Advanced Therapies

Lee R. Goldberg, MD, MPHMedical Director, Heart Failure and Cardiac

Transplant ProgramUniversity of Pennsylvania

What Are Advanced Therapies?• Heart Transplant• Ventricular Assisted Devices (VAD)– Mechanical Circulatory Support (MCS)

The Paradox

Sick enough toneed transplant or VAD without any other threatment options

Well enough to survive VAD and/or transplant and have good quality of life

Desperately sick and otherwise healthy at the same time!

For Transplant, limited organ availability creates a societal responsibility to carefully select recipients most likely to survive and have a good quality of life

The Evaluation – Three Components

• The Heart– There are no other therapies that will improve the status of

the heart, survival or quality of life• The rest of you

– All the organs except the heart are healthy and can survive the surgery and the effects of either the VAD or the medications after transplant

• Everything outside of you– “Family” support– Psychosocial – Transportation– Finances, insurance, disability, prescription coverage

Timing• Move forward with VAD or Transplant before

irreversible organ damage, malnutrition, muscle weakness or infection

• Avoid going “too soon” – “Up front risk” – can shorten life– New therapies always being developed and improved

• Can use VAD as bridge to heart transplant– Correct heart failure – Better candidate when it comes time to transplant

Transplant Wait List• The current UNOS system divides the waiting list by “risk

of death”– Status 1A – On IV medications at high dose with a monitoring

(PA) catheter, VAD complication, 30 days following VAD– Status 1B – On IV medication or VAD– Status 2 – Not on IV medications or VAD– Status 7 – Inactive

• Body Size• Blood Group• Antibodies (PRA)

Heart Transplant• Rejection versus infection• Immunosuppression

– Calcineurin inhibitor (Tacrolimus, Cyclosporine)– Anti-proliferative (Mycophenolate mofetil, azathiprine– Steroid (Prednisone)

• Immunosupporession issues– Must take for life– Increases risk of infection– Increases risk for cancers– Toxicity to kidneys– Other side effects – tremor, GI, weight gain, diabetes

• Frequent visits– Weekly, biweekly, monthly in first year– Heart biopsies, blood draws, frequent adjustment of medications

Ventricular Assist Device• Need to take anticoagulation (blood thinners) for life– Risk of clot in VAD– Risk of bleeding especially in GI tract– Risk of stroke – clots and bleeding

• Infection– Drive line infections

• Blood pressure control– Challenging – No pulse!

• Tethered to batteries– Can impact travel – need to plan ahead

Palliative Care• For people who are not candidates or who do

not want advanced therapies– Focus on quality of life and symptom control– Define wishes for heroic therapies – CPR, Shocks

from ICD device, admission to hospital• For people who have had advanced therapies– Define goals of care – When to deactivate VAD

Conclusion• Advanced therapies include ventricular assist devices

and heart transplants• You need to be “sick” and “healthy” at the same

time to really benefit from these therapies• A comprehensive evaluation is necessary to

determine who is a good candidate• Each therapy has advantages and disadvantages• Communicating your wishes and goals to your family

and clinical team is critical