Hepatic Veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a severe complication following hematopoietic stem cell transplantation (HSCT), traditionally diagnosed based on clinical criteria. This study aimed to evaluate the diagnostic performance of liver stiffness measurement (LSM) as a non-invasive tool for non invasive diagnosis of VOD/SOS. A multicentre clinical trial was conducted in Italy from April 2018 to December 2021, screening 1089 patients across 25 centers. VOD/SOS diagnosis followed established clinical guidelines, and patients underwent comprehensive clinical, laboratory, and imaging evaluations up to +100 days post-HSCT or until VOD/SOS diagnosis. LSM was measured pre-HSCT and on specific post-transplant days (ClinicalTrials.gov: NCT03426358). The study enrolled 774 adults and 167 children. The +100-day incidence of VOD/SOS HSCT was 5.53 and 5.26 in the overall and allo-HSCT population, higher in children (14.3%) than in adults (3.68%). The 100-day overall survival (OS) probability was 89.5% (overall) and 89.0% (allo-HSCT) while one-yr OS 79% and 78%, respectively, with outcomes varying by VOD/SOS occurrence and severity. LSM significantly differed between VOD/SOS patients and non-affected individuals at all post-HSCT time points, correlating with disease severity. A diagnostic algorithm was proposed, achieving ≥95% sensitivity and specificity, with a 6 kPa rule-out and 25 kPa rule-in cut-off, enhanced by the “three-time pre-HSCT rule.” Survivors showed declining LSM over time, while non-survivors did not. Fully recovered patients had lower LSM than non-improvers. LSM also distinguished VOD/SOS from other liver complications within +100 days post-HSCT in both adults and children. In conclusion, LSM is a reliable, non-invasive diagnostic tool for VOD/SOS. LSM contribute to differential diagnosis and to treatment response as well. This study underscores the potential of LSM, combined with multidisciplinary expertise, to guide VOD/SOS diagnosis and management in HSCT patients, improving potentially the clinical outcomes.
Ravaioli, F., Colecchia, A., Peccatori, J., Pagliara, D., Grassi, A., Barbato, F., et al. (2025). Diagnostic accuracy of liver stiffness measurement for the diagnosis of veno-occlusive disease/sinusoidal obstruction syndrome after hematopoietic stem cell transplantation (HSCT), the ELASTOVOD STUDY: an investigator-initiated, prospective, multicentre diagnostic clinical trial. BONE MARROW TRANSPLANTATION, 60(7), 978-993 [10.1038/s41409-025-02570-w].
Diagnostic accuracy of liver stiffness measurement for the diagnosis of veno-occlusive disease/sinusoidal obstruction syndrome after hematopoietic stem cell transplantation (HSCT), the ELASTOVOD STUDY: an investigator-initiated, prospective, multicentre diagnostic clinical trial
Ravaioli F.Primo
;Barbato F.;Masetti R.;Colecchia L.;Belotti T.;Alemanni L. V.;Ursi M.;Marasco G.;Roberto M.;Vestito A.;Dajti E.;Prete A.;Pession A.;Festi D.;Zama D;Zagari R. M.;Arpinati M.;
2025
Abstract
Hepatic Veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a severe complication following hematopoietic stem cell transplantation (HSCT), traditionally diagnosed based on clinical criteria. This study aimed to evaluate the diagnostic performance of liver stiffness measurement (LSM) as a non-invasive tool for non invasive diagnosis of VOD/SOS. A multicentre clinical trial was conducted in Italy from April 2018 to December 2021, screening 1089 patients across 25 centers. VOD/SOS diagnosis followed established clinical guidelines, and patients underwent comprehensive clinical, laboratory, and imaging evaluations up to +100 days post-HSCT or until VOD/SOS diagnosis. LSM was measured pre-HSCT and on specific post-transplant days (ClinicalTrials.gov: NCT03426358). The study enrolled 774 adults and 167 children. The +100-day incidence of VOD/SOS HSCT was 5.53 and 5.26 in the overall and allo-HSCT population, higher in children (14.3%) than in adults (3.68%). The 100-day overall survival (OS) probability was 89.5% (overall) and 89.0% (allo-HSCT) while one-yr OS 79% and 78%, respectively, with outcomes varying by VOD/SOS occurrence and severity. LSM significantly differed between VOD/SOS patients and non-affected individuals at all post-HSCT time points, correlating with disease severity. A diagnostic algorithm was proposed, achieving ≥95% sensitivity and specificity, with a 6 kPa rule-out and 25 kPa rule-in cut-off, enhanced by the “three-time pre-HSCT rule.” Survivors showed declining LSM over time, while non-survivors did not. Fully recovered patients had lower LSM than non-improvers. LSM also distinguished VOD/SOS from other liver complications within +100 days post-HSCT in both adults and children. In conclusion, LSM is a reliable, non-invasive diagnostic tool for VOD/SOS. LSM contribute to differential diagnosis and to treatment response as well. This study underscores the potential of LSM, combined with multidisciplinary expertise, to guide VOD/SOS diagnosis and management in HSCT patients, improving potentially the clinical outcomes.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
