International Journal of Hematology最新文献_第10页

Intravenous umbilical cord-derived mesenchymal stromal cell therapy may improve overall survival in Japanese patients with idiopathic pneumonia syndrome after hematopoietic stem cell transplantation: a multicenter, single-arm, phase II trial. 静脉输注脐带间充质间质细胞治疗可提高日本特发性肺炎综合征患者造血干细胞移植后的总生存率：一项多中心、单臂、II期试验。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2025-07-15 DOI: 10.1007/s12185-025-04024-x

Noriko Doki, Nobuharu Fujii, Shinichi Kako, Emiko Sakaida, Yoshinobu Kanda

{"title":"Intravenous umbilical cord-derived mesenchymal stromal cell therapy may improve overall survival in Japanese patients with idiopathic pneumonia syndrome after hematopoietic stem cell transplantation: a multicenter, single-arm, phase II trial.","authors":"Noriko Doki, Nobuharu Fujii, Shinichi Kako, Emiko Sakaida, Yoshinobu Kanda","doi":"10.1007/s12185-025-04024-x","DOIUrl":"https://doi.org/10.1007/s12185-025-04024-x","url":null,"abstract":"Idiopathic pneumonia syndrome (IPS) is a serious complication of allogeneic hematopoietic stem cell transplantation (HSCT) and has a poor prognosis. Although IPS is often treated with steroids, the disease can become resistant to or dependent on steroid treatment, and there is no effective cure for patients with refractory or steroid-dependent IPS. This multicenter, open-label, single-arm, phase II clinical trial investigated the efficacy and safety of HLC-001 (allogeneic umbilical cord-derived mesenchymal stromal cells) in patients with progressive steroid-dependent or refractory IPS after HSCT. Seven male patients (all male; mean age: 43.3 years) received HLC-001 and three completed the trial. The survival rate at day 56 (primary endpoint) was 71.4% (5/7 patients; 95% confidence interval: 29.0%-96.3%) and was sustained at day 100, suggesting that HLC-001 was more effective than previously reported treatment. Three of the five patients with ≥ 100 days of follow-up died. Five patients experienced at least one adverse drug reaction, none of which were serious. These findings indicate that HLC-001 was potentially effective and generally well tolerated in Japanese patients with steroid-dependent or refractory IPS after HSCT. Given there is no effective cure for steroid-dependent or refractory IPS, HLC-001 may be a promising treatment option and further clinical evaluation is warranted.Trial registration: Japan Registry of Clinical Trials identifier: jRCT2063220014.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.7,"publicationDate":"2025-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144642525","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

Growth differentiation factor-15 as a non-invasive biomarker of liver fibrosis in sickle cell disease. 生长分化因子-15作为镰状细胞病肝纤维化的非侵入性生物标志物

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2025-07-13 DOI: 10.1007/s12185-025-04035-8

Konstantinos Manganas, Sophia Delicou, Emilia Hadziyannis, Stavroula Giannouli, John Koskinas

{"title":"Growth differentiation factor-15 as a non-invasive biomarker of liver fibrosis in sickle cell disease.","authors":"Konstantinos Manganas, Sophia Delicou, Emilia Hadziyannis, Stavroula Giannouli, John Koskinas","doi":"10.1007/s12185-025-04035-8","DOIUrl":"https://doi.org/10.1007/s12185-025-04035-8","url":null,"abstract":"This study aimed to evaluate the relationship between growth differentiation factor-15 (GDF-15) levels and liver fibrosis in patients with sickle cell disease (SCD) and assess the diagnostic performance of GDF-15 as a non-invasive biomarker. Thirty patients with SCD were categorized into two groups based on fibrosis severity (≥ F2 vs. < F2) determined by Fibroscan, AST to Platelet Ratio Index (APRI) and FIB-4. GDF-15 levels were compared between groups, and independent predictors of GDF-15 were identified by multiple linear regression. Patients with significant liver fibrosis (≥ F2) had significantly higher GDF-15 levels. Multivariate linear regression revealed that GDF-15 concentration was independently associated with liver elastography values (β = 0.619, p = 0.002). The area under the curve for detecting significant fibrosis using GDF-15 as a diagnostic test was 0.835 (95% CI: 0.686-0.983, p = 0.002). A GDF-15 cut-off of 4200 pg/ml had a positive predictive value of 73.6%, a negative predictive value of 81.8%, sensitivity of 87.5% and specificity of 64.3%. In conclusion, GDF-15 is strongly associated with liver fibrosis in SCD and demonstrates high diagnostic accuracy as a non-invasive biomarker. Given its role in inflammation, oxidative stress, and iron metabolism, GDF-15 may serve as a valuable tool for early fibrosis detection and disease monitoring.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.7,"publicationDate":"2025-07-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144617374","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

Real-world safety and effectiveness of alemtuzumab as a conditioning regimen for hematopoietic stem cell transplantation. 阿仑单抗作为造血干细胞移植调理方案的安全性和有效性。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2025-07-07 DOI: 10.1007/s12185-025-04033-w

Yukie Sasakura, Makiko Hatanaka, Yoshinobu Kanda

{"title":"Real-world safety and effectiveness of alemtuzumab as a conditioning regimen for hematopoietic stem cell transplantation.","authors":"Yukie Sasakura, Makiko Hatanaka, Yoshinobu Kanda","doi":"10.1007/s12185-025-04033-w","DOIUrl":"https://doi.org/10.1007/s12185-025-04033-w","url":null,"abstract":"Alemtuzumab, a humanized monoclonal antibody directed against CD52, is indicated for administration prior to allogeneic hematopoietic stem cell transplantation (HSCT) in Japan. This post-marketing surveillance study was conducted as part of a risk management plan to confirm the safety and effectiveness of alemtuzumab in clinical practice, as mandated by the Japanese health authorities. Fifty-nine patients aged 0 to 69 years received alemtuzumab prior to HSCT for hematologic malignancies (n = 22), aplastic anemia (n = 7), or other diseases (n = 30). The number of mismatched human leukocyte antigens between donor and recipient was ≥ 2 in 39 patients (66.1%), 1 in seven patients (11.9%) and 0 in 11 patients (18.6%). Overall, 38 of 59 patients (64.4%) developed an adverse drug reaction (ADR), most commonly fever associated with infusion reactions (n = 22); 24 patients (40.7%) had a serious ADR (most commonly cytomegalovirus-related events [n = 7]), and 16 had a grade ≥ 3 ADR (mainly febrile neutropenia [n = 3]). Engraftment was achieved in 55 of 58 patients (94.8%) at a median of 16 days after HSCT, and predefined success (engraftment without grade ≥ 3 graft-versus-host disease in hematologic malignancies or grade ≥ 2 in other diseases) was met in 51 of 58 patients (87.9%). These results support the manageable safety profile and effectiveness of alemtuzumab as preconditioning for HSCT in Japanese patients.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.7,"publicationDate":"2025-07-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144575416","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

Post-transplant changes in physical functioning and quality of life in patients undergoing two allogeneic hematopoietic stem cell transplants. 接受两次同种异体造血干细胞移植的患者移植后身体功能和生活质量的变化。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2025-07-05 DOI: 10.1007/s12185-025-04030-z

Takahiro Takekiyo, Yoshikiyo Ito, Takayoshi Miyazono, Masahito Tokunaga, Nobuaki Nakano, Jun Odawara, Satoshi Fujino, Koichiro Dozono, Soichiro Nara, Shuichiro Shimoyama, Toshiyuki Okamura, Atae Utsunomiya

{"title":"Post-transplant changes in physical functioning and quality of life in patients undergoing two allogeneic hematopoietic stem cell transplants.","authors":"Takahiro Takekiyo, Yoshikiyo Ito, Takayoshi Miyazono, Masahito Tokunaga, Nobuaki Nakano, Jun Odawara, Satoshi Fujino, Koichiro Dozono, Soichiro Nara, Shuichiro Shimoyama, Toshiyuki Okamura, Atae Utsunomiya","doi":"10.1007/s12185-025-04030-z","DOIUrl":"https://doi.org/10.1007/s12185-025-04030-z","url":null,"abstract":"This study retrospectively examined changes in physical functioning and quality of life (QOL) in allogeneic hematopoietic stem cell transplantation (HSCT) recipients at their first (HSCT-1) and second (HSCT-2) transplantations. Seventeen patients who underwent HSCT-2 after relapse following HSCT-1 at our hospital between May 2013 and November 2023 and who underwent physical therapy evaluations before and after both transplantations were analyzed. Physical functioning was evaluated using handgrip strength as a measure of muscle strength, and exercise tolerance using the 6-min walk test (6MWT). QOL was assessed using the Medical Outcome Study 36-Item Short Form Health Survey (SF-36). Changes in physical functioning included handgrip strength (HSCT-1: - 9.3%; HSCT-2: - 18.3%; p < 0.05) and 6MWT findings (HSCT-1: - 5.1%; HSCT-2: - 12.6%; p < 0.05), which showed a significantly greater decline at HSCT-2. Physical functioning scores (SF-36) showed changes in QOL (HSCT-1: - 4.3 points; HSCT-2: - 23.4 points; p < 0.05), with a significantly greater reduction at HSCT-2. The findings indicate that the decline in physical functioning and QOL was more pronounced during the second transplantation than during the first. This highlights the importance of prioritizing strategies to maintain physical activity levels, particularly during the second transplantation process.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.7,"publicationDate":"2025-07-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144567407","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 features and outcomes of primary ocular adnexal follicular lymphoma: a retrospective analysis. 原发性眼附件滤泡性淋巴瘤的临床特点和预后：回顾性分析。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2025-07-03 DOI: 10.1007/s12185-025-04029-6

Shohei Nakamura, Shinichi Makita, Akiko Miyagi-Maeshima, Yoshikazu Hori, Haruhi Makino, Hirokazu Sasaki, Daiki Hattori, Noriko Iwaki, Suguru Fukuhara, Wataru Munakata, Hiroshi Igaki, Shigenobu Suzuki, Koji Izutsu

{"title":"Clinical features and outcomes of primary ocular adnexal follicular lymphoma: a retrospective analysis.","authors":"Shohei Nakamura, Shinichi Makita, Akiko Miyagi-Maeshima, Yoshikazu Hori, Haruhi Makino, Hirokazu Sasaki, Daiki Hattori, Noriko Iwaki, Suguru Fukuhara, Wataru Munakata, Hiroshi Igaki, Shigenobu Suzuki, Koji Izutsu","doi":"10.1007/s12185-025-04029-6","DOIUrl":"https://doi.org/10.1007/s12185-025-04029-6","url":null,"abstract":"Extranodal follicular lymphoma (FL) demonstrates unique clinicopathological features and prognostic implications, and the clinical characteristics of ocular adnexal FL are poorly documented. In this study, we retrospectively analyzed histologically confirmed cases of ocular adnexal FL at our institution between 1997 and 2019. Of the 261 patients with lymphoma involving the ocular adnexa, 28 had FL, and staging revealed that 10 had primary ocular adnexal FL. Of these 10 patients, one was excluded because of insufficient treatment information. The median age of the remaining nine patients (six women) was 62 (range: 24-81) years. Lesions were located in the orbit (three patients) or conjunctiva (six patients). Three patients had bilateral conjunctival lesions. Eight patients were initially treated with radiotherapy (including one who received radiotherapy after 1.5 years of observation); the other patient remained untreated for 6.4 years. With a median follow-up period of 7.7 years, three of eight patients who received radiotherapy relapsed (two in the contralateral conjunctiva, one in the subcutaneous tissue of the back), and no in-field relapses occurred. The 5-year progression-free and overall survival rates were 75% and 100%, respectively, without histological transformation. In conclusion, primary ocular adnexal FL has a favorable prognosis after radiotherapy and/or watchful waiting.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.7,"publicationDate":"2025-07-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144553497","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

Stopping bosutinib reverses bosutinib-induced elevation of serum creatinine in patients with chronic myeloid leukemia. 停用博舒替尼可逆转慢性髓性白血病患者博舒替尼诱导的血清肌酐升高。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2025-07-01 Epub Date: 2025-02-25 DOI: 10.1007/s12185-025-03954-w

Maiko Abumiya, Ayano Saito, Yuki Fujioka, Masatomo Miura, Naoto Takahashi

{"title":"Stopping bosutinib reverses bosutinib-induced elevation of serum creatinine in patients with chronic myeloid leukemia.","authors":"Maiko Abumiya, Ayano Saito, Yuki Fujioka, Masatomo Miura, Naoto Takahashi","doi":"10.1007/s12185-025-03954-w","DOIUrl":"10.1007/s12185-025-03954-w","url":null,"abstract":"Bosutinib is known to increase serum creatinine levels, and its mechanism of action is believed to involve a decrease in tubular creatinine excretion due to inhibition of tubular transporters and organic cation transporter 2. This study aimed to determine whether discontinuation of bosutinib could reverse bosutinib-induced elevation of serum creatinine levels. Serum creatinine levels were compared immediately before and after bosutinib administration and after bosutinib discontinuation in 11 patients with chronic myeloid leukemia. The median serum creatinine concentration significantly increased from 0.66 mg/dL before bosutinib to 0.76 mg/dL after bosutinib (P = 0.003) and decreased from 0.79 mg/dL before discontinuation of bosutinib to 0.66 mg/dL after discontinuation of bosutinib at 3 months (P = 0.005). This study revealed that bosutinib-induced elevation of serum creatinine, which was more pronounced in patients with the SLC22A2 808G/G genotype, does not indicate chronic kidney disease, but rather is simply a laboratory abnormality. If bosutinib-induced chronic kidney disease is suspected, renal function should be assessed by urinalysis and cystatin C levels to differentiate from simple elevation of serum creatinine.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"66-72"},"PeriodicalIF":1.7,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12202614/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143500764","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

JSH practical guidelines for hematological malignancies, 2023: II. Lymphoma5. Diffuse large B-cell lymphoma, not otherwise specified (DLBCL, NOS). 恶性血液病实用指南，2023:II。Lymphoma5。弥漫性大b细胞淋巴瘤，无其他特异性（DLBCL， NOS）。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2025-07-01 Epub Date: 2025-05-28 DOI: 10.1007/s12185-025-03997-z

Ken Ohmachi

引用次数: 0

Mechanism of antithrombin deficiency due to the novel variant C32W in the C-terminus of the signal peptide. 信号肽c端新变异C32W导致抗凝血酶缺乏的机制。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2025-07-01 Epub Date: 2025-02-10 DOI: 10.1007/s12185-025-03945-x

Yuika Kikuchi, Satomi Nagaya, Tomoki Togashi, Yuta Imai, Maki Togashi, Yuhei Araiso, Takumi Nishiuchi, Eriko Morishita

{"title":"Mechanism of antithrombin deficiency due to the novel variant C32W in the C-terminus of the signal peptide.","authors":"Yuika Kikuchi, Satomi Nagaya, Tomoki Togashi, Yuta Imai, Maki Togashi, Yuhei Araiso, Takumi Nishiuchi, Eriko Morishita","doi":"10.1007/s12185-025-03945-x","DOIUrl":"10.1007/s12185-025-03945-x","url":null,"abstract":"Introduction: We identified a novel variant of the antithrombin (AT) gene (SERPINC1), c.96 T>G, p.Cys32Trp (C32W), located at the signal peptide cleavage site in a patient with congenital AT deficiency. The impact of signal peptide variants on the intracellular trafficking and secretion of AT proteins has not been previously studied. Thus, we analyzed the intracellular dynamics and signal peptide cleavage of the C32W variant of AT (AT-C32W).Materials and methods: Wild-type AT (AT-WT) and AT-C32W expression vectors were transfected into HEK293 cells. Functional analyses were performed using western blotting and proteasome inhibition experiments. Signal peptide cleavage was evaluated by peptide sequencing.Results: The AT antigen levels in the cell lysates and culture supernatants of the AT-C32W were reduced to 3.8% and 4.8%, respectively. Following proteasome inhibition, the AT-C32W level increased to 71.5% of that for AT-WT. Peptide sequencing identified a fragment corresponding to the N-terminal end of the signal peptide exclusively in AT-C32W.Discussion: These results suggest that the signal peptide of AT-C32W is not cleaved properly, which causes intracellular degradation of AT-C32W by proteasomes that results in type I AT deficiency. Further studies on the intracellular dynamics of such variants may clarify the mechanisms underlying AT deficiency.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"35-44"},"PeriodicalIF":1.7,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143382330","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

Association between gene mutations and outcomes in Japanese high-risk AML patients: a phase 1/2 study of NS-87/CPX-351. 基因突变与日本高危AML患者预后之间的关系：NS-87/CPX-351的1/2期研究

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2025-07-01 Epub Date: 2025-02-27 DOI: 10.1007/s12185-025-03956-8

Hideki Makishima, Taisuke Mikasa, Kento Isogaya, Toshihiro Miyamoto, Takuji Yamauchi, Akira Yokota, Masahiro Onozawa, Kiyoshi Ando, Yoshiaki Ogawa, Kensuke Usuki, Takahiro Yamauchi, Shuichi Ota, Satoru Takada, Yasuyoshi Morita, Takayuki Ishikawa, Katsuto Takenaka, Junya Kuroda, Naohiro Sekiguchi, Toshiro Kawakita, Yasushi Miyazaki

{"title":"Association between gene mutations and outcomes in Japanese high-risk AML patients: a phase 1/2 study of NS-87/CPX-351.","authors":"Hideki Makishima, Taisuke Mikasa, Kento Isogaya, Toshihiro Miyamoto, Takuji Yamauchi, Akira Yokota, Masahiro Onozawa, Kiyoshi Ando, Yoshiaki Ogawa, Kensuke Usuki, Takahiro Yamauchi, Shuichi Ota, Satoru Takada, Yasuyoshi Morita, Takayuki Ishikawa, Katsuto Takenaka, Junya Kuroda, Naohiro Sekiguchi, Toshiro Kawakita, Yasushi Miyazaki","doi":"10.1007/s12185-025-03956-8","DOIUrl":"10.1007/s12185-025-03956-8","url":null,"abstract":"This phase 1/2 study investigated the association between genetic characteristics and outcomes for NS-87/CPX-351 in Japanese patients with high-risk acute myeloid leukemia. Blood samples collected from 29 patients were analyzed using a 70-gene next-generation sequencing panel. The most frequently mutated genes were TP53 (44.8%), TET2 (24.1%), DNMT3A (13.8%), and NRAS (13.8%). The rates of composite complete remission (CRc; complete remission [CR] or CR with incomplete hematologic recovery [CRi]) were comparable between patients with and without mutations in TP53, TET2, DNMT3A, and NRAS (P = 0.571 for all). Notably, patients with TP53 mutations had a similar CRc rate (69.2% vs. 56.3%), but shorter overall survival (OS), event-free survival (EFS), and relapse-free survival (RFS) compared to patients with wild-type TP53 (median OS: 7.43 vs. 18.18 months; P = 0.108, median EFS: 2.43 vs. 6.28 months; P = 0.012, median RFS: 1.48 vs. 10.19 months; P = 0.012). In conclusion, no gene mutations directly associated with the efficacy of NS-87/CPX-351 were found. While NS-87/CPX-351 achieved remission even in patients with TP53 mutations, relapse risk was higher in these patients. Therefore, it is advisable to consider treatment strategies such as early transplantation after achieving remission with NS-87/CPX-351, especially in patients with TP53 mutations.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"57-65"},"PeriodicalIF":1.7,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12202506/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143523479","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

Effects of tucidinostat in adult T-cell leukemia/lymphoma in clinical practice. 图西他汀治疗成人t细胞白血病/淋巴瘤的临床疗效。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2025-07-01 Epub Date: 2025-03-11 DOI: 10.1007/s12185-025-03963-9

Ayako Kamiunten, Takuro Kameda, Masaaki Sekine, Hiroshi Kawano, Takanori Toyama, Keiichi Akizuki, Noriaki Kawano, Kouichi Maeda, Seiichi Sato, Masanori Takeuchi, Junzo Ishizaki, Koshiro Nagamine, Ayuka Kuroki, Ryoma Ikeda, Kengo Matsumoto, Masayoshi Karasawa, Yuki Tahira, Taisuke Uchida, Haruko Shimoda, Tomonori Hidaka, Kiyoshi Yamashita, Hideki Yamaguchi, Yoko Kubuki, Kazuya Shimoda, Kotaro Shide

{"title":"Effects of tucidinostat in adult T-cell leukemia/lymphoma in clinical practice.","authors":"Ayako Kamiunten, Takuro Kameda, Masaaki Sekine, Hiroshi Kawano, Takanori Toyama, Keiichi Akizuki, Noriaki Kawano, Kouichi Maeda, Seiichi Sato, Masanori Takeuchi, Junzo Ishizaki, Koshiro Nagamine, Ayuka Kuroki, Ryoma Ikeda, Kengo Matsumoto, Masayoshi Karasawa, Yuki Tahira, Taisuke Uchida, Haruko Shimoda, Tomonori Hidaka, Kiyoshi Yamashita, Hideki Yamaguchi, Yoko Kubuki, Kazuya Shimoda, Kotaro Shide","doi":"10.1007/s12185-025-03963-9","DOIUrl":"10.1007/s12185-025-03963-9","url":null,"abstract":"Adult T-cell leukemia/lymphoma (ATL) is a peripheral T-cell malignancy with a poor prognosis. We conducted a retrospective study across six institutions in Miyazaki Prefecture, Japan, to assess the efficacy of tucidinostat in patients with relapsed/refractory ATL who had not undergone transplantation. Between October 2021 and July 2023, 24 patients aged 41 to 88 years (median, 73.4 years) who had undergone prior therapies, including intensive chemotherapy (79.2%) and mogamulizumab immunotherapy (79.2%), received tucidinostat. Objective response rate (ORR), disease control rate (DCR), progression-free survival (PFS), and overall survival (OS) were evaluated as key outcomes. ORR and DCR reached 54.2% and 91.7%, respectively. The median PFS was 3.95 months, and OS was 8.04 months, which were not inferior to the results of a phase IIb study. The influential factors for PFS were age ≥ 75 years and high soluble IL-2 receptor (sIL-2R) levels above 5000 U/mL at the start of treatment. Favorable patients without these factors achieved a PFS of 11.4 months. Treatment-related adverse events were mainly hematologic but were managed over the course of treatment. Our findings indicate that tucidinostat provides survival benefits in patients with relapsed/refractory ATL in clinical practice and highlight key clinical factors for better outcomes.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"83-92"},"PeriodicalIF":1.7,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12202688/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143604967","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