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

Hemophilia is a X-linked recessive hemorrhagic disease. Bleeding is the most common complication of hemophilia, and it is also the main cause leading to death and disability or reducing the quality of life of hemophilia patients. Rapid identification and standardized management of the bleeding events is of great significance to improve the prognosis of hemophilia patients. Emergency department is the frontline department for hemophilia patients with bleeding. The emergent management process of hemophilia hemorrhage is complex and often needs multidisciplinary team cooperation. To increase the awareness of the related professionals who may involve in the management of bleeding events of hemophilia patients, in collaboration with the Thrombosis and Hemostasis Group, Chinese Society of Hematology, Chinese Medical Association, Hemophilia Treatment Center Collaborative Network of China issued the Chinese guidelines for emergency management of bleeding in hemophilia patients.

Congenital dysfibrinogenemia (CDF) is the most common type of congenital fibrinogen disorders, characterized by dysfunctional fibrinogen. Its prevalence is significantly underestimated due to the absence of obvious clinical symptoms in most patients. In addition to bleeding manifestations, patients with CDF may experience thrombotic events or pregnancy-related complications. Fibrinogen antigen assays and molecular heritability analyses can help differentiate CDF from other types of congenital fibrinogen disorders. The clinical presentation of CDF varies significantly among individuals, and there is a lack of routine laboratory methods to effectively predict the risk of bleeding or thrombosis in these patients, in addition to their personal and family histories. This poses challenges in the clinical management of patients with CDF, particularly during the perioperative period or pregnancy. Further registry-based and prospective studies are needed to improve our understanding of this disease and guide clinical management.

Multiple myeloma (MM) is a malignant plasma cell disease that currently cannot be cured. Several new drugs have continuously been introduced in the recent years. New drugs targeting B-cell maturation antigen (BCMA) have greatly improved the efficacy and prognosis of MM compared with traditional treatments. This article reports the case of an IgD type relapsed and refractory MM patient with poor efficacy of BCMA×CD3 bispecific antibody. The patient achieved deep remission after receiving BCMA-targeted CAR-T cell therapy after initial seven lines of treatment. Literature review was also conducted to improve the clinical physicians' understanding of BCMA target therapy for relapsed and refractory MM patients.

Objectives: To analyze the value of the WT1 mRNA expression level in the diagnosis and prognostic evaluation of myelodysplastic syndrome (MDS) . Methods: A total of 403 patients with MDS, suspected MDS, and acute myeloid leukemia secondary to MDS (AML-MDS) from eight clinical trial centers in China were included in this multicenter, prospective study. Nucleic acid was extracted from the peripheral blood (PB) and bone marrow (BM) samples and WT1 mRNA expression was measured using the WT1 mRNA assay kit. Results: A good correlation (r=0.778) was observed between the expression levels of WT1 mRNA in PB and BM. The expression levels of WT1 mRNA in both PB and BM increased with increasing FAB (French-American-British) or WHO (2008) (world health organization) classification scores, and increasing IPSS-R or WPSS-R prognostic scores. A statistically significant difference was observed in the expression levels of WT1 mRNA in PB and BM between MDS and AML-MDS patients (PB: 3.11±0.98 vs 4.57±0.53, P<0.05; BM: 3.73±0.93 vs 4.92±0.81, P<0.05). A statistically significant difference also existed in the expression levels of WT1 mRNA in PB and BM between the IPSS-R relatively low-risk group (extremely low-risk + low-risk) and the relatively high-risk group (medium risk + high risk + extremely high risk) MDS patients (PB: 2.60±0.76 vs 3.48±0.91, P<0.05; BM: 3.50±0.82 vs 3.89±0.97, P<0.05). Statistically significant differences were observed in the WT1 mRNA expression levels between the IPSS-R low-risk group (extremely low-risk + low-risk + moderate risk) and the high-risk group (high-risk + extremely high-risk) MDS patients in PB and BM (PB: 2.82±0.89 vs 3.61±0.85, P<0.05; BM: 3.61±0.84 vs 3.92±1.05, P<0.05). Statistically significant differences were also observed in the expression levels of WT1 mRNA in PB and BM of MDS patients between the WPSS-R relatively low-risk group (extremely low-risk + low-risk + moderate risk) and the relatively high-risk group (high-risk + extremely high-risk) (PB: 2.56±0.79 vs 3.61±0.82, P<0.05; BM: 3.45±0.83 vs 3.93±1.00, P<0.05) . Conclusion: A good correlation was observed between the expression levels of WT1 mRNA in PB and BM specimens of MDS patients, and the expression level of WT1 mRNA is related to the disease risk of MDS.

The onset of myasthenia gravis (MG) after allogeneic hematopoietic stem cell transplantation (allo-HSCT) seriously threatens the survival of patients, since it is acute, and is prone to rapid progression. Two patients with acute myeloid leukemia (AML), who had undergone allo-HSCT developed shortness of breath, and gradually developed cervical weakness and dyspnea. The acetylcholine receptor (AChR) antibody and neostigmine test enabled the diagnosis of MG. The condition of the patients improved after treatment with pyridostigmine bromide, glucocorticoids and rituximab.

Objective: To explore the clinical characteristics and prognosis of children with chronic myeloid leukemia in the blast phase (CML-BP) . Methods: The clinical characteristics, treatment measures, and survival outcomes of 28 children with CML-BP were analyzed in our hospital from January 2008 to November 2022. Results: The male to female ratio of the 28 children with CML-BP was 1.15∶1. The median age of diagnosis of CML-BP was 10 years, and the median follow-up time was 79 months. During the diagnosis of CML, four children were in the BP, one was in the accelerated phase (AP) and 23 children were in the chronic phase (CP). Among the 23 children with CML-CP, 75% had progressed directly from CP to BP without experiencing the AP. Among the children diagnosed with CML-BP, 71.4% were classified as chronic myeloid leukemia lymphoid blast phase (CML-LBP), 25.0% belonged to the chronic myeloid leukemia myeloid blast phase (CML-MBP), and 3.6% belonged to the chronic myeloid leukemia mixed phenotype acute leukemia (CML-MPAL). Treatment with hemaopoietic stem cell transplantation (HSCT) after tyosine kinase inhibitor (TKI) combined with chemotherapy was administered to 19 children, two children received HSCT after TKI alone, and seven children received TKI combined with chemotherapy but without HSCT. The 5-year overall survival of the 28 children with CML-BP was 59.3%. Conclusion: The direct progression of BP from CP is greater in children with CML-BP compared with adults, and the overall prognosis of children with CML-BP is poor.

Objective: To investigate the prognosis of newly diagnosed multiple myeloma (MM) patients with t (14;16) abnormality. Methods: Clinical data from 564 patients diagnosed with initial MM from January 2018 to November 2020 at Beijing Chaoyang Hospital affiliated with Capital Medical University and the First Affiliated Hospital of Sun Yat-sen University were collected and retrospectively analyzed. The prognoses of patients with t (14;16) were analyzed and compared with the prognoses of patients with normal FISH, and those with t (4;14) and del (17p) . Results: Among 564 newly diagnosed MM patients, 19 (3.4%) exhibited t (14;16) abnormalities, with 14 cases diagnosed with 1q21+ and three cases with del (17p). Progression-free survival (PFS) and overall survival (OS) of patients with t (14;16) were significantly shorter compared with patients with normal FISH (the median PFS: 14 months vs not reached, P<0.001; the median OS: 42 months vs not reached, P=0.002). No statistically significant difference was detected in PFS and OS between the 15 patients with t (14;16) and the 15 with t (4;14) after propensity score matching (the median PFS: 13.0 months vs not reached, P=0.247; the median OS: 42 months vs not reached, P=0.609). Similarly, no statistically significant difference was observed in PFS and OS between 15 patients with t (14;16) and 15 with del (17p) (the median PFS: 13 months vs 31 months, P=0.939; the median OS: 42 months vs 37.3 months, P=0.557). Propensity score matching indicated that when combined with 1q21+, no statistically significant differences were present in PFS and OS between patients with t (14;16) and patients with t (4;14) or patients with del (17p) (all P>0.05). Whether or not the patients with t (14;16) had undergone auto-HSCT did not significantly impact the PFS and OS (all P>0.05) . Conclusion: t (14;16) is often associated with high-risk cytogenetic abnormalities in newly diagnosed MM patients, and its adverse prognostic value is similar to that of t (4;14) and del (17p) .

Objective: To analyze the distribution and drug resistance of pathogens of bacterial bloodstream infection in patients with hematological diseases in the Department of Hematology of Tianjin Medical University General Hospital, and to provide etiological data for clinical empirical anti-infection treatment. Methods: A retrospective analysis was conducted on the general clinical information, pathogenic bacteria and drug susceptibility test results of patients with hematological diseases diagnosed with bacterial bloodstream infection by menstrual blood culture in our center from January 2016 to December 2022. Results: Patients included 498 inpatients, with a total of 639 bacterial strains. Among the patients, 86.9% patients had malignancies, and 76.7% had agranulocytosis. Symptoms of concurrent infections, including those of the respiratory tract, oral mucosa, skin and soft tissues, and abdominal sources were observed in 68.3% patients. Gram-negative bacteria (G(-)) accounted for 79.0% of the isolated bacteria, and gram-positive bacteria (G(+)) accounted for 21.0%. The top five isolated pathogens were Klebsiella pneumoniae (22.5%), Escherichia coli (20.8%), Pseudomonas aeruginosa (15.0%), Enterococcus faecium (5.5%), and Stenotrophomonas maltophilum (5.0%). Escherichia coli exhibited a decreasing trend of resistance to quinolones, cephalosporins, and carbapenems. Klebsiella pneumoniae exhibited increasing rates of resistance to quinolones and cephalosporins between 2016 and 2018, but the rated decreased after 2019. The resistance rate to carbapenems exhibited by Pseudomonas aeruginosa was approximately 20%. Carbapenem-resistant strains of Pseudomonas aeruginosa strains were first detected in 2017, with a peak resistance rate of 35.7%, detected in 2019. A 60.0% resistance rate to methicillin was observed in methicillin-resistant coagulase-negative staphylococci (MRCNS), and one case of linezolid-resistant MRCNS was detected. Conclusions: Pathogenic bacteria of bacterial bloodstream infections were widely distributed in our center, and precautions are warranted against carbapenem resistant P. aeruginosa and Klebsiella pneumoniae.