TY - JOUR
T1 - Preterm neonates benefit from low prophylactic platelet transfusion threshold despite varying risk of bleeding or death
AU - PlaNeT-2 MATISSE Collaborators
AU - Fustolo-Gunnink, Susanna F.
AU - Fijnvandraat, Karin
AU - van Klaveren, David
AU - Stanworth, Simon J.
AU - Curley, Anna
AU - Onland, Wes
AU - Steyerberg, Ewout W.
AU - de Kort, Ellen
AU - D'Haens, Esther J.
AU - Hulzebos, Christian V.
AU - Huisman, Elise J.
AU - de Boode, Willem P.
AU - Lopriore, Enrico
AU - van der Bom, Johanna G.
PY - 2019
Y1 - 2019
N2 - The Platelets for Neonatal Thrombocytopenia (PlaNeT-2) trial reported an unexpected overall benefit of a prophylactic platelet transfusion threshold of 25 3 109/L compared with 50 3 109/L for major bleeding and/or mortality in preterm neonates (7% absolute-risk reduction). However, some neonates in the trial may have experienced little benefit or even harm from the 25 3 109/L threshold. We wanted to assess this heterogeneity of treatment effect in the PlaNet-2 trial, to investigate whether all preterm neonates benefit from the low threshold. We developed a multivariate logistic regression model in the PlaNet-2 data to predict baseline risk of major bleeding and/or mortality for all 653 neonates. We then ranked the neonates based on their predicted baseline risk and categorized them into 4 risk quartiles. Within these quartiles, we assessed absolute-risk difference between the 50 3 109/L- and 25 3 109/L-threshold groups. A total of 146 neonates died or developed major bleeding. The internally validated C-statistic of the model was 0.63 (95% confidence interval, 0.58-0.68). The 25 3 109/L threshold was associated with absolute-risk reduction in all risk groups, varying from 4.9% in the lowest risk group to 12.3% in the highest risk group. These results suggest that a 25 3 109/L prophylactic platelet count threshold can be adopted in all preterm neonates, irrespective of predicted baseline outcome risk. Future studies are needed to improve the predictive accuracy of the baseline risk model. This trial was registered at www.isrctn.com as #ISRCTN87736839.
AB - The Platelets for Neonatal Thrombocytopenia (PlaNeT-2) trial reported an unexpected overall benefit of a prophylactic platelet transfusion threshold of 25 3 109/L compared with 50 3 109/L for major bleeding and/or mortality in preterm neonates (7% absolute-risk reduction). However, some neonates in the trial may have experienced little benefit or even harm from the 25 3 109/L threshold. We wanted to assess this heterogeneity of treatment effect in the PlaNet-2 trial, to investigate whether all preterm neonates benefit from the low threshold. We developed a multivariate logistic regression model in the PlaNet-2 data to predict baseline risk of major bleeding and/or mortality for all 653 neonates. We then ranked the neonates based on their predicted baseline risk and categorized them into 4 risk quartiles. Within these quartiles, we assessed absolute-risk difference between the 50 3 109/L- and 25 3 109/L-threshold groups. A total of 146 neonates died or developed major bleeding. The internally validated C-statistic of the model was 0.63 (95% confidence interval, 0.58-0.68). The 25 3 109/L threshold was associated with absolute-risk reduction in all risk groups, varying from 4.9% in the lowest risk group to 12.3% in the highest risk group. These results suggest that a 25 3 109/L prophylactic platelet count threshold can be adopted in all preterm neonates, irrespective of predicted baseline outcome risk. Future studies are needed to improve the predictive accuracy of the baseline risk model. This trial was registered at www.isrctn.com as #ISRCTN87736839.
UR - https://www.scopus.com/inward/record.uri?partnerID=HzOxMe3b&scp=85077295332&origin=inward
UR - https://www.ncbi.nlm.nih.gov/pubmed/31697817
U2 - https://doi.org/10.1182/blood.2019000899
DO - https://doi.org/10.1182/blood.2019000899
M3 - Article
C2 - 31697817
SN - 0006-4971
VL - 134
SP - 2354
EP - 2360
JO - Blood
JF - Blood
IS - 26
ER -