Allogeneic Hematopoietic Stem Cell Transplantation in Juvenile Myelomonocytic Leukemia / 소아과

PURPOSE: The purpose of this study was to evaluate the outcome of children with juvenile myelomonocytic leukemia(JMML) treated with allogeneic hematopoietic stem cell transplantation(allo- HSCT). METHODS: Eleven JMML patients aged 8-39 months underwent allo-HSCT. The sources of grafts were unrelated donors(n=7), HLA-matched siblings(n=3) and an HLA 1-antigen mismatched familial donor. All patients had received chemotherapy +/- 13-cis-retinoic acid(CRA) before transplant, and CRA was used, posttransplant, in six patients. RESULTS: Only three patients were in complete remission(CR) at the time of transplantation. Initial chimeric status revealed complete donor chimerism(CC) in five patients, mixed chimerism(MC) in five and autologous recovery(AR) in one. One patient with MC having persistent splenomegaly eventually turned to CC and CR after rapid tapering of cyclosporine, combined with daily use of CRA. An AR case relapsed shortly after transplant but was rescued with second, unrelated cord blood transplantation. Ultimately, six patients are alive, event-free, with a median follow-up of 15.5 months posttransplant. All three deaths occurred in patients who failed to achieve CC, leading to disease progression. CONCLUSION: We suggest that graft-versus-leukemia effect play an important role and CRA a possible role in posttransplant leukemic involution in JMML. In patients whose leukemic burden is still high with MC after transplant, early tapering of immunosuppressants and introduction of CRA might provide a chance of a cure for some patients.

Responses and Toxicities of Risk-adapted Chemotherapy in Pediatric Intracranial Germ Cell Tumors / 소아과

PURPOSE: The purpose of this study was to evaluate the responses and toxicities of risk-adapted chemotherapy in pediatric intracranial germ cell tumors(IC-GCT). METHODS: Fourteen patients who were diagnosed as IC-GCT from October 2002 to December 2003 received chemotherapy as an initial treatment modality. The low risk(LR) group was defined as follows: Pure germinoma and normal AFP level. Beta-hCG level 50 mIU/mL or less. The others belonged to the high risk(HR) group. Chemotherapy was composed of cisplatin, cyclophosphamide, etoposide and vincristine. Double doses of cisplatin and cyclophosphamide was used in HR patients. RESULTS: Pathologic confirmation was done in all but one. Median age at diagnosis was 11.6 yr (1.2-18.7 yr), and nine patients belonged to the HR group. Tumor markers were normalized after chemotherapy in all patients whose tumor markers had been elevated. Four LR patients(80 percent) and seven HR patients(77.8 percent) showed complete response(CR) at the end of chemotherapy. An additional two of the three patients with partial response(PR) achieved CR after radiation therapy (RT), and the remaining one relapsed before RT. Four LR and all HR patients experienced infectious episodes that required hospitalization. Four of the nine HR patients(44.4 percent) suffered from tinnitus, three of whom developed sensorineural hearing loss. All but one are surviving, event-free, with a median follow-up of 13.9 mo(8.1-22.3 mo). CONCLUSION: Risk-adapted cisplatin-based chemotherapy was effective even in HR patients, but regimen modification seems to be necessary to avoid an unacceptably high toxicity rate.

Analysis of the Etiologies, Radiologic Findings, Bronchoscopic Findings, and Clinical Courses of Right Middle Lobe Syndrome in Children / 소아알레르기및호흡기학회지

PURPOSE: Right middle lobe syndrome is defined as chronic atelectasis of the middle lobe of the right lung. The purpose of this study was to analyze the etiologies, radiologic findings, bronchoscopic findings, and clinical manifestations of right middle lobe syndrome in children. METHODS: We retrospectively reviewed the medical records of 28 children, who were admitted to the Samsung Medical Center from June 1998 to January 2003. These children had persistent atelectasis in the right middle lobe in plain chest radiography for more than a month. RESULTS: In 28 children, the most common etiology was pneumonia, followed by tuberculosis, bronchiectasis, and asthma. Most of the patients manifested nonspecific respiratory symptoms, such as coughing. The computerized tomography showed various findings including atelectasis, air bronchogram, or bronchietasis. While normal patent airway was found in 50% of the patients by bronchoscopy, narrowing of bronchus, large amount of secretion, and granulation nodules were noted in another half of the patients. In comparison with tuberculosis, atelectasis caused by pneumonia was relived more frequently by bronchoscopic therapeutic intervention (P=0.008), but there was no significant difference between them after approximately 2 years of follow-up. (P=0.232) Final outcomes in patients whose duration of atelectasis was 2 months or less tended to be better than 12 months or more, but it was not statistically significant. (P= 0.067) CONCLUSION: Common causes of right middle lobe syndrome in Korean children are pneumonia and tuberculosis. A high index of suspicion is required for early diagnosis and proper treatment which leading to better outcomes.

The Comparison of Severity according to Preceding Causes of Bronchopulmonary Dysplasia in Very Low Birth Weight Infants

PURPOSE: This report attempts to reveal the incidence and prevalence of bronchopulmonary dysplasia (BPD) and compare the severity according to preceding causes of BPD in very low birth weight (VLBW) infants. METHOD: Retrospective study was done on 293 VLBW infants who were born and admitted to neonatal intensive care unit in Samsung medical center between October, 1995 and December, 2001. Classical BPD was defined as oxygen dependency at 36 week's postmenstrual age (PMA). Ogawa BPD was defined as oxygen dependency at 28 days after birth, with respiratory distress symptoms and the change on chest X-ray finding. This classification further classified as BPD into 5 subtypes by the presence of respiratory distress syndrome (RDS), pathologic chorioamnionitis and the type of chest X-ray finding. BPD by Jobe and Bancalari was defined as oxygen dependency at 28 days after birth and classified as 3 subtypes (severe, moderate, mild) by the severity of oxygen dependency. Comparisons were made among classifications. RESULTS: Classical BPD infants were 56 (19.1%), Ogawa BPD infants were 76 (25.9 %), BPD by Jobe and Bancalari infants were 124 (42.3%). In Ogawa classification, Infants with RDS and the change on chest X-ray were 58 infants (76.4%). There was no statistical difference of mortality between each type of Ogawa BPD. In classification by Jobe and Bancalari, 35 infants (28.2%) belonged to severe BPD and 75 infants (60.5%) belonged to mild BPD. The mortality was highest in severe BPD infants but there was no statistical difference after correction by birth weight. There was no statistical correlation between Ogawa classification and classification by Jobe and Bancalari. CONCLUSION: There was no statistical difference in mortality or severity between each subtype of classifications according to the severity or preceding cause of BPD in very low birth weight infants.

A Study on the Incidence and Risk factors of Cystic Periventricular Leukomalacia in very Low Birth Weight Infants

PURPOSE: Our study was carried out to estimate the incidence of cystic periventricular leukomalacia (CPVL) and to identify the risk factors for CPVL. METHODS: The medical records and cranial ultrasound scan were reviewed for 321 infants weighing less than 1, 500 g who lived more than 28 days and admitted to the NICU at Samsung Medical Center from October 1995 to December 2001. A multiple logistic regression was performed to identify which factors were independently associated with CPVL. RESULT: CPVL developed in 19 (5.9%) infants of 1, 188+/-236 g birth weight and 28(+6)+/-2(+4) weeks gestational age. Incidence of CPVL according to birth weight and gestational age were as follows respectively: <750 g 5.3%, 750-999 g 5.5%, 1, 000-1, 249 g 3.9%, 1, 250-1, 499 g 7.9% and <25weeks 8.3%, 25-26weeks 6.7%, 27-28weeks 6.5%, 29-30weeks 2.7%, 31-32weeks 11.1%. The mean day of diagnosis of CPVL was 41+/-33 days. Univriate analysis indicate that two clinical variables, prolonged ventilator duration (CPVL: control, 35+/-64 days vs 17+/-26 days, P=0.0184) and severe intraventricular hemorrhage (IVH) (21% vs 2.7%, P=0.0324), were significant predictors of CPVL. The odds ratio estimate and 95% confidence limits are 1.012 and 1.003 to 1.022, respectively for prolonged ventilator duration; 2.6 and 1.044 to 6.602, respectively for severe IVH. CONCLUSIONS: These data suggest that prolonged ventilator duration and severe IVH increase the risk for development of CPVL.

Clinical Characteristics and Treatment Outcome of Pediatric Patients with Burkitt Lymphoma / 대한소아혈액종양학회지

PURPOSE: Burkitt lymphoma (BL) occurs mainly in pediatric populations. Data on the clinical characteristics and treatment results are scarce in Korea. We report our single center experience on BL in children to improve the treatment efficacy while minimizing treatment-related toxicities. METHODS: We undertook a retrospective analysis of 15 patients diagnosed as BL or Burkitt leukemia-lymphoma (BLL) between Aug., 1995 and Feb., 2002. Several induction chemotherapy regimens were used including CCG 106B (prednisolone, cyclophosphamide, daunorubicin, vincristine, L-asparaginase; N=10). Post-induction regimens consisted of CCG 106B (N=12), high dose chemotherapy and autologous stem cell transplantation (N=1), and others (N= 2). RESULTS: The incidence of BL and BLL was 27.2% of Non-Hodgkin's lymphoma diagnosed at our institution. Abdominal mass was the most common presentation (80%) and many patients had advanced stage diseases. Six patients suffered from tumor lysis syndrome, all of whom eventually improved. None died from infection or bleeding. All patients are alive disease-free for median 20 months (range 2~26 months) of follow-up duration except for one who is alive with a residual liver mass. CONCLUSION: Though recent therapeutic trials of repeated intensified chemotherapy including high dose cytarabine and methotrexate led to improvement of survival in patients with BL, many patients suffers from therapy-related toxicities. We successfully treated pediatric BL patients with tolerable toxicities using CCG 106B regimen which is known to be highly effective in high-risk acute lymphoblastic leukemia. More experiences are needed to establish the optimal duration of therapy.