Introduction Hematopoietic stem cell transplantation (HSCT) and chemotherapy are considered potentially curative options for post-remission therapy in acute myeloid leukemia (AML). However, the comparative effectiveness of these approaches in favorable- and intermediate-risk AML remains unclear and requires further investigation.Methods In this retrospective study, 111 patients diagnosed with de novo favorable- and intermediate-risk AML, categorized according to the ELN 2022 guidelines, were investigated to compare outcomes following autologous HSCT (auto-HSCT), matched sibling donor HSCT (MSD-HSCT), and chemotherapy. Through propensity score matching for disease status before HSCT, 42 cases in first complete remission were selected for each of the auto-HSCT group and the MSD-HSCT group. Additionally, 27 cases in the chemotherapy group, excluding patients with early relapse or death, were included for comparison.Results In the overall population, the 3-year overall survival (OS) rates were 85.7%, 83.1%, and 70.4% (p = 0.043), while the disease-free survival (DFS) rates were 78.6%, 83.2%, and 57.1% (p = 0.002) in the auto-HSCT, MSD-HSCT, and chemotherapy groups, respectively. Notably, both auto-HSCT and MSD-HSCT demonstrated significantly improved DFS compared to chemotherapy in patients with favorable-risk AML. Multivariate analysis further revealed that chemotherapy was significantly associated with inferior DFS compared to auto-HSCT (HR=2.82; 95% CI, 1.26-6.32, p=0.012), while DFS was similar between the MSD-HSCT and auto-HSCT groups (HR=0.80; 95% CI, 0.31-2.09, p=0.645).Discussion The findings suggested the advantages of both MSD-HSCT and auto-HSCT over chemotherapy as post-remission therapy for AML patients with favorable and intermediate risk. Further research is needed to support these conclusions.

BACKGROUND:Myeloid sarcoma (MS) is a rare extramedullary manifestation of myeloid neoplasms and usually has poorer outcomes than acute myeloid leukemia without extramedullary disease. Allogeneic hematopoietic stem cell transplantation is the most effective consolidation, but evidence on allogeneic peripheral blood stem cell transplantation (allo-PBSCT), especially from haploidentical donors, is limited. METHODS:We retrospectively reviewed 20 consecutive patients with biopsy-confirmed MS who underwent allo-PBSCT at our center (2012-2025). All grafts were peripheral blood. Donors were haploidentical in 14 patients (70.0%), matched related in 4 (20.0%), and matched unrelated in 2 (10.0%). Overall survival (OS) and disease-free survival (DFS) were estimated by the Kaplan - Meier method; relapse incidence (RI), non-relapse mortality (NRM), and graft-versus-host disease (GVHD) were analyzed as competing risks. RESULTS:Twenty patients (median age, 28.5 years) were included; 45.0% had isolated extramedullary MS and 55.0% had concurrent bone marrow disease. At transplantation, 16 (80.0%) were in first complete remission, 18 (90.0%) were measurable residual disease (MRD) negative, and all had HCT-CI = 0. After a median follow-up of 38 months, 1- and 2-year OS and DFS were both 90.0%, with RI 0% and NRM 10.0%; at 3 years, OS/DFS were 81.8%, RI 8.2%, and NRM 10.0%. Haploidentical recipients (14/20) had comparable outcomes (3-year OS/DFS 92.9%, no relapse, NRM 7.1%). Acute GVHD occurred in 60.0%, but grade II - IV in only 20.0%; chronic GVHD was 30.0%, mostly mild. CONCLUSION:Allo-PBSCT can achieve durable remission in selected MS patients and is a practical option when an HLA-matched donor is unavailable.

OBJECTIVES:To evaluate posaconazole (POS) gastro-resistant tablets for preventing invasive fungal disease (IFD) in haematopoietic stem cell transplantation (HSCT) patients and analyse POS plasma concentrations. METHODS:A single-arm trial was designed with a historical cohort as a control. Patients aged 13 years and older undergoing HSCT at the HSCT Center of Blood Diseases Hospital, Chinese Academy of Medical Sciences between December 2020 and May 2022 were enrolled, prospectively taking POS gastro-resistant tablets orally from day 1 to day 90 post-transplant and monitoring plasma concentrations. We also identified a retrospective cohort treated with alternative antifungal prophylaxis between January 2018 and December 2020, matched using propensity score methods. The primary outcome was the cumulative incidence of IFD at day 90 post-transplant. RESULTS:The prospective study involved 144 patients receiving POS gastro-resistant tablets for IFD prevention, contrasting with 287 patients receiving non-POS tablets. By day 90 post-transplant, the POS tablet group exhibited a significantly lower cumulative incidence of IFD (2.81%; 95% CI, 0.09-5.50% vs. 7.69%; 95% CI, 4.60-10.78%; p 0.044). Adverse events were comparable between the groups with liver changes in 33/144 (22.92%) vs. 84/287 (29.27%) (p 0.162), and renal injuries in 15/144 (10.41%) vs. 37/287 (12.89%) (p 0.457). Mean POS plasma concentrations on days 4, 8, 15, and 22 post-administration were 930.97 ng/mL, 1143.97 ng/mL, 1569.8 ng/mL, and 1652.57 ng/mL, respectively. DISCUSSION:Patients administered POS gastro-resistant tablets for antifungal prophylaxis experienced a lower cumulative incidence of IFD. POS plasma concentrations in HSCT patients stabilized by day 15 of medication.

The 2022 European LeukemiaNet (ELN) updated the previous risk classification published in 2017 but the prognostic significance for allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains unclear. We enrolled 600 acute myeloid leukemia (AML) patients who underwent allo-HSCT to validate ELN-2022 genetic risk system and compared it with ELN-2017. There were 214 (35.67%), 162 (27.0%), and 224 (37.33%) patients in ELN-2022 favorable-, intermediate-, and adverse-risk group respectively and 86 patients (14.33%) experienced a shift in risk stratification compared to ELN-2017. Median and maximum follow-up time were 2.89 (95% CI 2.67 to 3.03) years and 8.78 years. The median overall survival (OS) was 73.8% (95% CI 67.5% to 80.3%), 63.9% (95% CI 56.7% to 72.0%) and 57.6% (95% CI 50.4% to 65.9%) in ELN-2022 favorable-, intermediate-, and adverse-risk group (P < 0.001). OS shortened significantly as the ELN-2022 risk stratification increased but didn’t significantly in ELN-2017 intermediate-risk compared to favorable-risk. Both ELN-2022 and ELN-2017 adverse-risk were associated with increased cumulative incidence of relapse (CIR). Time-dependent receiver operating characteristic (ROC) analysis showed that both ELN-2017 and ELN-2022 risk systems had limited prognostic ability for OS. We modified ELN-2022 risk system with pre-transplant minimal residual disease (MRD) and the modified risk system performed a significantly superior efficacy to ELN-2022 system.

Aims: To explore a new method for treating failure of rituximab plus immunosuppressant dose reduction in EB virus-related post-transplant lymphoproliferative disorders (PTLD). Methods: This study retrospectively analyzed the clinical data of 60 patients diagnosed with PTLD at the Institute of Hematology & Blood Diseases Hospital and Chinese Academy of Medical Sciences from May 2017 to June 2024, including confirmed and clinically diagnosed cases. Among them, 22 patients underwent lymph node biopsy, and except for 3 patients whose biopsy specimens were not marked with CD38, the remaining 19 patients had positive CD38, of whom 10/19 were strongly positive. We treated 4 patients with failed rituximab plus immunosuppressant dose reduction therapy (1 case of acute T-cell leukemia, 1 case of acute myeloid leukemia, 1 case of acute B-cell leukemia, and 1 case of Ph+ mixed cell leukemia) with daratumumab. Results: Of the 4 patients, 3 had obvious disease progression after receiving rituximab 375mg/m2/w twice (EBV-DNA progressively increased to more than 100 times the baseline level or lymph nodes progressively enlarged), and 1 had an initial EBV-DNA level of 107,310 copies/ml, which increased to 354,787 copies/ml after receiving rituximab. All 4 patients received daratumumab 16mg/kg qw 2-4 times, and all patients achieved complete remission (CR). They were followed up for 2.5 to 22.5 months, and all patients were alive and in complete remission from PTLD. 2/4 patients experienced a clear viral infection after receiving the CD38 monoclonal antibody, including one case of widespread adenovirus infection and one case of herpes simplex virus infection in the mouth, nose, and eyes, which improved after treatment. Conclusion: This study is the first to reveal high expression of CD38 in PTLD and successfully treated 4 patients who failed to reduce their treatment with rituximab and immunosuppressants. Daratumumab may become a new option for the treatment of PTLD patients.

Disease recurrence is the leading cause of treatment failure in patients with RUNX1::RUNXT1-positive acute myeloid leukemia (AML) after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Post-transplant maintenance therapy, guided by monitoring minimal residual disease (MRD), is commonly administered; however, relapse rates remain high. This prospective study aimed to assess the effectiveness and safety of epigenetic agents as prophylactic therapy in patients with RUNX1::RUNXT1-positive AML. Thirty high-risk patients received prophylactic therapy (n = 17 and n = 13 in the chidamide and AZA groups, respectively) between January 2019 and July 2023. 34 high-risk patients who received preemptive treatment due to molecular relapse were included in the analysis. The two-year relapse-free survival (RFS) and overall survival (OS) were significantly higher in the prophylactic group compared to the preemptive group (82.82

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) maintains the only promising curative option for patients with refractory/relapsed (R/R) acute myeloid leukemia (AML). However, the long-term survival results are suboptimal. Optimization of the conditioning regimen aims to eradicate leukemia blasts and reduce early relapse. Here we reported of the preliminary result of the prospective multicenter single arm study to evaluate the efficacy and safety of a modified dual alkylator-conditioning regimen, MCBC (regimen including Melphalan, Cladribine, Busulfan and Cyclophosphamide) (ChiCTR Registration ID: ChiCTR2000029936). This trial enrolled 56 patients from July 2020 to January 2022. With a median follow-up of 854 days (range 48 to 1343), the 2-year overall survival (OS) and relapse-free survival (RFS) were 60.7 +/- 6.5% (95% CI 47.5-73.9) and 57.1 +/- 6.6% (95% CI 43.870.5), respectively, the estimated 3-year OS and RFS rates were 58.9 +/- 6.6% (95% CI 45.6-72.2) and 55.4 +/- 6.6% (95% CI 41.9-68.8), respectively. A total of 19 patients experienced relapse, the 2-year cumulative incidence relapse (CIR) rate was 34.2 +/- 6.6% (95% CI 19.5-44.8), the estimated 3-year CIR rate was 36.3 +/- 6.7% (95% CI 21.1-46.7). Six patients died of severe infection or graft-versus-host disease (GVHD). The non-relapse mortality (NRM) rate was 11.8 +/- 4.5% (95% CI 2.4-19.1). Mucositis was the main reported regimen-related toxicity, and it was well controlled. Subgroup analyses illustrated that blasts count >= 20% before HSCT and the absence of maintenance treatment after HSCT were poor predictors. Our study confirmed the excellent anti-leukemia activity and acceptable toxicity of the MCBC conditioning regimen in R/R AML. Opportune maintenance treatment after HSCT led to significantly improved OS and RFS.

AbstractChimeric antigen receptor (CAR) T cells show suboptimal efficacy in acute myeloid leukemia (AML). We find that CAR T cells exposed to myeloid leukemia show impaired activation and cytolytic function, accompanied by impaired antigen receptor downstream calcium, ZAP70, ERK, and C-JUN signaling, compared to those exposed to B-cell leukemia. These defects are caused in part by the high expression of CD155 by AML. Overexpressing C-JUN, but not other antigen receptor downstream components, maximally restores anti-tumor function. C-JUN overexpression increases costimulatory molecules and cytokines through reinvigoration of ERK or transcriptional activation, independent of anti-exhaustion. We conduct an open-label, non-randomized, single-arm, phase I trial of C-JUN-overexpressing CAR-T in AML (NCT04835519) with safety and efficacy as primary and secondary endpoints, respectively. Of the four patients treated, one has grade 4 (dose-limiting toxicity) and three have grade 1–2 cytokine release syndrome. Two patients have no detectable bone marrow blasts and one patient has blast reduction after treatment. Thus, overexpressing C-JUN endows CAR-T efficacy in AML.

Introduction: Allogeneic hematopoietic cell transplantation (allo-HCT) is a promising treatment for patients with high-risk B-lineage acute lymphoblastic leukemia (B-ALL). To reduce the risk of relapse, we explored the role of blinatumomab as maintenance therapy post allo-HCT in patients with high-risk B-ALL. This study aimed to investigate the efficacy and safety of blinatumomab therapy post allo-HCT in patients with B-ALL. Methods: This is a prospective, single-arm phase II study. The efficacy of 4 cycles of blinatumomab administered every 3 months post allo-HCT was investigated. Eligible patients received blinatumomab at a dose of 8.75 µg/day from day 1-4, 17.5 µg/day on days 5 and 6, and 28 µg/day from day 7-16. Patients administered with bevacizumab prior allo-HCT, received blinatumomab at a dose of 17.5 µg/day on days 1 and 2, 28 µg/day on day 3-12. The primary endpoints were the 3-year cumulative recurrence rate (CIR) and drug related toxicity. Secondary endpoints included overall survival (OS), progression free survival (RFS), non-relapse mortality (NRM), incidence of acute and chronic graft-versus-host disease (GVHD). Inclusion criteria included patients: (1) aged ≥14 years (2) Undergone allo-HCT, including HLA-matched sibling, haploidentical donor, and unrelated donor transplantations (3) High risk acute B-ALL (such as Ph+B-ALL, Ph like B-ALL, high white blood cell counts at onset, and no remission after 2 cycles of chemotherapy) (4) Complete remission with minimal residual disease (MRD) negativity including absolute neutrophil and platelet counts of ≥1.0×109/L and ≥50×109/L, respectively, for >1 week (5) ≤ grade 3 acute GVHD or moderate to severe chronic GVHD within 30 days prior to enrollment (6) Signed the informed consent form. Exclusion criteria included: (1) Patients with hematological or MRD recurrence (2) Presence of severe active infections (3) Significant abnormalities in routine laboratory tests (4) ECOG physical fitness status score ≥3 (5) Expected survival period < 3 months. OS and non-relapse mortality were calculated from the date of allo-HCT to the last recorded vital sign. PFS was calculated from the date of allo-HCT to the date of disease progression or death. OS and PFS were estimated by Kaplan-Meier method. The cumulative incidences of relapse, NRM, and GVHD were evaluated by competing risks method. The competing risk for relapse was death, for NRM was relapse, and for GVHD were relapse and death. Group differences in OS and PFS were evaluated by stratified log-rank test, while cumulative incidences were assessed by stratified Gray's test. Results ：Eleven patients were enrolled to date and received at least 1 cycle of blinatumomab (range:1-4) during the first-year post allo-HCT. Of the patients, 73% were male with a median age of 34 (16-62) years. The median number of days from allo-HCT to cycle 1 of blinatumomab was 78 days (range: 44-105). Decrease of immunoglobulin A and G was the most common side effects, occurred in 10 patients. During medication, 3 patients developed fever, 2 patients developed headaches and 1 patient developed mild pulmonary infection, which improved after anti-infectious treatment. Hematologic cytopenia, including leukopenia was reported in 5 patients, neutropenia in 2 patients with acute grade 1 GVHD in 1 patient. The median time of follow-up from diagnosis to allo-HCT was 2.5 months (range: 3-67). Only 1 patient reported molecular biology recurrence, while the remaining patients remained in durable remission. No cases of non-relapse mortality were reported. Conclusion: Blinatumomab maintenance therapy post allo-HCT has shown initial feasibility and efficacy for B-ALL and is well tolerated. Future studies with a large number of patients are required to confirm its efficacy.

Background Olverembatinib is an oral, third-generation BCR-ABL1 tyrosine kinase inhibitor (TKI) developed in China that could effectively target wild-type and mutant BCR-ABL1 kinase. It is effective and well-tolerated for patients with chronic myeloid leukemia. However, its efficacy and safety in patients with Ph/BCR-ABL1-Positive acute lymphoblastic leukemia are not entirely clear. This study aims to analyze the efficacy and safety of Olverembatinib-based therapies in patients with Ph/BCR-ABL1-Positive acute lymphoblastic leukemia. Methods Patients with Ph/BCR-ABL1-positive acute lymphoblastic leukemia who received the Olverembatinib-based therapies in our center were included to conduct this retrospective study. Results Totally 33 patients with Ph/BCR-ABL1-positive acute lymphoblastic leukemia were included in this study with a median age of 41 years, of whom 21 (63.63%) were female. Among them, 19 subjects who had been previously treated with standard chemotherapy and three newly diagnosed patients received Olverembatinib-based therapies, while 11 patients received Olverembatinib-based therapies after hematopoietic stem cell transplantation. After the first course of the Olverembatinib-based regimen, 33 (100%) patients experienced remission (CR/CRi/ Morphologic leukemia-free state), and 22 (66.67%) achieved the negative minimal residual disease (MRD) of BCR-ABL1/ ABL. Followed by the first course of the Olverembatinib-based regimen, five patients with negative MRD were treated with HSCT successfully and 27 received the second course of Olverembatinib-based therapy, of whom 21 (77.78%) acquired negative MRD. For six patients with negative MRD of BCR-ABL1/ ABL1, Olverembatinib-based therapies could remain the negative MRD. These patients with negative MRD will receive allogeneic hematopoietic stem cell transplantation after Olverembatinib-based therapy. Olverembatinib-based therapies were generally well tolerated. Common hematologic adverse events included leukopenia, neutropenia, anemia, and thrombocytopenia. Common nonhematologic adverse events included hepatotoxicity, nephrotoxicity, myalgia, arthralgia, rash, and edema, of which most were mild. Conclusion Olverembatinib-based therapies are generally well tolerated and efficacious for patients with Ph/BCR-ABL1-positive acute lymphoblastic leukemia.

Objective: To investigate the safety and efficacy of a modified busulfan (Bu)/cyclophosphamide (Cy) conditioning regimen incorporating Selinexor for allogeneic hematopoietic stem cell transplantation in relapsed/refractory hematologic malignancies. Methods: We retrospectively evaluated the clinical outcomes of patients with hematologic malignancies who had received allo-HSCT with conditioning incorporating Selinexor at the Institute of Hematology and Blood Disease Hospital. Results: Between September 2022 and June 2023, 13 adult patients with high-risk hematologic malignancies were enrolled including 6 males and 7 females with a median age of 49 (13-58) years. There were 7 patients with acute myeloid leukemia (AML), 4 with myelodysplastic syndromes (MDS)/AML, 1 with multiple myeloma (MM) and 1 with extramedullary plasmacytoma. Six patients were in complete remission (CR), four in CR1, two in ≥CR2, five in non-remission (NR), and two patients were in a progressive disease status (PD) prior to conditioning. Three patients received matched-sibling donors transplant, while ten received related haploid hematopoietic stem cell transplantation. All patients had received myeloablative conditioning regimen incorporating Selinexor. Eleven patients received the conditioning: Bu (3.2mg/kg/d*2d); Melphalan (Mel, 60mg/m 2/d*2d), Fludarabine(Flu 30mg/m 2/d*3d) or CLA (5mg/m 2/d*3d), Cy (40mg/kg/d*2d), Selinexor 60mg/d*2d. The other 2 patients received the conditioning: Decitabine (DAC, 20mg/m 2/d*5d), Bu (3.2mg/kg/d*3d), Cy (40mg/kg/d *2d), Flu 30mg/m2/d*3d, Selinexor 60mg/d*2d. The median amount of infused mononuclear cell and CD34 + stem cell were 11.72×10 8/kg(range, 9.66 to 14) and 2.92×10 6/kg(range, 2.35 to 9.61), respectively. All the 13 patients achieved neutrophil engraftment with a median time of 12 (range, 9 to 18) days and 9 patients achieved platelet engraftment with a median time of 13 (range, 11 to 30) days. Four patients were still experiencing prolonged isolated thrombocytopenia. All patients achieved complete donor chimerism and minimal residual disease (MRD) negative at 30 days post-transplantation.The cumulative incidence of cytomegalovirus (CMV) reactivation was 15.4%, while Epstein-Barr virus (EBV) reactivation was not observed. Six patients (46.2%) experienced mucosal barrier injury, comprising three cases of oral mucosal ulcer, one case of digestive tract hemorrhage, and two cases of hemorrhagic cystitis. The cumulative incidence of II-IV and III-IV acute GVHD was 23.1% and 15.4%. The median follow-up for all the participants was 93 (40-316) days after transplantation. Only one patient was dead due to Thrombotic Microangiopathy (TMA) 78 days after transplant. One patient experienced molecular relapse 156 days after transplant, and another patient experienced hematology relapse 20 days after transplant. The two relapsed patients had received donor lymphocyte infusions after chemotherapy. The other 10 patients were still in CR with complete donor chimerism and MRD negative. The cumulative transplant-related mortality (TRM) and relapse rates are 7.7% (1 case) and 15.4% (2 cases), respectively. Conclusions: Incorporating Selinexor into the modified BU/CY conditioning regimen for allogeneic HSCT in patients with relapsed/refractory hematologic malignancies demonstrated a favorable safety profile. Ongoing prospective trials will provide valuable insights into the potential benefits of Selinexor in patients undergoing HSCT.

Background In 2022 European LeukemiaNet (ELN) recommendations for diagnosis and management of acute myeloid leukemia (AML), one of the most important changes made to the genetic risk classification of AML is that the FLT3-ITD allelic ratio (AR) is no longer considered in the risk classification; consequently, AML with FLT3-ITD (without adverse-risk genetic lesions) are now categorized in the intermediate-risk group, irrespective of the allelic ratio or concurrent presence of NPM1 mutation. Aims To investigate the survival outcomes of FLT3-ITD mutated AML patients underwent allogeneic hematopoietic stem cell transplantation (allo-HSCT), based on prognostic stratification defined by the 2017 and 2022 ELN guidelines, and to analyze the risk factors affecting transplant prognosis. Methods A total of 163 FLT3-ITD mutated AML patients underwent allo-HSCT were enrolled in this study. Bone marrow aspirates were obtained, cytogenetic and molecular characteristics were collected at the time of diagnosis. Genetic risk category was classified according to the 2017 and 2022 edition of the European LeukemiaNet (ELN) recommendations. HLA-haploidentical donor transplants were performed if no HLA matched sibling or unrelated donor was available. Patients received myeloablative conditioning with a modified Bu/Cy/Flu/Ara-c (busulfan/cyclophosphamide/fludarabine/cytarabine) or MCBC (melphalan/cladribine/busulfan/cyclophosphamide) preparative regimen. The therapeutic process and clinical outcomes were retrospectively analyzed. Results We assessed 163 adults with FLT3-mutated AML (median age 37 years) allografted between 2017 and 2022 in our clinical center. The median follow-up of alive patients was 24.9 months. The 2-year relapse-free survival (RFS) and overall survival (OS) were 67.2% and 71.9%, respectively. The 2-year incidences of relapse and non-relapse mortality were 27.9% and 12.5%, respectively. There was no significant difference in survival outcomes between the intermediate and high-risk groups after transplantation, both defined by the 2017 and 2022 ELN guidelines. High FLT3-ITD allelic ratio (≥0.5) at initial diagnosis was associated with elevated WBC counts ( P<0.001), but did not affect the response ratefollowing induction chemotherapy ( P=0.059) . Patients with high allelic ratio were more likely to benefit from maintenance therapy after transplantation ( P=0.048 for OS). In univariate analysis, presence of NPM1 mutation and the allelic ratio of FLT3-ITD did not affect any of the transplant outcomes. In multivariate analysis, MRD-negative at transplantation, occurrence of chronic GVHD and posttransplant maintenance therapy improved OS and RFS. Conclusion Allo-HSCT can overcome the poor prognosis in patients with FLT3-ITD mutated AML. Neither the presence of NPM1 mutation nor the FLT3-ITD allelic ratio affected survival. Prevention of disease relapse after transplantation remains a challenge. The main prognostic factors affecting transplant outcomes included pre-transplant MRD status and the application of maintenance therapy. Thus, introduction of FLT3 inhibitors as post-transplant maintenance therapy and the development of highly sensitive MRD assays will further reduce disease recurrence and improve survival.

Hepatitis-associated aplastic anemia (HAAA) is a rare variant of acquired aplastic anemia characterized with a syndrome of bone marrow failure after hepatitis. We retrospectively analyzed the outcomes of consecutive severe HAAA patients who received immunosuppressive therapy (IST, n = 70), matched-sibling donor hematopoietic stem cell transplantation (MSD-HSCT, n = 26) or haploidentical-donor (HID) HSCT (n = 11) as the first-line treatment. In the IST group, the hematologic response (HR) rate was 55.71% at 6 months. In contrast, HSCT recipients exhibited significantly more rapid and sustained hematopoiesis (HR 76.92%, 96.15% and 96.15% at 3, 6 and 12months, respectively). The 5-year overall survival (OS) was not different among IST (83.7 ± 4.9%), MSD-HSCT (93.3 ± 6.4%) and HID-HSCT group (80.8 ± 12.3%). Compared with IST, MSD and HID-HSCT demonstrated a trend of superiority in the estimated 5-year failure-free survival rates (93.3 ± 6.4% vs 64.3 ± 6.0%, p = 0.05; 80.8 ± 12.3% vs 64.3 ± 6.0%, p = 0.57). In subsequent stratified analysis on age, we found that HID-HSCT showed its efficacy and safety among young patients. In sum, MSD-HSCT remains first-line treatment choice for HAAA, whereas HID-HSCT represents an alternative treatment choice in addition to IST for young patients (< 40 years) without a matched sibling donor.

恶性血液病(HM)患者在omicron时期的感染率和死亡率高于其它人群.血液病疾病进展状态、高龄、基础病、造血干细胞移植(HSCT)和嵌合抗原受体T细胞治疗等均与HM新冠病毒感染患者的预后密切相关,而系统性抗肿瘤治疗(SACT)对患者的预后影响较小,建议在权衡患者血液病状态及新冠病毒感染严重程度的情况下决定是否延迟治疗或更换治疗方案.在预防新冠病毒感染方面,接种疫苗及加强针、加强个人防护是降低感染率和死亡率的重要方式,必要时可以注射Evusheld以增强抗感染能力.在治疗方面常采用抗病毒结合免疫调节的方式,但抗S单抗、激酶抑制剂、抗IL-6单抗、粒细胞集落刺激因子(G-CSF)、抗凝药等药物在HM患者中的有效性和安全性还需进一步评估.

SET-NUP214 fusion gene, also known as TAF-1-CAN and SET-CAN, is observed in acute myeloid leukemia (AML) and T-cell lymphoblastic leukemia (T-ALL). SET-NUP214 fusion in T-cell lymphoblastic leukemia is associated with chemotherapy resistance, but the prognosis of patients with AML with SET-NUP214 has rarely been reported. In the present study, we retrospectively analyzed all patients with acute leukemia including AML and T-ALL patients with SET-NUP214 fusion who underwent allogeneic stem cell transplantation (alloHSCT) in our center from July 2017 to November 2022. Of the total 11 patients, 5 patients were diagnosed with AML and 6 patients were diagnosed with T-ALL de novo. All patients received myeloablative regimens in CR1, and there were three (60%) AML patients who relapsed post-alloHSCT and three T-ALL (50%) patients who relapsed post-alloHSCT. Only one patient with AML who relapsed post-alloHSCT responded to subsequent chemotherapy plus donor lymphocyte infusion and survived the last follow-up. The estimated 1-year overall survival and 3-year overall survival for all these 11 patients were 69.3% and 38.5%, respectively. The estimated 1-year leukemia-free survival and 3-year leukemia-free survival for all patients were 69.3% and 38.5%, respectively. The research shows a high incidence of relapse for patients with acute leukemia with the SET-NUP214 fusion gene, even after alloHSCT. More clinical trials or research with larger samples are urgently needed for this group of patients.

•Long-term antibiotic use linked to multidrug-resistant infections.•Bloodstream infection prognosis is closely related to time to effective treatment.•The use of antibiotic prophylaxis in patients with neutropenia need to be weighed.•There are still many drugs with in vitro activity against CR P. aeruginosa.

Objectives To explore the role and dynamics of MAIT (Mucosal-associated invariant T) cells in blood stream infection of AML (Acute myeloid leukemia) patients. Methods Healthy adults and hospitalized adult AML patients that with blood stream infection, non-infection, or in completely remission (CR) were prospectively enrolled. Characteristics of MAIT cells in peripheral blood of enrolled subjects were analyzed by flow cytometry. Results Both gram negative and gram positive bacteria could induce sepsis in AML patients. MAIT cells significantly increased in AML patients with blood stream infection comparing with non-infected AML patients, healthy subjects and AML patients in completely remission. Increased MAIT cells were mainly consists of CD8+ T cells and were activated. There is no significant difference in the recruitment and death of MAIT cells, while their proliferation is significantly increased. The proliferation of MAIT cells occurs before traditional infection indicators such as CRP, PCT, endotoxin in plasma and fever. MAIT cells in neutropenic blood stream infected mice were increased as we observed in AML patients. Conclusions MAIT cells increased without bacterial specificity, and were activated and proliferated in AML patients with blood stream infection that caused by various bacteria. They may enhance host defense and play a vital role in fighting against pathogenic microbial infections. Proliferation of MAIT cells can be applied as a monitoring indicator for blood stream infection in AML patients, which helps to reduce the mortality rate of them.