Lipstein, Ellen A.; Lovell, Daniel J.; Denson, Lee A.; Moser, David W.; Saeed, Shehzad A.; Dodds, Cassandra M.; Britto, Maria T.
The growing use of treatments with significant risk-benefit tradeoffs has led to an increasing number of complex treatment decisions in pediatrics. One model of such decisions is that to initiate treatment with tumor necrosis factor-α inhibitor (TNFαi), which occurs in both Crohn disease (CD) and juvenile idiopathic arthritis (JIA). The importance of this decision is amplified by the relatively high prevalence of each disease, approximately 0.4 of 1000 for CD (1) and 1 of 1000 for JIA (2). Furthermore, in both pediatric and adult settings, chronic disease decisions such as this often occur over time (3–7), increasing the complexity of decision-making interactions compared with those in acute decisions (8).
Limited existing research in pediatric chronic conditions indicates that parents reconsider difficult medical and surgical decisions and may experience negative decision outcomes, such as worry, stress, and regret (3,9–11). Some of this distress and reconsideration are likely because of inadequate, complex, or poorly timed information and decision support (9–13). For parents wanting to participate in treatment decisions for their children, it is critical they have access to accurate, unbiased information as a component of high-quality decision making (14).
Although studies have shown problems with information delivery and support of parents making treatment decisions (9–13), it is unknown what information parents seek during decision making. Given the use of TFNαi in inflammatory bowel disease, as well as its high prevalence, understanding decisions in these conditions may significantly affect our ability to improve decision making in pediatric gastroenterology. Furthermore, the opportunity to compare CD and JIA likely provides a broader perspective and greater variation in parent experiences. This will promote the eventual development of decision-making interventions that work in diverse settings with diverse individuals.
In both CD and JIA, TNFαi can slow disease progression and alleviate symptoms but has limited long-term data and potentially significant risks, such as immunosuppression or malignancy (15,16). Moreover, the TNFαi treatment decision is likely to become more common, as physicians begin to consider using TNFαi earlier in the course of disease, before other treatments have been tried (17,18). To date, limited research has addressed decision making in pediatric chronic conditions; therefore, we used qualitative methods to begin studying the breadth and depth of parents’ experiences making decisions about TNFαi (19,20). The goal of the study was to develop an understanding of how parents presently make decisions about TNFαi treatment and determine how these decisions vary between CD and JIA, to guide future interventional work aimed at improving the decision-making process.
Parents were recruited from a large academically oriented children's hospital that has 20 gastroenterology and 8 rheumatology attendings, as well as rotating students, residents, and fellows. We conducted interviews with parents of children (of any age) diagnosed as having CD or JIA who had considered TNFαi treatment. Parents of children with ulcerative colitis were not included because the possible option of surgery makes the TNFαi treatment decision distinctly different than that in CD or JIA. The goal of our sampling strategy was to include parents at different points in the decision process, rather than to obtain a demographically representative sample. First, we used local registries to identify all patients with CD and JIA who initiated TNFαi treatment within the last year. Second, we requested providers refer families just starting the decision process or known to have struggled with the decision. Participants were eligible if they were English speaking and their child did not have a comorbid diagnosis that could also be treated with TNFαi. We sent letters to potential participants with an opt-out phone number.
The Cincinnati Children's Hospital Medical Center institutional review board approved the present study. Informed consent was obtained from all the participants. Participants received $30 remuneration.
Based on existing adult and pediatric literature about information needs when making treatment decisions (9,13,21–26), as well as input from relevant clinicians, we developed a semistructured interview guide (available upon request) focused on decisions related to CD or JIA. Interviews started with general questions regarding the parent's experience having a child with a chronic disease. They then moved toward an in-depth discussion of parents’ experiences with treatment decisions and their associated information needs. In discussing TNFαi treatment decisions, we focused on parents’ discussions with providers, factors considered in the decision, and information used. After the first 4 interviews, minor clarifications were made to the interview guide.
Participants were contacted to schedule an in-person or telephone interview with 1 of 2 trained interviewers. Telephone interviews were used for families who lived >1 hour from Cincinnati Children's Hospital and did not have an upcoming appointment. Field notes were taken, and interviews were audio-recorded for transcription. All transcriptions were verified by study staff. We continued recruiting until we reached thematic saturation (19,27), the point at which no new concepts were discussed after 3 consecutive interviews in each disease.
Data Coding and Analysis
We used thematic analysis (28) for data coding and analysis. For each disease, a multidisciplinary team, including the relevant subspecialist, annotated and open-coded 2 transcripts. The team then discussed the interview themes and developed a preliminary coding structure. The 2 teams’ structures were combined, approved by all study team members, and used for the remainder of coding. Two researchers independently coded the remaining transcripts and resolved differences through discussion. Once they reached a high level of consistency in their coding patterns (after 5 more interviews), coding was completed by one person and reviewed by the second. Following coding, the data were analyzed by thematic groupings, such as information sources, decision-making process or influencing factors, and then compared between CD and JIA. NVivo 8 (QSR International, Victoria, Australia) was used for coding and analysis.
The results are organized according to major themes, with the majority of illustrative quotations appearing in tables. When parents of children with CD (pcCD) and parents of children with JIA (pcJIA) had similar results the table has a single column. Differences between pcCD and pcJIA are highlighted by dividing some tables into 2 columns. Finally, to maintain participant confidentiality, parents are identified only by disease and whether their affected child was young (<13) or a teen (≥13).
We interviewed 35 parents from 34 families. On average, children with CD were older, had a longer duration of disease, and a shorter duration of TNFαi treatment (Table 1). All children had tried other medications before discussing TNFαi; in some cases, this was limited to nonsteroidal anti-inflammatories. Most participants were mothers and nearly all participants self-identified as the family leader of medical decisions for their children. Most children with CD received treatment with infliximab (Remicade, Jannsen Biotech Inc, Horsham, PA), whereas JIA was typically treated with etanercept (Enbrel, Immunex Corp, Thousand Oaks, CA) or adalimumab (Humira, Abbott Laboratories, North Chicago, IL). We interviewed 2 pcCD and 1 pcJIA whom physicians identified as being especially hesitant about TNFαi treatment. We found no differences in the information needs or decision processes for these families.
When asked which CD-related decision was most challenging, pcCD quickly and nearly uniformly named TNFαi treatment as the most challenging decision. They expressed anxiety and distress about this decision, as well as concern about whether they made the right decision. pcCD reported weighing the severity of their child's disease, with an emphasis on quality of life and growth, against the lack of long-term data about TNFαi and the potential risk of lymphoma. In contrast, pcJIA often had to think about their answer. Most, but not all pcJIA, believed the decision about TNFαi treatment was the most challenging owing to potential adverse effects and having to administer injections (Table 2).
Although pcCD and pcJIA used information from similar sources, the timing of their information seeking differed. In CD, parents typically were introduced to the idea of TNFαi by their child's health care provider and then sought information to help them decide whether or not to initiate treatment. As such, their decision process often took place over an extended period of time. In contrast, in JIA, TNFαi treatment typically was introduced by the provider and a decision made in the same appointment. Therefore, pcJIA tended to access other sources of information to confirm their TNFαi treatment decision.
In both groups, nearly everyone named starting TNFαi treatment as one of the major decisions they had made. At the same time, most believed there was no choice. The framing of this lack of choice differed. pcCD frequently referred to having no choice because of failing other treatments or because “nothing else was working”(pcCD, teen). pcJIA were more likely to describe initiating TNFαi treatment as “moving to the next step” (pcJIA, young child).
The vast majority of both pcCD and pcJIA sought information from multiple sources (Table 3). Most commonly, parents used information from the care team. This information was typically verbal information presented during clinic appointments, but also took the form of disease- and treatment-related handouts. Additionally, many pcJIA mentioned receiving a binder of information at the time of diagnosis. Although families highly valued such information, they occasionally struggled to balance conflicting recommendations, particularly when their child received care from multiple subspecialty services.
The Internet also served as a key information source, with parents frequently using search engines to find disease or drug information. They accessed both general medical sites, such as WebMD, and disease-specific Web sites. Most parents believed the Internet provided helpful and positive information; however, several commented that some sites, particularly those of pharmaceutical companies, were “biased, but (made them) feel better” (pcJIA, young child). Specific to CD was the frequent mention of the Internet as a source for scientific reports, such as from MEDLINE.
In addition to information from their care team and the Internet, parents in both groups received information from social contacts. For some this included friends and family with first-hand knowledge of the disease or treatment, including those they met at disease-related conferences. Others wanted information and advice from friends or community members known to have medical backgrounds. Several parents mentioned that discussing the decision with others led them to learn about mutual acquaintances who had the disease or had used TNFαi. They then sought information from those people.
Types of Information
The information parents used in making TNFαi treatment decisions fell into 3 dominant categories: disease-related information; TNFαi-specific information; and the experience of other affected families.
In both CD and JIA, information about disease severity, such as lab values and the child's quality of life was considered in the TNFαi treatment decision, as was the risk of future disability and disease-related complications (Table 4). No parent mentioned needing more information about their child's illness, except as it related to the efficacy of TNFαi treatment; however, pcCD and pcJIA expressed different expectations and understanding of disease progression. pcJIA typically had a sense, prior to discussion of TNFαi treatment, of the typical disease progression and plan for treatment escalation. In contrast pcCD expressed having less specific understanding of common treatment pathways. They also wished that, before needing TNFαi treatment, they had a better understanding of how and when treatment may be escalated.
Parents sought information about the effectiveness, adverse effects, and logistic details of TNFαi treatment (Table 5). Parents of children with either disease reported knowing that the medication was typically effective or that there had been local success with TNFαi treatment, saying things such as “the doctors told us…what kind of success they have seen” (pcJIA, young child). In this setting, pcJIA talked about the local and overall effectiveness in a more positive manner and with greater apparent confidence, whereas pcCD reported feeling it would work but seemed less certain. Both groups of parents wanted more long-term data, as well as information on how quickly they would see improvements in their child's symptoms and when they would assess the effect of TNFαi treatment on their child's disease. Many recognized that the data they sought may not yet exist.
In addition to understanding the benefits of treatment, all parents had some sense of TNFαi risks (Table 5). pcCD discussed and wanted detailed information about risks, whereas pcJIA often made general statements about adverse effects, such as “there is a certain amount of side effects” (pcJIA, young child). The risk most often mentioned by pcCD was the development of cancer and several mentioned receiving letters about the FDA black box warning (29). The distress associated with this risk of cancer was evident from parents’ tears during the interviews and from the frequency with which they reiterated concerns about the risk. In contrast to pcCD, pcJIA were as likely to mention the risk of immunosuppression as that of cancer. Several parents from each group expressed that they had a detailed understanding of how the length of TNFαi treatment and combination with other medications contributed to the cancer risk. Some reported knowing that the data on the cancer risk were evolving.
Parents valued information from other parents or patients who had experience with TNFαi treatment. Many parents mentioned reading “patient reviews” on the Internet. They most often found such narratives on pharmaceutical Web sites, chat boards, and blogs. Many parents believed the reviews helped them understand the treatment experience (such as pain of injections), “humanized the choices” (pcCD, teen), and “helped me convince myself” (pcJIA, young child). Others found they “have no substance…just get you worried” (pcCD, teen).
Similarly, parents from both diseases wished for a personal connection to other families who had been through this decision. Although some parents specifically wanted a connection to other parents, others only mentioned wanting their child to meet another child. Several commented that it would be difficult, because of confidentiality, but they would like their physician to connect them to another family, to help them understand what the treatment is like and to not feel so alone; however, the desire for a personal connection was not universal. One mother stated that a referral from her child's physician would be “…all marketing… it (would) be a family who had had a great experience and (would not) address our concerns” (pcCD, teen).
Role of Information in Decision Making
Parents desired more information than they received, specifically about TNFαi rather than general disease information. pcCD generally expressed an understanding of the range of possible adverse effects but wanted more long-term data and especially wanted to read the original studies about both efficacy and risk. In contrast, pcJIA rarely commented about needing further information about efficacy but did want more information about short- and long-term risks associated with treatment. Both groups believed that additional information was unlikely to make them change their decision, but may make the decision process less stressful. This perspective was highlighted by one parent (pcJIA, teen) who, after choosing TNFαi treatment, looked online and was “just shocked about a number of things” but did not consider changing the treatment plan.
Our study provides an in-depth view of parents’ experiences making a difficult treatment decision to use TNFαi in pediatric CD or JIA. Our data suggest that although they see it as a major decision, many parents may not recognize the choice as being, at a minimum, between starting and not starting TNFαi treatment. In adult settings, a lack of understanding of the choices is a recognized barrier to engaging patients in decision making (30). TNFαi treatment was a difficult decision for parents even in the perceived absence of a choice. It may become more challenging when TNFαi treatment is offered earlier in the disease, before other treatments have been exhausted (17,18).
Consistent with other studies of decision making in pediatric chronic conditions (3,5,6), decisions about TNFαi treatment are a process rather than an event. This is particularly true for the many pcCD who typically were introduced to the decision in a clinic visit, sought information, did research on their own, and then made their decision days to weeks after the initial appointment. Regardless of whether parents sought information before (pcCD) or after (pcJIA) telling the physician of their decision, they accessed and sought information from diverse sources.
In results reflective of our own, one large survey of Dutch adults with inflammatory bowel disease found that more than half wanted to be better informed about their disease and about new medication options (31). Other surveys, from both the United States (32) and the Netherlands (33), found that adult patients, similar to parents of pediatric patients (32), overestimate benefits and underestimate risks associated with infliximab treatment. Likewise, parents and adult patients with CD have similar tolerance for risks associated with treatment (34). Thus, the information needs and support found among parents interviewed for our study are not unique to the pediatric setting. Work is needed in both adult and pediatric settings that will ensure that patients and families engaged in decision making about TNFαi treatment have and understand information about treatment risks and benefits.
Parents are looking for guidance and resources from the physicians with whom they are making the decision. Because families depend so heavily upon physicians, it is essential that they provide families with correct, timely, unbiased information (35). Some of the difference seen in parent distress may be because of pcCD feeling that TNFαi treatment signals their child's failure on other medications, compared with the pcJIA framing the decision as a next step. Additionally, some of the decision-making stress is related to parents’ understanding about adverse effects. Other research has shown that framing and presentation of numerical data significantly affect patient interpretation (36). Overall, the variation in parents’ concern is likely a combination of adverse effect differences, parents’ temperaments, and information framing by the provider. Moreover, providers and families need to understand that there is not one right decision, but a right decision for each family. Therefore, high-quality, shared decision making requires an understanding of treatment risks and benefits, as well as families’ values and desires (37,38).
Previous pediatric decision-making studies have focused on the information provided by the care team and the timing of such information (9,24). Studies focused on health information seeking consider the sources patients use after a diagnosis but not how they are used (39–41). By considering the timing of information seeking and the sources used, our data demonstrate that parents seek information from a wide variety of sources throughout their decision-making experience. By the time parents reach the point of making a decision about TNFαi treatment, their information needs are focused on the treatment, rather than the underlying disease. Providing parents with straightforward information on risks and benefits is a first step toward assisting them in making informed, high-quality decisions; however, some parents may require further assistance, such as exposure to scientific reports, experiential information from other families, and a timeline of how and when treatment effectiveness will be reassessed. Determining how to provide information in a way that limits bias and manages expectations should be a focus of future research. Furthermore, because the use of TNFαi moves earlier in the disease, the need for disease and treatment information may become intermingled. This will force providers and researchers to consider how to address these needs simultaneously.
Although the opportunity to compare patients from the rheumatology and gastroenterology clinic increases the depth of our work, our data's generalizability is limited by this being a single-institution study. This limits our ability to disentangle disease effects from clinic-level practice variation, as well as to understand decision making in a nontertiary care setting. Furthermore, some of the difference between pcCD and pcJIA may be related to the active role providers in the rheumatology clinic have had in TNFαi clinical trials. Additionally, we encountered difficulty in identifying families that were in the process of making a decision or had declined TNFαi treatment. This difficulty likely reflects both high levels of eventual treatment acceptance and the relative ease of identifying families (using registries) who chose to initiate treatment.
Although almost all participants mentioned that they take a leading role in family medical decisions, to truly understand the decision-making dynamics and develop family-centered interventions, other family members’ perspectives should be explored. Future work should consider the role of others, most importantly the pediatric or adolescent patient. Finally, we interviewed parents at variable times after the decision and parent report is subject to recall bias. In the future, prospective study of the decision-making process will assist in understanding the interaction between physicians and families and how to provide decision support that will meet parents’ needs. For an understudied area such as pediatric decision making, our approach is a key first step.
Our study adds to the existing literature on pediatric decision making by comparing the experience of decision making about TNFαi treatment in CD and JIA. This comparison highlights variations in the decision experience, but also reveals that in both diseases decision making is a process and information needs continue after the treatment decision is made. For at least some parents, this on-going information seeking is tied closely to the distress and conflict they feel related to the decision. Improving access to information and supporting parents’ involvement in decision making are important components of high-quality, family-centered care (42). In adult settings, the use of structured decision aids that help patients become informed and consider their personal values related to the decision has been shown to improve decision outcomes, including conflict and regret (43). Future work in pediatrics needs to consider how to adapt such an approach to a setting that includes the triad of parents, patients, and providers. By assisting families with decision making, we may be able to improve decision outcomes, facilitate timely, high-quality decision making, and ultimately improve clinical outcomes for children with inflammatory bowel disease, as well as other chronic conditions.
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