Table 5 Preferred treatment modality
From: Decision-making about gene therapy in transfusion dependent thalassemia
Key Quotes | |
|---|---|
Chronic Transfusions | If they were equally available? Um, I mean, I think for right now, it would be transfusions, because – just exactly what we’re doing, because their quality of life is so good right now. [Parent] Um… I mean, it – if it – if we knew that the gene therapy would work for them, um, being double-zero, I think we would probably be, um, more heavily pursuing that. But, um… I – I guess there’s just still some unknowns in our mind of – of – of the kiddos that have double-zero, or zero zero, or however you say it, um, so… For now, we’re hanging out with the transfusions [Parent] So, right now I would probably choose status quo, um, just because I don’t think I have the, I don’t have enough information about gene therapy, I would like to see where the trial goes, before I go down that route, although it is a pretty close second, um, I’m very interested in, in the possibility of gene therapy but I’m not completely there yet, because right now it just isn’t there yet [Adult patient] |
BMT | No, so I would say the only option that I would consider would be a sibling match, or um, someone that comes very close, like 100% or 99% sort of um, match, otherwise, I wouldn’t consider bone marrow, um, it wouldn’t be even be in the table, so then I would wait for gene therapy to prove its success. [Parent] Honestly what would have been the best for me would have been when I was like five or six, my parents just were able to…., like I just have a sibling match donor and if I just got a bone marrow transplant, I think that would have probably been the best option. Um, now, I would say probably, I would prefer, I would prefer a successful gene therapy, assuming all my criteria were met, that leaves the other options that you mentioned [Patient] |
Gene Therapy | Um, I would be between um, gene therapy and a transplant, um, but I might want to wait a little bit and see how um, we’ll like do some research, do some more research about gene therapy and see what I find, but um, to me right now right now that one stacks better in theory because you would be using your own cells [Patient] No, I would consider gene therapy more because I know that there are lots of other risks of rejection, so gene therapy over BMT [Parent] |