Lived experience experts: a name created by us for us

Expert Review of Hematology, Год журнала: 2023, Номер 16(sup1), С. 7 - 11

Опубликована: Март 15, 2023

People affected by a medical disorder, usually called patients, develop very special expertise living with it every day. They know, better than anyone else, how affects their lives, what they go through to get diagnosis and treatment, treatments affect them, symptoms or side effects impact daily life, is like interact the health care system. The people who share close family members parents, partners, siblings, similar knowledge. When comes research, patients are seen only as subjects. In recent National Hemophilia Foundation State of Science Research Summit subsequent Blueprint project, inherited bleeding disorders were invited participate in creating an agenda most important research that needs be done, designing approach do research. As full Working Groups, leadership roles Blueprint, realized needed title recognizes clearly communicates unique expertise, so work understand bring table. chose term lived experience expert (LEE). Especially rare disorders, LEEs have unique, valuable contribute all stages (e.g. planning designing, participating recruiting participants, communicating its importance results). Including will make stronger.

Язык: Английский

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Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research to advance the health of people with inherited bleeding disorders with the potential to menstruate

Expert Review of Hematology, Год журнала: 2023, Номер 16(sup1), С. 71 - 86

Опубликована: Март 15, 2023

Background People who have or had the potential to menstruate (PPM) with inherited bleeding disorders (BD) face particular challenges receiving appropriate diagnosis and care participating in research. As part of an initiative create a National Research Blueprint for future decades research, Hemophilia Foundation (NHF) American Thrombosis Hemostasis Network conducted extensive all-stakeholder consultations identify priorities PPM BDs those them.Research design methods Working group (WG) 4 NHF State Science Summit distilled community-identified into concrete research questions scored their feasibility, impact, risk.Results WG4 identified important gaps foundational knowledge upon which base optimal BDs. They defined 44 top-priority concerning lifespan sex biology, pregnancy post-partum context, uterine physiology bleeding, bone joint health, health delivery, patient-reported outcomes quality-of-life.Conclusions The needs will best be advanced designed across spectrum gender methodologies outcome measures tailored this population, involving them throughout.

Язык: Английский

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Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities in health services; diversity, equity, and inclusion; and implementation science

Expert Review of Hematology, Год журнала: 2023, Номер 16(sup1), С. 87 - 106

Опубликована: Март 15, 2023

Background The National Hemophilia Foundation (NHF) conducted extensive all-stakeholder inherited bleeding disorder (BD) community consultations to inform a blueprint for future research. Sustaining and expanding the specialized comprehensive Treatment Center care model, better serve all people with BDs (PWIBD), increasing equitable access optimal health emerged as top priorities.Research Design Methods NHF, American Thrombosis Hemostasis Network (ATHN), convened multidisciplinary expert working groups (WG) distill priority research initiatives from consultation findings. WG5 was charged prioritizing services (HSR); diversity, equity, inclusion (DEI); implementation science (IS) advance community-identified priorities.Results identified multiple themes essential capitalizing on this potential. Formative studies using qualitative mixed methods approaches should be characterize issues meaningfully investigate interventions. Investment in HSR, DEI IS education, training, workforce development are vital.Conclusions An enormous amount of work is required areas DEI, IS, which have received inadequate attention BDs. This has great potential evolve experiences PWIBD, deliver transformational community-based care, equity.

Язык: Английский

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Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities for ultra-rare inherited bleeding disorders

Expert Review of Hematology, Год журнала: 2023, Номер 16(sup1), С. 55 - 70

Опубликована: Март 15, 2023

Background Ultra-rare inherited bleeding disorders (BDs) present important challenges for generating a strong evidence foundation optimal diagnosis and management. Without disorder-appropriate treatment, affected individuals potentially face life-threatening bleeding, delayed diagnosis, suboptimal management of invasive procedures, psychosocial distress, pain, decreased quality-of-life.Research design methods The National Hemophilia Foundation (NHF) the American Thrombosis Hemostasis Network identified priorities people with BDs their caregivers, through extensive inclusive community consultations, to inform blueprint future decades research. Multidisciplinary expert Working Group (WG) 3 distilled highly feasible transformative ultra-rare BD research opportunities from community-identified priorities.Results WG3 three focus areas potential advance needs all scored feasibility, impact, risk priority initiatives, including 13 in systems biology mechanistic science; 2 clinical research, data collection, infrastructure; 5 regulatory process novel therapeutics required collection.Conclusions Centralization expansion expertise resources, flexible innovative approaches, inclusion health care professionals will be essential capitalize on outlined herein.

Язык: Английский

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Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities to transform the care of people with hemophilia

Expert Review of Hematology, Год журнала: 2023, Номер 16(sup1), С. 19 - 37

Опубликована: Март 15, 2023

Background Decades of research have transformed hemophilia from severely limiting children's lives to a manageable disorder compatible with full, active life, for many in high-income countries. The direction future will determine whether exciting developments truly advance health equity all people (PWH). National Hemophilia Foundation (NHF) and American Thrombosis Hemostasis Network conducted extensive inclusive all-stakeholder consultations identify the priorities inherited bleeding disorders those who care them.Research design methods Working group (WG) 1 NHF State Science Research Summit distilled community-identified A B into concrete questions scored their feasibility, impact, risk.Results WG1 defined 63 top priority concerning arthropathy/pain/bone health, inhibitors, diagnostics, gene therapy, pediatric adult transition care, disparities faced by community, cardiovascular disease. This has potential empower PWH thrive despite lifelong comorbidities achieve new standards wellbeing, including psychosocial.Conclusions Collaborative delivery be key capitalizing on current horizon treatments harnessing technical advances improve diagnostics testing, PWH.

Язык: Английский

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The National Hemophilia Foundation State of the Science Research Summit initiative: executive summary

Expert Review of Hematology, Год журнала: 2023, Номер 16(sup1), С. 129 - 134

Опубликована: Март 15, 2023

Introduction The National Hemophilia Foundation State of the Science Research Summit initiative sought to unify research efforts in US inherited bleeding disorders (BDs) community around key topics importance people living with BDs, lived experience experts.Areas covered This community-led and -informed project focused on six broad areas – hemophilia A or B; von Willebrand Disease (VWD), platelet dysfunctions other mucocutaneous BDs; ultra-rare unique challenges potential menstruate diversity, equity inclusion, health services research, implementation science; facilitating BD through designing an optimizied infrastructure, enabling resources funding, furthering workforce capabilities required execute priorities.Expert opinion work summarized here, accompanying supplement manuscripts , has implications not only for population but globally who have BDs. information is equally relevant hemophilia, VWD, spectrum disorders, factor deficiencies, all BDs as it care providers researchers treatment globally.

Язык: Английский

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From Vision to Action: National Bleeding Disorders Foundation’s Roadmap for Achieving Health Equity, Diversity, and Inclusion

Health Equity, Год журнала: 2025, Номер 9(1), С. 144 - 160

Опубликована: Янв. 1, 2025

Язык: Английский

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Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: facilitating research through infrastructure, workforce, resources and funding

Expert Review of Hematology, Год журнала: 2023, Номер 16(sup1), С. 107 - 127

Опубликована: Март 15, 2023

Background The National Hemophilia Foundation (NHF) conducted extensive, inclusive community consultations to guide prioritization of research in coming decades alignment with its mission find cures and address prevent complications enabling people families blood disorders thrive.Research Design Methods With the American Thrombosis Hemostasis Network, NHF recruited multidisciplinary expert working groups (WG) distill community-identified priorities into concrete questions score their feasibility, impact, risk. WG6 was charged identifying infrastructure, workforce development, funding resources facilitate prioritized research. Community input on conclusions gathered at State Science Research Summit.Results detailed a minimal capacity infrastructure threshold, opportunities enable attainment, for bleeding centers participate prospective, multicenter national registries. They identified challenges recruit, retain, train diverse care required future. Innovative collaborative approaches trial design, resource networking, surmount obstacles facing rare were elucidated.Conclusions innovations proposed herein may contribute facilitating Blueprint Inherited Bleeding Disorders.

Язык: Английский

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The National Hemophilia Foundation’s State of the Science Research Summit: the foundation of a national research blueprint for inherited bleeding disorders

Expert Review of Hematology, Год журнала: 2023, Номер 16(sup1), С. 1 - 5

Опубликована: Март 15, 2023

KEYWORDS: Bleeding disorderblueprintcommunityhealth equityNational Hemophilia Foundationpatient-centricresearch

Язык: Английский

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Prevalence and risk factors for bleeding in hereditary hemorrhagic telangiectasia: a National Inpatient Sample study

Blood Advances, Год журнала: 2023, Номер 7(19), С. 5843 - 5850

Опубликована: Авг. 11, 2023

Hereditary hemorrhagic telangiectasia (HHT) is a common bleeding disorder, but little known regarding prevalence and risk factors for bleeding. Adult discharges with HHT were identified by International Classification of Disease, 10th edition (ICD-10) codes in the National Inpatient Sample (NIS), 2016-2018. Prevalence estimates weighted using NIS discharge-level weights to reflect national estimates. Risk determined multivariable logistic regression. Among 18 170 849 discharges, 2528 (0.01%) had HHT, whom 648 (25.6%) Arteriovenous malformation (AVM) (31.9% vs 1.3%), angiodysplasia (23.5% 2.3%), (2.3% 0.2%), epistaxis (17.9% 0.6%) more than non-HHT patients (non-HHT), each P < .001. In contrast, menstrual (HMB) postpartum (PPH) less reproductive-age non-HHT, Anemia associated iron deficiency (IDA), was equally or without (15.7% 16.0%), (7.5%), Comorbidities, including gastroesophageal reflux (25.9% 20.0%) cirrhosis (10.0% 3.6%) greater regression, peptic ulcer disease (OR, 8.86; .001), portal vein thrombosis 3.68; = .006), hepatitis C, 2.13; .017) significantly HHT. conclusion, AVM are HMB PPH those non-HHT. IDA as bleeding, suggesting ongoing blood loss need universal screening.

Язык: Английский

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