Organ Allocation in Pennsylvania: Current concepts and future directions

Transcription

1 Organ Allocation in Pennsylvania: Current concepts and future directions David Goldberg, MD, MSCE Assistant Professor of Medicine and Epidemiology Medical Director of Living Donor Liver Transplantation Senior Fellow, Leonard Davis Institute of Health Economics May 19, Disclosures Funded by an NIH K08 Career Development Award focused on evaluating population-level outcomes in end-stage liver disease. Consulting: Merck Additional research support: Merck (investigator-initiated grant), Intercept (research support) 2 Page 1 1

2 Outline Define concepts of organ allocation and possible methods of waitlist prioritization Review current system of liver allocation Review geographic distribution of livers in the US Discuss Share 35 policy and impact on prioritization Discuss access to livers for HCV-positive patients Review current proposal for liver distribution and how it could affect your patients in Pennsylvania 3 Allocation versus distribution in transplant Allocation: Algorithm for prioritizing patients (process by which patients ranked) MELD score (liver) LAS score (lung) Status 1, 2, 3 (heart) Distribution: Where the organs end up (i.e., geographic unit) UNOS regions and donors service areas 4 Page 2 2

3 Principles of potential allocation schemes Equity: First-come, first-served Example: Kidney waiting time system with some utility and benefit Limitation: Pure equity doesn t account for efficient use of scarce resource Urgency: Sickest-first (liver) Example: Liver MELD system Limitations: Sickest don t always benefit the most Need to correctly define sickest Utility: Expected post-transplant outcomes Example: Prioritization of pediatric patients and EPTS Limitation: Organs survive longest but not necessarily those in greatest need (i.e., may favor youngest) Transplant benefit: Difference between expected survival with transplant and mortality without Minimize mortality for population as whole Example: Lung 5 Urgency-based MELD system Use MELD score to prioritize patients Meant to give highest priority to sickest patients MELD score well validated for patients without renal disease Implemented system of exception points Standardized exceptions: Follow specific criteria HCC: Prolonged waiting time may lead to being ineligible for LT Concerns Limited evidence-base to base exact points May change patient care (i.e., wait and watch 1.5cm HCC) Non-standardized: Case-by-case basis Reviewed by regional review boards without standardized criteria Concerns: RRBs vary in awarding exception points Regions can develop own criteria 6 Page 3 3

4 Timeline of allocation of livers in US Pre-2002: CTP score + waiting time + ICU status Subjective nature of ICU and CTP score Benefited those with access to early listing 2002: Introduction of MELD-based allocation Objective measure of severity of illness MELD score predictor of 3-month mortality 2005: Share 15 Local patients with MELD<15 not offered liver until all patients with MELD 15 offered liver 2015: Share 35 Higher priority for patients within each region with MELD score 35 7 HCC exception point system Diagnosed with HCC within Milan 1 tumor 5cm or 2 or 3 tumors all 3cm Waitlisted No exception points on listing Wait 6 months with q3 month imaging 28 points 29 points 31, 32, 33, 34 points capped at 34 8 Page 4 4

5 Non-standardized exception points Current system Regional review boards review case-by-case basis Examples: PSC and cholangitis, refractory ascites Variability in: Applications for exceptions Approval of exceptions Points awarded Future policy change National Liver Review Board National board to accept Points based on MELD at transplant More evidence-base to guide acceptance of exception applications 9 MELD of 15 magic number for allocation Data from 2005 demonstrated patients with MELD <15 on average don t derive survival benefit 1 Survival benefit only measured over 1 year Benefit exists at longer follow-up (i.e., 5 years) Didn t consider patients sicker than their MELD Cachectic, ascites, muscle wasting Share 15 changed priority for patients with a MELD <15 Only offered to local patients with MELD<15 after all national patients (centers) decline organ offer Rare to get transplanted or get offer when MELD<15 Benefit of waiting patient with MELD<15 Some patients may benefit for a transplant Probability of transplant low 1-Merion et al, Liver Transplantation Page 5 5

6 Current units of distribution for livers 11 UNOS Regions 58 Donor Service Areas 11 Manner in which organs are distributed Local priority first Historically local priority matching all donors and recipients Regional priority next level Previously only after local offers declined Local and regional now for patients with MELD 35 Exceptions to local priority Status 1a (acute liver failure, hepatic artery thrombosis) Status 1b (pediatric) 12 Page 6 6

7 What is Share 35 Implemented in June 2013 Goal was to implement broader sharing of organs to the sickest' patients as defined by MELD score Highest waitlist mortality for patients with highest MELD scores Sought to minimize excess travel unless transplant for sicker patient Policy: Regional sharing of livers to MELD/PELD 35+ patients before local patients with MELD<35 13 Key points of current allocation and distribution Allocation Highest priority to Status 1a (acute liver failure) MELD/PELD: MELD/PELD: MELD<15 only offered organs if everyone else in country with MELD 15 declines the organ offer Exception points HCC exceptions capped at 34 6-month waiting period Exceptions still not standardized but NLRB soon to be implemented Distribution Local first (within DSA) Regional distribution to sickest patients 14 Page 7 7

8 Example of match run for liver 50 year-old brain dead donor in Philadelphia, blood group O Rank Center MELD 1 Pittsburgh 38 2 Johns Hopkins 37 3 Penn 36 4 Jefferson 35 5 Einstein 34* 6 Einstein 31* 7 Temple 30 8 Penn 29* * Signifies exception points for HCC 15 Drug overdose as a cause of death of deceased donors 1400 According to OPTN/UNOS data as of March 15, Page 8 8

9 Deceased donors with a drug overdose in our region Not all opioid overdoses are heroin PHS-IR criteria for drug use: People who have injected drugs by IV, IM, or subq route for nonmedical reasons in preceding 12 months 1-Compton W, et al. NEJM 2016; 374: Page 9 9

10 Geographic differences in HCV status of overdose deceased donors 35.0% 30.0% 25.0% 20.0% 15.0% 10.0% 5.0% 0.0% OPTN/UNOS Region 19 HCV-positive deceased donors in Region Page 10 10

11 What is the benefit of remaining HCV-positive Median MELD at transplant in HCV- donor HCV+ donor PADV PATF Region 2 United States 21 Treating HCV in patients with decompensated cirrhosis Limited trial data in MELD 20 Trial data in decompensated cirrhosis Subset will have decrease in MELD/CTP score 30% will have stable or worsening MELD score Even if MELD improves may remain decompensated HCV cure data similar if treatment pre- vs post-transplant My approach If transplant definite option (i.e., ascites on diuretics, HE)->don t treat If may improve (intermediate MELD, minimal to no decomps)- >consider treatment HCC: depends on region Key point: Refer to transplant center for discussion of treatment options before treating HCV locally in patient with decompensated cirrhosis or MELD Page 11 11

12 Liver Distribution Proposal In 2016, across the current OPTN/UNOS regions in 2016, the median MELD at transplant by DSA ranged from 20 to 40, which equates to an estimated risk of 3-month mortality without a liver transplant of 11% to nearly 100%. Goal: Normalize median match MELD at transplant across the US Local lab MELD, Share 29 Local: Patients at transplant center within 150 miles of donor hospital receive 5 priority points (added to lab MELD) Share 29 Share within region miles of donor hospital Share at lab MELD 29 (or ) 23 Philadelphia, PA Courtesy of Rick Hasz, GLDP 24 Page 12 12

13 New York City, NY Courtesy of Rick Hasz, GLDP 25 Pittsburgh, PA Courtesy of Rick Hasz, GLDP 26 Page 13 13

14 Concerns about liver distribution proposal Focus on allocation MELD, not lab MELD Based on disparities pre-nlrb Doesn t account for waitlist mortality Uses allocation MELD to equate to waitlist mortality 90-day waitlist mortality for patients with a MELD score of Region 1: 8.6% Region 2: 8.6% Region 9: 6.6% Does not account for variability in donation rates and OPO performance 27 Number of deceased donors per year 600 PADV, population 11.5 million PATF, population 5.5 million NYRT, population 13.5 million Page 14 14

15 Impact of proposal on transplant in Pennsylvania 350 Current Proposed NYRT PADV PATF 29 How to make your voice heard 30 Page 15 15

16 Conclusions Liver follows a sickest-first prioritization Imperfect way to measure who is sickest Patients getting progressively sicker in order to be transplanted Continued modifications to allocation hopefully will continue to prioritize the sickest patients Changing donor demographics favor keeping patients positive for HCV if transplant is likely in the horizon Proposals for broader organ sharing will increase costs and logistics for Pennsylvania residents, and would lead to fewer transplants in Eastern Pennsylvania Page 16 16