PB1326 - Clinical severity of the first episode and risk of recurrent neurological symptoms in relapses among patients with immune-mediated thrombotic thrombocytopenic purpura

Tuesday, June 25, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Presenting Author(s)

Addolorata Truma, MD

Research fellow
Università degli studi di Milano, Department of Pathophysiology and Transplantation, Milan, Italy.
milano, Lombardia, Italy

Co-Author(s)

Andrea Artoni

MD
Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy
Milan, Lombardia, Italy
Pasquale Agosti, MD, PhD

Assistant Professor Clinician
Università degli Studi di Milano, Department of Pathophysiology and Transplantation, Milan, Italy; Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy.
Milan, Lombardia, Italy
IM

Ilaria Mancini

Biologist
Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy
Milan, Lombardia, Italy
Juri Alessandro Giannotta, Medical Doctor

MD
Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy
Milano, Lombardia, Italy
BF

Barbara Ferrari

MD
Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy
Milan, Lombardia, Italy
PD

Pasqualina De Leo

Biologist
Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy
Milan, Lombardia, Italy
Flora Peyvandi, MD, PhD

Full Professor
Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Università degli Studi di Milano, Department of Pathophysiology and Transplantation
Milan, Lombardia, Italy

Background: Immune-mediated acquired thrombotic thrombocytopenic purpura (iTTP) is a life-threatening thrombotic microangiopathy often characterized by neurological symptoms (NS) at onset. It is unclear whether NS are associated with a more severe disease course and if patients with NS at onset are at higher risk of experiencing NS at relapses.

Aims: To compare disease severity parameters in patients with and without NS at iTTP onset and to assess the recurrence of NS in subsequent relapses.

Methods: A cohort-study using data from the Milan TTP Registry, including patients at their 1st acute iTTP event was conducted. We excluded patients with previous ischemic stroke, patients with only headache as NS, and patients treated with caplacizumab for its influence on acute event outcomes.

Results: One-hundred patients were enrolled, 67 with NS and 33 without NS at onset of the 1st episode. The median age was slightly higher in the NS than in those without (44 vs 34). NS presence at onset was not associated with abnormal laboratory findings, plasmapheresis sessions, need for early rituximab, and exacerbations. Cardiovascular risk factors such as hypertension, diabetes, hyperlipidemia, and previous major cardiovascular events, were more common in patients with NS, although without statistical significance (Table). During a median follow-up time of 8 years (IQR 4, 12), 14 out of 30 patients with NS at the 1st episode (47%), and none of the 15 patients without-NS had a recurrence of NS at relapse, 4 of whom with ischemic strokes (Figure).

Conclusion(s): Our study suggests that the presence of NS is not a risk factor of disease severity. However, patients displaying NS at their 1st episode are at a higher risk of experiencing NS also during relapses, even exhibiting a more severe phenotype. This pattern suggests an underlying, individual-specific risk factor influencing disease expression that remains to be elucidated.