Therapeutic Advances in Hematology最新文献

{"title":"Prevalence and factors associated with anemia among people living with HIV taking dolutegravir-based antiretroviral therapy at debre markos compressive specialized hospital, debre markos, northwest Ethiopia, 2024: A cross-sectional study.","authors":"Mohammed Jemal, Hassen Ahmed Yesuf, Mulu Shiferaw Asfaw, Gashaw Abebe, Addis Alem, Zeleke Geto, Altaseb Beyene Kassaw, Adane Adugna, Desalegn Abebaw, Tabarak Malik, Mihiret Bogale Abera, Mohammed Abdu Seid","doi":"10.1177/20406207261475903","DOIUrl":"https://doi.org/10.1177/20406207261475903","url":null,"abstract":"<p><strong>Background: </strong>Anemia is a common hematological complication in people living with HIV (PLHIV), contributing to increased morbidity and mortality. PLHIV are vulnerable to anemia due to chronic inflammation, opportunistic infections, nutritional deficiencies, and the adverse effects of antiretroviral therapy (ART). Despite the favorable safety and efficacy profile of dolutegravir (DTG)-based regimens, emerging evidence has raised concerns about potential hematologic abnormalities, underscoring the need to investigate anemia among PLHIV on DTG-based therapy.</p><p><strong>Objective: </strong>To assess the prevalence of anemia and its associated factors among PLHIV receiving DTG-based ART at Debre Markos Comprehensive Specialized Hospital (DMCSH) in Northwest Ethiopia.</p><p><strong>Design: </strong>An institutional-based cross-sectional study.</p><p><strong>Methods: </strong>This study was conducted among 417 participants enrolled between March 1, 2024, and June 30, 2024, at the ART clinic of DMCSH. Participants were selected using a systematic sampling technique. Socio-demographic data were collected through a structured questionnaire, anthropometric measurements were taken, and clinical data were extracted from patient medical records using a standardized checklist. Hemoglobin levels were measured using the HemoCue® Hb 301 System. The collected data were coded and entered into Epidata version 4.6, then exported to SPSS version 26 for analysis. Logistic regression was conducted to identify factors associated with anemia, with a p-value of < 0.05 considered statistically significant.</p><p><strong>Result: </strong>The prevalence of anemia among PLHIV on DTG-based ART was 32.6% (95% CI: 28.1, 37.1). Among those diagnosed with anemia, 69.1% had mild anemia, 26.5% had moderate anemia, and 4.4% had severe anemia. Factors significantly associated with anemia included increasing age (AOR = 1.04; 95% CI: 1.02-1.07), female sex (AOR = 1.80; 95% CI: 1.12-2.89), duration of HIV infection ≥ 7 years (AOR = 1.88; 95% CI: 1.10-3.23), CD4 T-cell count < 500 cells/mm³ (AOR = 1.89; 95% CI: 1.18-3.01), a family history of anemia (AOR = 3.48; 95% CI: 1.63-7.44), and a history of parasitic infection (AOR = 1.83; 95% CI: 1.03-3.27).</p><p><strong>Conclusion: </strong>Anemia is a moderate public health issue among PLHIV on DTG-based ART. Factors such as female sex, increasing age, duration of HIV infection ≥ 7 years, CD4 T-cell counts < 500 cells/mm³, a family history of anemia, and a history of parasitic infections were identified as significant factors of anemia. Thus, routine monitoring of hemoglobin levels is essential, particularly for individuals with one or more of these risk factors, to enable timely intervention when necessary.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261475903"},"PeriodicalIF":2.8,"publicationDate":"2026-08-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13487123/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148798156","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":"High-altitude polycythemia: State-of-the-art review.","authors":"Xiaoming Chen, Ran Cheng, Shipeng Xu, Wuyang Tong","doi":"10.1177/20406207261479590","DOIUrl":"10.1177/20406207261479590","url":null,"abstract":"<p><p>This review summarizes the research progress on high-altitude polycythemia (HAPC). At the fundamental theoretical level, it provides an in-depth analysis of the epidemiological characteristics influenced by factors such as altitude, genetics, and gender, as well as the pathological mechanisms triggered by chronic hypoxia, including alterations in gene expression, immune imbalance, and disorders of iron metabolism. In the section on clinical manifestations and diagnosis, the multisystem symptoms are elaborated in detail, diagnostic criteria based on hematological indicators combined with high-altitude residence history are clarified, and the key points for differentiating HAPC from primary and other secondary polycythemia are summarized. Regarding treatment strategies, the current application and efficacy of pharmaceutical interventions, non-pharmaceutical methods such as therapeutic erythrocytapheresis (TE), and comprehensive treatment regimens are outlined. Technological advancements focus on genetic research, biomarker development, and imaging technology innovation, providing support for precise diagnosis and treatment. A review of historical development and current status reveals the evolution of research, clarifies present trends in disease prevalence, and identifies challenges in diagnosis and treatment. In the outlook for the future, innovative preventive strategies are proposed, including health education, genetic screening, and the development of novel drug formulations. The research direction of multidisciplinary integration is emphasized, such as elucidating the gene-environment interaction mechanism and developing targeted drugs. Meanwhile, controversial issues including understanding of etiology, evaluation of treatment efficacy, and ethical and social implications, aiming to provide a systematic reference for HAPC research and clinical practice.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261479590"},"PeriodicalIF":2.8,"publicationDate":"2026-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13477526/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148761581","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 combination of alternating reduced-dose blinatumomab and hyper-CVAD as consolidation therapy in patients with newly-diagnosed adult B-cell acute lymphoblastic leukemia.","authors":"Zixuan Li, Fang Liu, Chun Zhang, Jing He, WeiMing Li, Dairong Xie, Yuting Jiang, Ruofeng Jin, Tianran Gao, Mei Hong, Qiuling Wu","doi":"10.1177/20406207261479931","DOIUrl":"10.1177/20406207261479931","url":null,"abstract":"<p><strong>Background: </strong>Blinatumomab has gained attention for its effectiveness in improving overall survival in relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL) and eradicating minimal residual disease (MRD).</p><p><strong>Objectives: </strong>We conducted a retrospective study to assess whether combining reduced-dose blinatumomab with chemotherapy for consolidation could improve outcomes in newly diagnosed B-ALL.</p><p><strong>Design: </strong>Patients with Philadelphia chromosome (Ph)-positive or Ph-negative B-ALL who achieved complete remission (CR) after induction received consolidation therapy consisting of blinatumomab (cycle 1, 3, 5, and 7) and hyper-CVAD (course B for cycles 2 and 6; course A for cycle 4 and 8). After completion of the cycle 2, the decisions to continue treatment or receive hematopoietic stem-cell transplantation were made based on multiple factors.</p><p><strong>Methods: </strong>The final endpoint was molecular remission, overall survival (OS), relapse-free survival (RFS). Molecular remission referred to MRD-related methods, including MFC (multiparameter flow cytometry)-based MRD, next generation sequencing (NGS)-based MRD, and complete molecular remission (CMR). Meanwhile, we assessed the safety profile of this combination regimen, including adverse events such as cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS).</p><p><strong>Results: </strong>32 newly diagnosed B-ALL patients (14 Ph+ B-ALL and 18 Ph- B-ALL) achieving CR were analyzed. At the time of study inclusion, 12 Ph+ B-ALL and 12 Ph- B-ALL patients were MFC-MRD negative, and 5 Ph+ B-ALL patients achieved CMR. After the first blinatumomab consolidation and hyper-CVAD B cycle, seven additional patients (one Ph+ B-ALL and six Ph- B-ALL) achieved MFC-MRD negativity, and six additional Ph+ B-ALL patients achieved CMR. With a median follow-up of 16.5 months, the overall rate of MFC-MRD was 96.88% (Ph+ B-ALL 92.86% and Ph- B-ALL 100%), and CMR was 78.57%. The estimated RFS and OS rates at 30 months for whole patients were 83.5% (95% CI, 79.1%-100%) and 96.8% (95% CI, 90.8%-100%), respectively. The most common adverse events were observed during chemotherapy cycles due to myelosuppression. Eight patients developed a grade 1-2 blinatumomab-related CRS with a prevalence of 17.78%, which was completely reversible.</p><p><strong>Conclusion: </strong>Reduced-dose blinatumomab combined with hyper-CVAD chemotherapy as consolidation therapy seems to be feasible for adult B-ALL with acceptable side effects.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261479931"},"PeriodicalIF":2.8,"publicationDate":"2026-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13477527/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148763978","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":"Hypo-fractionated radiotherapy combined with PD-1 inhibitor for relapsed/refractory B-cell lymphoma after failure to CD19 CAR-T therapy.","authors":"Min Li, Fang Bao, Ran Peng, Hongmei Jing, Hao Wang","doi":"10.1177/20406207261478443","DOIUrl":"10.1177/20406207261478443","url":null,"abstract":"<p><p>B-cell lymphoma relapsed after chimeric antigen receptor (CAR) T-cell therapy lacks effective standard treatment options and generally has a poor prognosis, largely due to mechanisms such as immune escape and tumor heterogeneity. Radiotherapy combined with immune checkpoint inhibitors (ICIs) may exert synergistic effects, including local tumor control through radiation-induced DNA damage and systemic immune activation via modulation of the tumor immune microenvironment. This case report describes a patient with relapsed/refractory (R/R) B-cell lymphoma who experienced multiple relapses and treatment failures, including CD19 CAR-T therapy. Almost 7 years after CAR-T treatment, the patient developed disease relapse and was diagnosed with follicular lymphoma (FL). The patient received a personalized treatment strategy combining hypo-fractionated radiotherapy (30 Gy/5 fractions) and the Programmed Death-1 (PD-1) inhibitor camrelizumab, achieving complete remission (CR) and remaining disease-free for over 43 months. This case highlights the potential efficacy of this combination strategy in R/R lymphoma, particularly in patients with limited treatment options.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261478443"},"PeriodicalIF":2.8,"publicationDate":"2026-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13477522/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148761791","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 US Experience with Voxelotor for Sickle Cell Disease: A Plain Language Summary of the RETRO and PROSPECT studies.","authors":"Biree Andemariam","doi":"10.1177/20406207261474411","DOIUrl":"https://doi.org/10.1177/20406207261474411","url":null,"abstract":"<p><p>What is this summary about? This summary describes two studies in people with sickle cell disease, or SCD. Both studies looked at a medicine called voxelotor, also known as Oxbryta^®, which was previously available for treating people with SCD. This is a summary of an article published in <i>Blood Advances</i>. What were the results of the studies? In the RETRO and PROSPECT studies, researchers looked at people with SCD taking voxelotor in everyday, real-world medical practice. In other words, they took voxelotor as part of their usual care when it was available for prescription in the United States. In RETRO, 216 participants were enrolled and the length of voxelotor treatment was about 1 year, on average. In PROSPECT, 265 participants were enrolled, and the length of voxelotor treatment was almost 3 years, on average. After taking voxelotor, participants had increases in hemoglobin, the protein in red blood cells that carries oxygen. Their red blood cells also broke apart less quickly when taking voxelotor. The most common symptom related to SCD was acute pain crisis, but participants did not experience it more often after starting voxelotor. What do the results mean? All medicines are tested in clinical trials before they are approved as treatments. However, it is always important to study a medicine in real-world medical practice as well. In RETRO and PROSPECT, the effects of voxelotor were like the effects seen in the clinical trials, and researchers did not see any unexpected symptoms related to SCD or unexpected side effects related to the study drug.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261474411"},"PeriodicalIF":2.8,"publicationDate":"2026-07-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13420057/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148631509","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":"Plain language summary: How safe and effective is long-term treatment with intravenous plasminogen concentrate for people with type 1 plasminogen deficiency?","authors":"Amy D Shapiro, Heather McDaniel, Robert W Decker, Charles Nakar, Jeremy Lorber, Neelam Thukral, Joseph M Parker, Karen Thibaudeau","doi":"10.1177/20406207261463031","DOIUrl":"https://doi.org/10.1177/20406207261463031","url":null,"abstract":"<p><p>What is this study about? Type 1 plasminogen deficiency (PLGD-1 for short) is a long-term, ultra-rare condition where the body does not make enough of a protein called plasminogen. This can cause thick layers made of a fibrous, insoluble protein called fibrin (known as lesions) to develop in different parts of the body. Intravenous plasminogen concentrate (IV PLG concentrate for short, also known as RYPLAZIM®) is the first medicine approved to treat this condition. It works by replacing plasminogen in the body. In this study, adults and children with PLGD-1 received long-term treatment with IV PLG concentrate. They had already received IV PLG concentrate before, as part of the clinical trial that led to the approval of the treatment, or through the expanded access programs. The researchers wanted to know how safe and effective long-term treatment with IV PLG concentrate was. They also wanted to explore whether giving IV PLG concentrate more or less often would affect how well the treatment worked. What were the results? Study participants had no new lesions associated with PLGD-1 or return of previous lesions when they received the treatment as prescribed. Some participants had previous lesions come back when they received the treatment less often than prescribed. These lesions rapidly improved or went away again when they started more frequent treatment, as originally prescribed. Most of the adverse events that participants experienced were classified as mild, and none were related to the study treatment. What do the results mean? Overall, long-term treatment with IV PLG concentrate is effective for people living with PLGD-1 and is well tolerated. Treatment with IV PLG concentrate can be personalized to meet people's individual needs. What is the purpose of this plain language summary? The purpose of this plain language summary is to help you understand the findings from recent research. Where can I find the original article on which this summary is based? You can read the original article titled 'Safety and efficacy of long-term treatment of type 1 plasminogen deficient patients with intravenous plasminogen replacement therapy' published in the journal Haemophilia for free at: https://doi.org/10.1111/hae.70019 Who sponsored the study and this summary? The study was <b>sponsored</b> by Prometic Biotherapeutics Inc., currently Kedrion Biopharma Inc. This summary was sponsored by Kedrion Biopharma Inc. Who is this summary for? This summary may be of interest to people living with PLGD-1, their families, and caregivers. Patient advocates, patient organizations, healthcare professionals, and funders of healthcare services may also find this summary useful.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261463031"},"PeriodicalIF":2.8,"publicationDate":"2026-07-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13401630/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148593242","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":"Treatment preferences of people with hemophilia and caregivers: a plain language summary.","authors":"Hui Lu, Travis Gould, Carmine Colavecchia, Joshua Coulter, Lisa J Wilcox, Joseph C Cappelleri, Stephanie A Christopher, Brett Hauber, Álvaro Alberto Gutiérrez-Vargas, Nicola Dunn, Pratima Chowdary, Jennifer A Whitty","doi":"10.1177/20406207261437965","DOIUrl":"10.1177/20406207261437965","url":null,"abstract":"<p><p>What is this summary about? This is a summary of the results from a study that looked at the treatment preferences of people with hemophilia and caregivers of children with hemophilia in the US and UK. The study was published in a research journal called Haemophilia. People with hemophilia need regular treatments to help their blood clot properly and to prevent bleeding. Treatments to prevent bleeding are often injected into a vein. For some people, the injections can be painful or hard to use. If treatments are hard to use, people may be less likely to stick to their treatment plan. This can make it harder for them to manage their hemophilia. New treatments that work in a different way can be given as an injection under the skin instead of into a vein. These treatments may be easier for people to use. Newer treatments have different benefits and risks compared with earlier types of treatment, so researchers wanted to understand what features of treatments are most important to people with hemophilia and caregivers What were the results of the study? Adults with hemophilia and caregivers of children with hemophilia in the US and UK took an online survey. They were asked to choose between made-up (hypothetical) treatments with different benefits, risks, and ways of administration (how the treatment is delivered into the body). The study showed that people with hemophilia and caregivers had mostly the same preferences for treatments to prevent bleeds. People cared most about having a treatment they can take less often. People were willing to accept some additional risks or lower treatment benefits if they could have injections under the skin instead of into a vein. What do the results mean? People with hemophilia and caregivers found some treatment features more important than others. It is important for healthcare teams to know which treatment features are most important to people. If treatments match people's preferences, they are more likely to be satisfied. For example, if people prefer injections under the skin, they may be more likely to stick to their treatment plan if they use a treatment delivered under the skin instead of into a vein. People who stick to their treatment plan are likely to have better health outcomes.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261437965"},"PeriodicalIF":2.8,"publicationDate":"2026-06-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13305631/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148346379","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 plain language summary of the BASIS study looking at people living with severe hemophilia A or B with inhibitors and being treated with marstacimab.","authors":"Davide Matino, Suchitra S Acharya, Carrie Turich Taylor, Pengling Sun, Delphine Agathon, Sangeeta Raje, Travis Gould, Andrew Palladino, Johnny Mahlangu","doi":"10.1177/20406207261442896","DOIUrl":"https://doi.org/10.1177/20406207261442896","url":null,"abstract":"<p><p>What is this summary about? This summary describes a clinical study looking at how well marstacimab works in people living with hemophilia with inhibitors. The study was published in the medical journal <i>Blood</i>. Hemophilia is caused by not having enough blood clotting factors, which are proteins found in the blood that help form a clot and stop bleeding. People living with hemophilia either don't make these proteins, have low amounts of these proteins, or these proteins do not work well. Hemophilia is usually inherited from biological parents, meaning it is passed on to their children through genes. In some cases, hemophilia happens even if no one in the family has it. There are some treatments to replace the missing clotting factor. This type of treatment is called factor replacement therapy and contains the missing clotting factor proteins. Some people make antibodies against the replacement clotting factors, so the replacement clotting factors do not work as well as they need to for the blood to clot. This is called hemophilia with inhibitors. People who have hemophilia with inhibitors need to take medicines called bypassing agents instead of replacement clotting factors. These medicines aim to activate the body's blood clotting system, but do not always work well. They also need to be injected into a vein, known as intravenous infusion, or IV infusion for short. IV infusions are taken several times a week. Different options to IV factor replacement therapy for people living with hemophilia A or hemophilia B are called non-factor replacement therapy. A protein in the blood called Tissue Factor Pathway Inhibitor, or TFPI, can also prevent blood clotting. Marstacimab is a type of non-factor replacement therapy that attaches to TFPI and stops its activity. For people living with hemophilia, marstacimab can help blood to form a clot when needed. Marstacimab is injected under the skin. This can be an easier way to receive medicine than an injection into a vein. Marstacimab offers a new way to treat people living with hemophilia. What were the results of the study? The study showed that people living with hemophilia with inhibitors who were treated with marstacimab had fewer bleeding events over 1 year compared to previous therapy with bypassing agents. Most side effects with marstacimab treatment were minor or could be easily managed. One person stopped taking marstacimab treatment because they developed a skin rash that got better over time. What do the results mean? The study showed that marstacimab could help prevent bleeding in people living with hemophilia A or B with inhibitors.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261442896"},"PeriodicalIF":2.8,"publicationDate":"2026-06-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13269730/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148273055","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":"Plain language summary: What have research studies shown about plasma-derived human factor X concentrate treatment for people living with hereditary factor X deficiency?","authors":"Miguel A Escobar, Kaan Kavakli","doi":"10.1177/20406207261448285","DOIUrl":"https://doi.org/10.1177/20406207261448285","url":null,"abstract":"<p><p>What are these studies about? Researchers looked at the latest research studies about plasma-derived human factor X concentrate (pdFX for short) for people living with hereditary factor X deficiency (HFXD for short). These people's health and daily lives were impacted in different ways by HFXD, and their condition was classified as mild, moderate, or severe. The researchers assessed information from 5 clinical trials and 1 case series that described the bleeding symptoms and treatment that a group of people living with HFXD had. What were the results? Researchers found that pdFX has been successfully used in adults and children with HFXD to treat bleeds when they happen, to prevent bleeds, and to manage bleeding around having surgery. Researchers found that people living with HFXD had minimal side effects when they received pdFX. What do the results mean? Research studies to date have shown that pdFX is safe and effective as a treatment for people living with mild, moderate, or severe HFXD. This information will help healthcare experts to develop recommendations based on research results about pdFX treatment for people living with HFXD. What is the purpose of this plain language summary? The purpose of this plain language summary is to help you to understand the findings from recent research.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261448285"},"PeriodicalIF":3.1,"publicationDate":"2026-06-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13241605/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148200009","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":"Effect of marstacimab in young people living with severe hemophilia A or moderate to severe hemophilia B without inhibitors: A plain language summary from the BASIS study.","authors":"Priya Patel","doi":"10.1177/20406207261439648","DOIUrl":"https://doi.org/10.1177/20406207261439648","url":null,"abstract":"<p><p>What is this summary about? This summary describes a study that looked at how well the study treatment (marstacimab) works in people living with hemophilia. The summary describes how well marstacimab reduced bleeding episodes in young people (adolescents) 12 to 17 years old. Hemophilia is a condition that causes people to bleed more easily because their blood is unable to clot properly. Clotting is when blood turns from liquid into gel to stop bleeding. Hemophilia is caused by not having enough blood clotting factors, which are proteins found in the blood that help in forming a clot and stop bleeding. People living with hemophilia either don't make these proteins, have low amounts of these proteins, or their proteins do not work well. There are different types of hemophilia, depending on which clotting factor is affected. For example, people living with hemophilia A lack clotting factor 8, and people living with hemophilia B lack clotting factor 9. Some treatments for hemophilia work by replacing clotting factors. These treatments are called factor replacement therapy, and are given directly into a vein (called intravenous, or IV for short). They are given frequently, sometimes even more than once a week. In some people living with hemophilia, the body develops antibodies called inhibitors that block replacement clotting factors from working properly. Not all people living with hemophilia produce these antibodies - those who don't are classified as having hemophilia without inhibitors. A different way to treat people living with hemophilia is by using treatment called non-factor replacement therapy. One type of non-factor treatment called marstacimab is given as a regular preventive treatment (called prophylaxis). It works without replacing the missing clotting factors but brings blood clotting and bleeding back to a normal balance. Marstacimab is given once a week as a flat-dose (not based on body weight) injection under the skin (called subcutaneous) using an auto-injector device. This injection is sometimes called a shot and can be easier than injecting directly into a vein. In some cases, marstacimab can be taken less often than clotting factors. Marstacimab offers a new way to treat people living with hemophilia A or B without inhibitors. What were the results of the study? In this study, young people treated with marstacimab for 1 year had on average fewer bleeding episodes than on previous clotting factor replacement treatment. Marstacimab was well tolerated in young people (similar to adults), which means that most side effects that people had were mild to moderate and resolved by the end of the study. What do the results mean? This study showed that a once weekly injection of marstacimab could help reduce bleeding episodes in young people living with hemophilia A or B, without inhibitors.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261439648"},"PeriodicalIF":3.1,"publicationDate":"2026-05-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13219864/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148138997","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}