New and Promising Treatments for Myelodysplastic Syndrome

Fri, Aug 15, 2008

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Family Cancer Center

I was about to slide my hand into the sterile glove when my patient, George, interrupted the quiet business of the procedure room

“Hey, Doc… did you hear the one about the family doctor who bumped into his patient at the mall? The patient was wearing a Hawaiian shirt, sunglasses and had a twenty-some hottie on his arm.”

“No,” I smiled, feeling that this would be a good one. George loved telling jokes and he wasn’t about to miss the opportunity to try his material out on a captive audience.

“Well, the doctor asked him, ‘What do you think you are doing, Bob?’ and Bob says ‘Just doing what you said, Doctor – Get a hot mamma and be cheerful.’ The doctor replied, ‘Bob! I said, you got a heart murmur. Be careful.’”

Laughter filled the room briefly as I reached for my scalpel.

At 70, George was still a natural entertainer with a sunny disposition. Recently, not even his zest for life and great sense of humor could keep him from noticing that it became difficult to keep up with his routine. He frequently tired. He noticed he felt breathless when walking or talking with his wife. His son who visited from out of town noticed his father looked pale. And he began feeling dizzy each time he stood up. A visit to his doctor for a simple blood test revealed the problem: George was severely anemic and had low platelets which explained his easy bruising. The most important question was…why?

The Real Reason

Anemia, a low red-blood cell count, can have many causes, ranging from simple iron deficiency to more sinister reasons, such as myelodysplasia of the bone marrow or acute myelogenous leukemia. George’s blood count revealed not only severe anemia, but also decreased platelet count.
Bone marrow, a liquid organ nestled in the hollow spaces of pelvis, ribs and long bones produces three kinds of cells. It makes white blood cells which protect us from infections; red blood cells, the vital link in delivering oxygen breathed through the lungs to the cells; and platelets, tiny particles that stick together and help blood clotting when injured or bleeding.

On this day, I performed a bone marrow biopsy on George. This is a relatively minor procedure in which a small chip of the bone and a syringe-full of juicy-looking marrow is removed from the hip bone. The procedure takes only 10 minutes in a doctor’s office under local anesthesia and usually causes only minor discomfort.

After receiving a blood transfusion during the biopsy, George’s dizziness disappeared and color returned to his face.

The results of the biopsy revealed George had an advanced form of myelodysplastic syndrome (MDS), a disorder in which bone marrow is making too few white blood cells, red blood cells and platelets. MDS, if left untreated, leads to bone marrow failure, severe anemia, life threatening infections, bleeding and death. MDS is also known as pre-leukemia as some patients experience progression of MDS to acute myelogenous leukemia.

George’s Treatment Options

With the approval of the several new drugs in the past five years, MDS treatment options have improved. In the past, the mainstay of treatment focused on support with blood transfusions and use of epoetin (Procrit) or darbopoetin (Aranesp), with no good options for a disease that was significant, but not yet severe enough to require a either bone marrow transplant or heavy doses of chemotherapy administered in the hospital.

George started treatment with azacitidine (Vidaza), a medication given as a simple injection seven days each month. The drug works by reworking impaired function of the bone marrow, improving the blood counts, reducing the infection and bleeding episodes as well as the need for blood transfusions. Azacitidine causes mild nausea but is well tolerated otherwise and is easy to give in older patients whom disease affects most frequently. Some patients experience a great response with complete normalization of their blood counts, but the majority have partial improvement of their blood counts, adequate enough to require less or no blood transfusions.

If azacitidine is not a treatment option or patient’s disease requires it, a drug called decitabine (Dacogen) is a very good next choice. Lenalidomide (Revlimid), an oral drug also used for multiple myeloma is yet another effective treatment option that can be used.

Not long after, George sauntered in the treatment room with a worried look on his face.

“Doc, I just talked to the nurse. The shots you’ve been giving me–are they really a birth control shots?”

“No!” I explained with surprised look, rushing to offer reassurance when a mischievous smile, wink and laughter interrupted me.

And I knew that George felt like his old self.

By Dr. Alex Jankov - Dr. Alex Jankov is a board certified medical oncologist and hematologist. He specializes in treating patients with cancer, blood disorders, clotting and bleeding problems. Dr. Jankov practices with Family Cancer Center and sees patients in the Memphis area.

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