ASH - Blood Disorders

Pharmacy Times interviewed Jason Valent, MD, of the Cleveland Clinic the next steps for his CAEL-101 findings, a treatment for patients living with amyloid light chain amyloidosis.

Pharmacy Times interviewed Jason Valent, MD, of the Cleveland Clinic his recent CAEL-101 findings, and how it can help shape treatment for patients living with amyloid light chain amyloidosis.

Pharmacy Times interviewed Jason Valent, MD, of the Cleveland Clinic about what CAEL-101 is, how it works, and how it is used to treat patients with amyloid light chain amyloidosis.

Pharmacy Times interviewed Jason Valent, MD, of the Cleveland Clinic about his new research surrounding CAEL-101, a treatment for patients living with amyloid light chain amyloidosis.

Per Morten Sandset, MD, of the University of Oslo and Oslo University Hospital, discusses the treatment considerations necessary to properly manage a patient with congenital plasminogen deficiency after she became pregnant.

Per Morten Sandset, MD, of the University of Oslo and Oslo University Hospital, discusses the details of a patient who had a successful pregnancy despite documented infertility due to congenital plasminogen deficiency.

Per Morten Sandset, MD, of the University of Oslo and Oslo University Hospital, discusses the disease impact on the gynecologic system and how often patients become infertile due to this impact.

Per Morten Sandset, MD, of the University of Oslo and Oslo University Hospital, details the treatment options available for patients with congenital plasminogen deficiency.

Per Morten Sandset, MD, of the University of Oslo and Oslo University Hospital, describes common manifestations of congenital plasminogen deficiency and their impact on patient outcomes.

Per Morten Sandset, MD, of the University of Oslo and Oslo University Hospital, discusses why congenital plasminogen deficiency is often underdiagnosed and describes the causes and characteristics of the disorder.

In an interview with Pharmacy Times, Helen Thackray, MD, Thackray describes what she hopes to see for future trials involving sickle cell disease and rivipansel for acute vaso-occlusive crisis.

The data presented at ASH 2020 build on results previously observed in the pivotal HAVEN clinical trial among adults, adolescents, and children with hemophilia A, with and without factor VIII inhibitors.

In an interview with Pharmacy Times, Helen Thackray, MD, Thackray emphasizes the importance of administering rivipansel early in vaso-occlusive crisis and how it is meaningful for both adults and children with sickle cell disease.

In an interview with Pharmacy Times, Helen Thackray, MD, reveals what finding she believes was the most interesting after analyzing the results of the RESET clinical trial, which covers the early initiation with rivipansel for acute vaso-occulusive crisis in sickle cell disease.

Acquired hemophilia A is a rare bleeding disorder caused by autoantibodies that inhibit coagulation factor VIII (FVIII), and the disorder is understudied given its rarity and the lack of randomized prospective trials to guide therapy.

The update outlines that all 5 patients in the high dose 3 x 1013 vg/kg cohort have had at least 1 year of follow-up and showed sustained factor VIII (FVIII) activity levels, with a group median FVIII activity of 56.9% and a group geometric mean FVIII activity of 70.4% via chromogenic assay from week 9 to 52.

BIVV001 is currently being studied as a recombinant factor VIII treatment for hemophilia A.

A single infusion of damoctocog alfa pegol resulted in 25% higher area under the curve and 20% lower clearance in patients with severe hemophilia A.

The early stage of COVID-19 is typically characterized by lymphocytopenia and thrombocytopenia, whereas the late stage may be characterized by more severe lymphocytopenia and other changes.

Helen Thackray, MD, dives deeper into how the researchers tested rivipansel on the study participants and the implications compared to previous trials.

Study supports a more individualized approach to pharmacological thromboprophylaxis over a universal one in patients with COVID-19.

Intravenous immunoglobulin administered to hypoxic non-ventilated COVID-19 patients with an A-a gradient of more than 200 mg Hg significantly decreases the rates of progression to mechanical ventilation.

Helen Thackray, MD, provides a brief overview and highlights of the RESET clinical trial.

The study found that the infusion helped to prevent spontaneous bleeding in patients with hemophilia A.

The proportion of patients with hemophilia A receiving Hemlibra who experienced zero treated bleeds increased with each consecutive 24-week period.

Pharmacy Times® interviewed Christopher Moxham of Fulcrum Therapeutics, on his closing thoughts on the research he conducted into the induction of fetal hemoglobin by FTX6058.

Pharmacy Times® interviewed Charles Nakar, MD and Amy Shapiro, MD, of the Indiana Hemophilia & Thrombosis Center, on 3 new patient cases from their presentation and the significance of these cases to their research.

Pharmacy Times® interviewed Christopher Moxham of Fulcrum Therapeutics, on the results observed in his research when treating donors who were healthy or had sickle cell disease with FTX-6058.

Pharmacy Times® interviewed Charles Nakar, MD and Amy Shapiro, MD, of the Indiana Hemophilia & Thrombosis Center, on the process for the diagnosis of patients with congenital plasminogen deficiency.

Pharmacy Times® interviewed Christopher Moxham of Fulcrum Therapeutics, on what their parallel target identification efforts were able to identify, and how this identification was important in his research.