Epidemiologic and Clinical Outcomes of Pediatric Renal Tumors in Korea: A Retrospective Analysis of The Korean Pediatric Hematology and Oncology Group (KPHOG) Data / Journal of the Korean Cancer Association, 대한암학회지

Purpose@#Renal tumors account for approximately 7% of all childhood cancers. These include Wilms tumor (WT), clear cell sarcoma of the kidney (CCSK), malignant rhabdoid tumor of the kidney (MRTK), renal cell carcinoma (RCC), congenital mesoblastic nephroma (CMN) and other rare tumors. We investigated the epidemiology of pediatric renal tumors in Korea. @*Materials and Methods@#From January 2001 to December 2015, data of pediatric patients (0–18 years) newly-diagnosed with renal tumors at 26 hospitals were retrospectively analyzed. @*Results@#Among 439 patients (male, 240), the most common tumor was WT (n=342, 77.9%), followed by RCC (n=36, 8.2%), CCSK (n=24, 5.5%), MRTK (n=16, 3.6%), CMN (n=12, 2.7%), and others (n=9, 2.1%). Median age at diagnosis was 27.1 months (range 0-225.5) and median follow-up duration was 88.5 months (range 0-211.6). Overall, 32 patients died, of whom 17, 11, 1, and 3 died of relapse, progressive disease, second malignant neoplasm, and treatment-related mortality. Five-year overall survival and event free survival were 97.2% and 84.8% in WT, 90.6% and 82.1% in RCC, 81.1% and 63.6% in CCSK, 60.3% and 56.2% in MRTK, and 100% and 91.7% in CMN, respectively (p < 0.001). @*Conclusion@#The pediatric renal tumor types in Korea are similar to those previously reported in other countries. WT accounted for a large proportion and survival was excellent. Non-Wilms renal tumors included a variety of tumors and showed inferior outcome, especially MRTK. Further efforts are necessary to optimize the treatment and analyze the genetic characteristics of pediatric renal tumors in Korea.

A Recent Update on Histiocytic Disorder in Children: Focus on Diagnosis and Treatment / 임상소아혈액종양

The histiocytosis is rare disorder characterized by the accumulation of macrophages, dendritic cells, or monocyte-derived cells in various tissues and organs of children and adults. Classifying histiocytic disorders is difficult and has changed over time as an understanding of the biology of these cells has evolved. The most recently revised 2016 WHO classification of histiocytosis and neoplasms of the macrophagedendritic cell lineages has proposed grouping this diverse group of over 100 clinical entities into five main groups based on clinical, histologic, and molecular relevance. Comprehensive genomic studies for histiocytosis have been described and our understanding of the pathogenesis and biology has increased over the past decade. These advances will be able to make precision medicine and targeted therapy possible in patients with histiocytosis. Among the histiocytosis, this review mainly focuses on the updated diagnosis and treatment of Langerhans cell histiocytosis (LCH) and hemophagocytic lymphohistiocytosis (HLH) in children.

Epidemiology of Congenital Bleeding Disorders: a Nationwide Populationbased Korean Study

Background@#Except for data in the Korea Hemophilia Foundation Registry, little is known of the epidemiology of congenital bleeding disorders in Korea. @*Methods@#Data were obtained from the Korean Health Insurance Review and Assessment Service (HIRA) database. @*Results@#From 2010 to 2015, there were 2,029 patients with congenital bleeding disorders in the Korean HIRA database: 38% (n = 775) of these patients had hemophilia A (HA), 25% (n = 517) had von Willebrand disease (vWD), 7% (n = 132) had hemophilia B (HB), and 25% (n = 513) had less common factor deficiencies. The estimated age-standardized incidence rate (ASR) of HA and HB was 1.78–3.15/100,000 and 0.31–0.51/100,000, respectively. That of vWD was 1.38–1.95/100,000. The estimated ASR of HA showed increase over time though the number of new patients did not increase. Most patients with congenital bleeding disorders were younger than 19 years old (47.8%), and most were registered in Gyeonggi (22.1%) and Seoul (19.2%). @*Conclusion@#This is the first nationwide population-based study of congenital bleeding disorders in Korea. This study provides data that will enable more accurate estimations of patients with vWD. This information will help advance the comprehensive care of congenital bleeding disorders. We need to continue to obtain more detailed information on patients to improve the management of these diseases.

Risk Factor Analysis for Secondary Malignancy in Dexrazoxane-Treated Pediatric Cancer Patients / Journal of the Korean Cancer Association, 대한암학회지

PURPOSE: Dexrazoxane has been used as an effective cardioprotector against anthracycline cardiotoxicity. This study intended to analyze cardioprotective efficacy and secondary malignancy development, and elucidate risk factors for secondary malignancies in dexrazoxane-treated pediatric patients. MATERIALS AND METHODS: Data was collected from 15 hospitals in Korea. Patients who received any anthracyclines, and completed treatment without stem cell transplantation were included. For efficacy evaluation, the incidence of cardiac events and cardiac event-free survival rates were compared. Data about risk factors of secondary malignancies were collected. RESULTS: Data of total 1,453 cases were analyzed; dexrazoxane with every anthracyclines group (D group, 1,035 patients) and no dexrazoxane group (non-D group, 418 patients). Incidence of the reported cardiac events was not statistically different between two groups; however, the cardiac event-free survival rate of patients with more than 400 mg/m2 of anthracyclines was significantly higher in D group (91.2% vs. 80.1%, p=0.04). The 6-year cumulative incidence of secondary malignancy was not different between both groups after considering follow-up duration difference (non-D, 0.52%±0.37%; D, 0.60%±0.28%; p=0.55). The most influential risk factor for secondary malignancy was the duration of anthracycline administration according to multivariate analysis. CONCLUSION: Dexrazoxane had an efficacy in lowering cardiac event-free survival rates in patients with higher cumulative anthracyclines. As a result of multivariate analysis for assessing risk factors of secondary malignancy, the occurrence of secondary malignancy was not related to dexrazoxane administration.

Clinical Course of Neutropenia in Previously Healthy Children / 임상소아혈액종양

BACKGROUND: Neutropenia can be easily found in previously healthy children associated with various medical conditions, and the clinical course ranges from transient benign to life threatening. This study aimed to investigate the etiology, clinical characteristics, and clinical courses of neutropenia in previously healthy children. METHODS: We evaluated 215 previously healthy children under aged 18 years who diagnosed with neutropenia in two hospitals. Clinical and laboratory features were analyzed retrospectively based on the medical records. RESULTS: Transient infectious neutropenia (TIN) accounted for 97.7% of cases and chronic neutropenia (CN), for 2.3%. An infectious agent was identified in 128/210 (61%) patients with TIN, and the most frequent agents were viruses (46.5%). The most common viral agent was respiratory syncytial virus (RSV) (29%). TIN subgroups exhibited no differences in severity according to infectious agent (virus, bacteria, Mycoplasma); however, neutropenia severity differed among viral agents [mild-to-moderate neutropenia in the RSV group (857.3±293.3/µL) and moderate-to-severe neutropenia in the parainfluenza group (567.3±198.1/µL); P=0.017]. All patients with CN had anti-neutrophil antibody positivity (autoimmune neutropenia, AIN), and moderate-to-severe neutropenia predominated. The median duration of TIN was 8 days (range, 3–286 days), and it was significantly longer for AIN at 330 days (range, 217–730 days) (P=0.000). The median duration of neutropenia was also different according to each viral agent, with 4 days (range, 3–11 days) for the RSV group and longer durations for 3 other groups (influenza, parainfluenza, other respiratory viruses) (P=0.015). CONCLUSION: Neutropenia in previously healthy children is usually of transient infectious origin, with mild-to-moderate severity, and it resolves spontaneously without complications.

Management of Spontaneous Spinal Epidural Hematoma Developed in Children with Hemophilia / 임상소아혈액종양

Spinal epidural hematoma (SEH) is a rare neurosurgical emergency in which pressure on the spinal cord leads to acute neurological deficits, and is a rare complication in children with hemophilia. We report three cases of SEH in severe hemophilia A. An 8-month-old boy who presented with non-traumatic acute-onset irritability was found to have SEH and was later diagnosed with hemophilia. The two other patients presented with neck pain and magnetic resonance imaging confirmed the diagnosis of SEH. Two patients who received conservative management fully recovered, however the patient who presented with progressive neurological abnormalities at the time of diagnosis, received surgery but later developed breathing difficulties and quadriplegia. Early diagnosis and immediate, aggressive, clotting factor replacement therapy are crucial when managing SEH in children with hemophilia. Immediate and aggressive factor replacement, accompanied by both neurological monitoring and early imaging, are essential for hemophiliac with suspected SEH.

Successful Non-operative Management of Intra-abdominal Hemorrhage in Two Patients with Hemophilia A / 임상소아혈액종양

Spontaneous intra-abdominal hemorrhages are uncommon in hemophilic patients. They can cause complications in patients with severe hemophilia, and are associated with a high mortality rate. To date, there is no guideline for the management of intra-abdominal hemorrhage in patients with hemophilia. Management of intra-abdominal hemorrhage ranges from conservative treatment to emergent embolization or surgery. We describe two children with hemophilia A, who were successfully treated non-operatively by administering coagulation factor concentrates and embolization, and were later discharged from the hospital. We emphasize the role of an active approach in the evaluation and management of intra-abdominal hemorrhage without any surgical intervention.

2 Cases of Beta-thalassemia Minor in Korea / 임상소아혈액종양

In Korea, recent epidemiologic studies show that the incidence of β-thalassemia is increasing as the influx of South-East Asian population increases and molecular technologies develop. However, many patients are still misdiagnosed as iron deficiency anemia (IDA). All patients with microcytic anemia need to perform evaluation including reticulocyte index, Mentzer index, and iron studies. Considering the increasing incidence of β-thalassemia, hemoglobin beta globulin (HBB) gene sequencing should be performed if suspicious. In our cases, patients whose parents were both Koreans were confirmed to have β-thalassemia with a substitution in c1, ATG>GTG, and deletion of the HBB gene. In Korea, initiation condon ATG>AGG (20.9%) is most common mutation, followed by codon 17 (A>T) (17.6%), codon 121 (G>T) (12.1%), and so on. We report two cases of β-thalassemia diagnosed by genetic testing for microcytic anemia.

2 Cases of Beta-thalassemia Minor in Korea / 임상소아혈액종양

In Korea, recent epidemiologic studies show that the incidence of β-thalassemia is increasing as the influx of South-East Asian population increases and molecular technologies develop. However, many patients are still misdiagnosed as iron deficiency anemia (IDA). All patients with microcytic anemia need to perform evaluation including reticulocyte index, Mentzer index, and iron studies. Considering the increasing incidence of β-thalassemia, hemoglobin beta globulin (HBB) gene sequencing should be performed if suspicious. In our cases, patients whose parents were both Koreans were confirmed to have β-thalassemia with a substitution in c1, ATG>GTG, and deletion of the HBB gene. In Korea, initiation condon ATG>AGG (20.9%) is most common mutation, followed by codon 17 (A>T) (17.6%), codon 121 (G>T) (12.1%), and so on. We report two cases of β-thalassemia diagnosed by genetic testing for microcytic anemia.

Successful Non-operative Management of Intra-abdominal Hemorrhage in Two Patients with Hemophilia A / 임상소아혈액종양

Spontaneous intra-abdominal hemorrhages are uncommon in hemophilic patients. They can cause complications in patients with severe hemophilia, and are associated with a high mortality rate. To date, there is no guideline for the management of intra-abdominal hemorrhage in patients with hemophilia. Management of intra-abdominal hemorrhage ranges from conservative treatment to emergent embolization or surgery. We describe two children with hemophilia A, who were successfully treated non-operatively by administering coagulation factor concentrates and embolization, and were later discharged from the hospital. We emphasize the role of an active approach in the evaluation and management of intra-abdominal hemorrhage without any surgical intervention.

High Remission Rate of Chronic Immune Thrombocytopenia in Children: Result of 20-Year Follow-Up

PURPOSE: This study examined the outcomes of children with chronic immune thrombocytopenia (ITP). MATERIALS AND METHODS: We retrospectively analyzed the medical records of all patients diagnosed with ITP from January 1992 to December 2011 at our institution. RESULTS: A total of 128 patients (64%) satisfied the criteria for newly diagnosed ITP, 31 (15%) for persistent ITP, and 41 (21%) for chronic ITP. The median age at diagnosis was 4.5 years (range, 1 month to 18 years). The median platelet count at diagnosis was 32x109/L. A comparison of the initial treatment data from 2001 to 2011 with those from 1992 to 2000 showed that the number of bone marrow examinations decreased, whereas observation increased. Chronic ITP presented at an older age than newly diagnosed and persistent ITP (6.6 years vs. 3.8 years vs. 4.1 years, respectively); however, the difference did not reach statistical significance (p=0.17). The probability of complete remission of chronic ITP was 50% and 76% at 2 and 5 years after diagnosis, respectively. Patients aged <1 year at diagnosis had a significantly better prognosis than did older patients (hazard ratio, 3.86; p=0.02). CONCLUSION: Children with chronic ITP showed a high remission rate after long-term follow-up. This study suggests that invasive treatments such as splenectomy in children with chronic ITP can be delayed for 4 to 5 years if thrombocytopenia and therapeutic medication do not affect the quality of life.

Recent advances in pediatric hemato-oncologic disease

No abstract available.

Differential diagnosis of childhood hemorrhagic disorders

Bruising and bleeding are common events in children. The pediatrician must be able to determine whether a child's symptoms are normal or perhaps indicative of hemorrhagic disorders. A thorough medical history and physical examination should enable the pediatricians to identify those patients warranting further evaluation. This review describes the characteristics of the medical history, physical examination, and clinical laboratory testing that are important in recognizing clinically significant bleeding disorders. This review will help the pediatrician to perform the initial laboratory evaluation, differentiate for patients with bleeding tendency and make the correct diagnosis for a variety of cases.

A Korean Case of De Novo 18q Deletion Syndrome With a Large Atrial Septal Defect and Cyanosis

No abstract available.

Respiratory distress in children and adolescents

Respiratory distress is not uncommon in children and adolescents, who are at higher risk of progressing into respiratory failure than adults. When managing dyspneic pediatric patients, a clinical history and physical examination should be performed simultaneously and must be followed up by prompt first aid, including oxygen supplementation. Prior to initiating an invasive therapy such as endotracheal intubation, noninvasive positive pressure ventilation should first be considered. This method may be effective in patients with chronic respiratory distress and failure. The prevention and proper management of respiratory infections are also important in affected patients. If chronic respiratory distress deteriorates into respiratory failure, a portable home mechanical ventilator may be needed.

Biologic response modifiers

Biotherapy, often called biological therapy or immunotherapy, aims at supporting and helping in the treatment of human disease without chemical drugs and invasive therapies, by restoring the natural immune system. It is also used to reduce certain side effects that may be caused by some treatments against cancer, autoimmune diseases, or other diseases. Biotherapy employs substances called biological response modifiers (BRMs). The term BRM is often used synonymously with the terms immunomodulator and immunostimulant. BRMs are agents that modify the host's response to pathogens with resultant beneficial prophylactic or therapeutic effects. The use of BRMs had rapidly expanded since the introduction of the first diagnostic antibodies. They are now employed in oncology, autoimmune diseases, inflammatory diseases, and transplantation medicine. BRMs used in biological therapy include interferones, interleukins, colony-stimulating factors, monoclonal antibodies, differentiation agents, tyrosine kinase inhibitor, tumor necrosis factor, vaccines, and nonspecific immunomodulating agents. BRMs are widely accepted in the treatment of certain types of cancer and rheumatoid arthritis, while others are being tested in research studies. This article reviewed the clinical use and side effects of BRMs in cancer and other diseases.

Complications of Resection in a Patient with Intracranial Growing Teratoma Syndrome: the Optimal Timing of Surgical Removal? / 임상소아혈액종양

No abstract available.

Complications of Resection in a Patient with Intracranial Growing Teratoma Syndrome: the Optimal Timing of Surgical Removal? / 임상소아혈액종양

No abstract available.

A Case of Benign Multiple Pulmonary Nodules in a Patient with Osteosarcoma / 임상소아혈액종양

When pulmonary nodules are detected in children receiving chemotherapy for osteosarcoma, it is often assumed that they have metastatic disease. However, these pulmonary nodules do not always represent metastatic disease. There are considerable similarities in the computed tomography (CT) images of benign pulmonary nodules and those of malignant nodules. Therefore, we have to be careful when diagnosing pulmonary metastases based on chest CT scan alone. We report a case of benign multiple pulmonary nodules in a patient with osteosarcoma.

Four Children with Multidrug-resistant Acinetobactor baumanii Infections in the Intensive Care Units of a University Hospital / 소아감염

The Multidrug-resistant Acinetobactor baumanii (MDRAB) is an opportunistic pathogen. Patients with long periods of hospital stay and/or under intensive care unit (ICU) receiving invasive management are more susceptible to this pathogen. In this report, four children with MDRAB infection are reviewed and described their clinical characteristics. There had been concurrent outbreaks of MDRAB infection in adult patients in the ICU at this period of time. The first child had received a craniotomy and epidural hematoma evacuation. The second child was admitted for status epilepticus with hydrocephalus. The third child had pneumonia with status epilepticus with hydrocephalus. The fourth child had poor activity due to hypoxic ischemic encephalopathy and convulsive disorder. Except the fourth child, all had not been exposed to carbapenem prior to infection of MDRAB. That imply the cause of MDRAB infections may be associated with invasive management and prolonged hospitalization together with the previous exposure to carbapenem in our cases. We would like to emphasize the importance and minimizing the spread of hospital infection in patients under prolonged intensive care management regardless of the use of carbapenem.