Mitoxantrone side effects: what 5,310 FDA adverse event reports show
Drug class
Not stated in label
Who reports
5,310 reports · 97% serious · 1,441 with death outcome · 47% female
Used for
Mitoxantrone injection, USP (concentrate) is indicated for reducing neurologic disability and/or the frequency of clinical relapses in patients with secondary (chronic) progressive, progressive relapsing, or worsening relapsing-remitting multiple sclerosis (i.e., patients whose neurologic status is significantly abnormal between relapses).
What it is
DESCRIPTION Mitoxantrone injection, USP (concentrate) is a synthetic antineoplastic anthracenedione for intravenous use. It is supplied as a concentrate that MUST BE DILUTED PRIOR TO INJECTION.
Boxed warning
Mitoxantrone injection (concentrate) should be administered under the supervision of a physician experienced in the use of cytotoxic chemotherapy agents. Mitoxantrone injection (concentrate) should be given slowly into a freely flowing intravenous infusion. It must never be given subcutaneously, intramuscularly, or intra-arterially.
Causality screen of the 40 strongest signals
What the badges mean
- Labeled — boxed warning The term (or its clinical synonym) appears in the FDA boxed warning: causality is regulator-recognised and considered serious.
- 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.
- 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. 187 further terms are in the interactive explorer. Reaction links open the term's cross-drug page.
| Reaction | Reports | PRR 95% CI · ROR · χ²/p | EBGM EB05 · IC025 | Label screen | Serious-only & strata | Trial cross-check |
|---|---|---|---|---|---|---|
| Acute myeloid leukaemia | 275 | 71.0 63.2–79.7ROR 74.8 · χ² 18,582 / p <1e-300 | 69.4 EB05 62.8 · IC025 +5.8 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 41.2 · 96% clinician | no trial data cached |
| Myelodysplastic syndrome | 220 | 60.9 53.5–69.4ROR 63.5 · χ² 12,721 / p <1e-300 | 59.7 EB05 53.4 · IC025 +5.5 3/3 methods agree | Not in label — unassessed | serious PRR 35.6 · 94% clinician | no trial data cached |
| Febrile neutropenia | 591 | 35.6 32.9–38.4ROR 39.9 · χ² 19,695 / p <1e-300 | 35.2 EB05 32.9 · IC025 +5.0 3/3 methods agree | Labeled — boxed warning synonym match | serious PRR 20.9 · 95% clinician | no trial data cached |
| Haematotoxicity | 82 | 35.2 28.4–43.7ROR 35.8 · χ² 2,670 / p <1e-300 | 34.7 EB05 28.8 · IC025 +4.5 3/3 methods agree | Not in label — unassessed | serious PRR 21.2 · 93% clinician | no trial data cached |
| Bone marrow failure | 122 | 23.7 19.9–28.2ROR 24.2 · χ² 2,615 / p <1e-300 | 23.5 EB05 20.2 · IC025 +4.1 3/3 methods agree | Labeled (related wording) related wording | serious PRR 13.9 · 97% clinician | no trial data cached |
| Bacteraemia | 54 | 18.2 13.9–23.7ROR 18.4 · χ² 857 / p 1e-188 | 18.0 EB05 14.3 · IC025 +3.5 3/3 methods agree | Not in label — unassessed | serious PRR 10.8 · 96% clinician | no trial data cached |
| Mucosal inflammation | 116 | 17.7 14.7–21.1ROR 18.0 · χ² 1,800 / p <1e-300 | 17.5 EB05 15.0 · IC025 +3.7 3/3 methods agree | Labeled (related wording) related wording | serious PRR 10.9 · 90% clinician | no trial data cached |
| Pancytopenia (all blood cells low) | 203 | 14.4 12.6–16.5ROR 15.0 · χ² 2,522 / p <1e-300 | 14.4 EB05 12.8 · IC025 +3.6 3/3 methods agree | Labeled (related wording) related wording | serious PRR 8.6 · 93% clinician | no trial data cached |
| Septic shock | 151 | 13.8 11.8–16.2ROR 14.2 · χ² 1,784 / p <1e-300 | 13.7 EB05 12.0 · IC025 +3.5 3/3 methods agree | Not in label — unassessed | serious PRR 8.1 · 99% clinician | no trial data cached |
| Cardiomyopathy | 49 | 13.1 9.9–17.3ROR 13.2 · χ² 535 / p 1e-118 | 12.8 EB05 10.0 · IC025 +3.1 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 7.8 · 93% clinician | no trial data cached |
| Cytopenia | 39 | 12.9 9.4–17.7ROR 13.0 · χ² 416 / p 8e-93 | 12.6 EB05 9.4 · IC025 +3.0 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 7.9 · 100% clinician | no trial data cached |
| Multiple organ dysfunction syndrome | 86 | 12.2 9.9–15.1ROR 12.4 · χ² 872 / p 6e-192 | 12.0 EB05 10.0 · IC025 +3.2 3/3 methods agree | Labeled (related wording) related wording | serious PRR 7.2 · 95% clinician | no trial data cached |
| Neutropenia (low neutrophils (infection-fighting cells)) | 392 | 11.5 10.4–12.6ROR 12.3 · χ² 3,744 / p <1e-300 | 11.4 EB05 10.5 · IC025 +3.3 3/3 methods agree | Labeled — boxed warning exact term | serious PRR 7.0 · 95% clinician | no trial data cached |
| Thrombocytopenia (low platelets) | 312 | 11.1 9.9–12.3ROR 11.7 · χ² 2,857 / p <1e-300 | 11.0 EB05 10.0 · IC025 +3.3 3/3 methods agree | Not in label — unassessed | serious PRR 6.7 · 95% clinician | no trial data cached |
| Drug resistance | 71 | 10.9 8.7–13.8ROR 11.1 · χ² 631 / p 2e-139 | 10.7 EB05 8.7 · IC025 +3.0 3/3 methods agree | Reporting / coding artifact | serious PRR 7.0 · 99% clinician | no trial data cached |
| Hyperbilirubinaemia | 28 | 10.8 7.4–15.6ROR 10.8 · χ² 238 / p 6e-54 | 10.1 EB05 7.0 · IC025 +2.6 3/3 methods agree | Not in label — unassessed | serious PRR 6.5 · 100% clinician | no trial data cached |
| Lymphoma | 42 | 10.7 7.9–14.5ROR 10.8 · χ² 360 / p 1e-80 | 10.3 EB05 7.8 · IC025 +2.8 3/3 methods agree | Not in label — unassessed | serious PRR 6.4 · ♀ 9% (drug 47%) · 98% clinician | no trial data cached |
| Cytomegalovirus infection | 44 | 10.5 7.8–14.1ROR 10.5 · χ² 367 / p 5e-82 | 10.0 EB05 7.6 · IC025 +2.7 3/3 methods agree | Not in label — unassessed | serious PRR 6.1 · 95% clinician | no trial data cached |
| Ejection fraction decreased | 40 | 10.2 7.5–13.9ROR 10.2 · χ² 321 / p 4e-72 | 9.7 EB05 7.2 · IC025 +2.7 3/3 methods agree | Lab value / disease marker | serious PRR 6.6 · 94% clinician | no trial data cached |
| Candida infection (yeast infection) | 42 | 10.0 7.4–13.5ROR 10.0 · χ² 329 / p 7e-74 | 9.5 EB05 7.2 · IC025 +2.7 3/3 methods agree | Not in label — unassessed | serious PRR 8.4 · 98% clinician | no trial data cached |
| Disseminated intravascular coagulation | 35 | 9.8 7.0–13.7ROR 9.9 · χ² 268 / p 2e-60 | 9.2 EB05 6.8 · IC025 +2.6 3/3 methods agree | Not in label — unassessed | serious PRR 5.8 · 100% clinician | no trial data cached |
| Cytokine release syndrome | 46 | 9.6 7.2–12.7ROR 9.6 · χ² 343 / p 6e-77 | 9.1 EB05 7.0 · IC025 +2.6 3/3 methods agree | Not in label — unassessed | serious PRR 5.7 · 98% clinician | no trial data cached |
| Hepatotoxicity (liver damage) | 53 | 9.5 7.2–12.4ROR 9.5 · χ² 392 / p 1e-87 | 9.1 EB05 7.1 · IC025 +2.7 3/3 methods agree | Not in label — unassessed | serious PRR 5.7 · 98% clinician | no trial data cached |
| Sepsis (blood infection) | 265 | 9.4 8.4–10.6ROR 9.8 · χ² 1,986 / p <1e-300 | 9.3 EB05 8.4 · IC025 +3.0 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 5.6 · 96% clinician | no trial data cached |
| Lymphopenia | 33 | 9.3 6.6–13.0ROR 9.3 · χ² 235 / p 2e-53 | 8.6 EB05 6.3 · IC025 +2.5 3/3 methods agree | Not in label — unassessed | serious PRR 5.9 · 94% clinician | no trial data cached |
| Leukaemia | 22 | 9.2 6.1–14.0ROR 9.3 · χ² 153 / p 2e-35 | 8.3 EB05 5.6 · IC025 +2.2 3/3 methods agree | Labeled — boxed warning exact term | serious PRR 5.5 | no trial data cached |
| Therapy partial responder | 34 | 8.9 6.4–12.5ROR 9.0 · χ² 231 / p 2e-52 | 8.3 EB05 6.1 · IC025 +2.4 3/3 methods agree | Reporting / coding artifact | serious PRR 6.4 · ♀ 17% (drug 47%) · 100% clinician | no trial data cached |
| Neurotoxicity | 39 | 8.9 6.5–12.1ROR 8.9 · χ² 265 / p 9e-60 | 8.3 EB05 6.3 · IC025 +2.5 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 5.3 · 95% clinician | no trial data cached |
| Leukopenia (low white blood cells) | 109 | 8.6 7.2–10.4ROR 8.8 · χ² 729 / p 6e-161 | 8.4 EB05 7.2 · IC025 +2.7 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 5.3 · 94% clinician | no trial data cached |
| Clostridium difficile colitis | 23 | 8.6 5.7–12.9ROR 8.6 · χ² 146 / p 6e-34 | 7.7 EB05 5.2 · IC025 +2.2 3/3 methods agree | Not in label — unassessed | serious PRR 5.2 · ♀ 14% (drug 47%) · 91% clinician | no trial data cached |
| Fungal infection | 72 | 8.6 6.8–10.8ROR 8.7 · χ² 473 / p 4e-105 | 8.2 EB05 6.7 · IC025 +2.6 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 9.2 · 91% clinician | no trial data cached |
| Appendicitis | 20 | 8.5 5.5–13.2ROR 8.5 · χ² 125 / p 3e-29 | 7.5 EB05 5.0 · IC025 +2.1 3/3 methods agree | Not in label — unassessed | serious PRR 5.1 · 95% clinician | no trial data cached |
| Madarosis | 23 | 7.8 5.2–11.7ROR 7.8 · χ² 129 / p 4e-30 | 6.9 EB05 4.8 · IC025 +2.1 3/3 methods agree | Not in label — unassessed | ♀ 100% (drug 47%) | no trial data cached |
| Metastases to bone | 32 | 7.7 5.5–10.9ROR 7.8 · χ² 181 / p 2e-41 | 7.1 EB05 5.2 · IC025 +2.2 3/3 methods agree | Not in label — unassessed | serious PRR 4.6 | no trial data cached |
| Myelosuppression | 66 | 7.7 6.0–9.8ROR 7.8 · χ² 376 / p 4e-84 | 7.4 EB05 6.0 · IC025 +2.5 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 4.5 · 95% clinician | no trial data cached |
| Hair colour changes | 24 | 7.5 5.1–11.3ROR 7.6 · χ² 130 / p 2e-30 | 6.8 EB05 4.7 · IC025 +2.0 3/3 methods agree | Not in label — unassessed | ♀ 100% (drug 47%) | no trial data cached |
| Acute respiratory distress syndrome | 33 | 7.4 5.2–10.4ROR 7.4 · χ² 175 / p 2e-40 | 6.8 EB05 5.0 · IC025 +2.2 3/3 methods agree | Not in label — unassessed | serious PRR 4.4 · ♀ 72% (drug 47%) · 94% clinician | no trial data cached |
| Encephalopathy | 45 | 7.3 5.4–9.7ROR 7.3 · χ² 237 / p 1e-53 | 6.8 EB05 5.3 · IC025 +2.3 3/3 methods agree | Not in label — unassessed | serious PRR 4.3 | no trial data cached |
| Metastases to liver | 32 | 7.2 5.1–10.2ROR 7.3 · χ² 165 / p 4e-38 | 6.6 EB05 4.9 · IC025 +2.1 3/3 methods agree | Not in label — unassessed | serious PRR 4.3 | no trial data cached |
| Multi-organ failure | 32 | 7.2 5.1–10.2ROR 7.3 · χ² 165 / p 5e-38 | 6.6 EB05 4.9 · IC025 +2.1 3/3 methods agree | Not in label — unassessed | serious PRR 4.3 | no trial data cached |
Trial evidence (ClinicalTrials.gov)
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 |
|---|---|---|---|---|---|---|
| Febrile neutropenia | 591 | 35.6 32.9–38.4ROR 39.9 · χ² 19,695 / p <1e-300 | 35.2 EB05 32.9 · IC025 +5.0 3/3 methods agree | Labeled — boxed warning synonym match | serious PRR 20.9 · 95% clinician | no trial data cached |
| Off label use (used outside approved label) | 462 | 2.1 1.9–2.3ROR 2.2 · χ² 277 / p 1e-62 | 2.1 EB05 1.9 · IC025 +0.9 | Reporting / coding artifact | serious PRR 2.3 · 97% clinician | no trial data cached |
| Neutropenia (low neutrophils (infection-fighting cells)) | 392 | 11.5 10.4–12.6ROR 12.3 · χ² 3,744 / p <1e-300 | 11.4 EB05 10.5 · IC025 +3.3 3/3 methods agree | Labeled — boxed warning exact term | serious PRR 7.0 · 95% clinician | no trial data cached |
| Thrombocytopenia (low platelets) | 312 | 11.1 9.9–12.3ROR 11.7 · χ² 2,857 / p <1e-300 | 11.0 EB05 10.0 · IC025 +3.3 3/3 methods agree | Not in label — unassessed | serious PRR 6.7 · 95% clinician | no trial data cached |
| Pyrexia (fever) | 283 | 3.2 2.9–3.6ROR 3.4 · χ² 440 / p 5e-98 | 3.2 EB05 2.9 · IC025 +1.5 3/3 methods agree | Labeled (related wording) related wording | serious PRR 2.4 · 94% clinician | no trial data cached |
| Acute myeloid leukaemia | 275 | 71.0 63.2–79.7ROR 74.8 · χ² 18,582 / p <1e-300 | 69.4 EB05 62.8 · IC025 +5.8 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 41.2 · 96% clinician | no trial data cached |
| Sepsis (blood infection) | 265 | 9.4 8.4–10.6ROR 9.8 · χ² 1,986 / p <1e-300 | 9.3 EB05 8.4 · IC025 +3.0 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 5.6 · 96% clinician | no trial data cached |
| Myelodysplastic syndrome | 220 | 60.9 53.5–69.4ROR 63.5 · χ² 12,721 / p <1e-300 | 59.7 EB05 53.4 · IC025 +5.5 3/3 methods agree | Not in label — unassessed | serious PRR 35.6 · 94% clinician | no trial data cached |
| Pneumonia (lung infection) | 216 | 2.6 2.3–3.0ROR 2.7 · χ² 222 / p 2e-50 | 2.6 EB05 2.3 · IC025 +1.2 3/3 methods agree | Labeled — boxed warning synonym match | serious PRR 1.6 | no trial data cached |
| Disease progression | 203 | 6.8 5.9–7.8ROR 7.0 · χ² 1,004 / p 1e-220 | 6.7 EB05 6.0 · IC025 +2.5 3/3 methods agree | Reporting / coding artifact | serious PRR 4.5 · 99% clinician | no trial data cached |
| Pancytopenia (all blood cells low) | 203 | 14.4 12.6–16.5ROR 15.0 · χ² 2,522 / p <1e-300 | 14.4 EB05 12.8 · IC025 +3.6 3/3 methods agree | Labeled (related wording) related wording | serious PRR 8.6 · 93% clinician | no trial data cached |
| Infection | 179 | 4.9 4.2–5.7ROR 5.0 · χ² 556 / p 3e-123 | 4.8 EB05 4.3 · IC025 +2.0 3/3 methods agree | Labeled — boxed warning exact term | serious PRR 3.6 · 93% clinician | no trial data cached |
| Product use in unapproved indication | 175 | 2.8 2.4–3.3ROR 2.9 · χ² 208 / p 2e-47 | 2.8 EB05 2.5 · IC025 +1.2 3/3 methods agree | Reporting / coding artifact | serious PRR 3.5 · 96% clinician | no trial data cached |
| Anaemia (low red blood cells) | 160 | 3.3 2.8–3.9ROR 3.4 · χ² 258 / p 2e-58 | 3.3 EB05 2.9 · IC025 +1.5 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 2.1 · 94% clinician | no trial data cached |
| Septic shock | 151 | 13.8 11.8–16.2ROR 14.2 · χ² 1,784 / p <1e-300 | 13.7 EB05 12.0 · IC025 +3.5 3/3 methods agree | Not in label — unassessed | serious PRR 8.1 · 99% clinician | 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.
- Myelodysplastic syndrome — n=220, PRR 60.9
- Haematotoxicity — n=82, PRR 35.2
- Bacteraemia — n=54, PRR 18.2
- Septic shock — n=151, PRR 13.8
- Thrombocytopenia — n=312, PRR 11.1
- Hyperbilirubinaemia — n=28, PRR 10.8
- Lymphoma — n=42, PRR 10.7
- Cytomegalovirus infection — n=44, PRR 10.5
- Candida infection — n=42, PRR 10.0
- Disseminated intravascular coagulation — n=35, 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 Mitoxantrone?
In FDA FAERS, the most frequently co-reported reaction terms for Mitoxantrone are febrile neutropenia, neutropenia, thrombocytopenia, pyrexia, acute myeloid leukaemia, sepsis. 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 Mitoxantrone have an FDA boxed warning?
Yes. The current FDA label carries a boxed warning: Mitoxantrone injection (concentrate) should be administered under the supervision of a physician experienced in the use of cytotoxic chemotherapy agents. Mitoxantrone injection (concentrate) should be given slowly into a freely flowing intravenous infusion. It must never be given subcutaneously, intramuscularly, or intra-arterially.
Do FAERS signals prove that Mitoxantrone 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 1634528a-30c5-4309-acc8-4c0293ae14ae, effective 20250424; matched on
openfda.generic_name.exact:"MITOXANTRONE") - 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, 227 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.