Klinische Pädiatrie
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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.
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Klinische Pädiatrie · Jul 1999
Case Reports[Thrombocytic alpha-delta-storage-pool-disease: shortening of bleeding time after infusion of 1-desamino-8-D-arginine vasopressin].
The synthetic vasopressin derivate desmopressin (1-desamino-8-D-arginine vasopressin) has been reported to shorten the bleeding time in patients with hemophilia A, von Willebrand's disease and several functional platelet disorders. In addition to substitution of platelets, vasopressin is therefore used to prevent bleeding complications. ⋯ Infusion of desmopressin appears to be effective in shortening bleeding time in thrombocytic storage-pool-disease. Its use could prevent bleeding complications after trauma and surgical interventions and may possibly help to spare the need for platelet concentrates.
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Klinische Pädiatrie · May 1999
Review[Babesiosis--a dangerous infection for splenectomized children and adults].
Babesiosis is a world-wide distributed protozoal zoonosis, and Babesia spp. are the most ubiquitous of the blood parasites of mammals, except the trypanosomes. The tick-transmitted protozoa infect various vertebrate reservoir hosts, like rodents, cattle and horses. Approximately 100 million cattle is exposed to the disease. ⋯ Most of the patients suffer from mild to moderate symptoms, whereas in asplenic individuals a fatal course of the infection prevails. Tick-bites in areas where human babesiosis occurs--mainly parts of Europe and the USA--can result in a deadly illness for persons without a spleen. The effect of treatment in splenectomized patients and when treatment is delayed, still is poor.