Bleeding Disorders
News
EMA recommends drug receive orphan designation for PNH
The European Medicines Agency (EMA) has recommended orphan drug designation for the complement C3 inhibitor APL-2 as a treatment for paroxysmal...
Conference Coverage
Watchful waiting a suitable option for pediatric acute ITP
MONTREAL – Treatment of acute idiopathic thrombocytopenic purpura does not appear to increase risk of later chronic ITP, except in children up to...
Conference Coverage
Cord blood/placental cell combo induces rapid immune recovery
MONTREAL – The probability of 1-year survival in children with severe diseases requiring stem cell transplants was 81%.
From the Journals
FDA boxed warning leads to drop off in use of ESAs
The FDA warning language was linked with a sharp decrease in use of erythropoietin-stimulating agents in colorectal, breast, and lung cancer...
From the Journals
Blood donor age, sex do not affect recipient survival
The age and sex of blood donors do not affect the recipient’s survival and do not need to be considered in blood allocation, according to a report...
Commentary
DTC genetic health risk tests: Beware
The Food and Drug Administration recently authorized 23andMe to provide consumers with results of germline DNA sequence variants associated with...
From the Journals
Pegylated interferon alfa-2a induces durable responses in MPNs
Pegylated interferon alfa-2a can induce durable hematologic and molecular responses in patients with advanced essential thrombocythemia and...
From the Journals
New diagnostic tool identifies severe ADAMTS13 deficiency
A new diagnostic tool – the PLASMIC score – predicts severe ADAMTS13 deficiency, effectively distinguishing thrombotic thrombocytopenic purpura...
News
Safety of N9-GP for hemophilia B needs further study, FDA committee agrees
The investigational factor IX product nonacog beta pegol for the treatment of hemophilia B has a possible safety issue that had the Food and Drug...
News
Drug granted fast track designation for PNH
The US Food and Drug Administration (FDA) has granted fast track designation to Coversin™ for the treatment of paroxysmal nocturnal hemoglobinuria...
Conference Coverage
Replacement factors, bypassing agents safely manage fitusiran bleed events
Fitusiran appears to promote hemostasis and reduce the frequency of bleeding in hemophilia.