Klinische Pädiatrie
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Klinische Pädiatrie · Jul 1999
Clinical Trial[Neoadjuvant therapy for localized osteosarcoma of extremities. Results from the Cooperative osteosarcoma study group COSS of 925 patients].
Owing to twenty years of multicentric interdisciplinary cooperation, the COSS group has been able to collect data on a large group of osteosarcoma patients treated by neoadjuvant therapy. This paper reviews results achieved in patients with localized extremity tumors. ⋯ Intensive multiagent chemotherapy and delayed surgery for localized extremity osteosarcoma led to excellent oncologic results in the COSS-studies. Tumor-size, -site, and -response as well as the intensity of upfront chemotherapy correlated with outcome. Giving doxorubicin and cisplatin by continuous infusions did not result in discernible prognostic disadvantages.
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Currently, several clinical studies explore the therapeutic potential of tumor vaccines which are genetically modified to produce immunostimulatory molecules as a complementary approach for conventional cancer therapy. In this review the immunological basis and the preclinical design of such vaccine strategies are described with particular emphasis to acute leukemia and neuroblastoma. The role of cytokines, chemokines and costimulatory surface molecules for generation of tumor vaccines is summarized, and the advantages and disadvantages of autologous, allogenic and dendritic cell vaccines are discussed. Finally, combination-immunogens are introduced as a potent means of enhancing the anti-tumor response.
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Klinische Pädiatrie · Jul 1999
Multicenter Study Comparative StudyCerebral venous sinus thrombosis in children with acute lymphoblastic leukemia carrying the MTHFR TT677 genotype and further prothrombotic risk factors.
The present study was designed to prospectively evaluate the role of prothrombotic risk factors in leukemic children treated according to the ALL-BFM 90/95 study protocols with respect to the onset of cerebral venous sinus thrombosis. ⋯ Prothrombotic risk factors should be included in a screening program in ALL children treated according to the BFM study protocols. Further prospective studies are recommended to establish adequate prophylactic anticoagulant treatment during ALL (BFM) polychemotherapy.
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Klinische Pädiatrie · Jul 1999
ReviewFanconi anemia and beta c deficiency-associated pulmonary alveolar proteinosis as two hereditary diseases of childhood which are potentially curable by stem cell gene therapy but require different therapeutic approaches.
Fanconi anemia (FANC) and pulmonary alveolar proteinosis associated with deficiency of the beta-chain common to the GM-CSF/IL3/IL5 receptors (beta c-PAP) are rare inherited disorders of childhood or adolescence. Hematopoietic stem cell gene therapy aiming at reintroducing the wildtype cDNA as a new concept for the treatment of hereditary diseases may be applicable to FANC and PAP, as both disorders can be successfully treated by allogeneic stem cell transplantation. However, there are important distinctions to be made between the two diseases: FANC seems to be a disorder with functional stem cell deficiency. ⋯ The introduction of a second selectable cDNA into the vector might be used to provide selective growth for modified cells and thus overcome a low gene transfer efficiency of stem cells. The correction of rare monogenetic diseases may serve as a model for gene therapy prior to attempts to treat more common and complex polygenetic diseases. The studies outlined here will be helpful envisioning new treatment strategies for other inherited monogenetic diseases such as mucopolysaccharidosis, Gauchers disease or adrenoleukodystrophy.
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Klinische Pädiatrie · Jul 1999
Multicenter Study Clinical Trial[High-dosage chemotherapy in primary metastasized and relapsed Ewing's sarcoma. (EI)CESS].
Patients (pts) with primary metastatic Ewing tumours (ET) have a poor prognosis for event free survival (EFS) compared to pts with localised disease. Following relapse the prognosis is extremely poor. Therefore these primary metastatic and relapsed pts were piloted for high dose therapy (HDT) for the last years. ⋯ The total group of primary metastatic ET pts showed no obvious benefit from HDT, based on melphalan and/or etoposide. Pts with metastases to multiple organ systems, and early relapse seemed to benefit from HDT. The value of HDT should be assessed in prospective clinical trials.