The Phase 2 TITAN study (n=75) evaluated the safety and efficacy of caplacizumab with standard of care of plasma exchange and immunosuppression.
The primary analysis of the study arm showed a statistically and clinically significant reduction in bleeding by 87% (risk rate [RR] 0.13; P<0.0001] with emicizumab prophylaxis.
Tavalisse is an oral treatment designed to inhibit SYK kinase, a key signaling member in the immune process that leads to platelet destruction in ITP.
Rebinyn, a recombinant DNA-derived coagulation Factor IX concentrate, temporarily replaces the missing coagulation Factor IX in hemophilia B patients.
The Food and Drug Administration (FDA) has granted Fast Track designation to SB-525 (Sangamo Therapeutics) for the treatment of hemophilia A.
Rigel announced its submission of a New Drug Application (NDA) to the Food and Drug Administration (FDA) for fostamatinib for the treatment of patients with chronic and persistent immune thrombocytopenic purpura (ITP).
The interim results from HAVEN 2, after a median of 12 weeks of treatment, showed that prophylaxis with emicizumab is associated with clinically meaningful reduction in the number of bleeds over time.
Commenting on the study's results, lead author Robert J. Adams, MD, said, "Having sickle cell disease did not adversely affect any of the indicators we measured."
While the GHR tests may provide genetic risk information, they cannot determine a person's overall risk for developing a disease, as other factors beyond genetics (ie, environment, lifestyle) may also influence risk.
Patients taking apixaban showed significantly less intracranial hemorrhage (0.33% per year) vs. patients taking warfarin (0.80% per year) no matter the type and location.
Fifty-two (10.9%) of the patients who experienced an ischemic event, and 41 (17.7%) who suffered experieced a bleeding event, died.
Nipride RTU is approved for immediate blood pressure reduction and also carries a Boxed Warning describing the possibility of excessive hypotension and cyanide toxicity.
The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to the investigational gene therapy, AMT-060 (uniQure), for the treatment of severe hemophilia B.
LFB S.A. announced that the FDA has accepted for review the Biologic License Application (BLA) of its recombinant Coagulation Factor VIIa (eptacog beta, activated) for the potential treatment of congenital hemophilia A or B in adolescents and adults with inhibitors.
The American Red Cross has announced an emergency call for blood and platelet donations today following a severe winter blood shortage. Eligible donors are being asked to schedule an appointment to give blood.
Adynovate, an extended circulating half-life recombinant Factor VIII (rFVIII), was initially approved for use in patients aged ≥12 years with hemophilia A for on-demand treatment and control of bleeding episodes, and routine prophylaxis to reduce the frequency of bleeding episodes.
The primary endpoint was the number of bleeds over time among patients with emicizumab prophylaxis or no prophylaxis.
For patients who received transfusions in Sweden and Denmark from 2003 to 2012, there was no correlation between the length of red blood cell (RBC) storage and mortality, according to a study published online December 20 in the Annals of Internal Medicine.
A single dose of an experimental gene therapy may benefit patients with hemophilia B, according to trial results presented at the annual meeting of the American Society of Hematology
In a report published online November 8 in Pediatrics, authors present the case of recurrent immune thrombocytopenia (ITP) after influenza vaccination.
Tranexamic acid is associated with a lower risk of bleeding than placebo for patients undergoing coronary artery surgery without increased risk of death or thrombotic complications, according to a study published online in the New England Journal of Medicine
The risk-to-benefit ratio must be considered before discontinuing a selective serotonin reuptake inhibitor (SSRI) in patients prior to an elective operative procedure
Platelet transfusion 48 hours after clopidogrel has small reversing effect post-transfusion
Rigel Pharmaceuticals announced positive data from the Phase 3 study of fostamatinib for the treatment of chronic or persistent immune thrombocytopenia (ITP) in adults.