Hepatocellular carcinoma (HCC) is a rare primary liver malignancy in children and adolescents with poor prognosis.

The aim of the study – to evaluate the clinical characteristics, treatment strategies, and outcomes in pediatric and adolescent patients with HCC, and to identify factors influencing survival.

Materials and methods. A retrospective analysis was conducted on 39 patients aged 4 to 16 years with histologically confirmed HCC treated and consulted at the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology between 2012 and 2023. Demographic and clinical features, underlying predisposing conditions, laboratory findings, intrahepatic tumor spread (PRETEXT staging), surgical interventions, and systemic treatment regimens were analyzed.

Results. Median age was 133.5 months (11.1 years; range 49.7–199.4 months). Male to female ratio was 1.1:1. HCC developed de novo in 77 % of patients, while 23 % had predisposing conditions – most commonly chronic hepatitis B. The clinical presentation was predominantly non-specific, and in 28.9 % of cases, the tumor was discovered incidentally. Serum alpha-fetoprotein levels were within normal limits in 45% of patients, particularly in those with fibrolamellar HCC.

Advanced intrahepatic tumor spread (PRETEXT stage III–IV) was observed in 60 % of cases, and distant metastases were present in 18 % of patients. The 5-year overall survival (OS) was 37 %, and the event-free survival was 26 %. The presence of distant metastases was significantly associated with inferior outcomes (5-year OS: 45 % in non-metastatic vs. 0 % in metastatic cases, p < 0.001). PRETEXT stage and histological subtype showed no significant impact on OS or event-free survival.

Surgical treatment (hepatic resection or orthotopic liver transplantation) was performed in 66 % of patients and was significantly associated with improved outcomes: 5-year OS was 54 % in the surgical group versus 0 % in those who did not undergo surgery (p < 0.001). Liver transplantation showed a trend toward better outcomes compared to resection, although the difference was not statistically significant. Initial treatment strategy (primary resection vs. biopsy with neoadjuvant chemotherapy) had no impact on survival.

Conclusions. Pediatric HCC is a rare but highly aggressive malignancy that is often diagnosed at an advanced stage. Radical surgical resection, including liver transplantation in certain cases, remains the primary determinant of prognosis. Improved outcomes require early diagnosis, early discussion of therapeutic tactics in center specialized in hepatobiliary surgery and inclusion of pediatric patients in multicenter clinical trials.

Timely identification, analysis, and prevention of potential errors in radiotherapy are crucial for successful comprehensive treatment.

The aim of this study – to establish a system of peer review of radiation therapy plans in pediatric practice prior to treatment initiation in order to avoid possible major errors in treatment planning as well as to assess and improve the quality of radiation therapy, and consequently, treatment outcomes of pediatric patients.

In study established a 42 % utilization rate for radiation therapy in pediatric practice.

Here, we present the results of a peer review of 589 radiation therapy plans in children conducted from 2019 to 2024, with 43.3 % of them requiring corrections. It was demonstrated that the most significant challenge in ensuring the quality of radiation therapy remained target volume delineation: the most common errors were related to target contouring (36.7 %), less frequently to organs-at-risk contouring (2.3 %), and dose prescription (5.4 %). Radiation therapy planning was most difficult in patients with Hodgkin lymphoma (55.2 % of errors) and those requiring craniospinal irradiation (39.5 % of errors).

 A peer review of radiation therapy plans resulted in a significant (p < 0.001) reduction of errors: from 51.1 % at first review to 23.6 % after the fourth one. These numbers confirm the educational value of the peer review system and its role in furthering professional development of specialists. A peer review of radiation therapy plans in children is an important tool for improving the quality of treatment and minimizing associated risks.

The results of our study emphasize the importance of continuing and expanding the use of radiotherapy planning so that to ensure high quality care and reduce risks for patients.

Background. Medulloblastoma (MB) is one of the most common malignant brain tumors in children. In the standard-risk group, 5-year overall survival reaches 75–85 %, however, high treatment efficacy is accompanied by a considerable risk of late toxic effects that substantially impact patients’ quality of life. The most prevalent complications include cognitive and endocrine disorders, sensorineural hearing loss, and scoliosis. Long-term follow-up of survivors remains non-standardized.

The aim of this study was to comprehensively assess the spectrum and frequency of late effects after chemoradiotherapy in children with standard-risk MB and to identify factors influencing their development.

Materials and methods. A retrospective analysis was performed in 144 patients with standard-risk MB treated at the Dmitry Rogachev National Medical Research Center between 2013 and 2023. All patients received craniospinal irradiation at a dose of 23.4–24.0 Gy or 35.2–36.0 Gy with a posterior fossa boost to 54–55 Gy, followed by maintenance chemotherapy (lomustine, cisplatin, vincristine). To assess late effects (endocrine dysfunction, sensorineural hearing loss, scoliosis, chronic nutritional deficiency, polyneuropathy), detailed clinical and laboratory data were available for 55 patients, neurocognitive outcomes were analyzed in 30, and oncologic follow-up for secondary tumors in all 144. The cumulative incidence of complications was estimated using competing-risks methodology, subgroup comparisons were performed with Gray’s test, with p < 0.05 considered statistically significant.

Results. The median age at the start of radiotherapy was 8.2 years, median follow-up 3.2 years. The most frequent late effects were cognitive (96.7 %) and endocrine disorders (89.1 %). Less frequent complications included scoliosis (50.9 %), sensorineural hearing loss (47.3 %), nutritional deficiency (32.7 %), and polyneuropathy (25.5 %), secondary neoplasms occurred in 4.2 % of patients. The most common cognitive impairments involved reduced psychomotor speed, attention, memory, emotional stability, and learning ability. Following therapy, a statistically significant increase was observed in the frequency of memory impairment (p = 0.01) and learning difficulties (p = 0.027). Growth hormone deficiency was detected in 70.1 % of patients (median time to onset – 1.7 years), the diagnosis was confirmed in 43 %, and growth hormones replacement therapy was initiated in only one-third. Hypothyroidism was diagnosed in 69.1 %, secondary adrenal insufficiency in 21.8 %, and gonadal dysfunction in 16.4 %. Growth hormone deficiency and hypothyroidism developed significantly earlier in patients who received craniospinal irradiation 35.2 Gy, although their long-term cumulative incidence was similar to those treated with 23.4 Gy. The 6-year cumulative incidence of secondary neoplasms was 4.7 %; most were high-grade diffuse gliomas (n = 3) with a median survival not exceeding one year.

Conclusions. Chemoradiotherapy for standard-risk MB is associated with a wide spectrum of late toxicities, predominantly cognitive and endocrine, most of which can be effectively managed when detected in a timely manner. Unwarranted de-escalation of therapy without reliable prognostic justification is unacceptable due to the high risk of fatal relapse. Systematic long-term monitoring of late effects, application of modern toxicity-reduction approaches, and comprehensive rehabilitation programs are essential components of optimal survivorship care for patients with MB.

Beta-thalassemia is a hereditary disorder caused by pathogenic variants in the HBB gene that lead to impaired synthesis of β-globin chains, resulting in ineffective erythropoiesis, development of microcytic hypochromic anemia, and a complex of systemic complications. This article reviews current therapeutic strategies for this condition. Adequate transfusion support combined with regular chelation therapy remains the cornerstone of standard treatment. In current practice, other methods of conservative therapy are also used, such as those targeting fetal hemoglobin induction, stimulation of erythropoiesis, and other pathogenetic mechanisms. Allogeneic hematopoietic stem cell transplantation was until recently the only method capable of providing a complete cure for β-thalassemia. However, its application is limited by the availability of a fully compatible donor and the risks of transplantation complications, including a high risk of graft failure in this disease and graft-versushost disease. The development of gene therapy aimed at restoring β-globin synthesis or inducing fetal hemoglobin expression offers prospects for functional cure without the need for allogeneic hematopoietic stem cell transplantation, overcoming many of its limitations, including the lack of an optimal donor, graft-versus-host disease, and graft rejection. This review provides an analysis of existing treatment methods, their limitations, and the potential for clinical application of gene therapy in β-thalassemia.

This article describes the only case of unstable hemoglobin Calgary in Russia in a two-year-old Russian boy with severe transfusiondependent anemia caused by a de novo point mutation in the β-globin gene (HBB: c.194G>T). The combination of the clinical picture of severe anemia, occurring as a major form of β-thalassemia, with the absence of specific laboratory signs complicated the diagnostic search, which was completed only thanks to modern molecular genetic methods. Currently, the only radical method of treating transfusion-dependent hemoglobinopathies is allogeneic hematopoietic stem cell transplantation. A literature review is presented demonstrating general information about the disease, international literature data on allogeneic hematopoietic stem cell transplantation for unstable hemoglobinopathies, as well as our own experience of successful use of this method. The patient's parents have given consent for the use of information, including photographs of the child, in scientific research and publications.

Over the past decades, survival rate for patients with refractory tumors have been increasing at a very slow. The success of the last decade has been the formation of new classes of drugs – targeting, created to affect certain intracellular molecular targets, as well as immuno-oncological, aimed at surface receptor targets. This article presents a clinical case of a patient with a GD2-positive Wilms tumor who received chemoimmunotherapy using anti-GD2 monoclonal antibodies as reinduction treatment. Within the framework of peripheral blood immunomonitoring, the picture of the main parameters of cellular immunity, as well as their dynamics during treatment, was assessed. During the analysis of the immune status dynamics, all observed parameters associated with an unfavorable outcome, namely the number of regulatory T cells and myeloid suppressor cells, were at low levels. The exception was the absolute neutrophils to lymphocytes ratio, which exceeded the norm by 3–4 times. In addition, after 2 courses of chemoimmunotherapy, the number of NK (CD3^–CD16⁺CD56⁺) and activated HLA^–DR⁺CD8⁺ cells, which are also present in the immunosuppressive lymphocyte cluster, increased. Subsequently, despite an increase in the levels of immunoactivating subpopulations of cells by 1.5–3.0 times (with the exception of γδ T cells), the immunosuppressive pattern of lymphocytes also reacted upward, but the increase in its indicators was not so significant. Again, the exception was regulatory T cells, the number of which tripled. These changes were associated with the progression of the malignant disease. A combination of immuno-oncology agents may need to be considered in the future to enhance T cell effector functions and/or inhibit immunosuppressive pathways. In this context, analyzed systemic immunomarkers in peripheral blood can be used to optimize the combination of chemoimmunotherapy.

Urothelial carcinomas are extremely rare in pediatric patients. Unlike in adults, exogenous risk factors do not play a leading role in the development of this disease in children. This article presents a clinical case of urothelial bladder carcinoma with a relapsing course in a 10-year-old patient.

Infantile acute B-cell lymphoblastic leukemia (B-ALL) is a rare leukemia that develops in children of the first year of life, prone to an aggressive course, a combination of unfavorable prognostic factors, frequent relapses and “switching” of tumor cell differentiation from lymphoid to myeloid. In addition, the physiological features and high vulnerability of the infant's body to the influence of unfavorable external factors cause a high frequency of severe complications of both the underlying disease and the specific treatment, which limits the possibilities of therapy and reduces the likelihood of a favorable outcome in such patients. In this regard, there is no doubt that the search for new curative options that increase the effectiveness and reduce the toxicity of therapy for B-ALL in infants is highly relevant. Given the rarity of this type of leukemia, fundamental studies of the biology of infantile leukemia, extensive clinical trials, and exchange of experience between transplant centers involved in the treatment of such patients are important for improving the effectiveness of its therapy. The clinical case presented in this article demonstrates the main problems of therapy for infantile B-ALL: the initial presence of a set of unfavorable prognostic factors (MLL gene rearrangement, lack of CD10 expression on tumor cells, age under 3 months, hyperleukocytosis, and central nervous system damage at manifestation), “switching” of the blast cell line from lymphoid to myeloid, poor response to glucocorticosteroids and development of severe complications limiting the possibilities of therapy intensification already at the stage of induction therapy, refractory course, relapse at late stages after allogeneic hematopoietic stem cell transplantation. Moreover, an individualized approach and the use of new treatment methods, including immunotherapy, cell therapy and apoptosis inducers, allowed the patient to achieve clinical and hematological remission with negative minimal residual disease a year after allogeneic hematopoietic stem cell transplantation, even after a relapse of leukemia.

This work analysed of the influence of scientific and practical events on the formation of the service of pediatric oncology and hematology in the regions of the Russian Federation. Various formats of events (webinars, clinical rounds and diagnostic colloquiums), aimed at transferring advanced knowledge and technologies, are considered. The effectiveness of these measures was assessed in terms of improving the skills of regional specialists, introducing new diagnostic and treatment methods, and improving treatment outcomes in children with specialized diseases. The article demonstrates the need to expand cooperation between the federal and regional centers to ensure equal access to modern medical care for all children in Russian Federation.

The article presents issues related to the payment for medical care in oncology/oncohematology profile in 24-hour and day inpatient hospital hospital settings. The main changes that have occurred in recent years in the formation of profile clinical and statistical groups are presented, and emphasis is placed on the correctness of determining the total cost of a case when paying for it according to the clinical and statistical groups and the cost-effectiveness of hospitalization of profile patients in a 24-hour hospital.

In 2025, the St. Petersburg State Pediatric Medical University celebrates the anniversary dates – the 100^th anniversary of the University and the 120^th anniversary of the clinical hospital of the St. Petersburg State Pediatric Medical University. These glorious figures are a reason to once again recall the history of the University in the context of the formation of individual specializations in pediatrics and pediatric surgery, in particular, pediatric oncology – one of the most difficult and priority areas of modern medicine.

Objective: to analyze the main stages of the development of pediatric oncology care at St. Petersburg Pediatric University

In 1925, the city hospital on the Vyborgskaya side was transformed into a completely new institution in terms of format and objectives, as the first specialized higher medical educational institution of the pediatric profile in the world – the Leningrad Institute of Maternal and Infant Care. This created a unique environment for the development of all areas of pediatrics, including pediatric oncology.

The pediatric oncology service was formed in the middle of the twentieth century, thanks to the efforts of two pioneering doctors in our country: Lev Abramovich Durnov (1931–2005) in Moscow and Genrikh Arsenievich Fedoreev (1930–1980) in Leningrad. Professor Lev Abramovich Durnov was the founder of the first pediatric oncology department in the USSR in 1962. The establishment of the first specialized department was a crucial step in the development of pediatric oncology. A school of pediatric oncologists is being formed. Active scientific research in the field of children's tumors has begun.

Pediatric oncology has always been one of the most challenging and relevant issues in both pediatric surgery and pediatrics as a whole.

It is a multidisciplinary field that requires collaboration and a comprehensive approach from doctors of various specialties. In the 1960s and 1970s, the Pediatric Institute's Department of Pediatric Surgery, led by Girey Alievich Bairov, continued to develop specific areas of pediatric surgery, including the importance of pediatric oncology, training specialists, and instilling "oncological awareness" in young pediatric students. Pediatric oncology was taught to students in the “Pediatric Surgery” course since 1974. The department was opened on September 1, 2007. This event marked a new chapter in the history of the university and the training of medical professionals in St. Petersburg. From the very beginning, the department's primary goal was to establish an organizational, methodological, and scientific foundation for the qualified teaching of clinical oncology and pediatric oncology. The establishment of the Department of Pediatric Oncology at St. Petersburg State Pediatric Medical University was a significant milestone that elevated the training of specialists to a new, specialized level. In recent years, significant progress has been made in the treatment of pediatric cancer. The cure rate for children with malignant tumors has reached 70 % due to the use of intensive chemotherapy programs and improved supportive treatment. The department's staff actively participates in optimizing treatment programs.

The St. Petersburg State Pediatric Medical University is rightfully considered one of the fundamental and leading centers for pediatric oncology in Russia, where the traditions established by the pioneers continue and develop in modern conditions.