BACKGROUND: Beta thalassemia major is a severe inherited form of hemolytic anemia that results from ineffective erythropoiesis. Allogenic hematopoietic stem cell transplantation (HSCT) remains the only potentially curative therapy. Unfortunately, the subgroup of adult thalassemia patients with hepatomegaly, portal fibrosis and a history of irregular iron chelation have an elevated risk for transplantation-related mortality that is currently estimated to be about 29 percent. DISCUSSION: Thalassemia patients may be faced with a difficult choice: they can either continue conventional transfusion and iron chelation therapy or accept the high mortality risk of HSCT in the hope of obtaining complete recovery.Throughout the decision making process, every effort should be made to sustain and enhance autonomous choice. The concept of conscious consent becomes particularly important. The patient must be made fully aware of the favourable and adverse outcomes of HSCT. Although it is the physician's duty to illustrate the possibility of completely restoring health, considerable emphasis should be put on the adverse effects of the procedure. The physician also needs to decide whether the patient is eligible for HSCT according to the "rule of descending order". The patient must be given full details on self-care and fundamental lifestyle changes and be fully aware that he/she will be partly responsible for the outcome. SUMMARY: Only if all the aforesaid conditions are satisfied can it be considered reasonable to propose unrelated HSCT as a potential cure for high risk thalassemia patients.
BACKGROUND:Beta thalassemia major is a severe inherited form of hemolytic anemia that results from ineffective erythropoiesis. Allogenic hematopoietic stem cell transplantation (HSCT) remains the only potentially curative therapy. Unfortunately, the subgroup of adult thalassemiapatients with hepatomegaly, portal fibrosis and a history of irregular iron chelation have an elevated risk for transplantation-related mortality that is currently estimated to be about 29 percent. DISCUSSION: Thalassemiapatients may be faced with a difficult choice: they can either continue conventional transfusion and iron chelation therapy or accept the high mortality risk of HSCT in the hope of obtaining complete recovery.Throughout the decision making process, every effort should be made to sustain and enhance autonomous choice. The concept of conscious consent becomes particularly important. The patient must be made fully aware of the favourable and adverse outcomes of HSCT. Although it is the physician's duty to illustrate the possibility of completely restoring health, considerable emphasis should be put on the adverse effects of the procedure. The physician also needs to decide whether the patient is eligible for HSCT according to the "rule of descending order". The patient must be given full details on self-care and fundamental lifestyle changes and be fully aware that he/she will be partly responsible for the outcome. SUMMARY: Only if all the aforesaid conditions are satisfied can it be considered reasonable to propose unrelated HSCT as a potential cure for high risk thalassemiapatients.
Authors: G Lucarelli; M Galimberti; P Polchi; E Angelucci; D Baronciani; C Giardini; M Andreani; F Agostinelli; F Albertini; R A Clift Journal: N Engl J Med Date: 1993-09-16 Impact factor: 91.245
Authors: Salvatore Pisu; Giovanni Caocci; Ernesto d'Aloja; Fabio Efficace; Adriana Vacca; Eugenia Piras; Maria Grazia Orofino; Carmen Addari; Michela Pintor; Roberto Demontis; Federica Demuru; Maria Rita Pittau; Gary S Collins; Giorgio La Nasa Journal: Philos Ethics Humanit Med Date: 2014-08-12 Impact factor: 2.464
Authors: Andrea Z Pereira; Ricardo Hellman; Nelson Hamerschlak; Andrea Kondo; Polianna Mara Rodrigues de Souza; Wilson Leite Pedreira; Luiz Fernando Alves Lima Mantovani; Eduardo Juan Troster; Henrique Grunspun; Marco Aurélio Scarpinella Bueno Journal: Case Rep Hematol Date: 2017-02-23