Clinical Hematology International最新文献

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Emicizumab Associated Rhabdomyolysis in Hemophilia A. 血友病A的Emicizumab相关横纹肌溶解。
Clinical Hematology International Pub Date : 2020-10-07 eCollection Date: 2020-12-01 DOI: 10.2991/chi.k.200924.001
Joseph A Wilson, Stephanie Hayden, Alexander Asamoah, Vivek R Sharma, David C Jennings, Ashok B Raj
{"title":"Emicizumab Associated Rhabdomyolysis in Hemophilia A.","authors":"Joseph A Wilson,&nbsp;Stephanie Hayden,&nbsp;Alexander Asamoah,&nbsp;Vivek R Sharma,&nbsp;David C Jennings,&nbsp;Ashok B Raj","doi":"10.2991/chi.k.200924.001","DOIUrl":"https://doi.org/10.2991/chi.k.200924.001","url":null,"abstract":"<p><p>Emicizumab is increasingly the front-line treatment for patients with Hemophilia A with or without inhibitors. Rhabdomyolysis is a syndrome of muscle necrosis and release of intracellular muscle constituents into the circulation. Creatine kinase (CK) levels are typically markedly elevated, and muscle pain and myoglobinuria may be present. The severity of illness ranges from asymptomatic elevations in serum muscle enzymes to life-threatening disease associated with extreme enzyme elevations, electrolyte imbalances, acute kidney injury and disseminated intravascular coagulation. We present a case of an African American male with severe hemophilia A and history of factor VIII inhibitor, maintained on emicizumab prophylaxis, who developed rhabdomyolysis with a symptomatic hyperCKemia. To date, there is no known link between rhabdomyolysis to emicizumab. This report brings to light the possibility of symptomatic rhabdomyolysis as a potential side effect of emicizumab after moderate exertional activity.</p>","PeriodicalId":10368,"journal":{"name":"Clinical Hematology International","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2020-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/5d/81/CHI-2-4-165.PMC8432407.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39476340","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
COVID-19 in Hematologic Malignancies: Big Challenges. COVID-19在血液恶性肿瘤中的应用:重大挑战。
Clinical Hematology International Pub Date : 2020-10-07 eCollection Date: 2020-12-01 DOI: 10.2991/chi.k.200919.001
Yongxian Hu, Linqin Wang, Qu Cui, Elaine Tan Su Yin, Hengwei Wu, Arnon Nagler, He Huang
{"title":"COVID-19 in Hematologic Malignancies: Big Challenges.","authors":"Yongxian Hu,&nbsp;Linqin Wang,&nbsp;Qu Cui,&nbsp;Elaine Tan Su Yin,&nbsp;Hengwei Wu,&nbsp;Arnon Nagler,&nbsp;He Huang","doi":"10.2991/chi.k.200919.001","DOIUrl":"https://doi.org/10.2991/chi.k.200919.001","url":null,"abstract":"1Bone Marrow Transplantation Center, The First Affiliated Hospital, School of Medicine, Zhejiang University, Hangzhou, China 2Zhejiang Province Engineering Laboratory for Stem Cell and Immunity Therapy, Hangzhou, China 3Institute of Hematology, Zhejiang University, Hangzhou, China 4Department of Hematology, Beijing Tiantan Hospital, Capital Medical University, Beijing, China 5Hematology and Bone Marrow Transplantation Division, Chaim Sheba Medical Center, Tel-Hashomer, Israel","PeriodicalId":10368,"journal":{"name":"Clinical Hematology International","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2020-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/e8/cd/CHI-2-4-173.PMC8432404.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39475190","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
B cell deficiency in patients with relapsed and refractory acute myeloid leukemia. 复发和难治性急性髓性白血病患者的B细胞缺乏症。
Clinical Hematology International Pub Date : 2020-09-01 Epub Date: 2020-07-29 DOI: 10.2991/chi.k.200712.001
Meghali Goswami, Katherine E Lindblad, Rahul Ramraj, Pradeep K Dagur, Julie Thompson, J Philip McCoy, Christopher S Hourigan
{"title":"B cell deficiency in patients with relapsed and refractory acute myeloid leukemia.","authors":"Meghali Goswami,&nbsp;Katherine E Lindblad,&nbsp;Rahul Ramraj,&nbsp;Pradeep K Dagur,&nbsp;Julie Thompson,&nbsp;J Philip McCoy,&nbsp;Christopher S Hourigan","doi":"10.2991/chi.k.200712.001","DOIUrl":"https://doi.org/10.2991/chi.k.200712.001","url":null,"abstract":"Immune checkpoint inhibition for the treatment of acute myeloid leukemia (AML) has been investigated clinically but has not yet been shown to be more effective than the standard of care treatment [1]. Tumor-specific autologous T cells expressing inhibitory receptors (iRs) are an inherent foundation to this immunotherapy; thus, a detailed picture of cells infiltrating the tumor microenvironment in AML, i.e., the bone marrow (BM), is warranted [2,3]. In this study, we characterized the frequencies and immunophenotypes of adaptive immune cells in the BM of relapsed or refractory AML (R-AML) patients in tandem with age-matched healthy donors (HD) for better contextualization of immunophenotypic variation in both cohorts. We performed deep immunological profiling using multi-parameter flow cytometry of BM of 10 R-AML and 20 age-matched HD, and considered the frequency, composition and expression of clinically relevant iRs across T and B cell subsets in HD and R-AML. Our group previously assessed the BM of this same HD cohort using single-cell RNA sequencing, mass cytometry and flow cytometry to cross-validate these technological approaches and to develop a reference data set for normal immune cell variation in healthy BM [4].","PeriodicalId":10368,"journal":{"name":"Clinical Hematology International","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2020-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/b4/01/CHI-2-3-125.PMC7485901.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38472637","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: 2
The Incidence and Timing of Blood Cultures in Multiple Myeloma - Results from a Retrospective, Single Center, Real-World Study. 多发性骨髓瘤患者血液培养的发生率和时机--一项回顾性、单中心、真实世界研究的结果。
Clinical Hematology International Pub Date : 2020-08-10 eCollection Date: 2020-12-01 DOI: 10.2991/chi.k.200719.001
Agoston Gyula Szabo, Katrine Fladeland Iversen, Flemming Schønning Rosenvinge, Sören Möller, Torben Plesner
{"title":"The Incidence and Timing of Blood Cultures in Multiple Myeloma - Results from a Retrospective, Single Center, Real-World Study.","authors":"Agoston Gyula Szabo, Katrine Fladeland Iversen, Flemming Schønning Rosenvinge, Sören Möller, Torben Plesner","doi":"10.2991/chi.k.200719.001","DOIUrl":"10.2991/chi.k.200719.001","url":null,"abstract":"","PeriodicalId":10368,"journal":{"name":"Clinical Hematology International","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2020-08-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/e5/63/CHI-2-4-168.PMC8432406.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39475189","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
What's Important and New in Hemochromatosis? 血色素沉着病有哪些重要的新发现?
Clinical Hematology International Pub Date : 2020-08-05 eCollection Date: 2020-12-01 DOI: 10.2991/chi.k.200726.001
Pierre Brissot, Eolia Brissot
{"title":"What's Important and New in Hemochromatosis?","authors":"Pierre Brissot,&nbsp;Eolia Brissot","doi":"10.2991/chi.k.200726.001","DOIUrl":"https://doi.org/10.2991/chi.k.200726.001","url":null,"abstract":"<p><p>Major advances in the understanding of genetic iron overload have led to a clarification of the nosology and terminology of the related diseases. The term hemochromatosis should be reserved to the entities where iron overload is related to hepcidin deficiency or hepcidin resistance. The diagnosis of hemochromatosis is non-invasive, based on clinical examination, blood investigations and, whenever possible, magnetic resonance imaging. Phlebotomies remain the mainstay of the treatment, but new therapeutic approaches should, in the future, constitute a valuable advance, hopefully both as an adjunct to bleeding in the induction phase and as its replacement in the maintenance phase. The goal of the present review is to update the terminology of hemochromatosis in light of major pathophysiological advances, and the main features of its diagnostic and therapeutic approaches.</p>","PeriodicalId":10368,"journal":{"name":"Clinical Hematology International","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2020-08-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/b5/0e/CHI-2-4-143.PMC8432403.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39476338","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: 5
Sorafenib Induced Complete Cytogenetic and Molecular Response in a Chronic Eosinophilic Leukemia Case with t(12;13) Translocation. 索拉非尼诱导慢性嗜酸性粒细胞白血病t(12;13)易位的完全细胞遗传学和分子反应。
Clinical Hematology International Pub Date : 2020-07-27 eCollection Date: 2020-09-01 DOI: 10.2991/chi.k.200714.001
Federica Ricci, Serena Balducci, Francesca Guerrini, Susanna Grassi, Elena Ciabatti, Claudia Baratè, Maria Immacolata Ferreri, Cecilia Giuliani, Angelo Valetto, Mario Petrini, Sara Galimberti
{"title":"Sorafenib Induced Complete Cytogenetic and Molecular Response in a Chronic Eosinophilic Leukemia Case with t(12;13) Translocation.","authors":"Federica Ricci,&nbsp;Serena Balducci,&nbsp;Francesca Guerrini,&nbsp;Susanna Grassi,&nbsp;Elena Ciabatti,&nbsp;Claudia Baratè,&nbsp;Maria Immacolata Ferreri,&nbsp;Cecilia Giuliani,&nbsp;Angelo Valetto,&nbsp;Mario Petrini,&nbsp;Sara Galimberti","doi":"10.2991/chi.k.200714.001","DOIUrl":"https://doi.org/10.2991/chi.k.200714.001","url":null,"abstract":"Hypereosinophilia (HE) is defined as a persistent increase in absolute eosinophil counts (AEC) to levels >1.5 × 109/L. HE can be ‘primary’ or ‘secondary’, more often accompanying infections, allergic disorders, autoimmune diseases or myeloproliferative/lymphoproliferative neoplasms [1,2]. In 2016, the WHO revised the classification of eosinophilic disorders, distinguishing myeloid/lymphoid neoplasms with eosinophilia and recurrent genetic lesions (such as rearrangement of PDGFRA, PDGFRB, FGFR1 and PCM1-JAK2), chronic eosinophilic leukemia (CEL) not otherwise specified (NOS), and idiopathic HE [3,4].","PeriodicalId":10368,"journal":{"name":"Clinical Hematology International","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2020-07-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/e8/e4/CHI-2-3-129.PMC8432331.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39476336","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
Plasma Cell Leukemia - Facts and Controversies: More Questions than Answers? 浆细胞白血病-事实与争议:问题多于答案?
Clinical Hematology International Pub Date : 2020-07-19 eCollection Date: 2020-12-01 DOI: 10.2991/chi.k.200706.002
Anna Suska, David H Vesole, Jorge J Castillo, Shaji K Kumar, Hari Parameswaran, Maria V Mateos, Thierry Facon, Alessandro Gozzetti, Gabor Mikala, Marta Szostek, Joseph Mikhael, Roman Hajek, Evangelos Terpos, Artur Jurczyszyn
{"title":"Plasma Cell Leukemia - Facts and Controversies: More Questions than Answers?","authors":"Anna Suska,&nbsp;David H Vesole,&nbsp;Jorge J Castillo,&nbsp;Shaji K Kumar,&nbsp;Hari Parameswaran,&nbsp;Maria V Mateos,&nbsp;Thierry Facon,&nbsp;Alessandro Gozzetti,&nbsp;Gabor Mikala,&nbsp;Marta Szostek,&nbsp;Joseph Mikhael,&nbsp;Roman Hajek,&nbsp;Evangelos Terpos,&nbsp;Artur Jurczyszyn","doi":"10.2991/chi.k.200706.002","DOIUrl":"https://doi.org/10.2991/chi.k.200706.002","url":null,"abstract":"<p><p>Plasma cell leukemia (PCL) is an aggressive hematological malignancy characterized by an uncontrolled clonal proliferation of plasma cells (PCs) in the bone marrow and peripheral blood. PCL has been defined by an absolute number of circulating PCs exceeding 2.0 × 10<sup>9</sup>/L and/or >20% PCs in the total leucocyte count. It is classified as primary PCL, which develops <i>de novo</i>, and secondary PCL, occurring at the late and advanced stages of multiple myeloma (MM). Primary and secondary PCL are clinically and biologically two distinct entities. After the diagnosis, treatment should be immediate and should include a proteasome inhibitor and immunomodulator-based combination regimens as induction, followed by stem cell transplantation (SCT) in transplant-eligible individuals who have cleared the peripheral blood of circulating PCs. Due to the rarity of the condition, there have been very few clinical trials. Furthermore, virtually all of the myeloma trials exclude patients with active PCL. The evaluation of response has been defined by the International Myeloma Working Group and consists of both acute leukemia and MM criteria. With conventional chemotherapy, the prognosis of primary PCL has been ominous, with reported overall survival (OS) ranging from 6.8 to 12.6 months. The use of novel agents and autologous SCT appears to be associated with deeper response and an improved survival, although it still remains low. The PCL prognostic index provides a simple score to risk-stratify PCL. The prognosis of secondary PCL is extremely poor, with OS of only 1 month.</p>","PeriodicalId":10368,"journal":{"name":"Clinical Hematology International","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2020-07-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/98/e3/CHI-2-4-133.PMC8432408.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39476337","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: 5
Cold Agglutinin Disease. 感冒凝集素病。
Clinical Hematology International Pub Date : 2020-07-17 eCollection Date: 2020-09-01 DOI: 10.2991/chi.k.200706.001
Amy P Gabbard, Garrett S Booth
{"title":"Cold Agglutinin Disease.","authors":"Amy P Gabbard,&nbsp;Garrett S Booth","doi":"10.2991/chi.k.200706.001","DOIUrl":"https://doi.org/10.2991/chi.k.200706.001","url":null,"abstract":"<p><p>Cold agglutinin disease (CAD) is an uncommon form of cold autoimmune hemolytic anemia (AIHA). It should be considered in the differential diagnosis of elderly patients with unexplained chronic anemia presenting with or without cold-induced symptoms in the extremities, such as the fingers, ears, and nose. CAD is a complement-mediated process which leads to intravascular and extravascular hemolysis. A stepwise approach to laboratory testing can help confirm the diagnosis. Nearly all cold agglutinins are positive for the C3d direct antiglobulin test (DAT). A negative C3d DAT should prompt investigation of a possible warm AIHA. Ninety percent of cold agglutinins are of the IgM immunoglobulin class and should have a titer of 1:64 or higher at 4°C. Distinction from a warm AIHA is important, as therapy differs for the two entities. Corticosteroids are not effective at treating CAD and should not be used as therapy in these patients. Approximately 45-60% of patients with CAD respond to rituximab monotherapy. Combination therapy of rituximab and fludarabine has been shown to be effective in up to 76% of patients; however, patients experience more mild side effects with this treatment. New anti-complement drugs, such as eculizumab and sutimlimab, are currently in phase-3 trials to determine their efficacy and safety in patients with CAD.</p>","PeriodicalId":10368,"journal":{"name":"Clinical Hematology International","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2020-07-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/ce/87/CHI-2-3-95.PMC8432332.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39477349","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: 112
Framingham Risk Score Is an Ineffective Screening Strategy for Coronary Heart Disease in Long-Term Allogeneic Hematopoietic Cell Transplant Survivors. 弗雷明汉风险评分对长期同种异体造血细胞移植幸存者冠心病筛查无效。
Clinical Hematology International Pub Date : 2020-06-12 eCollection Date: 2020-09-01 DOI: 10.2991/chi.d.200508.001
Natasha A Jain, Marcus Y Chen, Sujata Shanbhag, Prathima Anandi, Xin Tian, Sawa Ito, Priyanka A Pophali, Kimberly Doucette, Robert Q Le, Upneet Chawla, Eleftheria Koklanaris, Richard W Childs, A John Barrett, Minoo Battiwalla
{"title":"Framingham Risk Score Is an Ineffective Screening Strategy for Coronary Heart Disease in Long-Term Allogeneic Hematopoietic Cell Transplant Survivors.","authors":"Natasha A Jain,&nbsp;Marcus Y Chen,&nbsp;Sujata Shanbhag,&nbsp;Prathima Anandi,&nbsp;Xin Tian,&nbsp;Sawa Ito,&nbsp;Priyanka A Pophali,&nbsp;Kimberly Doucette,&nbsp;Robert Q Le,&nbsp;Upneet Chawla,&nbsp;Eleftheria Koklanaris,&nbsp;Richard W Childs,&nbsp;A John Barrett,&nbsp;Minoo Battiwalla","doi":"10.2991/chi.d.200508.001","DOIUrl":"https://doi.org/10.2991/chi.d.200508.001","url":null,"abstract":"<p><p>Long-term allogeneic hematopoietic cell transplant (allo-HCT) survivors suffer an elevated risk of coronary heart disease (CHD). We conducted a prospective, nonrandomized, cross-sectional study to screen asymptomatic survivors at a single allo-HCT center using cardiac computed tomography (CT) involving coronary CT angiography (CCTA) and the coronary artery calcium (CAC) score. Seventy-nine subjects with a median age of 39 years at allo-HCT and a median follow-up interval of 8 years were evaluated for CHD by Framingham Risk Score (FRS) and cardiac CT. CHD was detected in 33 of 79 (42%) subjects; 91% of lesions were nonobstructive, 19.5% of were noncalcified and 30% had associated valvular calcification. Overall, CAC was significantly superior to FRS in detecting early CHD in allo-HCT survivors [∆C = 0.25; <i>P</i> < 0.0001]. While both FRS and CAC were highly, >95% specific, FRS had a sensitivity, positive and negative predictive values of only 28% (95% CI, 14%-47%), 90% (95% CI, 55%-100%) and 60% (95% CI, 47%-73%), respectively. In contrast, the sensitivity, positive and negative predictive values of CAC were 78% (95% CI, 60%-91%), 96% (95% CI, 80%-100%) and 83% (95% CI, 69%-93%), respectively. Significantly, cardiac CT detected CHD in 23 of the 68 (34%) survivors deemed to have a low Framingham risk. Radiation exposure during cardiac CT was negligible, and there were no adverse events. In conclusion, CAC score with or without CCTA is a safe, feasible and sensitive screening technique for CHD. The FRS greatly underestimates CHD in allo-HCT survivors.</p>","PeriodicalId":10368,"journal":{"name":"Clinical Hematology International","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2020-06-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/1b/2c/CHI-2-3-109.PMC8432330.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39476334","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: 2
The Evolving Landscape of Myelodysplastic Syndrome Prognostication. 骨髓增生异常综合征预后的发展前景。
Clinical Hematology International Pub Date : 2020-06-01 Epub Date: 2020-04-19 DOI: 10.2991/chi.d.200408.001
Jacob Shreve, Aziz Nazha
{"title":"The Evolving Landscape of Myelodysplastic Syndrome Prognostication.","authors":"Jacob Shreve,&nbsp;Aziz Nazha","doi":"10.2991/chi.d.200408.001","DOIUrl":"10.2991/chi.d.200408.001","url":null,"abstract":"<p><p>Myelodysplastic syndromes (MDSs) are potentially devastating monoclonal deviations of hematopoiesis that lead to bone marrow dysplasia and variable cytopenias. Predicting severity of disease progression and likelihood to undergo acute myeloid leukemia transformation is the basis of treatment strategy. Some patients belong to a low-risk cohort best managed with conservative supportive care, whereas others are included in a high-risk cohort that requires decisive therapy with hematopoietic cell transplantation or hypomethylating agent administration. Risk scoring systems for MDS prognostication were traditionally based on karyotype characteristics and clinical factors readily available from chart review, and validation was typically conducted on de novo MDS patients. However, retrospective analysis found a large subset of patients incorrectly risk-stratified. In this review, the most commonly used scoring systems are evaluated, and pitfalls therein are identified. Emerging technologies such as personal genomics and machine learning are then explored for efficacy in MDS risk modeling. Barriers to clinical adoption of artificial intelligence-derived models are discussed, with focus on approaches meant to increase model interpretability and clinical relevance. Finally, a guiding set of recommendations is proposed for best designing an accurate and universally applicable prognostic model for MDS, which is supported by more than 20 years of observation of traditional scoring system performance, as well as modern efforts in creating hybrid genomic-clinical scoring systems.</p>","PeriodicalId":10368,"journal":{"name":"Clinical Hematology International","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2020-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/6e/f2/CHI-2-2-43.PMC7462414.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38340066","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: 5
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