Results
Twenty families were interviewed (13 patients, 15 parents; Table 1 ), at which point we achieved thematic saturation. Of the patient interviews, six were completed with their parent, two separate from their parent, and five without a corresponding parent interview. Of the parent interviews, seven were completed without a corresponding patient interview. Ten patients were treated at SCH, six at SJ, and four at the NCI. The median age of the patient participant at time of enrollment was 16.5 years (range: 8-24). Patients predominantly identified as female (65%) and white (80%). Most patients received CAR T cell therapy for treatment of ALL (80%). The parent participant median age was 47 years (range: 31-54). Parents predominantly identified as female (93%) and white (93%). All interview participants spoke English as a primary language. Three global themes summarize the family-centered experience with CAR T cell therapy: “A clear choice,” “Coping with symptoms,” and “Unforeseen psychosocial challenges.” (Table 2 ). Responses from the patient perspective did not significantly differ thematically from the parent perspective.
A clear choice
Deciding to pursue CAR T cell therapy was described as a “clear choice” by all families. Two decision-making paths were cited. Most commonly (n=13), families felt that CAR T cell therapy was unequivocally “the next best step.” For most, this reflected recommendations for the best chance for cure. Other families described CAR T cell therapy as the best choice to buy time until another treatment or to extend their child’s life. Alternatively, five families felt they reached the “end of a road” and were faced with no other options. One patient explained, “It was really the only thing I had left, after 12 rounds of chemo, radiation, and a bone marrow transplant not working” (CTP041). Families also described toxicities from prior therapies leaving few other safe options, including ineligibility for otherwise standard therapies, such as stem cell transplant. One parent shared, “There’s no way his body could’ve handled such large doses to do that again, to get him into remission, to get him to transplant. There’s just no way” (CTP051P). Of the remaining two families, one chose CAR T cell therapy for quality of life and the other to avoid toxicity. Factors facilitating the family decision-making process were organized into three themes: hope, trust, and self-efficacy.
Regardless of the rationale described, expressions of hope were pervasive when families reflected upon their decision-making process. Primarily, this took the shape of optimism in CAR T cell therapy for cure. Hope for comfort and minimal toxicity was also common. For example, one parent shared, “We really want what’s best for her, what was her best shot, and what will be the best way for her to be comfortable” (CTP055P). Hope for more time was also expressed. One parent explained, “That’s my goal is just to keep him going as long as possible in the hope that somebody figures it out.” (CTP029P).
Families expressed tremendous trust in their medical teams when reflecting on their decision-making process. Most families disclosed that they avoided the internet or social connections for information, but rather turned to their primary oncologist. One patient shared, “I mean I have all the trust in the world in my Oncology doctor, and so I told my mom from the beginning, whatever <Doctor> suggested is what I was gonna do. So I didn’t really want to spend my free time looking at it <the internet>” (CTP041). Another parent expressed, “I think getting the doctor’s opinion, from a doctor that knows <Child> and has been with her through her whole treatment is probably the most helpful, because I think he’s got her best interest at heart.” (CTP026P).
Despite expressing deep trust in their medical teams and hope for treatment success, families voiced a critical need for taking ownership in preparing themselves for treatment. This was organized into the theme “self-efficacy.” Multiple families shared they felt a duty to ask questions to advocate for their child. When asked about advice for future families preparing for CAR T cell therapy, one parent offered, “Ask a lot of questions and just make sure that you’re going into it as informed as you possibly can. And I think that’s really served <Child> well, because we know our child better than anybody else. So just having the ability to not be afraid to ask questions and follow up with things and make sure that things aren’t forgotten is really important, just to be a part of the medical team” (CTP002P).
Regardless of the decision-making mechanism, all but one family reported being satisfied with their decision and would make the same choice again. Notably, none of the families interviewed had a child who died before the interview. The sole family expressing decision dissatisfaction experienced a severe adverse event from treatment.
Coping with symptoms
The three most prevalent symptoms included pain, fatigue, and nausea. Overwhelmingly, families reported that physical symptoms during CAR T cell therapy were tolerable. Families were struck by this “feeling normal,” both physically and as a reflection of life returning to ordinary. This sentiment was organized into the theme “Normalcy.” Multiple patient participants noted a resolution of baseline physical symptoms, such as pain or nausea, and gaining a sense of independence, with n=4 participants using the word “normal.” One participant shared, “I really enjoyed being somewhat independent, a little bit, considering the past four years I’ve kind of been pretty dependent on my parents and caregivers” (CTP017). This participant then described being able to resume college classes in the time since their infusion. Another parent reflected, “Its been really nice to be able to be at home and to kind of feel like we are living a somewhat normal life because he feels probably better than he can remember feeling… He just feels well. He feels like a normal little boy” (CTP015P).
Most of the symptoms were attributed to lymphodepleting chemotherapy. Symptoms from CRS and ICANS were infrequently discussed, including among patients with reported severe toxicity. Symptoms were often compared to experiences with prior therapies, including intensive chemotherapy or hematopoietic stem cell transplant, reporting that symptoms experienced with these prior therapies were more significant than those experienced with CAR T cells. To reflect this viewpoint, we organized this experience into the theme “Perspective.”
Finally, families acknowledged grappling with a “symptom paradox,” a counterintuitive desire for symptoms to occur with therapy, attributing the presence of symptoms to be a sign of efficacy. One family explained: “Strangely enough, it made us sort of feel comforted when he did spike a high fever because it made us think that maybe it was working, ‘cause that’s what we were told beforehand… It might not happen, but if it does happen, that could be a good indication that it’s working.’ So as soon as it did happen and he spiked a fever, my husband was inpatient with him and he called me and he was like ‘He spiked a fever!’ and we both (it was weird) got a little bit excited like ‘Okay, this is good!”’ (CTP015P).
Unforeseen psychosocial challenges
Challenges of CAR T cell therapy were organized into three themes. First, families reflected on the emotional and financial sacrifices of being away from home, organized into the theme “The toll of being away from home.” Many families relocated for treatment, requiring the family to live local to the treating center for weeks. Although families acknowledged the benefits of technology for staying connected, they lamented the physical separation. One parent shared, “You just don’t see the people you’re used to seeing every day. She made a lot of new friends and with video chat, life has turned out to be a whole lot different, but it’s still hard to not sit and watch movies together, or eat dinner together as a family, or call her brothers to come over and visit. You have to call them on video chat. It wasn’t awful; it was just different” (CTP066P). Other families described the financial costs of transportation or lodging. For example, one family shared, “I mean it’s a financial strain. Things work out. You give-and-take in different places, but just being that far away from home is costly” (CTP020P).
The second challenge described was navigating unexpected scenarios, organized into the theme “The unexpected.” Predominantly this related to development of toxicity, requiring longer than expected hospitalizations or changes in subsequent therapy. One parent shared, “The last thing I would expect is that the clinical trial would then cause a reason to not get treatment after that. I mean and in <Patient’s> case, she’s had a lot of progression since then, so I can’t blame anyone and I don’t have a magic ball, but it does make us wonder like if we didn’t have that issue and we were able to get her on another treatment after the trial, would she have this level of progression since the trial” (CTP028P).
Finally, families endorsed the challenges of navigating novel therapies, organized into the theme “The unfamiliar.” One family explained, “I feel like if it was farther along in the CAR T development and research and knowledge, that maybe they could’ve ironed out a few more kinks and understand more about how it works and why it works… and maybe we could’ve had more options about which kinds of CAR Ts we could’ve done here, and maybe we would’ve had better results and been able to get straight to transplant, but it is what it is and we are where we are” (CTP047).