Immune thrombocytopenia (ITP) is characterized by isolated low platelet count in the absence of other etiologies for thrombocytopenia. Despite published guidelines (Neunert, Blood Adv 2019), controversy exists in the management of patients with pediatric refractory ITP. Data on second-line treatment approaches is limited and no randomized controlled trials exist for this patient group. A comparative-effectiveness trial demonstrated variation in prescribing practices, with parental or patient preference involved in decision making in 55% of cases (Grace, Am J Hematol, 2018). Despite this data, no qualitative studies have been done to explore the patient experience. We aim for the first time to explore thematic areas surrounding rationale for treatment choice for patients with refractory ITP from the patient/parent point of view, as a first step to creating a decision aid. Our goal is that this aid will inform individuals with regards to options, encourage engagement of patients in decision, and assist patients with processing their preferences so they can make choices reflective of their values.
Purposeful sampling identified participants locally from Columbia University Medical Center as well as nationally through the Platelet Disorder Support Association. Focus groups were conducted with an average of 5-7 parents of children with ITP in each group, and each lasting approximately 90 minutes. A written guide with open-ended questions was created to elicit participant opinions and foster guided discussion between them, illuminating particular concepts and permitting researchers to gain an understanding of patient experiences in the context of their own lives. Focus groups were audio recorded and transcribed. The de-identified transcripts were uploaded into Dedoose, a qualitative software. A thematic analysis was conducted to identify the nature, strength and prevalence of key concepts.
Three major themes were identified with regards to treatment choice for second line therapy: 'Experiences with Treatment Decision Making', 'Factors Important in Decision Making', and 'Concept of Remission'. Excerpts demonstrating findings from each theme, as well as some of the variability amongst participants' opinions, are shown in Table 1. With regards to experiences with treatment decision making, many parents expressed that they felt their hematologist presented the options with adequate descriptions of risks and benefits, though the extent to which parents seem to feel included in the decision was not consistent. Often, even if presented with options, parents did not view themselves as critical decision makers and some patients still reported not feeling well-educated about options. Additionally, many participants reported that they went through a "trial and error" process with regards to different second-line therapies, with no notable or specific conversations defining any one person's experience.
The data collected suggests that parents of children with refractory ITP have strong ideas about communications around their treatment options. The factors important to decision making appeared consistent between participants, and would provide valuable input in the ultimate decision making tool and also be important to capture in future research trials of different agents. Of interest, the concept of remission came up frequently, and did not have the meaning that the research team expected. Participants noted that the term had a negative connotation, and meant only that there was a possibility of the disease returning. They felt that the term gave a false sense of relief, because their disease was unlikely to be truly cured. This was emphasized as a value in treatment selection.
The information provided by these focus groups indicates that physicians' views on choosing therapies "based on patient preference" are not completely aligned with the patient experience. Additional focus group work, creation of a patient decision-aid, and inclusion of patient relevant outcomes in research will hopefully close this gap. We ultimately hope to create a clinical decision aid developed from input of these focus groups in order to increase patient understanding of their options, improve targeted treatment effects with regards to patient-related outcomes of importance, and help to guide management decisions.
Neunert:Platelet Disorder Support Association: Research Funding.
Asterisk with author names denotes non-ASH members.