Postnatal treatment for children with fetal and neonatal alloimmune thrombocytopenia: a multicentre, retrospective, cohort study

Vos, T.W. de; Winkelhorst, D.; Arnadottir, V.; Bom, J.G. van der; Suris, C.C.; Caram-Deelder, C.; ... ; Lopriore, E. (2022)

Article / Letter to editor

Background Children affected by fetal and neonatal alloimmune thrombocytopenia (FNAIT) are at risk of severe intracranial haemorrhage. Management in the postnatal period is based on sparse evidence. We aimed to describe the contemporary management and outcomes of patients with FNAIT in high-income countries.Methods In this multicentre, retrospective, cohort study, we set up a web-based registry for the collection of deidentified data on the management and course of neonates with FNAIT. Eight centres from seven countries (Australia, Norway, Slovenia, Spain, Sweden, the Netherlands, and the USA) participated. Eligibility criteria comprised neonates with FNAIT being liveborn between Jan 1, 2010, and Jan 1, 2020; anti-human platelet antigen (HPA) alloantibodies in maternal serum; confirmed maternal and fetal HPA incompatibility; and bleeding detected at antenatal ultrasound, neonatal thrombocytopenia (<150 x 10(9) platelets per L), or both in the current or previous pregnancy. Clinical data were retrieved from local medical records of the first neonatal admission and entered in the registry. The key outcome was the type of postnatal treatment given to neonates with FNAIT. Other outcomes were daily median platelet counts in the first week of life, median platelet count increment after first unmatched versus first matched transfusions, and the proportion of neonates with mild or severe bleeding.Findings 408 liveborn neonates with FNAIT were entered into the FNAIT registry, of whom 389 from Australia (n=74), Norway (n=56), Slovenia (n=19), Spain (n=55), Sweden (n=31), the Netherlands (n=138), and the USA (n=16) were included in our analyses. The median follow-up was 5 days (IQR 2-9). More neonates were male (241 [64%] of 379) than female (138 [36%]). Severe thrombocytopenia (platelet count <50 x 10(9) platelets per L) was reported in 283 (74%) of 380 neonates, and extreme thrombo-cytopenia (<10 x 10(9) platelets per L) was reported in 92 (24%) neonates. Postnatal platelet count nadir was higher in the no-treatment group than in all other groups. 163 (42%) of 389 neonates with FNAIT received no postnatal treatment. 207 (53%) neonates received platelet transfusions, which were either HPA-unmatched (88 [43%] of 207), HPA-matched (84 [41%]), or a combination of both (35 [17%]). The proportion of neonates who received HPA-matched platelet transfusions varied between countries, ranging from 0% (Slovenia) to 63% (35 of 56 neonates; Norway). Postnatal intravenous immunoglobulin treatment was given to 110 (28%) of 389 neonates (alone [n=19] or in combination with platelet transfusions [n=91]), with the proportion receiving it ranging from 12% (17 of 138 neonates; the Netherlands) to 63% (ten of 16 neonates; the USA) across countries. The median platelet increment was 59 x 10(9) platelets per L (IQR 35-94) after HPA-unmatched platelet transfusions and 98 x 10(9) platelets per L (67-134) after HPA-matched platelet transfusions (p<0 center dot 0001). Severe bleeding was diagnosed in 23 (6%) of 389 liveborn neonates, with one having a severe pulmonary haemorrhage and 22 having severe intracranial haemorrhages. Mild bleeding was diagnosed in 186 (48%) neonates.Interpretation Postnatal management of FNAIT varies greatly between international centres, highlighting the absence of consensus on optimal treatments. Our data suggest that HPA-matched transfusions lead to a larger median platelet count increment than HPA-unmatched transfusions, but whether HPA matching is also associated with a reduced risk of bleeding remains unknown. Copyright (c) 2022 Published by Elsevier Ltd. All rights reserved.

• All authors: Vos, T.W. de; Winkelhorst, D.; Arnadottir, V.; Bom, J.G. van der; Suris, C.C.; Caram-Deelder, C.; Deschmann, E.; Haysom, H.E.; Hverven, H.B.C.; Krivec, J.L.; McQuilten, Z.K.; Muniz-Diaz, E.; Nogues, N.; Oepkes, D.; Porcelijn, L.; Schoot, C.E. van der; Saxonhouse, M.; Sola-Visner, M.; Tiblad, E.; Tiller, H.; Wood, E.M.; Young, V.; Zeleznik, M.; Haas, M. de; Lopriore, E.
• Date: 2022-11-01
• Journal: The Lancet Haematology
• Volume: 9
• Issue: 11
• Pages: E844 - E853
• DOI: doi:10.1016/S2352-3026(22)00243-5
• Link: https://www.sciencedirect.com/science/article/pii/S2352302622002435?via%...