Peer-Reviewed Journal Summaries

WFH Publishes First Ever Guidelines for Hemophilia AAV Gene Therapy

Read more about WFH Publishes First Ever Guidelines for Hemophilia AAV Gene Therapy

The new comprehensive and holistic guidelines offer physicians and other prescribers access to the latest evidence-based clinical guidance and help patients make more informed decisions about gene therapy.

Pilot Study Yields Insights on Underserved Groups in Bleeding Disorders Community

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The study authors looked for potential trends in health equity, diversity, and inclusion among Black/African Americans, indigenous people of color (e.g. Native Americans and Asian Pacific Islanders), and Hispanic/Latino people with bleeding disorders.

Authors Discuss Slow Uptake of Hemophilia Gene Therapies

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In terms of hemophilia gene therapy, the authors assert the need for more favorable risk/benefit ratios especially considering the “one-and-done” nature of currently FDA approved AAV-vectors based therapies.

Literature Review Reaffirms Clinical Relevance of Von Willebrand Disease Guideline

Read more about Literature Review Reaffirms Clinical Relevance of Von Willebrand Disease Guideline

A comprehensive literature review was conducted to ensure that the von Willebrand disease guidelines were still clinically appropriate and informed by the research described in more current peer-reviewed papers.

New Paper Features Narrative History of Hemophilia Screening in Women

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A newly published paper takes a sweeping view of hemophilia testing in women, a historically underserved population.

New Article Describes Hemophilia Gene Editing Lexicon

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NBDF, ASGCT, and ISTH publish a new paper offering a standardized lexicon to guide communication on gene editing in hemophilia research and care.

ASH Led Initiative Focuses on Greater Representation in Hematology Clinical Trials

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ASH explores barriers to underrepresentation in hematology clinical trials. Learn about their initiative for greater diversity, equity, and inclusion.

Paper Finds Bone Health Screening Rates at HTCs Represent Clinical Care Gap

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A newly published journal article examined current practices of HTCs for screening and assessing bone health in people with hemophilia and von Willebrand disease.

NBDF Publishes Health Equity Framework to Advance Care for the Bleeding Disorders Community

Read more about NBDF Publishes Health Equity Framework to Advance Care for the Bleeding Disorders Community

NBDF research tackles health equity barriers in bleeding disorders. Learn about our framework for change & access to equitable healthcare.

HTC Looks at Family Pedigrees and Hemophilia Carrier Screening

Read more about HTC Looks at Family Pedigrees and Hemophilia Carrier Screening

Female hemophilia carriers often go undiagnosed. Learn about the importance of identifying and supporting these individuals to ensure equitable access to care.

New Study Looks at GI Bleeding in von Willebrand Disease

Read more about New Study Looks at GI Bleeding in von Willebrand Disease

The authors of this new paper conducted a retrospective medical chart review focused on GI bleeding in von Willebrand disease patients, which poses significant challenges for clinicians.

Study Provides New Data on Neurodevelopment in Younger Hemophilia Population

Read more about Study Provides New Data on Neurodevelopment in Younger Hemophilia Population

Study investigators assessed the impacts of hemophilia type and severity, intracerebral hemorrhage, hemophilia treatment type, socioeconomic status, and neurodevelopmental disorders or symptoms on cognitive, adaptive, and social-emotional functioning compared with the general population.

New Review Weighs Bleeding and Clotting Risk in ITP

Read more about New Review Weighs Bleeding and Clotting Risk in ITP

Primary immune thrombocytopenia is a rare autoimmune disorder that causes a significant reduction in platelets, which are necessary for effective coagulation.

Researchers Look at Impact of Factor X Deficiency on Patients/Caregivers

Read more about Researchers Look at Impact of Factor X Deficiency on Patients/Caregivers

Hereditary factor X deficiency is an ultra-rare bleeding disorder that occurs in approximately 1 in 500,000 to 1 in a million people across the globe.

Study Suggests Hemophilia Patients' Preferences Evolving Along with Treatments

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Study authors looked at hemophilia treatment preferences in light of less burdensome novel therapies.

Pilot Study Explores Educational Needs of HTC Physical Therapists

Read more about Pilot Study Explores Educational Needs of HTC Physical Therapists

The new study explored how new training opportunities based on a peer-to-peer model might be received by U.S. physical therapists at hemophilia treatment centers.

Paper Highlights Treatment for Ultra-Rare Type 1 Plasminogen Deficiency

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Type 1 plasminogen deficiency, also known as hypoplasminogenemia, is a very rare genetic disorder

Gynecologists Have Significant Part to Play in Shrinking Diagnostic Gaps

Read more about Gynecologists Have Significant Part to Play in Shrinking Diagnostic Gaps

People with inheritable bleeding disorders who have the potential or ability to menstruate will likely experience especially heavy menstrual bleeding in their lifetime.

Patient Safety in the Age of Gene Therapy a Central Focus of Review Article

Read more about Patient Safety in the Age of Gene Therapy a Central Focus of Review Article

This paper takes a broad, holistic view of patient safety in the era of hemophilia gene therapy.

Inequities in Thrombosis and Hemostasis Lab Testing the Focus of Illustrated Review

Read more about Inequities in Thrombosis and Hemostasis Lab Testing the Focus of Illustrated Review

Explore a review on biases in laboratory reference intervals for thrombosis and hemostasis, highlighting disparities and action items for health equity.

Gene Therapy as a Novel Approach to Achieving ITI in FVIII Inhibitors Reviewed

Read more about Gene Therapy as a Novel Approach to Achieving ITI in FVIII Inhibitors Reviewed

Explore the potential of gene therapy for hemophilia A with inhibitors. Learn about challenges, current strategies, and its impact on patients.

MLOF Genotyping Project Informed Inhibitor Risk

Read more about MLOF Genotyping Project Informed Inhibitor Risk

Learn about the MLOF Genotyping Project and its insights into inhibitor risk among individuals with hemophilia A. Genetic variations can inform future plans.

Summit Emphasizes Pharmacovigilance in the Inherited Bleeding Disorders Community

Read more about Summit Emphasizes Pharmacovigilance in the Inherited Bleeding Disorders Community

Learn how stakeholders are improving safety communication in the inherited bleeding disorders community. Read more.

Study Suggests Significant Rates of Depression and Anxiety for Individuals with VWD

Read more about Study Suggests Significant Rates of Depression and Anxiety for Individuals with VWD

New study shows significant depression and anxiety rates in von Willebrand disease patients. Learn about the impact and the need for mental health screening.

Paper Features Q&A Resource on Shared Decision Making and Gene Therapy

Read more about Paper Features Q&A Resource on Shared Decision Making and Gene Therapy

The Q&A resource highlighted here is meant to encompass all stages of a patient's decision making journey in gene therapy.

Diabetes Risk in Hemophilia vs Unaffected Groups Compared in Retrospective Study

Read more about Diabetes Risk in Hemophilia vs Unaffected Groups Compared in Retrospective Study

The hemophilia data for this study was drawn from deidentified medical records from four North American hemophilia treatment centers.

New Paper Highlights Integrated Care Model and the National HTC Network

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This paper provides a comprehensive description of the critical services and dynamic coordinating capacity of HTCs within a regional and national framework.

Patient-Reported Data Informs New Study on Sexual Health and Hemophilia

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The data used for this study came from the Patient Reported Outcomes Burdens and Experiences (PROBE) initiative.

Descriptive Study Explores HTC Social Worker Roles

Read more about Descriptive Study Explores HTC Social Worker Roles

While social workers have functioned as essential members of the multidisciplinary care teams at the core of U.S. hemophilia treatment center (HTC) network since its establishment in 1975, a breakdown of their primary roles, both actual and “ideal,” have not been clearly established.

Vector Biology and Hemophilia Gene Therapy the Focus of Review

Read more about Vector Biology and Hemophilia Gene Therapy the Focus of Review

This review provides a comprehensive overview of the scientific underpinnings of several hemophilia gene therapy platforms, with particular focus on the delivery of genetic payloads at the heart of these technologies.

New Descriptive Study Looks at Bleeding Disorder Camps

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While it has been well established that bleeding disorders camps (BDCs) in the U.S.

Review Offers Scientific Overview of AAV Vectors and Hemophilia Gene Therapy

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In this review, the authors discuss some of the primary considerations relevant to investigational gene therapies that employ adeno-associated viral

New Paper Explores Shared Decision Making in Hemophilia

Read more about New Paper Explores Shared Decision Making in Hemophilia

A new paper published in The Journal of Haemophilia Prac

Article Offers Scientific Overview of AAV Vectors and Hemophilia Gene Therapy

Read more about Article Offers Scientific Overview of AAV Vectors and Hemophilia Gene Therapy

In this review, the authors discuss some of the primary considerations relevant to investigational hemophilia gene therapies that employ adeno-associated viral (AAV) vectors.

The First National Patient Satisfaction Survey of U.S. HTCs

Read more about The First National Patient Satisfaction Survey of U.S. HTCs

This undertaking represents the first-of-its kind, a nationally uniform and comprehensive patient satisfaction survey of HTC clinicians, services and care processes.

Establishing a New Paradigm to Realize Health Equity in Hemophilia

Read more about Establishing a New Paradigm to Realize Health Equity in Hemophilia

The authors of this paper assert that an updated treatment model is needed globally for people with hemophilia, one that ulitmately strives for health equity for all.

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