{"title":"Current Status and Challenges in Delivering Comprehensive Care for Patients with Hemophilia.","authors":"Shosaku Nomura","doi":"10.2147/JBM.S446204","DOIUrl":"https://doi.org/10.2147/JBM.S446204","url":null,"abstract":"<p><p>The importance of comprehensive care as a treatment strategy for patients with hemophilia is recognized worldwide. Comprehensive care entails addressing full spectrum of medical and psychological aspects impacting both patients and their families. The primary objective of comprehensive care for individuals with hemophilia is to enable them to lead their daily lives just as anyone else would. To achieve this goal, it is necessary to have a positive and collaborative approach across various healthcare disciplines. This extends beyond clinical specialists, encompassing pediatricians, hematologists, orthopedic surgeons, dental and oral surgeons, gynecologists, nurses, physical therapists, clinical psychologists, and other professionals from diverse fields. This review article discusses the current status and challenges associated with comprehensive care for patients with hemophilia. We categorize these challenges as follows: hemophilic arthritis, rehabilitation, oral care, transitioning from pediatric to adult care, addressing carrier issues, and providing psychological care. There is still substantial work to be undertaken in addressing these hurdles and advancing the quality of comprehensive care for hemophilia patients.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"14 ","pages":"629-637"},"PeriodicalIF":2.0,"publicationDate":"2023-12-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10730945/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138829886","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}
Marie M Plante, ErinMarie O Kimbrough, Amit K Agarwal, Liuyan Jiang, Kirk Bourgeois, Greta C Stamper, Michael W Stewart, Han W Tun
{"title":"Hyperviscosity Syndrome Induced Bilateral Visual and Auditory Impairment in Therapy Resistant Waldenström Macroglobulinemia with MYD88 and CXCR4 Mutations.","authors":"Marie M Plante, ErinMarie O Kimbrough, Amit K Agarwal, Liuyan Jiang, Kirk Bourgeois, Greta C Stamper, Michael W Stewart, Han W Tun","doi":"10.2147/JBM.S424072","DOIUrl":"10.2147/JBM.S424072","url":null,"abstract":"<p><p>Hyperviscosity syndrome (HVS) is an emergent complication of Waldenström macroglobulinemia (WM) characterized by visual, neurologic, and rarely auditory impairment. We report a 69-year-old female with MYD88 and CXCR4-mutant WM who developed HVS resulting in bilateral blindness and deafness associated with neurologic manifestations including confusion, severe generalized weakness, and imbalance. Ophthalmologic evaluation revealed bilateral central retinal vein occlusion (CRVO), diffuse retinal hemorrhages, macular edema, and serous macular detachments (SMD). Magnetic resonance imaging of the brain showed bleeding in the inner ears. Management was challenging as her WM was resistant to systemic therapies including bendamustine + rituximab (BR) and rituximab + bortezomib + dexamethasone (RVD). Bruton's tyrosine kinase inhibitors could not be used initially due to ongoing lower gastrointestinal bleeding. She required five total sessions of plasma exchange and was finally initiated on zanubrutinib, achieving a partial response. She also received intravitreal bevacizumab with rapid resolution of the retinal hemorrhages but with little improvement of the SMD. She had partial restoration of her hearing in the right ear and only slight improvement in her bilateral visual deficits. The management of HVS in frail, elderly patients with therapy-resistant WM can be challenging. In these cases, plasma exchange is required until an effective systemic therapy can be safely instituted. Genomic profiling is important in the management of WM as it can predict treatment resistance and guide therapeutic decisions.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"14 ","pages":"639-648"},"PeriodicalIF":2.0,"publicationDate":"2023-12-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10729678/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138797634","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}
Katsuyuki Fukutake, K. Togo, Lin Xu, Leona Markson, José Maria Alvir, Ian Winburn, T. Karumori
{"title":"Real-World Amount of Clotting Factor Concentrates Dispensed and Annual Medical Expenditures for Japanese Patients with Hemophilia B","authors":"Katsuyuki Fukutake, K. Togo, Lin Xu, Leona Markson, José Maria Alvir, Ian Winburn, T. Karumori","doi":"10.2147/jbm.s418818","DOIUrl":"https://doi.org/10.2147/jbm.s418818","url":null,"abstract":"Methods: This retrospective study comprised patients with hemophilia B (N=197) who had filled prescriptions for FIX concentrates reported in Japan’s Medical Data Vision database","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"44 7","pages":""},"PeriodicalIF":2.0,"publicationDate":"2023-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139013290","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Paroxysmal Nocturnal Hemoglobinuria: Current Management, Unmet Needs, and Recommendations","authors":"Monika Oliver, C. Patriquin","doi":"10.2147/JBM.S431493","DOIUrl":"https://doi.org/10.2147/JBM.S431493","url":null,"abstract":"Abstract Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-rare, acquired clonal abnormality, which renders hematopoietic cells exquisitely sensitive to complement-mediated destruction. Classical features of PNH include intravascular hemolytic anemia, increased thrombotic risk, and manifestations related to end-organ damage (eg fatigue, chest pain, dyspnea, renal failure, and pulmonary hypertension). With supportive care alone, mortality rate of patients with PNH is approximately 35%. The anti-C5 monoclonal antibody, eculizumab, was the first targeted therapy approved for PNH, and led to improved hemoglobin, quality of life, reduced transfusion need, reduced thrombosis, and greater overall survival. More recently, therapeutics such as longer acting anti-C5 (ravulizumab) and anti-C3 (pegcetacoplan) medications have been approved, along with other novel therapeutics in late-stage clinical trials. Biosimilars of eculizumab are also now available. Proximal inhibitors (against C3, factor B, and factor D) have shown significant improvements in hemoglobin and transfusion-avoidance in patients who remain anemic despite C5 inhibition. Despite these novel therapies, some unmet challenges remain, including management of breakthrough hemolysis, clinically significant iatrogenic extravascular hemolysis, optimal management in pregnancy, and infection risk mitigation as new targets in the complement system are blocked. In addition, the use of self-administered subcutaneous and oral therapies raises concerns around treatment adherence and the risks of uncontrolled terminal complement. Given the ultra-rare nature of PNH, development is underway of a centralized international registry to capture and analyze the data as they mature for various new therapies and characterize the clinical challenges related to PNH management.","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":" 34","pages":"613 - 628"},"PeriodicalIF":2.0,"publicationDate":"2023-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138611480","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Richard R Gammon, Rounak Dubey, Gaurav K Gupta, Colleen Hinrichsen, Aikaj Jindal, Divjot Singh Lamba, Sadhana Mangwana, Amita Radhakrishnan Nair, Shaughn Nalezinski, Christopher Bocquet
{"title":"Patient Blood Management and Its Role in Supporting Blood Supply.","authors":"Richard R Gammon, Rounak Dubey, Gaurav K Gupta, Colleen Hinrichsen, Aikaj Jindal, Divjot Singh Lamba, Sadhana Mangwana, Amita Radhakrishnan Nair, Shaughn Nalezinski, Christopher Bocquet","doi":"10.2147/JBM.S387322","DOIUrl":"10.2147/JBM.S387322","url":null,"abstract":"<p><p>Blood donors and voluntary blood donations are essential for ensuring the blood supply that can be maintained by good patient blood management (PBM) practices. This review article explores the role of blood donation in PBM and highlights the importance of donor screening and selection processes in different regions worldwide. The donor health questionnaires and the focused physical examination guidelines have changed in the last decade to increase donor and recipient safety. This article also discusses the status of transfusion practices, including the challenges of ensuring a safe blood supply. Significant among these are the effects of the COVID-19 pandemic on the blood supply chain and the impact of an aging donor population, especially. Promoting autologous donations and other blood conservation strategies are suggested to mitigate these issues. The role of replacement donors and the upper age limit for voluntary blood donation may be decided based on the demography and donor pool. The involvement of C-suite executives is also critical in implementing and running a successful PBM program. The review highlights how these different aspects of blood donation are integral to a successful PBM program and the safety of patients who receive blood transfusions.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"14 ","pages":"595-611"},"PeriodicalIF":2.0,"publicationDate":"2023-11-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10695136/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138487551","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":"Deferasirox in Patients with Chronic Kidney Disease: Assessing the Potential Benefits and Challenges.","authors":"Abdulqadir J Nashwan, Mohamed A Yassin","doi":"10.2147/JBM.S415604","DOIUrl":"10.2147/JBM.S415604","url":null,"abstract":"<p><p>Chronic kidney disease (CKD) is a major global health concern, affecting millions of people worldwide. The progressive decline in kidney function often necessitates renal replacement therapy, such as hemodialysis (HD) or peritoneal dialysis (PD), to maintain a patient's health. Iron overload, which is common in CKD patients on dialysis, can lead to severe complications, including cardiovascular disease and infections where most of the existing iron chelators are deemed unsuitable due to their suboptimal clearance in patients with compromised renal function, it becomes a significant challenge to effectively manage iron overload. Deferasirox (DFX), an oral iron chelator, has emerged as a promising treatment option for managing iron overload in these patients. However, the use of DFX comes with its unique set of challenges, such as its cost, potential side effects, and the need for close monitoring of patients, as well as the noticeable scarcity of comprehensive and rigorous clinical studies confirming its efficacy and safety of DFX. In this review, we delve into both the promising prospects and the emerging challenges associated with DFX use in managing CKD patients on HD or PD, striving for a comprehensive understanding that informs better clinical practice and patient care.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"14 ","pages":"589-594"},"PeriodicalIF":2.0,"publicationDate":"2023-11-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10693276/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138477786","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":"Acquired Clotting Factor Deficits During Treatment with Asparaginase in an Institutional Cohort.","authors":"Vasiliki Papadopoulou, Giulia Schiavini","doi":"10.2147/JBM.S428159","DOIUrl":"10.2147/JBM.S428159","url":null,"abstract":"<p><p>We invariably see prolongation of activated partial thromboplastin time in patients treated with asparaginase in our clinical practice, but have noted that, contrary to hypofibrinogenemia and low antithrombin, clotting times' prolongation by asparaginase is largely unreported in the literature and guidelines and is not widely known to clinicians. We report on aPTT prolongations in a small cohort of patients, and on their origin, as investigated by measurements of clotting factors, fibrinogen, and D-dimers before and after asparaginase administration. We observed significant reductions in FIX and FXI (median post-treatment values of 27 IU/dl and 52 IU/dl, respectively), confirming one previous observation. A decrease in FXII was less pronounced but contributed to the prolonged aPTTs (FXII has no effect on in vivo haemostasis). The factor deficits are not due to consumption, as evidenced by unchanged D-dimer levels, and are, therefore, probably caused by disturbed factor synthesis. Our observations and insights contribute to elucidation of the profile of clotting assays during asparaginase treatment, and thus, to optimally monitor for undesirable events or steer situations of therapeutic anticoagulation without the risk of suboptimal or excessive anticoagulation.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"14 ","pages":"569-574"},"PeriodicalIF":2.0,"publicationDate":"2023-11-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10641023/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"107606236","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}
Mouhammad J Alawad, Mohammad Abu-Tineh, Awni Alshurafa, Alaa Al-Taie, Anil Yousaf, Mohamed A Yassin
{"title":"Glanzmann Thrombasthenia Associated with Siderotic Synovitis and Arthropathy: A Case Report.","authors":"Mouhammad J Alawad, Mohammad Abu-Tineh, Awni Alshurafa, Alaa Al-Taie, Anil Yousaf, Mohamed A Yassin","doi":"10.2147/JBM.S418937","DOIUrl":"10.2147/JBM.S418937","url":null,"abstract":"<p><p>Glanzmann thrombasthenia is a bleeding disorder with a low incidence. It typically manifests as superficial bleeding episodes, which tend to be mild. Deep organ involvement is not uncommon but remains rare due to the rarity of the disease itself and the unusual association between platelet disorders and deep organ implications. A 17-year-old boy with Glanzmann thrombasthenia since infancy developed ankle pain after a minor trauma. His initial workup was negative, but he continued to experience ankle pain. A magnetic resonance imaging (MRI) done after four weeks suggested siderotic synovitis. The patient was lost to follow-up after that and returned after two years with recurrent left ankle pain. Imaging and studies have shown evidence of chronic arthropathy. A specialized orthopedic team assessed the patient. The patient underwent intra-articular steroid injection for pain relief and was referred to continue physical therapy. In conclusion, hemarthrosis is more common in hemophilia than in platelet disorders and has potential morbidity and quality-of-life implications.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"14 ","pages":"563-567"},"PeriodicalIF":2.0,"publicationDate":"2023-11-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10629506/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71521574","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 and Associated Factors of Anemia Among Hospital Admitted Patients in Eastern Ethiopia","authors":"Mohammed Umer Yusuf, Nuredin Abdurahman, Haftu Asmerom, Tesfaye Atsbaha, Adisu Alemu, Fitsum Weldegebreal","doi":"10.2147/jbm.s431047","DOIUrl":"https://doi.org/10.2147/jbm.s431047","url":null,"abstract":"Background: Anemia is one of the most common comorbidities frequently seen in admitted patients. However, there is a scarcity of evidence regarding anemia among hospital admitted patients in Ethiopia, particularly in the Harari Region. Therefore, this study aimed to assess the prevalence and associated factors of anemia among hospital admitted patients in Eastern Ethiopia. Methods: A hospital-based cross-sectional study was conducted from October 25 to December 30, 2022. Four milliliters of venous blood were collected and complete blood count was done using the DxH 800 (Beckman Coulter, Inc, Miami, FL) hematology analyzer. The data were entered in Epi-data version 4 and exported to SPSS version 26 for statistical analysis. Bivariable and multivariable logistic regression models were fitted. The level of significance was declared at a p-value of < 0.05. Results: Of the 381 hospital admitted patients, 64.8% (95% CI = 60.01, 69.65) of the participants were anemic. Admitted patients who drank standard alcohol daily (AOR = 3.78, 95% CI = 1.71, 8.30), underweight (AOR = 9.39, 95% CI = 2.90, 30.46), and undernourished patients (AOR = 2.59, 95% CI = 1.15, 5.84), patients admitted with chronic kidney disease (AOR = 11.16, 95% CI = 4.06, 30.64), chronic liver disease (AOR = 3.20, 95% CI = 1.21, 8.47), deep vein thrombosis (AOR = 6.22, 95% CI = 1.98, 19.52), infectious disease (AOR = 9.71, 95% CI = 2.77, 34.02), and chronic non-communicable disease (AOR = 7.01, 95% CI = 1.90, 25.99) were all significantly associated with anemia. Conclusion: Anemia was common among hospital admitted patients and should prompt the focus on admission diagnoses that are likely to play leading roles in etiology. This information indicates a need for routine screening of anemia for all admitted patients to improve their health. Keywords: anemia, prevalence, associated factors, admitted patients, Ethiopia","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"5 4","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135765131","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"The Correlation Between sP-Selectin and Platelet Count in COVID-19 Patients in Referral Hospital, West Java Indonesia.","authors":"Delita Prihatni, Frany Charisma Budianto, Basti Andriyoko, Suryarini Trisa","doi":"10.2147/JBM.S425667","DOIUrl":"https://doi.org/10.2147/JBM.S425667","url":null,"abstract":"<p><strong>Introduction: </strong>sP-selectin is a glycoprotein located in α granules of platelet and endothelial's Weibel Palade body, as expression to platelet activation and endothelial cell stimulation by SARS-CoV-2 binding with ACE2 receptor. Consumptive thrombocytopenia is also related to platelet activation. Elevation of sP-selectin and thrombocytopenia are related to COVID-19 complication and often correlated with severity of COVID-19.</p><p><strong>Purpose: </strong>Assess the correlation between sP-selectin and platelet in COVID-19 patients at intensive care and non-intensive care.</p><p><strong>Patients and methods: </strong>The study population was hospitalized COVID-19 patients confirmed by Real-Time PCR that underwent platelet examination within 48 hours upon admission, divided into intensive care and non-intensive care group. sP-selectin examination using ELISA methods. Platelet cell count and sP-selectin divided based on normal reference range.</p><p><strong>Results: </strong>The subjects consist of 24 were in intensive care, 25 were in non-intensive care group. A 66.7% of subject in intensive care group has an elevation in sP-selection (>44.0 ng/mL). Thrombocytopenia was significantly correlated with intensive group (r =-0.32, p<0.05). The combination of platelet count <150.000/mm<sup>3</sup> and sP-selectin >44.0ng/mL was not correlated with the intensive and non-intensive group. Platelet and sP-selectin were not correlated with each other.</p><p><strong>Conclusion: </strong>Thrombocytopenia is able to induce the expression of sP-selectin.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"14 ","pages":"555-561"},"PeriodicalIF":2.0,"publicationDate":"2023-10-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10617529/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71423912","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}