DEEP-3 is a long-term observational safety study which evaluates the nature and incidence of adverse effects of deferiprone (DFP) in children and adolescents with beta-thalassaemia major by conducting a multi-centre, multi-national, observational cohort study with both retrospective (using chart review) and prospective data collection. In addition, incidence of non-serious ADRs and risk factors for ADRs related to DFP treatment are also investigated.
What is the progress of the study that is to date in the last collection rush for most of the centres involved and which are its main progresses and achievements? We talked about them with Assoc. Prof. Antje Neubert from Universitätsklinikum Erlangen (UKER).
“The study has recently successfully received the ethical approval for two new centres in Casablanca (Hôpital 20 août and Hôpital Universitaire d’enfants Abderrahim Harrouchi) and the recruitment started at Centre National de Greffe de Moelle Osseuse (CNGMO) of Tunis. To date we have enrolled 301 paediatric patients (92% of 328 patients) from 16 centres in 6 different countries. All centres put huge efforts into the data collection. This data are sufficient to provide a comprehensive safety profile of deferiprone treatment in children and adolescents within our defined sample size and set confidence interval.”
With reference to how the long-term study results will increase deferiprone post-marketing evidence, Prof. Neubert pointed out that “the latest product information of deferiprone states that insufficient data on safety and efficacy in children below 10 years of age are available at the moment. The results of the long-term observational safety study DEEP-3 will close this gap and provide valuable safety data for the treatment of transfusional iron overload in children and adolescents with beta-thalassaemia major“. She also explained that adverse reactions will be more predictable and monitoring for known adverse reactions in children will make the treatment safer in general.
Considering that the prospective arm of the study has been concluded in October 2015, while retrospective data will be collected until termination of the study with reference to the same period (up to October 2015), she further clarified that “data collection is still on-going, but in 286 patients we already have the complete data set. Our participating investigators are doing their best collating all the requested clinical data. Nearly 60% of our enrolled patients started deferiprone treatment before the age of 10 years. This gives us good confidence that the generated safety data adds to the existing data from other clinical trials and observational studies. So far we collected about 423 adverse events which may or may not be related to deferiprone therapy. Of those, our adverse reaction assessment team already evaluated 134 events including 49 serious adverse events. The team found that 42 adverse reactions and 20 serious adverse reactions related to deferiprone treatment were found. These reactions – she continued – comprise elevated liver enzymes, arthropathy and mild to moderate neutropenia. However, these are only very early results and we need to wait for the final results before drawing any conclusions about the safety profile of deferiprone in children.”