Question, @PaolaSaroufim and I were wondering how a gene therapy treatment like Strimvelis might be represented in the OMOP Vocabulary. Would we consider Strimbelis a drug exposure correct?
The treatment is personalized for each patient; hematopoietic stem cell (HSCs) are extracted from the patient and purified so that only CD34-expressing cells remain. Those cells are cultured with cytokines and growth factors and then transduced with a gammaretrovirus containing the human adenosine deaminase gene and then reinfused into the patient. These cells take root in the person’s bone marrow, replicating and creating cells that mature and create normally functioning adenosine deaminase protein, resolving the problem. As of April 2016, the transduced cells had a shelf life of about six hours.
This came up because there is an AE listed for it in the European Consortium (PROTECT), Adverse Drug Reactions Database and we were trying to map it to RxNorm for CEM.