A collection of features and news articles published in ASH Clinical News related to bleeding disorders.
New HOPE for Hemophilia B: One-Time Gene Therapy Abolishes Bleeding in Most Patients
A single dose of the gene therapy drug etranacogene dezaparvovec increased factor IX (FIX) activity levels, and eliminated the need for prophylactic FIX, in...
Adding Mycophenolate to Corticosteroids Improves Response as Frontline Option in Immune Thrombocytopenia
In results from the randomized, controlled FLIGHT trial, patients with immune thrombocytopenia (ITP) who received mycophenolate mofetil (MMF) plus corticosteroids as first-line therapy were...
Does Blood Group Affect Risk of Bleeding Events or Bleeding Severity?
According to research published in Blood Advances, people who experience bleeding of unknown causes are 48% more likely to have blood group O, compared...
Steven Pipe: Etranacogene Dezaparvovec Gene Therapy for Hemophilia B
Steven Pipe, MD, shares safety and efficacy data from a phase III study of etranacogene dezaparvovec gene therapy in patients with hemophilia B.
Tippi MacKenzie: In Utero Hematopoietic Cell Transplantation for Hematologic Conditions
In utero hematopoietic cell transplantation showed promise in a phase 1 clinical trial involving fetuses with alpha thalassemia major. Here, Tippi Mackenzie, MD, discusses...
What Is the Optimal Treatment of ITP?
In recent years, the number of treatment options available for patients with immune thrombocytopenia (ITP) has expanded. In addition to the classical trio of...
Treatment With Caplacizumab Allows Patients to Delay or Omit Plasma Exchanges
Treatment of acquired thrombotic thrombocytopenic purpura (aTTP) with caplacizumab, but without plasma exchange, increased platelet counts and improved surrogate parameters of organ damage in...
Targeting Higher Factor VIII Troughs Reduced Bleed Rates in Patients With Severe Hemophilia A
Targeting factor VIII (FVIII) trough levels of 1 to 3% and 8 to 12% with rurioctocog alfa pegol effectively reduced bleeding rates in patients...
Fresh Frozen Plasma May Help Maintain Successful Pregnancy in Women With Congenital TTP
Prophylactic fresh frozen plasma (FFP) infusions may be necessary to replenish ADAMTS13 activity and maintain a successful pregnancy in expectant mothers with congenital thrombotic...
PF4-Dependent P-Selectin Assay Highly Accurate in Diagnosing Heparin-Induced Thrombocytopenia
A study published in Blood established the diagnostic accuracy of a PF4-dependent P-selectin expression assay (PEA) for heparin-induced thrombocytopenia (HIT), with a sensitivity similar...
