Thalassemias

COVID-19 Pandemic Exacerbated Existing Problems With Thalassemia Care

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

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.

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.

The Efficacy of Intrabone Gene Therapy for Transfusion-Dependent Beta Thalassemia

By

John Schieszer, MA

Allogeneic HSC gene therapy may be an alternative to transplantation for patients with transfusion-dependent thalassemia.

Luspatercept May Improve Hemoglobin Levels in Patients With Beta Thalassemia

By

John Schieszer, MA

A high percentage of patients with beta thalassemia demonstrated an increase in hemoglobin levels after receiving luspatercept.

New Iron Chelator May Improve Cardiac Function in Patients With Beta Thalassemia

By

Lauren Dembeck, PhD

A new oral iron chelator, deferasirox, may confer long-term benefits to patients with beta thalassemia major.

A Novel Health-Related Quality of Life Assessment Tool for Nontransfusion-Dependent Beta Thalassemia

By

Megan Garlapow, PhD

Modern tools for measuring health-related quality of life may not capture symptoms unique to patients with nontransfusion-dependent thalassemia.

Managing Atrial Fibrillation in Patients With Beta Thalassemia Major

By

Lauren Dembeck, PhD

Patients with beta thalassemia major may be at higher risk for atrial fibrillation, creating a need for attentive clinical management.

Peripheral Blood: An Alternative Source of Stem Cells in Thalassemia

By

Megan Garlapow, PhD

Accessing peripheral blood as a graft source is easier and less invasive than accessing bone marrow.

Serum Ferritin May Unreliably Predict Iron Overload in Patients with Thalassemia Major

By

Sonali Gattani

Iron overload that persists after HSCT can cause long-term organ damage.

Managing Medical Complications of Beta-Thalassemia in Adults: An Experience-Based Perspective

By

Lauren Dembeck, PhD

Mortality in beta-thalassemia has decreased due to medical advances, allowing physicians to observe and manage disease-related complications in adult patients.