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Table 2 Motivating factors for gene therapy

From: Decision-making about gene therapy in transfusion dependent thalassemia


Key Quotes


 Transfusion independence

‘Well, primary motivating factor for me would probably be to be uh, just transfusion independent so, so I don't have to deal with all the medical bills and the uh, stresses of uh, going to the doctor so much. And I don't know… just think it'd be nice to live my life without thalassemia’ [Patient]

‘I say his wellbeing, where he doesn’t have to do this anymore. He doesn’t have to rely on a system to take care of him and he’ll be able to take care of himself because this is a huge system that helps him to stay alive’ [Parent]

‘I’m uh, been doing this over 50 years and it would be nice to retire. You know, if I put in my time and it would be nice to somewhat retire my uh, efforts, you know, my transfusions, and uh, regimen and at the very least, if it were a couple of times a year that would be uh, you know, reduce the amount of times that I’m having to go to uh, get that done, would be a nice switch’ [Patient]

 Healthcare Utilization

‘it would be nice to not uh, um, occupy so much time in the ….you know doctor’s office and getting treatments and being able to spend more time with my family’ [Patient]

‘if I have less frequent visits in a year with my son once he’s cured or his frequency of coming into the hospital for transfusion is reduced then that would also be a great thing for me’ [Parent]

 Iron overload and chelation

‘our main motivator would be that, eventually, hopefully, she wouldn’t have to take the chelators’ [parent]

‘but chelation is certainly an um… a daily uh… on its own schedule. I don’t know the right word for it, but it, fairly minor compared to a blood transfusion, but it’s something that has to be dealt with everyday versus once every 2 to 4 weeks, so that’s definitely a factor’ [parent]


‘If you can get a child to become transfusion-free, then it opens up a whole other world that they otherwise wouldn’t have had. You know, he can, if this works, he can go to college and not have to be pinned down to only going to a college where there’s a nearby place for him to get blood. He won’t have to worry about how he would handle, you know, six months overseas while he’s in college as, you know, trying to do something cool like other kids do because he’s not sure how insurance and all that’s going to work in a foreign country’ [parent]

As a parent I am kind of um, stuck to uh, to some of the roles or some of the positions or some of the cities where I can get her uh, the transfusion done ……….., so I can’t explore a lot of career options for myself, uhm, because of uh, her medical condition. [parent]


‘I have really good insurance, so it pays for his….. But after he’s 26, he’ll have to foot the bill for this his self, and I don’t know what that looks like for him. [parent]