Matched-unrelated-donor bone marrow transplantation for children with leukemia.

BACKGROUND AND PURPOSE: This report describes the results of matched-unrelated-donor transplant for leukemia or myelodysplasia in the first 23 recipient children at a single medical center in Taiwan. METHODS: Between August 1994 and February 2003, 23 consecutive children with leukemia or myelodysplasia underwent matched-unrelated-donor bone marrow transplantation (BMT). The preparative regimen consisted of fractionated total body irradiation and cyclophosphamide in 6 patients; busulfan in combination with etoposide and cyclophosphamide in 4 patients who received cranial irradiation before transplantation; and busulfan and cyclophosphamide in 13 patients. RESULTS: Engraftment was achieved in 91.3% of cases. Acute graft-versus-host disease (GVHD) occurred in 18 of 21 patients who engrafted (85.7%). Event-free survival for all patients was 24.46 +/- 9.24%. The 12 children with standard-risk disease had better event-free survival than the 11 children with high-risk disease (46.88 +/- 15.03% vs 0%, p < or = 0.001). CONCLUSIONS: The major obstacles to successful matched-unrelated-donor BMT are acute GVHD, relapse and infection. Early transplantation and patient selection, prophylactic and therapeutic maneuvers for GVHD, as well as appropriate donor selection and virus prophylaxis may improve the results.     

Ureteral obstruction caused by L-asparaginase-induced pancreatitis in a child with acute lymphoblastic leukemia.

L-asparaginase, an effective antileukemia and antilymphoma agent, is toxic to many organ systems. We report a case of ureteral obstruction caused by L-asparaginase via the inflammatory complication of acute pancreatitis. The patient was an 11-year-old boy with acute lymphoblastic leukemia. Six days after completing a 4-week induction therapy containing 9 doses of L-asparaginase, severe left abdominal pain developed. Abdominal computed tomography showed phlegmon formation anterior to the pancreatic head and in the left posterior pararenal space. The strands of inflammatory soft tissues encased the upper third of the left ureter, causing left hydroureter and left hydronephrosis. The ureteral obstruction resolved after insertion of a double-J catheter that remained in place for 66 days. This case suggests that L-asparaginase may play a role in the pathogenesis of ureteral obstruction in children receiving chemotherapy.     

Disseminated tuberculosis presenting as multiple hepatosplenic microabscesses and pancytopenia in a teenage boy.

Disseminated tuberculosis usually occurs in immunocompromised hosts. Involvement of bone marrow, liver, and spleen is infrequent. A previously healthy 15-year-old boy presented with body weight loss, prolonged fever, neck lymphadenopathy, pancytopenia, and hepatosplenic microabscesses within the recent month and was transferred to our hospital. Bone marrow studies showed hypocellular marrow. Based on his clinical manifestations, hemophagocytic syndrome was initially suspected. Pancytopenia resolved after administration of intravenous immunoglobulin but caseous necrosis and/or positive acid-fast stain were subsequently demonstrated in the lymph node biopsy and sputum. Cultures from these 2 specimens grew Mycobacterium tuberculosis. Fever continued in a low-grade pattern even under antituberculous therapy with rifampin, isoniazid, pyrazinamide and ethambutol. Five months after admission, fever subsided after splenectomy and liver wedge resection. Microscopic examinations of both the liver and spleen showed mycobacteria-related granulomatous inflammation and caseating necrosis. This report suggests that tuberculosis infection should be considered in the differential diagnosis in patients with prolonged fever, pancytopenia and hepatosplenic abscesses.     

The design and content validity of an infection control health education program for adolescents with cancer

The purpose of this article is to describe the process of designing an Infection Control Health Education Program (ICP) for adolescents with cancer, to describe the content of that program, and to evaluate its validity. The program consisted of an audiovisual "Infection Control Health Education Program in Video Compact Disc (VCD)" and "Self-Care Daily Checklist (SCDC)". The VCD was developed from systematic literature reviews and consultations with experts in pediatric oncology care. It addresses the main issues of infection control among adolescents. The content of the SCDC was designed to enhance adolescents' self-care capabilities by means of twice daily self-recording. The response format for content validity of the VCD and SCDC was a 5-point Likert scale. The mean score for content validity was 4.72 for the VCD and 4.82 for the SCDC. The percentage of expert agreement was 99% for the VCD and 98% for the SCDC. In summary, the VCD was effective in improving adolescents' capacity for self-care and the extensive reinforcement SCDC was also shown to be useful. In a subsequent pilot study, the authors used this program to increase adolescent cancer patients' self-care knowledge and behavior for, and decrease their levels of secondary infection.     

A trend of improved survival of childhood hepatoblastoma treated with cisplatin and doxorubicin in Taiwanese children

Hepatoblastoma is the second most common childhood malignant hepatic tumor in Taiwan. Its prognosis used to be poor. We reviewed our cases in this decade to see if there has been any improvement of survival in our patients with hepatoblastoma. From 1988 to 2000, 19 patients with hepatoblastoma in this institution were included in the study. These patients clinical manifestations, laboratory and image studies, histological findings, treatment modalities and prognostic significance were analyzed. The mean age at diagnosis was 13.5 months, ranging from 0 to 4 years old (male:female =11:8). Abdominal distension was the most common symptom, and hepatomegaly was the most common physical finding. Laboratory abnormalities included elevated alpha-fetoprotein, thrombocytosis and abnormal liver function profiles. Treatment modalities included primary surgery with postoperative chemotherapy in three, chemotherapy only in four and preoperative chemotherapy plus surgery with or without postoperative chemotherapy in nine patients. The overall 2-year survival rate is 38.6%. The significant prognostic factors include patients compliance, resectability and chemotherapy protocol. The introduction of a new chemotherapy protocol designed by the International Society of Pediatric Oncology Study (SIOPEL) in 1994 improved the 2-year survival rate from 12.5 to 58.4% (P=0.01). In conclusion, the improved chemotherapy protocol enhances the survival rate of hepatoblastoma in Taiwanese children.     

Post-hepatitic aplastic anaemia in children in Taiwan, a hepatitis prevalent area

Aplastic anaemia is a rare but serious complication of hepatitis, and hepatitis is an unusual cause of aplastic anaemia in children in the West. However, the relative frequencies of acquired aplastic anaemia in children in Taiwan, a hepatitis prevalent area, differ from those in the West, in the very high frequency of post-hepatitic aplastic anaemia (23.9% of all cases of aplastic anaemia). This may account for the higher incidence of aplastic anaemia in children in Taiwan. Although the prognosis of post-hepatitic severe aplastic anaemia was very poor, the present study using bone marrow transplantation, antithymocyte (or antilymphocyte) globulin, high-dose methylprednisolone and cyclosporin, etc., has improved the response rate and the survival.     

Acute lymphoblastic leukemia occurring as a second malignant neoplasm in a child.

The effective treatment of childhood malignancies has increased the importance of early detection and treatment of second malignant neoplasms. Anticancer drugs may also be leukemogenic agents, by the same mechanisms that kill cancer cells. We report the case of a 10-year-old boy who had received radiotherapy and chemotherapy for the treatment of Ewing's sarcoma and developed acute lymphoblastic leukemia 22 months after the diagnosis of primary malignancy. Although chemotherapy is well known to potentiate the development of second acute nonlymphocytic leukemia, the pathogenic factors leading to second acute lymphoblastic leukemia remain obscure.     

Hematopoietic stem cell transplantation in Taiwanese children with primary immunodeficiency.

BACKGROUND AND PURPOSE: Since 1968 it has been known that hematopoietic stem cell transplantation (HSCT) can ameliorate primary immunodeficiencies, but data on the long-term efficacy of this treatment in Taiwan are limited. This study analyzed the outcome of HSCT and the immune reconstruction in 10 children with primary immunodeficiencies in Taiwan. METHODS: We retrospectively analyzed the outcome of HSCT in 10 children with primary immunodeficiencies between 1986 and 2002. The primary immunodeficiencies in these children included severe combined immunodeficiency (SCID) in 4, Wiskott-Aldrich syndrome (WAS) in 4, Chediak-Higashi syndrome (CHS) in 1, and leukocyte adhesion deficiency (LAD) in 1. The conditioning protocols included busulfan and cyclophosphamide in 2 patients with SCID, 3 patients with WAS, 1 patient with LAD and 1 patient with CHS. Anti-thymocyte globulin was given to only 1 patient with WAS and no conditioning therapy was given in 2 SCID patients. Graft-versus-host disease (GVHD) prophylaxis with cyclosporine (CsA) and methotrexate was prescribed in 6 children, CsA alone in 1, and CsA and T-cell depletion in 1. RESULTS: Six patients were cured and 1 improved during a follow-up period from 3.5 years to 13 years after transplantation. Three patients died of severe sepsis. Three patients developed acute GVHD, which was grade 2 in 2 patients, and grade 3 in 1. Veno-occlusive disease developed in 1 patient and chronic GVHD with contracture of joints in 1. CONCLUSIONS: Our results support the benefits of allo-HSCT in children with primary immunodeficiencies. However, HSCT should be performed as soon as possible before severe infection develops.     

Unstratified chemotherapy for non-metastatic osteosarcoma of the extremities in children.

BACKGROUND AND PURPOSE: The superiority of changing postoperative chemotherapy of osteosarcoma based on histological response of the primary tumor over non-tailored chemotherapy has not been confirmed. This multicenter study evaluated the effectiveness of an intensive unstratified chemotherapy regimen in Taiwanese children with osteosarcoma. METHODS: Fifty patients younger than 18 years of age with previously untreated non-metastatic osteosarcoma of the extremities were enrolled. Patients were treated with pre- and postoperative chemotherapy, and surgery. Definitive surgery was scheduled in week 7 and postoperative chemotherapy was uniform without stratification regardless of histologic response. RESULTS: Chemotherapy toxicities were considerable, but manageable. Treatment delay and decreased dose-intensity were common. There was one treatment-related mortality. Forty three patients (86%) received limb salvage surgery and 14 patients (33%) had a good histologic response to preoperative chemotherapy. With a median follow-up of 47.1 months, the 7-year event-free and overall survival rates were 51.6% and 67.6%, respectively. CONCLUSIONS: This was the first multicenter study on the treatment of osteosarcoma from Taiwan. The results suggest that a non-tailored regimen may serve as an alternative treatment strategy in the management of osteosarcoma, particularly when histologic assessment of the tumor response is not available.     

Clinical features, prognostic factors, and their relationship with antiplatelet antibodies in children with immune thrombocytopenia

We investigated and evaluated the demographics, clinical and laboratory features, treatment responses, and disease duration of 25 children with immune thrombocytopenia (ITP) eligible for detection of antiplatelet antibodies. We found that patients without antecedent of preceding infection (API) were more likely to have anti-GPIa/IIa than those with API (42.9% vs. 5.5%, P=0.048). Age groups of <2 years and 2 to 10 years were more likely to show response (R) or complete response (CR) to given treatments, whereas none of the patients whose onset age >10 years showed R or CR to given treatments (88.9% and 100% vs 0%, P=0.001). The percentage of newly diagnosed ITP was higher in age groups of <2 years (100%) and in 2 to 10 years (90%) than the age group of >10 years (16.7%, P=0.001). Patients without API (71.4%) were more likely to develop chronic ITP than those with API (5.6%, P=0.002). In conclusion, younger age was a favorable prognostic factor, especially in patients <2 years of age with respect to treatment responses and disease duration. In addition, API was associated with a short disease course as well as absence of anti-GPIa/IIa.