This page shows what was measured, who it was measured in, and what that does not settle.
What Eltrombopag does in the body
Raising dangerously low levels of the blood cells that help form clots.
Platelets are shed by giant cells in the bone marrow, which are told how many to make by a hormone called thrombopoietin. Eltrombopag switches the same receptor on, but it binds a different part of it — the section buried in the cell membrane rather than the part the hormone docks to. So it acts as an extra signal rather than a substitute one. The marrow makes more megakaryocytes, the megakaryocytes shed more platelets, and the count rises over about two weeks.
What happened in people
About four in five reached a safer clot-forming-cell level versus one in four without it, with fewer serious bleeds.
✓ Reviewed first-read answer
Where this came from
A person wrote this and a reviewer approved it against this exact record. It carries no effect size.
A reviewer approved this against this record. The reviewed-claim record carrying the exact population and effect size does not exist yet.
No source is stored against this line.
The limit that matters most
In liver disease, a higher platelet count did not reduce bleeding and caused vein clots.
Where it acts
Bone marrow — the megakaryocyte, the giant cell that platelets are shed from
Kind of result
A number that stands in for health
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
Its recorded molecular formula is C30H35N5O5, weighing 545.63 g/mol.
US prescribing information · ed51e463-7a03-4858-bbd2-882ce4753d5a · read 2026-08-30
Where each sentence above came from
No recorded sentence describes the change this makes in a way that stands on its own. The page opens with what it is taken for instead, and the recorded explanation follows below.
Shown as the opening line on this page.
The limit a reviewer approved as the one that matters most here.
The four opening statements run to 114 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.
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.
Randomisation
Randomisation means chance decides who gets which treatment.
A picture of it, and where the picture fails
It is like a coin toss deciding the groups.
Where that stops being true. A coin toss is fair once. Fairness here comes from doing it for everyone.
What people get wrong. It is thought to make groups identical. It makes them similar on average, including on things nobody measured.
Allocation of participants to arms by a chance process, so that unmeasured factors are balanced in expectation.
Absolute difference
An absolute difference is how many more people in a hundred were affected.
A picture of it, and where the picture fails
It is like counting heads in two rooms of a hundred.
Where that stops being true. Heads are easy to count. Study results carry a margin of error too.
What people get wrong. It is confused with a percentage change, which can look far larger.
The arithmetic difference in event rates between arms.
Confidence interval
A confidence interval is the range the true answer is likely to sit in.
A picture of it, and where the picture fails
It is like a weather forecast giving a range rather than one number.
Where that stops being true. A forecast range covers tomorrow. This one covers what the study could resolve.
What people get wrong. The middle number is read as the answer. A range crossing zero means no change is still possible.
An interval estimate that would contain the true parameter in a stated proportion of repeated studies.
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.
Odds of achieving a platelet count between 50,000 and 400,000 per microlitre during the treatment period
✓ The study showed what it set out to show
Who was studied
RAISE (NCT00370331) — eltrombopag in chronic immune thrombocytopenia
What this does not prove. Meeting one endpoint in one population does not carry to other goals or other people.
The limits of this study, and where it came from
Main limit. Three thromboembolic events on eltrombopag against none on placebo, nine mild alanine aminotransferase rises against two, and five total bilirubin rises against none. The primary endpoint is a platelet count, not a bleeding outcome.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Oral tablet, taken once daily on an empty stomach
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
Avoidance of a platelet transfusion before, during and up to 7 days after an elective invasive procedure
✓ The study showed what it set out to show
Who was studied
ELEVATE (NCT00678587) — eltrombopag before procedures in cirrhosis
How many people
292
Study design
Randomised double-blind placebo-controlled trial, terminated early for safety
Compared against
A dummy treatment
Kind of result
A number that stands in for health
What was found
72% versus 19%, p < 0.001
Repeated elsewhere
Unreplicated
What this does not prove. Meeting one endpoint in one population does not carry to other goals or other people.
The limits of this study, and where it came from
Main limit. The key secondary endpoint, WHO grade 2 or higher bleeding, showed no significant difference (17% versus 23%). Portal venous system thrombosis occurred in 6 patients on eltrombopag against 1 on placebo, which terminated the study.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Oral tablet, taken once daily on an empty stomach
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
22% versus 10%, odds ratio 3.2 (95% CI 1.3 to 7.8), p = 0.01
Repeated elsewhere
Unreplicated
What this does not prove. Meeting one endpoint in one population does not carry to other goals or other people.
The limits of this study, and where it came from
Main limit. Open-label. Somatic mutations rose from 29% and 31% at baseline to 66% and 55% at six months in the two arms without affecting response or two-year outcome; a karyotypic abnormality classified as myelodysplastic syndrome developed in 1 and 2 patients at a median 24 months.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Oral tablet, taken once daily on an empty stomach
Interval reported. 95% CI 1
Written into the record, not signed off as a reviewed claim.
Platelet response and safety in myelodysplastic syndrome with thrombocytopenia receiving azacitidine
✗ The study did not show it
Who was studied
SUPPORT — eltrombopag plus azacitidine in intermediate-1, intermediate-2 and high risk myelodysplastic syndrome
How many people
356
Study design
Randomised double-blind placebo-controlled multicentre trial, terminated early
Compared against
A dummy treatment
Kind of result
A number that stands in for health
What was found
Death 32% versus 29%, hazard ratio 1.42 (95% CI 0.97 to 2.08); progression to acute myeloid leukaemia 12% versus 6%, hazard ratio 2.66 (95% CI 1.31 to 5.41)
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. Terminated for lack of efficacy and for safety, including increased progression to acute myeloid leukaemia. The label now carries an explicit limitation of use excluding myelodysplastic syndrome.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Oral tablet, taken once daily on an empty stomach
Interval reported. 95% CI 0
Written into the record, not signed off as a reviewed claim.
RNAWiki holds 4 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 it changes in the body◇Read from sources, not yet reviewed
The path through the body
From what a person takes to what changes. A solid line is a step measured in people. A dashed line is a step nobody has measured that way.
Start
Eltrombopag
What a person takes: Oral tablet, taken once daily on an empty stomach.
The measurement behind this step
A film-coated tablet containing eltrombopag olamine equivalent to 12.5, 25, 50 or 75 mg of free acid, taken once a day. It must be separated by several hours from dairy products, antacids and mineral supplements, because it chelates calcium, magnesium, aluminium, iron, selenium and zinc, and absorption falls sharply when they are present. A powder for oral suspension exists for young children.
Getting in
A daily tablet, taken well away from food
Swallowed once a day, but it must be separated by several hours from dairy, antacids and mineral supplements, because it grabs onto calcium, magnesium and iron and stops being absorbed.
╌╌Described, with no measurement named. No measurement is named for this step, so it is drawn as an unconfirmed link.
The measurement behind this step
The free acid is practically insoluble in aqueous buffer from pH 1 to 7.4, which is why the marketed form is the bis-olamine salt. The molecule chelates polyvalent cations, so co-administration with calcium-rich food, antacids or mineral supplements substantially reduces absorption. The tablets contain eltrombopag olamine equivalent to 12.5, 25, 50 or 75 mg of free acid.
It reaches the bone marrow and enters the cell membrane itself
Its target is not on the outside of the cell but inside the greasy membrane that surrounds it, so the molecule has to partition into that membrane rather than dock onto a surface.
╌╌Described, with no measurement named. No measurement is named for this step, so it is drawn as an unconfirmed link.
The measurement behind this step
The binding site is in the transmembrane domain of c-Mpl, which is why a lipophilic small molecule can do a job that otherwise requires a protein hormone. Its distribution and its food interaction both follow from the same physical chemistry: high lipophilicity and avid chelation of divalent cations.
It switches the receptor on at a different place from the hormone
Thrombopoietin docks onto the outside of the receptor. Eltrombopag pushes on a part buried in the membrane. Because they act at different places, the two can work together rather than getting in each other’s way.
──Measured in people. Written by a person from the study named beside the step. Not signed off as a reviewed claim.
The measurement behind this step
The label states that eltrombopag interacts with the transmembrane domain of the human thrombopoietin receptor and initiates signalling cascades that induce megakaryocyte proliferation and differentiation. Preclinical work confirms it acts without competing with thrombopoietin, and that it activates STAT signalling only in human and chimpanzee platelets among the species tested.
The signal tells stem cells in the marrow to become the giant platelet-producing cells, and tells the ones already there to mature further.
──Measured in people. Written by a person from the study named beside the step. Not signed off as a reviewed claim.
The measurement behind this step
Receptor engagement activates the STAT and mitogen-activated protein kinase pathways, driving proliferation and differentiation of primary human CD34-positive bone marrow cells into CD41-positive megakaryocytes. The effect requires receptor expression, so it is confined to the megakaryocyte lineage and the progenitors that carry c-Mpl.
The platelet count climbs over about two weeks — and falls back just as fast
Counts rise steadily and peak roughly a fortnight after starting. Stop the drug and they are back to baseline within about another fortnight. It is a tap, not a cure.
──Measured in people. Written by a person from the study named beside the step. Not signed off as a reviewed claim.
The measurement behind this step
The label reports dose-dependent increases in platelet count reaching a maximum approximately two weeks after initiation and returning to baseline within approximately two weeks after the last dose. That reversibility is why treatment is generally continuous, and why patients in the pivotal trials who stopped simply relapsed.
Whether that stops bleeding depends entirely on why the count was low
In immune thrombocytopenia, rescue treatment and serious bleeding both fell. In cirrhosis, the same rise in count avoided transfusions and changed bleeding not at all, while causing clots in the portal vein.
──Measured in people. Written by a person from the study named beside the step. Not signed off as a reviewed claim.
The measurement behind this step
RAISE: serious bleeding in <1% against 7% on placebo, rescue treatment in 18% against 40%. ELEVATE: transfusion avoided in 72% against 19% (p<0.001), WHO grade 2 or higher bleeding 17% against 23% — not significant — and portal venous thrombosis in 6 patients against 1, terminating the study. The pharmacology is identical in both; the clinical consequence is not.
No suggested links are held for this record, so nothing is hidden from this path.
What we know
The record describes 6 steps from taking it to an effect in a person.
How we know it
Each step names the study behind it in the technical detail beside it.
What this does not prove
A change inside the body is a reason to look. It is not a result in a person.
Why it matters
A reader can see exactly where the chain stops being measured in people.
What we still do not know
No reviewed claim binds any of these steps to a named population.
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.
People with chronic immune thrombocytopenia who have already failed steroids, immunoglobulin or splenectomy, and people with severe aplastic anaemia. It is taken by mouth, daily, often for years.
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 safety and effectiveness of eltrombopag in pediatric patients 6 years and older with persistent or chronic ITP were evaluated in two double-blind, placebo-controlled trials [see Adverse Reactions ( 6.1 ), Clinical Studies ( 14.1 )] .”
US prescribing information · ed51e463-7a03-4858-bbd2-882ce4753d5a · read 2026-08-30
On older people, the label states: “Of the 106 patients in two randomized clinical trials of eltrombopag 50 mg in persistent or chronic ITP, 22% were 65 years of age and over, while 9% were 75 years of age and over.”
US prescribing information · ed51e463-7a03-4858-bbd2-882ce4753d5a · read 2026-08-30
On people who are pregnant, the label states: “Risk Summary Available data from a small number of published case reports and postmarketing experience with eltrombopag use in pregnant women are insufficient to assess any drug-associated risks for major birth defects, miscarriage, or adverse maternal or fetal outcomes.”
US prescribing information · ed51e463-7a03-4858-bbd2-882ce4753d5a · read 2026-08-30
On people who are breastfeeding, the label states: “Risk Summary There are no data regarding the presence of eltrombopag or its metabolites in human milk, the effects on the breastfed child, or the effects on milk production.”
US prescribing information · ed51e463-7a03-4858-bbd2-882ce4753d5a · read 2026-08-30
On people with reduced liver function, the label states: “In a clinical trial in patients with severe aplastic anemia who had not received prior definitive immunosuppressive therapy, patients with baseline ALT or AST > 5 x ULN were ineligible to participate [see Dosage and Administration ( 2.3), Warnings and Precautions ( 5.2 ), Clinical Pharmacology ( 12.3 )] .”
US prescribing information · ed51e463-7a03-4858-bbd2-882ce4753d5a · read 2026-08-30
Where the result stopped carrying
ELEVATE was terminated early after six portal venous thromboses against one on placebo, having met its transfusion endpoint and missed its bleeding endpoint
The myelodysplastic syndrome trial was terminated for lack of efficacy and for increased progression to acute myeloid leukaemia, and the label now excludes that population outright
Two controlled trials in chronic hepatitis C found ascites and encephalopathy in 7% on eltrombopag plus antivirals against 4% on placebo plus antivirals, producing half of the boxed warning
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.
The product may not be what it says
Contents of a sold product are not always what the label states.
On this record: This is sold as a supplement, so no agency checked what is in a given tub before it was sold.
Other reasons RNAWiki checked and found nothing for (10)
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.
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
Oral tablet, taken once daily on an empty stomach
✎ 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: S6.
No source is stored against this line.
What is in the pack
5, 25, 50 or 75 mg of free acid, taken once a day.
✎ 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. The rest of the recorded wording: It must be separated by several hours from dairy products, antacids and mineral supplements, because it chelates calcium, magnesium, aluminium, iron, selenium and zinc, and absorption falls sharply when they are present. A powder for oral suspension exists for young children.
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◇Read from sources, not yet reviewed
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.
·Worth asking a clinician aboutWritten into the record from the studies named on this page
Carries a boxed warning with two parts: risk of hepatic decompensation when combined with interferon and ribavirin in chronic hepatitis C, and risk of severe and potentially life-threatening hepatotoxicity generally, with liver function monitoring required before and during therapy. The label also warns of increased risk of death and progression of myelodysplastic syndrome to acute myeloid leukaemia, and of thrombotic and thromboembolic complications including portal vein thrombosis in chronic liver disease. It inhibits UGT1A1 and OATP1B1, which can produce indirect hyperbilirubinaemia that is not itself liver injury. Platelet counts must be monitored regularly, both to guide dosing and because they fall back to baseline within about two weeks of stopping.
Nobody counted how many people took this and were fine, so this cannot be turned into a rate.
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
Oral tablet, taken once daily on an empty stomach
✎ 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
5, 25, 50 or 75 mg of free acid, taken once a day.
✎ 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 which forms are sold. The rest of the recorded wording: It must be separated by several hours from dairy products, antacids and mineral supplements, because it chelates calcium, magnesium, aluminium, iron, selenium and zinc, and absorption falls sharply when they are present. A powder for oral suspension exists for young children.
No source is stored against this line.
What is recorded as being sold
1 product lists this as an active ingredient in the United States drug directory. 1 of them contain it and nothing else.
FDA National Drug Code directory · 11722-071 · read 2026-08-29
They are sold as powder.
FDA National Drug Code directory · 11722-071 · 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 · ed51e463-7a03-4858-bbd2-882ce4753d5a · read 2026-08-29
Those labels are classed as human prescription drug.
US prescribing information · ed51e463-7a03-4858-bbd2-882ce4753d5a · read 2026-08-29
ALVAIZ is oral at 3 DOSAGE FORMS AND STRENGTHS 9 mg, round, biconvex, blue film-coated tablets, debossed with “TV” on one side and “Z9” on the other side. 18 mg, round, biconvex, off-white film-coated tablets debossed with “TV” on one si…, recorded as fda label in effect 2026-01-01 in the United States.
US prescribing information · ed51e463-7a03-4858-bbd2-882ce4753d5a · 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 Eltrombopag 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
Claims that go past the evidence
Things said about this substance that sound reasonable, and the exact step that is missing between the evidence and the claim.
✗Goes past the evidence
That raising the platelet count reduces bleeding wherever the count is low — in cirrhosis it demonstrably did not, and the same manoeuvre caused portal vein thrombosis
Why it sounds right. It follows from something real on this page: a body step, an animal result or a related finding.
The missing step. Nobody measured this in people for this use, or the study that did measure it did not show it.
What would change this
A study in people, for this exact use, measuring this exact thing, that a reviewer signs off.
RNAWiki has not yet published a reviewed conclusion for this use.
✗Goes past the evidence
That a receptor agonist which drives megakaryocyte proliferation is safe in a marrow that is already pre-malignant; in myelodysplastic syndrome the hazard ratio for progression to acute myeloid leukaemia was 2.66
Why it sounds right. It follows from something real on this page: a body step, an animal result or a related finding.
The missing step. Nobody measured this in people for this use, or the study that did measure it did not show it.
What would change this
A study in people, for this exact use, measuring this exact thing, that a reviewer signs off.
RNAWiki has not yet published a reviewed conclusion for this use.
✗Goes past the evidence
That the licensed hepatitis C indication describes current practice, when it specifies interferon-based therapy that has not been standard for over a decade
Why it sounds right. It follows from something real on this page: a body step, an animal result or a related finding.
The missing step. Nobody measured this in people for this use, or the study that did measure it did not show it.
What would change this
A study in people, for this exact use, measuring this exact thing, that a reviewer signs off.
RNAWiki has not yet published a reviewed conclusion for this use.
✗Goes past the evidence
That efficacy demonstrated in human cell culture and in chimpanzees can be corroborated in any conventional animal model — the drug does not work in any other species tested
Why it sounds right. It follows from something real on this page: a body step, an animal result or a related finding.
The missing step. Nobody measured this in people for this use, or the study that did measure it did not show it.
What would change this
A study in people, for this exact use, measuring this exact thing, that a reviewer signs off.
RNAWiki has not yet published a reviewed conclusion for this use.
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.
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 Eltrombopag 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.
Every line above can be traced to the study named beside it. Follow the link and read it.
Four in five responded against fewer than one in three on placebo
In plain words
In 197 patients with chronic immune thrombocytopenia, 79% on eltrombopag reached a platelet count in the target range at least once over six months, against 28% on placebo. Fewer needed rescue treatment and fewer had serious bleeds.
What was measured
Odds of achieving a platelet count of 50,000 to 400,000 per microlitre during six months of treatment
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
RAISE was a phase 3, double-blind, placebo-controlled trial in adults with previously treated immune thrombocytopenia of more than six months’ duration and baseline platelet counts below 30,000 per microlitre, randomised 2:1 to local standard of care plus 50 mg eltrombopag or matching placebo for six months. Of 197 randomised, 106 of 135 (79%) on eltrombopag responded at least once against 17 of 62 (28%) on placebo; the odds of responding across the treatment period gave an odds ratio of 8.2 (99% CI 3.59 to 18.73, p<0.0001). Concomitant immune thrombocytopenia treatment was reduced in 59% against 32% (p=0.016) and rescue treatment was needed by 18% against 40% (p=0.001). Serious bleeding events occurred in 1 of 135 (<1%) against 4 of 62 (7%). Three eltrombopag patients had thromboembolic events against none on placebo, and nine had mild alanine aminotransferase rises against two.
Written into the record, not signed off as a reviewed claim
In liver disease it avoided transfusions, changed no bleeding, and caused clots
In plain words
Given before procedures in cirrhosis, eltrombopag let 72% of patients avoid a platelet transfusion against 19% on placebo — and bleeding was no different. Six patients developed clots in the portal vein against one on placebo, and the trial was stopped early.
What was measured
Avoidance of platelet transfusion before, during and up to 7 days after an elective invasive procedure; WHO grade 2 or higher bleeding
Effect estimate
No reviewed effect estimate exists for this record.
Limits
This entry records a failure.
From the source
ELEVATE randomised 292 patients with chronic liver disease of diverse causes and platelet counts below 50,000 per cubic millimetre to eltrombopag 75 mg daily or placebo for 14 days before an elective invasive procedure. A platelet transfusion was avoided in 104 of 145 (72%) against 28 of 147 (19%), p<0.001. The key secondary endpoint, bleeding of WHO grade 2 or higher, showed no significant difference: 17% against 23%. Thrombotic events of the portal venous system occurred in 6 patients on eltrombopag against 1 on placebo, which caused the study to be terminated early. This is the cleanest natural experiment in the whole thrombopoietin agonist class: the surrogate moved enormously, the clinical endpoint did not move at all, and the harm was real.
Written into the record, not signed off as a reviewed claim
In myelodysplastic syndrome it increased leukaemia progression and death
In plain words
A trial adding eltrombopag to azacitidine in 356 patients with myelodysplastic syndrome was terminated. Progression to acute myeloid leukaemia was 12% against 6%, and deaths were 32% against 29%.
What was measured
Overall survival and incidence of progression to acute myeloid leukaemia in myelodysplastic syndrome
Effect estimate
No reviewed effect estimate exists for this record.
Limits
This entry records a failure.
From the source
The PROMACTA label reports a randomised, double-blind, placebo-controlled, multicentre trial in patients with IPSS intermediate-1, intermediate-2 or high risk myelodysplastic syndrome with thrombocytopenia receiving azacitidine plus either eltrombopag (n=179) or placebo (n=177), at 200 mg daily rising to a maximum of 300 mg for at least six cycles. It was terminated for lack of efficacy and for safety, including increased progression to acute myeloid leukaemia. Death occurred in 57 of 179 (32%) against 51 of 177 (29%), hazard ratio 1.42 (95% CI 0.97 to 2.08). Progression to acute myeloid leukaemia occurred in 21 of 179 (12%) against 10 of 177 (6%), hazard ratio 2.66 (95% CI 1.31 to 5.41). The label now carries an explicit limitation of use stating the drug is not indicated for myelodysplastic syndrome. A receptor agonist that drives proliferation of a marrow progenitor lineage was always going to raise this question in a pre-leukaemic marrow, and the trial answered it.
Source
PROMACTA (eltrombopag) prescribing information, section 5.3 Increased Risk of Death and Progression of Myelodysplastic Syndromes to Acute Myeloid Leukemia, and section 1.4 Limitations of Use
Role in the trial
Not matched to a registered study
Identity check
no open identity hold
Audit mark
caution
Review state
Written into the record, not signed off as a reviewed claim
Added to standard therapy in aplastic anaemia it doubled complete responses
In plain words
In 197 previously untreated patients with severe aplastic anaemia, adding eltrombopag to horse antithymocyte globulin and ciclosporin raised complete responses at three months from 10% to 22%, and the responses came about six months sooner.
What was measured
Haematologic complete response at 3 months in previously untreated severe aplastic anaemia
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
RACE was a prospective, investigator-led, open-label, multicentre randomised phase 3 trial comparing horse antithymocyte globulin plus ciclosporin with or without eltrombopag as front-line therapy. Complete response at three months, the primary endpoint, was 10% in 101 patients on immunosuppression alone against 22% in 96 patients with eltrombopag added, odds ratio 3.2 (95% CI 1.3 to 7.8, p=0.01). Overall response at six months was 41% against 68%, and median time to first response 8.8 months against 3.0 months. Severe adverse events were similar. At a median 24 months, a karyotypic abnormality classified as myelodysplastic syndrome developed in 1 patient against 2, and event-free survival was 34% against 46%. Somatic mutations were present at baseline in 29% and 31% and rose to 66% and 55% at six months without affecting response or two-year outcome. This is the strongest efficacy evidence the drug has, and the endpoint is a haematological response rather than survival.
Written into the record, not signed off as a reviewed claim
A licensed indication that outlived the therapy it was licensed to enable
In plain words
The label still carries an indication for raising platelets in hepatitis C so that interferon treatment can be given. Interferon has not been standard hepatitis C treatment for a decade, and the label says the drug has not been shown safe or effective with the drugs that replaced it.
What was measured
That a licensed indication reflects current practice; here it preserves a use case that the field abandoned more than a decade ago
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Section 1.2 of the PROMACTA label indicates the drug for thrombocytopenia in chronic hepatitis C to allow initiation and maintenance of interferon-based therapy. Section 1.4 states that safety and efficacy have not been established in combination with direct-acting antiviral agents used without interferon. Direct-acting antivirals displaced interferon-based regimens for hepatitis C from around 2014, so the indication describes a treatment pathway that has essentially ceased to exist, while the drug carries a boxed warning specifically about hepatic decompensation in that same population — ascites and encephalopathy in 7% on eltrombopag plus antivirals against 4% on placebo plus antivirals in two controlled trials. The indication remains on a label effective 18 December 2025.
Source
PROMACTA (eltrombopag) prescribing information, sections 1.2, 1.4 and 5.1, label effective 18 December 2025
Role in the trial
Not matched to a registered study
Identity check
no open identity hold
Audit mark
caution
Review state
Written into the record, not signed off as a reviewed claim
No animal can tell you whether it works
In plain words
Eltrombopag activates the platelet receptor in humans and chimpanzees and in no other species tested. There is no mouse or rat model of its efficacy, which is unusual for a small molecule and shaped what the early evidence could be.
What was measured
Species-specific STAT pathway activation in platelets, and platelet count increase in chimpanzees
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Preclinical characterisation showed that the compound depends on thrombopoietin receptor expression and activates STAT and MAP kinase signalling, and that measurements in platelets across several species indicated it specifically activates only the human and chimpanzee STAT pathways. In vivo activity was demonstrated by up to a 100% increase in platelet numbers in chimpanzees given 10 mg/kg per day orally for five days. The authors also report that it interacts with the receptor without competing with thrombopoietin, which is the pharmacological basis for the two acting additively. The consequence for the evidence base is that rodent toxicology cannot address efficacy, and that the transition from cell culture to human trials happened with an unusually thin animal layer in between.
Written into the record, not signed off as a reviewed claim
How many documents were read
8 documents were read for this substance.
RNAWiki source record
8 of them state the same proteinBinding, and they agree.
RNAWiki source record
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No registered study is classified as testing this substance.
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Suppression classes recorded: S6.
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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.
Withdrawn in United States, 2019, for "Cross Contamination with Other Products: product is being recalled due to possible cross-contamination with peanut flour." (openFDA drug enforcement Class I recall)
What the approval register records
1 approved application covers products containing this substance. The earliest was ANDA219121, approved 20260316 to DR REDDYS.
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What is not here
7 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.
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Felt, measured, or meaningful — found nothing in the sources checked.
How long anything takes — found nothing in the sources checked.
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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.
The older medicine-wide conclusion held in this record
Written for a clinical reader, and about the medicine as a whole rather than about one indication, population and set of trials. RNAWiki does not treat it as a programme conclusion and no reviewer has signed it off in that form. It is here word for word because it is what the record holds.
An oral molecule that jams itself into the transmembrane part of the thrombopoietin receptor and switches the platelet factory on without competing with the hormone that normally does it, raising the platelet count in about four out of five people with chronic immune thrombocytopenia against about one in four on placebo — a reliable effect that failed to reduce bleeding when it was finally measured in liver disease, caused portal vein thrombosis there, and raised both death and leukaemia progression in myelodysplastic syndrome.
Recorded evidence blocks (11)
Q2
On the Eltrombopag label: indicated for what?
"Eltrombopag tablet is a thrombopoietin receptor agonist indicated: • for the treatment of thrombocytopenia in adult and pediatric patients 1 year and older with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. Eltrombopag…": indications and usage on Eltrombopag's label. DailyMed label · 9af1e314-91ca-4ebc-ac12-f9fddc6d7902 · 2026-07-02
Q3
161 registered trials of Eltrombopag — at which phases?
Registered studies posting no result
104 of 161
161 registered studies of Eltrombopag: 86 phase2, 28 phase3, 27 phase1, 15 na or unstated, 13 phase4, 4 na, 1 early phase1. CLINICALTRIALS_SNAPSHOT · 2026-09-01
128 with a PubMed record
Show the evidence
phase2
86
phase3
28
phase1
27
na or unstated
15
phase4
13
na
4
11 more recorded rows
early phase1
1
completed
96
unknown
18
terminated
17
recruiting
11
not yet recruiting
8
active not recruiting
5
suspended
2
withdrawn
2
available
1
enrolling by invitation
1
recorded 2026-09-01 · last checked 2026-09-04
Q4
19 of Eltrombopag's trials stopped: safety, futility/efficacy, accrual/recruitment, funding/business, other?
safety (1), futility/efficacy (2), accrual/recruitment (6), funding/business (1) and other (9): Eltrombopag's stop wording, clustered. CLINICALTRIALS_SNAPSHOT · 2026-09-01
"GSK decision"; 19 of 161 registered studies
Show the evidence
Trial
NCT00678587
terminated; "GSK decision"
NCT00909363
terminated; "retirement of PI"
NCT01055600
withdrawn; "Protocol opened to recruitment in Nov 2009. No potential subjects identified in 5 years; therefore, study was terminated in Jul 2014 due to lack of feasibility"
NCT01194167
withdrawn; "Inability to identify eligible patients"
NCT01286038
terminated; "Lack of efficacy"
NCT01397149
terminated; "Insufficient recruitment"
13 further recorded trials
NCT01433978
terminated; "Study was terminated early due to significant enrollment challenges."
NCT01484314
terminated; "Low accrual."
NCT01491594
terminated; "Unable to accrue subjects to the study."
NCT01500538
terminated; "Poor recruitment rate"
NCT01550185
terminated; "Sponsor wanted study rewritten"
NCT01656252
terminated; "Study stopped per Novartis request due to futility from another study."
NCT01821625
terminated; "Interferon use for hepatitis C plummeted, eliminating the need for study drug."
NCT02010645
terminated; "PI Request"
NCT02446145
terminated; "Due to COVID-19 pandemia, recruitment was insufficient to reach required sample size in an acceptable time line. Furthermore, new drugs have recently been licensed resulting in alternative treatment regimens with a better prognosis."
NCT03718533
terminated; "In the context of COVID-19 pandemic, the benefit / risk ratio of the participation of a patient with pancytopenia could be compromised. The decision of this premature termination was not the consequence of any safety reason inherent to the…"
NCT03948529
terminated; "inclusions difficulties."
NCT04485416
suspended; "PI discretion; suspended while CAPA is reviewed"
NCT06630221
suspended; "Funding unavailable"
recorded 2026-09-01 · last checked 2026-09-04
Q5
Human studies of Eltrombopag used SB-497115-GR 12.5mg — over how long?
studies of Eltrombopag used the recorded amount. ClinicalTrials.gov · 2026-09-01
10 recorded entries; human; tablet; also "SB-497115-GR 12.5mg", "SB-497115-GR 25mg", "SB-497115-GR 12.5mg matching placebo"
Show the evidence
human
NCT00540423
SB-497115-GR 12.5mg
NCT00540423
SB-497115-GR 25mg
NCT00540423
SB-497115-GR 12.5mg matching placebo
NCT00540423
SB-497115-GR 50 mg
NCT00540423
SB-497115-GR 50mg
NCT00861601
tablet; eltrombopag 25 mg tablet
4 more recorded rows
humanNCT02148133
Eltrombopag 12.5 mg
humanNCT02148133
Eltrombopag 25 mg
humanNCT05466201
Eltrombopag 25 MG Oral Tablet
humanNCT06531018
Eltrombopag 25 MG
recorded 2026-09-01 · last checked 2026-09-04
Q6
Eltrombopag's half-life is 21 to 32 hours — which schedules were studied?
21 to 32 hours, the half-life Eltrombopag's label states: "Elimination The plasma elimination half-life of eltrombopag is approximately 21 to 32 hours in healthy subjects and 26 to 35 hours in patients with ITP." DailyMed label · 9af1e314-91ca-4ebc-ac12-f9fddc6d7902 · 2026-07-02
tmax 2 to 6 hours.
Show the evidence
half lifepharmacokinetics
21 to 32 hours; Elimination The plasma elimination half-life of eltrombopag is approximately 21 to 32 hours in healthy subjects and 26 to 35 hours in patients with ITP.
tmaxpharmacokinetics
2 to 6 hours; Absorption Eltrombopag is absorbed with a peak concentration occurring 2 to 6 hours after oral administration.
metabolismpharmacokinetics
Metabolism: Absorbed eltrombopag is extensively metabolized, predominantly through pathways, including cleavage, oxidation, and conjugation with glucuronic acid, glutathione, or cysteine.
recorded 2026-07-02 · last checked 2026-09-04
Q7
Which running trial of Eltrombopag could settle lifespan?
NCT03603795 measures Overal survival rate, reading out 2026-07-30.
1 open trial; n 110; "Study Impact on Outcome of Eltrombopag in Elderly Patients with Acute Myeloid Leukemia Receiving Induction Chemotherapy"
Show the evidence
TrialNCT03603795
"Study Impact on Outcome of Eltrombopag in Elderly Patients with Acute Myeloid Leukemia Receiving Induction Chemotherapy"; n 110; "Overal survival rate"; 2026-07-30
Q8
Which 47 trials of Eltrombopag posted no result?
Posted no result
47 of 47 completed trials
Registrations
NCT00110799, NCT00102739, NCT00102726, NCT00359463, NCT00442871 and NCT00688272, and 41 more
Completion dates
oldest 2006-11; newest 2024-07-16
Show the evidence
Trial
NCT00110799
2006-11
NCT00102739
2007-01
NCT00102726
2007-02
NCT00359463
2007-03-07
NCT00442871
2008-01-03
NCT00688272
2008-09-25
14 further recorded trials
NCT00833378
2009-03-11
NCT01235988
2009-10
NCT01236014
2009-10
NCT01072162
2010-04-07
NCT00358540
2010-10-22
NCT00888901
2011-05
NCT01439321
2011-06
NCT01657552
2012-10-26
NCT00643929
2013-03
NCT01481220
2013-05
NCT01458080
2013-11
NCT01652599
2013-11
NCT00903422
2013-12-05
NCT01064336
2014-07
Q9
At the median, Eltrombopag's trials enrolled 36 people — anything larger?
Median enrolment
36
Largest enrolment
5797
Registered trials counted
160
Q10
What do 1746 spontaneous reports say about Eltrombopag — 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.
Eltrombopag appears in spontaneous reports to regulators. Across the 10 most-reported reaction terms, 1746 reaction mentions were counted: platelet count decreased 629; thrombocytopenia 193; platelet count increased 182; pulmonary embolism 145. FAERS via Open Targets · CHEMBL3989691 · 2026-06-24
Show the evidence
platelet count decreased
629
thrombocytopenia
193
platelet count increased
182
pulmonary embolism
145
deep vein thrombosis
113
haemorrhage
100
4 more recorded rows
thrombosis
100
therapy non-responder
97
contusion
94
epistaxis
93
recorded 2026-06-24 · last checked 2026-09-04
Q11
Which 10 reactions does Eltrombopag's label not list?
7.2 Transporters Use caution when concomitantly administering eltrombopag and drugs that are substrates of OATP1B1 (e.g., atorvastatin, bosentan, ezetimibe, fluvastatin, glyburide, olmesartan, pitavastatin, pravastatin, rosuvastatin, repaglinide, rifampin, simvastatin acid, SN-38 [active metabolite of irinotecan], valsartan) or breast cancer resistance protein (BCRP) (e.g., imatinib, irinotecan,…
drug_interactions
Monitor patients closely for signs and symptoms of excessive exposure to the drugs that are substrates of OATP1B1 or BCRP and consider reduction of the dose of these drugs, if appropriate.
pharmacokinetics
Eltrombopag is a substrate of BCRP, but is not a substrate for P-glycoprotein (P-gp) or OATP1B1.
pharmacokinetics
In vitro studies suggest that CYP1A2 and CYP2C8 are responsible for the oxidative metabolism of eltrombopag.
pharmacokinetics
UGT1A1 and UGT1A3 are responsible for the glucuronidation of eltrombopag.
pharmacokinetics
Effect of Cyclosporine on Eltrombopag: The coadministration of a single dose of eltrombopag (50 mg) with a single dose of an OATP and BCRP inhibitor cyclosporine (200 mg or 600 mg) decreased plasma eltrombopag AUC 0-INF by 18% to 24% and C max by 25% to 39%.
2 more recorded rows
Interaction statementpharmacokinetics
Effect of Eltrombopag on Other Drugs Effect of Eltrombopag on Cytochrome P450 Enzymes Substrates: The coadministration of multiple doses of eltrombopag (75 mg once daily for 7 days) did not result in the inhibition or induction of the metabolism of a combination of probe substrates for CYP1A2 (caffeine), CYP2C19 (omeprazole), CYP2C9 (flurbiprofen), or CYP3A4 (midazolam) in humans.
Interaction statementpharmacokinetics
Effect of Eltrombopag on Rosuvastatin: The coadministration of multiple doses of eltrombopag (75 mg once daily for 5 days) with a single dose of rosuvastatin (OATP1B1 and BCRP substrate;
Withdrawn in United States, 2019, for "Cross Contamination with Other Products: product is being recalled due to possible cross-contamination with peanut flour." (openFDA drug enforcement Class I recall)
ChEMBL 37 — CC BY-SA 3.0 Unported · 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 · openFDA enforcement — 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: 7 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
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