International Journal of Hematology最新文献

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KMT2A-CBL fusion gene in the first reported case of T-cell acute lymphoblastic leukemia associated with Wiedemann-Steiner syndrome. KMT2A-CBL融合基因在首例报道的与Wiedemann-Steiner综合征相关的t细胞急性淋巴细胞白血病中的应用
IF 1.7 4区 医学
International Journal of Hematology Pub Date : 2025-03-28 DOI: 10.1007/s12185-025-03975-5
Akihiro Nishimura, Akihiro Tamura, Tomoko Fujikawa, Shotaro Inoue, Naoko Nakatani, Kandai Nozu, Nobuyuki Yamamoto
{"title":"KMT2A-CBL fusion gene in the first reported case of T-cell acute lymphoblastic leukemia associated with Wiedemann-Steiner syndrome.","authors":"Akihiro Nishimura, Akihiro Tamura, Tomoko Fujikawa, Shotaro Inoue, Naoko Nakatani, Kandai Nozu, Nobuyuki Yamamoto","doi":"10.1007/s12185-025-03975-5","DOIUrl":"https://doi.org/10.1007/s12185-025-03975-5","url":null,"abstract":"<p><p>Wiedemann-Steiner syndrome (WSS) is a congenital malformation syndrome characterized by intellectual disability, developmental delay, and distinctive facial features, caused by germline mutations in the KMT2A gene. Despite the key role of KMT2A in hematopoiesis, leukemia has not been previously reported in WSS patients. This report presents the first documented case of acute lymphoblastic leukemia (ALL) in a WSS patient. A 16-year-old boy with developmental delay, distinct facial features, and genital abnormalities was diagnosed with WSS following the identification of a heterozygous frameshift mutation in KMT2A. At age 17, he developed T-cell ALL harboring the KMT2A-CBL fusion gene, of which only nine cases have been reported so far. cDNA sequence analysis of the KMT2A-CBL transcript at the site of the germline KMT2A pathogenic variant revealed a wild-type sequence, indicating that the KMT2A-CBL fusion occurred on the wild-type allele. While this observation suggests a potential cooperative role of the KMT2A-CBL chimeric gene and the germline KMT2A pathogenic mutation in leukemogenesis, the rarity of leukemia in WSS underscores the need for cautious interpretation. This case provides preliminary insights into a possible mechanism of leukemogenesis in WSS, but further studies are required to clarify the relationship between WSS and ALL.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.7,"publicationDate":"2025-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143735819","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
Oral beclomethasone dipropionate therapy and prognostic plasma biomarkers for gastrointestinal graft-versus-host disease. 口服二丙酸倍氯米松治疗和胃肠道移植物抗宿主病的预后血浆生物标志物
IF 1.7 4区 医学
International Journal of Hematology Pub Date : 2025-03-25 DOI: 10.1007/s12185-025-03973-7
Yoshihiro Inamoto, Ayumu Ito, Toshihisa Nakashima, Asako Usui, Wataru Takeda, Takashi Tanaka, Sung-Won Kim, Shigehisa Kitano, Keisuke Watanabe, Kana Kusaba, Yu Aruga, Chiaki Ikeda, Minoru Kojima, Naoki Maezawa, Hirotaka Matsui, Hironobu Hashimoto, Chitose Ogawa, Takahiro Fukuda
{"title":"Oral beclomethasone dipropionate therapy and prognostic plasma biomarkers for gastrointestinal graft-versus-host disease.","authors":"Yoshihiro Inamoto, Ayumu Ito, Toshihisa Nakashima, Asako Usui, Wataru Takeda, Takashi Tanaka, Sung-Won Kim, Shigehisa Kitano, Keisuke Watanabe, Kana Kusaba, Yu Aruga, Chiaki Ikeda, Minoru Kojima, Naoki Maezawa, Hirotaka Matsui, Hironobu Hashimoto, Chitose Ogawa, Takahiro Fukuda","doi":"10.1007/s12185-025-03973-7","DOIUrl":"https://doi.org/10.1007/s12185-025-03973-7","url":null,"abstract":"<p><p>The real-world outcomes of oral beclomethasone dipropionate (BDP) for gastrointestinal graft-versus-host disease (GVHD) were evaluated in a single-center, prospective, observational study of 167 patients who developed histologically confirmed gastrointestinal GVHD. The median patient age was 55 years (range 10-71). The initial GVHD grade was mostly IIa (n = 138). BDP was used without systemic corticosteroids in 73 patients (44%), resulting in a decreased proportion of patients who received systemic corticosteroid administration from 76 to 58% (P = 0.001). The 4-week gastrointestinal response rate after BDP therapy, the primary endpoint, was 73% (95% CI 66-80%) compared with 68% (95% CI 55-78%) before BDP implementation. The proportion of patients with maximum gastrointestinal stage ≥ 2 was lower after than before BDP implementation (18% versus 35%, respectively, P = 0.004). The 1 year cumulative incidence of nonrelapse mortality (NRM) after gastrointestinal GVHD therapy was 15% after and 22% before BDP implementation (P = 0.12). The 4-week gastrointestinal response rate was lower in patients with elevated ST2 or REG3α levels than the remaining patients (36% versus 73%, P = 0.03). The 1 year NRM was higher in patients with elevated ST2 or ANG2 levels than the remaining patients (64% versus 12%, P < 0.001). This study characterized the outcomes of BDP therapy in real-world patients.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.7,"publicationDate":"2025-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143709695","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
JSH practical guidelines for hematological malignancies, 2023: II. Lymphoma 4. Mantle cell lymphoma (MCL). 恶性血液病实用指南,2023:II。淋巴瘤4。套细胞淋巴瘤(MCL)。
IF 1.7 4区 医学
International Journal of Hematology Pub Date : 2025-03-24 DOI: 10.1007/s12185-025-03960-y
Kenichi Ishizawa, Kazuhito Yamamoto
{"title":"JSH practical guidelines for hematological malignancies, 2023: II. Lymphoma 4. Mantle cell lymphoma (MCL).","authors":"Kenichi Ishizawa, Kazuhito Yamamoto","doi":"10.1007/s12185-025-03960-y","DOIUrl":"https://doi.org/10.1007/s12185-025-03960-y","url":null,"abstract":"","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.7,"publicationDate":"2025-03-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143700260","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
Early cardiotoxicity in post-transplant cyclophosphamide-based graft-versus-host disease prophylaxis after HLA-haploidentical hematopoietic stem cell transplantation. hla -单倍体造血干细胞移植后以环磷酰胺为基础的移植物抗宿主病预防的早期心脏毒性
IF 1.7 4区 医学
International Journal of Hematology Pub Date : 2025-03-23 DOI: 10.1007/s12185-025-03970-w
Toshihiro Matsukawa, Junichi Sugita, Daigo Hashimoto, Masayuki Aiba, Kohei Okada, Takanori Teshima
{"title":"Early cardiotoxicity in post-transplant cyclophosphamide-based graft-versus-host disease prophylaxis after HLA-haploidentical hematopoietic stem cell transplantation.","authors":"Toshihiro Matsukawa, Junichi Sugita, Daigo Hashimoto, Masayuki Aiba, Kohei Okada, Takanori Teshima","doi":"10.1007/s12185-025-03970-w","DOIUrl":"https://doi.org/10.1007/s12185-025-03970-w","url":null,"abstract":"<p><strong>Introduction: </strong>Post-transplant cyclophosphamide (PTCy)-based prophylaxis for graft-versus-host disease (GVHD) is widely used in HLA-haploidentical hematopoietic cell transplantation (haplo-HCT). One of the major drawbacks of PTCy is the risk of rare but potentially lethal cardiotoxicity, which prompted the development of regimens with reduced doses of PTCy.</p><p><strong>Methods: </strong>We retrospectively compared the incidence of early cardiotoxicity with standard-dose of PTCy (cyclophosphamide 50 mg/kg/day for 2 days, PTCy100) versus reduced-dose (40 mg/kg/day for 2 days, PTCy80). In total, 179 patients underwent PTCy-based haplo-HCT, including PTCy100 (n = 111) and PTCy80 (n = 68).</p><p><strong>Results: </strong>The PTCy80 group included significantly more elderly patients, patients who received reduced-intensity conditioning, and patients with a history of HCT than the PTCy100 group. Nine eligible patients (5.0%) experienced severe cardiotoxicity. The incidence of severe cardiotoxicity did not differ significant between PTCy80 and PTCy100 (4.4% vs. 5.4%; p = 1). The incidence of fatal cardiotoxicity was lower with PTCy80, but the small size may have prevented the difference from reaching statistical significance.</p><p><strong>Conclusion: </strong>Reducing the cyclophosphamide dose in PTCy-based GVHD prophylaxis may lower the risk of fatal cardiotoxicity without significantly altering the overall incidence of severe cardiotoxicity.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.7,"publicationDate":"2025-03-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143692048","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
DLI-induced remission in recurrent EBV-associated T/NK-cell lymphoproliferative disease following HSCT: a case report. HSCT后复发ebv相关T/ nk细胞淋巴增生性疾病dli诱导缓解:1例报告
IF 1.7 4区 医学
International Journal of Hematology Pub Date : 2025-03-21 DOI: 10.1007/s12185-025-03965-7
Toru Nagata, Hirohito Kubota, Itaru Kato, Kazushi Izawa, Ryo Akazawa, Tomoyasu Jo, Yasuyuki Arai, Shohei Azumi, Tatsuya Kamitori, Satoshi Saida, Katsutsugu Umeda, Ken-Ichi Imadome, Takahiro Yasumi, Hidefumi Hiramatsu, Junko Takita
{"title":"DLI-induced remission in recurrent EBV-associated T/NK-cell lymphoproliferative disease following HSCT: a case report.","authors":"Toru Nagata, Hirohito Kubota, Itaru Kato, Kazushi Izawa, Ryo Akazawa, Tomoyasu Jo, Yasuyuki Arai, Shohei Azumi, Tatsuya Kamitori, Satoshi Saida, Katsutsugu Umeda, Ken-Ichi Imadome, Takahiro Yasumi, Hidefumi Hiramatsu, Junko Takita","doi":"10.1007/s12185-025-03965-7","DOIUrl":"https://doi.org/10.1007/s12185-025-03965-7","url":null,"abstract":"<p><strong>Background: </strong>Epstein-Barr virus (EBV)-associated T/natural killer (NK)-cell lymphoproliferative diseases (T/NK-LPDs) are characterized by clonal proliferation of EBV-infected T or NK cells, and the only curative treatment for progressive T/NK-LPDs is allogeneic hematopoietic stem cell transplantation (HSCT). There are limited reports concerning optimal management strategies for disease relapse after HSCT in patients with EBV-associated T/NK-LPDs.</p><p><strong>Case presentation: </strong>A 2-year-old girl was diagnosed with severe mosquito bite allergy, a cutaneous form of EBV-associated T/NK-LPD. Following HSCT from her human leukocyte antigen-matched sister, complete donor-type chimerism was achieved and whole blood EBV-DNA load became undetectable. However, the whole blood EBV-DNA load became elevated again and donor type chimerism decreased. Donor lymphocyte infusion (DLI) was performed to control the disease relapse. A complete virological response and complete donor-type chimerism were attained after four doses of DLI. No adverse effects were observed during the treatment course.</p><p><strong>Conclusions: </strong>This case highlights the potential of DLI as an alternative therapeutic approach to re-transplantation for recurrent EBV-associated T/NK-LPDs.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.7,"publicationDate":"2025-03-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143673841","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 effectiveness of ixazomib, lenalidomide and dexamethasone in Asians with relapsed/refractory multiple myeloma. 伊唑唑米、来那度胺和地塞米松在亚洲复发/难治性多发性骨髓瘤患者中的实际疗效
IF 1.7 4区 医学
International Journal of Hematology Pub Date : 2025-03-20 DOI: 10.1007/s12185-025-03927-z
Soo Chin Ng, Joon-Ho Moon, Sung Soo Park, Youngil Koh, Ji Hyun Lee, Hyeon-Seok Eom, Ho-Jin Shin, Sung Hoon Jung, Young Rok Do, Gilbert Wilfred, Azlan Husin, Hyo Jung Kim, SFadilah Abdul Wahid, Myung-Won Lee, Hye-Won Heo, Kihyun Kim, Suporn Chuncharunee
{"title":"Real-world effectiveness of ixazomib, lenalidomide and dexamethasone in Asians with relapsed/refractory multiple myeloma.","authors":"Soo Chin Ng, Joon-Ho Moon, Sung Soo Park, Youngil Koh, Ji Hyun Lee, Hyeon-Seok Eom, Ho-Jin Shin, Sung Hoon Jung, Young Rok Do, Gilbert Wilfred, Azlan Husin, Hyo Jung Kim, SFadilah Abdul Wahid, Myung-Won Lee, Hye-Won Heo, Kihyun Kim, Suporn Chuncharunee","doi":"10.1007/s12185-025-03927-z","DOIUrl":"https://doi.org/10.1007/s12185-025-03927-z","url":null,"abstract":"<p><p>Randomized clinical trials have shown ixazomib, lenalidomide and dexamethasone (IRd) to be efficacious and safe in Asian patients with relapsed/refractory multiple myeloma (RRMM); however, real-world data are limited. The APEX study was a multicenter, observational cohort study of IRd conducted at 16 sites across South Korea, Malaysia, and Thailand. Overall, 104 patients treated with IRd during 2016-2023 were enrolled; data were collected by retrospective chart review and 6-month prospective follow-up. Median age at IRd initiation was 64.0 years. The primary endpoints of median time to next treatment (TTNT) and overall response rate (ORR) were 32.1 months and 72.1%, respectively (though ORR varied across countries). The secondary endpoint of median progression-free survival was 27.7 months, while median overall survival was not reached. Median TTNT and ORR were higher in elderly patients (≥65 and/or ≥70 years) than in the overall population. Adverse events occurred in 90.4% and serious adverse events occurred in 29.8% of all patients; common Grade ≥ 3 adverse drug reactions were pneumonia (9.6%), neutropenia (7.7%), and gastroenteritis (2.9%). This study demonstrated that IRd was safe and effective in real-world practice in Asia, including for elderly patients, and the results are aligned with TOURMALINE-MM1 and other real-world studies.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.7,"publicationDate":"2025-03-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143669762","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
Engraftment outcome of patients with anti-HLA antibodies in HLA-mismatched peripheral blood stem cell transplantation. hla不匹配外周血干细胞移植中抗hla抗体患者的移植结果。
IF 1.7 4区 医学
International Journal of Hematology Pub Date : 2025-03-20 DOI: 10.1007/s12185-025-03952-y
Takeshi Hagino, Kazuhiro Ikegame, Hidenori Tanaka, Yoshinobu Kanda, Katsuji Kaida, Takahiro Fukuda, Yukio Kondo, Maho Sato, Noriko Doki, Hirohisa Nakamae, Ken-Ichi Matsuoka, Yasuo Mori, Hideki Sano, Tetsuya Eto, Toshiro Kawakita, Yoshiko Hashii, Tatsuo Ichinohe, Yoshiko Atsuta, Junya Kanda
{"title":"Engraftment outcome of patients with anti-HLA antibodies in HLA-mismatched peripheral blood stem cell transplantation.","authors":"Takeshi Hagino, Kazuhiro Ikegame, Hidenori Tanaka, Yoshinobu Kanda, Katsuji Kaida, Takahiro Fukuda, Yukio Kondo, Maho Sato, Noriko Doki, Hirohisa Nakamae, Ken-Ichi Matsuoka, Yasuo Mori, Hideki Sano, Tetsuya Eto, Toshiro Kawakita, Yoshiko Hashii, Tatsuo Ichinohe, Yoshiko Atsuta, Junya Kanda","doi":"10.1007/s12185-025-03952-y","DOIUrl":"https://doi.org/10.1007/s12185-025-03952-y","url":null,"abstract":"<p><p>Anti-human leukocyte antigen (HLA) antibodies, particularly donor-specific HLA antibodies (DSA), negatively impact engraftment in hematopoietic cell transplantation. Past studies have proposed various interventions to reduce DSA, but these were primarily from single centers and not from large-scale registry data. Therefore, we conducted a retrospective analysis of nationwide registry data to examine the effects of anti-HLA antibodies on engraftment. Evaluable patients were classified into an anti-HLA antibody-negative group (n = 3657), an anti-HLA antibody-positive group (without high DSA) (n = 137), and a high-DSA (MFI > 5000) group (n = 8). Patient characteristics differed significantly between the anti-HLA antibody-negative and anti-HLA antibody-positive groups, and the number of patients with DSA was lower than expected. Statistical analyses revealed that the anti-HLA antibody-positive group had better neutrophil engraftment than the anti-HLA antibody-negative group (94.0% vs 84.2%, p < 0.001) but worse platelet engraftment (60.3% vs 64.9%, p = 0.047). In the high DSA group, two patients received a DSA-depleting intervention. Only one patient with an MFI of 5832 (without intervention) developed primary graft failure, while the remaining seven achieved engraftment. In this study, the effect of anti-HLA antibodies remained inconclusive, and the possibility of neutrophil engraftment with high-DSA levels was confirmed.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.7,"publicationDate":"2025-03-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143669760","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
Late-onset radiation necrosis of the larynx attributed to hydroxyurea taken for essential thrombocythemia. 因服用羟基脲治疗原发性血小板增多症而导致晚期喉部放射性坏死。
IF 1.7 4区 医学
International Journal of Hematology Pub Date : 2025-03-17 DOI: 10.1007/s12185-025-03974-6
Masahiro Tokunaga, Akiyuki Yamato, Hidenori Inohara, Tetsuo Maeda
{"title":"Late-onset radiation necrosis of the larynx attributed to hydroxyurea taken for essential thrombocythemia.","authors":"Masahiro Tokunaga, Akiyuki Yamato, Hidenori Inohara, Tetsuo Maeda","doi":"10.1007/s12185-025-03974-6","DOIUrl":"https://doi.org/10.1007/s12185-025-03974-6","url":null,"abstract":"","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.7,"publicationDate":"2025-03-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143648281","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
JSH Practical Guidelines for Hematological Malignancies, 2023: II. Lymphoma-2. Marginal zone lymphoma: MZL (extranodal marginal zone lymphoma: EMZL [extranodal marginal zone lymphoma of mucosa associated lymphoid tissue], nodal marginal zone lymphoma: NMZL, splenic marginal zone lymphoma: SMZL). 2023 年血液恶性肿瘤实用指南》:II.淋巴瘤-2。边缘区淋巴瘤:MZL(结节外边缘区淋巴瘤:EMZL[粘膜相关淋巴组织结外边缘区淋巴瘤],结节边缘区淋巴瘤:结节边缘区淋巴瘤:NMZL;脾边缘区淋巴瘤:SMZL):SMZL)。
IF 1.7 4区 医学
International Journal of Hematology Pub Date : 2025-03-17 DOI: 10.1007/s12185-025-03947-9
Isao Yoshida
{"title":"JSH Practical Guidelines for Hematological Malignancies, 2023: II. Lymphoma-2. Marginal zone lymphoma: MZL (extranodal marginal zone lymphoma: EMZL [extranodal marginal zone lymphoma of mucosa associated lymphoid tissue], nodal marginal zone lymphoma: NMZL, splenic marginal zone lymphoma: SMZL).","authors":"Isao Yoshida","doi":"10.1007/s12185-025-03947-9","DOIUrl":"https://doi.org/10.1007/s12185-025-03947-9","url":null,"abstract":"","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.7,"publicationDate":"2025-03-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143648264","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
Favorable outcomes of de novo advanced phases of pediatric chronic myeloid leukemia in the tyrosine kinase inhibitor era. 酪氨酸激酶抑制剂时代儿童慢性髓性白血病晚期新生患者的有利结局。
IF 1.7 4区 医学
International Journal of Hematology Pub Date : 2025-03-15 DOI: 10.1007/s12185-025-03953-x
Toshihide Yoshikawa, Hisashi Ishida, Akihiro Watanabe, Yuki Yuza, Haruko Shima, Masaki Ito, Yukari Sakurai, Dai Keino, Takuya Ichimura, Keisuke Kato, Yuko Osugi, Shosuke Sunami, Kunihiro Shinoda, Toshihiko Imamura, Katsuyoshi Koh, Yuri Okimoto, Chikako Tono, Hiroyuki Shimada, Akihiko Tanizawa
{"title":"Favorable outcomes of de novo advanced phases of pediatric chronic myeloid leukemia in the tyrosine kinase inhibitor era.","authors":"Toshihide Yoshikawa, Hisashi Ishida, Akihiro Watanabe, Yuki Yuza, Haruko Shima, Masaki Ito, Yukari Sakurai, Dai Keino, Takuya Ichimura, Keisuke Kato, Yuko Osugi, Shosuke Sunami, Kunihiro Shinoda, Toshihiko Imamura, Katsuyoshi Koh, Yuri Okimoto, Chikako Tono, Hiroyuki Shimada, Akihiko Tanizawa","doi":"10.1007/s12185-025-03953-x","DOIUrl":"https://doi.org/10.1007/s12185-025-03953-x","url":null,"abstract":"<p><p>Chronic myeloid leukemia (CML) is a rare disease during childhood, and accelerated phase (AP) and blast phase (BP) CML, also called advanced phases, are even rarer. We retrospectively collected and analyzed clinical data of children younger than 20 years with de novo advanced-phase CML between 1996 and 2017 in Japan. Median follow-up time was 8.9 years for AP-CML (n = 15) and 3.7 years for BP-CML (n = 32). The 5-year overall survival (OS) was 93.3% for AP-CML, and 100.0% for patients who received tyrosine kinase inhibitors (TKIs) in first-line therapy (n = 10). Four of the ten patients who received TKIs in first-line therapy remained in molecular remission without transplantation (median follow-up 5.5 years). The 5-year OS of patients with BP-CML was 79.0%, and most patients received chemotherapy before transplantation, with regimen selection based on blast immunophenotype. Furthermore, among patients who received transplantation after TKI therapy, the 5-year OS was 100.0% for AP and 84.8% for BP. In conclusion, our study confirmed excellent outcomes in children with de novo advanced-phase CML, especially in the TKI-era.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.7,"publicationDate":"2025-03-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143633625","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
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