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

{"title":"[The Chinese clinical expert consensus for the BCMA bispecific T cell engager in the treatment of multiple myeloma (2025)].","authors":"","doi":"10.3760/cma.j.cn121090-20250202-00050","DOIUrl":"10.3760/cma.j.cn121090-20250202-00050","url":null,"abstract":"<p><p>Multiple myeloma is an incurable disease. The bispecific T cell engager, one of the immune therapies targeting tumor cell surface antigens, has shown promising clinical efficacy. However, bispecific T cell engager therapy has a unique anti-myeloma mechanism and adverse effects. To further standardize the clinical application of B cell maturation antigen bispecific T cell engager, the Chinese Myeloma Committee of the Chinese Hematology Association, the Plasma Cell Disease Group, Chinese Society of Hematology, Chinese Medical Association and the Society of Hematology of Chinese Geriatrics Association organized experts to formulate the expert consensus by referring to recent domestic and international guidelines, consensus, research progress, and clinical practice, to guide clinical applications better.</p>","PeriodicalId":24016,"journal":{"name":"Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi","volume":"46 3","pages":"209-215"},"PeriodicalIF":0.0,"publicationDate":"2025-03-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12038466/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144050665","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"[Analysis of two cases of hereditary protein C deficiency causing venous thrombosis].","authors":"M Z Wen, Y F Lu, M N Liu, L Y Qin, Y H Jin, M S Wang, L L Yang","doi":"10.3760/cma.j.cn121090-20240628-00236","DOIUrl":"10.3760/cma.j.cn121090-20240628-00236","url":null,"abstract":"<p><p><b>Objective:</b> To investigate the molecular pathogenic mechanism of venous thrombosis caused by heterozygous missense mutations in two protein C (PROC) genes through laboratory phenotype analysis, genetic mutation analysis, and in vitro expression experiments. <b>Methods:</b> Two probands presented with venous thromboembolism at the First Affiliated Hospital of Wenzhou Medical University. Clinical data and blood samples were collected from the probands and their family members to evaluate the plasma protein C (PC) activity (PC∶A), PC antigen (PC∶Ag) levels, and other relevant coagulation parameters. The anticoagulant capacity was assessed using the thrombin generation test (TGT). The mutation sites of the PROC gene were identified using direct DNA sequencing. Bioinformatics software was used to analyze the conservation and pathogenicity of the mutated gene. PyMOL software was used for the analysis of the protein three-dimensional models and interactions between mutated amino acids. Wild-type and two mutant expression vectors were constructed and HEK293T cells were transiently transfected. Total cellular RNA was extracted from positively transfected cells to investigate the transcriptional levels of the mutant PROC gene. Enzyme-linked immunosorbent assay, Western blot, and cellular immunofluorescence assays were used to investigate the translation levels of the mutant PROC protein. <b>Results:</b> Probands 1 and 2 exhibited PC∶A levels of 35% and 40% and PC∶Ag levels of 44% and 39%, with increasing D-dimer levels to 4.42 mg/L and 0.83 mg/L, respectively. Meanwhile, other coagulation parameters revealed no significant abnormalities. TGT demonstrated impaired anticoagulant function in both proband witnesses and their familial PC carriers. Sequencing analysis revealed heterozygous missense mutations c. 833T>C (p. Leu278Pro) in proband 1 and c. 1330T>C (p. Trp444Arg) in proband 2 within exon 9 of the PROC gene. Conservation analysis revealed that Leu278 and Trp444 were highly conserved across homologous species. Pathogenicity analysis indicated that both p. Leu278Pro and p. Trp444Arg mutations are deleterious. Protein modeling analysis demonstrated that both mutations induce structural alterations in the protein. In vitro expression experiments revealed that compared with the wild-type, both p. Leu278Pro and p. Trp444Arg mutations showed no significant differences in the mRNA expression level of the PC protein. However, both mutations caused significantly lower PC∶Ag content and protein expression levels in the cell culture supernatant compared with the wild-type, whereas higher levels were observed in the cell culture lysate. This indicates the association of both mutations with the secretion function of the PC protein. <b>Conclusion:</b> The heterozygous missense mutations p. Leu278Pro and p. Trp444Arg in exon 9 of the PROC gene in both probands are associated with decreased PC levels.</p>","PeriodicalId":24016,"journal":{"name":"Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi","volume":"46 3","pages":"244-251"},"PeriodicalIF":0.0,"publicationDate":"2025-03-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12038477/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144048068","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"[Clinical analysis of 16 cases of adult acute B-lymphoblastic leukemia treated with blinatumomab].","authors":"Z Y Liu, S J Zhang, Z Y Yan, H M Sun, Y B Chen","doi":"10.3760/cma.j.cn121090-20240611-00219","DOIUrl":"10.3760/cma.j.cn121090-20240611-00219","url":null,"abstract":"<p><p>This study aimed to investigate the efficacy and safety of blinatumomab in adult patients with acute B-lymphoblastic leukemia (B-ALL) by conducting a retrospective analysis of the clinical data from 16 patients with B-ALL receiving blinatumomab at the Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, from June 2022 to April 2024. Among the 16 patients, 10 were classified as relapsed/refractory B-ALL and 6 were newly diagnosed Ph(-) B-ALL. Of the 10 patients with relapsed/refractory B-ALL, 8 achieved complete remission (CR) and minimal residual disease (MRD) negativity after one blinatumomab treatment cycle. In the 6 newly diagnosed patients, the bone marrow MRD was negative after one blinatumomab treatment cycle after initial induction chemotherapy followed by sequential blinatumomab treatment. Among them, four completed allogeneic hematopoietic stem cell transplantation and continuously maintained CR. This indicates that blinatumomab exhibits a high remission rate in both patients with relapsed/refractory and newly diagnosed B-ALL, thereby providing the possibility of bridging to transplantation and extending patient survival, with manageable adverse reactions.</p>","PeriodicalId":24016,"journal":{"name":"Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi","volume":"46 3","pages":"269-272"},"PeriodicalIF":0.0,"publicationDate":"2025-03-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12038468/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144052503","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"[Differences in clinical and laboratory features and survival between Chinese and Western patients with myelodysplastic neoplasm].","authors":"L L Liu, B Li, T J Qin, Z F Xu, S Q Qu, L J Pan, Q Y Gao, M Jiao, Y J Ja, C W Li, Q Sun, H J Wang, Z J Xiao","doi":"10.3760/cma.j.cn121090-20241210-00555","DOIUrl":"10.3760/cma.j.cn121090-20241210-00555","url":null,"abstract":"<p><p><b>Objective:</b> To compare the clinical and laboratory characteristics and survival between Chinese and Western patients with myelodysplastic neoplasms (MDS) . <b>Methods:</b> Clinical and laboratory data were collected from 1,464 primary adult patients diagnosed with MDS at the Institute of Hematology & Blood Diseases Hospital from August 2016 to June 2024. Collected data were retrospectively analyzed and compared with 2,191 patients from the International Working Group for the Prognosis of Myelodysplastic Syndromes (IWG-PM) . <b>Results:</b> Chinese patients were significantly younger (median age: 56 years <i>vs</i>. 72 years, <i>P</i><0.001) and experienced more severe hematopenia (<i>P</i><0.001) compared with patients from the IWG-PM. Further, Chinese patients exhibited a higher percentage of isolated del (20q), +8, and complex karyotypes as well as a lower percentage of normal karyotypes, del (5q), and -Y (<i>P</i><0.001). Higher U2AF1, NRAS, and NPM1 mutation rates and lower ASXL1, SF3B1, and RUNX1 mutation rates were observed in Chinese patients than in participants from the IWG-PM (<i>P</i><0.05). No significant difference in overall survival (OS) was found between the two groups (median OS: 48 [95% <i>CI</i>: 40 - 56]months, <i>vs</i>. 45[95% <i>CI</i>: 40 - 49] months; <i>P</i>=0.449). Among participants aged ≤45 years, Chinese patients demonstrated more trisomy 8 (<i>P</i>=0.070) and U2AF1 mutation (<i>P</i><0.001) and higher 4-year OS rate compared with those from the IWG-PM (75.5% <i>vs</i>. 62.1%, <i>P</i>=0.001). Among participants aged ≥70 years, Chinese patients exhibited more complex karyotypes but fewer del (5q) as well as more NPM1 but less SF3B1 and TET2 compared with those from the IWG-PM (<i>P</i><0.05). Chinese patients demonstrated shorter survival (median OS: 20 [95% <i>CI</i>: 13 - 27] months <i>vs</i>. 37 [95% <i>CI</i>: 32 - 42] months, <i>P</i><0.001) . <b>Conclusion:</b> Chinese and Western MDS patients differ in age of onset, clinical features, and cytogenetic or molecular genetic abnormalities, with significant differences persisting in age-matched groups. Although the OS is similar, disparities exist in survival for younger and older patients between the two populations.</p>","PeriodicalId":24016,"journal":{"name":"Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi","volume":"46 3","pages":"223-230"},"PeriodicalIF":0.0,"publicationDate":"2025-03-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12038469/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144049910","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"[Application of next-generation sequencing technology for the investigation of immunoglobulin variable region characteristics and their prognostic significance in patients with chronic lymphocytic leukemia].","authors":"Z Guo, H M Jin, T L Qiu, L Y Zhu, Y J Wu, H R Qiu, Y Wang, Y Miao, H Jin, L Fan, J Y Li, Y Xia, C Qiao","doi":"10.3760/cma.j.cn121090-20240819-00310","DOIUrl":"10.3760/cma.j.cn121090-20240819-00310","url":null,"abstract":"<p><p><b>Objective:</b> To elucidate the genomic characteristics of the immunoglobulin (IG) heavy-chain variable region and light-chain variable region, the expression of subclones, and the prognostic significance in patients with CLL. <b>Methods:</b> Blood and/or bone marrow specimens were gathered from a cohort of 36 patients with CLL diagnosed at Jiangsu Province Hospital from December 2018 to May 2023, including 12 cases of B cell receptor (BCR) stereotyped patients. IG heavy-chain (IGH) and light-chain (IG Kappa [IGK] and IG lambda [IGL]) gene rearrangements were performed using next-generation sequencing (NGS) technology to analyze the characteristics and prognostic value in CLL. <b>Results:</b> NGS detection of IG variable region (IGHV) demonstrated a significant correlation and superior consistency with Sanger sequencing (<i>r</i>=0.957, <i>P</i> < 0.001). Among the 36 patients, the IGH variant (IGHV) was observed in 9 (25.0%) but not in 27 (75.0%) participants. The incidence of the MYD88 mutation was higher among patients with mutated IGHV [1/27 (3.7%) <i>vs</i> 4/9 (44.4%), <i>P</i>=0.00]. A high incidence of trisomy 12 was observed in the IGHV #8/#8B subset [4/11 (36.4%) <i>vs</i> 1/25 (4.0%), <i>P</i>=0.023], which were more likely to develop Richter transformation [8/11 (72.7%) <i>vs</i> 4/25 (16.0%), <i>P</i>=0.002]. In the patient cohort, 36 individuals (36/36, 100.0%) used the IGK variable, whereas 15 individuals (15/36, 41.7%) employed the IGL variable (IGLV). IGLV3 - 21 reported the highest utilization rate in IGLV (5/15, 33.3%). Remarkably, patients with CLL with IGLV3-21 fragments were exclusively observed in the Binet C stage and Rai Phase Ⅲ-Ⅳ, with an incidence of del (13) (q14) at 60.0% (3/5). The median time to first treatment (TTFT) of patients with or without IGLV3 - 21 fragments was 5.2 (1.1 - 41.5) and 9.9 (0.1 - 94.4) months, respectively. Using the total reads threshold of 2.5%, 4 (4/36, 11.1%) samples were detected to have two IGHV productive clones. The median TTFT and overall survival (OS) time were 2.8 (0.9-72.7) and 12.8 months in patients with one mutated clone and 57.5 (32.0-120.7) and 51.8 months in those with two mutated clones, respectively. The median TTFT and OS time were 10.9 (0.3-94.4) and 6.3 (0.1 - 12.5) months in patients with one unmutated clone and 49.9 (22.2 - 211.1) and 30.0 (9.6 - 50.3) months in those with multiple unmutated clones, respectively (<i>P</i>>0.05) . <b>Conclusions:</b> Detection of IG gene rearrangements using NGS technology not only facilitates the analysis of the IGHV mutation status, dominant clones, and prognostic value but also contributes to the exploration of IGK/IGL gene rearrangement fragments and the utilization of subclones. Further, it provides information about the poor prognosis of IGLV3 - 21 CLL. The shortened survival of the two unmutated clone groups in the IGHV unmutated group may indicate a poor prognosis.</p>","PeriodicalId":24016,"journal":{"name":"Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi","volume":"46 3","pages":"261-268"},"PeriodicalIF":0.0,"publicationDate":"2025-03-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12038473/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144052688","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"[Clinical characteristics and treatment outcomes of adult patients with phytosterolemia presenting with Thrombocytopenia].","authors":"Y J Hu, W L Chen, M Xue, Y J Ding, H Mei, Y D Wang","doi":"10.3760/cma.j.cn121090-20240710-00257","DOIUrl":"10.3760/cma.j.cn121090-20240710-00257","url":null,"abstract":"<p><p><b>Objective:</b> To analyze the clinical characteristics of adult patients with phytosterolemia presenting with thrombocytopenia as the initial manifestation. <b>Methods:</b> A retrospective analysis was conducted on eight adult patients with phytosterolemia who visited Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, from December 2020 to December 2023. <b>Results:</b> ① The participants consisted of 2 (25%) male and 6 (75%) female patients, with a median age at diagnosis of 55 years (range: 29-66 years). The median duration from the discovery of thrombocytopenia to diagnosis was 10 years (range: 0.2-50 years). ② Compared with the normal control group (30 healthy adult volunteers) and the immune thrombocytopenia (ITP) control group (20 patients with ITP), patients with phytosterolemia exhibited significantly higher mean platelet volume and large platelet ratio. Peripheral blood smears revealed that the mean platelet diameter and the proportion of large platelets (diameter> 4 μm) were significantly higher in patients with phytosterolemia than those in the normal and ITP control groups (<i>P</i><0.01). ③ After a low-plant-sterol diet and ezetimibe treatment, five patients demonstrated decreased serum sitosterol and campesterol levels, increased hemoglobin concentration and platelet counts, and reduced platelet volume. <b>Conclusion:</b> Adult-onset phytosterolemia presenting with thrombocytopenia as the initial manifestation is prone to misdiagnosis. The presence of hemolytic anemia, splenomegaly, increased large platelets and schistocytes on peripheral blood smears, and xanthomas are crucial diagnostic indicators. Restricting dietary plant sterol intake and using ezetimibe to inhibit sterol absorption effectively lowers serum plant sterol levels and improves hematological abnormalities.</p>","PeriodicalId":24016,"journal":{"name":"Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi","volume":"46 3","pages":"238-243"},"PeriodicalIF":0.0,"publicationDate":"2025-03-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12038474/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144036780","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"[Advancements in CRISPR-Cas9 for Fanconi anemia].","authors":"Y M Gao, L X Chang, X F Zhu","doi":"10.3760/cma.j.cn121090-20240825-00321","DOIUrl":"10.3760/cma.j.cn121090-20240825-00321","url":null,"abstract":"<p><p>Fanconi anemia (FA) is a hereditary bone marrow failure syndrome that is characterized by genomic instability and heightened sensitivity to DNA cross-linking agents. In recent years, the CRISPR-Cas9 technology has exhibited groundbreaking progress in the field of gene therapy for FA. The traditional CRISPR-Cas9 technology has been successfully applied in FA gene editing. Further, single-base editing technology, based on the CRISPR/Cas9 system, performs precise and efficient gene repair for prevalent gene mutations in patients with FA. The prime editing technology provides new possibilities for gene editing; however, its application in FA has not been initiated. Despite significant advancements in FA gene editing technology, several challenges remain, including the collection of sufficient hematopoietic stem cells, the risk of increased tumorigenesis postgene editing, chromosomal instability, and off-target effects. Future research is recommended to focus on optimizing sgRNA and Cas9 nucleases, designing stricter PAM sequences to reduce off-target effects, and devising personalized gene editing strategies. Further, ethical and regulatory issues as well as long-term follow-ups are crucial priorities for future gene editing work. With continuous technological advancements and in-depth clinical trials, we expect more breakthroughs in FA treatment using the CRISPR-Cas9 technology in the future. This article reviews the latest research progress of CRISPR technology in FA treatment and analyzes the advantages and disadvantages of this technology in FA gene therapy.</p>","PeriodicalId":24016,"journal":{"name":"Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi","volume":"46 3","pages":"276-280"},"PeriodicalIF":0.0,"publicationDate":"2025-03-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12038472/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143988410","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"[Clinical efficacy analysis of haploidentical high-dose in vitro non-T-cell-depleted peripheral blood hematopoietic stem cell transplantation for the treatment of adult patients with Ph(+) acute lymphoblastic leukemia].","authors":"J L Xu, X F Du, H L Yuan, H B Wang, G Chen, R X Yang, K L Zhang, Aizezi Gulibadanmu, J H Qu, M Jiang","doi":"10.3760/cma.j.cn121090-20240411-00134","DOIUrl":"10.3760/cma.j.cn121090-20240411-00134","url":null,"abstract":"<p><p><b>Objective:</b> To investigate the clinical efficacy of haploidentical high-dose in vitro non-T-cell-depleted peripheral blood hematopoietic stem cell transplantation (haplo-HDPSCT) in treating adult patients with Ph(+) acute lymphoblastic leukemia (Ph(+) ALL) . <b>Method:</b> This retrospective analysis was conducted on the clinical efficacy of 25 adult patients with Ph(+) ALL who underwent haplo-HDPSCT from July 2011 to June 2022 at our hospital. <b>Results:</b> This study included 25 patients with a median age of 27 (16-61) years, consisting of 12 males and 13 females. CR1 and ≥CR2 before transplantation were found in 23 and 2 cases, positive and negative minimal residual lesions were observed in 8 and 17 cases, and myeloablative conditioning and reduced-intensity conditioning were reported in 21 and 4 cases, respectively. Hematopoietic function was restored in all 25 patients after stem cell infusion. Of the 25 patients who underwent transplantation, 16 developed acute graft-versus-host disease (aGVHD). The cumulative incidence rates of Ⅱ-Ⅳ and Ⅲ-Ⅳ aGVHD were (40.4±11.3) % and (4.8±4.6) %, respectively. Four patients experienced relapse after transplantation, the cumulative relapse rates at 1 and 2 years after transplantation were (4.0±3.9) % and (14.5±7.9) %, respectively. The 2-year overall survival rate after transplantation was (81.3±8.5) % and the disease-free survival rate was (77.1±9.1) %. <b>Conclusion:</b> This study reveals that the unique haplo-HDPSCT protocol achieves good clinical efficacy in Ph(+) ALL treatment.</p>","PeriodicalId":24016,"journal":{"name":"Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi","volume":"46 3","pages":"231-237"},"PeriodicalIF":0.0,"publicationDate":"2025-03-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12038476/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144049909","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"[BK virus nephropathy after allogeneic hematopoietic stem cell transplantation: a case report and literature review].","authors":"W L Zhang, Y L Zu, Z H Huang, Z Li, R R Gui, J Wang, X J Wang, H L Wang, X X Fan, Y P Song, B J Fang, J Zhou","doi":"10.3760/cma.j.121090-20240810-00298","DOIUrl":"10.3760/cma.j.121090-20240810-00298","url":null,"abstract":"<p><p>A 20-year-old male patient with T-lymphoblastic lymphoma/leukemia received 9/10 human leukocyte antigen-compatible unrelated peripheral blood stem cell transplantation. He was transplanted with 5.91×10(8) mononuclear cells/kg and 2.88×10(6) CD34(+) cells/kg, and neutrophil engraftment was obtained at +11 days and platelet engraftment at +9 days. After transplantation, he presented with repeatedly increased serum creatinine levels, BK virus (BKV) -associated hemorrhagic cystitis, and BKV viremia. BK virus nephropathy was diagnosed based on renal biopsy and metagenomic next-generation sequencing. After adjusting the immunosuppressant, intravenous immunoglobulin, and donor lymphocyte infusion treatment, the patient's renal function deteriorated progressively, and he eventually died of multiple organ failure at +289 days.</p>","PeriodicalId":24016,"journal":{"name":"Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi","volume":"46 3","pages":"273-275"},"PeriodicalIF":0.0,"publicationDate":"2025-03-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12038467/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144043225","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"[Limitations and challenges of glucocorticoids in the treatment of paroxysmal nocturnal hemoglobinuria].","authors":"B Han","doi":"10.3760/cma.j.cn121090-20241213-00568","DOIUrl":"10.3760/cma.j.cn121090-20241213-00568","url":null,"abstract":"<p><p>Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired hematopoietic stem cell disease that mainly occurs in young adults and is characterized by bone marrow failure, persistent intravascular hemolysis and thrombosis, all of which can cause severe end-organ damage, increase the risk of early death, and cause a severe disease burden in patients. In China, based on the historic reasons, glucocorticoids are still routinely used in many places. However, the effects of glucocorticoids on PNH hemolysis are uncertain. Evidence-based medical data and clinical benefits for glucocorticoid on PNH are missing, but the long-term use of glucocorticoids significantly increases the risk of adverse reactions in patients. Since PNH needs a lifelong follow-up and management, long-term glucocorticoid therapy will unavoidably seriously damage the health of patients. Therefore, glucocorticoids are not recommended for the treatment of PNH, either from domestic or overseas guidelines, or expert consensus. In this article, the limitations and challenges of glucocorticoids in the treatment of PNH were expounded upon, in order to encourage more effective and safe strategies to be accepted in China.</p>","PeriodicalId":24016,"journal":{"name":"Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi","volume":"46 3","pages":"193-197"},"PeriodicalIF":0.0,"publicationDate":"2025-03-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12038475/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144037776","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}