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).