{"title":"Acute myeloid leukemia with NUP98::RARG rearrangement: a case report and review of the relevant literature.","authors":"Junki Inamura, Takeshi Taketani, Miho Mochida, Tsukimi Goto, Ritsuro Suzuki, Sho Igarashi, Nodoka Tsukada, Masayo Yamamoto, Motohiro Shindo, Kazuya Sato","doi":"10.1007/s12185-024-03881-2","DOIUrl":"10.1007/s12185-024-03881-2","url":null,"abstract":"<p><p>We herein report a rare case of acute myeloid leukemia (AML) with t(11;12)(p15;q13) and NUP98::RARG, which seems to be involved in the development of AML. The morphological features were similar to those of classic acute promyelocytic leukemia (APL), but unlike classic APL, this leukemia was resistant to treatment with all-trans retinoic acid (ATRA). We decided to use standard chemotherapy for AML with monitoring of minimal residual disease (MRD) by qualitative reverse transcriptase-polymerase chain reaction (RT-PCR) analysis for NUP98::RARG mRNA. Although MRD disappeared after induction chemotherapy, it later reappeared, and hematological relapse occurred during subsequent chemotherapies. The patient received haploidentical hematopoietic stem cell transplantation while not in remission and achieved a second molecular remission. However, relapse occurred 4 months after transplantation. The specific mechanism of ATRA resistance in this unique case of AML remains unclear, and no standard treatment has been determined. This is the first case report of AML with NUP98::RARG rearrangement in Japan. Qualitative RT-PCR analysis for NUP98::RARG mRNA was helpful for the accurate diagnosis and evaluation of MRD to choose an adequate treatment for this type of AML.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"265-271"},"PeriodicalIF":1.7,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142768687","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}
Feng Chen, Shi Qiu, Ailing Gui, Shiyu Jiang, Yichen Yan, Jichuan Wu, Guangliang Chen, Shun Zhu, Yizhen Liu, Zuguang Xia, Baohua Yu, Xiaojian Sun, Juan Jennifer Gu, Lan Wang, Wen Liu, Ling Yang, Qunling Zhang, Ji Zuo
{"title":"Reduced OTUD7B expression correlates with poor prognosis in PTCL via non-canonical NF-κB.","authors":"Feng Chen, Shi Qiu, Ailing Gui, Shiyu Jiang, Yichen Yan, Jichuan Wu, Guangliang Chen, Shun Zhu, Yizhen Liu, Zuguang Xia, Baohua Yu, Xiaojian Sun, Juan Jennifer Gu, Lan Wang, Wen Liu, Ling Yang, Qunling Zhang, Ji Zuo","doi":"10.1007/s12185-024-03877-y","DOIUrl":"10.1007/s12185-024-03877-y","url":null,"abstract":"<p><p>Peripheral T cell lymphoma (PTCL) is an aggressive and highly heterogeneous lymphoma with a bleak prognosis, highlighting the urgent need for an effective biomarker to guide therapeutic strategies. Ovarian tumor domain-containing 7B (OTUD7B) has been shown to have a critical function in the progression of cancers. However, the prognostic significance of OTUD7B in PTCL remains unexplored. In this study, we demonstrated for the first time that PTCL patients with low expression of OTUD7B had shorter progression-free survival (PFS) and overall survival (OS). In addition, OTUD7B knockdown promoted chemoresistance to doxorubicin in PTCL cell lines, and led to increased translocation of p52 from the cytoplasm to the nucleus. Inhibition of non-canonical NF-κB partially restored the sensitivity of PTCL cells to doxorubicin. Remarkably, 5-azacytidine and cytarabine upregulated the expression of OTUD7B and exhibited a synergistic anti-lymphoma effect in PTCL. In summary, our study confirmed the prognostic role of OTUD7B in PTCL and the promising therapeutic potential of combining 5-azacytidine or cytarabine and doxorubicin for PTCL treatment.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"194-205"},"PeriodicalIF":1.7,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142785647","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}
{"title":"DREAMM-11, Part 2: Japanese phase I trial of belantamab mafodotin combination therapies in relapsed/refractory multiple myeloma.","authors":"Kazutaka Sunami, Shinsuke Iida, Nobuhiro Tsukada, Taku Fujii, Hitomi Kato, Ryuichi Fukushima, Satoshi Wakabayashi, Hirofumi Nakano, Sumita Roy-Ghanta, Brandon E Kremer","doi":"10.1007/s12185-024-03889-8","DOIUrl":"10.1007/s12185-024-03889-8","url":null,"abstract":"<p><p>DREAMM-11 (NCT03828292) was a Phase 1, open-label, dose-escalation study of belantamab mafodotin in Japanese patients with relapsed/refractory multiple myeloma (RRMM). In Part 1, belantamab mafodotin monotherapy (2.5 or 3.4 mg/kg every 3 weeks) was tolerated and demonstrated clinical activity and a manageable safety profile. Part 2 investigated the tolerability, safety, clinical activity and pharmacokinetics of belantamab mafodotin (2.5 mg/kg on Day [D]1 of each 21-day cycle) plus bortezomib and dexamethasone (Arm A; N = 3) or belantamab mafodotin (2.5 mg/kg on D1 of the first 28-day cycle; 1.9 mg/kg on D1 of subsequent cycles) plus pomalidomide and dexamethasone (Arm B; N = 4) in Japanese patients with RRMM and ≥ 1 prior line of therapy. No dose-limiting toxicities were reported in Arm A; 1 (non-serious liver injury) was reported in Arm B. Safety profiles of each treatment combination were consistent with those of the individual agents and those in Western populations. An overall response was achieved by 3/3 (100%) patients in Arm A and 2/4 (50%) in Arm B. Pharmacokinetics were consistent between Japanese and Western populations. The clinical pharmacokinetics, safety, and efficacy data from this study can inform future use of belantamab mafodotin plus bortezomib/pomalidomide and dexamethasone in Japanese patients with RRMM.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"174-186"},"PeriodicalIF":1.7,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11782446/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142882054","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}
{"title":"Clinical significance of NOTCH1 and FBXW7 alterations in adult T-cell leukemia/lymphoma.","authors":"Yuma Sakamoto, Takashi Ishida, Ayako Masaki, Takayuki Murase, Eiichi Ohtsuka, Morishige Takeshita, Reiji Muto, Ilseung Choi, Hiromi Iwasaki, Asahi Ito, Shigeru Kusumoto, Nobuaki Nakano, Masahito Tokunaga, Kentaro Yonekura, Yukie Tashiro, Youko Suehiro, Shinsuke Iida, Atae Utsunomiya, Ryuzo Ueda, Hiroshi Inagaki","doi":"10.1007/s12185-024-03880-3","DOIUrl":"10.1007/s12185-024-03880-3","url":null,"abstract":"<p><p>Here, we investigated the clinical significance of NOTCH1 and FBXW7 alterations for adult T-cell leukemia/lymphoma (ATLL) treatment outcomes. NOTCH1 alterations were identified in 37 (14.4%) of 257 patients, of which 33 were single nucleotide variants/insertion-deletions in the PEST domain, and 7 were in the heterodimerization or LIN-12/Notch repeats domains. FBXW7 alterations were observed in nine ATLL patients (3.5%). For patients without allogeneic hematopoietic stem cell transplantation (HSCT), NOTCH1, but not FBXW7, alterations were significantly and independently associated with worse overall survival (median OS 0.5 years, 95% confidence interval [CI] 0.4-0.5 years for 27 patients with NOTCH1 alterations vs 1.8 years, 95% CI 1.3-2.2 years for 170 patients without). Also, for patients receiving mogamulizumab, but not allogeneic-HSCT, NOTCH1, but not FBXW7, alterations were significantly associated with worse survival (median survival from the first dose of mogamulizumab 0.4 years, 95% CI 0.3-0.5 years for 12 patients with NOTCH1 alterations vs 1.4 years, 95% CI 0.9-2.0 years for 87 without). In contrast, NOTCH1 alterations had no significant impact on survival of patients who did receive allogeneic-HSCT. Thus, mogamulizumab-containing treatment was unable to overcome treatment refractoriness of ATLL with NOTCH1 alterations. Therefore, patients with NOTCH1 alterations are recommended for allogeneic-HSCT.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"206-221"},"PeriodicalIF":1.7,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142716160","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}
Francesca Magnoli, Marco Brociner, Stefano La Rosa
{"title":"Nodal mature plasmacytoid dendritic cell proliferation mimicking lymphoma in a patient with CALR-mutated myelofibrosis.","authors":"Francesca Magnoli, Marco Brociner, Stefano La Rosa","doi":"10.1007/s12185-024-03902-0","DOIUrl":"10.1007/s12185-024-03902-0","url":null,"abstract":"","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"147-148"},"PeriodicalIF":1.7,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142909643","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}
{"title":"Outcomes of allogeneic SCT versus tisagenlecleucel in patients with R/R LBCL and poor prognostic factors.","authors":"Kenta Hayashino, Toshiki Terao, Hisakazu Nishimori, Wataru Kitamura, Hiroki Kobayashi, Chihiro Kamoi, Keisuke Seike, Hideaki Fujiwara, Noboru Asada, Daisuke Ennishi, Keiko Fujii, Nobuharu Fujii, Ken-Ichi Matsuoka, Yoshinobu Maeda","doi":"10.1007/s12185-024-03888-9","DOIUrl":"10.1007/s12185-024-03888-9","url":null,"abstract":"<p><p>This study investigated the efficacy of tisagenlecleucel (tisa-cel) and allogeneic hematopoietic stem cell transplantation (allo-SCT) for patients with relapsed and/or refractory (r/r) large B-cell lymphoma (LBCL) with poor prognostic factors, defined as performance status (PS) ≥ 2, multiple extranodal lesions (EN), chemorefractory disease, or higher lactate dehydrogenase (LDH). Overall, the allo-SCT group demonstrated worse progression-free survival (PFS), higher non-relapse mortality, and a similar relapse/progression rate. Notably, the tisa-cel group showed better PFS than the allo-SCT group among patients with chemorefractory disease (3.2 vs. 2.0 months, p = 0.092) or higher LDH (4.0 vs. 2.0 months, p = 0.018), whereas PFS in the two cellular therapy groups was similar among those with PS ≥ 2 or multiple EN. Survival time after relapse post-cellular therapy in patients with poor prognostic factors was 1.6 with allo-SCT and 4.6 months with tisa-cel. These findings were confirmed in a propensity score matching cohort. In conclusion, tisa-cel resulted in better survival than allo-SCT in patients with poor prognostic factors. However, patients who relapsed post-cellular therapy had dismal outcomes regardless of therapy. Further strategies are warranted to improve outcomes in these patients.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"232-243"},"PeriodicalIF":1.7,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11782353/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142828403","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}
Tsuyoshi Muta, Yosuke Masamoto, Go Yamamoto, Shingo Kurahashi, Yoshihiro Kameoka, Shuichi Ota, Eri Matsuki, Kazutaka Ozeki, Takanori Toyama, Naoki Takahashi, Takahiro Kumode, Nobuyuki Aotsuka, Takuro Yoshimura, Hideto Tamura, Ai Omi, Kazuhiro Shibayama, Aki Watanabe, Yasushi Isobe, Kensuke Kojima, Jun Takizawa, Hirokazu Nagai, Junji Suzumiya, Sadao Aoki
{"title":"Real-world effectiveness and safety of ibrutinib in patients with chronic lymphocytic leukemia in Japan: the Orbit study.","authors":"Tsuyoshi Muta, Yosuke Masamoto, Go Yamamoto, Shingo Kurahashi, Yoshihiro Kameoka, Shuichi Ota, Eri Matsuki, Kazutaka Ozeki, Takanori Toyama, Naoki Takahashi, Takahiro Kumode, Nobuyuki Aotsuka, Takuro Yoshimura, Hideto Tamura, Ai Omi, Kazuhiro Shibayama, Aki Watanabe, Yasushi Isobe, Kensuke Kojima, Jun Takizawa, Hirokazu Nagai, Junji Suzumiya, Sadao Aoki","doi":"10.1007/s12185-024-03875-0","DOIUrl":"10.1007/s12185-024-03875-0","url":null,"abstract":"<p><p>Ibrutinib is a first-in-class Bruton's tyrosine kinase inhibitor that is approved for the treatment of chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) in Japan based on randomized clinical trial data. The aim of the real-world, retrospective Orbit study was to describe long-term clinical outcomes and management in adults (aged ≥ 20 years) with CLL/SLL treated with ibrutinib, either as first-line (1L) treatment or for relapsed or refractory (RR) disease, in routine clinical practice in Japan between July 2018 and December 2020. A total of 246 patients were registered, and the safety and per-protocol sets included 237 and 234 patients, respectively. After a median follow-up of 35.7 months, the 36-month progression-free survival rate was 80.9% in the 1L CLL cohort and 67.2% in the RR CLL cohort, and the 36-month overall survival rates were 90.8% and 83.7%, respectively. Common Terminology Criteria for Adverse Events (CTCAE) Grade ≥ 3 adverse events of special interest were atrial fibrillation (2.1%), infections (herpesvirus infection, fungal infection, or Pneumocystis jiroveci pneumonia; 1.7%), bleeding (3.8%), and second primary malignancy (2.5%). These findings confirm the long-term, real-world effectiveness and safety of ibrutinib for the treatment of Japanese patients with newly diagnosed or RR CLL/SLL.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"161-173"},"PeriodicalIF":1.7,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11782388/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142716193","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}
{"title":"Comparison of busulfan pharmacokinetics between four-times-daily and once-daily administration in pediatric patients: a preliminary prospective observational trial.","authors":"Atsushi Yamaguchi, Shinsuke Hirabayashi, Kazuko Niki, Keisuke Kagami, Yukayo Terashita, Yuko Cho, Atsushi Manabe, Mitsuru Sugawara, Yoh Takekuma","doi":"10.1007/s12185-024-03891-0","DOIUrl":"10.1007/s12185-024-03891-0","url":null,"abstract":"<p><p>Therapeutic drug monitoring (TDM) of busulfan (BU) is useful for achieving the target area under the curve (AUC) because its effective plasma-concentration range is narrow. This preliminary observational study evaluated the pharmacokinetic (PK) parameters of BU administered four times daily or once daily to pediatric patients. The plasma concentrations were measured at both the test dose and first dose, and the doses on day 1 and days 2-4 were determined based on each TDM. A comparison of PK parameters between four-times-daily and once-daily administration was performed for both the test dose and first dose of BU. Of the 11 patients, five received four-times-daily BU and six received once-daily BU. The V<sub>d</sub> for once-daily administration was higher than that for four-times-daily administration for the first dose but not for the test dose. The ratio of actual AUC for the first dose to estimated AUC guided by the test dose was lower with once-daily administration than with four-times-daily administration. These results indicate that the PK parameters of BU administered once daily are challenging to predict based on the TDM of the test dose. TDM should be considered on day 1 to achieve the target AUC, especially with once-daily administration.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"244-251"},"PeriodicalIF":1.7,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142768688","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}
{"title":"Assessment and management of pregnancy in patients with myeloproliferative neoplasms: insights from a single-institution study of 29 neonates.","authors":"Yoko Edahiro, Shuichi Shirane, Jun Takeda, Hajime Yasuda, Tadaaki Inano, Miyuki Tsutsui, Yasuharu Hamano, Makoto Sasaki, Jun Ando, Atsuo Itakura, Miki Ando, Norio Komatsu","doi":"10.1007/s12185-024-03893-y","DOIUrl":"10.1007/s12185-024-03893-y","url":null,"abstract":"","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"281-283"},"PeriodicalIF":1.7,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142785645","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}
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