{"title":"Outcomes of allogeneic hematopoietic stem cell transplantation for acquired pure red cell aplasia.","authors":"Yuma Noguchi, Takehiko Mori, Yasushi Onishi, Yukinori Nakamura, Minoru Kanaya, Shinichi Ito, Kosei Matsue, Shingo Yano, Makoto Onizuka, Tetsuya Nishida, Seitaro Terakura, Satoshi Yoshihara, Takeshi Maeda, Tatsuo Ichinohe, Yoshiko Atsuta, Katsuto Takenaka","doi":"10.1007/s12185-025-04137-3","DOIUrl":"10.1007/s12185-025-04137-3","url":null,"abstract":"<p><p>Acquired pure red cell aplasia (PRCA) affects only the red blood cell lineage and responds to immunosuppressive therapy. Few reports describe the outcome of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for refractory PRCA. This study investigated the outcomes of allo-HSCT for PRCA using Japanese nationwide registry data. Eight patients aged 16 years or older who underwent allo-HSCT for acquired PRCA were analyzed. Median ages at diagnosis of PRCA and HSCT were 39.5 (range 19-68) and 47.5 (range 21-68) years, respectively. Stem cell sources and donors were bone marrow (related, n = 2; unrelated, n = 3), umbilical cord blood (n = 2), and peripheral blood stem cells (related, n = 1). All patients achieved neutrophil engraftment with a median time to engraftment of 21 days. Four patients died within 3 months after HSCT; the causes of death were acute myocardial infarction, thrombotic microangiopathy, capillary leak syndrome, and bacterial pneumonia. The remaining four patients were alive and transfusion-independent at a median follow-up of 99.4 months (range 21-148). Allo-HSCT could be a curative treatment option for refractory PRCA. However, early post-transplant mortality is an obstacle, and thus, optimal transplant procedures should be carefully determined for each patient.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"600-606"},"PeriodicalIF":1.8,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145810148","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":"Large atypical cells with foamy cytoplasm in bone marrow in primary splenic angiosarcoma: a case report.","authors":"Takehiro Okuda, Naomi Katsuki, Yasuko Miyahara","doi":"10.1007/s12185-026-04186-2","DOIUrl":"10.1007/s12185-026-04186-2","url":null,"abstract":"","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"471-472"},"PeriodicalIF":1.8,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147305496","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":"Genome-epigenome crosstalk in T-cell lymphomas: from maps to mechanisms.","authors":"Makoto Yamagishi","doi":"10.1007/s12185-025-04115-9","DOIUrl":"10.1007/s12185-025-04115-9","url":null,"abstract":"<p><p>T-cell lymphomas are clinically and biologically heterogeneous malignancies that comprise ~ 10% of non-Hodgkin lymphomas. Outcomes with first-line chemotherapy remain poor. Over the past decade, integrative genomic and epigenomic studies have defined recurrent abnormalities converging on proximal T-cell antigen receptor/costimulatory signaling to the NF-κB/NFAT, JAK/STAT, PI3K/AKT/mTOR, and NOTCH pathways, alongside pervasive alterations in chromatin modifiers and the DNA methylation machinery. In this review, we frame the biology of peripheral T-cell lymphoma as two interdependent layers, including genetic events that establish constitutive signaling programs and epigenomic remodeling that stabilizes these outputs. We overview genomic alterations across major peripheral T-cell lymphoma entities and analyze epigenomic dysregulation, focusing on DNA methylation, enhancer regulation, and polycomb-mediated gene control. We highlight adult T-cell leukemia/lymphoma as a paradigmatic dual-layer disease, summarize therapeutic approaches based on epigenetic traits, and discuss biomarker-guided strategies and challenges in translating integrated maps into durable disease control.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"487-495"},"PeriodicalIF":1.8,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13083410/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145603977","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":"CYCS-related thrombocytopenia in three Japanese families with a novel variant in one family.","authors":"Natsuhiko Yamada, Atsushi Sakamoto, Rintaro Nagoshi, Saori Endo, Masaki Yamamoto, Shoji Saito, Yuji Yamada, Toru Uchiyama, Kumiko Yanagi, Tadashi Kaname, Shinji Kunishima, Akira Ishiguro","doi":"10.1007/s12185-025-04145-3","DOIUrl":"10.1007/s12185-025-04145-3","url":null,"abstract":"<p><p>CYCS encodes somatic cytochrome c, which plays a central role in the electron transport chain and oxidative phosphorylation. We report nine cases of autosomal dominant, non-syndromic CYCS-related thrombocytopenia in three Japanese families. Patients showed no or mild bleeding tendency, with preserved platelet size and morphology, and without other abnormalities in blood cell lineages. Using targeted gene sequencing for congenital thrombocytopenia, we identified pathogenic missense variants in CYCS, shared by all affected individuals in each family. Two were previously reported: c.274A > G,p.(Arg92Gly) and c.309C > T,p.(Thr103Ile); one was novel: c.212A > C,p.(Asn71Thr). Genetic testing should be considered in unexplained familial thrombocytopenia.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"611-616"},"PeriodicalIF":1.8,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145846594","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":"Cost-effectiveness analysis of ropeginterferon alfa-2b for the management of patients with polycythemia vera in Japan.","authors":"Hiroki Yamaguchi, Yuka Sugimoto, Akihiko Gotoh, Shuichi Ota, Hiromi Igari, Tatsunori Murata, Keigo Hanada, Norio Komatsu, Keita Kirito","doi":"10.1007/s12185-025-04136-4","DOIUrl":"10.1007/s12185-025-04136-4","url":null,"abstract":"<p><strong>Objective: </strong>To verify the clinical benefit of ropeginterferon alfa-2b (ropegIFN) and evaluate its cost-effectiveness compared with hydroxycarbamide or ruxolitinib for patients with polycythemia vera (PV) under the National Health Insurance (NHI) system in Japan.</p><p><strong>Methods: </strong>The cost-effectiveness of ropegIFN compared with hydroxycarbamide or ruxolitinib was evaluated for patients with PV requiring cytoreductive therapy (without prior cytoreductive therapy) and resistant or intolerant to hydroxycarbamide. According to previous reports, patients with PV who achieve a molecular response with a JAK2V617F allele burden < 10% tend to maintain hematologic responses even after discontinuing interferon treatment. Therefore, in these analyses, patients who achieved a molecular response with JAK2V617F burden < 10% discontinued ropegIFN treatment.</p><p><strong>Results: </strong>Compared with hydroxycarbamide, ropegIFN yielded an incremental effectiveness of 0.440 quality-adjusted life years (QALYs), indicating a higher QALY gain. The incremental costs were 128,001,730 yen, and the incremental cost-effectiveness ratio (ICER) was 291,092,030 yen/QALY. Compared with ruxolitinib, the incremental effectiveness of ropegIFN was 1.278 QALYs, while the total costs were reduced by 18,025,182 yen, which resulted in a dominant ICER.</p><p><strong>Conclusions: </strong>These results suggest that ropegIFN may be cost-effective compared with ruxolitinib in patients with PV who are resistant or intolerant to hydroxycarbamide under the NHI system in Japan.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"556-569"},"PeriodicalIF":1.8,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13083320/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145819393","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":"Talquetamab in Japanese patients with relapsed/refractory multiple myeloma in the MonumenTAL-1 study.","authors":"Shigeki Ito, Yoshiaki Kuroda, Kazutaka Sunami, Kosei Matsue, Kazunori Imada, Hideto Tamura, Ei Fujikawa, Hiroshi Yamazaki, Mikihiro Takamoto, Lixia Pei, Xiang Qin, Tara J Masterson, Michela Campagna, Veronique Vreys, Bonnie W Lau, Yasushi Takamatsu","doi":"10.1007/s12185-025-04134-6","DOIUrl":"10.1007/s12185-025-04134-6","url":null,"abstract":"<p><p>Talquetamab is the first G protein-coupled receptor family C group 5 member D (GPRC5D) × CD3 bispecific antibody approved for relapsed/refractory multiple myeloma (RRMM). We report the first efficacy and safety results of talquetamab in Japanese patients enrolled as a separate cohort in the global MonumenTAL-1 study. Between July 2022 and December 2023, 36 patients were enrolled and received 0.4 mg/kg talquetamab weekly. Median follow-up was 13.4 months. The overall response rate was 77.8% (55.6% achieved complete response or better). Median time to first response was 1.2 months. Twelve-month duration of response, progression-free survival, and overall survival rates were 66.4%, 56.3%, and 74.1%, respectively. On-target, off-tumor adverse events (AEs), including taste-, skin- (non-rash), nail-, and rash-related AEs, were common (80.6%, 66.7%, 55.6%, and 36.1%, respectively) and mostly grade 1/2. Cytokine release syndrome occurred in 75.0% of patients; all events were grade 1/2. The rate of infection was 52.8%; the rate of grade 3/4 infection was 16.7% (one led to death [pneumonia]). One patient discontinued talquetamab and three patients died due to AEs. Results were generally consistent with the global MonumenTAL-1 population, demonstrating talquetamab as an important novel treatment for Japanese patients with RRMM. Clinical trial registration number: NCT03399799/NCT04634552.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"580-592"},"PeriodicalIF":1.8,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13083461/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145767996","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":"Safety and efficacy of clofarabine for preconditioning intervention in patients undergoing allogeneic hematopoietic stem cell transplantation for relapsed/refractory acute lymphoblastic leukemia.","authors":"Akihiko Izumi, Takayoshi Tachibana, Takuya Miyazaki, Takaaki Takeda, Hiroaki Konishi, Maasa Abe, Takahiro Suzuki, Shuku Sato, Taisei Suzuki, Etsuko Yamazaki, Kana Bando, Yu Uemura, Masatsugu Tanaka, Hideaki Nakajima","doi":"10.1007/s12185-026-04201-6","DOIUrl":"https://doi.org/10.1007/s12185-026-04201-6","url":null,"abstract":"<p><p>Previous studies have suggested that intensive chemotherapy to induce bone marrow hypoplasia before allogeneic hematopoietic stem cell transplantation (HSCT) may improve outcomes in relapsed/refractory acute lymphoblastic leukemia. In this retrospective single-center study, we analyzed 14 patients who received clofarabine (CLO) as a preconditioning intervention (PCI) before HSCT between 2019 and 2024. PCI was defined as initiation of conditioning within 2 weeks after CLO. The median age was 34 years, and seven patients were not in remission at the time of CLO. CLO (30 mg/m<sup>2</sup> for 5 days) was given in one or two cycles. WBC and bone marrow nucleated cells significantly decreased after CLO. The 1-year overall survival, relapse incidence, and non-relapse mortality rates were 67.5%, 32.2%, and 21.6%, respectively. Neutrophil engraftment was achieved in all patients. Acute and chronic graft-versus-host disease occurred in four and two patients, respectively. Bloodstream infections within 100 days after HSCT were observed in nine patients. Thrombotic microangiopathy (n = 2), sinusoidal obstruction syndrome/veno-occlusive disease (n = 2), drug-induced cardiomyopathy (n = 1), and organizing pneumonia (n = 1) were also observed but were clinically manageable. Considering the high-risk nature of this cohort, CLO-based PCI followed by HSCT appears to be a feasible treatment strategy with acceptable toxicity, warranting further investigation.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.8,"publicationDate":"2026-03-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147581258","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}
Merve Yilmaz Kars, Taha Ulutan Kars, Mustafa Cayci, Metin Bagci, Salih Cirik, Ayse Gunay, Seda Yılmaz, Abdulkadir Basturk, Muhammet Cemal Kizilarslanoglu
{"title":"Psoas and pectoralis muscle indices and brown adipose tissue activity may be prognostic indicators in older patients with multiple myeloma.","authors":"Merve Yilmaz Kars, Taha Ulutan Kars, Mustafa Cayci, Metin Bagci, Salih Cirik, Ayse Gunay, Seda Yılmaz, Abdulkadir Basturk, Muhammet Cemal Kizilarslanoglu","doi":"10.1007/s12185-026-04206-1","DOIUrl":"https://doi.org/10.1007/s12185-026-04206-1","url":null,"abstract":"<p><p>Changes in body composition, particularly sarcopenia and adipose tissue remodeling, have prognostic implications in multiple myeloma (MM). This retrospective study evaluated the prognostic value of PET/CT-derived muscle indices and brown adipose tissue (BAT) activity in 75 MM patients aged ≥ 60 years treated between January 2023 and March 2025. Autologous stem cell transplantation (ASCT) was performed in 41.3% of patients. Median progression-free survival was 6.06 months, with a median follow-up of 25.70 months. ASCT was independently associated with improved overall survival (OR: 0.249, p = 0.060) and a reduced risk of final disease progression (OR: 0.260, p = 0.019). Higher Charlson Comorbidity Index scores independently predicted increased mortality (OR: 1.677, p = 0.017). Reduced skeletal muscle mass, particularly a lower pectoralis major index, was independently associated with disease progression (OR: 0.003, p = 0.012). Among ASCT recipients, higher BAT activity (SUVmean) showed a strong negative correlation with post-transplant progression-free survival (rho = -0.717, p < 0.05). PET/CT-based body composition analysis may provide additional prognostic value in MM.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.8,"publicationDate":"2026-03-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147592015","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":"Clinical significance of TP53 mutations in patients with acute myeloid leukemia: the HM-SCREEN-JAPAN 01/02 study.","authors":"Fumiya Ogasawara, Yosuke Minami, SungGi Chi, Tomoaki Ueda, Kentaro Fukushima, Kensuke Kojima","doi":"10.1007/s12185-026-04192-4","DOIUrl":"https://doi.org/10.1007/s12185-026-04192-4","url":null,"abstract":"<p><p>Mutant TP53 (mTP53) variants are diverse, and residual wild-type p53 function (functional, non-functional), gain-of-function (GOF) mTP53, and the number of mTP53 (single-hit, multi-hit) can affect prognosis. We used next-generation sequencing to evaluate the association between qualitative and quantitative mTP53 abnormalities and overall survival (OS) in 330 patients with acute myeloid leukemia (AML) enrolled in the Japanese multicenter study HM-SCREEN-JAPAN 01/02. Patients with single- and multi-hit mTP53 had a worse prognosis than patients with wild-type TP53 (median OS: 16.1 months vs. 7.5 months vs. 41.6 months). Patients with multi-hit mTP53 had a worse prognosis (P = 0.011). Patients with both mTP53 and complex karyotype (CK) had a worse prognosis than patients with either mTP53 or CK (median OS: 7.6 months vs. 15.0 months vs. 18.9 months; P = 0.03). Residual function did not affect prognosis in patients with single-hit mTP53 (median OS: 24.9 months vs. 16.1 months; P = 0.985), and prognosis did not differ between patients with GOF mTP53 and those with non-GOF mTP53 (median OS: 9.5 months vs. 9.8 months; P = 0.913). Quantitative abnormalities in the TP53 gene affected prognosis, suggesting that qualitative abnormalities did not.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.8,"publicationDate":"2026-03-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147581213","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":"Postmarketing surveillance study of asciminib in patients with resistant/intolerant chronic myeloid leukemia in Japan.","authors":"Kazuaki Yamaguchi, Makoto Aoki, Ryohei Osako","doi":"10.1007/s12185-026-04199-x","DOIUrl":"https://doi.org/10.1007/s12185-026-04199-x","url":null,"abstract":"<p><p>Following asciminib's initial approval in Japan for chronic myeloid leukemia (CML) resistant or intolerant to previous therapy, this all-patient, postmarketing surveillance study was initiated. Predefined safety specifications, overall safety, and effectiveness were assessed for 48 weeks from asciminib initiation (safety analysis set, n = 523). The approved daily dose of asciminib (80 mg [40 mg twice daily]) was most frequently used (63.9%) and not exceeded. Discontinuations (28.5%) were primarily due to adverse events (18.2%), including disease progression. Incidences of the safety specifications myelosuppression, pancreatitis, QT interval prolongation, infections, vascular occlusive events, and photosensitivity were 8.6%, 4.4%, 2.5%, 1.3%, 0.2%, and 0%, respectively, with low rates of treatment discontinuation for these events. There were no notable trends in the rates or types of adverse drug reactions in patients aged ≥ 65 years or with concurrent renal impairment, hepatic impairment, or cardiac dysfunction. The cumulative major molecular response rate was 61.2% by week 48 and was not affected by age (≥ 65 years) or comorbidities. Cumulative MR<sup>4.0</sup> and MR<sup>4.5</sup> rates by week 48 were 42.3% and 26.5%, respectively, with some patients harboring baseline BCR::ABL1 mutations showing these responses. These real-world outcomes support the safety and effectiveness of asciminib for patients with resistant/intolerant CML.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.8,"publicationDate":"2026-03-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147592010","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}