Molecular stratification of patients with giant cell arteritis to tailor glucocorticoid and tocilizumab therapy

Objectives of the Project

The present research aims to develop and validate biomarkers whose quantification in temporal artery biopsies (TABs) can predict response to glucocorticoids and tocilizumab in patients with giant cell arteritis (GCA) and to stratify patients according to molecular signatures in TABs. Glucocorticoids are the standard of care in GCA but about 40% of patients relapse when glucocorticoids are tapered. Tocilizumab plus glucocorticoids has recently been proven effective at increasing the percentage of GCA patients in remission and sparing glucocorticoids

Specific aims:

1) identification of biomarkers in TABs whose quantification allow to predict at diagnosis patients’ response to glucocorticoids and tocilizumab;

 2) stratification of GCA patients according to molecular signatures in TABs and correlation of such signatures to the clinical characteristics of patients;

 3) validation of the potential predictors and signatures.

The long-term objective is to create the basis for a therapeutic approach in patients with GCA, tailored to molecular characteristics in TABs at diagnosis, aiding physicians to achieve the best outcome in each patients (maximum efficacy with minimal adverse effects) in the shortest time.

Facts and Figures

Project lead
MD, PhD N Pipitone
Azienda Unità Sanitaria Locale - IRCCS
2018 - 2021
FOREUM research grant: € 600.000

Meet the Team

MD, PhD N Pipitone
Azienda Unità Sanitaria Locale - IRCCS
Dr. S Croci
Azienda Unità Sanitaria Locale-IRCCS
Dr. F Ciccia
University of Palermo
Prof. R Alessandro
University of Palermo
Prof. S Fontana
University of Palermo
Prof. M Gonzalez-Gay
Hospital Universitario Marqués de Valdecilla
Dr. R Lopez-Meijas
Hospital Universitario Marqués de Valdecilla
Prof. S Castaneda
Hospital La Princesa, Universidad Autonoma
Prof. J Martin
Institute of Parasitology and Biomedicine López-Neyra
Prof. P Liò
University of Cambridge
Prof. D Saadoun
Pitie-Salpetriere Hospital
Prof. P Villiger
University Hospital Bern
Mr. D Conti
Associazione Malati Reumatici Emilia Romagna
Mr. J Baquero
Foro Español de Pacientes

Patient Voice

One Italian (AMRER) and two Spanish (FEP and LIRE) associations of patients are involved as patient research partners (PRPs). We will further involve patient associations in France and Switzerland.

EULAR guidelines for Patient Research Partners (PRP) inclusion have been followed. We have discussed the design of the project and the burden for patients with the PRPs integrating their feedback. PRPs will be involved in all phases of the project.

PRPs will be involved in writing the patient consent forms and the clinical record forms. Patients who will consent to participate to the study will be asked to report any adverse effects of the therapies and exacerbation of symptoms. PRPs will help with patient recruitment and education by organizing events to make patients gaining knowledge about GCA which will empower them to actively participate in the project (e.g. leaflets about the disease and the research project, meetings, information on the PRP web sites). PRPs will be asked to constantly contribute by bringing patient perspective and experiential knowledge. They will participate to the dissemination of the results among patients and non-academic institutions.