Retrospective comparative analysis of two medical evacuation systems for Ukrainian patients affected by war.

Introduction: Coordinated medical evacuations represent an important strategy for emergency response when healthcare systems are impaired by armed conflict, particularly for patients diagnosed with life-threatening conditions such as cancer. In this study, we compare the experiences of two parallel medical evacuation systems developed to meet the medical needs of Ukrainians affected by war.

Methods: This retrospective study compared outcomes of two medical evacuation systems, developed by the European Union Emergency Response Coordination Centre (ERCC) and Supporting Action for Emergency Response in Ukraine (SAFER Ukraine) collaborative, in the first 10 months after the war's intensification in Ukraine (February 24 to December 21, 2022).

The HyperPed-COVID international registry: Impact of age of onset, disease presentation and geographical distribution on the final outcome of MIS-C.

Objectives: The aim of the study was to establish an international multicenter registry to collect data on patients with Multisystem Inflammatory Syndrome in Children (MIS-C), in order to highlight a relationship between clinical presentation, age of onset and geographical distribution on the clinical outcome.

Study Design: Multicenter retrospective study involving different international societies for rare immunological disorders.1009 patients diagnosed with MIS-C between March and September 2022, from 48 centers and 22 countries were collected.

Caring for children with cancer evacuated from Ukraine: The patients' perception.

Background And Aims: Since the beginning of the war in Ukraine on February 24, 2022, many pediatric oncology centers welcomed evacuated patients. To better understanding the needs of patients and families arriving at two Lombardy hospitals in the period March to November 2022, an anonymous questionnaire investigated the families' backgrounds, feelings, and impressions about hospitality and care.

Methods: Twenty questions investigated how patients had reached Italy, from whom they had received help (logistically/financially); the emotions regarding their status as war refugees; the knowledge, expectations, and opinions about Italy and Italians; the quality of medical care received and the relationships with the healthcare staff; lastly, suggestions to improve assistance.

Simoctocog alfa (Nuwiq®) in previously untreated patients with severe haemophilia A-Final efficacy and safety results from the NuProtect study.

Introduction: Simoctocog alfa (Nuwiq®) is a 4th generation recombinant FVIII with proven efficacy for the prevention and treatment of bleeding episodes (BEs) in previously treated patients with severe haemophilia A. The NuProtect study assessed the immunogenicity, efficacy and safety of simoctocog alfa in 108 previously untreated patients (PUPs). The incidence of high-titre inhibitors was 16.

Long-term immunogenicity, efficacy and tolerability of simoctocog alfa in patients with severe haemophilia A who had completed the NuProtect study in previously untreated patients.

Background: The NuProtect study reported data on the immunogenicity, efficacy and tolerability of simoctocog alfa (Nuwiq ) in 108 previously untreated patients with severe haemophilia A planned to be treated for ≥100 exposure days or up to 5 years. The NuProtect-Extension study collected long-term prophylaxis data in children with severe haemophilia A.

Methods: Patients who completed the NuProtect study according to the protocol were eligible for the NuProtect-Extension study, a prospective, multinational, non-controlled, Phase 3b study.

FEATURES OF DIAGNOSIS AND ADVERSE COURSE OF NEONATAL JUVENILE XANTHOGRANULOMA: CASE REPORT.

The work describes a case of rare neonatal systemic juvenile xanthogranuloma with an initial damage of the scalp, limbs, back and abdomen, multiple damages of the parenchyma of both lungs, spleen and liver with the development of a severe form of congenital cholestatic hepatitis. The diagnosis was established on the basis of histopathological and immunohistochemical examination of the skin nodules. The child on the background of therapy under the Langerhans cell histiocytosis III program achieved a partial response, which was manifested by a reduction of granulomatous formations on the skin, elimination of liver failure, but retained hepatosplenomegaly, specific lesions of the lung parenchyma, liver, and left kidney.

Monogenic early-onset lymphoproliferation and autoimmunity: Natural history of STAT3 gain-of-function syndrome.

Background: In 2014, germline signal transducer and activator of transcription (STAT) 3 gain-of-function (GOF) mutations were first described to cause a novel multisystem disease of early-onset lymphoproliferation and autoimmunity.

Objective: This pivotal cohort study defines the scope, natural history, treatment, and overall survival of a large global cohort of patients with pathogenic STAT3 GOF variants.

Methods: We identified 191 patients from 33 countries with 72 unique mutations.

Translating Research to Action: The Development of a Pediatric Palliative Cancer Care Advocacy Tool in Eurasia.

Purpose: The Assessing Doctors' Attitudes on Palliative Treatment study was conducted in 11 Eurasian countries to assess physician knowledge of and structural barriers to integration of palliative care into pediatric oncology. After publication, regional collaborators identified the need to disseminate country-specific study results locally and provide policy recommendations to inform stakeholders.

Methods: The Assessing Doctors' Attitudes on Palliative Treatment report was developed with Eurasian and St Jude pediatric palliative care and oncology experts to summarize study findings and deliver country-level data to local stakeholders.

TREC/KREC levels in children with ataxia-telangiectasia.

The aim of the study was to determine the TREC/KREC levels in the patients diagnosed with ataxia-telangiectasia (AT) and to establish their informative value for early diagnosis of this pathology. TRECs and KREC assay was performed using real-time polymerase chain reaction on the DNA of 25 patients diagnosed with AT aged 3 to 14 years and of 173 healthy individuals of the control group aged 1 to 12 years. Clinical and laboratory characteristics of patients were ascertained using their medical records.

Simoctocog Alfa (Nuwiq) in Previously Untreated Patients with Severe Haemophilia A: Final Results of the NuProtect Study.

Introduction:  FVIII inhibitor development is the most serious contemporary treatment complication in haemophilia A, particularly in previously untreated patients (PUPs). No inhibitors developed in clinical trials in previously treated patients treated with simoctocog alfa (Nuwiq), a fourth-generation recombinant FVIII produced in a human cell line.

Methods:  The NuProtect study investigated the immunogenicity of simoctocog alfa in PUPs.

Woman with Turner syndrome and her child with acute leukemia (a case report).

Turner syndrome (TS) is a chromosomal condition that affects development in females. The case of TS in the mother whose child was diagnosed with acute leukemia at the age of 1.5 years is presented.

Determinants of Discordance Between Criteria for Inactive Disease and Low Disease Activity in Juvenile Idiopathic Arthritis.

Objective: To assess concordance among criteria for inactive disease (ID) and low disease activity (LDA) in juvenile idiopathic arthritis (JIA) and to seek factors driving discordance.

Methods: The frequency of fulfillment of existing criteria was evaluated in information on 10,186 patients extracted from 3 cross-sectional data sets. Patients were divided up according to the functional phenotypes of oligoarthritis and polyarthritis.

Hematopoietic Stem Cell Transplantation Positively Affects the Natural History of Cancer in Nijmegen Breakage Syndrome.

Purpose: Nijmegen breakage syndrome (NBS) is a DNA repair disorder with a high predisposition to hematologic malignancies.

Experimental Design: We describe the natural history of NBS, including cancer incidence, risk of death, and the potential effectiveness of hematopoietic stem cell transplantation (HSCT) in preventing both pathologies: malignancy and immunodeficiency.

Results: Among 241 patients with NBS enrolled in the study from 11 countries, 151 (63.

Blood calprotectin in children with juvenile idiopathic arthritis: relationship to flare development after discontinuation of treatment.

Objectives: The study aim was to prospectively evaluate the relationship between disease flare development in children with juvenile idiopathic arthritis (JIA) after discontinuation of treatment and serum calprotectin levels (MRP8/14).

Material And Methods: Determination of blood serum level of calprotectin was performed in 54 patients with inactive JIA from various regions of Ukraine. The inclusion criterion was the existence of an inactive state of the disease in children with JIA for at least 6 months.

Difficulties in diagnosing of measles inclusion body encephalitis in a child with acute lymphoblastic leukemia.

The measles epidemic was observed in Ukraine during 20172019. According to WHO, in Ukraine there was registered the highest number of measles cases in Europe during that period [8]. Measles is characterized by an acute course with fever, maculopapular rash, cough, conjunctivitis and can lead to central nervous system complications (encephalitis, encephalomyelitis) and bronchopulmonary system complications (laryngotracheobronchitis, bronchiolitis, pneumonia).

Distinct molecular response patterns of activating STAT3 mutations associate with penetrance of lymphoproliferation and autoimmunity.

Germline STAT3 gain-of-function (GOF) mutations have been linked to poly-autoimmunity and lymphoproliferation with variable expressivity and incomplete penetrance. Here we studied the impact of 17 different STAT3 GOF mutations on the canonical STAT3 signaling pathway and correlated the molecular results with clinical manifestations. The mutations clustered in three groups.

Immunogenicity, efficacy and safety of Nuwiq (human-cl rhFVIII) in previously untreated patients with severe haemophilia A-Interim results from the NuProtect Study.

Introduction: Nuwiq (Human-cl rhFVIII) is a fourth generation recombinant FVIII, produced in a human cell line, without chemical modification or protein fusion. No inhibitors developed in studies with Nuwiq in 201 previously treated patients with haemophilia A (HA). The immunogenicity, efficacy and safety of Nuwiq in previously untreated patients (PUPs) with severe HA are being assessed in the ongoing NuProtect study.

Measurement of blood calprotectin (MRP-8/MRP-14) levels in patients with juvenile idiopathic arthritis.

Objectives: The aim of the investigation was to compare blood calprotectin (MRP8/14, S100A 8/9) levels in patients with systemic-onset, polyarticular, RF-negative and oligoarticular subtypes of juvenile idiopathic arthritis (JIA), and to explore links between blood calprotectin levels and clinical and laboratory markers of JIA activity.

Material And Methods: Measurement of calprotectin in blood serum was performed in 160 patients with JIA followed up at Lviv Regional Council Public Institution "Western-Ukrainian Specialised Children's Medical Centre". Seventeen patients with systemic-onset JIA (sJIA) and 49 patients with other JIA subtypes (RF-negative polyarthritis and oligoarthritis) in the active phase of the disease were included in this study.

Nijmegen Breakage Syndrome: Clinical and Immunological Features, Long-Term Outcome and Treatment Options - a Retrospective Analysis.

Purpose: Nijmegen Breakage Syndrome (NBS) is a rare inherited condition, characterized by microcephaly, chromosomal instability, immunodeficiency, and predisposition to malignancy. This retrospective study, characterizing the clinical and immunological status of patients with NBS at time of diagnosis, was designed to assess whether any parameters were useful in disease prognosis, and could help determine patients qualified for hematopoietic stem cell transplantation.

Methods: The clinical and immunological characteristics of 149 NBS patients registered in the online database of the European Society for Immune Deficiencies were analyzed.

Predictors of juvenile idiopathic arthritis course.

Introduction: Juvenile idiopathic arthritis (JIA) is a heterogeneous group of inflammatory diseases of joints in children with various and often unfavourable prognosis. It is possible to improve the outcome of the disease in patients with JIA by a correct therapeutic choice made at disease onset - one that enables fast achievement of an inactive disease state and remission. The aim of the investigation was to develop a model/application for automatic calculation of risk of treatment-refractory JIA taking into account the combined action of clinical and cytokine factors.