RESUMO
Objective@#To investigate the safety and efficacy of haploidentical hematopoietic stem cell transplantation with different intensity conditioning regimen in the treatment of childhood aplastic anemia (AA) .@*Methods@#Thirty-seven AA patients who underwent haploidentical transplantation in BaYi Children's Hospital Affiliated to PLA Army General Hospital from January 2013 to January 2017 were enrolled. According to the dosage of conditioning regimen, 34 patients excluding 3 other conditioning regimens were divided into high-dosage group (regimen 2, 22 cases) and low-dosage group (regimen 3, 12 cases). The data of Engraftment, graft-vs-host disease (GVHD), hematopoietic reconstitution, relapse, infection, overall survival (OS) were analyzed. The comparison between the two groups was tested by χ2 test.@*Results@#A total of 35 of 37 patients achieved primary engraftment; 2 cases died of regimen-related toxicity and severe infection before the infusing of the grafts. The activation rate of CMV and EBV was 60% (21/35) . Post-transplant lymphocyte disease (PTLD) of lung occurred in one case. The cumulative incidences of acute GVHD grade Ⅰ-Ⅳ and chronic GVHD were 29% (10/35) and 34% (12/35) respectively and the incidence of extensive chronic GVHD was 6% (2/35) . The median follow-up time was 18.8 (2.9-44.1) months, the OS was 92% (34/37) .All survived patients were no longer dependent on blood transfusion and none of them had recurrence. Comparing the rates of overall survival(86%(19/22) vs.100%(12/12)) and rates of chronic GVHD(40%(8/20) vs. 17%(2/12)) in regimen 2 and regimen 3 group, there were no significant difference (χ2=1.742, 1.841, all P>0.05) . Significant difference was found at the incidence of Ⅰ-Ⅳ acute GVHD (10% (2/20) vs. 50% (6/12) ,χ2=6.200, P=0.013).@*Conclusions@#Haploidentical hematopoietic stem cell transplantation is effective and safe. It is suitable for patients who are not eligible for matched donor transplantation. Application of reduced dose preconditioning in haploid transplantation is worth exploring.
RESUMO
Significant improvements in hematopoietic stem cell transplantation (HSCT) with haploidentical family donors (HFD) have confirmed its therapeutic role in severe aplastic anemia (SAA) and led to the evolution of treatment algorithms. However, the optimal conditioning regimen for HFD-HSCT remains undefined, especially the dosage of cyclophosphamide (Cy). A total of 77 patients with SAA from two research centers, who received HFD-HSCT with reduced-intensity fludarabine + cyclophosphamide + thymoglobulin ± busulfan conditioning regimen plus third-party cells infusion were included in this study, of which 67 pairs had 4-5 loci mismatched. We were particularly interested in whether the dosage of Cy significantly impacted graft failure (GF) and overall survival (OS). All patients showed sustained hematopoietic engraftment without any increase in severe aGVHD and transplantation-related mortality (TRM). The incidences of grade II-IV aGVHD, grade III-IV aGVHD and extensive cGVHD were 18%, 10% and 7%, respectively. The probabilities of 1-year and 5-year OS were 93.1% and 87.9%, respectively. Furthermore, patient age <15 years, MNC cells >8×108/kg and donor age <45 years were associated with better survival (P=0.043, P=0.023, and P=0.037, respectively) and engraftment (P=0.019, P=0.008, and P=0.001, respectively). Our findings indicated that SAA patients lack MSD benefited the most if HFD-HSCT was performed with reduced-intensity fludarabine-based conditioning regimen. Improved outcomes with HFD-HSCT may lead to a salvaged therapy and an expanded direct role for SAA in the future.
RESUMO
Haploidentical hematopoietic stem cell transplantation (Haplo-HSCT) is widely carried out in China, and transplantation related complications decreased gradually with the transplant technology improving, and the overall survival(OS) increased year by year. However, relapse after transplantation is still one of the main causes of death in patients with hematological malignancy. In order to reduce the recurrence after HSCT, we set a tumorablative conditioning regimen (TAC ) regimen; the aim is as much as possible to eliminate the malignant clone to reduce the recurrence without increasing the conditioning toxicity. We retrospectively analyzed 102 cases of haplo-HSCT in our hospital from 2012 to 2017. Ninety-eight out of the 99 (99.0%) patients achieved primary engraftment. The 2-year OS and disease free survival (DFS) are 81.4% (83/102) and 77.45% (79/102). The cumulative incidence of leukemia relapse is 16.2% (16/99), Twenty-nine patients developed II-IV acute graft-versus-host disease (aGVHD) (29%) within 100 days and only nine patients have grade III-IV aGVHD (9%) in measurable 99 patients. The conditioning regimen was relatively well tolerated with limited regimen-related toxicity. The preliminary results show that TAC is safe and effective in haplo-HSCT of children with hematologic malignancies. This study will provide a clinical basis for the individualized conditioning regimen.
RESUMO
Objective To evaluate the efficacy of haploidentical hematopoietic stem cell transplantation (HSCT) with supplemental umbilical cord blood (UCB) infusion in treatment of malignant hematological diseases.Method Clinical data of 66 patients with hematological malignancies treated with HSCT in our hospital between January 2010 and May 2013,were retrospectively analyzed.Among them 25 cases received infusion of human UCB before HSCT (experimental group) and other 41 cases had no UCB injection before HSCT (control group).Results There were no differences in age,gender,donor type,disease categories,disease status before transplant between two groups (P ＞ 0.05).There was a significant difference in conditioning regimes between two groups (P ＜ 0.05),but no clinical implication.The infused mononuclear cell (MNC) count in experimental group was higher than that in control group (9.94 ± 2.88 × 108/kg vs.7.80 ±0.82 × 108/kg,P =0.00),while there were no difference in infused CD34 + cell count (5.46 ±3.54 × 106/kg vs.3.54 ± 1.60 × 106/kg,P =0.16).Neutrophil recovery time in experimental group was shorter than that in control group (13.7 ±2.9 d vs.16.6 ±2.9 d,P =0.023).The incidences of grade Ⅲ-Ⅳ acute graft versus host disease (aGVHD,P =0.036),bacterial infection (P =0.001) and fungal infection (P =0.001)and hemorrhagic cystitis (P =0.00)in experimental group were lower than those in control group.There were no significant differences in platelet recovery time(P =0.43),the incidence of grade Ⅰ-Ⅱ aGVHD (P =0.27),implanted syndrome (P =0.24),sinusoidal obstruction syndrome (P =0.57)and viraemia (P =0.31)between two groups.Conclusion HSCT with supplemental infusion of human UCB may alleviate the degree of aGVHD,but the long-term outcome remains to be studied.
RESUMO
Objective To explore the efficiency and prognosis of hematological malignancies treated by haploidentical hematopoietic stem cell transplantation.Methods 70 patients who received haploidentical hematopoietic stem cell transplantation were analyzed retrospectively.According to tumor burden before transplantation,the patients were divided into three groups,the low tumor burden group,the mediate tumor burden group and the high tumor burden group.And then the effection of the tumor burden to survival was analyzed,and the engraftment,GVHD,infection,conditioning related toxicity,relapse and survival rate were also observed.Results The follow-up was terminated on January 1,2014.Follow-ups were performed for a median of 34.05 (7.4-83.6) months after transplantation.All patients achieved engraftments.The cumulative incidence of GVHD of grades 2-4 was 47.14 ％ (33/70) and that of grades 3-4 was 21.4 ％ (15/70).The chronic extensive GVHD was 20.0 ％ (14/70).The overall survival was 68.6 ％.Transplant-related mortality was 12.8 ％ and the relapse was 18.6 ％.The overall survivals in low tumor burden group,mediate tumor burden group,high tumor burden group were 91.67 ％,72.7 ％,33.3 ％ respectively.By SPSS 20.0,tumor burden was the high risk factor affecting the survival (low tumor group vs high tumor group,mediate tumor group vs high tumor group,low tumor group vs high tumor group,P =0.000,P =0.038,P =0.016).Conclusions Haploidentical hematopoietic stem cell transplantation in hematological malignancies is safe and effective.And for hematological malignancies with poor prognosis disease,it should be accepted the HSCT as soon as possible after remission in order to reduce the recurrence rate of malignancy.
RESUMO
Objective To deepen the understanding of chronic eosinophilic leukemia (CEL).Methods The course of diagnosis and treatment in a case of FIP1L1/PDGFRα fusion gene negative CEL was reported. Flow cytometry was used to analyze the immunophenotype of the cells in peripheral blood and pleural fluid. Karyotype was analyzed with G-banding. The expression of FIP1L1/PDGFRα fusion gene was detected by RT-PCR technique. Routine pathological examination of the tissues from bone marrow, lung and spleen were performed. Result A sixteen-year-old girl had severe anemia, fever, splenomegaly,thrombocytopenia and dominant hypereosinophilia lasting for 22 months. Trephine biopsy showed a hypercellular marrow with eosinophilic proliferation and moderate reticular fibrosis. Eosinophilic infiltration was found in lung and spleen and embolism was also found in spleen. She had a clonal chromosomal abnormality t(5;12)(q31;p13). The expression of FIP1L1/PDGFRα was negative. An abnormal clone of T cells expressing CD3-,CD4-,CD8- was found in peripheral blood and pleural fluid, in which the cional T cell accounted for 5.43% and 1.66% of the total lymphocytes respectively. The patient was refractory to treatment with hydroxyurea, prednisone and interferon alpha. She had poor response to a combination of therapy with low dose cytosine arabinoside, mitoxantrone, vincristine, cyclophosphamide, methotrexate and prednisone. She did not respond to imatinib and died of multiple organ failure. Conclusion The present case fulfilled the WHO diagnostic criteria of FIP1L1/PDGFRα(-) CEL which did not respond to routine treatment and imatinib. Allogenic stem cell transplantation should be considered as early as possible in this case. It is noteworthy that clonal CD3-,CD4-,CD8- T-cell abnormality is related to the pathogenesis of CEL.
