Clinical Edge

Summaries of Must-Read Clinical Literature, Guidelines, and FDA Actions

Gene therapy cut need for blood transfusions in beta thalassemia

Marktel S et al. ASH 2017 Abstract 355

Key clinical point: Gene therapy engraftment was achieved sooner using intrabone delivery.

Major finding: After a median of 16 months of follow-up, five of seven patients who received this novel gene therapy needed markedly fewer blood transfusions than at baseline.

Data source: An interventional phase 1/2 trial of seven patients with transfusion-dependent beta thalassemia.

Disclosures: Telethon Foundation provided funding. Dr. Marktel disclosed research funding from GlaxoSmithKline, which has licensed the therapy.

Source: Marktel S et al. ASH 2017 Abstract 355.

Read the full article.

Citation:

Marktel S et al. ASH 2017 Abstract 355

This Week's Must Reads

Consider bleeding disorders in heavy menstrual bleeding, O’Brien B et al. J Pediatr Adolesc Gynecol. 2018 Nov 22 . doi: 10.1016/j.jpag.2018.11.005

Lower-dose treatment could save money in aTTP, Zwicker JI et al. ASH 2018, Abstract 374

Low rates of bleeding with DOAC interruption, Douketis J et al. ASH 2018, Abstract LBA-5

Gene therapy in severe hemophilia B, Reiss UM et al. ASH 2018, Abstract 491

Luspatercept reduced transfusion burden in trial, List AF et al. ASH 2018, abstract 1

Must Reads in Bleeding Disorders

Consider bleeding disorders in heavy menstrual bleeding, O’Brien B et al. J Pediatr Adolesc Gynecol. 2018 Nov 22 . doi: 10.1016/j.jpag.2018.11.005

Lower-dose treatment could save money in aTTP, Zwicker JI et al. ASH 2018, Abstract 374

Low rates of bleeding with DOAC interruption, Douketis J et al. ASH 2018, Abstract LBA-5

Gene therapy in severe hemophilia B, Reiss UM et al. ASH 2018, Abstract 491

Luspatercept reduced transfusion burden in trial, List AF et al. ASH 2018, abstract 1