Journal of Blood Medicine最新文献

{"title":"Use and Effectiveness of Carboximaltose Iron in Preoperative Anemia Treatment: A Multicenter and Retrospective Study.","authors":"Angel Manuel Yuste Gutierrez, Marta Alonso-Moreno, Jose Luis Perez Blanco, David Berlana, Maria Angeles Peña Fernandez, Maria Teresa Perez Maroto, Miguel Torralba","doi":"10.2147/JBM.S460422","DOIUrl":"10.2147/JBM.S460422","url":null,"abstract":"<p><strong>Aim: </strong>Anemia, primarily due to iron deficiency, is a key risk factor in both elective and emergency surgeries. Immediate preoperative treatment with ferric carboxymaltose (FCM) in anemic patients can reduce the need for transfusions and the length of hospital stay, thereby optimizing surgical outcomes. The objective of this study was to assess the effectiveness and describe the use of administering intravenous FCM prior to elective scheduled surgery for patients diagnosed with anemia.</p><p><strong>Methods: </strong>Multicenter, retrospective cohort study that encompassed patients aged 18 years and older who underwent surgery between January 2017 and December 2018. Demographic variables, dose scheme, baseline and perioperative haemoglobin (Hb), transfusion requirements, and admission days were collected. The primary endpoints were the response rate and effectiveness of FCM, defined as the proportion of patients with Hb preoperative levels of ≥13 g/dL. A patient response was deemed to occur when Hb level increased by 1 g/dL or more. The secondary endpoints were the appropriateness of FCM dose, transfusion requirement rate, and length of hospital stay.</p><p><strong>Results: </strong>446 patients (55.2% women, median age 69 IQR:52-78 years) were included. The median total dose of FCM administered was 1000 mg over a span of 5 day (IQR: 0-16) days before surgery. 62.8% of patients received lower doses, 24.9% had an INCREASE of Hb ≥ 1 g/dL, 11.6% had Hb ≥ 13 g/dL and 21.3% required blood transfusions, with a mean of 0.73 units transfused. The length of the hospital stay was 12 days (IQR:6-23).</p><p><strong>Conclusion: </strong>Low percentage of patients achieved a hemoglobin level of 13 g/dL or experienced an increase in hemoglobin of 1 g/dL or more following the administration of FCM, indicating the low effectiveness of FCM in treating perioperative anaemia in our surgical patients. There is underdosing of FCM and insufficient time between FCM administration and surgery in most patients. Both transfused and non-transfused patients show similar Hb increases, while those receiving a standard 1000 mg dose of FCM experience shorter hospital stays compared to those receiving 500 mg, and patients with more transfusions have longer hospital stays.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"15 ","pages":"477-486"},"PeriodicalIF":2.1,"publicationDate":"2024-11-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11577930/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142681773","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":"Paving the Road for Haematopoietic Stem Cell Transplantation in the United Arab Emirates: A Single Centre's Experience.","authors":"Panayotis Kaloyannidis, Biju George, Ahmad Alrustamani, Charbel Khalil, Basmah Al-Charfli, Nour Al-Moghrabi, Dima Ibrahim, Mohammed Alfar, Mohammed Daryahya, Kayane Mheidly, Amro El-Saddik, Humaid O Al-Shamsi","doi":"10.2147/JBM.S468665","DOIUrl":"https://doi.org/10.2147/JBM.S468665","url":null,"abstract":"<p><strong>Background: </strong>Despite its long-term history, haematopoietic stem cell transplantation (HSCT) still faces challenges in several countries under development and especially in the private health sector.</p><p><strong>Objectives design and methods: </strong>In this retrospective analysis, we present our experience and the results of 48 adult patients who underwent HSCT (autologous 37, allogeneic 9) at a private-sector hospital in Abu Dhabi, United Arab Emirates (UAE). The main indications were multiple myeloma and acute myeloid leukaemia in the autologous and allogeneic setting, respectively.</p><p><strong>Results: </strong>All patients successfully engrafted, and after a median follow-up of 6 (range: 1-11) months, 42 patients are alive (36 autografted and 6 allografted). The 1-year overall survival rates were 97% and 62% for autografted and allografted patients, respectively, while 4 patients died within 100 days post-transplant from treatment-related causes (1 patient from the autografted group and 3 patients from the allografted group).</p><p><strong>Conclusion: </strong>Our data confirm that HSCT is a feasible, safe, and effective treatment approach, offering high success rates to UAE patients with haematological malignant diseases.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"15 ","pages":"471-475"},"PeriodicalIF":2.1,"publicationDate":"2024-11-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11546142/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142621097","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":"Evaluation of IgG and Complement Component C4 Levels in Low-Income Countries, Yemen Republic in Light of Their Proposed Role in the Hemolysis of Stored CPDA-1 Whole Blood.","authors":"Jamil M A S Obaid, Khawla A A S Sakran, Shaima A H Mohammed, Shifa A L A Al-Salahi, Nawal A N Mahdi, Mohammed A M Al-Sharabi, Asadaddin S M Al-Gaadi, Mohammed N M Al-Fatahi","doi":"10.2147/JBM.S472605","DOIUrl":"10.2147/JBM.S472605","url":null,"abstract":"<p><strong>Objective: </strong>Hemolysis is the most severe change that occurs in stored blood and can cause severe consequences in patients after transfusion. This study examines the potential role of IgG and complement, exampled by C4, in the hemolysis of stored CPDA-1 blood under poor storage conditions in low-income countries.</p><p><strong>Methods: </strong>The study was performed on 30 whole blood units (250 mL) drawn from convenience healthy volunteer donors with CPDA-1 anticoagulant and stored at 2-6 °C for 35 days. Each well-mixed blood bag was sampled at 0, 7, 21 and 35 days and examined for CBC, plasma hemoglobin, hemolysis percent and determination of IgG and C4.</p><p><strong>Results: </strong>The plasma hemoglobin level and hemolysis percent increased continuously to reach 1.56 g/dl and 7.05% at the end of storage time. Hemolysis increased alongside the mean IgG concentration that was increased significantly from day 0 of storage (7.68±1.75 g/L) and peaked on day 7 (11.55±1.57 g/L), then declined to reach 8.33±2.09 g/L on day 35. Also, the mean concentration of C4 increased from day 0 of storage (0.15±0.06 g/L) to a peaked on day 21 (0.18±0.04) then declined on day 35 (0.17±0.06 g/L). The coordinated action of IgG and C4 is reflected by the positive correlation of their delta changes (r=0.616, p<0.0001).</p><p><strong>Conclusion: </strong>Elevated hemolysis percent in whole CPDA-1 stored blood in Yemen was accompanied by initial increase of IgG and C4 followed by final decline, which indicate their activation and consumption during hemolysis. Further studies for other hemolysis markers and analyses will give a full idea about that.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"15 ","pages":"459-469"},"PeriodicalIF":2.1,"publicationDate":"2024-10-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11526732/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142557897","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 Dosimetric Comparison Study for Blood Irradiation Employing Different Medium and Algorithms in Clinical Linear Accelerator.","authors":"Sarath S Nair, Jyothi Nagesh, Shambhavi C, Anshul Singh, Shirley Lewis, Umesh Velu, Deepika Chenna","doi":"10.2147/JBM.S476581","DOIUrl":"10.2147/JBM.S476581","url":null,"abstract":"<p><strong>Objective: </strong>To identify a suitable approach for blood irradiation other than the commonly used water medium and to study the impact of different algorithm dose computations.</p><p><strong>Methods: </strong>Water is the commonly used medium for blood irradiation. In this study computed tomography scans were taken with locally made blood irradiation phantoms other than water, by using air, rice powder and thermocole using parallel beam for 25 Gy. Plans were recalculated for different algorithms such as collapsed cone (CC), Monte Carlo (MC) and pencil beam (PB). The dose-volume parameters and measured doses were collected and analyzed for each medium and algorithm.</p><p><strong>Findings: </strong>The monitor unit (MU) for rice powder and water are close (2461±57 and 2469±61, respectively), with a maximum dose of 28.0±1.8 and 28.0±1.9 Gy. The PB algorithm resulted in lower monitor unit values regardless of the medium used, generating values of 2418, 2406, 2382, and 2362 for water, rice powder, air, and Thermocol, respectively. A significant increase in dose was observed irrespective of the medium used when the MC algorithm was employed, with a maximum of 30.26 Gy in rice powder; a smaller dose was used when the CC algorithm was employed, with 26.3 Gy in water medium. The average maximum doses of all groups were equal using the one-way Anova statistical test. Regarding the impact of field size, rice powder appears to have consistent doses across various field sizes, with slight increases as field size grows, which is similar to water.</p><p><strong>Novelty/applications: </strong>While water is the conventional medium, this study highlights the potential benefits of rice powder, such as eliminating the risks associated with bubble formation and water spillage, which can lead to equipment malfunction and safety hazards. Although previous studies have explored rice powder as a bolus and tissue-equivalent material, this study uniquely applies this knowledge to blood irradiation, an area where rice powder has not been thoroughly investigated.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"15 ","pages":"449-458"},"PeriodicalIF":2.1,"publicationDate":"2024-10-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11523925/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142545680","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":"Newer Modalities and Updates in the Management of Sickle Cell Disease: A Systematic Review.","authors":"Zeel Vishnubhai Patel, Priyadarshi Prajjwal, Lakshmi Deepak Bethineedi, Divyakshi J Patel, Kaarvi Khullar, Hinal Patel, Kanishka Khatri, Mohammed Dheyaa Marsool Marsool, Srikanth Gadam, Soumya Aleti, Omniat Amir","doi":"10.2147/JBM.S477507","DOIUrl":"https://doi.org/10.2147/JBM.S477507","url":null,"abstract":"<p><p>Sickle cell disease (SCD), the most common autosomal recessive genetic disorder, affects the hemoglobin (Hb) chains in human red blood cells. It is caused by mutations in the β-globin genes, leading to the production of hemoglobin S, which results in the formation of sickle-shaped red blood cells (RBCs). These abnormal cells cause hemolysis, endothelial damage, and small vessel occlusion, leading to both acute and long-term complications. According to the World Health Organization's 2008 estimates, SCD affects approximately 2.28 per 1000 individuals globally. Despite this high prevalence, therapeutic advancements have been slow. For many years, the only FDA-approved medications for managing SCD complications were hydroxyurea and deferiprone. However, recent years have seen the approval of several new therapies, including L-glutamine (2017), voxelotor and crizanlizumab (2019), as well as exagamglogene autotemcel (Casgevy) and lovotibeglogene autotemcel (Lyfgenia) (2023). These treatments have proven effective in managing both the acute and chronic effects of SCD, including hemolytic anemia, chronic pain, stroke, vaso-occlusive crises, and multiple organ damage syndromes. This review explores the mechanisms of action, practical considerations, and side effects of these emerging therapies, drawing from a comprehensive search of databases such as PubMed, Medline, and Cochrane.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"15 ","pages":"435-447"},"PeriodicalIF":2.1,"publicationDate":"2024-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11404495/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142287961","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":"Management of Advanced Systemic Mastocytosis: Clinical Challenges.","authors":"Douglas Tremblay, Nicole E Wagner, John Mascarenhas","doi":"10.2147/JBM.S366367","DOIUrl":"https://doi.org/10.2147/JBM.S366367","url":null,"abstract":"<p><p>Advanced systemic mastocytosis (AdvSM) is a rare hematologic malignancy with organ damage and compromised life expectancy arising from organ accumulation of neoplastic mast cells. Identification of the gain-of-function <i>KIT</i>D816V in the majority of cases has accelerated pharmaceutical development culminating with the development of selective KIT inhibitors such as avapritinib. While the advent of these therapies has improved the quality and quantity of life in patients with AdvSM, current challenges remain in the management of this disease. In this review, we summarize the present and future therapeutics landscape of AdvSM, highlighting the development of novel KIT inhibitors including elenestinib and bezuclastinib. We also explore the continued role of additional treatment modalities including allogeneic stem cell transplantation before discussing unresolved clinical challenges in the management of AdvSM.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"15 ","pages":"421-433"},"PeriodicalIF":2.1,"publicationDate":"2024-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11402342/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142287960","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":"Specific Mutation Predict Relapse/Refractory Diffuse Large B-Cell Lymphoma.","authors":"Jing Wang, Lei Tian, Weilong Zhang, Shuhan Tang, Wei Zhao, Yu Guo, Chaoling Wu, Yuansheng Lin, Xiaoyan Ke, Hongmei Jing","doi":"10.2147/JBM.S471639","DOIUrl":"https://doi.org/10.2147/JBM.S471639","url":null,"abstract":"<p><strong>Background: </strong>The application of rituximab has significantly enhanced the overall survival rates in patients with diffuse large B-cell lymphoma (DLBCL). Regrettably, a significant number of patients still progress to relapse/refractory DLBCL (rrDLBCL).</p><p><strong>Methods: </strong>Herein, we employed targeted sequencing of 55 genes to investigate if gene mutations could predict the progression to rrDLBCL. Additionally, we compared the mutation profiles at the time of DLBCL diagnosis with those found in rrDLBCL cases.</p><p><strong>Results: </strong>Our findings highlighted significantly elevated mutation frequencies of <i>TP53, MEF2B</i> and <i>CD58</i> in diagnostic biopsies from patients who progressed to relapse or refractory disease, with CD58 mutations exclusively observed in the rrDLBCL group. In assessing the predictive power of mutation profiles for treatment responses in primary DLBCL patients, we found that the frequency of <i>CARD11</i> mutations was substantially higher in non-response group as compared with those who responded to immunochemotherapy. In addition, we revealed mutations in <i>HIST2H2AB, BCL2, NRXN3, FOXO1, HIST1H1C, LYN</i> and <i>TBL1XR1</i> genes were only detected in initial diagnostic biopsies, mutations in the EBF1 gene were solely detected in the rrDLBCL patients.</p><p><strong>Conclusion: </strong>Collectively, this study elucidates some of the genetic mechanisms contributing to the progression of rrDLBCL and suggests that the presence of <i>CD58</i> mutations might serve as a powerful predictive marker for relapse/refractory outcomes in primary DLBCL patients.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"15 ","pages":"407-419"},"PeriodicalIF":2.1,"publicationDate":"2024-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11401521/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142287962","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":"Management of Congenital Methemoglobinemia in the Perioperative Setting: A Case Report and Review of Current Literature.","authors":"Moncef Ben Ghoulem Ben Saad, Arunabha Karmakar, Tayseer Salih Mohamed Salih, Wajeeha Arshad, Muhammad Jaffar Khan","doi":"10.2147/JBM.S468072","DOIUrl":"10.2147/JBM.S468072","url":null,"abstract":"<p><strong>Background: </strong>Methemoglobin is an altered state of hemoglobin where iron in hemoglobin is oxidized and incapable of binding oxygen; leading to complications such as cyanosis, dyspnea, headache, and heart failure. Methemoglobinemia can be congenital or acquired. Congenital methemoglobinemia is a rare disease and its worldwide incidence is unclear. We recently encountered the first documented case of congenital methemoglobinemia at our institution, necessitating perioperative care.</p><p><strong>Case presentation: </strong>In the present case, a 22-year-old man with congenital methemoglobinemia underwent general anesthesia for dental extraction. The surgeon was informed to avoid local anesthetics and oxygenation was performed with FiO₂ of 1.0. Arterial blood gas analysis showed a PH of 7.337, PaO₂ of 302 mm Hg, PaCO₂ of 44 mm Hg, oxyhemoglobin level of 63.4%, and methemoglobin level of 37.8%. The patient had a stable course. No methylene blue therapy was required, although cyanosis was observed during surgery.</p><p><strong>Conclusion: </strong>In summary, though rare, congenital methemoglobinemia poses fatal risks during surgery. Its management involves preoperative recognition and optimization, oxygenation status, multidisciplinary care, avoiding precipitating or oxidizing agents, discussing treatment options, maintaining cardiopulmonary stability, and ensuring perioperative safety measures with the medical team.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"15 ","pages":"395-405"},"PeriodicalIF":2.1,"publicationDate":"2024-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11368142/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142119881","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":"Prediction of Viable CD34 Count in Harvested Product by Peripheral Blood Hematopoietic Progenitor Count of Automated Hematology Analyzer Undergoing Hematopoietic Stem Cell Transplantation.","authors":"Siew Lian Chong, Asral Wirda Ahmad Asnawi, Tengku Amatullah Madeehah Tengku Mohd, Sen Mui Tan","doi":"10.2147/JBM.S460820","DOIUrl":"10.2147/JBM.S460820","url":null,"abstract":"<p><strong>Introduction: </strong>The CD34+ hematopoietic cell count was used to define cell harvest goals. Successful peripheral blood stem cell transplantation depends on infusion of an appropriate number of HPCs to achieve rapid and durable hematologic recovery.</p><p><strong>Purpose: </strong>In this study, we evaluated the use of the Hematopoietic Progenitor Cell count program on the Sysmex XN-3000 hematology analyzer as an effective parameter for enumerating CD34+ cells.</p><p><strong>Patients and methods: </strong>Whole blood samples from 144 subjects who are either healthy donors or patients scheduled to undergo peripheral blood stem cell collection were collected and hemopoietic stem cells were quantified using CD34 cell enumeration by flow cytometry and XN-HPC by hematology analyzer.</p><p><strong>Results: </strong>The correlation between the two methods was high (r = 0.766; 95% CI: 0.702-0.818). Passing-Bablok showed an intercept at 3.45 (2.54 to 4.74) with a slope of 0.78 (95% CI 0.69 to 0.89). Residual analysis of this model indicated no significant deviation from linearity (p = 0.360). The receiver operating characteristic curve demonstrated an area under curve to be 0.88 (0.82 to 0.92), with a positive predictive value of 80.3%. The correlation between CD34+ and XN-HPC showed a strong relationship and good agreement with minimal bias.</p><p><strong>Conclusion: </strong>The XN-HPC showed good analytical performance. With the increasing requirements for stem cell transplantation, a technically simple and rapid alternative for stem cell enumeration that is sustainable is highly useful.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"15 ","pages":"387-394"},"PeriodicalIF":2.1,"publicationDate":"2024-08-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11346492/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142072897","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 Systematic Review of the Epidemiology and Disease Burden of Congenital and Immune-Mediated Thrombotic Thrombocytopenic Purpura.","authors":"Ping Du, Tiffany Cristarella, Camille Goyer, Yola Moride","doi":"10.2147/JBM.S464365","DOIUrl":"10.2147/JBM.S464365","url":null,"abstract":"<p><p>Congenital (cTTP) and immune-mediated (iTTP) thrombotic thrombocytopenic purpura are serious and rare clotting disorders resulting from a deficiency in the ADAMTS13 enzyme. A systematic review was conducted using the Ovid^® MEDLINE & Embase databases to synthesize the epidemiology and burden of cTTP and iTTP worldwide (from January 1, 2010, to February 6, 2020, with an update that covered the period January 1, 2020-February 11, 2022). Outcomes of interest were incidence and prevalence of TTP, incidence of acute episodes, mortality, burden of illness (eg complications, healthcare utilization, patient-reported outcomes) and disease management. A total of 221 eligible observational studies were included. The incidence rate of acute episodes ranged from 0.19-0.35 person-years in adult patients with cTTP, and 1.81-3.93 per million persons per year for iTTP in the general population. Triggers of acute episodes were similar for cTTP and iTTP, with pregnancy and infection the most commonly observed. Exacerbation in patients with iTTP varied widely, ranging from 2.4-63.1%. All-cause mortality was observed in 0-13.4% of patients with cTTP, across studies and follow-up periods, and in 1.1% (median follow-up: 0.4 years) to 18.8% (1 year) of patients with iTTP during acute episodes. Cardiovascular, renal, and neurological disease were common complications. TTP also led to work disturbances, feelings of anxiety and depression, and general activity impairment. TTP treatment regimens used were generally reflective of current treatment guidelines. The evidence identified describes a high patient burden, highlighting the need for effective treatment regimens leading to improvements in outcomes. Considerable evidence gaps exist, particularly for disease epidemiology, patient-reported outcomes, costs of disease management, and associated healthcare resource utilization. This review may help increase disease awareness and highlights the need for additional real-world studies, particularly in geographical regions outside the United States and Western Europe.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"15 ","pages":"363-386"},"PeriodicalIF":2.1,"publicationDate":"2024-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11330749/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142004267","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}