Background/Aims: Veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is one of the most fatal complications of hematopoietic cell transplantation (HCT), and defibrotide is the only curative drug. We conducted this study to confirm the survival rate of VOD/SOS patients diagnosed in Korea and assess the efficacy of defibrotide. Methods: Patients diagnosed with VOD/SOS after allogenic HCT between 2003 and 2020 were enrolled. We investigated day +100 survival rates and associated risk factors in patients who satisfied the modified Seattle criteria within 50 days of HCT. Results: A total of 110 patients satisfied the modified Seattle criteria, of which 65.5% satisfied the Baltimore criteria. Thirty-seven patients were treated with defibrotide. The day +100 survival rate of the 110 patients was 65.3%. The survival rates in patients who did not meet the Baltimore criteria and in those who did were 86.8% and 53.7%, respectively (p = 0.001). The day +100 survival rate of patients treated with defibrotide was 50.5%. Among the patients receiving defibrotide, those whose creatinine levels were more than 1.2 times the baseline had a significantly lower survival rate at 26.7% (p = 0.014). On multivariate regression analysis, the hazard ratio of satisfaction of the Baltimore criteria was 4.54 (95% confidence interval [CI], 1.69 to 12.21; p = 0.003). In patients treated with defibrotide, the hazard ratio was 8.70 (95% CI, 2.26 to 33.45; p = 0.002), when creatinine was more than 1.2 times the baseline on administration. Conclusions The day +100 survival rate was significantly lower when the Baltimore criteria were satisfied, and when there was an increase in creatinine at the time of defibrotide administration.

Purpose: Acute promyelocytic leukemia (APL) is a rare disease in children and there are some different characteristics between children and adult. We aimed to evaluate incidence, clinical characteristics and treatment outcomes of pediatric APL in Korea. Materials and Methods: Seventy-nine pediatric APL patients diagnosed from January 2009 to December 2016 in 16 tertiary medical centers in Korea were reviewed retrospectively. Results: Of 801 acute myeloid leukemia children, 79 (9.9%) were diagnosed with APL. The median age at diagnosis was 10.6 years (range, 1.3 to 18.0). Male and female ratio was 1:0.93. Thirty patients (38.0%) had white blood cell (WBC) count greater than 10×109/L at diagnosis. All patients received induction therapy consisting of all-trans retinoic acid and chemotherapy. Five patients (6.6%) died during induction chemotherapy and 66 patients (86.8%) achieved complete remission (CR) after induction chemotherapy. The causes of death were three intracranial hemorrhage, one cerebral infarction, and one sepsis. Five patients (7.1%) suffered a relapse during or after maintenance chemotherapy. The estimated 4-year event-free survival and overall survival (OS) rates were 82.1%±4.4%, 89.7%±5.1%, respectively. The 4-year OS was significantly higher in patients with initial WBC < 10×109/L than in those with initial WBC ≥ 10×109/L (p=0.020). Conclusion This study showed that the CR rates and survival outcomes in Korean pediatric APL patients were relatively good. The initial WBC count was the most important prognostic factor and most causes of death were related to serious bleeding in the early stage of treatment.

Background: Hereditary hemolytic anemia (HHA) is a rare disease characterized by premature red blood cell (RBC) destruction due to intrinsic RBC defects. The RBC Disorder Working Party of the Korean Society of Hematology established and updated the standard operating procedure for making an accurate diagnosis of HHA since 2007. The aim of this study was to investigate a nationwide epidemiology of Korean HHA. Methods: We collected the data of a newly diagnosed pediatric HHA cohort (2007–2016) and compared this cohort's characteristics with those of a previously surveyed pediatric HHA cohort (1997–2006) in Korea. Each participant's information was retrospectively collected by a questionnaire survey. Results: A total of 369 children with HHA from 38 hospitals distributed in 16 of 17 districts of Korea were investigated. RBC membranopathies, hemoglobinopathies, RBC enzymopathies, and unknown etiologies accounted for 263 (71.3%), 59 (16.0%), 23 (6.2%), and 24 (6.5%) of the cases, respectively. Compared to the cohort from the previous decade, the proportions of hemoglobinopathies and RBC enzymopathies significantly increased (P < 0.001 and P = 0.008, respectively). Twenty-three of the 59 hemoglobinopathy patients had immigrant mothers, mostly from South-East Asia. Conclusion In Korea, thalassemia traits have increased over the past 10 years, reflecting both increased awareness of this disease and increased international marriages. The enhanced recognition of RBC enzymopathies is due to advances in diagnostic technique; however, 6.5% of HHA patients still do not have a clear diagnosis. It is necessary to improve accessibility of diagnosing HHA.

BACKGROUND: von Willebrand disease (VWD) is one of the most common inherited bleeding disorders. However, the number of patients who register to the Korea Hemophilia Foundation (KHF) is much lower than the expected prevalence rate and only few hospitals perform tests for diagnosis autonomously. Thus, we surveyed practical realities of VWD in Yeungnam region. METHODS: Patients with VWD (N=267) who were diagnosed at eleven university hospitals from March 1995 to March 2018 were enrolled in this study. We evaluated the medical records from each hospital retrospectively. RESULTS: Two hundred and twenty-eight children and 39 adults met the diagnostic criteria for VWD. Seventy-eight (57.4%) patients had the blood type O. Fifty-eight patients were definite type 1 (21.7%), 151 were possible type 1 (56.6%), and the others were type 2. Abnormal laboratory findings were the most common factor for the diagnosis in children. VWF mutations were detected in 17 patients. Patients with a family history showed age of diagnosis of 9 y, which is higher than in those with no family history (6 yr), and also showed a higher rate of significant bleeding (32.1% vs. 14.2%). VWF:RCo and VWF:Ag tests were performed in-hospital at only 1 of 11 hospitals. Twelve of 267 patients were enrolled at the KHF (4.5%). CONCLUSION: A high rate of out-sourcing studies may result in inaccurate diagnosis. The registration rate to the KHF is still lower than the prevalence rate. A comprehensive nationwide registration system is necessary in order to identify the actual prevalence rate and promote the diagnosis of VWD in Korea.
Adult ; Child ; Diagnosis ; Hemophilia A ; Hemorrhage ; Hospitals, University ; Humans ; Korea ; Medical Records ; Prevalence ; Retrospective Studies ; von Willebrand Diseases

BACKGROUND: Venous thromboembolism (VTE) is rare in pediatric patients compared to adults, but it's incidence is gradually increasing. The purpose of this study was to analyze the incidence, risk factors, and prognosis of pediatric patients with VTE in Korea. METHODS: Between January 2000 and July 2017, 249,312 medical records of the patients older than 1 year who were hospitalized in the department of pediatrics of 10 university hospitals in Yeungnam region were retrospectively reviewed. RESULTS: The overall incidence of VTE was 4.9 per 10,000 admissions. Of the total 123 patients, 80 (65.0%) were male and the median age was 10.8 years (range, 1.0–23.5 years). Magnetic resonance imaging was performed most frequently to confirm the diagnosis of VTE (43.1%). Thrombosis occurred in the cerebral vessels (46.3%), lower extremities (23.8%), pulmonary (19.5%), abdomen (9.8%), and upper extremities (4.1%). One hundred and six patients had underlying causes such as cancer (27.6%), infection (26.8%), intravenous catheter insertion (17.9%), and surgery (14.6%). Protein C was evaluated in 39 patients (31.7%), protein S in 40 (32.5%), antithrombin (AT) III in 52 (42.3%), and homocysteine in 21 (17.1%). Among them, one patient with a family history of AT III deficiency had SERPINC gene mutation. Seventy-seven patients (62.6%) started anticoagulation treatment. Most (52.0%) were treated for more than 90 days. CONCLUSION: Healthcare providers must be aware of the potential for VTE development in childhood. In the near future, a nationwide survey should be investigated to determine the incidence rate and the trends in VTE among Korean children.
Abdomen ; Adult ; Catheters ; Child ; Diagnosis ; Epidemiology ; Health Personnel ; Homocysteine ; Hospitals, University ; Humans ; Incidence ; Korea* ; Lower Extremity ; Magnetic Resonance Imaging ; Male ; Medical Records ; Pediatrics ; Prognosis ; Protein C ; Protein S ; Retrospective Studies ; Risk Factors ; Thrombosis ; Upper Extremity ; Venous Thromboembolism*

PURPOSE: Blood culture is an essential diagnostic tool and requires clear indications, proper techniques, and quality control. We aimed to investigate whether blood cultures in children are appropriate for indications, are performed correctly, and receive proper quality control. METHODS: We conducted an online survey targeting pediatric infectious diseases (ID) specialists working in general hospitals and neonatologists (Neo) working at hospitals operating a neonatal intensive care unit in Korea. RESULTS: Approximately 81.1% (30/37) of pediatric ID specialists and 72.2% (52/72) of Neo responded to the survey. Some of the respondents (33.3% of ID and 59.6% of Neo) performed blood culture as a regular test irrespective of the indication. Approximately 40% of ID and 65.4% of Neo ordered only one set of blood culture in patients suspected with bacteremia. The most commonly used disinfectant for skin preparation was povidone-iodine, while the skin preparation method varied by institution. Approximately two-thirds of the institutions were monitoring the blood culture contamination rate, whereas relatively few provided staff with feedback on that rate. In addition, less than half of the institutions were providing regular staff training on blood culture (40% of ID and 28.8% of Neo). CONCLUSIONS: The indication and methods of blood culture for children varied according to institution, and few hospitals exert effort in improving the quality of blood culture. Institutions have to strive constantly toward improvement of blood culture quality and evidence-based recommendations for pediatric blood cultures should be standardized.
Bacteremia ; Child ; Communicable Diseases ; Hospitals, General ; Humans ; Infant, Newborn ; Intensive Care, Neonatal ; Korea ; Methods ; Povidone-Iodine ; Quality Control ; Skin ; Specialization ; Surveys and Questionnaires

Massive fetomaternal hemorrhage (FMH) is a major cause of unexplained fetal death and neonatal anemia. FMH can be diagnosed using the Kleihauer-Betke test or flow cytometry by identifying the presence of fetal red cells in the maternal blood. However, timely diagnosis is a challenge because many hospitals lack the equipment needed to perform such tests. The authors experienced a case of FMH diagnosed via high-performance liquid chromatography (HPLC) which is generally used in measuring glycated hemoglobin (HbA1c) in a patient with unexplained neonatal anemia. A girl aged 2 days was transferred to our hospital for showing pallor and a hemoglobin level of 5.0 g/dL. HPLC revealed 3% fetal hemoglobin (HbF) in the maternal blood. HPLC is a quick test for quantifying HbF that is readily available in many hospitals and could serve as a promising alternative for diagnosing FMH.
Anemia, Neonatal ; Chromatography, High Pressure Liquid ; Chromatography, Liquid ; Diagnosis ; Female ; Fetal Death ; Fetal Hemoglobin ; Fetomaternal Transfusion ; Flow Cytometry ; Hemoglobin A, Glycosylated ; Humans ; Infant, Newborn ; Pallor ; Pregnancy ; Prothrombin Time

BACKGROUND: This study investigated the clinical characteristics and risk factors of the severity of pandemic influenza A (H1N1) 2009 infection in pediatric patients in Busan and Gyeongsangnam-do. METHODS: Cases of influenza A (H1N1) 2009 in patients under the age of 18 years, confirmed by reverse transcription polymerase chain reaction, at Pusan National University Hospital and Pusan National University Yangsan Hospital from the last week of August 2009 through the last week of February 2010 were retrospectively analyzed. RESULTS: Of the 3,777 confirmed cases of influenza A (H1N1) 2009, 2,200 (58.2%) were male and 1,577 (41.8%) were female. The average age of the patients was 8.4+/-4.8 years. The total cases peaked during 44th to 46th week. Most of the patients were in the 5- to 9-year-old age group. Oseltamivir was administered to 2,959 (78.3%) of the patients. 221 patients (5.9%) were hospitalized, age an average of 6.7+/-4.5 years. The average duration of hospitalization was 7.4+/-5.6 days. One hundred cases (45.2%) had pneumonia. Risk factors for hospitalization included male gender, <2 years of age, and underlying disease. Children with asthma were at very high risk of hospitalization, over 20 times the non-asthmatic children (odds ratio [OR], 21.684; confidence interval [CI], 13.295~39.791). Likewise the children with neurologic deficits faced a 16 times higher risk (OR, 15.738; CI, 7.961~31.111). Ten of the patients (4.5%) were admitted to the intensive care unit, and eight (3.6%) required mechanical ventilation. CONCLUSION: Of the pediatric patients with pandemic influenza A (H1N1) 2009, most of the patients were in the 5- to 9-year-old age group. Risk factors for hospitalization included male gender, <2 years of age, and underlying disease. The most common complication was pneumonia. The very high risk of severe morbidity in children with asthma or neurologic disease shows the critical importance of targeted vaccine coverage, special awareness and swift care by both guardians and primary care providers.
Asthma ; Child ; Epidemiologic Study Characteristics as Topic ; Female ; Hospitalization ; Humans ; Influenza A Virus, H1N1 Subtype ; Influenza, Human ; Intensive Care Units ; Male ; Neurologic Manifestations ; Oseltamivir ; Pandemics ; Pediatrics ; Pneumonia ; Polymerase Chain Reaction ; Primary Health Care ; Retrospective Studies ; Reverse Transcription ; Risk Factors

BACKGROUND: The neurological complications of childhood patients with cancer are caused by brain metastasis, chemotherapy, and infection. Seizure is one of the most important neurological complications of pediatric patients receiving chemotherapy. This study was designed to identify the causes, clinical manifestations, and prognosis of seizures in children receiving cancer treatment.METHODS: We retrospectively reviewed the medical records of pediatric cancer patients with seizures at Pusan National University Hospital from January 2005 to December 2010.RESULTS: Among 271 patients, 31 experienced seizures (19 males and 12 females), and the mean age at the first seizure was 8.6 years (1.7-18.0 years). Seizures frequently occurred in patients with brain tumors (27.8%), and the types of seizures were generalized seizure in 15 patients (48.4%) and partial in 16 cases (51.6%). Eighteen (62.0%) of 29 patients who underwent a brain magnetic resonance imaging study and 21 (75.0%) of 28 cases who received an electroencephalogram recorded abnormal findings. The causes of seizures were drugs, CNS infections, and brain metastasis each in five cases (16.2%). Drugs were the most common cause in patients with a hematological malignancy (16.2%), whereas brain metastasis was the most common cause in patients with solid tumors (12.9%). Eight (40.0%) of 20 patients who survived received anticonvulsants. Eleven patients (35.5%) expired, and the most common cause of death was progression of the malignancy.CONCLUSION: Drugs, CNS infection, and brain metastasis of tumors were the most common causes of seizure in children with malignancy.
Anticonvulsants ; Brain ; Brain Neoplasms ; Cause of Death ; Child ; Electroencephalography ; Hematologic Neoplasms ; Humans ; Magnetic Resonance Imaging ; Male ; Medical Records ; Neoplasm Metastasis ; Prognosis ; Retrospective Studies ; Seizures

PURPOSE: This study aimed to analyze the clinical spectrum of foreign body aspiration in children and explore the clinical features which could facilitate early diagnosis. METHODS: We studied 206 pediatric patients who had aspirated foreign bodies in Pusan National University Hospital between 1980 and 2009. Age, sex, symptoms, signs, the type and location of foreign bodies, radiologic findings and clinical courses were investigated retrospectively. We compared these findings of children diagnosed within 24 hours (early-diagnosis group: EDG) with those of the delayed-diagnosis group (DDG). RESULTS: The majority of patients (76.2%) were yonger than 3 years of age. Cough (67.9%) and decreased breathing sound (43.6%) were the predominant symptoms and signs. Obstructive emphysema (50.0%) was the most frequent radiologic finding. Peanut was the most common foreign body. Acute severe airway obstruction by aspirated foreign bodies tended to occur in younger children because of their small airway. Choking was more frequent in the EDG group than in the DDG group (P=0.018). For the location of foreign bodies, the larynx and trachea were more common in the EDG group (P=0.031). Fever (P=0.024), persistent pneumonia (P=0.011) and bronchiectasis (P=0.041) were more common in the DDG group than in the EDG group. Bronchial asthma, upper respiratory infection and pneumonia were the most common wrong diagnosis. CONCLUSION: Reducing the number of accidents associated with foreign body aspiration is the best way to promote public prevention policies. Physicians must consider the possibility of foreign body aspiration in children with chronic respiratory symptoms.
Airway Obstruction ; Asthma ; Bronchiectasis ; Child ; Cough ; Emphysema ; Fever ; Foreign Bodies ; Humans ; Larynx ; Pneumonia ; Respiratory Sounds ; Retrospective Studies ; Trachea