PB1280 - Assessment of platelet aggregation and thrombin generation in patients with familial chylomicronemia syndrome treated with volanesorsen

Tuesday, June 25, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Presenting Author(s)

IC

Ilenia Calcaterra

Assistant Professor
Federico II University of Naples
Napoli, Campania, Italy

Co-Author(s)

Rs

Renata santoro

Biotech student
University Federico II of Naples
Naples, Campania, Italy
NV

Nicoletta Vitelli

Resident
University Federico II of Naples
Naples, Campania, Italy
VP

Vincenzina Palermo

Resident
University Federico II of Naples
Naples, Campania, Italy
SD

Sofia Donnarumma

Resident
University Federico II of Naples
Naples, Campania, Italy
FD

Francesca De Ruberto

Resident
University Federico II of Naples
Naples, Campania, Italy
CG

Cornelia Guerrino

Pharmacist
University Federico II of Naples
Naples, Campania, Italy
mD

maria Donata Di Taranto

Professor
University Federico II of Naples
Naples, Campania, Italy
GI

Gabriella Iannuzzo

Physician
University Federico II of Naples
Naples, Campania, Italy
FC

Ferdinando Cirillo

biologist
DEPARTMENT OF CLINICAL MEDICINE AND SURGERY. Federico II University of Naples
Naples, Campania, Italy
gC

giovanna Cardiero

Biologist
University Federico II of Naples
Naples, Campania, Italy
GF

Giuliana Fortunato

Professor
University Federico II of Naples
Naples, Campania, Italy
MD

Matteo Di Minno

Professor
Department of Clinical Medicine and Surgery, Federico II University of Naples, Naples, Italy
Naples, Campania, Italy

Background: The antisense oligonucleotide against Apolipoprotein CIII (APOC3) mRNA volanesorsen was recently introduced to treat Familial Chylomicronemia Syndrome (FCS). Cases of thromobocytopenia are reported among patients treated with volanesorsen.

Aims: The aim of the study is to evaluate platelet function and thrombin generation (TG) assessment in FCS patients receiving volanesorsen.

Methods: We performed a cross-sectional study FCS patients treated with volanesorsen. To assess the thrombin generation, samples were processed by CAT (with PPP-reagent LOW). The results were expressed by the trombogram graphic (thrombin variation over time); lagTime; Endogenous Thormbin Potential (ETP); peak; time to reach peak (ttpeak), startTail e velocity Index. Platelet aggregation was assessed by testing different agonists (adenosine diphosphate, arachidonic acid, ristocetin, collagen) using turbidimetry method.

Results: Four FCS patients and 4 matched healthy controls were included in the present study. Thrombin generation results show values in normal range (for patients and controls respectively lagTime: 10,42 ± 4,40 and 9,25 ± 0,99 p=0,685; Peak 76,84 ± 1,07 123,30 ± 39,45 p=0,104; ttPeak: 14,33 ± 4,01 and 13,10 ± 0,67 p=0,620; startTail: 32,13 ± 3,54 and 29,46 ± 1,69 p=0,143; velocityIndex: 20,21 ± 3,63 and 33,05 ± 13,21 p=0,143). We observed a difference not statistically significant between cases and controls in ETP, thrombin amount generates (599,80±73,47 in cases and 900,2±210,99 in controls, MD: -33,4% p=0,102), and in maximum concentration reached, the peak value (76,84±1,07 in cases and 123,30±39,45 in controls). It was possible to test platelet aggregation only in 1 patient because the turbidimetry method was not applicable because of the presence of plasma CM and showed normal values.

Conclusion(s): Our study showed for the first time that no significant changes in the general haemostasis assessed by thrombin generation and in platelet function have been observed in FCS patients receiving volanesorsen.