Therapeutic Advances in Hematology最新文献

{"title":"Clinical considerations for ruxolitinib in the treatment of steroid-refractory acute graft-versus-host disease (GVHD) and steroid-refractory or -dependent chronic GVHD: a plain language summary.","authors":"Zahra Mahmoudjafari, Valkal Bhatt, Zhenyi Xue, Franco Locatelli, Gérard Socié, Robert Zeiser","doi":"10.1177/20406207261442908","DOIUrl":"https://doi.org/10.1177/20406207261442908","url":null,"abstract":"<p><p>What is this summary about? Ruxolitinib (JAKAFI^®) is a Janus kinase (JAK) 1 and JAK2 inhibitor that is approved for acute and chronic graft-versus-host disease (GVHD). The phase III REACH2 and REACH3 trials tested efficacy and safety of ruxolitinib for patients with acute and chronic GVHD, respectively, who were no longer responding to steroids. This summary describes additional analyses from REACH2 and REACH3 that evaluated the effect of treatment initiation time, use of antifungal azole treatments, and development of cytopenias (low number of blood cells) on ruxolitinib effectiveness. What happened in the analyses? Results from REACH2 and REACH3 were analyzed to inform whether time to start treatment, use of azoles, and developing cytopenias affected how well ruxolitinib worked. Treatment effectiveness was measured as any response to treatment, including complete resolution of all GVHD symptoms. What were the results? When analyzing time to start treatment, more patients taking ruxolitinib had a better response than those taking control treatments, regardless of when treatment started. In acute GVHD, treatment responses generally appeared higher when ruxolitinib was started early (within 3 days of steroids not working). The effectiveness of ruxolitinib persisted even among patients taking azoles or for patients who developed cytopenias, with some patients having their dosage adjusted. What do the results mean? The treatment benefit of ruxolitinib compared with control treatments is consistently observed across patients with acute and chronic GVHD who no longer respond to steroids, regardless of time to start of treatment, use of azoles, or developing cytopenias. Dose modifications of ruxolitinib may need to occur to ensure treatment is well tolerated. <b>ClinicalTrials.gov registration ID:</b> NCT02913261 (REACH2) and NCT03112603 (REACH3).</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261442908"},"PeriodicalIF":3.1,"publicationDate":"2026-05-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13191118/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148017441","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":"Final data from the German AHEAD study: effectiveness and safety of octocog alfa and rurioctocog alfa pegol for hemophilia A in a real-world setting.","authors":"Robert Klamroth, Karin Kurnik, Swee Wenning, Carmen Escuriola-Ettingshausen, Stephan Regensburger, Leilei Tang, Joan Gu, Randy Guerra, Johannes Oldenburg","doi":"10.1177/20406207261419337","DOIUrl":"10.1177/20406207261419337","url":null,"abstract":"<p><strong>Background: </strong>Octocog alfa and rurioctocog alfa pegol have demonstrated efficacy in the prevention and management of bleeding associated with hemophilia A (HA) in clinical trials. The German AHEAD study (DRKS00000556) was designed to provide real-world data over a long period to complement these studies.</p><p><strong>Objectives: </strong>To assess real-world long-term outcomes in patients with moderate/severe HA receiving octocog alfa or rurioctocog alfa pegol in routine clinical practice.</p><p><strong>Design: </strong>Noninterventional, prospective, study conducted between 2010 and 2022.</p><p><strong>Methods: </strong>The primary endpoint was the incidence of hemophilia-affected joint arthropathy. Secondary endpoints included annualized bleeding rate and joint bleeding rate (ABR/AJBR), and octocog alfa and rurioctocog alfa pegol consumption. Final data are presented.</p><p><strong>Results: </strong>The analysis included 377 patients aged 1-80 years who received octocog alfa (severe HA, <i>n</i> = 309; moderate HA, <i>n</i> = 68) and 99 patients aged ⩾12 years who received rurioctocog alfa pegol (severe, <i>n</i> = 82; moderate, <i>n</i> = 17). Over 8 years of follow-up, average Gilbert scores were maintained with prophylaxis. In patients with severe HA, median ABRs/AJBRs were generally lower with prophylaxis versus on-demand treatment, whereas in moderate HA, bleeding rates were similar. Treatment-related adverse events (AEs) and serious treatment-related AEs were reported in 5.3% and 4.2% (35 events including hemorrhage, arthropathy, and factor FVIII (FVIII) inhibitor development) of patients receiving octocog alfa, respectively, and 5.1% and 4.0% of patients receiving rurioctocog alfa pegol, respectively. One previously treated patient receiving octocog alfa developed de novo FVIII inhibitors.</p><p><strong>Conclusion: </strong>These real-world data corroborate previous clinical trial and real-world data demonstrating the long-term effectiveness and safety of octocog alfa and rurioctocog alfa pegol in patients with HA. In patients with severe HA, octocog alfa prophylaxis was associated with reduced bleeding rates compared with on-demand therapy.</p><p><strong>Trial registration: </strong>Deutsches Register Klinischer Studien (German Clinical Trials Register): DRKS00000556.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261419337"},"PeriodicalIF":2.8,"publicationDate":"2026-05-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13153523/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147869620","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":"Management of bleeds in people living with hemophilia A or B without inhibitors and receiving marstacimab: A plain language summary of publication from the BASIS study.","authors":"Hae Kyung Kim","doi":"10.1177/20406207261435225","DOIUrl":"https://doi.org/10.1177/20406207261435225","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 people treated bleeding events while taking marstacimab. 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 will 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 these proteins do not work well. 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. Some people living with hemophilia develop antibodies called inhibitors against the factor replacement therapy, which causes the treatment to be ineffective. If people make these antibodies, it is called hemophilia with inhibitors. A different way to treat people living with hemophilia is using treatment called non-factor replacement therapy. One type of non-factor treatment targets a protein in the blood called tissue factor pathway inhibitor, or TFPI for short. A treatment called marstacimab attaches to TFPI and stops its activity. Marstacimab is given as a regular preventative (prophylaxis) treatment. For people living with hemophilia, marstacimab can help blood to form a clot when needed. Marstacimab is taken less often than clotting factors and is given once a week as an injection under the skin (called subcutaneous), which can be easier than injecting directly into a vein. This injection is sometimes called a shot. Marstacimab offers a new way to treat people living with hemophilia without inhibitors. What were the results of the study? In this study, people who received regular preventative treatment with marstacimab and experienced bleeds were able to manage their bleeds with factor replacement therapy. People could usually treat bleeds with a single infusion of factor replacement therapy. None of the people in the study developed harmful blood clots when they took regular preventative treatment with marstacimab and infusions of factor replacement therapy as needed to treat their bleeds. What do the results mean? The study showed that people taking marstacimab can still use factor replacement therapy to treat a bleed.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261435225"},"PeriodicalIF":3.1,"publicationDate":"2026-04-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13150083/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147869430","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":"Long-term effect of marstacimab on joints that bleed frequently in people living with severe hemophilia without inhibitors: A plain language summary of publication.","authors":"Hae Kyung Kim","doi":"10.1177/20406207261435233","DOIUrl":"https://doi.org/10.1177/20406207261435233","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. Hemophilia is a condition that causes people to bleed more easily because their blood is unable to clot properly. Clotting is when liquid blood turns gel-like to stop uncontrolled bleeding. Bleeding can happen inside the body, in joints and muscles. A joint that has a lot of bleeds in a short period of time is called a target joint. The summary describes how well marstacimab stopped bleeding in the target joints of people living with hemophilia. 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 these proteins do not work well. Some treatments for hemophilia work by replacing these clotting factors. These treatments are known as 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. Some people living with hemophilia develop antibodies called inhibitors against the replacement clotting factors, which causes the treatment to be ineffective. If people make these antibodies, it is called hemophilia with inhibitors. A different way to treat people living with hemophilia is using treatments called non-factor replacement therapy. One type of non-factor treatment targets a protein in the blood called tissue factor pathway inhibitor, or TFPI for short. A treatment called marstacimab attaches to TFPI and stops its activity. Marstacimab is given as a regular preventative (prophylaxis) treatment. For people living with hemophilia, this helps blood to form a clot when needed. Marstacimab is taken less often than clotting factors and is given as a once a week injection under the skin (called subcutaneous), which may be easier than injecting directly into a vein. This injection is sometimes called a shot. Marstacimab offers a new way to treat people living with hemophilia without inhibitors. What were the results of the study? In this study, marstacimab reduced bleeding in target joints in people living with hemophilia without inhibitors for up to 2 years of treatment compared to previous clotting factor replacement treatment. Most target joints got better with marstacimab treatment. What do the results mean? This study showed that marstacimab once a week could help reduce bleeding in target joints for people living with hemophilia A or B without inhibitors. Marstacimab may also help resolve target joints in people living with hemophilia A or B without inhibitors who already have target joints.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261435233"},"PeriodicalIF":3.1,"publicationDate":"2026-04-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13150088/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147869352","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":"Marstacimab for People with Severe Hemophilia A or Moderate to Severe Hemophilia B Without Inhibitors: A Plain Language Summary of Publication of the BASIS Study.","authors":"Davide Matino, Andrew Palladino, Carrie Turich Taylor, Eunhee Hwang, Sangeeta Raje, Satyaprakash Nayak, Regina McDonald, Suchitra S Acharya, Johnny Mahlangu, Victor Jimenez-Yuste, Nirmalkumar Choraria, Renchi Yang, Chi Kong Li, Murtadha Al-Khabori, Yasser Wali, Javier Morales Adrian, Young-Shil Park, O Bülent Zülfikar, John Teeter","doi":"10.1177/20406207261430259","DOIUrl":"https://doi.org/10.1177/20406207261430259","url":null,"abstract":"<p><p>What is this summary about? This is a summary of the results from a clinical study of treatment for people with severe hemophilia A or moderately severe to severe hemophilia B without inhibitors. Because severe hemophilia A and B predominantly affect men and boys, this study only included men and boys aged 12 to 74 years. The study was published in <i>Blood</i>. People with hemophilia either have low amounts of clotting factors or are missing certain clotting factors in their blood. There are medicines that people with hemophilia can take to replace the missing clotting factor. These medicines must be injected into a vein and are usually given more than once a week. Marstacimab is an antibody that works by attaching to a protein in the blood called tissue factor pathway inhibitor (or TFPI). TFPI works separately from clotting factors. Marstacimab helps the balance between blood flowing freely and clotting. Marstacimab is given by a simple injection under the skin (not in a vein or muscle) and can be taken less often than clotting factors. Marstacimab may offer a new way to help people with hemophilia. What were the results of the study? The study showed that men with hemophilia had fewer treated bleed events during 1 year with marstacimab treatment compared to previous factor replacement therapy. The researchers reported that marstacimab was safe and most side effects were mild to moderate. What do the results mean? These studies showed that marstacimab could help prevent bleeding in men with hemophilia A or B, without inhibitors.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261430259"},"PeriodicalIF":3.1,"publicationDate":"2026-04-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13145024/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147843217","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":"Efgartigimod as a treatment for adults with primary immune thrombocytopenia: a plain language summary of the ADVANCE IV study.","authors":"Catherine Broome, Yoshitaka Miyakawa, Monica Carpenedo, Hanny Al-Samkari, Jaume Ayguasanosa, John Phillips, Francesco Rodeghiero","doi":"10.1177/20406207261445668","DOIUrl":"10.1177/20406207261445668","url":null,"abstract":"<p><p>What is this summary about? This is a plain language summary of an article published in the scientific journal The Lancet in 2023. The ADVANCE IV study involved people living with immune thrombocytopenia, called ITP. In primary ITP your immune system attacks platelets in the blood. People with ITP have a lower number of platelets than normal. Some people may bruise or bleed more easily, and bleeding can become serious if platelet counts are very low. ITP is called persistent when low platelet counts continue for 3 to 12 months and chronic if low platelet counts continue for more than a year from diagnosis. What happened in this study? Participants received efgartigimod or a placebo (inactive medicine) through a needle into the vein (intravenous) weekly. The main aim was to see how many people with chronic ITP had increased platelet counts over several weeks during the treatment period. Researchers looked at how well intravenous efgartigimod worked in improving symptoms such as bleeding in adults with chronic or persistent primary ITP. They also looked at the side effects people had during the study. What were the results of this study? Overall, 131 people with ITP participated in the study between December 2019 and February 2022. On average, people had received their ITP diagnosis over 10 years before the start of the study. Around 7 in 10 people (67%) had previously received at least 3 other treatments for ITP. More people (22%) who received intravenous efgartigimod had increased platelet counts than those who received a placebo (5%). Over half of the people who received efgartigimod had improvements when taking platelet counts and bleeding into account. The most common side effects were tiny purple, red, or brown spots on the skin (petechiae), red blood cells in the urine, and headaches. These were seen at similar rates in people receiving efgartigimod and those receiving a placebo. What do the results mean? These results show that intravenous efgartigimod helps increase platelet counts in people with chronic or persistent primary ITP who had tried several previous treatments for ITP.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261445668"},"PeriodicalIF":2.8,"publicationDate":"2026-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13100365/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147781912","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":"Individualized decitabine dosing for post-HSCT maintenance in MDS and secondary AML from MDS: long-term outcomes from the PODAC trial and matched controls.","authors":"Silvia Park, Suein Choi, Seunghoon Han, Yoo-Jin Kim","doi":"10.1177/20406207261432568","DOIUrl":"10.1177/20406207261432568","url":null,"abstract":"<p><strong>Background: </strong>Relapse after allogeneic hematopoietic stem cell transplantation (HSCT) remains a major clinical challenge in higher-risk myelodysplastic neoplasms (MDS). Although hypomethylating agents have been evaluated as posttransplant maintenance therapy, their efficacy remains uncertain.</p><p><strong>Objective: </strong>We aimed to evaluate the long-term clinical outcomes of individualized decitabine maintenance therapy following HSCT in patients with MDS or secondary acute myeloid leukemia from MDS.</p><p><strong>Design: </strong>This was a retrospective comparative study based on a long-term follow-up of the phase I PODAC trial, which individualized posttransplant decitabine maintenance dosing.</p><p><strong>Methods: </strong>Nineteen PODAC participants were included in the treatment group and compared with 52 matched control patients selected through target trial emulation using identical eligibility criteria.</p><p><strong>Results: </strong>For the 19 PODAC participants, a median of 6 cycles was administered, starting at 5 mg/m²/day and escalating to a median maintenance dose of 7 mg/m²/day. After a median follow-up of 11.4 years in the PODAC group and 9.7 years in the control group (<i>p</i> = 0.90), no significant differences were observed between the two groups in overall survival, relapse-free survival, graft-versus-host disease (GVHD)-free relapse-free survival, or the incidence of relapse, nonrelapse mortality, and grades II-IV acute GVHD. However, the incidence of moderate-to-severe chronic GVHD was significantly lower in the PODAC group and decitabine maintenance emerged as an independent predictor for longer chronic GVHD-free relapse-free survival (cCRFS).</p><p><strong>Conclusion: </strong>Decitabine maintenance using an adaptive dosing approach enabled prolonged treatment and was associated with a lower incidence of chronic GVHD and improved cGRFS. However, it did not improve survival or relapse outcomes compared to the control group.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261432568"},"PeriodicalIF":2.8,"publicationDate":"2026-04-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13077147/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147691931","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":"Precision medicine in thrombotic thrombocytopenic purpura: a narrative review.","authors":"Rafal Al-Shibly, Rola Ghasoub, Muna AlRasheed, Lulwa AlTourah, Taghreed Al-Eisa, Mohamed A Yassin","doi":"10.1177/20406207261430423","DOIUrl":"10.1177/20406207261430423","url":null,"abstract":"<p><p>Thrombotic thrombocytopenic purpura (TTP) is a life-threatening thrombotic microangiopathy driven by severe ADAMTS13 deficiency with consequent ultra-large von Willebrand factor-mediated microthrombosis. Over the last two decades, understanding of the ADAMTS13-vWF axis has transformed management and made TTP a model for precision medicine. This narrative review (adult focus) synthesizes contemporary evidence on diagnosis, treatment, genetics, and emerging therapies in immune-mediated TTP. We summarize a pragmatic, biomarker-guided algorithm integrating early therapeutic plasma exchange and corticosteroids, upfront rituximab to suppress anti-ADAMTS13 autoimmunity, and caplacizumab to block vWF-platelet interactions; define escalation strategies for refractory disease (e.g., proteasome inhibition); and formalize treat-to-target de-escalation anchored to ADAMTS13 recovery. We emphasize the role of rapid ADAMTS13 assays for front-door triage, routine ADAMTS13 monitoring in remission, and preemptive rituximab at biochemical relapse to avert clinical recurrence. Precision diagnostics are extended by genetic markers (e.g., HLA associations) and emerging biomarkers that may refine relapse risk. Finally, we discuss near-term opportunities-point-of-care ADAMTS13 assays, longer-acting vWF-pathway inhibitors, and data-driven risk tools-that can further individualize timing and intensity of therapy. Collectively, a biomarker-first strategy offers a clear path to fewer exacerbations, fewer relapses, and more consistent outcomes.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261430423"},"PeriodicalIF":2.8,"publicationDate":"2026-04-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13069169/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147676779","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":"Prophylaxis for von Willebrand disease: Is it time for parity with established practice in hemophilia A?","authors":"Robert F Sidonio, Fernando F Corrales-Medina, Jill M Johnsen, Michelle Sholzberg, Caroline Malcolmson, Nathan T Connell","doi":"10.1177/20406207261431617","DOIUrl":"10.1177/20406207261431617","url":null,"abstract":"<p><p>A deficiency and/or dysfunction of von Willebrand factor (VWF) or factor VIII (FVIII) results in the bleeding disorders of von Willebrand disease (VWD) and hemophilia A (HA), respectively. Whereas HA impacts coagulation, VWD primarily impairs hemostasis through defective platelet adhesion and aggregation. In addition, because VWF protects FVIII from proteolytic degradation, a deficiency in VWF can also reduce FVIII levels and affect coagulation. While regular prophylaxis to restore FVIII activity is the standard of care for severe HA, its use to correct the dual VWF/FVIII defect in severe VWD is less well established. Current treatment guidelines suggest the use of long-term prophylaxis rather than no prophylaxis in persons with VWD with a history of severe and frequent bleeds. In this narrative review, we discuss the barriers to the broader adoption of prophylaxis in persons with severe VWD and results of recent clinical studies that provide further evidence to support its use. A growing body of evidence suggests that prophylaxis should be established as the standard care for individuals with severe VWD and recurrent bleeding.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261431617"},"PeriodicalIF":2.8,"publicationDate":"2026-04-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13051177/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147634221","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":"Neutrophil-to-lymphocyte ratio half reduction as a surrogate of molecular response in polycythemia vera treated with ropeginterferon alfa-2b.","authors":"Seug Yun Yoon, Sung-Soo Yoon, Deok-Hwan Yang, Gyeong-Won Lee, Sang Kyun Sohn, Ho-Jin Shin, Sung Hwa Bae, Chul Won Choi, Eun-Ji Choi, June-Won Cheong, Soo-Mee Bang, Joon Seong Park, Yong Park, Young Hoon Park, Sung-Eun Lee","doi":"10.1177/20406207261431904","DOIUrl":"10.1177/20406207261431904","url":null,"abstract":"<p><strong>Background: </strong>Polycythemia vera (PV) is a myeloproliferative neoplasm driven by <i>JAK2</i> V617F mutations. Ropeginterferon alfa-2b effectively reduces both hematologic parameters and <i>JAK2</i> allele burden, but molecular monitoring is not always readily available.</p><p><strong>Objectives: </strong>To evaluate whether a ⩾50% reduction in the neutrophil-to-lymphocyte ratio (NLR) can serve as a surrogate marker of hematologic and molecular response during ropeginterferon therapy.</p><p><strong>Design: </strong>Secondary analysis of a multicenter, phase II, open-label trial in South Korea.</p><p><strong>Methods: </strong>Ninety-five patients with PV received ropeginterferon alfa-2b biweekly for 48 weeks. NLR and <i>JAK2</i> allele burden were serially measured, and generalized estimating equations and logistic regression were used to assess associations.</p><p><strong>Results: </strong>NLR half reduction significantly predicted hematologic response (week 24 OR 6.42, <i>p</i> = 0.001) and molecular response consistently across all time points (week 24 OR 27.94, <i>p</i> < 0.001).</p><p><strong>Conclusion: </strong>NLR half reduction is a simple, cost-effective biomarker that may reflect molecular response and treatment efficacy in PV.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"17 ","pages":"20406207261431904"},"PeriodicalIF":2.8,"publicationDate":"2026-03-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13036332/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147594801","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}