| Autor: |
Lu Y; Department of Bone Marrow Transplantation, Hebei Yanda Lu Daopei Hospital, Langfang, China., Zhao YL; Department of Bone Marrow Transplantation, Hebei Yanda Lu Daopei Hospital, Langfang, China., Xiong M; Department of Bone Marrow Transplantation, Hebei Yanda Lu Daopei Hospital, Langfang, China., Sun RJ; Department of Bone Marrow Transplantation, Hebei Yanda Lu Daopei Hospital, Langfang, China., Cao XY; Department of Bone Marrow Transplantation, Hebei Yanda Lu Daopei Hospital, Langfang, China., Wei ZJ; Department of Bone Marrow Transplantation, Hebei Yanda Lu Daopei Hospital, Langfang, China., Lu DP; Department of Bone Marrow Transplantation, Hebei Yanda Lu Daopei Hospital, Langfang, China. |
| Abstrakt: |
We analyzed the outcomes of 32 patients with paroxysmal nocturnal hemoglobinuria (PNH) who underwent either a haploidentical donor (HID) or a matched unrelated donor (MUD) hematopoietic stem cell transplantation (HSCT). Seventeen patients received an HSCT from an HID and 15 patients received an HSCT from an MUD. The median follow-up time of the surviving patients was 36 months (range: 12-96 months). No significant differences were observed in the 3-year overall survival (OS) between the HID and MUD cohorts (74.1%±11.4% vs. 93.3%±6.4%, respectively, p =.222) or in the 3-year failure-free survival (68.8%±11.8% vs. 86.7%±8.8%, respectively, p =.307). Treatment-related mortality occurred in five patients. A univariate analysis of risk factors revealed platelet engraftment failure negatively impacted OS and FFS. We conclude that HID and MUD-HSCT are feasible and can be effective options for those PNH patients with concomitant bone marrow failure, recurrent life-threatening thrombosis, and uncontrollable hemolysis. |
