Lusutrombopag is currently indicated for the treatment of severe thrombocytopaenia in patients suffering from liver disease undergoing invasive surgery. Following the approval from NICE it is now set to be funded by the NHS.
Severe thrombocytopaenia is associated with an increased risk of bleeding following surgery due to decreased platelet levels (platelets < 50 x 10^9/L). Platelet concentrations are controlled by thrombopoietin (TPO). This is produced in the liver and is responsible for initiating a cell signalling cascade resulting in the genesis of platelets following megakaryocyte differentiation (WEKSLER, 2007). In normal circumstances, TPO is released at a constant rate, with platelet levels being mediated through a negative feedback loop (Gurney et al., 1994). TPO production in patients with chronic liver disease is depressed, resulting in low platelet concentrations and prevents blood form clotting normally (Peck-Radosavljevic et al., 1997).
How does Lusutrombopag work?
Lusutrombopag is designed as a treatment to increase platelet concentrations in the days leading to an invasive procedure. Lusutrombopag acts as TPO receptor agonist. It binds megakaryocytes to promote the production of platelets. Lusutrombopag therefore compensates the inability of the liver to produce TPO at a normal rate (Peck‐Radosavljevic et al., 2019). Two key studies, L-PLUS 1 and 2 assessed the efficacy and safety of lusutrombopag as a treatment to prevent the need for platelet transfusions. L-PLUS 2 was a global, phase 3, double-blind, placebo-controlled study which assessed whether lusutrombopag affected the need for platelet transfusions before surgery and whether it increased platelet levels. The results showed that of patients receiving lusutrombopag, 64.8% avoided a platelet transfusion compared to 29% in the control group (fig.1).
Decision on cost effectiveness
Lusutrombopag has been deemed cost effective by NICE (12th December) and will be available across the NHS. The recommendations are based on the likely benefits to patients. These include fewer transfusions, as well as a reduced possibility of transfusion related complications. Furthermore, invasive surgeries would not require treatment windows in which to operate, which would lead to fewer surgery cancellations and hospital visits. These patient benefits would also help offset the cost of the once a day oral tablet.
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Gurney, A., Carver-Moore, K., de Sauvage, F. and Moore, M. (1994). Thrombocytopenia in c-mpl-deficient mice. Science, 265(5177), pp.1445-1447.
Peck‐Radosavljevic, M., Simon, K., Iacobellis, A., Hassanein, T., Kayali, Z., Tran, A., Makara, M., Ben Ari, Z., Braun, M., Mitrut, P., Yang, S., Akdogan, M., Pirisi, M., Duggal, A., Ochiai, T., Motomiya, T., Kano, T., Nagata, T. and Afdhal, N. (2019). Lusutrombopag for the Treatment of Thrombocytopenia in Patients With Chronic Liver Disease Undergoing Invasive Procedures (L‐PLUS 2). Hepatology, 70(4), pp.1336-1348.
Peck-Radosavljevic, M., Zacherl, J., Meng, Y., Pidlich, J., Lipinski, E., Längle, F., Steininger, R., Mühlbacher, F. and Gangl, A. (1997). Is inadequate thrombopoietin production a major cause of thrombocytopenia in cirrhosis of the liver?. Journal of Hepatology, 27(1), pp.127-131.
WEKSLER, B. (2007). Review article: the pathophysiology of thrombocytopenia in hepatitis C virus infection and chronic liver disease. Alimentary Pharmacology & Therapeutics, 26, pp.13-19.