Rare Blood

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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

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.
Mutated Genes Model | image credit: imaginuma - stock.adobe.com
January 29th 2024

Myeloid Panel Analysis Imperative for Risk Assessment in Patients With CCUS

Kyle Munz
A myeloid panel mutation analysis should be prioritized for patients with clonal cytopenia of undetermined significance (CCUS) as it can better assess patient risks for progression to a myeloid malignancy, a recent study argues.
Red Blood Cells | image credit: Svetlana - stock.adobe.com
January 24th 2024

Emicizumab Demonstrates Efficacy in Treatment of Hemophilia

Kyle Munz
Patients with hemophilia A experienced prognostic and quality of life benefits from emicizumab (Hemlibra; Genentech) prophylaxis treatment.
Omicron Variant Poster | image credit: Nikolay - stock.adobe.com
January 19th 2024

Complement Inhibitors may be Ineffective for Reducing Omicron Infection Severity

Kyle Munz
A single-center study suggests complement inhibitors do not reduce the severity of a COVID-19 Omicron infection in patients with paroxysmal nocturnal hemoglobinuria (PNH).
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.
Top 5 Most-Read Rare Blood Articles of 2023
December 27th 2023

Top 5 Most-Read Rare Blood Articles of 2023

Julia Bonavitacola
The top 5 articles about rare blood featured information on pseudo–thrombotic thrombocytopenic purpura (TTP), immune TTP, and hemophilia.
Brain illustration | Image credit: jolygon - stock.adobe.com
December 21st 2023

White Matter Damage Leads to Depression, Cognitive Impairment in Patients With TTP

Julia Bonavitacola
Patients with immune-mediated thrombotic thrombocytopenic purpura (TTP) were found to have white matter damage in the frontal lobe and cingulate cortex that could affect short-term memory and depressive scores.
Patient in Hospital | Image credit: Gorodenkoff - stock.adobe.com
December 18th 2023

Patients With TTP, COVID-19 Had Higher Mortality, More Complications

Julia Bonavitacola
Patients who had concurrent thrombotic thrombocytopenic purpura (TTP) and COVID-19 had elevated risk of mortality and other complications.
Asian doctor checking on African baby boy in mother's arms | Image credit: Dollydoll - stock.adobe.com
December 13th 2023

VOC Frequency Negatively Associated With HRQOL in Children, Caregivers Living With SCD

Julia Bonavitacola
Children with sickle cell disease and their caregivers experience an increased burden on health-related quality of life (HRQoL) due to the frequency of vaso-occlusive crises (VOCs).
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