Journal of Blood Medicine最新文献

{"title":"The Incidence of Hemolytic and Serologic Transfusion Reactions Among Patients with Hematological Malignancies at Mbarara Regional Referral Hospital and the Uganda Cancer Institute, in Uganda.","authors":"Ivan Mugisha Taremwa, Nixon Niyonzima, Scholastic Ashaba, Deusdedit Tusubira, May Y Choi, Craig N Jenne, Guido van Marle, Bernard Natukunda","doi":"10.2147/JBM.S589504","DOIUrl":"10.2147/JBM.S589504","url":null,"abstract":"<p><strong>Background: </strong>Blood transfusions are essential in the supportive care of patients with hematological malignancies but carry a risk of adverse reactions. Data on the incidence of transfusion reactions remain scarce in Uganda. This study evaluated the incidence of hemolytic and serologic transfusion reactions among patients with hematological malignancies at Mbarara Regional Referral Hospital and the Uganda Cancer Institute in Uganda.</p><p><strong>Materials and methods: </strong>This prospective cohort study enrolled hospitalized patients aged ≥2 years with hematological malignancies and a history of prior red blood cell (RBC) transfusions. Participants received additional transfusions and were monitored for up to 14 days. Blood samples were collected on day 0 (transfusion day), days 7, and 14 to assess hemoglobin (Hb) levels, lactate dehydrogenase (LDH), and perform direct and indirect antiglobulin tests. Participants' medical records were also reviewed for transfusion and pregnancy histories. Repeated Measures-Analysis of Variance was used to compare mean Hb and LDH levels over time points.</p><p><strong>Results: </strong>Of the 467 participants enrolled (median age: 36.4 years, interquartile range: 27.3-46.9), 382 completed the follow-up period. A progressive increase in mean Hb levels (g/dL) was observed: 6.6 (95% confidence interval [CI]: 6.1-8.9) on day 0, 7.5 (7.3-10.1) on day 7, and 8.9 (8.4-10.6) on day 14. No acute or delayed hemolytic reactions occurred. The incidence of delayed serologic transfusion reactions (DSTRs) was 0.86 in 100 (95% CI: 0.02-1.69), while the overall RBC alloantibody prevalence was 2.4% (95% CI: 0.98-3.74). The identified alloantibodies were directed against antigens in the Rh, Kell, Lewis, and MNS group systems.</p><p><strong>Conclusion: </strong>Despite a low incidence of DSTRs, a notable prevalence of RBC alloimmunization was observed among patients with hematological malignancies. These findings underscore the need to strengthen pre-transfusion antibody testing to prevent hemolytic complications and improve transfusion outcomes in this population.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"17 ","pages":"589504"},"PeriodicalIF":2.1,"publicationDate":"2026-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13098692/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147771946","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Impact of Transfusion Dependence on Clinical and Economic Burden in Patients with Lower-Risk Myelodysplastic Syndromes: A 28-Year Retrospective Study.","authors":"Jun Ho Jang, Chul Won Jung, Dae-Ho Choi, Ji-Hyun Kim, Kyungah Lee, Hyojin Kim","doi":"10.2147/JBM.S561536","DOIUrl":"10.2147/JBM.S561536","url":null,"abstract":"<p><strong>Background: </strong>Myelodysplastic syndromes comprise a diverse group of hematopoietic stem-cell malignancies characterized by ineffective hematopoiesis, cytopenia, and increased risk of acute myeloid leukemia. Anemia is a common manifestation of lower-risk myelodysplastic syndrome (LR-MDS) and often necessitates red blood cell (RBC) transfusions, although reliance on transfusions is associated with adverse outcomes and substantial burden. This 28-year, single-center, retrospective study aimed to assess the burden of RBC transfusion-dependence (RBC-TD) in terms of healthcare resource utilization (HCRU), medical costs, and clinical outcomes among patients with LR-MDS in South Korea.</p><p><strong>Patients and methods: </strong>Adult patients with LR-MDS initiating treatment between 1994 and 2022 at the Samsung Medical Center were included. HCRU, medical costs, and clinical outcomes were analyzed based on RBC-TD status.</p><p><strong>Results: </strong>Among 349 patients with LR-MDS, 103 (29.5%) were RBC-TD. RBC-TD was associated with increased HCRU and medical costs compared with non-transfusion-dependent (NTD) patients. Baseline erythropoietin (EPO) levels before erythropoiesis-stimulating agent (ESA) treatment were available for 76 patients with RBC-TD. Among 51 TD patients with baseline EPO levels ≤500U/L, 20 initiated ESA treatment after RBC-TD developed, to observe only limited effectiveness. Moreover, RBC-TD patients exhibited significantly shorter overall survival, leukemia-free survival and progression-free survival compared with NTD patients.</p><p><strong>Conclusion: </strong>RBC-TD in LR-MDS imposes significant clinical and economic burdens, including poor outcomes, heightened HCRU, elevated risks of disease progression, and premature mortality. These trends were particularly pronounced in patients with LR-MDS compared with the overall MDS population, underscoring that \"lower-risk\" does not necessarily equate to \"lower-burden\".</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"17 ","pages":"561536"},"PeriodicalIF":2.1,"publicationDate":"2026-04-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13094568/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147771918","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Interpreting Blood Transfusion in Hip Fracture Surgery: Marker of Severity Rather Than Mediator of Harm? [Response to Letter].","authors":"Lou'i Al-Husinat, Fadi Haddad, Sarah Al Sharie, Mohammad Araydah, Laith Al Hseinat, Mohamad Kharashgah, Adel Alsharei, Zaid Al Modanat, Mohammed S Alisi, Jihad Al-Ajlouni, Mohd Said Dawod, Silvia De Rosa, Denise Battaglini","doi":"10.2147/JBM.S610748","DOIUrl":"10.2147/JBM.S610748","url":null,"abstract":"","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"17 ","pages":"610748"},"PeriodicalIF":2.1,"publicationDate":"2026-03-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13049359/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147622866","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Interpreting Blood Transfusion in Hip Fracture Surgery: Marker of Severity Rather Than Mediator of Harm? [Letter].","authors":"Aisha Ahmad, Benjamin H L Harris, Michael B Fertleman, Louis J Koizia","doi":"10.2147/JBM.S608457","DOIUrl":"10.2147/JBM.S608457","url":null,"abstract":"","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"17 ","pages":"608457"},"PeriodicalIF":2.1,"publicationDate":"2026-03-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13034777/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147592305","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Joint Damage Prediction in Non-Severe Hemophilia A with Artificial Intelligence.","authors":"Ana Marco-Rico, José Manuel Calvo-Villas, Francisco-Jose Lopez-Jaime, Mariana Canaro Hirnyk, María Del Mar Nieto-Hernández, Sonia Herrero Martín, Laura Entrena-Ureña, Shally Marcellini-Antonio, Bolivar L Diaz-Jordan, Sergio Jurado-Herrera, Noelia F Pérez-González, Covadonga García-Díaz, Faustino García-Candel, Ihosvany Fernández-Bello, Jorge Mateo-Sotos, Pascual Marco-Vera","doi":"10.2147/JBM.S569311","DOIUrl":"10.2147/JBM.S569311","url":null,"abstract":"<p><strong>Purpose: </strong>Patients with non-severe hemophilia A (PwnSHA) can develop joint damage (JD). The objective was to identify a machine learning model based on routinely collected variables to predict the presence of JD in PwnSHA.</p><p><strong>Patients and methods: </strong>A nationwide, multicenter, cross-sectional study was conducted. Clinical and laboratory variables to assess joint health were included. Predictors were age, target joint history, thrombin generation capacity, baseline factor VIII (FVIII) measured by one-stage clotting (FVIII-CLOT) and chromogenic (FVIII-CHR) assays, and the FVIII-CLOT/FVIII-CHR ratio. The joint condition was described using the HEAD-US score. JD was defined as HEAD-US >0. A Random Forest (RF) ensemble was trained with regression-based multiple imputation, z-scaling, and Synthetic Minority Oversampling within a stratified five-fold stratified cross-validation repeated 100 times. Support Vector Machine, Decision Tree, Gaussian Naïve Bayes and k-Nearest Neighbors were used as comparators. Model performance was assessed on held-out test folds, and 95% confidence intervals (CIs) were obtained by bootstrap resampling with 10,000 repetitions.</p><p><strong>Results: </strong>Eighty-four Spanish males ≥12 years old were enrolled. Forty-two percent (35/84) had JD. JD was present in 30% (3/10) of patients with moderate hemophilia and 43% (32/74) with mild hemophilia. The RF achieved an accuracy of 92.0% (95% CI: 90.72-93.31), a recall of 92.1% (95% CI: 90.87-93.41), a specificity of 91.9% (95% CI: 90.58-93.27), and an AUC-ROC of 0.92 (95% CI: 0.907-0.938), outperforming all alternative classifiers. Permutation-based feature importance identified age, target joint history, thrombin generation and the FVIII-CLOT/FVIII-CHR ratio as the most influential variables.</p><p><strong>Conclusion: </strong>The RF model identifies PwnSHA more likely to have prevalent, occult JD in a cross-sectional setting, enabling rapid triage for targeted HEAD-US evaluation. External and prospective validation in larger cohorts is now warranted to confirm generalizability and to facilitate integration into electronic health-record decision-support systems aimed at preserving long-term joint health in PwnSHA.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"17 ","pages":"569311"},"PeriodicalIF":2.1,"publicationDate":"2026-03-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13011837/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147512373","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical and Genetic Pattern of β-Thalassemia Major in East Java, Indonesia.","authors":"Pradana Zaky Romadhon, Ami Ashariati, Siprianus Ugroseno Yudho Bintoro, Nasronudin Nasronudin, Bagus Aulia Mahdi, Aditea Etnawati Putri, Kartika Prahasanti, Afifah Zahra Dzakiyah, Kamila Auliya, Inswasti Cahyani","doi":"10.2147/JBM.S573064","DOIUrl":"10.2147/JBM.S573064","url":null,"abstract":"<p><strong>Background: </strong>Beta thalassemia major is the most common monogenic mutation disorder in Indonesia, with steadily increasing frequency. However, there are limited studies regarding genetic distribution and its relationship with the patient's clinical manifestation. This study aimed to identify the genetic mutation frequency and its association with the clinical phenotype pattern among β-thalassemia major patients in East Java, Indonesia.</p><p><strong>Methods: </strong>In this observational study, we include subjects who have diagnosed with β-thalassemia previously through Hb electrophoresis. Demographic distribution with several ethnicities of Javanese, Sundanese, Chinese, Maduranese, and Batak was recorded. From each subject, a total of 6 mL of blood sample was collected and divided into two ethylene diamine tetraacetic acid (EDTA) tubes for CBC and DNA extraction. DNA samples were analyzed by PCR and followed by Sanger sequencing.</p><p><strong>Results: </strong>A total of 91 subjects were included in this study, with a median age of 22.25 ± 7.56 years old; consisting of 52 females and 39 males, with Javanese as the most common ethnicity. There are 22 types of mutation were identified through Sanger sequencing. The most common mutation was IVS-1-5/CD 26 and the CD 35/CD 26 observed in 36 (39.5%) and 19 (20.8%), respectively. While 9 subjects (9.8%) had no mutation detected. Several clinical phenotypes, including iron overload, short stature, severe anemia, and splenomegaly, were most prevalent among the two most common genetic mutations.</p><p><strong>Conclusion: </strong>There is variability in clinical phenotype in β-thalassemia observed in several types of genotype mutations. Among all the mutations found in East Java, the genotypes IVS-1-5/CD 26 and CD 35/CD 26 were the two most frequent genotypes. Those genotypes are linear with the severity of the phenotype in β-thalassemia, such as severe anemia, iron overload, short stature, and splenomegaly.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"17 ","pages":"573064"},"PeriodicalIF":2.1,"publicationDate":"2026-03-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13011833/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147512417","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Study the Correlation of Red Blood Cell Indices and Electrolytes Imbalance in Anemic Patients in Ibb City, Yemen.","authors":"Abdul Baset Abbas, Reham Beshr, Rasha Al-Areqi, Kholod Al-Khateeb, Manar Aljabry, Kawthar Saif, Yasmin Al-Khateeb, Maisa Al-Halemi, Mohammed Gamah, Eihab Al-Herwi","doi":"10.2147/JBM.S563480","DOIUrl":"10.2147/JBM.S563480","url":null,"abstract":"<p><strong>Background: </strong>Anemia is a widespread illness. It is often associated with an electrolyte imbalance. The correlation between electrolytes and anemia is crucial. Electrolyte balance is essential for maintaining the shape and function of red blood cells (RBC), including the exchange of O₂ and CO₂. Therefore, this study aimed to investigate the correlation between RBC indices and electrolyte imbalance in anemic patients, Ibb City, Yemen.</p><p><strong>Methods: </strong>This study included 400 participants, consisting of 200 anemic patients and 200 non-anemic (controls). Approximately 5 mL of blood was collected directly from newly diagnosed anemic treatment-naïve patients and divided into tubes with and without EDTA to estimate RBC indices and serum electrolytes (sodium, potassium, chloride, calcium, and magnesium) directly.</p><p><strong>Results: </strong>Red cell indices (Hb, RBC, MCV, HCT, MCH, and MCHC) and electrolyte levels (potassium, calcium, and magnesium) were decreased significantly among patients compared to controls. Serum sodium was not significantly different. Notably, levels of chloride were considerably higher in anemic individuals. Odds ratio (OR) of hypo-electrolytemia with anemia, including hypo-magnesemia (OR=4.928, 95% CI: 2.278-10.47), hypo-kalemia (OR=2.967, 95% CI: 1.551-5.581), and hypo-calcemia (OR=2.833, 95% CI: 1.887-4.236), was significantly higher in anemic patients compared to controls. In contrast, OR of hyponatremia did not show a significant difference (OR =1.438, 95% CI: 0.9522-2.143). Notably, hyper-natremia was identified in the patient group, with no existence in controls, resulting in an infinite odds ratio (OR=∞, 95% CI: 0.5262-∞). Additionally, there were significant positive correlations between sodium and calcium levels with Hb, RBC, and HCT, as well as between magnesium levels with MCV and MCH. In contrast, significant negative correlations were found between chloride, magnesium, and calcium levels and Hb, RBC, and MCV, respectively.</p><p><strong>Conclusion: </strong>The study concluded a correlation and a significant disparity in serum electrolytes, particularly calcium, potassium, magnesium, and chloride, among anemic patients.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"17 ","pages":"563480"},"PeriodicalIF":2.1,"publicationDate":"2026-03-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12998301/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147486139","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prevalence, Morphological Types, and Associated Factors of Neonatal Anemia at a Rural Referral Hospital, Uganda.","authors":"Martha Amoding, Joachim Ndawula, Bright Hajusu, Isaac Morunyang, Christopher Lakwera, Robert Wagubi, Clinton Olong, Elizabeth A John, Enoch Muwanguzi, Benson Okongo","doi":"10.2147/JBM.S582356","DOIUrl":"10.2147/JBM.S582356","url":null,"abstract":"<p><strong>Purpose: </strong>This study aimed to determine the prevalence, morphological classification, and factors associated with anemia among neonates at Soroti Regional Referral Hospital neonatal unit.</p><p><strong>Patients and methods: </strong>We conducted a hospital-based cross-sectional study among 239 neonates between June and August 2025. Data on sociodemographic characteristics of neonates and mothers were collected using structured questionnaires and a review of medical records. Venous blood was collected from mothers during labor and cord blood was collected within 30 minutes of delivery or in situation where cord blood was not collected, then venous blood was collected from neonates. Hemoglobin estimation was done using a Nihon Kohden automated analyser. Neonates with umbilical cord hemoglobin <13 g/dL and mothers with venous blood haemoglobin <11 g/dL were classified as anemic. For anemic neonates, Giemsa-stained blood smears were examined to determine morphological type of anemia and screen for malaria parasites. Bivariate and multivariate logistic regression analysis were employed to identify factors associated with neonatal anemia, with a p-value ≤0.05 considered statistically significant.</p><p><strong>Results: </strong>The median age for neonates in days was 1 day and ranged from 0 to 27 days. The prevalence of neonatal anemia was 11.7% (95% CI: 7.6-15.8). Normocytic normochromic anemia was the most frequent morphological type (78.6%), followed by normocytic hypochromic (10.7%) and microcytic hypochromic anemia (10.7%). Multivariate analysis identified four factors independently associated with neonatal anemia: maternal history of anemia (AOR: 5.39 [95% CI: 1.15-25.32], p=0.033), lack of iron-folate supplementation during pregnancy (AOR: 6.62 [95% CI: 2.23-19.7], p=0.001), infrequent consumption of fruits and vegetables (AOR: 6.52 [95% CI: 1.23-34.48], p=0.027), and the presence of maternal anemia at delivery (AOR: 5.48 [2.11-14.21], p<0.001).</p><p><strong>Conclusion: </strong>This study confirms that neonatal anemia is a persistent health issue in eastern Uganda. The identified risk factors are primarily rooted in maternal health and nutrition. Our findings underscore the imperative for integrative antenatal strategies that combine nutritional education, promotion of iron-folate supplementation, and proactive management of maternal anemia to effectively reduce the burden of neonatal anemia.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"17 ","pages":"582356"},"PeriodicalIF":2.1,"publicationDate":"2026-03-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12996034/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147486103","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Phase 1/2, Open-Label, Single-Dose, Multicenter Study to Evaluate the Pharmacokinetics and Safety of Human Plasma-Derived Protein C Concentrate in Japanese Patients with Severe Congenital Protein C Deficiency (SCPCD).","authors":"Katsuyoshi Koh, Keiji Nogami, Harumi Kakuda, Yuri Okimoto, Moeko Hino, Yusuke Tanigawara, Zhaoyang Li, Chihiro Suzuki, Souhei Terashio, Hitoshi Ueda, Shouichi Ohga","doi":"10.2147/JBM.S535025","DOIUrl":"10.2147/JBM.S535025","url":null,"abstract":"<p><strong>Purpose: </strong>Severe congenital protein C deficiency (SCPCD) is a rare, life-threatening disorder. Plasma-derived protein C concentrate is recommended for the acute and long-term management of SCPCD; however, data in Japanese patients are lacking. In this study, the pharmacokinetics (PK) and safety of protein C concentrate were investigated in a Japanese population.</p><p><strong>Patients and methods: </strong>This study was an open-label, phase 1/2, nonrandomized, noncontrolled, multicenter clinical trial in Japanese patients with SCPCD (ClinicalTrials.gov: NCT04984889). Patients received a single intravenous dose (80 IU/kg) of human plasma-derived protein C concentrate. The primary endpoints were plasma protein C activity levels and PK parameters. Secondary endpoints for safety included adverse events (AEs).</p><p><strong>Results: </strong>All five enrolled patients (mean age, 15.2 years; mean weight, 34.0 kg) received the predefined dose of protein C concentrate and were included in PK and safety analyses. The geometric mean (coefficient of variation [CV]%) maximum concentration (C_max) was 1.679 IU/mL (31.7%) and the geometric mean (CV%) area under the curve (AUC_inf) was 21.88 IU·h/mL (47.1%). The median (range) half-life for protein C in plasma was 10.7 (7.35-12.4) hours. C_max and AUC_inf tended to be higher in older patients (≥20 years old) than in younger patients (<20 years old), whereas half-life was similar regardless of age. One patient had a mild treatment-related AE of pyrexia. No serious AEs or deaths were reported.</p><p><strong>Conclusion: </strong>PK parameters for protein C concentrate in Japanese patients with SCPCD were determined to be comparable to studies in western populations. A single intravenous 80 IU/kg dose was well tolerated, with no serious treatment-related AEs.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"17 ","pages":"535025"},"PeriodicalIF":2.1,"publicationDate":"2026-03-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12996032/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147486180","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Systematic Literature Review of the Economic and Quality of Life Burden of Congenital and Immune-Mediated Thrombotic Thrombocytopenic Purpura.","authors":"Harneet Kaur, Debjit Ghoshal, Inderpreet Singh Khurana","doi":"10.2147/JBM.S568965","DOIUrl":"10.2147/JBM.S568965","url":null,"abstract":"<p><strong>Background: </strong>Thrombotic thrombocytopenic purpura (TTP) is a life-threatening blood disorder resulting from ADAMTS13 deficiency, caused by mutations in the <i>ADAMTS13</i> gene in congenital TTP (cTTP), and by neutralizing antibodies against ADAMTS13 in immune-mediated TTP (iTTP). This systematic review summarizes available economic evaluations, healthcare resource utilization (HCRU), cost, utility, and health-related quality of life (HRQoL) literature in TTP, with a focus on cTTP. Given the limited availability of cTTP-specific data, broader TTP evidence was also reviewed to infer potential implications for cTTP where appropriate.</p><p><strong>Methods: </strong>Three systematic literature searches were conducted in January 2024 in accordance with PRISMA guidelines, each tailored to identify studies reporting economic evaluations, HCRU/cost data, and utility/HRQoL data respectively. For the economic evaluation and utility/HRQoL reviews, inclusion criteria were broadened to include studies reporting data on iTTP and unspecified TTP from which findings for cTTP were inferred.</p><p><strong>Results: </strong>In total 431, 989, and 849 records were identified for the economic evaluation, HCRU/cost, and utility/HRQoL reviews, respectively. No economic evaluations were identified for treatments in cTTP populations. Eight economic studies (all in iTTP) were included. Overall, 5 studies found caplacizumab to be cost-effective, whereas 3 did not, highlighting variability across model assumptions and healthcare settings. Six HCRU/cost studies reported that cTTP was associated with frequent hospital interactions, missed work time, and high costs. Fourteen studies (1 in cTTP, 10 in iTTP, and 3 in unspecified TTP) were included in the utility/HRQoL review and showed that TTP is associated with reduced utility scores and lower HRQoL, irrespective of TTP subtype.</p><p><strong>Conclusion: </strong>These findings underscore the meaningful patient burden observed across the TTP spectrum and highlight the need for further research to evaluate the cost-effectiveness of emerging therapies in cTTP. However, the predominance of iTTP data reflects ongoing challenges in evidence generation for rare TTP subtypes such as cTTP.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"17 ","pages":"568965"},"PeriodicalIF":2.1,"publicationDate":"2026-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12977066/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147443995","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}