Myelodysplastic syndromes (MDS) are complex and confusing diseases, and healthcare providers and patients have disparate views on their characteristics and course, as well as on the value and limitations of treatment. For patients, this multiplicity of information often translates into poor adherence and response. Better education and communication may improve outcomes.
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Most MDS patients — even those with lower-risk disease at diagnosis — will die of related infectious or hemorrhagic complications. Many factors contribute to poor outcomes, including:
- Incomplete pathobiological understanding of MDS by the biomedical community
- A paucity of effective treatments
- Suboptimal use of available therapies
- Premature discontinuation of potentially effective treatments
Treatment options are limited. The only potentially curative therapy, allogeneic hematopoietic stem cell transplantation (HSCT), is attempted in less than 5 percent of cases. Epoetin alfa and darbepoetin alfa are widely used off-label to augment red blood cell production. Only three drugs are currently approved by the FDA:
- azacitidine (AZA)
- decitabine (DAC)
- lenalidomide (LEN)
These relieve symptoms and slow progression in some patients. However, they require repeated treatment cycles on specific schedules to have a clinical effect and maintain durable hematopoietic improvement. Still, health-related QoL and outcomes are poor. A median survival of less than two years is seen for patients with high-risk forms of the disease.
Usually, MDS patients aren’t aware of poor prognoses. Results from a survey conducted by the Aplastic Anemia and MDS International Foundation indicated that only 10 percent of patients report they were told by their healthcare providers that MDS were cancer. The majority reported that the diseases were described as bone marrow failure (60 percent), a blood disease (38 percent), a form of anemia (23 percent) or a pre-leukemic condition (17 percent). Only 29 percent believed MDS were ever curable. Mikkael Sekeres, MD, Director of Cleveland Clinic’s Leukemia Program, was senior author on the survey.
Among non-physician healthcare providers, 33 percent believed that MDS were curable versus 52 percent of physicians. However, treatment cycles thought to be therapeutically adequate by physicians were inconsistent with National Comprehensive Cancer Network (NCCN) guidelines for AZA, DAC, and LEN.
NCCN guidelines suggest that an adequate therapeutic trial of LEN is eight to 12 weeks, with a median time to response of five weeks. Appropriate treatment for AZA is six monthly cycles; for DAC, it’s four monthly cycles (if tolerated). By those time points, 90 percent of ADA or DAC responders will start to show a favorable response. Nonetheless, data suggest that discontinuation prior to these lengths of time is common. Physicians reported fewer than six cycles for AZA and fewer than four for DAC.
“One of the major findings of this study is that patients and doctors diverge dramatically in the reasons they report for stopping MDS therapy,” Dr. Sekeres says. “For example, physicians were twice as likely as patients to report burden of treatment or patient symptoms as a reason for therapy cessation.”
Patients also play a role in undertreatment. In 2012, only 66 percent of those prescribed LEN refilled their first prescription (i.e., stayed with therapy for at least four weeks), and only 50 percent refilled their second. Some of the early discontinuations are due to progressive disease or intolerance of therapy. However, others may be caused by inadequate education or by healthcare providers and patients giving up too soon.
Education, communication essential
The survey demonstrated that healthcare providers and patients have disparate views of the clinical courses of MDS and the value and limitations of treatment for the syndromes. Improved communication and education of all parties may increase understanding of the diseases and achieve improved treatment outcomes.
“This study clearly shows that doctors make assumptions regarding why our patients stop taking active therapies to treat their cancers,” Dr. Sekeres says. “Side effects and burdens on patients and their families may be more troubling to us than they are to our patients. We need to be better about listening to our patients before recommending that they stop cancer treatments.”