Anemia of PrematurityPhase 4ETTNONCT01393496
Effects of Transfusion Thresholds on Neurocognitive Outcome of Extremely Low Birth Weight Infants
A Phase 4 study testing red blood cell transfusion in people with Anemia of Prematurity, run by University Hospital Tuebingen at 32 sites in 2 countries.
Trial summary
To compare the effect of restrictive versus liberal red blood cell transfusion thresholds on long-term neurodevelopmental outcome in extremely low birth weight infants. Read more about this study
- Who is it for?
- Ages 0 to 3 days
- Conditions the study is for
- Anemia of Prematurity
- What is being tested?
- red blood cell transfusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1,013 participants
- Where
- 32 sitesin 2 countries
- Study dates
- Jul 2011to 31 Oct 2018
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 3 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Infants with a birth weight of 400 - 999g
Who cannot join
- Missing written parental consent.
- Gestational age > 29 + 6/7 weeks
- Major congenital anomalies (including chromosomal aberrations, cyanotic congenital heart defects, syndromes likely affecting long-term outcome, and major congenital malformations requiring surgical correction during newborn period).
- Infants who died before 48 hours, infants in whom the clinical decision to withhold intensive care was made, infants who were not considered viable
- Participation in another study with ongoing use of an unlicensed investigational product from 28 days before study enrollment until the end of the study
Trial locations and contacts
32 sites in 2 countries.
32 sites in 2 countries
Germany31 sites
University Hospital of Aachen
Aachen, 52074
Children's Hospital
Augsburg, 86156
Vivantes Children's Hospital
Berlin, 12351
Charité University Children's Hospital
Berlin, 13353
University Children's Hospital
Bochum, 44791
Children's Hospital
Cologne, 50375
University Children's Hospital
Cologne, 50924
Children's Hospital
Datteln, 45711
University Children's Hospital
Dresden, 01307
University of Duesseldorf
Düsseldorf, 40225
Helios Children's Hospital
Erfurt, 99089
Children's Hospital
Erlangen, 91054
University Children's Hospital
Essen, 45122
University Children's Hospital
Frankfurt, 60590
University Children's Hospital
Giessen, 35385
University of Greifswald
Greifswald, 17475
Children's Hospital Eppendorf
Hamburg, 20246
Children's Hospital Altona
Hamburg, 22763
Children's Hospital
Hanover, 30625
University Hospital of Leipzig
Leipzig, 04103
University of Luebeck
Lübeck, 23538
University Children's Hospital
Magdeburg, 39120
University Hospital of Marburg
Marburg, 35033
University Children's Hospital
Munich, 80337
University Children's Hospital
Münster, 48149
Children's Hospital
Nuremberg, 90471
Children's Hospital St. Hedwig
Regensburg, 93049
DRK Kinderklinik
Siegen, 57072
Children's Hospital
Stuttgart, 70176
University Hospital of Tuebingen
Tübingen, 72076
University Children's Hospital
Ulm, 89075
About this study
Official title: Effects of Transfusion Thresholds on Neurocognitive Outcome of Extremely Low Birth Weight Infants (ETTNO)- a Blinded Randomized Controlled Multicenter Trial
To compare the effect of restrictive versus liberal red blood cell transfusion thresholds on long-term neurodevelopmental outcome in extremely low birth weight infants.
Read the detailed description
Extremely low birth weight (ELBW) infants uniformly develop anemia of prematurity and frequently require multiple red blood cell transfusions (RBCT) during neonatal intensive care. The criteria currently applied to indicate RBCT in this population are based on expert opinion rather than evidence and conclusive data of long-term effects of RBCT practices do not exist. Both, giving RBCT to improve oxygen carrying capacity and restricting RBCT to avoid RBCT associated risks and costs potentially impair long-term development. The proposed blinded randomized controlled trial was designed and will be powered to compare the effect of restrictive versus liberal red blood cell transfusion guidelines on long-term neurodevelopmental outcome in ELBW infants. ELBW infants will be randomized to receive RBCT according to liberal or restrictive RBCT guidelines, which both reflect current practice in Germany and aim for a clinically relevant difference in mean hemoglobin concentrations. The primary outcome measure is the incidence of death or major neurodevelopmental impairment determined at 24 months of age corrected for prematurity. Key secondary outcomes are the incidences of individual components of the composite primary outcome, the mental and physical developmental index scores of the Bayley Scales of Infant Development (II edition), and growth. Safety analyses will assess the incidences of all major diseases of prematurity. The results of this trial may help to improve the quality of life of these patients and reduce long-term health care costs.
- Conditions
- Anemia of Prematurity
- Being tested
- red blood cell transfusion
- Keywords
- prematurity, anemia, transfusion
- Medical subject headings
- Anemia, Premature Birth
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
red blood cell transfusionOther
Implementation of "liberal" guidelines for red blood cell transfusions in extremely low birth weight infants
red blood cell transfusionOther
Implementation of "restrictive" guidelines for red blood cell transfusions in extremely low birth weight infants
| Study group | Role | What they receive |
|---|---|---|
| "liberal" transfusion triggers | Experimental | "liberal" guidelines for red blood cell transfusions Receives: Other: red blood cell transfusion |
| "restrictive" transfusion triggers | Active comparator | "restrictive" guidelines for red blood cell transfusions Receives: Other: red blood cell transfusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of death or major neurodevelopmental impairment
The primary outcome measure of this study will be the incidence of death or major neurodevelopmental impairment determined at 24 months of age corrected for prematurity (where major neurodevelopmental impairment is defined as any of the following: cognitive delay defined as mental developmental index (MDI) score of the Bayley Scales of Infant Development (2nd edition) < 85, cerebral palsy, or severe visual or hearing impairment
Measured: 24 months of age corrected for prematurity
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jul 2011 (estimated)
- Study startJul 2011
- Primary completion30 Apr 2017
- Study completion31 Oct 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Anemia of PrematurityStudies of the same condition, open and under way.
- More trials run by University Hospital TuebingenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 1,013 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital Tuebingen
- Sponsor type
- University, hospital or other
- Collaborators
- German Research Foundation
- Responsible party
- Principal investigator, Axel Franz, University Hospital Tuebingen
- Investigators
- Axel R Franz, MD, Principal investigator, University Hospital of Tuebingen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01393496
- Sponsor's protocol ID
- DFG Fr 1455/6-1
- Other IDs
- 2010-021576-28
- Acronym
- ETTNO
- Status last verified
- Sep 2021
- First submitted
- 12 Jul 2011
- Last updated
- 16 Sep 2021
- Results posted
- No
- Data monitoring committee
- Yes
Publications
ETTNO Investigators. The 'Effects of Transfusion Thresholds on Neurocognitive Outcome of Extremely Low Birth-Weight Infants (ETTNO)' Study: Background, Aims, and Study Protocol. Neonatology. 2012 Jun;101(4):301-305. doi: 10.1159/000335030. Epub 2012 Jan 27.
PubMed 22298226Franz AR, Engel C, Bassler D, Rudiger M, Thome UH, Maier RF, Krageloh-Mann I, Kron M, Essers J, Buhrer C, Rellensmann G, Rossi R, Bittrich HJ, Roll C, Hohn T, Ehrhardt H, Avenarius S, Korner HT, Stein A, Buxmann H, Vochem M, Poets CF; ETTNO Investigators. Effects of Liberal vs Restrictive Transfusion Thresholds on Survival and Neurocognitive Outcomes in Extremely Low-Birth-Weight Infants: The ETTNO Randomized Clinical Trial. JAMA. 2020 Aug 11;324(6):560-570. doi: 10.1001/jama.2020.10690.
PubMed 32780138Carson JL, Stanworth SJ, Dennis JA, Fergusson DA, Pagano MB, Roubinian NH, Turgeon AF, Valentine S, Trivella M, Doree C, Hebert PC. Transfusion thresholds and other strategies for guiding red blood cell transfusion. Cochrane Database Syst Rev. 2025 Oct 20;10(10):CD002042. doi: 10.1002/14651858.CD002042.pub6.
PubMed 41114449Franz AR, Engel C, Bassler D, Rudiger M, Thome UH, Maier RF, Krageloh-Mann I, Essers J, Buhrer C, Bittrich HJ, Roll C, Hohn T, Ehrhardt H, Boettger R, Korner HT, Stein A, Neuberger P, Henriksen TB, Greisen G, Poets CF; ETTNO Investigators. Effects of liberal versus restrictive transfusion strategies on intermittent hypoxaemia in extremely low birthweight infants: secondary analyses of the ETTNO randomised controlled trial. Arch Dis Child Fetal Neonatal Ed. 2025 Oct 17;110(6):564-570. doi: 10.1136/archdischild-2024-327643.
PubMed 40139741Radford M, Estcourt LJ, Sirotich E, Pitre T, Britto J, Watson M, Brunskill SJ, Fergusson DA, Doree C, Arnold DM. Restrictive versus liberal red blood cell transfusion strategies for people with haematological malignancies treated with intensive chemotherapy or radiotherapy, or both, with or without haematopoietic stem cell support. Cochrane Database Syst Rev. 2024 May 23;5(5):CD011305. doi: 10.1002/14651858.CD011305.pub3.
PubMed 38780066Carson JL, Stanworth SJ, Dennis JA, Trivella M, Roubinian N, Fergusson DA, Triulzi D, Doree C, Hebert PC. Transfusion thresholds for guiding red blood cell transfusion. Cochrane Database Syst Rev. 2021 Dec 21;12(12):CD002042. doi: 10.1002/14651858.CD002042.pub5.
PubMed 34932836
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effects of Transfusion Thresholds on Neurocognitive Outcome of Extremely Low Birth Weight Infants (ETTNO)- a Blinded Randomized Controlled Multicenter Trial
- ClinicalTrials.gov ID
- NCT01393496
- Acronym / sponsor ID
- ETTNO / DFG Fr 1455/6-1
- Conditions
- Anemia of Prematurity
- Keywords
- prematurity, anemia, transfusion
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 1,013 participants (actual)
- Lead sponsor
- University Hospital TuebingenUniversity, hospital or other
- Collaborators
- German Research Foundation
- Study start
- Jul 2011
- Primary completion
- 30 Apr 2017 (actual)
- Study completion
- 31 Oct 2018 (actual)
- First posted
- 13 Jul 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To compare the effect of restrictive versus liberal red blood cell transfusion thresholds on long-term neurodevelopmental outcome in extremely low birth weight infants.
Detailed description
Extremely low birth weight (ELBW) infants uniformly develop anemia of prematurity and frequently require multiple red blood cell transfusions (RBCT) during neonatal intensive care. The criteria currently applied to indicate RBCT in this population are based on expert opinion rather than evidence and conclusive data of long-term effects of RBCT practices do not exist. Both, giving RBCT to improve oxygen carrying capacity and restricting RBCT to avoid RBCT associated risks and costs potentially impair long-term development. The proposed blinded randomized controlled trial was designed and will be powered to compare the effect of restrictive versus liberal red blood cell transfusion guidelines on long-term neurodevelopmental outcome in ELBW infants. ELBW infants will be randomized to receive RBCT according to liberal or restrictive RBCT guidelines, which both reflect current practice in Germany and aim for a clinically relevant difference in mean hemoglobin concentrations. The primary outcome measure is the incidence of death or major neurodevelopmental impairment determined at 24 months of age corrected for prematurity. Key secondary outcomes are the incidences of individual components of the composite primary outcome, the mental and physical developmental index scores of the Bayley Scales of Infant Development (II edition), and growth. Safety analyses will assess the incidences of all major diseases of prematurity. The results of this trial may help to improve the quality of life of these patients and reduce long-term health care costs.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| "liberal" transfusion triggers | Experimental | Other: red blood cell transfusion"liberal" guidelines for red blood cell transfusions |
| "restrictive" transfusion triggers | Active comparator | Other: red blood cell transfusion"restrictive" guidelines for red blood cell transfusions |
Who may qualify
- Ages
- 0 to 3 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- Infants with a birth weight of 400 - 999g
Key exclusion criteria (5 listed)
- Missing written parental consent.
- Gestational age > 29 + 6/7 weeks
- Major congenital anomalies (including chromosomal aberrations, cyanotic congenital heart defects, syndromes likely affecting long-term outcome, and major congenital malformations requiring surgical correction during newborn period).
- Infants who died before 48 hours, infants in whom the clinical decision to withhold intensive care was made, infants who were not considered viable
- Participation in another study with ongoing use of an unlicensed investigational product from 28 days before study enrollment until the end of the study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| red blood cell transfusionImplementation of "liberal" guidelines for red blood cell transfusions in extremely low birth weight infants | Other | "liberal" transfusion triggers |
| red blood cell transfusionImplementation of "restrictive" guidelines for red blood cell transfusions in extremely low birth weight infants | Other | "restrictive" transfusion triggers |
Primary outcome
Incidence of death or major neurodevelopmental impairment
Time frame: 24 months of age corrected for prematurity
The primary outcome measure of this study will be the incidence of death or major neurodevelopmental impairment determined at 24 months of age corrected for prematurity (where major neurodevelopmental impairment is defined as any of the following: cognitive delay defined as mental developmental index (MDI) score of the Bayley Scales of Infant Development (2nd edition) < 85, cerebral palsy, or severe visual or hearing impairment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2021.
- Background: PubMed 22298226ETTNO Investigators. The 'Effects of Transfusion Thresholds on Neurocognitive Outcome of Extremely Low Birth-Weight Infants (ETTNO)' Study: Background, Aims, and Study Protocol. Neonatology. 2012 Jun;101(4):301-305. doi: 10.1159/000335030. Epub 2012 Jan 27.
- Results publication: PubMed 32780138Franz AR, Engel C, Bassler D, Rudiger M, Thome UH, Maier RF, Krageloh-Mann I, Kron M, Essers J, Buhrer C, Rellensmann G, Rossi R, Bittrich HJ, Roll C, Hohn T, Ehrhardt H, Avenarius S, Korner HT, Stein A, Buxmann H, Vochem M, Poets CF; ETTNO Investigators. Effects of Liberal vs Restrictive Transfusion Thresholds on Survival and Neurocognitive Outcomes in Extremely Low-Birth-Weight Infants: The ETTNO Randomized Clinical Trial. JAMA. 2020 Aug 11;324(6):560-570. doi: 10.1001/jama.2020.10690.
- Derived from this study: PubMed 41114449Carson JL, Stanworth SJ, Dennis JA, Fergusson DA, Pagano MB, Roubinian NH, Turgeon AF, Valentine S, Trivella M, Doree C, Hebert PC. Transfusion thresholds and other strategies for guiding red blood cell transfusion. Cochrane Database Syst Rev. 2025 Oct 20;10(10):CD002042. doi: 10.1002/14651858.CD002042.pub6.
- Derived from this study: PubMed 40139741Franz AR, Engel C, Bassler D, Rudiger M, Thome UH, Maier RF, Krageloh-Mann I, Essers J, Buhrer C, Bittrich HJ, Roll C, Hohn T, Ehrhardt H, Boettger R, Korner HT, Stein A, Neuberger P, Henriksen TB, Greisen G, Poets CF; ETTNO Investigators. Effects of liberal versus restrictive transfusion strategies on intermittent hypoxaemia in extremely low birthweight infants: secondary analyses of the ETTNO randomised controlled trial. Arch Dis Child Fetal Neonatal Ed. 2025 Oct 17;110(6):564-570. doi: 10.1136/archdischild-2024-327643.
- Derived from this study: PubMed 38780066Radford M, Estcourt LJ, Sirotich E, Pitre T, Britto J, Watson M, Brunskill SJ, Fergusson DA, Doree C, Arnold DM. Restrictive versus liberal red blood cell transfusion strategies for people with haematological malignancies treated with intensive chemotherapy or radiotherapy, or both, with or without haematopoietic stem cell support. Cochrane Database Syst Rev. 2024 May 23;5(5):CD011305. doi: 10.1002/14651858.CD011305.pub3.
- Derived from this study: PubMed 34932836Carson JL, Stanworth SJ, Dennis JA, Trivella M, Roubinian N, Fergusson DA, Triulzi D, Doree C, Hebert PC. Transfusion thresholds for guiding red blood cell transfusion. Cochrane Database Syst Rev. 2021 Dec 21;12(12):CD002042. doi: 10.1002/14651858.CD002042.pub5.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Axel R Franz, MD principal investigator
University Hospital of Tuebingen
32 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing red blood cell transfusion in people with Anemia of Prematurity, run by University Hospital Tuebingen at 32 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Sep 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 32 sites in Germany, Denmark. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of death or major neurodevelopmental impairment (time frame: 24 months of age corrected for prematurity). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01393496, which also lists 6 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Sep 2021.