A randomised trial of granulocyte-macrophage colony-stimulating factor for neonatal sepsis: childhood outcomes at 5 years
File(s)
Published version
Author(s)
Type
Journal Article
Abstract
Objective: We performed a randomised trial in very
preterm, small for gestational age (SGA) babies to
determine if prophylaxis with granulocyte macrophage
colony stimulating factor (GM-CSF) improves outcomes
(the PROGRAMS trial). GM-CSF was associated with
improved neonatal neutrophil counts, but no change in
other neonatal or 2-year outcomes. As subtle benefits in
outcome may not be ascertainable until school age we
performed an outcome study at 5 years.
Patients and methods: 280 babies born at 31 weeks
of gestation or less and SGA were entered into the trial.
Outcomes were assessed at 5 years to determine
neurodevelopmental and general health status and
educational attainment.
Results: We found no significant differences in
cognitive, general health or educational outcomes
between 83 of 106 (78%) surviving children in the GMCSF
arm compared with 81 of 110 (74%) in the control
arm. Mean mental processing composite (equivalent to
IQ) at 5 years were 94 (SD 16) compared with 95
(SD 15), respectively (difference in means −1 (95%CI −6
to 4), and similar proportions were in receipt of special
educational needs support (41% vs 35%; risk ratio 1.2
(95% CI 0.8 to 1.9)). Performance on Kaufmann-ABC
subscales and components of NEPSY were similar. The
suggestion of worse respiratory outcomes in the GM-CSF
group at 2 years was replicated at 5 years.
Conclusions: The administration of GM-CSF to very
preterm SGA babies is not associated with improved or
more adverse neurodevelopmental, general health or
educational outcomes at 5 years.
preterm, small for gestational age (SGA) babies to
determine if prophylaxis with granulocyte macrophage
colony stimulating factor (GM-CSF) improves outcomes
(the PROGRAMS trial). GM-CSF was associated with
improved neonatal neutrophil counts, but no change in
other neonatal or 2-year outcomes. As subtle benefits in
outcome may not be ascertainable until school age we
performed an outcome study at 5 years.
Patients and methods: 280 babies born at 31 weeks
of gestation or less and SGA were entered into the trial.
Outcomes were assessed at 5 years to determine
neurodevelopmental and general health status and
educational attainment.
Results: We found no significant differences in
cognitive, general health or educational outcomes
between 83 of 106 (78%) surviving children in the GMCSF
arm compared with 81 of 110 (74%) in the control
arm. Mean mental processing composite (equivalent to
IQ) at 5 years were 94 (SD 16) compared with 95
(SD 15), respectively (difference in means −1 (95%CI −6
to 4), and similar proportions were in receipt of special
educational needs support (41% vs 35%; risk ratio 1.2
(95% CI 0.8 to 1.9)). Performance on Kaufmann-ABC
subscales and components of NEPSY were similar. The
suggestion of worse respiratory outcomes in the GM-CSF
group at 2 years was replicated at 5 years.
Conclusions: The administration of GM-CSF to very
preterm SGA babies is not associated with improved or
more adverse neurodevelopmental, general health or
educational outcomes at 5 years.
Date Issued
2015-04-28
Date Acceptance
2015-03-15
Citation
Archives of Disease in Childhood. Fetal and Neonatal Edition, 2015, 100 (4), pp.F320-F326
ISSN
1359-2998
Publisher
BMJ Publishing Group
Start Page
F320
End Page
F326
Journal / Book Title
Archives of Disease in Childhood. Fetal and Neonatal Edition
Volume
100
Issue
4
Copyright Statement
© 2015 The Author(s). Published by the BMJ Publishing Group Limited. This is an Open Access article distributed in accordance with the terms of the Creative Commons Attribution (CC BY 4.0) license, which permits others to distribute, remix, adapt and build upon this work, for commercial use, provided the original work is properly cited. See: http://creativecommons.org/licenses/by/4.0/
Sponsor
Wellcome Trust
Identifier
http://gateway.webofknowledge.com/gateway/Gateway.cgi?GWVersion=2&SrcApp=PARTNER_APP&SrcAuth=LinksAMR&KeyUT=WOS:000357528600010&DestLinkType=FullRecord&DestApp=ALL_WOS&UsrCustomerID=1ba7043ffcc86c417c072aa74d649202
Grant Number
068499/Z/02/Z
Subjects
Science & Technology
Life Sciences & Biomedicine
Pediatrics
EXTREMELY PRETERM BIRTH
DEVELOPMENTAL-DISABILITY
FOLLOW-UP
CHILDREN
NEUTROPENIA
INFANTS
AGE
Child Psychology
Haematology
Neonatology
Neurodevelopment
Child Development
Female
Granulocyte-Macrophage Colony-Stimulating Factor
Hematologic Agents
Humans
Infant, Extremely Premature
Infant, Newborn
Infant, Small for Gestational Age
Leukocyte Count
Male
Neuropsychological Tests
Neutrophils
Outcome Assessment (Health Care)
Sepsis
Time
1114 Paediatrics And Reproductive Medicine
Publication Status
Published