Soliciting international perspectives on an American national research agenda for inherited bleeding disorders DOI Open Access
Michelle Witkop, Fiona Robinson, Donna DiMichele

и другие.

Expert Review of Hematology, Год журнала: 2023, Номер 16(sup1), С. 13 - 17

Опубликована: Март 15, 2023

KEYWORDS: Communityglobal health equityinherited bleeding disordersinternational perspectivelived experience expertNational Hemophilia Foundationresearch prioritiesresource constraints

Язык: Английский

Lived experience experts: a name created by us for us DOI Open Access

Esmeralda Vázquez,

Michelle Kim, Maria E. Santaella

и другие.

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

Процитировано

27

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 DOI Creative Commons
Maureen K. Baldwin, Homa K. Ahmadzia,

Diane L. Bartlett

и другие.

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

Процитировано

19

Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities for ultra-rare inherited bleeding disorders DOI Creative Commons
Diane J. Nugent,

Suchitra S. Acharya,

Kimberly J. Baumann

и другие.

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

Процитировано

13

The National Hemophilia Foundation State of the Science Research Summit initiative: executive summary DOI Creative Commons
Leonard A. Valentino, Michelle Witkop, Maria E. Santaella

и другие.

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

Процитировано

12

Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities to transform the care of people with hemophilia DOI Creative Commons
Duc Quang Tran,

Craig C Benson,

Judith A. Boice

и другие.

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

Процитировано

12

Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities for mucocutaneous bleeding disorders DOI Creative Commons
Robert F. Sidonio,

Paulette Bryant,

Jorge Di Paola

и другие.

Expert Review of Hematology, Год журнала: 2023, Номер 16(sup1), С. 39 - 54

Опубликована: Март 15, 2023

Background Excessive or abnormal mucocutaneous bleeding (MCB) may impact all aspects of the physical and psychosocial wellbeing those who live with it (PWMCB). The evidence base for optimal diagnosis management disorders such as inherited platelet disorders, hereditary hemorrhagic telangiectasia (HHT), hypermobility spectrum (HSD), Ehlers-Danlos syndromes (EDS), von Willebrand disease (VWD) remains thin enormous potential targeted research.Research design methods National Hemophilia Foundation American Thrombosis Hemostasis Network initiated development a Research Blueprint Inherited Bleeding Disorders extensive all-stakeholder consultations to identify priorities people care them. They recruited multidisciplinary expert working groups (WG) distill community-identified into concrete research questions score their feasibility, impact, risk.Results WG2 detailed 38 high priority concerning biology MCB, VWD, qualitative function defects, HDS/EDS, HHT, disorder unknown cause, novel therapeutics, aging.Conclusions Improving our understanding basic large cohort longitudinal natural history studies, collaboration, creative approaches therapeutics will be important in maximizing benefit future entire MCB community.

Язык: Английский

Процитировано

12

The National Hemophilia Foundation’s State of the Science Research Summit: the foundation of a national research blueprint for inherited bleeding disorders DOI Open Access
Leonard A. Valentino, Michelle Witkop, Maria E. Santaella

и другие.

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

Процитировано

9

Generative AI for Threat Intelligence and Information Sharing DOI
Siva Raja Sindiramutty,

Krishna Raj V. Prabagaran,

N. Z. Jhanjhi

и другие.

Advances in digital crime, forensics, and cyber terrorism book series, Год журнала: 2024, Номер unknown, С. 191 - 234

Опубликована: Сен. 12, 2024

Collaboration in providing threat intelligence and disseminating information enables cyber security professionals to embrace digital most successfully, whose risks are ever-changing. This article dwells on the capacity of machine change by categorising indicators compromise (IOC) actors, then highlights limits traditional methods. Among Artificial tools such as generative adversarial networks (GANs) Variational autoencoders (VAEs), which key innovators, one can create synthetic or fake data that emulates real attack scenarios past. allows cyber-related be analysed differently from before. In addition, this feature secure stakeholder collaborations. It is also meant mainly for factual protects private but exchange helpful information. clear fact showcasing real-world examples demonstrates Al's automation through cybersecurity detection.

Язык: Английский

Процитировано

3

Soliciting international perspectives on an American national research agenda for inherited bleeding disorders DOI Open Access
Michelle Witkop, Fiona Robinson, Donna DiMichele

и другие.

Expert Review of Hematology, Год журнала: 2023, Номер 16(sup1), С. 13 - 17

Опубликована: Март 15, 2023

KEYWORDS: Communityglobal health equityinherited bleeding disordersinternational perspectivelived experience expertNational Hemophilia Foundationresearch prioritiesresource constraints

Язык: Английский

Процитировано

4