Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi最新文献

Data and guidelines for a second attempt at treatment-free remission (TFR) in patients with chronic myeloid leukemia in the chronic phase (CML-CP) are limited. We retrospectively analyzed the clinical data of six patients with CML-CP who attempted a second discontinuation of tyrosine kinase inhibitors (TKIs) for TFR at Peking University People's Hospital from October 2006 to October 2024. Before the first TKI discontinuation, all six patients had received imatinib treatment for a median of 82.5 months (range, 40-87 months) and maintained a sustained deep molecular response, specifically MR(4.5), for a median of 34.5 months (range, 24-62 months). They lost major molecular response (MMR) at a median of 3 months (range, 2-4 months) after discontinuing imatinib treatment. All six patients resumed TKI treatment within 4 weeks following failure of the first TFR (TFR1). After a median of 4.5 months (range, 3-7 months) on TKI, they achieved MR(4.5) again and voluntarily attempted a second TKI discontinuation at 93, 84, 76, 76, 33, and 44 months, respectively, after resuming therapy. Four out of six patients maintained TFR, with sustained TFR durations of 42, 23, 21, and 58 months, respectively. All four patients who had resumed TKI treatment for >6 years and maintained MR(4.5) for >5 years achieved a sustained second TFR of ≥21 months; among these, three had received second-generation TKI for >1 year during the resumption of TKI treatment. The other two patients lost MMR at 4 and 3 months, respectively, after the second TKI discontinuation but regained MR(4.5) at 2 and 3 months, respectively, after resuming TKI treatment again.

Human adenovirus (HAdV) hepatitis is a rare but rapidly progressive and often fatal complication following allogeneic hematopoietic stem cell transplantation. The present study reported the management of two patients with lymphoproliferative disease who developed HAdV infection after haploidentical hematopoietic stem cell transplantation and subsequently died of liver failure. These cases highlight the importance of early multi-site screening, dynamic monitoring, and timely intervention for HAdV in high-risk patients to reduce mortality risk and improve prognosis.

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired clonal disorder of hematopoietic stem cells with diverse clinical manifestations, making it susceptible to missed diagnosis or misdiagnosis. Early clinical screening through multidisciplinary collaboration is therefore essential. Treatment strategies should be guided by precise clinical classification. For patients with classic PNH, complement inhibitors are the first-line treatment, whereas for patients with bone marrow failure, the primary treatment should be focused on the underlying bone marrow failure. In cases accompanied by hemolysis, a combination of immunosuppressants and complement inhibitors can be used. During treatment with complement inhibitors, close monitoring of treatment efficacy and complications is required. Based on three representative cases admitted to our hospital, this article highlights the importance of early screening, summarizes key points for standardized diagnosis and treatment of PNH, and provides recommendations for managing breakthrough hemolysis and extravascular hemolysis, with the aim of improving clinical practices.

We retrospectively analyzed the RUNX1 mutation status and clinical characteristics of 323 newly diagnosed acute myeloid leukemia (AML) patients treated in the Second Affiliated Hospital of Anhui Medical University from February 2018 to May 2023. The mutation rate of RUNX1 in AML patients was 11.5% with a median mutation frequency of 40.4%. RUNX1 mutations often coexisted with other mutations, such as ASXL1, but were mutually exclusive with NPM1 mutations. AML patients with RUNX1 mutations were older (median age: 65 vs 55 years, P<0.01), had a lower complete remission rate (39.4% vs 79.8% P<0.01), higher relapse (75.0% vs 48.8%, P<0.05) and mortality (91.4% vs 59.5%, P<0.01) rates, shorter median overall survival (OS) (5.9 vs 20.1 months, P<0.01) and relapse-free survival (RFS) (9.5 vs 46.5 months, P<0.01). A high RUNX1 mutation frequency was associated with increased mortality (100.0% vs 76.5%, P<0.05) and shorter RFS (7.2 vs 12.2 months, P<0.05). Different induction therapies (chemotherapy/low-intensity treatment) showed no significant impact on the efficacy or prognosis of patients with RUNX1 mutations (P>0.05). Patients with concurrent RUNX1 and DNMT3A or ASXL1 mutations portend a poorer prognosis, and the cooccurrence of FLT3-ITD mutation further leads to inferior therapeutic outcomes.

Objective: To compare the long-term efficacy and safety of the CCCG-ALL-2015 and CCLG-ALL-2008 protocols in treating adolescents and older children (aged ≥10 years) with acute lymphoblastic leukemia (ALL) and explore the key factors affecting prognosis. Methods: A retrospective analysis was conducted on the clinical data of patients aged 10-18 years who were admitted to the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences between January 2008 and October 2020. The patients were treated with either the CCCG-ALL-2015 (Group Ⅰ) protocol or the CCLG-ALL-2008 (Group Ⅱ) protocol. The Kaplan-Meier analysis was used to evaluate the overall survival (OS) and event-free survival (EFS) rates, and for univariate analysis. Multivariate analysis was performed using the Cox proportional-hazards regression model. Results: Altogether, 355 patients were enrolled, including 210 in Group Ⅰ and 145 in Group Ⅱ. There were 214 men and 141 women; 286 and 69 had B-ALL and T-ALL, respectively. The complete remission rate after induction therapy in Group Ⅰ was 99.5%, which was higher than the in Group Ⅱ at 91.7% (P<0.001, χ(2)=14.596). The median follow-up time was 84 (range: 2-206) months. A total of 89 patients (25.1%) relapsed, and 73 (20.6%) died. The predicted 10-year EFS and OS rates for all 355 patients were (65.9 ± 2.6) % and (79.1 ± 2.2) %, respectively. The predicted 10-year EFS and OS rates for Group Ⅰ were (68.3 ± 3.3) % and (80.7 ± 2.7) %, respectively, whereas those for Group Ⅱ were (62.6 ± 4.0) % and (76.8 ± 3.5) %, respectively, showing no significant difference (P(EFS)=0.201, P(OS)=0.305). The rates of infectious events (54.3% vs 69.7%, P=0.004) and asparaginase allergy (0.9% vs 9.0%, P<0.001) were significantly lower in Group Ⅰ than in Group Ⅱ. Multivariate analysis identified an initial WBC ≥50×10(9)/L (HR=1.583, 95% CI:1.060-2.365, P=0.025) and minimal residual disease (MRD) value ≥0.01% at the end of induction therapy (HR=1.300, 95% CI: 1.174-1.439, P<0.001) as the independent risk factors for EFS. MLL rearrangement (HR=3.144, 95%CI: 1.093-9.046, P=0.034) and MRD value ≥0.01% at the end of the induction therapy (HR=1.310, 95%CI: 1.152-1.488, P<0.001) were the independent risk factors for OS. Conclusion: By incorporating MRD-guided dynamic risk stratification and optimized drug regimens, the CCCG-ALL-2015 protocol significantly improved the quality of induction remission, reduced adverse events, and improved the long-term survival in adolescents (aged 10-18 years) with ALL. The MRD status at the end of induction therapy is a critical prognostic indicator and provides important guidance for individualized therapy.

Acute graft-versus-host disease (aGVHD) remains a severe complication following allogeneic hematopoietic stem cell transplantation, and traditional corticosteroid therapy often demonstrates limited efficacy. This review highlights the recent advances in cellular and targeted immunotherapies for aGVHD. Mesenchymal stem cells show considerable efficacy in steroid-resistant aGVHD and are already in clinical use. Regulatory T cells (Treg), including CAR-engineered Tregs, effectively induce immune tolerance and reduce GVHD incidence. Targeted agents, such as CTLA-4 fusion proteins and α4β7 integrin antibodies, mitigate severe aGVHD by blocking immune activation or T-cell homing. Additionally, tissue-repair factors (e.g., IL-22, GLP-2 analogs) and immunomodulatory molecules (e.g., α1-antitrypsin) offer novel strategies that combine tissue protection with immunomodulation. Collectively, these innovative therapies are driving aGVHD treatment toward precision, high efficacy, and low toxicity, demonstrating a promising clinical potential.

Objective: To evaluate the efficacy of haploidentical hematopoietic stem cell transplantation (haplo-HSCT) in advanced myelodysplastic syndrome (MDS) patients with recurrent genetic abnormalities. Methods: We conducted a retrospective analysis of the clinical characteristics and prognosis of 14 advanced MDS patients with recurrent genetic abnormalities who underwent haplo-HSCT in the Hematology Department of Peking University People's Hospital from January 2015 to December 2020. The present study employed a 1∶4 propensity score matching method to select 56 contemporaneous advanced MDS patients without recurrent genetic abnormalities as the control group. The matching factors included age and disease stage (EB-1 or EB-2) . Results: The 5-year overall survival and disease-free survival after haplo-HSCT for MDS patients with and without recurrent genetic abnormalities were (64.3± 12.8) % vs (66.6±6.6) % (χ(2)=0.044, P=0.833) and (57.1±13.2) % vs (59.8±6.7) % (χ(2)=0.031, P=0.861) respectively. The 5-year cumulative non-relapse mortality rates after haplo-HSCT for MDS patients with and without recurrent genetic abnormalities were (21.4±11.5) % and (21.4±5.5) % (χ(2)=0.004, P=0.952), respectively; the relapse rates were (21.4±11.5) % and (16.1±5.0) % (χ(2)=0.096, P=0.757), respectively. Conclusion: These results suggest that the prognosis of MDS patients with recurrent genetic abnormalities is comparable to that of MDS patients without these abnormalities after haplo-HSCT. Haplo-HSCT may be an effective therapeutic option for advanced MDS patients with recurrent genetic abnormalities.

Genetic abnormalities are a cornerstone for prognostic assessment in multiple myeloma (MM) and form the basis of contemporary risk-stratification systems. In routine clinical practice, cytogenetic karyotyping, fluorescence in situ hybridization, and next-generation sequencing should be performed in a standardized manner to identify high-risk patients and guide individualized therapy. With recent advances in genomic technologies, such as whole-genome sequencing and single-cell sequencing, our understanding of clonal evolution and genetic heterogeneity in MM has deepened. Meanwhile, the use of triplet and quadruplet combination regimens has altered the prognostic impact of certain genetic lesions, highlighting the need to update the existing risk frameworks. To standardize genetic testing for MM in China, the Multiple Myeloma Expert Committee of the Chinese Society of Clinical Oncology (CSCO) and Myeloma & Plasma Cell Disease Group, Hematology Oncology Committee of the China Anti-Cancer Association (CACA) have revised the 2019 consensus based on the latest evidence. This updated consensus focuses on refining the testing workflows, harmonizing the interpretation and reporting of high-risk genetic abnormalities, and discussing the incorporation of emerging molecular markers into risk stratification. This new consensus aims to promote standardized and precision-based MM care in China and improve the identification and management of high-risk patients.

To analyze the clinical characteristics of post-allogeneic hematopoietic stem cell transplantation (allo-HSCT) lymphoproliferative disorders and to explore the clinical guidance provided by the latest nomenclature for post-transplant lymphoproliferative disorders (PTLD) in the 2022 World Health Organization Classification of Hematologic and Lymphoid Tumors (WHO-HAEM5). A retrospective analysis was conducted on the clinical data of 35 patients diagnosed with PTLD after allo-HSCT at the Department of Hematology, Fifth Medical Center, Chinese PLA General Hospital, from January 1, 2016, to December 15, 2024. Among these, 11 cases were clinically diagnosed, and 24 cases were pathologically confirmed. The 24 pathologically confirmed PTLD cases were renamed according to the latest WHO-HAEM5 nomenclature. Monomorphic PTLD, corresponding to the lymphoma subtype of PTLD, exhibited the poorest prognosis with a over survival rate of only 54.5% (6/11). All deceased patients had severe aplastic anemia (SAA) as the primary disease; one death resulted from chemotherapy-related complications, while the remaining four died due to explosive PTLD progression. PTLD patients with SAA as the primary diagnosis exhibit poor prognosis, necessitating more aggressive clinical intervention strategies.

Objective: This study aimed to investigate the clinical characteristics and prognosis of primary immune thrombocytopenia (ITP) complicated with diffuse alveolar hemorrhage (DAH) . Methods: This study retrospectively analyzed the clinical data of five patients with ITP complicated with DAH admitted to Peking University People's Hospital from January 2015 to June 2024. Basic characteristics, clinical symptoms, laboratory tests, imaging findings, and treatment responses were summarized, and survival and prognostic factors were evaluated. Results: All patients were female, with a median age of 56 (38-66) years. Four cases were diagnosed with chronic ITP, and three cases were complicated by infection. The main symptoms included mucocutaneous bleeding (3/5), dyspnea (3/5), and hemoptysis (2/5). The median PLT was 51 (1-83) ×10(9)/L. D-dimer was significantly increased [median 870 (220-8 578) ng/ml]. Chest computed tomography revealed diffuse ground-glass opacities in both lungs (3/5), consolidation (1/5), and air bronchogram (1/5). Three patients achieved complete remission, whereas two patients died. Conclusion: ITP complicated by DAH mainly manifests as acute respiratory failure. Increased D-dimer and failure to control infection are high-risk factors for death. Therefore, early combined immunosuppression and anti-infection treatment improves prognosis.