A call for help
Waiting for a match
Ten-year-old LJ is waiting for a match to help treat a rare disease of the blood. (Photo courtesy of live.drjays.com)
But LJ is not a typical kid. He has a rare blood disease that is almost impossible to pronounce but even more difficult to live with.
In hypereosinophilic syndrome (HES) the body produces too many of a particular type of white blood cell, which can result in damage to the heart, lungs, liver and other organs. Not only is HES rare, it is basically unheard of in children, and is typically found in males over the age of 50.
Finally knowing the diagnosis was a good first step for LJ’s mother, Crystal Robinson, 47. At least she now knew why her child suffered repeated bouts of pneumonia. But knowing the diagnosis was only half the battle.
The cure is an entirely different matter. Because of his chemotherapy treatments, LJ’s condition is relatively stable. But, according to Robinson, his doctors state that the only chance of a cure is a marrow transplant. His three siblings are not a match, so the family’s only hope depends on a stranger.
As the chief of the Stem Cell Transplantation Program at Dana-Farber Cancer Institute, Dr. Joseph H. Antin knows all too well about the life-saving procedure.
Success of a marrow transplant depends on several factors. The age and condition of the recipient, the type and stage of disease and complications all have an impact. But matching — how closely the donated cells match the cells being replaced — is crucial.
Proteins on the surface of cells warn the immune system of unrecognized intruders. If the match is dissimilar, the immune system sets out to destroy the donated cells. That is why proteins of the donor are “typed” with proteins of the recipient to find the most suitable match.
Most recipients receive stem cell donation from complete strangers taken from a pool of donors worldwide. But this pool is lacking in donors from people of color. Race in itself does not matter. “We are pretty much the same,” said Antin. “It’s more how proteins are distributed.”
Some groups of people are more insular with little influx of outsiders making them very similar. But some people — often people of color — have a lot of mixing, which makes matching more difficult. “There’s a little bit of this, a little bit of that,” he said. “You can’t put a light bulb from a Toyota into a Honda. It’s still a light bulb, but the wiring is different.”
Joseph H. Antin, M.D.
Chief, Stem Cell Transplantation Program
Dana-Farber Cancer Institute
A successful transplantation can cure the disease and stem cells are the key. “They provide for the next generation of cells,” said Antin. “It’s like planting seeds to re-establish normal function.”
And that would be a great relief to LJ. He seems to take his condition in stride. To avoid the prevalence of germs in most school settings, LJ is home-schooled as suggested by his doctors. He does go outside but often under the protection of a mask. His mother says that he is somewhat of a prisoner in his own house.
LJ admits there are some things that are hard to take. Being in the hospital is one of them. And the illness and medication sometimes make him break out in a rash and cause an occasional headache. Even worse, LJ complained, “I can’t skateboard.”
LJ has become a celebrity of sorts. His mother’s never-ending search for a donor has brought his dilemma into the media limelight. The two have pleaded his case on television, radio and at countless donor drives. So far, no match has been found.
There have been many advances in transplantation techniques over the year. One major improvement has been the introduction of reduced intensity transplantations that has allowed the inclusion of previously exempt candidates.
At one time the age limit for a transplant was 35 years old, according to Antin. Because some of the diseases of the blood occur in older people, “We were not treating the bulk of people,” he said.
With reduced intensity chemotherapy and radiation, older people can now take part. “People in their early seventies are now being treated,” he said.
Even those with reduced organ function are now potential candidates. High dosage chemotherapy — used in preparation for stem cell transplantation — was not advised for such patients.
Courtesy of Dana-Farber Cancer Institute, Stem Cell Transplantation Program
There are three methods to donate stem cells. They can be harvested from the marrow of bone or extracted from the bloodstream.
A newer option is umbilical cord blood donation, which poses no threat to the mother or baby. If not donated, it is thrown away. Cord blood is a good option for people of color. “It’s a clean slate,” said Antin. The cord blood has no functional immunity, which allows hematologists to take some liberties. “The match can be less perfect, but not damage the host,” he said.
For her part, Robinson stresses the need for donors of color and cautions people to not believe the myths about donation. “Just do a little research,” she advised. She admits she gets a little frustrated. “If you can take the time to get a tattoo or a body piercing, you can take the time to do this,” she said. “And it is less painful.”
LJ is quick to chime in. “It’s not hard to register,” he said. “Waiting [for a donor] is hard.”