Therapeutic Advances in Hematology最新文献

{"title":"Efficacy and safety of different TPO-RAs combined with cyclosporine in the treatment of NSAA.","authors":"Ting Wang, Boyi Wang, Donglan Liu, Nianbin Li, Shuai Gao, Huaquan Wang, Lijuan Li, Limin Xing, Hong Liu, Guojin Wang, Yuhong Wu, Wen Qu, Jia Song, Jing Guan, Zonghong Shao, Rong Fu","doi":"10.1177/20406207261479584","DOIUrl":"10.1177/20406207261479584","url":null,"abstract":"<p><strong>Background: </strong>TPO receptor agonists (TPO-RAs) have significantly improved the treatment outcomes of patients with aplastic anemia. Currently, three small-molecule TPO-RAs (eltrombopag, hetrombopag, and avatrombopag) are available for clinical use and work well.</p><p><strong>Objectives: </strong>This article aims to compare the efficacy and safety of these TPO-RAs in treating non-severe aplastic anemia (NSAA).</p><p><strong>Design: </strong>This was a single-center, retrospective observational cohort study.</p><p><strong>Methods: </strong>A retrospective analysis was performed to assess the efficacy and side effects of cyclosporine A (CsA) combined with different TPO - RAs in 67 NSAA patients. Patients were divided into three groups according to the type of TPO-RA received: eltrombopag, hetrombopag, or avatrombopag. The overall response rate (ORR) was evaluated at 3 months and 6 months after treatment initiation. Adverse events, including liver impairment, infection, and death, were recorded and compared among the three groups.</p><p><strong>Results: </strong>In total,the 3-month overall response rate (ORR) of CsA combined with TPO-RAs was 71.6%, the 6-month ORR was 82.1%. The ORRs among eltrombopag, hetrombopag, and avatrombopag groups were no statistically significant difference at the 3-month (P=0.334) or 6-month (P=0.776). Due to the pre-screening of patients with underlying liver conditions, the occurrence of side effects including new liver impairment, infection and death, showed no significant differences among the three groups. High level of absolute reticulocyte count at enrollment and adequate initial treatment dose of TPO-RAs are independent factors for better efficacy at 6 months. Old age (P=0.034) and infection(P=0.000) were the independent factors affecting the duration of hospitalization.</p><p><strong>Conclusions: </strong>This study shows that eltrombopag, hetrombopag, and avatrombopag combined with CsA have comparable efficacy in the treatment of NSAA. Identifying patients at high risk for liver injury before treatment and initiating avatrombopag therapy represents a viable clinical treatment option for NSAA patients with pre-existing liver injury. Similar to SAA, NSAA patients should also be initiated with an adequate dose of TPO-RAs therapy. Elderly patients and those with concurrent infections are at higher risk of mortality, prolonged hospitalizations, and require more clinical attention.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261479584"},"PeriodicalIF":2.8,"publicationDate":"2026-09-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13583317/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148991553","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Efficacy and safety of daratumumab-based quadruplet therapy vs non-daratumumab-based triplet therapy in newly diagnosed multiple myeloma patients: A GRADE assessed systematic review and meta-analysis of randomized controlled trials.","authors":"Muhammad Osama, Muhammad Haris Khan, Ammara Tahir, Amna Hussain, Ahmad Iftikhar, Aizaz Ali, Safeena Khan, Malak Bilal Hassan, Intikhab Alam, Zeeshan Khan, Abdullah Afridi, Mohammad Ebad Ur Rehman, Amna Gul, Pawan Kumar Thada","doi":"10.1177/20406207261489040","DOIUrl":"10.1177/20406207261489040","url":null,"abstract":"<p><strong>Background: </strong>Multiple myeloma (MM) is the second most common hematologic malignancy worldwide. Recent therapeutic innovations, including the addition of daratumumab to standard regimens, have shown promising improvements in patient outcomes.</p><p><strong>Objectives: </strong>This study aims to compare the efficacy and safety of daratumumab-based quadruplet therapy regimens with standard triplet therapy regimens in newly diagnosed multiple myeloma (NDMM) patients.</p><p><strong>Design: </strong>Systematic review and meta-analysis of randomized controlled trials.</p><p><strong>Data sources and methods: </strong>A comprehensive literature search was conducted on PubMed, Cochrane Library, Embase, and ClinicalTrials.gov from inception to April 2026. Primary outcomes were Progression-free survival (PFS) and Minimal residual disease (MRD) negativity. Subgroup analysis based on cytogenetic risk was performed. The statistical assessment was done using The Review Manager (RevMan, Version 5.4).</p><p><strong>Results: </strong>Our meta-analysis included 7 RCTs, comprising 3 studies in transplant-eligible multiple myeloma (TE-MM) and 4 in transplant-ineligible multiple myeloma (TIE-MM) patients, containing 3,443 patients (1,759 in the daratumumab group and 1684 in the non-daratumumab group). The median age across studies ranged from approximately 59 to 76 years. Daratumumab-based therapy demonstrated significantly higher PFS (HR 0.50, 95%CI: 0.43- 0.58, p < 0.00001). The analysis showed statistically significant high MRD negativity with daratumumab based quadruplet therapy compared to non-Dara triplet arm (RR 1.99, 95%CI 1.59-2.50, P<0.00001). Subgroup analysis based on cytogenetic risk demonstrated significant improved PFS in standard-risk (HR 0.45) as well as high-risk (HR 0.67), whereas MRD negativity increased significantly only in standard-risk (RR 1.76) but not high-risk (RR 1.23). Daratumumab-based quadruplet therapy was associated with increased neutropenia (56.2% vs 40.3%), thrombocytopenia (40.7% vs 32.5%), and infections (33.1% vs 23.3%).</p><p><strong>Conclusion: </strong>The meta-analysis shows higher efficacy yet higher adverse events in Daratumumab-based quadruplet therapy for NDMM patients (TE, and TIE).</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261489040"},"PeriodicalIF":2.8,"publicationDate":"2026-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13570013/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148956458","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Fulminant extramedullary relapse of multiple myeloma with rare cardiac, vascular, and multivisceral involvement: A case report.","authors":"Yuhui Wei, Xuejiao Yang, Yulan Zheng, Dongdong Zhang","doi":"10.1177/20406207261488942","DOIUrl":"10.1177/20406207261488942","url":null,"abstract":"<p><p>Multiple myeloma (MM) is a plasma cell malignancy that typically follows a protracted clinical course. Extramedullary disease (EMD) represents an aggressive manifestation of MM with a relatively low incidence, traditionally involving the skin and soft tissues. While MM usually progresses indolently, fulminant disease progression characterized by extensive multivisceral and vascular infiltration is exceptionally rare and seldom documented in the medical literature. This case illustrates a 64-year-old woman who presented with left chest wall mass and pain and was diagnosed with high-risk IgG-λ-type MM (R-ISS Stage II), harboring 1q21 amplification and IGH/FGFR3 translocation. Following 12 cycles of VCD (bortezomib, cyclophosphamide, and dexamethasone) induction, she achieved partial remission and proceeded to maintenance therapy with bortezomib and ixazomib. Fifteen months post-diagnosis, the patient presented with a rapidly enlarging abdominal wall mass. 18F-FDG PET-CT revealed widespread hypermetabolic activity involving the heart, major blood vessels, liver, spleen, lungs, and multiple soft tissue sites, indicating extensive EMD. Bone marrow examination showed only 1% plasma cells, demonstrating a marked dissociation between systemic EMD and medullary involvement. Despite salvage therapy with carfilzomib, liposomal doxorubicin, and dexamethasone, the patient's condition deteriorated rapidly. Compounded by severe hypoproteinemia, electrolyte disturbances, and respiratory infection, the disease followed a fulminant course, ultimately leading to the patient's death. This case represents a catastrophic transition of genetically high-risk MM from initial medullary involvement to widespread multivisceral and cardiovascular infiltration upon relapse. It highlights a rare phenotype of relapsed/refractory MM where aggressive extramedullary proliferation occurs independently of bone marrow progression. Such patients face a dismal prognosis and present a formidable challenge for current salvage therapeutic strategies.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261488942"},"PeriodicalIF":2.8,"publicationDate":"2026-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13554540/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148920488","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Corticosteroids plus statins in patients with primary immune thrombocytopenia and hyperlipidemia: A multicenter retrospective cohort study.","authors":"Xiran Hou, Yajing Zhao, Jian Chen, Lin Sun, Qiaofeng Dong, Changyong Yuan, Jian Lv, Shuang Liu, Na Gao, Ming Hou, Jun Peng, Zhenyu Yan, Xinguang Liu","doi":"10.1177/20406207261484030","DOIUrl":"10.1177/20406207261484030","url":null,"abstract":"<p><strong>Background: </strong>Hyperlipidemia is prevalent in primary immune thrombocytopenia (ITP) and may exacerbate bleeding severity and compromise treatment responses. Statins possess lipid-lowering and anti-inflammatory properties, but their influence on corticosteroid therapy in ITP patients with hyperlipidemia remains unclear.</p><p><strong>Objectives: </strong>This study aimed to compare the efficacy and safety of initial corticosteroid therapy between patients with or without concomitant statin use for pre-existing hyperlipidemia in a cohort of newly diagnosed ITP patients.</p><p><strong>Design: </strong>A multicenter retrospective propensity score-matched study.</p><p><strong>Methods: </strong>Newly diagnosed ITP patients with pre-existing hyperlipidemia from 9 medical centers were analyzed. Patients were divided into 2 groups based on statin use (statin <i>vs</i>. non-statin). Propensity score matching (PSM) was employed to balance baseline characteristics. Treatment outcomes, including response rates, duration of response (DOR), and safety, were compared.</p><p><strong>Results: </strong>After PSM, 104 patients (52 per group) were included. Baseline characteristics were comparable. Initial response rates were similar between the statin and non-statin groups (67.3% <i>vs.</i> 65.4%, <i>P</i> = 0.836), with the 95% confidence interval (CI) for the between-group risk difference ranging from -15.83% to 19.51%. The median time to response was numerically shorter in the statin group, although this difference did not reach statistical significance (4 [2-8] days <i>vs.</i> 5 [2-12] days, <i>P</i> = 0.054). Notably, the sustained response (SR) rate at 6 months was significantly higher in the statin group compared to the non-statin group (50.0% <i>vs.</i> 30.8%, <i>P</i> = 0.046), with the 95% CI for the between-group risk difference ranging from 0.41% to 36.25%. Kaplan-Meier analysis also demonstrated a significant longer duration of response (DOR) in the statin group (<i>P</i> = 0.044). No serious adverse events were observed in either group during follow-up.</p><p><strong>Conclusion: </strong>In this retrospective, propensity score-matched cohort, concomitant statin use was associated with higher 6-month SR rates and longer DOR in newly diagnosed ITP patients with pre-existing hyperlipidemia, without an apparent increase in serious adverse events. These observational findings warrant further validation in larger, prospective cohorts.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261484030"},"PeriodicalIF":2.8,"publicationDate":"2026-09-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13554654/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148920363","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prognostic value of early lymphocyte dynamics during blinatumomab therapy in relapsed or refractory B-cell precursor acute lymphoblastic leukemia: A real-world retrospective study.","authors":"Seonghan Lee, Daehun Kwag, Gi June Min, Sung-Soo Park, Silvia Park, Sung-Eun Lee, Byung-Sik Cho, Ki-Seong Eom, Yoo-Jin Kim, Hee-Je Kim, Chang-Ki Min, Seok-Goo Cho, Jae-Ho Yoon","doi":"10.1177/20406207261487003","DOIUrl":"10.1177/20406207261487003","url":null,"abstract":"<p><strong>Background: </strong>Blinatumomab, a bispecific T-cell engager, mediates cytotoxicity through CD3+ T-cell activation and has shown efficacy in relapsed or refractory (R/R) B-cell precursor acute lymphoblastic leukemia (BCP-ALL). Although T-cell expansion correlates with favorable outcomes, routine immunophenotyping is impractical in clinical settings.</p><p><strong>Objectives: </strong>To determine whether early changes in absolute lymphocyte count (ALC), a readily available laboratory marker, can serve as a surrogate indicator of therapeutic response and long-term survival in patients receiving blinatumomab.</p><p><strong>Design: </strong>A retrospective cohort study of adult patients with R/R BCP-ALL treated with blinatumomab.</p><p><strong>Methods: </strong>We analyzed 162 adults with R/R BCP-ALL who received up to two cycles of blinatumomab. The primary outcome was complete remission (CR), and secondary outcomes included overall survival (OS). ALC change from day 1 to day 14 (ΔALC14) was calculated, and its association with outcomes was assessed using logistic and Cox regression models.</p><p><strong>Results: </strong>Of the 162 patients, 106 (65.4%) achieved CR. ΔALC14 was significantly greater in responders than in non-responders (median 0.344 vs. 0.146 × 10⁹/L; <i>P</i> < 0.001). A high ΔALC14 (≥0.100× 10⁹/L) was independently associated with higher response rates (OR: 2.96; P=0.004) and longer OS (median 15.3 vs. 7.0 months; P=0.007). Multivariate analysis confirmed high ΔALC14 as an independent predictor of response and favorable OS. Subgroup analyses suggested that the association between high ΔALC14 and response was more evident in first salvage than in later salvage (OR 8.40 vs. 1.82), whereas its association with OS was more evident in second or later salvage (median OS 14.5 vs. 4.3 months; P=0.005).</p><p><strong>Conclusion: </strong>Early lymphocyte expansion during blinatumomab therapy, reflected by ΔALC14, is an independent predictor of treatment response. ALC monitoring may offer a simple, cost-effective tool for risk stratification.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261487003"},"PeriodicalIF":2.8,"publicationDate":"2026-09-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13554671/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148920545","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A real-world, multi-center, prospective, observational study for paroxysmal nocturnal haemoglobinuria (PNH) in China: Baseline characteristics, disease burden and treatment patterns.","authors":"Bing Han, Hui Liu, Guangsheng He, Jinsong Jia, Mei Hong, Jianyu Weng, Shunqing Wang, Fengkui Zhang, Ting Niu, Tianjun Ma, Jinghan Xu, Rong Fu","doi":"10.1177/20406207261481224","DOIUrl":"10.1177/20406207261481224","url":null,"abstract":"<p><strong>Background: </strong>Paroxysmal nocturnal hemoglobinuria (PNH) is a rare life-threatening hematologic disorder with high thromboembolic mortality. In the context of large patient population and limited use of complement inhibitors, the clinical characteristics and disease progression of patients in China have not been well studied.</p><p><strong>Objectives: </strong>The China PNH Registry was initiated to advance understanding of the disease by collecting data and describing PNH disease burden, progression, and clinical outcomes with different medical interventions.</p><p><strong>Design: </strong>This is a multi-center, prospective observational study in patients with PNH regardless of treatment for PNH.</p><p><strong>Methods: </strong>This real-world study enrolled 716 PNH patients irrespective of treatment, collecting baseline demographics, clinical/laboratory data, and treatment patterns (including eculizumab dosing and safety) for descriptive analyses across PNH subtypes.</p><p><strong>Results: </strong>52.0% of the enrolled patients had classic PNH, 47.2% had bone marrow failure (BMF/PNH), and 0.8% had subclinical PNH. Classic PNH patients had a higher proportion of PNH red blood cells (RBCs) (39.0% vs. 18.0%), PNH neutrophils (86.1% vs. 52.4%), and PNH monocytes (90.2% vs. 47.7%) than BMF/PNH patients. 343/496 patients had LDH > 1.5 ULN, with a higher proportion in classic PNH than BMF/PNH (74.7% vs. 63.1%). 582 patients had at least one of the PNH-related symptoms, the most common being fatigue (63.7%), red/dark urine (46.6%). Although eculizumab has become a reimbursable complement inhibitor in China, only 28.5% of the patients received eculizumab treatment during the study period. The other main treatment methods were supportive care (46.6%), corticosteroids (28.1%), and RBC transfusion (7.5%).</p><p><strong>Conclusion: </strong>Current data revealed a gap between real-world practice and guideline recommendations, indicating that standard treatment - particularly complement inhibitors - remains underutilized despite being essential for improving long-term patient outcomes.</p><p><strong>Trial registry name: </strong>The China Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry.</p><p><strong>Registration number: </strong>NCT06154512.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261481224"},"PeriodicalIF":2.8,"publicationDate":"2026-09-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13547791/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148906674","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"From toxicity to immunity: Evaluating CAR-T and conventional therapies in multiple myeloma through quality-adjusted life year (QALY) outcomes.","authors":"Guluzar Gulnur Itez, Asuman Sunguroğlu","doi":"10.1177/20406207261484713","DOIUrl":"10.1177/20406207261484713","url":null,"abstract":"<p><p>Multiple myeloma (MM) remains characterized by recurrent relapse and cumulative treatment burden despite major therapeutic advances. This study compared conventional stepwise regimens with CAR-T cell therapy in terms of QoL, toxicity, cost, and ethical considerations within a model-based comparative framework. This comparative pharmacoeconomic modeling analysis was based on published clinical trial data. Kaplan-Meier survival curves were digitized to estimate mean overall survival (OS) and progression-free survival (PFS) using area-under-the-curve integration. Treatment costs were calculated based on published pricing and trial-derived treatment durations. A simple and transparent ECOG-based utility model was developed to enable clinicians, researchers, and health policy authorities to estimate quality-adjusted life years (QALY) and incremental cost-effectiveness ratios (ICER) across regimens spanning heterogeneous treatment lines. The present analysis synthesizes outcome data from 19 pivotal trials and real-world cohorts (N = 7,793 patients). Among heavily pretreated patients, CAR-T therapy approximately doubled OS and PFS compared with other late-line regimens and yielded higher QALY estimates with lower cumulative treatment burden. Daratumumab-based combinations improved outcomes but reached very high costs (∼USD 1 million/patient), while carfilzomib-based regimens remained costly but clinically important for high-risk disease. VMP represented a practical lower-cost option for transplant-ineligible or resource-limited patients. In treatment-line-stratified analysis, later-line/RRMM regimens had higher median ICER-equivalent values than early-line/induction regimens (USD 739,050/QALY vs USD 218,687/QALY; 3.4-fold higher), whereas CAR-T regimens showed a median ICER-equivalent estimate of USD 299,723/QALY, approximately 2.5-fold lower than later-line/RRMM conventional regimens. Within this model-based comparative framework, CAR-T provided substantial survival and quality-adjusted outcome gains after three or more prior therapy lines, with promising potential for earlier use. Despite its upfront single-payment structure, CAR-T did not show a disproportionate ICER-equivalent burden compared with later-line conventional regimens. However, limited global availability raises ethical concerns regarding access, infrastructure, reimbursement, and equity. Further studies incorporating longer follow-up and patient-level QoL data are needed to refine its role in multiple myeloma care.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261484713"},"PeriodicalIF":2.8,"publicationDate":"2026-09-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13542552/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148897769","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comparative efficacy and safety of JAK inhibitor-based strategies in JAK inhibitor-naïve myelofibrosis: A systematic review and network meta-analysis.","authors":"Zhongwang Wang, Linfeng Li, Ting Niu, He Li","doi":"10.1177/20406207261484707","DOIUrl":"10.1177/20406207261484707","url":null,"abstract":"<p><strong>Background: </strong>Myelofibrosis is a debilitating neoplasm with progressive bone marrow fibrosis, splenomegaly, and leukemic transformation risk. While ruxolitinib remains the standard for intermediate/high-risk MF, new strategies, including novel JAK inhibitors and ruxolitinib-based combinations, aim to improve efficacy and safety. However, direct comparisons of these strategies in JAK inhibitor-naïve patients remain limited.</p><p><strong>Objectives: </strong>To compare the efficacy and safety of ruxolitinib, novel inhibitors, and combinations in JAKi-naïve MF.</p><p><strong>Design: </strong>A systematic review and network meta-analysis of RCTs.</p><p><strong>Data sources and methods: </strong>We searched electronic databases through November 2025 for RCTs evaluating JAK inhibitors in JAKi-naïve MF. The efficacy endpoints were spleen volume reduction of ≥35% (SVR35) and total symptom score reduction of ≥50% (TSS50) at week 24. Safety outcomes included adverse events (AEs) as well as treatment discontinuation rates.</p><p><strong>Results: </strong>Seven RCTs (n=2,254) were analyzed. Ruxolitinib combinations significantly improved SVR35 over ruxolitinib (OR = 3.63) but not TSS50. Momelotinib showed comparable SVR35 (OR = 0.886), while selective JAK2 inhibitors (fedratinib and pacritinib) were numerically inferior in efficacy. Regarding safety, fedratinib had the lowest risk of grade ≥3 thrombocytopenia (OR = 0.204); pacritinib, the lowest risk of all-grade thrombocytopenia (OR = 0.209); momelotinib, the lowest risk of all-grade anemia (OR = 0.226) and grade ≥3 anemia (OR=0.187). JAK2 inhibitors significantly increased diarrhea risk (OR=6.19). Notably, ruxolitinib monotherapy maintained the lowest AEs-related discontinuation rates in patients with platelets ≥ 50×10^9/L.</p><p><strong>Conclusion: </strong>In first-line therapy, our findings support a stratified approach. Ruxolitinib combinations are preferred for patients where maximal splenic debulking is the primary goal. Selective JAK2 inhibitors provide a safe option for patients with profound thrombocytopenia. Momelotinib matches ruxolitinib's efficacy via JAK1/2 inhibition while being optimal for baseline anemia via ACVR1 inhibition. Crucially, ruxolitinib remains the benchmark for symptom control and treatment retention in patients with adequate platelets.</p><p><strong>Registration: </strong>CRD420261330640.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261484707"},"PeriodicalIF":2.8,"publicationDate":"2026-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13646515/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149138270","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Quality improvement project - Implementation of subcutaneous immunoglobulin in patients with haematological disorders and secondary immunodeficiency.","authors":"Amira Chaudry, Rebecca Culpin, Shereen Elshazly","doi":"10.1177/20406207261475918","DOIUrl":"10.1177/20406207261475918","url":null,"abstract":"<p><strong>Background: </strong>Immunoglobulins (Ig) play a crucial role in the immune system's ability to fight infections. In incidences when their production and function is disrupted, such as in secondary immunodeficiency (SID), patients often face increased frequency and severity of infection, particularly respiratory tract infections. Ig replacement therapy administered intravenously (IVIG) or subcutaneously (SCIG) is recommended for treatment of SID, including SID caused by haematological malignancies. Subregional Ig assessment panels (SRIAPs) and prefilled syringes (PFS) for SCIG may positively impact services across the Trust and wider National Health Service (NHS) and increase patient satisfaction and treatment outcomes.</p><p><strong>Objectives: </strong>Investigate the improvement in service delivery and patient satisfaction following the switch from IVIG to SCIG PFS manual push in patients with SID following an East of England Immunoglobulin Assessment Panel (EOE IAP) review.</p><p><strong>Design: </strong>Following establishment of the EOE IAP, three cohorts of patients with haematological disorders and SID, currently receiving IVIG across the Mid and South Essex NHS Foundation trust, were reviewed before switching to SCIG PFS. This manuscript reports the findings from one of the three cohorts, based at Broomfield Hospital.</p><p><strong>Methods: </strong>Following clinical screening, patients were reviewed by the local SRIAP to assess Ig use and suitability for transfer from IVIG to SCIG PFS. Patient outcomes and satisfaction, financial measurements, and prescribing information after transition were recorded.</p><p><strong>Results: </strong>SRIAP review of Ig prescribing for ten patients resulted in six transitioning from IVIG to SCIG PFS. Patients reported improved infection rates and satisfaction with home-based SCIG PFS treatment. Service outcomes reported savings in prescribing, pharmacy, and nursing time, as well as an increase in therapy chair capacity.</p><p><strong>Conclusion: </strong>SRIAP reviews and switching to SCIG PFS can improve patient satisfaction and outcomes, help clinically vulnerable patients shield via home-based treatment, improve service efficacy and lower total treatment costs, whilst promoting Ig stewardship.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261475918"},"PeriodicalIF":2.8,"publicationDate":"2026-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13538992/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148888168","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"More precise risk stratification for de novo acute myeloid leukemia with double-mutation CEBPA.","authors":"Jing Jing Liu, Hai Ping Yang, Meng Hu","doi":"10.1177/20406207261485720","DOIUrl":"10.1177/20406207261485720","url":null,"abstract":"<p><strong>Background: </strong>CEBPA double-mutation (CEBPAdm) defines a favorable-risk subgroup of acute myeloid leukemia (AML), but marked heterogeneity exists, and relapse remains common. More precise prognostic stratification is urgently needed for this population.</p><p><strong>Objectives: </strong>To identify reliable prognostic factors and establish a refined risk stratification model for de novo AML with CEBPAdm.</p><p><strong>Design: </strong>Single-center retrospective observational cohort study.</p><p><strong>Methods: </strong>Samples were collected from AML patients at diagnosis for analysis. The proportions of different surface antigens in the bone marrow were evaluated via flow cytometry. The detection of mutations in AML patients was based on next-generation sequencing (NGS). Multiparametric flow cytometry (MFC) can detect minimal residual disease (MRD). The data were analysed via Statistical Program for Social Sciences Version 27.0 (SPSS 27.0).</p><p><strong>Results: </strong>①Univariate analysis revealed that high expression of cluster of differentiation antigen 33 (CD33)was unfavourable prognostic factor (<i>P</i>=0.028). ②There was a statistically significant difference between patients with more than 2 companion gene mutations (χ²=9.868, <i>P</i>=0.002). ③Notably, patients who became MRD-negative after induction chemotherapy had more favourable outcomes (χ²=3.847, <i>P</i>=0.05). ④Overall survival (OS) was worse in the high expression of CD33 group (43.00 months vs. 39.00 months, Log Rank <i>P</i>=0.024).</p><p><strong>Conclusions: </strong>These findings provide a more precise restratification for CEBPAdm patients on the basis of the expression of CD33.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261485720"},"PeriodicalIF":2.8,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13535027/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148881366","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}