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
Study Profile
- Design
- systematic review of 92 studies (2002-2023) on non-HCT SOS/VOD burden of illness and management
- Sample size (n)
- 0
- Intervention
- Defibrotide and other treatments for non-HCT-related sinusoidal obstruction syndrome/veno-occlusive disease
- Comparator
- Standard of care or natural history
- Primary endpoint
- Incidence, diagnosis, management, clinical and economic burden of non-HCT SOS/VOD
- Primary result
- 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 duration
- Variable across included studies (2002-2023 window)
- PMID
- 39640784
Key Findings
- 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
Limitations
- 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
Clinical Implications
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.
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