Rare Blood

Latest News
August 28th 2024

Pegcetacoplan May Reduce Fatigue in PNH Accompanied by Anemia

August 18th 2024

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

August 16th 2024

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

August 5th 2024

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

July 31st 2024

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

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)
Video Interviews
View All
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

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.
Sickled Cells in Blood Vessel | image credit: Dr_Microbe - stocke.adobe.com
April 3rd 2024

Processing Speed Decline Over Time Identified in Individuals With Sickle Cell Disease

Kyle Munz
A retrospective study reveals that measures of cognitive function, specifically processing speeds, decline in individuals with sickle cell disease as they grow older.
AstraZeneca Building | image credit: Sundry Photography - stock.adobe.com
April 1st 2024

Factor D Inhibitor Danicopan Approved as Add-On Therapy for PNH

Kyle Munz
The first-in-class oral factor D inhibitor danicopan (Voydeya) has been approved for the management of extravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria (PNH).
CDC Logo on Laptop | image credit: monticellllo - stock.adobe.com
March 21st 2024

Researchers Evaluate Consequences of 2016 CDC Guidance for Patients With SCD

Kyle Munz
A retrospective, qualitative cohort study concludes that patients with sickle cell disease (SCD) may have been negatively impacted by the 2016 CDC guidance.
Formation of a blood clot | image credit: © Tatiana Shepeleva - stock.adobe.com
March 14th 2024

How Patients Navigate Anticoagulation, Bleeding After Venous Thromboembolism

Kyle Munz
A descriptive, qualitative study investigated the experiences and perspectives of patients with venous thromboembolism on shared decision-making and treatment with anticoagulants.
Clinician Holding COVID-19 Vaccine Bottle and Syringe | image credit: Leigh Prather - stock.adobe.com
March 12th 2024

Managing Rare Thrombotic Events Following COVID-19 Vaccination

Kyle Munz
A single-center, retrospective study examined the management of thrombotic events in patients presenting with thrombocytopenia following vaccination against COVID-19.
Red Blood Cells in Blood Vessel | image credit: Artur - stock.adobe.com
March 9th 2024

Immunosuppressive Therapies Shown to Significantly Impact Remission in Acquired Hemophilia A

Kyle Munz
A single-center, retrospective study demonstrated the major success of immunosuppressive therapies in contributing to complete remission in patients with acquired hemophilia A.
Mouth Illustration | image credit: udaix - stock.adobe.com
February 29th 2024

Children With SCD Exhibit Distinctive Palatal Rugae Patterns

Kyle Munz
A cross-sectional study analyzing morphological and dimensional features of palatal rugae patters revealed that distinct anomalies exist in children with sickle cell disease (SCD) compared with children without.
Red Blood Cell Concept | image credit: AI DREAMS - stock.adobe.com
February 22nd 2024

Complement Inhibitors Suggested to Benefit Treatment Outcomes in PNH

Kyle Munz
A meta-analysis on complement inhibitors in the management of paroxysmal nocturnal hemoglobinuria (PNH) reveals an association between these interventions and positive treatment outcomes.
Teddy Bear in Waiting Room, Pediatric Care Concept | Image credit: Studio Romantic - stock.adobe.com
February 16th 2024

Pain Screening Benefits Care for Pediatric Patients With SCD

Kyle Munz
Pain screening in young patients with sickle cell disease (SCD) has demonstrated benefits for the administration of integrative health and medicine modalities for pain management.
Hearing Loss Model | image credit: peterschreiber.media - stock.adobe.com
February 9th 2024

Hearing Impairment Disproportionately Affects Patients With SCD, Further Studies Required

Kyle Munz
A cross-sectional comparative study demonstrated the increased prevalence of hearing impairment in children and adults with sickle cell disease (SCD) or sickle cell traits compared with healthy individuals.
Clinician Preparing COVID-19 Vaccine | image credit: myskin - stock.adobe.com
February 4th 2024

COVID-19 Vaccination Rates Far Lower in Patients With SCD Compared With General Population

Kyle Munz
A state-wide analysis draws attention to the fact individuals with sickle cell disease (SCD) completed a vaccination against COVID-19 at nearly half the rate of individuals without SCD.
Red Blood Cell Model | image credit: Bipul Kumar - stock.adobe.com
February 2nd 2024

Hospitalization, ER, and Medication Discontinuation Rates Still High in PNH

Kyle Munz
A retrospective analysis surveys the treatment landscape and patterns of patients with paroxysmal nocturnal hemoglobinuria (PNH), illuminating high hospitalization rates and emergency department use despite the availability of multiple treatment options.
123456>

2 Commerce Drive
Suite 100
Cranbury, NJ 08512

© 2024 MJH Life Sciences® and AJMC®.
All rights reserved.

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