This page shows what was measured, who it was measured in, and what that does not settle.
What Treosulfan does in the body
2 Pharmacodynamics Increase in treosulfan exposure was associated with an increased incidence of infection-related death.
From the FDA-approved label: Mechanism of Action Treosulfan is an alkylating agent. DNA alkylation is thought to be responsible for the cytotoxic activities of treosulfan. Treosulfan showed hematopoietic stem cell depleting activity as well as immunosuppressive and antitumor activity in mouse models of leukemia. 12. Treosulfan time course of pharmacodynamic response is unknown. 3 Pharmacokinetics Treosulfan pharmacokinetic parameters were observed at the approved recommended dosage, unless otherwise specified. Treosulfan is a prodrug.
Why people take it. GRAFAPEX is an alkylating drug indicated for: Use in combination with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation (alloHSCT) in adult and pediatric patients 1 year of age and older with acute myeloid leukemia (AML).
What happened in people
RNAWiki has not yet published a reviewed conclusion for this use.
§ A fixed RNAWiki sentence
Where this came from
Wording RNAWiki always uses, not a finding about this substance.
No reviewed claim names a result for any goal on this record.
No source is stored against this line.
The limit that matters most
Not recorded.
Where it acts
Not recorded.
Kind of result
No result is published, so no kind of result applies yet
Supervision
Professional supervision is normally required. A regulator classifies this substance in a way that restricts how it is supplied.
What the registries record it as
The substance registry classes this as chemical.
FDA substance registry · CO61ER3EPI · read 2026-08-29
Its recorded molecular formula is C6H14O8S2, weighing 278.3 g/mol.
US prescribing information · 2d06b03c-17aa-0492-e063-6294a90abd8f · read 2026-08-30
Where each sentence above came from
A person wrote this explanation into the record, with the studies named in the path below.
The use the label states, quoted from it. It is written for a clinician, not for a reader without medical training.
No statement of the main limit is recorded.
The four opening statements run to 137 words.
Words this page uses
Four words worth knowing first
Chosen from what this page shows, with each one explained before the word it depends on.
Biomarker
A biomarker is a number from a test that stands in for something about health.
A picture of it, and where the picture fails
A biomarker is like a fuel gauge.
Where that stops being true. A gauge is wired to the tank. Many biomarkers are only loosely tied to health.
What people get wrong. A better number is read as a better life. Several medicines improved a number and helped nobody.
A measurable indicator used as a substitute for a clinical outcome of interest.
Placebo
A placebo is a dummy treatment given so the real one can be compared with it.
A picture of it, and where the picture fails
A placebo is like a blank control in an experiment.
Where that stops being true. A blank does nothing. People given a placebo often do get better.
What people get wrong. A placebo effect is read as imaginary. The improvement is measured and real.
An inactive intervention matched in appearance to the test intervention, used to control for non-specific effects.
Comparator
A comparator is whatever the treatment was measured against.
A picture of it, and where the picture fails
It is like the other runner in a race.
Where that stops being true. A race has one winner. A study can show both arms improved.
What people get wrong. Results are read without asking what the other group got. Beating nothing is not beating a treatment.
The control condition against which the experimental intervention is assessed.
What happened in people◇Read from sources, not yet reviewed
What happened in people
Each card is one study: the exact question it asked, who was in it, and whether it showed what it set out to show.
What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.
The limits of this study, and where it came from
Main limit. No limitation is recorded for this study.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Intravenous
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
openFDA Drugs@FDA · a recorded source, not a stored snapshot
openFDA NDC Directory · a recorded source, not a stored snapshot
openFDA SPL label · a recorded source, not a stored snapshot
PubChem PUG-REST · a recorded source, not a stored snapshot
Vomiting post prediction time
✗ The study did not show it
Who was studied
NCT06886451
How many people
1332
Study design
Not applicable
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated
What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.
The limits of this study, and where it came from
Main limit. No limitation is recorded for this study.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Intravenous
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
openFDA Drugs@FDA · a recorded source, not a stored snapshot
openFDA NDC Directory · a recorded source, not a stored snapshot
openFDA SPL label · a recorded source, not a stored snapshot
PubChem PUG-REST · a recorded source, not a stored snapshot
Genetic variants in participants as a marker of risk of Adverse events and/or Efficacy of the studied agents
✗ The study did not show it
Who was studied
NCT02670564
How many people
1000
Study design
Phase 4
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated
What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.
The limits of this study, and where it came from
Main limit. No limitation is recorded for this study.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Intravenous
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
openFDA Drugs@FDA · a recorded source, not a stored snapshot
openFDA NDC Directory · a recorded source, not a stored snapshot
openFDA SPL label · a recorded source, not a stored snapshot
PubChem PUG-REST · a recorded source, not a stored snapshot
Event free survival
✗ The study did not show it
Who was studied
NCT00987636
How many people
907
Study design
Phase 3
Compared against
Not recorded for this study
Kind of result
Living longer, or avoiding a major event
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated
What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.
The limits of this study, and where it came from
Main limit. No limitation is recorded for this study.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Intravenous
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
openFDA Drugs@FDA · a recorded source, not a stored snapshot
openFDA NDC Directory · a recorded source, not a stored snapshot
openFDA SPL label · a recorded source, not a stored snapshot
PubChem PUG-REST · a recorded source, not a stored snapshot
Event-free survival (EFS)
✗ The study did not show it
Who was studied
NCT00822393
How many people
570
Study design
Phase 3
Compared against
Not recorded for this study
Kind of result
Living longer, or avoiding a major event
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated
What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.
The limits of this study, and where it came from
Main limit. No limitation is recorded for this study.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Intravenous
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
openFDA Drugs@FDA · a recorded source, not a stored snapshot
openFDA NDC Directory · a recorded source, not a stored snapshot
openFDA SPL label · a recorded source, not a stored snapshot
PubChem PUG-REST · a recorded source, not a stored snapshot
Event free survival
✗ The study did not show it
Who was studied
NCT01423500
How many people
405
Study design
Phase 3
Compared against
Not recorded for this study
Kind of result
Living longer, or avoiding a major event
What was found
Result not recorded on this page
Repeated elsewhere
Unreplicated
What this does not prove. A study that did not show an effect does not show that no effect exists anywhere.
The limits of this study, and where it came from
Main limit. No limitation is recorded for this study.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Intravenous
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
openFDA Drugs@FDA · a recorded source, not a stored snapshot
openFDA NDC Directory · a recorded source, not a stored snapshot
openFDA SPL label · a recorded source, not a stored snapshot
PubChem PUG-REST · a recorded source, not a stored snapshot
What we know
RNAWiki holds 6 written-up human studies for this substance, each with the question it asked.
How we know it
Each card names the study, the number of people and what the study set out to measure.
What this does not prove
These summaries were written by a person and have not been signed off as reviewed claims.
Why it matters
A study that failed is as informative as one that worked, and both are here.
What we still do not know
No reviewed claim exists, so no exact population, effect size or interval is published yet.
What is missing or unclear◇Read from sources, not yet reviewed
Were people like you studied?
RNAWiki cannot tell whether a study fits you. It can show who was in it, and where the result stops carrying.
Who was studied
People similar to this profile were included.
GRAFAPEX is an alkylating drug indicated for: Use in combination with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation (alloHSCT) in adult and pediatric patients 1 year of age and older with acute myeloid leukemia (AML). ( 1.1 ).
Who is missing from the studies
Which groups were under-represented in the studies has not been recorded for this substance. Pregnancy, breastfeeding, children, older adults, and people with liver or kidney conditions are commonly excluded from trials; nothing here says whether they were.
What the label states about particular groups
On pediatric, the label states: “The incidence of hepatic and gastrointestinal adverse reactions was higher in pediatric patients than in adults.”
US prescribing information · 2d06b03c-17aa-0492-e063-6294a90abd8f · read 2026-08-30
On older people, the label states: “Of the total number of GRAFAPEX-treated patients with AML or MDS in Study MC-FludT.14/L Trial II (n=270), 73 (27%) were 65 to 74 years of age, and none were 75 years of age and older [see Clinical Studies ( 14 )] .”
US prescribing information · 2d06b03c-17aa-0492-e063-6294a90abd8f · read 2026-08-30
On people who are pregnant, the label states: “Risk Summary Based on its mechanism of action, GRAFAPEX can cause fetal harm when administered to a pregnant woman because it is genotoxic and affects dividing cells [see Clinical Pharmacology ( 12 ) and Nonclinical Toxicology ( 13 )] .”
US prescribing information · 2d06b03c-17aa-0492-e063-6294a90abd8f · read 2026-08-30
On people who are breastfeeding, the label states: “Risk Summary There is no data on the presence of treosulfan or its metabolites in human milk, the effects on the breastfed child, or on milk production.”
US prescribing information · 2d06b03c-17aa-0492-e063-6294a90abd8f · read 2026-08-30
Where the result stopped carrying
This is a scope explorer, not a diagnosis engine.
It shows who was studied so you can see whether people similar to you were included.
RNAWiki cannot tell whether a study fits you. It can show who was in it.
What it would be like to take◇Read from sources, not yet reviewed
Why it might seem to do nothing
A real effect and a noticed effect are different things. These are the recorded reasons the two come apart.
It was studied in different people
The people in the studies were not much like the reader.
On this record: Which groups were under-represented in the studies has not been recorded for this substance. Pregnancy, breastfeeding, children, older adults, and people with liver or kidney conditions are commonly excluded from trials; nothing here says whether they were.
The evidence may simply be wrong
Small early studies often do not hold up.
On this record: RNAWiki has not yet published a reviewed conclusion for this use.
Other reasons RNAWiki checked and found nothing for (11)
It was studied for a different goal
The studies measured something else entirely. Nothing in this record points to this reason.
A different form was studied
The studied form is not the form on the shelf. Nothing in this record points to this reason.
There was nothing to correct
Where a level is already normal, topping it up may change nothing. Nothing in this record points to this reason.
Something else had to happen too
In the studies it was paired with training, a diet or another treatment. Nothing in this record points to this reason.
The change is too small to feel
A real change can still sit below what a person notices. Nothing in this record points to this reason.
Day-to-day swing hides it
Sleep, food, stress and time of day move most numbers more than this would. Nothing in this record points to this reason.
Not enough time yet
The studies ran longer than the person has waited. Nothing in this record points to this reason.
It was not taken as studied
Missed days change the result, and studies count them. Nothing in this record points to this reason.
Something else changed at the same time
Two changes at once cannot be told apart afterwards. Nothing in this record points to this reason.
The product may not be what it says
Contents of a sold product are not always what the label states. Nothing in this record points to this reason.
No recorded reason
RNAWiki has nothing stored that would explain it. Nothing in this record points to this reason.
None of these is a reason to take more. Taking more is not a step this page ever suggests.
What it would be like to take◇Read from sources, not yet reviewed
What taking it involves
The recorded facts about getting hold of it and using it. Nothing here is a suggestion about what you should do.
How it is supplied
Given by a clinician
❝ Quoted from a source
Where this came from
Copied from a source RNAWiki stored, with the source named.
Read from the recorded approval status.
No source is stored against this line.
Form and route
Intravenous
✎ Source-linked record
Where this came from
A person wrote this into the record with the study named beside it. No reviewer has signed it off.
The form and route recorded on this substance.
No source is stored against this line.
Who oversees it
Professional supervision is normally required. A regulator classifies this substance in a way that restricts how it is supplied.
❝ Quoted from a source
Where this came from
Copied from a source RNAWiki stored, with the source named.
Suppression classes recorded: S1, S3, S4, S6, S7.
No source is stored against this line.
What is in the pack
Sold as powder, injection, powder, lyophilized, for solution, given by the intravenous route.
✎ Source-linked record
Where this came from
A person wrote this into the record with the study named beside it. No reviewer has signed it off.
Recorded notes on how this is sold and what that means for what is in the pack.
No source is stored against this line.
Where it is registered
Regulatory records are listed in the technical disclosure at the foot of this page.
# Counted from records
Where this came from
A count of rows RNAWiki holds. It describes our records, not your body.
Register entries are stored per jurisdiction and shown with their dates.
No source is stored against this line.
Why people stop
Not recorded.
∅ Nothing recorded
Where this came from
RNAWiki holds nothing here and says so rather than guessing.
No record of why people stopped taking it is stored for this substance.
No source is stored against this line.
What it would be like to take∅Nothing found in the sources checked
What can go wrong
Sorted by where each line came from. A regulator's label and a report somebody sent in are not the same kind of fact.
∅Nothing found in the sources checked
No harm is recorded against this substance in the sources RNAWiki checked. Finding nothing is not the same as showing there is nothing.
The sources listed were searched and held nothing. That is not the same as nothing existing.
Over a long time. No completed tested study window is recorded, so long-term effects are not established by the registry record.
Groups the studies covered thinly. Which groups were under-represented in the studies has not been recorded for this substance. Pregnancy, breastfeeding, children, older adults, and people with liver or kidney conditions are commonly excluded from trials; nothing here says whether they were.
What is missing or unclear◇Read from sources, not yet reviewed
Does the exact form matter?
Evidence belongs to the exact thing that was tested. A different salt, a different mixture or a different preparation is a different question.
The form that was studied
Intravenous
✎ Source-linked record
Where this came from
A person wrote this into the record with the study named beside it. No reviewer has signed it off.
The form used in the studies cited on this page.
No source is stored against this line.
What is sold
Not recorded.
∅ Nothing recorded
Where this came from
RNAWiki holds nothing here and says so rather than guessing.
Nothing further is recorded about which forms are sold.
No source is stored against this line.
What is recorded as being sold
2 products list this as an active ingredient in the United States drug directory. 2 of them contain it and nothing else.
FDA National Drug Code directory · 59137-365 · read 2026-08-29
They are sold as injection, powder, lyophilized, for solution, taken intravenous.
FDA National Drug Code directory · 59137-365 · read 2026-08-29
1 published label names it as an active ingredient. 1 of them describe this substance alone, which is where its own label text on this page comes from.
US prescribing information · 2d06b03c-17aa-0492-e063-6294a90abd8f · read 2026-08-29
Those labels are classed as human prescription drug.
US prescribing information · 2d06b03c-17aa-0492-e063-6294a90abd8f · read 2026-08-29
GRAFAPEX is intravenous at 3 DOSAGE FORMS AND STRENGTHS For injection: 1 g/vial or 5 g/vial treosulfan as a white, sterile, lyophilized powder in single-dose vials for reconstitution., recorded as fda label in effect 2025-01-31 in the United States.
US prescribing information · 2d06b03c-17aa-0492-e063-6294a90abd8f · read 2026-08-30
Evidence carries across two names for one substance. It does not carry across a salt, a mirror form or a mixture.
What it would be like to take◇Read from sources, not yet reviewed
What you could measure
This one is decided with a clinician, so this section holds questions to ask rather than a plan to run.
Self-experiment planning is not offered for a prescription or clinician-supervised medicine. The questions below are for a clinician or pharmacist.
Questions worth asking
Which of the trials of Treosulfan studied people like me?
What was measured, and for how long?
Was the result a laboratory value or a health outcome?
What would we watch for, and when would we stop?
Tracking can show whether something changed for you. It cannot show what caused it.
RNAWiki records evidence. It does not say whether this substance is right for you.
What is missing or unclear◇Read from sources, not yet reviewed
What nobody knows yet
Open questions, each with why it is open and what would close it.
How much did people take in the studies?
The sources RNAWiki checked hold nothing for this field.
Why it matters. A result belongs to an amount. Without the amount the result floats free.
What would answer it
A stored source that records it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
Missing populations
Which groups were under-represented in the studies has not been recorded.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
Missing long-term data
No completed tested study window is recorded.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
No reviewed conclusion
RNAWiki has not yet published a reviewed conclusion for this use.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
Missing interaction studies
No interaction was found in the registers checked. Not finding one is not the same as showing there is none.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
Formulation uncertainty
Several salts, forms or products of Treosulfan are recorded. Results from one form may not transfer to another.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
Mechanism not reviewed
No reviewed mechanism story exists for this substance.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
What happened in people◇Read from sources, not yet reviewed
Check any of this yourself
Every line above traced back to the study it came from. This is the technical layer, and the vocabulary changes here.
Where else this substance is registered
FDA substance identifier (UNII)
CO61ER3EPI
RxNorm concept
2703570
Checks this page had to pass
✓ Passed
Identity resolved
no open identity hold
✓ Passed
No unresolved merge across substance families
no quarantine open
✓ Passed
Every public sentence names a source
The opening statement carries the origin: Written into the record, not signed off.
✗ Not passed
Trial roles classified for highlighted evidence
No registered study is classified as testing this substance.
✓ Passed
No internal keys in reader text
enforced by the copy-contract test over the rendered page
✓ Passed
Safety mode resolved
Suppression classes recorded: S1, S3, S4, S6, S7.
✓ Passed
Canonical metadata present
slug and display name present
What is missing or unclear◇Read from sources, not yet reviewed
How this medicine reached us
Kept near the foot of the page. A historical event moves up only when it changes something about this substance today.
What the approval register records
1 approved application covers products containing this substance. The earliest was NDA214759, approved 20250121 to MEDEXUS.
This order is fixed in code and does not count clicks or time on the page.
What is not here
9 questions this page could not answer
These sections were prepared and left out because there was nothing to put in them. They are listed rather than hidden, so the gaps in this record are visible.
What was measured, goal by goal — found nothing in the sources checked.
How close this is to real life — found nothing in the sources checked.
The path through the body — found nothing in the sources checked.
Felt, measured, or meaningful — found nothing in the sources checked.
How long anything takes — found nothing in the sources checked.
What it may clash with — found nothing in the sources checked.
Other ways to the same goal — found nothing in the sources checked.
Claims that go past the evidence — found nothing in the sources checked.
What changed on this page — found nothing in the sources checked.
The record as stored
The full record, for auditing
Everything above is built from what is below. This layer keeps the technical vocabulary, record identifiers and every stored row, so a reader who wants to check the page can.
Recorded evidence blocks (10)
Q2
On the Treosulfan label: indicated for what?
"GRAFAPEX is an alkylating drug indicated for: Use in combination with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation (alloHSCT) in adult and pediatric patients 1 year of age and older with acute myeloid leukemia (AML). ( 1.1 ).": indications and usage on Treosulfan's label. DailyMed label · 2d06b03c-17aa-0492-e063-6294a90abd8f · 2025-01-31
Q3
36 registered trials of Treosulfan — at which phases?
1.7 ± 0.4 hours; Elimination Treosulfan mean (± standard deviation) terminal half-life is 1.7 ± 0.4 hours.
metabolismclinical_pharmacology
Metabolism The pharmacologically inactive treosulfan is converted spontaneously under physiological conditions into the active monoepoxide intermediate (2S,3S)‑1,2‑epoxybutane‑3,4‑diol‑4‑methanesulfonate) and finally to active L‑diepoxibutane (2S,3S)‑1,2:3,4‑diepoxybutane).
recorded 2025-01-31 · last checked 2026-09-04
Q6
Which running trial of Treosulfan could settle lifespan?
NCT07232953 measures The 1-year leukemia -free survival (LFS) after allo-SCT, reading out 2027-06.
6 open trials; n 82; "Safety and Efficacy of FT14 Conditioning for Allogeneic HSCT in Acute Myeloid Leukemia"
Show the evidence
Trial
NCT07232953
"Safety and Efficacy of FT14 Conditioning for Allogeneic HSCT in Acute Myeloid Leukemia"; n 82; "The 1-year leukemia -free survival (LFS) after allo-SCT"; 2027-06
NCT07025824
"Evaluation of Treosulfan Versus Melphalan Conditioning Followed by PTCy in Patients With AML and MDS Undergoing Allogeneic Transplantation"; n 220; "overall survival (OS)"; 2028-12
NCT06198842
"Low Dose Treosulfan Based Conditioning Regimen and PTCy in HSCT for Nijmegen Breakage Syndrome"; n 10; "Event-free survival"; 2029-01-31
NCT01949129
"Allogeneic Stem Cell Transplantation for Children and Adolescents With Acute Lymphoblastic Leukaemia"; n 1800; "Overall Survival (OS) Stratum 1a (randomisation TBI+ chemo-conditioning vs. chemo-conditioning only)"; 2030-04
NCT07316595
"Study of Treosulfan-Based Conditioning for HSCT in Nijmegen Breakage Syndrome"; n 24; "To evaluate event-free survival (EFS), defined by the occurrence of death, graft rejection, or development of secondary malignancy/relapse."; 2030-12-31
NCT07493538
"MT2025-35 Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning Treosulfan and Fludarabine, With Post-Transplant Cytoxan (PTCy) for the Treatment of Hematological Diseases"; n 132; "Overall Survival"; 2035-03
Q7
Which 12 trials of Treosulfan posted no result?
Posted no result
12 of 12 completed trials
Registrations
NCT01063647, NCT01063660, NCT01062490, NCT00170690, NCT00491634 and NCT01772953, and 6 more
Completion dates
oldest 2006-06; newest 2023-02-13
Show the evidence
Trial
NCT01063647
2006-06
NCT01063660
2007-07
NCT01062490
2009-10
NCT00170690
2012-04
NCT00491634
2014-06
NCT01772953
2015-08
6 further recorded trials
NCT02048800
2017-07-31
NCT00822393
2018-01-25
NCT00987636
2019-03-31
NCT02333058
2019-09-30
NCT03961919
2021-12-31
NCT02349906
2023-02-13
Q8
At the median, Treosulfan's trials enrolled 64 people — anything larger?
Median enrolment
64
Largest enrolment
1800
Registered trials counted
36
Q9
What do 574 spontaneous reports say about Treosulfan — and not say?
These are reports people sent to a regulator. They do not show the medicine caused the reaction. Nobody counted how many people took the medicine and reported nothing. The same event can be reported more than once, and many reports are incomplete. News coverage, lawsuits and new warnings change how often people report. A count is not a rate and not a risk.
Treosulfan appears in spontaneous reports to regulators. Across the 10 most-reported reaction terms, 574 reaction mentions were counted: cytomegalovirus infection 105; adenovirus infection 79; mucosal inflammation 67; acute graft versus host disease in skin 59. FAERS via Open Targets · CHEMBL455186 · 2026-06-24
Show the evidence
cytomegalovirus infection
105
adenovirus infection
79
mucosal inflammation
67
acute graft versus host disease in skin
59
graft versus host disease in skin
56
neutropenia
53
4 more recorded rows
thrombocytopenia
43
human herpesvirus 6 infection
39
cytomegalovirus infection reactivation
38
pyrexia
35
recorded 2026-06-24 · last checked 2026-09-04
Q10
Which 10 reactions does Treosulfan's label not list?
acute graft versus host disease in skin, adenovirus infection and cytomegalovirus infection and 7 more reported for Treosulfan, absent from its label. FAERS via Open Targets · CHEMBL455186 · 2026-06-24
2 label terms; 10 reported and unlisted; 2d06b03c-17aa-0492-e063-6294a90abd8f
Show the evidence
acute graft versus host disease in skin
count not stated
adenovirus infection
count not stated
cytomegalovirus infection
count not stated
cytomegalovirus infection reactivation
count not stated
graft versus host disease in skin
count not stated
human herpesvirus 6 infection
count not stated
4 more recorded rows
mucosal inflammation
count not stated
neutropenia
count not stated
pyrexia
count not stated
thrombocytopenia
count not stated
recorded 2026-06-24 · last checked 2026-09-04
Q11
Treosulfan and CYP3A4, CYP2C8 and CYP2D6: shared by which compounds?
Certain CYP2C19 and CYP3A4 Substrates: Monitor for adverse reactions of these substrates where minimal concentration changes may lead to serious or life-threatening toxicities.
drug_interactions
( 7.1 ) 7.1 Effect of GRAFAPEX on Other Drugs Certain CYP2C19 and CYP3A4 Substrates Monitor for adverse reactions of certain CYP2C19 or CYP3A4 substrates where minimal concentration changes may lead to serious or life-threatening toxicities, and reduce the dosage, as needed, if recommended in the prescribing information of these substrates.
drug_interactions
Treosulfan is a CYP2C19 and CYP3A4 inhibitor [see Clinical Pharmacology ( 12 )] .
drug_interactions
Concomitant use of GRAFAPEX is predicted to increase the exposure of CYP2C19 and CYP3A4 substrates based on a mechanistic understanding of treosulfan metabolism, which may increase the risk of their adverse reactions.
clinical_pharmacology
Drug Interaction Studies In Vitro Studies CYP Enzymes : Treosulfan is a CYP2D6 substrate and its monoepoxide intermediate is a CYP2C8 substrate.
clinical_pharmacology
Treosulfan inhibits CYP2C19 and CYP3A4 (using midazolam as substrate), but does not inhibit CYP1A2, CYP2B6, CYP2C8, CYP2C9, CYP2D6, or CYP3A4 (using testosterone as substrate).
1 more recorded row
Interaction statementclinical_pharmacology
Transporter Systems : Treosulfan does not inhibit BCRP, BSEP, MATE1, OATP1B1, OATP1B3, OAT1, OAT3, OCT1, or OCT2.
ChEMBL 37 — CC BY-SA 3.0 Unported · ChEMBL ATC CC BY-SA · ClinicalTrials.gov — US Government work · Open Targets 26.06 — CC0 · mixed register set; per-register licences in docs/specs/corpus-20k-sources.md · openFDA / DailyMed — US Government work
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