MDS: Treatment Criteria and When to Start Targeted Therapy

MDS disease issues

In the case of MDS RAEB-1, is it necessary to have a specific blood cell count decrease in order to use targeted therapy? I have normal or elevated peripheral blood counts, but I was diagnosed with this condition due to issues found in a bone marrow biopsy.
Currently, I am not receiving any treatment.
Do I need to wait for my condition to worsen before starting treatment?

chun, 30~39 year old female. Ask Date: 2015/04/16

Dr. Chen Yunfang reply Oncology

You have been diagnosed with MDS RAEB-1, correct? It is important to note that for this diagnosis, the presence of abnormal blast cells in the bone marrow examination should account for 5-9% of the total blood cells.
Although this does not qualify as acute leukemia (commonly referred to as blood cancer, which requires blasts to be greater than 20%), it is considered a precursor condition that may progress to leukemia in the coming years.
Currently, the National Health Insurance covers a chemotherapy drug called Azacitidine for your diagnosis (not the targeted therapy you mentioned).
Due to its high cost, with a single injection priced at nearly $20,000, the physician must submit a request to the National Health Insurance Administration for prior review, which takes about a week.
Once approved, the patient can receive the medication under insurance coverage without out-of-pocket expenses.
Clinically, it is primarily administered subcutaneously, making it convenient and requiring no hospitalization, with minimal side effects.
After a four-month treatment course, the efficacy is approximately 30-40%, which can improve the blast situation in the bone marrow.
If your diagnosis is indeed MDS RAEB-1, you can apply for the use of Azacitidine under the current National Health Insurance coverage.
I hope this information is helpful, and I will also provide you with the relevant insurance regulations for your reference.

9.44.
Azacitidine (e.g., Vidaza) (102/1/1)
1.
For high-risk patients with myelodysplastic syndromes: refractory anemia with excess blasts (RA with excess blasts, RAEB), refractory anemia with excess blasts in transformation (RAEB in transformation, RAEB-T), and chronic myelomonocytic leukemia (CMMoL).

2.
Use requires prior review and approval; it must be confirmed that the patient's condition has not progressed to acute myeloid leukemia before continuing treatment.

(1) The first application is for four treatment cycles.

(2) Subsequent applications are made every three cycles starting from the second application.

Reply Date: 2015/04/16

More Info

Myelodysplastic syndromes (MDS) are a group of disorders caused by poorly formed or dysfunctional blood cells. They are often characterized by ineffective hematopoiesis, leading to various degrees of cytopenias (low blood cell counts). The classification of MDS includes several subtypes, including Refractory Anemia with Excess Blasts (RAEB), which is a more advanced form of MDS.
In the case of RAEB-1, the criteria for initiating treatment, including targeted therapy, typically depend on the presence of specific cytopenias. While it is true that many patients with MDS may present with low blood counts—such as anemia (low red blood cells), thrombocytopenia (low platelets), or leukopenia (low white blood cells)—the decision to start treatment is not solely based on these parameters.
For patients with RAEB-1, treatment is often recommended when there is evidence of significant cytopenias or when the disease shows signs of progression, such as an increase in the percentage of blasts in the bone marrow. The presence of high peripheral blood counts does not necessarily preclude the initiation of treatment, especially if the bone marrow findings indicate a significant risk of progression to acute myeloid leukemia (AML).
In your case, since you mentioned that your peripheral blood counts are normal or elevated but you have been diagnosed with MDS due to issues found in your bone marrow biopsy, it is essential to monitor your condition closely. The absence of treatment at this stage does not mean you must wait for your condition to worsen before starting therapy. The goal of treatment in MDS is to manage symptoms, improve blood counts, and reduce the risk of progression to AML.

Targeted therapies, such as hypomethylating agents (e.g., Azacitidine or Decitabine), are often used in patients with higher-risk MDS, including those with RAEB-1. These therapies can help improve blood counts and may also reduce the risk of progression to AML. However, the initiation of such treatments should be based on a comprehensive evaluation by a hematologist, considering your overall health, the specific characteristics of your MDS, and any symptoms you may be experiencing.

It is crucial to have open discussions with your healthcare provider about your treatment options. If you are not experiencing significant symptoms or cytopenias, your doctor may recommend a watchful waiting approach, with regular monitoring of your blood counts and bone marrow status. However, if there are signs of deterioration or increased blast counts, it may be time to consider starting treatment.

In summary, while certain blood count abnormalities often guide treatment decisions in MDS, the presence of normal or elevated peripheral blood counts does not automatically exclude the need for therapy. It is essential to work closely with your healthcare team to determine the best course of action based on your specific situation and the characteristics of your disease. Regular follow-ups and monitoring will be key in managing your condition effectively.

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