Burden of illness of non-hematopoietic stem cell transplant-related hepatic sinusoidal obstruction syndrome: A systematic review
Fan L, Stewart F, Ruiz K, Devani D, Fusco N, Gill M, Amber V, Su W, Gangi A, Hanvesakul R (2024) · Heliyon · n=0
Studienprofil
- Design
- systematic review of 92 studies (2002-2023) on non-HCT SOS/VOD burden of illness and management
- Stichprobengröße (n)
- —
- Intervention
- Defibrotide and other treatments for non-HCT-related sinusoidal obstruction syndrome/veno-occlusive disease
- Komparator
- Standard of care or natural history
- Primärer Endpunkt
- Incidence, diagnosis, management, clinical and economic burden of non-HCT SOS/VOD
- Primäres Ergebnis
- Non-HCT SOS/VOD occurs in ~35% of oxaliplatin-treated colorectal liver metastases (CRLM) patients and ~5% of acute leukemia patients; defibrotide most frequently reported treatment; significant unmet need for diagnostic and treatment guidelines
- Follow-up-Dauer
- Variable across included studies (2002-2023 window)
- PMID
- 39640784
Wichtigste Ergebnisse
- 35% non-HCT SOS/VOD incidence in oxaliplatin-treated CRLM patients
- 5% incidence in acute lymphoblastic and acute myeloid leukemia
- Defibrotide most frequently reported treatment
- Wide diagnostic criteria variability across disease settings
- Identifies unmet need for guidelines
Einschränkungen
- 57% retrospective cohort studies (low evidence quality)
- Industry-affiliated authorship (Jazz Pharmaceuticals)
- Excluded pulmonary VOD and pyrrolizidine alkaloid cases
- Methodological quality varied across included studies
- Not applicable to whole-leech hirudotherapy
Klinische Implikationen
Fan 2024 quantifies the substantial off-label use of defibrotide for non-HSCT VOD, particularly in oncology populations. For ASH, this systematic review illustrates how the pharmaceutical pathway expands beyond approved indications through clinical adoption rather than regulatory action - a phenomenon also visible in the K040187 device-leech US clinical practice but with different scale and economic implications.
Verwandte Studien
Defibrotide for prophylaxis of sinusoidal obstruction syndrome/veno-occlusive disease (SOS/VOD) in pediatric high-risk patients: consensus guidelines from the European Society for Blood and Marrow Transplantation (EBMT)
Corbacioglu S, Bajwa R, Antmen AB, Balduzzi A, Boelens JJ, Bonifazi F, Cesaro S, Ciceri F, Colecchia A, Dignan F, Kleinschmidt K, Mahadeo KM, Pagliuca A, Sedlacek P, Shaw PJ, Stein J, Szmit Z, Tambaro F, Ince E, Verna M, Yesilipek A, Zecca M, Richardson PG, Mohty M, Kalwak K (2026)
Hepatic Sinusoidal Obstruction Syndrome/Veno-Occlusive Disease (SOS/VOD) Primary Prophylaxis in Patients Undergoing Hematopoietic Stem Cell Transplantation: A Network Meta-Analysis of Randomized Controlled Trials
Sousa-Pimenta M, Martins Â, Estevinho LM, Pinho Vaz C, Leite L, Mariz J (2024)
Impact of Oral Eicosapentaenoic Acid on Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome Onset Prevention: Single-Center Retrospective Analysis
Miyazaki Y, Narumi H, Azuma T, Tanimoto K, Masuda Y, Kato J, Konishi T, Nabe S, Maruta M, Ochi T, Yamanouchi J, Takenaka K (2025)