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Introduction: Neonatal thrombocytopenia is a common and important clinical problem in preterm neonates. A trial assessing clinically relevant outcomes in relation to the different platelet count thresholds used to trigger transfusion has never been undertaken in preterm neonates with severe thrombocytopenia. Objectives: Platelets for Neonatal Transfusion - Study 2 (PlaNeT-2) aims to assess whether a higher prophylactic platelet transfusion threshold is superior to the lower thresholds in current standard practice in reducing the proportion of patients who have a major bleed or die up to study day 28. Methods: PlaNeT-2 is a two-stage, randomised, parallel-group, superiority trial. PlaNet-2 compares clinical outcomes in preterm neonates (<34 weeks' gestation at birth) randomised to receive prophylactic platelet transfusions to maintain platelet counts at or above either 25 × 10 9 /l or 50 × 10 9 /l. The primary outcome measure is the proportion of patients who either die or experience a major bleed up to and including study day 28. A total of 660 infants will be randomised. Results and Conclusions: This trial will help define optimal platelet transfusion support for severely thrombocytopenic preterm neonates by evaluating the risks and benefits of two different prophylactic neonatal platelet transfusion thresholds.

Objective: Pregnancies at risk of fetal and neonatal alloimmune thrombocytopenia (FNAIT) are commonly treated using weekly intravenous immunoglobulin (IVIG) at 1 g/kg maternal weight. IVIG is an expensive multidonor human blood product with dose-related side effects. Our aim was to evaluate the effectiveness of IVIG at a lower dose, i.e. 0.5 g/kg. Methods: This was a randomized controlled multicenter trial conducted in Sweden, the Netherlands and Australia. Pregnant women with human platelet antigen alloantibodies and an affected previous child without intracranial hemorrhage (ICH) were enrolled. The participants were randomized to IVIG at 0.5 or 1 g/kg per week. The analyses were per intention to treat. The primary outcome was fetal or neonatal ICH. Secondary outcomes were platelet count at birth, maternal and neonatal IgG levels, neonatal treatment and bleeding other than ICH. Results: A total of 23 women were randomized into two groups (low dose: n = 12; standard dose: n = 11). The trial was stopped early due to poor recruitment. No ICH occurred. The median newborn platelet count was 81 × 10 9 /l (range 8-269) in the 0.5 g/kg group versus 110 × 10 9 /l (range 11-279) in the 1 g/kg group (p = 0.644). Conclusion: The risk of adverse outcomes in FNAIT pregnancies treated with IVIG at 0.5 g/kg is very low, similar to that using 1 g/kg, although our uncompleted trial lacked the power to conclusively prove the noninferiority of using the low dose.

Background Severe neonatal thrombocytopenia, as a rare but life-threatening disease with multiple etiologies, has limited relevant reports in China. The single-center study was performed in a severe thrombocytopenic cohort to improve the prognosis of this disease. Methods We included all the patients diagnosed with severe thrombocytopenia (platelet counts ≤ 50 × 10 3 /µL) in our institution between October 2016 and February 2021, and retrospectively reviewed their electronic records. Comparisons were made according to etiology and outcome. Results Among the 5819 inpatients, 194 with severe thrombocytopenia were included in this study, with 64.4% of the cases manifesting thrombocytopenia within 72 h of life. The highest incidence was recorded among extremely low birth weight neonates (6.5%). The main etiologies included sepsis (22.2%), genetic syndromes (14.4%), perinatal asphyxia (9.8%), necrotizing enterocolitis (NEC; 8.8%), and cytomegalovirus infection (6.7%). The median platelet nadir was 5.0 × 10 3 /µL [IQR:2.0 × 10 3 /µL-16.0 × 10 3 /µL], and 112 patients developed very severe thrombocytopenia (platelet counts ≤ 30 × 10 3 /µL), of which 21.4% were associated with late-onset sepsis. In 45 culture-positive cases, the gram-negative group had a lower level of platelets (mean [SD]: 28 [11]×10 3 /µL) as compared to the gram-positive group (mean [SD]: 39 [12]×10 3 /µL). A total of 120 cases (61.9%) exhibited evidence of hemorrhage, with patients diagnosed with NEC demonstrating the highest incidence of hemorrhage at 58.8%. The platelet counts took a median of 10 days to recover: 11 and 7 days for early and late-onset cases; 15 days without and 21 days with platelet transfusions, respectively. The overall mortality rate was 26.8%. The causes of severe thrombocytopenia in 32.7%, 19.2%, and 17.3% of patients who died were identified as sepsis, birth asphyxia, and NEC, respectively. The levels of PT ( P  = 0.025), APTT ( P  = 0.046), and lactate ( P  = 0.028) were lower among surviving patients. Conclusions Sepsis, genetic syndromes, and perinatal asphyxia are the predominant etiologies of severe neonatal thrombocytopenia in China. The overall prognosis of severe neonatal thrombocytopenia is poor, but its severity and outcome were related to laboratory results (PT, APTT, and lactate) and the underlying etiology.

Fetal and neonatal alloimmune thrombocytopenia (FNAIT) is a serious disorder that arises when a mother produces alloantibodies against specific human platelet antigens (HPAs) expressed on fetal platelets. These maternal alloantibodies cross the placenta and destroy fetal platelets, significantly increasing the risk of fetal or neonatal intracranial hemorrhage (ICH). Indeed, FNAIT is the leading cause of isolated severe thrombocytopenia in otherwise healthy neonates. In this review, we summarize recent advances to provide obstetricians and maternal-fetal medicine specialists with an updated overview of FNAIT, encompassing its pathophysiology, clinical features, diagnostic strategies, antenatal management, delivery planning, and postnatal care. Although the prophylactic interventions remain at an early stage, HPA histo-incompatibility prescreening is already available at a suitable cost, and the development of NAITgam, a targeted immunoglobulin preparation, represents a promising advance. While the widespread implementation of screening and prophylaxis may take years, such measures have the potential to significantly reduce the incidence and severity of FNAIT. Currently, the antenatal administration of intravenous immunoglobulin (IVIg), with or without corticosteroids, remains the safest and most effective treatment for high-risk pregnancies. Looking ahead, animal model data continue to provide valuable insights that may inform the development of novel preventive and therapeutic strategies. Ultimately, the implementation of a national screening program could prevent severe complications and help mitigate the long-term societal and healthcare burden of FNAIT.

Only isolated occurrences of neonatal Jk hemolytic disease have been identified, and no cases of Jk hemolytic disease combining fetal and neonatal alloimmune thrombocytopenia (FNAIT) have been reported. The majority of medical professionals lack sufficient knowledge regarding Jk hemolytic disease, which could result in missed diagnoses and early misdiagnoses. In this study, a case of a male newborn with extreme anemia and thrombocytopenia is reported. The newborn and his mother were identified as blood groups O RhD + and Jk(a + b+), and O RhD + and Jk(a-b+). Anti-Jk was identified in the plasma of both the mother and newborn. Thrombocytopenia and upper gastrointestinal bleeding were observed in the newborn, and both mother and newborn tested positive for platelet antibodies. The extreme anemia and thrombocytopenia were successfully treated with red cell transfusions and immunoglobulin. Co-existence of neonatal Jk hemolytic disease and FNAIT is very rare in newborns with significant clinical manifestations. Early diagnosis and timely treatment are crucial for improving patient outcomes.

A low platelet count (thrombocytopenia) is a common finding especially in neonates who are admitted to the neonatal intensive care unit. Due to the varied causes that can lead to neonatal thrombocytopenia, assessment and management can be challenging. Having an understanding of the causes of neonatal thrombocytopenia and their natural progression would help guide subsequent management. Therefore, we will be exploring the different circumstances where thrombocytopenia occurs and how to interpret and manage the neonate with thrombocytopenia in this interpretations article.

Background Neonatal thrombocytopenia is common in preterm and term neonates admitted to neonatal intensive care units. The etiology behind neonatal thrombocytopenia is complex. Inherited thrombocytopenia is rare and usually results from genetic mutations. Case presentation Here we report a case of twins with severe inherited thrombocytopenia presented in the neonatal period who were shown to be compound heterozygotes for 2 UDP-N-acetylglucosamine 2-epimerase ( GNE ) gene mutations, c.1351C > T and c.1330G > T, of which c.1330G > T is a novel mutation. Conclusion These two GNE mutations may help in the diagnosis and management of thrombocytopenia diagnosed in neonates.

Neonatal alloimmune thrombocytopenia (NAIT) is an immune disorder characterized by maternal antibodies that destroy fetal platelets, leading to thrombocytopenia. The prevalence of NAIT is approximately 0.05% to 0.15%. Fetal and neonatal severe thrombocytopenia represents the most common form of the disease, primarily occurring in firstborn children. It poses a greater risk and harm to the fetus and newborn. Neonatal intracranial hemorrhage is a severe complication of NAIT, resulting in irreversible damage to cranial nerves and potential neonatal death. This study aims to assess the current advancements in the pathogenesis, clinical characteristics, laboratory evaluation, and therapeutic interventions for neonatal alloimmune thrombocytopenia. This narrative review explores neonatal alloimmune thrombocytopenia through a thorough literature review. The study encompasses the pathogenesis, clinical features, laboratory examination, and treatment options associated with this condition. The results of this study highlight that despite the extremely low incidence of NAIT, it carries a high risk. Currently, there is no timely and effective prevention method available. However, using HPA-1a as a screening item for prenatal prevention shows the potential to reduce the mortality rate of NAIT fetuses. Further research is required to evaluate its accuracy and specificity. The findings of this review emphasize the need for further research to develop effective prevention methods. The use of HPA-1a as a screening tool holds promise but requires additional investigation. Enhancing clinical understanding of NAIT will contribute to improved management and outcomes for affected infants.

IntroductionIn pregnant women with a history of fetal and neonatal alloimmune thrombocytopenia (FNAIT), prenatal intervention in subsequent pregnancies may be required to prevent fetal bleeding. Several invasive and non-invasive protocols have been published: amniocentesis for fetal genotyping, fetal blood sampling for the determination of fetal platelet count, intrauterine platelet transfusions, and weekly maternal i.v. immunoglobulin (IVIG) infusion with or without additional corticosteroid therapy. This is the first retrospective study that report the experience with a non-invasive protocol focused on side effects of maternal IVIG treatment and neonatal outcome.MethodsPregnant women with proven FNAIT in history and an antigen positive fetus were treated with IVIG (1 g/kg/bw) every week. To identify potential IVIG-related hemolytic reactions isoagglutinin titer of each IVIG lot and maternal blood count were controlled. IVIG-related side effects were prospectively documented and evaluated. Furthermore, ultrasound examination of the fetus was performed before starting IVIG administration and continued regularly during treatment. Outcome of the index and subsequent pregnancy was compared. Corresponding data of the newborns were analyzed simultaneously.ResultsIVIG was started at 20 weeks of gestation (median). Compared to the index pregnancy, platelet counts of the newborns were higher in all cases. No intracranial hemorrhage occurred (Index pregnancies: 1 case). Platelet counts were 187 × 109/l (median, range 22–239, 95% CI) and one newborn had mild bleeding. No severe hemolytic reaction was observed and side effects were moderate.ConclusionAmong pregnant women with FNAIT history, the use of non-invasive fetal risk determination and maternal IVIG resulted in favorite outcome of all newborns. Invasive diagnostic or therapeutic procedures in women with a history of FNAIT should be abandoned.