centerofexcellence
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prader-willi syndrome
I tell insurers all the time that there’s no place in the world like The Children’s Institute to get this
Rare — and Incurable Prader-Willi syndrome is rare, genetically caused and incurable. Its primary effect is insatiable hunger. Other effects can include developmental delay, short stature, low muscle tone, behavioral issues and obesity. People with severe cases never feel satiated, and unless food availability and intake are carefully limited, they can literally eat themselves to death, either ts Patien a through sudden stomach ge rupture or obesity-related avera se a e r medical complications. c in o What makes The i d in car ce Children’s Institute’s an program unique — and endur excellent — is its rigorous multidisciplinary approach that includes medical and nursing care; psychiatry and psychology; physical, occupational and speech/language therapies; diet and nutrition; and a carefully planned and monitored exercise regimen. Treatment is always completely tailored to the needs and abilities of each patient. The care takes place on a bright, sunny unit designed to meet the needs of both pediatric and adult patients. (This is The Children’s Institute’s only program that serves non-pediatric patients.) “For many patients, this is the first time they’ve been in a community of other people with Prader-Willi syndrome. It’s greatly helpful to know they’re not alone,” says Dr. Cynthia Smith, a pediatric physiatrist who is the attending physician on the unit. The unit has its own well-equipped gym, funded primarily through the Pittsburgh Pirates Wins for Kids program.
56%
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So that patients don’t have to deal with the temptation of being surrounded by food, they are served meals brought to the unit’s dining area on trays individualized to each patient’s caloric and nutritional needs. A music therapist helps patients develop emotional expression and control, self-esteem and social skills. A speech/language pathologist holds regular “meet the author” sessions in which patients present, with great pride, essays and poetry they’ve written. Over each patient’s stay (an average admission is 67 days) weight comes off, health improves and, just as importantly, patients learn strategies for coping with their aggression and anger. Along the way, families and caregivers learn how to help manage patients’ difficult behaviors and stringent dietary needs. The program treats the most challenging patients in the world. Yet patient records from a typical year show impressive results: an average 56% increase in cardio endurance, an average weight loss of 36 pounds and increased compliance with behavioral management. Reduction or elimination of oxygen use was achieved in 87.5% of patients, and, among patients with diabetes, 40% were able to discontinue insulin use, transitioning to oral medications alone, or discontinuing diabetic medications altogether. “For so many of these patients, their stay here is the first time in their lives that they have been able to achieve success,” says staff psychiatrist Dr. greg Cherpes. Importantly, those good results persist. That was certainly the case with William Weaver, now down more than 100 pounds and doing well in his school for kids with special needs.
Avera weight ge loss
36 pou is nds
remarkable quality of care.
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Jennifer Miller, MD
– William 00 1 minus S !! POUND
“He’s Not the Child We Brought to Pittsburgh” William’s family was in despair when they brought him to The Children’s Institute. Now, says Faith Weaver, “He’s amazing. He’s not the child we brought to Pittsburgh. He’s more cooperative, and for the first time we can plan things as a family. At The Children’s Institute, William himself and we as a family were given the tools we need to manage this difficult condition. We’re so grateful.” Program staff members are often asked to share their knowledge and expertise, for example by presenting, speaking and taking part in panels at professional meetings. Two staff members — Dr. greg Cherpes and Prader-Willi Syndrome Supports Coordinator Ken Smith — are on the Board of the national Prader-Willi Syndrome Association. Outpatient clinics at several hospitals across the country treat patients with Prader-Willi Syndrome, and The Children’s Institute is working on plans to offer professional development programming for the clinic professionals. The staff also participates in research to generate more knowledge about the condition. With the national association and partners at Duquesne university, they are investigating metabolic issues associated with the use of certain medications. Another study is comparing the impact of two different forms of exercise on patients’ Body Mass Index. And another will examine swallowing function in patients with Prader-Willi syndrome.
Staff members advocate strongly on behalf of patients with the syndrome, for example, for improved insurance coverage, for an increase in the availability of group homes specifically for people with PraderWilli syndrome, and for the availability of day programming and vocational programs. Meanwhile, the program has been praised as “exemplary” by the independent Commission on Accreditation of Rehabilitation Facilities (CARF), an international accrediting organization. Physicians who refer patients to the program are among its strongest supporters. jennifer Miller, MD, a pediatric endocrinologist in gainesville, Florida, follows 450 patients with Prader-Willi syndrome. When she sees a child who can’t improve even with excellent outpatient treatment, she refers that child to The Children’s Institute. She says, “The doctors and staff there are just phenomenal with these kids. I tell insurers all the time that there’s no place in the world like The Children’s Institute to get this remarkable quality of care.” Dr. Cherpes adds, “until there is a cure, refrigerators and pantries will need to stay locked. In creating the Center of Excellence, we are dedicated to unlocking human potential and clearing the barriers to healthy and fulfilling lives. Community support helps with program development and research to discover new keys to success.” ❖
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