Treatment outcomes of pediatric cancers have improved greatly with the development of improved treatment protocols, new drugs, and better supportive measures, resulting in overall survival rates greater than 70%. Survival rates are highest in acute lymphoblastic leukemia, reaching more than 90%, owing to risk-based treatment through multicenter clinical trials and protocols developed to prevent central nervous system relapse and testicular relapse in boys. New drugs including clofarabine and nelarabine are currently being evaluated in clinical trials, and other targeted agents are continuously being developed. Chimeric antigen receptor-modified T cells are now attracting interest for the treatment of recurrent or refractory disease. Stem cell transplantation is still the most effective treatment for pediatric acute myeloid leukemia (AML). However, in order to reduce treatment-related death after stem cell transplantation, there is need for improved treatments. New drugs and targeted agents are also needed for improved outcome of AML. Surgery and radiation therapy have been the mainstay for brain tumor treatment. However, chemotherapy is becoming more important for patients who are not eligible for radiotherapy owing to age. Stem cell transplant as a means of high dose chemotherapy and stem cell rescue is a new treatment modality and is often repeated for improved survival. Drugs such as temozolomide are new chemotherapeutic options. In order to achieve 100% cure in children with pediatric cancer, every possible treatment modality and effort should be considered.
Brain Neoplasms* ; Central Nervous System ; Child ; Clinical Protocols ; Drug Therapy ; Humans ; Leukemia* ; Leukemia, Myeloid, Acute ; Pediatrics ; Precursor Cell Lymphoblastic Leukemia-Lymphoma ; Radiotherapy ; Recurrence ; Stem Cell Transplantation ; Stem Cells ; Survival Rate ; T-Lymphocytes

PURPOSE: Malignant rhabdoid tumor (MRT) is a rare and highly aggressive tumor that affects young children. Due to its extreme rarity, most of the available data are based on retrospective case series. To add to the current knowledge of this disease, we reviewed the patients treated for extra-cranial MRT in our institute. MATERIALS AND METHODS: A retrospective medical record review was conducted on children treated for pathologically confirmed extra-cranial MRT at Seoul National University Children's Hospital between January 2003 and May 2013. RESULTS: Eleven patients (7 boys, 4 girls) were diagnosed with extra-cranial MRT at a median age of 9 months old. INI1 staining was important in the pathological confirmation. Six patients (55%) had renal MRT and five (45%) had soft tissue MRT. Five patients (45%) had metastases at diagnosis. All patients underwent chemotherapy, eight patients (73%) underwent surgery, six patients (55%) received therapeutic radiotherapy, and four patients (36%) underwent high dose chemotherapy with autologous stem cell rescue (HDCT/ASCR) with melphalan, etoposide, and carboplatin. Five patients (45%) died of disease following progression (n=3) or relapse (n=2), however, there was no treatment related mortality. The overall survival of the cohort was 53.0% and the event-free survival was 54.5% with a median follow-up duration of 17.8 months (range, 2.3 to 112.3 months). CONCLUSION: Extra-cranial MRT is still a highly aggressive tumor in young children. However, the improved survival of our cohort is promising and HDCT/ASCR with melphalan, etoposide, and carboplatin may be a promising treatment option.
Carboplatin ; Child* ; Cohort Studies ; Diagnosis ; Disease-Free Survival ; Drug Therapy ; Etoposide ; Follow-Up Studies ; Humans ; Kidney Neoplasms ; Medical Records ; Melphalan ; Mortality ; Neoplasm Metastasis ; Radiotherapy ; Recurrence ; Retrospective Studies ; Rhabdoid Tumor* ; Seoul ; Soft Tissue Neoplasms ; Stem Cells

PURPOSE: To identify the clinical and epidemiological characteristics of human metapneumovirus infections (hMPV) in children compared to respiratory syncytial virus A (RSV A) and B (RSV B). METHOD: A retrospective review of medical records was performed in 36 patients with hMPV infection, 106 with RSV A infection, and 51 with RSV B infection, from September 2007 to July 2012. RESULTS: The peak incidence of hMPV infection was observed in May, whereas for RSV infections in November and December. hMPV infection occurred in older patients compared to RSV A and B infection (29.9+/-32.5 months vs. 13.6+/-15.4 months, P<0.001; 29.9+/-32.5 months vs. 12.1+/-13.5 months, P<0.001, respectively). hMPV infection was more often associated with fever compared to RSV A (97.2% vs. 67.9%, P<0.001), while wheezing was less frequent compared to RSV A and B infection (16.7% vs. 47.2%, P=0.001; 16.7% vs. 37.3%, P=0.037, respectively). hMPV infection was more often diagnosed as pneumonia compared to RSV A infection (72.2% vs. 50.0%, P=0.047) while bronchiolitis was less frequent than in RSV A (5.6% vs. 34.9%, P=0.001) or RSV B infection (5.6% vs. 29.4%, P=0.006). In addition, intravenous antibiotic was more often prescribed for patients with hMPV infection than those with RSV A and B (69.4% vs. 39.6%, P=0.002; 69.4% vs. 43.1, P=0.015, respectively). CONCLUSION: This study identified characteristics of hMPV infection compared to RSV A and B infection. Seasonality in spring, higher age group, and higher proportion of pneumonia in hMPV infections may be a useful guide for management of respiratory viral infections in children.
Bronchiolitis ; Child ; Fever ; Humans* ; Incidence ; Medical Records ; Metapneumovirus* ; Pneumonia ; Respiratory Sounds ; Respiratory Syncytial Viruses* ; Retrospective Studies ; Seasons

Transplantation-associated thrombotic microangiopathy (TA-TMA) is an uncommon but devastating complication in patients who undergo hematopoietic stem cell transplantation (SCT). However, the optimal treatment strategy for TA-TMA is unclear. We report a rare case of TA-TMA in a 39-month-old boy who underwent tandem autologous SCT (autoSCT) for high-risk medulloblastoma. TA-TMA developed 64 days after the second autoSCT with microangiopathic hemolytic anemia, fever, renal impairment, acute respiratory distress syndrome and posterior reversible encephalopathy syndrome. The patient recovered after plasmapheresis and methylprednisolone therapy. He had mild to moderate deficiency of a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13 (ADAMTS-13). The patient's clinical course would suggest that plasmapheresis and methylprednisolone therapy could be a treatment option for TA-TMA. Early intervention is needed to aid the recovery of the patient who is suspected for TA-TMA.
Anemia, Hemolytic ; Child, Preschool ; Early Intervention (Education) ; Fever ; Hematopoietic Stem Cell Transplantation ; Humans ; Male ; Medulloblastoma ; Methylprednisolone ; Pediatrics ; Plasmapheresis ; Posterior Leukoencephalopathy Syndrome ; Respiratory Distress Syndrome, Adult ; Stem Cell Transplantation ; Thrombospondins ; Thrombotic Microangiopathies

BACKGROUND: Nasopharyngeal carcinoma (NPC) is very rare in children and adolescents. The aim of this study was to evaluate clinical characteristics and treatment outcomes of pediatric NPC.METHODS: Medical records of 9 patients treated for NPC at the Seoul National University Children's Hospital between 1988 and 2012 were analyzed retrospectively.RESULTS: The median age at diagnosis was 11 years (range, 9-13 years). One patient had stage II disease, 3 had stage III disease, and 5 had stage IV disease. The histologic subtypes were undifferentiated carcinoma and squamous cell carcinoma in 7 and 2 patients, respectively. All patients were initially treated with cisplatin (100 mg/m2 intravenous [IV] every 4 weeks for 4-6 months), bleomycin (15 unit/m2 IV every 1 weekx7), and fluorouracil (1,000 mg/m2 IV every 4 weeks for 1 year). Eight patients received radiotherapy with doses of 45-59.4 Gy at the primary site and neck nodes. Seven patients (77.8%) achieved complete remission, 1 (11.1%) achieved partial remission, and 1 (11.1%) showed disease progression. Six patients developed fluorouracil-related neurotoxicity; the regimen was changed to cisplatin, epirubicin, and bleomycin in five of the 6 patients. One patient died of progressive disease without responding to treatment. Treatment-related mortality occurred in 1 patient owing to septic shock. Secondary osteosarcoma developed in 1 patient 6 years after treatment. The overall survival was 77.8%, with a median follow-up of 40.8 months (range, 4.5-287.6 months).CONCLUSION: Children and adolescents with advanced NPC treated with combined chemotherapy and radiotherapy have a good survival rate.
Adolescent ; Bleomycin ; Carcinoma ; Carcinoma, Squamous Cell ; Chemoradiotherapy ; Child ; Cisplatin ; Diagnosis ; Disease Progression ; Drug Therapy ; Epirubicin ; Fluorouracil ; Follow-Up Studies ; Humans ; Korea ; Medical Records ; Mortality ; Nasopharyngeal Neoplasms ; Neck ; Osteosarcoma ; Pediatrics ; Radiotherapy ; Retrospective Studies ; Seoul ; Shock, Septic ; Survival Rate

A 1.1 year old boy was admitted to the Seoul National University Children's Hospital because of incidental findings of hepatosplenomegaly, skin lesion and multiple intra- abdominal lymphadenopathies. Anemia and thrombocytopenia were found based on the initial complete blood count (CBC) measurements. Because of bicytopenia and hepatosplenomegaly, bone marrow examination was performed which revealed hypercellular marrow with increased monocytes and granulopoiesis. The hemoglobin F level was high for his age, and monocyte production was increased. The patient was diagnosed with juvenile myelomonocytic leukemia at the age of 1.2 years. Chemotherapy with cytarabine, etoposide, vincristine, and isotretinoin was initiated. After 6 cycles of chemotherapy, the CBC normalized. He underwent double cord blood transplantation (dCBT), but chimerism studies showed autologous recovery. However, he did not show relapse during the 5 years post-transplant during which he received isotretinoin. He is surviving disease-free 9 years after dCBT.
Anemia ; Blood Cell Count ; Bone Marrow ; Bone Marrow Examination ; Chimerism ; Cytarabine ; Drug Therapy ; Etoposide ; Fetal Blood ; Fetal Hemoglobin ; Humans ; Incidental Findings ; Isotretinoin ; Leukemia, Myelomonocytic, Juvenile ; Male ; Monocytes ; Recurrence ; Seoul ; Skin ; Thrombocytopenia ; Vincristine

BACKGROUND: Desmoplastic small round cell tumor (DSRCT) is an aggressive malignancy with a poor prognosis. DSRCT is a rare disease, and therefore a standard treatment regimen has not been established. In this study, we reviewed the clinical characteristics and treatment outcomes of pediatric DSRCT patients.METHODS: We retrospectively reviewed the medical records of 5 DSRCT patients (2 boys, 3 girls) that were diagnosed and treated with DSRCT at Seoul National University Children's Hospital from January 1999 to January 2015.RESULTS: The median age at diagnosis was 11 years 5months (range 4 years 10 months-17 years 2 months). The most frequent symptoms were abdominal pain (60%). The primary sites were gastrointestinal tract, bladder, and omentum, and the involved sites were the liver, gastrointestinal tract, bladder and bone. Three patients had multiple metastases at diagnosis. Two patients underwent upfront surgical excision of primary tumor, and the remaining 3 patients received neo-adjuvant chemotherapy after the diagnosis was confirmed by using needle biopsy. Combination chemotherapy was administered to all patients in addition to radiotherapy (median dose 45 Gy, range 17.5-54 Gy). Four patients showed disease progression or relapse, resulting in a 20% overall survival rate. At the time of analysis, one patient is alive. She had localized disease at the time of diagnosis and were treated with upfront surgery, chemotherapy, and high-dose chemotherapy with autologous stem cell transplantation and radiotherapy.CONCLUSION: Patients with DSRCT have a poor prognosis, even after multimodal treatment. Further studies are needed to determine the prognostic factors of DSRCT.
Abdominal Pain ; Biopsy, Needle ; Combined Modality Therapy ; Desmoplastic Small Round Cell Tumor ; Diagnosis ; Disease Progression ; Drug Therapy ; Drug Therapy, Combination ; Gastrointestinal Tract ; Humans ; Korea ; Liver ; Medical Records ; Neoplasm Metastasis ; Omentum ; Pediatrics ; Prognosis ; Radiotherapy ; Rare Diseases ; Recurrence ; Retrospective Studies ; Seoul ; Stem Cell Transplantation ; Survival Rate ; Treatment Outcome ; Urinary Bladder

BACKGROUND: Soft tissue clear cell sarcoma is a rare tumor which originates from neural crest cells. Due to its rarity and lack of established treatment, the prognosis of clear cell sarcoma is poor. Here, we reviewed the clinical data and outcome of patients diagnosed with soft tissue clear cell sarcoma in our institution.METHODS: A retrospective study was conducted on pediatric patients who were treated for pathologically confirmed soft tissue clear cell sarcoma at the Seoul National University Hospital, between January 2000 and July 2017.RESULTS: Six patients (3 boys and 3 girls) were diagnosed with soft tissue clear cell sarcoma at a median age of 14 years 4 months (range 11 years 7 months - 19 years 3 months). The median size of the tumor was 5.6 cm (range, 0.6 cm to 7.9 cm). The most frequent symptom was pain (67%), and the most common primary site was the lower limb (67%). Three patients (50%) presented with metastases at diagnosis. Four patients underwent chemotherapy with various therapeutic combinations. Four patients received surgical resection. Only one patient received local radiotherapy. One patient died of primary refractory disease, three patients relapsed, while the remaining two survive event-free.CONCLUSION: Soft tissue clear cell sarcoma is a rare and highly aggressive tumor, for which there is no established treatment. All surviving patients received surgery, indicating that surgery is a key treatment modality. Further genetic studies of soft tissue clear cell sarcoma are needed to find a better treatment strategy.
Diagnosis ; Drug Therapy ; Humans ; Korea ; Lower Extremity ; Neoplasm Metastasis ; Neural Crest ; Pediatrics ; Prognosis ; Radiotherapy ; Retrospective Studies ; Sarcoma, Clear Cell ; Seoul ; Treatment Outcome

No abstract available.
Child* ; Humans

Chronic granulomatous disease (CGD) is a primary immunodeficiency disease caused by impaired phagocytic function. Hematopoietic stem cell transplantation (HSCT) is a definitive cure for CGD; however, the use of HSCT is limited because of associated problems, including transplantation-related mortality and engraftment failure. We report a case of a patient with CGD who underwent successful HSCT following a targeted busulfan and fludarabine reduced-toxicity myeloablative conditioning. Intravenous busulfan was administered once daily for 4 consecutive days (days –8 to –5), and the target area under the curve was 75,000 µg·hr/L. Fludarabine (40 mg/m2) was administered once daily for 6 consecutive days from days –8 to –3. Antithymocyte globulin (2.5 mg/kg/day) was administered from days –4 to –2. The patient underwent successful engraftment and did not have any severe toxicity related to the transplantation. Conditioning with a targeted busulfan and fludarabine regimen could provide a better outcome for HSCT in CGD, with close regulation of the busulfan dose.
Antilymphocyte Serum ; Bone Marrow Transplantation* ; Bone Marrow* ; Busulfan* ; Granulomatous Disease, Chronic* ; Hematopoietic Stem Cell Transplantation ; Humans ; Mortality ; Transplantation Conditioning