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

November 7th 2024

The future of hemophilia care lies not only in technological advancements but also in the pursuit of health equity, ensuring that all patients have access to effective, affordable treatments.

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

November 5th 2024

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High Health Care Burden of Transfusion-Dependent β-Thalassemia

October 30th 2024

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New AI Tool Identifies Undiagnosed PNH in Health Records

October 30th 2024

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HSSP Pharmacists Demonstrate Substantial Cost Avoidance in Specialty Medication Use

October 28th 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

image of newspapers and coffee on a table with text "What We're Reading: Your top daily health care news from AJMC®"
January 17th 2023

What We’re Reading: Sickle Cell Cure Complexity; Free Care for Veterans in Suicidal Crisis; Pfizer to Offer Low-Cost Drugs to Underserved Countries

AJMC Staff
The reality of a sickle cell disease cure brings fear, optimism, and questions to patients’ lives; veterans who are in suicidal crisis can receive free emergency care at any Department of Veterans Affairs or private facility; Pfizer to increase access to progressive treatments through sale of drugs at non-profit prices to poor countries.
Top 5 Most-Read Rare Blood Content of 2022
December 22nd 2022

Top 5 Most-Read Rare Blood Content of 2022

Pearl Steinzor
The most-read rare blood articles included studies on cold agglutinin disease , hemophilia B gene therapy, autoimmune hemolytic anemia, and hereditary thrombotic thrombocytopenia purpura.
Image of blood in a test tube
December 22nd 2022

Conquering SCD by Confronting Treatment Barriers, Investigating PROs

Maggie L. Shaw
A pair of abstracts presented during this year’s 64th American Society of Hematology Annual Meeting and Exposition explored barriers to treatment for sickle cell disease (SCD).
Image of blood flowing
December 20th 2022

Analysis of Beti-Cel for Severe β-Thalassemia Shows Extensive, Long-term Patient Improvement

Maggie L. Shaw
A pair of abstracts presented at this year’s 64th American Society of Hematology Annual Meeting and Exposition bear out the significant transfusion-free rate at 3 years following beti-cell administration and marked improvements in patient-reported outcomes, including the ability to work and be physically active.
Blood
December 9th 2022

Phase 3 CADENZA Trial Validates QOL Improvements Reported by Patients With CAD Receiving Sutimlimab

Pearl Steinzor
Mental and physical health aspects of quality of life (QOL) improved after sutimlimab treatment in patients with cold agglutin disease (CAD) during a phase 3 trial.
Image of a doctor and patient talking
November 18th 2022

Some Patients With CAD Experience Sustained Remission After Sutimlimab Discontinuation

Rose McNulty
A research letter published in Blood Advances details the first clinical evidence of sustained hematologic remission for cold agglutinin disease (CAD) after sutimlimab treatment discontinuation.
Child in exam room
November 14th 2022

Study Highlights Value of PROs in Children With Sickle Cell Disease During Acute Pain Episodes

Rose McNulty
Sufficiently powered studies are necessary to investigate associations between clinical measures and patient-reported outcomes (PROs) in children with sickle cell disease experiencing acute pain episodes, according to a recent study.
Teddy Bear
November 12th 2022

Report Suggests Best Practices for Care of Pediatric Sickle Cell Disease

Rose McNulty
A panel of specialists proposed 26 essential elements for comprehensive pediatric sickle cell disease care, laying groundwork for standardized guidelines and the establishment of accredited care centers in the future.
Image of sickled cells
November 11th 2022

Real-World Study of Voxelotor for Sickle Cell Disease Shows Safety, Efficacy Similar to Clinical Trials

Rose McNulty
Over the course of voxelotor treatment, hemoglobin levels and hemolysis markers improved, with results sustained over the treatment period.
image of high health care costs
November 7th 2022

Sickle Cell Disease With Recurrent Vaso-Occlusive Crises Carries a Substantial Economic Burden, Study Finds

Rose McNulty
The analysis emphasizes the association between vaso-occlusive crisis frequency and health care costs, which are inflated largely due to inpatient visits.
Picture of red cells
October 27th 2022

Case Report Describes Thrombocytopenia Secondary to Iron-Deficiency Anemia

Rose McNulty
A recently published report describes the diagnosis and treatment of a case of iron-deficiency anemia–associated thrombocytopenia secondary to recurrent nosebleeds.
graphic depicting patient data
October 21st 2022

Improved Surveillance, Registry Data Could Improve Outcomes in Sickle Cell Disease

Rose McNulty
Population-based data is a crucial aspect to evaluating current care quality and improving outcomes for patients with sickle cell disease.
Blood cells
October 15th 2022

Review Addresses Proper Applications of Sutimlimab in Cold Agglutinin Disease

Rose McNulty
A review published in the journal Immunotherapy summarizes the applications of sutimlimab and emphasizes the importance of individualizing treatment for patients with cold agglutinin disease.
Sickle cells
October 12th 2022

Sickle Cell Disease Management May Overlook Impactful Complications, Study Finds

Rose McNulty
Findings of a recent study suggest that complications of sickle cell disease (SCD) significantly contribute to morbidity and are more prevalent among older patients.
Image of a happy life
October 3rd 2022

Sutimlimab Improves Patient-Reported QOL in Cold Agglutinin Disease

Rose McNulty
An analysis of patient-reported outcomes from the pivotal CARDINAL trial of sutimlimab in cold agglutinin disease found that quality of life (QOL) improved and persisted throughout treatment.
Blood cells
October 1st 2022

Review: More Research on Treatment for Extramedullary Hematopoiesis in Transfusion-Dependent Beta Thalassemia Is Needed

Rose McNulty
Currently, there are no clear guidelines for managing extramedullary hematopoiesis in transfusion-dependent beta thalassemia.
Image of a sick child
September 26th 2022

Guidance for Evaluating for Bleeding Disorders in Suspected Child Abuse

Rose McNulty
The recommendations aim to make this type of evaluation more consistent in cases when children have suspicious bruising or bleeding patterns.
picture of teddy bear
September 20th 2022

Children With Sickle Cell Anemia Using Medicaid Less Likely to Get Stroke Screening, Pain Treatment

Allison Inserro
In 2019, less than half of children aged 2 to 16 years with sickle cell anemia received the recommended screening for stroke, a common complication of the disease.
sickle cell disease
September 17th 2022

Review Highlights Advances in SCD Treatment, Potential Targets for Novel Agents

Rose McNulty
The review discusses hydroxyurea and 3 additional drugs approved by the FDA—L-glutamine, crizanlizumab, and voxelotor—as well as agents currently being investigated to treat sickle cell disease (SCD).
Image of blood flow
September 17th 2022

Sutimlimab Is Effective, Well Tolerated in Refractory Chronic Immune Thrombocytopenia

Rose McNulty
Sutimlimab demonstrated rapid efficacy in the study cohort, and platelet level improvements were sustained for the duration of treatment in nearly half of the participants.
Image of red blood cells
September 13th 2022

Bendamustine/Rituximab vs Sutimlimab Monotherapy for CAD Explored in New Review

Rose McNulty
A review published in Transfusion Medicine Reviews discussed the 2 therapeutic approaches and their pros and cons for treating cold agglutinin disease (CAD).

Test tubes in a lab
September 9th 2022

The Impact of Orphan Drug Legislation on the Beta Thalassemia Treatment Landscape

Rose McNulty
Orphan drug designation incentives have helped boost enthusiasm for researching and developing drugs for beta thalassemia, a new review concludes.
Image of blood in test tubes
September 1st 2022

Hemoglobin Improvement Correlates With Better HRQOL in Paroxysmal Nocturnal Hemoglobinuria

Rose McNulty
Post hoc analyses of the phase 3 PEGASUS trial found that clinical and hematological improvements were associated with better patient-reported fatigue and physical function outcomes in paroxysmal nocturnal hemoglobinuria, for an overall bettering of health-related quality of life (HRQOL).
Specialist recommendations
August 21st 2022

Patients With Sickle Cell Disease Less Likely to Receive Specialized Care

Rose McNulty
A recent analysis of Medicaid claims data found that individuals with sickle cell disease are seeing hematologists at a lower rate than patients with other chronic genetic diseases.
FDA approval text
August 17th 2022

FDA Approves First Gene Therapy for Beta-thalassemia

Allison Inserro
The FDA Wednesday approved betibeglogene autotemcel (Zynteglo), the first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.
Image of a sick older man
August 15th 2022

Impact of Cold Agglutinin Disease on Daily Life Seen in New Survey

Rose McNulty
Symptoms associated with cold agglutinin disease fluctuated throughout the course of the disease, and many patients were not satisfied with management measures.
rare disease
August 11th 2022

Report Details Rare Case of Warm AIHA Following Epstein-Barr Virus Infection

Rose McNulty
The case of warm autoimmune hemolytic anemia (AIHA) highlights the potential for life-threatening acute hemolysis if patients do not receive timely treatment.
image of sickled cells
August 6th 2022

Young Adults With SCD Show Better Hydroxyurea Adherence Than Children, Adolescents

Rose McNulty
With adherence estimated at less than 50% in children, adolescents, and young adults, a recent study stresses the importance of treatment adherence for patients with sickle cell disease receiving hydroxyurea and the benefit for those who do.
Image of genetics
August 1st 2022

Heavy/Light Chain Assay Useful Marker in Cold Agglutinin Disease, Study Suggests

Rose McNulty
Current standard modalities for detecting and quantifying monoclonal immunoglobin in patients with cold agglutinin disease lack adequate sensitivity. Heavy chain/light chain assay may be more effective.
Image of a thrombosis
July 29th 2022

Study Identifies Risk Factors for Thrombosis in Patients With AIHA

Rose McNulty
This retrospective study and prospective follow-up provide insight into predictors of thrombotic episodes in patients with autoimmune hemolytic anemia (AIHA) and indicators that anticoagulant prophylaxis may be beneficial to certain patients.
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