Liz Jones remembers sitting in support groups and feeling scared listening to parents of older children. The group was for parents and caregivers of profoundly disabled children who require help with every basic need. As the children grow, it becomes more difficult to bathe, transport and care for these children. One mom in the group had multiple back surgeries after years of lifting her son. Another mom put her son in a nursing home because she could no longer lift him on her own.
Jones wondered, “What am I going to do when Daniel gets bigger?”
Her son Daniel, now 17, was born with profound developmental and cognitive disabilities and will remain in the same condition for the rest of his life. He’s nonverbal, non-ambulatory and tube fed. Like many severely disabled children, there is no specific diagnosis. He is considered medically fragile and has a slew of health complications.
X-rays of his growth plates when he was elementary-school age predicted he would be over 6 feet tall.
As his primary caregiver, Jones listened with angst to the parents saying, “I wish my child was just 10 pounds lighter” and “I wish my child was a couple inches shorter.” In 2007, her friend sent an email with the subject line, “Your prayers have been answered.” It was an article about growth attenuation therapy.
Growth attenuation therapy stunts growth. High doses of estrogen, delivered as a daily pill, patch or injection, speed up bone maturation to close the growth plates, or cartilage at the end of long bones in children and adolescents. The earlier the treatment is started, the greater the height reduction.
The therapy started in the 1950s as a means to keep women from growing too tall. As it became accepted for women to be taller, estrogen is no longer used for this purpose, but has been adapted for a new use in the past 10 years. Parents of severely disabled, non-ambulatory children with cognitive impairments are seeking the therapy to provide a better quality of life for themselves and their children. A shorter and lighter child requires only one person to lift and no hoisting apparatus so the child can be more easily bathed and transported.
The article Jones read described the first case in 2006 where estrogen was used for this purpose. A 6-year-old girl, known as Ashley, with profound cognitive disabilities relied on support for every aspect of daily life, including lifting her head. Ashley’s parents sought the treatment so they could lift and transport her more easily.
A common side effect of high-dose estrogen is breast bud growth. Ashley’s parents chose to remove her breast buds because they thought breast growth would be uncomfortable for her. Also, to avoid uncomfortable menses and bleeding, which is difficult for caregivers, Ashley’s parents decided to have her undergo a hysterectomy. Their decision received national attention.
Due to the overwhelming attention, the therapy became known nationwide as the “Ashley Treatment.”
Despite the backlash, parents of severely disabled children began asking their doctors about growth attenuation. As Jones read the article from her friend, her spirits lifted. She had had no idea growth attenuation existed; Daniel’s doctors had never mentioned the therapy. She imagined the benefit of Daniel being a size she could lift on her own.
When she brought it up to Daniel’s doctors it had been a year since the Ashley case. There had been few cases at the time, and research was limited. And the backlash deterred many providers from discussing the treatment. Daniel’s doctors were tentative, but agreed to consider the case with an ethics committee.
More than 20 people, including community members, attorneys, doctors and specialists, met to examine Daniel’s case. The committee only focused on Daniel’s medical conditions; they never brought up any benefits to the family members. For several hours they discussed the pros and cons. Ultimately, they decided it was an ethical treatment. Before the scheduled treatment, Daniel’s endocrinologist backed out. “It really saddened me,” Jones says. She believes he backed out because he feared the media’s reaction and how it would affect his career.
Jones reached out to about 150 doctors around the country asking about growth attenuation. Some did support it but had never administered the treatment; others said it was against their personal beliefs. Jones thought she was out of luck, until she coordinated with Dr. Philip Zeitler and Dr. Michael Kappy at Children’s Hospital Colorado.
Zeitler is the head of Pediatric Endocrinology at Children’s and Kappy is a practicing pediatric endocrinologist. Together, they have administered growth attenuation therapy to about 20 children.
They are well aware of the negative opinions about growth attenuation but reiterate the therapy is only for a very specific population. Disability rights advocates argue how can you really know the cognitive functioning of these children? What if they are more aware of what’s happening than you realize?
Developmental disabilities fall on a massive spectrum, encompassing everything from attention-deficit/hyperactivity disorder, cerebral palsy, autism, and vision impairment. Candidates who qualify for growth attenuation fall on the most severe end of the spectrum, where IQs are so low there is no way to even test cognitive capacity.
Like Jones, families within this network oftentimes struggle to find a doctor who will administer the therapy. Families have come from all over the U.S. and abroad seeking growth attenuation at Children’s Hospital Colorado. “We are not going to people saying we need to make your child smaller. They are coming to us,” Zeitler says. “These are people who are recognizing their child is profoundly affected and not going to improve.” Zeitler and Kappy also have a few colleagues outside of Colorado who are willing to do the therapy.
They are aware of the lack of evidence for the therapy. Because of the limited number of cases, there have been no randomized trials, so many answers remain unclear. How much height is reduced and how quickly varies case by case. Most treatments so far have taken two to three years. Because many children with developmental disabilities start puberty early, the ideal age to begin treatment is elementary school age.
Although exact results cannot be predicted or guaranteed, Children’s Hospital Colorado has made a conscious decision to administer the therapy anyway. Without evidence in the medical literature to guide them, the endocrinology team at Children’s practices what they call “compassionate use.” This refers to a treatment or drug that has not been widely tested, and therefore is restricted to people who meet very specific conditions, according to the U.S. Food and Drug Administration. With the help of an ethics committee, Children’s Hospital Colorado endocrinologists and the family discuss whether the child is an appropriate candidate. And if the child is a good match, do they proceed with the treatment even though there is not a guaranteed effect?
Of the 20 cases at Children’s Hospital Colorado so far, all the families have said it was a success and they are grateful for the treatment. One family decided to stop treatment after their son developed breast buds. Neither Children’s nor their few colleagues who administer the therapy perform breast bud removal or a hysterectomy on these children.
“We don’t have a lot of information because hospitals are not collecting and sharing results. Our hope is that if we can make this a little less controversial, there could be a sharing of outcomes for folks who are doing this,” Zeitler says.
Daniel completed growth attenuation therapy six years ago and is 5 feet tall, a foot shorter than his predicted height. “His height has no correlation with his cognitive abilities,” Jones says. “Just because we attenuated his height doesn’t mean we attenuated his brain. My son continues to mature.” Every year, friends comment on how much Daniel has grown and she chuckles to herself. “The reason why they think that is because he continues to mature. He’s developing into a young man.” She is grateful for the treatment, as she can care for Daniel on her own. They even travel.