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1.
Neonatal Medicine ; : 37-43, 2020.
Article | WPRIM | ID: wpr-837006

ABSTRACT

Kaposiform hemangioendothelioma (KHE) is a rare, locally aggressive vascular neoplasm that often develops a coagulopathy known as Kasabach­Merritt phenomenon (KMP). Visceral involvement denotes a poor prognosis. We report a case of visceral KHE with KMP successfully treated with corticosteroids and vincristine. The infant had been born vaginally at 34 weeks 6 days’ gestation, weighing 2,360 g. He was admi tted for the management of respiratory failure. Blood tests showed anemia and thrombocytopenia 1 hour after delivery. Additional blood tests revealed a prothrombin time of 12.1 seconds, activated partial thromboplastin time of 60.6 seconds, fibrinogen levels of 72.4 mg/dL, and D­dimer levels >3,200 ng/mL. Despite supportive measures and daily transfusions, the clinical condition and coagulopathy gradually worsened. Renal ultrasonography performed to find the origin of the coagulopathy revealed an echogenic mass measuring >3 cm in the abdominal cavity. A magnetic resonance imaging scan showed an ill­marginated, infiltrative mass like lesion in the right anteromedial and posteromedial perirenal space that was hypointense with mild enhance ment on T1­ and T2­weighted images. Large vascular tumors adherent to other visceral organs were noted during exploratory laparotomy but could not be resected. Treatment with methylprednisolone was ineffective. Vincristine was administered weekly from the 17th hospital day, and the coagulation profile showed gradual impro vement after its initiation. Intravenous methylprednisolone was switched to oral prednisolone on the 57th hospital day. He was discharged on the 73rd hospital day and continued vincristine treatment every 2 weeks and oral prednisolone administration as an outpatient treatment for 8 weeks. He remained symptom­free at the 39­month follow­up.

2.
Yonsei Medical Journal ; : 542-546, 2020.
Article | WPRIM | ID: wpr-833355

ABSTRACT

Activated phosphoinositide 3-kinase δ syndrome (APDS)1 is caused by gain-of-function mutations in PIK3CD, which encodes the catalytic p110δ subunit of phosphoinositide 3 kinase. We describe three patients with APDS1, the first thereof in Korea. Therein, we investigated clinical manifestations of APDS1 and collected data on the efficacy and safety profile of sirolimus, a mammalian target of rapamycin inhibitor and pathway-specific targeted medicine. The same heterozygous PIK3CD mutation was detected in all three patients (E1021K). After genetic diagnosis, all patients received sirolimus and experienced an excellent response, including amelioration of lymphoproliferation and improvement of nodular mucosal lymphoid hyperplasia in the gastrointestinal tract. The median trough level of sirolimus was 5.5 ng/mL (range, 2.8–7.5) at a dose of 2.6–3.6 mg/m2. Two patients who needed highdose, short-interval, immunoglobulin-replacement treatment (IGRT) had a reduced requirement for IGRT after initiating sirolimus, and the dosing interval was extended from 2 and 3 weeks to 4 weeks. The IgG trough level after sirolimus treatment (median, 594 mg/dL; range, 332–799 mg/dL) was significantly higher than that before sirolimus treatment (median, 290 mg/dL; range, 163–346 mg/dL) (p<0.001). One episode of elevated serum creatinine with a surge of sirolimus (Patient 2) and episodes of neutropenia and oral stomatitis (Patient 1) were observed. We diagnosed the first three patients with APDS1 in Korea. Low-dose sirolimus may alleviate clinical manifestations thereof, including hypogammaglobulinemia.

3.
Article in English | WPRIM | ID: wpr-831569

ABSTRACT

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.

4.
Article in English | WPRIM | ID: wpr-176880

ABSTRACT

Little is known about platelet dynamics and the effect of antiplatelet therapy in Kawasaki disease (KD). This study sought to define platelet activation dynamics in KD patients by assaying platelet-derived microparticles (PDMPs). We measured plasma PDMPs levels in 46 patients with KD using an enzyme-linked immunosorbent assay (ELISA). Blood samples were collected before, at 2–5 days, and 9–15 days after intravenous immunoglobulin (IVIG) infusion, 2 months and 4–5 months after the onset of KD. We measured PDMP levels in 23 febrile and 10 afebrile control patients. In the acute phase of KD patients, PDMP levels increased significantly after IVIG treatment (12.04 ± 5.58 nmol before IVIG infusion vs. 19.81 ± 13.21 nmol at 2–5 days after IVIG infusion, P = 0.006). PDMP levels were negatively correlated with age and positively correlated with procalcitonin levels in the acute phase of KD. No significant difference was found in PDMP levels between KD patients with and without coronary artery lesion (CAL). Elevated PDMP levels after IVIG therapy significantly decreased below the pre-IVIG level in subacute phase (19.81 ± 13.21 nmol at 2–5 days after IVIG infusion vs. 8.33 ± 2.02 nmol at 9–15 days after IVIG infusion, P < 0.001), and PDMP levels stayed below the pre-IVIG level in the convalescent phase, during which antiplatelet therapy was given. However, PDMP levels rebounded after discontinuing aspirin in 17 patients. In conclusion, enhanced platelet activation was noted before treatment of KD and peaked immediately after IVIG treatment. Recurrent rising of PDMP levels was observed after discontinuing aspirin, although there were no significant differences between the PDMP levels at 2 months after the onset of KD and those at 4–5 months after the onset of the disease.


Subject(s)
Aspirin , Blood Platelets , Coronary Vessels , Enzyme-Linked Immunosorbent Assay , Humans , Immunoglobulins , Immunoglobulins, Intravenous , Mucocutaneous Lymph Node Syndrome , Plasma , Platelet Activation
5.
Article in Korean | WPRIM | ID: wpr-788615

ABSTRACT

BACKGROUND: Immune thrombocytopenic purpura (ITP) is an acquired bleeding disorder in which the immune system destroys platelets. There were many studies which predicted the factors associated with the prognosis of childhood ITP, but controversies remained. We analyzed the predicting factors associated with the clinical outcome and prognosis of pediatric patients with newly diagnosed ITP in a single institution.METHODS: We reviewed retrospectively the medical records of 170 patients with newly diagnosed ITP at Chungnam National University Hospital (CNUH) from January 2005 to December 2015. The demographics, complete blood count (CBC), leukocyte differential counts and treatment of patients with ITP were reviewed.RESULTS: The median age at diagnosis were 20 months old (range, 0 to 189 months) for acute ITP and 52 months old for chronic ITP. After initial diagnosis of ITP, 20 of 170 patients (11.8%) were later diagnosed as chronic ITP. Age at diagnosis and absolute lymphocyte count (ALC) at diagnosis were statistically correlated with development of chronic ITP. ALC at diagnosis and at discharge were significantly higher in acute ITP patients than chronic ITP patients. We determined that ALC >4,109/μL at diagnosis and ALC >3,825/μL at discharge were associated with platelet recovery after 12 months.CONCLUSION: This study demonstrated that that high ALC at admission and discharge predict a favorable outcome in children with newly diagnosed ITP. Further studies are warranted to validate these findings.


Subject(s)
Blood Cell Count , Blood Platelets , Child , Demography , Diagnosis , Hemorrhage , Humans , Immune System , Leukocytes , Lymphocyte Count , Medical Records , Prognosis , Purpura, Thrombocytopenic, Idiopathic , Retrospective Studies
6.
Article in Korean | WPRIM | ID: wpr-23113

ABSTRACT

BACKGROUND: Immune thrombocytopenic purpura (ITP) is an acquired bleeding disorder in which the immune system destroys platelets. There were many studies which predicted the factors associated with the prognosis of childhood ITP, but controversies remained. We analyzed the predicting factors associated with the clinical outcome and prognosis of pediatric patients with newly diagnosed ITP in a single institution. METHODS: We reviewed retrospectively the medical records of 170 patients with newly diagnosed ITP at Chungnam National University Hospital (CNUH) from January 2005 to December 2015. The demographics, complete blood count (CBC), leukocyte differential counts and treatment of patients with ITP were reviewed. RESULTS: The median age at diagnosis were 20 months old (range, 0 to 189 months) for acute ITP and 52 months old for chronic ITP. After initial diagnosis of ITP, 20 of 170 patients (11.8%) were later diagnosed as chronic ITP. Age at diagnosis and absolute lymphocyte count (ALC) at diagnosis were statistically correlated with development of chronic ITP. ALC at diagnosis and at discharge were significantly higher in acute ITP patients than chronic ITP patients. We determined that ALC >4,109/μL at diagnosis and ALC >3,825/μL at discharge were associated with platelet recovery after 12 months. CONCLUSION: This study demonstrated that that high ALC at admission and discharge predict a favorable outcome in children with newly diagnosed ITP. Further studies are warranted to validate these findings.


Subject(s)
Blood Cell Count , Blood Platelets , Child , Demography , Diagnosis , Hemorrhage , Humans , Immune System , Leukocytes , Lymphocyte Count , Medical Records , Prognosis , Purpura, Thrombocytopenic, Idiopathic , Retrospective Studies
7.
Blood Research ; : 254-263, 2017.
Article in English | WPRIM | ID: wpr-21833

ABSTRACT

Management options for patients with immune thrombocytopenia (ITP) have evolved substantially over the past decades. The American Society of Hematology published a treatment guideline for clinicians referring to the management of ITP in 2011. This evidence-based practice guideline for ITP enables the appropriate treatment of a larger proportion of patients and the maintenance of normal platelet counts. Korean authority operates a unified mandatory national health insurance system. Even though we have a uniform standard guideline enforced by insurance reimbursement, there are several unsolved issues in real practice in ITP treatment. To optimize the management of Korean ITP patients, the Korean Society of Hematology Aplastic Anemia Working Party (KSHAAWP) reviewed the consensus and the Korean data on the clinical practices of ITP therapy. Here, we report a Korean expert recommendation guide for the management of ITP.


Subject(s)
Anemia, Aplastic , Clothing , Consensus , Evidence-Based Practice , Hematology , Humans , Insurance , National Health Programs , Platelet Count , Purpura, Thrombocytopenic, Idiopathic
8.
Article in English | WPRIM | ID: wpr-137677

ABSTRACT

BACKGROUND: Prescription of inappropriate medicine to elderly patients is a major public health care concern. The Beers criteria have been commonly employed as a screening tool to identify the use of potentially inappropriate medications (PIMs). The present study investigated the prevalence of PIM use according to the Beers criteria as well as factors related to PIM use. METHODS: Data obtained from a retrospective survey included 25,810 outpatients aged ≥65 years from a university medical center in Seoul, Korea. PIMs were defined using the Beers criteria. Factors associated with PIM use were evaluated using multiple regression analysis. RESULTS: Of all participants, 7,132 (27.6%) were prescribed at least one PIM. The most commonly prescribed PIMs were alprazolam (11.2%), clonazepam (10.8%), zolpidem (8.7%), quetiapine (8.4%), and hydroxyzine (5.4%). In multivariate logistic regression analysis, having five or more prescription medicines (odds ratio [OR], 11.32; 95% confidence interval [CI], 9.38 to 13.66) and five or more prescribing doctors (OR, 4.40; 95% CI, 3.59 to 5.39) were strongly associated with PIM. In a likelihood ratio test for trend, an increasing number of medications and prescribing doctors were both significantly associated with PIM. CONCLUSION: At a university medical center, the number of medications and the number of prescribing doctors was associated with PIM in older outpatients.


Subject(s)
Academic Medical Centers , Aged , Alprazolam , Beer , Clonazepam , Humans , Hydroxyzine , Korea , Logistic Models , Mass Screening , Outpatients , Potentially Inappropriate Medication List , Prescriptions , Prevalence , Public Health , Quetiapine Fumarate , Retrospective Studies , Risk Factors , Seoul
9.
Article in English | WPRIM | ID: wpr-137676

ABSTRACT

BACKGROUND: Prescription of inappropriate medicine to elderly patients is a major public health care concern. The Beers criteria have been commonly employed as a screening tool to identify the use of potentially inappropriate medications (PIMs). The present study investigated the prevalence of PIM use according to the Beers criteria as well as factors related to PIM use. METHODS: Data obtained from a retrospective survey included 25,810 outpatients aged ≥65 years from a university medical center in Seoul, Korea. PIMs were defined using the Beers criteria. Factors associated with PIM use were evaluated using multiple regression analysis. RESULTS: Of all participants, 7,132 (27.6%) were prescribed at least one PIM. The most commonly prescribed PIMs were alprazolam (11.2%), clonazepam (10.8%), zolpidem (8.7%), quetiapine (8.4%), and hydroxyzine (5.4%). In multivariate logistic regression analysis, having five or more prescription medicines (odds ratio [OR], 11.32; 95% confidence interval [CI], 9.38 to 13.66) and five or more prescribing doctors (OR, 4.40; 95% CI, 3.59 to 5.39) were strongly associated with PIM. In a likelihood ratio test for trend, an increasing number of medications and prescribing doctors were both significantly associated with PIM. CONCLUSION: At a university medical center, the number of medications and the number of prescribing doctors was associated with PIM in older outpatients.


Subject(s)
Academic Medical Centers , Aged , Alprazolam , Beer , Clonazepam , Humans , Hydroxyzine , Korea , Logistic Models , Mass Screening , Outpatients , Potentially Inappropriate Medication List , Prescriptions , Prevalence , Public Health , Quetiapine Fumarate , Retrospective Studies , Risk Factors , Seoul
10.
Article in Korean | WPRIM | ID: wpr-788540

ABSTRACT

Germ cell tumors the designation given to neoplasm arising from the cells of the germline, the cells that are destined to become either the egg or the sperm. These tumors have a number of unique features that includes bimodal and wide age distribution, remarkable phenotypic diversity, and varying biologic behavior. During infancy, sacrococcygeal locations predominate with either teratomas in neonates or endodermal sinus tumors in infants above three months. After puberty, non-germinomatous germ cell tumors predominate with gonadal, mediastinal or intracranial tumor. Specific subtypes of germ cell tumors secrete proteins as tumor markers. Surgical resection of the tumor is necessary to establish the diagnosis and for staging of the extent of tumor spread. Except for teratoma, germ cell tumors are highly sensitive to chemotherapy in particular cisplatin. The most commonly used chemotherapy regimen for malignant germ cell tumors is PEB (cisplatin, etoposide and bleomycin). Prognosis is good even in metastatic diseases. Patients with relapsed or recurrent disease may be candidates for high dose chemotherapy and autologous hematopoietic stem cell transplantation.


Subject(s)
Adolescent , Age Distribution , Choriocarcinoma , Cisplatin , Diagnosis , Drug Therapy , Endodermal Sinus Tumor , Etoposide , Female , Germinoma , Gonads , Hematopoietic Stem Cell Transplantation , Humans , Infant , Infant, Newborn , Neoplasms, Germ Cell and Embryonal , Ovum , Pediatrics , Pregnancy , Prognosis , Puberty , Spermatozoa , Teratoma , Biomarkers, Tumor
11.
Article in Korean | WPRIM | ID: wpr-13546

ABSTRACT

Germ cell tumors the designation given to neoplasm arising from the cells of the germline, the cells that are destined to become either the egg or the sperm. These tumors have a number of unique features that includes bimodal and wide age distribution, remarkable phenotypic diversity, and varying biologic behavior. During infancy, sacrococcygeal locations predominate with either teratomas in neonates or endodermal sinus tumors in infants above three months. After puberty, non-germinomatous germ cell tumors predominate with gonadal, mediastinal or intracranial tumor. Specific subtypes of germ cell tumors secrete proteins as tumor markers. Surgical resection of the tumor is necessary to establish the diagnosis and for staging of the extent of tumor spread. Except for teratoma, germ cell tumors are highly sensitive to chemotherapy in particular cisplatin. The most commonly used chemotherapy regimen for malignant germ cell tumors is PEB (cisplatin, etoposide and bleomycin). Prognosis is good even in metastatic diseases. Patients with relapsed or recurrent disease may be candidates for high dose chemotherapy and autologous hematopoietic stem cell transplantation.


Subject(s)
Adolescent , Age Distribution , Choriocarcinoma , Cisplatin , Diagnosis , Drug Therapy , Endodermal Sinus Tumor , Etoposide , Female , Germinoma , Gonads , Hematopoietic Stem Cell Transplantation , Humans , Infant , Infant, Newborn , Neoplasms, Germ Cell and Embryonal , Ovum , Pediatrics , Pregnancy , Prognosis , Puberty , Spermatozoa , Teratoma , Biomarkers, Tumor
12.
Article in English | WPRIM | ID: wpr-221892

ABSTRACT

We describe a very rare case of 6.9-year-old boy with Down syndrome (DS) and a prior history of transient myeloproliferative disorder. He was diagnosed with acute megakaryoblastic leukemia and found to have a novel GATA1 gene mutation, as well as a complex karyotype without recurrent acute myeloid leukemia (AML) aberrations. The patient achieved an early bone marrow response to chemotherapy. However, relapse occurred during treatment, 9 months after the initial diagnosis. Although GATA1 mutations are closely associated with DS-AML, we speculate that factors other than the presence of the GATA1 mutation can affect the overall outcome in older pediatric patients.


Subject(s)
Bone Marrow , Down Syndrome , Humans , Karyotype , Leukemia, Megakaryoblastic, Acute , Leukemia, Myeloid, Acute , Myeloproliferative Disorders , Recurrence
13.
Article in English | WPRIM | ID: wpr-788479

ABSTRACT

We describe a very rare case of 6.9-year-old boy with Down syndrome (DS) and a prior history of transient myeloproliferative disorder. He was diagnosed with acute megakaryoblastic leukemia and found to have a novel GATA1 gene mutation, as well as a complex karyotype without recurrent acute myeloid leukemia (AML) aberrations. The patient achieved an early bone marrow response to chemotherapy. However, relapse occurred during treatment, 9 months after the initial diagnosis. Although GATA1 mutations are closely associated with DS-AML, we speculate that factors other than the presence of the GATA1 mutation can affect the overall outcome in older pediatric patients.


Subject(s)
Bone Marrow , Down Syndrome , Humans , Karyotype , Leukemia, Megakaryoblastic, Acute , Leukemia, Myeloid, Acute , Myeloproliferative Disorders , Recurrence
14.
Yonsei Medical Journal ; : 238-245, 2013.
Article in English | WPRIM | ID: wpr-17423

ABSTRACT

PURPOSE: We evaluated the effect of human parathyroid hormone (hPTH) on the engraftment and/or in vivo expansion of hematopoietic stem cells in an umbilical cord blood (UCB)-xenotransplantation model. In addition, we assessed its effect on the expression of cell adhesion molecules. MATERIALS AND METHODS: Female NOD/SCID mice received sublethal total body irradiation with a single dose of 250 cGy. Eighteen to 24 hours after irradiation, 1x107 human UCB-derived mononuclear cells (MNCs) and 5x106 human UCB-derived mesenchymal stem cells (MSCs) were infused via the tail vein. Mice were randomly divided into three groups: Group 1 mice received MNCs only, Group 2 received MNCs only and were then treated with hPTH, Group 3 mice received MNCs and MSCs, and were treated with hPTH. RESULTS: Engraftment was achieved in all the mice. Bone marrow cellularity was approximately 20% in Group 1, but 70-80% in the hPTH treated groups. Transplantation of MNCs together with MSCs had no additional effect on bone marrow cellularity. However, the proportion of human CD13 and CD33 myeloid progenitor cells was higher in Group 3, while the proportion of human CD34 did not differ significantly between the three groups. The proportion of CXCR4 cells in Group 3 was larger than in Groups 1 and 2 but without statistical significance. CONCLUSION: We have demonstrated a positive effect of hPTH on stem cell proliferation and a possible synergistic effect of MSCs and hPTH on the proportion of human hematopoietic progenitor cells, in a xenotransplantation model. Clinical trials of the use of hPTH after stem cell transplantation should be considered.


Subject(s)
Animals , Bone Marrow/metabolism , Cell Proliferation , Female , Fetal Blood/cytology , Flow Cytometry , Hematopoietic Stem Cell Transplantation , Hematopoietic Stem Cells/drug effects , Humans , Leukocytes, Mononuclear/cytology , Mesenchymal Stem Cell Transplantation , Mesenchymal Stem Cells/cytology , Mice , Mice, Inbred NOD , Mice, SCID , Parathyroid Hormone/therapeutic use , Stem Cells/cytology , Transplantation, Heterologous
15.
Article in English | WPRIM | ID: wpr-720311

ABSTRACT

BACKGROUND: Gelsolin and matrix metalloproteinase 12 (MMP12) expression has been reported in Langerhans cell histiocytosis (LCH), but the clinical significance of this expression is unknown. We investigated the associations of these proteins with clinical manifestations in patients diagnosed with LCH. METHODS: We performed a retrospective analysis of clinical data from patients diagnosed with LCH and followed up between 1998 and 2008. Available formalin-fixed, paraffin-embedded specimens were used for gelsolin and MMP12 immunohistochemical staining. We analyzed the expression levels of these proteins and their associations with LCH clinical features. RESULTS: Specimens from 36 patients (20 males, 16 females) with a diagnosis of LCH based on CD1a positivity with clinical manifestations were available for immunohistochemical staining. Median patient age was 62 months (range, 5 to 207). The expression of gelsolin varied; it was high in 17 patients (47.2%), low in 11 patients (30.6%), and absent in 8 patients (22.2%). The high gelsolin expression group had a higher tendency for multi-organ and risk organ involvement, although the trend was not statistically significant. MMP12 was detected only in 7 patients (19.4%) who showed multi-system involvement (P=0.018) and lower event-free survival (P=0.002) in comparison to patients with negative MMP12 staining. CONCLUSION: Gelsolin and MMP12 expression may be associated with the clinical course of LCH, and MMP12 expression may be particularly associated with severe LCH. Further studies of larger populations are needed to define the precise role and significance of gelsolin and MMP12 in the pathogenesis of LCH.


Subject(s)
Disease-Free Survival , Gelsolin , Histiocytosis , Histiocytosis, Langerhans-Cell , Humans , Immunohistochemistry , Langerhans Cells , Male , Matrix Metalloproteinase 12 , Proteins , Retrospective Studies
16.
Article in English | WPRIM | ID: wpr-39059

ABSTRACT

The triad of rash, arthritis, and uveitis seems to be characteristic for early-onset childhood sarcoidosis. We describe an interesting case of early-onset childhood sarcoidosis coexisting enchondromatosis, which clinically masquerade as Langerhans cell histiocytosis. A 33 months old girl presented with skin rash, subcutaneous nodules with polyarthritis, and revealed the involvement of lymph nodes as well as spleen during work-up. She also presented with multiple osteolytic lesions which pathologically proven enchondromatosis. Oral prednisone was prescribed at 2 mg/kg/day for 2 months until when subcutaneous nodules and joint swellings almost disappeared, and then slowly tapered over a period of 5 months. We report an unusual case of early-onset childhood sarcoidosis presented with osteolytic bone lesions which were irrelevant to sarcoidosis.


Subject(s)
Administration, Oral , Anti-Inflammatory Agents/therapeutic use , Arthritis/complications , Child, Preschool , Diagnosis, Differential , Enchondromatosis/complications , Exanthema/etiology , Female , Humans , Positron Emission Tomography Computed Tomography , Prednisone/therapeutic use , Sarcoidosis/complications , Whole Body Imaging
18.
Article in English | WPRIM | ID: wpr-720270

ABSTRACT

BACKGROUND: In this study, we investigated the effects of reduced-dose craniospinal radiotherapy (CSRT) followed by tandem high-dose chemotherapy (HDCT) with autologous stem cell rescue (ASCR) in children with a newly diagnosed high-risk medulloblastoma (MB) or supratentorial primitive neuroectodermal tumor (sPNET). METHODS: Between March 2005 and April 2007, patients older than 3 years with a newly diagnosed high-risk MB or sPNET were enrolled. The patients received two cycles of pre-RT chemotherapy consisting of cisplatin, etoposide, vincristine, and cyclophosphamide (cycle A), and carboplatin, etoposide, vincristine, and ifosphamide (cycle B), followed by CSRT with 23.4 Gy and local RT with 30.6 Gy. After four cycles of post-RT chemotherapy (cycles A, B, A, and B), tandem double HDCT with ASCR was performed. RESULTS: A total of 13 patients (MB=11, sPNET=2) were enrolled. Of these, one patient progressed, one patient died of septic shock after the second cycle of B, and one patient relapsed after the third cycle of B. The 3-year event-free survival (EFS) rate of the patients intended for HDCT was 76.9%, whereas the 3-year EFS rate of the patients who received HDCT was 100%. No treatment-related mortality occurred during HDCT. CONCLUSION: Although the follow-up period was short and the patient cohort was small in size, the results of this study are encouraging. The limited toxicity and favorable EFS rate observed in children treated with reduced-dose CSRT followed by HDCT and ASCR warrant further exploration in a larger study population.


Subject(s)
Carboplatin , Child , Cisplatin , Cohort Studies , Cyclophosphamide , Disease-Free Survival , Etoposide , Follow-Up Studies , Humans , Medulloblastoma , Neuroectodermal Tumors, Primitive , Shock, Septic , Stem Cells , Vincristine
19.
Article in English | WPRIM | ID: wpr-188011

ABSTRACT

We describe a girl with Diamond-Blackfan anemia with accompanying red cell enolase deficiency. At the age of 9 yr old, the patient received allogeneic bone marrow transplantation from her HLA-identical sister who had normal red cell enolase activity. While the post transplant DNA analysis with short tandem repeat has continuously demonstrated a stable mixed chimerism on follow-up, the patient remains transfusion independent and continues to show a steady increase in red cell enolase activity for over two and a half years following bone marrow transplantation.


Subject(s)
Anemia, Diamond-Blackfan/blood , Bone Marrow Cells/cytology , Bone Marrow Transplantation , Child , Child, Preschool , Erythrocytes/enzymology , Female , Humans , Infant , Phosphopyruvate Hydratase/genetics , Transplantation, Homologous
20.
Article in English | WPRIM | ID: wpr-155857

ABSTRACT

Functioning adrenocortical oncocytomas are extremely rare and most reported patients are 40-60 yr of age. To our knowledge, only 2 cases of functioning adrenocortical oncocytomas have been reported in childhood. We report a case of functioning adrenocortical oncocytoma in a 14-yr-old female child presenting with virilization. She presented with deepening of the voice and excessive hair growth, and elevation of plasma testosterone and dehydroepiandrosterone sulfate. She had an adrenalectomy. The completely resected tumor composed predominantly of oncocytes without atypical mitosis and necrosis. A discussion of this case and a review of the literature on this entity are presented.


Subject(s)
Adenoma, Oxyphilic/complications , Adolescent , Adrenal Cortex Neoplasms/complications , Adrenalectomy , Adult , Female , Humans , Male , Middle Aged , Virilism/etiology
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