Disease perception, quality of life and mental health among Chinese patients with transplant-eligible newly diagnosed multiple myeloma following treatment: a cross-sectional survey study

This cross-sectional survey study was conducted between January and June 2025 at two tertiary hospitals in China. This study received ethical clearance from the ethics committee of the study hospital, and all participants provided written informed consent prior to enrollment. The research was performed in strict accordance with the ethical principles outlined in the Declaration of Helsinki and complied with all relevant national guidelines for clinical research involving human subjects. This study was reported following the Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) reporting guideline for observational studies.

A total of 59 transplant-eligible patients with NDMM after induction therapy were initially screened for eligibility. After excluding two patients whose interval between ASCT and survey completion was less than 1 year, 57 eligible patients were invited to participate in the survey. Among them, four patients declined participation. Ultimately, 53 patients were included in the final analysis and constituted the study cohort. The detailed inclusion and exclusion flow is presented in Supplementary Figure 1.

In this cross-sectional survey of transplant-eligible Chinese patients with NDMM after induction therapy, responses to individual disease-perception items varied, with notable knowledge gaps regarding less apparent complications. HRQoL was relatively preserved overall. Anemia was associated with lower EQ-5D utility, while progressive disease was associated with greater anxiety and depressive symptoms. Patients in the non-ASCT group also reported more financial and access-related treatment challenges. These findings highlight areas that may warrant greater attention in patient education, symptom management, psychological support and the assessment of treatment-related challenges.

The disease-perception results showed a clear contrast between greater recognition of symptomatic or visible manifestations and lower awareness of less apparent organ involvement. Most patients recognized bone pain, fatigue, pathological fractures, infection risk and long-term functional impairment, which is consistent with the high prevalence of pathological fractures in the cohort and with qualitative studies showing that patients with MM often focus on symptoms that directly disrupt daily life [11,17]. In contrast, only half of participants recognized renal impairment as a disease-related risk, despite renal dysfunction being a core component of MM-related end-organ damage. This pattern may differ from clinician-centered descriptions of MM because patients may base their understanding of the disease primarily on experienced symptoms rather than laboratory abnormalities or occult organ injury. These findings highlight the potential value of structured education that explicitly links less apparent complications, such as renal impairment and anemia, to disease management and treatment decisions.

The mean EQ-5D utility score of 0.849 and EQ-VAS score of 71.8 suggest better overall HRQoL than might be expected in broader real-world MM populations, but this difference is plausible given the study design. Unlike studies enrolling patients across disease stages, treatment lines, relapse states or frailer clinical profiles [10,11], this cohort was restricted to transplant-eligible patients who had completed induction therapy and achieved at least a partial response. Only patients who survived and were able to complete the survey were included in the final analysis. Patients surveyed during acute peri-transplant recovery were also excluded. These eligibility features may have resulted in selection toward patients with better performance status and more stable post-treatment functioning, potentially contributing to the relatively favorable HRQoL observed in this cohort. At the same time, anemia was the variable significantly associated with lower HRQoL in the multivariable analysis, which is consistent with prior work emphasizing the central role of anemia-related fatigue and physical limitation in MM-related quality of life [18]. This finding suggests that routine assessment of anemia-related symptom burden and appropriate management of hematologic abnormalities may warrant greater attention in clinical practice, particularly given the potential contribution of anemia to patients’ perceived health status. Whether timely correction of anemia can translate into meaningful improvements in HRQoL, however, remains to be determined in longitudinal or interventional studies. Differences from rehabilitation or survivorship studies may also reflect differences in instruments, timing of assessment, symptom burden and supportive-care exposure [19].

The mental health findings also require interpretation in relation to the cohort and measurement approach. The proportions of patients with moderate-to-severe anxiety and mild-or-greater depressive symptoms were clinically meaningful, but may appear lower than reports emphasizing the broad unmet psychological needs of people living with MM [12]. Several factors may explain this difference, including use of GAD-7 and PHQ-9 threshold-based classifications, assessment after induction rather than at diagnosis or relapse, tertiary-center follow-up and exclusion of patients in the acute post-ASCT period. Nevertheless, progressive disease was significantly associated with both anxiety and depressive symptoms in the multivariable analyses, which is consistent with literature linking fear of progression, fatigue, impaired functioning and poorer global quality of life among hematologic cancer survivors [20]. However, the logistic regression estimates were imprecise, as reflected by the wide confidence intervals for moderate-to-severe anxiety and mild-or-greater depressive symptoms. Given the small number of outcome events, these estimates should be regarded as exploratory rather than as robust estimates for clinical prediction. Nevertheless, the observed associations highlight the potential importance of psychological screening among patients experiencing disease progression, while requiring confirmation in larger prospective studies.

The ASCT and non-ASCT subgroup findings were exploratory and should be interpreted as patient-reported signals rather than causal effects of transplantation. ASCT recipients were younger, had longer disease duration, were less likely to be covered by Urban Resident Basic Medical Insurance, and were more likely to report no major treatment challenges. Non-ASCT patients more frequently reported high treatment costs, limited reimbursement, adverse effects or drug toxicity and poor long-term adherence. These findings describe differences in patient-reported treatment experiences and preferences between the two treatment pathways and should not be interpreted as evidence that these factors determined whether patients underwent ASCT. Although treatment pathway was included as a covariate in the multivariable regression models, the clinical and treatment heterogeneity within the non-ASCT group could not be fully accounted for given the small sample size. Published studies of ASCT often describe an early deterioration in HRQoL during transplantation followed by recovery over time [21]. In our study, patients surveyed within 1 year after ASCT were excluded, so the assessment likely captured a more stable survivorship phase. In addition, baseline differences between groups, together with the small sample size, limit the interpretability of between-group differences in PROs and preclude conclusions regarding treatment effects.

The financial findings also differ from many Western cost-effectiveness studies, in which ASCT is often evaluated as a clinically effective and economically favorable strategy over longer horizons [22,23]. In the present Chinese tertiary-care cohort, however, the patient-reported barrier was not only whether ASCT is cost-effective at a system level, but whether patients could afford upfront costs and whether insurance reimbursement was sufficient at the point of care. This distinction may provide context for understanding why some transplant-eligible patients did not proceed to ASCT despite guideline-supported treatment pathways and evidence that ASCT and maintenance strategies can extend disease control [24–26], although these patient-reported treatment challenges should not be interpreted as established determinants of treatment selection. The high frequency of reported financial burden among non-ASCT patients is also consistent with emerging evidence that financial toxicity affects outcomes in hematologic malignancies and may be modifiable through targeted financial-support interventions [27]. Taken together, these findings suggest that differences between this study and published PRO studies may reflect not only clinical status and assessment timing, but also health-system financing, reimbursement design and patient-level affordability.

The findings of this study carry several important implications for clinical practice in transplant-eligible patients with NDMM in China. First, structured patient education should be strengthened at the time of diagnosis and during post-induction treatment, with particular emphasis on silent but prognostically critical complications such as renal impairment. Improving patients' understanding of long-term organ damage and the potential benefits and limitations of ASCT may facilitate informed discussions and shared decision-making regarding treatment options. Second, routine assessment of anemia-related symptom burden and timely management of hematologic abnormalities may help improve HRQoL. This is particularly relevant because anemia was associated with worse patient-perceived health status in our cohort. Third, systematic psychological assessment using brief tools such as GAD-7 and PHQ-9 should be integrated into standard MM care, particularly for patients experiencing disease progression, who appear to be at markedly elevated risk for anxiety and depressive symptoms. These findings underscore the potential value of early psychological assessment and supportive care, which may help preserve treatment adherence and resilience during intensive therapy. Finally, given the substantial financial barriers identified among non-ASCT patients, multidisciplinary care models that incorporate social work consultation and financial counseling are warranted. At a broader level, these findings highlight the potential importance of insurance reimbursement and financial-support mechanisms in addressing patient-reported financial challenges and may warrant further evaluation as part of efforts to improve equitable access to guideline-recommended care.

This study has several limitations that should be considered when interpreting the results. First, the cross-sectional design precludes causal inference between clinical factors, such as disease progression or anemia, and observed quality of life and psychological outcomes. Although multivariable adjustment was performed, residual confounding cannot be excluded because relevant factors may not have been measured or adequately captured. Longitudinal studies are required to track how these perceptions and mental health states evolve throughout the treatment continuum. Second, the sample size was relatively small (N = 53) and limited to two tertiary hospitals in a single province, which may restrict generalizability to the broader Chinese population, particularly patients treated in lower-tier hospitals or rural areas with different access to care. Third, restricting the study population to patients who had achieved at least a partial response and survived to complete the survey may have introduced selection bias. This selection may have contributed to the relatively favorable HRQoL observed in our cohort compared with broader MM populations. In addition, excluding patients surveyed within 1 year after ASCT may have introduced survivor bias and time-related selection bias.This may have led to underestimation of the early adverse effects of ASCT on HRQoL and mental health and may have made the ASCT group appear more recovered. Moreover, the ASCT and non-ASCT groups differed in baseline characteristics, including age and disease duration, and treatment allocation was not randomized. Accordingly, these exploratory subgroup differences should be interpreted descriptively rather than as evidence of treatment effects. The non-ASCT group may also have comprised patients with diverse clinical and treatment characteristics. Although treatment pathway was included as a covariate in the multivariable regression models, the small sample size limited our ability to account more comprehensively for this within-group heterogeneity. Fourth, all PROs were based on self-administered questionnaires, which may introduce recall or social desirability bias. Disease-perception and treatment-challenge items were self-developed and not formally psychometrically validated, limiting comparability with validated disease-knowledge or financial-toxicity instruments. Fifth, the regression analyses should be interpreted cautiously given the relatively small sample size (N = 53) and limited number of outcome events, particularly for disease progression, moderate-to-severe anxiety and depressive symptoms. The low events-per-variable ratio in some logistic regression models may have increased the risk of overfitting and contributed to wide confidence intervals and uncertainty in effect estimates. Diagnostic analyses of the linear regression models did not indicate obvious violations of the assessed model assumptions, although some effect estimates varied in sensitivity analyses excluding potentially influential observations. Therefore, these regression findings should be considered exploratory and require validation in larger prospective cohorts. Missing data were minimal, with only one patient lacking initial ISS stage data, while all variables included in the regression models were complete; thus, missingness was unlikely to have materially influenced the regression estimates. Potentially unmeasured factors, including social support, caregiver availability, treatment regimen, coping style and objective financial toxicity, may therefore have contributed to residual confounding. Finally, although we identified perceived financial barriers, detailed objective cost, income, reimbursement and out-of-pocket expenditure data were not collected. Future research incorporating objective economic data alongside patient-reported financial toxicity would provide a stronger basis for health policy recommendations.

In conclusion, transplant-eligible patients with NDMM in China showed substantial awareness of symptomatic and visible disease manifestations, but important knowledge gaps regarding less apparent complications, such as renal impairment, persisted. Anemia was significantly associated with lower HRQoL, while progressive disease was associated with higher anxiety and depressive symptoms. Exploratory subgroup findings suggested that patients who did not undergo ASCT reported greater perceived financial burden and medical insurance limitations, but these findings should be interpreted with caution given baseline differences between groups, limited sample size and the subjective nature of patient-reported economic measures. These findings highlight the potential value of patient-centered care integrating psychological screening, targeted symptom management and structured disease education, while the effectiveness of specific interventions warrants further prospective evaluation.

J Meng, X Liu and W Chen formulated the study concept. Y Zhang, J You and C Chen developed the study protocol. Y Zhang, J You, Y Meng, Y Zhao, H Sun, J Wang and GS Sah conducted patient enrollment, collect patient consent, patient interview and data collection from medical records. C Chen conducted data analysis. Y Zhang and J You drafted the manuscript under the guidance of J Meng, X Liu and W Chen. All authors critically reviewed and revised the manuscript and approved the manuscript submission.

The authors thank all the patients who participated in this survey study for their valuable contributions. The authors also acknowledge the medical and nursing staff, as well as the research teams at the participating tertiary hospitals, for their support in facilitating participant recruitment and data collection.

This work was supported by a grant from the International Science and Technology Cooperation/Hong Kong–Macao–Taiwan Science and Technology Cooperation Program of the Hebei Provincial Department of Science and Technology (grant no. 25297702D).

The authors have no competing interests or relevant affiliations with any organization or entity with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

No funded writing assistance was utilized in the production of this manuscript.

The study received ethical clearance from the ethics committee of the study hospital, and all participants provided written informed consent prior to enrollment. The research was performed in strict accordance with the ethical principles outlined in the Declaration of Helsinki and complied with all relevant national guidelines for clinical research involving human subjects.

This manuscript reports the results of a cross-sectional survey study. The study protocol was not publicly registered. The datasets generated and/or analyzed during the current study are not publicly available due to ethical and privacy restrictions. The anonymized datasets, analytic methods, and study materials are available from the corresponding authors upon reasonable request.

This work is licensed under the Attribution-NonCommercial-NoDerivatives 4.0 Unported License. To view a copy of this license, visit https://creativecommons.org/licenses/by-nc-nd/4.0/