Myelodysplastic Syndromes and Their Treatment Options: A Guide for Pharmacists

Myelodysplastic Syndromes

Myelodysplastic syndromes (MDS) are a group of diseases caused by blood cells that are not formed completely or do not work as they should. This syndrome has to do with red blood cells production in the bone marrow.¹

3d image of bone marrow | Image credit: © CHA | stock.adobe.com

An estimated 10,000 individuals in the US are diagnosed with MDS each year. It is uncommon to be diagnosed with this disease before age of 50, but the risk increases as individuals age. MDS is mostly commonly diagnosed among individuals in their 70s.² MDS has several types, including MDS with single-lineage dysplasia, MDS with multilineage dysplasia, MDS with ring sideoblasts, MDS with isolated chromosome abnormalities, MDS with excess blasts, and MDS that are simply unclassifiable.¹

The management of MDS is primarily targeted at controlling or slowing the disease and preventing future complications as much as possible. Some of these interventions include blood transfusions, which could help boost blood cell production. If appropriate, some patients may receive stem cell transplants to replace the diseased bone marrow with healthy bone marrow cells from the donor.¹

Individuals with MDS might not present with any symptoms in the beginning stages of the disease, but as the disease progresses patients may begin to develop symptoms. These symptoms may vary from person to person and may include fatigue, shortness of breath, unusual paleness, unusual bruising or bleeding, and red spots on the skin caused by petechiae. Some patients may also experience frequent infections due to low white blood cell count, also called leukopenia.¹

Patients with MDS do not have a proper process of making red blood cells in the bone marrow. In these patients, for a number of reasons, the process of building red blood cells is disrupted, causing the red blood cells to die in the bone marrow or just after entering the bloodstream. Over time, repeating this process causes the individual to have less healthy red blood cells and present with symptoms of fatigue, anemia, leukopenia, and bleeding problems. Some patients have MDS because of genetic factors, whereas some experience the syndrome due to chemotherapy, radiation, or toxic chemicals, or a number of other unknown reasons.¹

Treatments for MDS

Treatment of MDS includes medications to help increase the blood cells in the body, treatments to stimulate the blood cells to mature, drugs to suppress the immune system, and especially treatments to treat the infections.¹ Some of these include hypomethylating agents, such as azacitidine (Vidaza; Bristol Myers Squibb), decitabine (Dacogen; Otsuka America Pharmaceutical, Inc), or the combination of decitabine and cedazuridine (Inqovi; Taiho Oncology, Inc). Immunomodulating drugs may include thalidomide (Thalomid; Bristol Myers Squibb) or lenalidomide (Revlimid; Bristol Myers Squibb) and immunosuppressant treatment options include anti-thymocyte globulin and cyclosporine (Neoral; Novartis).⁴ In some patients, erythropoiesis stimulating agents (ESAs) are also given to increase the number of mature red blood cells made in the body and to reduce anemia. Granulocyte colony-stimulating factors may also be given with ESAs.³

Depending on the patient-specific factors such as age, limiting factors, and advancement of disease, the choices of these therapies or their combinations may vary. Adverse effects of these therapies can include hair loss, mouth sores, loss of appetite, nausea, vomiting, diarrhea, and constipation.

Bone marrow transplant, also known as stem cell transplant, is another option for those candidates who are qualified to receive it. This is the only current treatment option to potentially cure the syndrome. However, this option carries major risks of complications, and it is generally reserved for those who are young and healthy enough to go through the process. During such transplant, high doses of chemotherapy drugs are used to clear out the defective blood cells from the bone marrow before replacing them with the donated healthier cells.1

Depending how early the diagnosis is and how healthy the patient is, there are various treatment options available. Supportive care plays a key role in helping these patients go through the stages of their treatment courses for MDS.

REFERENCES

1. Myelodysplastic syndromes. Mayo Clinic. October 25, 2024. Accessed December 3, 2024. https://www.mayoclinic.org/diseases-conditions/myelodysplastic-syndrome/symptoms-causes/syc-20366977

2. Key Statistics for Myelodysplastic Syndromes (MDS). American Cancer Society. Updated November 21, 2024. Accessed December 3, 2024. https://www.cancer.org/cancer/types/myelodysplastic-syndrome/about/key-statistics.html

3. Myelodysplastic Syndromes Treatment (PDQ)–Patient Version. National Cancer Institute. Updated October 4, 2024. Accessed December 3, 2024. https://www.cancer.gov/types/myeloproliferative/patient/myelodysplastic-treatment-pdf

4. Medicines for Myelodysplastic Syndromes (MDS). American Cancer Society. Updated November 21, 2024. Accessed December 3, 2024. https://www.cancer.org/cancer/types/myelodysplastic-syndrome/treating/chemotherapy.html