Hemophilia

Latest News
March 28th 2025

FDA Approves Fitusiran for Reducing Bleeds in Patients With Hemophilia A/B

July 26th 2024

Hemophilia A Gene Therapy Superior to Standard of Care in Phase 3 Trial

June 28th 2024

Field Study Highlights Differences in Factor IX Variant Lab Results

June 26th 2024

In Patients With Hemophilia A, Physical Activity Levels Low

June 20th 2024

AAV5 Gene Therapy for Severe Hemophilia A Successfully Enables FVIII Production

Video Interviews
View All
October 14th 2023

Dr Sarah Wall: Challenges Faced by Older Patients With Hematologic Disorders

September 6th 2023

Dr Margaret Ragni Sheds Light on the Necessity of Testing Hemophilia Carriers

December 10th 2022

Dr Steven Pipe: What to Watch for in the Hemophilia Therapy Pipeline

December 2nd 2022

Dr Steven Pipe on Current Trends in Hemophilia Incidence

May 13th 2022

Walgreens’ Ray Tancredi Weighs in on Expensive Hemophilia Gene Therapies in the Pipeline

Continuing Medical Education
View All
March 29th 2025

Addressing the Gaps and Clinical Challenges in Chronic Lymphocytic Leukemia: Updates and Strategies for Managed Care

1.5 Credits / Hematology, Hematologic Cancer, Oncology
March 29th 2025

Keeping Pace With New Developments in Multiple Myeloma: Updates and Strategies to Optimize Bispecific Antibodies and CAR T-Cell Therapy

1.5 Credits / Oncology, Hematology, Hematologic Cancer
March 29th 2025

ASH Annual Meeting Coverage: Highlighting the Recent Updates in TKI Use in the Treatment of CML – Insights and Application for Managed Care

1.0 Credit / Oncology, Hematology, Hematologic Cancer

More News

hemophilia | Image Credit: jarun011-stock.adobe.com
June 13th 2024

Emicizumab Has Strong Safety, Efficacy Record in Real-World Usage

Jared Kaltwasser
The most common treatment-related adverse events were injection site reactions, the study found.
Blood Cells - Катерина Євтехова - stock.adobe.com
June 7th 2024

CHESS II: Hemophilia Severity Correlates With Economic Burden, Patient QOL

AJMC Contributor
The data come from the observational Cost of Hemophilia in Europe: A Socioeconomic Survey II (CHESS II) study of 288 Spanish patients with hemophilia A and B, which showed certain differences between disease subtypes but overall similar trends in disease impact.
Hemophilia test | Image Credit: jarun011-stock.adobe.com
May 29th 2024

Simoctocog Alfa Effective, Safe in Children With Severe Hemophilia A

Kimberly Rath, PharmD
Recent studies reveal that simoctocog alfa (Nuwiq) offers effective prophylaxis and treatment for severe hemophilia A in children, with low inhibitor rates and strong long-term safety and efficacy.
Children in school | Image credit: Vasyl - stock.adobe.com
May 24th 2024

Hemophilia Not Linked With Cognitive and Behavioral Outcomes in Children

Jared Kaltwasser
However, investigators said there is some evidence suggesting attention-deficit/hyperactivity disorder may be more common in certain age groups of children with hemophilia.
Red blood drop with double helix | Image Credit: Елена Бутусова-stock.adobe.com
May 18th 2024

Enhancing Outpatient Hemophilia Care May Improve Health Outcomes and Costs

AJMC Contributor
Prospective data from patients attending a rural practice in West Virginia between 2016 and 2023 show that a quality improvement program that followed guideline recommendations and was tailored to specific patient needs reduced preventable bleeds and lowered costs.
Bleeds and Resource Use in Hemophilia B: Retrospective Observational Study
May 15th 2024

Bleeds and Resource Use in Hemophilia B: Retrospective Observational Study

Lisa Young, MSc Lu Ban, PhD Yong Chen, PhD, MHS Patrick F. Fogarty, MD
This real-world US study describes individuals with hemophilia B who experience bleeds despite factor replacement therapy and quantifies the associated comorbidity and health care resource utilization burden.
Worried young woman | Image Credit: © SB Arts Media - stock.adobe.com
May 10th 2024

Health Equity, Accessibility Essential to Supporting Women Affected by Hemophilia

Luke Halpern
Due to often being overlooked as only carriers of hemophilia, the review authors sought to highlight the needs of female caretakers for patients with the disorder and the possibility of women themselves being affected by it.
heartbeats | Image Credit: Robert - stock.adobe.com
May 8th 2024

Coexistence of HIV, Hemophilia May Lead to Increased Presence of Coronary Artery Stenosis

Luke Halpern
In patients living with HIV, the cardiovascular risk factors that come with infection may lead to an increased prevalence of coronary artery stenosis, especially in those who also have hemophilia.
DNA helix inside blood drop | Image Credit: © Елена Бутусова - stock.adobe.com
May 3rd 2024

Review: One-Shot Etranacogene Dezaparvovec Represents Game-Changing Gene Therapy for Hemophilia B

Gayle Turim Dickstein
More than 20 years of gene therapy clinical research have led to etranacogene dezaparvovec’s use in hemophilia B, according to authors of a recent review—and it takes its place in a line of current and potentially many future genomic medicines for a range of diseases.
Concept of FDA approved | Image credit: wladimir1804-stock.adobe.com
April 26th 2024

FDA Approves One-Time Gene Therapy Fidanacogene Elaparvovec for Hemophilia B

Rose McNulty
The FDA has approved fidanacogene elaparvovec-dzkt (Beqvez; Pfizer), a gene therapy administered in a one-time dose, for certain patients with moderate to severe hemophilia B.
HemophiliaA | Image Credit: MQ-Illustrations - stock.adobe.com
April 25th 2024

Males With Hemophilia A Report Physical, Mental Burdens of Disease Regardless of Severity

AJMC Contributor
Self-reported data from adult and pediatric males with hemophilia A show that burden of the disease persists regardless of severity, highlighting a need for improved prophylactic treatment.
Hemophilia defined | Image Credit: VitezslavVylicil - stock.adobe.com
April 16th 2024

Long-Term Data Demonstrate Emicizumab Safety, Efficacy in Hemophilia A

Jared Kaltwasser
The therapy bridges activated factors IX and X to mimic the function of missing or deficient factor VIII.
Hemophilia | Image Credit: lexiconimages - stock.adobe.com
April 12th 2024

Study Suggests Walking 10K Steps Daily Improves QOL in Severe Hemophilia

AJMC Contributor
Among a small group of patients with severe hemophilia, researchers found that getting in more than 10,000 steps each day was linked to significant improvements in perceived physical health and quality of life (QOL).
Blood Cells | Image Credit: © Катерина Євтехова - stock.adobe.com
April 6th 2024

Clot Formation, Fibrinolysis Assays May Help Show Important Differences Between Hemophilia A Treatments

AJMC Contributor
Researchers found that in vitro clot formulation and fibrinolysis assays were able to determine the hemostatic effects of different replacement agents, as well as identify distinct pharmacodynamic responses across the different mechanisms of action.
blood samples | Image credit: Daniel CHETRONI – stock.adobe.com
April 5th 2024

Small Study Shows Safety, Efficacy of Prophylactic rIX-FP in Previously Untreated Hemophilia B

AJMC Contributor
Findings from this new study demonstrate the safety and efficacy of rIX-FP among a group of treatment-naive patients often characterized by young age and particular risk for severe bleeding.
Menstruation Period Accessories - Atlas - stock.adobe.com
January 8th 2024

Breaking Barriers in Bleeding Disorders: Experts Call for More Data on Women

Giuliana Grossi
“These autosomal diseases, from an inheritance standpoint, affect men and women equally, but because of their impact on the gynecological system and menses, women are often affected much more than men," hematologist Donna DiMichele, MD, explains.
Red Blood Cells - Design Cells stock.adobe.com.jpeg
November 24th 2023

Subcutaneous OCTA101 Trial in Severe Hemophilia A Halted Due to Safety Concerns

Giuliana Grossi
In a trial assessing subcutaneous OCTA101 in patients with severe hemophilia A, promising results in pharmacokinetics and efficacy were overshadowed by safety concerns.
Professional Nurse Preparing Treatment - Viacheslav Yakobchuk - stock.adobe.com
November 14th 2023

Study Reveals Racial Disparities in Immune Tolerance Induction Treatment for Hemophilia

Giuliana Grossi
New research highlights racial disparities in hemophilia care, revealing that Black and Hispanic patients are significantly less likely to receive immune tolerance induction treatment compared with White patients, even after considering clinical factors.
Genetic engineering and gene manipulation concept - andrianocz - stock.adobe.com.jpeg
November 1st 2023

Etranacogene Dezaparvovec Gene Therapy Outperforms FIX Products in Hemophilia B

Giuliana Grossi
The findings from a recent study unveiled notable distinctions in bleeding rates between etranacogene dezaparvovec and the standard prophylactic factor IX (FIX) products among individuals diagnosed with hemophilia B.
Asian doctor woman image credit Nattakorn stock.adobe.com.jpeg
October 28th 2023

Study Identifies Core Instruments for Monitoring Physical Function in Hemophilia

Giuliana Grossi
The study identified practical instruments for monitoring physical function in people with hemophilia by pinpointing 11 potential markers and establishing performance-based tests for 5 of these activities.
Brain illustration | Image credit: jolygon - stock.adobe.com
October 27th 2023

ANNEXA-I: Andexanet Alfa Shows Efficacy, Safety for Factor Xa Reversal in Acute ICH

Rose McNulty
The phase 4 ANNEXA-I trial was stopped early after showing superior hemostatic efficacy and the capability to limit potentially life-threatening intracerebral hemorrhage (ICH) compared with usual care in patients taking oral factor Xa (FXa) inhibitors.
Set IV Fluid | Image Credit: Trsakaoe stock.adobe.com
October 26th 2023

Damoctocog Alfa Pegol Demonstrates Safety and Efficacy in Hemophilia A

Giuliana Grossi
While clinical trials offer controlled data, real-world studies like HEM-POWR bridge gaps, confirming damoctocog alfa pegol’s effectiveness and tolerance, establishing it as a valuable hemophilia A treatment option.
Red Blood Cells | Image Credit: Design Cells - stock.adobe.com
October 12th 2023

Evaluating WHO's Hemophilia Treatment Choices: Patient Safety, Access Challenges

Giuliana Grossi
A commentary questions the characterization of cryoprecipitate, which is not pathogen-reduced, as an alternative modality for treating hemophilia, despite its substantial risk of transmitting blood-borne pathogens to patients.
What We’re Reading: Rising Breast Cancer Rates; Pharmacists Walkout Over Working Conditions; School Blood Drives on Hold
October 11th 2023

What We’re Reading: Rising Breast Cancer Rates; Pharmacists Walkout; School Blood Drives on Hold

AJMC Staff
Advances in treatments and early detection methods have led to improved survival rates; staffing shortages and rising demands take their toll; the US blood supply is under increasing strain.
Doctor using digital technology | Image credit: otello-stpdc - stock.adobe.com
September 30th 2023

The Framework for Embracing Gene Therapy in Hemophilia Treatment

Giuliana Grossi
Although gene therapy is anticipated to bring positive changes for people with hemophilia, choosing to receive gene therapy will require empowerment, participation, and shared decision-making, investigators write.
Image credit: Катерина Євтехова | stock.adobe.com
September 29th 2023

Altuviiio sBLA for Severe Hemophilia A in Children Accepted by FDA

Ashley Gallagher
The supplemental biologics license application (sBLA) is supported by the results of the XTEND-Kids study, demonstrating a once-weekly 50 IU/kg dose of antihemophilic factor (recombinant) provides effective bleed protection in children with hemophilia A and can be used across clinical scenarios.
Blood Transfusion | Image credit: smolaw11 – stock.adobe.com
September 23rd 2023

Hemophilia Survival Rates Improve, Black Men Still Fall 10 Years Behind

Giuliana Grossi
The median age of death remained consistently 10 years lower for Black and Hispanic males in comparison to White males, underscoring a critical area for further attention.
Doctor Conducting Ultrasound | Image credit: Africa Studio – stock.adobe.com
September 14th 2023

Study Reveals Prevalence of Subclinical Synovial Proliferation in Hemophilia A

Giuliana Grossi
The findings highlight the critical role that subclinical synovial proliferation plays in the progression of hemophilic arthropathy.
Hemophilia Carriers' Reproductive Decisions Linked to Childhood Trauma
September 9th 2023

Hemophilia Carriers' Reproductive Decisions Linked to Childhood Trauma

Giuliana Grossi
A study found that negative experiences and concern for future children's health drive hemophilia carriers to choose prenatal tests, with cognitive factors playing a role.
patient and doctor sitting down talking
August 23rd 2023

Patients, Clinicians Need Hemophilia Gene Therapy Shared Decision-Making Tool

Victoria Johnson
Clinicians and nurses who treat patients with hemophilia with a gene therapy identified important factors and considerations needed in a potential shared decision making tool.
123>

2 Commerce Drive
Suite 100
Cranbury, NJ 08512

© 2025 MJH Life Sciences® and AJMC®.
All rights reserved.

About
Contact
Editorial Boards
Advertise
Do Not Sell My Information
Terms & Conditions
Privacy Policy
AJMC Managed Markets Network Logo
CH LogoCenter for Biosimilars Logo