Therapeutic Advances in Hematology最新文献

{"title":"Ruxolitinib treatment in patients with polycythemia vera reduces <i>JAK2</i> variant allele frequency and improves symptom burden and hematocrit control.","authors":"Claire N Harrison, Jean-Jacques Kiladjian, J E Hamer-Maansson, Ahmad Naim, Francesca Palandri, Francesco Passamonti","doi":"10.1177/20406207261425083","DOIUrl":"10.1177/20406207261425083","url":null,"abstract":"<p><strong>Background: </strong>In RESPONSE/RESPONSE-2, ruxolitinib was superior to best available therapy (BAT) in patients with polycythemia vera (PV).</p><p><strong>Objective: </strong>Report a post hoc pooled RESPONSE/RESPONSE-2 analysis.</p><p><strong>Design: </strong>RESPONSE/RESPONSE-2 were randomized, open-label phase III trials. Adults with hydroxyurea-resistant/intolerant PV were randomized 1:1 to ruxolitinib or BAT; crossover to ruxolitinib was allowed after primary analysis.</p><p><strong>Methods: </strong><i>JAK2</i>V617F allele burden, hematocrit control, and Myeloproliferative Neoplasm Symptom Assessment Form total symptom score (MPN-SAF TSS) were assessed.</p><p><strong>Results: </strong>Among 371 patients, mean <i>JAK2</i>V617F allele burden declined from baseline (ruxolitinib, 66.1%; crossover, 69.5%) through week 208 (41.4%; 37.1%). Significantly more ruxolitinib versus BAT patients achieved hematocrit control at week 28 (62.0% vs 18.2%; <i>p</i> < 0.0001) and ⩾50% reduction in MPN-SAF TSS at week 16 (48.7% vs 18.0%; <i>p</i> < 0.0001).</p><p><strong>Conclusion: </strong>Patients receiving ruxolitinib experienced decreased <i>JAK2</i>V617F allele burden, durable hematocrit control, and better symptom improvements versus BAT, reinforcing ruxolitinib clinical benefit.</p><p><strong>Trial registration: </strong>RESPONSE: https://clinicaltrials.gov/study/NCT01243944; RESPONSE-2: https://clinicaltrials.gov/study/NCT02038036.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261425083"},"PeriodicalIF":2.8,"publicationDate":"2026-03-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13014013/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147521979","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":"Prefibrotic myelofibrosis: recent updates in diagnosis, prognostication, and management 2026.","authors":"Noor Al-Zubaidi, Katherine Makris, Priya Sriskandarajah","doi":"10.1177/20406207261432438","DOIUrl":"10.1177/20406207261432438","url":null,"abstract":"<p><p>Prefibrotic primary myelofibrosis (pre-PMF) is a subtype of primary myelofibrosis, having only been formally defined as a distinct entity since 2016. Diagnosis, using clinical assessment, peripheral blood analysis, genetic and molecular analysis, and bone marrow biopsy, is needed to distinguish pre-PMF from other myeloproliferative neoplasms, in particular essential thrombocythemia. While the 2022 International Consensus Criteria and World Health Organization diagnostic criteria are used in current clinical practice to aid diagnosis, prognostication for pre-PMF remains challenging. Advances in molecular testing and cytogenetics may enable new risk stratification and prognostic tools to inform treatment decisions for pre-MF. In this review, we aim to give an overview on pre-PMF including the latest updates on diagnostics and prognostic indicators. Furthermore, due to limited reviews on pre-PMF, there still remains no standardized treatment pathway; thus, we summarize the current management advice as well as suggest further trials and areas of research that may improve care in pre-PMF patients.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261432438"},"PeriodicalIF":2.8,"publicationDate":"2026-03-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13010013/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147515031","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":"Assessment of flonoltinib maleate versus ruxolitinib phosphate in intermediate- to high-risk myelofibrosis (FMF-02): study protocol for a multicenter, randomized, open-label phase IIB trial.","authors":"Linyu Yang, Ke Tan, Rui Liang, Wei Zhang, Yiqun Wang, Lijuan Chen","doi":"10.1177/20406207261424845","DOIUrl":"10.1177/20406207261424845","url":null,"abstract":"<p><strong>Background: </strong>Myelofibrosis (MF) is a chronic myeloproliferative neoplasm characterized by progressive bone marrow fibrosis, splenomegaly, debilitating constitutional symptoms, and cytopenias. Despite the benefits of ruxolitinib in controlling symptoms and spleen volume, challenges such as myelosuppression and limited impact on underlying fibrosis persist, particularly in cytopenic patients. Flonoltinib maleate (FM), a novel JAK2/FLT3/CDK6 inhibitor, shows preliminary potential in improving hematologic parameters and reducing fibrosis.</p><p><strong>Objectives: </strong>To evaluate the efficacy of low-/high-dose FM compared with RUX (primary objective), along with safety and the pharmacokinetic profile of FM (secondary objectives), in patients with intermediate- to high-risk MF (Trial registration: NCT06457425).</p><p><strong>Design: </strong>FMF-02 is a multicenter, randomized, open-label, active-controlled, phase IIb clinical trial.</p><p><strong>Methods: </strong>Approximately 75 adults with primary or secondary MF will be randomized in a 1:1:1 ratio to receive low-dose FM (50 mg once daily), high-dose FM (100 mg once daily), or RUX (5, 15, or 20 mg twice daily, based on platelet count), with randomization stratified by the Dynamic International Prognostic Scoring System risk category. The primary endpoint is the proportion of patients achieving ⩾35% spleen volume reduction (SVR35) at week 24, as assessed by a blinded Independent Review Committee. Key secondary endpoints include the proportion with ⩾50% reduction in Total Symptom Score (TSS50), changes in myelofibrosis grade, objective remission rate [International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) criteria], and safety. Subjects in the RUX group who complete the 24-week treatment or experience disease progression due to splenomegaly will crossover to receive FM. All subjects will continue long-term therapy until meeting discontinuation criteria, followed by survival follow-up.</p><p><strong>Results: </strong>The study commenced in June 2024 and is currently ongoing. The results will provide comparative data on the efficacy and safety profiles of FM versus RUX, including analyses of spleen response, symptom burden, hematologic parameters, and bone marrow fibrosis.</p><p><strong>Conclusion: </strong>The FMF-02 trial is the first randomized phase IIb study directly comparing the novel inhibitor FM against RUX. Its findings are expected to generate pivotal evidence regarding whether FM offers superior or differentiated clinical benefits, thereby informing its potential as a frontline therapy for intermediate- to high-risk MF and guiding the design of future phase III studies.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261424845"},"PeriodicalIF":2.8,"publicationDate":"2026-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13009813/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147515025","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":"Direct and indirect medical costs associated with transfusion-dependent beta-thalassemia in Brazil, Thailand, and India.","authors":"Tulika Seth, Adisak Tantiworawit, Sandra R Loggetto, Mrudula B Glassberg, Pimwadee Khaikham, Paola Marinheiro, Jason Williams, Camila F Roubik, Christopher Lee, Nelly F Ly, Pooja Hindocha, Anita Mallya, Dorothea von Bredow, Yogesh Punekar, Ahmed Hnoosh","doi":"10.1177/20406207261416795","DOIUrl":"10.1177/20406207261416795","url":null,"abstract":"<p><strong>Background: </strong>The economic burden of blood transfusions (BTs) in transfusion-dependent β-thalassemia (TDT) is not well characterized in adults from Brazil, Thailand, and India.</p><p><strong>Objective: </strong>To assess direct and indirect costs of BTs in adults with TDT in three geographically distinct countries: Brazil, Thailand, and India.</p><p><strong>Design: </strong>Healthcare professionals (HCPs) and administrative personnel completed a cross-sectional survey to assess transfusion-associated direct costs and indirect costs among patients with TDT.</p><p><strong>Methods: </strong>Direct (blood collection, BTs, iron chelation therapy (ICT), transfusion-related adverse events (AEs)), and indirect costs (blood supply shortages, waiting time, patient, and caregiver time) per patient per year (PPPY) were calculated from survey data, and publicly available cost data.</p><p><strong>Results: </strong>Between February 2, 2024, and March 12, 2024, 54, 104, and 125 participants in Brazil, Thailand, and India, respectively, were included, mostly hematologists or pharmacists. Median number of BTs PPPY was 12 (Brazil), 6 (Thailand), and 20 (India). Physicians and nurses conducted pretransfusion monitoring, whereas nurses monitored during BTs and posttransfusion appointments. AEs were estimated in 15% (Brazil), 5% (Thailand), and 10% (India) of BTs. In the past 12 months, 72%, 50%, and 54% of participants experienced blood supply shortages in Brazil, Thailand, and India, respectively; 40%, 25%, and 70% of patients experienced iron overload due to BTs, and 30%, 20%, and 70% of patients received ICT due to BTs. Total direct costs PPPY (USD) were 4438 in Brazil, 1775 in Thailand, and 1991 in India; total indirect costs PPPY (USD) were 831 in Brazil, 392 in Thailand, and 715 in India.</p><p><strong>Conclusion: </strong>Results showed a significant burden of BTs in TDT on HCPs, patients, and caregivers, along with shortages and delays in blood supplies. These findings underscore the importance of enhancing supportive care for BTs and exploring alternative approaches to alleviate this burden.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261416795"},"PeriodicalIF":2.8,"publicationDate":"2026-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13010036/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147515053","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 phase I/II study of gilteritinib in combination with chemotherapy in newly diagnosed patients with AML in Asia: final analysis.","authors":"Masashi Sawa, Toshihiro Miyamoto, Hee-Je Kim, Yasushi Hiramatsu, June-Won Cheong, Takayuki Ikezoe, Tomoki Naoe, Koichi Akashi, Satoshi Morita, Masanori Kosako, Masaki Shimura, Wataru Terada, Takeshi Kadokura, Jason Hill, Shuichi Miyawaki, Stanley C Gill, Alexandra Heinloth, Nahla Hasabou","doi":"10.1177/20406207261419953","DOIUrl":"10.1177/20406207261419953","url":null,"abstract":"<p><strong>Background: </strong>Mutations in the <i>FMS</i>-like tyrosine kinase 3 (<i>FLT3</i>) gene are present in approximately 30% of patients with newly diagnosed (ND) acute myeloid leukemia (AML), and are associated with worse therapy outcomes compared to the general AML population. Gilteritinib, a selective oral FLT3 inhibitor, is a promising treatment option for this patient population.</p><p><strong>Objectives: </strong>To assess the safety and efficacy of gilteritinib in combination with induction and consolidation chemotherapy in Asian patients with ND, <i>FLT3</i>-mutated (<i>FLT3</i> ^mut+) AML.</p><p><strong>Design: </strong>This study was a phase I/II open-label, single-arm study. Herein, we present the final results from phase II.</p><p><strong>Methods: </strong>A total of 84 patients were enrolled in 33 centers across Japan, Korea, and Taiwan. All patients enrolled in phase II received induction and consolidation therapy with gilteritinib 120 mg/day plus chemotherapy (induction: ⩽2 cycles, idarubicin/cytarabine once-daily; consolidation: ⩽4 cycles, cytarabine twice-daily) followed by maintenance with gilteritinib 120 mg/day monotherapy (⩽26 cycles). The primary efficacy endpoint was the complete remission (CR) rate after induction therapy.</p><p><strong>Results: </strong>The primary endpoint of CR rate after induction was 50.0% (90% CI: 40.4-59.6). Gilteritinib in combination with chemotherapy achieved high composite CR (CRc; 86.6%, 95% CI: 77.3-93.1) rates after induction. The overall survival (OS) rate at 3 years was 71.6%, and the median OS was 48.2 months; however, due to the immaturity of the data, the median OS should be interpreted with caution. In addition, 51.2% of patients underwent hematopoietic stem cell transplantation during the study period. The safety profile of gilteritinib was as expected, and no new safety signals were identified.</p><p><strong>Conclusion: </strong>Induction and consolidation with gilteritinib plus chemotherapy, and maintenance with gilteritinib monotherapy were well tolerated in ND patients in Asia with <i>FLT3</i> ^mut+ AML and had favorable efficacy compared with historical data.</p><p><strong>Trial registration: </strong>This trial was registered with the ClinicalTrials.gov identifier NCT02310321.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261419953"},"PeriodicalIF":2.8,"publicationDate":"2026-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12982847/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147463904","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":"An analysis of attitudes toward gene therapy in people with severe hemophilia in Germany, a survey-based cross-sectional study.","authors":"Svetlana Babayeva, Aida Peyvandi, Christian Schepperle, Günter Auerswald, Steffen Rauchfuss, Flora Peyvandi, Ilaria Cutica, Wolfgang Miesbach","doi":"10.1177/20406207261428774","DOIUrl":"10.1177/20406207261428774","url":null,"abstract":"<p><strong>Background: </strong>Severe hemophilia traditionally requires lifelong prophylactic treatment, which cannot fully prevent joint damage and significantly impacts patients' quality of life. In recent years, gene therapy has been extensively researched as a potential cure with a single infusion, but it has not yet gained widespread acceptance.</p><p><strong>Objectives: </strong>This study examines the knowledge and attitudes of people with severe hemophilia regarding gene therapy to provide guidance to practitioners in the context of shared decision-making.</p><p><strong>Design: </strong>Cross-sectional observational study based on a standardized questionnaire.</p><p><strong>Methods: </strong>A questionnaire developed by Cutica, Ilaria et al. was evaluated in a self-reported, anonymized survey for a German cohort of 59 people with severe hemophilia. It covered sociodemographic and clinical characteristics, knowledge, attitudes, risk tolerance, and decision-making preferences.</p><p><strong>Results: </strong>Among 59 people with severe hemophilia A and 10 with severe hemophilia B, 59% expressed a predominantly rejective attitude toward gene therapy. Most had only general knowledge of gene therapy. Concerns about long-term side effects (e.g., cancer development) significantly contribute to a negative attitude toward gene therapy (<i>p</i> = 0.005). People with hemophilia and an annual bleeding rate ⩾1 show a significantly higher willingness to consider or accept gene therapy (<i>p</i> = 0.001). Rejection of gene therapy is significantly associated with high expectations regarding its duration of efficacy and the minimum clotting factor level.</p><p><strong>Conclusion: </strong>Further studies are needed to refine educational concepts for people with hemophilia and clear out misconceptions about gene therapy.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261428774"},"PeriodicalIF":2.8,"publicationDate":"2026-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12979895/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147463876","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":"Real-world efficacy and tolerability of ixazomib-based combination therapies in advanced multiple myeloma and other plasma cell neoplasms.","authors":"Xiang Zhou, Julia Mersi, Christine Riedhammer, Maximilian J Steinhardt, Max Bittrich, Stefan Knop, Hermann Einsele, Leo Rasche, Klaus Martin Kortüm, Johannes M Waldschmidt","doi":"10.1177/20406207261421841","DOIUrl":"10.1177/20406207261421841","url":null,"abstract":"<p><strong>Background: </strong>Ixazomib is an oral proteasome inhibitor for relapsed/refractory multiple myeloma (RRMM). Our study aimed to analyze the efficacy and tolerability of ixazomib-based combination therapies.</p><p><strong>Methods: </strong>We performed a single-center retrospective analysis of 126 patients with RRMM and other plasma cell neoplasms.</p><p><strong>Results: </strong>The median age was 65 years, with a median of two prior therapy lines, and 16.7% were triple-class refractory. The overall response rate (ORR) was 52.5%; triple-class refractory patients had a significantly lower ORR than non-refractory controls (10.5% vs 60.2%, <i>p</i> < 0.001). After a median follow-up of 27.0 months, the median progression-free survival (PFS) was 7.9 months (95% CI: 6.9-11.0), and the median overall survival (OS) was 84.1 months (95% CI: 68.1-not reached). In multivariate analysis, a glomerular filtration rate of ⩽70 ml/min/1.73 m² was linked to worse PFS (hazard ratio (HR): 2.11, <i>p</i> = 0.008) and OS (HR: 7.27, <i>p</i> = 0.003). Furthermore, triple-class refractory patients showed a trend toward inferior PFS (HR: 3.76, <i>p</i> = 0.05) and significantly worse OS (HR: 20.46, <i>p</i> = 0.002) compared to non-refractory patients. Grade ⩾3 hematologic adverse events occurred in 15.1% patients, while the most common non-hematologic adverse events were fatigue (5.6%) and peripheral neuropathy (26.2%), with the majority classified as grade 1-2.</p><p><strong>Conclusion: </strong>Altogether, ixazomib regimens are potent in RRMM but are less effective in heavily pretreated patients and those with renal impairment, suggesting earlier use may yield greater benefits.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261421841"},"PeriodicalIF":2.8,"publicationDate":"2026-02-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12953948/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147356537","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 immunosuppressive therapy in connective tissue disease-related immune thrombocytopenia: a systematic review and meta-analysis.","authors":"Yupei Zhang, Xiaxiao Yan, Alyssa A Grimshaw, Yanhong Wang, Mengtao Li, Xiaofeng Zeng, Evelyn Hsieh, Nan Jiang","doi":"10.1177/20406207261422963","DOIUrl":"10.1177/20406207261422963","url":null,"abstract":"<p><strong>Background: </strong>Connective tissue diseases (CTDs) are a group of autoimmune disorders in which immune thrombocytopenia (ITP) represents a common and important manifestation. Current CTD-ITP management mainly relies on glucocorticoids and immunosuppressive agents.</p><p><strong>Objectives: </strong>This systematic review and meta-analysis aims to evaluate the efficacy and safety of immunosuppressive agents in patients with CTD-ITP.</p><p><strong>Design: </strong>Systematic review and meta-analysis.</p><p><strong>Methods: </strong>This study was conducted using eight databases up to July 22, 2024. Observational studies and experimental trials with relevant efficacy and safety data were included. Methodological quality were evaluated using the Newcastle-Ottawa Scale and the ROBINS-I tool. The Mantel-Haenszel formula with a random effect model was employed to estimate the overall effect size. Subgroup analyses were performed based on the study characteristics, clinical features, and treatment regimen.</p><p><strong>Results: </strong>Through integrating 24 studies (1 single-arm clinical trial, 1 case-control, and 22 cohort studies) involving 775 CTD-ITP patients, the pooled estimates of the optimal overall and complete response rate for immunosuppressive therapy were 82% (95% CI: 75-88) and 64% (95% CI: 56-72), with stable results after sensitivity analysis. The combined side-effect incidence was 31% (95% CI: 24-39). The pooled relapse rate was 30% (95% CI: 18-43) among 403 immunosuppressive therapy responders.</p><p><strong>Conclusion: </strong>Immunosuppressive treatments exhibited favorable efficacy and safety in CTD-ITP patients. Future larger-scale multicenter studies are needed.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261422963"},"PeriodicalIF":2.8,"publicationDate":"2026-02-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12954039/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147356406","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":"Effectiveness of caplacizumab in the first-line treatment for acquired thrombotic thrombocytopenic purpura: single center experience.","authors":"Kristina Maria Belakova, Renata Pizurova, Tomas Simurda, Ivana Plamenova, Pavol Holly, Ingrid Skornova, Sohaib Mukhtar Agouba, Miroslava Drotarova, Monika Brunclikova, Alena Stryckova, Richard Jusko, Jan Stasko, Juraj Sokol, Alena Jezikova","doi":"10.1177/20406207261419903","DOIUrl":"10.1177/20406207261419903","url":null,"abstract":"<p><p>Acquired thrombotic thrombocytopenic purpura (aTTP) is a very rare life-threatening disorder, which manifests as a profound thrombocytopenia, microangiopathic hemolytic anemia, renal insufficiency, neurological dysfunction, and other ischemic organ damage. It is caused by antibodies blocking ADAMTS13 metalloprotease. The current standard of the treatment is a triple combination consisting of therapeutic plasma exchange (TPE), immunosuppressive therapy, and caplacizumab, an anti-vWF nanobody. We retrospectively analyzed the medical records and management of three patients with newly diagnosed acute form of aTTP. Three Slovak female patients (mean age at onset 57 years) with severe anemia and thrombocytopenia, one with acute renal insufficiency and two with neurological symptoms were admitted to our institution for suspected aTTP. All patients had ADAMTS13 activity below 2%, laboratory signs of hemolysis, and the presence of ADAMTS13 antibodies. In our laboratory, we also examined the ultra-large high-molecular-weight vWF multimers. The patients were urgently indicated for TPE and high-dose immunosuppressive therapy. Shortly thereafter, caplacizumab was added to the treatment. In the following days platelet count stabilized, ADAMTS13 activity increased and biochemical parameters were gradually adjusted. Two patients experienced exacerbations and one patient experienced relapse of aTTP with mild thrombocytopenia and reduced ADAMTS13 activity, without thrombotic microangiopathy. They were successfully treated with rituximab or cyclophosphamide. Clinical remission was achieved later in all patients. Data collected from our center show that addition of caplacizumab to the standard aTTP treatment helps normalize platelet count faster, reduces the number of TPE sessions and length of hospitalizations, and significantly improves the clinical outcome of patients.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261419903"},"PeriodicalIF":2.8,"publicationDate":"2026-02-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12949255/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147326995","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 retrospective real-world study assessing diagnostic pattern of light-chain amyloidosis in Japan based on data from the medical data vision claims database.","authors":"Moe Yogo, Mami Kasahara-Kiritani, Kazuki Oshima, Tadao Ishida","doi":"10.1177/20406207251379852","DOIUrl":"10.1177/20406207251379852","url":null,"abstract":"<p><strong>Background: </strong>Early diagnosis of primary or light chain (AL) amyloidosis is crucial for initiating appropriate therapeutic interventions. However, diagnosis is getting delayed (several months-to-years) in clinical practice.</p><p><strong>Objective: </strong>To investigate the real-world patterns of clinical procedures until initial diagnosis of AL amyloidosis in Japan.</p><p><strong>Design: </strong>Retrospective longitudinal, observational cohort study.</p><p><strong>Methods: </strong>This study included adults with AL amyloidosis using Medical Data Vision claims database (2003-2022). The primary endpoint was time from initial hospital visit until confirmed AL amyloidosis diagnosis. Symptoms, lab tests, and medical department visits until diagnosis, and mortality were analyzed.</p><p><strong>Results: </strong>Overall, 323 patients with AL amyloidosis were included (median age: 73.0 years). Median time to confirmed diagnosis was 81.5 days; reported longer in patients aged ⩾65 years versus <65 years, and Charlson Comorbidity Index ⩾4 than <4. Specific tests (tissue and bone marrow biopsy) were conducted 28-40 days close to the diagnosis. Patients visited internal medicine (<i>n</i> = 158), hematology medicine (<i>n</i> = 139), dermatology (<i>n</i> = 97), and nephrology (<i>n</i> = 93) departments for confirmed diagnosis. Time to confirmed diagnosis was shorter for patients who visited hematology (median: 7.5 days). Early diagnosed (⩽1 year) patients had longer time-to-in-hospital death than late diagnosis (>1 year).</p><p><strong>Conclusion: </strong>These real-world data from Japanese AL amyloidosis patients are crucial for early and effective treatment, leading to better prognosis.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207251379852"},"PeriodicalIF":2.8,"publicationDate":"2026-02-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12929834/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147310366","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}