Evolution of Pediatric Chronic Disease Treatment Decisions

Sample

Physicians and one nurse practitioner, hereafter referred to as providers, who treat patients with either JIA or IBD were recruited from the rheumatology and gastroenterology clinics of a large academic children’s hospital. All of the approached providers, except one, agreed to participate, and written consent was obtained.

Eligible families were those who were English-speaking, whose child had JIA or IBD, who had a clinic appointment scheduled with a consented provider, and whose provider anticipated discussing biologic treatment initiation at that visit. At the time of the clinic appointment, written consent and assent (for children age 8–17) were obtained from anyone who would be in the room during the visit. We recruited families until we reached informational saturation for each disease group, ²¹ defined, for this study, as the point at which 2 consecutive visits revealed no new approaches to discussing treatment decisions. Families who discussed biologic treatment initiation during the recorded visit (21 out of 30) then participated in sequential telephone interviews.

Families were compensated $30 for video-recording of the clinic visit and $10 per telephone interview. Providers received no compensation. Cincinnati Children’s Hospital Medical Center’s Institutional Review Board approved this study.

Data Collection

Overview

In total, 21 families, including 29 parents, and 10 providers participated (Table 2). All patients were white and non-Hispanic, reflecting the general makeup of the clinics from which families were recruited. Results are organized to highlight the evolving nature of treatment decision making, including patterns of decision evolution and descriptions of the ways in which decision-making elements evolve or remain constant over time. Illustrative quotations are presented in Table 3.

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Table 2

Participants

	Patient Condition
Participants and Characteristics	Inflammatory Bowel Disease	Juvenile Idiopathic Arthritis
Visit participants—providers
Years in subspecialty, median (range)	9.5 (3–19)	16.2 (1–33)
Sex, n
Male	3	3
Female	1	3
Provider type, n
Physician	4	5
Nurse practitioner	0	1
Ethnicity, n
Non-Hispanic	4	6
Race, n
White	3	5
Asian	1	1
Visit participants—family members
Patient, n	12	9
Median age in years (range)	11.5 (7–16)	9 (2–18)
Mother, n	12	9
Father, n	2	6 a
Maternal education, n
Not a college graduate	2	5
College graduate	6	1
Graduate or professional degree	4	3

a.

One father present at the visit declined to participate in interviews.

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Table 3

Illustrative Quotations

Themes and Subthemes	Quotations
Decision evolution: Decision events
Research	“I spend hours on forums, threads, on blogs, drug company websites.” (mother #12)
	“I search the internet once in a while.” (mother #4)
Conversations	“I had actually discussed it with [2 doctors] and had asked the few questions I have because I had looked into it before.” (mother #8)
	“Mostly me and [the patient] have talked about it.” (mother #21)
	“I spoke with [the nurse] and we had a 20-minute discussion on the phone.” (mother #1)
Medical events	“They did a Doppler [of his swollen knee] in one of the visits.” (mother #19)
	“We take her off of those medications and then she gets sick again.” (mother #21)
	“ . . . then his pain came back between the two visits and when I called we just enacted the plan we already had in place.” (mother #28)
Constant element: Role perceptions	“[The doctor] pretty much explained what the new medicine was and how it works and me and my wife pretty much decided that we wanted to stay with the methotrexate.” (father #31; two-week interview)
	“He just kind of discussed it with us and then we were all pretty much like, we’ll all just stay on the methotrexate.” (father #31; six-week interview)
	“[The doctor] offered the suggestion and then my husband and I talked it over and I think both of us were on board with trying something.” (mother #19; two-week interview)
	“Dr. L suggested it and explained it to us and we decided that was the next, natural step to take.” (mother #19; ten-week interview)
	“Well, we listened to the doctor’s advice . . . we talked it over and we just made the decision.” (father #19; ten-week interview)
Constant element: Internal processing	“We’re trying to figure out what to do next.” (mother #6; two-week interview)
	“I still think about which one will be the best one to start.” (mother #6; ten-week interview)
	“I guess we were just trying to decide with all of her other medical issues.” (mother #25; two-week interview)
	“We were trying to decide what would be the next course of treatment.”(mother #25; six-week interview)
	“Basically I thought, ‘well, are we at that point? Are we at the end of the rope?’ I was just thinking about the [biologic therapy].” (mother #1; two-week interview)
	“ . . . now that she did start it . . . I’m thinking, ‘Oh no, did we do the right thing?’” (mother #1; six-week interview)
Evolving element: Expectations	“Definitely, I just think letting him and seeing him feel better is my biggest goal. Trying to kick it in to remission where he’s not walking stiff in the morning and things like that.” (mother #28; two-week interview)
	“Just, you know, minimal side effects since that seems to be what was bothering him the most with [prior treatment].” (mother #28; six-week interview)
Evolving element: Worry and hope	“Can we get her off of her 15 pills and go down to the [biologic therapy] in one pill? You know, is this going to be the right treatment? Are we going to have to switch again? Just a lot of unknowns right now.” (mother #9; two-week interview)
	“I hope she stays well with less flare-ups.” (mother #9; six-week interview)
	“We still both had some reservations about the medication particularly because of the severity of the side effects.” (mother #3; two-week interview)
	“It seems like he’s feeling much better. . . . I’m just glad at this point that we moved forward with that decision.” (mother #3; six-week interview)
	“I, eventually, would like to see [my son] go into remission with is. That’s our hope.” (mother #21; two-week interview)
	“You always worry about any medications that something shows up years later . . . you know, this has been taken off the market because of this, this and this.” (mother #21; six-week interview)
Element with constant and evolving components: Option framing	“So that was the biggest decision . . . whether to try a different treatment option or hold off on therapy or to continue with the therapy and try to get rid of side effects.” (mother #28; two-week interview)
	“So one is the biological, then they also talked about leflunomide and then we talked about methotrexate.” (mother #28; six-week interview)
	Interviewer: What were you choosing between?
	“You know, letting the methotrexate do a slow, I guess you could say slow healing process where when we add the [biologic therapy], it will be a faster heal.” (mother #23; two-week interview)
	“Whether it was the right time to put her on an additional injection. If it would be worth her having the two shots a week instead of one shot.” (mother #23; six-week interview)
	“Well, I know [the nurse practitioner] had gone over [injected biologic] and I know there’s [different biologic] and I know she talked about one, I think it was [third biologic] and I can’t remember there might have been a couple other ones.” (mother #26; two-week interview)
	“[We were] choosing between her starting the [biologic] to make her feel better or her staying where she was without that.” (mother #26; six-week interview)

Decision Evolution

For all participating families, the treatment decision evolved over time, although the time over which it evolved varied from weeks to years, according to participants’ descriptions of their experience. During this time, numerous decision-making events occurred. Such events included having conversations with health care providers and other family members, researching treatment options, experiencing disease flares and related medical testing, and seeking insurance approval. The specific decision-making events varied between families, especially with regard to the order of events.

The type of event that occurred most often was conversation about the treatment options. Participants’ first awareness of biologics as a treatment option commonly occurred around the time of diagnosis, during a conversation with members of their child’s health care team or from parents’ own research. However, many parents recalled having thought, at that early time point, that such treatment was not for their child or might be an option “down the line” rather than now. For other families, early conversations mentioned biologics in passing, as part of a larger conversation about treatment options. Most parents reported that prior to the visit recorded for this study, at least one conversation about biologics as a treatment option had occurred with a health care provider.

In reviewing the timeline for each participant’s decision-making process, we found that these conversations and the other decision events consistently occurred as part of a process that followed 1 of 3 patterns. The first is a slow, steady progression in which families are aware of the choices and, over time, progress to a decision about biologics. The second pattern is more step-wise than the first. In this, the family members perceived the decision about biologics as the next step in a series of treatment decisions. These families articulated having understood, from an early point in the patient’s disease course, that there were established, ordered treatment steps. The final pattern is one of abrupt decision making. Here an acute event, such as a disease flare, leads to a rapid decision regarding biologics. In all 3 patterns, after a treatment decision is made, family members nearly immediately began considering the next decision, such as when another medication could be discontinued.

Constant Elements of Decision Making

As decisions evolved, individuals’ roles and perceptions of others’ roles in decision making remained constant. Most participants described the same people as participating in decision making, in the same manner, in each sequential interview. For example, one mother described the physician’s role as providing information, hers as weighing that information. Another mother (#1) said that she and her family “were at a point that [they were] just going to do whatever [the doctor] thought was in the best interest of my child.” Additionally, when more than one parent participated in the study, each member of the pair gave a similar description of the decision participants’ roles. Although most individuals’ role perceptions were constant, two families did have changes in the perception of who made the final decision. In one, the mother initially stated that she made the final decision but in later interviews stated that the doctor made the decision. In the other, in the first interview the mother described making the decision together with the doctor but in a later interview stated that she made the decision alone.

Although specifics varied both within and between families, another constant was parents’ descriptions of a prominent role for internal processing throughout the decision-making process. For example, many parents mentioned thinking about the options. Others talked about “deciding what’s best” or “coming around to a decision.”

Evolving Elements of Decision Making

Other aspects of the decision process, such as expectations regarding treatment with biologics, changed over time. Commonly, when participants had chosen to start biologics, their expectations initially had focused on the anticipated improvement in symptoms. However, once symptoms improved, participants expressed new expectations, often related to limiting side effects or decreasing other medications.

For most participants, the decision about biologics was characterized by a combination of worry and hope that fluctuated over the course of the process. Parents expressed that sometimes they felt deep worry about side effects and other times felt more hopeful that the new treatment would be effective. Some parents, after making the treatment decision, experienced a sense of relief, especially when their child’s symptoms improved and the child had not yet experienced any side effects. Others, even if their child’s symptoms improved, seemed to experience little relief and began to worry whether they made the right decision, when side effects would appear, or how long the treatment would continue to work. Within families, the emotional changes over time tended to be similar. For example, if one parent continued to worry after the decision was made, the other parent often did as well. In a few cases, one parent would refer to the other’s emotions rather than describing his or her own, saying something like “my wife was concerned.” This cycle of worry, decision making, and relief, often followed by worrying about the next decision, overlaid the general patterns of decision evolution.

Elements of Decision Making with Both Constant and Evolving Components

Many aspects of decision making had both constant and evolving components. During decision making, the options considered by most participants did not change, but the framing of the options changed for some families. For example, one family initially discussed the options as being between 2 classes of medications but in a follow-up interview described the options as a choice between ongoing symptoms or using biologics.

The concrete factors that parents mentioned as influencing their decision-making process changed in emphasis but not content over time. In other words, some factors that were initially minor influences became more influential over time, and vice versa. These shifts in emphasis often appeared situational rather than temporal. Such factors included, but were not limited to, data on long-term risks and benefits, disease severity, experiences with other treatments, and the patients’ other diagnoses. For example, as one patient’s symptoms worsened over time, her mother discussed less about side effects and more about the patient’s disease severity as influencing the decision. When more than one parent participated in interviews, the parents typically mentioned the same influences but may have emphasized them differently.

Similarly, we found that participants’ treatment concerns did not change over time, but for many the emphasis placed on such concerns did change. For example, once families made a decision to start treatment with a biologic, many placed less emphasis on previously expressed concerns about treatment. One father (#19), whose child started biologics between the first and second interview, stated in the first interview that his concern was “ . . . the side effects . . . Even when I’m at work, I’m thinking about the side effects.” Four weeks later he said, “The only concern is I want to know why it isn’t working.” In this instance, the lack of effect from treatment had apparently led to his concern about side effects having less salience. In contrast, in families whose decision was not to start treatment with biologics, the participants continued to emphasize their treatment concerns. In only one family did concerns notably differ between family members, with the mother concerned about side effects and the father concerned about the treatment’s impact on the patient’s social life. Nevertheless, this family, who chose not to start treatment, followed the overall pattern of continuing to emphasize their treatment concerns.

Disease-Specific Differences

Our analysis was not designed to compare and contrast families of patients with IBD with those of patients with JIA. During the course of analysis, however, some differences were observed. The first difference related to the pattern of decision evolution, in that abrupt decision making associated with an acute event such as a disease flare occurred more frequently in IBD than JIA. Also, compared with parents of patients with JIA, parents of patients with IBD more often reported having had multiple conversations with other individuals, such as friends or family members, and having done independent research about treatment options.

As to the process of decision making, nearly all parents of children with JIA reported making a decision either to start or not to start biologics during the recorded visit. In contrast, about half the parents of children with IBD indicated that the decision was made some time after the visit. Similarly, after the recorded visit and within the follow-up period of this study, few parents of children with JIA had additional conversations with a health care provider, but nearly half of parents of children with IBD had such conversations.