Promacta in MDS – pro
Thrombocytopenia is a frequent symptom in patients with myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). Eltrombopag is a small molecule thrombopoietin receptor agonist that might be an option to treat thrombocytopenia in these diseases. There is significant literature supporting the use of this drug in MDS. To the extent that ATG may be used, it is not connected to the use of Prmacata and is not relevant to the use of this drug.
Eltrombopag is well-tolerated in patients with lower-risk MDS and severe thrombocytopenia and is clinically effective in raising PLT count and reducing bleeding events. QoL improves with response to treatment. There are two randomized phase II studies supporting its efectiveness.
Vicente A, Patel BA, Gutierrez-Rodrigues F, Groarke E, Giudice V, Lotter J, Feng X, Kajigaya S, Weinstein B, Barranta E, Olnes MJ, Parikh AR, Albitar M, Wu CO, Shalhoub R, Calvo KR, Townsley DM, Scheinberg P, Dunbar CE, Young NS, Winkler T. Eltrombopag monotherapy can improve hematopoiesis in patients with low to intermediate risk-1 myelodysplastic syndrome. Haematologica. 2020 Dec 1;105(12):2785-2794.
Mittelman M, Platzbecker U, Afanasyev B, et al. Eltrombopag for advanced myelodysplastic syndromes or acute myeloid leukaemia and severe thrombocytopenia (ASPIRE):
a randomised, placebo-controlled, phase 2 trial. Lancet Haematol. 2017 December 11. [Epub ahead of print]
E N Oliva et al, Long Term Effects of Eltrombopag Treatment Versus Placebo for Low-Risk Myelodysplastic Syndromes with Thrombocytopenia (EQoL-MDS): Interim Results of a Single-Blind, Randomised, Controlled, Phase 2 Superiority Trial. Blood (2019) 134 (Supplement_1): 3000.