A first in home treatment

A first in home treatment
Children’s hospital leading the way. New infusion product means fewer side effects, less fatigue for kids with rare genetic disorder
AARON DERFEL, The Gazette
Published: Friday, September 14

Since he was stricken with severe pneumonia at age 4, Akeel Knight-Lee has been very careful not to catch the slightest infection.

That’s because the boy, now 10, has a rare genetic disorder called X-linked agammaglobulinemia. The disease has robbed his immune system of the ability to make antibodies that fight infection.

For six years, Akeel has had to spend one day a month at the Montreal Children’s Hospital, hooked up to an intravenous line so he can be infused with immunoglobulin – a solution of pooled antibodies from blood donors.
Katherine Knight with her son Akeel at their Dollard des Ormeaux home. Akeel Knight-Lee has a rare immunodeficiency and used to have to go to the Children’s Hospital for treatment that left him weakened. Now, he receives treatment at home and has more energy.View Larger Image View Larger Image
Katherine Knight with her son Akeel at their Dollard des Ormeaux home. Akeel Knight-Lee has a rare immunodeficiency and used to have to go to the Children’s Hospital for treatment that left him weakened. Now, he receives treatment at home and has more energy.

The treatment is lifesaving, but it can cause side effects like seizure-inducing headaches and nausea in about 25 per cent of cases. And everyone who receives the treatment – regardless whether they suffer from the side effects – feels sapped of energy for about three weeks after the infusion.

“Akeel would say to me, ‘Mommy, when do I have to go to the hospital?’ ” Katherine Knight recounted. “And I would say, ‘Next week.’

“Oh,” Akeel would respond dejectedly. “I’m feeling so tired.”

But now, thanks to the pioneering efforts of the Children’s, Akeel and others with similar disorders now can infuse themselves in the comfort of their homes. And since the treatment is weekly, their energy levels never drop.

“We’re so happy,” Akeel’s mother said. “His energy level is high. He played soccer in the summer.”

Akeel is one of about 25 children with primary immuno-deficiency who are followed by the Children’s, the first hospital in Canada to set up the at-home treatment.

Primary immunodeficiency is the umbrella name for a range of 20 to 30 genetic disorders. The disorders are not like AIDS, which is a secondary immuno-deficiency. Since the mid-1980s, people with primary immuno-deficiency have been treated with replacement immunoglobulin or antibodies.

The driving force behind the at-home treatment is Christine McCusker, a pediatric immunologist. In an interview, she said she wasn’t satisfied with the in-hospital treatment, not only because it could produce side effects, but because it proved to be a major inconvenience for the children and their parents.

“Can you imagine a 4-year-old having to come into the hospital, get picked (with a needle) three times to have his IV started and then sit for four hours while he gets his infusion?” she asked.

“Mummy has to take time off work and the child has to be off from his usual activities. And if he’s older, he has to miss school for that day.”

So McCusker made some inquiries and discovered nearly three years ago that a group of physicians in Britain had switched to a new infusion product that could be used at home. The treatment, Vivaglobin, does not require an intravenous line, but is delivered subcutaneously – that is, just under the skin.

A child’s mother can inject the needle into the belly and, over the course of an hour, deliver an ounce (about 28 grams) of the antibodies. The only side effects are redness at the site of the injection and a bulge that goes away after a few hours.

At first, Health Canada approved the treatment on a case-by-case basis. But last year, Vivaglobin was approved for use without special authorization.

Children’s hospital leading the way. New infusion product means fewer side effects, less fatigue for kids with rare genetic disorder

And since the Children’s developed expertise with the treatment, it’s now a training centre for nurses from hospitals across the country.

“When you can make a small change that improves a child’s life, it’s the greatest feeling in the world,” McCusker said.

“Now, not only do these children not have to come to hospital, with all the attendant problems, but they become independent sooner and it’s better for their self-esteem and their social development.”

Katherine Knight said she’s greatly relieved that her son no longer misses school exams and field trips.

For Akeel, who wants to be a doctor, the at-home treatment is simply fun.

“I can just relax,” he said, “and play games like Transformer.”

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