Bone marrow transplants: life-saving, life-giving
April 30, 2009
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Noor Javed
STAFF REPORTER
Every month for the first 15 years of her life, Fatana Mirzazada would spend one day in the hospital getting a blood transfusion. Every night, she would give herself a needle, a procedure to keep her iron levels in control.
Then, one day, the young patient who was born with thalassemia, a blood-related genetic disorder, decided she was tired of this temporary solution.
"I really thought about it," says Mirzazada, now 20. "I realized, I'd rather not have to deal with blood transfusions and taking needles every night for the rest of my life," says the Mississauga nursing student.
So she decided to proceed with the only other treatment option available to her: a bone-marrow transplant. This procedure, doctors told her, if successful, would be the closest thing to a cure.
Years ago, patients with metabolic or non-malignant blood diseases would have little choice but to undergo lifelong medical interventions and, in some cases, accept the fatal outcome of their illness.
But now bone-marrow transplants, a procedure normally associated with cancerous diseases, is proving to be a vital way to treat and save the lives of patients with a range of non-cancerous, life-threatening diseases, such as thalassemia, sickle-cell anemia and several metabolic diseases.
"Leukemia is our most common indication for why we are going to take someone to transplantation," says Dr. John Doyle, a hematology/oncology specialist at The Hospital for Sick Children. "But there is this whole other group of kids who have non-malignant diseases where it is a very useful modality, as well.
"By replacing the bone marrow, you can replace a function they are missing in their blood," Doyle explains. Due to the thalassemia, a disease that Mirzazada inherited from her parents, her body made fewer healthy red blood cells and less hemoglobin than normal. The bone marrow transplant helps her body to make red blood cells.
Since Mirzazada's disease was not life-threatening, her choice was more of a "lifestyle" one, says Dr. Adam Gassas, a hematology/oncology specialist at SickKids who treated her in 2004. Her brother turned out to be a near-perfect match, so she was able to undergo a transplant when she was 15.
But there are many others, explains Gassas, for whom a bone-marrow transplant is the only option.
Evan Santos was diagnosed with a rare, genetic metabolic disease called Hurler's Syndrome when he was 8 months old. "We had never even heard about it," says mom Melissa Santos.
Radiologists noticed a defect in his spine during a routine chest X-ray, and told the Santos family that because of the disease, Evan lacked a specific enzyme to break down a type of sugar in the body.
"When they are born, they are born normal, and then, as the sugar builds up in his organs, it starts to have multiple organ damage – so his brain, his liver, his spleen, his joint, and everything is affected – and ultimately, his heart, would give," explains Santos.
Doctors told them that most kids with Hurler's Syndrome don't survive past 10 years. But Evan's early diagnosis made him the perfect candidate for a bone-marrow transplant. They first tested his 2-year-old sister, to see if her bone marrow matched. It did. The transplant took place and was "extremely successful." Although it cannot reverse any damage done, it has essentially saved Evan's life.
Over the past 20 years, SickKids has done 44 bone-marrow transplants for 43 children with metabolic disorders. "Eighty per cent are now surviving," says Gassas.
Toronto Star