NCCN Guidelines for Patients® | Myelodysplastic Syndromes
43 NCCN Guidelines for Patients ® : Myelodysplastic Syndromes, 2018 5 Treatment guide Lower-risk MDS with anemia The goal is to increase red blood cell counts and lessen the need for red blood cell transfusions. Doctors often assess the response about 2 to 4 months after the start of treatment with lenalidomide. If tests do not show a treatment response, then your next options are the same as those listed for MDS without del(5q). If tests show a response, you should continue this treatment until it stops working or side effects get too severe. Over time, your doctor may lower the dose of lenalidomide to lessen the side effects. (See page 33 for more details about lenalidomide.) Treatment options for MDS without del(5q) If tests show serum EPO ≤500 mU/mL, treatment with blood cell growth factors is recommended. The two main options are epoetin alfa and darbepoetin alfa. These drugs are red blood cell growth factors— also called ESAs. ESAs act like EPO and stimulate the body to make more red blood cells. Thus, they work best when there is a lower level of natural EPO in the blood. ESAs may be given alone or along with G-CSF. G-CSF is a white blood cell growth factor. Studies show that adding G-CSF can improve how well ESAs work for some patients. See Next steps at the end of this section. If tests show serum EPO >500 mU/mL, there are four main treatment options to choose from. The options are divided into two groups. The first group includes IST drugs and the second one does not. To decide between options, your doctor will check for factors linked with a higher chance that MDS will respond to IST. Such factors include: hypocellular bone marrow, ≤5 percent blast cells in bone marrow, and MDS cells with the HLA-DR15 protein. IST also tends to work better in patients who are 60 years of age or younger. If MDS is likely to respond to IST, then you will receive ATG (equine) with cyclosporine. If MDS is not likely to respond to IST, then you have a few other 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. The second option is to receive lenalidomide. This drug works best against MDS with the del(5q) chromosome change (described above). But, it may also be helpful for some other patients, particularly those with a normal karyotype. The third option is to receive treatment within a clinical trial. (See Part 4 for more details about each treatment.) Your doctor will give tests during treatment to check if it is working well to improve blood cell counts and other signs of MDS. How long it takes to see a treatment response depends on which treatment you have. If tests show a response, treatment may be continued until it stops working. But, treatment may need to be stopped early if side effects become too severe. This is called treatment intolerance. Next steps If tests do not show a treatment response, see Guide 6 on page 44 for the next treatment options. Guide 6 also includes the next options for if initial treatment stops working or side effects become too severe. For patients with serum EPO <500 mU/mL, see Guide 10 on page 49 and Guide 11 on page 50 for more details about anemia treatment with blood cell growth factors.