Long-Term Outcomes in Heart Transplanted Patients with Anderson-Fabry Disease: Effectiveness of a Combined Medical and Surgical Therapy.

ABSTRACT

BACKGROUND: Transplantation in the management of end-stage organ failure in patients with Anderson- Fabry disease is still a matter of debate due to a lack of data regarding long-term outcomes and prognosis. OBSERVATIONS Most of the cases in the literature did not receive enzyme-replacement therapy (ERT), or received it only years after transplantation. Based on our experience, we describe here the long-term results of heart transplant in combination with early ERT. CONCLUSIONS: Transplantation combined with lifelong ERT should be considered to offer patients a chance of good long-term outcomes and quality of life.