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

December 24th 2024

The high health care burden faced by individuals with hemophilia underscores the urgent need for innovative treatments and improved diversity in clinical trials.

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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

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Rare Blood Disease Innovations Address Unmet Needs, According to ASH Data

December 11th 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

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.
Image of genetic code
July 27th 2022

ICER Releases Final Evidence Report on Beti-Cel to Treat Beta Thalassemia

Rose McNulty
The Institute for Clinical and Economic Review (ICER) released the report, which supports the long-term value of betibeglogene autotemcel (beti-cel) for the treatment of beta thalassemia.
Image of blood in a test tube
July 25th 2022

Sutimlimab Has Potential to Treat Warm Autoimmune Hemolytic Anemia

Jared Kaltwasser
A patient who failed to respond to rituximab and steroids later saw dramatic improvement with sutimlimab
graphic to depict genetics
July 20th 2022

Hemophilia B Gene Therapy Produces Durable Response for 90% of Participants

Allison Inserro
Nine out of 10 patients with hemophilia B dosed with FLT180a were able to stop their weekly infusions of replacement factor IX.
image of blood droplet
July 18th 2022

Interim Results From CARDINAL Trial Show Sutimlimab Effective in CAD

Jared Kaltwasser
Patients with cold agglutinin disease (CAD) had durable responses at week 53, and no new safety signals were reported.
Close up of a microscope
July 14th 2022

Management of Autoimmune Hemolytic Anemia Needs Improvement, Investigators Say

Rose McNulty
Additional population-based studies of autoimmune hemolytic anemia and its management are needed to reduce mortality in this patient population.
Image of blood-filled test tubes in a centrifuge
July 4th 2022

Which Malignancies Are Most Linked With Langerhans Cell Histiocytosis?

Jared Kaltwasser
New data show the types of malignancies, such as leukemias and myeloproliferative disorders, that are most common in children with Langerhans cell histiocytosis (LCH).
Image of liver surgery
July 1st 2022

Case Report Shows Successful Liver Transplant Following HSCT in Patient With SCD

Jared Kaltwasser
The patient was 52 years old and underwent a liver transplant 13 years after his hematopoietic stem cell transplant (HSCT) to treat his sickle cell disease (SCD).
Image of bleeding
June 28th 2022

Risk Factors for Hemophilia-Related Bleeding From Prophylaxis Explored in New Study

Rose McNulty
Creating and validating effective risk assessment models could help facilitate appropriate prophylaxis for hemophilia, but more research is needed to confirm the prognostic value of various clinical risk factors and biomarkers.
Image of a genetic blood test
June 20th 2022

In Hereditary TTP, Neuropsychiatric Symptoms, Stroke Are Common

Jared Kaltwasser
A new report offers novel insights into a very small patient population.
doctor holding patient hand
June 17th 2022

Survey Results Highlight Unmet Needs and Burdens Facing Patients With SCD

Laura Joszt, MA
Surveys of health care professionals, patients, and caregivers explored unmet needs in sickle cell disease (SCD), as well as the significant impact the disease has on everyday life.
Image of red blood cells
June 15th 2022

Sutimlimab Shows Efficacy in Patients With CAD Without Recent Transfusion

Jared Kaltwasser
Investigators said the benefits appeared relatively early in treatment for many patients and persisted throughout the study period.
Image of sickled cells
June 10th 2022

Evidence Unclear About Superiority of Exchange Transfusion Types in Sickle Cell Disease

Jared Kaltwasser
A systematic review and meta-analysis found no clear advantage for automated red cell exchange.
blood cells
June 3rd 2022

Routine Mental Health Screening Critical for Patients With Sickle Cell Disease, Caregivers Amid Pandemic

Jared Kaltwasser
Prior to the pandemic, youth with sickle cell disease were more likely to report depressive symptoms, but the opposite was true during the pandemic.
coins
June 2nd 2022

Sickle Cell Cure Would Have Wide-Ranging Economic Impacts, Study Finds

Jared Kaltwasser
A cure for sickle cell disease could more than double the lifetime incomes of patients, the study found.
droplet of blood
May 28th 2022

Unmet Need Remains in iTTP Treatment, but Caplacizumab Could Be Major Step Forward

Jared Kaltwasser
A panel of experts suggests adding caplacizumab to the historic treatment of plasma exchange and immunosuppression could lead to significant improvement in outcomes for patients with immune-mediated thrombotic thrombocytopenic purpura (iTTP).
sickle cell
May 25th 2022

Patients With SCD Face OOP Costs 4 Times Higher Than Those Without the Disorder

Allison Inserro
The findings highlight the significant financial burden of sickle cell disease.
blood cells
May 20th 2022

Case Study: Caplacizumab May Salvage Refractory Immune TTP Following COVID-19 Vaccine

Jaime Rosenberg
While suggesting that the disorder be included in the differential diagnosis of thrombocytopenia following vaccination, the group emphasized that the benefits of global vaccination against the virus outweigh the risk of this rare complication.
Image of red blood cells
May 18th 2022

Mortality Risk Appears to Spike in Winter Among Patients With Cold Agglutinin Disease

Jared Kaltwasser
Patients with cold agglutinin disease faced a 4.5-times greater risk of death during winter months, new data show.
graphic of brain
May 14th 2022

Montreal Cognitive Assessment May Hold Promise for Assessing Cognitive Impairment in SCD

Jaime Rosenberg
Among their sample cohort of 49 patients, the researchers found that scores on the Montreal Cognitive Assessment correlated with measures of both literacy and health literacy.
sickle cell graphic
May 12th 2022

Adolescent, Young Adult Males With Sickle Cell Disease Lack Knowledge of Fertility Implications, Study Shows

Rose McNulty
Improved management of sickle cell disease has vastly improved life expectancy and reduced morbidity for affected patients, but the disease itself and its treatments carry fertility risks that many young patients and their caregivers are unaware of.
doctor with tablet as various symbols related to healthcare pass by
October 20th 2021

Cancer Treatments, Orphan Drugs, Gene Therapies Represent Big Areas to Watch in Specialty Drug Pipeline

Skylar Jeremias
During her presentation at the Academy of Managed Care Pharmacy Nexus 2021 meeting, Aimee Tharaldson, PhD, highlighted the most anticipated specialty pharmacy drugs awaiting FDA approval, including several treatments for cancer and rare diseases.
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