J. Pena

Liver Transplantation for Hereditary Transthyretin Amyloidosis: After 20 Years Still the Best Therapeutic Alternative?

Background Until recently, liver transplantation (Ltx) was the only available treatment for hereditary transthyretin (TTR) amyloidosis; today, however, several pharmacotherapies are tested. Herein, we present survival data from the largest available database on transplanted hereditary TTR patients to serve as a base for comparison. Methods Liver transplantation was evaluated in a 20-year retrospective analysis of the Familial Amyloidosis Polyneuropathy World Transplant Registry. Results From April 1990 until December 2010, data were accumulated from 77 liver transplant centers. The Registry contains 1940 patients, and 1379 are alive. Eighty-eight Ltx were performed in combination with a heart and/or kidney transplantation. Overall, 20-year survival after Ltx was 55.3%. Multivariate analysis revealed modified body mass index, early onset of disease (<50 years of age), disease duration before Ltx, and TTR Val30Met versus non-TTR Val30Met mutations as independent significant survival factors. Early-onset patients had an expected mortality rate of 38% that of the late-onset group (P < 0.001). Furthermore, Val30Met patients had an expected mortality rate of 61% that of non-TTR Val30Met patients (P < 0.001). With each year of duration of disease before Ltx, expected mortality increased by 11% (P < 0.001). With each 100-unit increase in modified body mass index at Ltx, the expected mortality decreased to 89% of the expected mortality (P < 0.001). Cardiovascular death was markedly more common than that observed in patients undergoing Ltx for end-stage liver disease. Conclusions Long-term survival after Ltx, especially for early-onset TTR Val30Met patients, is excellent. The risk of delaying Ltx by testing alternative treatments, especially in early-onset TTR Val30Met patients, requires consideration.

Presumed Consent and Cadaver Organ Donation: Is There a Place for Family Involvement?

In 1976, Portugal adopted presumed consent legislation concerning cadaver organ donation for transplantation. This law took its inspiration from several attempts to introduce a similar contracting-out system in Britain, and from the proceedings of the joint committee of experts of the Council of Europe which led to the Resolution (78) adopted by the Committee of Ministers on the 11 May 1978.

Immunological Criteria in Kidney Allocation

In Portugal, shortage of organs is the factor limiting the development of kidney transplantation. As the annual number of new hemodialysis patients acceptable to transplant largely exceeds the annual number of grafted patients [1], waiting lists tend to grow. Kidney allocation is done primarily according to immunological criteria: ABO blood group compatibility and number of DR, B, and A locus compatibilities in this order. Clinical classification, second grafts, age and duration of hemodialysis come next. Patient priority is defined through clinical classification as Super urgent (SU; only ABO blood group compatibility and negative crossmatch required); U1, U2, and U3 (1, 2, or 3 human leukocyte antigen, HLA locus compatibilities required); IR (second graft patients), and H (hyperimmunized patients with more than 80% preformed antibodies to a population panel). Using these allocation criteria, O group and incomplete HLA locus identified patients should be prejudiced, as O kidneys are diverted to other blood group patients, and incomplete HLA-typed patients have less chances to be grafted. In countries with large waiting time to transplant, differences between these groups may became clinically significant, and as a result, we analyzed waiting time to transplant according to the allocation criteria, at the Red Cross Hospital Transplant Unit.