Hematology

Top Story

Gene therapy appears effective in patients with transfusion-dependent beta-thalassemia

Gene therapy appears effective in patients with transfusion-dependent beta-thalassemia
February 17, 2017

SAN DIEGO — The infusion of a lentiviral vector containing an engineered betaA-T87Q-globin gene effectively reduced or eliminated the transfusion needs of patients with beta-thalassemia major without leading to severe adverse events, according to updated results of a full cohort from the Northstar Study presented at the ASH Annual Meeting and Exposition.

Researchers developed the gene therapy drug product, LentiGlobin BB305 (Bluebird Bio Inc.), which uses a non-communicable virus to deliver a fully functioning HBB gene to the patient’s blood-producing stem cells.

In the Journals Plus

Factor XI vaccination may help prevent thrombosis

February 17, 2017
A novel coagulation Factor XI vaccine may serve as a promising strategy in the prevention of thromboembolic diseases, according to a report published in Journal of

New ASH president: 'Extraordinary science' at genomic level must be brought to bedside

February 14, 2017
Kenneth C. Anderson, MD, director of the Lebow Institute for Myeloma Therapeutics and Jerome Lipper Myeloma Center at Dana-Farber Cancer Institute, took over as…
In the Journals Plus

Adverse events result in high rate of low–molecular-weight heparin discontinuation

February 13, 2017
One in five patients with cancer-associated venous thromboembolism
CME

Diagnostic and Therapeutic Challenges in Von Willebrand Disease

This activity is supported by an educational grant from Shire.

With the significant variability in the subtypes and clinical manifestations of von Willebrand disease (VWD), diagnosis…
More »
Video

VIDEO: ‘March toward a cure’ continues with rapid developments in myeloma treatment

February 17, 2016
More »
Featured
CME

The Patient with Castration-resistant Prostate Cancer

No commercial support for this activity.

Castration-resistant prostate cancer (CRPC) is a heterogeneous condition that encompasses a continuum of disease…
More »
In the Journals PlusAbstract Only

Excess mortality in the myelodysplastic syndromes

February 19, 2017
AUTHORS:
Nomdedeu M, et al.

In the Journals PlusAbstract Only

The role of acuity of illness at presentation in early mortality in black children with acute myeloid leukemia

February 18, 2017
AUTHORS:
Winestone LE, et al.

In the Journals PlusAbstract Only

Arterio-venous fistula for automated red blood cells exchange in patients with sickle cell disease: Complications and outcomes

February 17, 2017
AUTHORS:
Delville M, et al.

Meeting News

Gene therapy appears effective in patients with transfusion-dependent beta-thalassemia

February 17, 2017
SAN DIEGO — The infusion of a lentiviral vector containing an engineered betaA-T87Q-globin gene effectively reduced or eliminated the…

In the Journals Plus

Factor XI vaccination may help prevent thrombosis

February 17, 2017
A novel coagulation Factor XI vaccine may serve as a promising strategy in the prevention of thromboembolic diseases, according to a report published…

In the Journals PlusAbstract Only

Progressive refractory light chain amyloidosis and multiple myeloma patients are responsive to the addition of clarithromycin to IMiD based therapy

February 15, 2017
AUTHORS:
Shaulov A, et al. ...

New ASH president: 'Extraordinary science' at genomic level must be brought to bedside

February 14, 2017
Kenneth C. Anderson, MD, director of the Lebow Institute for Myeloma Therapeutics and Jerome Lipper Myeloma Center at Dana-Farber Cancer Institute…

In the Journals Plus

Adverse events result in high rate of low–molecular-weight heparin discontinuation

February 13, 2017
One in five patients with cancer-associated

In the Journals Plus

HLA–identical sibling HSCT leads to high rate of 5-year survival in sickle cell disease

February 10, 2017
HLA–identical sibling transplantation showed promising long-term survival for children and adults with sickle cell disease, according to an…

In the Journals Plus

Lower HbA1c levels linked to sickle cell trait in black adults

February 10, 2017
Black adults with sickle cell trait have lower HbA1c values compared with participants without sickle cell trait despite similar fasting and 2-hour…

More Headlines »
Advertisement
Advertisement