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  • 1
    Keywords: tumor ; Germany ; THERAPY ; CT ; imaging ; INFORMATION ; TOOL ; VISUALIZATION ; VOLUME ; NEW-YORK ; DIFFERENTIATION ; TUMORS ; computed tomography ; NUCLEAR-MEDICINE ; PATIENT ; kidney ; MRI ; MAGNETIC-RESONANCE ; magnetic resonance imaging ; TRIAL ; TRIALS ; METASTASIS ; chemotherapy ; COMPUTED-TOMOGRAPHY ; CHILDREN ; nuclear medicine ; MANAGEMENT ; PREOPERATIVE CHEMOTHERAPY ; radiology ; ONCOLOGY ; CHILDHOOD ; THERAPIES ; monitoring ; NUCLEAR ; USA ; EXTENT ; CHILD ; pediatric ; BONE METASTASES ; nephroblastoma ; SIOP ; MEDICINE ; three-dimensional ; INTERNATIONAL-SOCIETY ; PARTIAL NEPHRECTOMY ; IMAGING CHARACTERISTICS ; INTRAVASCULAR EXTENSION ; UNILATERAL WILMS-TUMOR
    Abstract: Magnetic resonance imaging (MRI) presents the main diagnostic tool for differentiation and staging of renal tumors in childhood. Nephroblastoma is the most common malignant tumor in children. Radiological findings play an important role in therapy study trials of SIOP (International Society of Pediatric Oncology), especially for indicating preoperative chemotherapy. In the past few years MRI has gained great importance in imaging of nephroblastoma and has replaced computed tomography (CT). The aim of this review is to present the diagnostic possibilities of MRI in relation to the requirements of therapy studies. For nephroblastoma, MRI provides important information about tumor extent and distant metastasis. A special focus of MRI in distant staging is venous extent of the tumor into the inferior vena cava. In addition, MRI has an important role in monitoring chemotherapy and in preoperative planning by volume rendering and three-dimensional postprocessing
    Type of Publication: Journal article published
    PubMed ID: 18193429
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  • 2
    Keywords: CELLS ; EXPRESSION ; Germany ; VOLUME ; DISEASE ; GENES ; PATIENT ; kidney ; ACID ; chemotherapy ; HIGH-RISK ; CHILDREN ; RETINOIC ACID ; signaling ; development ; USA ; nephroblastoma ; WILMS-TUMOR ; pediatrics ; nephroblastomatosis ; von Willebrand disease ; Wilms Tumor
    Abstract: A 9-month-old girl presented with massive bilateral diffuse nephroblastomatosis. After response to actinomycin D and vincristine over a period of 1 year, the nephroblastomatosis continuously progressed under this treatment. As retinoic acid signaling is critical for normal renal development and nephroblastomatosis seems histologically as undifferentiated embryonal tissue, we added 13-cis retinoic acid to the chemotherapy regimen. Three months thereafter, kidney volumes declined significantly over a period of 1 year. Interestingly, nephroblastomatosis-associated acquired von Willebrand disease also resolved. Retinoic acid maybe a novel nontoxic treatment option for nephroblastomatosis requiring further systematic evaluation
    Type of Publication: Journal article published
    PubMed ID: 19346886
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  • 3
    Abstract: Rare diseases are an important public health issue with high unmet need. The introduction of the EU Regulation on orphan medicinal products (OMP) has been successful in stimulating investment in the research and development of OMPs. Despite this advancement, patients do not have universal access to these new medicines. There are many factors that affect OMP uptake, but one of the most important is the difficulty of making pricing and reimbursement (P&R) decisions in rare diseases. Until now, there has been little consensus on the most appropriate assessment criteria, perspective or appraisal process. This paper proposes nine principles to help improve the consistency of OMP P&R assessment in Europe and ensure that value assessment, pricing and funding processes reflect the specificities of rare diseases and contribute to both the sustainability of healthcare systems and the sustainability of innovation in this field. These recommendations are the output of the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL), a collaboration between rare disease experts, patient representatives, academics, health technology assessment (HTA) practitioners, politicians and industry representatives. ORPH-VAL reached its recommendations through careful consideration of existing OMP P&R literature and through a wide consultation with expert stakeholders, including payers, regulators and patients. The principles cover four areas: OMP decision criteria, OMP decision process, OMP sustainable funding systems and European co-ordination. This paper also presents a guide to the core elements of value relevant to OMPs that should be consistently considered in all OMP appraisals. The principles outlined in this paper may be helpful in drawing together an emerging consensus on this topic and identifying areas where consistency in payer approach could be achievable and beneficial. All stakeholders have an obligation to work together to ensure that the promise of OMP's is realised.
    Type of Publication: Journal article published
    PubMed ID: 28283046
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  • 4
    Keywords: tumor ; evaluation ; Germany ; PERFUSION ; CT ; DIAGNOSIS ; imaging ; INFORMATION ; SYSTEM ; VISUALIZATION ; VOLUME ; liver ; SITE ; TISSUE ; TUMORS ; SURGERY ; PATIENT ; IMPACT ; primary ; DYNAMICS ; MR ; MRI ; AGE ; magnetic resonance imaging (MRI) ; CHILDREN ; CHILDHOOD ; monitoring ; SOLID TUMORS ; ENHANCEMENT ; methods ; technique ; CHILD ; AGREEMENT ; pediatric ; nephroblastoma ; MEDIA ; three-dimensional ; 3D imaging ; discussion ; pediatrics ; interactive
    Abstract: Introduction: 3D imaging and surgical planning for the treatment of embryonal tumors using different techniques (CT versus MRI) are presently under discussion. Up to now, the main focus has been on visualizing the anatomy. Contrast medium dynamics have not been taken into consideration. The aim of the present study was to establish the technical means of integrating the 3D images from functional MRI data into the anatomical images and to determine clinical applications for this approach. Material and Methods: In 11 patients (mean age: 2.4 years) with solid tumors, 26 diagnostic MRI examinations were performed for primary diagnosis, treatment monitoring, or as part of the surgical planning. Seven children presented with neuroblastomas, three with Wilms' tumor, and one with advanced bilateral nephroblastomatosis. The MRI data were acquired using a 1.5-T system. For post-processing, we used volume rendering software, including an evaluation of perfusion. By using color-coded parametric images and integrating functional information, perfusion could be visualized and used for interactive surgical planning. Macroscopic and microscopic sections served as the gold standard for assessing tissue viability. Results: We were able to integrate the dynamic data into the anatomical images for all patients. A good agreement was found between the results of surgical planning, including perfusion mapping, with the surgical site, subsequently produced macroscopic sections and the results of random microscopic examinations. Conclusions: Perfusion mapping using color-coded parametric images of pediatric abdominal tumors extends the diagnostic techniques currently available. We provide first proof of the possibility of integrating functional information into 3D MR images in children. Monitoring the treatment of nephroblastoma and surgical planning for pediatric embryonal tumors represent potential applications of this technique
    Type of Publication: Journal article published
    PubMed ID: 18302062
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  • 5
    ISSN: 0013-4686
    Source: Elsevier Journal Backfiles on ScienceDirect 1907 - 2002
    Topics: Chemistry and Pharmacology , Physics
    Type of Medium: Electronic Resource
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