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The Role of 11C-Methionine PET Imaging for the Evaluation of Lymphomas: A Systematic Review. 11c -蛋氨酸PET成像在淋巴瘤评估中的作用:一项系统综述。
IF 1.1
Hematology Reports Pub Date : 2024-11-27 DOI: 10.3390/hematolrep16040072
Francesco Dondi, Maria Gazzilli, Gian Luca Viganò, Antonio Rosario Pisani, Cristina Ferrari, Giuseppe Rubini, Francesco Bertagna
{"title":"The Role of 11C-Methionine PET Imaging for the Evaluation of Lymphomas: A Systematic Review.","authors":"Francesco Dondi, Maria Gazzilli, Gian Luca Viganò, Antonio Rosario Pisani, Cristina Ferrari, Giuseppe Rubini, Francesco Bertagna","doi":"10.3390/hematolrep16040072","DOIUrl":"10.3390/hematolrep16040072","url":null,"abstract":"<p><p><b>Background</b>: In the last years, different evidence has underlined a possible role for [11C]-methionine ([11C]MET) positron emission tomography (PET) imaging for the evaluation of lymphomas. The aim of this paper was, therefore, to review the available scientific literature focusing on this topic. <b>Methods</b>: A wide literature search of the PubMed/MEDLINE, Scopus and Cochrane Library databases was conducted in order to find relevant published articles investigating the role of [11C]MET in the assessment of lymphomas. <b>Results</b>: Eighteen studies were included in the systematic review and the main fields of application of this imaging modality were the evaluation of disease, therapy response assessment, prognostic evaluation and differential diagnosis with other pathological conditions. <b>Conclusion</b>: Even with heterogeneous evidence, a possible role for [11C]MET PET imaging in the assessment of lymphomas affecting both the whole body and the central nervous system was underlined. When compared to [18F]fluorodesoxyglucose ([18F]FDG) imaging, in general, similar results have been reported between the two modalities in these settings.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"752-768"},"PeriodicalIF":1.1,"publicationDate":"2024-11-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11675220/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142893940","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
Serum Vitamin D in Children with Hemophilia A and Its Association with Joint Health and Quality of Life. 血友病A患儿血清维生素D及其与关节健康和生活质量的关系
IF 1.1
Hematology Reports Pub Date : 2024-11-26 DOI: 10.3390/hematolrep16040071
Aida M S Salem, Takwa Mohamed AbdEltwwab, Hanan Hosni Moawad, Marwa O Elgendy, Reham S Al-Fakharany, Ahmed Khames, Mohamed Hussein Meabed
{"title":"Serum Vitamin D in Children with Hemophilia A and Its Association with Joint Health and Quality of Life.","authors":"Aida M S Salem, Takwa Mohamed AbdEltwwab, Hanan Hosni Moawad, Marwa O Elgendy, Reham S Al-Fakharany, Ahmed Khames, Mohamed Hussein Meabed","doi":"10.3390/hematolrep16040071","DOIUrl":"10.3390/hematolrep16040071","url":null,"abstract":"<p><p><b>Background/Objectives</b>: Hemophilia A is an X-linked recessive illness produced by a deficiency of coagulation factor VIII. This study aimed to evaluate serum vitamin D in hemophilic pediatric patients and its correlation with joint health and quality of life. <b>Methods</b>: This case-control study was performed on ninety children under the age of 18 years old and separated into two groups: study group of 45 children with hemophilia A and control group of 45 healthy children at an outpatient pediatric hematology clinic at the Beni-Suef University hospitals. <b>Results</b>: Serum vitamin D levels were significantly lower in hemophilia A patients than in controls (<i>p</i> < 0.001). The level of serum vitamin D was deficient in 38 (84.4%), insufficient in 4 (8.8%) and sufficient in 3 (6.6%) in the study group while deficient in 8 (17.7%), insufficient in 16 (35.5%) and sufficient in 21 (46.6%) in the control group. Total hemophilia joint health score (HJHS) had a significant negative correlation with serum total calcium (R = -0.31, <i>p</i> = 0.038) and serum vitamin D level (R = -0.974, <i>p</i> < 0.001) while also positively correlated with alkaline phosphatase (R = 0.834, <i>p</i> < 0.001). A quality-of-life index that is specific to total hemophilia (Haemo-Qol/Haem-A-QoL) had a significant positive correlation with total hemophilia joint health score (HJHS) (R = 0.934, <i>p</i> < 0.001) and negatively correlated with serum vitamin D level (R = -0.924, <i>p</i>-value lower than 0.001), alkaline phosphatase (R = 0.842, <i>p</i> < 0.001), and severity of hemophilia (R = 0.67, <i>p</i> < 0.001). <b>Conclusions</b>: patients with hemophilia A had lower vitamin D levels than healthy controls. The severity of vitamin D deficiency is related positively to (HJHS) hemophilia and quality of life hemophilia cases according to Haemo-QoL.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"742-751"},"PeriodicalIF":1.1,"publicationDate":"2024-11-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11727777/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142894032","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
Mesenchymal Stem Cells and Reticulated Platelets: New Horizons in Multiple Myeloma. 间充质干细胞和网状血小板:多发性骨髓瘤的新视野。
IF 1.1
Hematology Reports Pub Date : 2024-11-23 DOI: 10.3390/hematolrep16040070
Cristian Alejandro Mera Azaín, Johan Leandro Vargas Pasquel, Sandra Milena Quijano Gómez, Viviana Marcela Rodríguez-Pardo
{"title":"Mesenchymal Stem Cells and Reticulated Platelets: New Horizons in Multiple Myeloma.","authors":"Cristian Alejandro Mera Azaín, Johan Leandro Vargas Pasquel, Sandra Milena Quijano Gómez, Viviana Marcela Rodríguez-Pardo","doi":"10.3390/hematolrep16040070","DOIUrl":"10.3390/hematolrep16040070","url":null,"abstract":"<p><p>Multiple myeloma (MM) is a malignant plasma cell disorder characterized by the accumulation of abnormal plasma cells in the bone marrow. Mesenchymal stem cells (MSCs) and reticulated platelets (RPs) have been implicated in the pathogenesis of MM. This narrative review aims to explore the role of MSCs and RPs in the pathophysiology of MM, particularly their clinical use as possible variables of prognostic value in this hematologic neoplasia. The interaction between MSCs and MM cells within the bone marrow microenvironment supports MM cell survival, proliferation, and drug resistance. MSCs contribute to the development and maintenance of MM through the secretion of various factors, including cytokines, chemokines, and growth factors. Moreover, RPs, young and highly reactive platelets, have been implicated in promoting angiogenesis, tumor growth, and metastasis in MM. Several studies show that cells such as MSCs and platelets participate actively in the biology of the disease. Still, in clinical practice, they are not considered part of evaluating affected patients. In this review, we explore the possibility of including the evaluation of MSCs and PRs in the clinical practice for patients with MM as part of the strategies to improve the outcomes of this disease.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"732-741"},"PeriodicalIF":1.1,"publicationDate":"2024-11-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11627159/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142709534","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
A Case of B-Cell Lymphoblastic Lymphoma Presenting with an Isolated Epidural Mass Treated Successfully with Radiotherapy Followed by United Kingdom Acute Lymphoblastic Leukemia (UKALL) Chemotherapy Protocol. 英国急性淋巴细胞白血病 (UKALL) 化疗方案成功治疗了一例出现孤立硬膜外肿块的 B 细胞淋巴细胞淋巴瘤患者。
IF 1.1
Hematology Reports Pub Date : 2024-11-23 DOI: 10.3390/hematolrep16040069
Musa Fares Alzahrani
{"title":"A Case of B-Cell Lymphoblastic Lymphoma Presenting with an Isolated Epidural Mass Treated Successfully with Radiotherapy Followed by United Kingdom Acute Lymphoblastic Leukemia (UKALL) Chemotherapy Protocol.","authors":"Musa Fares Alzahrani","doi":"10.3390/hematolrep16040069","DOIUrl":"10.3390/hematolrep16040069","url":null,"abstract":"<p><strong>Background: </strong>B-cell lymphoblastic lymphoma (B-LBL) is an aggressive type of non-Hodgkin lymphoma that usually involves lymph nodes, skin and soft tissue. Bone marrow and peripheral blood are normally spared from involvement in the disease. B-LBL typically forms solid masses that have similar pathologic and immunophenotypic features to their liquid counterpart, B-cell acute lymphoblastic leukemia (B-ALL). The presentation of B-LBL with a solitary epidural mass at the cervical spine is very rare and the optimal treatment of such cases is unknown. Most of the literature on the management of B-LBL comes from small case series, pediatric patients, or as part of retrospective data that combine B-LBL with B-ALL cases.</p><p><strong>Case presentation: </strong>The case presented herein is a unique presentation that was treated using three modalities, namely surgical resection, radiotherapy and consolidation with systemic chemotherapy, adopted from the United Kingdom acute lymphoblastic leukemia (UKALL14) protocol.</p><p><strong>Conclusions: </strong>The patient attained complete remission following the planned treatment and is still in remission for more than four and half years from the time of his initial diagnosis.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"724-731"},"PeriodicalIF":1.1,"publicationDate":"2024-11-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11586985/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142709201","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
Efficacy of Anti-CD38 Monoclonal Antibodies for Relapsed or Refractory Multiple Myeloma in Stem Cell Transplant-Ineligible Patients Aged over 65 Years: A Propensity Score-Matched Study. 抗CD38单克隆抗体对不符合干细胞移植条件的65岁以上复发性或难治性多发性骨髓瘤患者的疗效:倾向评分匹配研究
IF 1.1
Hematology Reports Pub Date : 2024-11-18 DOI: 10.3390/hematolrep16040068
Satoshi Yamasaki, Michitoshi Hashiguchi, Nao Yoshida-Sakai, Hiroto Jojima, Koichi Osaki, Takashi Okamura, Yutaka Imamura
{"title":"Efficacy of Anti-CD38 Monoclonal Antibodies for Relapsed or Refractory Multiple Myeloma in Stem Cell Transplant-Ineligible Patients Aged over 65 Years: A Propensity Score-Matched Study.","authors":"Satoshi Yamasaki, Michitoshi Hashiguchi, Nao Yoshida-Sakai, Hiroto Jojima, Koichi Osaki, Takashi Okamura, Yutaka Imamura","doi":"10.3390/hematolrep16040068","DOIUrl":"10.3390/hematolrep16040068","url":null,"abstract":"<p><strong>Background: </strong>The development of newer agents, including anti-CD38 monoclonal antibodies (mAbs), has significantly improved overall survival (OS) in patients with relapsed or refractory multiple myeloma (RRMM). However, the treatment of older patients with RRMM who are transplant-ineligible remains challenging.</p><p><strong>Methods: </strong>We retrospectively evaluated OS in 78 transplant-ineligible patients with RRMM who were aged ≥ 65 years and treated at our institution between February 2012 and November 2023.</p><p><strong>Results: </strong>Unadjusted OS was significantly longer in the anti-CD38 mAb-exposed group (i.e., those previously treated with daratumumab and receiving isatuximab plus pomalidomide and low-dose dexamethasone because of disease progression during treatment with daratumumab [<i>n</i> = 6], daratumumab plus pomalidomide and low-dose dexamethasone [<i>n</i> = 9], or isatuximab plus pomalidomide and low-dose dexamethasone without daratumumab-exposure [<i>n</i> = 14]) than in the anti-CD38 mAb-naïve group (no exposure to daratumumab or isatuximab [<i>n</i> = 49]) (<i>p</i> < 0.001). To address potential confounder factors associated with use or nonuse of anti-CD38 mAbs, we performed propensity score matching (PSM) using age, sex, performance status, and Geriatric 8 and Instrumental Activities of Daily Living scores. PSM identified 14 subjects from the anti-CD38 mAb-exposed group with baseline characteristics similar to those of 14 subjects from the anti-CD38 mAb-naïve group. After PSM, the adjusted OS was significantly longer in the anti-CD38 mAb-exposed group than in the anti-CD38 mAb-naïve group (<i>p</i> < 0.001).</p><p><strong>Conclusion: </strong>These findings provide insights into the optimal use of anti-CD38 mAbs in patients with RRMM who are transplant-ineligible and aged ≥65 years and on candidates who are appropriate for novel approaches, such as chimeric antigen receptor T-cell or bispecific T-cell engager therapy.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"714-723"},"PeriodicalIF":1.1,"publicationDate":"2024-11-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11587159/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142709529","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
Treatment Strategies Used in Treating Myelofibrosis: State of the Art. 用于治疗骨髓纤维化的治疗策略:最新技术。
IF 1.1
Hematology Reports Pub Date : 2024-10-30 DOI: 10.3390/hematolrep16040067
Massimo Martino, Martina Pitea, Annalisa Sgarlata, Ilaria Maria Delfino, Francesca Cogliandro, Anna Scopelliti, Violetta Marafioti, Simona Polimeni, Gaetana Porto, Giorgia Policastro, Giovanna Utano, Maria Pellicano, Giovanni Leanza, Caterina Alati
{"title":"Treatment Strategies Used in Treating Myelofibrosis: State of the Art.","authors":"Massimo Martino, Martina Pitea, Annalisa Sgarlata, Ilaria Maria Delfino, Francesca Cogliandro, Anna Scopelliti, Violetta Marafioti, Simona Polimeni, Gaetana Porto, Giorgia Policastro, Giovanna Utano, Maria Pellicano, Giovanni Leanza, Caterina Alati","doi":"10.3390/hematolrep16040067","DOIUrl":"10.3390/hematolrep16040067","url":null,"abstract":"<p><strong>Background: </strong>Current drug therapy for myelofibrosis does not alter the natural course of the disease or prolong survival, and allogeneic stem cell transplantation is the only curative treatment modality. For over a decade, the Janus kinase (JAK) inhibitor ruxolitinib has been the standard of care. More recently, newer-generation JAK inhibitors have joined the ranks of accepted treatment options.</p><p><strong>Objectives: </strong>The primary goal of treatment is to reduce spleen size and minimize disease-related symptoms. Prognostic scoring systems are used to designate patients as being at lower or higher risk. For transplant-eligible patients, transplant is offered to those with a bridge of a JAK inhibitor; patients who are not eligible for transplant are usually offered long-term therapy with a JAK inhibitor. Limited disease-modifying activity, dose-limiting cytopenias, and other adverse effects have contributed to discontinuation of JAK inhibitor treatment.</p><p><strong>Conclusions: </strong>Novel JAK inhibitors and combination approaches are currently being explored to overcome these shortcomings. Further research will be essential to establish optimal therapeutic approaches in first-line and subsequent treatments.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"698-713"},"PeriodicalIF":1.1,"publicationDate":"2024-10-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11587016/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142709722","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
Real-World Study of US Adults with Paroxysmal Nocturnal Hemoglobinuria Treated with Pegcetacoplan. 美国成人阵发性夜间血红蛋白尿症患者接受培基他克普兰治疗的真实世界研究。
IF 1.1
Hematology Reports Pub Date : 2024-10-29 DOI: 10.3390/hematolrep16040065
Brian Mulherin, Apeksha Shenoy, Lily Arnett, Weiqi Jiao, Joseph Guarinoni, Sujata Sarda, Jinny Min, David Dingli
{"title":"Real-World Study of US Adults with Paroxysmal Nocturnal Hemoglobinuria Treated with Pegcetacoplan.","authors":"Brian Mulherin, Apeksha Shenoy, Lily Arnett, Weiqi Jiao, Joseph Guarinoni, Sujata Sarda, Jinny Min, David Dingli","doi":"10.3390/hematolrep16040065","DOIUrl":"10.3390/hematolrep16040065","url":null,"abstract":"<p><p><b>Background</b>: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, life-threatening disease characterized by complement-mediated hemolysis. OPERA is the first US longitudinal real-world study on C3 inhibitor therapy, known as pegcetacoplan. <b>Methods</b>: OPERA enrolled US patients with PNH, age ≥18, who were prescribed pegcetacoplan, and data were collected from routine care. Hemoglobin was reported by patients during regular follow-up (censored from transfusions). The Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue (0-52 score) and Patient-Reported Outcomes Measurement Information System scale for Cognitive Function Abilities (PROMIS-CF; 23.27-67.09 t-score) were completed electronically (low score = negative outcome). Patients self-reported incidence of healthcare resource utilization (HCRU). <b>Results</b>: By January 2024, 70 patients (mean age 44.6 years; 57.1% female) reported up to 9 months of pegcetacoplan treatment, with a median [IQR] follow-up of 6.6 [3.8] months. The latest reported hemoglobin levels improved by a mean (SD) of 2.6 (1.9) g/dL from baseline. At 3, 6 and 9 months, patients reported clinically meaningful improvements (≥5 points) in FACIT-F (53.3-69.0%) and (≥2 points) PROMIS-CF (46.7-55.2%). Patients reported a <10% incidence rate per person month of all HCRU events. <b>Conclusions</b>: This first longitudinal real-world US study indicates a positive trend in Hb, fatigue, and cognition with limited HCRU during pegcetacoplan treatment in adults with PNH.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"669-681"},"PeriodicalIF":1.1,"publicationDate":"2024-10-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11587045/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142709629","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
Alternative Splicing: A Potential Therapeutic Target in Hematological Malignancies. 替代剪接:血液恶性肿瘤的潜在治疗靶点
IF 1.1
Hematology Reports Pub Date : 2024-10-29 DOI: 10.3390/hematolrep16040066
Gazmend Temaj, Silvia Chichiarelli, Sarmistha Saha, Pelin Telkoparan-Akillilar, Nexhibe Nuhii, Rifat Hadziselimovic, Luciano Saso
{"title":"Alternative Splicing: A Potential Therapeutic Target in Hematological Malignancies.","authors":"Gazmend Temaj, Silvia Chichiarelli, Sarmistha Saha, Pelin Telkoparan-Akillilar, Nexhibe Nuhii, Rifat Hadziselimovic, Luciano Saso","doi":"10.3390/hematolrep16040066","DOIUrl":"10.3390/hematolrep16040066","url":null,"abstract":"<p><p>Leukemia represents the most prevalent malignancy in children, constituting 30% of childhood cancer cases, with acute lymphoblastic leukemia (ALL) being particularly heterogeneous. This paper explores the role of alternative splicing in leukemia, highlighting its significance in cancer development and progression. Aberrant splicing is often driven by mutations in splicing-factor genes, which can lead to the production of variant proteins that contribute to oncogenesis. The spliceosome, a complex of small nuclear RNAs and proteins, facilitates RNA splicing, a process critical for generating diverse mRNA and protein products from single genes. Mutations in splicing factors, such as U2AF1, SF3B1, SRSF2, ZRSR2, and HNRNPH1, are frequently observed across various hematological malignancies and are associated with poor prognosis and treatment resistance. This research underscores the necessity of understanding the mechanisms of RNA splicing dysregulation in order to develop targeted therapies to correct these aberrant processes, thereby improving outcomes for patients with leukemia and related disorders.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"682-697"},"PeriodicalIF":1.1,"publicationDate":"2024-10-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11587037/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142709360","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
Gene Therapy: A Revolutionary Step in Treating Thalassemia. 基因疗法:治疗地中海贫血症的革命性一步。
IF 1.1
Hematology Reports Pub Date : 2024-10-21 DOI: 10.3390/hematolrep16040064
Jhancy Malay, Rasha Aziz Attia Salama, Ghania Shehzad Alam Qureshi, Ali Raafat Ali Ahmed Ammar, Gayatri Janardhan, Maryam Safdar, Hesham Amin Hamdy Elshamy
{"title":"Gene Therapy: A Revolutionary Step in Treating Thalassemia.","authors":"Jhancy Malay, Rasha Aziz Attia Salama, Ghania Shehzad Alam Qureshi, Ali Raafat Ali Ahmed Ammar, Gayatri Janardhan, Maryam Safdar, Hesham Amin Hamdy Elshamy","doi":"10.3390/hematolrep16040064","DOIUrl":"https://doi.org/10.3390/hematolrep16040064","url":null,"abstract":"<p><p>Beta thalassemia is an inherited blood disorder that results in inefficient erythropoiesis due to genetic mutation that leads to the reduction or absence of the hemoglobin beta-globulin protein. Approximately 8.5% of UAE residents suffer from β-thalassemia, a significant health and financial problem. The treatment options available for β-Thalassemia major are limited and associated with a wide range of complications. β-thalassemia gene therapy is emerging as a potential novel treatment option that eliminates the complications caused by the current long-term treatment modalities and the associated economic burden. This paper reviews the scientific literature related to emerging gene therapy for β-Thalassemia by analyzing all the articles published from January 2010 to December 2023 in the English language on Databases like PubMed, Scopus, ProQuest, and CINAHL. The use of gene therapy has demonstrated promising outcomes for a permanent cure of β-Thalassemia. To conclude, gene therapy is an innovative solution. It demonstrates a promising future, but does come with its own setbacks and is something that must be tackled in order to revolutionize it in the medical world. FDA-approved ZYNTEGLO is a potentially one-time curative treatment for β-Thalassemia. Although cutting-edge, its use is limited because of the high cost-a price of USD 2.8 million per patient.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"656-668"},"PeriodicalIF":1.1,"publicationDate":"2024-10-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11503351/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142499158","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
Pancytopenia Related to Splenic Angiosarcoma: A Case Report and Literature Review. 与脾脏血管肉瘤相关的全血细胞减少症:病例报告和文献综述。
IF 1.1
Hematology Reports Pub Date : 2024-10-18 DOI: 10.3390/hematolrep16040063
Jakub Misiak, Bernard Sokołowski, Norbert Skrobisz, Mateusz Matczak, Marcin Braun
{"title":"Pancytopenia Related to Splenic Angiosarcoma: A Case Report and Literature Review.","authors":"Jakub Misiak, Bernard Sokołowski, Norbert Skrobisz, Mateusz Matczak, Marcin Braun","doi":"10.3390/hematolrep16040063","DOIUrl":"https://doi.org/10.3390/hematolrep16040063","url":null,"abstract":"<p><strong>Background: </strong>Angiosarcomas are highly aggressive malignancies with endothelial differentiation, presenting considerable challenges in oncology, especially when arising in rare locations such as the spleen. These tumors predominantly affect adults and are commonly found in the skin, breast, liver, or soft tissues, with more unusual occurrences in other organs. Angiosarcomas have a high propensity for metastasis, typically spreading to the liver, lungs, lymph nodes, and gastrointestinal tract. Splenic angiosarcoma, with fewer than 300 documented cases, is an especially rare and complex form of this malignancy.</p><p><strong>Case presentation: </strong>This report details a case of splenic angiosarcoma in a 45-year-old male, where bone marrow metastases were the first clinical presentation, initially mimicking myelodysplastic syndrome (MDS) due to persistent pancytopenia.</p><p><strong>Conclusions: </strong>The eventual identification of the splenic origin underscores the diagnostic difficulties and clinical challenges inherent in managing such atypical and rare presentations.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"648-655"},"PeriodicalIF":1.1,"publicationDate":"2024-10-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11503393/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142499161","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
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