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November 7th 2024

Next-Gen Hemophilia Treatments Offer Hope for Joint Health, but Accessibility Challenges Remain

November 5th 2024

Polycythemia Vera Management: Addressing the Burden of Symptoms and Phlebotomy Dependence

October 30th 2024

High Health Care Burden of Transfusion-Dependent β-Thalassemia

October 30th 2024

New AI Tool Identifies Undiagnosed PNH in Health Records

October 28th 2024

HSSP Pharmacists Demonstrate Substantial Cost Avoidance in Specialty Medication Use

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)
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June 7th 2024

Ongoing Research in Plasminogen Deficiency Type 1: Dr Amy Shapiro

May 28th 2024

Patients With Plasminogen Deficiency Should Receive Care at Hemophilia Treatment Centers: Dr Amy Shapiro

May 20th 2024

Ryplazim Marks First FDA Approval for Plasminogen Deficiency: Dr Amy Shapiro

May 14th 2024

Ligneous Lesions Often Misdiagnosed in Rare Plasminogen Deficiency: Dr Amy Shapiro

August 19th 2023

Dr Kevin Davies Discusses Challenges of Streamlining CRISPR Technology Delivery

April 20th 2023

Dr David Eagle: Progress in Hematology/Oncology Is Not Without Its Difficulties

More News

hand in rubber gloves holding a hormonal implant - Celso Pupo - stock.adobe.com
October 23rd 2024

Etonogestrel Contraceptive Implants Reduce Pain Crises in Women With Sickle Cell Disease

Giuliana Grossi
Etonogestrel-releasing contraceptive implants in women with sickle cell disease significantly reduced pain intensity and frequency of pain crises over 12 months, with no adverse changes in metabolic or liver function markers.
Hands holding blood, give blood donation, blood transfusion, world blood donor day, world hemophilia day concept - SewcreamStudio - stock.adobe.com
Published: October 16th 2024 | Updated: October 16th 2024

Access Has Improved for Diverse Patients Needing Umbilical Cord Blood Transplant, but Disparities Remain

Giuliana Grossi
Despite advancements in umbilical cord blood transplants, social determinants of health, such as poverty and public insurance, continue to impact outcomes, underscoring the need for equitable access to lifesaving treatments.
Red blood cells | Image credit: Getty Images
October 11th 2024

FDA Approves Marstacimab, First Weekly Sub-Q Option for Hemophilia B

Mary Caffrey
Switching patients with hemophilia B to gene therapy may compound immense health care savings in the long term | image credit: ejgrubbs - stock.adobe.com
September 25th 2024

Gene Therapy for Hemophilia B Could Lower Health Plans’ Budget Over Time

Jared Kaltwasser
Health plans would see savings faster if they switched to etranacogene dezaparvovec sooner, the investigators found.
Sickle cell anemia affects an estimated 100,000 people in the US | image credit: Studios - stock.adobe.com
September 20th 2024

Urinary Angiotensinogen Predicts Persistent Albuminuria in Sickle Cell Anemia

Karen Jacobson-Sive
Patients with sickle cell anemia (SCA) and persistent albuminuria (PA) exhibited significantly higher levels of urinary angiotensinogen (AGT).
Genetic variation and sex-specific differences may be related to pain in SCD | image credit: Lila Patel - stock.adobe.com
September 18th 2024

Pain in SCD: Are Genetic Variations Responsible?

Nameera Temkar, MD
Genetic variability and sex-specific differences may play a role in sickle cell disease (SCD)-related pain; however, more research is needed.
Patients with SCD have reportedly felt stigmatized due to their race, pain, and more | image credit: Andrey Popov - stock.adobe.com
September 11th 2024

Study: Patients With SCD Report Discrimination Based on Race, Pain, and Other Factors

Jared Kaltwasser
A new report based on patient focus groups suggests many clinicians can do more to help patients with sickle cell disease (SCD) feel heard and understood.
Despite treatment, some patients with PNH still experience anemia | image credit: ImageFlow - stock.adobe.com
August 28th 2024

Pegcetacoplan May Reduce Fatigue in PNH Accompanied by Anemia

Kyle Munz
Findings from a post hoc analysis of 3 clinical trials support the switching to or initiation of pegcetacoplan to manage fatigue for patients with paroxysmal nocturnal hemoglobinuria (PNH) that is accompanied by mild to moderate anemia.
Quality of life in patients with hemophilia A or B benefits from the use of concizumab | image credit: Patrick Daxenbichler - stock.adobe.com
August 18th 2024

Patient-Reported Outcomes Favor Concizumab Prophylaxis in Hemophilia A or B

Connor Iapoce
Analysis of the phase 3 explorer7 (NCT04083781) study indicates a preference for concizumab prophylaxis over no prophylaxis in patients with hemophilia A or B with inhibitors.
Patients with PNH may become more active following ravulizumab therapy | image credit: sutadimages - stock.adobe.com
August 16th 2024

Ravulizumab Boosts Activity Levels, Quality of Life in Patients With PNH

Connor Iapoce
Ravulizumab therapy may enable patients with paroxysmal nocturnal hemoglobinuria (PNH) to comparable achieve activity levels and patient-reported outcomes to the US general population.
Sickle cell retinopathy is a manifestation of SCD that occurs in the eye and can cause detriments to patients’ vision| image credit: Катерина Євтехова - stock.adobe.com
August 5th 2024

The Benefits of More Sickle Cell Retinopathy Screening for Patients With SCD

Kyle Munz
A systematic analysis that reviewed screening for sickle cell retinopathy in patients with sickle cell disease (SCD) underscores the importance of advanced imaging measures and further research on screening implementation methods.
COVID-19 vaccine injection | image credit: insta_photos - stock.adobe.com
July 31st 2024

COVID-19 Vaccination Shows Favorable Risk-Benefit in Sickle Cell

Connor Iapoce
A study from the American Society of Hematology (ASH) Research Network reports positive antibody response and tolerability after mRNA vaccination in people with sickle cell disease.
Efanesoctocog alfa is a high-sustained factor VIII replacement therapy for the treatment of hemophilia A | image credit: Ekaterina - stock.adobe.com
July 25th 2024

Efanesoctocog Alfa Prophylaxis Promising for Pediatric Severe Hemophilia A

Kyle Munz
The phase 3, international, open-label, single-group XTEND-Kids study concluded that children with severe hemophilia A younger than 12 years benefited from treatment with efanesoctocog alfa prophylaxis.
Hemolysis is the destruction of red blood cells, a common symptom of PNH | image credit: Red Lemon - stock.adobe.com
July 22nd 2024

Risk Mitigation for Hemolysis Is Needed During PNH Treatment, Study Finds

Kyle Munz
The phase 3 PEGASUS trial found that patients with paroxysmal nocturnal hemoglobinuria (PNH) may still experience anemia following recommended therapies, underscoring the need for further research.
Fatigue is one of the most prevalent non-painful symptoms reported in SCD | image credit: Feng Yu - stock.adobe.com
July 20th 2024

Pain Threshold Predictive of Fatigue Severity in SCD, Study Finds

Kyle Munz
A cross-sectional study found that static pain measures could be useful for predicting fatigue severity in patients with sickle cell disease (SCD).
Clotting complications are a severe symptom of TTP | image credit: Artur - stock.adobe.com
July 15th 2024

Patients With ADAMTS13 Deficiency and/or TTP Experience High Clinical Burden

Kyle Munz
A retrospective database analysis demonstrated the high clinical burden experienced by patients with severe deficiencies of ADAMTS13 and thrombotic thrombocytopenic purpura (TTP)–related diagnoses.
Children and adults with SCD can exhibit various cognitive deficits or neurological injury that could be related to their disease | image credit: Werckmeister - stock.adobe.com
July 9th 2024

Cognitive Health Unaffected by Diluted Perivascular Spaces in Children With SCD

Kyle Munz
An investigation into the burden of dilated perivascular spaces (dPVS) in children with sickle cell disease (SCD) confirmed that those with SCD experience more severe manifestations of dPVS compared with the general population.
Patients with PNH stand at an increased risk for thrombotic, among other, events | image credit: Bipul Kumar - stock.adobe.com
June 30th 2024

Crovalimab Offers Effective, Less Burdensome Alternative to Eculizumab for PNH Treatment

Kyle Munz
For patients with paroxysmal nocturnal hemoglobinuria (PNH), crovalimab has emerged as an additional treatment option with a similar safety and efficacy profile to eculizumab,
The fertility risks that accompany SCD are not completely understood by impacted patients | image credit: Prostock-studio - stock.adobe.com
June 28th 2024

Fertility Risks and Treatments Misunderstood by Patients With SCD

Kyle Munz
Results from a cross-sectional study revealed the gaps in knowledge related to fertility risks among patients with sickle cell disease (SCD), as well as shed light on patient perspectives on fertility treatments.
Data shows that pregnant individuals have 3 times the risk of contracting COVID vs the general population | image credit: Evgeniya Primavera - stock.adobe.com
June 20th 2024

Sickle Cell Trait Can Exacerbate COVID-19 Risk During Pregnancy

Kyle Munz
Sickle cell trait (SCT) and pregnancy each exacerbate the symptoms of COVID-19. As a result, pregnant individuals with SCT may have a higher risk of COVID-19 infection.
World Sickle Cell Awareness Day was established on June 19, 2009 | image credit: SaturnO_27 - stock.adobe.com
June 19th 2024

World Sickle Cell Awareness Day: Navigating the Present and Shaping the Future of Sickle Cell Disease

Kyle Munz Giuliana Grossi
June 19 is marked as World Sickle Cell Awareness Day, granting the opportunity to reflect on the current state of treatment, access, equity, and more that impacts patients living with sickle cell disease.
Blood clot forming | image credit: Tatiana Shepeleva - stock.adobe.com
June 5th 2024

Prophylaxis With Recombinant ADAMTS13 Demonstrates Superiority vs Standard Therapy in Congenital TTP

Kyle Munz
The multinational, open-label, controlled, randomized, crossover, phase 3 TAK-755 (NCT03393975) trial demonstrated superior efficacy and safety of recombinant ADAMTS13 as prophylaxis in patients with congenital thrombotic thrombocytopenic purpura (cTTP) compared with standard therapy.
Blood for Transfusion PNH Concept | image credit: sebgross - stock.adobe.com
May 29th 2024

Pegcetacoplan Contributes to Better Outcomes in PNH vs Other Treatments

Kyle Munz
An analysis of 2 phase 3 trials supporting the use of pegcetacoplan (PEG) in the treatment of paroxysmal nocturnal hemoglobinuria (PNH) assessed the benefits of this treatment compared with eculizumab (ECU) and supportive care measures.
Hands Holding Newborn Baby | image credit: BazziBa - stock.adobe.com
May 9th 2024

SCD Birth Rate Higher in Socially Vulnerable Areas, Study Finds

Kyle Munz
A cross-sectional analysis assessed the birth prevalence of sickle cell disease (SCD) alongside data on social vulnerability to highlight the needs of communities impacted by SCD.
Immune Cells and Antibodies Concept | image credit: vipman4 - stock.adobe.com
May 4th 2024

Immune Irregularities Detected in Patients With PNH

Kyle Munz
A retrospective analysis suggested that immune cells play a role in the progression of paroxysmal nocturnal hemoglobinuria (PNH).
Electronic Health Record Access | image credit: pandpstock001 - stock.adobe.com
April 30th 2024

EHR Alerts Improve Quality of Care in Heparin-Induced Thrombocytopenia, Study Finds

Kyle Munz
A team of researchers developed 3 quality improvement measures embedded in electronic health records (EHRs) to improve care delivery in patients with heparin-induced thrombocytopenia.
Blood Transfusion | image credit: Kiryl Lis - stock.adobe.com
April 30th 2024

Exagamglogene Autotemcel Shows Promise in Treatment of Transfusion-Dependent β-Thalassemia

Kyle Munz
An ongoing, phase 3, open-label study demonstrated the efficacy of exagamglogene autotemcel (exa-cel) infusion to promote transfusion dependence in patients with transfusion-dependent β-thalassemia (TDT).
Weight, scale, cost | Image Credit: © Cagkan - stock.adobe.com
April 18th 2024

Pegcetacoplan for PNH More Cost-Effective Than Anti-C5 Monoclonal Antibodies

Christina Mattina
A cost-utility analysis conducted from the perspective of the Italian health system found that pegcetacoplan was more effective and less costly than 2 complement 5 (C5) inhibitors for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).
Child Receiving Care for COVID-19 Infection | image credit: amedeoemaja - stock.adobe.com
April 15th 2024

Children With Sickle Cell Disease Experience Severe COVID-19 Infection

Connor Iapoce
In a registry-based analysis, more hospitalized children with SCD and COVID-19 had severe infection requiring supplemental oxygen, compared with the general population.
Definition of Opioids | image credit: Feng Yu - stock.adobe.com
April 10th 2024

Study: Children With SCD Not Found to Abuse Opioids Long-Term

Kyle Munz
A retrospective cohort study assessed patterns of opioid use in children with sickle cell disease (SCD) following their initial opioid prescription for pain management, finding that concerning patterns of opioid abuse did not develop.
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