NCCN Guidelines for Patients® | Myelodysplastic Syndromes
45 NCCN Guidelines for Patients ® : Myelodysplastic Syndromes, 2018 5 Treatment guide Lower-risk MDS without anemia Lower-risk MDS without anemia Guide 7. Treatment for lower-risk MDS without anemia Treatment options Disease progression/ no response Next options • Azacitidine or decitabine • IST for certain patients • Clinical trial ª • Consider azacitidine or decitatbine (if not taking already) ª • Clinical trial • Consider allogeneic HCT for certain patients Guide 7 shows the treatment options for patients with lower-risk MDS but without anemia. Instead, the number of platelets or white blood cells is low. Or, the number of blast cells in the bone marrow is high. Treatment options There are three main treatment options to choose from. The first option is to receive azacitidine or decitabine. Both are low-intensity chemotherapy drugs. They are also a lot alike in how they work against MDS. They can help to increase blood cell counts and lessen the need for transfusions. They can also help to slow MDS growth and progression to AML. With either drug, at least 4 to 6 cycles of treatment should be given to decide if it is working. Treatment with IST may also be an option for some patients. Your doctor will look at the features of the MDS cells and other factors to decide if this treatment might work well for you. If there is a good chance that MDS will respond to IST, then you will receive ATG (equine) with or without cyclosporine. The third option is to receive treatment within a clinical trial. Treatment results and next options Your doctor will give tests during treatment to check if it is working well to improve blood cell counts and other signs of MDS. This is called a treatment response. How long it takes to see a treatment response depends on which treatment you have. Sometimes MDS grows or gets worse during treatment. This is called progression. The next treatment options depend on how well initial treatment worked. If tests show a response, then you will stay on the same treatment as long as it keeps working. Once the MDS grows or gets worse, the next treatment options will be tried. If tests don’t show a response, or show progression, there are other options to choose from next. You may start azacitidine or decitabine, if you are not taking it already. Your doctor may consider eltrombopag or romiplostim, if thrombocytopenia is severe or the treatment is not working to control it. Another option is to receive treatment within a clinical trial. An allogeneic HCT may also be an option for some patients. But, it is only recommended for patients with intermediate-risk MDS and very low blood cell counts. Next steps If tests show progression to higher-risk MDS, see Guide 8 on page 46 for treatment options, which includes treatment with an allogeneic HCT.