Newswise — Plagued by a growing weakness that left her unable to walk, talk and even take a normal breath, 11-year-old Iraqi Aram Ali was a shell of the bright little girl she used to be.
No one could figure out what was wrong. Not the 13 doctors her distraught parents saw in Syria, not the specialists in Britain who had reviewed her chart, not the additional physicians she had seen in Iraq, the war-torn country the family had fled in a desperate effort to find the medical help the little girl needed.
In early 2009, her sobbing mother, Kamila Ali, sat in an office at the United Nations in Syria and gave an ultimatum: she wasn’t leaving the building until they gave her an answer. She had to know whether they could emigrate to Europe or the United States to find someone who could help her ailing daughter.
Ten days later they had their answer and, for the first time since their nightmare began when Aram was 9, they had hope.
Their journey would lead them to The University of Texas Medical School at Houston, where pediatric neurologists and a gifted surgeon would put Aram on a very different path, one that led to a healthy life.
Ramy El Khoury, M.D., chief neurology resident at the UT Medical School at Houston, was one of the first physicians to see Aram after she arrived at the emergency room at Children’s Memorial Hermann Hospital. She could barely breathe or move, he remembers.
“My first thought was that this girl is really sick and it is probably a complex mechanism that had not been diagnosed yet. She required pediatric ICU monitoring,” said El Khoury, who was able to talk to the family in their native Arabic. “My second thought was that the family really needs help because it was their first experience with hospitalization in this country and they had to interact with so many people who spoke a different language.”
According to the records the family brought with them, Aram had already been tested for myasthenia gravis, a rare neuromuscular disorder that seldom affects children. So physicians were looking for something even rarer, a “zebra,” (as opposed to a horse) as El Khoury described it. They also didn’t know if a seizure disorder, a remnant of meningitis when she was 2, possibly played a role.
“I didn’t know what I would find,” said Pedro Mancias, M.D., associate professor of pediatrics and neurology at the UT Medical School at Houston and the university’s neuromuscular specialist in childhood disease. “Through testing we ascertained that her sensory responses were normal but her motor responses were on the small side, so it had to be something with the motor system.”
It turned out that it was myasthenia gravis, but an extremely severe case. “Her blocking antibodies were sky high,” Mancias said.
Myasthenia gravis is an autoimmune neuromuscular disease that causes weakness of the voluntary muscles in the body. It can be thought of as a bridge leading to nowhere. A nerve impulse travels from the brain and attempts to cross the neuromuscular junction in order to make the muscle move. But when the neurotransmitter tries to reach the muscle, the “bridge” ends abruptly in mid-air, leaving the transmitter with no place to land. The necessary landing spot has been wiped out by an overactive antibody produced by the thymus gland, which plays an important role in the development of the immune system in early life.
The disease affects 7 to 14 people in every 100,000 and most commonly occurs in young adult women and older men, according to the U.S. Department of Health and Human Services. It can be difficult to diagnose because symptoms can mimic those of other diseases. Aram was unusually young to have the disease.
“It’s usually diagnosed within a year in the United States,” Mancias said. “Without treatment, people who suffer from myasthenia gravis eventually can’t swallow their own saliva and so they aspirate it into their lungs and may die of pneumonia. She would have died over there (without treatment).”
A single dose of oral steroids administered by UT physicians acted almost like a magic serum. Aram began to speak more clearly and talk to her family. “We usually don’t see that much improvement that quickly,” Mancias said.
“It’s like a miracle in the sense she was unable to move for so long,” El Khoury said.
For the family, it was the break they had prayed about.
The first sign that something was wrong with Aram came in 2007 when she began to lose her balance and fall for no apparent reason. In Iraq and Syria, her mother and father, Mohammad Yahia Ali, spent nearly all their income on physicians and tests trying to get Aram well. Her mother worked as the equivalent of a physician’s assistant to a midwife while Mohammad worked in a restaurant.
They told their story recently while seated in their apartment living room, where they served tea, lemonade and cookies to their guests. Aram, wearing a princess shirt and tiara, sat nearby in a wheelchair but was eager to show her guests that she could take a few unassisted steps.
Acting as interpreter, El Khoury explained that Aram’s parents were delighted when they finally received word that they would be able to travel to the United States for treatment.
“It was like a rebirth of Aram,” translated El Khoury, as Kamila’s eyes brimmed with tears. “She (Kamila) was convinced she would find an answer here. She didn’t accept the verdict from the doctors they had seen. Because they felt the hope when they got the news they would be coming here, it changed their whole perspective.”
They were told by the UN that their destination would be Houston because it was “a good place to treat neurological problems.”
Aram was also looking forward to the trip for another reason. “Aram’s only dream is to go to school,” El Khoury translated. Her younger brother has now started school in Houston, much to their parents’ joy.
Shortly after arriving in Houston, her breathing problems became worse, resulting in the emergency room visit to Children’s Memorial Hermann.
An option for the treatment of myasthenia gravis is removal of the thymus gland, which is located behind the sternum in the upper chest. It can take up to two years for the effects of the operation to be fully realized.
“Taking out the thymus gland results in a complete remission in almost half the patients with myasthenia gravis and at least a partial remission in almost 90 percent of patients, so it’s worthwhile to remove it,” said Larry Kaiser, M.D., thoracic surgeon and president of The University of Texas Health Science Center at Houston.
Typically, the surgery involves a highly invasive procedure of splitting the sternum (the central breast bone) in order to reach the thymus. Kaiser is one of the few surgeons in the world who is an expert at using a less invasive technique called a transcervical thymectomy, which is done through a very small incision in the neck.
“The standard surgery requires four days in the hospital. The operation we do through a small neck incision is far less invasive and I usually send the patient home the same day,” said Kaiser, who wrote the chapter on thymectomy in the 2006 book he co-authored, “Mastery of Cardiothoracic Surgery.”
“Obviously this situation was a little bit different,” Kaiser said of Aram’s case. “She came in very weak from the myasthenia. Once she was better controlled medically, we were able to do the operation and one of the advantages is, even in a person like this who is still quite weak, she did not have any setback from her operation.”
The outlook, say all her physicians, is bright.
“She had some muscle atrophy and we have to rebuild all the receptors but there’s no reason to suspect she won’t have a normal life,” said Mancias, who sees her in follow-up visits at UT Physicians’ Pediatric Neurology Clinic. “It’s one of the few neuromuscular disorders that we can treat and potentially cure.”
As Aram takes some of those first steps, her mother smiles broadly and says, “We got a treasure from the United States: Aram’s health.”
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