Clinical Medicine Insights-Blood Disorders最新文献

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Management of Primary Plasma Cell Leukemia Remains Challenging Even in the Era of Novel Agents. 即使在新药物的时代,原发性浆细胞白血病的治疗仍然具有挑战性。
IF 3
Clinical Medicine Insights-Blood Disorders Pub Date : 2021-02-26 eCollection Date: 2021-01-01 DOI: 10.1177/2634853521999389
Chakra P Chaulagain, Maria-Julia Diacovo, Amy Van, Felipe Martinez, Chieh-Lin Fu, Antonio Martin Jimenez Jimenez, Wesam Ahmed, Faiz Anwer
{"title":"Management of Primary Plasma Cell Leukemia Remains Challenging Even in the Era of Novel Agents.","authors":"Chakra P Chaulagain,&nbsp;Maria-Julia Diacovo,&nbsp;Amy Van,&nbsp;Felipe Martinez,&nbsp;Chieh-Lin Fu,&nbsp;Antonio Martin Jimenez Jimenez,&nbsp;Wesam Ahmed,&nbsp;Faiz Anwer","doi":"10.1177/2634853521999389","DOIUrl":"https://doi.org/10.1177/2634853521999389","url":null,"abstract":"<p><p>Primary plasma cell leukemia (PCL) is a rare and aggressive variant of multiple myeloma (MM). PCL is characterized by peripheral blood involvement by malignant plasma cells and an aggressive clinical course leading to poor survival. There is considerable overlap between MM and PCL with respect to clinical, immunophenotypic, and cytogenetic features, but circulating plasma cell count exceeding 20% of peripheral blood leukocytes or an absolute plasma cell count of >2000/mm<sup>3</sup> distinguishes it from MM. After initial stabilization and diagnosis confirmation, treatment of PCL in a fit patient typically includes induction combination chemotherapy containing novel agents typically, with proteasome inhibitors (such as bortezomib) and immunomodulatory drugs (eg, lenalidomide), followed by autologous hematopoietic stem cell transplant (HSCT) and multidrug maintenance therapy using novel agents post-HSCT. Long-term outcomes have improved employing this strategy but the prognosis for non-HSCT candidates remains poor and new approaches are needed for such PCL patients not eligible for HSCT. Here, we report a case of primary PCL, and a comprehensive and up to date review of the literature for diagnosis and management of PCL. We also present the findings of Positron Emission Tomography (PET) scan. Since PCL is often associated with extra-medulary disease, including PET scan at the time of staging and restaging may be a novel approach particularly to evaluate the extra-medullary disease sites.</p>","PeriodicalId":43083,"journal":{"name":"Clinical Medicine Insights-Blood Disorders","volume":"14 ","pages":"2634853521999389"},"PeriodicalIF":3.0,"publicationDate":"2021-02-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1177/2634853521999389","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"25476004","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}
引用次数: 4
Efficacy of Nuwiq® (Simoctocog Alfa) in Patients with Hemophilia A Who Changed and Adhered to a Pharmacokinetic-Guided Prophylaxis Regimen in the NuPreviq Study. 在NuPreviq研究中,Nuwiq®(simotocog Alfa)在改变并坚持药代动力学指导预防方案的A型血友病患者中的疗效
IF 3
Clinical Medicine Insights-Blood Disorders Pub Date : 2021-02-26 eCollection Date: 2021-01-01 DOI: 10.1177/2634853521991517
K John Pasi
{"title":"Efficacy of Nuwiq<sup>®</sup> (Simoctocog Alfa) in Patients with Hemophilia A Who Changed and Adhered to a Pharmacokinetic-Guided Prophylaxis Regimen in the NuPreviq Study.","authors":"K John Pasi","doi":"10.1177/2634853521991517","DOIUrl":"https://doi.org/10.1177/2634853521991517","url":null,"abstract":"","PeriodicalId":43083,"journal":{"name":"Clinical Medicine Insights-Blood Disorders","volume":"14 ","pages":"2634853521991517"},"PeriodicalIF":3.0,"publicationDate":"2021-02-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1177/2634853521991517","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"25476080","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}
引用次数: 1
A Clinical Triad with Fatal Implications: Recrudescent Diffuse Large B-cell Non-Hodgkin Lymphoma Presenting in the Leukemic Phase with an Elevated Serum Lactic Acid Level and Dysregulation of the TP53 Tumor Suppressor Gene - A Case Report and Literature Review. 具有致命意义的临床三位一体:复发性弥漫性大b细胞非霍奇金淋巴瘤在白血病期表现为血清乳酸水平升高和肿瘤抑制基因TP53失调-一例报告和文献综述
IF 3
Clinical Medicine Insights-Blood Disorders Pub Date : 2021-02-19 eCollection Date: 2021-01-01 DOI: 10.1177/2634853521994094
Catherine S Hwang, Dick G Hwang, David M Aboulafia
{"title":"A Clinical Triad with Fatal Implications: Recrudescent Diffuse Large B-cell Non-Hodgkin Lymphoma Presenting in the Leukemic Phase with an Elevated Serum Lactic Acid Level and Dysregulation of the TP53 Tumor Suppressor Gene - A Case Report and Literature Review.","authors":"Catherine S Hwang,&nbsp;Dick G Hwang,&nbsp;David M Aboulafia","doi":"10.1177/2634853521994094","DOIUrl":"https://doi.org/10.1177/2634853521994094","url":null,"abstract":"<p><p>Despite representing 30% to 40% of newly diagnosed cases of adult non-Hodgkin lymphoma, diffuse large B-cell lymphoma (DLBCL) rarely presents (1) in the leukemic phase (2) with dysregulation of the TP53 tumor suppressor gene and (3) an elevated serum lactic acid level. In this case report and literature review, we highlight this unfortunate triad of poor prognostic features associated with an aggressive and fatal clinical course in a 53-year-old man with recrudescent DLBCL. A leukemic presentation of de novo or relapsed DLBCL is rare and may be related to differential expressions of adhesion molecules on cell surfaces. In addition, TP53 gene mutations are present in approximately 20% to 25% of DLBCL cases and foreshadow worse clinical outcomes. Finally, an elevated serum lactic acid level in DLBCL that is not clearly associated with sepsis syndrome is a poor prognostic factor for survival and manifests as type B lactic acidosis through the Warburg effect.</p>","PeriodicalId":43083,"journal":{"name":"Clinical Medicine Insights-Blood Disorders","volume":"14 ","pages":"2634853521994094"},"PeriodicalIF":3.0,"publicationDate":"2021-02-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1177/2634853521994094","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"25443684","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}
引用次数: 3
Occurrence of a Clonal T-Cell Population in a Case of Chronic Myelomonocytic Leukemia. 慢性髓单细胞白血病一例克隆t细胞群的发生。
IF 3
Clinical Medicine Insights-Blood Disorders Pub Date : 2021-02-12 eCollection Date: 2021-01-01 DOI: 10.1177/2634853521991509
Anupama Patil, Balasaheb Wanve, Pradeep Kar, Shanthi Velusamy
{"title":"Occurrence of a Clonal T-Cell Population in a Case of Chronic Myelomonocytic Leukemia.","authors":"Anupama Patil,&nbsp;Balasaheb Wanve,&nbsp;Pradeep Kar,&nbsp;Shanthi Velusamy","doi":"10.1177/2634853521991509","DOIUrl":"https://doi.org/10.1177/2634853521991509","url":null,"abstract":"<p><p>Chronic myelo-monocytic leukemia (CMML) is an aggressive myeloid neoplasm with some features of a myelodysplastic syndrome (MDS) and others of a myeloproliferative neoplasm (MPN). Rarely, patients with CMML have a co-existing lympho-proliferative disorder (LPD). In most cases, the lymphoid neoplasm is diagnosed first, and the CMML is considered to be a secondary therapy-induced form of leukemia. We report herein a unique case of de-novo CMML, with an underlying clonal T-cell population and describe its clinical presentation and laboratory findings. A 70-year old male presented with a 3-month history of cough, dsypnea, abdominal distension, and low-grade fever. Physical and radiological examination revealed hepatosplenomegaly but no lymphadenopathy. Peripheral blood had absolute monocytosis with marrow showing CMML with 10% blasts along with dysplasia in myeloid and erythroid lineages. Flow cytometry indicated possibility of chronic myelo-monocytic leukemia with 13% monocytic cells along with an additional clonal population of gamma/delta T cells (15%) with aberrant immunophenotype. Polymerase chain reaction (PCR) analysis was positive for clonal T-cell rearrangement. A diagnosis of CMML with an underlying clonal T-CLPD was made. The synchronous occurrence of CMML and T-cell neoplasm may be attributed to a genetic mutation common to both. Currently, there are no treatment guidelines for group of patients; hence individualized therapeutic strategies should be implemented to enable symptomatic improvement and provide optimum care.</p>","PeriodicalId":43083,"journal":{"name":"Clinical Medicine Insights-Blood Disorders","volume":"14 ","pages":"2634853521991509"},"PeriodicalIF":3.0,"publicationDate":"2021-02-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1177/2634853521991509","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"25402396","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}
引用次数: 0
Factors Involved in the Development of Inhibitory Antibodies in Patients with Hemophilia in Colombia: A Case-Control Study. 哥伦比亚血友病患者抑制抗体产生的相关因素:一项病例对照研究
IF 3
Clinical Medicine Insights-Blood Disorders Pub Date : 2020-10-06 eCollection Date: 2020-01-01 DOI: 10.1177/2634853520962467
Jorge E Machado-Alba, Laura A Chica-Quintero, Manuel E Machado-Duque, Andrés Gaviria-Mendoza, Juan David Wilches-Gutierrez, Diana Rocio Arias-Osorio
{"title":"Factors Involved in the Development of Inhibitory Antibodies in Patients with Hemophilia in Colombia: A Case-Control Study.","authors":"Jorge E Machado-Alba,&nbsp;Laura A Chica-Quintero,&nbsp;Manuel E Machado-Duque,&nbsp;Andrés Gaviria-Mendoza,&nbsp;Juan David Wilches-Gutierrez,&nbsp;Diana Rocio Arias-Osorio","doi":"10.1177/2634853520962467","DOIUrl":"https://doi.org/10.1177/2634853520962467","url":null,"abstract":"<p><strong>Background: </strong>The appearance of inhibitory antibodies against antihemophilic factors is one of the most serious complications related to hemophilia.</p><p><strong>Objective: </strong>The objective of this study was to identify variables and factors related to the development of inhibitory antibodies in a group of patients undergoing antihemophilic therapy in Colombia.</p><p><strong>Methods: </strong>A case-control study in patients with hemophilia treated in Specialized Healthcare Provider Institutions (IPS-E) in 21 cities of Colombia of any age and with a diagnosis of inhibitory antibodies against factor VIII or IX during 2016. Four controls per case paired by age and type of hemophilia were used. Sociodemographic, clinical, and pharmacological variables were identified and analyzed.</p><p><strong>Results: </strong>Seventeen patients with inhibitory antibodies and 68 controls with hemophilia were identified. The mean age was 28.3 ± 17.8 years. A total of 94.1% had hemophilia A, and 88.2% of the cases and 50.0% of the controls had severe hemophilia; 47.1% of the cases and 54.4% of the controls were receiving prophylaxis with coagulation factors. Multivariate analysis showed that having severe hemophilia (OR:17.0, 95%CI:1.32-219.60) and lack of knowledge of the coagulation factor with which the patient was treated before entering the care program in the IPS-E (OR:8.9, 95%CI:1.82-43.75) were significantly associated with a higher probability of developing inhibitory antibodies.</p><p><strong>Conclusion and relevance: </strong>Coagulation factors associated with the development of inhibitory antibodies were severe hemophilia and lack of knowledge of the type of factor used prior to entering the follow-up cohort.</p>","PeriodicalId":43083,"journal":{"name":"Clinical Medicine Insights-Blood Disorders","volume":" ","pages":"2634853520962467"},"PeriodicalIF":3.0,"publicationDate":"2020-10-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1177/2634853520962467","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38521512","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}
引用次数: 1
Rare Causes of Isolated and Progressive Splenic Lesions: Challenges in Differential Diagnosis, Evaluation, and Treatment of Primary Splenic Lymphomas. 孤立性和进行性脾病变的罕见病因:原发性脾淋巴瘤的鉴别诊断、评估和治疗难题。
IF 3
Clinical Medicine Insights-Blood Disorders Pub Date : 2020-06-10 eCollection Date: 2020-01-01 DOI: 10.1177/1179545X20926188
Ryan B Sinit, Russell K Dorer, John Paul Flores, David M Aboulafia
{"title":"Rare Causes of Isolated and Progressive Splenic Lesions: Challenges in Differential Diagnosis, Evaluation, and Treatment of Primary Splenic Lymphomas.","authors":"Ryan B Sinit, Russell K Dorer, John Paul Flores, David M Aboulafia","doi":"10.1177/1179545X20926188","DOIUrl":"10.1177/1179545X20926188","url":null,"abstract":"<p><p>The spleen is among the most common extranodal sites for Hodgkin and non-Hodgkin lymphomas (NHLs); however, among lymphomas arising from the spleen, primary splenic lymphomas (PSLs) are rare. The group of PSLs includes primary splenic diffuse large B-cell lymphoma (PS-DLBCL), splenic red pulp small B-cell lymphoma, splenic marginal zone lymphoma (SMZL), and a splenic hairy cell leukemia variant. Delineating between the PSL variants can be challenging, especially as fine-needle aspirate and core needle biopsy of the spleen are not routinely offered at most medical centers. Herein, we describe the clinical course of 2 representative patients who presented with non-specific gastrointestinal symptoms, the first who was diagnosed with PS-DLBCL and the second who was diagnosed with SMZL. We review and contrast the clinical presentations, imaging techniques, and laboratory findings of these discrete lymphoma variants and offer strategies on how to delineate between these varied splenic processes. We also examine the use of splenectomy and splenic needle biopsy as diagnostics and, in the case of splenectomy, a therapeutic tool. Finally, we also briefly review treatment options for these varied lymphoma sub-types while acknowledging that randomized trials to guide best practices for PSLs are lacking.</p>","PeriodicalId":43083,"journal":{"name":"Clinical Medicine Insights-Blood Disorders","volume":" ","pages":"1179545X20926188"},"PeriodicalIF":3.0,"publicationDate":"2020-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7288794/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38067751","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}
引用次数: 0
Corrigendum. 勘误表。
IF 3
Clinical Medicine Insights-Blood Disorders Pub Date : 2020-01-06 eCollection Date: 2020-01-01 DOI: 10.1177/1179545X19900859
{"title":"Corrigendum.","authors":"","doi":"10.1177/1179545X19900859","DOIUrl":"https://doi.org/10.1177/1179545X19900859","url":null,"abstract":"<p><p>[This corrects the article DOI: 10.1177/1179545X19875105.].</p>","PeriodicalId":43083,"journal":{"name":"Clinical Medicine Insights-Blood Disorders","volume":"13 ","pages":"1179545X19900859"},"PeriodicalIF":3.0,"publicationDate":"2020-01-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1177/1179545X19900859","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"37539820","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}
引用次数: 0
The Essential Thrombocythemia in 2020: What We Know and Where We Still Have to Dig Deep. 2020年的原发性血小板增多症:我们所知道的和我们还需要深入挖掘的地方。
IF 3
Clinical Medicine Insights-Blood Disorders Pub Date : 2020-01-01 DOI: 10.1177/2634853520978210
Vincenzo Accurso, Marco Santoro, Salvatrice Mancuso, Mariasanta Napolitano, Melania Carlisi, Marta Mattana, Chiara Russo, Alessandro Di Stefano, Davide Sirocchi, Sergio Siragusa
{"title":"The Essential Thrombocythemia in 2020: What We Know and Where We Still Have to Dig Deep.","authors":"Vincenzo Accurso,&nbsp;Marco Santoro,&nbsp;Salvatrice Mancuso,&nbsp;Mariasanta Napolitano,&nbsp;Melania Carlisi,&nbsp;Marta Mattana,&nbsp;Chiara Russo,&nbsp;Alessandro Di Stefano,&nbsp;Davide Sirocchi,&nbsp;Sergio Siragusa","doi":"10.1177/2634853520978210","DOIUrl":"https://doi.org/10.1177/2634853520978210","url":null,"abstract":"<p><p>The Essential Thrombocythemia is a Chronic Philadelphia-negative Myeloproliferative Neoplasm characterized by a survival curve that is only slightly worse than that of age- and sex-adjusted healthy population. The criteria for diagnosis were reviewed in 2016 by WHO. The incidence varies from 0.2 to 2.5:100 000 people per year, with a prevalence of 38 to 57 cases per 100 000 people. The main characteristics of ET are the marked thrombocytosis and the high frequency of thrombosis. The spectrum of symptoms is quite wide, but fatigue results to be the most frequent. Thrombosis is frequently observed, often occurring before or at the time of diagnosis. The classification of thrombotic risk has undergone several revisions. Recently, the revised-IPSET-t has distinguished 4 risk classes, from very low risk to high risk. Driver mutations seem to influence thrombotic risk and prognosis, while the role of sub-driver mutations still remains uncertain. Antiplatelet therapy is recommended in all patients aged ⩾ 60 years and in those with a positive history of thrombosis or with cardiovascular risk factors, while cytoreductive therapy with hydroxyurea or interferon is reserved for high-risk patients.</p>","PeriodicalId":43083,"journal":{"name":"Clinical Medicine Insights-Blood Disorders","volume":"13 ","pages":"2634853520978210"},"PeriodicalIF":3.0,"publicationDate":"2020-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1177/2634853520978210","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10283353","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}
引用次数: 22
Autoimmune Hemolytic Anemia After Relapse of Chronic Myeloid Leukemia: A Case Report. 慢性髓性白血病复发后自身免疫性溶血性贫血1例报告
IF 3
Clinical Medicine Insights-Blood Disorders Pub Date : 2019-12-23 eCollection Date: 2019-01-01 DOI: 10.1177/1179545X19894578
Tahseen Hamamyh, Mohamed A Yassin
{"title":"Autoimmune Hemolytic Anemia After Relapse of Chronic Myeloid Leukemia: A Case Report.","authors":"Tahseen Hamamyh,&nbsp;Mohamed A Yassin","doi":"10.1177/1179545X19894578","DOIUrl":"https://doi.org/10.1177/1179545X19894578","url":null,"abstract":"Autoimmune hemolytic anemia is one of the differential diagnoses for anemia in patients with lymphoproliferative neoplasia, such as chronic lymphocytic leukemia, who experience sudden drop in hemoglobin. The association between autoimmune hemolytic anemia and chronic myeloid leukemia on the contrary is unusual. Here we present a patient with a background of chronic myeloid leukemia treated previously with Tyrosine Kinase Inhibitors, then developed autoimmune hemolysis simultaneously with chronic myeloid leukemia relapse. Hemolysis was treated with steroids with good response.","PeriodicalId":43083,"journal":{"name":"Clinical Medicine Insights-Blood Disorders","volume":"12 ","pages":"1179545X19894578"},"PeriodicalIF":3.0,"publicationDate":"2019-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1177/1179545X19894578","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"37512934","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}
引用次数: 3
Current Advance in Thrombopoietin Receptor Agonists in the Management of Thrombocytopenia Associated With Chronic Liver Disease: Focus on Avatrombopag 血小板生成素受体激动剂治疗慢性肝病伴血小板减少症的最新进展:以阿伐曲波帕为重点
IF 3
Clinical Medicine Insights-Blood Disorders Pub Date : 2019-10-01 DOI: 10.1177/1179545X19875105
Jemal Abdela
{"title":"Current Advance in Thrombopoietin Receptor Agonists in the Management of Thrombocytopenia Associated With Chronic Liver Disease: Focus on Avatrombopag","authors":"Jemal Abdela","doi":"10.1177/1179545X19875105","DOIUrl":"https://doi.org/10.1177/1179545X19875105","url":null,"abstract":"Chronic liver disease (CLD) is a condition that progresses over time toward advanced disease state which is known as liver cirrhosis. Liver cirrhosis leads to dangerous health problems among people living across the world. One such problem that observed in about 75% of cirrhotic patients is thrombocytopenia; which in turn associated with poor prognosis and recovery from CLD. Beyond these, thrombocytopenia in cirrhotic patients led to impairment of coagulation cascade and significantly influenced the utilization of effective mechanism in the management of CLD. By nature, treatment of CLD involves invasive diagnostic and treatment procedures; therefore, in the presence of thrombocytopenia implementing these methods put the lives of patients in a critical health problem due to increased risk of bleeding and mortality. Because of these reasons, prophylactic transfusion of platelets is considered to be one of the most effective options that reduce the risk of bleeding in patients with CLD that required to undergo an invasive procedure. Although platelet transfusion presented with significant advantages in facilitating the invasive procedure in patients with CLD, refractoriness with repeated use and various problems associated with its transfusion limit the continuous utilization of this important option. With these challenges and current advance in the knowledge of thrombopoiesis, the development of relatively safe and alternative drugs that enhance the production of platelets by interacting with thrombopoietin receptor agonists provides a promising option to platelet transfusion. The discovery and approval of romiplostim and eltrombopag in August 2008 and November 2008, respectively, for the treatment of chronic immune thrombocytopenia paved a way and followed by the Food and Drug Administration (FDA) approval of 2 potentially advantageous drugs, lusutrombopag, and avatrombopag, in 2018 for the treatment of thrombocytopenia in patients with CLD that required to undergo elective surgery. Therefore, this review aims to assess pathogenesis of thrombocytopenia and its challenges in the management of liver-related issues and, more importantly, gives emphasis to address the potential use of avatrombopag in the treatment of thrombocytopenia underlying CLD, its pharmacokinetics and pharmacodynamics, as well as its toxicological profiles by presenting the most commonly reported adverse events in various trials.","PeriodicalId":43083,"journal":{"name":"Clinical Medicine Insights-Blood Disorders","volume":"12 1","pages":""},"PeriodicalIF":3.0,"publicationDate":"2019-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"82856925","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}
引用次数: 13
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