Myelodysplastic syndromes (MDS) are a group of blood disorders characterized by the ineffective blood production, increased risk of infection, and chance of progression to acute myeloid leukemia (AML). Disease severity can range from mild MDS, with low impact on a patient's quality of life, to severe MDS, with severely low blood counts, recurrent infections, need for transfusions and increasing risks of progression to AML. Blood and marrow transplant (BMT) is currently the only cure for MDS patients; however, the clinical heterogeneity of MDS makes candidate identification and optimization of transplant timing a challenge.
Physicians in our BMT program individualize treatment plans for MDS patients by evaluating not only the disease biology, but also the patient’s overall health and goals for treatment. Upon diagnosis, MDS patients are classified into groups ranging from low to very high risk to predict prognosis and the natural history of their MDS. For those patients who are elderly or who are not good candidates for transplant therapy, numerous chemotherapy choices exist including azacitidine, decitabine, and lenalidomide with the preferred options depending on the features of their MDS. For those patients whose treatment goal is potential cure and are transplant candidates, the preferred timing for transplant is then determined. Given the potential complications of BMT, some patients with lower risk MDS have better outcomes if transplant is delayed until their disease progresses further, whereas higher risk MDS patients benefit with planned transplant early in their disease course. Careful collaboration with the patient’s referring physician to monitor their disease course can help determine the best timing for transplantation.
Patients are determined to be eligible for transplant based on their general health and multi-organ function including measures of any heart, kidney, or liver disease. BMT is performed on patients up to age 75; however, even more stringent criteria are used for patients 70-75 years old, given somewhat lesser resilience in this group to withstand transplant complications.
If a patient’s disease characteristics, health, and treatment goals warrant them eligible for BMT, the timing of transplant is discussed; often in collaboration with their referring physician. BMT is more effective if patients have fewer blasts in their bone marrow. Therefore, many patients will require therapy prior to BMT to reduce their disease burden. Options include outpatient therapy with hypomethylating agents such as azacitidine or decitabine, or inpatient therapy with AML-type induction chemotherapy. Physicians and patients discuss these options together to determine the ideal treatment plan based on the patient’s disease severity and personal preferences. Outpatient therapy may require months of treatment and is suitable for patients whose disease biology would not outpace the possible response, whereas the more intensive induction chemotherapy is best for high-risk MDS patients with more aggressive disease features. Tailoring the choices and timing of treatment to limit toxicities and achieve the best outcomes is a regular part of developing any patient’s treatment plan.
-Contributed by Erica Warlick, MD
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