Patient-preferred outcomes in patients with vestibular schwannoma: a qualitative content analysis of symptoms, side effects and their impact on health-related quality of life

Vestibular schwannomas (VS) are rare benign tumours of the hearing nerve. They typically cause hearing loss, tinnitus and balance disorders, but also headaches, a lack of energy and anxiety. Management of vestibular schwannomas is aimed at maintaining health-related quality of life (HRQoL) while preventing symptoms to get worse as a result of the tumour or treatment. In this study, we have explored the patients’ perspective on VS, its impact on HRQoL and the outcomes that patients consider most important when measuring and evaluating their disease-burden (patient-preferred outcomes). This study indicates that many patients with VS encounter participation difficulties in their daily physical and social activities and are mostly hampered by symptoms and side effects related to hearing, balance and energy. Treatment outcomes that patients place most importance on are hearing preservation, balance preservation and reduced tinnitus. Healthcare professionals should consider these key points and use these and the patient-preferred outcomes in consultation, treatment and follow-up to optimize patient-centred care.

Vestibular schwannoma (VS) is a rare, slow-growing, benign tumour originating from the Schwann cells surrounding the eighth cranial nerve. It is the most common cerebellopontine angle tumour, with an incidence of about 34 per 1,000,000 [1] and a median age at diagnosis of 50 years [2]. VS is most commonly characterized by unilateral hearing loss, tinnitus and dizziness but may also lead to a lack of energy, anxiety, headaches and balance problems. Current treatment for patients with VS is generally aimed at clinical outcomes such as tumour control, hearing and facial nerve preservation [3‐7]. Treatment options include observation with serial imaging in a wait and scan protocol, stereotactic radiosurgery and microsurgery [3, 7]. Unfortunately, neither one of these management strategies can improve the experienced hearing loss, tinnitus or balance problems. Additionally, all treatment options have a risk of deteriorating hearing, balance or facial nerve function. As VS is a benign tumour and usually not a life-threatening disease, patients will face the associated symptoms and/or management-related side effects (hereafter shortened to side effects) for the rest of their lives [8]. Consequently, VS is considered a chronic disease with a lifelong impact on the patient’s health-related quality of life (HRQoL). As of such, especially in patients with the chronic sequelae of VS, HRQoL is emerging as an increasingly important outcome in decision for treatment and treatment evaluation [9, 10].

QoL is characterized by the World Health Organization as: ‘The individual’s perception of their position in life in the context of the culture and value system in which they live’, and is influenced by six domains [11]. Four of these domains (physical, psychological, functional, and social) imply the influence of health on the QoL, also defined as the HRQoL [12]. Covering physical as well as mental needs, HRQoL is an important concept in disease perception, disease assessment and decision making [13]. HRQOL is driven by patient preferences, recognizing patients may prioritize and prefer additional or other treatment outcomes than clinicians, which are shaped by the patient’s beliefs, perspective, expectations and goals for health and life [14].

In order to incorporate the patient’s HRQoL in management strategy decision, HRQoL should be periodically measured, or at least the factors that impact HRQoL. HRQoL in patients with VS has been assessed by several researchers, mainly using generic questionnaires such as the 36-Item Short Form Health Survey (SF-36) and Glasgow Benefit Inventory (GBI) [5, 13, 15‐25], and less frequently, with the disease-specific Penn Acoustic Neuroma Quality-of-Life (PANQOL) questionnaire [3, 4, 26‐28]. Although these questionnaires measure HRQoL, they all restrict patients to pre-determined domains and answer options. Likewise, patient-preferred outcomes in patients with VS are highly understudied with only one study limited to prefilled possible impairments [29].

Understanding the patients’ perspective on VS, its impact on HRQoL and the patient-preferred outcomes (what outcomes matter most to patients) is important and essential in the process of shared decision making and, thus, requires deeper investigation. Taking into account, the considerable morbidity and impact on HRQoL with which VSs and their treatments are associated [30], insights in the patient perspective could be used during consultations, in counselling, and decision making. Therefore, the current study aimed to identify patient-preferred outcomes and to explore the patient-reported disease symptoms and management-related side effects in patients with VS in order to understand how these affect their HRQoL and daily life.

The expected outcomes of this study are a rich understanding of the experiences of patients with vestibular schwannoma and the factors that influence their HRQoL and daily life activities. We think these will mainly concern restrictions in societal participation and employment affecting HRQoL rather than the classical management outcomes such as tumour control and functional nerve preservation influencing HRQoL. The study will contribute to a deeper understanding of the challenges faced by patients with VS and inform the development of patient-centred care strategies that address their unique needs and preferences.

In total, 237 surveys were completed throughout the multiple platforms (e.g. Stichting Hoormij website and email, Facebook). After exclusion of patients with neurofibromatosis type 2 (n = 3), meningiomas (n = 1), and removal of duplicates (n = 2), 231 completed surveys remained eligible for analysis. The demographics of these patients are presented in Table 1. Patients had a mean age of 58 years (SD 11.5, range 24–86 years), and a slight majority was female (n = 144; 62.3%). Mean time since VS diagnosis was 8.3 years (SD 7.5). Most of the patients did not report any comorbidities (n = 153, 66.2%). Those patients with comorbidities (n = 78, 33.8%) mainly described a variety of diseases such as migraine, glaucoma, and Graves’ disease. In total, 164 patients (71%) were actively treated (stereotactic radiosurgery, microsurgery or a combination of both) for their VS, with a mean time since treatment of 8.4 years (SD 8.2). The other 67 patients (29%) were observed in a wait and scan protocol with serial imaging.

This qualitative study aimed to assess patient-preferred outcomes in patients with VS. Of the 231 respondents in this study population, the majority was actively treated (71%). Patients mainly prioritize preservation of hearing and balance, tinnitus reduction and restoring energy as patient-preferred outcomes. Second, patient-perceived symptoms and management-related side effects and their impact on HRQoL in patients with VS were explored. For both symptoms and side effects, patients mentioned reduced hearing, reduced balance and energy loss as most frequent issues. Overall, patients were most frequently affected in the socio-economic aspects of life (social engagements, work and tasks) and avoided busy environments.

Patient-preferred outcomes in patients with VS are highly understudied. A systematic PubMed search combining all search terms for VS with a standardized search query on patient preferences for treatment outcomes [33] retrieved one article for full-text assessment [29]. In that study, 576 VS patients ranked prefilled impairments on a scale of 1 (not too bad) to 5 (unbearable). Complete loss of hearing on both sides was ranked highest among the patient-preferred outcomes. Correspondingly, hearing preservation, in our open-ended questionnaire, turned out to be the most favoured patient-preferred outcome. Furthermore, patients in our study ranked balance preservation, tinnitus reduction and restored energy as important patient-preferred outcomes, but these may differ between patients and need to be enquired individually. Clinicians may, however, use and incorporate these preferred outcomes to improve counselling, better support their patients and improve the patients’ satisfaction [33, 34].

Patients often cited hearing and communication problems altogether with problems in their social interactions (Table 3, Supplementary Table 3). Deafness and hearing loss had a high impact on HRQoL and because of their symptoms or side effects patients reported to be hampered especially during social engagements. Although several studies showed that hearing impairment was less substantial in patients’ HRQoL, which might be attributed to a gradual adaptation to the hearing impairment over the years [27, 35], Breivik et al. found that VS patients had significant worsening of social functioning due to hearing loss [36]. Additionally, Carlson et al. developed and validated a new disease-specific QoL instrument for patients with VS as they felt domains that frequently impact well-being such as social engagement and occupational limitations were missing in the PANQOL [37].

Energy-related issues, especially fatigue, had a high impact on HRQoL and restored energy is among the most reported patient-preferred outcomes [9]. Unfortunately, studies specifically on fatigue or a lack of energy in VS patients are scarce. Despite this, Dhayalan et al. recently showed that patients with VS significantly suffer from fatigue complaints. They found a strong relationship between fatigue and other complaints such as depression and anxiety as also vertigo, one of the hallmarks of VS [38].

According to the patients in our study, balance issues—and especially vertigo—did not have the highest impact on HRQoL. An explanation for this phenomenon might be that symptoms like dizziness or vertigo are episodic and transient while hearing loss, deafness and facial weakness may be continuously present. Patients, however, prioritized vertigo among balance as the second highest patient-preferred outcome (Supplementary Table 1), which is in line with the many studies that showed a strong negative association between vertigo and HRQoL [30, 35, 36, 39] and ongoing dizziness being associated with a deterioration of long-term HRQoL in patients with VS [27].

Regarding side effects, facial nerve issues were often mentioned, and deafness was more frequent in treated than observed patients. This might be explained by the fact that treated patients often have larger tumours [40]. Another explanation is that treatment itself, either surgical removal or radiation therapy, might have caused nerve damage and facial dysfunction. Although not pointed out by our results as this was not explicitly investigated, Blom et al. showed that facial issues or paresis are debit to a lower HRQoL [41]. However, permanent facial weakness after surgery or radiosurgery is reported to be less than 10% [7, 42]. Similar to facial dysfunctions, headaches (both transient and permanent) are a well-known consequence of VS surgery and more commonly associated with the retrosigmoid approach compared to those treated by the translabyrinthine and middle fossa approaches [43]. Headaches in general, however, though the causal relation with VS is not exactly clear, might severely affect HRQoL [3], as demonstrated by the high impact of headache as VS-related symptom on HRQoL in our results.

Our study has several key strengths. Most importantly, in the current era in which HRQoL is highly prioritized, our study is the first to assess patient-preferred outcomes in patients with VS. Moreover, this study combined qualitative and quantitative data on symptoms, management-related side effects, and their impact on HRQoL and daily life. The use of open-ended questions in our questionnaire allowed participants to express themselves freely about living with VS. Other strengths of this study were the high number of included patients and the methodology of independent coding by two of the authors.

Several limitations should, however, also be acknowledged. First of all, even though the large number of participants is a strength, the multiple platforms through which the patients were included form a limitation. While we know Stichting Hoormij send 332 VS patients an email for participation, the call for participation was spread on multiple platforms by both the patient association and patients themselves with an unknown reach. Assuming that most of our included patients were members of Stichting Hoormij, our results might have faced some bias, since members of a VS association are known to report symptoms more actively than patients with VS seen in hospitals [44]. On top of that, 71% of the patients reported to have received active treatment in our study while a large population-based study by Reznitsky et al. shows that 48.4% of 3637 patients were actively treated during a mean 7.33 years of follow-up [45], limiting the generalizability of our results. However, when looking at questionnaire studies in the field of VS, actively treated patients tend to respond more to surveys (70.3–72.5%), comparable to our findings [3, 27, 46]. Yet patients who were actively treated might have had larger tumours upfront with more complaints and a higher burden, though there is a limited relation with tumour size or growth and for example hearing loss [47]. Secondly, our questionnaire did not ask patients about their tumour size since this was not expected to be accurately known. However, this may certainly be an important factor influencing symptoms, management strategy and side effects. Meanwhile, several factors might have contributed to either over- or underestimation of symptoms and self-reported side effects, as some patients mentioned having difficulty distinguishing these two, possibly caused by recall bias with a median time of 8.4 years since treatment. Yet, patients do report differences in symptoms and self-reported side effects, emphasizing the necessity for a distinction between these two. Lastly, data were collected using self-report questionnaires, which may be subject to biases such as social desirability and recall bias. In future research, incorporating qualitative methods, such as interviews or focus groups, could provide a more in-depth understanding of participants' perspectives and experiences, allowing for a richer exploration of their perceptions and insights. Additionally, conducting a pilot phase with a critical reference group to refine the study procedures, materials, and measures in the preparation, organization and reporting phase could further enhance the trustworthiness and validity of the findings [48]. By incorporating these qualitative methods and a pilot phase, future research could build on the strengths of our study and provide a more extensive and comprehensive understanding of the patient-preferred outcomes, symptoms and side effects in patients with VS, enhancing the overall trustworthiness of the findings.

Although in management of VS, priorities are shifting from tumour control towards preservation of HRQoL, there are still improvements to make when it comes to patient-centred care for patients with VS. This study, in which the majority of patients with VS was actively treated, not only showed that the well-known classical symptoms hampered patients (e.g. hearing problems, balance disturbances and energy impairments), but it, more importantly, showed that patients encountered participation difficulties in their daily physical and social activities. This ranged from exclusion in social engagements (especially conversations or activities in groups and noisy environments) to being precluded from doing their jobs and societal participation. Healthcare providers should consider these key points when managing patients with VS in consultation, treatment and follow-up.