Thalassemias

Addressing Gaps in Care for Patients With Thalassemia

Researchers sought to identify and address gaps in care for patients with thalassemia.

Beti-Cel, a Gene Therapy for Beta-Thalassemia, Gets FDA Panel Support

By

Brian Park, PharmD

Beti-cel is an investigational one-time gene therapy that works by adding functional copies of a modified form of the beta-globin gene into a patient’s own hematopoietic stem cells.

Bristol Myers Squibb Withdraws Luspatercept sBLA for NTD Beta Thalassemia

By

Brian Park, PharmD

The application included data from the phase 2 BEYOND study.

Lipid Profiles in Beta-Thalassemia With IVS Mutations and Hydroxyurea Therapy

By

Vicki Moore, PhD

Researchers sought to identify lipid profiles of red blood cell membranes in patients with beta-thalassemia, in the context of IVS mutations and hydroxyurea treatment.

Mortality With Pulmonary Arterial Hypertension and Beta-Thalassemia

By

Vicki Moore, PhD

Researchers sought to determine mortality rates among patients with beta-thalassemia and pulmonary arterial hypertension who had right heart catheterization.

Natural Iron-Chelating Agent May Be Effective for Serum Ferritin Reduction in Beta-Thalassemia

By

Lauren Dembeck, PhD

Researchers sought to determine whether silymarin would be safe and effective in decreasing serum ferritin levels in patients with beta-thalassemia intermedia.

Thalassemia May Not Increase Risk for Poor Outcomes From COVID-19

By

Andrea S. Blevins Primeau, PhD, MBA

Researchers sought to determine whether patients with thalassemia would be at an increased risk for excess morbidity or mortality from COVID-19.

Brand of Deferasirox Does Not Appear to Affect Response in Beta Thalassemia

By

Jonathan Goodman, MPhil

Researchers sought to determine whether the brands of deferasirox would have different outcomes in patients with major beta thalassemia.

Gene Therapy Potentially Curative for β-Thalassemia

Transfusion independence, near-normal Hb levels seen for most children and adults receiving beti-cel for non-β⁰/β⁰ genotype β-thalassemia

Adherence to Iron Chelation Therapy in Young Patients With Thalassemia

By

Vicki Moore, PhD

Researchers sought to determine the factors linked to adherence to iron chelation therapy in young patients with thalassemia.

Stored Vs Fresh Blood for Patients With Transfusion-Dependent Thalassemia

By

Vicki Moore, PhD

Researchers sought to assess the outcomes for patients with transfusion-dependent thalassemia based on the use of a fresh blood product vs a stored blood product.

Calcium Channel Blocker Plus Iron Chelation Therapy Reduces Cardiac Iron Overload in Children With Transfusion-Dependent Thalassemia

By

Lauren Dembeck, PhD

Researchers sought to determine whether amlodipine with chelation therapy would be more effective in reducing cardiac iron overload in transfusion-dependent thalassemia.

Bleeding and Thrombotic Risks in Young Patients With Beta-Thalassemia Major

By

Vicki Moore, PhD

Researchers sought to determine whether pediatric patients with beta-thalassemia major are at increased risk of both bleeding and thrombosis.

Thalidomide Appears Safe, Effective in Transfusion-Dependent Beta-Thalassemia

By

Jonathan Goodman, MPhil

Researchers sought to determine whether thalidomide would be an effective therapy for patients with transfusion-dependent beta-thalassemia.

One-Time Gene Therapy Gets Priority Review for Beta-Thalassemia

By

Brian Park, PharmD

Betibeglogene autotemcel (beti-cel) is a one-time gene therapy that works by adding functional copies of a modified form of the β-globin gene into a patient’s own hematopoietic stem cells.

Transfusion Duration Predicts Risk of Abnormal Kidney Function in Patients With Thalassemia

By

Jonathan Goodman, MPhil

Researchers sought to determine whether there is a need for regular kidney surveillance to detect dysfunction among pediatric patients with transfusion-dependent thalassemia.

COVID-19 Pandemic Exacerbated Existing Problems With Thalassemia Care

By

Dr Androulla Eleftheriou

Τhe COVID-19 pandemic has exacerbated challenges in thalassemia care worldwide.

Luspatercept Under Review for Nontransfusion Dependent Beta Thalassemia

By

Brian Park, PharmD

The application is supported by data from the phase 2 BEYOND study.

Droplet-Based Digital PCR May Be Effective for Prenatal Alpha-Thalassemia Detection

By

Patricia Chaney

Researchers sought to determine whether a droplet digital PCR would be effective for noninvasive prenatal diagnosis of thalassemia.

Prenatal Diagnosis Results in Early Diagnosis of Transfusion-Dependent Thalassemia and Better Growth Outcomes

By

Lauren Dembeck, PhD

Researchers sought to determine the clinical outcomes of children with thalassemia whose parents did or did not undergo prenatal diagnosis testing.

Beta Thalassemia Carriers May Be More Susceptible to Severe COVID-19

By

Patricia Chaney

Researchers sought to determine whether patients with beta thalassemia heterozygosity are at higher risk of morbidity and mortality from COVID-19.

Correlation Seen Between Thyroid Function, Iron Overload in Patients With Beta Thalassemia

By

Patricia Chaney

Researchers sought to determine a correlation between endocrine levels and iron overload in patients with transfusion-dependent beta thalassemia and growth retardation.

Patients With Beta-Thalassemia Receiving Therapy May Suffer From Growth Issues

By

Jonathan Goodman, MPhil

Researchers sought to determine whether patients with thalassemia may have growth impairments from their disease.

Economics of Betibeglogene Autotemcel for Patients With Transfusion-Dependent Beta-Thalassemia

By

Vicki Moore, PhD

Researchers sought to conduct an economic evaluation of the use of beti-cel gene therapy in the treatment of transfusion-dependent -thalassemia.

MRI T2 Effective at Detecting Iron Overload in Patients With Thalassemia

By

Patricia Chaney

Researchers sought to determine whether MRI T2 is an effective way to detect iron concentrations in the heart, liver, and pancreas during chelation therapy for patients with thalassemia major.

Carvedilol Improves Left Ventricular Diastolic Dysfunction in Patients With Transfusion‑Dependent Thalassemia

By

Lauren Dembeck, PhD

Researchers sought to determine whether carvedilol would improve left ventricular diastolic function in patients with transfusion‑dependent thalassemia who had iron overload cardiomyopathy.

Mitapivat Induces Responses in Patients With Thalassemia

By

Jonathan Goodman, MPhil

Researchers sought to determine whether mitapivat would improve clinical outcomes in patients with thalassemias who are not transfusion-dependent.

Cardiac T2* Magnetic Resonance Imaging of Siderosis Validated in Patients With Thalassemia Major

By

Vicki Moore, PhD

Researchers sought to validate the usefulness of cardiac T2* magnetic resonance imaging to evaluate cardiac siderosis in patients with thalassemia major.

Abatacept May Be Additional Prophylaxis for Graft-vs-Host Disease in Patients With Beta-Thalassemia

By

Patricia Chaney

Researchers sought to determine whether adding abatacept to standard prophylaxis may reduce rates of GVHD in patients with beta-thalassemia.

Dose of Total Body Irradiation With Transplantation for Hemoglobinopathies

By

Vicki Moore, PhD

Researchers sought to determine whether TBI could help improve treatment with allogeneic blood or marrow transplantation in patients with severe hemoglobinopathies.

Deferiprone Non-Inferior to Deferasirox for Patients With Pediatric Hemoglobinopathy

By

Jonathan Goodman, MPhil

Researchers sought to determine whether deferiprone could be a good option to treat pediatric patients with transfusion-dependent hemoglobinopathy.

Deferasirox FCT Good Option for Management of Iron Overload in Iron Chelation Therapy

By

Patricia Chaney

Researchers sought to determine whether deferasirox would be a good option for long-term management of iron overload in patients who receive iron chelation therapy.

An Overview of the Use of Luspatercept for Thalassemia

By

Patricia Chaney

Luspatercept, an activin receptor ligand trap, has shown efficacy and safety in patients with 𝛽-thalassemia.

Promising Follow-Up of Trials Using CRISPR-Cas9 for Inherited Hemoglobinopathies

By

Vicki Moore, PhD

Researchers sought to determine whether CRISPR-Cas9 gene therapy would be an effective treatment for patients with sickle cell disease or -thalassemia.

Ethnographic Factors May Negate Need for Matched Sibling Donors With Beta-Thalassemia

By

Jonathan Goodman, MPhil

In communities where consanguineous marriages are commonplace, nonsibling matched donor transplants may represent a viable option in patients with beta-thalassemia.

CRISPR-Cas9 Gene Editing May Be Effective in β-Thalassemia, Sickle Cell Disease

By

Jonathan Goodman, MPhil

Preliminary results suggest that CRISPR-Cas9 editing of BCL11A may benefit patients with sickle cell disease and β-thalassemia by potentially eliminating vaso-occlusive episodes or the need for transfusion.

Comorbidity Rate Remains High in Transfusion-Dependent β-Thalassemia Compared With General Population

By

Jonathan Goodman, MPhil

Among patients with transfusion-dependent β-thalassemia, comorbidity and mortality rates remain high, suggesting an unmet need among patients.

Treatment Considerations for Thalassemia in the Emergency Department

By

Caleb Rans, PharmD

Antoine N. Saliba, MD, and colleagues summarized current literature surrounding the frequent complications that bring patients with thalassemia into the ED.

FDA Grants Orphan Drug Status to Mitapivat for Thalassemia

By

Brian Park, PharmD

The FDA has granted Orphan Drug designation to mitapivat (AG-348; Agios Pharmaceuticals) for the treatment of thalassemia.

Promising New Noninvasive Test Developed for β-Thalassemia in At-Risk Pregnancies

By

Lauren Dembeck, PhD

Compared to invasive prenatal diagnosis, a noninvansive assay may be a viable alternative for genotyping a fetus in pregnancy at high-risk for β-thalassemia.

Ferriprox Twice-a-Day Tablet Dosing Approved for Transfusional Iron Overload Due to Thalassemia Syndromes

By

Brian Park, PharmD

The FDA has approved a twice-a-day formulation of Ferriprox® (deferiprone; Chiesi) for the treatment of transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate.

Altered Bone Marrow Microenvironment Induces Defect of Hematopoietic Stem Cells in β-Thalassemia

By

Dibash Kumar Das, PhD

For patients with β-thalassemia who undergo HSCT, exposure to a normal microenvironment and the impairment of HSCs may be reversible in the presence of a normal microenvironment and in vivo protein restoration.

Luspatercept Improves Transfusion Burden in Patients with Transfusion-Dependent Beta-Thalassemia

By

Lauren Dembeck, PhD

When compared with placebo, luspatercept increased the percentage of patients with transfusion-dependent beta-thalassemia who had a reduction in transfusion burden.

Managing Complications Associated With Increased Longevity in Beta Thalassemia

By

Roxanne Nelson, RN, BSN

As the life expectancy for patients with beta thalassemia continues to increase, more and more patients are experiencing age-related complications.

Novel Conditioning Regimen for Haploidentical HSCT in Severe Thalassemia

By

Vicki Moore, PhD

Both event-free and overall survival at 3 years were 96% for patients with transfusion-dependent thalassemia who underwent transplant.

Mineral and Hormonal Dysfunction Associated With Lower BMD in Thalassemia Major

By

Morgan E. Meissner

Hypothyroidism, hyperglycemia, and low adrenocorticotrophic hormone levels are associated with lower bone mass in patients with thalassemia major.

Improving Bone Mass With Iron Chelation Therapy in Transfusion-Dependent Beta Thalassemia

By

Vicki Moore, PhD

Iron chelation therapy with deferasirox and deferoxamine led to significant improvements in bone mineral density and serum ferritin levels.

As Survival Increases in Beta Thalassemia, Renal Complications Emerge

By

Roxanne Nelson, RN, BSN

Renal complications occur in more than half of patients with beta thalassemia, but research on their epidemiology and treatment is limited.

Utility of Flow Cytometry to Detect Red Blood Cell Alloimmunization in Thalassemia

By

Sharon Ben Barouch, MD

Red blood cell alloantibodies were detected more than 4 times as frequently with flow cytometry compared with direct Coombs test.

Reducing Transfusion Burden and Addressing Unmet Needs in Beta Thalassemia

By

Caleb Rans, PharmD

Lack of resources and poor awareness of thalassemia can make it difficult for patients with beta thalassemia to receive necessary blood transfusions.

White Matter Changes and Headache in Thalassemia

By

Sonali Gattani

Recurrent headache was reported by 61% of patients with thalassemia compared with 22.5% of healthy individuals in a control arm.