International Journal of Hematology最新文献_第9页

Long-term safety and effectiveness of romiplostim for chronic idiopathic thrombocytopenic purpura in real-world settings. 罗米波司汀治疗慢性特发性血小板减少性紫癜的长期安全性和有效性。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2024-12-01 Epub Date: 2024-09-29 DOI: 10.1007/s12185-024-03847-4

Naoshi Obara, Shigeki Hatanaka, Yukie Tsuji, Koji Higashi

{"title":"Long-term safety and effectiveness of romiplostim for chronic idiopathic thrombocytopenic purpura in real-world settings.","authors":"Naoshi Obara, Shigeki Hatanaka, Yukie Tsuji, Koji Higashi","doi":"10.1007/s12185-024-03847-4","DOIUrl":"10.1007/s12185-024-03847-4","url":null,"abstract":"Idiopathic thrombocytopenic purpura (ITP), an autoimmune hematologic disorder characterized by severe platelet count reduction, can be treated with romiplostim. However, post-marketing safety and effectiveness data for romiplostim in Japan are scarce. This prospective, observational, post-marketing Specified Use-Results Survey evaluated the real-world safety and effectiveness of romiplostim for 2 years. All patients treated with romiplostim during the survey period were eligible. Of the 1622 patients in the safety analysis set, 94.08% (1526/1622) had chronic ITP. The mean single dose of romiplostim was stable after 12 weeks and remained < 6 μg/kg in approximately 70% of patients until 104 weeks. Within 2 years, 14.92% of patients discontinued romiplostim because of adverse events, while 6.47% discontinued because of suspected adverse drug reactions. In contrast, 14.00% of patients discontinued romiplostim because of symptom improvement. Before romiplostim initiation, platelet count was < 2.0 × 104/µL in 60.54% of patients, and the mean platelet count was 2.84 ± 5.76 × 104/µL. Platelet count was 9.19 ± 13.01 × 104/µL after 4 weeks, and remained between 10.34 ± 10.72 and 12.38 ± 12.63 × 104/µL from 8 to 104 weeks of treatment. No specific concerns were revealed regarding the safety and effectiveness of romiplostim in chronic ITP; the findings demonstrated a favorable risk-benefit balance for romiplostim in this population. Trial registration: UMIN000047864 ( www.umin.ac.jp/ctr ).","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"665-674"},"PeriodicalIF":1.7,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11588964/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142346187","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Silent inactivation of asparaginase in Japan: results of the prospective ALL-ASP19 trial. 日本天冬酰胺酶无声失活：前瞻性 ALL-ASP19 试验结果。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2024-12-01 Epub Date: 2024-09-30 DOI: 10.1007/s12185-024-03856-3

Hidemi Shimonodan, Kimiyoshi Sakaguchi, Takashi Ishihara, Yasuhiro Okamoto, Takuro Nishikawa, Dai Keino, Reo Tanoshima, Souichi Suenobu

{"title":"Silent inactivation of asparaginase in Japan: results of the prospective ALL-ASP19 trial.","authors":"Hidemi Shimonodan, Kimiyoshi Sakaguchi, Takashi Ishihara, Yasuhiro Okamoto, Takuro Nishikawa, Dai Keino, Reo Tanoshima, Souichi Suenobu","doi":"10.1007/s12185-024-03856-3","DOIUrl":"10.1007/s12185-024-03856-3","url":null,"abstract":"Silent inactivation (SI) of L-asparaginase (ASP) is a phenomenon by which a neutralizing antibody for ASP (AAA) decreases ASP activity (ASA) in patients without a clinical allergy to ASP. Acute lymphoblastic leukemia (ALL) has a poor prognosis in patients with SI. Therefore, measurement of ASA levels, not AAA levels, is needed to identify patients with SI. We herein report the results of the prospective clinical trial ALL-ASP19, the first study in Japan to measure ASA and AAA to identify patients with SI. A total of 110 newly diagnosed ALL patients were enrolled, and ASA levels were measured three times during ALL treatment. Besides the 12 patients who discontinued the study, 32 were excluded due to inappropriate frequency and timing of ASA measurements and inappropriate ASP dosing. The remaining 66 patients were analyzed, and 3 patients with SI (4.5%) were identified. The incidence of SI is lower in Japan than in other countries. AAA was detected in all patients with SI, but four of the seven patients with AAA did not develop SI. Clinical characteristics did not significantly differ between patients with and without SI. Therefore, ASA levels must be measured to identify patients receiving insufficient ASP treatment.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"725-734"},"PeriodicalIF":1.7,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142346190","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Current treatment approach and future perspectives in B cell lymphoma. B 细胞淋巴瘤的当前治疗方法和未来展望。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2024-11-21 DOI: 10.1007/s12185-024-03879-w

Nobuhiko Yamauchi, Dai Maruyama

{"title":"Current treatment approach and future perspectives in B cell lymphoma.","authors":"Nobuhiko Yamauchi, Dai Maruyama","doi":"10.1007/s12185-024-03879-w","DOIUrl":"https://doi.org/10.1007/s12185-024-03879-w","url":null,"abstract":"Diffuse large B cell lymphoma (DLBCL) and follicular lymphoma (FL) represent the two major subtypes of mature B cell lymphoma. A deeper understanding of tumor biology, as well as molecular classification characterized by targetable gene alterations, and the introduction of novel treatment options, including targeted drugs (e.g., antibody-drug conjugates and small molecules [e.g., Bruton tyrosine kinase inhibitor]) and immune therapies (e.g., chimeric antigen receptor [CAR] T cell therapy and bispecific antibody [BsAb]), has changed the treatment paradigms for DLBCL and FL. In clinical practice, however, treatment regimens are determined mainly based on prior treatment history, duration of response after previous treatment, patient age, and patient frailty because there have been few randomized trials to inform treatment selection for patients with relapsed or refractory disease and because there is no single prognostic index that guides suitable treatment for each patient. In this review, we summarize the treatment options for DLBCL and FL and discuss the treatment strategies for these two subtypes. We also discuss future perspectives for the treatment of these subtypes.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.7,"publicationDate":"2024-11-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142686904","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Treatment of relapsed acute lymphoblastic leukemia in children: an observational study of the Japan Children's Cancer Group. 儿童急性淋巴细胞白血病复发的治疗：日本儿童癌症小组的观察研究。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2024-11-01 Epub Date: 2024-08-27 DOI: 10.1007/s12185-024-03838-5

Hiroaki Goto, Akiko Kada, Chitose Ogawa, Ritsuo Nishiuchi, Junko Yamanaka, Akihiro Iguchi, Masanori Nishi, Kimiyoshi Sakaguchi, Tadashi Kumamoto, Shinji Mochizuki, Hideaki Ueki, Yoshiyuki Kosaka, Akiko M Saito, Hidemi Toyoda

{"title":"Treatment of relapsed acute lymphoblastic leukemia in children: an observational study of the Japan Children's Cancer Group.","authors":"Hiroaki Goto, Akiko Kada, Chitose Ogawa, Ritsuo Nishiuchi, Junko Yamanaka, Akihiro Iguchi, Masanori Nishi, Kimiyoshi Sakaguchi, Tadashi Kumamoto, Shinji Mochizuki, Hideaki Ueki, Yoshiyuki Kosaka, Akiko M Saito, Hidemi Toyoda","doi":"10.1007/s12185-024-03838-5","DOIUrl":"10.1007/s12185-024-03838-5","url":null,"abstract":"The Japan Children's Cancer Group Relapsed Acute Lymphoblastic Leukemia (ALL) Committee conducted a prospective observational study (ALL-R14) to explore promising reinduction therapy regimens for relapsed ALL to investigate in future trials. In Japan, clofarabine- and bortezomib-based regimens were of interest since they were newly introduced for ALL in the study period (2015-2018). Seventy-five pediatric patients were enrolled in total. The 2-year event-free/overall survival rates in patients with first (n = 59) or second (n = 11) relapse were 40.1% (95% confidence interval [CI]: 25.5-52.3%)/66.3% (95% CI 52.3-77.0%) and 34.1% (95% CI 9.1-61.6%)/62.3% (95% CI 27.7-84.0%), respectively. Clofarabine- or bortezomib-based regimens were used only in patients with high-risk disease. The first reinduction therapy used in the 41 patients with early or multiple relapsed B-cell precursor ALL was clofarabine in 7 patients and bortezomib in 9 patients. The odds ratio for reinduction failure risk with a clofarabine- or bortezomib-based regimen compared with other regimens was 9.0 (95% CI 0.9-86.4, P = 0.057) or 1.9 (95% CI 0.4-8.7, P = 0.42), respectively. Thus, clofarabine- or bortezomib-based regimens had no obvious advantage as reinduction therapy for relapsed ALL in children.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"631-638"},"PeriodicalIF":1.7,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142072783","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Comparison of disease and risk classifications of AML before and after incorporation of NGS analysis of bone marrow samples. 骨髓样本纳入 NGS 分析前后急性髓细胞白血病的疾病和风险分类比较。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2024-11-01 Epub Date: 2024-09-02 DOI: 10.1007/s12185-024-03841-w

Hiroyuki Sugiura, Tatsunori Ishikawa, Taiga Kuroi, Sachiyo Okamoto, Naho Nomura, Taro Masunari, Nobuo Sezaki, Seishi Ogawa, Yasuhito Nannya, Masanori Makita

{"title":"Comparison of disease and risk classifications of AML before and after incorporation of NGS analysis of bone marrow samples.","authors":"Hiroyuki Sugiura, Tatsunori Ishikawa, Taiga Kuroi, Sachiyo Okamoto, Naho Nomura, Taro Masunari, Nobuo Sezaki, Seishi Ogawa, Yasuhito Nannya, Masanori Makita","doi":"10.1007/s12185-024-03841-w","DOIUrl":"10.1007/s12185-024-03841-w","url":null,"abstract":"Mutation profiling by next-generation sequencing (NGS) has facilitated understanding of the molecular pathogenesis of acute myeloid leukemia (AML), and has been incorporated into the new disease classification (International Consensus Classification; ICC) and risk classification (European LeukemiaNet [ELN] 2022; ELN2022). We compared disease subtypes between the previous disease classification (4th edition of the WHO classification; WHO-4) and the ICC in 91 patients with AML diagnosed at our institution. We also compared disease risk classifications using the previous risk classification (ELN2017) and the ELN2022. Targeted sequencing of bone marrow samples was conducted at Kyoto University. We found that entities under AML with recurrent genetic abnormalities were well-established, with almost no change from the WHO-4 to the ICC. In contrast, 16.7% of cases of AML, not otherwise specified in the WHO-4 were reclassified into AML with mutated TP53, and 36.7% were reclassified into AML with myelodysplasia-related gene mutations or cytogenetic abnormalities per the ICC. Meanwhile, the ELN2017 and ELN2022 showed no difference in concordance indexes in multivariate Cox regression analysis for progression-free and overall survival. The superiority of the ELN2022 over the ELN2017 could not be confirmed in our single-center retrospective study, and further investigation including multicenter prospective studies is needed.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"594-600"},"PeriodicalIF":1.7,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142107144","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Tazemetostat for relapsed/refractory B-cell non-Hodgkin lymphoma with EZH2 mutation in Japan: 3-year follow-up for a phase II study. 在日本，Tazemetostat 用于治疗 EZH2 突变的复发/难治性 B 细胞非霍奇金淋巴瘤：一项 II 期研究的 3 年随访。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2024-11-01 Epub Date: 2024-08-23 DOI: 10.1007/s12185-024-03834-9

Koji Izutsu, Kiyoshi Ando, Momoko Nishikori, Hirohiko Shibayama, Hideki Goto, Junya Kuroda, Koji Kato, Yoshitaka Imaizumi, Kisato Nosaka, Rika Sakai, Maho Abe, Seiichiro Hojo, Tadashi Nakanishi, Shinya Rai

{"title":"Tazemetostat for relapsed/refractory B-cell non-Hodgkin lymphoma with EZH2 mutation in Japan: 3-year follow-up for a phase II study.","authors":"Koji Izutsu, Kiyoshi Ando, Momoko Nishikori, Hirohiko Shibayama, Hideki Goto, Junya Kuroda, Koji Kato, Yoshitaka Imaizumi, Kisato Nosaka, Rika Sakai, Maho Abe, Seiichiro Hojo, Tadashi Nakanishi, Shinya Rai","doi":"10.1007/s12185-024-03834-9","DOIUrl":"10.1007/s12185-024-03834-9","url":null,"abstract":"Previously, we reported the efficacy and safety of tazemetostat in Japanese patients with relapsed/refractory follicular lymphoma (FL) and diffuse large B-cell lymphoma (DLBCL) harboring the EZH2 mutation in a multicenter, open-label, phase II study. Here, we present a follow-up analysis of tazemetostat at a long-term median follow-up of 35.0 months. Twenty patients were enrolled: 17 in the FL cohort and three in the DLBCL cohort. In the FL cohort, the objective response rate was 70.6%, consistent with the primary analysis, and the median progression-free survival (PFS) was not reached. The 24-month and 36-month PFS rates were 72.1% (95% confidence interval [CI] 41.5%-88.6%) and 64.1% (95% CI 33.7%-83.4%), respectively. The median duration of treatment was 30.2 months. After the primary analysis at a median follow-up of 12.9 months, grade 1-2 urinary tract infection, peripheral motor neuropathy, and hypogammaglobulinemia newly emerged, but the incidence of adverse events (AEs) did not increase notably during this follow-up period. No unexpected grade ≥ 3 treatment-related AEs were reported. Long-term oral monotherapy with tazemetostat showed favorable efficacy and safety profiles, indicating that it may be a useful third-line or later treatment option for patients with relapsed/refractory FL harboring the EZH2 mutation. Trial registration: ClinicalTrials.gov: NCT03456726.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"621-630"},"PeriodicalIF":1.7,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11513718/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142046600","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Venetoclax treatment for chronic lymphocytic leukemia/small lymphocytic leukemia in Japan: post-marketing surveillance. 日本对慢性淋巴细胞白血病/小淋巴细胞白血病的 Venetoclax 治疗：上市后监测。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2024-11-01 Epub Date: 2024-08-21 DOI: 10.1007/s12185-024-03832-x

Tomoki Ito, Tomohiko Kamimura, Toru Kiguchi, Koji Kato, Risa Takenaka, Mariko Kobayashi, Ayumi Ito, Mizu Sakai, Koji Izutsu

{"title":"Venetoclax treatment for chronic lymphocytic leukemia/small lymphocytic leukemia in Japan: post-marketing surveillance.","authors":"Tomoki Ito, Tomohiko Kamimura, Toru Kiguchi, Koji Kato, Risa Takenaka, Mariko Kobayashi, Ayumi Ito, Mizu Sakai, Koji Izutsu","doi":"10.1007/s12185-024-03832-x","DOIUrl":"10.1007/s12185-024-03832-x","url":null,"abstract":"Venetoclax was approved for relapsed/refractory chronic lymphocytic leukemia (R/R CLL) and small lymphocytic leukemia (SLL) in Japan in September 2019; however, clinical data in Japanese patients are limited. This all-case post-marketing surveillance assessed efficacy and safety in Japanese patients with R/R CLL/SLL who started venetoclax treatment between November 2019 and August 2020. Overall, the safety and efficacy analysis sets included 129 and 114 patients, respectively. The overall response rate (ORR) was 57.0%; ORRs were higher in patients with versus without concomitant rituximab (65.4% vs. 54.7%), and in patients with 1 versus ≥ 2 prior lines of therapies (72.5% vs. 44.4%). Adverse events (AEs) were reported in 66.7% of patients (86/129); the most common AEs were neutrophil count decreased (22.5%), white blood cell count decreased (7.8%), and tumor lysis syndrome (TLS; 6.2%). AEs of special interest (TLS, myelosuppression, and infection) were manageable in clinical practice in Japan. Venetoclax is efficacious and safe for R/R CLL/SLL patients in the real-world setting in Japan. ClinicalTrials.gov ID: NCT04198415.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"613-620"},"PeriodicalIF":1.7,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11513738/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142017367","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

A case of posttransplant isolated extramedullary relapse of acute lymphoblastic leukemia achieving durable treatment-free remission with blinatumomab and donor lymphocyte infusion. 一例移植后孤立性髓外复发的急性淋巴细胞白血病患者，通过使用 blinatumomab 和输注供体淋巴细胞获得了持久的无治疗缓解。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2024-11-01 Epub Date: 2024-08-30 DOI: 10.1007/s12185-024-03839-4

Makoto Nishijima, Kentaro Ido, Yusuke Okayama, Hiroshi Okamura, Masatomo Kuno, Yosuke Makuuchi, Mitsutaka Nishimoto, Yasuhiro Nakashima, Hideo Koh, Mika Nakamae, Masayuki Hino, Hirohisa Nakamae

{"title":"A case of posttransplant isolated extramedullary relapse of acute lymphoblastic leukemia achieving durable treatment-free remission with blinatumomab and donor lymphocyte infusion.","authors":"Makoto Nishijima, Kentaro Ido, Yusuke Okayama, Hiroshi Okamura, Masatomo Kuno, Yosuke Makuuchi, Mitsutaka Nishimoto, Yasuhiro Nakashima, Hideo Koh, Mika Nakamae, Masayuki Hino, Hirohisa Nakamae","doi":"10.1007/s12185-024-03839-4","DOIUrl":"10.1007/s12185-024-03839-4","url":null,"abstract":"Acute lymphoblastic leukemia (ALL) relapsed after allogeneic hematopoietic cell transplantation (allo-HCT) has a catastrophic prognosis. Blinatumomab, a CD3/CD19-directed bispecific T cell engager, is reportedly effective for advanced B-cell ALL (B-ALL), even after allo-HCT. However, the efficacy of blinatumomab in extramedullary relapse (EMR) is controversial. Donor lymphocyte infusion (DLI) is another immunological treatment worth considering for ALL relapsed after allo-HCT. We report the case of a 56-year-old woman with B-ALL. Allo-HCT was performed during the second complete remission (CR). Thirteen months after allo-HCT, isolated EMR (iEMR) of B-ALL developed without bone marrow lesions. A third CR was achieved with 2 cycles of blinatumomab. An additional four cycles each of blinatumomab and DLI were then administered. The patient did not develop graft-versus-host disease and has confirmed 2-year treatment-free remission without a second allo-HCT. Therefore, blinatumomab was considered an effective salvage therapy for iEMR of B-ALL after allo-HCT, because iEMR could have a lower tumor burden than that seen in systemic relapse, and low tumor burden was a prognostic factor for response to blinatumomab. Furthermore, immunological consolidation therapies could only provoke graft-versus-leukemia effects if the imbalanced effector/target ratio was restored and the tumor burden was lowered through immunosurveillance.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"645-650"},"PeriodicalIF":1.7,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142107143","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Changes in coagulation potential over time after administration of recombinant activated factor VII in an emicizumab-treated hemophilia A patient with inhibitors. 一名接受埃米珠单抗治疗的 A 型血友病患者在使用重组活化因子 VII 后凝血潜能随时间的变化。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2024-11-01 Epub Date: 2024-08-07 DOI: 10.1007/s12185-024-03828-7

Mitsumasa Osuna, Yuto Nakajima, Kenichi Ogiwara, Keiji Nogami

{"title":"Changes in coagulation potential over time after administration of recombinant activated factor VII in an emicizumab-treated hemophilia A patient with inhibitors.","authors":"Mitsumasa Osuna, Yuto Nakajima, Kenichi Ogiwara, Keiji Nogami","doi":"10.1007/s12185-024-03828-7","DOIUrl":"10.1007/s12185-024-03828-7","url":null,"abstract":"We describe a 67-year-old patient with hemophilia A and inhibitors (PwHA-I) receiving emicizumab prophylaxis who underwent surgical treatment for pseudoaneurysm. He was treated with a bolus infusion of recombinant factor VIIa (rFVIIa; 79 μg/kg) immediately before surgery, and a second dose of rFVIIa after an initial treatment on day 1. A third rFVIIa bolus was infused 17 h after the second dose on day 2, and the treatment was continued every 24 h on day 3 and day 4. Treatment with rFVIIa was discontinued on day 4. No perioperative bleeding or thrombotic events were observed. Coagulation potentials at 8 h after rFVIIa administration determined by clot waveform analysis (CWA) and thrombin generation assay (TGA) were within near-normal ranges, and results at 17 h after rFVIIa administration showed coagulation function comparable to that in the patient without rFVIIa. Our experimental data suggest that the coagulation potential in FVIII-deficient plasma spiked with both 0.28 µg/mL (11.2 μg/kg) rFVIIa and emicizumab was equivalent to or greater than that spiked with 2.2 µg/mL (90 μg/kg) rFVIIa alone. Thus, administration of rFVIIa every 8 h may be feasible for managing perioperative treatment in emicizumab-treated PwHA-I.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"639-644"},"PeriodicalIF":1.7,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141897398","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Recent progress in AML with recurrent genetic abnormalities. 具有复发性基因异常的急性髓细胞性白血病的最新进展。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2024-11-01 Epub Date: 2024-10-01 DOI: 10.1007/s12185-024-03848-3

Yuichi Ishikawa

{"title":"Recent progress in AML with recurrent genetic abnormalities.","authors":"Yuichi Ishikawa","doi":"10.1007/s12185-024-03848-3","DOIUrl":"10.1007/s12185-024-03848-3","url":null,"abstract":"Acute myeloid leukemia (AML) is a heterogeneous disease characterized by various molecular abnormalities that significantly impact its pathogenesis and prognosis. Currently, the prognosis of AML patients is stratified on the basis of co-existing chromosomal and genetic abnormalities. AML patients with NPM1 or CEBPA mutations, which are frequently identified in cytogenetically normal AML, are classified in the favorable-risk group, although approximately 40% of patients relapse. Similarly, a clinical high-risk group has been identified among patients with acute promyelocytic leukemia, but the underlying molecular abnormalities remain unclear. FLT3 mutations frequently overlap in these favorable-risk AMLs, including core binding factor AML, and their prognostic impact is still controversial. As such, further risk stratification and treatment optimization based on various molecular abnormalities are warranted to improve the prognosis of favorable-risk AMLs. These molecular abnormalities are also considered therapeutic targets, and targeted therapies have been developed over the years. In recent years, several targeted agents have been approved and demonstrated to improve the prognosis of AML. However, resistance to targeted therapies is also a challenge. This Progress in Hematology features current trends and challenges in favorable-risk AML and FLT3 mutations that are frequently identified in these patients.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"525-527"},"PeriodicalIF":1.7,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142346188","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0