An analysis of dose modifications for infants in 29 Children’s Oncology Group protocols across 10 cancer types revealed 11 sets of criteria defining the infant population using age, weight, body surface area (BSA), or a combination of these parameters and eight dose modification methods. A new method of dosing anticancer drugs in infants was developed based on the rationale that prior modifications were implemented to reduce toxicity, which is not cancer-specific. The new method uses BSA dose banding in dosing tables for infants and children with a BSA < 0.6 m2 and gradually transitions from body weight based to BSA-based dosing. 

The purpose of the current work was the estimation of prognostic significance of cytogenetic and molecular markers, assessed by multiplex ligation-dependent probe amplification (MLPA) in 142 cases of pediatric B-cell precursor acute lymphoblastic leukemia (BCP-ALL) patients. Good-risk genetic (GEN-GR) group consisted of 114 patients carrying either ETV6-RUNX1 or high hyperdiploidy together with normal copy-number status for all 8 genes (IKZF1, PAX5, ETV6, RB1, BTG1, EBF1, CDKN2A/2B and PAR1) or isolated deletions affecting ETV6/ PAX5/BTG1 and ETV6 deletions with a single additional deletion of BTG1/PAX5/CDKN2A/2B. All other patients (n = 28) were classified to genetic poor risk (GEN-PR) group. GEN-PR features were older age (p = 0.015), stratification to high-risk group of ALL-MB 2008 protocol (p = 0.001), higher initial WBC (p = 0.008), M3 marrow status on day 15 (p = 0.002) and lack of remission on day 36 (p = 0.039). GEN-PR patients had statistically significant lower event-free survival (EFS) (0.59 ± 0.11 vs 0.88 ± 0.03; p = 0.0008), overall survival (OS) (0.63 ± 0.15 vs 0.93 ± 0.02; p = 0.0050) and higher cumulative incidence of relapse (CIR) (0.38 ± 0.12 и 0.06 ± 0.02; p < 0.0001) in comparison to GEN-GR patients. Genetic risk group stratification retained its negative prognostic value in multivariate analysis affecting EFS (hazard ratio (HR) – 2.659; 95 % CI 1.047–6.755; p = 0.040) and CIR (HR – 3.864; 95 % CI 1.226–12.183; p = 0.021), nut did not influenced to OS (HR – 1.479; 95 % CI 0.356–6.139; p = 0.590). There was no prognostic significance of genetic risk group classifier in the “B-other ALL” group. Majority of unfavorable events (9 out of 10) and relapse (8 out of 9) in GEN-PR patients were revealed in case of IKZF1 deletion co-occurrence. Moreover all 15 patients carrying IKZF1 deletions were stratified to GEN-PR group. So when we added IKZF1 deletion as extra variable in the multivariate analysis genetic risk group classification lost its prognostic significance on EFS (HR – 0.696; 95 % CI 0.086–5.636; p = 0,735), and CIR (HR – 0.511; 95 % CI 0.053–4.924; p = 0.561), while IKZF1 deletion remained its prognostic value both to risk of unfavorable event (HR – 4.292; 95 % CI 1.521–12.911; p = 0.006) and risk of relapse (HR – 9.163; 95 % CI 3.131–26.815; p < 0.001). Thus, combination of cytogenetic risk group and MLPA markers did not bring any advantage over detection of isolated IKZF1 deletion for the estimation of prognosis in pediatric BCP-ALL. 

Generalized osteopetrosis is a rare hereditary disease characterized by systemic sclerosis of the bones of the skeleton, disorder of bone marrow hematopoiesis and, as a consequence, development of severe anemia and appearance of extramedullary hematopoiesis foci in various parenchymal organs. The problem of osteopetrosis is also relevant for the Russian Federation. A radical approach is the allogeneic hematopoietic stem cells transplantation (allo-HSCT). The article presents the experience of treating patients with osteopetrosis in the department of bone marrow transplantation of the Russian Children Clinical Hospital (RCCH).

In the period from 2010 to 2017 allo-HSCT was performed in 6 patients with autosomal recessive form of osteopetrosis. The median age was 5.5 years (1–11 years). Allo-HSCT was performed using the myeloablative regimen of conditioning with treosulfan, fludarabine and melphalan. Reconstitution of donor leukopoiesis was recorded in 5 of 6 patients. In 1 patient, a recipient of umbilical cord blood, there was no reconstitution of donor leukopoiesis. In the early posttransplant period, severe toxic complications were not recorded. One patient had a graft-versus-host reaction, a cutaneous form, I degree. In 5 patients, hypercalcemia was observed in 2–5 months after HSCT, relieved by the admission of bisphosphonates. One patient with a neurodegenerative form of the disease died in the early posttransplant period during deterioration of a hydrocephalic-hypertensive syndrome (donor hematopoiesis was confirmed). In 4 patients with successful allo-HSCT, the follow-up period was from 5 to 42 months (median – 26 months). All patients have complete hematologic recovery, partial correction of the skull deformity, acceleration of growth rates of the body length.

Thus, allo-HSCT is an effective method of systemic disease control. The defeat of the central nervous system is not corrected by allo-HSCT, which requires earlier diagnosis and the initiation of radical therapy before the onset of severe disability. 

Classical Hodgkin’s lymphoma (HL) in children and adolescents is one of the most well-cured tumors. However, during a long period of follow-up, the death rate from secondary tumors, cerebrovascular diseases and diseases of the cardiovascular system is increased in the reconvalescents. Radiation therapy plays a major role in the occurrence of delayed complications. Modern protocols for the treatment of HL in children and adolescents optimize therapy depending on risk groups and the level of the early response to induction therapy in order to minimize the number of possible side effects.

Nowadays for the most efficient allocation of patients to risk currently there are several risk factors based on clinical, instrumental and laboratory indicators. The leading place in the staging took positron emission tomography. Modern approaches to diagnosis and treatment of HL require a multicenter study with a centralized revision of histological preparations and visualization data to optimize therapy in children and adolescents. 

A vitamin K deficiency is one of the most common causes of increased bleeding associated with disorders of coagulation path of hemostasis in newborns and children of first year of life. Coagulopathy is a manifestation of vitamin K deficiency by the time of birth or due to secondary causes. In the latter case, we should not forget about the more rare causes of vitamin K dependent coagulopathy in children. As an example, the article presents the case of a patient at 6 month of age with intracranial hemorrhage on the background of the secondary deficiency of vitamin K. The child had severe coagulopathy, recurrent course за the disease and resistance to oral therapy with menadione sodium bisulfite, which led to the idea of the presence of secondary coagulopathy. After carrying out the targeted sequencing the child was revealed with pathological changes in the gene ABCB11, which are described in the 2nd type of familial syndrome of intrahepatic cholestasis, established the true reason for the deficiency of vitamin K was established. The girl’s therapy was optimized, by which clinical and laboratory manifestations of hemorrhagic disease of newborns were leveled.

Thus, a comprehensive examination of children with recurrent bleeding associated with vitamin K deficiency due to unclear etiology can indicate the true cause of the disease. 

Neuroblastoma (NB) is a malignant pediatric tumor originating from undifferentiated neural crest cells. About 20 % of high-risk group patients are high-risk group patients are refractory to first line therapy and about 50 % of initial responders later develop a relapse. There is no common tactics for salvage therapy. In spite of a c relatively high response rate observed for most second- and third-line therapy regimens used, the long-term event-free survival is still as low as 5 %, which implies a need for consolidation, e.g. by allogeneic hematopoietic stem cell transplantation (allo-HSCT) from haploidentical donor. 

We present a case report of a patient with systemic NB relapse achieving a long-term disease stabilization after a haplo-HSCT and post-transplant therapy. 

The article presents information on the current development of continuing medical education (CME) in the Russian Federation. The work of a particular specialist in the system and the influence of professional organizations on the development of this issue are shown. Schematically shows the features of passing certification / accreditation in the current period, the regulatory framework is described. The work of the specialists of the National Society of Pediatric Hematologists and Oncologists in the system of CME is presented in the context of the development of the regulatory framework, the accreditation of educational activities and the evaluation of the quality of the events. 

Angiomatoid fibrous histiocytoma (AFH) is an extremely rare soft tissue tumor in children characterized by intermediate biological behavior. This article describes a case of AFH of soft tissue of the lumbar region in 10-year-old child. The literature review describes the state-of-the-art data on epidemiology, molecular genetics, diagnostics and treatment of AFH in children. 

The article shows that, as an oncologist, Max Wilms devoted a number of his studies to purely theoretical questions of oncology. On the other hand, he promoted the development of clinical surgery and oncology, offering a whole range of new methods of diagnosis and therapy of the main forms of malignant tumors of the genital organs and kidneys, making a significant contribution to the international clinical practice. M. Wilms is the author of classical studies of teratoid tumors of the ovary and testis, mixed tumors and kidney tumors. It is noted that the Wilms tumor is a dysontogenetic malignant tumor of the kidney. This tumor, described by M. Wilms in 1899, is one of the most frequent malignant neoplasms in children, usually occurring at the age of 2–5 years old, but it can appear at any age, even among the elderly. In the same year his famous monograph “Die Mischgeschwulste Der Niere” or “The Mixed Tumors of the Kidney” was published. In 1904 M. Wilms was awarded the title of professor, and two years later he published his fundamental work “Intestinal obstruction – pathomorphology and clinic”, which made him famous and recognized among surgeons, oncologists and pathomorphologists around the world. Wilms did not leave pediatric surgery unheeded. As early as 1906, he proposed to determine the type of intestinal invagination, starting from the name of the intestine, which forms the head of the intussusception (ileo-colonic, ceco-colonic, etc.). It is said that according to the aetiology of Hirschsprung’s disease, Max Wilms adhered to the theory of prolonged spasm of the anal canal and as a consequence of the appearance of a secondary megacolon (1904). He confirmed his assumptions by performing manometric studies at the level of the anal canal of the rectum, and by using the method of bougie for the treatment. As a talented scientist and teacher, demanding with himself and his students, Max Wilms published more than 130 scientific papers, being the only author in the overwhelming majority among these papers. He was a principled man and he never conspired with his conscience; at the same time, Wilms was an unusually sympathetic and sensitive teacher, always helping those who wished to study and work.