Therapeutic Advances in Hematology最新文献

{"title":"Nine areas with outstanding challenges for hemophilia B research.","authors":"Jan Astermark, Cédric Hermans, Peter J Lenting, James S O'Donnell","doi":"10.1177/20406207251409300","DOIUrl":"10.1177/20406207251409300","url":null,"abstract":"<p><p>Hemophilia A and B are rare, X-linked bleeding disorders characterized by deficiencies in coagulation factor VIII (FVIII) and factor IX (FIX), respectively. Numerous advances have helped to reduce disease burden. However, hemophilia B is not as well studied as hemophilia A, likely reflecting its lower prevalence. Clinical management of hemophilia B has often relied on inference and extrapolation from hemophilia A. Despite being part of the same tenase complex, as enzyme (FIX) and cofactor (FVIII) when activated, with the main task being to activate factor X in the intrinsic pathway, the FIX and FVIII proteins display several molecular differences. These have the potential to impact the clinical phenotype of hemophilia, affect monitoring, and influence treatment options. Consequently, hemophilia B presents several outstanding challenges, requiring a greater degree of understanding and/or attention across a range of areas. Some of these challenges relate to the FIX molecule, with more knowledge needed in relation to: the biological/clinical impact of underlying genetic changes; hemostatic implications of the extravascular distribution of FIX; and FIX clearance. Other challenges relate to clinical management: determining the best ways to monitor the true biological activity of FIX; clarifying the relationship between FIX plasma levels and clinical outcomes when treating patients; inhibitors; affected girls and women; and appreciating the value of novel treatment approaches, while considering possible breakthrough bleeds, thrombosis, and monitoring. In addition, concerted effort is required to address global disparities, which can particularly affect hemophilia B. Identifying such challenges may help to facilitate research that will further existing knowledge, with better understanding being crucial for achieving health equity between hemophilia A and B.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207251409300"},"PeriodicalIF":3.1,"publicationDate":"2026-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12847661/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146087155","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":"Transfusion-induced hemolysis in warm autoimmune hemolytic anemia with autoantibodies that have anti-e specificity: a case report and systematic review of the literature.","authors":"Faramarz Jabbari-Zadeh, Ortenc Hoxha, Alan Gob, Ziad Solh, Benjamin Chin-Yee, Cyrus C Hsia","doi":"10.1177/20406207251410168","DOIUrl":"10.1177/20406207251410168","url":null,"abstract":"<p><p>Warm autoimmune hemolytic anemia (wAIHA) is characterized by autoantibodies against proteins that comprise the Rh antigen system along with hemolysis. The presence of specific autoantibodies, especially autoantibodies with anti-e specificity, is rare. The pathophysiology, management, and outcomes in this condition, including with transfusion of e-antigen positive packed red blood cells (pRBCs), remain poorly understood. A 63-year-old woman presented with fatigue, jaundice, tea-colored urine, and dyspnea. She was diagnosed with wAIHA with anti-e specificity and developed worsening hemolysis after receiving a unit of e-antigen positive pRBCs with a drop in hemoglobin from 85 to 71 g/L. She achieved a complete response with a tapering course of prednisone and folate. We conducted a systematic review of the literature by searching MEDLINE, EMBASE, and Cochrane for articles on management of wAIHA with anti-e specificity published between 1946 and 2025. We identified eighteen cases of wAIHA mediated through autoantibodies with anti-e specificity, only one of which demonstrated hemolysis after transfusion with e-antigen positive pRBCs. Treatment with corticosteroids and rituximab was effective in many of these cases. This is the one of the few cases to demonstrate increased hemolysis after transfusion with e-antigen positive pRBCs in a patient with wAIHA and autoantibodies with anti-e specificity. In these patients, transfusion of e-antigen positive pRBCs should be avoided and if transfusion is necessary, it will be important to closely monitor for worsening hemolysis. Additionally, our case and those reported in the literature highlight the effectiveness of standard corticosteroid treatment for this rare type of wAIHA.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207251410168"},"PeriodicalIF":3.1,"publicationDate":"2026-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12847652/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146087206","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":"The impact of clinical manifestations on the prognosis of diffuse large B-cell lymphoma.","authors":"Siqian Wang, Xi Xu, Yongyong Ma, Shanhu Qian, Liyuan Tang, Lan Sun, Zhijian Shen, Haige Ye, Honglan Qian, Songfu Jiang, Shujuan Zhou","doi":"10.1177/20406207251410405","DOIUrl":"10.1177/20406207251410405","url":null,"abstract":"<p><strong>Objective: </strong>This study aimed to investigate the recurrence patterns of diffuse large B-cell lymphoma (DLBCL) and assess the significance of clinical manifestation consistency at the time of recurrence.</p><p><strong>Methods: </strong>A total of 141 patients who were newly diagnosed with DLBCL between January 2015 and October 2024 and who initially achieved complete remission but eventually developed relapsed disease during follow-up were retrospectively identified, their recurrence circumstances were assessed, and their clinical manifestations at diagnosis and recurrence were compared. The timing of recurrence was categorized as within 1 year of postchemotherapy or later.</p><p><strong>Results: </strong>A total of 113 (80.1%) patients presented with clinical manifestations leading to the diagnosis of recurrence. A total of 87 (61.7%) patients presented similar clinical manifestations at recurrence to those observed during initial diagnosis. Importantly, for patients who relapsed within 1 year of chemotherapy, those with inconsistent manifestations at recurrence had significantly poorer prognoses and lower survival rates (complete remission (CR) rate, 7.4%; 1-year overall survival (OS), 37.5%; 1-year progression-free survival (PFS), 18.5%) than those with consistent manifestations (CR rate, 20.8%; 1-year OS, 34.0%; 1-year PFS, 22.6%; PFS, <i>p</i> = 0.046; OS, <i>p</i> = 0.049). In contrast, for patients whose recurrence occurred more than 1 year postchemotherapy, consistent manifestations were associated with better survival outcomes (CR rate, 44.1%; 1-year OS, 61.8%; 1-year PFS, 52.9%).</p><p><strong>Conclusion: </strong>The timing of recurrence and the consistency of clinical manifestations are important factors influencing the prognosis of DLBCL. Specifically, early recurrence with inconsistent manifestations is associated with worse outcomes, whereas recurrence with consistent manifestations 1 year postchemotherapy is correlated with an improved prognosis.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207251410405"},"PeriodicalIF":3.1,"publicationDate":"2026-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12847682/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146087224","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":"Outcomes in participants switching from FVIII replacement therapy to efanesoctocog alfa prophylaxis in XTEND-1: a post hoc analysis.","authors":"Sophie Susen, Roshni Kulkarni, Keiji Nogami, Flora Peyvandi, Barbara Konkle, Elena Santagostino, Umer Khan, Annemieke Willemze, Linda Bystrická, Jennifer Dumont, Pratima Chowdary","doi":"10.1177/20406207251398986","DOIUrl":"10.1177/20406207251398986","url":null,"abstract":"<p><strong>Background: </strong>Efanesoctocog alfa is a first-in-class high-sustained factor VIII (FVIII) replacement therapy. In the phase III XTEND-1 (NCT04161495) study, once-weekly efanesoctocog alfa prophylaxis (50 IU/kg) was well-tolerated and achieved high-sustained factor levels in the normal to near-normal range (>40%) for most of the week.</p><p><strong>Objective: </strong>To report outcomes in previously treated participants with severe haemophilia A aged ⩾12 years from an observational study who switched to efanesoctocog alfa prophylaxis during XTEND-1.</p><p><strong>Design: </strong>Paired assessment of participants from an observational study who enrolled in the phase III XTEND-1 study.</p><p><strong>Methods: </strong>Seventy-eight participants switched from marketed standard half-life (SHL) or extended half-life (EHL) FVIII prophylaxis to once-weekly efanesoctocog alfa prophylaxis (50 IU/kg). Endpoints included annualized bleed rates (ABRs), treatment of bleeding episodes, injection frequency and FVIII consumption.</p><p><strong>Results: </strong>Pre-study, 44 (56%) and 34 (44%) participants received SHL FVIII or EHL FVIII prophylaxis, respectively. In the overall population, a significant reduction in ABR from 2.96 to 0.69 (<i>p</i> < 0.0001) was observed following the switch to efanesoctocog alfa prophylaxis as well as reductions in spontaneous, traumatic, joint and spontaneous joint ABRs (<i>p</i> < 0.0001). Significant reductions in mean weekly injection frequency were observed, from 2.8 to 1.0 in the SHL FVIII cohort (<i>p</i> < 0.0001) and from 1.8 to 1.0 in the EHL FVIII cohort (<i>p</i> < 0.0001). Mean annualized factor consumption reduced by 47% in the SHL FVIII cohort and 30% in the EHL FVIII cohort.</p><p><strong>Conclusion: </strong>Collectively, the results of this post hoc analysis demonstrate the benefits of once-weekly efanesoctocog alfa prophylaxis over SHL or EHL FVIII prophylaxis on bleed rates, injection frequency and consumption.</p><p><strong>Trial registration: </strong>Observational study: 242HA201/OBS16221; XTEND-1: NCT04161495 (https://clinicaltrials.gov/study/NCT04161495).</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207251398986"},"PeriodicalIF":3.1,"publicationDate":"2026-01-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12824134/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146053589","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 Busulfan-Fludarabine versus Busulfan-Cyclophosphamide as a conditioning regimen prior to hematopoietic stem cell transplant in hematologic malignancy patients: a meta-analysis of randomized controlled trials and observational studies.","authors":"Aizaz Ali, Muhammad Abdullah Ali, Abdullah Afridi, Hammad Ali, Umair Ul Haq, Muhammad Osama, Muhammad Umar Afridi, Touba Azeem, Mian Aban Masaud, Fazia Khattak, Fiza Safeer, Areeba Khan, Muhammad Sufyan Darwesh, Nimra Ehsan, Wahab Zia, Gulmeena Riffat, Abdul Moeez, Kamil Ahmad Kamil","doi":"10.1177/20406207251407534","DOIUrl":"10.1177/20406207251407534","url":null,"abstract":"<p><strong>Background: </strong>Conditioning regimen selection significantly impacts the outcomes of allogeneic hematopoietic stem cell transplantation (HSCT) in patients with hematologic malignancies. However, comparative evidence between Busulfan-Fludarabine and Busulfan-Cyclophosphamide remains inconclusive.</p><p><strong>Objectives: </strong>To compare the efficacy and safety of Busulfan-Fludarabine versus Busulfan-Cyclophosphamide as conditioning regimens prior to HSCT.</p><p><strong>Design: </strong>Systematic review and meta-analysis conducted in accordance with PRISMA 2020 guidelines.</p><p><strong>Data sources and methods: </strong>MEDLINE, CENTRAL, and Embase were searched through October 2024. Eligible studies included randomized controlled trials and cohort studies comparing Busulfan-Fludarabine and Busulfan-Cyclophosphamide in HSCT recipients. Primary outcomes included overall survival and acute graft-versus-host disease (GVHD). Risk ratios (RRs) with 95% confidence intervals (CIs) were pooled using random-effects models. Risk of bias was assessed using RoB 2.0 and the Newcastle-Ottawa Scale.</p><p><strong>Results: </strong>Eighteen studies (6 randomized controlled trials, 12 cohorts) comprising 2888 patients (1539 received Busulfan-Fludarabine, 1349 received Busulfan-Cyclophosphamide) were included. Busulfan-Fludarabine showed higher 1-year overall survival (RR 1.13, 95% CI: 1.01-1.26), but no significant difference at 2 or 5 years. Grade III-IV acute GVHD was significantly lower with Busulfan-Cyclophosphamide (RR 0.45, 95% CI: 0.21-0.98). Busulfan-Fludarabine resulted in lower 5-year non-relapse mortality (RR 0.63, 95% CI: 0.48-0.83), and significantly reduced pulmonary and gastrointestinal toxicities. Event-free survival favored Busulfan-Fludarabine at 2 and 5 years. No significant differences were found for relapse-related mortality, chronic GVHD, cytomegalovirus infection, or total mortality. Meta-regression identified conditioning regimen and graft source as contributors to 1-year survival variability.</p><p><strong>Conclusion: </strong>Busulfan-Fludarabine offers early survival and toxicity advantages, while Busulfan-Cyclophosphamide may reduce severe acute GVHD. Conditioning regimen selection should consider patient-specific factors. Further prospective trials are needed to guide clinical decisions.</p><p><strong>Trial registration: </strong>PROSPERO ID: CRD42025630836.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207251407534"},"PeriodicalIF":3.1,"publicationDate":"2026-01-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12811581/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145998829","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":"Clinical practice of the endothelial activation and stress index and its modified versions in multiple myeloma and the effects on prognosis.","authors":"Vildan Gursoy, Ozgur Omer Gul, Elif Kose, Sevil Sadri","doi":"10.1177/20406207251387409","DOIUrl":"10.1177/20406207251387409","url":null,"abstract":"<p><strong>Objective: </strong>Here, we aimed to evaluate the prognostic values of the endothelial activation and stress index (EASIX) and its derivatives, modified EASIX (mEASIX) and simplified EASIX (sEASIX), in patients with multiple myeloma (MM).</p><p><strong>Methods: </strong>The data of 134 newly diagnosed MM patients between January 2020 and December 2024 were retrospectively analyzed. Patients were divided into groups based on EASIX and its derivatives, and the outcomes of survival rates in groups were compared. Optimal cut-off points were determined using the receiver-operating characteristic analysis, and mortality predictive values of the scores were investigated. The independent prognostic factors were evaluated through univariate and multivariate Cox regression analyses.</p><p><strong>Results: </strong>The optimal cut-off point for EASIX was detected as >1.03, and survival times were significantly shorter in patients with higher EASIX scores (<i>p</i> < 0.001). Even so, the optimal cut-off point for mEASIX was >26.5, showing higher specificity (area under curve (AUC) = 0.663; <i>p</i> = 0.003). The sEASIX score predicted lower mortality (AUC = 0.586; <i>p</i> = 0.123). In multivariate analysis, high EASIX scores, not performing autologous stem cell transplantation, and not receiving immunomodulatory therapy were identified as independent negative prognostic factors for survival.</p><p><strong>Conclusion: </strong>EASIX, especially mEASIX, is a valuable prognostic tool for predicting survival in MM patients. EASIX can be easily integrated into clinical practice due to its simple computability and reliance on commonly used laboratory parameters. However, larger prospective studies are needed to determine how these scores can be integrated with traditional prognostic systems such as the International Staging System (ISS) and Revised-ISS.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207251387409"},"PeriodicalIF":3.1,"publicationDate":"2026-01-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12775338/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145935122","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 chimeric antigen receptor T-cell therapy in relapsed/refractory large B-cell lymphoma: a systematic review and meta-analysis.","authors":"Qiang Li, Shenghua Xu, Yun Zhong, Peng Rao, Hui Huang, Yan Huang","doi":"10.1177/20406207251407511","DOIUrl":"10.1177/20406207251407511","url":null,"abstract":"<p><strong>Background: </strong>Individuals diagnosed with relapsed or refractory large B-cell lymphoma (R/R LBCL) typically exhibit a dismal prognosis when treated with conventional therapeutic modalities. CD19-targeted chimeric antigen receptor T (CAR-T) cell therapy has brought about a paradigm shift in the treatment paradigm of this disease. Nevertheless, a comprehensive assessment of the efficacy and safety profiles of diverse CAR-T products (e.g., axicabtagene ciloleucel (axi-cel), tisagenlecleucel (tisa-cel), and lisocabtagene maraleucel (liso-cel)) is imperative.</p><p><strong>Objective: </strong>This systematic review aims to systematically summarize and evaluate the efficacy and safety of CAR T-cell therapies for R/R LBCL through systematic review and meta-analytic approaches.</p><p><strong>Design: </strong>This is a systematic review and meta-analysis.</p><p><strong>Data sources and methods: </strong>Relevant studies were identified by systematically searching PubMed and Web of Science until November 29, 2023. Cohort studies and clinical trials were incorporated, with the inclusion of single-arm studies. CAR T-cell therapies are involved in tisa-cel, axi-cel, and liso-cel. Proportions and their 95% confidence intervals were calculated using standard meta-analytic approaches.</p><p><strong>Results: </strong>Thirty-seven studies were included in meta-analyses, liso-cel demonstrated equivalent overall survival rates (70.3%) to axi-cel (65.6%) at 12 months post-treatment, whereas tisa-cel was 48.0%. Objective response rate for liso-cel was comparable to axi-cel (79.0% vs 76.8%, <i>p</i> _{meta-regression} = 0.74), both of which were notably higher than those observed for tisa-cel (58.3%, both <i>p</i> _{meta-regression} < 0.05). Regarding safety assessments, liso-cel exhibited the lowest cytokine release syndrome rate at 43.0%, followed by tisa-cel at 70.9%, and axi-cel at 87.9%. However, tisa-cel had the lowest incidence of neurologic events (14.9%), in contrast to liso-cel (21.1%) and axi-cel (52.3%).</p><p><strong>Conclusion: </strong>Based on the available evidence, liso-cel has shown promising efficacy and a manageable safety profile in patients with R/R LBCL, when compared to axi-cel and tisa-cel. However, real-world data on liso-cel are limited.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207251407511"},"PeriodicalIF":3.1,"publicationDate":"2026-01-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12764758/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145906703","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 avatrombopag in aplastic anemia: a comprehensive review of clinical evidence.","authors":"Mostafa F Mohammed Saleh, Abdulrahman Nasiri, Ahmed Kotb Abdrabou, Alfadil Haroon, Hadeel Samarkandi, Hazza Alzahrani, Mahmoud Aljurf, Ali Alahmari","doi":"10.1177/20406207251409305","DOIUrl":"10.1177/20406207251409305","url":null,"abstract":"<p><p>Aplastic anemia is a bone marrow failure disorder marked by cytopenias that impair oxygen delivery, immune defense, and hemostasis. Standard therapy traditionally combines immunosuppression with or without hematopoietic stem cell transplantation, and more recently incorporates thrombopoietin receptor agonists to stimulate residual hematopoiesis. Eltrombopag improved outcomes when added to immunosuppressive therapy, but its association with hepatotoxicity limits its suitability for patients with underlying liver disease or elevated baseline liver enzymes. Avatrombopag is a newer oral thrombopoietin receptor agonist that does not require dietary restrictions and does not undergo significant hepatic metabolism, which offers a potential therapeutic advantage in settings where liver function is compromised. This review evaluated ten clinical studies published from 2023 to October 2025 that investigated avatrombopag in acquired aplastic anemia across varied patient populations, including treatment-naive, relapsed or refractory cases, older adults, and patients with secondary aplastic anemia related to chemoradiation. Across these studies, overall response rates ranged from 55% to 85%, and complete response rates reached up to one-third of treated patients. Response onset typically occurred within 1-2 months, which aligns with clinical decision timelines for assessing therapeutic benefit. Avatrombopag supported reductions in transfusion requirements and sustained hematologic improvement in both severe and non-severe disease. Patients previously intolerant or non-responsive to eltrombopag also demonstrated clinical improvement, which suggests pharmacologic differences translate into meaningful therapeutic effects. Importantly, avatrombopag demonstrated a favorable safety profile in all reviewed settings. Reports did not identify clinically relevant hepatotoxicity, clonal evolution, or treatment-limiting adverse effects. Its tolerability in patients with liver dysfunction distinguishes it from earlier agents in this drug class. Ongoing trials will clarify optimal dosing strategies and define its future role within first-line therapy and salvage treatment pathways for aplastic anemia.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207251409305"},"PeriodicalIF":3.1,"publicationDate":"2026-01-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12759122/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145901204","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":"Mogamulizumab plus etoposide in the management of mycosis fungoides with blood involvement: a case report.","authors":"Daniel Nieto Rodríguez, Elena Sendagorta","doi":"10.1177/20406207251388057","DOIUrl":"10.1177/20406207251388057","url":null,"abstract":"<p><p>Mycosis fungoides (MF) and Sézary syndrome are primary cutaneous T-cell lymphomas with widely varying courses of disease and prognosis. Here, we report the case of a patient with MF with blood involvement who experienced many lines of skin-directed and systemic therapies throughout their disease course. The male patient presented with scaly, itchy, and painful patches on his trunk and upper extremities in 2012 and was diagnosed with MF, which progressed to erythroderma with blood involvement. Various skin-directed therapies were prescribed; however, in 2014, progression of skin lesions ensued. The patient achieved a partial response with methotrexate but discontinued after ~12 months due to elevated transaminases. Following treatment with bexarotene then gemcitabine, CHOP chemotherapy was initiated in December 2019, but, after a period of partial skin response, the patient relapsed with progression of skin lesions. Mogamulizumab was initiated in May 2020 (1 mg/kg intravenously on days 1, 8, 15, and 22 of the first 28-day cycle, then on days 1 and 15 of subsequent cycles) with a good initial response in skin and blood. Disease progression in the skin occurred in December 2020; mogamulizumab was continued, and the patient achieved remission with the addition of etoposide and prednisone in August 2021. Onset of progression occurred in July 2022, which did not respond to repeat combination treatment. In October 2022, the patient was diagnosed with large cell CD30⁺ transformation, and the therapeutic approach was changed to extracorporeal photopheresis, brentuximab vedotin, and topical steroids. The patient died in February 2023 due to sepsis. Our experience adds to the limited evidence that mogamulizumab may be continued in combination with etoposide following disease progression in patients with MF with blood involvement; however, more research is needed on the efficacy and safety of this approach.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207251388057"},"PeriodicalIF":3.1,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12759120/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145901172","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":"Ibrutinib oral suspension bioavailability and compatibility for optimal enteral administration route.","authors":"Jonas Paludo, Lisa Nodzon, Xavier Woot de Trixhe, Michelle Pacia, Heena Mavani, Harisha Atluri, Thomas J Huemann, Jessica Shelly, Yemin Liu, Shayna Sarosiek","doi":"10.1177/20406207251399466","DOIUrl":"10.1177/20406207251399466","url":null,"abstract":"<p><strong>Background: </strong>Ibrutinib is the only Bruton tyrosine kinase inhibitor (BTKi) with once-daily oral capsule, tablet, and oral suspension formulations approved in the United States across indications of chronic lymphocytic leukemia/small lymphocytic lymphoma, Waldenström macroglobulinemia, and previously treated chronic graft-versus-host disease, and for mantle cell lymphoma in Europe. Patients with difficulty swallowing capsules or tablets may require alternative formulations or administration options to optimize treatment.</p><p><strong>Objectives: </strong>To evaluate the relative bioavailability of ibrutinib oral suspension relative to capsule or tablet formulations and to examine compatibility for optimal administration methods.</p><p><strong>Methods: </strong>Relative bioavailability of ibrutinib oral suspension was evaluated in healthy volunteers or patients in comparison to capsule or tablet formulations. Dose recovery and delivery, presence of impurities, particle size, and hold time were evaluated after in vitro mock oral suspension administration via syringe and nasogastric and percutaneous endoscopic gastrostomy (PEG) tubes.</p><p><strong>Results: </strong>Clinical bioavailability was comparable for ibrutinib oral suspension and capsules (420 mg/day) under the fasted state in healthy volunteers. Dose-normalized pharmacokinetic values were similar across ibrutinib formulations in patient studies. All evaluated enteral tubes achieved 90%-110% ibrutinib dose recovery regardless of tube type or syringe after 2 × 3 mL water rinses. Oral suspension preservative may be adsorbed into enteral tubes after a 60-minute hold time.</p><p><strong>Conclusion: </strong>Ibrutinib oral suspension bioavailability was comparable with ibrutinib tablet and capsule formulations. When dosed via standard enteral tube administration methods, ibrutinib oral suspension is stable and compatible with polyurethane, silicone, or polyvinyl nasogastric or PEG tubes. To ensure full dose recovery and prevent drug preservative adsorption into tubes, ibrutinib oral suspension should be administered immediately and followed by two rinses of 3 mL of water each. Ibrutinib oral suspension is a viable BTKi treatment option for patients requiring or preferring alternatives to oral capsules and tablets.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"16 ","pages":"20406207251399466"},"PeriodicalIF":3.1,"publicationDate":"2025-12-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12743846/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145858079","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}