Journal of Blood Medicine最新文献

{"title":"Determine Complete Blood Count Reference Values Among Healthy Adult Populations.","authors":"Abdul Baset Abbas, Abeer Aldomaini, Amah Alrahman Al-Qadri, Zahra'a Algorbani, Sara Aljamali, Safa Alsiri, Khadeeja Alghorbani, Saba Abo Osba'a","doi":"10.2147/JBM.S488050","DOIUrl":"10.2147/JBM.S488050","url":null,"abstract":"<p><strong>Background: </strong>Complete blood counts (CBC) are commonly used in diagnostic medicine to evaluate normal and abnormal hematological status. Furthermore, reference values (RVs) of CBC supplied by researchers are the most reliable means of the judgment-making stage and can aid interpretation and accurate diagnosis of diseases. Reference values vary between peoples because of differences in lifestyle, dietary habits, ethnicity and environment. Moreover, the Clinical and International Standards Institute (CISI) advises determining the RVs for each area. There are no RVs for CBC in Yemen. Therefore, this study aimed to determine the common RVs of CBC for healthy adults in Ibb City in the middle of Yemen.</p><p><strong>Methods: </strong>A cross-sectional study was conducted from April 1, to November 30, 2023. Of the 623 adults who participated in this study, 433 (aged 18-80 years) were included in the final analysis after applying exclusion criteria. The mean, median, and 95^th percentile RVs (2.5^th-97.5^th percentiles) were calculated for gender, age, and residence by the GraphPad Prism 8.0.1.</p><p><strong>Results: </strong>The RVs of hemoglobin (Hb) 11.16-17.54g/dl, red blood cells (RBC) 3.890-6.340×10¹²/l, hematocrit (HCT) 33.03-49.30%), mean corpuscular volume (MCV) 72.83-94.55fl), mean corpuscular hemoglobin (MCH) 23.95-33.55pg, mean corpuscular hemoglobin concentration (MCHC) 32.97-36.7354g/dl, platelet (PLT) count 140.0-418.6×10⁹/l, total white blood cells (WBC) 2.810-8.797 ×10⁹/l and WBC differential count (basophils 0.000-1.000%, neutrophils 30.10-69.17%, eosinophils 1.500-5.000%, lymphocytes 23.86-63.45% and monocytes1.873-5.600%). Significantly higher median values were observed in males compared to females for Hb (<i>P</i><0.0001), RBC (<i>P</i><0.0001), HCT (<i>P</i><0.0001), lymphocyte (<i>P</i>=0.0197) and monocytes (<i>P</i>=0.0009). Contrariwise, females demonstrated significantly higher neutrophils (<i>P</i>=0.0009), eosinophils (<i>P</i>=0.0020), basophils (<i>P</i><0.0001) and platelets (<i>P</i>=0.0324) than males. This study showed differences in the RVs of CBC compared to those reported in other countries in the Middle East, Asia, Africa, and Europe.</p><p><strong>Conclusion: </strong>In this study, the reference values of CBC are considered as a benchmark that may assist in accurately judging laboratory results and enhancing medical and clinical services for adults in Ibb City, Yemen.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"15 ","pages":"513-522"},"PeriodicalIF":2.1,"publicationDate":"2024-12-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11668252/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142885833","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":"Thrombotic Risk Assessment, P-Selectin, and Thromboprophylaxis Use Among, Cancer Patients at the University of Calabar Teaching Hospital, Calabar.","authors":"Kingsley Akaba, Edakabasi Akaba, Olukayode Oshatuyi, Brian Ssenkumba","doi":"10.2147/JBM.S478192","DOIUrl":"10.2147/JBM.S478192","url":null,"abstract":"<p><strong>Background: </strong>Venous thromboembolism is the second leading cause of mortality among cancer patients. The Khorana Risk Assessment Score (KRAS) is widely acknowledged as the most validated tool in this context.</p><p><strong>Aim: </strong>To assess the thrombotic risk in cancer patients using the modified Khorana Risk Assessment Score, examine the association between modified KRAS and soluble P-selectin levels, and document the utilization of thromboprophylaxis among cancer patients at the University of Calabar Teaching Hospital.</p><p><strong>Methods: </strong>This was a cross-sectional hospital-based recruiting 100 cancer patients. Seven millilitres of blood were collected for complete blood count and P-selectin assay. Continuous variables were expressed as mean and standard deviation, while categorical variables were summarized using frequencies. Chi-square was employed to compare VTE risk status across genders, different cancer types, and guideline compliance. The significance level was set at 0.05.</p><p><strong>Results: </strong>Participants age ranged from 19 to 87 years, with a male-to-female ratio of 1:1.6. The most common female cancer was Breast at 40.32% and prostate cancer at 65.79% was the most common in males. Seventy nine percent and 21% of participants had intermediate and high-risk modified KRAS scores respectively. The median level of soluble P-selectin among cancer patients was 23.00 within the interquartile range. Significant associations were observed between cancer types and sex, VTE risk assessment and cancer types, and cancer types and risk score.</p><p><strong>Conclusion: </strong>The risk of VTE among cancer patients ranges from intermediate to high, going by the modified Khorana risk score irrespective of the P selectin level, with underutilization of thromboprophylaxis. There is little adherence to the Khorana score in our setting, hence the need for greater application and knowledge of this predictive score in clinical practice to improve outcomes and quality of life.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"15 ","pages":"501-512"},"PeriodicalIF":2.1,"publicationDate":"2024-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11654208/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142853871","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":"COVID-19-Associated Immune Thrombocytopenic Purpura in a Hemodialysis Patient.","authors":"Shuqin Mei, Cheng Xue, Zheng Zhang, Lingling Liu, Wenwen Cai, Xuelian Gong, Zhiguo Mao, Xiaojing Tang, Bing Dai","doi":"10.2147/JBM.S496127","DOIUrl":"10.2147/JBM.S496127","url":null,"abstract":"<p><strong>Background: </strong>Severe acute respiratory syndrome coronavirus (SARS-CoV-2) infection has been a global threat since the end of 2019. Although the main clinical manifestation of coronavirus disease 2019 (COVID-19) is respiratory, its range of clinical manifestation is extensive and may include various systems, including hematological disorders, such as lymphopenia, thrombotic events, thrombocytopenia and immune thrombocytopenic purpura (ITP). The present case was the first one that aimed to raise awareness of ITP induced by COVID-19 in patients undergoing maintenance hemodialysis.</p><p><strong>Case presentation: </strong>This is the case of a 75-year-old Asian woman who was diagnosed COVID-19 positive 15 days before attending our Emergency Department on January 19th, 2023, with a three-day history of severe bleeding symptoms, including gastrointestinal, mucosal bleeding, epistaxis, and the platelet count of 5×10⁹/L. She suffered from end-stage kidney disease due to autosomal dominant polycystic kidney disease and has received thrice-weekly maintenance hemodialysis (MHD) since 2012. Platelet count recovery was observed after 45 days of combined treatment with corticosteroids, intravenous immunoglobulin, thrombopoietin receptor agonists, and rituximab. The count of platelets rose to 180×10⁹/L after four dosages of Rituximab.</p><p><strong>Conclusion: </strong>In brief, SARS-CoV-2 infection might trigger the onset of ITP. To our knowledge, this is the first case with severe and refractory ITP secondary to COVID-19 in MHD patients and no guidelines were able to be referred on the therapy. Nephrologists must be concerned with clinical characteristics, diagnostic flowcharts, and therapy for SARS-CoV-2-induced ITP.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"15 ","pages":"495-500"},"PeriodicalIF":2.1,"publicationDate":"2024-11-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11614712/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142780009","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":"Blood Donation: Fears and Myths in Healthcare Workers of the Future.","authors":"Maham Arshad, Ayesha Ellahi, Fahad Ahmed, Javaid Usman, Saleem Ahmed Khan","doi":"10.2147/JBM.S446697","DOIUrl":"https://doi.org/10.2147/JBM.S446697","url":null,"abstract":"<p><strong>Objective: </strong>To determine the fears and myths related to blood donation in future health care workers.</p><p><strong>Study design: </strong>Cross-sectional study.</p><p><strong>Place and duration of study: </strong>This study was carried out from October to December 2022 at the National University of Medical Sciences (NUMS), Rawalpindi, Pakistan. Donors were selected according to the, WHO recommended, Safe Blood Transfusion Program of Pakistan criteria.</p><p><strong>Results: </strong>In total, 411 participants were included in the study. The individuals were 21-24 years of age, with a mean age of 21 years. In our study, females dominated (232/411); the remaining 179 were males. Out of the total 411, 145 participants had previously donated blood while the other 266 had never donated blood. Our study analyzed both of these groups. The most common symptoms experienced by blood donors were dizziness, post-donation weakness, and bodily aches and pains. Most non-donors feared problems related to their general health (42.3%) and developing infections (12.7%). <i>P-</i>value was 0.002, which reveals a significant association between fears and intention to donate blood.</p><p><strong>Conclusion: </strong>These results suggest that fears and concerns related to blood donation play a leading role in forecasting donors' attitudes and intentions. Motivation leads to inspiration and potential donors can be motivated by addressing their fear.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"15 ","pages":"487-493"},"PeriodicalIF":2.1,"publicationDate":"2024-11-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11608004/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142769269","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":"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}