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.

Clinical Characteristics and Treatment Outcomes of Childhood Acute Promyelocytic Leukemia in Korea: A Nationwide Multicenter Retrospective Study by Korean Pediatric Oncology Study Group / Journal of the Korean Cancer Association, 대한암학회지

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.

Effectiveness and Safety of Clofarabine Monotherapy or Combination Treatment in Relapsed/Refractory Childhood Acute Lymphoblastic Leukemia: A Pragmatic, Non-interventional Study in Korea / Journal of the Korean Cancer Association, 대한암학회지

Purpose@#Effectiveness and safety of clofarabine (one of the treatment mainstays in pediatric patients with relapsed/refractory acute lymphoblastic leukemia [ALL]) was assessed in Korean pediatric patients with ALL to facilitate conditional coverage with evidence development. @*Materials and Methods@#In this multicenter, prospective, observational study, patients receiving clofarabine as mono/combination therapy were followed up every 4-6 weeks for 6 months or until hematopoietic stem cell transplantation (HSCT). Response rates, survival outcomes, and adverse events were assessed. @*Results@#Sixty patients (2-26 years old; 65% B-cell ALL, received prior ≥ 2 regimen, 68.3% refractory to previous regimen) were enrolled and treated with at least one dose of clofarabine; of whom 26 (43.3%) completed 6 months of follow-up after the last dose of clofarabine. Fifty-eight patients (96.7%) received clofarabine combination therapy. Overall remission rate (complete remission [CR] or CR without platelet recovery [CRp]) was 45.0% (27/60; 95% confidence interval [CI], 32.4 to 57.6) and the overall response rate (CR, CRp, or partial remission [PR]) was 46.7% (28/60; 95% CI, 34.0 to 59.3), with 11 (18.3%), 16 (26.7%), and one (1.7%) patients achieving CR, CRp, and PR, respectively. The median time to remission was 5.1 weeks (95% CI, 4.7 to 6.1). Median duration of remission was 16.6 weeks (range, 2.0 to 167.6 weeks). Sixteen patients (26.7%) proceeded to HSCT. There were 24 deaths; 14 due to treatment-emergent adverse events. @*Conclusion@#Remission with clofarabine was observed in approximately half of the study patients who had overall expected safety profile; however, there was no favorable long-term survival outcome in this study.

Clinical Characteristics of Human Parvovirus B19 Infection in Children

Purpose@#Human parvovirus B19 infection is widespread and has a heterogeneous clinical spectrum, ranging from asymptomatic infection to potentially life-threatening complications. We investigated the various clinical features of human parvovirus B19 infection during an outbreak of the virus in our community. @*Methods@#A retrospective chart review study was conducted at the Pusan National University Children's Hospital from December 2017 to April 2019. We investigated the clinical features of children with parvovirus B19 immunoglobulin M or parvovirus B19 DNA detected using polymerase chain reaction. @*Results@#A total of 24 children were diagnosed with parvovirus B19 infection. Twelve (50%) had lace form rashes, and four (16.7%) had petechial rashes. Two (8.3%) were diagnosed with fever without a focus. Six (25%) developed aplastic crisis as a complication of infection, of whom three were previously diagnosed with hereditary spherocytosis and three with acute lymphoblastic leukemia. @*Conclusions@#In addition to erythema infectiosum, the parvovirus B19 infection can present clinically with various types of rashes and fever without a focus. Furthermore, hematologic manifestations such as neutropenia and aplastic crisis can occur during infection.

Clinical Characteristics and Treatment Outcomes in Children, Adolescents, and Young-adults with Hodgkin's Lymphoma:a KPHOG Lymphoma Working-party, Multicenter, Retrospective Study

Background@#Hodgkin's lymphoma (HL) constitutes 10%–20% of all malignant lymphomas and has a high cure rate (5-year survival, around 90%). Recently, interest has increased concerning preventing secondary complications (secondary cancer, endocrine disorders) in long-term survivors. We aimed to study the epidemiologic features and therapeutic outcomes of HL in children, adolescents, and young adults in Korea. @*Methods@#We performed a multicenter, retrospective study of 224 patients aged < 25 years diagnosed with HL at 22 participating institutes in Korea from January 2007 to August 2016. @*Results@#A higher percentage of males was diagnosed at a younger age. Nodular sclerosis histopathological HL subtype was most common, followed by mixed cellularity subtype.Eighty-one (36.2%), 101 (45.1%), and 42 (18.8%) patients were classified into low, intermediate, and high-risk groups, respectively. Doxorubicin, bleomycin, vinblastine, dacarbazine was the most common protocol (n = 102, 45.5%). Event-free survival rate was 86.0% ± 2.4%, while five-year overall survival (OS) rate was 96.1% ± 1.4%: 98.7% ± 1.3%, 97.7% ± 1.6%, and 86.5% ± 5.6% in the low, intermediate, and high-risk groups, respectively (P = 0.021). Five-year OS was worse in patients with B-symptoms, stage IV disease, highrisk, splenic involvement, extra-nodal lymphoma, and elevated lactate dehydrogenase level.In multivariate analysis, B-symptoms and extra-nodal involvement were prognostic factors for poor OS. Late complications of endocrine disorders and secondary malignancy were observed in 17 and 6 patients, respectively. @*Conclusion@#This is the first study on the epidemiology and treatment outcomes of HL in children, adolescents, and young adults in Korea. Future prospective studies are indicated to develop therapies that minimize treatment toxicity while maximizing cure rates in children, adolescents, and young adults with HL.

Epidemiological Study of Hereditary Hemolytic Anemia in the Korean Pediatric Population during 1997–2016: a Nationwide Retrospective Cohort Study

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.

Clinical Features and Treatment Outcomes of Immune Thrombocytopenic Purpura in Infants: A Single Center Retrospective Study / 임상소아혈액종양

BACKGROUND: Immune thrombocytopenic purpura (ITP) in children less than one year of age is less well characterized compared to ITP in toddlers and school-age children. Since children of different ages may have differing clinical courses, better delineation of the natural history of ITP in infants is needed. METHODS: We retrospectively reviewed the admission records of 248 consecutive pediatric patients between 1 month and 15 years of age who were admitted and treated for acute ITP at Pusan National University Children's Hospital from 2009 through 2017. All patients less than 1 year of age were identified and enrolled in this study. We investigated their demographics, clinical features, laboratory examinations, response to treatment, and long-term outcomes and made a comparison to those of children aged 1 to 10 years of age. RESULTS: Ninety nine infants were identified. Male to female ratio was highest in infants and decreased with age. Seventy nine (79.8%) of the 99 infant were found to be under 6 months old. The median platelet counts at diagnosis was 6×10⁹/L. Minor bleeding (bleeding score 0–2) was significantly dominant in infant compared to older subjects. Eighty two (96.5%) out of 85 patients achieved complete remission after initial intravenous immunoglobulin (IVIG) treatment. The relapse rate after initial CR was significantly lower than older ages (P=0.003). The platelet count after IVIG treatment in infant showed more rapid response compared to older subjects (P=0.04). Follow up information at 12 months was available for 70 infants. Chronic ITP at 12 month was seen less frequently in infants than in children 1 to 10 years of age (1.4% vs. 20.2%, P<0.001). CONCLUSION: Infants with acute ITP respond more favorably to IVIG treatment and are less likely to develop chronic ITP compared to children 1 to 10 years of age.

A Multicenter Study on von Willebrand Disease Realities in Yeungnam Region / 임상소아혈액종양

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.

Prevalence of Unexpected Antibodies in Pregnant Korean Women and Neonatal Outcomes / 대한수혈학회지

BACKGROUND: In pregnant women, the frequency of irregular antibodies that cause hemolytic disease of the fetus and newborn (HDFN) vary between study populations. The clinical manifestations of HDFN differ according to the specificities and degree of irregular antibodies. This study examined the frequency and nature of maternal alloimmunization and neonatal outcomes. METHODS: Pregnant women, who underwent irregular antibody screening for prenatal testing at an obstetrics clinic in a single center, were enrolled. Those who screened positive for irregular antibodies were selected as the test group, and age- and obstetrics history-matched pregnant women were selected as the control group to evaluate the pregnancy outcomes according to irregular antibodies. RESULTS: The prevalence of irregular antibodies was 2.78% (42/1,508). With the exception of an unidentified antibody, anti-D was the most frequently identified antibody, followed in order by anti-E and anti-Le(a). The rate of fetal death was higher in the test group (6/37, 16.2%) than in the control group (1/37, 2.7%) (P=0.047). Eight pregnant women had anti-C or anti-D, one woman had a stillbirth, and four living neonates developed hyperbilirubinemia. Of six pregnant women with anti-E alone or with other alloantibodies, three experienced a spontaneous abortion or stillbirth. Among the six newborns with maternal anti-Le(a) and anti-Jk(a), four developed hyperbilirubinemia, but their mothers did not experience a spontaneous abortion or stillbirth. CONCLUSION: The prevalence of unexpected antibodies among pregnant Korean women was 2.78%. A significant difference in neonatal outcomes was observed, including the death rate, prematurity, and hyperbilirubinemia, depending on the specificity of the unexpected antibody.

Two Pediatric Cases of Spontaneous Ruptured Solid Tumors Successfully Treated with Transcutaneous Arterial Embolization / 임상소아혈액종양

Spontaneous rupture with internal bleeding of solid tumors has rarely been described at the time of diagnosis or during chemotherapy. This rare event must be regarded as a life threatening condition. In these emergency situations, control of hemorrhage, which is life-saving, can be achieved by transcatheter arterial embolization (TAE) and/or surgical resection. This report describes two infants presenting with acute hemorrhagic shock due to spontaneous tumor rupture of hepatoblastoma and neuroblastoma during chemotherapy. TAE successfully arrested the tumor bleeding and a visibly reduced the tumor size in both children. Spontaneous rupture of solid tumors occur infrequently in children, but is a life threatening situation. Careful monitoring for the occurrence of this rare event especially in very young children presenting with a large tumor mass.

Childhood Venous Thromboembolism in Yeungnam Region in Korea: Multicenter Study / 임상소아혈액종양

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.

Spontaneous Tumor Lysis Syndrome Presenting Acute Kidney Injury with Extreme Hyperuricemia and Urinary Stone: A Rare Case of Spontaneous Tumor Lysis Syndrome

Tumor lysis syndrome is a serious complication of malignancy, resulting from the massive and rapid release of cellular components into the blood. Generally, it occurs after initiation of chemotherapy. The onset of spontaneous tumor lysis syndrome (STLS) before anti-cancer treatment is rare and occurs mostly in Burkitt lymphoma and non-Hodgkin's lymphoma. There are only a few case reports in children. Here, we report a case of STLS secondary to T-cell acute lymphoblastic leukemia (ALL), which presented with urinary stone and subsequent acute kidney injury with severe hyperuricemia. Occult malignancy should be considered in case of unexplained acute kidney injury with extreme hyperuricemia.

Experience of Failure Mode and Effect Analysis in Transfusion Medicine / 대한수혈학회지

BACKGROUND: Blood transfusions are complicated procedures, and are highly sensitive to mistakes that could seriously endanger the life of patients. The failure mode and effect analysis (FMEA) can be used to inspect and improve high risk processes. Here, we aimed to identify the risk factors of a blood transfusion process and to improve its safety by optimizing the process. METHODS: We conducted a weekly meeting from March to April 2014. We investigated the frequency of events for 2013 (before FMEA) and 2015 (after FMEA). The FMEA process was performed in eight steps and the improvement priorities were determined in accordance with the magnitude of calculated fatalities (multiplied by severity, occurrence, and detection scores). RESULTS: The whole process of blood transfusion was analyzed by detailed steps: Decision of blood transfusion, blood transfusion request, pre-transfusion test, blood product discharge, delivery, and administration process. Then, we identified the types of failures and likelihood of occurrence, discovery, and severity. Based on the calculated risk priority number, strategies to improve the highest failure modes were developed. Eleven transfusion-related events occurred before FMEA, and three events occurred after FMEA. CONCLUSION: In this study, we analyzed the failure modes that may occur during a transfusion procedure. The FMEA was a useful tool for analyzing and reducing the risks associated with a blood transfusion procedure. Continuous efforts to improve the failure modes would be helpful to further improve the safety of patients undergoing blood transfusion.

Effects of low-dose topiramate on language function in children with migraine / 소아과

PURPOSE: This study aimed to verify the safety of low-dose topiramate on language development in pediatric patients with migraine. METHODS: Thirty newly diagnosed pediatric patients with migraine who needed topiramate were enrolled and assessed twice with standard language tests, including the Test of Language Problem Solving Abilities (TOPs), Receptive and Expressive Vocabulary Test, Urimal Test of Articulation and Phonology, and computerized speech laboratory analysis. Data were collected before treatment, and topiramate as monotherapy was sustained for at least 3 months. The mean follow-up period was 4.3±2.7 months. The mean topiramate dosage was 0.9 mg/kg/day. RESULTS: The patient's mean age was 144.1±42.3 months (male-to-female ratio, 9:21). The values of all the language parameters of the TOPs were not changed significantly after the topiramate treatment as follows: Determine cause, from 15.0±4.4 to 15.4±4.8 (P>0.05); making inference, from 17.6±5.6 to 17.5±6.6 (P>0.05); predicting, from 11.5±4.5 to 12.3±4.0 (P>0.05); and total TOPs score, from 44.1± 13.4 to 45.3±13.6 (P>0.05). The total mean length of utterance in words during the test decreased from 44.1±13.4 to 45.3±13.6 (P0.05). In the articulation and phonology validation in both groups, speech pitch and energy were not significant, and all the vowel test results showed no other significant values. CONCLUSION: No significant difference was found in the language-speaking ability between the patients; however, the number of vocabularies used decreased. Therefore, topiramate should be used cautiously for children with migraine.

Effects of lamotrigine on attention-deficit hyperactivity disorder in pediatric epilepsy patients / 소아과

PURPOSE: The purpose of this study was to investigate the effects of lamotrigine for the treatment of attention-deficit hyperactivity disorder (ADHD) symptoms in children with epilepsy. METHODS: Pediatric patients newly diagnosed with epilepsy (n=90 [61 boys and 29 girls]; mean age, 9.1±3.4 years) were enrolled. All patients were evaluated with the Korean ADHD rating scale (K-ARS)-IV before treatment with lamotrigine and after doses had been administered. The mean interval of ADHD testing was approximately 12.3 months. The initial dosage of lamotrigine was 1 mg/kg/day (maximum 25 mg/day for the first 2 weeks), and increased by 1 mg/kg every 2 weeks until titrated up to 7 mg/kg/day (or maximum 200 mg/day). RESULTS: The mean ADHD test score of the 90 subjects was 17.0±1.8 at baseline. It was slightly reduced to 15.6±1.7 after lamotrigine monotherapy (P >0.01). Prior to treatment, a total of 31 patients (34.4%) met the diagnostic criteria for ADHD according to Diagnostic and Statistical Manual of Mental Disorders, 4th Edition, Text Revision, Of these 31 patients, 27 (87.1%) had significantly improved ADHD scores with lamotrigine monotherapy (28.0±1.6 reduced to 18.1±2.6, P<0.001). Among these 27 patients, 25 (92.6%) showed normalized electroencephalogram (EEG) and 26 (96.3%) achieved total freedom from seizures within 12 months of the initiation of lamotrigine monotherapy. CONCLUSION: The results from our study show that lamotrigine had a positive effect in pediatric epilepsy patients by reducing ADHD symptoms, preventing seizures, and normalizing EEG. However, further research is required to determine whether lamotrigine is efficacious against ADHD symptoms independent of its effects on epileptic seizures.

Tacrolimus and Mini-dose Methotrexate for Prevention of Graft-Versus-Host Disease after Unrelated Hematopoietic Stem Cell Transplantation in Children: A Single-center Study in Korea / 임상소아혈액종양

BACKGROUND: Currently, data on the role of tacrolimus and mini-dose methotrexate (MTX) in pediatric unrelated hematopoietic stem cell transplantation (HSCT) is limited. We report the outcomes of unrelated hematopoietic stem cell recipients, evaluating engraftment status, incidence of acute and chronic graft-versus-host disease (GVHD) and toxicities after use of tacrolimus and mini-dose MTX for GVHD prophylaxis.METHODS: Thirty-five children who received tacrolimus and mini-dose MTX as prophylaxis from January 2004 to December 2013 were reviewed. All patients received tacrolimus beginning the day prior to transplant at a dose of 0.03 mg/kg/day by continuous intravenous infusion. MTX was administered at a dose of 5 mg/m2 IV on days 1, 3, 6 and 11.RESULTS: Median age at transplantation was 8.42 years (range 0.75-18.9 years). Seventeen patients received human leukocyte antigen (HLA) fully matched donor transplants and 18 received partially mismatched transplants. All but two patients who received unrelated cord blood transplants showed successful engraftment. The median time to ANC recovery was 12 days. The incidence of acute GVHD was 33.3% including 15.1% grade III-IV GVHD. Localized chronic GVHD developed in only 2 of 27 (7.4%) evaluable patients. Lower tacrolimus levels during days 1-21 were associated with a higher incidence of acute GVHD (P=0.033). The estimated 4-year event free survival and overall survival of the patients were 71.2% and 80.0%.CONCLUSION: Overall, the combination of tacrolimus and mini-dose MTX could be effectively administered in the setting of pediatric unrelated HSCT.

A Case of Blastic Plasmacytoid Dendritic Cell Neoplasm in a Child / 임상소아혈액종양

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a very rare hematological malignancy in children that is characterized by bruise-like skin lesions, with or without bone marrow involvement. Because the clinical course of BPDCN is highly aggressive and fatal in adults, allogeneic hematopoietic stem cell transplantation (HSCT) is recommended as the optimal treatment. Due to its rarity, the pediatric experience with BPDCN is limited and standard treatment has so far not been defined. We report a case of a 14-year-old boy with BPDCN involving the skin, bone marrow and lymph nodes. The patient was treated with high-risk group acute lymphoblastic leukemia protocol. He achieved a complete remission after induction chemotherapy and still maintains clinical remission without HSCT for 17 months after initial diagnosis.

Tacrolimus and Mini-dose Methotrexate for Prevention of Graft-Versus-Host Disease after Unrelated Hematopoietic Stem Cell Transplantation in Children: A Single-center Study in Korea / 임상소아혈액종양

BACKGROUND: Currently, data on the role of tacrolimus and mini-dose methotrexate (MTX) in pediatric unrelated hematopoietic stem cell transplantation (HSCT) is limited. We report the outcomes of unrelated hematopoietic stem cell recipients, evaluating engraftment status, incidence of acute and chronic graft-versus-host disease (GVHD) and toxicities after use of tacrolimus and mini-dose MTX for GVHD prophylaxis. METHODS: Thirty-five children who received tacrolimus and mini-dose MTX as prophylaxis from January 2004 to December 2013 were reviewed. All patients received tacrolimus beginning the day prior to transplant at a dose of 0.03 mg/kg/day by continuous intravenous infusion. MTX was administered at a dose of 5 mg/m2 IV on days 1, 3, 6 and 11. RESULTS: Median age at transplantation was 8.42 years (range 0.75-18.9 years). Seventeen patients received human leukocyte antigen (HLA) fully matched donor transplants and 18 received partially mismatched transplants. All but two patients who received unrelated cord blood transplants showed successful engraftment. The median time to ANC recovery was 12 days. The incidence of acute GVHD was 33.3% including 15.1% grade III-IV GVHD. Localized chronic GVHD developed in only 2 of 27 (7.4%) evaluable patients. Lower tacrolimus levels during days 1-21 were associated with a higher incidence of acute GVHD (P=0.033). The estimated 4-year event free survival and overall survival of the patients were 71.2% and 80.0%. CONCLUSION: Overall, the combination of tacrolimus and mini-dose MTX could be effectively administered in the setting of pediatric unrelated HSCT.

A Case of Blastic Plasmacytoid Dendritic Cell Neoplasm in a Child / 임상소아혈액종양

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a very rare hematological malignancy in children that is characterized by bruise-like skin lesions, with or without bone marrow involvement. Because the clinical course of BPDCN is highly aggressive and fatal in adults, allogeneic hematopoietic stem cell transplantation (HSCT) is recommended as the optimal treatment. Due to its rarity, the pediatric experience with BPDCN is limited and standard treatment has so far not been defined. We report a case of a 14-year-old boy with BPDCN involving the skin, bone marrow and lymph nodes. The patient was treated with high-risk group acute lymphoblastic leukemia protocol. He achieved a complete remission after induction chemotherapy and still maintains clinical remission without HSCT for 17 months after initial diagnosis.

Ultrasound-guided internal jugular vein catheterization in critically ill pediatric patients / 소아과

PURPOSE: Continuous intravenous access is imperative in emergency situations. Ultrasound-guided internal jugular vein (IJV) catheterization was investigated in critically ill pediatric patients to assess the feasibility of the procedure. METHODS: Patients admitted to the pediatric intensive care unit between February 2011 and September 2012 were enrolled in this study. All patients received a central venous catheter from attending house staff under ultrasound guidance. Outcome measures included successful insertion of the catheter, cannulation time, number of cannulation attempts, and number and type of resulting complications. RESULTS: Forty-one central venous catheters (93.2%) were successfully inserted into 44 patients (21 males and 23 females; mean age, 6.54+/-1.06 years). Thirty-three patients (75.0%) had neurological disorders. The right IJV was used for catheter insertion in 34 cases (82.9%). The mean number of cannulation attempts and the mean cannulation time was 1.57+/-0.34 and 14.07+/-1.91 minutes, respectively, the mean catheter dwell time was 14.73+/-2.5 days. Accidental catheter removal was observed in 9 patients (22.0%). Six patients (13.6%) reported complications, the most serious being catheter-related sepsis, which affected 1 patient (2.3%). Other complications included 2 reported cases of catheter malposition (4.6%), and 1 case each of arterial puncture (2.3%), pneumothorax (2.3%), and skin infection (2.3%). CONCLUSION: The results suggest that ultrasound-guided IJV catheterization can be performed easily and without any serious complications in pediatric patients, even when performed by visiting house staff. Therefore, ultrasound-guided IJV catheterization is strongly recommended for critically ill pediatric patients.