{"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}
{"title":"Renal recovery and clinical outcomes with daratumumab, lenalidomide, and dexamethasone in transplant-ineligible newly diagnosed multiple myeloma with severe renal impairment.","authors":"Yuichi Horigome, Hirotoshi Kamata, Koichi Ehata, Noriyuki Tadera, Kei Hayama, Takahiro Suzuki","doi":"10.1007/s12185-026-04205-2","DOIUrl":"https://doi.org/10.1007/s12185-026-04205-2","url":null,"abstract":"<p><p>Severe renal impairment (RI) is a major contributor to early morbidity and mortality in transplant-ineligible newly diagnosed multiple myeloma (Ti-NDMM). Although daratumumab, lenalidomide, and dexamethasone (DRd) is an established frontline regimen, patients with creatinine clearance ≤ 30 mL/min were excluded from the MAIA trial, leaving evidence in this high-risk population limited. We retrospectively analyzed Ti-NDMM patients with severe RI, defined as estimated glomerular filtration rate (eGFR) ≤ 30 mL/min/1.73 m<sup>2</sup>, treated with frontline DRd between 2019 and 2024 at our institution. Ten patients met inclusion criteria, all classified as stage III according to the Second Revision of the International Staging System. The overall hematologic response rate was 80%, including very good partial response or better in 50%. Complete renal response occurred in 40% and was associated with deeper hematologic response and significantly prolonged progression-free survival and time to next treatment. Median eGFR improved from 21 to 50.5 mL/min/1.73 m<sup>2</sup>. These findings suggest that DRd is feasible and may provide clinically meaningful renal recovery and disease control in Ti-NDMM patients with severe RI, supporting its potential role as a frontline therapeutic option in this underrepresented population.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.8,"publicationDate":"2026-03-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147573920","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":"Phase-specific impact of hypogammaglobulinemia on bacterial infections after allogeneic hematopoietic cell transplantation.","authors":"Hidekatsu Yae, Mitsutaka Nishimoto, Hiroshi Okamura, Hideo Miyagawa, Mika Nakamae, Yasuhiro Nakashima, Masayuki Hino, Hirohisa Nakamae","doi":"10.1007/s12185-026-04204-3","DOIUrl":"https://doi.org/10.1007/s12185-026-04204-3","url":null,"abstract":"<p><p>Infections are a major complication after allogeneic hematopoietic cell transplantation (allo-HCT). Although humoral immunity is essential, the phase-specific role of IgG remains unclear. We analyzed 579 allo-HCT recipients, stratified into early, intermediate, and late post-transplant phases. The incidence of bacterial infections during the intermediate phase decreased with increasing IgG trough levels (incidence rate ratio [IRR], 0.85; 95% confidence interval [CI]: 0.78-0.94, p < 0.01), whereas no association was observed in the early or late phases. During the intermediate phase, multivariable analyses demonstrated that hypogammaglobulinemia (IRR, 1.70; 95% CI: 1.11-2.62, p = 0.02) and acute graft-versus-host disease with grade II-IV (IRR, 2.10; 95% CI: 1.39-3.18, p < 0.01) were associated with bacterial infections. In models including high-dose steroid exposure, steroid therapy was independently associated with bacterial infections, whereas the effect of hypogammaglobulinemia was attenuated. There was a trend toward an interaction between hypogammaglobulinemia and high-dose steroid exposure. A stratified analysis suggested that hypogammaglobulinemia had a more pronounced impact among patients without high-dose steroids (IRR 1.86; 95% CI: 1.09-3.16; p = 0.02). These findings suggest the clinical relevance of hypogammaglobulinemia is phase-specific and influenced by the intensity of immunosuppressive therapy. Monitoring IgG during this period may help identify candidates for targeted immunoglobulin replacement therapy.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.8,"publicationDate":"2026-03-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147573936","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":"Daratumumab plus VRd in Japanese transplant-ineligible/deferred NDMM patients: Japanese subgroup of the CEPHEUS trial.","authors":"Kenshi Suzuki, Morio Matsumoto, Hiroyuki Takamatsu, Hiroshi Kosugi, Tadakazu Kondo, Tomoaki Fujisaki, Thierry Facon, Sonja Zweegman, Miku Ito, Chika Sakai, Satoshi Kanai, Tomohiko Nakatogawa, Melissa Rowe, Robin Carson, Saad Z Usmani","doi":"10.1007/s12185-026-04185-3","DOIUrl":"10.1007/s12185-026-04185-3","url":null,"abstract":"<p><p>The phase 3 CEPHEUS trial was conducted in 13 countries starting December 11, 2018 in patients with newly diagnosed multiple myeloma (NDMM) who were transplant-ineligible or for whom transplantation was not planned as initial therapy. Bortezomib, lenalidomide, and dexamethasone (VRd) with daratumumab (D-VRd) provided deeper, more durable responses and lowered the risk of disease progression or death compared with VRd. This analysis evaluated the efficacy and safety of D-VRd specifically in the Japanese subpopulation of the CEPHEUS trial (D-VRd: n = 9; VRd: n = 13). At a median follow-up of 59.0 months, the overall minimal residual disease (MRD) negativity rate was 77.8% with D-VRd and 46.2% with VRd (odds ratio: 4.08 [95% confidence interval: 0.60, 27.65]). The complete response or better rate was 88.9% with D-VRd and 76.9% with VRd. Median progression-free survival was not reached in either group, with a hazard ratio of 0.34 favoring D-VRd. The sustained (≥ 12 months) MRD negativity rate was 55.6% with D-VRd and 38.5% with VRd. Efficacy and safety results were similar to those observed in the global CEPHEUS population, supporting the use of D-VRd in Japanese patients with NDMM.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.8,"publicationDate":"2026-03-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147528801","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":"Acute megakaryoblastic leukemia with RBM15::MKL1 fusion presenting as neonatal acute liver failure: rescued by living-donor liver transplantation.","authors":"Hiroki Yoshinari, Yuta Kawahara, Hitomi Niijima, Shiho Aoki, Atsuko Ishihara, Toshio Horiuchi, Yuta Hirata, Yukihiro Sanada, Yasuharu Onishi, Yasunaru Sakuma, Kentaro Tsuji, Yukichi Tanaka, Hideki Kumagai, Hitoshi Osaka, Akira Shimada","doi":"10.1007/s12185-026-04194-2","DOIUrl":"https://doi.org/10.1007/s12185-026-04194-2","url":null,"abstract":"<p><p>Acute liver failure in the neonatal period is commonly caused by infections, metabolic disorders, immune disorders, or neonatal hepatitis-like diseases of unknown etiology; however, malignant diseases are rarely included in the differential diagnosis, and leukemia may be overlooked. We report a case of neonatal acute liver failure that required living-donor liver transplantation from the patient's father, which was later diagnosed as acute megakaryoblastic leukemia (AMKL) harboring an RBM15::MKL1 fusion transcript. After liver transplantation, leukemic blasts infiltrated the transplanted liver, necessitating the initiation of chemotherapy. The patient achieved remission and has survived for four years after completion of reduced-intensity AML-type chemotherapy. RBM15::MKL1-positive AMKL is characteristically associated with both myelofibrosis and hepatic fibrosis. Congenital cases have also been reported, suggesting that RBM15::MKL1 AMKL may originate in the intrauterine fetal liver. A literature search for \"acute liver failure\" and \"acute megakaryoblastic leukemia\" revealed five cases reported since 2000. All patients had an RBM15::MKL1 fusion transcript and all died. In contrast, survival has been reported in patients with RBM15::MKL1-AMKL who did not present with acute liver failure. We speculate that our patient survived because acute liver failure was successfully managed with living-donor liver transplantation.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.8,"publicationDate":"2026-03-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147511926","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}
Tatsuro Hirao, Hisashi Yamamoto, Mika Kuno, Otoya Watanabe, Kyosuke Yamaguchi, Kosei Kageyama, Daisuke Kaji, Yuki Taya, Aya Nishida, Shinsuke Takagi, Yuki Asano-Mori, Go Yamamoto, Atsushi Wake, Shuichi Taniguchi, Naoyuki Uchida
{"title":"Cord blood versus matched related donor transplantation in AML not in remission: role of pre-engraftment immune reactions.","authors":"Tatsuro Hirao, Hisashi Yamamoto, Mika Kuno, Otoya Watanabe, Kyosuke Yamaguchi, Kosei Kageyama, Daisuke Kaji, Yuki Taya, Aya Nishida, Shinsuke Takagi, Yuki Asano-Mori, Go Yamamoto, Atsushi Wake, Shuichi Taniguchi, Naoyuki Uchida","doi":"10.1007/s12185-026-04198-y","DOIUrl":"https://doi.org/10.1007/s12185-026-04198-y","url":null,"abstract":"<p><p>Although HLA-matched related donor transplantation (MRDT) is considered the preferred graft source when available, cord blood transplantation (CBT) is an alternative source, with several studies suggesting a potent graft-versus-leukemia effect. We compared outcomes between CBT and MRDT in acute myeloid leukemia (AML) not in remission and examined how pre-engraftment immune reaction (PIR), graft-versus-host disease (GVHD), and HLA mismatch affect CBT outcomes. We retrospectively analyzed 334 patients with AML not in remission who underwent first allo-HSCT using either single-unit CBT (n = 309) or MRDT (n = 25). At 5 years, CBT recipients showed significantly better leukemia-free survival (LFS) (42.0% vs. 17.6%) and lower relapse rates (24.6% vs. 54.0%), with no difference in NRM. Among CBT recipients, patients who developed mild PIR had a lower relapse rate compared with those without PIR (hazard ratio [HR], 0.48). In contrast, severe PIR was associated with higher NRM (HR, 3.13) and worse overall survival (HR, 2.12). Acute GVHD, chronic GVHD, and HLA disparity were not significantly associated with relapse. CBT was associated with superior LFS compared with MRDT in patients with AML not in remission, and PIR occurrence and severity were associated with CBT outcomes.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.8,"publicationDate":"2026-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147485853","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":"Real-world treatment patterns and clinical outcomes in patients with AML from 65 to 74 years unfit for first-line intensive chemotherapy in Japan.","authors":"Fumiaki Fujii, Masahiro Onozawa, Shota Yoshida, Naoki Miyashita, Daisuke Hidaka, Reiki Ogasawara, Mutsumi Takahata, Junichi Hashiguchi, Shota Yokoyama, Masahiro Chiba, Tomoyuki Saga, Taku Shimizu, Ikumi Kasahara, Akio Shigematsu, Katsuya Fujimoto, Satoshi Iyama, Tetsuyuki Igarashi, Shinichi Ito, Yoshihito Haseyama, Mizuha Kosugi-Kanaya, Takeshi Kondo, Takanori Teshima","doi":"10.1007/s12185-026-04193-3","DOIUrl":"https://doi.org/10.1007/s12185-026-04193-3","url":null,"abstract":"<p><p>Venetoclax (VEN), a BCL-2 inhibitor, was approved in Japan in March 2021, for acute myeloid leukemia (AML). We retrospectively analyzed the impact of VEN approval on treatment patterns and outcomes in older AML patients aged 65-74 years unfit for intensive chemotherapy in Japan. Using the Hokkaido Leukemia Net database, we categorized 101 patients into pre-VEN (n = 46) and post-VEN (n = 55) cohorts, excluding those who had acute promyelocytic leukemia or received intensive chemotherapy. Following VEN approval, VEN + azacitidine (AZA) became the most frequently used initial regimen (56%). Despite higher rates of TP53 mutations and complex karyotypes (35.5%), VEN + AZA achieved comparable response rates (CR + CRi: 64.5%) and overall survival (OS, median 11.7 months) to r7 + 3 (CR + CRi: 64.5%, median OS: 13.1 months), and superior outcomes to cytarabine + aclarubicin + G-CSF (CAG, CR + CRi 37.5%, median OS 6.8 months) or AZA monotherapy (CR + CRi 12.5%, median OS 4.5 months). Early mortality at 60 days from diagnosis was lower with VEN + AZA (3.2%) than with reduced-dose cytarabine plus anthracycline (r7 + 3) (12.9%), CAG (26.7%), or AZA monotherapy (18.8%). Our findings demonstrate a substantial shift in real-world treatment practices following VEN approval and suggest that VEN + AZA is an effective option for older AML patients with adverse genetic features.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.8,"publicationDate":"2026-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147485814","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":"Evaluation of Real-world treatment patterns in Japanese patients with cGVHD: A retrospective claims database study.","authors":"Junya Kanda, Kittima Wattanakamolkul, Hideyuki Muromine, Kaname Shiga","doi":"10.1007/s12185-026-04158-6","DOIUrl":"https://doi.org/10.1007/s12185-026-04158-6","url":null,"abstract":"<p><p>This retrospective cohort study using the medical data vision (MDV) database included adult patients who had confirmed diagnosis of cGVHD between 2003 and 2023, were prescribed a steroid prior to diagnosis of cGVHD, and received mycophenolate mofetil (MMF), ibrutinib, or ruxolitinib as second- or later-line therapy. Duration of treatment (DoT) and steroid dose reduction during second-line therapy were assessed. Of the 1489 patients whose data were retrieved, 854 were included (median [range] age: 54 [18.0-83.0] years; males: 518 [60.7%]; mean [SD] Charlson Comorbidity Index score: 7.5 [3.5]). Data on second or later lines of treatment were available for 226 patients. The most common second-line therapy used after first-line steroid treatment was MMF (110 [48.67%]), followed by ibrutinib (88 [38.94%]) and ruxolitinib (28 [12.39%]). Median DoT (days) was 95 for MMF, 86 for ibrutinib, and 30 for ruxolitinib. Steroid doses were mostly kept below 0.5 mg/kg/day under all the 3 second-line treatments. These real-world data provide valuable insights into the management of cGVHD with the therapies currently used in Japan.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.8,"publicationDate":"2026-03-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147480516","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 hepatic indices serve as predictive markers for sinusoidal obstruction syndrome after allogeneic HSCT.","authors":"Hiroya Ichikawa, Kimikazu Yakushijin, Yumiko Inui, Naoko Takemoto, Takahiro Tsuji, Kotaro Iida, Sakura Kamido, Isamu Harima, Yuri Okazoe-Hirakawa, Sakuya Matsumoto, Rina Sakai, Keiji Kurata, Akihito Kitao, Yasuyuki Saito, Shinichiro Kawamoto, Katsuya Yamamoto, Mitsuhiro Ito, Tohru Murayama, Hiroshi Matsuoka, Hironobu Minami","doi":"10.1007/s12185-026-04191-5","DOIUrl":"https://doi.org/10.1007/s12185-026-04191-5","url":null,"abstract":"<p><p>Prediction and early diagnosis are critical for the effective treatment of sinusoidal obstruction syndrome/veno-occlusive disease (SOS/VOD) after hematopoietic stem cell transplantation (HSCT). This study aimed to investigate the utility of clinical hepatic indices easily calculated in routine practice, namely the Endothelial Activation and Stress Index (EASIX), aspartate aminotransferase-to-platelet ratio index (APRI), Fibrosis-4 (FIB-4), reversed Albumin-Bilirubin grade (rALBI), Model for End-Stage Liver Disease (MELD), and MELD score and the serum sodium concentration (MELDNa). We retrospectively analyzed longitudinal clinical data from 175 allogeneic HSCTs at our institution. The 22 patients who eventually developed clinical SOS/VOD were used as the reference standard. At baseline, EASIX, APRI, FIB-4, rALBI, MELD, and MELDNa were analyzed using the receiver operating characteristic method, with resulting area under the curve (95% confidence interval) of 0.739 (0.607-0.871), 0.770 (0.641-0.898), 0.760 (0.645-0.875), 0.672 (0.530-0.815), 0.577 (0.440-0.713), and 0.642 (0.449-0.784), respectively. After HSCT, these indices were also associated with SOS/VOD, even 7 days before diagnosis (all p-values < 0.001). In conclusion, clinical hepatic indices are useful for prediction and early diagnosis of SOS/VOD in allogeneic HSCT recipients. Further studies are required to determine their optimal clinical application.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.8,"publicationDate":"2026-03-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147467143","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":"Thrombosis in paroxysmal nocturnal hemoglobinuria in the complement inhibitor era: mechanisms, risk stratification, and clinical management.","authors":"Bruno Fattizzo, Christoph Q Schmidt","doi":"10.1007/s12185-026-04190-6","DOIUrl":"https://doi.org/10.1007/s12185-026-04190-6","url":null,"abstract":"<p><p>With the advent of complement inhibition therapy, the severe thrombotic complications of paroxysmal nocturnal hemoglobinuria (PNH)-once described as the most vicious acquired thrombophilic state-no longer pose the same imminent clinical threat. Nevertheless, thrombotic events still occur, albeit at much lower frequency, raising both clinical and mechanistic questions. Among the most pressing are: Under what circumstances does anti-complement therapy fail to prevent thrombosis, and which patient- or therapy-specific factors contribute to this risk? When and where should anticoagulation, anti-platelet therapy, or even prophylaxis be considered? At a mechanistic level, what are the drivers of complement-mediated thrombosis in PNH, and how do they differ from those in other thrombotic conditions involving complement activation? This review addresses these questions by summarizing current evidence on complement-induced thrombosis, integrating clinical and experimental findings. It highlights unresolved issues, including when complement blockade is insufficient and explores the distinction between complement-driven thrombotic states, such as PNH, and intrinsic complement-related diseases without thrombotic complications, such as C3 glomerulopathy. Finally, it proposes a pragmatic framework for anticoagulation and prophylaxis and provides an outlook on critical directions for basic and clinical research, with the goal of further elucidating the complex interplay between complement activation and thrombosis.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.8,"publicationDate":"2026-03-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147457024","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}