied Disorders (EAHAD) and the World Federation of Hemophilia (WFH). The EHC has long sought to represent and serve women with bleeding disorders and others affected by a bleeding disorder. In 2015, it organised a preconference workshop entitled Starting a European Conversation for Women with Bleeding Disorders at its annual scientific conference in Belgrade, Serbia. The work of a group of dedicated volunteers from across Europe, has since enabled the EHC to build on this foundation to increase its focus on women, organising sessions specific to issues concerning women with bleeding disorders at scientific conferences, youth debates and its annual Leadership Conference. In 2017, the EHC created a Committee for Women and Bleeding Disorders to promote awareness, recognition, support, and education about and for women in the bleeding disorder community at a European level. The Committee organised a survey of women, NMOs and treatment centres to assess the prevalence and experience of bleeding disorders and the effectiveness of the support available. The survey showed that the needs of women with bleeding disorders are being neglected and provided the evidence on which the EHC can build a campaign to improve recognition and services for women with a bleeding disorder . The Committee invited EHC members to propose the topics to be addressed in this conference. The response was so great that it was a struggle to accommodate everything in the time available. However, the Conference Programming Committee ensured that the meeting covered a wide range of topics in plenary sessions, also providing time for delegates to talk about issues that affected them in Q&A and breakout sessions. The programme covered an overview of bleeding disorders affecting women, genetic transmission, gynaecological and nongynaecological issues, managing monthly menses, the impact of bleeding disorders on quality of life, and tackling psychosocial issues. Breakout sessions focused on family planning, and support from NMOs and treatment centres. The conference marked the premiere of the EHC’s film Women and Bleeding Disorders: Untold Stories, DEBRA POLLARD Lead Nurse, Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free London NHS Foundation Trust, London, UK
{"title":"The First European Conference on Women and Bleeding Disorders Frankfurt, Germany, 24-26 May 2019","authors":"D. Pollard, A. Bok","doi":"10.17225/jhp00145","DOIUrl":"https://doi.org/10.17225/jhp00145","url":null,"abstract":"T he European Haemophilia Consortium’s (EHC) conference Women and Bleeding Disorders, held in May 2019, was the first such meeting dedicated entirely to discussing women’s experience of living with a bleeding disorder, the activities of national member organisations (NMOs), and diagnosis and medical management. The conference welcomed almost 150 delegates from nearly 30 countries, including representatives from the European Association for Haemophilia and Allied Disorders (EAHAD) and the World Federation of Hemophilia (WFH). The EHC has long sought to represent and serve women with bleeding disorders and others affected by a bleeding disorder. In 2015, it organised a preconference workshop entitled Starting a European Conversation for Women with Bleeding Disorders at its annual scientific conference in Belgrade, Serbia. The work of a group of dedicated volunteers from across Europe, has since enabled the EHC to build on this foundation to increase its focus on women, organising sessions specific to issues concerning women with bleeding disorders at scientific conferences, youth debates and its annual Leadership Conference. In 2017, the EHC created a Committee for Women and Bleeding Disorders to promote awareness, recognition, support, and education about and for women in the bleeding disorder community at a European level. The Committee organised a survey of women, NMOs and treatment centres to assess the prevalence and experience of bleeding disorders and the effectiveness of the support available. The survey showed that the needs of women with bleeding disorders are being neglected and provided the evidence on which the EHC can build a campaign to improve recognition and services for women with a bleeding disorder . The Committee invited EHC members to propose the topics to be addressed in this conference. The response was so great that it was a struggle to accommodate everything in the time available. However, the Conference Programming Committee ensured that the meeting covered a wide range of topics in plenary sessions, also providing time for delegates to talk about issues that affected them in Q&A and breakout sessions. The programme covered an overview of bleeding disorders affecting women, genetic transmission, gynaecological and nongynaecological issues, managing monthly menses, the impact of bleeding disorders on quality of life, and tackling psychosocial issues. Breakout sessions focused on family planning, and support from NMOs and treatment centres. The conference marked the premiere of the EHC’s film Women and Bleeding Disorders: Untold Stories, DEBRA POLLARD Lead Nurse, Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free London NHS Foundation Trust, London, UK","PeriodicalId":372940,"journal":{"name":"The Journal of Haemophilia Practice","volume":"12 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2019-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"115100435","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}
T here are many aspects of living with haemophilia that make people feel different from others. But imagine the impact of having a bleeding disorder so rare that you feel different from almost everyone else with a bleeding disorder. Evelyn Grimberg and Minette van der Ven from The Netherlands are two such people: they have Glanzmann’s thrombasthenia, an inherited platelet function disorder. There are perhaps 2,000 – 3,000 people worldwide living with this diagnosis – a prevalence of about 1 in 4 million population worldwide. However, there are many more cases than expected in some countries – for example, in some areas of Iran, where the prevalence is estimated at 1 in 200,000. Whichever figure is correct, the odds of having two people with Glanzmann’s in the same room at random are inconceivably small. But this is not a random meeting: Evelyn and Minette are close friends who have come together, not because they share this unusual diagnosis, but because they have the same outlook on living with it. And they have also bonded as women who see services for bleeding disorders heavily geared towards boys and men with haemophilia, to the extent that the impact on women with bleeding disorders has been underestimated or ignored. For Evelyn and Minette, early life with Glanzmann’s was similar to that for many of the current generation of adults with a bleeding disorder. Suspicion first arose after an episode of uncontrolled bleeding – at age four months for Evelyn and two years for Minette. Both are the only family members known to be affected. For their parents, the uncertainty of a future living with a bleeding disorder was compounded by difficulty identifying the cause – it was two years before Minette’s diagnosis was confirmed – but both families adopted a robust approach to raising their child.
血友病患者在生活中有许多方面会让他们感觉与他人不同。但想象一下，患有出血性疾病的影响是如此罕见，以至于你觉得自己与几乎所有患有出血性疾病的人都不一样。来自荷兰的Evelyn Grimberg和Minette van der Ven就是这样的两个人:他们患有格兰兹曼血栓症，一种遗传性血小板功能紊乱。全世界可能有2000 - 3000人患有这种疾病，患病率约为400万人中有1人。然而，一些国家的病例比预期的要多得多，例如，在伊朗的一些地区，患病率估计为20万分之一。无论哪个数字是正确的，在同一个房间里随机出现两个患有格兰兹曼氏症的人的几率都小得难以想象。但这不是一次偶然的相遇:伊芙琳和米内特是亲密的朋友，她们走到了一起，不是因为她们都有这种不寻常的诊断，而是因为她们对生活的看法相同。她们也因为看到出血性疾病的服务主要面向患有血友病的男孩和男性而联系在一起，以至于对患出血性疾病的女性的影响被低估或忽视了。对于伊芙琳和米内特来说，患有格兰兹曼氏症的早期生活与当今许多患有出血性疾病的成年人的生活相似。伊芙琳四个月大的时候，米内特两岁大的时候，一次无法控制的出血事件引起了人们的怀疑。两人都是已知的唯一受影响的家庭成员。对于他们的父母来说，未来是否会患有出血性疾病的不确定性，加上难以确定病因——两年后米内特的诊断才得到证实——但两个家庭都采取了强有力的方法来抚养孩子。
{"title":"Doubly rare: Evelyn Grimberg and Minette van der Ven","authors":"S. Chaplin","doi":"10.17225/JHP00130","DOIUrl":"https://doi.org/10.17225/JHP00130","url":null,"abstract":"T here are many aspects of living with haemophilia that make people feel different from others. But imagine the impact of having a bleeding disorder so rare that you feel different from almost everyone else with a bleeding disorder. Evelyn Grimberg and Minette van der Ven from The Netherlands are two such people: they have Glanzmann’s thrombasthenia, an inherited platelet function disorder. There are perhaps 2,000 – 3,000 people worldwide living with this diagnosis – a prevalence of about 1 in 4 million population worldwide. However, there are many more cases than expected in some countries – for example, in some areas of Iran, where the prevalence is estimated at 1 in 200,000. Whichever figure is correct, the odds of having two people with Glanzmann’s in the same room at random are inconceivably small. But this is not a random meeting: Evelyn and Minette are close friends who have come together, not because they share this unusual diagnosis, but because they have the same outlook on living with it. And they have also bonded as women who see services for bleeding disorders heavily geared towards boys and men with haemophilia, to the extent that the impact on women with bleeding disorders has been underestimated or ignored. For Evelyn and Minette, early life with Glanzmann’s was similar to that for many of the current generation of adults with a bleeding disorder. Suspicion first arose after an episode of uncontrolled bleeding – at age four months for Evelyn and two years for Minette. Both are the only family members known to be affected. For their parents, the uncertainty of a future living with a bleeding disorder was compounded by difficulty identifying the cause – it was two years before Minette’s diagnosis was confirmed – but both families adopted a robust approach to raising their child.","PeriodicalId":372940,"journal":{"name":"The Journal of Haemophilia Practice","volume":"38 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2019-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"122345772","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}
M onday 24th September 2018 was the first day of the preliminary hearings of the UK Infected Blood Inquiry. Two earlier inquiries, dismissed as whitewashes, failed to provide answers and committed campaigners had now been rewarded. The Inquiry would examine the circumstances in which people under the care of the National Health Service (NHS) were given blood and blood products contaminated with HIV and hepatitis viruses, and possibly Creuzfeldt-Jakob disease (CJD); the impact on those infected and affected; the nature, adequacy and timeliness of the response from agencies involved; to what extent individuals may have been misled and denied treatment; and whether those agencies and individuals covered up the facts. The first three days were held in public, given over to opening statements that included witness accounts from people with haemophilia and their families. Their stories are profoundly moving. The Inquiry is truly an historic step towards accountability for the government, the NHS, health professionals and all involved in haemophilia care in the UK decades since 1970. The man who worked for many years to make this a reality is Bruce Norval. Bruce was born in Edinburgh in 1965. At age three he was admitted to hospital with bleeding from a fall; after three months on the ward, he was diagnosed with haemophilia B. It did not make much difference to him at the time.
{"title":"Redressing the balance: Bruce Norval","authors":"S. Chaplin","doi":"10.17225/JHP00119","DOIUrl":"https://doi.org/10.17225/JHP00119","url":null,"abstract":"M onday 24th September 2018 was the first day of the preliminary hearings of the UK Infected Blood Inquiry. Two earlier inquiries, dismissed as whitewashes, failed to provide answers and committed campaigners had now been rewarded. The Inquiry would examine the circumstances in which people under the care of the National Health Service (NHS) were given blood and blood products contaminated with HIV and hepatitis viruses, and possibly Creuzfeldt-Jakob disease (CJD); the impact on those infected and affected; the nature, adequacy and timeliness of the response from agencies involved; to what extent individuals may have been misled and denied treatment; and whether those agencies and individuals covered up the facts. The first three days were held in public, given over to opening statements that included witness accounts from people with haemophilia and their families. Their stories are profoundly moving. The Inquiry is truly an historic step towards accountability for the government, the NHS, health professionals and all involved in haemophilia care in the UK decades since 1970. The man who worked for many years to make this a reality is Bruce Norval. Bruce was born in Edinburgh in 1965. At age three he was admitted to hospital with bleeding from a fall; after three months on the ward, he was diagnosed with haemophilia B. It did not make much difference to him at the time.","PeriodicalId":372940,"journal":{"name":"The Journal of Haemophilia Practice","volume":"79 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2019-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"129420043","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}
Srila Gopal, R. Barnes, D. Quon, H. Sun, S. Jackson, A. von Drygalski
Abstract Background Patients with haemophilia have a higher prevalence of hypertension than the general population that cannot be explained by traditional cardiovascular risk factors such as age, race, diabetes or obesity. Patients with severe haemophilia, who are on clotting factor prophylaxis, have a higher prevalence of hypertension compared to patients with milder forms of haemophilia, who infuse clotting factor less frequently. This raises the question of whether there is a link between clotting factor usage and blood pressure in haemophilia patients. Methods Data was collected from 193 patients with severe haemophilia presenting to three haemophilia treatment centres in the United States and Canada, including age, body mass index (BMI), blood pressure (BP), Hepatitis C (HCV) and Human Immunodeficiency Virus (HIV) infection status, and clotting factor usage from pharmacy prescriptions (units/kg/year). The correlation between BP and factor usage was examined using quantile regression models. Results Systolic and diastolic BP plotted against factor use showed a cone-shaped scatter of points. There was no association between clotting factor usage and higher systolic or diastolic BP. Conclusion Our observations provide no evidence for an association between increased clotting factor usage and high BP.
{"title":"Blood pressure in haemophilia and its relation to clotting factor usage","authors":"Srila Gopal, R. Barnes, D. Quon, H. Sun, S. Jackson, A. von Drygalski","doi":"10.17225/jhp00147","DOIUrl":"https://doi.org/10.17225/jhp00147","url":null,"abstract":"Abstract Background Patients with haemophilia have a higher prevalence of hypertension than the general population that cannot be explained by traditional cardiovascular risk factors such as age, race, diabetes or obesity. Patients with severe haemophilia, who are on clotting factor prophylaxis, have a higher prevalence of hypertension compared to patients with milder forms of haemophilia, who infuse clotting factor less frequently. This raises the question of whether there is a link between clotting factor usage and blood pressure in haemophilia patients. Methods Data was collected from 193 patients with severe haemophilia presenting to three haemophilia treatment centres in the United States and Canada, including age, body mass index (BMI), blood pressure (BP), Hepatitis C (HCV) and Human Immunodeficiency Virus (HIV) infection status, and clotting factor usage from pharmacy prescriptions (units/kg/year). The correlation between BP and factor usage was examined using quantile regression models. Results Systolic and diastolic BP plotted against factor use showed a cone-shaped scatter of points. There was no association between clotting factor usage and higher systolic or diastolic BP. Conclusion Our observations provide no evidence for an association between increased clotting factor usage and high BP.","PeriodicalId":372940,"journal":{"name":"The Journal of Haemophilia Practice","volume":"6 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2019-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"130546041","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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