Idursulfase and idursulfase beta currently used for enzyme replacement therapy of mucopolysaccharidosis type II (MPS II) do not cross the blood-brain barrier (BBB), which significantly limits the treatment of neurocognitive impairment in patients. Verenafusp alfa (Clotilia®, GENERIUM JSC) contains a recombinant modified iduronate-2-sulfatase enzyme covalently linked to the Fab fragment of a monoclonal antibody against the human insulin receptor, which crosses the BBB. Objective: The aim of this multicenter, open-label, multicohort phase II–III study was to evaluate the safety, pharmacokinetics, pharmacodynamics (PD), efficacy, and optimal dose selection of verenafusp alfa in different age groups of patients. Materials and methods: The interim analysis included data from 18 patients under 18 years of age in 6 age cohorts. Cohort enrollment was performed sequentially with decreasing patient age, first for the 2 mg/kg dose (Group 1) in cohorts 2, 3, 5, and then for the 3 mg/kg dose (Group 2) in cohorts 4, 6, 7. The study included 52 weekly infusions of verenafusp alfa. The PD criterion was the level of glycosaminoglycans (GAGs) in urine, blood, and cerebrospinal fluid (CSF). Efficacy parameters included assessment of somatic manifestations and neurocognitive functions; safety parameters included assessment of adverse events (AEs) and the dynamics of anti-drug antibody (ADA) formation. Results: The increase in verenafusp alfa concentration in CSF was more pronounced in Group 2 (3 mg/kg) – 252.5 [221.4; 351.54] pg/mL, as well as in the group of children under 6 years of age – 302.02 [244.72; 388.83] pg/mL. The decrease in heparan sulfate (HS) levels in CSF at week 52 was more pronounced in Group 2 (3 mg/kg) – 49% (p=0.084), and also in the group of patients under 6 years of age – 53% (p=0.027). Urinary and blood GAG levels remained stable, taking into account prior idursulfase therapy in 94% of patients. An 18% increase in the 6-minute walk test distance was observed in the overall group of patients under 18 years of age (p=0.020), accompanied by a statistically significant increase in range of motion in the shoulder, knee, and left hip joints. Liver and spleen volumes remained stable. Stabilization and/or improvement of neurocognitive function parameters were observed in patients with MPS II. The AEs observed in 50% of patients were non-serious, classified as infusion reactions of mild or moderate severity, with a predictable and manageable spectrum and frequency. ADA formation against the drug was observed in 40% of patients. Conclusion: Weekly intravenous administration of verenafusp alfa for one year achieved control of urinary GAG levels, stabilization and/or improvement of neurocognitive functions, and reduction of somatic manifestations of MPS II in previously treated patients with idursulfase drugs aged 2 to 16 years. After one year of therapy, an increase in verenafusp alfa concentration and a decrease in HS levels in CSF were observed, confirming its ability to cross the BBB and prevent neurodegenerative changes in the CNS.
Recent technological advantages of ultrasound sonography (USG) have made it possible to visualize peripheral nerves over a large area in real time with high resolution and with the ability to assess the structure and the degree of vascularization, to measure the size and the stiffness of the nerves in the studying area. The spreading of the use of visual ultrasound assessment of peripheral nerves in the diagnosis of tunnel neuropathies, traumatic injuries and hereditary polyneuropathies creates prerequisites for studying the possibilities of diagnosing diabetic polyneuropathy (DPN). This bibliographical review represents the results of ultrasonic diagnostics of DPN by researchers from all-over the World. There are a lot of reports with positive experience in registration of an increase in peripheral nerves and their stiffness in patients with DPN. Further development of the USG techniques aimed at the DPN signs assessment would improve the disease prognoses coupled with an undoubtedly positive effect on the patients’ quality-of-life.
Intrahospital transportation (IHT) of newborns is an integral part of intensive care and nursing of patients in neonatal ICUs and neonatal departments. Transfer within a medical facility ensures the availability of medical technologies required by the patient and is associated with significant risks of deterioration of the condition, potential increase in the need for certain types of therapy and worsening of hospital stage outcomes at the same time. Nevertheless, the newborns’ IHT problematics remains poorly studied. Undeservedly insufficient amount of attention is paid to the standardization of this section of practical healthcare. Meanwhile, modern requirements for the organization of high-quality and high-safety medical care imply the formation of an optimal technology associated with minimalization of all the risks. Authors summarize the available scientific and practical experience in the field of newborns’ IHT organizing highlighting the key points of the process and inviting neonatologists to further discussion on this important problem.
Spondylocostal dysostosis is a rare hereditary disease that is accompanied by a violation of the segmentation of the spine. The features of the appearance of patients are characterized by spondylocostal dysplasia in the form of a shortened neck and a “crab-shaped” deformation of the chest due to the fusion of the distal and divergence of the proximal ribs. It belongs to orphan genetic diseases, Worldwide frequency of which is 1:200,000 newborns. To date, six genetic variants of this disease with different types of inheritance have been described as follows: autosomal recessive forms associated with the genes DLL3, MESP2, LFNG, HES7 (spondylocostal dysplasia-1), autosomal dominant form with the TBX6 gene (spondylocostal dysplasia-2), the inheritance of changes in the RIPPLY2 gene has not been established. Since the syndrome is a disease with low prevalence, patients need various diagnostic and screening tests with interdisciplinary evaluation of the results at different stages of their life. Early diagnosis of the disease is crucial for timely treatment and rehabilitation measures and further improvement of the quality-of-life prognosis for the child. Authors represent a clinical case of genetically confirmed spondylocostal dysostosis-4 in a child, during the examination of which a previously undescribed pathogenic variant of the nucleotide sequence in exon 4 of the HES7 gene in a homozygous state, a Sifrim-Hitz-Weiss syndrome, was detected. The presented clinical case is of a particular practical interest to geneticists, radiation diagnosticians, obstetricians-gynecologists and neonatologists in the context of the timely diagnosis problematics.
Kawasaki disease (KD) is an acute systemic disease characterized by predominant damage to small and medium-sized arteries with the development of destructive-proliferative vasculitis, damage to the coronary (CA) and visceral arteries. In addition to the symptoms included in the diagnostic criteria for KD, other clinical manifestations are observed in the Article. Facial nerve damage in KD is a rare but serious manifestation which can be a marker for severe CA damage. Authors represent their own clinical case report of a 5-months-old male patient with late diagnosis of immunoglobulin-resistant KD with giant aneurysms of CA and facial nerve paralysis.
Testicular dysfunction is a serious medical and social problem that leads to infertility, endocrine disorders and impaired sexual development. Existing treatment methods are often limited to surgery and replacement therapy that in its turn cannot restore the structural elements of already damaged testicular tissue fully. The purpose of this research was to evaluate the efficacy of allogeneic Sertoli cell transplantation as a treatment for testicular dysfunction caused by experimental cryptorchidism. Materials and methods used: a single-center experimental randomized controlled trial was conducted on 24 outbred rats that were subjected to abdominal cryptorchidism for 90 days, after which the animals were divided into three groups of 8 animals each. In the experimental group, the animals were injected with a medium containing undifferentiated Sertoli cells into the testes (“Group O”); animals from the control group 1 were injected with DMEM medium into the testes (“Group K”); and from the second control group 2 they’ve underwent the usual lowering of the testes into the scrotum (“Group K+B”). Ninety days after the lowering of the testes into the scrotum, the animals were taken out of the experiment and the testes were removed for subsequent histological examination. Results: the average testis weight 90 days after the transplantation of undifferentiated Sertoli cells in Group O was 530 milligrams which was significantly higher than the testis weight in the animals from both of the control groups. In animals from the control Group K, the average testis weight was statistically significantly lower than in animals from the experimental group and amounted to 282 mg (p=0.003). Also, in the experimental group of animals, 90 days after the transplantation, the seminiferous tubules were found with spermatogenesis completely restored in them. In both of the control groups, no signs of spermatogenesis restoration were recorded (p<0.001). Conclusion: undifferentiated Sertoli cells when transplanted into the testes are capable of forming the new seminiferous tubules and supporting spermatogenesis processes in them. The positive transplantation outcome was expressed as the increase in the weight of the testes compared to those of the control groups coupled with the presence of germ cells in the seminiferous tubules and differentiation processes in them.
Functional maturity of premature infants is not characterized by low weight and height indicators only but also by the delayed development of organs and systems that in its turn leads to disruption of their successful adaptation to extrauterine life. The purpose of this research was to identify the features of clinical adaptation and expression of IGF and FGF in cord blood in premature infants born at 22 to 31.6 weeks of gestation with different stages of lung development. Materials and methods used: a single-center cohort prospective study was conducted in 2022-2023 that included 51 premature infants with a gestational age (GA) of 22 to 31.6 weeks of which 22 had canalicular stage (CS) and 29 had saccular stage (SS) of lung development, accordingly. Results: patients with CS had longer duration of general respiratory support (both invasive and noninvasive) (p=0.032), grade 2 intraventricular hemorrhages were diagnosed more often (p<0.001), blood and plasma transfusion procedures were performed (p=0.026 and p=0.018, respectively); patients in this group were mainly prescribed with imipenem+cilastatin and ceftazidime therapy (p=0.035 and p=0.009, respectively). When assessing the acid-base balance of the blood in the first hours of life, children with CS showed increase in sodium ions (p<0.001) compared to those with SS. In the general blood test taken in the first day of life an increase in the relative number of segmented neutrophils (p<0.001) and a decrease in the number of lymphocytes in patients of the CS group (p=0.015) were recorded. In the umbilical cord blood, increased levels of IGF and FGF were observed in children of the CS group (p=0.021 and p=0.006, respectively). Conclusion: the severity of the premature infants born at 22 to 31.6 weeks of gestation condition depends on the stage of lung development. Determination of the level of fibroblast growth factor expression in the umbilical cord blood can serve as an additional criterion in assessing the degree of maturity of lung tissue, which will ensure a personalized approach to therapy in premature infants.
Eosinophilic esophagitis (EoE) is a chronic inflammatory disease with a complex pathobiological mechanism. Understanding the differences in the cytokine profile in patients with different phenotypes of the disease can contribute to a more personalized approach to the management of children with EoE. The purpose of this research was to identify clinical and anamnestic features and differences in the cytokine profile in children with inflammatory and fibrostenotic phenotypes of EoE and to evaluate the significance of biomarker levels in predicting the severity of the disease in children. Materials and methods used: a single-center retrospective study was performed that included 40 patients, all under 18 years old, with EoE, who were then divided into two groups as follows: with fibrostenotic (G1; 7) and inflammatory (G2; 33) phenotypes. A comprehensive assessment of clinical and instrumental examination data was performed including endoscopic and morphological changes in the esophageal mucosa. Blood serum samples were taken from all patients in order to determine the levels of T2 inflammation biomarkers using the enzyme immunoassay. Results: in G1 compared with G2, an earlier onset of disease symptoms (5.7 (4.2; 11.5) v. 10.7 (5.3; 14.8) y/o, p=0.012) and a later diagnosis (38 (2.0; 64.0) v. 9 (6.0; 22.0) months, p=0.004) were revealed. The concentration of IL-33 was statistically significantly higher in G1 (p<0.001) while the IL-5 level was statistically significantly higher in G2 (p=0.012). The area under the ROC curve, corresponding to the relationship between the prediction of the presence of the esophageal stenosis and the concentration of IL-33, was 0.951±0.045 with 95% CI: 0.845-0.986 (p<0.001). The IL-33 threshold value was 1315.0 pg/ml: with an increase in the IL-33 concentration a high risk of esophageal stenosis was predicted. The sensitivity of the parameter was 85.7%, the specificity was 82.2%. Conclusion: IL-33 can be a useful diagnostic marker for predicting the fibrostenotic phenotype of EoE. Comprehensive assessment of clinical characteristics and T2 inflammation biomarkers has significant diagnostic value for personalized treatment of children with EoE.
Despite the rapid relief of respiratory disorders in transient tachypnea of the newborn (TTN) in most of the cases, these patients keep showing evidence of changes in the level of cerebral oxygenation and activity of neurotrophic factors as well as a high frequency of the “cerebral ischemia” (CI) diagnosis at discharge. There are no current data on the frequency of cerebral damage in patients with a history of TTN in the long term. The purpose of this research was to evaluate the effectiveness of the CPAP standardized protocol in the delivery room on the long-term outcomes of cerebral pathology (CP). Materials and methods used: a single-center cohort retrospective study was conducted that included both full-term and late preterm patients with TTN, both before the introduction of the CPAP standardized protocol in the delivery room and after the its implementation. The follow-up data of the full-term infants - for the first two years of life (2020, 2021, 2022, 2023) and of the premature infants - for the first one year of life (2020 and 2022, 2023) were collected. The frequency and the nature of the identified CP were assessed. Results: the full-term follow-up control (FFC) group consisted of 116, the full-term follow-up study (FFS) group of 84, the premature follow-up control (PFC) group of 60 and the premature follow-up study (PFS) group of 50 children. In the FFS group the CP was statistically significantly less frequently diagnosed at 6, 9, 12 months, 1.5 and 2 years of life in contrast to the FFC group (p<0.05). In the PFS group the CP was also statistically significantly less frequently diagnosed, but only at the age of 1 year of life, unlike the PFC group (p˂0.05). However, the results of the study do not exclude a high frequency of hyperdiagnosis that requires the introduction of a standardized approach. Conclusion: the study showed that standardization of the primary respiratory strategy in the delivery room, CPAP implementation according to the protocol that the Aurors have developed, leads to a decrease in the frequency of CP not only upon discharge from the perinatal center, but also in the long term. Authors see the introduction of the protocol into the maternity hospitals’ routine practice as one of the important steps in the strategy for protecting and maintaining children's health.
Development of molecular genetics and cellular immunology has fundamentally changed the understanding of the relationship between mother and child during pregnancy. For a successful pregnancy, it is necessary for the mother's immune system to recognize and accept the semi-allogeneic fetus, ensuring effective penetration of fetal trophoblasts into the uterine body and protecting the fetus from external pathogens and the mother's microbiota. This is achieved through the exchange of immune and hematopoietic stem cells of the mother and the child with the formation of a cellular chimera called feto-maternal microchimerism as well as vice versa, the formation of the innate immunity of the fetus, ensuring its tolerance to the microbiota and chronic infections of the mother. Innate immunity and hematopoiesis are formed from the cells of the yolk sac mesoderm and extraembryonic mesenchymal tissue due to granule-macrophage-like precursors and pluripotent erythroid cells. During the first trimester of pregnancy and prior to the formation of the thymus and bone marrow hematopoiesis and, therefore, the implementation of the adaptive immune response, the fetus has a formed innate immune defense and cellular control over the biology of its own development represented by neutrophils, eosinophils, basophils, monocytes, macrophages, dendritic cells and the five types of innate lymphoid cells. Innate immune cells are activated by molecular pattern molecules mediated by pattern recognition receptors which provides rapid protection against microbiota pathogens and chronic infections and helps get rid of dying and/or damaged innate cells. Analysis of the interaction between the microbiome, acute and chronic maternal infections, development and functioning of innate lymphoid cells in the fetal and neonatal period is of a great clinical importance aiming at detailing the pathogenesis and developing pathogenetically based therapy, prevention and treatment of eclampsia, pregnancy complications, such as premature birth and other problems associated with placental abruption, bronchopulmonary dysplasia, necrotizing enterocolitis, pneumonia and sepsis in premature and newborn infants. Author presents an analysis of the recent data on the role of innate immunity in terms of evidence-based links with perinatal disorders in newborns and proposals for planning of further scientific research in this important area.
Diaphanospondylodysostosis is a rare genetic skeletal disorder caused by biallelic variants in the BMPER gene. The term “diaphanospondylodysostosis” includes ischiospinal dysotosis, which was previously known as a separate entity with milder clinical features. The clinical phenotype of diaphanospondylodysostosis is quite variable and some cases have been lethal in the early postnatal period. The main clinical and radiographic features of the disease are a narrow chest, segmentation defects of the spine, rib anomalies, decreased ossification of the axial skeleton and nephrogenic cysts. Authors represent a clinical case report of a newborn female with diaphanospondylodysostosis who was found to have two novel heterozygous variants in the bone morphogenetic protein endothelial progenitor-binding regulator (BMPER) gene.
Authors present an analysis of the prevalence of the best type of infant nutrition, breastfeeding, both Worldwide and in Russia. In spite of over 40 years of implementation of breastfeeding support measures developed by the WHO/UNICEF, the provision of infants with breastfeeding remains unsatisfactory in many countries of the World including Russia. Authors outline possible reasons for this situation, substantiate promising strategies for increasing the effectiveness of measures aimed at increasing the frequency and the duration of infant breastfeeding and propose a few specific directions for increasing the effectiveness of breastfeeding support strategies for further discussion by the medical community opinion leaders, decision-makers.
Populational screening with TREC/KREC determination is a universal method for detecting severe combined immunodeficiency prior to the onset of infectious diseases. The prognostic significance of TREC/KREC low levels in preterm infants with secondary lymphopenia has not been sufficiently studied as yet. The purpose of this research was to determine the value of TREC/KREC indicators for predicting severe infections in preterm infants. Materials and methods used: the study was conducted on the basis of the Stavropol Krai Regional Clinical Perinatal Center (Stavropol, Stavropol Krai, Russia). An analysis of neonatal screening results for 100 preterm infants was conducted in Jan. 01, 2023-June 30, 2024, and clinical and laboratory data of 18 patients who were hospitalized in the ICU were studied retrospectively. Statistical analysis utilized the Mann-Whitney non-parametric test and ROC curve analysis. Results: among the causes of secondary lymphopenia not associated with congenital immune defects, preterm birth accounts for 77.8%. Preterm infants have reduced levels of TREC and KREC with statistically significant differences observed in those with gestational ages below 28 weeks. The median TREC in preterm infants admitted to the ICU was significantly lower than in the comparison group. A TREC value below 509 copies/105 may be used as a predictor for life-threatening infections. Conclusion: preterm infants with low and abnormal TREC/KREC values are at high risk for adverse outcomes and therefore require throughout and careful monitoring.
MN1 gene is localized in the q12.1 region of the Chromosome 22, consists of two exons and encodes a protein of 1320 amino acids. In 2020 it was for the first time shown that dysfunction of the MN1 gene causes a specific disease, CEBALID syndrome, which is characterized by malformations of the central nervous system and craniofacial skeleton. There are also isolated reports that overlapping microdeletions of 22q12.1, including the entire sequence of the MN1 gene, are associated with various clinical phenotypes, including developmental anomalies of the nervous system, facial dysmorphia and congenital heart defects. Authors represent the two new clinical case reports of dysfunction of the MN1 gene caused by a heterozygous deletion of 22q12.1 and a pathogenic variant of the nucleotide sequence in exon 2 of the MN1 gene, which were diagnosed in two patients with different phenotype anomalies. Conclusion: the variability of clinical features among the two presented cases was caused by different types of genetic variants. Deletion of the chromosomal region 22q12.1 leads to less pronounced clinical symptoms and difficulties in diagnosis.
Data on the clinical course and treatment of polycythemia vera (PV) in children are extremely limited; the results of any treatment are currently unknown. Authors represent a clinical case report of a PV pediatric patient who has been receiving long-term cytoreductive therapy with pegylated interferon α-2a (PEG-IFN-α2a): 14 y/o male, first visit to the National Scientific and Practical Center for Pediatric Hematology, Oncology and Immunology named after Dmitry Rogachev (Moscow, Russia) in Apr., 2017 at the age of 8 y/o with pronounced changes in the hemogram. In the general blood test: an increase in hemoglobin - 175 g/l, hematocrit - 50.3%, erythrocytosis - up to 6.99x1012/l, other indicators were normal. Molecular genetic examination of bone marrow using high-throughput sequencing revealed a somatic mutation of the JAK2 gene c.1849G>T, p.Val617Phe with an allelic load of 30.23%. Based on the examination results, the patient was diagnosed with PV. PEG-IFN-α2a therapy was initiated. In total, the patient has been receiving the therapy for 7 years; according to the results of control examinations, a persistent partial clinical, hematological and molecular response is maintained throughout the treatment period. Mild adverse events were observed against the background of the therapy: grade 2 neutropenia, grade 1 increase in liver transaminases according to CTCAE v5.0. Conclusion: the use of the PEG-IFN-α2a effectively normalizes the clinical and hematological manifestations of PV in children with no significant adverse events of its use recorded so far.
T.E. Borovik, O.I. Simonova, S.V. Voronin, T.V. Bushuyeva, Ina Sokolov, A.A. Pashkevich, A.E. Lavrova, N.A. Ilyenkova, D.F. Sergiyenko, A.A. Agafonova, N.Yu. Filina, I.P. Karimova. "Recent developments in the nutrition of children with cystic fibrosis," Resolution by the Expert Council dated December 12, 2024, Moscow, Russia. Pediatria n.a. G.N. Speransky. 2025; 104 (1): 151-154. DOI: 10.24110/0031-403X-2025-104-1-151-154.
Diagnosis of rare causes of cyanosis is if a certain difficulty. A clinical case report of cyanosis caused by the presence of abnormal Hemoglobin (Hb) Kansas with reduced oxygen affinity is presented. This is the Russia’s first detected and recorded case in a Russian child with cyanosis and mild anemia. As a result of routine examination using capillary electrophoresis and high-performance liquid chromatography, a pathological fraction of Hb was detected. Molecular genetic research according to Sanger determined a mutation in codon 102 of the β-globin gene with the replacement of aspartic acid with threonine, corresponding to rare abnormal Hb Kansas with reduced oxygen affinity.
Some patients with bronchopulmonary dysplasia (BPD) whose status requires long-term mechanical ventilation (MV), undergo tracheostomy (TS), however, the characteristics of such patients are not presented in domestic studies as yet. The purpose of this research was to calculate the frequency of TS in children with BPD, to assess the clinical features of such patients’ status, the comorbidity and the outcomes in these patients. Materials and methods used: 73 children in their first year of life - aged 2.0 [1.0; 5.0] months old, 45 (62%) boys/28 (38%) girls, with diagnosed BPD participated in a single-center cohort retrospective study and were divided into two groups (with BPD and TS; 14, and with BPD but without TS; 59), who were admitted at the Morozov Children’s City Clinical Hospital (Moscow, Russia) in 2020-2023. The demographic characteristics of patients, their comorbidity and mortality as well as the reasons for TS were assessed. Results: the frequency of TS in infants with BPD was 4.3% [95% CI 2.4%; 7.2%]. In the structure of infant patients with TS, children with BPD accounted for 18.7% [95% CI 10.6%; 29.3%]. In 14 children with BPD, TS was performed at median age of 3.5 [2.0; 6.0] months old with an average age of 4.4±2.7 months old. These patients had severe comorbidities, such as: perinatal CNS damage (all of them), congenital heart defects (6), Edwards syndrome (2), Down syndrome (2) and CHARGE syndrome (2). Patients with BPD and TS, compared with patients with BPD but without TS, had statistically significantly greater gestational age (p=0.005) and birth weight (p=0.046), rather frequently needed the MV (p=0.007) and ware assigned with the palliative patient status (p<0.001). Of the 8 children with BPD with TS with known follow-up, 5 (63%) had died. Conclusion: the need for TS and long-term MV in children with BPD is determined by severe comorbidity.
Changes in the optic nerve lead to decreased vision with a risk of complete loss in up to 20% of cases. Chronic hyperglycemia in diabetes mellitus is a key cause for the development of diabetic retinopathy and optic neuropathy. Insufficient attention is paid to the diagnosis of the optic nerve in children and adolescents, which leads to the development of pronounced structural changes and loss of time for reversible changes. Ultrasound is a safe, accessible method for assessing the optic nerve. The purpose of this research was to evaluate the ultrasound characteristics of the optic nerve in patients with type 1 diabetes mellitus compared with healthy individuals in childhood and adolescence depending on the anthropometric, laboratory data and the duration of the disease. Methods used: there was a single-center, prospective cohort study performed that involved 223 children aged 7 to 18 y/o (113 boys/110 girls), of which 173 had type 1 diabetes mellitus (92 boys/81 girls) and 50 represented the control group (21 boys/29 girls). All of the children had undergone the ophthalmological examination with the assessment of the fundus. Ultrasonic examination of the eyeball and orbit included the assessment of the anterior and posterior sections of the eyeball, optic nerve using B-mode and Doppler modes. Results: an increase in the thickness of the optic nerves with involvement of the membranes (р<0,05) in the groups “7 to 12 y/o” and “13 to 17 y/o” and without involvement of the membranes (р<0,05) in the group “13 to 17 y/o” were revealed in children and adolescents with type 1 diabetes mellitus in comparison with healthy children. Correlation analysis did not determine any significant connection between ultrasonic characteristics of the optic nerves and age, weight, height, lipid profile and glycated hemoglobin level (p<0.05). Conclusion: ultrasonic method allows characterization of the optic nerve, assessment of its structure and the condition of the membranes in children and adolescents with type 1 diabetes mellitus.
Development of reference values for both absolute and relative blood levels of various types of leukocytes as well as the neutrophil index (NI) in both the conditionally “healthy” full-term and the late premature newborns (LPN) is primarily aimed at determining the standards obtained using modern laboratory technologies that would be of help in recognizing hematological disorders in the early neonatal period. This is the continuation of the research with its initial results previously published in the Journal “Pediatria. Journal named after G.N. Speransky” in 2022, i.e.: A.L. Karpova. Complete blood count: reference intervals for full-term and late premature infants in the first day of life (part I). Pediatria n.a. G.N. Speransky. 2022; 101 (1): 62-70. DOI: 10.24110/0031-403X-2022-101-1-62-70. https://doi.org/10.24110/0031-403X-2022-101-1-62-70. The purpose of the research was to determine and compare the general blood test (GBT) reference values in full-term infants and LPN who did not require intensive care and stayed at the obstetrics/physiological departments during the first day of life in order to use them as normative indicators in clinical practice. Materials and methods used: multicenter prospective cohort study was conducted in 2019-2020: 4,376 full-term infants and LPN were examined. Inclusion criteria: 1) the first minute APGAR score of 7 points or above; 2) aged 18 to 24 hours of life; 3) absence of diseases in newborns by the end of the first day of life; 4) gestational age at birth of 35 0/7 to 41 6/7 weeks. In order to avoid errors in obtaining the reference values for white blood cell counts depending on the gestational age (GA), only those children whose GA was specified more precisely, and not as a range - e.g., “37 to 38 weeks,” were included in the analysis. As a result, 3,334 newborns were selected and divided into 2 comparison groups: G1 (n=3,225) of full-term newborns and G2 (n=109) of LPN. Results: reference values of absolute and relative content of different types of leukocytes, leukocytes, lymphocytes, monocytes are presented as tables and nomograms depending on GA. When comparing the GBT parameters, statistically significant differences were found between full-term infants and LPN in the absolute number of segmented neutrophils (13.5 [0.7-44.3] v. 10.1 [3.6-22.0], p<0.001); lymphocytes (5.3 [0.0-25.7] v. 4.7 [2.2-9.5], p<0.001); monocytes (2.1 [0.0-9.1] v. 1.5 [0.2-4.3], p<0.001); eosinophils (0.4 [0.0-4.6] v. 0.2 [0.0-1.4], p<0.001) and basophils (0.0 [0.0-0.3] v. 0.0 [0.0-0.3], p<0.001), which were statistically significantly lower in LPN than in full-term newborns. Conclusion: the absolute number of segmented neutrophils, lymphocytes, monocytes, eosinophils and basophils in the blood of LPN in the first 24 hours of life is lower compared to those of full-term infants.
