Results from expanded access programs: a review of academic literature 165 7♦ Several limitations to the study are mentioned: ‘There was no protocol for this program and all reporting of patient data and outcomes was voluntary, without on-site monitoring. As such, in some cases, outcome forms were either not returned or not fully completed for all patients, which may have resulted in under-reporting of AEs that did not lead to death. In addition, the relatively shorter recommended duration of treatment in this program, compared with more recent studies also may have influenced efficacy and safety outcomes.’ Conclusion The authors conclude that: ‘For patients in the CUP (…) the survival rate (…) is consistent with prior studies of defibrotide. The overall profile of serious and fatal events in this large study population of more than 700 patients was consistent with what has been observed in other studies of defibrotide for the treatment of VOD/SOS and was consistent with the manageable toxicities seen with defibrotide use in this setting.’ As defibrotide received marketing approval at the time of writing, the authors give no advice on future conduct of trials.
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