منابع مشابه
Flexible trial design in a rare condition
Running clinical trials in relapsed Ewing sarcoma (ES) is challenging due to its rarity. However randomised clinical trials are essential to evaluate the effectiveness of treatments reliably. Four chemotherapy regimens are currently most often used within Europe for relapsed disease based on small single-arm series with different eligibility criteria, short follow up and variable definitions of...
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Background & Importance: Iniencephaly is a rare cranio-cervical deformity. It belongs to neural tube defects. This disorder is characterized by a retroflexion of head, a very short neck, varying degrees of incomplete posterior closure of vertebrae and cervical and thoracic spinal synostosis. This malformation is associated with central nervous system and systemic malformations. Most o...
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Neuroblastoma is a common tumor in pediatric cancer and which might present with usual or unusual clinical features. One of the rare clinical finding of neuroblastoma is paraneoplastic syndromes. We report a two year-old girl with presentation of bilateral ptosis and abdominal pain which was diagnosed as having neuroblastoma. Our case is a very rare case of neuroblastoma accompanied by bilat...
متن کاملA three-stage clinical trial design for rare disorders.
Many clinical trials of uncommon diseases are underpowered because of the difficulty of recruiting adequate numbers of subjects. We propose a clinical trial design with improved statistical power compared to the traditional randomized trial for use in clinical trials of rare diseases. The three-stage clinical trial design consists of an initial randomized placebo-controlled stage, a randomized ...
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A total of 298 children with a history of adverse reactions in connection with oral penicillin treatment were investigated with a radioallergosorbent test for penicillin metabolites, the skin prick test, and oral challenge with penicillin V. No severe reactions were seen. In 30 (10%) of the subjects slight to moderate skin reactions were observed on the seventh to 10th day of the challenge peri...
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ژورنال
عنوان ژورنال: Trials
سال: 2013
ISSN: 1745-6215
DOI: 10.1186/1745-6215-14-s1-p24