This page shows what was measured, who it was measured in, and what that does not settle.
What Busulfan does in the body
Busulfan injection is indicated for use in combination with cyclophosphamide as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous leukemia.
From the FDA-approved label: Busulfan is a bifunctional alkylating agent in which two labile methanesulfonate groups are attached to opposite ends of a four-carbon alkyl chain. In aqueous media, busulfan hydrolyzes to release the methanesulfonate groups. This produces reactive carbonium ions that can alkylate DNA. DNA damage is thought to be responsible for much of the cytotoxicity of busulfan.
Why people take it. Busulfan injection is indicated for use in combination with cyclophosphamide as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous leukemia.
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 · G1LN9045DK · read 2026-08-29
Where each sentence above came from
The use the label states, quoted from it. No plain-language version of this sentence has been written.
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 120 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.
Event Free Survival (R1: MAT therapy)
✗ The study did not show it
Who was studied
NCT01704716
How many people
3300
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. Injection
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
Timing of treatment Substudy or Tier Advancement Pathway (TAP) assignment
✗ The study did not show it
Who was studied
NCT05564390
How many people
2000
Study design
Phase 2
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. Injection
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
Overall survival
✗ The study did not show it
Who was studied
NCT00454480
How many people
2000
Study design
Phase 2/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. Injection
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
Duration of overall survival and disease-free survival after first randomization
✗ The study did not show it
Who was studied
NCT00004128
How many people
2000
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. Injection
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 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. Injection
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
Combination Chemotherapy Followed by Bone Marrow or Peripheral Stem Cell Transplantation in Treating Patients With Acute Myelogenous Leukemia
✗ The study did not show it
Who was studied
NCT00002549
How many people
1520
Study design
Phase 3
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. Injection
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.
Busulfan Injection is indicated for use in combination with cyclophosphamide as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous leukemia.
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 effectiveness of busulfan in the treatment of CML has not been specifically studied in pediatric patients.”
US prescribing information · ac9448da-37ad-4609-9aef-eedf8be0f69c · read 2026-08-30
On older people, the label states: “Clinical studies of busulfan did not include sufficient numbers of subjects aged 65 and over to determine whether they respond differently from younger subjects.”
US prescribing information · ac9448da-37ad-4609-9aef-eedf8be0f69c · read 2026-08-30
On people who are pregnant, the label states: “Risk Summary Busulfan can cause fetal harm when administered to a pregnant woman based on animal data.”
US prescribing information · ac9448da-37ad-4609-9aef-eedf8be0f69c · read 2026-08-30
On people who are breastfeeding, the label states: “8.3 Females and Males of Reproductive Potential Contraception Females Busulfan can cause fetal harm when administered to a pregnant woman [see Use in Specific Populations (8.1)].”
US prescribing information · ac9448da-37ad-4609-9aef-eedf8be0f69c · 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
Prescription only
❝ 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
Injection
✎ 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.
No source is stored against this line.
What is in the pack
Sold as injectable, tablet, tablet, film coated, injection, given by the injection, oral, 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
Injection
✎ 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
17 products list this as an active ingredient in the United States drug directory. 17 of them contain it and nothing else.
FDA National Drug Code directory · 51817-170 · read 2026-08-29
They are sold as injection, injection, solution, injection, solution, concentrate, powder and tablet, film coated, taken intravenous and oral.
FDA National Drug Code directory · 51817-170 · read 2026-08-29
The regulator's established pharmacologic class for it is alkylating activity [moa] and alkylating drug [epc].
FDA National Drug Code directory · 51817-170 · read 2026-08-29
9 published labels name it as an active ingredient. 9 of them describe this substance alone, which is where its own label text on this page comes from.
US prescribing information · ac9448da-37ad-4609-9aef-eedf8be0f69c · read 2026-08-29
Those labels are classed as human prescription drug.
US prescribing information · ac9448da-37ad-4609-9aef-eedf8be0f69c · read 2026-08-29
Busulfan is intravenous at 3 DOSAGE FORMS AND STRENGTHS Injection: 60 mg/10 mL (6 mg/mL) as a clear, colorless, sterile, solution in a single-dose vial for intravenous use only ., recorded as fda label in effect 2020-09-23 in the United States.
US prescribing information · ac9448da-37ad-4609-9aef-eedf8be0f69c · 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 Busulfan 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 Busulfan 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)
G1LN9045DK
RxNorm concept
253113
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: Quoted from a stored source.
✗ 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.
✓ 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
17 approved applications cover products containing this substance. The earliest was NDA009386, approved 19540626 to WAYLIS THERAP.
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 (8)
Q2
On the Busulfan label: indicated for what?
"Busulfan Injection is indicated for use in combination with cyclophosphamide as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous leukemia. Busulfan Injection is an alkylating drug indicated for: Use in combination with cyclophosphamide as a conditioning…": indications and usage on Busulfan's label. DailyMed label · 086a715e-4958-4bb5-9ebd-86171e6b6203 · 2026-07-29
Q3
484 registered trials of Busulfan — at which phases?
Registered studies posting no result
328 of 484
484 registered studies of Busulfan: 329 phase2, 124 phase1, 69 phase3, 36 na, 5 early phase1, 5 phase4, 3 na or unstated. CLINICALTRIALS_SNAPSHOT · 2026-09-01
"PI left institution"; 81 of 484 registered studies
Show the evidence
Trial
NCT00002761
withdrawn; "PI left institution"
NCT00002961
terminated; "poor accrual"
NCT00003060
terminated; "lack of patient accrual"
NCT00003926
terminated; "Withdrawn due to slow accrual"
NCT00005641
terminated; "low study accrual"
NCT00005986
terminated; "Principal investigator left the university."
14 further recorded trials
NCT00006126
withdrawn; "Unable to accrue subjects."
NCT00034528
terminated; "Due to slow recruitment"
NCT00040469
terminated; "accrual was slow and sporadic so the study was closed"
NCT00113919
terminated; "Due to poor accrual"
NCT00176826
terminated; "Replaced by another protocol"
NCT00176839
terminated; "Replaced by a different study"
NCT00176930
terminated; "replaced by another study ; Trial re-written as MT2015-29"
NCT00245115
terminated; "Expiration of study supply"
NCT00253552
terminated; "Terminated at request of PI as study was outdated."
NCT00290628
terminated; "Replaced with another study"
NCT00301912
withdrawn; "Withdrawn because study never opened to accrual"
NCT00363467
terminated; "Low accrual"
NCT00446173
withdrawn; "Terminated due to low accrual."
NCT00478244
terminated; "Competing studies"
recorded 2026-09-01 · last checked 2026-09-04
Q5
Which running trial of Busulfan could settle lifespan?
NCT03121014 measures Relapse free survival of approximately 30% in high-risk patients conditioned with the Fludarabine/ Busulfan regimen, reading out 2026-04.
33 open trials; n 38; "Study of Intensity Modulated Total Marrow Irradiation (IM-TMI) in Addition to Fludarabine/Busulfan Conditioning for Allogeneic Transplantation in High Risk AML and Myelodysplastic Syndromes"
Show the evidence
Trial
NCT03121014
"Study of Intensity Modulated Total Marrow Irradiation (IM-TMI) in Addition to Fludarabine/Busulfan Conditioning for Allogeneic Transplantation in High Risk AML and Myelodysplastic Syndromes"; n 38; "Relapse free survival of approximately 30% in high-risk patients conditioned with the Fludarabine/ Busulfan regimen"; 2026-04
NCT04547049
"A Study Comparing Haploidentical Hematopoietic Stem Cell Transplantations (HSCTs) From Young Non-first-degree and Older First-degree Donors in Hematological Malignancies"; n 232; "Overall survival (OS)"; 2026-04-01
NCT04217278
"A Trial of Treatments to Assess the Effects on Outcome of Adults With AML and MDS Undergoing Allogeneic SCT"; n 333; "Overall survival (all randomisations)"; 2026-05
NCT01704716
"High Risk Neuroblastoma Study 1.8 of SIOP-Europe (SIOPEN)"; n 3300; "Event Free Survival (R1: MAT therapy)"; 2026-09
NCT04904588
"HLA-Mismatched Unrelated Donor Hematopoietic Cell Transplantation With Post-Transplantation Cyclophosphamide"; n 300; "Overall Survival"; 2026-12
NCT04644016
"Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders"; n 31; "Treatment related mortality at 1 year after myeloablative cord transplant"; 2026-12-20
14 further recorded trials
NCT06001385
"HLA-Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation With Reduced Dose Post Transplantation Cyclophosphamide GvHD Prophylaxis"; n 313; "Infection Free Survival"; 2026-12-30
NCT02727803
"Personalized NK Cell Therapy in CBT"; n 100; "Progression free survival (PFS) time in C2C2 patients"; 2027-05-31
NCT05303727
"Allogeneic Hematopoietic Stem Cell Transplantation for 4/M Neuroblastoma"; n 64; "overall survival(OS) at 3 year"; 2027-08
NCT02250937
"Venetoclax and Sequential Busulfan, Cladribine, and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Acute Myelogenous Leukemia or Myelodysplastic Syndrome"; n 116; "Progression free survival"; 2027-10-31
NCT06809699
"Human Leukocyte Antigen (HLA) Mismatched Related Allogeneic Hematopoietic Stem Cell Transplantation"; n 29; "Overall survival"; 2027-10-31
"Allogeneic Stem Cell Transplantation in Relapsed/Refractory T-, NK/T-cell Lymphomas"; n 34; "2-year progression-free survival"; 2027-12
NCT04708054
"Venetoclax to Improve Outcomes of Fractionated Busulfan Regimen in Patients With High-Risk AML and MDS"; n 324; "1-year progression free survival (PFS)"; 2027-12-31
NCT06297772
"Compare the Efficacy and Safety of Dec-FB4 and FB4 as Conditioning Regimen for AML-MR"; n 220; "disease-free survival"; 2028-05-01
NCT06954987
"Venetoclax or Placebo in Combination With Reduced-Intensity Conditioning Hematopoietic Cell (Bone Marrow/Blood Stem Cell) Transplant and as Maintenance Therapy After Transplant in Patients With Acute Myeloid Leukemia (A MyeloMATCH Treatment Trial)"; n 244; "Event free survival (EFS)"; 2028-05-15
NCT02861417
"Busulfan, Fludarabine Phosphate, and Post-Transplant Cyclophosphamide in Treating Patients With Blood Cancer Undergoing Donor Stem Cell Transplant"; n 204; "Non-relapse mortality rate"; 2028-08-31
NCT04451200
"Sequential and Personalized PK-guided Busulfan Administration in the Frame of the Conditiong Regimen for Allo-HSCT in Patients With Malignant Hemopathies Ineligible for the Standard Myeloablative Conditioning"; n 82; "non-relapse mortality evaluation"; 2028-12
NCT07441967
"The Safety and Efficacy of the Regimen of Thiotepa + Fludarabine +Granulocyte Colony-Stimulating Factor+ Cytarabine + Busulfan in Single Unrelated Umbilical Cord Blood Transplantation"; n 44; "180-day non-recurrence mortality rate"; 2028-12-30
NCT07762508
"A Study of VABu Conditioning Regimen in Allo-HSCT for AML Patients"; n 42; "2-Year Overall Survival (OS)"; 2029-07
Q6
Which 106 trials of Busulfan posted no result?
Posted no result
106 of 106 completed trials
Registrations
NCT00002554, NCT00002502, NCT00003195, NCT00003662, NCT00003462 and NCT00003146, and 100 more
Completion dates
oldest 1999-12; newest 2024-04-30
Show the evidence
Trial
NCT00002554
1999-12
NCT00002502
2000-05
NCT00003195
2000-11
NCT00003662
2001-01
NCT00003462
2001-02
NCT00003146
2001-06
14 further recorded trials
NCT00002789
2001-09
NCT00003960
2001-09
NCT00005948
2001-10
NCT00003081
2002-01
NCT00003101
2002-01
NCT00004171
2002-06
NCT00002592
2002-08
NCT00002831
2002-12-31
NCT00003972
2003-03
NCT00002792
2003-04
NCT00027924
2003-05
NCT00002547
2003-10
NCT00006367
2004-01
NCT00003619
2004-04
Q7
At the median, Busulfan's trials enrolled 38 people — anything larger?
Median enrolment
38
Largest enrolment
3300
Registered trials counted
454
Q8
What do 2595 spontaneous reports say about Busulfan — 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.
Busulfan appears in spontaneous reports to regulators. Across the 10 most-reported reaction terms, 2595 reaction mentions were counted: mucosal inflammation 363; cytomegalovirus infection 347; venoocclusive liver disease 343; graft versus host disease 300. FAERS via Open Targets · CHEMBL820 · 2026-06-24
Show the evidence
mucosal inflammation
363
cytomegalovirus infection
347
venoocclusive liver disease
343
graft versus host disease
300
febrile neutropenia
286
acute graft versus host disease
214
4 more recorded rows
cystitis haemorrhagic
202
chronic graft versus host disease
201
venoocclusive disease
170
acute graft versus host disease in skin
169
recorded 2026-06-24 · last checked 2026-09-04
Q9
Which 10 reactions does Busulfan's label not list?
acute graft versus host disease, acute graft versus host disease in skin and chronic graft versus host disease and 7 more reported for Busulfan, absent from its label. FAERS via Open Targets · CHEMBL820 · 2026-06-24
2 label terms; 10 reported and unlisted; 086a715e-4958-4bb5-9ebd-86171e6b6203
Show the evidence
acute graft versus host disease
count not stated
acute graft versus host disease in skin
count not stated
chronic graft versus host disease
count not stated
cystitis haemorrhagic
count not stated
cytomegalovirus infection
count not stated
febrile neutropenia
count not stated
4 more recorded rows
graft versus host disease
count not stated
mucosal inflammation
count not stated
venoocclusive disease
count not stated
venoocclusive liver disease
count not stated
recorded 2026-06-24 · last checked 2026-09-04
Where it is registeredIdentifiers, relations and other names
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
✗ required summary fields resolved: 4 required field(s) not terminal: Why people use it, Best-supported result, Biggest unanswered question, Human evidence
✓ public claims reviewed: 0 reviewed claim(s); drafts are never rendered
✓ source coverage passed: 5 source rows
✓ no critical contamination: no quarantine open
✓ canonical metadata passed: slug and display name present
✓ no raw internal fields: enforced by the copy-contract test over the rendered page
This is a record of evidence. It is not medical advice, and it does not say this substance suits you. Nothing here says any substance on RNAWiki is appropriate for a child.