Cross-Cultural Adaptation of the Korean Version of the Minneapolis-Manchester Quality of Life Instrument-Adolescent Form

We verified the reliability and validity of the Korean version of the Minneapolis-Manchester Quality of Life Instrument-Adolescent Form (KMMQL-AF) among Korean childhood cancer survivors. A total of 107 childhood cancer patients undergoing cancer treatment and 98 childhood cancer survivors who completed cancer treatment were recruited. To assess the internal structure of the KMMQL-AF, we performed multi-trait scaling analyses and exploratory factor analysis. Additionally, we compared each domains of the KMMQL-AF with those of the Karnofsky Performance Status Scale and the Revised Children's Manifest Anxiety Scale (RCMAS). Internal consistency of the KMMQL-AF was sufficient (Cronbach's alpha: 0.78-0.92). In multi-trait scaling analyses, the KMMQL-AF showed sufficient construct validity. The "physical functioning" domain showed moderate correlation with Karnofsky scores and the "psychological functioning" domain showed moderate-to-high correlation with the RCMAS. The KMMQL-AF discriminated between subgroups of different adolescent cancer survivors depending on treatment completion. The KMMQL-AF is a sufficiently reliable and valid instrument for measuring quality of life among Korean childhood cancer survivors.

Time to establish multidisciplinary childhood cancer survivorship programs in Korea / 대한혈액학회지

Improved treatment strategies and better supportive care have resulted in increased survival rates for childhood cancers. However, most of the survivors may have complex, long-term health issues. In 2004, Childhood Cancer Survivorship Study of the United States confirmed that both survivors and the medical community need to be educated about the late effects of childhood cancer treatment. Korea, with an estimated number of childhood cancer survivors of 20,000 to 25,000, faces similar challenges that the United States had experienced earlier. Despite of the tight budgetary situation on the part of government and hospitals, nationwide cohort study for Korean childhood cancer survivors was proposed and the versions of instruments to measure the quality of life of childhood cancer survivors were already validated. Nationwide registry of long-term survivors as well as the multi-center study is to be developed not only for the care of the survivors but also to raise the patient's awareness of the importance of regular follow-up. In addition to education of primary care providers regarding the survivorship, the need for special education program in the school system is emphasized.

Normalization of Red Cell Enolase Level Following Allogeneic Bone Marrow Transplantation in a Child with Diamond-Blackfan Anemia

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.

Hematopoietic Stem Cell Transplantation for Children with Malignant Lymphoma in Korea: Multicenter Retrospective Study / 대한소아혈액종양학회지

PURPOSE: Children with Malignant lymphoma who is in the advanced stage at diagnosis or relapses during treatment have a poor prognosis. Recently, hematopoietic stem cell transplantation (HSCT) for advanced stage or refractory/relapsed lymphoma performed frequently. However, the role for HSCT for children with malignant lymphoma is still controversial. In this study, we reviewed children with malignant lymphoma who received HSCT and analyzed the results. METHODS: Questionnaires were made and sent to a group of teaching hospitals, with a return of 37 questionnaires from 11 hospitals. 33 patients with Non-Hodgkin lymphoma (NHL) and 4 patients with Hodgkin disease (HD) who received HSCT from 1997 to 2004 in Korea were enrolled in this study. Disease state at diagnosis, relapses during treatment, disease state at HSCT, and survival record were analyzed. All Data were reviewed with the questionnaires from the 11 teaching hospitals. RESULTS: Four patients with HD received HSCT at the 2nd complete remission after relapse. Survival rate for HD was 100% and their follow up duration ranged from 0.2 to 6.2 years (median 2.4 years). The 2-year survival rate for NHL was 68.1+/-9.0% and their follow up duration ranged from 0.1 to 7.6 years (median 1.5 years). The 2-year survival rate in patients with advanced stage at diagnosis and in relapsed/refractory patients were 83.6+/-1.1% and 55.9+/-12.9%, respectively (P=0.12). The mortality asssociated with HSCT was only 1 case, and most of the transplantation related complications did not resulted in death. CONCLUSION: Our results suggest that high dose chemotherapy followed by HSCT in children with malignant lymphoma is a safe procedure, which at the same time improves the results of standard treatment.

Treatment Outcome of Childhood B-cell Lymphoma and L3 Acute Lymphoblastic Leukemia from a Single Institution / 대한소아혈액종양학회지

PURPOSE: High survival rate can be obtained in B-cell lymphoma (Burkitt's lymphoma, diffuse large B-cell lymphoma) and L3 acute lymphoblastic leukemia (ALL) with multiagent chemotherapy. Objectives of this study were to evaluate the treatment outcomes of B-cell lymphoma and L3 ALL diagnosed at the Department of Pediatrics, Asan Medical Center. METHODS: The medical records of 32 children who were diagnosed with Burkitt's lymphoma, diffuse large B-cell lymphoma and L3 ALL from March 1992 to July 2004 at Asan Medical Center were reviewed retrospectively. The 5 year event free survival (EFS) according to the diagnosis, age, risk group and lactic dehydrogenase (LDH) level were analyzed. RESULTS: There were 23 boys and 9 girls. Age ranged from 9 months to 14.4 years old with a median of 7.1 years. Fourteen patients had L3 ALL, 11 had Burkitt's lymphoma and 7 had diffuse large B-cell lymphoma. Five patients (15.6%) had CNS involvement and 5 with B-cell lymphoma (27.8%, 5/18) had BM involvement. All patients who received appropriate chemotherapy achieved a complete remission (CR), but 18.8% (6/32) relapsed. Among 6 relapsed patients, 5 achieved CR after reinduction chemotherapy. One who had no response to secondary chemotherapy and 2 with isolated CNS relapse died due to disease progression. The most common treatment-related toxicity was myelosuppression (87.5%) followed by neutropenic fever (81.3%). Median follow up is 25 months (3 months to 74 months). Four patients who achieved CR after proper induction therapy (4/32, 12.5%) died, 3 due to relapse and 1 due to toxicity-related complication (neutropenia and sepsis). The 5 year EFS for all patients was 77.5+/-7.5% and the 5 year overall survival was 84.6+/-7.3%. The 5 year EFS of B-cell lymphoma compared with that of L3, ALL was 94.4+/-5.4% versus 55.1+/-13.9% (P=0.012) and 5 year overall survival of relapsed patients was 50.0+/-13.9%. CNS disease at diagnosis, age, LDH had no significant influence on EFS. CONCLUSION: High survival rate of childhood B-cell lymphomas and L3 ALL was obtained with recent intensive multiagent chemotherapy and about 50% of relapsed patients were salvaged with reinduction. High incidence of the treatment-related toxicity such as myelosuppression, neutropenic fever and TLS was observed, but the treatment-related mortality was very low with recent supportive therapies. Survival rate was improved with prompt and appropriate management for the treatment-related toxicity of the intensive chemotherapy.

Therapy for Malignant Brain Tumors in Infant and Young Children / 대한소아혈액종양학회지

No abstract available.

KSPNO Protocol for Glioma / 대한소아혈액종양학회지

No abstract available.

Epidemiology and Clinical Outcomes in Children with Malignant Lymphoma in Korea: Retrospective Study / 대한소아혈액종양학회지

PURPOSE: Malignant lymphoma is the primary malignant tumor derived from lymphoid organs. It is composed of Hodgkin's disease and non-Hodgkin lymphoma. Recently, survival rate is on the rise due to improved combination chemotherapy, radiotherapy and high dose chemotherapy followed by hematopoietic stem cell transplantation. In South Korea, no epidemiologic studies concerning malignant lymphoma in the pediatric age group has been performed. Therefore, the Korean Society of Pediatric Hematology-Oncology retrospectively analyzed the incidence, pathologic subtypes, treatment strategies, and survival rates of pediatric malignant lymphomas in South Korea. METHOD: Questionnaires were made and sent to a group of training hospitals, with a return of 580 questionnaires from 24 hospitals. Among them, 517 reports were suitable for analysis. RESULTS: Among the 517 cases, Hodgkin's disease accounted for 58 cases and non-Hodgkin's lymphoma for 459 cases. Male to female ratio for malignant lymphoma was 2.7. Mean age at diagnosis was 8.3 years. Among the pathologic subtypes, mixed cellularity was the most frequent subtype for Hodgkin's disease. Most (70.7%) cases of non-Hodgkins lymphoma belonged to high grade NHL. Burkitt lymphoma accounted for 102 cases, and lymphoblastic lymphoma was found in 58 cases. Peripheral lymphadenopathy was the most common presenting sign upon diagnosis. B symptoms were significantly more frequent in Hodgkin's disease patients than in non-Hodgkin lymphoma patients. The Complete response rate was 62.1% for non-Hodgkin's lymphoma, and 82.8% for Hodgkin's disease. Overall 5 year survival rate was 60.0% in non-hodgkin's lymphoma, and 84.8% in Hodgkin's disease. CONCLUSION: The annual incidence of malignant lymphoma in Korea is 4.7 per million. In cases of chemotherapy-sensitive, refractory or relapsed malinant lymphoma, high dose chemotherapy followed by hematopoietic stem cell transplantation is vital for improved survival. For more systematic analysis of epidemiology on malignant lymphomas, better surveillance mechanisms on the occurrence of malignant lymphomas are crucial, and establishment of standardized treatment protocol for malignant lymphoma is required.

The Role of Adjunctive Therapy of Optic Pathway Glioma in Children

OBJECTIVE: This study is performed in order to compare the outcomes of surgical management and to define the role of adjunctive therapy in the management of optic pathway glioma in children. METHODS: Sixteen children with optic pathway glioma had been managed in various treatment methods during the last 8 years. The patients aged from 5 months to 14 years. The patients presented with progressive visual loss, increased ICP symptoms, endocrine dysfunction, seizure, and motor weakness. Optic pathway glioma associated with neurofibromatosis was excluded. Tumor involved chiasmatico-hypothalamus(12 patients), optic chiasm(3), and optic nerve(1). The extent of surgical resection were radical(3 patients), subtotal(12), and partial(1). RESULTS: Three patients treated with radical resection showed no evidence of tumor recurrence. Among 12 patients treated with subtotal resection and without adjunctive therapy, 6 patients(50%) developed recurrence in the postoperative period of average 20.5 months. Those patients with recurrence were managed by reoperation(3 patients), irradiation therapy(2), and chemotherapy(1). However three patients with residual tumors after subtotal(2), or partial(1) resection were treated with adjunctive chemotherapy in the postoperative period showed no evidence of recurrence. CONCLUSION: Although radical resection of optic pathway glioma might offer long-term control of tumor, adjunctive chemotherapy could be effective to prevent tumor recurrence in children with subtotally or partially resected optic pathway glioma. More experience is necessary to determine the optimal method of treatment of optic pathway gliomas in children.

Pediatric Myelodysplastic Syndrome in Korea: Clinical Characteristics and Comparison of Prognostic Scoring Systems / 대한소아혈액종양학회지

PURPOSE: Myelodysplastic syndromes (MDS) are heterogeneous clonal stem cell disorders characterized by morphological and functional abnormal hematopoiesis, resulting in cytopenias in the peripheral blood. MDS in children are very rare and their clinical characteristics, natural history, the most effective treatment and prognostic factors need to be elucidated. In addition, consensus for the classification of pediatric MDS has not been reached yet. This multicenter, retrospective study aimed to describe the characteristics and the disease courses of 75 MDS patients from 11 University Hospitals in Korea, seen between 1991 and 2001. METHODS: Kaplan-Meier probability of leukemic transformation and overall survival were plotted. And the usefulness of prognostic scoring systems, including French-American-British (FAB) classification, Bournemouth scoring system (BSS), and International Prognostic Scoring System (IPSS) in the prediction of transformation to acute myelogenous leukemia (AML) and overall survival was evaluated. RESULTS: The median age was 65 months (2~175 months) and the sex ratio was 2.6: 1 (M: F). Fourteen patients (18.7%) were unable to be allocated into any subtype of FAB. The frequency of FAB subtypes in Korea was similar to that of Western countries except for higher proportion of refractory anemia (RA, 47.5%). Median survival was 54 months with Kaplan-Meier 5-yr survival probability of 31.9% and 2-yr probability of transformation to AML was 23.7%. None of the FAB, BSS, and IPSS was capable of discriminating subgroup of patients for the prediction of survival. However, all of the FAB (P=0.004), BSS (P=0.001), and IPSS (P=0.02) were able to subdivide subgroups for the prediction of transformation to AML. CONCLUSION: The characteristics of pediatric MDS in Korea were different from those of other countries, in light of the higher proportion of RA, the low percentage of inherited diseases, and the low percentage of cytogenetic abnormalities. However, the reasons of the differences were not clear. Moreover, none of the prognostic scoring systems, including IPSS, was reliably predictive of survival, reflecting differences from adult cases. With this multicenter study, we suggest the necessity of a prospective study for the classification and treatment. A newer, effective method should be developed for the prediction of disease progression and survival in pediatric MDS.

Childhood Acute Immune Thrombocytopenic Purpura in Korea: Multicenter Study of Korean Society of Pediatric Hematology/Oncology / 대한소아혈액종양학회지

PURPOSE: Childhood acute immune thrombocytopenic purpura (ITP) is a benign hematologic disease. Therapy does not affect the natural history of the illness. We evaluated the clinical and laboratory findings, treatment and prognosis of childhood acute ITP in Korea through a retrospective multicenter study. METHODS: We analyzed retrospectively the data of 1, 829 children with acute ITP through survey of 33 hospitals among 43 hospitals in Korea from Sep. 1992 to Aug. 2001. RESULTS: Male to female ratio was 1.3: 1 and the median age at the diagnosis of ITP was 2.9 (0.1 17) years. Median duration of follow up was 6 months. One hundred and forty nine cases of the total 1, 829 patients (8.1%) received no treatment. The initial median platelet count of the non-treated group was 42, 500/mm3. Among the 861 cases who were followed up over 6 months, 315 cases (36.6%) progressed into chronic ITP. Associated with this high rate of chronicity of childhood acute ITP patients in Korea, we must consider the fact that acute ITP patients with fast improvement in the first episode tend not to follow up. Considering that fact, the rate of chronicity becomes 17.2% of the 1, 829 acute ITP patients. The treated group used many kinds of treatment methods. Intravenous immunoglobulins (IVIG) with or without prednisolone (PD) (67.5%) were the most commonly used regimens. In the group treated with IVIG alone, the platelet count began to rise above 50, 000/mm3 at 2.6 days, 100, 000/mm3 at 3.7 days and 150, 000/mm3 at 4.9 days. Four hundred and twenty two cases of the 1, 686 (25.0%) cases followed up after first episode of ITP relapsed. The relapse rate was significantly higher in older patients and in girls than in younger patients and in boys (P< 0.05). The chronicity of ITP statistically increased with age (P< 0.05) and that was the only valuable factor. CONCLUSION: Despite the fact that childhood acute ITP is a pretty common disaese, there is no agreement on the best treatment method for this disease. The establishment of Korean treatment guideline of childhood acute ITP, based on an analysis of multicenters, seems to be needed.

Clinical Characteristics of Biphenotypic Acute Leukemia in Childhood: A Single Institutional Experience / 대한소아혈액종양학회지

PURPOSE: The diagnostic criteria of the biphenotypic acute leukemia (BAL) are based on the number and degree of the specificity of the immunological markers expressed on the leukemic blasts. The newer diagnostic criteria proposed by The European Group for the Immunological Classification of Leukaemias (EGIL) is now well accepted. We have recently evaluated our BAL patients using the EGIL criteria and analyzed the clinical characteristics, treatment and clinical outcome. METHODS: Fourteen children diagnosed with BAL among 193 childhood acute leukemia patients in our hospital from January 1995 to December 2001 were retrospectively reviewed. RESULTS: The incidence of BAL was 7.3% (14 out of 193 cases). Of these 14 patients, 12 were de novo and 2 were secondary. In the de novo group, the immunological marker studies showed myeloid/B-lymphoid phenotype in 6 (50%), myeloid/T-lymphoid in 3 and B-lymphoid/T-lymphoid in 1. In addition, two patients showed trilineage differentiation. Cytogenetic analysis revealed various abnormal karyotypes in the majority of the cases, showing normal karyotype only in 3 cases. Among the karyotype abnormalities, two were t (9; 22) and one was t (4; 11). Chemotherapy was mostly based on the induction regimen of acute lymphoblastic leukemia therapy (12 of 14 cases). One patient was treated with acute myeloid leukemia regimen and one patient received combination of both acute lymphoid and myeloid regimen. Induction of complete remission was achieved in 100% of the patients and the median duration of induction therapy to complete remission was 33 days. Five of the 12 de novo patients died during the median follow-up of 16.5 months (4 months to 37.5 months) and the 2 year survival rate was only 52%. CONCLUSION: The incidence of BAL in children is relatively rare and the most common immunophenotypic expression consists of myeloid and B-lymphoid coexpression. Most of the cases showed chromosomal abnormalities. The outcome of BAL treated mostly with the traditional acute lymphoblastic leukemia induc without stem cell transplantation should be sought especially in those expressing poor prognostic cytogenetic abnormalities or strong myeloid marker expression.

The Frequency of HLA Alleles in Korean Children with Aplastic Anemia and the Correlation with the Response to Immunosuppressive Treatment / 대한소아혈액종양학회지

PURPOSE: Immune pathophysiology of aplastic anemia (AA) has been indirectly inferred from responses to immunosuppressive agents. An association between AA and HLA-A2, or HLA-DR2 (its serologic split, HLA-DR15; or its molecular correspondents, DRB1*1501) has been implicated. The presence of HLA-DR15 (including DR2 or DRB1*15) has been closely associated with a favorable response to immunosuppression in AA. This study was aimed to characterize Korean patients with AA by determining the association with certain HLA alleles, such as HLA-DR2 or HLA-A2, and their implications in terms of the response to immunosuppression. METHODS: One-hundred eighteen children with AA from 10 university hospitals between 1990 and 2001 were enrolled in this multicenter, retrospective study. Among them, HLA data were available from 80 patients. Tests of proportions were used to compare allelic frequencies. RESULTS: The frequency of HLA-A2 (58.8%) or HLA-DR2 (24.7%) in AA was not significantly different from those of the controls. Analysis of the patients treated with immunosuppression (N=86) showed that, 50.0% of patients showed a response, including 16.0% of complete response at 6 months. The presence of DR2 allele did not portend a favorable response to immunosuppressive therapy. CONCLUSION: Unlike Western countries, the association of AA with certain HLA alleles was not documented in the Korean population. Moreover, the presence of HLA-DR2 did not predict a favorable response to immunosuppression. This peculiar characteristics of Korean AA needs to be investigated whether these findings reflect ethnic differences, different contribution of immune-mediated AA, different immune mechanisms, or mere limitation by number of study patients.

A Case of Posttransplant Lymphoproliferative Disease Following Renal Transplantation in a Child

Posttransplant lymphoproliferative disease(PTLD) has emerged as a potential life-threatening complication of immunosuppressive therapy after organ transplantation. The occurrence of PTLD is usually associated with an Epstein-Barr virus(EBV) infection in patients who are treated by aggressive immunosuppressive therapy. PTLD is represented by diverse manifestations ranging from reactive lymphoid hyperplasia to high grade malignant lymphoma. This is a case report of a late PTLD in a child. The patient is a 14-year-old girl, who presented as malignant lymphoma 44 months after successful renal transplantation. There was no evidence of EBV infection. On bone marrow study, many neoplastic lymphoid cells were detected. Aggressive chemotherapy for PTLD had resulted in clinical remission. However the patient expired from uncontrolled sepsis and septic shock after 77 days.

Childhood Brain Tumors

No abstract available.

Atypical Teratoid/Rhabdoid Tumor in Central Nervous System: Report of 2 Cases

We report two cases of atypical teratoid/rhabdoid tumor(AT/RT) in the central nervous system. Primary central nervous system AT/RT is an extremely rare malignant tumor affecting infants and young children. These tumors have been diagnosed previously as primitive neuroectodermal tumors(PNETs) because AT/RT contains fields indistinguishable from classic PNETs. Separation of these two tumor types is crucial because the prognosis for AT/RT is poor even when treatment includes surgery with or without radiation therapy and/or chemotherapy. Clinical, radiological and histopathological features of AT/RT are presented.

Analysis of Etiology and Prognosis of Pulmonary Complications in Children with Hematological or Oncological Disorders in Pediatric Intensive Care Unit

PURPOSE: In the course of treatment, patients with hematological or oncological disorders often develop pulmonary complication. The patients who develop a severe pulmonary complication have a poor outlook. The causes of pulmonary complication are either infectious or non-infectious in origin. We have analyzed the etiology and outcome of these patients admitted to the pediatric intensive care unit of Asan Medical Center. METHODS: Medical records of 95 patients on Pediatric oncology service who were admitted to pediatric intensive care unit(PICU) of Asan Medical Center from Jan 1997 to May 2000 were retrospectively reviewed. RESULTS: The mean age of the patients was 8.5 years(2 months-18 years). The underlying malignancies of these 95 patients were as following; acute lymphoblastic leukemia(31 cases), lymphoma (11 cases), acute myeloid leukemia(nine cases), brain tumor(eight cases) and other solid tumors(25 cases). Pulmonary complications included pneumonia, acute respiratory failure, pneumothorax and pleural effusion. The most common cause of pulmonary complication was infection(88%) in etiology. The overall mortality rate was 56.8%. Pulmonary complications in these patients carried high rates of mortality regardless of whether they were immune compromised(76%) or not(69%). Even without pulmonary complications, the hematological or oncological patients admitted to PICU had high mortality rates of 43%. CONCLUSION: Pulmonary complications are frequent finding in the hematological or oncological patients admitted to Intensive Care Unit. The main etiology of these pulmonary complications was infection, which carried a high mortality rate regardless of their immune status at the time when they were admitted to PICU

The Implications of E2A-PBX1 Positivity and Effect of Delayed Intensification Chemotherapy in t(1;19)-positive Childhood Acute Lymphoblastic Leukemia / 대한혈액학회지

PURPOSE: We studied the E2A-PBX1 positivity in t(1;19)-positive childhood acute lymphoblastic leukemia (ALL) patients during chemotherapy and follow-up period to evaluate the clinical implications of minimal residual disease (MRD) and the effect of delayed intensification chemotherapy on long-term survival. METHOD: Fifty-six bone marrow or peripheral blood samples collected retrospectively or prospectively before or during chemotherapy from 14 childhood ALL patients who had t(1;19) or E2A-PBX1 transcript at initial diagnosis were studied for the presence of E2A-PBX1 by RT- PCR. All patients received delayed intensification chemotherapy regardless of standard prognostic grouping for childhood ALL to evaluate its effect on the improvement of long-term survival. RESULTS: There were 11 t(1;19)-positive cases documented by karyotyping and 3 E2A-PBX1 transcript-positive cases amplified by PCR from the initial bone marrow samples. There were 11 cases of FAB L1 and 3 cases of L2. Immunophenotypic classification revealed 10 cases of group V, 2 cases of group IV, and 1 case of group III. Among 11 cases with documented karyotype, 9 cases (81.8%) had der(19)t(1;19) and the other 2 had balanced t(1;19). Fifty-six samples collected at 2 to 7 different time points of 14 patients revealed 4 cases of MRD immediately after completion of induction chemotherapy despite hematologic complete remission. Three of these cases relapsed eventually, and 1 was lost to follow-up. Among 12 cases who received adequate delayed intensificiation chemotherapy, 10 are alive in complete remission. CONCLUSION: MRD detection by RT-PCR amplification of E2A-PBX1 transcript was feasible, and the sample after completion of induction chemotherapy was most informative for the prediction of long-term survival and relapse. The presence of MRD after completion of induction chemotherapy conferred poor prognosis. The addition of delayed intensification chemotherapy to standard chemotherapy regimen could abolish the adverse effect of t(1;19) in childhood ALL patients.

Mitoxantrone Based Chemotherapy Regimen for Remission Induction in Children with Refractory Acute Myeloid Leukemia / 대한소아혈액종양학회지

PURPOSE: The effect of salvage chemotherapy using mitoxantrone based regimen for refractory AML (RAML) was analyzed. METHODS: Between January 1990 and March 2001, we treated 26 RAML. Ten patients received salvage chemotherapy with mitoxantrone based regimen and 16 patients with regimen devoid of mitoxantrone. RESULTS: Of total 54 AML patients treated during this period, 48 cases were available for analysis. RAML patients were 26 cases (54.1%) and M:F ratio was 2.3:1. FAB M2 and M4 were 8 each, and 7 cases were M7. There were 1 case of M1, M5 and M6 each. Eleven (42.3%) of them showed chromosomal abnormalities with variable karyotypes. Patients on mitoxantrone regimen all achieved complete remission (CR), while mitoxantrone devoid regimen achieved CR in only 56.2%. Allogeneic bone marrow transplantation was done in 5 cases (19.2%) of RAML, who are all alive with the median survival duration of 18 months. In mitoxantrone based regimen, 4 cases developed congestive heart failure and 5 had severe infection due to prolonged myelosuppression. In non-mitoxantrone group, 5 of 7 cases who failed to achieve CR died of sepsis. CONCLUSION: The mortality of RAML patients who are refractory to salvage chemotherapy remains very high. This study suggests that mitoxantrone based chemotherapy is effective in achieving complete remission as well as in prolonging survival in RAML patients.

High Dose Chemotherapy with Autologous Peripheral Blood Stem Cell Transplantation in Patients with Medulloblastoma/Primitive Neuroectodermal Tumor / 대한소아혈액종양학회지

PURPOSE: To improve survival and/or to avoid radiotherapy, high dose chemotherapy (HDCT) with autologous peripheral blood stem cell transplantation (PBSCT) was given to patients with recurrent or high risk medulloblastoma (MB)/primitive neuroectodermal tumor (PNET) as well as patients younger than 3 years of age. METHODS: Six patients (3 recurrent, 1 high risk, 2 younger than 3 years; 5 MBs and 1 PNET) received single or double HDCT and PBSCT with or without immunotherapy using interleukin-2. Chemotherapeutic regimen in the first HDCT included cyclophosphamide (1,500 mg/m2/ day for 4 days) and melphalan (60 mg/m2/day for 3 days). Chemotherapeutic regimen in the second HDCT included carboplatin (400 mg/m2/day for 3 days), thiotepa (250 mg/ m2/day for 3 days), and etoposide (200 mg/m2/day for 3 days). RESULTS: Nine HDCTs were applied in 6 patients. Three double HDCTs were rescued with peripheral blood stem cells collected during single round leukapheresis. Rapid hematologic recovery occurred in 4 patients. Engraft failure occurred in 1 patient and delayed granulocyte recovery and platelet engraft failure occurred in 1 patient. Three patients who had minimal disease before HDCT had event free survival for 7~18 months after HDCT. Tumor relapsed 8 and 12 months after single HDCT in 2 patients among 3 patients with recurrent MB/PNET. One patient with recurrent MB died due to engraft failure and sepsis. CONCLUSION: HDCT with autologous PBSCT is expected to improve survival of patients with poor prognosis MB/PNET including younger patients less than 3 years. Subsequent trials with larger number of patients and long-term follow-up are needed.