Big data are used in the pursuit of precision medicine in the general population. Applying these tools to patients with sickle cell disease (SCD) is essential for ensuring that they receive the most appropriate customized therapy for their disease. For these tools to be applied, there must be a large number of willing, fully phenotyped participants in collaborative registries. Working collaboratively to respond to unmet clinical needs and the lack of a large multisite registry, SCD providers at ...
Feb 09, 2022•16 min•Ep. 28
Sickle cell disease (SCD) is an inherited blood condition resulting from abnormal hemoglobin production. It is one of the most common genetic diseases in the world. The clinical manifestations are variable and range from recurrent acute and debilitating painful crises to life-threatening pulmonary, cardiovascular, renal, and neurologic complications. The only curative treatment of SCD at this time is bone marrow transplantation (also called hematopoietic stem cell transplantation) using healthy ...
Jan 26, 2022•13 min•Ep. 27
The prospect of a clinical strategy using an adeno-associated virus (AAV) vector for expression of therapeutic levels of factor VIII (FVIII) has been highly desirable. This was initially anticipated by promising data from clinical studies on AAV5-FVIII in men with severe hemophilia A. However, long-term follow-up showed a unique efficacy concern on the sustainability and durability derived from a continuous decline in the FVIII transgene levels starting 1 year after vector injection through year...
Jan 26, 2022•31 min•Ep. 26
The availability of novel nonfactor therapeutics is revolutionizing the management of hemophilia in individuals with inhibitory antibodies, as well as making prophylaxis more convenient even in the absence of inhibitors. Unfortunately, the use of these products has been associated with thrombotic events that are not typically seen with factor replacement. These are primarily seen when a patient on a nonfactor therapy experiences breakthrough bleeding and concomitantly receives another hemostatic...
Oct 01, 2021•18 min•Ep. 25
Amy Shapiro discusses the recent approval of concizumab for the treatment of patients with hemophilia. This novel subcutaneous homeostatic rebalancing agent has proven to be useful for the treatment of patients with hemophilia A and B, and we hope you find this talk to be useful to understand it.
Jan 12, 2021•16 min•Ep. 24
Allogeneic hematopoietic stem cell transplantation (HCT) is curative in many patients with advanced hematopoietic malignancies. Donor T cells not only facilitate engraftment and protect against opportunistic pathogens and residual disease, but can also cause graft-versus-host disease (GVHD), with significant morbidity and mortality.
Oct 13, 2020•24 min•Ep. 23
The high incidence of thromboembolic disease, and in particular venous thromboembolism (VTE), has emerged as an important consideration in hospitalized and critically ill patients with coronavirus disease 2019 (COVID-19).
Sep 15, 2020•15 min•Ep. 22
The treatment of hemophilia, which has undergone many transformative changes over the past 60 years, is poised for yet another disruptive change: the use of gene therapy to produce functional cures in some persons with hemophilia A or B.
Sep 15, 2020•17 min•Ep. 21
The changing landscape of anticoagulation in cancer-associated thrombosis
Mar 10, 2020•16 min•Ep. 20
TA-TMA: state of the art for diagnosis and treatment by Blood Advances Talks
Jan 14, 2020•20 min•Ep. 19
Inherited predisposition to myeloid malignancies by Blood Advances Talks
Sep 10, 2019•19 min•Ep. 18
Managing patients who are receiving warfarin (or DOAC)and need an elective surgery or procedure by Blood Advances Talks
Jun 25, 2019•22 min•Ep. 17
Daily light-and-darkness onset regulates mouse hematopoietic stem cells by Blood Advances Talks
Feb 26, 2019•17 min•Ep. 16
Extended therapy for unprovoked venous thromboembolism: when is it indicated? by Blood Advances Talks
Feb 12, 2019•16 min•Ep. 15
Antiplatelet therapy in the management of atherothrombosis: recent clinical advances by Blood Advances Talks
Jul 27, 2018•16 min•Ep. 14
Challenges facing the benign hematology physician-scientist workforce by Blood Advances Talks
Feb 13, 2018•9 min•Ep. 13
Choosing for whom to recommend allogeneic transplantation for AML in CRL by Blood Advances Talks
Jan 23, 2018•18 min•Ep. 12
Catalyzing innovation in clinical trial design and analysis at NHLBI by Blood Advances Talks
Jan 11, 2018•15 min•Ep. 11
ITP and thrombosis: an intriguing association by Blood Advances Talks
Nov 16, 2017•24 min•Ep. 10
The current therapeutic landscape of FLT3 inhibitors by Blood Advances Talks
Nov 16, 2017•16 min•Ep. 9
How many mice? Design considerations for murine studies by Blood Advances Talks
Aug 15, 2017•16 min•Ep. 8
The Direct Oral Anticoagulants – can we finally stop using rat poison? by Blood Advances Talks
Jun 19, 2017•10 min•Ep. 7
Impact of the ICAL on the treatment of acute leukemia by Blood Advances Talks
Mar 15, 2017•23 min•Ep. 6
Venetoclax: a primer by Blood Advances Talks
Feb 28, 2017•19 min•Ep. 5
Technological Solutions For Global Hematology And Oncology by Blood Advances Talks
Feb 22, 2017•17 min•Ep. 4
The Lucky Iron Fish: a simple solution for iron deficiency by Blood Advances Talks
Jan 24, 2017•10 min•Ep. 3
New developments in the management of sickle cell disease by Blood Advances Talks
Dec 01, 2016•12 min•Ep. 2
Should we avoid heparin to eliminate HIT? by Blood Advances Talks
Dec 01, 2016•13 min•Ep. 1
