Medical Update (2003)

Volume1/2003_Springupdate University of Utah Health System Spring 2003 W H A T ' S I N S I DE • u 2 Tour De Force: Pediatric Liver Transplant Program page 4 Regaining Heart: New Pacemakers page 6 Continuing Medical Education Spring Schedule . page 8 Lou Gehrig's Disease: It Takes a Team Sixty years ago when a rare illness forced Lou Gehrig to retire from baseball, he told a stunned Yankee Stadium crowd he considered him-self " the luckiest man on the face of the earth." Anyone familiar with his disease knew better. Amyotrophic lateral sclerosis ( ALS), or Lou Gehrig's disease, as it has come to be called, robs people in their prime of independence and, ultimately, life itself. And people diagnosed with it today can't expect their luck to be much better than Gehrig's. There is still no cure for the disease that brought down the crown prince of baseball in 1941. It starts in a limb, hand, or foot, or in the muscles that control speech and swallowing, inexorably killing nerve cells that tell the voluntary muscles to move. It progresses gradually through the body until it reaches the muscles that control breathing, making them too weak to pump oxygen to sustain life. Survival rates remain grim. Half of all ALS patients live two to four years from symptom onset, and just 10 percent survive more than a decade. It strikes slightly more men than women, and about 30,000 people have it in continued on page 2 Lou Gehrig's Disease: It Takes a Team ... continuedfrom cover the U. S. About 5 to 10 percent of cases have been linked to gene mutations. But amid the disheartening facts of ALS, medical science has made some strides. Besides a new drug, Rilutek, which can prolong survival for three to five months, a new concept in treatment has emerged since Gehrig's time. " Team management is an intervention that pro-longs comfortable survival," said Mark Bromberg, M. D., Ph. D., professor of neurology in the University of Utah School of Medicine. " Ten to 15 years ago, when physicians made the diagnosis, they said, ' There's nothing we can do for you. Go home. Put your affairs in order. There's no need to make a return appointment.'" But now teams of specialists at the U of U and other high- volume ALS treatment facilities around the nation provide a range of therapies and help patients adapt to a whole catalog of assistive devices. The goal is to keep patients as independent as possible for as long as possible. Bromberg's team sees a caseload of 60- 80 ALS patients at the University of Utah's Motor Neuron Disease Clinic, funded in part by the Muscular Dystrophy Association ( MDA), at the U's health center in Sugar House. ALS clinic manager Barbara Miano, R. N., B. S. N., coordinates an 11- member team that includes a case aide, pharmacist, research coordinator, social worker, speech pathologist, and physical, occupational, and respiratory therapists. Patients visit the clinic with their caregivers, usually spouses or family members. A visit may take as long as three to five hours, depending on how much time is needed with each team member. On each visit, Miano evaluates patients' progress and assigns them an ALS functional rating score. " If the scores stay constant, things are working," said Miano. " If not, we make adjustments in their care." As the disease runs its devastating course, the team members give therapy in numerous tasks that most people take for granted: walking, getting dressed, hold-ing a cup of coffee, gesturing to save the vocal cords, learning how to swallow safely. Eventually, patients will even need help eating and breathing, so therapists will coach them to accept the need for a gastric feeding tube and ventilator. Alderman's symptoms first appeared in his speech and swallowing. " My speech was slow and very, very monotone," he said. " I noticed it before anyone else did." Two months later others began to notice and asked him if he had been drinking. But Alderman has managed to turn his speech problem into an asset: Everything he says is as carefully crafted as though he were writing it. " It's a luxury to have time to get things in order, to savor life and to say goodbye. I was being diagnosed at the same time 9/ 11 happened. I told my wife ' I'm so much luckier than those people.' They went to work that morning having no idea. They did not have time to say good-bye. I do have that time." Father of three, Alderman is a credit manager at a national finance company, but corporate heads have decided it's time for him to take medical retirement. He's not happy about that, but he's trying to see it as an opportunity to spend more time with his family while he's still fairly healthy. The only medication for ALS is expensive, costing from $ 800 to $ 900 or more a month. A power wheel-chair will cost $ 20,000. Although Alderman is cov-ered under Medicare, his wife, Shaun, has gone back to work as a marketing services representative for a credit card company so the rest of the family can have health insurance. " I think that we as a country need to do some-thing," said Alderman, " about the incredible personal and financial hardship ALS and other patients endure." Alderman has already done a lot to fight ALS. Last August he got his employer to sponsor a golf tourna-ment that raised $ 45,000 for the clinic, and his moth-er- in- law found a benefactor who is donating $ 32,000 On the cover . .. Alan Alderman ( upper right) gets a lot of support from the home team: ( clockwise from lower right) son Justin, dog Nikita, daughters Breanna and Jessica, and wife, Shaun. 2 Backing Alan Alderman in his battle to maintain independence are Mark Bromberg M. D., and ALS team members ( from left) Summer Davis, Natalie Bee R. T. T., Michele Halstenrude, Brenda Dee L. C. S. W., Barbara Miano R. N., B. S. N., Nancy Ivy, R. P. T., Judy Hendrickson O. T. R./ L., Michelle Taggert M. S./ C. C. C., and Orly Carter, Pharm. D. a year to help fund the research coordinator position. His disease has introduced Alderman to a world of friends he wouldn't have known otherwise. He is an active member of two monthly support groups, one in Salt Lake City sponsored by the U of U and MDA and one sponsored by the Amyotrophic Lateral Sclerosis Association in Roy, Utah. The groups give patients a chance to discuss quality of life issues and swap ideas and strategies for coping. Patients compare information about nutrition, hospice care, and assistive devices. Sometimes instructions are shared for assembling homemade devices that work as well as the more expensive gadgets in the catalogs. They laugh together, too. " They call ALS a nice guy's disease," said Alderman. " So I told our support group one month that, ' Well, if ALS is a nice guy's disease, then to cure it we all have to get mean. " The Internet has become a big part of Alderman's life. Between it and the library, he spends five to eight hours a week keeping up on research. He's in touch with a worldwide net-work of ALS friends via e- mail, and he often e- mails the team at the clinic. Team members are quick to give their opinions in response to his questions about research and alternative treatment. " When I was deciding which speech communication device to purchase," said Alderman, " the e- mail feedback I received from them was invaluable." " They're absolutely wonderful. Not only do they help us at the clinic, but they're willing to really help us anytime. One thing they do here is help us live with ALS. What I fear is living a life where I'm totally dependent on others for everything that 1 need, everything that I do. Even the most basic things in life that we have all done since we were 2 or 3 I'll eventually need help with, and giving up that independence frightens me much more than death." • © For more information call the Department of Neurology, 801- 585- 2443. University of Utah Hospitals & Clinics toll- free referral and consultation: 1 - 8 0 0 - 4 5 3 - 0 1 2 2 in Utah, I - 8 O O - 6 6 2 - O O 5 2 outside Utah Tour De Force Pediatric Liver Transplant Program Gives Infants, Children, Teens New Hope for Life Nicole Flores celebrated her first birthday in the hospital- desperately needing a liver transplant. Jaundiced, and getting sicker each day, Nicole would wait three more months and lapse into a coma before a suitable liver became available. But in a donor system that often can't provide an organ until a patient is gravely ill, Nicole received a liver just in time. Nicole was born in Ecuador and lives in West Jordan, Utah, with her mother, brother, and sister. Now 7, when Nicole received her liver several years ago she was one of the first patients in the pediatric liver transplant program of the University of Utah School of Medicine's departments of Surgery and Pediatrics at Primary Children's Medical Center ( PCMC) in Salt Lake City. The program is the only one in Utah, Idaho, Wyoming, and Montana, and provides lifesaving liver and combined liver- kidney transplants for infants, children, and teens. Since the program began in 1996, about 55 patients, ranging from newborns to people in their early 20s, have received livers, according to Linda S. Book, M. D., U professor of pediatrics and medical director of the program. " It's truly a cooperative effort for children," says Book, a gastroenterologist and liver specialist who was the driving force behind the program. Transplants involve a labyrinth of logistics, and a large team undertakes the sizable job of transplanting new livers for critically ill children. The pediatric team at Primary Children's includes four pediatric gastroen-terologists, two pediatric surgeons, two nurse coordinators, a social worker, a dietitian, and a secretary. The team collaborates with the LDS Hospital adult liver trans-plant program, which includes two liver transplant surgeons and four adult gastroenterologists, all of whom are adjunct faculty at the U medical school. During the transplant and postoperative period additional support comes from operating room nurses, anesthesiologists, radiologists, and physicians specially trained in critical care, also called intensivists. Teamwork is vital for success. When a donor organ becomes available, a coordinator is paged. In the ensuing four hours, the coordinator will make approximately 30 phone calls to mobilize the surgical team, alert the patient, and track the donor organ. And it will take several more hours and dozens more calls to line up every detail before surgery begins. In all, 24 hours can pass between when the coordinator is paged and the transplant completed. " Timing of the donor and recipient surgery is the most crucial aspect of coordinating," says coordinator Lacey Bruschke. Livers last only a short period outside the human body, so it's crucial to minimize the time between procuring the donor liver, preserving it, and transplanting it. Currently, seven children are on the active waiting list for liver transplants at Primary Children's. One child is designated status 1, which signifies an urgent need and top priority to receive a liver transplant. There are other candidates on the inactive list who will be activated for transplantation as their liver functions begin to deteriorate. These may not sound like large numbers, but finding donor organs is a race against time, and the demand far outpaces the supply. Nationally, the waiting list for all liver transplants has increased 25- fold in the past 10 4 When Nicole Flores, 7, needed a new liver, her mother, Martha Palacios, moved from Ecuador to Utah to get her daughter the operation. Nicole received a new liver when she was 15 months old. i When a liver becomes available for transplantation, the pediatric team swings into action. Team members: Front row, ( left to right) Lacey Bruschke, R. N., C. C. T. C., Molly O'Gorman, M. D., Linda Book, M. D., Cyndi Kawai, A. P. R. N. C. C. T. C. Back row, ( left to right) Valerie Stephens, L. C. S. W., Steve Guthery, M. D. Rebecka Meyers, M. D., Daniel Jackson, M. D. Missing: Eric Scaife, M. D. years. One- quarter of all infants and young chil-dren who need a liver transplant in 2003 will die waiting, according to Book. This year, 10 to 15 infants and children will receive livers through the pediatric transplant program. Most of those children, 75 percent, will receive livers from cadavers. The remaining 25 per-cent will receive organs from living donors, usually related to the recipients, who give part of their liver to the recipient. Most infants and children need liver transplants because of biliary atresia, a disease in which the liver does not have a bile duct. When this happens, the liver cannot drain bile into the small intestine and becomes poisoned and scarred. Jaundice is typically the first symptom. About 20 percent of children with biliary atresia are born with the condition. The remaining 80 percent acquire it through unknown circumstances, although Book and other physicians suspect a virus may cause it. If diagnosed before an infant reaches 3 months, biliary atresia can be surgically corrected. But after that, the liver usu-ally sustains too much damage and the child will need a trans-plant. Book has seen eight babies with biliary atresia in the past year, an unusually high number. " The disease can occur in epi-demics," she says. " And that's what is happening now." Although liver transplants are performed routinely, such surgery is never routine. These are complex operations that often take up to eight hours and can last as long as 20, accord-ing to Rebecka L. Meyers, M. D., U of U associate professor of surgery and chief of pediatric surgery at PCMC. Meyers, along with Eric R. Scaife, M. D., assistant professor of surgery, per-forms the pediatric liver transplants in conjunction with the adult transplant surgeons from LDS Hospital- John B. Sorenson, M. D., adjunct instructor of surgery and LeGrand P. Belnap, M. D., adjunct associate professor of surgery. Several factors make liver transplants challenging procedures. Unless the donated liver comes from a size- matched child, transplantation in infants and children involves cutting down an adult liver to fit into a smaller body. Adult cadaver livers sometimes can be split in two in a procedure called " split- liver." The smaller piece goes to a child and the larger to an adult. Alternately, the child's living parent or another relative might donate a small piece of liver. Working with infant and child blood vessels- less than 2 or 3 millimeters in diameter in the smallest babies- makes pediatric liver transplants more treacherous. Surgeons often use magnifying loops or even microscopes to get a clearer look at these tiny vessels. " These transplants can be a tour de force," Meyers says. " You're operating on tiny babies with tiny livers supplied by tiny blood vessels." The liver also makes the body's blood- clotting factors, and blood loss increases during transplantation because there's little or no clotting. For all the challenges of liver transplantation, children who undergo the operation through the U of U- PCMC program have the numbers on their side. At least 90 percent of patients in the program survive a year or longer. About 10 percent of the survivors receive a second transplant to treat complications such as blood clots or poor liver function in the transplanted liver. Both of those rates equal or better national averages, according to the United Network for Organ Sharing. The five- year patient survival rate is an impressive 90 percent. Nicole Flores wouldn't have survived without a new liver. But life after the transplant has been trying, and downright scary at times, according to her mother, Martha Palacios. Palacios learned her daughter had biliary atresia when Nicole was 3 months old. But a doctor in Ecuador said a transplant had only a 5 percent chance of succeeding, and he discouraged Palacios from seeking one for Nicole. She wasn't about to give up, though. When her sister, who then lived in Provo, Utah, told Palacios about the U of U-PCMC transplant program, she moved to give her daughter a chance to live. Despite Nicole's grave condition, the operation succeeded, and the liver started functioning almost immediately. But Nicole developed rejection problems and has battled them since. She's also highly vulnerable to infections because of the immunosup-pressants she takes to keep her body from rejecting the liver. Despite this, Nicole is a playful, chatty child who most people would not suspect takes a lot of medication and has had a transplant. " I think it's always going to be scary," Palacios says. " When you get a transplant, you trade one thing for another." But, as she watches her daughter grow up- playing and giggling like any other child- that's a trade- off Palacios gladly accepts. • For information on the pediatric liver transplant program call 801- 588- 2777. For information about organ donation call 866- yes- utah ( 937- 8824) or visit www. yesutah. org. University of Utah Hospitals & Clinics toll- free referral and consultation: 1 - 8 0 0 - 4 5 3 - 0 1 2 2 in Utah, 1 - 8 0 0 - 6 6 2 - 0 0 5 2 outside Utah Regaining Heart: New Pacemakers, Defibrillators Save Lives of Patients Whose Hearts Are Too Weak To Pump Enough Blood Jim Daniel did well in the annual Wyoming elk and moose hunts last fall. Rather than tend to the fire while his family tramped around the woods looking for game, Daniel, 70, was able to join the hunt. That's been rare for the Rock Springs, Wyo., resident in recent years because of heart failure that had drained his energy and left him sitting by the campfire. Daniel has low ejection fraction, also called weak heart, a condition in which his heart cannot pump enough blood when it beats. This puts him at risk for sudden cardiac death- his heart may stop functioning alto-gether and kill him without warning. He was diagnosed in 1988 and his heart had grown progressively weaker through the years until even some-thing as simple as going grocery shopping was too taxing. Daniel had resigned himself to sitting on the bench. But his for-tunes changed in May 2001 when he joined a national study through the University of Utah School of Medicine to test a combination pacemaker/ defibrillator for people with chronic heart failure. The device, made by Guidant Corp. and called CONTAK CD, uses a new generation of pace-maker that synchronizes the contraction of the ventricles so the heart pumps more blood, as well as reducing the risk of sudden cardiac death in people like Daniel. In the event the heart fibrillates, or starts beating too fast, and then stops, the defibrillator jump- starts it with an electrical jolt that restores the normal rhythm. 6 The CONTAK CD has made all the difference for Daniel. He started feeling more energetic almost immediately after receiving the device. " I feel like I'm going to live," he says. " Before, I didn't give a damn whether I lived or not." But the device also has proved to be a lifesaver- five times- by restarting his heart after it stopped beating. The first time happened while he was repairing a water line in a recreational vehicle. The next thing Daniel knew, he was waking up after the defibrillator had zapped his heart back to life. Four other times his heart stopped and the defibrillator brought him back. Physicians don't know why he or others develop low ejec-tion fraction, although they suspect a virus may cause it. When his problem was diagnosed, Daniel was put on medications- diuretics, potassium replacement, angiotensin converting enzyme inhibitors and beta blockers- that were the best course of treatment at the time. But as the years passed, the medica-tions became less effective and Daniel's heart grew progressively weaker, according to his physician, Edward M. Gilbert, M. D., a cardiologist and professor of internal medicine at the U medical school. The national trial, called COMPANION- Comparison of Medical Therapy, Pacing, and Defibrillation in Chronic Heart Failure- was started in January 2000. Participants had to have been hospitalized at least once in the prior 12 months for low ejection fraction or bundle branch block, a problem that pre-vents the ventricles from contracting synchronously and reduces the volume of blood they pump. Daniel was a good candidate. By the time he joined the trial in May 2001, he had up to a 50 percent chance of dying within a year, said Gilbert, the study's principal investigator at the U. Roger Freedman, M. D., professor of internal medicine and interim chief of cardiology, served as co- principal investigator. Trial participants- 600 congestive heart failure patients nationwide, 20 of them in Utah- were split into three groups: 20 percent received only medication for their condi-tion; 40 percent received Guidant CONTAK TR pacemakers; and 40 percent, like Daniel, received CONTAK CD pace-maker/ defibrillators. The results were dramatic. Compared with those who received medication only, patients with the pacemaker/ defibrillator experienced a 40 percent reduction in the risk of death or hospitalization. Those who received the pacemaker alone experienced a 20 percent reduction in the risk of death or hospitalization. Defibrillators appear to be particularly effective in saving lives of people with low ejection fraction. The results were so impressive that the trial, originally scheduled to run three years, was ended more than a year early last November. Results of the study were presented at the American Cardiology Convention in Chicago in early April. The trial results will change the standard of care for congestive heart failure patients, according to Gilbert. " Defibrillation therapy should strongly be considered in most patients with weak hearts," he says. " This device will reduce the risk of death." For people with bundle br