Drug · FAERS side-effect profile with label screen
Defibrotide side effects: what 3,546 FDA adverse event reports show
Drug class
Not stated in label
Label matched
Defibrotide Sodium (Defitelio)
closest FDA label found for this product string; brand list suppressed because the label is a related product, not an exact match
Who reports
3,546 reports · 84% serious · 1,730 with death outcome · 39% female
openFDA FAERS counts, all years, deduplication not applied
Used for
DEFITELIO is indicated for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT).
FDA label, Indications and Usage
What it is
Defibrotide sodium is an oligonucleotide mixture with profibrinolytic properties.
FDA label, Description section
Boxed warning
None in the current label
How to read this page. FAERS is a spontaneous-reporting system: counts are not incidence rates and a signal is not proof of causation.
Each reaction below is run through an automated screen — is the term in the FDA label (known), in the indications (the disease being treated), an administrative/coding term, or none of these (unlabeled)?
That screen is mechanical text matching, not a causality assessment. The 30 hand-written Bradford Hill assessments are on the causality assessment page. Not medical advice.
Causality screen of the 40 strongest signals
Labeled adverse reaction 6Labeled (related wording) 3Likely indication confounding 1Reporting / coding artifact 4Lab value / disease marker 1Not in label — unassessed 25
What the badges mean
- Labeled adverse reaction The exact term appears in the label's warnings or adverse-reactions sections: a known, expected effect. The FAERS signal confirms the label rather than discovering anything.
- Labeled (related wording) The label describes this effect under different wording (e.g. 'neutropenia' for 'neutrophil count decreased'). Treated as known.
- Likely indication confounding The term appears in the label's indications: reports probably describe the disease being treated (or its progression), not a drug effect. Classic confounding by indication.
- Reporting / coding artifact An administrative, monitoring or exposure term (product issue, off-label use, 'test normal', overdose...). Not a biological reaction; a high PRR here reflects how reports are coded.
- Lab value / disease marker A laboratory result, investigation or disease-activity marker (antibody positive, CRP abnormal, swollen joint count) not described in the label. These usually mirror the monitored disease or testing protocol rather than a drug effect; not screened further.
- Not in label — unassessed Not found in the label text, not an artifact pattern. This is a genuine candidate for a formal causality assessment (Bradford Hill / WHO-UMC); it has NOT had one here.
Strongest disproportionality signals
Flagged terms (PRR ≥ 2, χ² ≥ 4, n ≥ 10), ranked by PRR; infinite PRRs (no comparator reports) excluded. The EBGM column is a second, shrinkage-based estimate (MGPS) with its 5th percentile EB05 and the BCPNN IC025; a pair flagged by PRR alone is marked PRR-only and should be read as fragile. The strata column recomputes the PRR on serious reports only and flags pairs whose reports are unusually skewed by sex or come mostly from consumers rather than clinicians. 37 further terms are in the interactive explorer. Reaction links open the term's cross-drug page.
Show:
| Reaction | Reports | PRR 95% CI · ROR · χ²/p | EBGM EB05 · IC025 | Label screen | Serious-only & strata | Trial cross-check |
|---|---|---|---|---|---|---|
| Multiple organ dysfunction syndrome | 343 | 73.6 66.5–81.4ROR 81.4 · χ² 24,213 / p <1e-300 | 72.4 EB05 66.2 · IC025 +5.9 3/3 methods agree | Labeled (related wording) related wording | serious PRR 49.6 | no trial data cached |
| Thrombotic microangiopathy | 53 | 32.5 24.9–42.5ROR 33.0 · χ² 1,580 / p <1e-300 | 32.0 EB05 25.4 · IC025 +4.2 3/3 methods agree | Not in label — unassessed | serious PRR 22.1 | no trial data cached |
| Acute myeloid leukaemia | 54 | 20.6 15.8–26.8ROR 20.9 · χ² 983 / p 4e-216 | 20.4 EB05 16.2 · IC025 +3.7 3/3 methods agree | Not in label — unassessed | serious PRR 14.1 | no trial data cached |
| Acute respiratory distress syndrome | 61 | 20.5 15.9–26.2ROR 20.8 · χ² 1,106 / p 7e-243 | 20.3 EB05 16.3 · IC025 +3.7 3/3 methods agree | Not in label — unassessed | serious PRR 14.0 · ♀ 83% (drug 39%) | no trial data cached |
| Posterior reversible encephalopathy syndrome | 24 | 14.9 10.0–22.3ROR 15.0 · χ² 298 / p 5e-67 | 14.5 EB05 9.9 · IC025 +2.9 3/3 methods agree | Not in label — unassessed | serious PRR 10.2 | no trial data cached |
| Septic shock | 104 | 14.3 11.8–17.2ROR 14.7 · χ² 1,269 / p 3e-278 | 14.2 EB05 12.0 · IC025 +3.4 3/3 methods agree | Not in label — unassessed | serious PRR 9.7 | no trial data cached |
| Melaena (black stool) | 50 | 13.4 10.2–17.7ROR 13.6 · χ² 562 / p 1e-124 | 13.2 EB05 10.3 · IC025 +3.1 3/3 methods agree | Not in label — unassessed | serious PRR 8.8 | no trial data cached |
| Intentional product use issue | 198 | 13.3 11.7–15.3ROR 14.1 · χ² 2,252 / p <1e-300 | 13.3 EB05 11.8 · IC025 +3.5 3/3 methods agree | Reporting / coding artifact | serious PRR 12.4 · 78% consumer | no trial data cached |
| Cytomegalovirus infection | 33 | 11.8 8.4–16.5ROR 11.9 · χ² 314 / p 2e-70 | 11.2 EB05 8.1 · IC025 +2.8 3/3 methods agree | Not in label — unassessed | serious PRR 8.1 | no trial data cached |
| Haematuria (blood in urine) | 64 | 11.1 8.7–14.1ROR 11.2 · χ² 575 / p 2e-127 | 10.8 EB05 8.6 · IC025 +2.9 3/3 methods agree | Not in label — unassessed | serious PRR 6.9 | no trial data cached |
| Bacteraemia | 20 | 10.1 6.5–15.6ROR 10.1 · χ² 154 / p 1e-35 | 9.0 EB05 5.9 · IC025 +2.3 3/3 methods agree | Not in label — unassessed | serious PRR 6.9 | no trial data cached |
| Myelodysplastic syndrome | 24 | 9.8 6.6–14.6ROR 9.9 · χ² 181 / p 1e-41 | 8.9 EB05 6.1 · IC025 +2.4 3/3 methods agree | Not in label — unassessed | serious PRR 6.8 | no trial data cached |
| Blood bilirubin increased | 42 | 9.2 6.8–12.4ROR 9.3 · χ² 298 / p 4e-67 | 8.7 EB05 6.6 · IC025 +2.6 3/3 methods agree | Lab value / disease marker | serious PRR 6.7 | no trial data cached |
| Upper gastrointestinal haemorrhage | 26 | 9.1 6.2–13.4ROR 9.2 · χ² 180 / p 3e-41 | 8.3 EB05 5.8 · IC025 +2.3 3/3 methods agree | Labeled (related wording) related wording | serious PRR 6.2 · 92% clinician | no trial data cached |
| Haemorrhage | 155 | 8.8 7.5–10.2ROR 9.1 · χ² 1,064 / p 9e-234 | 8.6 EB05 7.5 · IC025 +2.8 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 6.9 | no trial data cached |
| Gastrointestinal haemorrhage | 123 | 8.7 7.3–10.3ROR 8.9 · χ² 828 / p 2e-182 | 8.5 EB05 7.3 · IC025 +2.8 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 5.9 | no trial data cached |
| Off label use (used outside approved label) | 1,236 | 8.4 8.1–8.8ROR 12.4 · χ² 8,427 / p <1e-300 | 8.4 EB05 8.0 · IC025 +3.0 3/3 methods agree | Reporting / coding artifact | serious PRR 7.7 | no trial data cached |
| Hepatic encephalopathy | 13 | 8.2 4.8–14.2ROR 8.3 · χ² 76 / p 2e-18 | 6.8 EB05 4.1 · IC025 +1.8 3/3 methods agree | Not in label — unassessed | serious PRR 5.6 | no trial data cached |
| Subarachnoid haemorrhage | 14 | 8.1 4.8–13.7ROR 8.1 · χ² 80 / p 2e-19 | 6.8 EB05 4.2 · IC025 +1.8 3/3 methods agree | Not in label — unassessed | serious PRR 5.5 | no trial data cached |
| Hepatic failure | 40 | 7.8 5.8–10.7ROR 7.9 · χ² 232 / p 1e-52 | 7.3 EB05 5.6 · IC025 +2.3 3/3 methods agree | Labeled (related wording) related wording | serious PRR 5.4 | no trial data cached |
| Subdural haematoma | 19 | 7.8 5.0–12.3ROR 7.9 · χ² 106 / p 3e-25 | 6.8 EB05 4.5 · IC025 +2.0 3/3 methods agree | Likely indication confounding shares a disease stem with the indications wording | serious PRR 5.1 | no trial data cached |
| Lymphoma | 20 | 7.6 4.9–11.8ROR 7.7 · χ² 109 / p 9e-26 | 6.7 EB05 4.5 · IC025 +2.0 3/3 methods agree | Not in label — unassessed | serious PRR 5.3 | no trial data cached |
| Interstitial lung disease | 62 | 7.6 5.9–9.7ROR 7.7 · χ² 349 / p 4e-78 | 7.3 EB05 5.8 · IC025 +2.4 3/3 methods agree | Not in label — unassessed | serious PRR 5.2 · 90% clinician | no trial data cached |
| Ascites | 37 | 7.4 5.4–10.3ROR 7.5 · χ² 200 / p 1e-45 | 6.9 EB05 5.2 · IC025 +2.2 3/3 methods agree | Not in label — unassessed | serious PRR 5.0 · 97% clinician | no trial data cached |
| Incorrect product administration duration | 25 | 7.3 4.9–10.8ROR 7.3 · χ² 129 / p 3e-30 | 6.6 EB05 4.6 · IC025 +2.0 3/3 methods agree | Reporting / coding artifact | serious n=20 | no trial data cached |
| Cerebral haemorrhage | 44 | 7.3 5.4–9.8ROR 7.4 · χ² 232 / p 9e-53 | 6.8 EB05 5.3 · IC025 +2.3 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 4.9 · 93% clinician | no trial data cached |
| Altered state of consciousness | 25 | 6.9 4.6–10.2ROR 6.9 · χ² 119 / p 4e-28 | 6.2 EB05 4.4 · IC025 +2.0 3/3 methods agree | Not in label — unassessed | serious PRR 4.7 · ♀ 14% (drug 39%) · 96% clinician | no trial data cached |
| Petechiae | 12 | 6.8 3.9–12.0ROR 6.8 · χ² 54 / p 1e-13 | 5.6 EB05 3.4 · IC025 +1.5 3/3 methods agree | Not in label — unassessed | serious PRR 5.6 | no trial data cached |
| Clostridium difficile colitis | 12 | 6.7 3.8–11.8ROR 6.7 · χ² 52 / p 2e-13 | 5.5 EB05 3.3 · IC025 +1.5 3/3 methods agree | Not in label — unassessed | serious PRR 4.7 | no trial data cached |
| Gastric haemorrhage | 13 | 6.6 3.9–11.4ROR 6.7 · χ² 57 / p 2e-14 | 5.5 EB05 3.4 · IC025 +1.5 3/3 methods agree | Not in label — unassessed | serious PRR 4.3 | no trial data cached |
| Hepatomegaly | 11 | 6.6 3.7–11.9ROR 6.6 · χ² 47 / p 4e-12 | 5.3 EB05 3.2 · IC025 +1.4 3/3 methods agree | Not in label — unassessed | serious PRR 4.9 | no trial data cached |
| Sepsis (blood infection) | 121 | 6.4 5.4–7.6ROR 6.6 · χ² 550 / p 5e-122 | 6.3 EB05 5.4 · IC025 +2.3 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 4.4 | no trial data cached |
| Coagulopathy | 17 | 6.1 3.8–9.7ROR 6.1 · χ² 67 / p 2e-16 | 5.3 EB05 3.5 · IC025 +1.6 3/3 methods agree | Not in label — unassessed | serious PRR 4.3 | no trial data cached |
| Nephropathy toxic | 11 | 5.8 3.2–10.5ROR 5.9 · χ² 39 / p 2e-10 | 4.8 EB05 2.9 · IC025 +1.2 3/3 methods agree | Not in label — unassessed | serious PRR 4.1 | no trial data cached |
| Respiratory distress | 25 | 5.4 3.6–7.9ROR 5.4 · χ² 84 / p 2e-20 | 4.9 EB05 3.5 · IC025 +1.6 3/3 methods agree | Not in label — unassessed | serious PRR 3.7 | no trial data cached |
| Candida infection (yeast infection) | 15 | 5.3 3.2–8.8ROR 5.3 · χ² 48 / p 2e-12 | 4.6 EB05 3.0 · IC025 +1.4 3/3 methods agree | Not in label — unassessed | serious PRR 5.2 | no trial data cached |
| Epistaxis (nosebleed) | 64 | 5.1 4.0–6.4ROR 5.1 · χ² 205 / p 1e-46 | 4.9 EB05 4.0 · IC025 +1.9 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 4.3 | no trial data cached |
| Respiratory failure | 62 | 5.0 3.9–6.4ROR 5.1 · χ² 197 / p 6e-45 | 4.9 EB05 3.9 · IC025 +1.9 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 3.4 | no trial data cached |
| Disease progression | 98 | 4.9 4.0–6.0ROR 5.0 · χ² 303 / p 3e-68 | 4.8 EB05 4.1 · IC025 +1.9 3/3 methods agree | Reporting / coding artifact | serious PRR 3.9 | no trial data cached |
| Haematemesis (vomiting blood) | 21 | 4.9 3.2–7.5ROR 4.9 · χ² 61 / p 3e-15 | 4.4 EB05 3.1 · IC025 +1.4 3/3 methods agree | Not in label — unassessed | serious PRR 3.3 | no trial data cached |
Trial evidence (ClinicalTrials.gov)
Trial cross-check not yet run for this drug (ClinicalTrials.gov pull covers the 800 most-reported drugs first).
Most frequently reported reactions
By raw co-report count, regardless of disproportionality. Common terms here are often the illness itself or generic outcomes.
| Reaction | Reports | PRR 95% CI · ROR · χ²/p | EBGM EB05 · IC025 | Label screen | Serious-only & strata | Trial cross-check |
|---|---|---|---|---|---|---|
| Off label use (used outside approved label) | 1,236 | 8.4 8.1–8.8ROR 12.4 · χ² 8,427 / p <1e-300 | 8.4 EB05 8.0 · IC025 +3.0 3/3 methods agree | Reporting / coding artifact | serious PRR 7.7 | no trial data cached |
| Death | 458 | 3.1 2.9–3.4ROR 3.5 · χ² 697 / p 9e-154 | 3.1 EB05 2.9 · IC025 +1.5 3/3 methods agree | Outcome field, not a reaction | serious PRR 2.2 | no trial data cached |
| Multiple organ dysfunction syndrome | 343 | 73.6 66.5–81.4ROR 81.4 · χ² 24,213 / p <1e-300 | 72.4 EB05 66.2 · IC025 +5.9 3/3 methods agree | Labeled (related wording) related wording | serious PRR 49.6 | no trial data cached |
| Intentional product use issue | 198 | 13.3 11.7–15.3ROR 14.1 · χ² 2,252 / p <1e-300 | 13.3 EB05 11.8 · IC025 +3.5 3/3 methods agree | Reporting / coding artifact | serious PRR 12.4 · 78% consumer | no trial data cached |
| Hypotension (low blood pressure) | 159 | 4.7 4.0–5.5ROR 4.9 · χ² 463 / p 5e-103 | 4.6 EB05 4.1 · IC025 +2.0 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 3.7 | no trial data cached |
| Haemorrhage | 155 | 8.8 7.5–10.2ROR 9.1 · χ² 1,064 / p 9e-234 | 8.6 EB05 7.5 · IC025 +2.8 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 6.9 | no trial data cached |
| Gastrointestinal haemorrhage | 123 | 8.7 7.3–10.3ROR 8.9 · χ² 828 / p 2e-182 | 8.5 EB05 7.3 · IC025 +2.8 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 5.9 | no trial data cached |
| Sepsis (blood infection) | 121 | 6.4 5.4–7.6ROR 6.6 · χ² 550 / p 5e-122 | 6.3 EB05 5.4 · IC025 +2.3 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 4.4 | no trial data cached |
| Septic shock | 104 | 14.3 11.8–17.2ROR 14.7 · χ² 1,269 / p 3e-278 | 14.2 EB05 12.0 · IC025 +3.4 3/3 methods agree | Not in label — unassessed | serious PRR 9.7 | no trial data cached |
| Renal failure (kidney failure) | 103 | 4.6 3.8–5.5ROR 4.7 · χ² 284 / p 5e-64 | 4.5 EB05 3.8 · IC025 +1.8 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 3.1 | no trial data cached |
| Disease progression | 98 | 4.9 4.0–6.0ROR 5.0 · χ² 303 / p 3e-68 | 4.8 EB05 4.1 · IC025 +1.9 3/3 methods agree | Reporting / coding artifact | serious PRR 3.9 | no trial data cached |
| Thrombocytopenia (low platelets) | 66 | 3.5 2.8–4.4ROR 3.5 · χ² 116 / p 2e-27 | 3.4 EB05 2.8 · IC025 +1.4 3/3 methods agree | Not in label — unassessed | serious PRR 2.5 · ♀ 59% (drug 39%) | no trial data cached |
| Epistaxis (nosebleed) | 64 | 5.1 4.0–6.4ROR 5.1 · χ² 205 / p 1e-46 | 4.9 EB05 4.0 · IC025 +1.9 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 4.3 | no trial data cached |
| Haematuria (blood in urine) | 64 | 11.1 8.7–14.1ROR 11.2 · χ² 575 / p 2e-127 | 10.8 EB05 8.6 · IC025 +2.9 3/3 methods agree | Not in label — unassessed | serious PRR 6.9 | no trial data cached |
| Respiratory failure | 62 | 5.0 3.9–6.4ROR 5.1 · χ² 197 / p 6e-45 | 4.9 EB05 3.9 · IC025 +1.9 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 3.4 | no trial data cached |
Unlabeled candidate signals
These flagged terms were not found in the FDA label text and do not match an artifact pattern. They are the pairs that would merit a structured assessment (temporality, dose-response, biological plausibility, consistency across databases, trial or cohort evidence). The screen has not done that; string-matching misses label wording, and a strong PRR can still be stimulated reporting, co-medication, or the disease itself.
- Thrombotic microangiopathy — n=53, PRR 32.5
- Acute myeloid leukaemia — n=54, PRR 20.6
- Acute respiratory distress syndrome — n=61, PRR 20.5
- Posterior reversible encephalopathy syndrome — n=24, PRR 14.9
- Septic shock — n=104, PRR 14.3
- Melaena — n=50, PRR 13.4
- Cytomegalovirus infection — n=33, PRR 11.8
- Haematuria — n=64, PRR 11.1
- Bacteraemia — n=20, PRR 10.1
- Myelodysplastic syndrome — n=24, PRR 9.8
Want to assess one of these properly? The runbook describes the Bradford Hill / trial-evidence workflow used on the assessment page, and handoff_top1000_signals.csv lists the strongest unassessed pairs across all drugs.
Common questions
What are the most reported side effects of Defibrotide?
In FDA FAERS, the most frequently co-reported reaction terms for Defibrotide are multiple organ dysfunction syndrome, hypotension, haemorrhage, gastrointestinal haemorrhage, sepsis, septic shock. These are report counts, not rates: FAERS has no denominator, reporting is voluntary and uneven, and a term can be reported because of the illness being treated rather than the drug.
Does Defibrotide have an FDA boxed warning?
No. The FDA label retrieved for Defibrotide has no boxed warning; see the label's Warnings and Precautions for the recognised risks.
Do FAERS signals prove that Defibrotide causes these side effects?
No. A disproportionality signal (PRR, ROR) means a reaction is reported more often with this drug than with others in the database. That can reflect a real effect, but also the disease being treated, stimulated reporting after news or litigation, co-medications, or how reports are coded. Causality needs temporality, dose-response, biological plausibility, consistency across data sources and, ideally, trial or cohort evidence. This page screens each term against the FDA label and ClinicalTrials.gov results; it does not establish causation.
Sources & methods
- FDA label via openFDA (set ID 2c3db989-d7ad-41ed-9ebf-698dcf6c24ec, effective 20250701; matched on
openfda.generic_name.exact:"DEFIBROTIDE SODIUM") - Bayesian shrinkage measures computed offline over all 1,317,300 cached pairs: BCPNN information component (IC, with Norén closed-form IC025) and MGPS empirical-Bayes geometric mean (EBGM, EB05) with a two-component gamma prior fitted by maximum marginal likelihood (α₁=0.57, β₁=0.02, α₂=1.42, β₂=0.70, w=0.17); unstratified
- FAERS pair statistics from the OpenPV build of 2026-09-14 (openFDA
count=aggregation, fieldpatient.drug.openfda.generic_name.exact, 77 reaction terms, min 3 co-reports); PRR/ROR with 95% CI, chi-square with Yates correction - Serious-only and stratified counts (serious:1, patient.patientsex, primarysource.qualification) from openFDA count queries; serious-only PRR uses the drug serious report total and the term global serious total (N_serious=11,882,979); strata shown only when n ≥ 20
- Seriousness and sex counts from openFDA
count=serious,patient.patientsex,seriousnessdeath
Page generated 2026-10-08. Label and PubChem text are retrieved automatically and may lag the current label; always check the current prescribing information. OpenPV is a beta research tool of the Open Source Medicine Foundation.