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

March 28th 2025

Data from the ATLAS research comprises a series of trials that have yielded years of long-term, comprehensive data on the safety and efficacy of fitusiran (Qfitlia; Sanofi).

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Research Supports Need for Diversity in Hemophilia Trials and Reducing Resource Strain

December 24th 2024

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First Subcutaneous Prophylaxis for Hemophilia A and B With Inhibitors Approved by FDA

December 23rd 2024

Top 5 Rare Blood
Top 5 Most-Read Articles on Rare Blood Disorders in 2024

December 20th 2024

There is a persistent unmet need for more effective therapies for hemophilia. Image credit: Mayava - stock.adobe.com.jpeg
Unmet Needs in Hemophilia: Prophylaxis, Bleeding, Quality of Life

December 12th 2024

Treating PNH: Complement Inhibitors, Administration Modes, and Coordinated Care Needs

Join experts as they explore PNH pathophysiology, treatment strategies, clinical trials, and management challenges. Gain essential insights to enhance patient outcomes and optimize PNH treatment.

View the latest AJMC Peer Exchange on paroxysmal nocturnal hemoglobinuria (PNH)

More News

Sickle Cell Anemia 3D Illustration | Image Credit: © Ezume Images - stock.adobe.com
December 12th 2023

Medication Monitoring Feasible in Patients With SCD

Julia Bonavitacola
A medication monitoring program using medication bottles waws found to be effective in patients with sickle cell disease (SCD), though the usage will need additional support.
FDA Approves Exagamglogene Autotemcel, First CRISPR Gene-Editing Therapy for SCD
December 8th 2023

FDA Approves Exagamglogene Autotemcel, First CRISPR Gene-Editing Therapy for SCD

Julia Bonavitacola
The FDA has approved a gene-editing therapy for sickle cell disease (SCD) for the first time.
FDA Approves Iptacopan, Marking First Oral Monotherapy for Adults With PNH
December 6th 2023

FDA Approves Iptacopan, Marking First Oral Monotherapy for Adults With PNH

Julia Bonavitacola
Iptacopan, the first oral monotherapy for patients with paroxysmal nocturnal hemoglobinuria (PNH), is now an FDA-approved option for improving hemoglobin levels.
Plans for Pregnant Individuals With TTP Should Be Developed for Better Management
December 5th 2023

Plans for Pregnant Individuals With TTP Should Be Developed for Better Management

Julia Bonavitacola
Pregnancy-onset thrombotic thrombocytopenic purpura (TTP) is a challenging condition with 3 distinct subtypes, which should be planned for in adults with a history of TTP.
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November 27th 2023

Poverty Linked to Health-Related Quality of Life in Children With SCD

Julia Bonavitacola
Health-related quality of life was found to be associated with poverty status through parenting stress in children with sickle cell disease (SCD).
Model Accurately Predicts SCD Pain Scores Using Apple Watch Data
November 14th 2023

Model Accurately Predicts SCD Pain Scores Using Apple Watch Data

Julia Bonavitacola
Patients with sickle cell disease (SCD) who wore an Apple Watch and reported daily pain scores could have their pain scores accurately predicted during hospital admission for vaso-occlusive crises and after discharge.
Sickle cell anemia, 3D illustration showing blood vessel with normal and deformed crescent | Image credit: tussik - stock.adobe.com
November 10th 2023

Mitapivat Effective, Safe in Treating SCD Through 1 Year

Julia Bonavitacola
Durable mechanistic effects and a good safety profile were found in patients who took mitapivat for sickle cell disease (SCD).
FDA Approves Adzynma for Treatment of Congenital Thrombotic Thrombocytopenic Purpura
November 10th 2023

FDA Approves Adzynma for Treatment of Congenital Thrombotic Thrombocytopenic Purpura

Julia Bonavitacola
Adzynma is the first product to be approved for the treatment of the rare blood disorder that has 90% mortality when left untreated.
Sickle-cell and normal red blood cells | Image credit: extender_01 - stock.adobe.com
November 7th 2023

Children Hospitalized for Sickle Cell Anemia More Commonly Have AKI

Julia Bonavitacola
Acute kidney injury (AKI) was found to be common in children aged 17 years and younger who were hospitalized for sickle cell anemia.
Pegcetacoplan Found to Be Cost-Effective Approach to Treating PNH
November 4th 2023

Pegcetacoplan Found to Be Cost-Effective Approach to Treating PNH

Julia Bonavitacola
Patients with paroxysmal nocturnal hemoglobinuria (PNH) who had suboptimal response to eculizumab can switch to pegcetacoplan for a cost-effective alternative, according to. acost analysis.
Caplacizumab Effective, Safe in Treating Immune TTP
November 1st 2023

Caplacizumab Effective, Safe in Treating Immune TTP

Julia Bonavitacola
Immune thrombotic thrombocytopenic purpura (TTP) can be treated safely and effectively with caplacizumab.
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November 1st 2023

What We’re Reading: Panel on Sickle Cell Gene Therapy; Infant Mortality Rises; Campaign Against Burnout

AJMC Staff
A panel of experts agreed that exa-cel, a sickle cell disease gene therapy, was safe enough for clinical use, setting the stage for a potential FDA approval; the United States saw an increase in infant mortality rates for the first time in more than 2 decades; the new antiburnout campaign from the CDC asks leaders to better support health care workers.
What We’re Reading: Pharmacy Staff Stage Nationwide Walkout; Dads Embrace Paid Family Leave; FDA Reviews Therapy for Sickle Cell Disease
October 30th 2023

What We’re Reading: Pharmacy Staff Walkout; Male Parental Leave; FDA Reviewing SCD Therapy

AJMC Staff
Pharmacy workers embark on a multiday strike; Fathers are taking paid family leave to bond with their newborns; the FDA is set to evaluate a pioneering gene therapy for sickle cell disease (SCD).
Eculizumab Shows Long-Term Efficacy, Safety in PNH
October 26th 2023

Eculizumab Shows Long-Term Efficacy, Safety in PNH

Julia Bonavitacola
Paroxysmal nocturnal hemoglobinuria (PNH) could be effectively treated by using eculizumab, which was found to be safe in patients.
Racial, Ethnic Differences Persist in Nurses, Nursing Assistants When Treating Sickle Cell Disease
October 25th 2023

Racial, Ethnic Differences Persist in Nurses, Nursing Assistants When Treating Sickle Cell Disease

Julia Bonavitacola
Biases in care for young adults with sickle cell disease could lead to lower quality of care for these patients.
Non-ADAMTS13 Parameters Could Benefit Differential Diagnosis in TTP
October 20th 2023

Non-ADAMTS13 Parameters Could Benefit Differential Diagnosis in TTP

Julia Bonavitacola
Including biomarkers for non-ADAMTS13 in routine clinical testing could benefit diagnosis and follow-up in patients with immune-mediated thrombotic thrombocytopenic purpura (TTP).
Heavy Menstrual Bleeding, Dysmenorrhea High in Adolescents With Sickle Cell Disease
October 17th 2023

Heavy Menstrual Bleeding, Dysmenorrhea High in Adolescents With Sickle Cell Disease

Julia Bonavitacola
Young women with sickle cell disease have a higher prevalence of heavy menstrual bleeding and dysmenorrhea.
Lactate Dehydrogenase Levels Associated With Fatigue, QOL in Patients With PNH
October 13th 2023

Lactate Dehydrogenase Levels Associated With Fatigue, QOL in Patients With PNH

Julia Bonavitacola
Patients with paroxysmal nocturnal hemoglobinuria (PNH) had decreased quality of life (QOL) and worse fatigue that could be linked to lactate dehydrogenase levels.
Platelet Count Acted as Predictor of Relapse in TTP
October 11th 2023

Platelet Count Acted as Predictor of Relapse in TTP

Julia Bonavitacola
The 3-year relapse and mortality rates for thrombotic thrombocytopenic purpura (TTP) were 23.6% and 26.3%, predicated on platelet count.
Children With SCD Have Limited Gastrointestinal Evaluations
October 6th 2023

Children With SCD Have Limited Gastrointestinal Evaluations

Julia Bonavitacola
Although children with sickle cell disease (SCD) frequently present with abdominal pain, limited gastrointestinal evaluations are performed for them.
Sickle Cell Disease Associated With Severe COVID-19, Mortality
October 3rd 2023

Sickle Cell Disease Associated With Severe COVID-19, Mortality

Julia Bonavitacola
Severe cases of COVID-19 and COVID-19–related deaths were associated with sickle cell disease.
Adverse Effects of Hospitalization for Vaso-Occlusive Crises Persist for 12 Months
September 28th 2023

Adverse Effects of Hospitalization for Vaso-Occlusive Crises Persist for 12 Months

Julia Bonavitacola
Children with sickle cell disease who require hospitalization for vaso-occlusive crises can experience adverse effects for up to 12 months following hospitalization, a study found.
Doctor talking to patient
September 22nd 2023

Patient-Reported Outcome Measures Pick Up Patient Experiences in Patients With TTP

Julia Bonavitacola
Patients with thrombotic thrombocytopenic purpura (TTP) benefited from patient-reported outcome measures to report their quality of life.
Relative Benefit in Survival, Reduction in TE/MAVE Risk With Eculizumab for Patients With PNH
September 19th 2023

Relative Benefit in Survival, Reduction in TE/MAVE Risk With Eculizumab for Patients With PNH

Julia Bonavitacola
When patients with paroxysmal nocturnal hemoglobinuria (PNH) received eculizumab, survival rates increased and thromboembolic events/major adverse vascular events (TE/MAVE) decreased.
Study Identifies Risk Factors for Thromboembolic Events in Patients With PNH
September 14th 2023

Study Identifies Risk Factors for Thromboembolic Events in Patients With PNH

Julia Bonavitacola
Researchers found indicators that increased the risk of thromboembolic events in patients with paroxysmal nocturnal hemoglobinuria (PNH).
Repeated Molecular Testing Indicates Dynamic Disease Profile in Patients With PV, ET
September 12th 2023

Repeated Molecular Testing Indicates Dynamic Disease Profile in Patients With PV, ET

Julia Bonavitacola
Patients with polycythemia vera (PV) and essential thrombocythemia (ET) require repeated molecular testing and improved prognostic tools.
Processing Speed May Be Slower in People Who Have SCD
September 8th 2023

Processing Speed May Be Slower in People Who Have SCD

Julia Bonavitacola
Patients who have with sickle cell disease (SCD) could have a slower processing speed and higher cerebral blood flow compared with controls.
Blood cells
September 5th 2023

Imatinib Could Be Therapeutic Option for Treating Painful Sickle Cell Crises

Julia Bonavitacola
Painful crises in sickle cell disease or sickle cell anemia could potentially be treated using imatinib as a therapeutic option, a small study found.
Group of doctors
August 28th 2023

Study Finds Simulation Useful to Boost Awareness of Osteomyelitis in SCD Among Residents, Fellows

Julia Bonavitacola
A simulation case for emergency medicine residents was found to be helpful as a review of the medical diagnosis and management of osteomyelitis in patients with pediatric sickle cell disease (SCD).
Brain
August 15th 2023

Pain, Fatigue Not Associated With Neurocognitive Performance in Adolescents With SCD

Julia Bonavitacola
In a study of adolescents and young adults with sickle cell disease (SCD), researchers did not find a link between neurocognitive performance and pain and fatigue.
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