Allogeneic Hematopoietic Stem Cell Transplantation for Paroxysmal Nocturnal Hemoglobinuria / 中国实验血液学杂志

OBJECTIVE@#To analyze the clinical efficacy and safety of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for paroxysmal nocturnal hemoglobinuria (PNH), and preliminarily explore the role of an improved post-transplantation cyclophosphamide (PTCy) based conditioning regimen in PNH patients receiving transplantation.@*METHODS@#Clinical related data of PNH sufferers receiving allo-HSCT in Department of Hematology, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology were collected, and hematopoietic reconstitution, chimerism, PNH cloning, graft-versus-host disease (GVHD), infection, and survival were analyzed.@*RESULTS@#Totally five PNH patients receiving allo-HSCT were enrolled, including 1 case with classic PNH, 3 cases with aplastic anemia-PNH syndrome, 1 case with myelodysplastic syndrome, three of them (case 1-3) received the improved PTCy based conditioning regimen before HSCT. All sufferers engrafted successfully within 28 days, the median time of neutrophil and platelet engraftment was 11 days and 12 days, respectively, no patient occurred acute or chronic GVHD, after a median follow-up of 16 months, all recipients survived and completely eliminated PNH cloning.@*CONCLUSION@#Allo-HSCT can completely clear PNH cloning and restore hematopoietic function with controllable complications, and the improved PTCy based conditioning regimen is proved to be effective in PNH transplantation.

The correlation between early lymphocyte count post-allogeneic hematopoietic stem cell transplantation and prognosis in leukemia patients / 中华血液学杂志

<p><b>OBJECTIVE</b>To analyze the correlation between early lymphocyte count (lymphocyte count on day 30, LC30) post-allogeneic hematopoietic stem cell transplantation (allo-HSCT) and transplant prognosis in leukemia patients.</p><p><b>METHODS</b>The data from 124 consecutive patients undergoing allo-HSCT for leukemia from January 2003 to April 2011 was analyzed retrospectively. LC30 post-allo-HSCT correlated with 5-year overall survival (OS), 5-year relapse rate (RR), 5-year nonrelapse mortality (NRM), accumulative rate of acute graft versus host disease (aGVHD) and chronic graft versus host disease (cGVHD) was studied.</p><p><b>RESULTS</b>Univariate analysis indicated that patients with LC30 ≥ 0.40×10(9)/L had higher 5-year OS than those with LC30 < 0.40×10(9)/L \[(62.2 ± 5.8)% vs (37.0 ± 8.6)%, P = 0.003\], lower 5-year RR\[(13.9 ± 4.7)% vs (32.0 ± 8.4)%, P = 0.027\], lower 5-year NRM \[(31.3 ± 5.8)% vs (45.0 ± 9.3)%, P = 0.048)\], and higher cGVHD cumulative incidence \[(82.9 ± 4.6)% vs (62.7 ± 11.1)%, P = 0.042)\]. Multivariate analysis also suggested that LC30 was associated with 5-year OS, 5-year RR, 5-year NRM, and cGVHD cumulative incidence. At the same time disease risk stratification was associated with prognosis.</p><p><b>CONCLUSIONS</b>Early lymphocyte count (LC30) post-allogeneic hematopoietic stem cell transplantation in leukemia is highly associated with prognosis, which can be the independent prognosis index after allo-HSCT in leukemia and can identify a group of patients who might be suitable candidates for early interventions treatment.</p>

Establishment of a stable transfectant 32D/Gfi1 cell line by recombinant lentiviral expression vector / 中国实验血液学杂志

The aim of study was to establish the packaging system of the recombinant lentiviral vector encoding Gfi1 gene for eukaryotic expression and to realize the efficient, stable expression of Gfi1 32D cells so as to provide effective platform for further studying the development of Gfi1 gene in hematologic malignancies. The three-plasmid recombinant lentiviral vector consisting of transfer plasmid (pLOX-Gfi1/pLOX), the packaging plasmid (pCMVDeltaR8.2) and the envelop plasmid (pMD.G) was prepared and purified. Human embryonic kidney 293T cells were cotransfected with the three plasmids by lipofectamine 2000. After transfection for 48 hours, the viral supernatant was collected and the target cell 32D was transfected with the recombinant lentivirus; the Gfi1 integration and expression in 293T and 32D cells were detected by Western-blot. The results showed that the three plasmids of lentivirus could be transfected into 293T with high efficiency and packaged successfully, and the Gfi1 protein could be detected by fluorescent microscopy. The recombinant lentiviruses carrying Gfi1 could transfer and deliver Gfi1 gene to 32D cells, and the Gfi1 expression in 293T and 32D cell could be detected by Western blot. It is concluded that the recombinant lentivirus carrying Gfi1 can deliver target gene to 32D cells with high efficiency, and the expression of Gfi1 protein is stable in 32D.