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Effects of Transfusion Thresholds on Neurocognitive Outcome of Extremely Low Birth Weight Infants

Phase 4
Completed
Conditions
Anemia of Prematurity
Interventions
Other: red blood cell transfusion
Registration Number
NCT01393496
Lead Sponsor
University Hospital Tuebingen
Brief 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.

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.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
1013
Inclusion Criteria
  • Infants with a birth weight of 400 - 999g
Exclusion Criteria
  • 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

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
"liberal" transfusion triggersred blood cell transfusion"liberal" guidelines for red blood cell transfusions
"restrictive" transfusion triggersred blood cell transfusion"restrictive" guidelines for red blood cell transfusions
Primary Outcome Measures
NameTimeMethod
Incidence of death or major neurodevelopmental impairment24 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

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (17)

Children's Hospital

🇩🇪

Stuttgart, Germany

University Hospital of Aachen

🇩🇪

Aachen, Germany

Neonatalklinikken

🇩🇰

Copenhagen, Denmark

Vivantes Children's Hospital

🇩🇪

Berlin, Germany

University Children's Hospital

🇩🇪

Ulm, Germany

Charité University Children's Hospital

🇩🇪

Berlin, Germany

University of Duesseldorf

🇩🇪

Duesseldorf, Germany

Helios Children's Hospital

🇩🇪

Erfurt, Germany

University of Greifswald

🇩🇪

Greifswald, Germany

Children's Hospital Eppendorf

🇩🇪

Hamburg, Germany

Children's Hospital Altona

🇩🇪

Hamburg, Germany

University of Luebeck

🇩🇪

Luebeck, Germany

University Hospital of Tuebingen

🇩🇪

Tuebingen, Germany

Children's Hospital St. Hedwig

🇩🇪

Regensburg, Germany

University Hospital of Marburg

🇩🇪

Marburg, Germany

DRK Kinderklinik

🇩🇪

Siegen, Germany

University Hospital of Leipzig

🇩🇪

Leipzig, Germany

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