Pediatric Hematology/Oncology and Immunopathology最新文献

{"title":"Quantitative bone marrow magnetic resonance imaging in children with lymphoblastic leukaemia","authors":"G. V. Tereshchenko, N. A. Kriventsova, D. A. Kupriyanov, M. I. Abu Jabal, A. D. Kopaneva, N. V. Myakova, D. V. Litvinov, A. I. Karachunskiy, G. A. Novichkova","doi":"10.24287/1726-1708-2023-22-3-80-86","DOIUrl":"https://doi.org/10.24287/1726-1708-2023-22-3-80-86","url":null,"abstract":"The aim of the study was to evaluate fat fraction (FF) changes in patients diagnosed with acute lymphoblastic leukaemia (ALL) in comparison with children without haematological disorders. All the patients or their legal representatives gave their informed consent to magnetic resonance imaging (MRI). The study was approved by the Independent Ethics Committee and the Scientific Council of the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology and was conducted in line with the Ethical Principles of the World Health Organization (the Declaration of Helsinki) for Medical Research Involving Human Subjects. The study included 33 healthy volunteers aged 13.4 ± 2.8 years (the control group) and 34 patients with acute phase ALL whose mean age was 12.2 ± 3.6 years (the group of interest). Imaging of the pelvic bones and lumbar vertebrae was performed on a Philips Achieva 3T scanner using the mDixon-quant sequence, with a subsequent construction of FF maps. The Mann–Whitney U-test was used to compare the FF data of the cases with each other and with the controls. Four regions of interest were selected, 100 mm2 each: in the bodies of the right and the left iliac bones as well as in the bodies of the L4 and L5 vertebras. For each group of subjects and each region of interest, mean FF was calculated. In the group of the patients with acute phase ALL, FF was the lowest: 3.53 ± 2.75% and 3,72 ± 3.09% in the bodies of the left and right iliac bones respectively, and 2.62 ± 1.86% and 2.47 ± 2.17% in the L4 and L5 vertebras respectively. In the control group, FF in the respective regions of interest was 51.3 ± 9.5%; 49.9 ± 11.0%; 31.3 ± 8.73% and 32.4 ± 10.3%. It is obvious that bone marrow FF in the patients with ALL differs significantly from the control group. Quantitative MRI can become a new method for the assessment of changes in the bone marrow of children with leukaemias.","PeriodicalId":38370,"journal":{"name":"Pediatric Hematology/Oncology and Immunopathology","volume":"27 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-09-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135032763","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"<i>Helicobacter pylori</i> infection in children with immune thrombocytopenia","authors":"S. M. Ragab, M. A. El-Hawy, S. M. Awad, W. A. Soliman, A. A. Mahmoud","doi":"10.24287/1726-1708-2023-22-3-68-73","DOIUrl":"https://doi.org/10.24287/1726-1708-2023-22-3-68-73","url":null,"abstract":"To detect the association between Helicobacter pylori (H. pylori) infection and immune thrombocytopenia in children and adolescents. Immune thrombocytopenia (ITP) is a common bleeding disorder in childhood. H. pylori is a widespread organism that is present in about 50% of the global population. There is an obvious relation between helicobacter pylori infection and chronic idiopathic thrombocytopenic purpura. A cross-sectional study was conducted in 95 patients with ITP who were recruited from the Hematology Unit, Department of Pediatrics, Menoufia University Hospital in the period from June 2021 to June 2022. The age of the patients ranged between 3.5 and 7.5 years. Fifty-five of them were males and 40 were females. The study was approved by the Ethical Committee of the Faculty of Medicine, Menoufia University. Out of the studied ITP children, 62 (65.3%) were positive for H. pylori antigen in stool, and 33 (34.7%) were negative. There was a significant difference between H. pylori-positive and H. pylori-negative patients regarding the grade of bleeding at presentation; 51 (82.3%) H. pylori-positive patients presented with grade 3 bleeding, 35 (56.5%) of them presented with skin and gum bleeding, 16 (25.8%) presented with skin bleeding and epistaxis. There was a statistically significant difference in the rate of recovery between H. pylorinegative patients (78.8%) and H. pylori-positive patients (22.6%). There was a significant rise in the platelet count in H. pylori-positive patients after the treatment of H. pylori. The prevalence of H. pylori infection in ITP pediatric patients was 65.3%. There was a significant rise in the platelet count in H. pylori-positive ITP children after the treatment of H. pylori.","PeriodicalId":38370,"journal":{"name":"Pediatric Hematology/Oncology and Immunopathology","volume":"68 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-09-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"136345063","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Association between the levels of serum vitamin D and trace elements and joint health in children with hemophilia","authors":"A. A.S. Mahmoud, A. E.A. Sharaf, N. R.M. Bayomy, B. S.T. Abdel Nour, A. A. Mahmoud","doi":"10.24287/1726-1708-2023-22-3-74-79","DOIUrl":"https://doi.org/10.24287/1726-1708-2023-22-3-74-79","url":null,"abstract":"Hemophilia is defined as X-linked recessive bleeding disorder. Recurrent bleeding episodes lead to hemarthrosis. Objectives: to investigate the levels of serum 25(OH) D and trace elements in children with hemophilia A and B and to identify the possible association of these factors with Hemophilia Joint Health Score (HJHS). This case-control study was conducted among children with hemophilia A and B. A total of 48 cases were recruited from the hematology units at the Menoufia University Hospital (n = 36) and Sohag University Hospital (n = 12) from December 2020 to February 2022. Forty healthy controls were matched to cases on age, sex and socioeconomic status. Serum zinc and magnesium levels in the hemophilia patients were significantly lower than in the controls, while serum alkaline phosphatase levels in the cases were significantly higher than in the controls. Informed consent was obtained from all the children's parents and ethical approval was acquired from the ethical committee (ID: 5/2020PEDI38), Faculty of Medicine, Menoufia University. The levels of phosphorus and calcium were the same in two groups. Serum 25(OH) D levels were deficient in 85.4% of the cases and insufficient in 14.6%. None of the hemophilia patients had sufficient levels of serum 25(OH) D. There was no significant correlation between HJHS and the levels of serum trace elements but there was a significant positive correlation between HJHS and annualized bleeding rate and a significant negative correlation between HJHS and serum vitamin D. There was no significant difference regarding the demographic data except for weight and body mass index. The patients had significantly higher weight and body mass index compared to the control group. The levels of serum vitamin D and trace elements were decreased in hemophilia patients, and these low values were associated with the worst joint health.","PeriodicalId":38370,"journal":{"name":"Pediatric Hematology/Oncology and Immunopathology","volume":"84 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-09-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"136345449","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Diagnostic significance of serum erythropoietin as a marker of perinatal brain damage in premature newborns with very low birth weight","authors":"D. R. Sharafutdinova, E. N. Balashova, Yu. V. Kessler, I. A. Vedikhina, Yu. V. Sukhova, А. R. Kirtbaya, A. Yu. Ryndin, T. Yu. Ivanets, O. V. Ionov","doi":"10.24287/1726-1708-2023-22-3-136-145","DOIUrl":"https://doi.org/10.24287/1726-1708-2023-22-3-136-145","url":null,"abstract":"The search for promising markers of brain damage in premature newborns is important for the development and optimization of individual diagnostic and therapeutic approaches to neuroprotection in neonatology. Objective: to evaluate the diagnostic significance of serum erythropoietin (sEPO) on the 1st day of life as a marker of perinatal brain damage in premature infants with very low birth weight (VLBW). The study protocol was approved by the Biomedical Research Ethics Committee (Minutes No.12 of 17 November 2016) and the Scientific Council (Minutes No.19 of 29 November 2016) of the National Medical Research Center for Obstetrics, Gynecology and Perinatology named after the Academician V.I. Kulakov of Ministry of Healthcare of the Russian Federation. Written informed consent to the patients' participation in the study was obtained from their parents. The study included 47 premature infants with VLBW born in 2018 at the National Medical Research Center for Obstetrics, Gynecology and Perinatology named after Academician V.I. Kulakov of the Ministry of Healthcare of the Russian Federation. In these patients, sEPO was determined on the 1st day of life. Depending on the level of sEPO, infants were divided into 3 groups: group 1 – premature infants with VLBW with a low sEPO level on the 1st day of life (< 20 IU/L, n = 24); group 2 – premature infants with VLBW with an average sEPO level of 20–39 IU/L (reference values) (n = 14) – control group; group 3 – premature infants with VLBW with an elevated sEPO level (≥ 40 IU/L, n = 9). We determined the frequency of brain damage, including intraventricular hemorrhages (IVH) and periventricular leukomalacia. sEPO was not correlated with gestational age. In group 1, IVH ≤ Grade II was observed in 4/24 (16.7%) infants; in group 2, IVH ≤ Grade II was observed in 3/14 (21.4%) infants, and 1/14 (7.1%) infant had IVH Grade III; in group 3, IVH ≤ Grade II was noted in 1/9 (11.1%) infant, and IVH Grade III – in 1/9 (11.1%) infant, p > 0.05. There were no cases of periventricular leukomalacia. A high sEPO level on the 1st day of life in premature infants with VLBW was not associated with an increased risk of perinatal brain damage. The clinical value and practical significance of the determination of sEPO on the 1st day of life as a marker of perinatal brain damage in premature infants with VLBW did not demonstrate any benefits. Further studies are required to assess the role of sEPO in predicting neonatal outcomes.","PeriodicalId":38370,"journal":{"name":"Pediatric Hematology/Oncology and Immunopathology","volume":"30 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-09-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135032766","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Assessment of erythroferrone levels in children with chronic kidney disease on regular hemodialysis","authors":"Mahmoud Ahmed El-Hawy, Ahmed Adel Nowir, Shimaa Abdelsatar Zaki, Mohamed Shokry El-Haruon","doi":"10.24287/1726-1708-2023-22-3-130-135","DOIUrl":"https://doi.org/10.24287/1726-1708-2023-22-3-130-135","url":null,"abstract":"Anemia is a common comorbidity in children with chronic kidney disease (CKD) and is associated with adverse outcomes. Erythroferrone (ERFE) is a hepcidin inhibitor whose synthesis is stimulated by erythropoietin, which increases iron absorption and mobilization. Aim of the study: to assess the levels of ERFE hormone in children with CKD on regular hemodialysis. This case–control study was carried out at Menoufia University Hospital and included 70 subjects: 38 healthy individuals (controls) and 32 children with CKD on regular dialysis (cases). The study was approved by the Faculty of Medicine Ethics Committee at Menoufia University. All children were subjected to full history taking, complete clinical examination, blood tests such as complete blood count, reticulocyte count, serum iron, ferritin, and total iron binding capacity, liver and renal function tests, and an immunoassay to measure human ERFE. There was a statistically significant difference in the levels of ERFE between the cases and controls (p < 0.001). There was a significant, strong correlation between the levels of hemoglobin and serum iron and the level of ERFE (r = –0.655, p < 0.001). There was no significant correlation between the administered dose of exogenous erythropoietin and the level of ERFE (p = 0.460). Serum ERFE levels in the children with CKD on regular hemodialysis were significantly higher than in the controls and were negatively correlated with hemoglobin and iron levels. There was no significant correlation between ERFE levels and both serum ferritin and total iron binding capacity levels.","PeriodicalId":38370,"journal":{"name":"Pediatric Hematology/Oncology and Immunopathology","volume":"44 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-09-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"136010891","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A somatostatin analogue and immunosuppressive therapy in the treatment of complex forms of chyloperitoneum and chylothorax in newborns and infants","authors":"A. S. Gurskaya, M. A. Sulavko, R. R. Bayazitov, I. V. Karnuta, E. V. Ekimovskaya, O. N. Nakovkin, D. M. Akhmedova, A. A. Klepikova, R. A. Khagurov, N. V. Petrova, V. А. Skvortsova","doi":"10.24287/1726-1708-2023-22-3-121-129","DOIUrl":"https://doi.org/10.24287/1726-1708-2023-22-3-121-129","url":null,"abstract":"Chyloperitoneum and chylothorax are rare conditions with high mortality rates whose optimal treatment strategy remains unclear. The aim of the study was to evaluate the results of chyloperitoneum and chylothorax treatment with a synthetic somatostatin analogue (octreotide) and immunosuppressive therapy with sirolimus. The study was approved by the Independent Ethics Committee and the Scientific Council of the National Medical Research Center for Children’s Health of Ministry of Healthсare of Russia. The patients' parents gave their consent to the use of their children's data, including photographs, for research purposes and in publications. We conducted a retrospective study of nine children diagnosed with congenital chyloperitoneum and chylothorax who had been treated from 2018 to 2022. All the children received either abdominal or pleural drainage, parenteral nutrition, and conservative therapy with drugs. The first line of therapy was octreotide for 14–20 days that was then switched to sirolimus if there had been no effect. The effectiveness of conservative therapy with octreotide at a dose of 5–10 µg/kg/hour was observed in 5 cases. If there had been no effect by day 14, the patients were started on sirolimus at a dose of 0.05–0.2 mg/day which proved to be effective in all the patients (n = 4). Our study showed that sirolimus is effective in complex cases of chyloperitoneum and chylothorax in newborns and infants. Because of the rarity of these disorders, our conclusions were based on the analysis of a small cohort. To confirm our results and develop uniform diagnostic and treatment guidelines, further, more targeted multicenter research is needed. Until such guidelines are adopted, decisions on the treatment of chyloperitoneum and chylothorax should be made on an individual basis and approved by the medical committee of a treatment center.","PeriodicalId":38370,"journal":{"name":"Pediatric Hematology/Oncology and Immunopathology","volume":"97 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-06-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135287048","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Extracorporeal photopheresis: how, why and for whom?","authors":"I. Kumukova, P. Trakhtman, E. Kurnikova","doi":"10.24287/1726-1708-2023-22-2-159-165","DOIUrl":"https://doi.org/10.24287/1726-1708-2023-22-2-159-165","url":null,"abstract":"   Extracorporeal photopheresis is a method of cell therapy that was developed and introduced into clinical practice of various specialties over 30 years ago but its mechanism of action, clinical application and the possibility of further modification are still on the minds of scientists around the world. Here we provide a review of the existing literature on the major critical aspects of the extracorporeal photopheresis technology as well as information on possible ways of modifying the method, the current understanding of its mechanism of effectiveness, the use in various diseases and pathological conditions and a list of possible side effects.","PeriodicalId":38370,"journal":{"name":"Pediatric Hematology/Oncology and Immunopathology","volume":"79 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-05-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"85533750","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Mycosis fungoides in an 11 year-old child: a case report","authors":"M. N. Korsantiya, D. Abramov, A. Efimova, A. V. Pshonkin, N. Myakova","doi":"10.24287/1726-1708-2023-22-2-113-122","DOIUrl":"https://doi.org/10.24287/1726-1708-2023-22-2-113-122","url":null,"abstract":"   Primary cutaneous lymphomas are quite rare in children. Clinical and histopathological manifestations of these diseases in children differ significantly from those in adults. Due to their rarity and complex clinical presentation, diagnosis may take long time. Mycosis fungoides (MF) is the most commonly diagnosed form of primary cutaneous lymphomas in childhood. There are no clinical guidelines for the treatment of children. Literature data on MF variants in children are scarce; the largest study includes 34 patients who were diagnosed on average 4 years after the onset of the first symptoms. In the present article we describe a clinical case of MF in an 11-year-old child with an 8-year history of multiple lesions of the skin and scalp. The patient's parents gave their consent to the use of their child's data, including photographs, for research purposes and in publications.   The aim of our article is to demonstrate the problems in the diagnosis of the disease, especially at an early stage, because its symptoms may be similar to those of many common pediatric inflammatory skin conditions.","PeriodicalId":38370,"journal":{"name":"Pediatric Hematology/Oncology and Immunopathology","volume":"5 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-05-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"80324966","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Results of hematopoietic stem cell transplantation in children with acute leukemia: a single-center experience","authors":"I. Kostareva, K. Kirgizov, E. Machneva, T. Aliev, Yu. V. Lozovan, K. Sergeenko, N. Burlaka, T. I. Potemkina, K. Mitrakov, A. Y. Yelfimova, A. S. Slinin, M. D. Malova, R. R. Fatkhullin, N. Stepanyan, N. Batmanova, T. Valiev, S. Varfolomeeva","doi":"10.24287/1726-1708-2023-22-2-16-23","DOIUrl":"https://doi.org/10.24287/1726-1708-2023-22-2-16-23","url":null,"abstract":"   Currently, allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective treatment option for relapsed / refractory (R / R) acute leukemia (AL) and high-risk AL. In this article, we present our own experience of allo-HSCT in children with R / R AL. The study was approved by the Independent Ethics Committee and the Scientific Council of the N. N. Blokhin National Medical Research Center of Oncology. Fifty-one patients with R / R AL were included in the study: 32 patients had acute lymphoblastic leukemia (ALL), 17 patients had acute myeloid leukemia (AML) and 2 patients had biphenotypic leukemia (BL). All patients underwent allo-HSCT from January 2021 to October 2022. The median age was 8.7 years (5 months – 17 years). At the time of allo-HSCT, 26 patients were in the second (and further) remission, the rest were in the first clinical and hematologic remission (high-risk AML and refractory ALL). Twenty-one (41.2 %) patients received allo-HSCT from a haploidentical donor, 19 (37.2 %) patients underwent allo-HSCT from an HLA-matched related donor and 11 (21.6 %) patients – from an HLA-matched unrelated donor. Pre-transplant conditioning in ALL: 27 patients received regimens based on total body irradiation at a dose of 12 Gy, 4 patients received busulfan-based conditioning regimens, and in 1 patient we used treosulfan. In AML and BL, we used conditioning regimens based on treosulfan/thiotepa (n = 10), treosulfan/melphalan (n = 8) or busulfan / melphalan (n = 1). Bone marrow (in 14 patients) and peripheral blood stem cells (in 37 patients) were used as a source of hematopoietic stem cells. In haploidentical allo-HSCTs in order to prevent graft-versus-host disease (GVHD) we performed TCRab/CD19 depletion followed by additional administration of abatacept / tocilizumab / rituximab on day –1 in 15 patients, 6 patients received post-transplant cyclophosphamide. In transplantations from HLA-matched related and unrelated donors, patients received combined immunosuppressive therapy with abatacept and rituximab on day –1, and calcineurin inhibitors were used as basic immunosuppressive therapy. All patients engrafted with a median time to engraftment of 13 (range, 9 to 24) days after allo-HSCT. Eight (15.7 %) patients developed a relapse of AL at different times after HSCT (five of them are alive). At the median follow-up of 9 (5–25) months, the overall and disease-free survival survival rates were 76.4 % and 68.8 %, respectively, for patients with AL. Acute GVHD was observed in 72.5 % of children, grade 3–4 GVHD was observed in 5.3 % of patients, and 13.7 % of children developed chronic GVHD. Most patients developed infectious complications in the early post-transplant period: febrile neutropenia (96.0 %), reactivation of viremia (47.3 %,) oropharyngeal mucositis (78.4 %), acute cystitis (12.3 %). The overall mortality rate was 17.6 %. Late mortality was associated with a relapse of AL.","PeriodicalId":38370,"journal":{"name":"Pediatric Hematology/Oncology and Immunopathology","volume":"3 2-3 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-05-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"79720808","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Experience with the use of Hizentra, an immunoglobulin preparation for subcutaneous administration, in patients with primary immunodeficiency diseases","authors":"A. Avedova, Y. Rodina, D. Yukhacheva, V. Burlakov, E. Deripapa, A. Shcherbina","doi":"10.24287/1726-1708-2023-22-2-92-97","DOIUrl":"https://doi.org/10.24287/1726-1708-2023-22-2-92-97","url":null,"abstract":"   Immunoglobulin replacement therapy is the gold standard of treatment for patients with antibody deficiencies.   We aimed to investigate the efficacy and safety of replacement therapy with subcutaneous immunoglobulin (SCIG) Hizentra in patients with primary immunodeficiencies.   This study was approved by the Independent Ethics Committee and the Academic Council of the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology. All patients and / or their legal representatives gave informed consent for this treatment. In our study, 12 patients under 18 years of age with various forms of primary immunodeficiencies who had previously received intravenous immunoglobulin were switched to SCIG treatment to receive weekly infusions of Hizentra. Therapy SCIG was administered weekly at a dose of 0.1–0.15 g/kg by rapid push infusion. All patients received Hizentra for at least 3 months. None of the patients included in the study developed severe infections. Immunoglobulin G levels in blood after 3 months of SCIG therapy were significantly higher compared to those achieved on previous intravenous immunoglobulin therapy. There were no severe adverse events associated with Hizentra administration. Our study demonstrated Hizentra to be effective and safe for the treatment of children with various forms of primary immunodeficiencies.","PeriodicalId":38370,"journal":{"name":"Pediatric Hematology/Oncology and Immunopathology","volume":"261 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-05-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"76771196","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}