Darbepoetin alfa to reduce transfusion episodes in infants with haemolytic disease of the fetus and newborn who are treated with intrauterine transfusions in the Netherlands: an open-label, single-centre, phase 2, randomised, controlled trial.

Autor:	Ree IMC; Division of Neonatology, Department of Paediatrics, Leiden University Medical Center, Leiden, Netherlands. Electronic address: i.m.c.ree@lumc.nl., de Haas M; Department of Haematology, Leiden University Medical Center, Leiden, Netherlands; Department of Immunohematology Diagnostics, Sanquin, Amsterdam, Netherlands., van Geloven N; Department of Biomedical Data Sciences, Leiden University Medical Center, Leiden, Netherlands., Juul SE; Department of Paediatrics, University of Washington, Seattle, WA, USA., de Winter D; Division of Neonatology, Department of Paediatrics, Leiden University Medical Center, Leiden, Netherlands; Department of Immunohematology Diagnostics, Sanquin, Amsterdam, Netherlands., Verweij EJT; Division of Foetal Therapy, Department of Obstetrics, Leiden University Medical Center, Leiden, Netherlands., Oepkes D; Division of Foetal Therapy, Department of Obstetrics, Leiden University Medical Center, Leiden, Netherlands., van der Bom JG; Department of Epidemiology, Leiden University Medical Center, Leiden, Netherlands., Lopriore E; Division of Neonatology, Department of Paediatrics, Leiden University Medical Center, Leiden, Netherlands.
Jazyk:	angličtina
Zdroj:	The Lancet. Haematology [Lancet Haematol] 2023 Dec; Vol. 10 (12), pp. e976-e984.
DOI:	10.1016/S2352-3026(23)00285-5
Abstrakt:	Background: Up to 88% of infants with haemolytic disease of the fetus and newborn who are treated with intrauterine transfusions require erythrocyte transfusions after birth. We aimed to investigate the effect of darbepoetin alfa on the prevention of postnatal anaemia in infants with haemolytic disease of the fetus and newborn. Methods: We conducted an open-label, single-centre, phase 2 randomised controlled trial to evaluate the effect of darbepoetin alfa on the number of erythrocyte transfusions in infants with haemolytic disease of the fetus and newborn. All infants who were treated with intrauterine transfusion and born at 35 weeks of gestation or later at the Leiden University Medical Center, Leiden, Netherlands, were eligible for inclusion. Included infants were randomised by computer at birth to treatment with 10 μg/kg darbepoetin alfa subcutaneously once a week for 8 weeks or standard care (1:1 allocation, in varying blocks of four and six, with no stratification). Treating physicians and parents were not masked to treatment allocation, but the research team, data manager, and statistician were masked to treatment allocation during the process of data collection. The primary outcome was the number of erythrocyte transfusion episodes per infant from birth up to 3 months of life in the modified intention-to-treat population. This trial is registered with ClinicalTrials.gov (NCT03104426) and has been completed. Findings: Between Oct 31, 2017, and April 31, 2022, we recruited 76 infants, of whom 44 (58%) were randomly assigned to a treatment group (20 [45%] were allocated to receive darbepoetin alfa and 24 [55%] were allocated to receive standard care). Follow-up lasted 3 months and one infant dropped out of the trial before commencement of treatment. A significant reduction in erythrocyte transfusion episodes was identified with darbepoetin alfa treatment compared with standard care (median 1·0 [IQR 1·0-2·0] transfusion episodes vs 2·0 [1·3-3·0] transfusion episodes; p=0·0082). No adverse events were reported and no infants died during the study. Interpretation: Darbepoetin alfa reduced the transfusion episodes after intrauterine transfusion treatment for haemolytic disease of the fetus and newborn. Treatment with darbepoetin alfa or other types of erythropoietin should be considered as part of the postnatal treatment of severe haemolytic disease of the fetus and newborn. Funding: Sanquin Blood Supply. Translation: For the Dutch translation of the abstract see Supplementary Materials section. Competing Interests: Declaration of interests We declare no competing interests. (Copyright © 2023 Elsevier Ltd. All rights reserved.)
Databáze:	MEDLINE
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