7:00 a.m.

Registration and Continental Breakfast—Exhibits Open

8:00 a.m.

Welcome and Opening Remarks

An Update on Liver Transplantation: Status of Live Donor Transplants for Adults, Michael Abecassis, MD, MBA
With nearly 17,000 patients currently awaiting liver transplant in the United States, a number of strategies have been developed to address this organ shortage. Living donor liver transplantation (LDLT) is among the strategies most likely to impact the need for donor organs. Dr. Abecassis will discuss the role living donors play in the management of liver disease.

Objectives:

• Discuss the role of living donor compared with deceased donor liver transplantation for the management of acute and chronic liver disease.
• Summarize the changing indications for living donor liver transplantation.

Portal Vein Thrombosis Before and After Transplantation: Surgical Alternatives, Rashmi Kabre, MD
Management of portal vein thrombosis after transplantation can be challenging in small children, and posttransplant portal venous problems may lead to serious portal hypertension. The incidence of portal vein narrowing and occlusion is best treated proactively so that retransplantation or other methods of shunting might be avoided. Meso-Rex bypass is a method of restoring blood flow to the portal vein in the liver after the vein has thrombosed posttransplant. During this session, Dr. Kabre will provide an explanation of Meso-Rex bypass and describe the options for patients who have congenital or acquired portal venous obstruction and are referred for portal vein reconstruction without transplantation.

Objectives:

• Discuss the management of portal vein problems after transplantation and surgical options to avoid or treat thrombosis of the portal vein.
• Describe options that will benefit the child with mesenteric vein thrombosis, without the need for transplantation.

9:45 a.m.

Break—Exhibits Open

Pediatric Acute Liver Failure (ALF), Estella Alonso, MD
ALF is a severe, life-threatening condition that is defined as liver injury that results in impaired liver synthetic function and hepatic encephalopathy occurring within six weeks of the onset of illness. The causes of ALF in children are varied and include infectious illness, drug-induced injury, metabolic diseases, and autoimmune processes. However, in approximately half of affected children, the cause of liver injury cannot be determined. Treatment includes supportive care and focused therapies if a diagnosis amenable to medical therapy is identified. Survival with medical therapy is the exception rather than the rule, and many patients require liver transplantation. Assessment of which patients do require transplantation to recover is guided primarily by clinical judgment, as predictive models based on clinical data are not accurate enough to individualize transplant decisions and biomarkers that signal spontaneous recovery are not available. The presentation will include a discussion of the early evaluation and management of children with ALF and a review of predictive models of outcomes including how these models can guide patient care. We will also detail typical outcomes following recovery from ALF and future directions in research to advance the care of these patients.

Objectives:

• Identify the causes of ALF in pediatrics and describe findings from the Pediatric Acute Liver Failure (PALF) Research Consortia.
• Discuss prognosis and management strategies in ALF, including the role of liver transplantation.

Infections in Transplantation: Has the Focus Shifted From Immunosuppression to Transmission? Michael Ison, MD, MS
Organ transplantation extends the life of more than 25,000 patients with end-stage organ failure each year in the United States. Patient outcomes have improved with the advent of potent immunosuppressive agents, improvement in surgical techniques and expanded use of antimicrobial prophylaxis. Despite these advances, infections remain a leading cause of morbidity and mortality among transplant recipients. A rare complication of organ transplantation is the transmission of infections and malignancies from the donor to the recipient. Existing data suggests that this is rare, complicating only about 0.2 percent of all organ transplants. Reviewing the donor’s medical and social history, conducting a thorough physical examination of the donor, and testing the donor’s blood for evidence of infection may reduce the risk of donor-derived disease transmission.

Objectives:

• Summarize the types of infections that complicate solid organ transplantation.
• Describe the current epidemiology of donor-derived infectious disease transmission.
• Identify the various ways to mitigate against transmission of infections from donors to recipients.

11:30 a.m.

Intestinal Rehabilitation: The Latest and Greatest, Valeria Cohran, MD
The diagnosis of short bowel syndrome usually necessitates parenteral feeding. Prolonged parenteral feeding requires aggressive management and close monitoring of the caregiver’s central venous catheter care and parenteral nutrition. Dr. Cohran will provide an overview of the major surgical procedures that are used to “extend” the intestines and present the characteristics of a good intestinal rehabilitation program.

Objectives:

• State the common strategies used by intestinal rehabilitation programs to manage patients with intestinal failure.
• Describe intestinal lengthening procedures commonly employed for the management of intestinal failure.
• Discuss the role of lipid minimization and newer lipid preparations.

12:15 p.m.

1:30 p.m.

The Changing Face of Kidney and Pancreas Transplantation: What’s Hot, What’s New? Joseph Leventhal, MD, PhD
As the demand for kidney transplants continues to grow, today’s strategies to increase the number of transplants encompasses concepts that were not found acceptable years ago. Strategies like altruistic donation, paired donation, pay-it-forward, and list exchanges are some of the concepts that are just beginning to contribute to decreasing the shortage. In addition to discussing novel concepts to increase kidney transplantation, Dr. Leventhal will also discuss the use of cell therapy to induce tolerance, which is finally a reality, and how certain patients with Type II diabetes can now be considered for pancreas transplants.

Objectives:

• Describe the challenges in transplantation that could be addressed by either inducing tolerance or tissue engineering.
• Summarize the treatment options for kidney transplant patients who are highly sensitized.
• Discuss the criteria for pancreas transplant in patients with Type II diabetes.

2:15 p.m.

Health Maintenance and Anticipatory Care: The Pediatric Kidney Recipient, Amy Bobrowski, MD, MS
Dr. Bobrowski will review the screening process for pediatric post-kidney-transplant patients to include: cardiovascular disease, infectious disease, malignancy screening and prevention, and mental health (neurodevelopmental issues). Utilizing case studies and scientific data, Dr. Bobrowski will explain how long-term health and transplant success depend on a team effort and good communication among families, primary care providers and transplant professionals.

Objectives:

• Identify the major causes of morbidity and the barriers to long-term health maintenance in the pediatric kidney transplant recipient.
• Describe how the pediatric transplant team screen for and treat risk factors associated with poor long-term posttransplant outcomes.

3:00 p.m.

Improving Transplant Opportunities With Kidney Paired Donation (KPD), John Friedewald, MD
KPD has evolved as an option for patients in need of kidney transplantation who have an incompatible living donor. By exchanging donors, two or more pairs can be successfully transplanted with a compatible live donor. The inclusion of compatible pairs and nondirected donors can increase transplant opportunities and have a multiplier effect in the system. The use of desensitization therapies can complement KPD for highly sensitized recipients and greatly shorten their time on renal replacement therapy, as well as their time to transplant.

Objectives:

• Discuss the advantages of living donor kidney transplantation over dialysis and over deceased donor transplantation.
• Describe the basic premise of kidney paired donation.
• Summarize the role of desensitization in a KPD program.

3:45 p.m.

Break—Exhibits Open

4:00 p.m.

Pediatric Heart Transplantation: Triumphs and Challenges, Steven Kindel, MD

Objectives:

• Understand the history and progression of pediatric heart transplantation.
• State the indications, contraindications and outcomes for pediatric heart transplantation.
• Explain the listing criteria for pediatric heart transplantation.
• Identify posttransplant complications and treatment options.

4:45 p.m.

The Surgical Approach in the Management of Heart Failure, Edwin McGee, MD
Cardiac transplantation has long been the gold standard for the treatment of heart failure. The shortage of donor organs however, has led to the emergence of other surgical interventions in the management of heart failure. Dr. McGee will describe therapies for heart failure and discuss the status of heart transplant and mechanical support in the management of this complex condition.

Objectives:

• Summarize the indications for using a VAD.
• Discuss the potential advantages of using an LVAD pump.

5:30–6:30 p.m.

Facility Tour: Inpatient and Outpatient Transplant Centers at Northwestern and Lurie Children’s (optional)
Space is limited; registration is required. More information.

7:00 a.m.

Registration and Continental Breakfast—Exhibits Open

8:00 a.m.

Welcome and Opening Remarks

8:15 a.m.

Pediatric Stem Cell Transplant for Primary Immunodeficiency Diseases, William Tse, MD, PhD
Primary immunodeficiency is a group of inherited disorders associated with defects in immune defense and susceptibility to serious infections. In the past decade, the stem cell transplant team at Lurie Children’s Hospital of Chicago studied the use of a novel, reduced-intensity conditioning regimen to prepare primary immunodeficiency patients for stem cell transplant. We demonstrated that this approach is associated with minimal toxicity and is well tolerated by the patients. The procedure resulted in good donor cell engraftment, excellent immune reconstitution, and effective correction of the underlying defects. In this presentation, the speaker will review the use of reduced-intensity conditioning regimen for primary immunodeficiency.

Objectives:

• Explain the rationale for performing stem cell transplant for primary immunodeficiency diseases.
• Discuss the pros and cons of utilizing a reduced-intensity conditioning approach for primary immunodeficiency diseases.

9:00 a.m.

Autologous Hematopoietic Stem Cell Transplantation (HSCT) for Myeloma, Jayesh Mehta, MD
HSCT has become an essential component of the treatment for a variety of diseases, including myeloma. Advances in HSCT have led to considerable widening of the indication for transplant and markedly improved clinical results. Dr. Mehta will review the process of adult SCT, discuss advances in this treatment regimen and outline developments in the treatment of myeloma.

Objectives:

• Summarize the role of autologous HSCT in myeloma.
• Describe alternative options to autografting in myeloma.
• Discuss appropriate candidates for autotransplantation.

9:45 a.m.

Break—Exhibits Open

10:00 a.m.

Pediatric Brain Tumors: Updates in Treatment and Care, Rishi Lulla, MD, MS
The care of children with brain and spinal cord tumors continues to change with newer therapies leading to improved outcomes. We will review the common pediatric central nervous system (CNS) tumors, as well as discuss current and future approaches to treatment, including chemotherapy and radiation therapy. Newer biologically targeted approaches will be discussed, as will late effects of treatment for pediatric CNS tumors.

Objectives:

• Identify the common pediatric brain tumors.
• Discuss current treatment strategies for pediatric brain tumors.

10:45 a.m.

Coming of Age: Fertility Preservation in Pediatric, Adolescent and Young-Adult Patients, Barbara Lockart, RN, MSN, CPNP, CPON
Cancer treatment, including stem cell transplant, may put children and adolescents at risk for infertility. There is a growing awareness that maintaining fertility is important to families and that infertility is a concern for patients throughout the treatment continuum. Fertility preservation is a rapidly evolving field with lifelong implications for the physical, emotional and spiritual health of patients. The state of fertility preservation science will be examined, as well as guidelines for determining which patients are appropriate for intervention.

Objectives:

• Identify those patients at risk for infertility.
• Describe the available options for fertility preservation in male and female patients.

11:30 a.m.

Alternative Donors and Stem Cell Transplantation: The “End Around” Play on the Immune System, Reggie Duerst, MD
To bring the advantages of allogeneic hematopoietic stem cell transplantation (HSCT) to all, alternate sources of stem cells other than matched sibling donors are required. The current sources have included matched unrelated adult volunteers and stored donated umbilical cord blood units vs nonmatched family donors. Each of these sources is considered an “alternative” donor and requires specific adjustments in planning the HSCT procedure. Pros and cons of different alternative donor sources will be reviewed and discussed.

Objectives:

• Recognize who would be considered as a standard hematopoietic stem cell (HSC) donor.
• Explain who would be considered an alternative HSC donor and how he or she can be safely used as a source.

12:15 p.m.

Lunch—Exhibits Open

1:30 p.m.

Liver Cancer Update, Talia Baker, MD
Patients with hepatobiliary cancer are living longer due to the growing number of available treatment options. For hepatocellular carcinoma, those treatment options include liver transplantation. During this presentation, Dr. Baker will identify key diagnosis factors for hepatocellular carcinoma and discuss the use of Living Donor Liver Transplant (LDLT) as an emerging treatment option for these patients.

Objectives:

• Summarize the methods used for the diagnosis and management of hepatocellular carcinoma.
• Discuss what factors to consider when matching locoregional therapy and disease severity in the patient with liver cancer.

2:15 p.m.

Relevance of Traditional and Novel Prognostic Markers for Acute Myeloid Leukemia (AML) Therapies, Olga Frankfurt, MD

The therapeutic success and prognosis of patients with AML largely depend on a variety of patient- and disease-specific characteristics. In recent years, with the development of sophisticated techniques to analyze the AML genome, there has been a wealth of accumulating data describing novel prognostic markers in AML. Dr. Frankfurt will describe the traditional, novel, and tentative prognostic markers in AML as well as their relevance for standard and emerging therapeutic strategies.

Objectives:

• Summarize the latest data regarding existing and novel approaches for the therapy of acute leukemias.
• Discuss clinical practice issues of importance for effective care of patients with acute leukemia, such as timing of allogeneic stem cell transplantation, including umbilical cord blood transplantation, maintenance therapy, and targeted therapy.

3:00 p.m.

Break—Exhibits Open

3:15 p.m.

Acute Lymphoblastic Leukemia (ALL) in Children; Recent Findings, Elaine Morgan, MD
ALL is the most common cancer in children. The survival rate has significantly improved in the last 30 years and now most patients survive. The excellent survival rate raises other issues such as long-term complications. Management of patients who relapse poses a significant challenge. Various classes of new agents have been studied to improve the outcome in this patient population. The presentation will review the current issues and findings in pediatric ALL.

Objectives:

• Describe the current treatment of childhood ALL.
• Discuss the current knowledge of the biology of childhood ALL.

4:00 p.m.

Cellular Transplantation: Islets, Tregs and Tolerance—a New Frontier, Xunrong Luo, MD, PhD
Cell therapies are playing an increasing role in the treatment of many diseases. Islet cell transplantation for the Type 1 diabetic is now ready to be considered as a treatment option covered by commercial and other insurances. We have finally stepped through the door of transplant tolerance and established durable chimerism through the use of donor stem cells. The advances in these fields have led to a new understanding of the role of T regulatory cells in the induction of tolerance and what the future may become with this new knowledge. Dr. Luo will discuss the current state of islet cell transplantation, its future and the strategies being utilized to induce tolerance in the kidney transplant population.

Objectives:

• Discuss the current state-of-the-art for islet cell transplantation.
• Summarize the role of regulatory T cells in the induction of transplant tolerance.
• Describe available strategies for transplant tolerance induction.

Hematopoietic Stem Cell Transplantation (HSCT) for Autoimmune Diseases, Richard Burt, MD
HSCT is an increasingly used therapy for the treatment of autoimmune diseases. Dr. Burt’s presentation will discuss the difference between autologous and allogeneic stem cell transplantation and identify autoimmune diseases treated with stem cells.

Objectives:

• Describe the difference between HSCT for autoimmune disease and malignant disease.
• Discuss what autoimmune diseases are treated with stem cells.

5:30 p.m.

Adjourn