Underlying Diseases

Organ transplants don't often "just happen." For a vital organ to lose so much function that it needs to be replaced, some disease has progressed to the point that it's causing serious problems. While the underlying cause is sometimes unknown (a single-digit percentage of cases), some underlying diseases are pretty frequent in the transplant world. Despite the commonalities of the transplant experience between patients, specific diseases tend to share an even more common experience. Also, many of these diseases have effects, stories, and communities that go beyond the subset that are transplant-specific. To highlight some of the places where transplant patients come from, this post is a summary of four specific underlying diseases.

Diabetes

Diabetes mellitus is what happens when the body has a lack of insulin activity, either because insulin can't be produced (Type 1 diabetes) or it can't be used (Type 2 diabetes). It's an extremely common disease, affecting 20.8 million Americans in 2005. Diabetes doesn't just affect sugar metabolism, but the use of carbohydrates, fats, and proteins. It can affect almost every organ system, including kidneys, peripheral nerves, eyes, and blood vessels of all types. A number of treatments are available, from medications of all types to injected insulin to insulin pumps to better regulate blood glucose levels to transplants.

Diabetes has a strong interplay with transplantation. It isn't just the indication for essentially all pancreas transplants; diabetic nephropathy is also the #1 disease causing the need for kidney transplants as well. Beyond those who have a transplant for diabetes, many transplant patients have diabetes as an additional disease. Also, diabetes can be a side effect of immunosuppression after transplantation, which is categorized as post-transplant diabetes mellitus (PTDM). Finally, type 1 diabetes is an autoimmune disease, and both diabetes and chronic rejection of transplants affect blood vessels, so basic research in diabetes and transplantation play off each other to lead to new advances for both groups.

Any disease that is so common is bound to have a large community on the Internet and in medicine in general. The American Diabetes Association is the center of this community, supporting everything from educational resources to research funding and advocacy. Unfortunately, there aren't too many blogs about diabetes or kidney or pancreas transplants related to diabetes. There are a couple in the "Adult Kidney & Pancreas Transplant Blogs" section, and other great blogs about diabetes in general such as Diabetes Mine and Six Until Me. Still, beyond the blogs or broadcasted stories, diabetes is an important part of transplantation.

Biliary Atresia

Biliary atresia is the most common underlying disease for pediatric liver transplants, responsible for about half of all pediatric cases and a much greater proportion of those in the youngest age groups. Biliary atresia is a rare and poorly understood disease. The number of new cases each year number in the hundreds, and the cause is not congenital, not genetic, not contagious (though possibly infectious), and...not known. The ultimate problem is that bile ducts "drop off" and don't develop fully enough to drain bile from the liver. No organ does well when it's not drained, and the liver is no exception, which leads many biliary atresia patients into cholestatic liver failure. The stories between patients are shockingly similar: initially assuming it was typical neonatal jaundice, undergoing a Kasai portoenterostomy to attempt to restore bile flow, and children going through the transplant experience at a very young age, with parents and families thrown into this world with them. Even though the condition is extremely rare, and everyone's life is different, the number of commonalities can make biliary atresia quickly seem like a relatively familiar disease.

Those with biliary atresia seem to have taken to blogging in great numbers, and an incredible number of individual stories are available in the "Pediatric Liver Blogs" section here. In addition, there are a few blogs of people facing biliary atresia but not requiring transplants, such as Imagine Bright Futures. Beyond blogs and Internet communities, those affected by pediatric liver diseases have assembled larger organizations such as Liver Families and C.L.A.S.S. (Children's Liver Association for Support Services).

Short Bowel Syndrome

Short bowel syndrome has a unique position in transplantation: the most common indication for the rarest type of transplant (intestinal transplant). The causes of short bowel syndrome fill an entire textbook of pediatric surgery, but the consequences are surprisingly uniform. The problems include intolerance to feeding (malabsorption, diarrhea, etc.), difficulties with vascular access for total parenteral nutrition (TPN), and the need for transplantation either from liver damage due to TPN or from losing sites for vascular access. "Tummy problems" sound a lot less exciting than heart, lung, or brain diseases, but the complications of short bowel syndrome can be as serious or deadly as any condition out there.

Short bowel syndrome and intestinal transplantation are a uniquely cloistered community. Only a handful of centers even attempt intestinal transplantation, and only a few of these have programs of any significant volume. Most people with short bowel syndrome end up at one of the institutions that have dedicated efforts in this area. Furthermore, many patients go between centers for second opinions, which leads to an even tighter network in this group. The short bowel community uses the Internet to the fullest, with a web site and mailing list (Gifts From Heaven), several blogs (such as The Short Gut News) and even a wiki.

Cystic Fibrosis

Cystic fibrosis is both simple and complex. For the simple part, it's caused by mutations in a single gene, for a single protein, with the general effect of impairing salt movement across membranes (specifically chloride ions). Water follows salt, and a lack of water movement makes various secretions thicker than usual. The complex part is the effects. Thick mucus in the respiratory system causes chronic and repeated lung infections (and sinus abnormalities) which get worse over time. In the gastrointestinal system, it can cause bowel obstruction in infants, liver disease, and pancreatic damage leading to diabetes. Cystic fibrosis is also unique in that it is always present from birth (though may not be recognized for years) and is a lifelong affliction. The survival of cystic fibrosis patients has improved incredibly, but just about all patients born with cystic fibrosis will die of cystic fibrosis, which means that there's far more to be done.

The vast majority of transplanted cystic fibrosis patients have lung transplants, with a few needing liver transplants, intestinal or multivisceral transplants, or heart-lung transplants. Cystic fibrosis is one of the top three diseases leading to the need for lung transplantation, and about two-thirds of pediatric lung transplants are for cystic fibrosis.

The cystic fibrosis community is uniquely organized and expressive. For the organizational end, the Cystic Fibrosis Foundation has led efforts ranging from research funding to center establishment to patient support. A recent New Yorker article by Atul Gawande discusses the extreme persistence and diligence needed in cystic fibrosis care, and the role of specific CF centers, the Cystic Fibrosis Foundation, and the CF community in striving for the absolute highest quality of care possible. As for Internet resources, the web site CysticFibrosis.com consolidates an incredible number of resources on the Internet, from foundations and support groups to mailing lists, blogs and wikis. The "Lung Blogs" section here has a number of blogs by cystic fibrosis patients and they tend to be especially well-written and vivid descriptions of living with both an organ transplant and chronic illness in general.

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Donation After Cardiac Death (DCD)

The very first experimental organ transplants came from donors who had recently died, whose hearts had stopped beating. These were superceded by living organ donations between twins and other relatives for the first "successful" kidney transplants, but "dead donors" would ultimately make up the bulk of organs donated. The earliest and most logical definition of "dead" was supremely intuitive--those whose heartbeats and breathing had stopped. Over the course of several years, brain death came into the picture as a new definition of death, and the 1968 Harvard Ad Hoc Committee on Brain Death formalized its definition and criteria. Brain death generated some controversy, but has become accepted as a medical and legal definition of death, and brain-dead patients became the pool of candidates for organ donation. However, some in the transplant profession had not forgotten the early donors, and in the mid-to-late 1990s the practice of recovering organs from donors whose hearts had stopped was resurrected as "donation after cardiac death" or DCD.

The principles of each are simple. Brain death is when the brainstem has been damaged beyond repair, when someone cannot breathe or maintain essential life functions without artificial support. Brain death can be diagnosed and declared by standard criteria, and after brain death is declared, death is declared as well and life support is removed. This time period is also the window for possible organ donation. DCD is for donors who do not meet brain death criteria but still have no meaningful chance of survival. In these cases, mechanical support is withdrawn as it commonly is for many people in these circumstances. The only difference from the usual practice is that organs are recovered after death for transplantation.

UNOS

In the decades between the early and the recent use of DCD, the biggest change in the US transplant system was the development of UNOS. Its history is another very long post, but the end result is that UNOS allocates organs for transplant, sets up the rules and systems by which organs are allocated, and sets conditions that member hospitals have to follow to legally get organs for transplant. UNOS is responsible for current DCD practices, and it also holds responsibility for an extremely long waiting list and a huge number of transplant centers. Recently, faced with criticism over its effectiveness, incoming UNOS President Sue McDiarmid, a pediatric liver transplant specialist at UCLA, set the amazing goal of eliminating death on the waiting list of children. No child would die waiting for a transplant...this is a result that could definitely show UNOS's effectiveness to be beyond question. It would obviously require an increase in organ donations (in addition to tweaking current practices) and this increase could logically come from DCD donation.

UNOS set forth with proposals to increase DCD donations, including requiring all hospitals to have a DCD policy. This effort was not met with unbridled enthusiasm, but instead with a lot of resistance. The report of a recent meeting of the UNOS Pediatric Committee details the resistance, which is especially strong for pediatric donors. This is ironic given that the push was partly designed to help children needing transplants, but it is also very understandable given the medical uncertainty of prognosis in children and the pure tragedy of a child's death. To understand and work through this concern, UNOS recently held a Summit on Pediatric Organ Donation and Transplantation, including both pediatric transplant professionals and pediatric intensivists (ICU physicians). A report from this summit is not available, but one fact that has been brought up is that pediatric intensivists are often in the unique position of caring for both potential organ donors and recipients. While most in the transplant field aren't in this same position, they are experienced medical professionals, so have undoubtedly dealt with issues surrounding the death of a patient.

Just as the initiatives to increase DCD were gaining momentum, a devastating blow was dealt to the practice by the misconduct of a transplant surgeon. The Los Angeles Times exposed the story of a transplant surgeon seeming to order medications for a potential DCD donor. The intent may or may not have been to hasten the donor's death, but even the presence of the transplant surgeon before death is improper and filled with conflict. Faced with such a blatant violation of DCD practices, the reaction of the transplant community was relatively poor, with a response from UNOS that was essentially a non-response of "we can't comment on an ongoing investigation" and grumblings from others in the transplant world that "The LA Times is at it again..." (my reaction being "don't shoot the messenger"). While this incident never should have happened, and cast DCD donation in a poor light, confusion is not limited to the DCD situation, with the LA Times recently reporting confusion in declaration of brain death of a potential organ donor. This confusion isn't even limited to cases where organ donation is a consideration, with the story a couple of years ago about a girl in California who was declared dead but was actually alive.

Current Opinions

Unfortunately, current opinions about DCD seem to be drastically negative. The Washington Post recently ran a feature article on DCD, discussing the resistance and including a number of quotes that are quite questionable. Jerry Menikoff of the University of Kansas stated that "we're starting to remove the organs a few minutes before they meet the legal definition of death" which is completely contradicted by the multiple statements earlier about how organ recovery begins several minutes after death, not a moment before. Dr. David Crippen of the University of Pittsburgh asks if "we're going to take organs from patients who have a prognosis of death but who do not meet the strict definition of death, might we become more interested in taking organs from patients who are not dead at all but who are incapacitated or disabled?" which is also false since organs are not taken from patients when they "have a prognosis of death" but rather when they are dead. The only published reply from the transplant community was a brief letter to the editor by UNOS President Sue McDiarmid, stating generally that some statements were not fully true. Since this article has been circulated so widely in the blogosphere and beyond, it's concerning to me that it's been contested or debunked so little.

Blogs have a great deal of insight into current opinions of DCD, insight that builds on the published works and expands on them. A comment to a post on the popular blog Kevin, MD include the information that all of the pediatric anesthesiologists at St. Louis Children's Hospital have refused to participate in the practice. A similar feeling has been reflected at other hospitals, but the bluntness and honesty of this response is particularly telling. A more deeply insightful post on pediatric DCD comes from Donorcycle, a blog by a transplant procurement coordinator with an excellent post titled "When You Watch A Child Die".

My opinion on DCD donations is that they're as rough as everything else. Donations after brain death also come under tragic circumstances and strike a family at the time of a loved one's death, and living organ donation is fraught with worries of its own. DCD strikes me as being equally viable and as beneficial as other forms of organ donation, so I see no reason why it shouldn't be supported and expanded. That said, it does seem like the transplant community's efforts in this area haven't panned out as well as they could. My suggestion for the transplant community is (1) set clear policies and regulations to enable DCD to happen in an ethical and respectful manner and (2) show no tolerance for breaches of these policies, affirming that we strive for maximal organ donation within strict bounds of ethics and dignity. My request to the rest of the world is much simpler: please bear with the transplant community during changing times, understand just how valuable organ donations are, and how profound it is to "donate life."

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Beth Israel Deaconess Medical Center

Like every transplant patient, the Beth Israel Deaconess Medical Center (BIDMC) has been through a lot, but sweeping changes have made the transplant program stronger than ever. Kidney transplantation began at the Beth Israel Hospital in 1973 under the direction of Dr. Anthony Monaco, and liver transplantation began at the New England Deaconess Medical Center in 1983 under the direction of Dr. Roger Jenkins. In 1996, amid drastic changes in the US health care system, the hospitals decided to merge. The merger led to a serious culture clash, prompting the entire liver transplant program to relocate to the Lahey Clinic. Bad turned to worse, as the hospital re-established a liver transplant program with grand plans: pediatric liver transplantation, living donor liver transplantation, and a "model program" for the rest of the world. One year later, the program was suspended in the wake of five deaths, which represented 25% of the transplants performed. The drama of the merger as a whole is the subject of the book Code Green, which thoroughly documents the events of the merger, mostly from the perspective of the nursing staff and with a seeming bias towards the Beth Israel side. The book ends with the recruitment of Paul Levy as CEO of BIDMC.

Resurrection

Paul Levy saved BIDMC, using every viable tactic imaginable. He offered joltingly honest assessments of the hospital's situation to the staff. He created a partnership with the Boston Red Sox. He even started a blog. However, development of clinical programs would rely on new clinical leadership as well as strong administrative support. Dr. Josef Fischer had recently joined the institution as Chairman of Surgery, and he in turn recruited top doctors to reinvigorate major divisions. Dr. Douglas Hanto was selected as the new chief of transplantation. He proceeded to recruit additional staff members and lead the existing ones, and together they set to the task of restoring the transplant program. All of the people in the multidisciplinary team, from surgeons to coordinators to administrators, were co-located to the same office and clinic area, fostering both patient convenience and close personal interactions among the staff across traditional boundaries. To ensure safe and thorough clinical care, detailed clinical pathways were established and a transplant-specific electronic medical record (EMR) system was implemented. The services of the transplant center were also expanded to include:

• a Dialysis Access Center to ensure essential care for patients with end-stage renal disease (often awaiting transplantation),
• a hepatobiliary surgery program for liver diseases that may not require transplantation but still require major surgery, and
• a strong relationship with the Joslin Diabetes Center, which includes a kidney-pancreas transplant clinic at Joslin as well as a program in the evolving procedure of islet transplantation.

These changes have been a success, and today Beth Israel Deaconess has a balanced and busy program in kidney, liver, and pancreas transplantation with solid outcomes. They have also used their clinical and research strengths to create educational programs for future transplant specialists, including a transplant nephrology fellowship that is the only one in Massachusetts accredited by the American Society of Transplantation and a transplant surgery fellowship that is the only one in all of New England accredited by the American Society of Transplant Surgeons to fully qualify surgeons to perform kidney, liver, and pancreas transplants (other fellowships in the area are limited to one or two of these organs). Dr. Hanto summarized the efforts and results of the transplant program: "What has occurred has required real team work, hard work, committment, and willingness to think outside the box."

BIDMC Transplant Center Today

With the crises of the past seemingly resolved, Beth Israel Deaconess has moved beyond fixing problems within itself to advancing the field of organ transplantation as a whole. They have sought to expand the deceased donor pool with the use of expanded criteria donors (ECD) and donation after cardiac death (DCD), and the institution received recognition from the Organ Donation Breakthrough Collaborative for recovering organs from more than 75% of eligible deceased donors. Also, to serve the overall goal of increasing the availabilty of transplantation, BIDMC is participating in a study of kidney and liver transplantation in patients with HIV, and is currently the second-most active center in this study (behind UCSF). This is one of several clinical trials taking place at BIDMC, and research efforts also include historically strong basic science research and recent funding to establish a Center for Transplant Outcomes and Quality Improvement within the Transplant Center.

Patients and Donors

In the past, the hospital has declined to perform living donor transplants facilitated through solicitations (Web sites, billboards, etc.). This view has prompted intense discussions and quite a bit of disagreement. However, BIDMC currently is willing to evaluate candidates and solicited donors as they do any other patients. The change is not due to a shift in their beliefs, but rather a recognition that the issue remains unresolved in the transplant community and is permitted under current regulations. Given this, their desire is to not force patients to go elsewhere, or have them "caught in the middle" of an ongoing global debate, if they do find a donor in these ways. Clearly, soliciting donors will continue to be controversial both at Beth Israel Deaconess and far beyond. BIDMC would prefer to increase living donation through altruistic donors and is part of the New England Program for Kidney Exchange, through which they have recently participated in a three-way kidney swap. Though their firm beliefs on solicited donors have brought criticism, BIDMC does support many efforts to meet patient needs, including educational programs, support groups, and a one-of-a-kind Behavioral Care Program for transplant donors and recipients.

The Beth Israel Deaconess transplant program has lived through a long history and is gradually pushing forward towards the future of organ transplantation. Their "style" does not seem to support radical innovation, yet their results in transplantation are solid and they devote a fair amount of energy towards collaborative programs and clinical trials on new therapies. Like every transplant patient asked "What happened? How are things?" the answer is "It's a long story..." Thankfully, in the case of Beth Israel Deaconess Medical Center, it's now a good one.

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About the Center Profiles

After the general post on how to look up a transplant center, I've decided to include profiles about specific centers on this blog. The purpose of these profiles is to describe transplant centers beyond the easily-obtainable information. They won't be simple links to the information described in the earlier post, but will include information (on the Internet and otherwise) that show what a center has done, what they're "up to" currently, and anything that is truly unique about one center versus most or all others. I'm thinking of doing approximately one a month, though this frequency may change depending on how the blog evolves and how busy a news day it is in the rest of the transplant world.

Centers will be chosen to be profiled with roughly the following priority:

1. Request of a transplant center to be included
2. An inquiry from a transplant patient or candidate, or a caregiver of one, about a specific center
3. Nomination by a previously-profiled center
4. A center that has been in the news, has a vivid history, or just flat-out intrigues me

I'll be writing these profiles myself, so any opinions expressed will be mine. I will attempt to contact the director of the transplant program being profiled and ask them to actively participate in two ways: first, to bring key features of their program to my attention, and second, to check what's written for factual accuracy. However, they won't be writing the profile and will not be able to censor unflattering-but-accurate content.

Comments will be open, and I hope that this will be the more interesting portion of the post. Anyone who has information or an opinion on a specific center is welcome to comment. Anonymous comments are fine. That said, "flaming" or personal attacks won't be permitted and will be deleted. Also, if you have a concern or question about a specific clinical situation, please contact the transplant center directly rather than post a comment.

Let's see how this goes...

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