Anna Olsson, Petra Elfvinge, Eva Zetterberg, Linda Myrin-Westesson
Introduction
Heavy menstrual bleeding (HMB) is a common and burdensome symptom in women with von Willebrand disease (VWD). Although VWD and HMB have been studied, the prevalence and impact of HMB across different age groups remains underexplored.
Aim
To investigate the prevalence and impact of HMB in women with VWD across different age groups.
Methods
This retrospective nationwide study included 136 women aged 18–55 years with type 1 (von Willebrand factor activity ≤ 0.35 IU/mL), 2 or 3 VWD. Data were collected using a questionnaire assessing HMB and its impact on life, and clinical data were retrieved from medical records.
Results
HMB was reported by 93% of women in one or more age groups. During adolescence, 85% reported HMB. Reported HMB decreased with age: 63% at 20–29 years, 45% at 30–39 years, and 36% at 40–55 years. Of the 136 women, 60% had sought medical care due to HMB. Forty-nine percent of these had sought medical care at two or more healthcare facilities. The majority (86%) stated that HMB had negatively impacted their lives. Several aspects were affected, including school/work performance, social and physical activities, with a decreasing impact observed with increasing age. Despite the use of hormonal therapy (76%) and/or haemostatic agents (76%), 54% had had iron deficiency. Thirty-five percent were diagnosed after age 19, and 27% were lost to follow-up.
Conclusions
The findings emphasise the need for increased awareness during adolescence, more structured follow-up and improved individualised management strategies to prevent and treat HMB.
{"title":"Prevalence and Impact of Heavy Menstrual Bleeding in Women With von Willebrand Disease Across Age Groups: A Retrospective Study","authors":"Anna Olsson, Petra Elfvinge, Eva Zetterberg, Linda Myrin-Westesson","doi":"10.1111/hae.70127","DOIUrl":"10.1111/hae.70127","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Heavy menstrual bleeding (HMB) is a common and burdensome symptom in women with von Willebrand disease (VWD). Although VWD and HMB have been studied, the prevalence and impact of HMB across different age groups remains underexplored.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Aim</h3>\u0000 \u0000 <p>To investigate the prevalence and impact of HMB in women with VWD across different age groups.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>This retrospective nationwide study included 136 women aged 18–55 years with type 1 (von Willebrand factor activity ≤ 0.35 IU/mL), 2 or 3 VWD. Data were collected using a questionnaire assessing HMB and its impact on life, and clinical data were retrieved from medical records.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>HMB was reported by 93% of women in one or more age groups. During adolescence, 85% reported HMB. Reported HMB decreased with age: 63% at 20–29 years, 45% at 30–39 years, and 36% at 40–55 years. Of the 136 women, 60% had sought medical care due to HMB. Forty-nine percent of these had sought medical care at two or more healthcare facilities. The majority (86%) stated that HMB had negatively impacted their lives. Several aspects were affected, including school/work performance, social and physical activities, with a decreasing impact observed with increasing age. Despite the use of hormonal therapy (76%) and/or haemostatic agents (76%), 54% had had iron deficiency. Thirty-five percent were diagnosed after age 19, and 27% were lost to follow-up.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>The findings emphasise the need for increased awareness during adolescence, more structured follow-up and improved individualised management strategies to prevent and treat HMB.</p>\u0000 </section>\u0000 </div>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"31 6","pages":"1243-1249"},"PeriodicalIF":3.0,"publicationDate":"2025-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/hae.70127","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145069314","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Arthroscopic Ankle Surgery Under Ultrasound-Guided Peripheral Nerve Block in Patients With Haemophilic Ankle Arthropathy—A Report of Five Cases","authors":"Yasunari Ikuta, Tomoyuki Nakasa, Teruhisa Fujii, Naoya Yamasaki, Saori Ishibashi, Satoru Sakurai, Dan Moriwaki, Nobuo Adachi","doi":"10.1111/hae.70132","DOIUrl":"10.1111/hae.70132","url":null,"abstract":"","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"31 6","pages":"1352-1355"},"PeriodicalIF":3.0,"publicationDate":"2025-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145069336","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Yesim Dargaud, Arianna Colombo, Maria Elisa Mancuso
Background
This article provides a critical analysis of the ‘factor equivalence’ concept as applied to non-factor therapies (NFTs) for haemophilia, highlighting its limitations and clinical implications. Although factor equivalence estimates serve as a comparative tool for evaluating pro-coagulant effects across different therapies, they may fail to accurately represent the true haemostatic potential or clinical efficacy due to the diverse mechanisms of action of different molecules and patient-specific factors.
Aim
The article challenges the widespread adoption of factor equivalence as a universal benchmark to quantify the haemostatic potential of NFTs.
Discussion and Conclusion
It advocates for patient- and context-specific laboratory assessments that reflect each therapy's unique pharmacological profile. This approach is particularly relevant to enhance personalised treatment strategies, especially in high-risk situations such as surgery or severe breakthrough bleeding, thereby optimising the use of novel haemostatic therapies.
{"title":"The Mirage of Factor Equivalence: Examining the Complexities of Non-Factor Therapies in Haemophilia","authors":"Yesim Dargaud, Arianna Colombo, Maria Elisa Mancuso","doi":"10.1111/hae.70131","DOIUrl":"10.1111/hae.70131","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>This article provides a critical analysis of the ‘factor equivalence’ concept as applied to non-factor therapies (NFTs) for haemophilia, highlighting its limitations and clinical implications. Although factor equivalence estimates serve as a comparative tool for evaluating pro-coagulant effects across different therapies, they may fail to accurately represent the true haemostatic potential or clinical efficacy due to the diverse mechanisms of action of different molecules and patient-specific factors.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Aim</h3>\u0000 \u0000 <p>The article challenges the widespread adoption of factor equivalence as a universal benchmark to quantify the haemostatic potential of NFTs.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Discussion and Conclusion</h3>\u0000 \u0000 <p>It advocates for patient- and context-specific laboratory assessments that reflect each therapy's unique pharmacological profile. This approach is particularly relevant to enhance personalised treatment strategies, especially in high-risk situations such as surgery or severe breakthrough bleeding, thereby optimising the use of novel haemostatic therapies.</p>\u0000 </section>\u0000 </div>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"31 6","pages":"1170-1175"},"PeriodicalIF":3.0,"publicationDate":"2025-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/hae.70131","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145069385","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Barbara Konkle, Donna Coffin, Cédric Hermans, Mayss Naccache, Brian O'Mahony, Glenn F. Pierce
<p>To the Editor:</p><p>Gene therapy for haemophilia has been in development for several decades, with the first clinical attempts initiated in the 1990s [<span>1</span>]. These early studies, including six initial trials, laid the foundation for subsequent systemic liver-directed adeno-associated virus (AAV) trials that ultimately led to regulatory approvals. A pivotal milestone came in 2011 with the publication by Nathwani et al. [<span>2</span>], which demonstrated the first successful systemic AAV8-FIX gene therapy for haemophilia B, achieving sustained therapeutic factor IX levels with immune modulation using prednisolone. Since then, 16 gene therapy products (seven for haemophilia A (PWH-A) and nine for haemophilia B (PWH-B)) have been assessed in 20 distinct trials (8 HEM-A, 12 HEM-B) [<span>3</span>], with more clinical trials expected in the future. Of these, three advanced to Phase III clinical trials (valoctocogene roxaparvovec, fidanacogene elaparvovec, and etranacogene dezaparvovec) while the remaining products did not progress beyond Phase I or II. One of the products among those that advanced to Phase III clinical trial was subsequently discontinued by the manufacturer after receiving regulatory approval but before any commercial dosing took place [<span>4</span>]. As a result, only two gene therapy products, from two manufacturers, are currently approved and available in some jurisdictions, primarily within FDA- and EMA-regulated markets. Manufacturers of all gene therapy clinical trials, regardless of whether the product receives regulatory approval, are required to conduct long-term follow-up of participants for at least 15 years [<span>5</span>]. Other countries, including China and India, are actively developing gene therapy products, but these are not yet reflected in international regulatory approvals or global commercialization data. Despite these advancements, several challenges have emerged [<span>1, 6</span>], leading to a more cautious outlook by some gene therapy manufacturers. While gene therapy remains a groundbreaking innovation for haemophilia and has advanced the field forward, uncertainties around long-term safety and efficacy, pricing, patient uptake, and competition from other existing and upcoming therapies have led to a more measured response from the global community, with several manufacturers becoming less optimistic about commercial viability. Global haemophilia organizations are calling for urgent international collaboration to address access, affordability and sustainability challenges in the evolving haemophilia treatment landscape [<span>6</span>].</p><p>An equally significant issue has arisen concerning the 395 people with haemophilia (211 HEM-A patients and 184 HEM-B patients) from 26 different countries (Figure 1) who participated in these clinical trials. This number will increase over time as additional PWH participate in ongoing and future gene therapy clinical trials. As many of the original ma
{"title":"The Critical Need to Consolidate All Gene Therapy Data in Haemophilia","authors":"Barbara Konkle, Donna Coffin, Cédric Hermans, Mayss Naccache, Brian O'Mahony, Glenn F. Pierce","doi":"10.1111/hae.70128","DOIUrl":"10.1111/hae.70128","url":null,"abstract":"<p>To the Editor:</p><p>Gene therapy for haemophilia has been in development for several decades, with the first clinical attempts initiated in the 1990s [<span>1</span>]. These early studies, including six initial trials, laid the foundation for subsequent systemic liver-directed adeno-associated virus (AAV) trials that ultimately led to regulatory approvals. A pivotal milestone came in 2011 with the publication by Nathwani et al. [<span>2</span>], which demonstrated the first successful systemic AAV8-FIX gene therapy for haemophilia B, achieving sustained therapeutic factor IX levels with immune modulation using prednisolone. Since then, 16 gene therapy products (seven for haemophilia A (PWH-A) and nine for haemophilia B (PWH-B)) have been assessed in 20 distinct trials (8 HEM-A, 12 HEM-B) [<span>3</span>], with more clinical trials expected in the future. Of these, three advanced to Phase III clinical trials (valoctocogene roxaparvovec, fidanacogene elaparvovec, and etranacogene dezaparvovec) while the remaining products did not progress beyond Phase I or II. One of the products among those that advanced to Phase III clinical trial was subsequently discontinued by the manufacturer after receiving regulatory approval but before any commercial dosing took place [<span>4</span>]. As a result, only two gene therapy products, from two manufacturers, are currently approved and available in some jurisdictions, primarily within FDA- and EMA-regulated markets. Manufacturers of all gene therapy clinical trials, regardless of whether the product receives regulatory approval, are required to conduct long-term follow-up of participants for at least 15 years [<span>5</span>]. Other countries, including China and India, are actively developing gene therapy products, but these are not yet reflected in international regulatory approvals or global commercialization data. Despite these advancements, several challenges have emerged [<span>1, 6</span>], leading to a more cautious outlook by some gene therapy manufacturers. While gene therapy remains a groundbreaking innovation for haemophilia and has advanced the field forward, uncertainties around long-term safety and efficacy, pricing, patient uptake, and competition from other existing and upcoming therapies have led to a more measured response from the global community, with several manufacturers becoming less optimistic about commercial viability. Global haemophilia organizations are calling for urgent international collaboration to address access, affordability and sustainability challenges in the evolving haemophilia treatment landscape [<span>6</span>].</p><p>An equally significant issue has arisen concerning the 395 people with haemophilia (211 HEM-A patients and 184 HEM-B patients) from 26 different countries (Figure 1) who participated in these clinical trials. This number will increase over time as additional PWH participate in ongoing and future gene therapy clinical trials. As many of the original ma","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"31 6","pages":"1349-1351"},"PeriodicalIF":3.0,"publicationDate":"2025-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/hae.70128","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145069353","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
J. Mahlangu, M. E. Mancuso, K. Fischer, et al., “Extension Study With rVIII-SingleChain in Previously Untreated Patients (PUPs) With Severe Hemophilia A,” Haemophilia 31, no. 2 (2025): 214–223, https://doi.org/10.1111/hae.15151.
In the Results section of the Abstract, the text'Median time to eradication was 14.3 weeks (IQR 9.8–53.8)' was incorrect. This should have read'Median time to eradication was 14.3 weeks (IQR 9.6–53.6)'.
In paragraph 2 of the Discussion, the text'Our study showed a high incidence of LT inhibitors that achieved inhibitor eradication (< 0.6 BU/mL) within a median time of 14 weeks'. was incorrect. This should have read'Our study showed that all PUPs with LT inhibitors achieved inhibitor eradication (< 0.6 BU/mL), within a median time of 11 weeks'.
In paragraph 4 of the Discussion, the text'The median number of EDs at the time of inhibitor development was 10.0 (range 5.0–23.0)' was incorrect. This should have read'The median number of EDs at the time of inhibitor development was 10.0 (range 4.0–23.0)'.
We apologize for these inconsistencies.
J. Mahlangu， M. E. Mancuso， K. Fischer等，“rviii -单链在未治疗的严重血友病A患者（PUPs）中的扩展研究”，《血友病》第31期，no。2 (2025): 214-223, https://doi.org/10.1111/hae.15151.In摘要的结果部分，文本“median time to eradication is 14.3 weeks （IQR 9.8-53.8）”是错误的。这应该是“根除的中位时间为14.3周（IQR 9.6-53.6）”。在Discussion的第2段中，文章“我们的研究显示，在14周的中位时间内，LT抑制剂的发生率很高（< 0.6 BU/mL）”。是不正确的。这应该是“我们的研究表明，所有使用LT抑制剂的幼犬在11周的中位时间内都实现了抑制剂根除（< 0.6 BU/mL）”。在Discussion的第4段中，“抑制剂开发时ed的中位数为10.0（范围5.0-23.0）”这一文本是不正确的。这应该是“抑制剂开发时ed的中位数为10.0（范围为4.0-23.0）”。我们为这些不一致之处道歉。
{"title":"Correction to “Extension Study With rVIII-SingleChain in Previously Untreated Patients (PUPs) With Severe Hemophilia A”","authors":"","doi":"10.1111/hae.70126","DOIUrl":"10.1111/hae.70126","url":null,"abstract":"<p>J. Mahlangu, M. E. Mancuso, K. Fischer, et al., “Extension Study With rVIII-SingleChain in Previously Untreated Patients (PUPs) With Severe Hemophilia A,” <i>Haemophilia</i> 31, no. 2 (2025): 214–223, https://doi.org/10.1111/hae.15151.</p><p>In the Results section of the Abstract, the text'Median time to eradication was 14.3 weeks (IQR 9.8–53.8)' was incorrect. This should have read'Median time to eradication was 14.3 weeks (IQR 9.6–53.6)'.</p><p>In paragraph 2 of the Discussion, the text'Our study showed a high incidence of LT inhibitors that achieved inhibitor eradication (< 0.6 BU/mL) within a median time of 14 weeks'. was incorrect. This should have read'Our study showed that all PUPs with LT inhibitors achieved inhibitor eradication (< 0.6 BU/mL), within a median time of 11 weeks'.</p><p>In paragraph 4 of the Discussion, the text'The median number of EDs at the time of inhibitor development was 10.0 (range 5.0–23.0)' was incorrect. This should have read'The median number of EDs at the time of inhibitor development was 10.0 (range 4.0–23.0)'.</p><p>We apologize for these inconsistencies.</p>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"31 6","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/hae.70126","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145075176","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Eve Justason, Anna M. Ishchuk, Benjamin P. Ott, M. E. Sophie Gibson, Paula D. James
Introduction
Von Willebrand disease (VWD) is a bleeding disorder characterized by a deficiency or dysfunction of Von Willebrand factor (VWF) and/or Factor VIII (FVIII), critical coagulation proteins. Individuals with VWD often use combination oral contraceptives (COCs) to manage heavy menstrual bleeding. However, the impact of COCs on VWF and FVIII levels and whether COC use affects VWD diagnosis is unclear.
Aim
To review the literature and assess the impact of COCs on FVIII and VWF.
Methods
This scoping review used the OVID platform in the MEDLINE, EMBASE and Cochrane databases. Keywords “combination oral contraceptives,” “von Willebrand Factor,” “Factor VIII” and “von Willebrand Disease” were searched. Primary studies exploring the impact of COCs on VWF and/or FVIII in patients of reproductive age were included. Article titles and abstracts were screened, followed by full-text reviews, data extraction and a meta-analysis.
Results
Twenty-seven studies were included. In healthy patients, 11 studies reported no change in VWF levels, while three found changes in VWF levels. Nine studies reported no change in FVIII levels, while 10 studies observed an increase. In patients with VWD, two studies found no significant change in VWF or FVIII levels. Meta-analysis revealed there was no significant difference in VWF% (Estimate: 2.62 (95%CI −0.5905, 5.831); p value: 0.4033) or FVIII% (Estimate: 2.99 (95%CI −4.85, 10.82); p value: 0.4552) with COC use.
Conclusion
The meta-analysis revealed no difference in VWD or FVIII levels between participants with and without COCs. The lack of observed differences suggests that COCs do not interfere with accurate VWD diagnosis.
{"title":"Determining the Impact of Combination Oral Contraceptives on Von Willebrand Factor and Factor VIII in Healthy Patients and Patients With Von Willebrand Disease: A Scoping Review and Meta-Analysis","authors":"Eve Justason, Anna M. Ishchuk, Benjamin P. Ott, M. E. Sophie Gibson, Paula D. James","doi":"10.1111/hae.70124","DOIUrl":"10.1111/hae.70124","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Von Willebrand disease (VWD) is a bleeding disorder characterized by a deficiency or dysfunction of Von Willebrand factor (VWF) and/or Factor VIII (FVIII), critical coagulation proteins. Individuals with VWD often use combination oral contraceptives (COCs) to manage heavy menstrual bleeding. However, the impact of COCs on VWF and FVIII levels and whether COC use affects VWD diagnosis is unclear.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Aim</h3>\u0000 \u0000 <p>To review the literature and assess the impact of COCs on FVIII and VWF.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>This scoping review used the OVID platform in the MEDLINE, EMBASE and Cochrane databases. Keywords “combination oral contraceptives,” “von Willebrand Factor,” “Factor VIII” and “von Willebrand Disease” were searched. Primary studies exploring the impact of COCs on VWF and/or FVIII in patients of reproductive age were included. Article titles and abstracts were screened, followed by full-text reviews, data extraction and a meta-analysis.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Twenty-seven studies were included. In healthy patients, 11 studies reported no change in VWF levels, while three found changes in VWF levels. Nine studies reported no change in FVIII levels, while 10 studies observed an increase. In patients with VWD, two studies found no significant change in VWF or FVIII levels. Meta-analysis revealed there was no significant difference in VWF% (Estimate: 2.62 (95%CI −0.5905, 5.831); <i>p</i> value: 0.4033) or FVIII% (Estimate: 2.99 (95%CI −4.85, 10.82); <i>p</i> value: 0.4552) with COC use.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>The meta-analysis revealed no difference in VWD or FVIII levels between participants with and without COCs. The lack of observed differences suggests that COCs do not interfere with accurate VWD diagnosis.</p>\u0000 </section>\u0000 </div>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"31 6","pages":"1158-1169"},"PeriodicalIF":3.0,"publicationDate":"2025-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/hae.70124","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145033191","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Marie-Anaïs Roques, Natacha Rosso-Delsemme, Amandine Celli, Ngoc Anh Thu Nguyen, Martin Postzich, Sabine Castet, Yoann Huguenin, Annie Harroche, Anne Lienhart, Sandrine Meunier, Christine Biron-Andréani, Florence Rousseau, Roseline d'Oiron, Yohann Repessé, Clémence Tabélé, Any Beltran Anzola, Thomas Sannié, Nicolas Giraud, Pascal Auquier, Hervé Chambost, Noémie Resseguier, The TRANSHEMO Study Group
Introduction
Haemophilia causes spontaneous or prolonged bleeding due to a deficiency in clotting factor VIII (haemophilia A) or IX (haemophilia B). Although substitutive therapies and regular follow-up can prevent severe haemorrhagic events, adherence to treatment remains a challenge. Transitioning from adolescence to adulthood and from paediatric to adult care is particularly complex for young people with severe haemophilia (PwSH), as it involves gaining autonomy in health management.
Objectives
This study aimed to explore factors influencing the success of the transition process in young PwSH, with a focus on adherence to healthcare.
Methods
This qualitative study was part of the mixed-methods TRANSHEMO project. Participants were selected from the quantitative phase of the TRANSHEMO project based on two criteria: adolescents/young adults and adherent/nonadherent to healthcare. Interviews were conducted via video conferencing, transcribed, and thematically analysed to identify key themes affecting the transition process.
Results
Twenty-two interviews were conducted. Four major themes emerged as critical to transition success: (1) Care factors [continuity of care, treatment rituals, and evolving therapies]; (2) Family and social factors [support from family, friends, peers, and overprotection]; (3) Personal factors [understanding haemophilia, risk management, optimism, and coping strategies]; and (4) Autonomy [secondary benefits, independence, proactivity in disease management, and accompaniment by caregivers].
Conclusion
Based on the enlightened determinants, supportive strategies and patient education programs should focus on the development of autonomy, personal factors such as acquisition and application of health literacy in haemophilia care, and family factors such as support from family.
{"title":"Navigating the Transition From Adolescence to Adulthood Among Young People With Severe Haemophilia: The Qualitative Phase of the TRANSHEMO Project","authors":"Marie-Anaïs Roques, Natacha Rosso-Delsemme, Amandine Celli, Ngoc Anh Thu Nguyen, Martin Postzich, Sabine Castet, Yoann Huguenin, Annie Harroche, Anne Lienhart, Sandrine Meunier, Christine Biron-Andréani, Florence Rousseau, Roseline d'Oiron, Yohann Repessé, Clémence Tabélé, Any Beltran Anzola, Thomas Sannié, Nicolas Giraud, Pascal Auquier, Hervé Chambost, Noémie Resseguier, The TRANSHEMO Study Group","doi":"10.1111/hae.70101","DOIUrl":"10.1111/hae.70101","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Haemophilia causes spontaneous or prolonged bleeding due to a deficiency in clotting factor VIII (haemophilia A) or IX (haemophilia B). Although substitutive therapies and regular follow-up can prevent severe haemorrhagic events, adherence to treatment remains a challenge. Transitioning from adolescence to adulthood and from paediatric to adult care is particularly complex for young people with severe haemophilia (PwSH), as it involves gaining autonomy in health management.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Objectives</h3>\u0000 \u0000 <p>This study aimed to explore factors influencing the success of the transition process in young PwSH, with a focus on adherence to healthcare.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>This qualitative study was part of the mixed-methods TRANSHEMO project. Participants were selected from the quantitative phase of the TRANSHEMO project based on two criteria: adolescents/young adults and adherent/nonadherent to healthcare. Interviews were conducted via video conferencing, transcribed, and thematically analysed to identify key themes affecting the transition process.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Twenty-two interviews were conducted. Four major themes emerged as critical to transition success: (1) Care factors [continuity of care, treatment rituals, and evolving therapies]; (2) Family and social factors [support from family, friends, peers, and overprotection]; (3) Personal factors [understanding haemophilia, risk management, optimism, and coping strategies]; and (4) Autonomy [secondary benefits, independence, proactivity in disease management, and accompaniment by caregivers].</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Based on the enlightened determinants, supportive strategies and patient education programs should focus on the development of autonomy, personal factors such as acquisition and application of health literacy in haemophilia care, and family factors such as support from family.</p>\u0000 </section>\u0000 </div>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"31 6","pages":"1197-1208"},"PeriodicalIF":3.0,"publicationDate":"2025-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/hae.70101","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145032699","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Mathieu Fiore, Andrea Artoni, Robert Klamroth, Mary Mathias, Roger Schutgens, Roseline d'Oiron, EAHAD Glanzmann Thrombasthenia Working Group
Introduction
Glanzmann's thrombasthenia is a rare inherited platelet disorder characterized by a lack of platelet aggregation. Patients tend to be diagnosed in early childhood with treatment strategies involving a multifaceted approach to prevent and manage bleeding episodes. Unfortunately, there is currently no European consensus regarding the management of GT.
Aim
This initiative aimed to gain an understanding of current clinical management of GT across Europe, with the aim of aligning best practice and improving patient outcomes.
Methods
The authors, on behalf of the EAHAD Glanzmann Working Group, administered an online survey of 57 questions to European haematologists currently involved in the management of patients with GT. The survey covered topics related to diagnosis, treatment access and selection, immunization, peri-operative management and use of second-line therapies.
Results
Responses reflected physician consensus around some topics, including peri-operative treatment, use of recombinant factor VIIa, and concerns around antibody development. However, more varied responses were received on topics such as antibody screening (anti-αIIbβ3 antibodies screening conducted by ≤53% of respondents in all countries of interest except France), access to HLA-matched platelet concentrates (none or limited for 55% of respondents) and duration of platelet transfusions for major surgery (13%–31% for 1, 2, 3 and 4 or more days of transfusions).
Conclusion
Establishing comprehensive guidelines to manage GT will enhance patient outcomes by ensuring patients receive high-quality and effective care as well as standardize care across different healthcare settings.
{"title":"European Management of Glanzmann's Thrombasthenia: A Survey of Current Clinical Practice","authors":"Mathieu Fiore, Andrea Artoni, Robert Klamroth, Mary Mathias, Roger Schutgens, Roseline d'Oiron, EAHAD Glanzmann Thrombasthenia Working Group","doi":"10.1111/hae.70114","DOIUrl":"10.1111/hae.70114","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Glanzmann's thrombasthenia is a rare inherited platelet disorder characterized by a lack of platelet aggregation. Patients tend to be diagnosed in early childhood with treatment strategies involving a multifaceted approach to prevent and manage bleeding episodes. Unfortunately, there is currently no European consensus regarding the management of GT.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Aim</h3>\u0000 \u0000 <p>This initiative aimed to gain an understanding of current clinical management of GT across Europe, with the aim of aligning best practice and improving patient outcomes.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>The authors, on behalf of the EAHAD Glanzmann Working Group, administered an online survey of 57 questions to European haematologists currently involved in the management of patients with GT. The survey covered topics related to diagnosis, treatment access and selection, immunization, peri-operative management and use of second-line therapies.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Responses reflected physician consensus around some topics, including peri-operative treatment, use of recombinant factor VIIa, and concerns around antibody development. However, more varied responses were received on topics such as antibody screening (anti-<i>αIIbβ3</i> antibodies screening conducted by ≤53% of respondents in all countries of interest except France), access to HLA-matched platelet concentrates (none or limited for 55% of respondents) and duration of platelet transfusions for major surgery (13%–31% for 1, 2, 3 and 4 or more days of transfusions).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Establishing comprehensive guidelines to manage GT will enhance patient outcomes by ensuring patients receive high-quality and effective care as well as standardize care across different healthcare settings.</p>\u0000 </section>\u0000 </div>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"31 6","pages":"1261-1270"},"PeriodicalIF":3.0,"publicationDate":"2025-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/hae.70114","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145033180","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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