Health System Requirements for Pediatric Liver Transplantation

Introduction

The ultimate goal for a pediatric liver transplant (LT) program is for transplant recipients to live a normal lifespan free from disease recurrence or long-term morbidity. The healthcare needs of transplant recipients range in extremes from critical care early on to the maintenance of allograft and patient health in the years following transplant. The needs of patients are distinct over the phases of transplant care, each of which presents unique challenges for the patient, family, and transplant program. Understanding the barriers to care at each phase will allow a transplant program to adequately mitigate potential issues that may arise. Consequently, the metrics used to monitor a program need to be specific to a patient’s phase of care. Finally, attaining this goal requires an institutional understanding of the interdisciplinary nature of transplant medicine and the key stakeholders needed to ensure the best outcomes. Members of the transplant team are needed in different capacities during each phase of transplant and often have diverse roles outside of transplant within a hospital system. As such, the transplant program needs to have mechanisms in place to ensure team cohesion.

Michael Porter’s definition of value may serve as a framework for the metrics used in a transplant program. Porter’s concept of value is defined as health outcomes achieved divided by cost and should be evaluated from the perspective of the patient. Under this concept, value has three tiers. Tier 1 is the survival benefit. Tier 2 refers to the recovery time and minimizing the disutility in care (e.g., making care more efficient and less wasteful). Finally, Tier 3 involves the sustainability of health and preventing complications related to treatment. Specific to pediatric liver transplant, tier 1 is the overall survival benefit of transplant. This metric is often tracked by national organizations (e.g., United Network for Organ Sharing [UNOS]). Tier 2 refers to the efficiency of the healthcare system (e.g., complications). Tier 3 refers to long-term allograft and recipient health without complications from transplant or medications (e.g., nephrotoxicity or developmental delay).

The needs of patients are vastly different over the continuum of the transplant course. The transplant course can roughly be categorized into four phases (see Fig. 3.1 ): (1) referral/candidate selection, (2) waitlist management, (3) transplant and immediate post-operative care, and (4) long-term management. Therefore, specific objectives for each phase of care need to be identified and, from those objectives, metrics developed to track program success. In addition to monitoring best-practice metrics across this continuum, identifying critical events at each phase of care serves as an important mechanism to ensure the best outcomes. When a critical event is identified, a determination will need to be made as to whether this event can be addressed with (1) change to clinical care, (2) system-wide quality improvement, or (3) original research.

Team cohesion is paramount to the success of a transplant program. Often in a hospital system, many of the team members are only occasionally involved in the care of transplant recipients (e.g., medical trainees, intensive care unit members). Furthermore, many individuals on a transplant team often have different reporting structures. Lessons from other disciplines can be applied to transplant medicine to build team cohesion and establish strong working relationships among team members. These include shared goals, strong interpersonal relationships among team members, and organizational support for interdisciplinary teamwork. Furthermore, the concepts of a team champion and a balanced culture are important to fostering strong interdisciplinary teams.

The rest of the chapter will address the role of teams in transplant and then address each phase of care and discuss specific objectives at each stage, potential barriers to care, and important metrics that should be tracked. At each phase of care, potential cost drivers will also be discussed. The chapter will also briefly present research on the financial implications in pediatric liver transplant. Finally, the concept of the ideal outcome (IO) will be discussed.

Team

Pediatric LT requires a sophisticated understanding of the diverse knowledge needed to deliver exceptional care. Wagner’s Chronic Care Model highlights the importance of teams in chronic disease management. Multidisciplinary teams are necessary to ensure that care being provided is population focused (i.e., specific to the population served), based on treatment plans, evidence based, and supportive of self-management. To attain these goals, an understanding of the interdisciplinary knowledge needed and the complexity of team-based care are necessary. Teams allow for complex problem-solving to occur; however, the healthcare setting poses potential barriers to team effectiveness. In traditional hospital structures, teams often include transient members (e.g., medical trainees) and members across reporting structures (e.g., members from different divisions).

The foundation for effective team-based care lies in the organization. Organizations with a commitment to total quality management have been found to have higher team effectiveness. In healthcare, this means an organizational commitment to the patient and patient outcomes. Although organizational commitment is external to the team, organizational commitment to total quality management likely fosters a culture that allows teams to attain the additional education, training, and resources necessary to succeed. Therefore the organizational culture cannot be understated.

Research has also identified several factors within a team that lead to increased effectiveness. Notably, a team champion has been identified as important to team effectiveness. A team champion can be thought of as someone who naturally facilitates change within the team. Team stability impacts the effectiveness of teams, and this can be challenging in a healthcare setting with revolving staff members (e.g., consultants, trainees, etc.). Therefore a shared vision is critical to ensure that the team functions effectively despite the barriers imposed by the healthcare system. Finally, although transparency has been identified as an important element of healthcare delivery, affording the team opportunities for private conversations has been found to lead to greater innovation and higher productivity.

Referral and Candidate Selection

Pediatric LT is best delivered at centers of excellence or those who perform a sufficient number so as to build institutional experience and expertise. Commensurate with regionalized care delivery, the issue of access to highly specialized care becomes an important consideration. In the United States, it was found that adult patients who lived farther away from the transplant center were less likely to be referred for transplant. This finding is troubling and highlights the attention needed for distant and potentially disadvantaged patients. For patients traveling a significant distance, initiating the transplant evaluation locally may relieve some of the financial, emotional, and logistical strain faced by these families. Professional relationships and ongoing dialogue between a pediatric LT program and the surrounding geographical communities is essential to ensuring that patients in need of a liver transplant are referred in a timely fashion.

Once patients are referred for transplant, efficiently evaluating and recommending whether to list the patient is necessary to ensure the best outcomes for the patients. Such an approach may include written criteria that result in fair and nondiscriminatory distribution of organs. It would be important to ensure that the criteria cannot exclude patients based on the following: race, ethnicity, religion, national origin, gender, or sexual orientation. However, centers could consider candidacy based on the ability to pay, age, ability to adhere to an immunosuppressive regimen, and clinical status. The goals of the transplant evaluation (as discussed in Chapter 6 ) include (1) evaluating the indications for transplant and the survival benefit offered by transplantation, (2) identifying and mitigating other risk factors that may affect outcomes after transplant, and (3) determining the specific psychosocial needs of the patient that may affect survival after transplantation.

Determining the survival benefit offered by transplantation, especially with respect to pediatric LT, poses a challenge to providers. Data are often insufficient to make accurate predictions about survival benefit, and in certain indications (e.g., urea cycle defects), the survival benefit is less clear than the anticipated improvement in quality of life. Furthermore, there is wide variation in what would be considered a meaningful survival benefit. Finally, with the rapid generation of new knowledge, survival benefit is likely to increase over time.

Comorbid conditions that could affect survival may preclude being listed for transplant. Generally, uncorrectable multisystem failure, malignancy not correctable by transplant, active infection, and advanced/uncorrectable central nervous system disease are contraindications to LT. Psychosocial variables may legitimately impact the outcome. However, care needs to be taken to ensure psychosocial considerations are based on actual past behavior and not anxiety or fear of future behavior. Candidates and their families should also be given an opportunity to correct their behavior. Candidate selection needs to have two distinct but important components. The first includes evaluating the candidate using the discussed framework while also minimizing the potential for bias. The second component entails the team decision-making process. This requires a strong interdisciplinary team where members can have candid discussions. Again, care is needed to ensure that bias is not introduced into the decision-making.

Waitlist

Scarcity of organs is a worldwide problem. Data from the World Health Organization (WHO) Global Observatory on Organ Donation estimate that only 10% of patients needing an organ receive one annually. In the United States, only about 60% of waitlisted pediatric patients receive liver transplants within 1 year of being listed on the waitlist. In the United States, waitlist mortality for children awaiting LT remains between 5% and 10%. Across 14 countries, the waitlist mortality for children younger than 2 years of age ranged from 0% and 20% . Once a patient is placed on the waitlist, patients and providers must wait for a suitable organ to be made available.

In the United States, organ distribution is regulated by the federal government. The federal government gives sole authority to UNOS to distribute organs in accordance with the Final Rule. The Final Rule was enacted in 2000 and is the basis for the current deceased donor organ allocation policy. The Final Rule (see Fig. 3.2 ) dictates that (1) allocation be based on sound medical judgment; (2) allocation is done in a manner that minimizes wasted organs, avoids futile transplants, promotes patient access to transplant, and promotes the efficient management of organ placement; (3) allocation is done without respect to a patient’s residence or place of listing; and (4) policies are reviewed periodically. Broadly, UNOS will first try to identify an appropriately matched recipient within the local area. If no suitable recipient is found, the organization will then broaden to the surrounding region before expanding to the national registry. Pediatric donors are offered preferentially to pediatric recipients.

In the United States, patients are offered organs based on their Mayo end-stage liver disease (MELD) (for patients ≥ 12 years of age) or pediatric end-stage liver disease (PELD) (for patients < 12 years of age) score. These scores are intended to predict waitlist mortality, and thus organs are theoretically offered to the patients with the highest mortality risk. The MELD and PELD scores are objective measures; however, there are standard and nonstandard exceptions for which a program can petition. Standard exceptions include tumors or metabolic diseases where mortality is high yet not reflected by liver biochemistries. Nonstandard exceptions are assigned when transplant programs appeal to regional review boards who review and approve additional MELD/PELD points on a case-by-case basis. In the United States, 86% of all pediatric nonstandard exception requests are approved. Given the high rate of approval, it may be incumbent on a transplant program to aggressively request exception points to increase the availability of organs for their patients. In addition to mortality risk, pediatric patients awaiting transplant encounter additional risks from remaining on the waitlist, including neurocognitive development and growth failure. Therefore additional work is needed to ensure that organ allocation across adult and pediatric patients reflects not only the true mortality risk to pediatric patients but also the long-term morbidity facing children living with end-stage liver disease.

Once an organ is made available to a patient, the transplant surgeon or hepatologist has to decide whether to accept the organ. This decision is complex and includes deciding between a living donor versus deceased donor organ. The decision to accept a deceased donor organ is impacted by the timing of the organ offer, the expertise of the transplant center, quality of the donor organs, and an understanding of the availability of more favorable organs. In a 2017 study of the UNOS database, of pediatric patients who died on the waitlist, 55% had received one or more offers for an organ that was subsequently transplanted into another child. Of organs refused for size mismatch, 50% of the organs offered were within the size range based on body surface area ratios. In a subsequent article, Mitchell et al. found higher waitlist mortality for patients who were at institutions with lower organ acceptance rates. They also found that graft survival was not superior at these institutions, suggesting that their more stringent donor selection criteria were not impacting graft survival. Finally, the study found that organs that were rejected more than four times were likely to continue to be rejected even after controlling for other available donor characteristics. These findings indicate that there is much to be learned in pediatric organ acceptance strategies and that institutions should have a policy in place regarding the review of organ acceptance to minimize bias. Transplant programs need to think critically about the availability and quality of deceased donor organs, the technical expertise of the surgeons, and the availability of living donors to properly determine the timing of organ acceptance.

Given the scarcity of organs and the specific size constraints that children have, efforts to expand the donor pool should be identified. Split liver transplant and living related donation are two strategies that increase organ availability for pediatric patients. Split liver transplant involves dividing the liver into a right extended segment graft and a left lateral segment graft. By splitting the liver, the pool of available organs is increased for pediatric patients who have size constraints on the type of organ they can receive. Worldwide adoption of this technique is variable, with European countries having the highest rates of utilization. The technical expertise necessary to perform split liver transplantation may be a barrier to widespread adoption. Split liver transplantation has a high rate of biliary complications, and performing split liver transplantation requires a detailed understanding of the segmental surgical anatomy of the liver.