Eltrombopag to allow chemotherapy in a patient with MYH9 -related inherited thrombocytopenia and pancreatic cancer
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CASE REPORT
Eltrombopag to allow chemotherapy in a patient with MYH9‑related inherited thrombocytopenia and pancreatic cancer Francesco Paciullo1 · Loredana Bury1 · Paolo Gresele1 Received: 10 February 2020 / Revised: 29 May 2020 / Accepted: 4 June 2020 © Japanese Society of Hematology 2020
Abstract Chemotherapy is the mainstay of treatment for advanced pancreatic cancer however, due to possible myelotoxicity, it is used with caution in patients with thrombocytopenia, especially when severe. TPO-receptor agonists have been employed for chemotherapy-induced thrombocytopenia, however treatment with TPO-receptor agonists to allow chemotherapy in patients with inherited thrombocytopenia has not been reported so far. We report the first successful use of eltrombopag to prevent chemotherapy-induced thrombocytopenia in a patient with MHY9-related disorder and pancreatic cancer. Treatment with eltrombopag allowed to attain a safe and stable platelet count for several months sufficient to permit chemotherapy and to allow the patient to undergo endoscopic placement of a biliary stent with no bleeding complications. Keywords Eltrombopag · Chemotherapy-induced thrombocytopenia · MYH9-RD · Thrombocytopenia
Introduction MHY9-related disorder (MYH9-RD), an autosomal dominant syndromic inherited platelet disorder, is the most frequent form of inherited thrombocytopenia and is caused by monoallelic variants of the gene coding for the heavy chain of non-muscle myosin II A. MYH9-RD usually associates with a low bleeding risk, but the phenotype can vary widely depending on the genotype, with some patients presenting with severe thrombocytopenia and mucocutaneous bleeding [1]. Although platelet transfusions remain the mainstay for the treatment of bleeding and preparation to surgery, in recent years eltrombopag, an oral TPO-mimetic indicated for chronic refractory immune thrombocytopenic purpura (ITP), severe aplastic anemia and HCV-related thrombocytopenia, has been shown to be effective in increasing transiently the platelet count in MYH9-RD as well as in some other inherited thrombocytopenias [2]. In a phase 2 clinical trial, eltrombopag given for up to 6 weeks raised the platelet count in 10 out of 11 patients * Paolo Gresele [email protected] 1
Department of Medicine, Section of Internal and Cardiovascular Medicine, University of Perugia, Perugia, Italy
with MYH9-RD and an initial platelet count
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