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.
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 / F. Ravaioli, A. Colecchia, J. Peccatori, D. Pagliara, A. Grassi, F. Barbato, R. Masetti, B. Sarina, S. Sica, S. Cesaro, C. Nozzoli, G.M. Assanto, L. Prezioso, S. Santarone, F. Saglio, E. Vanni, A. Olivieri, M. Delia, E. Benedetti, F. Zallio, F. Pane, C. Skert, M. Menconi, F. Benedetti, F. De Felice, L. Colecchia, T. Belotti, L.V. Alemanni, M. Ursi, G. Marasco, M. Roberto, A. Vestito, E. Dajti, M. Garcovich, S. Bramanti, D. Taurino, F. Quagliarella, F. Ciceri, A. Prete, A. Pession, D. Festi, N. Null, M. Arpinati, E. Maffini, S. Falcione, R.M. Zagari, Z. Daniele, L. Lazzari, A. Rambaldi, L. Riccardi, V.D. Ruscio, E. Boccieri, M. Falcomer, V. Chiara, V. Vitale, C. Garonzi, F. Vizzutti, U. Arena, A.D. Santis, A. Rinaldi, A. Olivani, D. Vaddinelli, E.D. Lembo, M. Spadea, V. Apolito, D. Chiara, A. Busca, G. Mancini, G. Argalia, P. Carluccio, F. Passerini, A.D. Gangi, P. Rivela, R. Carbone, G. Battipaglia, F. Carobolante, I. Franceschet, F. Patriarca, M. Malagola, R. Varaldo, F. Onida, P. Vincenzo, F. Bonifazi, F. Bonifazi. - In: BONE MARROW TRANSPLANTATION. - ISSN 0268-3369. - (2025). [Epub ahead of print] [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
F. Benedetti;S. Bramanti;D. Taurino;A. Rambaldi;F. Onida;
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.
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