Therapeutic Advances in Hematology最新文献

{"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":"https://doi.org/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":3.1,"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":"https://doi.org/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":3.1,"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":"https://doi.org/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":3.1,"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":"https://doi.org/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":3.1,"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":"https://doi.org/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":3.1,"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}

{"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":3.1,"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":3.1,"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":3.1,"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":3.1,"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}