A Prospective Observational Study for Evaluating CGVHD

NCT ID: NCT02991846

Last Updated: 2022-11-25

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 248 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2017-09-01
• Study Completion Date: 2022-11-22

Brief Summary

Prospective, observational, multicentre, spontaneous, non-interventional study This study will evaluate all consecutive patients who develop chronic graft-versus-host disease, reported by the Italian GITMO centers according to a standardized Web platform for real-time, onsite data collection. The platform for data collection will be based on a software prototype developed by the Clinica di Ematologia di Ancona Transplant Center for the management of patients with chronic graft-versus-host disease. This software has been integrated with algorithms that automatically determine: severity of chronic graft-versus-host disease and overall response according to the 2015 NIH consensus criteria.

Detailed Description

Chronic Graft-versus-Host Disease represents the first cause of transplant-related mortality and reduced quality of life after transplant (HSCT). The epidemiology of Chronic Graft-versus-Host Disease is largely unknown; moreover, diagnosis of Chronic Graft-versus-Host Disease can be easily missed because its onset is often late in the post-transplant period, requires specific follow up, and general practitioners are usually not familiar with this entity. Successful treatment of Chronic Graft-versus-Host Disease represents an unmet clinical need in the field of allogeneic transplantation. Steroids are standard treatment, but up to 60% of the patients will require second-line treatment but there is no standard second-line treatment for Chronic Graft-versus-Host Disease steroid refractory. To help standardise the management Chronic Graft-versus-Host Disease, the NIH Consortium proposed consensus definitions for diagnosis, scoring and response criteria in 2006 revised in 2015 which offers a shared framework to study this rare disease. These criteria are not yet validated and thus not suitable for clinical trials.

This study is prospective, observational, multicentre, spontaneous, non-interventional study that will evaluate all consecutive patients who develop chronic graft-versus-host disease, reported by the Italian GITMO centers according to a standardized Web platform for real-time, on-site data collection. The platform for data collection will be based on a software prototype developed by the Ancona Transplant Center for the management of patients with Chronic Graft-versus-Host Disease. This software has been integrated with algorithms that automatically determine: severity of Chronic Graft-versus-Host Disease and overall response by the 2015 NIH consensus criteria. Historical controls to compare Chronic Graft-versus-Host Disease incidence, toxicities, response rate and hard outcomes will be used.

The aim of this project is to evaluate prospectively the long-term effectiveness of different therapies by the hard outcome "failure free survival" commonly considered the most reliable one. The failure free survival is the result of a number of factors that influence the treatment failure and has been shown a reliable predictor of long-term survival. Main cause of failure is the change in immunosuppressive treatment although recurrent disease, treatment toxicity and mortality from Chronic Graft-versus-Host Disease (or other infectious complications) also contribute to failure free survival. Second, we aim to evaluate the prognostic ability of the latest NIH response criteria to predict main hard survival outcomes and to assess their suitability as a tool for decision-making that ultimately leads to treatment changes. Finally, we aim to evaluate the feasibility of the use of an electronic tool for data collection in daily clinical practice.

Conditions

Chronic Graft-Versus-Host Disease

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Patients with cGVHD

All consecutive patients undergoing allogenic stem cell transplant for any underlying disease who develop chronic graft-versus-host disease (cGVHD)

No interventions assigned to this group

Eligibility Criteria

Inclusion Criteria

• Any age
• All patients who develop cGVHD (any grade) by the NIH criteria after allogeneic transplantation
• Written and signed informed consent

Exclusion Criteria

• Absence of informed consent

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Gruppo Italiano Trapianto di Midollo Osseo

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Attilio Olivieri, MD

Role: PRINCIPAL_INVESTIGATOR

AOU Ospedali Riuniti di Ancona

Locations

Azienda Ospedaliera SS Antonio e Biagio

Alessandria, , Italy

Azienda Ospedaliero-Universitaria Ospedali Riuniti

Ancona, , Italy

Policlinico di Bari-Ematologia con trapianti

Bari, , Italy

Divisione di Ematologia - Ospedali Papa Giovanni XXIII

Bergamo, , Italy

Ospedale San Orsola

Bologna, , Italy

Ospedale Regionale Generale- Divisione Ematologia

Bolzano, , Italy

AO Spedali Civili di Brescia- USD - TMO Adulti

Brescia, , Italy

Ospedale Ferrarotto - Ematologia

Catania, , Italy

S.C. Ematologia - Azienda Ospedaliera S. Croce e Carle

Cuneo, , Italy

Cattedra di Ematologia - Azienda Ospedaliera di Careggi

Florence, , Italy

Ospedale Gaslini

Genova, , Italy

Osp. Card. Panico

Lecce, , Italy

Ospedale Maggiore - Policlinico

Milan, , Italy

Ospedale San Raffaele

Milan, , Italy

CTMO Fond MBBM Clinica pediatrica

Monza, , Italy

Uoc Sit Tmo

Napoli, , Italy

Azienda ospedaliera Universitaria di Parma

Parma, , Italy

Ospedale G. Da Saliceto di Piacenza

Piacenza, , Italy

Azienda Ospedaliero Universitaria Pisana

Pisa, , Italy

Centro Unico Regionale Trapianti di Midollo Osseo - Ospedale Bianchi-Melacino-Morelli

Reggio Calabria, , Italy

Arciospedale S. M. Novella

Reggio Emilia, , Italy

Cattedra di Ematologia - Policlinico Umberto I

Roma, , Italy

A.O.U. Citta della Salute e della Scienza

Torino, , Italy

Ospedale Regina Margherita

Torino, , Italy

A.O. Santa Maria della Misericordia

Udine, , Italy

Policlinico GB Rossi

Verona, , Italy

Ospedale S. Bortolo-Divisione Ematologia

Vicenza, , Italy

Countries

Italy

Other Identifiers

GITMO-GVCrOSy

Identifier Type: -

Identifier Source: org_study_id