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by Dr. C.D. Buckner M.D. Medically Reviewed by C.H. Weaver 01/2022

Myelodysplastic syndrome (MDS) is curable and the prognosis is dependent on an age, overall health, and specific genetic or cytogenetic abnormalities. Treatment is designed to manage the complications associated with ineffective blood cell production, extend survival, and cure the disease when possible.

Treatment of MDS is individualized and depends on:

  1. The severity of low blood counts
  2. The risk of progression to acute myeloid leukemia
  3. Genetic abnormalities detected on cytogenetic analyses
Myelodysplastic Syndrome MDS

The potential treatment options for MDS include the following:

  • Destruction of abnormal cells through administration of chemotherapy, at either low, conventional, or high doses, depending on the condition of the patient and the aggressiveness of their disease.
  • Supportive care through administration of growth factors to stimulate immature cells to development into mature blood cells.
  • Replacement of damaged bone marrow with healthy cells that develop into blood cells, a procedure called stem cell transplantation
  • Precision medicines or immunotherapies
  • Participation in a clinical trial.

Currently, only stem cell transplant utilizing cells from a donor—called an allogeneic transplant—can consistently cure patients with MDS. Other therapies are directed at prolonging survival and decreasing the symptoms.1,2

Planning Treatment for Myelodysplastic Syndrome

Since a stem cell transplant utilizing cells from a donor—called an allogeneic stem cell transplant—holds the most hope for cure, a major decision faced by some patients with MDS is not whether to undergo transplantation, but when. Patients with MDS that is likely to progress to leukemia early—which results in shorter survival—may be willing to accept higher risks of treatment and proceed quickly to a stem cell transplant. Patients with MDS that progresses more slowly are likely to live longer and may wish to pursue a more conservative treatment approach, opting to use supportive care and wait for the development of new treatments or a longer period before undergoing a stem cell transplant.

Patients who choose conservative treatment approaches should always be prepared to receive more aggressive treatment in case their disease progresses more rapidly than anticipated. To prepare for a possible stem cell transplant, patients should consider arranging for a stem cell donor and/or having their own stem cells collected and stored shortly after diagnosis. This is important because as MDS progresses and treatment is initiated, it becomes increasingly difficult to collect stem cells.

In order to better plan treatment, doctors try to identify how quickly patients are likely to progress to acute myeloid leukemia (AML). A score is assigned that reflects this tendency to progress and is based on a system called the International Prognostic Scoring System (IPSS). A higher score is associated with a type of MDS that is likely to progress to leukemia more quickly. The IPSS score takes into account three important factors in MDS:

  • The percent of bone marrow blasts—more blasts contributes to a higher score
  • Genetic (cytogenetic) abnormalities—more abnormalities contribute to a higher score
  • Severity of low white blood cell counts—lower counts of white blood cells, platelets, and red blood cells contribute to a higher score

Relationship between a patient’s risk of progressing to leukemia and timing of stem cell transplant:

Research shows that knowing a patient’s risk of progressing to leukemia is important for determining optimal timing of stem cell transplantation. Based on information about 1,000 patients who had been diagnosed with MDS, researchers from several U.S. cancer centers have determined that patients with a low or low-intermediate risk of progression to leukemia have better outcomes if their transplant was not performed at the time of diagnosis but was delayed. Patients with a high or high-intermediate risk experienced optimal survival if they underwent an allogeneic transplant at the time of diagnosis, without delay. Furthermore, the patients with lower risk achieved optimal outcomes if their transplant was administered prior to progression of their disease to acute myeloid leukemia compared to after progression.3c

Genetic Profiling Guides Stem Cell Transplantation 

Based on an analysis of blood samples from 1,514 patients with MDS investigators from the Center for Blood and Marrow Transplant Research have determined that using a single blood test to genetically profile a patient’s blood cells and basic information about a patient’s medical status can also indicate which patients likely to benefit from a stem cell transplant.4

  • Blood cells that carry a mutation in the gene TP53 tent to survive for a shorter time after a transplant, and also relapse more quickly, than patients whose cells lacked that mutation.
  • Individuals with RAS mutations have reduced survival and increased risk of only when treated with reduced-intensity conditioning. These patients may benefit from higher intensity conditioning regimens, the researchers indicated.
  • TP53 mutations and mutations in PPM1D, a gene that regulates TP53 function, were far more common in patients with MDS that resulted previous cancer treatment.

These and other factors will continue to be identified and help determine which treatment is most appropriate.

Supportive Care with Growth Factors

While patients may not initially experience symptoms of their condition, the ineffective blood cell production that characterizes MDS eventually results in uncomfortable and life-threatening side effects. Low red blood cell production results in anemia and associated fatigue; low white blood cell production causes neutropenia, which increases the risk of infection; and low platelets causes thrombocytopenia and an increased risk of bleeding.

Patients who are at low risk of progressing to acute myeloid leukemia may not have any of these symptoms or they may require supportive treatments in order to prevent the complications associated with low blood counts. Several strategies exist for improving blood cell production and decreasing complications:5-8

Anemia: Anemia can be treated with a red blood cell transfusion or by increasing red blood cell production with a naturally produced protein called erythropoietin. Erythropoietin stimulates the bone marrow to produce more red blood cells. When administered to some patients, it reduces the severity of anemia and can prevent red blood cell transfusions. Currently, there are two forms of erythropoietin available, epoetin alfa (Epogen® or Procrit®) and darbepoetin alfa (Aranesp®). Aranesp is a longer acting form that can be administered less frequently. Clinical trials have shown that Aranesp improves anemia and quality of life in patients with low or intermediate-risk MDS.

Neutropenia: Neutropenia, or a low white blood count, increases a patient’s risk of infection. The white blood count can be temporarily increased with white blood cell growth factors which stimulate the production of white blood cells. This can decrease the chance of additional or worsening infections.

Thrombocytopenia: Drugs referred to as “platelet growth factors” such as (Nplate) (romiplostim) and Promacta (eltrombopag) might help some people with MDS who have very low platelet levels, although this is still being studied. Neumega (interleukin-11, IL-11, can also be used to raise platelet counts after chemotherapy and in some other diseases. But none of these drugs are specifically approved for primary treatment of MDS.

AML Newsletter 490

Immunotherapy and Precision Cancer Medicines

The immune system is a network of cells, tissues, and biologic substances that defend the body against viruses, bacteria, and cancer. The immune system recognizes cancer cells as foreign and can eliminate them or keep them in check—up to a point. Cancer cells are very good at finding ways to avoid immune destruction however, so the goal of immunotherapy is to help the immune system eliminate cancer cells by either activating the immune system directly or inhibiting the mechanisms of suppression of the immune system. There are several substances that generally boost, direct or restore normal immune defenses and include interferons and interleukins.

Not all cancer cells are alike - they may differ from one another based on what genes have mutations. The purpose of precision cancer medicine is to define the genomic (genetic) alterations in a cancers DNA that are driving that specific cancer. Precision cancer medicine utilizes molecular diagnostic testing, including DNA sequencing, to identify cancer-driving abnormalities in a cancer’s genome.

Precision cancer medicine uses targeted drugs and immunotherapies (monoclonal antibodies, vaccines) engineered to directly attack the specific abnormalities causing the cancer and leave normal cells largely unharmed. Precision cancer medicines and immunotherapy can be used both instead of and in addition to chemotherapy to improve treatment outcomes.

Revlimid (lenalidomide) is thought to regulate the immune system and cause anticancer effects by killing abnormal white blood cells, reducing inflammation, and inhibiting growth of new blood vessels. Revlimid is effective in the treatment of patients with low - risk MDS and a specific cytogenetic abnormality, partial loss of chromosome 5 (5q-). Response to treatment is reported to be rapid and long-lasting. Patients experienced an increase in hemoglobin, a component of red blood cells, within approximately one month (4.4 weeks) after the start of treatment.9,10

Antithymocyte globulin (ATG): Administering a drug that suppresses the immune system—a technique called immunosuppression—appears to provide some benefit in the treatment of MDS. Clinical trial results suggest that over 43% of patients with MDS respond to immunosuppression with ATG. This response lasted nearly two and a half years (29 months) on average.11,12

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High-Dose Therapy with Stem Cell Transplant

Without functioning stem cells in the bone marrow, the body cannot produce red blood cells, white blood cells, or platelets, bone marrow function however can be restored by replacing the damaged stem cells with healthy ones. This is a procedure known as a stem cell transplant.

There are two possible sources of stem cells for transplantation; they may be collected from the patient prior to undergoing high-dose therapy or they may be collected from a donor. A stem cell transplant that utilizes the patient’s own cells is called an autologous stem cell transplant. When the stem cells are from a donor the procedure is called an allogeneic stem cell transplant. Currently an allogeneic stem cell transplant offers the greatest chance for cure.

Hematopoietic Stem Cell Transplant is a standard and potentially curative treatment resulting in 3 to 4 year survival rates of 30% to 40%.1,2,13,14 Side effects and mortality from a SCT can be significant however a recent research report revealed that age alone doesn’t affect survival outcomes for MDS patients undergoing SCT. The Center for International Blood and Marrow Transplant Research compared patients aged 65 and older to those aged 55 to 64 with MDS who receive a SCT and found similar outcomes.15


Individuals with MDS who are unable or unwilling to undergo a SCT using donor cells or those planning to have their own stem cells collected for a future transplant can receive a variety of treatments not requiring stem cell support. Many patients can achieve a remission but most ultimately experience disease progression. While some patients may experience a long remission following therapy, remissions after conventional chemotherapy typically average less than 12 months.

Vidaza® (azacitadine): was the first drug to be approved specifically for the treatment of MDS. Approximately 15-20% of patients will have an anticancer response, improved quality of life, and no longer required blood transfusions to maintain appropriate hemoglobin levels.17

Fludarabine plus cytarabine: Physicians from Italy have reported that the chemotherapy regimen consisting of fludarabine and cytarabine with the white blood cell growth factor, Neupogen® (filgrastim), produced remission in three-quarters of patients with advanced MDS. The 42 patients involved in this study survived 13 months, on average. Nine percent of patients died from complications of the treatment.18

Vyxeos: The U.S. Food and Drug Administration approved Vyxeos (liposome-encapsulated combination of daunorubicin and cytarabine) for the treatment of AML and MDS based on a study showing it prolonged survival compared to a standard combination of daunorubicin and cytarabine.19

Dacogen™ (decitabine): FDA approval for Dacogen was prompted by results of clinical trials demonstrating that it effectively improved outcomes in patients with MDS when compared to supportive care. Overall, Dacogen provided anticancer responses in approximately 17%-26% of patients in trials-demonstrating a significant improvement over supportive care measures and reducing the need for blood transfusions in a significant portion of patients.20

Topotecan/cytarabine may be more tolerable than standard idarubicin/cytarabine: A clinical study that involved 510 patients and compared four different chemotherapy combinations has revealed that anticancer responses and duration of survival were the same for all four treatments. However, treatment with topotecan/cytarabine resulted in fewer deaths than other regimens and may be considered an alternative to the standard idarubicin/cytarabine combination for the treatment of patients with progressive or high-risk MDS.2

Strategies to Improve Treatment

The current treatments for MDS remain limited and new therapies are being advanced. There is an increased need for patients to participate in clinical trials to change this therapeutic stagnation. Several promising treatment approaches are under investigation and include some of the following.

Researchers are working on improving stem cell transplant in several ways; all patients with MDS should be evaluated for SCT at a transplant center with a dedicated research interest in MDS because many new treatment strategies are being developed and only physicians at those centers can speak to the progress being made.

  • Autologous Stem Cell Transplant utilizes the patient’s own stem cells thereby eliminating the need to find an appropriate stem cell donor. High-dose chemotherapy and autologous stem cell transplant had not been widely used to treat MDS because stem cells collected from the patient with myelodysplasia were thought to be abnormal. Results of a clinical trial however have demonstrated that ~27% of patients with MDS or AML secondary to survived cancer-free four years after transplant was similar for both types of transplant; patients who experienced a complete remission during therapy and received their transplant while in remission had slightly better outcomes.2
  • Reduced-intensity Transplants: A reduced intensity or mini transplant involves lower doses of therapy prior to allogenic stem cell transplant and may allow more older patients to benefit from treatment. Mini-transplants are associated with fewer side effects and a lower treatment-related mortality than regular allogeneic stem cell transplants, while still providing the benefit of donor stem cells that can attack the patient’s cancer cells.13,14
  • Umbilical cord transplantation: Results from a clinical trial conducted in Japan suggests that an umbilical cord transplant may be an effective option for patients with myelodysplastic syndrome who are eligible for an allogeneic stem cell transplant but cannot find a suitable donor. However, the major disadvantage of using umbilical cord blood is the low number of stem cells collected, which has limited the use of this technique, particularly in larger patients who require more stem cells.16

Luspatercept (ACE-536): In a phase 2 trial 63% of 51 patients treated with higher-dose luspatercept achieved hematological improvement-erythroid and 38% of 42 patients eligible for RBC transfusion independence. Luspatercept was approved by the US Food and Drug Administration (FDA) for lower risk MDS in April 2020.22-24 

Imetelstat is a telomerase inhibitor - In one study in myelofibrosis patients Imetelstat achieved complete responses in the setting of U2AF1 or SF3B1mutations.35

Iron chelation therapy; is a key component of managing lower risk MDS patients with transfusion requirement. The randomized, double-blind, phase 2 TELESTO trial evaluated the iron chelator deferasirox compared to placebo in 225 adults diagnosed with low/intermediate-1 MDS who had iron overload. Patients in the deferasirox group experienced significantly longer median event-free survival compared with the placebo group; 1440 days vs 1091 days, however, the median overall survival was not significantly different between the two groups.25

Guadecitabine (SGI-110): In a phase 2 study focusing on patients with higher risk MDS and AML with 20% to 30% blasts, all of whom had previously failed azacitidine, the overall response rate with guadecitabine was 14.3% with an overall survival rate of 7.1 months. None of the 11 patients with TP53 mutation responded to treatment.26-27

Enasidenib is an inhibitor of mutant IDH2 protein and was granted FDA approval in 2017 for treatment of patients with relapsed or refractory AML with an isocitrate dehydrogenase 2 (IDH2) mutation which are present in approximately 5% of patients with MDS. In a phase 1 study of patients with IDH2-mutant MDS, more than half of the patients had a hematologic response with an overall response rate of 53%.28.29

Immunotherapy It has been reported that hypomethylating therapies may increase the expression of programmed death 1 (PD-1), programmed death ligand 1 and cytotoxic T-lymphocyte-associated antigen 4 (CTLA-4) in MDS CD34+ cells. Single-agent ipilimumab was capable of inducing responses in previously treated MDS patients, though single-agent nivolumab showed no clinical response. In a phase 1b study, pembrolizumab, a humanized monoclonal antibody against PD-1 has also shown activity.20,31

BCL2 inhibition: Clinical trials have shown that BCL-2 blockade by venetoclax alone and in combination with cytarabine induce responses in patients with AML, aged 65 and older and those not fit for intensive chemotherapy. Patients with mutations of NPM1, IDH1/2, and genetic alterations of chromatin-RNA splicing were also noted to have a better response to the venetoclax-based regimens.32-34

Chimeric antigen receptor (CAR) T-cell therapy

CAR therapies utilize T-cells (CAR T) which are a patient’s own immune cells that are re-programmed to recognize and kill cancer cells throughout the body. The process involves the removal of some T cells from a patient, and through laboratory processes these T cells are re-programmed to identify a patient’s cancer cells.

Once the T cells have been programmed to identify a patient’s cancer cells, they are replicated in the laboratory in very large numbers and infused back into the patient. The re-programmed T cells circulate throughout the body where the identify cancer cells and facilitate an immune attack against them. Simultaneously, the T cells are also replicating within the body, so that more of the immune cells can identify and attack the cancer cells. CAR-T cell therapy is being evaluated as a treatment in MDS.36


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