PB0332 - The role of thrombopoietin receptor agonists in preventing surgical bleeding in patients with inherited thrombocytopenia

Sunday, July 10, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: In-Person

Presenting Author(s)

Miguel Carneiro, MD

MD, resident in Hematology
Thrombosis and Haemostasis Unit, Centro Hospitalar Universitário do Porto, Porto, Portugal
Porto, Porto, Portugal

Co-Author(s)

ES

Eunice Silva

MD, resident in Hematology
Thrombosis and Haemostasis Unit, Centro Hospitalar Universitário do Porto, Porto, Portugal
Porto, Porto, Portugal
AF

Andreia Ferreira

MD, resident in Hematology
Centro Hospitalar Universitário do Porto, Porto, Portugal
Porto, Porto, Portugal
MO

Margarida Oliveira

MD, resident in Hematology
Thrombosis and Haemostasis Unit, Centro Hospitalar Universitário do Porto, Porto, Portugal
Porto, Porto, Portugal
MC

Maria Coutinho

MD, Assistent in Hematology
Thrombosis and Haemostasis Unit, Centro Hospitalar Universitário do Porto, Porto, Portugal
Porto, Porto, Portugal
EC

Eugénia Cruz, MD, PhD

Consultant Physician, MD, PhD
Thrombosis and Hemostasis Unit, Clinical Haematology Service, Centro Hospitalar Universitário do Porto, Porto, Portugal
Porto, Porto, Portugal
SM

Sara Morais

Senior Physician, MD
Thrombosis and Hemostasis Unit, Clinical Haematology Service, Centro Hospitalar Universitário do Porto, Porto, Portugal
Porto, Porto, Portugal

Background: Inherited thrombocytopenia (IT) are heterogeneous group of platelet disorders, characterized by diminished platelet count (PLT) and increased bleeding potential. Recent advances in diagnostic methods allowed the identification of an increasing number of IT. In contrast, the therapeutic options of IT patients remained unchanged and based mainly on PLT transfusion. Recently, thrombopoietin receptor agonists (TPO-RA) were introduced in the treatment of patients with IT.

Aims: To evaluate the efficacy of a TPO-RA (Eltrombopag) in preventing surgical bleeding in patients with IT.

Methods: Four patients, 3M/1F, median age 45 years-old (min-max 12-60) with different IT (MYH9-RD, ANKRD26-RT, ACTN1-RT and ITGA2B/ITGB3-RT), achieved five treatments with Eltrombopag (50mg/day) prior to surgical procedures, between 2017 and 2020.

Results: The procedures were tympanoplasty, two hydrocelectomies, a mitral valvuloplasty with tricuspid annuloplasty and a biopsy of pancreatic lesion. The median PLT was 43x103/µL (min-max 26-83x10^3/µL) at baseline, and 103x10^3/µL (min-max 56-211x10^3/µL) after 24 days of treatment in average (min-max 19-26). PLT increase was greater than 100% in 4 of the treatments and was only marginal (30%) in one treatment. No additional measures to improve haemostasis were needed and all procedures were performed without increase of the expected bleeding. Only one treatment was interrupted early, soon after surgery, due to severe headaches. The patient who underwent cardiac surgery had an acute coronary syndrome considered secondary to the surgery.

Conclusion(s): Treatment with 50 mg/day of Eltrombopag was confirmed to be effective in preventing haemorrhagic events in patients with IT. The limited duration of treatment allows to avoid potential adverse effects such as thrombosis or bone marrow fibrosis. A patient with ACTN1-RT, an IT associated with defects in cytoskeletal proteins, had an excellent response to treatment. This was the first time that TPO-RA treatment had been reported in a patient with this diagnosis.