Christoph Königs (Frankfurt, Germany) presented the final results of the phase 3 PUPs A-LONG study (NCT02234323). 103 previously untreated patients (<6 years) with haemophilia A were treated with an extended half-life recombinant factor VIII Fc fusion protein. The primary endpoint was the incidence of inhibitor development. A secondary endpoint was annualised bleeding rate. 87 patients completed...
In this issue of The Lancet Haematology, Matthias Stelljes and colleagues1 present a compelling exploration of an approach to allogeneic haematopoietic stem-cell transplantation (HSCT) in…
Non-inferiority of disease control could not be shown at the 2·5% significance level. The rate of treatment success was also not statistically better for patients…
Combination of sintilimab with P-GEMOX seems to be an active and safe first-line regimen for patients with advanced ENKTL.
The use of chimeric antigen receptor (CAR) T cells in haematological malignancies is becoming increasingly widespread, with several authorised drugs and many patients benefiting from…
The exciting therapeutic potential of nitric oxide-based pharmaceuticals is the inspiration for the work of Mark Gladwin and colleagues1 in this issue of The Lancet…
Riociguat was safe and had a significant haemodynamic effect on systemic blood pressure. The results of this study provide measures of effect and variability that…
