Bleeding Disorders

Bleeding Disorders

A collection of features and news articles published in ASH Clinical News related to bleeding disorders.

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Hemophilia B: The Next Target for Gene Therapy?

Treatment with a single dose of AMT-061, an investigational gene therapy, led to increased factor IX (FIX) activity and reduced spontaneous bleeding episodes for...

Testing the Testing Tool: ISTH Bleeding Assessment Accurately Predicts Patients With Platelet Function Disorders

In patients with suspected platelet function disorders, the International Society on Thrombosis and Hemostasis bleeding assessment tool (ISTH-BAT) proved to be a useful instrument...

IV Iron Superior to Oral Iron for Managing Postpartum Anemia

For women with postpartum anemia, treatment with intravenous (IV) iron was associated with higher hemoglobin concentrations at six weeks than oral iron, according to...
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Valoctocogene Roxaparvovec Continues to Improve Bleeding Outcomes in Patients with Hemophilia A

In three-year follow-up results from an ongoing phase I/II study of patients with hemophilia A, valoctocogene roxaparvovec gene therapy significantly reduced annual bleeding rates...

U.K. Denying Benefit to Patients With Sickle Cell Disease

The U.K. government has been withholding personal independence payments (PIPs) from patients with sickle cell disease because the condition does not fit government criteria...
Sickle cell anemia

Voxelotor Increases Hemoglobin, Decreases Hemolysis in Patients With Sickle Cell Disease

A report from the phase III HOPE trial found that once-daily voxelotor led to greater increases in hemoglobin (Hb) levels and larger reductions in...

Bluebird Bio Wins European Approval for Beta Thalassemia Gene Therapy

Bluebird Bio’s one-time gene therapy, Zynteglo, has been cleared by E.U. regulators for the treatment of beta thalassemia in patients aged 12 and older....
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Erythropoietin Increases Hemoglobin and Reticulocyte Counts in Patients with Treatment-Refractory AIHA

In patients with autoimmune hemolytic anemia (AIHA) unresponsive to ongoing and/or prior therapies, treatment with erythropoietin (EPO) effectively increased hemoglobin (Hb) and reticulocyte counts,...
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Evaluating Rozanolixizumab in Primary Immune Thrombocytopenia

Rozanolixizumab, at doses ranging from 4 to 15 mg/kg, induced platelet responses in patients with primary immune thrombocytopenia (ITP), according to an interim analysis...

FDA Approves First Anticoagulant for Pediatric Patients

The FDA approved dalteparin sodium, administered as a subcutaneous injection, to reduce the recurrence of symptomatic venous thromboembolism (VTE) in pediatric patients (≥1 month). Dalteparin...
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Current Issue

December 2019, Volume 5, Issue 15

This issue examines the hidden costs of participating in clinical trials, explores the pediatric hematology/oncology workforce, and more.