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Isatuximab plus pomalidomide and dexamethasone in frail individuals with relapsed/refractory multiple myeloma in Japan. Isatuximab联合泊马度胺和地塞米松治疗日本复发/难治性多发性骨髓瘤患者
IF 1.7 4区 医学 Q3 HEMATOLOGY Pub Date : 2024-12-27 DOI: 10.1007/s12185-024-03904-y
Nami Tagami, Michihiro Uchiyama, Kenshi Suzuki, Heigoroh Shirai, Takeshi Seto, Shinsuke Iida

This post-marketing surveillance (PMS) assessed the safety and effectiveness of isatuximab plus pomalidomide and dexamethasone (Isa-Pd) for relapsed or refractory multiple myeloma (RRMM) in frail individuals during real-world use in Japan. Data from all individuals with RRMM treated with Isa-Pd in Japan between October 2020 and October 2021 were collected, with follow-up continued up to 12 months after starting Isa-Pd or until discontinuation. In the overall PMS population, 40 participants were classified as frail (33.3%) and 29 as fit/intermediate (24.2%), and 51 had no frailty score (42.5%). Incidence of adverse drug reactions in each group was 77.5%, 65.5%, and 37.3%. In frail versus fit/intermediate participants, bone-marrow suppression occurred in 72.5% versus 44.8%, infectious diseases in 17.5% versus 10.3%, and infusion-related reactions in 7.5% versus 3.5%. Heart failure occurred in one participant with no frailty score. The rates of overall response and very good partial response or better were higher (p = 0.101) in fit/intermediate participants (56.0% and 36.0%) than in frail participants (38.5% and 18.0%). Rates of treatment discontinuation due to disease progression were similar between groups. These findings support the safety and effectiveness of Isa-Pd for frail individuals with RRMM in real-life settings in Japan.

这项上市后监测(PMS)评估了isatuximab联合泊马度胺和地塞米松(Isa-Pd)在日本实际使用期间治疗虚弱个体复发或难治性多发性骨髓瘤(RRMM)的安全性和有效性。收集了2020年10月至2021年10月期间日本接受Isa-Pd治疗的所有RRMM患者的数据,随访持续至开始Isa-Pd治疗后12个月或直到停药。在整个经前综合症人群中,40名参与者被分类为虚弱(33.3%),29名被分类为适合/中级(24.2%),51名没有虚弱评分(42.5%)。各组药物不良反应发生率分别为77.5%、65.5%和37.3%。在虚弱和健康/中度受试者中,骨髓抑制发生率分别为72.5%和44.8%,感染性疾病发生率分别为17.5%和10.3%,输液相关反应发生率分别为7.5%和3.5%。一名没有虚弱评分的参与者发生心力衰竭。健康/中度参与者(56.0%和36.0%)的总体缓解率和非常好的部分缓解率高于虚弱参与者(38.5%和18.0%)(p = 0.101)。两组间因疾病进展而停止治疗的比率相似。这些发现支持Isa-Pd在日本现实生活中对患有RRMM的体弱个体的安全性和有效性。
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引用次数: 0
Adjunctive effects of eltrombopag on immunosuppressive therapy for childhood aplastic anemia. 儿童再生障碍性贫血免疫抑制治疗中的辅助作用。
IF 1.7 4区 医学 Q3 HEMATOLOGY Pub Date : 2024-12-27 DOI: 10.1007/s12185-024-03903-z
Katsuhide Eguchi, Masataka Ishimura, Shouichi Ohga, Saori Endo, Shoji Saito, Sachiyo Kamimura, Dai Keino, Shota Kato, Yoshihiro Azuma, Atsushi Watanabe, Akiko Inoue, Takeshi Higa, Shuichi Ozono, Naoto Fujita, Kenichiro Watanabe, Yoshiyuki Takahashi

Eltrombopag is used with first-line immunosuppressive therapy for adult aplastic anemia, although its practical utility in childhood remains unclear. We retrospectively analyzed the outcomes of pediatric patients who received eltrombopag in Japan. Of the 27 eligible patients, 23 (85%) were previously treated, and 15 (56%) had severe or very-severe disease. Seventeen (63%) received eltrombopag with or after rabbit anti-thymocyte globulin plus cyclosporin-A. Within the first year of eltrombopag therapy, 12 patients showed a good or partial response, 15 showed no response, and 8 non-responders successfully underwent hematopoietic cell transplantation. Within the first 3 months after eltrombopag therapy, all but one of the transfusion-dependent responders became transfusion-independent. At 12 months, 6 of 12 responders were disease-free off-treatment. The one-year overall response rate was higher for severe or very-severe than non-severe cases (p = 0.006). Multivariable analysis showed that very-severe disease at the start of eltrombopag therapy was a predictor of being disease-free off-treatment (p = 0.03). No cytogenetic abnormalities developed, but myelofibrosis occurred 4 months after eltrombopag therapy in one non-responder with very-severe disease. The first 3 months' response to adjunctive eltrombopag may guide to the safe and effective use for the cure of disease, although prospective trials are needed to determine its long-term effects.

Eltrombopag用于成人再生障碍性贫血的一线免疫抑制治疗,尽管其在儿童中的实际应用尚不清楚。我们回顾性分析了日本接受电子曲巴格治疗的儿科患者的预后。在27例符合条件的患者中,23例(85%)以前接受过治疗,15例(56%)患有严重或非常严重的疾病。17例(63%)患者在兔抗胸腺细胞球蛋白加环孢素a的同时或之后接受了电曲巴。在治疗的第一年内,12例患者表现出良好或部分反应,15例无反应,8例无反应成功进行了造血细胞移植。在接受电子波包治疗后的前3个月内,除1例输血依赖应答者外,所有患者均转为输血不依赖。在12个月时,12名应答者中有6名无病停止治疗。严重或极严重病例的1年总有效率高于非严重病例(p = 0.006)。多变量分析显示,治疗开始时非常严重的疾病是治疗结束后无疾病的预测因子(p = 0.03)。没有出现细胞遗传学异常,但在一名病情非常严重的无应答患者中,在接受电子波包治疗4个月后发生了骨髓纤维化。虽然还需要前瞻性试验来确定其长期效果,但对辅助电子曲巴的前3个月反应可能会指导安全有效地治疗疾病。
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引用次数: 0
Cost-effectiveness of ferric citrate hydrate in patients with iron deficiency anemia. 水合柠檬酸铁治疗缺铁性贫血的成本-效果。
IF 1.7 4区 医学 Q3 HEMATOLOGY Pub Date : 2024-12-26 DOI: 10.1007/s12185-024-03905-x
Mikio Momoeda, Kyoko Ito, Sachie Inoue, Hidetoshi Shibahara, Yuko Mitobe, Norio Komatsu

We investigated the cost-effectiveness of treating iron deficiency anemia (IDA) with ferric citrate hydrate (FC) in Japan. We employed four treatment strategies: switching from sodium ferrous citrate (SF) to FC at (1) 500 mg (approximately 120 mg of iron) per day or (2) 1000 mg (approximately 240 mg of iron) per day in patients with SF-induced nausea/vomiting, or starting treatment with FC at (3) 500 mg/day or (4) 1000 mg/day. We evaluated the cost-effectiveness of these strategies compared with SF 100 mg (100 mg of iron) per day. Incremental effects over 26 weeks relative to SF 100 mg were 0.0052 quality-adjusted life years (QALYs) for (1) and (2), and 0.0044 QALYs for (3) and (4). From the payer's perspective, incremental cost-effectiveness ratios (ICERs: JPY/QALY) against SF 100 mg were: (1) 1,107,780, (2) 2,257,477, (3) 5,588,430, and (4) 11,544,816. All four FC strategies were dominant (less costly and more effective) from a limited societal perspective. Treatment with FC for IDA was cost-effective (ICER ≤ JPY 5,000,000/QALY) when switching strategies from the payer perspective, and cost-saving (all FC strategies) from limited societal perspectives. Individual patients' characteristics and cost-effectiveness should be considered in treatment selection.

我们在日本研究了用柠檬酸铁水合物(FC)治疗缺铁性贫血(IDA)的成本-效果。我们采用了四种治疗策略:从柠檬酸亚铁钠(SF)切换到FC,(1)每天500毫克(约120毫克铁)或(2)每天1000毫克(约240毫克铁),用于SF引起的恶心/呕吐患者,或以(3)500毫克/天或(4)1000毫克/天的FC开始治疗。我们评估了这些策略与每天SF 100 mg (100 mg铁)相比的成本效益。26周内相对于SF 100 mg的增量效应(1)和(2)为0.0052质量调整生命年(QALYs),(3)和(4)为0.0044 QALYs。从付款人的角度来看,SF 100 mg的增量成本-效果比(ICERs: JPY/QALY)为:(1)1,107,780,(2)2,257,477,(3)5,588,430和(4)11,544,816。从有限的社会角度来看,所有四种FC策略都占主导地位(成本更低,更有效)。从支付者的角度转换策略时,使用FC治疗IDA具有成本效益(ICER≤500万日元/QALY),从有限的社会角度来看,使用FC治疗节省成本(所有FC策略)。在选择治疗方案时应考虑患者个体的特点和成本效益。
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引用次数: 0
A 25-year clonal resurrection in adult T-cell leukemia-lymphoma relapse. 成人t细胞白血病淋巴瘤复发的25年克隆复活。
IF 1.7 4区 医学 Q3 HEMATOLOGY Pub Date : 2024-12-25 DOI: 10.1007/s12185-024-03901-1
Atae Utsunomiya, Jun-Ichirou Yasunaga, Tomohisa Tabuchi, Nobuaki Nakano, Jun Odawara, Ayumu Kubota, Masahito Tokunaga, Takayoshi Miyazono, Masao Matsuoka, Yoshikiyo Ito, Yukie Tashiro

Here, we report a rare case of relapsed adult T-cell leukemia-lymphoma (ATL) with evidence of clonal relapse 26 years after initial diagnosis. The patient had been diagnosed with an aggressive form of lymphoma-type ATL 26 years prior and did not receive further ATL treatment for approximately 26 years after achieving complete remission. We used nested PCR to identify the amplification of ATL clone-specific accumulation sites in DNA from hematoxylin and eosin-stained specimens from the patient. Furthermore, the sequence of amplicons obtained from peripheral blood mononuclear cells and lymphoma cells from the previously diagnosed ATL were identical, indicating that a human T-cell leukemia virus-type 1 (HTLV-1)-infected clone identical to the one that recently caused ATL was present in the original lymphoma tissue. Although we were unable to identify this clone as the cause of the previous ATL, the peripheral leukemia cells revealed an ATL clone that was present in the tumor cells of a lymph node diagnosed 26 years earlier. To our knowledge, this is the first report demonstrating survival of HTLV-1-infected clones for a quarter of a century in a patient with recurrent ATL.

在此,我们报告一例罕见的成人t细胞白血病淋巴瘤(ATL)复发,在初次诊断后26年有克隆性复发的证据。患者在26年前被诊断为侵袭性淋巴瘤型ATL,在完全缓解后大约26年没有接受进一步的ATL治疗。我们使用巢式PCR方法鉴定了患者苏木精和伊红染色标本DNA中ATL克隆特异性积累位点的扩增。此外,从先前诊断为ATL的外周血单个核细胞和淋巴瘤细胞中获得的扩增子序列相同,表明在原始淋巴瘤组织中存在与最近引起ATL的人t细胞白血病病毒1型(HTLV-1)感染的克隆相同。虽然我们无法确定这个克隆是否是先前ATL的原因,但外周白血病细胞显示,在26年前诊断的淋巴结肿瘤细胞中存在一个ATL克隆。据我们所知,这是第一个证明htlv -1感染克隆在复发性ATL患者中存活了25年的报告。
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引用次数: 0
Chimerism analysis by ABO blood group genotyping with digital droplet PCR. 用数字液滴PCR进行ABO血型基因分型的嵌合分析。
IF 1.7 4区 医学 Q3 HEMATOLOGY Pub Date : 2024-12-17 DOI: 10.1007/s12185-024-03898-7
Takuya Naruto, Maiko Sagisaka, Mieko Ito, Akiko Hayashi, Naoyuki Miyagawa, Dai Keino, Tomoko Yokosuka, Fuminori Iwasaki, Hiroaki Goto, Masakatsu Yanagimachi

Objective: Chimerism analysis is an important post-transplant assessment for allogeneic hematopoietic stem cell transplant (HCT) recipients. Although various chimerism analysis techniques are already established, they are limited in terms of sensitivity, versatility, and turnaround time. Our objective was to develop a digital droplet polymerase chain reaction (ddPCR) assay for chimerism analysis using ABO gene polymorphisms as markers.

Methods: Our new chimerism analysis method utilizes ddPCR to assess the ABO gene polymorphisms that encode the ABO blood genotype. ABO genotypes were determined in blood samples from 15 HCT recipients using the O panel (rs8176719) and B panel (rs8176746 and rs8176747).

Results: The two panels distinguished six ABO genotypes (AA, AO, BB, BO, AB, and OO). The results of chimerism analysis using ABO genotypes with ddPCR were compatible with those of established methods, such as SRY gene analysis and the use of short tandem repeat markers via standard PCR. Our method could distinguish chimerism in 77% of donor and recipient combinations in the Japanese population.

Conclusions: We developed a sensitive and rapid chimerism analysis method for HCT using ABO gene polymorphisms in ddPCR.

目的:嵌合体分析是异基因造血干细胞移植(HCT)受者移植后的一项重要评估。虽然已有多种嵌合体分析技术,但它们在灵敏度、多功能性和周转时间方面都有局限性。我们的目标是开发一种数字液滴聚合酶链反应(ddPCR)检测方法,利用 ABO 基因多态性作为标记物进行嵌合体分析:我们的新嵌合体分析方法利用 ddPCR 评估编码 ABO 血液基因型的 ABO 基因多态性。结果:这两组数据区分出了 6 个 ABO 血型基因多态性:结果:两个面板可区分六种 ABO 基因型(AA、AO、BB、BO、AB 和 OO)。利用 ddPCR 对 ABO 基因型进行嵌合体分析的结果与 SRY 基因分析和通过标准 PCR 使用短串联重复标记等成熟方法的结果一致。我们的方法可以区分日本人群中 77% 的供体和受体组合的嵌合体:我们在 ddPCR 中利用 ABO 基因多态性开发了一种灵敏、快速的 HCT 嵌合体分析方法。
{"title":"Chimerism analysis by ABO blood group genotyping with digital droplet PCR.","authors":"Takuya Naruto, Maiko Sagisaka, Mieko Ito, Akiko Hayashi, Naoyuki Miyagawa, Dai Keino, Tomoko Yokosuka, Fuminori Iwasaki, Hiroaki Goto, Masakatsu Yanagimachi","doi":"10.1007/s12185-024-03898-7","DOIUrl":"https://doi.org/10.1007/s12185-024-03898-7","url":null,"abstract":"<p><strong>Objective: </strong>Chimerism analysis is an important post-transplant assessment for allogeneic hematopoietic stem cell transplant (HCT) recipients. Although various chimerism analysis techniques are already established, they are limited in terms of sensitivity, versatility, and turnaround time. Our objective was to develop a digital droplet polymerase chain reaction (ddPCR) assay for chimerism analysis using ABO gene polymorphisms as markers.</p><p><strong>Methods: </strong>Our new chimerism analysis method utilizes ddPCR to assess the ABO gene polymorphisms that encode the ABO blood genotype. ABO genotypes were determined in blood samples from 15 HCT recipients using the O panel (rs8176719) and B panel (rs8176746 and rs8176747).</p><p><strong>Results: </strong>The two panels distinguished six ABO genotypes (AA, AO, BB, BO, AB, and OO). The results of chimerism analysis using ABO genotypes with ddPCR were compatible with those of established methods, such as SRY gene analysis and the use of short tandem repeat markers via standard PCR. Our method could distinguish chimerism in 77% of donor and recipient combinations in the Japanese population.</p><p><strong>Conclusions: </strong>We developed a sensitive and rapid chimerism analysis method for HCT using ABO gene polymorphisms in ddPCR.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.7,"publicationDate":"2024-12-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142835633","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
Clinical practice guidelines for management of disseminated intravascular coagulation in Japan 2024. Part 1: sepsis. 2024 年日本弥散性血管内凝血管理临床实践指南。第 1 部分:败血症。
IF 1.7 4区 医学 Q3 HEMATOLOGY Pub Date : 2024-12-16 DOI: 10.1007/s12185-024-03896-9
Kazuma Yamakawa, Kohji Okamoto, Yoshinobu Seki, Takayuki Ikezoe, Takashi Ito, Toshiaki Iba, Satoshi Gando, Noritaka Ushio, Takaaki Totoki, Takeshi Wada, Hidesaku Asakura, Hiroyasu Ishikura, Mitsuhiro Uchiba, Toshimasa Uchiyama, Kaoru Kawasaki, Noriaki Kawano, Shigeki Kushimoto, Shin Koga, Yuichiro Sakamoto, Toshihisa Tamura, Kenji Nishio, Mineji Hayakawa, Takeshi Matsumoto, Seiji Madoiwa, Toshihiko Mayumi, Shinya Yamada, Hideo Wada

The Japanese Society on Thrombosis and Hemostasis (JSTH) published the first-ever disseminated intravascular coagulation (DIC) guidelines in 2009. Fifteen years later, the JSTH developed new guidelines covering DIC associated with various underlying conditions. These guidelines were developed in accordance with the GRADE system to determine the strength of the recommendations and certainty of the evidence. This article was drafted as Part 1 of an overall DIC guideline covering various underlying conditions, with sepsis as the subject. In this section, seven key clinical issues (questions) are set. Question 1, regarding DIC diagnosis, introduces several diagnostic criteria, such as the JAAM-2, ISTH overt, SIC, and JSTH DIC criteria and recommends choosing the appropriate diagnostic criteria for DIC based on an understanding of their diagnostic properties. For pharmacotherapy in DIC patients with sepsis, we recommend the administration of antithrombin (Question 2) and recombinant thrombomodulin (Question 3) (both GRADE 1B). However, we do not make a clear recommendation regarding the administration of heparin (Question 6) and serine protease inhibitors (Question 7) because of the lack of evidence. Combination therapy, order of administration, and other administration methods for antithrombin and recombinant thrombomodulin are proposed as important future research questions (Questions 4 and 5).

日本血栓与止血学会(JSTH)于 2009 年首次发布了弥散性血管内凝血(DIC)指南。15 年后,日本血栓与止血学会又制定了新的指南,涵盖了与各种基础疾病相关的 DIC。这些指南是根据 GRADE 系统制定的,以确定建议的力度和证据的确定性。本文是以败血症为主题,作为涵盖各种基础疾病的整体 DIC 指南的第一部分而起草的。本部分设置了七个关键临床问题(提问)。问题 1 涉及 DIC 诊断,介绍了几种诊断标准,如 JAAM-2、ISTH overt、SIC 和 JSTH DIC 标准,并建议在了解其诊断特性的基础上选择合适的 DIC 诊断标准。对于脓毒症 DIC 患者的药物治疗,我们建议使用抗凝血酶(问题 2)和重组血栓调节蛋白(问题 3)(均为 GRADE 1B)。但是,由于缺乏证据,我们没有就肝素(问题 6)和丝氨酸蛋白酶抑制剂(问题 7)的使用提出明确建议。抗凝血酶和重组血栓调节蛋白的联合疗法、给药顺序和其他给药方法被列为今后的重要研究课题(问题 4 和 5)。
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引用次数: 0
A practice-oriented genome-profiling study for acute myeloid leukemia using the novel HANDLE system: HM-screen-JAPAN02. 利用新型 HANDLE 系统对急性髓性白血病进行以实践为导向的基因组谱分析研究:HM-screen-JAPAN02.
IF 1.7 4区 医学 Q3 HEMATOLOGY Pub Date : 2024-12-16 DOI: 10.1007/s12185-024-03895-w
Hironori Arai, Naoko Hosono, SungGi Chi, Kentaro Fukushima, Daisuke Ikeda, Satoshi Iyama, Akihiko Gotoh, Takayuki Ikezoe, Chikashi Yoshida, Goichi Yoshimoto, Junya Kanda, Naoto Takahashi, Emiko Sakaida, Kensuke Usuki, Takahiro Yamauchi, Yosuke Minami

HM-SCREEN-Japan is a multicenter collaborative project in Japan to evaluate the clinical utility of a cancer genome panel in the treatment of acute myeloid leukemia (AML). The HM-SCREEN-JAPAN02 study used the Amoy Myeloid Panel® with the HANDLE system, which enables efficient and rapid sequencing, as the genomic testing kit. The Amoy Myeloid Panel® targets 53 genes with established clinical significance or high prevalence. The study analyzed bone marrow fluid or peripheral blood. Multiple time points for submission were allowed to evaluate clonal changes over time. A total of 179 tests/145 patients with one or more pathogenic mutations (23 patients submitted specimens at multiple time points) were included in the analysis. A variety of patterns were detected, including acquisition of new resistance-associated genetic mutations and pathogenic mutations remaining after clinical remission. The median time required for sequencing and annotation was 8 days. TP53 and NRAS mutations were associated with increased risk of death (hazard ratio = 3.98 and 5.50, respectively). In a survey of physicians at the participating centers, 63% reported that the genomic panel was clinically useful, particularly for assessing clinical risk and evaluating indications for hematopoietic stem cell transplantation.

HM-SCREEN-Japan是日本的一个多中心合作项目,旨在评估癌症基因组面板在急性髓性白血病(AML)治疗中的临床应用。HM-SCREEN-JAPAN02研究使用了具有HANDLE系统的淘髓系面板®,该系统可实现高效快速的测序,作为基因组检测试剂盒。淘髓系面板®针对53个具有临床意义或高患病率的基因。该研究分析了骨髓液或外周血。允许多个提交时间点评估克隆随时间的变化。共有179项试验/145例具有一种或多种致病突变的患者(23例患者在多个时间点提交标本)被纳入分析。检测到多种模式,包括获得新的耐药相关基因突变和临床缓解后残留的致病性突变。测序和注释所需的中位时间为8天。TP53和NRAS突变与死亡风险增加相关(风险比分别为3.98和5.50)。在对参与中心的医生进行的一项调查中,63%的医生报告说基因组小组在临床上是有用的,特别是在评估临床风险和评估造血干细胞移植的适应症方面。
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引用次数: 0
Clinical practice guidelines for management of disseminated intravascular coagulation in Japan 2024. Part 2: hematologic malignancy. 2024 年日本弥散性血管内凝血管理临床实践指南。第二部分:血液系统恶性肿瘤。
IF 1.7 4区 医学 Q3 HEMATOLOGY Pub Date : 2024-12-15 DOI: 10.1007/s12185-024-03887-w
Noriaki Kawano, Takayuki Ikezoe, Yoshinobu Seki, Kazuma Yamakawa, Kohji Okamoto, Masahiko Fukatsu, Seiji Madoiwa, Toshimasa Uchiyama, Hidesaku Asakura, Shinya Yamada, Shin Koga, Hiroyasu Ishikura, Takashi Ito, Toshiaki Iba, Mitsuhiro Uchiba, Kaoru Kawasaki, Satoshi Gando, Shigeki Kushimoto, Yuichiro Sakamoto, Toshihisa Tamura, Kenji Nishio, Mineji Hayakawa, Takeshi Matsumoto, Toshihiko Mayumi, Hideo Wada

Disseminated intravascular coagulation (DIC) associated with hematologic malignancies, particularly acute promyelocytic leukemia (APL), is characterized by marked fibrinolytic activation, which leads to severe bleeding complications. Therefore, appropriate diagnosis and management of DIC are crucial for preventing bleeding-related mortality. However, to date, no clinical guidelines have specifically addressed hematologic malignancy-associated DIC. Therefore, we developed diagnostic and management algorithms for DIC based on a systematic literature review. Notably, these guidelines recommend using the JSTH DIC diagnostic criteria (2017 version) or the former Ministry of Health and Welfare DIC diagnostic criteria (1983 version) to diagnose DIC. Furthermore, in the management of DIC, it is essential to treat the underlying disease through transfusion of platelet concentrates and fresh frozen plasma, if necessary. A systematic review of antifibrinolytic and anticoagulant therapies concluded that tranexamic acid therapy is not strongly recommended for patients with APL undergoing treatment with all-trans retinoic acid (Grade 1C). The use of recombinant thrombomodulin is weakly recommended (Grade 2B), whereas the use of other anticoagulants, including heparin and serine protease inhibitors, is weakly not recommended (Grade 2C). Therefore, we hope that these guidelines will help physicians find the best possible solutions in clinical practice.

弥散性血管内凝血(DIC)与血液系统恶性肿瘤,特别是急性早幼粒细胞白血病(APL)相关,其特征是显著的纤维蛋白溶解激活,导致严重的出血并发症。因此,DIC的正确诊断和处理对于预防出血相关死亡至关重要。然而,到目前为止,没有临床指南专门针对血液恶性肿瘤相关的DIC。因此,我们在系统文献回顾的基础上开发了DIC的诊断和管理算法。值得注意的是,这些指南建议使用JSTH DIC诊断标准(2017年版)或前保健福利部DIC诊断标准(1983年版)来诊断DIC。此外,在DIC的治疗中,必要时通过输血血小板浓缩物和新鲜冷冻血浆治疗基础疾病是必要的。一项关于抗纤溶和抗凝治疗的系统综述得出结论,对于接受全反式维甲酸治疗的APL患者,不强烈推荐氨甲环酸治疗(1C级)。弱推荐使用重组血栓调节素(2B级),而弱不推荐使用其他抗凝剂,包括肝素和丝氨酸蛋白酶抑制剂(2C级)。因此,我们希望这些指南能够帮助医生在临床实践中找到最好的解决方案。
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引用次数: 0
Treatment trends and risks of corticosteroid use in adult primary immune thrombocytopenia: a claims database study in Japan. 在成人原发性免疫性血小板减少症中使用皮质类固醇的治疗趋势和风险:日本的一项索赔数据库研究
IF 1.7 4区 医学 Q3 HEMATOLOGY Pub Date : 2024-12-12 DOI: 10.1007/s12185-024-03897-8
Hirokazu Kashiwagi, Isao Miura, Naohiko Terasawa, Ken-Ichi Iwayama, Yuka Furukawa, Makoto Kanenishi

Recent trends in the treatment of primary immune thrombocytopenia (ITP) were investigated using a claims database that included data from 16,161 Japanese patients with ITP collected from April 2014 to August 2022. Of the 4144 adult patients analyzed, 1276 received corticosteroids. The mean and median durations of corticosteroid use were 115.31 and 41 days, respectively. The time to withdrawal of corticosteroids was significantly shorter in 2020 to 2021 than in 2015 to 2019. Additionally, the number of prescriptions for thrombopoietin receptor agonists increased from 2015 to 2021 and exceeded that of corticosteroids in 2021. While these results suggest a trend towards reduction in corticosteroid use in real-world settings in Japan, 12.00% of patients received a corticosteroid dose of ≥ 10 mg/day at Week 12. Furthermore, 23.05% of patients continued to receive corticosteroids at Week 24, indicating that some patients were still receiving long-term corticosteroid treatment. The risk of adverse outcomes was significantly associated with corticosteroid use. In conclusion, new treatment options may lead to more sophisticated ITP management with less corticosteroid use, although further research and reconsideration of clinical practice guidelines is needed.

使用索赔数据库调查原发性免疫性血小板减少症(ITP)治疗的最新趋势,该数据库包括2014年4月至2022年8月收集的16,161名日本ITP患者的数据。在分析的4144名成年患者中,1276名接受了皮质类固醇。皮质类固醇使用的平均和中位持续时间分别为115.31天和41天。2020 - 2021年停用皮质类固醇的时间明显短于2015 - 2019年。此外,从2015年到2021年,血小板生成素受体激动剂的处方数量有所增加,并在2021年超过了皮质类固醇。虽然这些结果表明在日本现实环境中皮质类固醇的使用有减少的趋势,但12.00%的患者在第12周接受了≥10mg /天的皮质类固醇剂量。此外,23.05%的患者在第24周继续接受糖皮质激素治疗,表明部分患者仍在接受长期糖皮质激素治疗。不良后果的风险与皮质类固醇的使用显著相关。总之,新的治疗方案可能导致更复杂的ITP管理和更少的皮质类固醇使用,尽管需要进一步的研究和重新考虑临床实践指南。
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引用次数: 0
Raising awareness may increase the likelihood of hematopoietic stem cell donation: a nationwide survey using artificial intelligence. 一项使用人工智能的全国性调查显示,提高意识可能会增加造血干细胞捐赠的可能性。
IF 1.7 4区 医学 Q3 HEMATOLOGY Pub Date : 2024-12-12 DOI: 10.1007/s12185-024-03894-x
Luana Conte, Giorgio De Nunzio, Roberto Lupo, Donato Cascio, Marco Cioce, Elsa Vitale, Chiara Ianne, Ivan Rubbi, Massimo Martino, Letizia Lombardini, Aurora Vassanelli, Simonetta Pupella, Simona Pollichieni, Nicoletta Sacchi, Fabio Ciceri, Stefano Botti

Background: In Italy, the demand for allogeneic transplantation exceeds the number of compatible donors in the Italian Bone Marrow Donor Registry (IBMDR). This study aimed to explore the knowledge, beliefs, opinions, values, and feelings of the Italian population regarding stem cell donation.

Methods: An online survey was shared via social media. Respondents were retrospectively identified as registered on the IBMDR (donor group) or never registered (non-donor group). Statistical analyses confirmed the relationship between knowledge level and willingness to donate. Six machine learning classifiers were trained using questionnaire responses to predict the probability of IBMDR registration.

Results: A total of 1518 respondents participated. Characteristics identified in the non-donor group were a lower level of knowledge regarding donation needs (51.7% vs 24.4%, p < 0.001) and negative feelings such as fear (Z = - 2.2642, p = 0.02), confusion (Z = 4.4821, p < 0.001), and uncertainty (Z = 3.3425, p < 0.001). Higher knowledge predicted a greater likelihood of IBMDR enrollment. Machine learning analysis showed an AUC ranging from 0.65 to 0.81, depending on the classifier.

Conclusions: The results underscore the need to improve strategies to raise awareness and knowledge of stem cell donation among the Italian population.

背景:在意大利,同种异体移植的需求超过了意大利骨髓供体登记(IBMDR)中匹配供体的数量。本研究旨在探讨意大利人关于干细胞捐赠的知识、信仰、观点、价值观和感受。方法:通过社交媒体进行在线调查。回答者被回顾性地确定为在IBMDR上登记(捐助者组)或从未登记(非捐助者组)。统计分析证实了知识水平与捐赠意愿之间的关系。使用问卷回答训练六个机器学习分类器来预测IBMDR注册的概率。结果:共1518人参与调查。在非供体组中发现的特征是关于捐赠需求的知识水平较低(51.7%对24.4%)。结论:结果强调需要改进策略,以提高意大利人群对干细胞捐赠的认识和知识。
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International Journal of Hematology
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