Identification of Patient and Caregiver Perspectives in Langerhans Cell Histiocytosis Using Focus Group Discussions

Background:

Langerhans Cell Histiocytosis (LCH) is a clinically heterogenous inflammatory myeloid disorder, typically affecting children. Because of the uncertainty of disease course, rarity of disease, and incorporation of chemotherapy in treatment, the patient/family experience of LCH may experience significant emotional stress in addition to the physical disease burden. Aligned with the focus in the literature in other rare diseases, this study explored patient and family perspectives to develop a better understanding of patient treatment priorities, gaps in psychosocial support, and health care needs for families diagnosed with LCH.

Methods:

IRB approval to conduct this study was obtained. 4 focus group discussions (FGDs) with 14 study participants (6 adolescent patients and 8 adult patient caregivers) were held to elicit factors impacting the experience of diagnosis, treatment and surveillance for LCH. Topics discussed included: understanding of the LCH disease process, interpersonal communication between providers and patients/caregivers, difficulties experienced during the diagnostic, treatment or surveillance process, and fears surrounding disease recurrence.

All patients and caregivers were recruited from the outpatient clinic at one institution via email or telephone using a consecutive sampling strategy. Further, the sample purposively included a range of patients in the treatment process (from recent diagnosis to active treatment to surveillance). The FGDs were all held virtually using Zoom videoconferencing software and subsequently audio-recorded and transcribed. The same researcher facilitated all FGDs.

Two investigators independently reviewed the initial transcripts to develop the provisional codebook for this analysis. The investigators then applied the comprehensive codebook to all transcripts for the final coding process, after which key themes and concepts were identified and classified using the methodological orientation of grounded theory.

Results:

Fourteen individuals were divided into four focus groups. 6 adolescent patients, ages 12-21 years (median 14) were split into 2 groups (3 each), and 8 caregivers, all female, were split into 2 groups (4 each). Participant characteristics were as follows: 79% female and 21% male, 66.6% non-Hispanic White (66.6%), 16.7% Asian, 16.7% Latinx; 83.3% single system disease and 16.7% multi-system involvement. Intentionally, 50% of participants (patients or caregivers) experienced recurrent disease.

Key themes from the FGDs were:

• 1. Length of time to diagnosis

• 2. Caregiver relationship with internet and social media as source of information

• 3. Lack of knowledge surrounding LCH disease, pathophysiology, and causes

• 4. Importance of peer and social support for families impacted by LCH

• 5. Dissatisfaction surrounding the disease classification of LCH - not cancer, perceived lack of access to resources

Discussion:

Accounting for patient and caregiver perspectives, especially for rare diseases like LCH, is critical in the delivery of patient centered care and optimizing outcomes. Notably, the patient/caregiver education surrounding disease treatment was found to be a central gap, even with a wealth of available materials. Additionally, peer support groups were recognized as a powerful tool for helping parents to cope.

The results also demonstrated that increased care coordination and assistance with navigation of complex care process could benefit families affected by LCH. Interestingly, the debate over classification of LCH as a cancer has implications as highlighted by the FGDs. The participants preferred the use of cancer to describe LCH. The results from this study could be used to develop a larger, quantitative patient preference study eliciting preferred methods of addressing these gaps.