CAR-T cell therapy has dramatically changed the treatment paradigm for relapsed/refractory multiple myeloma (R/R MM). Nevertheless, there is a notable paucity of comprehensive bibliometric analyses within this specialized domain. On January 26, 2026, publications on CAR-T cell therapy for MM (2013-2025) were retrieved from the Web of Science Core Collection using: TS = (CAR-T OR "chimeric antigen receptor T*" OR "chimeric antigen receptor modified T" OR "chimeric antigen receptor engineered T" OR "chimeric antigen receptor transduced T" OR "chimeric antigen receptor redirected T") AND TS = ("multiple myeloma*" OR "plasma cell myeloma*" OR "Kahler Disease"). English articles and reviews were included. Bibliometric and visual analyses used CiteSpace 6.2.R1 and VOSviewer 1.6.17. A total of 1,661 eligible documents (including 874 articles and 787 reviews) were included. The annual publication volume showed a continuous upward trend. The United States, Harvard University, Frontiers in Immunology, and Hermann Einsele were the leading contributors by country, institution, journal, and author, respectively. Research focuses on BCMA-CAR-T cell therapy efficacy and safety, comparisons with standard regimens, toxicity management, and combinatorial strategies with bispecific antibodies. Citation burst analysis highlighted emerging directions, including GPRC5D-CAR-T cell therapy and allogeneic "off-the-shelf" CAR-T cell products. The field of research concerning CAR-T cell therapy for multiple myeloma is experiencing rapid advancement. This study systematically summarizes the global research trends, research hotspots and emerging directions in this field, which provides valuable reference for researchers in this field.
BACKGROUND:Myeloablative chemotherapy supported by autologous stem cell transplantation (ASCT) is an option for primary central nervous system lymphoma (PCNSL) in both the relapse setting and as postremission consolidation, but the level of evidence in this field is still low. MATERIAL AND METHODS:We retrospectively analyzed 47 HIV-negative PCNSL patients from 2010 to 2021. To assess the outcomes in patients undergoing ASCT. RESULTS:Of the 47 patients, the median age was 51 (range, 21-77) years, and 28 (59.6%) were male. After induction, 33 (70.2%) patients achieved complete remission, and 6 (12.8%) patients achieved partial remission. At a median follow-up of 21.4 months (95% CI 8.86-33.95), the median progression-free survival (PFS) was 23.3 months (95% CI 14.87-31.73), and the 4-year PFS rate was 14.6%. The median overall survival (OS) time was 62.4 months (95% CI 41.93-82.87), and the 4-year OS rate was 71.5%. Among 20 patients who received ASCT (10 consolidation, 10 salvage), the 4-year PFS and 4-year OS rates were 57.3% and 71.2%, respectively. In the multivariate analysis, ASCT therapy (hazard ratio [HR] 0.16, P=0.016) and early remission (HR 0.12, p=0.003) were found to be independent prognostic factors for a longer PFS. Two treatment-related deaths occurred in patients with multiple relapses before ASCT. Pancytopenia and diarrhea were the most common adverse events. CONCLUSIONS:ASCT offers potential long-term PFS with good tolerability for patients with PCNSL. Our retrospective cohort adds to the currently available literature and identifies disease status after induction as a significant factor affecting survival.
The number of HLA-haploidentical allogeneic hematopoietic stem-cell transplantation (Haplo-HSCT) is increasing. Comparative studies about Haplo-HSCT versus allo-HSCT with HLA-matched sibling donors (MSD-HSCT) have been tried in leukemias and B-cell lymphomas. Few studies were reported in Peripheral T-cell lymphomas (PTCLs). We performed a multicenter retrospective study about 52 patients with PTCLs undergoing Haplo-HSCT (n = 20) or MSD-HSCT (n = 32). All Haplo-HSCT recipients received antithymocyte globulin (ATG) based graft versus host disease (GVHD) prophylaxis. The median follow-up for all survivors was 38 months. The 100-day cumulative incidence of grade II to IV acute GVHD was similar (19% in the MSD-HSCT group versus 28% in the Haplo-HSCT group, P = 0.52). The 2-year cumulative incidence of chronic GVHD (limited and extensive) after Haplo-HSCT (30%) was also similar with that in the MSD-HSCT group (50%, P = 0.15). The 3-year relapse rates (33% vs 27%, P = 0.84) and non-relapse mortality (21% vs 22%, P = 0.78) did not differ between these two groups. There were also no differences in 3-year overall survival (OS) (48% vs 50%, P = 0.78) and progression-free survival (47% vs 51%, P = 0.95) between these two groups. On multivariate analysis, prognostic index for T-cell lymphoma (PIT) score (higher than 1: hazard ratio [HR], 4.0; P = 0.003) and disease status (stable or progression disease before HSCT: HR, 2.8; P = 0.03) were independent variables associated with worse OS. We concluded that ATG-based haplo-HSCT platform could work as an alternative to MSD-HSCT for patients with PTCLs.
Background: There were few studies on real-world data about autologous hematopoietic stem cell transplantation (auto-HSCT) or allogeneic HSCT (allo-HSCT) in peripheral T-cell lymphoma (PTCL). This study aimed to investigate the clinical outcomes of patients who received auto-HSCT or allo-HSCT in China. Methods: From July 2007 to June 2017, a total of 128 patients who received auto-HSCT (n = 72) or allo-HSCT (n = 56) at eight medical centers across China were included in this study. We retrospectively collected their demographic and clinical data and compared the clinical outcomes between groups. Results: Patients receiving allo-HSCT were more likely to be diagnosed with stage III or IV disease (95% vs. 82%, P = 0.027), bone marrow involvement (42% vs. 15%, P = 0.001), chemotherapy-resistant disease (41% vs. 8%, P = 0.001), and progression disease (32% vs. 4%, P < 0.001) at transplantation than those receiving auto-HSCT. With a median follow-up of 30 (2-143) months, 3-year overall survival (OS) and progression-free survival (PFS) in the auto-HSCT group were 70%(48/63) and 59%(42/63), respectively. Three-year OS and PFS for allo-HSCT recipients were 46%(27/54) and 44%(29/54), respectively. There was no difference in relapse rate (34%[17/63] in auto-HSCT vs. 29%[15/54] in allo-HSCT, P = 0.840). Three-year non-relapse mortality rate in auto-HSCT recipients was 6%(4/63) compared with 27%(14/54) for allo-HSCT recipients (P = 0.004). Subanalyses showed that patients with lower prognostic index scores for PTCL (PIT) who received auto-HSCT in an upfront setting had a better outcome than patients with higher PIT scores (3-year OS: 85% vs. 40%, P = 0.003). Patients with complete remission (CR) undergoing auto-HSCT had better survival (3-year OS: 88% vs. 48% in allo-HSCT, P = 0.008). For patients beyond CR, the outcome of patients who received allo-HSCT was similar to that in the atuo-HSCT group (3-year OS: 51% vs. 46%, P = 0.300). Conclusions: Our study provided real-world data about auto-HSCT and allo-HSCT in China. Auto-HSCT seemed to be associated with better survival for patients in good condition (lower PIT score and/or better disease control). For patients possessing unfavorable characteristics, the survival of patients receiving allo-HSCT group was similar to that in the auto-HSCT group.
Objective:To explore difference of clinical effects and prognosis of autologous hematopoietic stem cell transplantation (auto-HSCT) and allogeneic hematopoietic stem cell transplantation (allo-HSCT) in treatment of patients with natural killer/T cell lymphoma (NKTL).Methods:From June 2007 and June 2017, a total of 37 patients underwent auto-HSCT or allo-HSCT from 8 Chinese Grade Ⅲ class A teaching hospitals were selected as research subjects. The median ages of patients in auto-HSCT group and allo-HSCT group were 34.0 years(27.5-43.5 years) and 33.0 years (25.8-41.3 years), respectively. There were 10 male and 7 female patients in auto-HSCT group, and 17 male and 3 female patients in allo-HSCT group, respectively.According to hematopoietic stem cell transplantation (HSCT) types of patients underwent, all subjects were divided into auto-HSCT group ( n=17) and allo-HSCT group ( n=20). Then clinical data of NKTL patients in auto-HSCT group and allo-HSCT group were collected, and clinical effect and prognosis of patients in two groups were analyzed by retrospectively cohort method. Patients in both groups were followed up until December 31, 2017. Chi-square test or Fisher probabilities were used to compare categorical data between two groups, including composition ratios of clinical stages, marrow metastases, types of chemotherapy regimen and so on. Mann-Whitney U test was used to compare measurement data with skewed distribution, including age, transfusion amount of mononuclear cells (MNC) and CD34 + cells etc.. Kaplan-Meier method was used to draw survival curve of cumulative relapse, cumulative transplant-related mortality (TRM), overall survival (OS) and progression-free survival (PFS) of two groups, which were compared by log-rank test. Cox proportional hazards regression model was performed for univariate and multivariate analysis of OS rates between two groups. This study was in line with World Medical Association Declaration of Helsinki revised in 2013, and informed contents were obtained from all subjects. Results:① In auto-HSCT group, there were 12, 4, 1 and 0 patients evaluated as complete remission (CR), partial remission (PR), stable disease (SD) and progressive disease (PD) before HSCT, respectively. There were 4, 11, 1 and 4 cases in allo-HSCT group, respectively.There were significant differences among composition ratios of bone marrow metastase, disease states before HSCT and types of conditioning regimen ( P=0.001; χ2=12.087, P=0.016; χ2=23.632, P<0.001) between two groups. ② In auto-HSCT group, all 17 NKTL patients had successful neutrophil and platelet engraftment, and median engraftment time were 11.0 d (9.5-11.0 d) and 11.0 d (9.5-14.0 d), respectively. In allo-HSCT group, there were 18 NKTL patients with sucsessful neutrophil and platelet engraftment, and median engraftment time were 15.0 d (13.0-16.0 d) and 16.0 d(13.0-20.3 d), respectively. There was no significant difference in 3-year cumulative TRM (6.2% vs 23.7%; χ2=1.658, P=0.198) between two groups. ③ There were 13, 6 and 9 patients with graft versus host disease (GVHD), acute GVHD (aGVHD) and chronic GVHD (cGVHD), respectively. Patients with GVHD acquired effective control and improved of GVHD-related clinical symptoms after therapy. ④ There were no significant differences in 3-year cumulative relapsed rates (35.5% vs 48.1%; χ2=0.796, P=0.372) and 2-year PFS rates (64.8% vs 43.1%; χ2=2.765, P=0.096) between two groups, but 2-year OS rate in auto-HSCT group was significantly higher than that of allo-HSCT group (82.0% vs 41.3%; χ2=4.697, P=0.03). ⑤ The results of multivariate Cox proportional hazard regression analysis showed that underwent allo-HSCT was not an independent risk factor influencing OS rate of NKTL patients ( HR=4.618, 95% CI: 0.992-21.497, P=0.051). Conclusions:Auto-HSCT and allo-HSCT are both effective therapeutic methods for NKTL patients. OS rate of NKTL patients who acquire CR before HSCT could be improved by auto-HSCT, and disease state of patients who are relapsed or refractory can be controlled by allo-HSCT. However, this study is analysis by retrospective mothod, and sample size is limited, so the differences in efficacy and prognosis between auto-HSCT and allo-HSCT in treatment of NKTL patients needs to be further confirmed by prospective, large sample size and clinical randomized controlled studies.
Objective A retrospective study was performed to compare intensive immunosuppressive therapy (IST) and haploidentical donor hematopoietic stem cell transplantation (HID-HSCT) for young patients with severe aplastic anaemia(SAA). Methods Fifty-five SAA patients aged 14-30 years were included. The patients were treated with anti-thymocyte immunoglobulin(ATG) combined with cyclosporine A(CsA)(29 cases, IST group) or HID-HSCT(26 cases, HID-HSCT group). Results The complete response(CR) rates in the IST and HID-HSCT groups were 58.6% vs 84.6%(P=0.034), and the overall response(OR) rates were 86.2% vs 84.6%. No significant difference was found in estimated 5-year overall survival(IST 77.9%±11.7% vs HID-HSCT 82.1%±8.4%) and event-free survival(IST 67.6%±11.7% vs HID-HSCT 69.2%±9.1%). The relapse rate of the IST group was 3.4%, and the clonal transformation rate was 13.8%. In HID-HSCT group, the relapse and clonal transformation rates were both 0%, and the incidence rates of grades 2-4 aGVHD and cGVHD were 19.2% and 15.4% respectively. The hospitalization expenses were significantly less in the IST group than in that of HID-HSCT group(median 168 000 yuan vs 378 000 yuan, P<0.001). Conclusions IST shows similar OR, OS and EFS but much less expense comparing with that of HID-HSCT. IST remains an appropriate choice for young SAA patients without sibling donors.
Objective: To explore the efficacy and prognostic factors of hematopoietic stem cell transplantation (HSCT) for the treatment of patients with anaplastic large cell lymphoma (ALCL) . Methods: The clinical records of 33 ALCL patients after HSCT were collected and analyzed retrospectively to evaluate the rates of overall survival (OS) and recurrence after autologous (auto-HSCT) and allogeneic HSCT (allo-HSCT) and the factors influencing prognosis. Results: The median-age of this cohort of 33 ALCL cases at diagnosis was 31 (12-57) years old with a male/female ratio of 23/10, 24 cases (72.7%) were ALK(+) and 9 ones (27.3%) ALK(-). Of them, 25 patients (19 ALK(+) and 6 ALK(-)) underwent auto-HSCT and 8 cases (5 ALK(+) and 3ALK(-)) allo-HSCT with a median follow-up of 18.7 (4.0-150.0) months. Disease states before HSCT were as follows: only 6 patients achieved CR status and received auto-HSCT, 16 patients achieved PR (14 cases by auto-HSCT and 2 ones allo-HSCT) , the rest 11 cases were refractory/relapse (5 cases by auto-HSCT and 6 ones allo-HSCT) . There were 7 cases died of disease progression (5 after auto-HSCT and 2 allo-HSCT) and 5 cases treatment-related mortality (TRM) (2 after auto-HSCT and 3 allo-HSCT) , TRM of two groups were 8.0% and 37.5%, respectively. Both the median progression-free survival (PFS) and OS were 15 months after auto-HSCT, the median PFS and OS after allo-HSCT were 3.7 (1.0-90.0) and 4.6 (1.0-90.0) months, respectively. There was no statistically significant difference in terms of survival curves between the two groups (OS and PFS, P=0.247 and P=0.317) . The 2-year OS rates in auto-HSCT and allo-HSCT groups were 72% and 50%, respectively. The 5-year OS rates in auto-HSCT and allo-HSCT groups were 36% and 25%, respectively. Conclusion: ALCL treated by chemotherapy produced high rates of overall and complete responses. Chemotherapy followed by auto-HSCT remained to be good choice for patients with poor prognostic factors. High-risk patients should be considered more beneficial from allo-HSCT.
Objective: To evaluate the clinical outcomes in patients with relapsed or refractory peripheral T-cell lymphoma (PTCL) who had undergone allogeneic hematological stem cell transplantation (allo-HSCT). Methods: From June 2007 to June 2017, the clinical data of PTCL patients who underwent HSCT from eight hospitals were assessed retrospectively. Results: There were 23 patients diagnosed as relapsed or refractory PTCL with chemoresistance who underwent allo-HSCT. Among these patients, 18 were identified as progressive disease (PD) status and 5 patients as stable disease (SD) status before allo-HSCT. Seventeen patients received allo-HSCT from matched sibling donor (MSD),2 patients from matched unrelated donor and 4 patients from related haplo-identical donor (HD). After a median follow-up of 29 months, 21 patients survived longer than 28 days after allo-HSCT. Hematopoietic reconstitution was achieved in 20 of the 21 patients. The median time of myeloid and platelet engraftment were+13 (9-22) d and+16(10-38) d, respectively. The 100-d treatment-related mortality rate was 13.1%. Acute GVHD occurred in 11(47.8%) patients at a median time of 22(6-82) d after transplantation. Grade Ⅱ~Ⅳ aGVHD occurred in 6 patients. Chronic GVHD occurred in 10 patients at a median of 7.9 (3.5-27) months. After a median follow-up of 29 months, 13 patients died after HSCT. Four of them died of complications associated with allo-HSCT, and other 9 patients died of the primary lymphoma. The 3-years cumulative overall survival (OS) and progress-free survival (PFS) were 43.03% (95%CI: 29.79-69.16) and 39.13% (95%CI: 23.50-65.14), respectively. No significant difference was found in the 3-year PFS between patients with PD status and SD status before allo-HSCT (P=0.133). Conclusion: Allo-HSCT can be a promising treatment for relapsed or refractory PTCL with chemoresistance.
Objective: To evaluate clinical outcomes of autologous (auto-HSCT) and allogeneic hematopoietic stem cell transplantation (allo-HSCT) for angioimmunoblastic T-cell lymphoma (AITL) . Methods: From June 2007 to June 2017, clinical data of AITL patients who underwent HSCT in eight hospitals were assessed retrospectively. Results: Of 19 patients, 13 male and 6 female with a median age of 50 (32-60) years old, 12 auto-HSCT and 7 allo-HSCT recipients were enrolled in this study, all donors were HLA-identical siblings. Two of allo-HSCT recipients were relapsed auto-HSCT ones. There were 5 patients (5/12) in complete response (CR) status and 7 (7/12) in partial remission (PR) status before transplantation in auto-HSCT group, and 2 (2/7) in PR status and 3 (3/7) in progression disease (PD) status before transplantation in allo-HSCT group. The median follow-up for the surviving patients was 46.5 months (range, 1-100 months) for the whole series, two patients lost in auto-HSCT group. Three patients developed acute graft-versus-host disease (aGVHD) and 5 chronic graft-versus-host disease (cGVHD) after allo-HSCT. Three patients died of primary disease and 1bleeding in auto-HSCT group. One patient died of primary disease and 2 transplantation-related mortality in allo-HSCT group. The 3-year cumulative overall survival (OS) were 56% (95%CI 32%-100%) and 57% (95%CI 30%-100%) for auto-HSCT and allo-HSCT, respectively (P=0.979) . The 3-year cumulative progression-free survival (PFS) were 34% (95%CI 14%-85%) and 57% (95%CI 30%-100%) for auto-HSCT and allo-HSCT, respectively (P=0.451) . Conclusion: Both auto-HSCT and allo-HSCT were optimal choices for AITL. In clinical practice, which HSCT was better for AITL patients should be based on comprehensive factors including sensitivity to chemotherapy, risk stratification and disease status at transplantation.
目的 观察硼替佐米联合化疗治疗难治性套细胞淋巴瘤(MCL)的疗效。 方法 选择2014年7月解放军总医院第一附属医院收治的1例难治性MCL患者为研究对象,患者连续接受不同化疗方案共13个疗程后无明显疗效,给予硼替佐米联合MOFP(米托蒽醌、长春碱、氟达拉滨、甲泼尼龙)方案化疗,分析该例患者的临床资料并复习相关文献。 结果 硼替佐米联合MOFP方案化疗5个疗程后,患者颈部淋巴结肿物缩小75%以上,且腹腔淋巴结肿物及胸腔积液消失,至截稿前无进展生存10个月余。 结论 难治性MCL患者予硼替佐米联合MOFP方案疗效显著。
Background: This study not only evaluated the clinical effects of treatment using haploidentical hematopoietic stem cells (haplo-HSCs) combined with human umbilical cord mesenchymal stem cells (UC-MSCs) in patients with severe aplastic anemia (SAA), but also investigated the factors related to graft versus host disease (GVHD). Methods: Cotransplantation of haplo-HSCs and UC-MSCs was performed in 24 SAA patients. The conditioning regimens consisted of rabbit anti-human T-lymphocyte immunoglobulin (ATG), cyclophosphamide, and fludarabine with or without busulfan. GVHD was prevented using cyclosporine A, ATG, anti-CD25 monoclonal antibody, and mycophenolate material. Results: The incidence of acute GVHD was 50%. The incidence of severe acute GVHD was not related to gender, age, donor-recipient relations, and patient/donor pair, while patient/donor pair (r = 0.541, P = 0.022) was significantly correlated with incidence of chronic GVHD. Upon follow-up for a median of 13 months, 5 of the 24 patients (20.8%) were dead. The survival rates at 3 and 6 months in all patients were 87.5% (21/24) and 83.3% (20/24), respectively. Conclusion: Cotransplantation of haplo-HSCs combined with UC-MSCs was an effective and safe approach for the treatment of patients with SAA. The appropriate conditioning regimen and early treatment for infection also played a critical role in the success of HSCT.
OBJECTIVETo explore the relationship between HLA-A, -B, -C, -DRB1, -DQB1 gene polymorphism and aplastic anemia (AA)of 65 cases in Northern China.METHODSThe high resolution genotyping of HLA-A, -B, -C, -DRB1, -DQB1 alleles in 65 AA patients and 772 healthy controls was performed with polymerase chain reaction-sequence specific oligonucleotide (PCR-SSO), the relationship between HLA-A, -B, -C, -DRB1, -DQB1 gene polymorphism and aplastic anemia was analyzed by Pearson Chi-square,Continuity Correction, Two-sided Fisher's Exact Test and Odds Ratio.RESULTSThe HLA-B*1302(10% vs 4.21%), B*3501(7.69% vs 3.89%), DRB1* 0701(10% vs 4.73%), DRB1*0901(19.23% vs 7.58%), DQB1*0202(9.23% vs 3.76%) gene frequency in AA patients was higher than those in health controls, the difference was statistically significant (P<0.05), the χ2 were 9.049, 4.336, 6.838, 20.974 and 8.968, OR ratio was 2.528, 2.061, 2.239, 2.904 and 2.605. However, the HLA-A*3303(1.54% vs 6.93%), DQB1*0302(1.54% vs 6.02%) gene frequency in AA patients was lower than those in healthy controls, the difference was statistically significant (P<0.05), the χ2 was 5.726 and 4.505, the OR ratio were 0.210 and 0.244.CONCLUSIONThe polymorphism of HLA-A, -B, -DRB1, -DQB1 alleles is associate with AA in these patient cases, the HLA-B*1302, HLA-B*3501, HLA-DRB1*0701, HLA-DRB1*0901 and HLA-DQB1*0202 may be sensitive genes to AA, while the HLA-A*3303 and HLA-DQB1*0302 may be protective genes on AA.
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.
Objective To observe the clinical outcome and safety of ultra low-dose of decitabine (DAC) combined with reduced-intensity CAG (aclacinomycin, cytarabine, granulocyte colony stimulating factor) therapeutic regimen on intermediate or high risk myelodysplastic syndrome (MDS). Methods Total of 30 patients treated by ultra low-dose DAC combined with reduced-intensity CAG therapeutic regimen in our center from Apr. 2015 to Oct. 2016 were included in this study, and the clinical outcome, adverse reactions, and survival data were recorded. Results Of all the patients, complete remission (CR) rate was 53% (16 cases), partial remission rate (PR) was 17% (5 cases), hematological improvement (HI) rate was 10% (3 cases), total failure (TF) was 20% (6 cases) and overall response rate (ORR) was 80%. Infection was the main adverse event caused by DAC and occurred in 15 patients (50%), with 2 of them severely infected. The median survival time was 11 months (2-18 months) with 24 survived and 4 died. Conclusion Ultra low-dose DAC combined with reduced-intensity CAG therapeutic regimen is effective and safe on intermediate or high risk MDS with lower rate of severe infection and hematological toxicities.
Matched sibling donor (MSD) or matched unrelated donor (MUD) hematopoietic stem cell transplantation is the front-line therapy for severe aplastic anemia. When MSD or MUD is not available, then intensive immunosuppressive therapy (IST) is indicated. However, IST has a high relapse rate due to lack of response. Haploidentical hematopoietic stem cell transplantation (Haplo-HSCT) has been developing so rapidly that they could now replace the transplantation from identical siblings with similar efficacy. The application of Haplo-HSCT terminate the era of donor shortage and bring benefit to the patients. This review will focus on the progress of the therapeutic effect of Haplo-HSCT in the treatment of SAA.
将比喻教学法引入血液病规范化培训教学中,明确比喻教学法在临床教学实践中的有效性,从而提高住院医师的规范化培训质量.
Objective To observe the clinical safety and efficacy of foscarnet prophylaxis and pre-emptive therapy for cytomegalovirus (CMV) infection in allogeneic hematopoietic stem cell transplantation (allo-HSCT). Methods Ninety-six patients undergoing allo-HSCT from October 2014 to December 2016 were retrospectively analyzed. Plasma CMV-DNA was monitored with real-time quantitative polymerase chain reaction (RQ-PCR) from beginning to 180 days after transplantation. Foscarnet was used not only for prophylaxis but also for first-line pre-emptive therapy when plasma CMV-DNA turned to positive. Foscarnet was given 60 mg·kg-1·d-1 and 120 mg·kg-1·d-1 respectively in prevention and pre-emptive therapy. Incidences of CMV infection and CMV disease were observed, influencing factors on CMV in faction and the efficacy and safety of foscarnet prophylaxis were analyzed, and survival of patients treated by all-HSCT was evaluated. Results Of the total 96 patients, 42 cases (43.8%) had CMV infection with the median time of 42 days after allo-HSCT. CMV-DNA became negative in 36 patients (85.7%, 36/42) after pre-emptive therapy. Six patients (14.3 %, 6/42) developed CMV disease, including 5 patients with CMV negative and 1 patient died for CMV pneumonia. Haploidentical donor and grade Ⅱ-Ⅳacute graft versus host disease (GVHD) were the risk factors for CMV reactivation (χ2 = 3.834, P< 0.05; χ2 = 16.807, P< 0.001). The side effects of foscarnet prophylaxis were mild without hematologic toxicities. 12 patients (28.6 %) died in 42 patients with CMV infection, and 6 patients (11.1 %) died in 54 patients without CMV infection. The difference of survival rates between both groups was not statistically significant. Conclusion Foscarnet is an effective agent for prophylaxis and pre-emptive therapy in CMV infection after allo-HSCT with mild adverse reactions, especially for patients following with hematopoietic recovering.
OBJECTIVE:To investigate the efficacy and clinical safety of posaconazoleon primary antifungal prophylaxis against invasive fungal disease (IFD) in patients with stem cell transplantation. METHODS:At the start from preconditioning regimen, 45 patients without IFD were administered with posaconazoleon until neutrophils greater than 0.5×109/L, 35 patients treated with micafungin were enrolled in control group. The incidence, risk factors of IFD and side effects of medicines were evaluated. RESULTS:Of the total 80 patients, 13(16%) had IFD within 100 days after allo-HSCT. The overall survival was significantly different between patients with or without IFD by Kaplan-Meier survival curve analysis (P<0.05). Out of the 45 cases in posaconazoleon group, IFD occurred in 4 cases (9%). In contrast, the incidence of IFD in control group was 26%(9 out of 35) (P<0.05). The risk factors of IFD and side effects were not significantly different between 2 groups(P>0.05). CONCLUSION:The primary prevention efficancy of IFD by posaconazoleon after allo-HSCT is much better than that of micafungin with well tolerability and satisfactory efficacy.
OBJECTIVE:To analyse the feasibility and compare differences between hematopoietic reconstitution and prognosis of patients with severe aplastic anemia(SAA) after matched sibling donor (MSD) or haploidentical family donor (HFD) hematopoietic stem cell transplantation (HSCT) using the modified FC/ATG conditioning.METHODS:The clinical data of 56 patients with SAA who received HSCT in First Affiliated Hospital of Chinese PLA General Hospital from January 2011 to June 2016 were analyzed retrospectively. The hematopoietic reconstitution, graft verus host disease (GVHD), transplantation related toxicity (TRT) and prognosis after transplantation were compared. Furthermore, the modifed conditioning FC/ATG included low-dose cyclophosphamide (total dose 100 mg/kg), infustion of third-party donor-derived mesenchymal stem cells.RESULTS:All 56 patients with MSD-HSCT or HFD-HSCT achieved hematopoietic reconstitution. Among them, not only the recovery of neutrophils and platelets, but also the incidences of III-IV aGVHD, extensive cGVHD and TRT were not significantly different (the P value were 0.58, 0.61, 0.73, 0.73 and 0.67, respectively). After following-up for 32(2-66) months, 48 patients alive well, the 1-year overall survival rates were 86% in HFD-HSCT group and 89% in MSD-HSCT group, respectively (P=0.58).CONCLUSION:After HSCT using the modifed FC/ATG conditioning, patients with SAA achieved stable engraftment, low toxicity, mild GVHD and excellent outcomes. Furthermore, the HFD-HSCT achieved comparable outcomes to MSD-HSCT and may be served as an alternate therapy for patients with SAA.
Objective:To investigate the effects of CsA on the proliferation,apoptosis,and cell cycle of human umbilical cord mesenchymal stem cells (UC-MSC).Methods:UC-MSC were treated with CsA at different concentrations.The inhibitory effect of CsA on cell proliferation was detected by MTT assay,the apoptosis and cell cycle were detected by flow cytometry.Results:CsA had no inhibitory effect on cell proliferation of UC-MSC,but could induce apoptosis of UC-MSC.There was a increase trend of apoptosis rate when the CsA concentration was beyond 240 ng/ml.CsA could also change cycle process of UC-MSC in concentration dependent manner,which could decrease cell proportion in G0/G1 phase and increase it in S phase.Conclusion:CsA has no obvious negative effect on the cell fuction of UC-MSC.