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Myelodysplastic Syndrome - Classification and Prognostication »

Myelodysplastic Syndrome - Treatment

Even though the IPSS scoring and risk group for the patient can be identified relatively easily, there are still no consensus when it comes to treatment guideline. In other words, when it comes to decision about treatment, the IPSS risk grouping may play just a small role. The more important consideration would be patient’s age and general well-being. As MDS is often a disease of the elderly, not all treatment modalities can be explored. The Haematologist need to assess the state of the patients heart, liver, lungs and kidney before final decision can be made. If the patient has other medical conditions such as diabetes and heart disease, giving too intense a treatment regime may be detrimental to the patient’s health and will not be suitable.

Long-term survival according to risk groupTo simplify matters, the treatment options for MDS can be broadly divided into 4 main groups; 1) supportive, 2) low-intensity, 3) high-intensity and 4) palliative. The low-intensity group proves to be the most controversial and will be discussed last. Supportive care is usually offered in patients in the low risk groups or in elderly patients in the higher risk groups. The idea here is to impart minimal treatment on the patient, and the purpose of the transfusion is not to normalise the patient’s counts, but to increase it enough to a level where the patient will not have symptoms. The emphasis here is to increase the quality of life while minimising potential harm.

Summary of the treatment optionsIn the very elderly, or advanced risk group, it may be appropriate just to palliate the patient. The idea here stems from the understanding that intensive therapy will be futile or will even hasten the patient’s demise. It may also impart unnecessary cost and suffering to the patient. The patient will be given transfusion if needed, and treated for infection if this becomes an issue. Palliation does not mean leaving the patient to die without any intervention. Most tertiary hospitals now have Specialist Palliative Care Physician who will help the patients and family through this trying time. Palliative care will be discussed in another entry.

In the young patient, where long-term cure is a reasonable target, the patient may be a candidate for bone marrow transplant. There is controversy however in terms of timing. Most centers, including ours believe that transplant should be offered early in suitable candidates where complications of transfusion and infection has not yet set in. It is imperative not to wait until transformation occurs as success rate from transplantation at this stage of MDS is very poor. Once transformation occur, chemotherapy similar to patients with acute leukaemia will be needed to take the patient into the remission phase. However, this remission phase is often hard to achieve and even if so will be short-lived. The patient then will need to be transplanted as soon as possible. Data also suggest that the relapse rate in MDS patients undergoing transplant especially once where transformation has occurred is much higher if compared to AML patients or in patients with MDS where transformation have not yet occurred. The take home message here is, bone marrow transplant offers the only possibility of long-term remission and is best to be done early in the disease course.

Low-intensity therapy form the grey area in MDS treatment. Here, a combination of patients’ age and unsuitability for bone marrow transplant put the chance of long-term disease control slim, but in the meantime, the patient may be fit enough for more than just supportive care. The options are broad and there is no right answer for everybody. Often the treatment offered will depend on patient’s preferences and the Haematologist’s experience in using the available drugs. Some of the drugs have horrendous side-effect while others, are very expensive - azacytidine, lenalidominde and decitabine to name a few. It is often sensible to start with growth factors, such as erythropoeitin and G-CSF, either separately or in combination. The next option would be immunosuppression. Steroids, danazol, cyclosporine, thalidomide and mycophenolate have been tried with various level of success. This may be especially appropriate in subgroup of patient where marrow aplasia - empty marrow - is prominent. In a special subgroup - the 5q- syndrome - thalidomide and lenalidomide have been proven to be very effective.

In the last few years, a new group of novel agents have emerged from clinical trials. These are the HDAC inhibitors and the hypomethylating agents. The two hypomethylating agents currently available in the market are decitabine and azacytidine. Both has been proven to prolong the patients live by a factor of a few months. It is not clear however if they both can prevent the progression of the disease into leukaemia. Our center has so far only been able to use decitabine. The main issue being the cost to the patient. Suffice to say it is prohibitive, and there will also be risk of complications that will incur further cost to the patient later on. However, these novel agent will be the option for the future and I am sure over time, the cost will become reasonable.

There are also patients who may benefit from low-dose chemotherapy to help stem down the population of blast cells in circulation. However, they do not prevent progression of disease or delay transformation into leukaemia. Its use should be weight against the risk of infection and organ damage.

All in all, decision about treating patients with MDS is never clear cut. Extensive discussions with the patients and relatives are very important.

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