International Journal of Hematology最新文献_第7页

Validation of an algorithm for identifying patients with febrile neutropenia: an epidemiological study using real-world data in Japan. 一种识别发热性中性粒细胞减少症患者的算法的验证：一项使用日本真实世界数据的流行病学研究。

IF 1.8 4区医学

International Journal of Hematology Pub Date : 2025-08-15 DOI: 10.1007/s12185-025-04053-6

Kazuhiro Ishikawa, Nobuyoshi Mori, Kiyohide Fushimi, Koichi Benjamin Ishikawa

{"title":"Validation of an algorithm for identifying patients with febrile neutropenia: an epidemiological study using real-world data in Japan.","authors":"Kazuhiro Ishikawa, Nobuyoshi Mori, Kiyohide Fushimi, Koichi Benjamin Ishikawa","doi":"10.1007/s12185-025-04053-6","DOIUrl":"https://doi.org/10.1007/s12185-025-04053-6","url":null,"abstract":"The epidemiology of febrile neutropenia (FN) remains poorly understood. Although the Diagnosis Procedure Combination (DPC) database offers valuable clinical information, it lacks FN-specific diagnostic codes. This study aimed to develop an algorithm to identify patients with FN using DPC data and to investigate the epidemiology of FN. Data from St. Luke's International Hospital were used to identify DPC-based FN cases in patients with hematological malignancies who underwent chemotherapy, blood culture collection, and antipseudomonal antibiotic therapy. True FN cases were confirmed through chart review, incorporating neutrophil counts and body temperature. The algorithm demonstrated a sensitivity of 79.6%, specificity of 94.1%, PPV of 81.9%, NPV of 93.2%, and an F-measure of 80.6%. A sensitivity analysis for 2015-2020 showed improved performance: sensitivity 98.9%, specificity 99.6%, PPV 82.4%, NPV 99.6%, and F-measure 89.9%. Among the 144,009 patients with hematological malignancies in the DPC database, 50,558 received chemotherapy, and 13,720 (27.1%) developed FN as indicated by the algorithm, with in-hospital mortality rates of 15.0%. Although 76.7% of patients with FN received guideline-recommended first-line antibiotics, 17.0% received carbapenem, suggesting a potential need for antimicrobial stewardship interventions. The algorithm represents a valuable tool for large-scale epidemiological studies and could help inform strategies for FN management in Japan.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.8,"publicationDate":"2025-08-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144855146","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

Safety of liposomal daunorubicin-cytarabine (CPX-351) in secondary AML: Japanese phase 1/2 study and global phase 3 study. 柔红霉素-阿糖胞苷（CPX-351）脂质体治疗继发性AML的安全性：日本1/2期研究和全球3期研究

IF 1.8 4区医学

International Journal of Hematology Pub Date : 2025-08-15 DOI: 10.1007/s12185-025-04047-4

Naoko Hosono, Yusaku Tomiyama, Nanako Emori, Kento Isogaya, Takahiro Yamauchi

{"title":"Safety of liposomal daunorubicin-cytarabine (CPX-351) in secondary AML: Japanese phase 1/2 study and global phase 3 study.","authors":"Naoko Hosono, Yusaku Tomiyama, Nanako Emori, Kento Isogaya, Takahiro Yamauchi","doi":"10.1007/s12185-025-04047-4","DOIUrl":"https://doi.org/10.1007/s12185-025-04047-4","url":null,"abstract":"CPX-351 is a dual-drug liposomal encapsulation of cytarabine and daunorubicin. The safety of CPX-351 in Japanese patients was verified by comparing safety data from the Japanese phase 1/2 study and the global phase 3 study. This analysis included 47 patients in the safety analysis set of the Japanese study and 153 patients who received CPX-351 and were included in the intention-to-treat analysis for the global study. In both the Japanese and global studies, the most frequent adverse events were febrile neutropenia (FN) (Japanese: 85.1%, global: 70.0%) and pneumonia (25.5% and 24.2%). Median time to occurrence was 8 and 11 days for FN, and 17 and 23 days for pneumonia. Median time to hematologic recovery was 36 and 41 days for neutrophils (> 1000/μL), and 36 and 44 days for platelets (> 100,000/μL). In the global study, univariate analysis was performed after one cycle of induction therapy to investigate factors that delay neutrophil and platelet recovery, but no specific factors were identified. In these analyses, the pattern and frequency of adverse events were similar in Japanese and non-Japanese patients, with no need for race- or region-specific management.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.8,"publicationDate":"2025-08-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144855145","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

Acute hemolytic crises and clinical course in a Japanese family with Hb Santander: a detailed case report. 急性溶血危机和临床过程在一个日本家庭与Hb桑坦德：一个详细的病例报告。

IF 1.8 4区医学

International Journal of Hematology Pub Date : 2025-08-13 DOI: 10.1007/s12185-025-04052-7

Sayaka Suzuki, Yuzuru Hosoda, Miku Umeda, Masaya Maegaki, Koji Adachi, Fumihito Tajima, Yuki Hatayama, Rina Hosoda, Kentaro Hara, Koji Kawamura, Yasuhiro Yamashiro, Yukio Hattori, Tetsuya Fukuda

{"title":"Acute hemolytic crises and clinical course in a Japanese family with Hb Santander: a detailed case report.","authors":"Sayaka Suzuki, Yuzuru Hosoda, Miku Umeda, Masaya Maegaki, Koji Adachi, Fumihito Tajima, Yuki Hatayama, Rina Hosoda, Kentaro Hara, Koji Kawamura, Yasuhiro Yamashiro, Yukio Hattori, Tetsuya Fukuda","doi":"10.1007/s12185-025-04052-7","DOIUrl":"https://doi.org/10.1007/s12185-025-04052-7","url":null,"abstract":"A Japanese woman presented with a history of neonatal jaundice and recurrent episodes of severe fatigue and jaundice during viral infections in her late teens. Her mother, aunt, and grandmother had similar clinical histories. During an admission for fever and fatigue, blood tests revealed acute hemolysis and an abnormal hemoglobin band on high-performance liquid chromatography. β-globin gene sequencing identified a codon 34 substitution from GTC (Val) to GAC (Asp), confirming the presence of the unstable hemoglobin variant Hb Santander. This very rare hemoglobinopathy was previously reported only in a single sporadic case involving a Spanish man; this is the first documented case in a Japanese family. Our observation of four affected individuals across three generations provided insight into the progression of Hb Santander from birth to old age. Unstable hemoglobin variants can lead to recurrent, severe episodes of acute hemolytic crisis. Chronic hemolysis in the steady state was compensated without anemia or polycythemia but was associated with an increased risk of gallstone formation. Hb Santander does not appear to be a life-shortening hemoglobinopathy; however, clinical vigilance is necessary for acute hemolysis triggered by drugs or infections and for gallstones at a younger age, particularly in patients with Gilbert syndrome.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.8,"publicationDate":"2025-08-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144845867","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

Reduced splenic volume and advanced age predict sepsis in patients with Howell-Jolly bodies: a retrospective cohort study. 脾体积减小和高龄预测Howell-Jolly体患者败血症：一项回顾性队列研究。

IF 1.8 4区医学

International Journal of Hematology Pub Date : 2025-08-12 DOI: 10.1007/s12185-025-04050-9

Kaori Uchino, Yuya Nakagami, Megumi Enomoto, Nozomi Shimizu, Kenichi Kondo, Takahiro Yamamoto, Yukie Sugita, Yuto Isaji, Sakura Saigusa, Yusuke Iida, Saki Shinohara, Tomohiro Horio, Satsuki Murakami, Shohei Mizuno, Kazuhiro Ikegame, Ichiro Hanamura, Akiyoshi Takami

{"title":"Reduced splenic volume and advanced age predict sepsis in patients with Howell-Jolly bodies: a retrospective cohort study.","authors":"Kaori Uchino, Yuya Nakagami, Megumi Enomoto, Nozomi Shimizu, Kenichi Kondo, Takahiro Yamamoto, Yukie Sugita, Yuto Isaji, Sakura Saigusa, Yusuke Iida, Saki Shinohara, Tomohiro Horio, Satsuki Murakami, Shohei Mizuno, Kazuhiro Ikegame, Ichiro Hanamura, Akiyoshi Takami","doi":"10.1007/s12185-025-04050-9","DOIUrl":"https://doi.org/10.1007/s12185-025-04050-9","url":null,"abstract":"Functional hyposplenism, defined as impaired splenic function in the absence of splenectomy, increases susceptibility to life-threatening infections. Although Howell-Jolly bodies (HJBs) are well-established markers for this condition, the predictive value of spleen volume for infection risk remains unclear. We retrospectively analyzed 95 non-splenectomized patients with HJBs from 2014 to 2024. We measured spleen volume by computed tomography and compared results with ideal values. We evaluated the associations between splenic volume and infections using univariate and multivariate logistic regression analyses. The median patient age was 66 years (range, 16-95); 72% were male. The median spleen volume was 34 mL, lower than the ideal median of 210 mL. Forty-eight percent of patients experienced at least one infection. Univariate analysis identified age ≥ 65 years and spleen volume < 34 mL as significantly associated with sepsis. Both factors remained independent predictors in multivariate analysis (age ≥ 65: odds ratio [OR], p = 0.039; spleen volume < 34 mL: OR 3.0, p = 0.047). Age ≥ 65 also predicted any infection (OR 3.1, p = 0.013), while low spleen volume demonstrated a trend toward significance (OR 2.2, p = 0.064). In non-splenectomized patients with HJBs, reduced spleen volume and older age independently increase susceptibility to sepsis. Computed tomography-based measurements may help identify functional hyposplenism and guide targeted prophylactic measures.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.8,"publicationDate":"2025-08-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144821396","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

Management of inpatient chimeric antigen receptor T-cell therapy for relapsed/refractory B-cell malignancies: an analysis using the Japanese Diagnosis Procedure Combination database. 住院患者嵌合抗原受体t细胞治疗复发/难治性b细胞恶性肿瘤的管理：使用日本诊断程序组合数据库的分析

IF 1.8 4区医学

International Journal of Hematology Pub Date : 2025-08-06 DOI: 10.1007/s12185-025-04043-8

Keisuke Tanaka, Hiroaki Kikuchi, Yoshihiro Umezawa, Takehiko Mori, Kiyohide Fushimi, Masahide Yamamoto

{"title":"Management of inpatient chimeric antigen receptor T-cell therapy for relapsed/refractory B-cell malignancies: an analysis using the Japanese Diagnosis Procedure Combination database.","authors":"Keisuke Tanaka, Hiroaki Kikuchi, Yoshihiro Umezawa, Takehiko Mori, Kiyohide Fushimi, Masahide Yamamoto","doi":"10.1007/s12185-025-04043-8","DOIUrl":"https://doi.org/10.1007/s12185-025-04043-8","url":null,"abstract":"Chimeric antigen receptor T-cell (CAR-T) therapy has shown remarkable efficacy in treating relapsed/refractory B-cell malignancies, as supported by real-world evidence (RWE). However, limited RWE exists on the management of adverse events during the perioperative period following CAR-T infusion. This study was conducted to obtain RWE on perioperative management using the Japanese Diagnosis Procedure Combination database, a comprehensive repository of Japanese health and medical service data. Between November 2019 and March 2022, 388 patients received CAR-T therapy. Of these, 312 had large B-cell lymphoma (LBCL) and 76 had B-cell acute lymphoblastic leukemia (B-ALL). The number of CAR-T infusions increased every 6-month interval, correlating with the rise in LBCL cases. Tocilizumab was administered for cytokine release syndrome in 56.1% of LBCL and 42.1% of B-ALL patients. Steroids were used for 22.9% and 81.3%, respectively. Prophylaxis for fungal infections was administered during CAR-T infusion in most LBCL and B-ALL patients. Treatment intensity was escalated in 2.8% of LBCL and 7.0% of B-ALL patients, and treatment for cytomegalovirus infection was initiated in approximately 7% of patients. This analysis elucidated perioperative management strategies based on patients' medication histories.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.8,"publicationDate":"2025-08-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144794373","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

Andrographolide drives dual apoptosis and ferroptosis via caspase-3 and FACL4 in T-ALL cell lines. 穿心莲内酯通过caspase-3和FACL4驱动T-ALL细胞系的双重凋亡和铁下垂。

IF 1.8 4区医学

International Journal of Hematology Pub Date : 2025-08-02 DOI: 10.1007/s12185-025-04044-7

Hiroki Doi, Hidehiko Akiyama, Taei Matsui, Kazuya Shiogama, Masaya Hirayama, Rie Nakagawa, Sumie Fujii, Hideaki Matsuura, Yasuo Miura

{"title":"Andrographolide drives dual apoptosis and ferroptosis via caspase-3 and FACL4 in T-ALL cell lines.","authors":"Hiroki Doi, Hidehiko Akiyama, Taei Matsui, Kazuya Shiogama, Masaya Hirayama, Rie Nakagawa, Sumie Fujii, Hideaki Matsuura, Yasuo Miura","doi":"10.1007/s12185-025-04044-7","DOIUrl":"https://doi.org/10.1007/s12185-025-04044-7","url":null,"abstract":"This study investigated the anti-tumor effects of andrographolide, a diterpene lactone derived from Andrographis paniculata, on T-cell acute lymphoblastic leukemia (T-ALL) cells. Andrographolide induced dose-dependent cytotoxicity and morphological changes in the T-ALL cell line Jurkat cells, including cell shrinkage and chromatin condensation. Mechanistically, andrographolide triggers apoptosis through reactive oxygen species (ROS) generation, mitochondrial membrane depolarization, and cytochrome c release. These effects were reversed by the ROS inhibitor N-acetyl-L-cysteine (NAC), indicating that andrographolide induces apoptosis through a ROS-dependent apoptotic pathway. In contrast, NAC treatment did not reverse cytarabine- and vincristine-induced apoptosis or the ROS-dependent apoptotic pathway in Jurkat cells. Intriguingly, andrographolide also induced ferroptosis, as evidenced by increased expression of the ferroptosis marker fatty acid-CoA ligase 4 and ultrastructural changes such as reduced mitochondrial area and disappearance of cristae. These effects were likewise reversed by NAC, further implicating ROS in the ferroptotic process. In MOLT-4 cells, where andrographolide suppressed viability, increased Annexin V positivity and ROS levels, and upregulated FACL4 expression in a NAC-sensitive manner. Unlike cytarabine and vincristine, andrographolide did not significantly alter cell cycle distribution. In conclusion, andrographolide induces both apoptosis and ferroptosis in T-ALL cells via ROS-dependent mechanisms that are distinct from those of conventional chemotherapeutic agents. These dual actions position andrographolide as a candidate for standalone or combination therapy in T-ALL.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.8,"publicationDate":"2025-08-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144768633","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

Incidence of herpes zoster in transplant-ineligible patients with newly diagnosed multiple myeloma treated with daratumumab, lenalidomide, and dexamethasone. 接受达拉单抗、来那度胺和地塞米松治疗的新诊断多发性骨髓瘤患者不适合移植的带状疱疹发病率

IF 1.8 4区医学

International Journal of Hematology Pub Date : 2025-08-01 Epub Date: 2025-03-31 DOI: 10.1007/s12185-025-03980-8

Yuichi Horigome, Hirotoshi Kamata, Yusuke Michishita, Maki Yokoyama, Noriyuki Tadera, Kei Hayama, Takahiro Suzuki

{"title":"Incidence of herpes zoster in transplant-ineligible patients with newly diagnosed multiple myeloma treated with daratumumab, lenalidomide, and dexamethasone.","authors":"Yuichi Horigome, Hirotoshi Kamata, Yusuke Michishita, Maki Yokoyama, Noriyuki Tadera, Kei Hayama, Takahiro Suzuki","doi":"10.1007/s12185-025-03980-8","DOIUrl":"10.1007/s12185-025-03980-8","url":null,"abstract":"Combination therapy with daratumumab, lenalidomide, and dexamethasone (D-Rd) has greatly improved outcomes for transplant-ineligible newly diagnosed multiple myeloma (TIE-NDMM). Effective management of herpes zoster (HZ) and other infections is critical to maximize therapeutic benefit and to maintain treatment continuity. However, antiviral prophylaxis for HZ in TIE-NDMM patients receiving D-Rd has unclear efficacy, and is currently not covered by health insurance in Japan. In this study, we retrospectively analyzed the incidence of HZ in 40 TIE-NDMM patients treated with D-Rd. Nine patients (22.5%) developed HZ at a median period of 10.7 months (range, 0.4-34.2 months) after starting D-Rd. The cumulative HZ incidence at 12, 24, and 36 months was 13.3%, 19.5%, and 28.6%, respectively. Development of HZ was not associated with patient characteristics, disease characteristics, or hematologic response. Our data indicate a high incidence of HZ in TIE-NDMM patients receiving D-Rd, and we anticipate that Japanese health insurance should soon cover prophylactic treatment of HZ in D-Rd.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"224-230"},"PeriodicalIF":1.8,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143752682","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

Synergistic effect of asciminib with reduced doses of ponatinib in human Ph + myeloid leukemia with the T315M mutation. 阿西米尼与减少剂量波纳替尼对T315M突变的人Ph +髓系白血病的协同作用。

IF 1.8 4区医学

International Journal of Hematology Pub Date : 2025-08-01 Epub Date: 2025-04-10 DOI: 10.1007/s12185-025-03981-7

Thao Nguyen, Daisuke Harama, Minori Tamai, Keiko Kagami, Chiaki Komatsu, Shin Kasai, Koshi Akahane, Kumiko Goi, Takeshi Inukai

{"title":"Synergistic effect of asciminib with reduced doses of ponatinib in human Ph + myeloid leukemia with the T315M mutation.","authors":"Thao Nguyen, Daisuke Harama, Minori Tamai, Keiko Kagami, Chiaki Komatsu, Shin Kasai, Koshi Akahane, Kumiko Goi, Takeshi Inukai","doi":"10.1007/s12185-025-03981-7","DOIUrl":"10.1007/s12185-025-03981-7","url":null,"abstract":"In Philadelphia chromosome-positive (Ph +) leukemia, substitution of threonine at the 315 position of BCR::ABL1 with isoleucine (T315I) induces severe resistance to tyrosine kinase inhibitors (TKIs). Of clinical importance, the substitution of the baseline T315I mutation by methionine (I315M) was reported in a Ph + leukemia patient treated with ponatinib. The resultant T315M mutation induces severe TKI-resistance in a murine Ba/F3 model. Asciminib, an allosteric inhibitor of BCR::ABL1, is reportedly active in ponatinib-resistant patients with the T315I mutation. Although asciminib alone is not active in a murine Ba/F3 model with the T315M mutation, asciminib and ponatinib show synergistic activities. In the present study, we introduced the T315M mutation into the intrinsic BCR::ABL1 gene of two Ph + myeloid and one Ph + lymphoid leukemia cell lines using the CRISPR/Cas9 system to directly verify the utility of the combined asciminib and ponatinib in human models. All three T315M-acquired sublines were more resistant to TKIs including ponatinib than T315I-acquired sublines. Notably, asciminib exhibited a stronger synergistic effect with reduced doses of ponatinib in the T315M-acquired sublines of two myeloid cell lines, but not in the lymphoid cell line. This indicates that the combination of ponatinib and asciminib may have a clinical utility in human Ph + myeloid leukemia.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"206-216"},"PeriodicalIF":1.8,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12304016/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143994550","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

Retrospective analysis of treatment discontinuation in minimal residual disease negative multiple myeloma. 微小残留病阴性多发性骨髓瘤停药的回顾性分析。

IF 1.8 4区医学

International Journal of Hematology Pub Date : 2025-08-01 Epub Date: 2025-03-09 DOI: 10.1007/s12185-025-03966-6

Shuku Sato, Emi Sawazaki, Shun Tsunoda, Wataru Kamata, Tomiteru Togano, Yotaro Tamai

{"title":"Retrospective analysis of treatment discontinuation in minimal residual disease negative multiple myeloma.","authors":"Shuku Sato, Emi Sawazaki, Shun Tsunoda, Wataru Kamata, Tomiteru Togano, Yotaro Tamai","doi":"10.1007/s12185-025-03966-6","DOIUrl":"10.1007/s12185-025-03966-6","url":null,"abstract":"Background: The prognostic significance of minimal residual disease (MRD) negativity in multiple myeloma (MM) is well-established, and MRD negativity serves as a surrogate marker for treatment outcomes. However, in various clinical trials, achieving MRD negativity often leads to treatment continuation until disease progression. In real-world clinical practice, discontinuing treatment could lower healthcare costs and reduce adverse events.Methods: We retrospectively analyzed patients who reached MRD negativity and discontinued treatment.Results: A total of 39 multiple MM cases were included (17 eligible and 22 ineligible for autologous stem cell transplantation). The median time to next treatment was 42.4 months. Ten patients (25%) required additional treatment due to paraproteins or clinical relapse. Cumulative incidence of relapse at 12 and 48 months was 11.7% (95% confidence interval [CI], 4.5-28.2%) and 26.4% (95% CI, 12.8-49.6%), respectively. Multivariate analysis revealed that elevated lactate dehydrogenase (LDH) at first visit and t(4;14) were the only baseline factors significantly associated with worse outcomes. Eight patients (20%) with International Staging System (ISS) = I and with no risk factors (history of extramural disease, elevated LDH, high-risk cytogenetics) had no recurrence.Conclusion: Although treatment discontinuation in high-risk cases is potentially unsafe, low-risk cases demonstrate potential for treatment-free remission.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"217-223"},"PeriodicalIF":1.8,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143585707","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

Quality of life of children and adolescents with hemophilia receiving low-dose prophylactic treatment. 接受低剂量预防性治疗的儿童和青少年血友病患者的生活质量

IF 1.8 4区医学

International Journal of Hematology Pub Date : 2025-08-01 Epub Date: 2025-04-03 DOI: 10.1007/s12185-025-03969-3

Zahra Badiei, Atefeh Mokhtari, Hamid Farhangi, Maryam Heidarian, Elham Bakhtiari

{"title":"Quality of life of children and adolescents with hemophilia receiving low-dose prophylactic treatment.","authors":"Zahra Badiei, Atefeh Mokhtari, Hamid Farhangi, Maryam Heidarian, Elham Bakhtiari","doi":"10.1007/s12185-025-03969-3","DOIUrl":"10.1007/s12185-025-03969-3","url":null,"abstract":"Objective: Compared to episodic treatment, low-dose prophylaxis (LDP) leads to better quality of life (QOL) by decreasing spontaneous bleeding in severe hemophilia A and B. LDP has been used to treat children under 16 years of age in Iran since 2017. The present study assesses the QOL of these patients.Methods: Patients aged 8-16 years with severe hemophilia A and B receiving escalating LDP were included. Patients and parents completed the Hemophilia Quality of Life Questionnaire for Children (Haemo-QOL).Results: Twenty-five children and 15 adolescents were included. The total score for children and parents was 39.7 ± 14.5 and 39.1 ± 15, respectively (p = 0.88). The total score for adolescents and parents was 36.5 ± 15 and 41 ± 15.3, respectively (p = 0.42). The domains of friends and treatment in children and domains of friends and family in parents of children had the highest and lowest scores, respectively. The domains of sport/school and coping in adolescents and the domains of physical health and coping in parents of adolescents had the highest and lowest scores, respectively. The overall QOL of adolescents was better than that of children.Conclusion: LDP resulted in a marked improvement in most domains of QOL. LDP can be appropriate for societies with limited access to factor concentrate.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"277-283"},"PeriodicalIF":1.8,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143779968","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