Annals of Hematology最新文献

{"title":"Improving outcome prediction in indolent ATL by subdividing intermediate risk into low- and high-intermediate groups.","authors":"Shigeo Fuji, Masahito Tokunaga, Atae Utsunomiya, Junya Makiyama, Youko Suehiro, Ki-Ryang Koh, Makoto Nakashima, Yoshihisa Yamano, Kaoru Uchimaru","doi":"10.1007/s00277-025-06319-9","DOIUrl":"https://doi.org/10.1007/s00277-025-06319-9","url":null,"abstract":"<p><p>The indolent adult T-cell leukemia-lymphoma prognostic index (iATL-PI) uses soluble interleukin-2 receptor (sIL-2R) levels of 1,000 and 6,000 U/mL as a cut-off. The disadvantage of the iATL-PI is that approximately half of the patients are classified as intermediate risk (1,000 ≤ sIL-2R < 6,000). Here, we aimed to develop a novel prognostic model for indolent ATL using a prospectively registered database. We identified 375 patients with indolent ATL. Median age was 61 years. The median follow-up of surviving patients was approximately 62 months. In multivariate analysis for overall survival (OS), sIL-2R level (as log(sIL-2R), HR 4.58), male sex (HR 1.41) and LDH > ULN (HR 1.68) were independent prognostic factors. The best cut-off value for sIL-2R to predict OS was 2,870 U/mL. Including the cut-off of 3,000 U/mL in the original iATL-PI, the probabilities of 5-year OS were 88.9% in the low group (sIL-2R < 1,000), 67.5% in the low-intermediate group (1,000 ≤ sIL-2R < 3,000), 34.0% in the high-intermediate group (3,000 ≤ sIL-2R < 6,000) and 17.0% in the high group (sIL-2R ≥ 6,000). The c-index for predicting OS was higher with the modified iATL-PI than with the original iATL-PI (0.749 vs. 0.725). The addition of sex and LDH levels did not improve the c-index. In conclusion, we developed a novel prognostic index of indolent ATL.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143708109","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Optimization of allogeneic hematopoietic cell transplantation for patients with myelofibrosis treated with ruxolitinib: eligibility, best practices, and improving transplant outcomes.","authors":"Sarah A Wall, Roni Tamari, Zachariah DeFilipp, Gabriela S Hobbs","doi":"10.1007/s00277-025-06270-9","DOIUrl":"https://doi.org/10.1007/s00277-025-06270-9","url":null,"abstract":"<p><p>Allogeneic hematopoietic cell transplantation (HCT) is the only curative treatment for myelofibrosis (MF), and current guidelines recommend assessing all patients with MF for eligibility. Several patient- and disease-specific factors impact transplantation outcomes, and timely assessment of potential transplant candidates is key to optimizing post-HCT outcomes. The role of HCT in the treatment of MF continues to evolve, with the adoption of newer and safer approaches, enhanced donor availability, use of reduced-intensity conditioning, improvements in graft-versus-host disease (GVHD) prophylaxis and treatment, and greater understanding of high-risk clinical and molecular features of the disease. These developments highlight the importance of early and ongoing assessment throughout the MF disease course to optimize eligibility and consideration for HCT. Ruxolitinib is approved for first-line treatment of intermediate- or high-risk MF, and emerging data have clarified the important role of ruxolitinib in not only optimizing clinical status before HCT but also mitigating and treating post-HCT complications in patients with MF, notably acute and chronic GVHD and relapse. Here we review strategies for optimizing clinical outcomes in patients considered for and undergoing HCT for MF treated with ruxolitinib. We discuss strategies for appropriate patient and donor selection, optimization of ruxolitinib therapy in the pre- and peri-HCT periods, choice of conditioning regimen, GVHD prophylaxis, post-HCT management of GVHD, continued monitoring for MF relapse, and the role of post-HCT ruxolitinib maintenance to reduce risks of GVHD and disease relapse.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-03-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143690700","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Gain or amplification of 1q21 in systemic light chain amyloidosis is associated with advanced Mayo stage, plasma cell disease and worse overall survival.","authors":"Sara Oubari, Maria Papathanasiou, Lars Michel, Tienush Rassaf, Andreas Thimm, Tim Hagenacker, Daniela Ehling, Stefan Wieczorek, Eyad Naser, Ute Hegenbart, Stefan Schönland, Ulrich Dührsen, Hans Christian Reinhardt, Alexander Carpinteiro","doi":"10.1007/s00277-025-06256-7","DOIUrl":"https://doi.org/10.1007/s00277-025-06256-7","url":null,"abstract":"<p><p>Systemic light-chain amyloidosis (AL) is an acquired protein misfolding disease characterized by deposition of immunoglobulin light-chain fibrils most often secreted from clonal plasma cells. In this retrospective study we analyzed the impact of iFISH aberrations on clinical characteristics and outcomes in 175 AL patients presented between 2015 and 2024. The most common aberrations were t(11;14) (57%), deletion 13q14 (33%), +1q21 (21%), hyperdiploidy (21%) and deletion 16q23 (17%). Significant elevations in dFLC levels were observed in patients with + 1q21 (median 407 vs. 213 mg/l, p = 0.04) and deletion 16q23 (median 476 vs. 204, p = 0.006). Only + 1q21 was associated with increased levels of cardiac biomarkers NTproBNP (median 9945 vs. 3538 pg/ml, p = 0.002) and hsTnT (median 110 vs. 53 ng/l, p = 0.002). This resulted in an increased proportion of patients with Mayo stage IIIb (53% vs. 26%, p = 0.01). Patients with + 1q21 had more advanced plasma cell disease (p = 0.0004). Our study highlights for the first time + 1q21 as the key aberration associated with advanced cardiac and plasma cell disease. After 17 months of follow-up, overall survival was significantly worse in patients with + 1q21 treated with daratumumab (7.2 months vs. not reached, p = 0.006). Alternative therapeutic approaches such as CAR-T therapies or bispecific antibodies should be further investigated.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-03-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143676699","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Racial disparities in cardiovascular manifestations among patients with sickle cell trait: analysis of national inpatient sample data (2016-2020).","authors":"Praneeth Reddy Keesari, Charan Thej Reddy Vegivinti, Medha Rajamanuri, Ahmad Mustafa, Ngowari Pokima, Lauren Haley White, Hasnain Chaudhry, Chamberline E Ozigbu, Khairul A Siddiqi, Suzanne El-Sayegh, Meekoo Dhar, Ugochi Olivia Ogu","doi":"10.1007/s00277-025-06306-0","DOIUrl":"https://doi.org/10.1007/s00277-025-06306-0","url":null,"abstract":"<p><p>Sickle cell trait (SCT) is a condition that affects up to 3 million individuals and 10% of African Americans (AA) in the United States (U.S.). Although often believed to be benign, the true burden of the condition remains understudied. Data is also lacking regarding the racial disparities in SCT and if African American individuals with SCT are at increased cardiovascular risk compared to individuals of other races. Using the National Inpatient Sample (NIS) database, we investigated cardiovascular outcomes in AA individuals with SCT compared to other racial groups. Our study reported statistically significant higher odds of acute heart failure in AA individuals with SCT (Adjusted OR: 1.39 (p < 0.01, 95% CI: 1.13-1.71). Further studies investigating the racial disparities and cardiovascular risk among individuals with SCT are warranted to measure the causal effects.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-03-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143668842","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Favorable correlation between neutrophil percentages obtained using automated hematology analyzers and microscopic examinations in malignant lymphoma patients receiving chemotherapy.","authors":"Hiroki Hosoi, Tadashi Okamura, Shotaro Tabata, Naoto Minoura, Ke Wan, Kazuhiro Hirayasu, Shogo Murata, Toshiki Mushino, Takashi Sonoki","doi":"10.1007/s00277-025-06313-1","DOIUrl":"https://doi.org/10.1007/s00277-025-06313-1","url":null,"abstract":"<p><p>Automated hematology analyzers are routinely used to assess blood cell counts and leukocyte differentials. However, the accuracy of assessing neutrophil percentages with automated hematology analyzers in patients undergoing chemotherapy has not been examined. In this study, the correlation between neutrophil percentages calculated by automated hematology analyzers and those obtained through manual microscopic examinations by laboratory technicians was investigated on the day of chemotherapy initiation in 125 patients receiving CHOP-based chemotherapy for malignant lymphoma. Peripheral blood counts and 5-part leukocyte differential percentages were analyzed using the Sysmex XE-5000 system. An examination of 797 samples from each chemotherapy course showed a favorable correlation between the two methods (Pearson's r = 0.917). However, this correlation significantly diminished in samples when the bone marrow nucleated cell count was ≤ 50 × 10⁹/L or granulocyte colony-stimulating factors were administered in previous chemotherapy courses (r = 0.881 and 0.886, respectively). Furthermore, a tendency towards a weaker correlation was noted when the percentage of immature neutrophils exceeded 3% (r = 0.894). Nevertheless, in all these conditions, the correlations consistently had coefficients of > 0.8, emphasizing the robustness of the correlation. Our study suggests that neutrophil percentages calculated by automated hematology analyzers on the first day of chemotherapy were strongly correlated with those obtained through microscopic examinations. Measuring neutrophil counts with an automated hematology analyzer is sufficient, even in patients receiving chemotherapy.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-03-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143668837","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Proinflammatory and prothrombotic conditions in JAK2V617F-positive MPN: a case of Lemierre's syndrome in essential thrombocythemia.","authors":"Takuya Izumi-Tamura, Kosuke Takano, Shigeki Nagao, Noriaki Tachi, Sho Sato, Masaya Nakagawa, Takehiro Sone, Kohei Takada, Hiraku Ogata, Keita Saito, Shoichiro Kato, Takaaki Maekawa, Akihide Yoshimi, Shinichi Kobayashi, Fumihiko Kimura","doi":"10.1007/s00277-025-06234-z","DOIUrl":"https://doi.org/10.1007/s00277-025-06234-z","url":null,"abstract":"<p><p>Lemierre's syndrome (LS) represents a rare yet potentially life-threatening systemic infection, characterized by thrombophlebitis of the internal jugular vein and abscess formation in distant organs. It typically follows episodes of tonsillitis or other infections of the oropharyngeal region. Pulmonary complications, including septic pulmonary emboli, are common. Essential thrombocythemia (ET) is a chronic myeloproliferative neoplasm (MPN) sometimes associated with the JAK2V617F mutation, which predisposes patients to thrombotic events. A 66-year-old male with JAK2V617F-positive ET presented with severe pulsatile pain radiating from the right temporal region to the occipital area following a recent dental infection. Although pain management was administered, the pain continued to persist. Computed tomography of the chest revealed multiple subpleural nodules, raising suspicion for septic pulmonary emboli. Further investigation with gadolinium-enhanced magnetic resonance imaging identified a thrombus extending from the right sigmoid sinus into the internal jugular vein, consistent with cerebral venous thrombosis. The patient was diagnosed with　LS, complicated by septic thrombosis. Blood cultures yielded alpha-hemolytic streptococcus. Empirical antimicrobial therapy combined with anticoagulation was initiated, resulting in a gradual improvement of symptoms, including the resolution of fever and pain. Follow-up imaging confirmed the resolution of both the infection and thrombosis. This is the first reported case of LS in a patient with JAK2V617F-positive ET. The coexistence of LS and JAK2V617F-positive MPN highlights the potential interplay between proinflammatory and prothrombotic conditions associated with the JAK2V617F mutation.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-03-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143662195","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Effect of major ABO blood group mismatched HSCT on blood transfusion and clinical outcomes in AA patients.","authors":"Dongdi Lai, Dandan Chen, Xiaowei Chen, Ruiqing Zhou, Minglu Zhong, Xiaojie Chen, Jianyun Huang, Yanfei Lan, Xinxin Tong, Zhen Liu, Xuexin Yang, Shunqing Wang, Yaming Wei, Zhaohu Yuan","doi":"10.1007/s00277-025-06213-4","DOIUrl":"https://doi.org/10.1007/s00277-025-06213-4","url":null,"abstract":"<p><p>To investigate the impact of the ABO blood group major match type on stem cell engraftment, blood transfusion and clinical outcomes in aplastic anemia patients with hematopoietic stem cell transplantation (HSCT), we retrospectively analyzed the data of 361 aplastic anemia patients treated with HSCT, and found ABO major mismatched resulted in delayed red blood cells (RBCs) engraftment and ABO blood group conversion. The patients in the ABO major mismatched group required more units of RBCs and PLTs transfusions. Multivariate linear regression analysis showed that ABO mismatched, acute graft-versus-host disease (aGVHD), time to RBCs and PLTs engraftment and blood group conversion, and baseline hemoglobin were significantly associated with RBCs transfusion, the factors associated with PLTs transfusion were the PLTs, RBCs and neutrophils engraftment, graft rejection, baseline PLTs, aGVHD grade II-IV, and severe chronic GVHD. Multivariate analysis showed that the time to neutrophils engraftment, baseline hemoglobin, and aGVHD were independent poor prognostic factors to both overall survival and failure-free survival. Moreover, the major ABO-mismatched HSCT group were hospitalized more often. These findings suggest that it's better to select a donor with an ABO major match to reduce the burden of transfusion and the impact of hospitalization, if conditions permit.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143655946","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Initial management of patients with acquired aplastic anemia in the United States: results from a large national claims database.","authors":"Jessica M Stempel, Rong Wang, Alfred I Lee, Amer M Zeidan, Xiaomei Ma, Nikolai A Podoltsev","doi":"10.1007/s00277-025-06307-z","DOIUrl":"https://doi.org/10.1007/s00277-025-06307-z","url":null,"abstract":"<p><p>Acquired aplastic anemia (AA) is an immune-mediated disorder leading to bone marrow failure characterized by pancytopenia, with infectious and bleeding complications. The disease course may be complicated by paroxysmal nocturnal hemoglobinuria (PNH), necessitating screening with flow cytometry (FC) at the time of AA diagnosis. Management strategies vary based on disease severity. Severe AA patients are usually heavily transfusion-dependent (HT-AA) and typically treated with antithymocyte globulin, calcineurin inhibitor (CNI) and eltrombopag (EPAG) as triple therapy, while allogeneic hematopoietic stem cell transplant (HSCT) is often reserved for younger patients with matched sibling donors. Moderate AA patients are less transfusion-dependent (LT-AA) and may be observed or treated with CNI or EPAG. We conducted a retrospective cohort study using Blue Cross Blue Shield Axis database, examining adult patients diagnosed with AA between 07/01/2016 and 06/30/2022. We evaluated their management within the first 6 months following the diagnosis. Of 793 identified individuals (542 LT-AA, 251 HT-AA), with a median age of 49 years, only 42.6% received AA-directed therapy. Triple therapy and HSCT were infrequently used for patients with HT-AA (4.4% and 18.7%, respectively), while the most common treatment was the combination of a CNI and EPAG (LT-AA 37.8%, HT-AA 51.7%). The median time from diagnosis to treatment was 22 days, with older patients (age ≥ 40 years) experiencing treatment initiation delays (p = 0.03). FC testing was underutilized with only 55.5% of patients undergoing evaluation. These findings highlight the need for better access to diagnostic evaluation and appropriate AA-directed therapy for patients with AA in real-world settings.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143656001","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Challenges in managing iTTP: insights into ADAMTS13 inhibitor boosting during caplacizumab therapy.","authors":"Haruyuki Tanaka, Kazuya Sakai, Shusuke Tamura, Hiroya Shiwaku, Junko Nakamura, Yasunori Ueda, Seiya Bamba, Masashi Nishikubo, Yuya Nagai, Masanori Matsumoto","doi":"10.1007/s00277-025-06318-w","DOIUrl":"https://doi.org/10.1007/s00277-025-06318-w","url":null,"abstract":"<p><p>Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare but life-threatening disorder characterized by severe thrombocytopenia, hemolytic anemia, and end-organ ischemic damage. The introduction of caplacizumab, an anti-von Willebrand factor A1 nanobody, has revolutionized the treatment of patients with iTTP by preventing fatal thrombotic events and shortening the time to platelet normalization. Despite its benefits, caplacizumab does not address the challenge of anti-ADAMTS13 autoantibody production, posing a risk of ADAMTS13 inhibitor boosting and delayed recovery of ADAMTS13 activity. Here, we highlight three challenging cases from the Japanese TTP registry involving patients with iTTP who experienced severe ADAMTS13 inhibitor boosting. This delayed the recovery of ADAMTS13, and extended administration of caplacizumab while requiring additional therapeutic plasma exchange (TPE) and immunosuppressive therapy. All patients demonstrated delayed recovery of ADAMTS13 activity despite initial clinical improvement. Prolonged use of caplacizumab masked the persistence of ADAMTS13 inhibitors, emphasizing the need for close monitoring and timely interventions. Although recent proposals for TPE-free regimens show promise, our findings underscore that TPE remains essential for removing residual autoantibodies and preventing disease exacerbation in certain patients. Stratifying patients based on initial ADAMTS13 inhibitor titers and optimizing immunosuppressive strategies may help identify those at risk of severe inhibitor boosting. Further research is required to refine treatment protocols and ensure the safe withdrawal of caplacizumab while achieving sustained recovery of ADAMTS13 activity.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143662175","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Efficacy and influencing factors of immunosuppressive therapy for pure red cell aplasia: meta-analysis and systematic review.","authors":"Muyassar Yusup, GuangSheng He, YuTing Qin, Niluopaer Tuerxun, JianPing Hao","doi":"10.1007/s00277-025-06315-z","DOIUrl":"https://doi.org/10.1007/s00277-025-06315-z","url":null,"abstract":"<p><p>Acquired pure red cell aplasia (aPRCA) is a rare hematological syndrome characterized by anemia and a significant reduction in erythroid progenitor cells. Immunosuppressive therapy (IST), including Corticosteroids (CS), Cyclosporine (CsA), and cyclophosphamide (CYC), is the primary treatment. However, variations in clinical efficacy and limited comparative studies have created uncertainty in therapeutic choices. This study aims to evaluate the efficacy of IST and the factors influencing treatment outcomes. A systematic search was conducted using PubMed, Embase, Cochrane Library, and Web of Science. Two researchers independently screened studies and extracted data. The quality of studies was assessed using the MINORS scale. Meta-analysis was performed using STATA/MP16, and effect size (ES) was calculated using fixed- or random-effects models based on heterogeneity. A total of 33 studies involving 1,193 patients were included. The overall efficacy of IST was significant, with a pooled ES of 0.656 (95% CI: 0.600-0.710). CsA demonstrated the highest efficacy (ES = 0.699; 95% CI: 0.615-0.779), followed by CYC (ES = 0.592; 95% CI: 0.423-0.752) and CS (ES = 0.568; 95% CI: 0.457-0.676). Subgroup analyses revealed that factors such as etiology, combination therapies, first- vs. second-line treatment, and genetic characteristics significantly influenced outcomes. Notably, the response to IST was higher in primary aPRCA (ES = 0.667; 95% CI: 0.598-0.733) compared to LGLL-associated (ES = 0.515; 95% CI: 0.393-0.637) and thymoma-associated (ES = 0.690; 95% CI: 0.492-0.864) aPRCA. The combination of CS and CsA yielded superior efficacy (ES = 0.761; 95% CI: 0.658-0.853) compared to combination of CS and CsA and monotherapy. First-line treatment demonstrated better efficacy than second-line treatment (ES = 0.659; 95% CI: 0.596-0.720) vs. (ES = 0.452; 95% CI: 0.199-0.715). The important finding was that (ES = 0.861; 95% CI: 0.595-1.000) in the STAT3 mutation (+) group and (ES = 0.375; 95% CI: 0.034-0.801) in the STAT3 mutation (-) group. IST demonstrates overall efficacy in aPRCA, with variations influenced by etiology, drug combinations, and genetic mutations such as STAT3. These findings highlight the need for personalized treatment strategies and further research to validate and optimize IST efficacy.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143662180","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}