ASH 2020 late-breaking abstracts: a preview

30 Nov 2020

The 62nd American Society of Hematology (ASH) Annual Meeting and Exposition will be held virtually from 5-8 December. Here are some of the late-breaking abstracts that will be featured during a general session on 8 December beginning at 12:30 GMT.

  • The addition of mycophenolate (MMF) to corticosteroid therapy resulted in significantly fewer treatment failures than corticosteroid alone in patients with immune thrombocytopenia, according to the randomised FLIGHT trial.

Importantly, the therapy was effective even with the inclusion of a substantial proportion of elderly patients. Adding MMF to the corticosteroid yielded about half the treatment failures of corticosteroid alone.

MMF is often used as a second-line therapy for the rare autoimmune disorder, but these results from the UK-based trial could yield a change to standard practice with the combination regimen used as a first-line treatment.

  • The novel agent asciminib yielded better outcomes than bosutinib in a trial of patients with chronic myeloid leukaemia in chronic phase (CML-CP) who had previously been treated with at least two tyrosine kinase inhibitors (TKIs).

Asciminib is a first-in-class agent known as a STAMP inhibitor, with a different mechanism of action from other TKIs used in CML therapy.

In the new randomised phase III trial, patients who received asciminib fared better with regard to major molecular response rate, and the agent also had a favorable safety profile, with fewer patients discontinuing therapy due to adverse events.

  • An experimental gene therapy known as etranacogene dezaparvovec showed promising results in patients with severe or moderate-severe haemophilia B, according to the first data from the phase III HOPE-B trial.

The treatment works by causing patients to begin making factor IX (FIX), potentially reducing the need for high levels of external FIX consumption given as prophylaxis.

The trial, which will follow patients for five years, showed rapid increases in FIX activity following a single dose of therapy, and a large reduction in bleeding in most patients after 26 weeks of follow-up. The treatment was generally well tolerated, and annualised FIX consumption rates dropped by 96%.

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