Peer-Reviewed Journal Summaries

New Review Weighs Bleeding and Clotting Risk in ITP

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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

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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

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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

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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

Review Offers Scientific Overview of AAV Vectors and Hemophilia Gene Therapy

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

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.

Nationwide Children’s Hospital Looks at Optimal Dosing in the Emergency Department

Read more about Nationwide Children’s Hospital Looks at Optimal Dosing in the Emergency Department

A team of physicians and pharmacists analyzed factor dosing of hemophilia patients at the emergency department of Nationwide Children’s Hospital, targeting out-of-range and optimal dosing data and subsequent educational interventions.

PT Study Focuses on Comprehensive Survey of Physical Activities Risks for 101 Activities

Read more about PT Study Focuses on Comprehensive Survey of Physical Activities Risks for 101 Activities

A veteran group of HTC physical therapists conduct comprehensive review of the ranges and risk drivers associated with 101 activities, including specific bleeding risks in six joints and three injury types, bruising, head injury, and muscle bleeding.

San Diego HTC Looks at Cardiovascular Disease in Young People with Hemophilia

Read more about San Diego HTC Looks at Cardiovascular Disease in Young People with Hemophilia

In this study, researchers uncovered a relatively high rate of established risk factor amongst young people hemophilia, then discuss possible interventions for prevention.

Non-Attendance of Clinic Visits at Emory HTC the Focus of New Study

Read more about Non-Attendance of Clinic Visits at Emory HTC the Focus of New Study

Emory HTC researchers uncovered some noteworthy findings in this unique study on clinic non-attendance rates, including a small at-risk subgroup that may warrant further investigations.

Updates on My Life Our Future Genotyping Project

Read more about Updates on My Life Our Future Genotyping Project

The MLOF Genotyping project is a valuable resource for the hemophilia community.The MLOF Genotyping project has enrolled over 11,000 participants

New Study on Cardiovascular Disease in Hemophilia Patients

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HTC researchers conducted this study in order to substantiate the long-held theory that individuals with hemophilia are largely protected from cardiovascular disease.

New Study on Aging, VWF Levels and Bleeding Risk

Read more about New Study on Aging, VWF Levels and Bleeding Risk

Investigators look to establish whether VWF levels increase as patients with VWD age, and if so, whether this can be associated with lower risk for bleeding.

Studying the Effectiveness of pdFX in Females with Factor X

Read more about Studying the Effectiveness of pdFX in Females with Factor X

In this ad hoc study based on a plasma-derived factor X concentrate, investigators focus on the therapy’s efficacy in a small but particularly vulnerable subgroup, females with factor X deficiency.

Study Suggests Prophylaxis Could Reduce Hospitalizations for VWD Patients

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Swedish and U.S. researchers sought to uncover potential correlations between VWD prophylaxis and hospitalizations.

Retrospective Study Reflects Successful Transition to Extended Half-Life Therapies

Read more about Retrospective Study Reflects Successful Transition to Extended Half-Life Therapies

Investigators at one HTC reported positive clinical outcomes for hemophilia patients who switched from a standard half-life to an extended half-life therapy.

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