Perceptions of Disease State, Treatment Expectations, and Prognosis Among Patients with Myelodysplastic Syndromes.

Myelodysplastic syndromes (MDS) are becoming the most commonly diagnosed forms of leukemia in the US. The degree to which patients (pts) with MDS are aware of their disease severity, likely treatment (Tx) outcome and prognosis, and to which doctors have communicated this information to them is unknown. Methods: We conducted an online survey of MDS pts registered with the Aplastic Anemia & MDS International Foundation over a 2-week period in March 2009 to assess pt perceptions regarding their disease, Tx and prognosis, and differences between groups. A $15 Amazon.com gift card was offered to the first 300 respondents (acceptance constituted consent). The protocol and consent were approved by a central Institutional Review Board. The survey consisted of 55 questions assessing disease knowledge, medical evaluation, prognosis, and Tx. Baseline and objective medical data were analyzed using proportions and medians, and comparisons between groups were calculated using t-test and Cochrane-Mantel-Haenszel tests where appropriate.

Of 3131 pts invited to participate, 358 (12%) responded and completed the survey (expected proportion for an on-line survey); 183 (51%) were female, median age for all pts was 65 years (yrs; range 19–91), and 46 US states were represented. Pts were diagnosed with MDS a median of 3 yrs prior to the survey (range 0–29 yrs); 54% reported abnormal blood tests a median of 6 yrs prior to the survey (range 0–59 yrs). Only 45% of pts knew their International Prognostic Scoring System (IPSS) score, with a distribution of Low (28%), Intermediate (Int)-1 (40%), Int-2 (23%), and High (9%); 42% of pts did not know their bone marrow blast %, and 28% did not know their cytogenetic status. Cytopenias included anemia (82%), thrombocytopenia (46%), and neutropenia (45%). Of 234 pts (65%) who had a blood transfusion, 121 (52%) had one within the past 3 months and 96 (27%) were receiving transfusions at the time of the survey. Darbepoetin had been used by 148 pts (41%) and epoetin by 158 (44%). Other Txs used included antithymocyte globulin (ATG; 11%), lenalidomide (19%), azacitidine (26%), decitabine (15%), stem cell or bone marrow transplantation (11%); 48 pts (13%) had been involved in a clinical trial. Pts reported that MDS had been described to them as a “bone marrow disorder” (80%), “anemia” (56%), “blood disorder” (32%), “low white blood cell” (19%), “low platelet count” (17%), “cancer” (7%), and “leukemia” (6%). Overall, pts understanding of Tx goals and prognosis was poor; 37% of pts felt that their most current Tx would increase their chances of survival (26% of all pts saying with a >50% chance), while 36% were uncertain; 16% of pts agreed that their most current Tx could be curative (10% saying with a >50% chance), while 30% were uncertain. Prognosis was not discussed according to 35% of pts. For pts receiving any active Tx during the last 3 months (n=98) vs those receiving only growth factors or supportive care (SC; n=260), there were no significant differences in how MDS was first described, their IPSS score, or their blast % being known. Pts on active Tx, however, were 67% more likely to agree that Tx would increase their chances of survival than pts on SC (52% vs 31%; p<.001) and 69% more likely than pts on SC to agree that Tx could be curative (23% vs 14%; p=.03). However, pts on SC were 46% more likely than pts on active Tx to not have prognosis discussed (39% vs 27%; p=.03). Comparing pts with higher- (IPSS Int-2 or High) vs lower-risk (Low or Int-1) disease, agreement that Tx would increase chances of survival was 43% vs 33% (p=.20). While there were no differences in knowledge of blast % or Txs received, higher-risk pts were 2.4 times more likely to believe that current Tx would be curative (26% vs 11%; p=.01); although only 4 pts had undergone stem cell or bone marrow transplantation within 3 months.

Most MDS pts have abnormal blood tests for years preceding their diagnosis, and have high transfusion needs. Few pts describe their MDS as a cancer, and most have misperceptions or lack knowledge about Tx efficacy and overall prognosis. Pts receiving Tx other than growth factors and SC were more likely to have their prognosis discussed, and felt that their Tx could be curative and increase their chances of survival, compared with pts receiving growth factors and SC. Clear presentation of prognostic information and active pt engagement may help clear misperceptions and improve pt understanding of MDS.

Maciejewski:Taligen: Membership on an entity's Board of Directors or advisory committees; Genzyme: Research Funding; Celgene: Speakers Bureau; Eisai: Membership on an entity's Board of Directors or advisory committees. Swern:Celgene: Employment. Stone:Celgene: Speakers Bureau; Merck: Membership on an entity's Board of Directors or advisory committees; Eisai: Membership on an entity's Board of Directors or advisory committees; Genzyme: Membership on an entity's Board of Directors or advisory committees.