background: the classical supportive therapy for î² -thalassemia major consists of regular blood transfusion, iron-chelation therapy and specific treatment of the related complications. a balance between the maintenance of the highest possible level of haemoglobin and lowest possible level of iron accumulation in heart and liver give the best chance of survival and the best quality of life. met...

Time-to-event endpoints show an increasing popularity in phase II cancer trials. The standard statistical tool for such one-armed survival trials is the one-sample log-rank test. Its distributional properties are commonly derived large sample limit. It however known from literature, that asymptotical approximations suffer when size small. There have already been several attempts to address this...

Increasingly, genetic studies of tumors of the same histologic diagnosis are elucidating subtypes that are distinct with respect to clinical endpoints such as response to treatment and survival. This raises concerns about the efficiency of using the simple log-rank test for analysis of treatment effect on survival in studies of possibly heterogeneous tumors. Furthermore, such studies, designed ...