Table 4 Burden of CTT

Impact on daily life and family

Work: Families report increased planning needs around work and challenges with having to miss work. Several describe challenges and increased demands of coordinating their schedules, and some report switching to night shifts to accommodate appointments, or having had to stop working.

School: Patient and parents reported missing school, and distress associated with missing school, schoolwork, tests and school events, and having to make up schoolwork. Some adolescents reported feeling ‘frustrated’ or, ‘mad’ about missing school. A few parents reported concerns about support from school.

Family: Parents report challenges with care for other children and family members, and increased planning/care-coordination needs.

Transportation: Parents and patients report challenges with long commute times, traffic, parking, and difficulties with transportation for families without a personal vehicle

“Because like I said I’m a single parent. So I have to work, so there’s not a lot of lee-way sometimes. Now we’re down to six employees and I had to take off today. I was so scared to call my supervisor today because, my God, I had to.” (Parent)

“So he’s out Friday and today, which means he’s behind on somethings. He’s already struggling to do what he’s already doing. And this puts us even farther behind and a little more challenging. Is our biggest challenge with him, is being behind.” (Parent)

“It’s kind of tough, I guess, if you’re learning. I mean it depends, cause if the teacher taught something new the day you’re out it’s kind of difficult to pick up on.” (Patient)

Pain and distress with venous access

Most patients and parents report pain, emotional distress, or fear associated with obtaining venous access. Some also report difficulty with obtaining venous access, need for central venous access and complications due to central venous access, and two adolescents commented on physical appearance of port.

“And it’s kind of hard seeing your child, your little child in so much pain, you know, getting stuck. You know a lot of the times when they’re small they can’t find the veins. You know, I’ve watched them stick over and over and over again, you know, veins collapse. It’s, it’s just really a test. It really is. It’s a lot to go through.” (Parent)

“Uh when I was younger they had people strap me down because I didn’t like needles, and I was terrified. And plus I couldn’t bear the pain.” (Patient)

Emotional distress and worry surrounding CTT

Parent participants expressed feelings of emotional distress surrounding their child receiving CTT, including feelings of stress and worry. Many parent participants specifically have uncertainty and worry about possible complications of CTT including iron overload, chelation, infection, organ damage, and unpredictable future events. Patient participants did not describe feelings of concern about potential future effects of transfusions.

“It’s been a tough journey, its just a lot. For him to go through, for me to see him go through” (Parent)

“Um, I don’t know, cause we still like, we haven’t really figured that out, like how that’s gonna affect her body. So hopefully, Dr. *** said they can do an MRI of her liver or whatever. But um, I’m hoping for the best. That’s something that I’m fearful, very fearful about right now because I don’t want anything else like wrong with her. You know, especially something like that. So um, I’m hoping that’s gonna be okay. Praying.” (Parent)

Burden of Chelation

The majority of patient and parent participants report challenges with taking chelation medications. Some of the challenges described include unpleasant taste of the powder forms of chelation medications, difficulty with remembering to take medications, distress with having to take multiple medications, and side effects.

“He doesn’t like to look at them. Like he has the pill case. He covers it up on his dresser, and that bothers me for some reason because you have to take it. I don’t know why you don’t want to see it. He says he doesn’t want to see it.” (Parent)

“And I hated it so much. Cause like, you have to mix it in your drinks and drink. And just does not feel good going down.” (Patient)