Differences In Perceptions Of Disease and Treatment Effectiveness and Adherence Between Physicians and Patients With Myelodysplastic Syndromes (MDS)

MDS are complex conditions, described with sometimes confusing terminology (e.g., “refractory anemia”), and contemporary drug therapies (tx) for MDS require repeated administration cycles to achieve clinical effect. Lack of disease understanding or premature tx discontinuation may result in poorer outcomes for patients (pts). To better understand physician (MD) and pt perceptions about MDS and tx decisions, we conducted two online surveys: one for pts with MDS and one for healthcare providers (HCP) registered with the non-profit Aplastic Anemia & MDS International Foundation. The protocol and consent were approved by a central Institutional Review Board. Pt and HCP surveys consisted of 57 and 49 questions, respectively, and assessed understanding of MDS, perceptions of specific tx, barriers to tx adherence, and overall tx experience. Data were analyzed using proportions, means, and medians; groups were compared using a Chi-squared test.

Of 4,039 pts invited to participate via e-mail, 477 (12%) complete responses were received from 42 US States. Of responders, 247 (52%) were men; 63% were ≥age 60; pts were diagnosed with MDS a median of 5 years prior to the survey (range, 0-32 years). Of 4,594 HCPs invited to participate, 120 (3%) complete responses were received. Due to low participation among other HCP groups, only MD responses were examined. Of the 61 MDs (from 23 US states), 35 (57%) practice in an academic setting and 26 (43%) in the community setting. Survey responses from self-designated academic and community MDs did not differ significantly. Among MDs, 48% reported they see 5-19 new MDS pts per year. Only 10% of pts reported MDS was described to them as “cancer”, compared to how 59% of MDs stated they described it (p<.001). Only 29% of pts reported that MDS is “curable”, compared to 52% of MDs (p<.001). Forty-two percent of pts had received at least one disease-modifying tx: azacitidine (AZA, 58%), decitabine (DAC, 27%), lenalidomide (LEN, 35%) or hematopoetic stem cell transplant (HSCT, 26%) (total >100% due to multiple answers).

MD and pt perceptions of active tx were significantly different, with MDs overestimating quality of life (QOL) benefits and underestimating the burden of tx on pt activities. [Table 1]

MDs interpreted the benefit of active tx significantly higher than pts, however pts perceived the actual tx experience more positively than MDs. [Table 2]

Most pts (81%) reported that MDs had the most influence on their tx decisions. Sixty-nine percent of MDs reported recommending stopping tx prior to the completion of tx regimen. Reported reasons diverged between pts and MDs, including burden of tx exceeding the benefit to the pt, as well as perceptions that the impact on the pt and family was too great. [Table 3]

Physicians and pts with MDS have distinct views of the value of tx for MDS, with MDs underestimating the impact of tx on pt QOL while overestimating it as justification for stopping tx. Improved communication may improve understanding of disease and impact of active treatment and achieve better tx adeherence and responses.

No relevant conflicts of interest to declare.