Background: The diagnosis of Veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS), a serious complication post-HSCT, relies primarily on clinical criteria. Our aim was to assess the diagnostic performance of liver stiffness measurement (LSM) for the non-invasive diagnosis of VOD/SOS. Methods: In Italy, a multicenter clinical trial was executed from April 2018 to December 2021, with 1089 screened patients across 25 centres. The diagnosis of VOD/SOS adhered to clinical guidelines, and patients underwent a thorough evaluation, including clinical, laboratory, and imaging assessments until VOD/SOS diagnosis or up to +100-days-post-HSCT. LSM measurements were taken pre-HSCT and on specific days post-transplant (ClinicalTrials.gov-number-NCT03426358). Findings: 774 adults and 167 children were enrolled in the study. The +100-day incidence of VOD/SOS was 5.53%, with significantly different rates between adults (3.62%) and children (14.37%). The 100-day and one-year overall survival rates were 89.5% and 79%, respectively, varying significantly according to VOD/SOS occurrence and severity. LSM analysis showed significant differences in all time points after HSCT between patients developing VOD and those who did not. The increase in LS was proportional to the disease severity as well. Then, a VOD/SOS diagnosis algorithm was proposed to achieve ≥95% sensibility and specificity, incorporating a 6 kPa rule-out cut-off and a 25 kPa rule-in cut-off, utilising the "three-time pre-HSCT rule" method for enhanced accuracy. Furthermore, VOD/SOS’survivors exhibited a reduction in LSM over time, while non-survivors did not, and fully recovered patients had notably lower LSM values than non-improvers. Noteworthy differences were also noted in LSM levels between VOD/SOS and other liver-related complications within +100 days after HSCT in both adult and children cohorts.
Sica, S., Diagnostic Accuracy of Liver Stiffness Measurement for the Diagnosis of Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome after Hematopoietic Stem Cell Transplantation (ELASTOVOD STUDY): An Italian Pragmatic Investigator-Initiated, Prospective, Multicentre Diagnostic Clinical Trial, <<BONE MARROW TRANSPLANTATION>>, 2025; (19): 1-3 [https://hdl.handle.net/10807/312625]
Diagnostic Accuracy of Liver Stiffness Measurement for the Diagnosis of Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome after Hematopoietic Stem Cell Transplantation (ELASTOVOD STUDY): An Italian Pragmatic Investigator-Initiated, Prospective, Multicentre Diagnostic Clinical Trial
Sica, Simona
Secondo
Membro del Collaboration Group
2025
Abstract
Background: The diagnosis of Veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS), a serious complication post-HSCT, relies primarily on clinical criteria. Our aim was to assess the diagnostic performance of liver stiffness measurement (LSM) for the non-invasive diagnosis of VOD/SOS. Methods: In Italy, a multicenter clinical trial was executed from April 2018 to December 2021, with 1089 screened patients across 25 centres. The diagnosis of VOD/SOS adhered to clinical guidelines, and patients underwent a thorough evaluation, including clinical, laboratory, and imaging assessments until VOD/SOS diagnosis or up to +100-days-post-HSCT. LSM measurements were taken pre-HSCT and on specific days post-transplant (ClinicalTrials.gov-number-NCT03426358). Findings: 774 adults and 167 children were enrolled in the study. The +100-day incidence of VOD/SOS was 5.53%, with significantly different rates between adults (3.62%) and children (14.37%). The 100-day and one-year overall survival rates were 89.5% and 79%, respectively, varying significantly according to VOD/SOS occurrence and severity. LSM analysis showed significant differences in all time points after HSCT between patients developing VOD and those who did not. The increase in LS was proportional to the disease severity as well. Then, a VOD/SOS diagnosis algorithm was proposed to achieve ≥95% sensibility and specificity, incorporating a 6 kPa rule-out cut-off and a 25 kPa rule-in cut-off, utilising the "three-time pre-HSCT rule" method for enhanced accuracy. Furthermore, VOD/SOS’survivors exhibited a reduction in LSM over time, while non-survivors did not, and fully recovered patients had notably lower LSM values than non-improvers. Noteworthy differences were also noted in LSM levels between VOD/SOS and other liver-related complications within +100 days after HSCT in both adult and children cohorts.
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