{"title":"Hematopoietic cell transplantation in pediatric EB virus-associated hemophagocytic lymphohistiocytosis.","authors":"Kenichi Sakamoto, Satoshi Miyamoto, Kohsuke Imai, Maho Sato, Katsuyoshi Koh, Takashi Koike, Masataka Ishimura, Tadashi Anan, Koji Kato, Atsushi Sato, Moeko Hino, Kimikazu Matsumoto, Ken Tabuchi, Katsutsugu Umeda","doi":"10.1007/s12185-025-04005-0","DOIUrl":"10.1007/s12185-025-04005-0","url":null,"abstract":"<p><strong>Background: </strong>Few reports exist on the transplant outcomes of Epstein-Barr virus-associated hemophagocytic lymphohistiocytosis (EBV-HLH). This nationwide survey evaluated the clinical outcomes of pediatric patients with EBV-HLH who underwent allogeneic HCT.</p><p><strong>Methods: </strong>Data from 32 pediatric patients with EBV-HLH who underwent their first allogeneic HCT between 2000 and 2020 were reviewed retrospectively.</p><p><strong>Results: </strong>Of the 32 patients, 12 had a performance status of 3-4 and 8 had multiple organ dysfunction. The cumulative incidence of engraftment on day 30 was 53.1% (95% confidence interval [CI] 34.1-68.9). The 1-year overall survival (OS) rate of the entire cohort was 56.2% (37.6-71.3). Univariate analysis revealed that poor ECOG-PS at HCT and a reduced-intensity conditioning regimen were significant risk factors for OS. Furthermore, multivariate analysis revealed that only ECOG-PS was a significant risk factor for OS. Sixteen of the 32 (50.0%) patients died after HCT, and the main causes of death were HLH progression (n = 12) and infection (n = 2). Most notably, 12 of the 13 patients with primary graft failure died of HLH.</p><p><strong>Conclusion: </strong>Good general condition prior to HCT, which is typically achieved through optimal disease control, is essential for stable engraftment and long-term survival following allogeneic HCT.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"598-604"},"PeriodicalIF":1.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144101861","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":"A successful control of cardiac amyloidosis by idecabtagene vicleucel in a patient with relapsed and refractory multiple myeloma: a case report and literature review.","authors":"Yayoi Ueda, Toshiki Terao, Nobuharu Fujii, Tatsuji Mino, Saya Kubota, Kenta Hayashino, Kanako Fujiwara, Takumi Kondo, Keisuke Seike, Hideaki Fujiwara, Noboru Asada, Daisuke Ennishi, Keiko Fujii, Hideo Tanaka, Yoshinobu Maeda","doi":"10.1007/s12185-025-04016-x","DOIUrl":"10.1007/s12185-025-04016-x","url":null,"abstract":"<p><p>A 50-year-old Japanese man with penta-drug refractory multiple myeloma (MM) was found to have stage IIIa cardiac immunoglobulin light-chain amyloidosis before idecabtagene vicleucel (ide-cel) therapy. Ide-cel therapy was started after careful consideration and hospital ethics committee approval. Grade 3 cytokine release syndrome and atrial flutter occurred in the acute phase after ide-cel infusion, but these were well-tolerated with supportive care in the intensive care unit. The patient achieved stringent complete response at day 60 and cardiac response at 9 months after ide-cel infusion with the addition of catheter ablation for sustained atrial flutter on day 133. He has maintained both hematological and cardiac remission for over 1 year since ide-cel therapy. This case highlights the effectiveness of ide-cel for disease control in heavily pretreated MM with cardiac amyloidosis.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"616-622"},"PeriodicalIF":1.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144293744","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":"Utility of tolvaptan sodium phosphate for refractory fluid retention in post-transplant sinusoidal obstruction syndrome.","authors":"Koshi Akahane, Shin Kasai, Minori Tamai, Yukihiro Sugita, Hiroko Oshiro, Kumiko Goi, Takeshi Inukai","doi":"10.1007/s12185-025-04022-z","DOIUrl":"10.1007/s12185-025-04022-z","url":null,"abstract":"<p><p>Sinusoidal obstruction syndrome (SOS) is a life-threatening complication of hematopoietic stem cell transplantation (HSCT), particularly in patients with a high HokUS-10 score after starting treatment. Tolvaptan sodium phosphate (TSP) is a novel intravenous aquaretic agent used to treat refractory fluid retention in congestive heart failure (CHF). Here, we report the successful treatment of severe post-HSCT SOS with refractory fluid retention and CHF using TSP plus defibrotide. A 22-year-old man with relapsed acute lymphoblastic leukemia underwent unrelated peripheral blood stem cell transplantation and developed SOS on day 13. Despite defibrotide therapy and standard management, fluid retention rapidly progressed, resulting in an 18.3% increase in body weight on day 21 and a high HokUS-10 score (10/13 points). TSP (16 mg) administered to treat the CHF immediately induced adequate urine output. Continued TSP treatment (8 mg/day) resulted in sustained diuresis and a return to baseline body weight on day 33. The only significant adverse event observed during the 5 weeks of TSP treatment was transient hypernatremia (148 mEq/L). Defibrotide was discontinued on day 72 because the HokUS-10 score had decreased to 1 point. Our experience suggests the utility of TSP in controlling refractory fluid retention due to post-HSCT SOS.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"611-615"},"PeriodicalIF":1.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12476439/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144234025","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}
Yoshimitsu Shimomura, Seitaro Terakura, Keitaro Matsuo, Yuri Ito, Tatsuo Ichinohe, Yoshiko Hashii, Hideki Goto, Koji Kato, Koji Kawamura, Makoto Onizuka, Fumihiko Ishimaru, Yoshiyuki Takahashi, Atsumi Yanagisawa, Marie Ohbiki, Ken Tabuchi, Yoshiko Atsuta, Takahiro Fukuda, Junya Kanda, Makoto Murata
{"title":"Impact of center volume on acute graft versus host disease in allogeneic stem cell transplant recipients.","authors":"Yoshimitsu Shimomura, Seitaro Terakura, Keitaro Matsuo, Yuri Ito, Tatsuo Ichinohe, Yoshiko Hashii, Hideki Goto, Koji Kato, Koji Kawamura, Makoto Onizuka, Fumihiko Ishimaru, Yoshiyuki Takahashi, Atsumi Yanagisawa, Marie Ohbiki, Ken Tabuchi, Yoshiko Atsuta, Takahiro Fukuda, Junya Kanda, Makoto Murata","doi":"10.1007/s12185-025-04003-2","DOIUrl":"10.1007/s12185-025-04003-2","url":null,"abstract":"<p><p>Prophylactic, diagnostic, and treatment strategies for acute graft-versus-host disease (aGVHD) may vary across medical centers and physicians. We aimed to investigate the relationship between center volume and the incidence and outcomes of aGVHD. This retrospective study included 28,786 patients who underwent their first hematopoietic stem cell transplantation (HSCT) (entire cohort) and 9498 patients who developed grade II-IV aGVHD (aGVHD cohort). Data were categorized into quartiles (very low, low, high, and very high) based on the number of HSCTs the treating center performed during the study period. We assessed the incidence of aGVHD using the entire cohort and overall survival (OS) using the aGVHD cohort. Higher center volume was associated with a higher incidence of grade II-IV aGVHD than very low center volume, with an adjusted hazard ratio of 1.07-1.11. Conversely, center volume was not associated with the incidence of grade III-IV aGVHD. OS after development of aGVHD was better in the higher center volume group than the very low-volume group, with an adjusted hazard ratio of 0.81-0.89. A very low-volume center was associated with a lower incidence of grade II-IV aGVHD in patients with allogeneic HSCT and poor survival in patients with aGVHD.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"559-571"},"PeriodicalIF":1.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144110617","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":"Comparison of clinical outcomes between preemptive and maintenance therapy after allogeneic hematopoietic stem cell transplantation for acute myeloid leukemia.","authors":"Hiroaki Konishi, Takayoshi Tachibana, Shota Arai, Akihiko Izumi, Takaaki Takeda, Natsuki Hirose, Jun Nukui, Shuku Sato, Taisei Suzuki, Ryuji Ishii, Etsuko Yamazaki, Manabu Matsunawa, Junichi Mukae, Atsushi Takahata, Masatsugu Tanaka, Takahiro Suzuki, Hideaki Nakajima","doi":"10.1007/s12185-025-04009-w","DOIUrl":"10.1007/s12185-025-04009-w","url":null,"abstract":"<p><p>This study aimed to compare the efficacy and safety of preemptive and maintenance therapies as post-transplant therapy for acute myeloid leukemia (AML). Patients with AML who underwent allogeneic stem cell transplantation and received post-transplant therapy were eligible. Preemptive therapy was initiated if elevated Wilms' tumor-1 mRNA was detected in the peripheral blood. Twenty-nine patients received either preemptive (n = 12) or maintenance therapy (n = 17). The median age was 56 years (range, 18-70). The median time from transplantation to intervention was 77 days (range, 43-203) for maintenance and 346 days (range, 104-1027) for preemptive therapy. Maintenance therapy consisted of azacitidine (AZA) monotherapy in six patients, venetoclax (VEN) + AZA in five, and VEN + cytarabine (AraC) in six. Preemptive therapy consisted of AZA monotherapy in two patients, VEN + AZA in nine, and VEN + AraC in one. One-year overall survival from intervention was 92% for maintenance and 73% for preemptive therapy (P = 0.28), and 1-year event-free survival was 83% and 66%, respectively (P = 0.29). No serious adverse events or treatment-related mortality were observed in either group. Both therapies were safe and effective in preventing disease relapse. A prospective study comparing these two groups is warranted.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"546-558"},"PeriodicalIF":1.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144132353","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":"Prognostic significance of age at diagnosis and preceding infection varies across age groups in childhood immune thrombocytopenia.","authors":"Masue Imaizumi, Junichi Kitazawa, Takeshi Rikiishi, Hisaya Nakadate, Kousaku Matsubara, Yukihiro Takahashi, Yoji Sasahara, Toshiaki Oka, Naoko Maeda, Akira Ishiguro","doi":"10.1007/s12185-025-04081-2","DOIUrl":"https://doi.org/10.1007/s12185-025-04081-2","url":null,"abstract":"","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145199233","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}
Sang Eun Yoon, Junhun Cho, Duck Cho, Eun-Sook Kang, Seok Jin Kim, Won Seog Kim
{"title":"Clinical outcomes of tisagenlecleucel in relapsed/refractory diffuse large B-cell lymphoma: insights from a single-center study.","authors":"Sang Eun Yoon, Junhun Cho, Duck Cho, Eun-Sook Kang, Seok Jin Kim, Won Seog Kim","doi":"10.1007/s12185-025-04006-z","DOIUrl":"10.1007/s12185-025-04006-z","url":null,"abstract":"<p><p>Chimeric antigen receptor (CAR) T-cell therapy has transformed the treatment landscape for relapsed and refractory large B-cell lymphoma (RR-DLBCL). This study evaluated the real-world efficacy and toxicities of 96 patients with RR-DLBCL who received tisagenlecleucel (tisa-cel) at a single institution. Among 81 patients who received bridging chemotherapy, most received a bendamustine and rituximab regimen (n = 48, 46.9%), and 31 (38.3%) responded to bridging chemotherapy (17.3% complete remission [CR] and 21.0% partial remission). Tisa-cel showed an overall response rate (ORR) of 71.9% at 1 month, which declined to 55.1% at 3 months. The median progression-free survival (PFS) was 4.5 months, with 1-year PFS at 33.3%. Median overall survival (OS) was 13.9 months, with a 1-year OS rate of 55.2%. Achieving CR at 3 months was correlated with significantly improved PFS and OS. Cytokine release syndrome occurred in 75% of patients (14.6% grade ≥ 3) and immune effector cell-associated neurotoxicity syndrome occurred in 22.9% of patients (7.3% grade ≥ 3). All adverse events were managed effectively. The results demonstrated significant survival benefits with manageable toxicities, supporting tisa-cel as a viable salvage therapy for RR-DLBCL.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"533-545"},"PeriodicalIF":1.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12476396/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144953228","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":"A pluripotent stem cell model of Emberger syndrome reveals reduced lymphatic endothelial differentiation.","authors":"Kagehiro Kouzuki, Katsutsugu Umeda, Takayuki Hamabata, Tatsuya Kamitori, Takashi Mikami, Yoshitaka Honda, Satoshi Saida, Itaru Kato, Shiro Baba, Hidefumi Hiramatsu, Takahiro Yasumi, Akira Niwa, Megumu K Saito, Junko Takita","doi":"10.1007/s12185-025-04004-1","DOIUrl":"10.1007/s12185-025-04004-1","url":null,"abstract":"<p><p>Emberger syndrome (ES), an autosomal dominant disorder characterized by congenital deafness, primary lymphedema, and predisposition to myeloid malignancies, is caused by mutations in the GATA2 gene. Although primary lymphedema is an important hallmark of ES, the pathophysiology remains unclear due to the lack of a suitable experimental model. In this study, we isolated induced pluripotent stem cells (iPSCs) from two patients with ES (i.e., ES-iPSCs) and analyzed their in vitro lymphatic differentiation potential via the mesodermal progenitor stage. KDR<sup>+</sup> CD34<sup>+</sup> early mesodermal progenitors generated from either ES-iPSCs or wild-type iPSCs during a 6-days serum- and feeder-free culture supplemented with bone morphogenetic protein 4 and vascular endothelial growth factor (VEGF) had almost equivalent developmental potential. However, upon co-culture with OP9 stromal cells, KDR<sup>+</sup> CD34<sup>+</sup> cells derived from ES-iPSCs developed into CD31<sup>+</sup> lymphatic vessel endothelial hyaluronan receptor-1<sup>+</sup> VEGF receptor 3<sup>+</sup> lymphatic endothelial cells less efficiently than KDR<sup>+</sup> CD34<sup>+</sup> cells derived from wild-type iPSCs. Thus, patient-derived iPSCs recapitulate impairments at an early stage of lymphangiogenesis, making them a useful experimental tool for dissecting the pathophysiology of primary lymphedema in ES and developing potential therapeutic approaches.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"590-597"},"PeriodicalIF":1.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144158412","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":"Transient lupus anticoagulant induced by adenovirus cystitis in a bone marrow transplant recipient.","authors":"Tatsuya Imi, Hidesaku Asakura, Keijiro Sato, Shinya Yamada, Takeshi Yoroidaka, Yoshitaka Zaimoku, Hiroyuki Maruyama, Kohei Hosokawa, Akiyo Yoshida, Hiroyuki Takamatsu, Ken Ishiyama, Hirohito Yamazaki, Hikaru Kobayashi, Shinji Nakao, Toshihiro Miyamoto","doi":"10.1007/s12185-025-04020-1","DOIUrl":"10.1007/s12185-025-04020-1","url":null,"abstract":"<p><p>Adenovirus-associated hemorrhagic cystitis (AdV-HC) is a serious complication of hematopoietic stem cell transplantation (HSCT) that requires hemostatic therapies to control severe hematuria. Here we present the case of a 65-year-old woman with acute myeloid leukemia who successfully underwent HSCT, but developed AdV-HC followed by adenovirus viremia. Marked prolongation of activated partial thromboplastin time (APTT) was observed, along with a decrease in all coagulation factors except prothrombin, raising suspicion of a coagulation factor deficiency. A workup including the APTT cross-mixing test, diluted Russell's viper venom time, and phospholipid neutralization assays revealed the presence of lupus anticoagulant (LA), indicating a thrombotic tendency due to LA associated with artifactual lowering of coagulation factors. Based on these findings, no hemostatic agents were used to manage macroscopic hematuria, and all coagulation test results spontaneously normalized with the resolution of adenovirus viremia, without any thrombus formation. Patients with AdV-HC and prolonged APTT should be screened for LA to avoid inappropriate use of hemostatic agents.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"605-610"},"PeriodicalIF":1.8,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12476403/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144225400","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":"Myelodysplastic neoplasms with unbalanced whole-arm translocation der(5;19)(p10;q10): association with double-hit TP53 mutations.","authors":"Kensuke Kojima, Noriko Tsuge, Shohei Yoshida, Dai Umebara, Yoshie Nishida, Shiori Miyazaki, Tadashi Asagiri","doi":"10.1007/s12185-025-04076-z","DOIUrl":"https://doi.org/10.1007/s12185-025-04076-z","url":null,"abstract":"<p><p>Unbalanced whole-arm translocation der(5;19)(p10;q10) is a rare but recurrent cytogenetic aberration noted in patients with myelodysplastic neoplasms (MDS) and acute myeloid leukemia (AML). Eight cases of MDS/AML with der(5;19)(p10;q10) have been previously reported. Here, we describe three additional cases of MDS with der(5;19)(p10;q10) at our institution, in which we identified myeloid malignancy-associated mutations using next-generation sequencing. All patients had two TP53 mutations, each with >10% variant allele frequency, suggesting double-hit TP53 mutations. Double-hit TP53 mutations are only found in approximately 2% of patients with MDS, and may be involved in the development of the cytogenetic abnormalities der(5;19)(p10;q10), +19, and complex karyotype, often associated with exposure to alkylating agents. We propose der(5;19)(p10;q10) as a potential cytogenetic indicator of biallelic TP53 inactivation through double-hit mutations. These data suggest that MDS with der(5;19)(p10;q10) is clinically characterized by aberrant CD7 expression in blasts, a tendency for leukemic transformation, resistance to anti-leukemia therapies, and poor survival outcomes. We also noted that cases of der(5;19)(p10;q10) MDS/AML have been reported exclusively by Japanese institutions. Geographical and ethnic factors may contribute to oncogenesis, which can be triggered by exposure to alkylating agents.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.8,"publicationDate":"2025-09-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145148757","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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