Background. Fanconi anemia (FA) – rare syndrome characterized by congenital malformations, high risk of bone marrow failure and
oncological diseases. Hematopoietic stem cell transplantation (HSCT) – is the only way of correction of hematopoietic insufficiency, but standard very intensive regimens of conditioning are highly toxic for patients with FA.

Patients and methods. During the period from November 1994 to April 2014 transplantations were performed for 29 patients with FA (18 females/11 males, age median – 9.5 years, variation – 3.7–15.4). Indication for HSCT was the transfusion-dependent aplastic anemia at 27 patients and transformation to myelodysplastic syndrome/acute myeloid leukemia at 2 patients. At 17 (58.6 %) patients HLA-matched family donor were used, 12 (41.4 %) patients received unrelated graft. Source: bone marrow (BM) – 18 patients, peripheral blood stem cells – 7 patients, BM + umbilical cord blood (UCB) – 3 patients, UCB – 1 patient. One of the related HSCT was performed from 9/10 donor and 2 unrelated HSCT were from 9/10 donor too. Conditioning regimens: busulphan 4–8 mg/kg, cyclophosphamide – 20–40 mg/kg, fludarabine 150 mg/sq.m., anti-thymocyte globulin. “Graft versus host” disease (GvHD) prophylaxis: cyclosporine A/tacrolimus, methotrexate 5 mg/sq.m. (days +1, +3, +6, +11) /+/– mycophenolate mofetil. Three recipients of unrelated HSCT received TCRα/β grafts.

Results. All patients engrafted. Secondary graft failure diagnosed at 4 (13.8 %) patients – after 1, 2, 6 and 12 months from HSCT. All these patients received second HSCT with conditioning with alemtuzumab, thoracoabdominal irradiation and fludarabine. Two patients died after the second HSCT, causes: GvHD and adenovirus pneumonia. Eighteen patients were free from GvHD, acute GvHD I–II gr. was revealed at 7 (24.1 %) patients, III–IV gr. – at 4 (13,7 %) patients. Limited chronic GvHD was observed at 4 patients, extensive – at 2 patients. Followup median is 31.9 (3.8–246) months. Overall 5 years survival was 67.4 %. Two patients suffered from oropharyngeal squamous cell cancer. Ten patients died in total – 10 from GvHD and one from the cancer.

Conclusion. HSCT with attenuated high immunosuppressive regimens of conditioning can obtain transplant engraftment with minimal visceral toxicity at patients with FA. Frequency of GvHD and infection complications remains high. Non-hematopoietic cancers are the cause of late mortality.

One of the main risk-factors of acute lymphoblastic leukemia (ALL) is age. The most favorable age group is children from 2 to 9 years old, and the adolescents and young adults have the worst prognosis. Treatment of ALL at adolescents and young adults is a unique problem of modern hematology now. This work presents the analysis of 368 cases of patients with primary ALL 15–18 years old, which were registered in the database of Russian-Belarussian group from April 2002 to November 2014. The aim of the analysis was the estimation of effectiveness of different generations of protocol for adolescents and prognostic significance of different factors to reveal the ways of future therapy optimizing in this age group. Event-free survival of adolescents 15–18 years old according our study was 56 ± 5 % in ALL-MB-2002 study and 57 ± 6 % in ALL-MB-2008 study. We did not received any differences in survival based on immunophenotype of blast cells. But the risk group analysis showed the worst results at patients with ALL from pre-B-precursor cells of high risk. No difference was revealed according the gender. One of the general problems remains high toxicity of therapy at patients of this age group. Treatment related mortality (TRM) was 13.2 and 12 % at ALL-MB-2002 and ALL-MB-2008 accordingly. Main cause of deaths remains infection.

Background. Despite of modern methods of diagnosing, prevention and treatment, invasive fungal disease (IFD) remains actual problem after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Data of IFD in adolescents and young adults (15–25 y.o.) after allo-HSCT are limited.

The aim of the study was to estimate of incidence, etiology, clinical signs and outcome of IFD in adolescents and young adults recipients
of allo-HSCT.

Materials and methods. In retrospective single center study from Jan 2013 to Dec 2014 were included 80 pts after first allo-HSCT in the clinic of Raisa Gorbacheva Memorial Research Institute of Children Oncology, Hematology and Transplantation, First Pavlov State Medical University of St. Petersburg. EORTC/MSG 2008 criteria for IFD diagnosis were used. “Active IFD” means IFD diagnosed just before HSCT.

Results. Incidence of IFD before allo-HSCT was 18.8 % (n = 15): invasive aspregillosis (IA) (n = 11), invasive candidiasis (IC) (n = 3) and 1 patient had two IFD (IA + IC). Complete response to antifungal therapy was in 40 %, partial – 26.7 %, “active” IFD – 33.3 %. Secondary antifungal prophylaxis was made predominantly with voriconazole (80%). Patients without IFD before HSCT (n = 65) received primary antifungal prophylaxis predominantly with fluconazole (85 %). Cumulative incidence of IFD-events at 1 year after allo-HSCT was 15 %, at 2 years – 18.8 % including new cases of IFD (n = 14) and relapse of IFD (n = 1) after allo-HSCT with median day of oncet +43 (14–577). Incidence of IA was 15 %, IC – 2.5 %, pneumocystis pneumonia (PCP) – 1.25 %. IFD risk factors after allo-HSCT (< 0.05) were: age < 18 years, non-malignant diseases, active disease at the moment of HSCT, neutropenia grade IV more than 20 days, acute “graft-versus-host” disease. First-line therapy were: IA – voriconazole (58.3 %), IC – echinocandins (100 %), PP – co-trimoxazole (100 %). 12-weeks overall survival (OS) from day of IFD diagnosis after allo-HSCT was 93.3 %, IA – 91.7 %. All patients with IC and PCP alive. 100-days OS after allo-HSCT was 85 %, 2-year OS after allo-HSCT – 67.5 %. There was no difference in OS in patients with or without IFD before allo-HSCT.

Conclusion. Incidence of IFD in adolescents and young adults before allo-HSCT was 18.8 %. Only one patient relapsed with IA after allo-HSCT. Incidence of IFD after allo-HSCT (1 year) was 15 %. The main IFD was invasive aspergillosis. 12-weeks overall survival from IFD diagnosis after allo-HSCT was 93.3 %. IFD before and after allo-HSCT did not impair the outcome of the transplantation in adolescents and young adults.

Primary immunedifficiencies (PID) are genetically driven defects of immune system. Apart of relapsing severe infections, PIDs characterized by immune tolerance disturbance, chronic inflammation, autoimmunity and high risk of malignant diseases. Early diagnosing and in-time pathogenic therapy allows treatment of the majority of patients with PIDs and providing normal quality of life in other cases provides. Main pathogenic methods of treatment are hematopoietic stem cell transplantation and intravenous immunoglobulins (IVIGs) substitutional therapy. Usage of 10 % IVIGs allows decreasing time and economical costs of PIDs therapy.

Nutrition status (NS) disturbance registered at majority of patients receiving treatment on malignancies due to both malignant substrate and specific treatment. Due to this situation a question on actual nutrition of this patients group remains actual. This work presents analysis of actual nutrition status of children with solid non-CNS and CNS tumors on the stage of intensive chemotherapy. Significant decreasing of protein and energy income with consumed ration was revealed. This situation can be a risk-factor of severe nutrition status disturbance. NC dynamics during our study showed that correction with the help of natural products is less-effective at children with malignancies. Received results showed the reasonability of preventive nutritive support for these patients.

Article presents the resume of 2 lectures performed in scientific-educational symposium on retinoblastoma treatment during International society of pediatric oncology congress (SIOP Congress) in Cape-town (South Africa). Lectures on new methods of diagnosing and treatment of retinoblastoma were given by one of the World leaders – prof. F. Doz (France) and A. Moll (Netherlands). New approaches to the therapy were presented: local control with thermotherapy, radiotherapy or intraarterial and intravitreal chemotherapy. Approaches to treatment of two variants of disease (unilateral and bilateral) were presented.