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Chart review across EU5 in MM post-ASCT patients. asct后MM患者EU5的图表回顾。
Pub Date : 2018-07-11 eCollection Date: 2018-03-01 DOI: 10.2217/ijh-2018-0004
John Ashcroft, Davneet Judge, Sujith Dhanasiri, Gavin Taylor-Stokes, Chloe Middleton

Aim: To understand the current treatment patterns, clinical outcomes and healthcare resource utilization-associated costs for multiple myeloma patients, post autologous stem cell transplant (ASCT) across Europe.

Patients & methods: Medical records were used to abstract data for 337 multiple myeloma patients who had received ASCT.

Results: Following ASCT, 7% received maintenance therapy prior to progression. Lenalidomide was the most frequently prescribed maintenance, second- and third-line therapy. Monthly resource use was considerably lower in patients who received maintenance therapy (€638.14 vs €1001.74). Median time to progression was longer for patients who had received maintenance therapy.

Conclusion: The study highlights the diversity in current treatment patterns post-ASCT. Results suggest patients who receive maintenance therapy have a prolonged remission period, and as a result their associated healthcare resource utilization is spread across the treatment pathway.

目的:了解欧洲多发性骨髓瘤患者自体干细胞移植(ASCT)后的当前治疗模式、临床结果和医疗资源利用相关成本。患者与方法:对337例行ASCT的多发性骨髓瘤患者病历进行数据提取。结果:ASCT后,7%的患者在病情进展前接受了维持治疗。来那度胺是最常用的维持、二线和三线治疗。接受维持治疗的患者每月资源使用量明显较低(638.14欧元对1001.74欧元)。接受维持治疗的患者中位进展时间更长。结论:该研究强调了asct后当前治疗模式的多样性。结果表明,接受维持治疗的患者缓解期延长,因此他们相关的医疗资源利用在整个治疗途径中分散。
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引用次数: 8
Isolated hypoglossal nerve palsy as a presenting symptom of metastatic peripheral T-cell lymphoma - not otherwise specified (PTCL-NOS): a unique case & a review of the literature. 孤立性舌下神经麻痹作为转移性外周t细胞淋巴瘤-非特异性(PTCL-NOS)的表现症状:一个独特的病例&文献回顾。
Pub Date : 2018-06-28 eCollection Date: 2018-03-01 DOI: 10.2217/ijh-2018-0002
Emily Bryer, David Henry

Extensive and significant technological advancements have enhanced the sensitivity and accuracy of the pathologic classification, diagnosis, and therapeutics of lymphoma. These advances have prompted a more comprehensive understanding of neoplastic behavior and have led to improvements in both treatment and prognosis. This paper presents a comprehensive review of lymphoma and features a case report of a unique presentation of peripheral T-cell lymphoma - not otherwise specified that presented with isolated hypoglossal nerve dysfunction.

广泛而重要的技术进步提高了淋巴瘤病理分类、诊断和治疗的敏感性和准确性。这些进展促使人们对肿瘤行为有了更全面的了解,并改善了治疗和预后。本文对淋巴瘤进行了全面的回顾,并报道了一例独特的外周t细胞淋巴瘤的病例报告-没有其他规定,表现为孤立的舌下神经功能障碍。
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引用次数: 4
Radioimmunotherapy-based conditioning regimen prior to autologous stem cell transplantation in non-Hodgkin lymphoma. 非霍奇金淋巴瘤自体干细胞移植前基于放射免疫治疗的调理方案。
Pub Date : 2018-06-13 eCollection Date: 2018-03-01 DOI: 10.2217/ijh-2017-0025
Mahsa Eskian, MirHojjat Khorasanizadeh, Alessandro Isidori, Nima Rezaei

Non-Hodgkin lymphoma (NHL) is the most common hematologic malignancy and the sixth cause of death from cancer in the USA. Autologous stem cell transplantation (ASCT) is a potentially curative therapeutic option for many NHL patients. Choosing the most effective conditioning regimen prior to ASCT can lead to longer survival in these patients, and, as in many cases of high risk NHL, the only potentially curative option is stem cell transplantation. Radioimmunotherapy (RIT) is based on using radiolabeled monoclonal antibodies against tumoral antigens. Since lymphoma cells are sensitive to radiation, RIT has become a potential approach in treating NHL. In this review, we have discussed the efficacy and safety of RIT as an alternative conditioning regimen prior to ASCT.

非霍奇金淋巴瘤(NHL)是最常见的血液系统恶性肿瘤,也是美国癌症死亡的第六大原因。自体干细胞移植(ASCT)是许多NHL患者的潜在治疗选择。在ASCT之前选择最有效的调理方案可以延长这些患者的生存期,并且,在许多高风险NHL病例中,唯一潜在的治疗选择是干细胞移植。放射免疫治疗(RIT)是基于使用放射标记的单克隆抗体来对抗肿瘤抗原。由于淋巴瘤细胞对辐射敏感,RIT已成为治疗非霍奇金淋巴瘤的潜在方法。在这篇综述中,我们讨论了RIT作为ASCT之前的替代调理方案的有效性和安全性。
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引用次数: 3
Management of older adults with acute lymphoblastic leukemia: challenges & current approaches. 老年人急性淋巴细胞白血病的管理:挑战和当前的方法。
Pub Date : 2018-04-10 eCollection Date: 2018-03-01 DOI: 10.2217/ijh-2017-0023
Yazeed Sawalha, Anjali S Advani

The management of acute lymphoblastic leukemia (ALL) in older patients is challenging. Older patients often have multiple comorbidities and poor performance status, and disease factors associated with poor prognosis are more common in this age group. Patient and disease-related factors should be taken into account to determine whether intensive therapy is appropriate. The use of comorbidity indices and comprehensive geriatric assessment tools can be valuable in this setting. Fit patients should be considered for aggressive therapies including allogeneic hematopoietic stem cell transplantation, whereas low intensity options may be more suitable for the frail. The Philadelphia (Ph) chromosome is present in up to half of the cases of ALL in older patients. The incorporation of TK inhibitors into the treatment plans of older patients with Ph-positive ALL has improved the outcomes significantly. For less fit patients with Ph-positive ALL, the use of TK inhibitors with reduced-intensity chemotherapy or steroids alone results in high rates of remission, but, without further consolidation, relapses are inevitable. Many novel targeted and immunotherapeutic agents are being developed, offering more effective and tolerable treatment options.

老年急性淋巴细胞白血病(ALL)的治疗具有挑战性。老年患者往往有多种合并症和不良的表现状态,与预后不良相关的疾病因素在该年龄组更为常见。在确定强化治疗是否合适时,应考虑患者和疾病相关因素。在这种情况下,使用合并症指数和综合老年评估工具可能是有价值的。健康的患者应该考虑积极的治疗,包括异体造血干细胞移植,而低强度的选择可能更适合体弱的患者。费城(Ph)染色体存在于老年ALL患者中多达一半的病例中。将TK抑制剂纳入老年ph阳性ALL患者的治疗计划可显著改善预后。对于ph阳性ALL患者,使用TK抑制剂联合低强度化疗或单独使用类固醇可导致高缓解率,但如果没有进一步巩固,复发是不可避免的。许多新的靶向和免疫治疗药物正在开发中,提供更有效和可耐受的治疗选择。
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引用次数: 24
A discussion of blood cancers and the MPN landmark survey. 关于血癌和MPN里程碑调查的讨论。
Pub Date : 2017-12-01 Epub Date: 2017-12-21 DOI: 10.2217/ijh-2017-0024
Claire Harrison

Professor Claire Harrison speaks to Roshaine Wijayatunga, managing commissioning editor: Professor Claire Harrison became a consultant at Guy's and St Thomas' hospital in 2001. She is also deputy clinical director of cancer and hematology. She has influenced major advances in her field, including the description of key mutations and designing/leading global clinical trials, for example, the first JAK inhibitor trial in Europe. She is the chief investigator for several other international and national clinical trials. She has strong international collaborations and authors many papers in peer-reviewed journals such as the New England Journal of Medicine and Blood. She has worked with enthusiastic patients to create a patient charity, MPN Voice. She is an internationally regarded expert in her field as a patient advocate.

克莱尔·哈里森教授在2001年成为盖伊和圣托马斯医院的顾问。她也是癌症和血液学临床副主任。她影响了她所在领域的重大进展,包括关键突变的描述和设计/领导全球临床试验,例如,欧洲的第一个JAK抑制剂试验。她是其他几个国际和国家临床试验的首席研究员。她拥有强大的国际合作关系,并在《新英格兰医学与血液杂志》等同行评议期刊上发表了许多论文。她与热心的病人一起创建了一个病人慈善机构MPN Voice。作为一名患者倡导者,她是该领域的国际知名专家。
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引用次数: 0
Interview with Dr Philip McCarthy. 采访菲利普·麦卡锡博士。
Pub Date : 2017-12-01 Epub Date: 2018-02-22 DOI: 10.2217/ijh-2017-0022
Philip McCarthy

Philip McCarthy speaks to Roshaine Wijayatunga, Senior Editor: Oncology Philip McCarthy completed his MD at Tufts University School of Medicine, Boston, MA, USA and his Internal Medicine Residency at Yale, New Haven Hospital, New Haven, CT, USA. His Fellowship was completed at Brigham and Women's Hospital and the Dana Farber Cancer Insitute, Harvard University, Boston, MA, USA. His research interests are devoted to developing novel intensive and reduced intensity allogeneic (allo) and autologous (auto) hematopoietic stem cell transplant (HSCT) approaches for the treatment of hematologic disorders, leading to improved patient outcomes and decreased toxicity. He has over 20 years of experience treating HSCT patients and directing clinical and translational HSCT research studies. He has served as chair or co-chair of several clinical trials including CALGB 100104, a Phase III clinical trial evaluating lenalidomide maintenance after auto-HSCT for multiple myeloma (MM). This study demonstrated an improved progression-free and overall survival for MM patients receiving lenalidomide maintenance therapy after auto-HSCT. The Roswell Park Comprehensive Cancer Center Blood and Marrow Transplant team has developed a systematic approach to the evaluation and treatment of HSCT patients with a specific focus on predicting and minimizing treatment-related mortality. The team participates with a core group of basic science and clinical researchers who are committed to the investigation of the complications of auto- and allo-HSCT and to the developing novel approaches to improve outcomes.

Philip McCarthy在美国马萨诸塞州波士顿的塔夫茨大学医学院完成了医学博士学位,并在美国康涅狄格州纽黑文的耶鲁大学纽黑文医院完成了内科住院医师。他的研究在美国马萨诸塞州波士顿的哈佛大学布里格姆妇女医院和达纳法伯癌症研究所完成。他的研究兴趣是致力于开发新的强化和降低强度的同种异体(allo)和自体(auto)造血干细胞移植(HSCT)治疗血液系统疾病的方法,从而改善患者的预后并降低毒性。他有超过20年的治疗HSCT患者和指导临床和转化HSCT研究的经验。他曾担任多项临床试验的主席或联合主席,包括CALGB 100104,这是一项评估多发性骨髓瘤(MM)自体造血干细胞移植后来那度胺维持的III期临床试验。这项研究表明,自体造血干细胞移植后接受来那度胺维持治疗的MM患者的无进展和总生存率有所改善。Roswell Park综合癌症中心的血液和骨髓移植团队已经开发出一种系统的方法来评估和治疗HSCT患者,并特别关注预测和最小化治疗相关的死亡率。该团队与基础科学和临床研究人员组成的核心小组一起参与,他们致力于研究自体和同种异体造血干细胞移植的并发症,并开发新的方法来改善结果。
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引用次数: 0
Long-term outcomes of rituximab, temozolomide and high-dose methotrexate without consolidation therapy for lymphoma involving the CNS. 利妥昔单抗、替莫唑胺和大剂量甲氨蝶呤非巩固治疗累及中枢神经系统淋巴瘤的长期疗效。
Pub Date : 2017-12-01 Epub Date: 2018-01-26 DOI: 10.2217/ijh-2017-0020
Sarah J Nagle, Nirav N Shah, Alex Ganetsky, Daniel J Landsburg, Sunita D Nasta, Anthony Mato, Stephen J Schuster, Ran Reshef, Donald E Tsai, Jakub Svoboda

Aim: To describe the long-term outcomes of patients with lymphoma in the CNS treated with rituximab, temozolomide and high-dose methotrexate without consolidation therapy.

Patients & methods: A retrospective cohort study of 46 consecutive patients with primary CNS lymphoma (PCNSL, 27 patients) or secondary CNS involvement of diffuse large B-cell lymphoma (DLBCL, 19 patients) who were treated with rituximab on day 1 in combination with high-dose methotrexate (days 1 and 15) and temozolomide (days 1-5) in 28-day cycles without further consolidation.

Results: Median follow-up was 21.2 months. Patients received a median of five cycles (range 1-15). Median overall survival (OS) was 26 months and median progression-free survival was 8.6 months. At 3 years, 37% of patients were alive and without evidence of disease. The patients with PCNSL had a significantly higher response rates (ORR 81 vs 47%; p = 0.015) and longer median OS (55.3 vs 4.8 months; p < 0.01) than those with secondary CNS DLBCL. Toxicities were mild and manageable.

Conclusion: The rituximab, temozolomide and methotrexate regimen is an effective therapy for patients with PCNSL without the toxicities typically associated with consolidation therapy.

目的:描述无巩固治疗的利妥昔单抗、替莫唑胺和大剂量甲氨蝶呤治疗中枢神经系统淋巴瘤患者的长期预后。患者和方法:一项回顾性队列研究,连续46例原发性中枢神经系统淋巴瘤(PCNSL, 27例)或继发性弥漫性大b细胞淋巴瘤(DLBCL, 19例)患者,在第1天接受利妥昔单抗联合高剂量甲氨蝶呤(第1天和第15天)和替莫唑胺(第1天至第5天)治疗,28天周期,无进一步巩固。结果:中位随访时间为21.2个月。患者平均接受5个周期(范围1-15)。中位总生存期(OS)为26个月,中位无进展生存期为8.6个月。3年时,37%的患者存活且无疾病迹象。PCNSL患者的缓解率明显更高(ORR为81比47%;p = 0.015)和更长的中位OS (55.3 vs 4.8个月;结论:利妥昔单抗、替莫唑胺和甲氨蝶呤联合治疗PCNSL是一种有效的治疗方案,且无巩固治疗的毒副作用。
{"title":"Long-term outcomes of rituximab, temozolomide and high-dose methotrexate without consolidation therapy for lymphoma involving the CNS.","authors":"Sarah J Nagle,&nbsp;Nirav N Shah,&nbsp;Alex Ganetsky,&nbsp;Daniel J Landsburg,&nbsp;Sunita D Nasta,&nbsp;Anthony Mato,&nbsp;Stephen J Schuster,&nbsp;Ran Reshef,&nbsp;Donald E Tsai,&nbsp;Jakub Svoboda","doi":"10.2217/ijh-2017-0020","DOIUrl":"https://doi.org/10.2217/ijh-2017-0020","url":null,"abstract":"<p><strong>Aim: </strong>To describe the long-term outcomes of patients with lymphoma in the CNS treated with rituximab, temozolomide and high-dose methotrexate without consolidation therapy.</p><p><strong>Patients & methods: </strong>A retrospective cohort study of 46 consecutive patients with primary CNS lymphoma (PCNSL, 27 patients) or secondary CNS involvement of diffuse large B-cell lymphoma (DLBCL, 19 patients) who were treated with rituximab on day 1 in combination with high-dose methotrexate (days 1 and 15) and temozolomide (days 1-5) in 28-day cycles without further consolidation.</p><p><strong>Results: </strong>Median follow-up was 21.2 months. Patients received a median of five cycles (range 1-15). Median overall survival (OS) was 26 months and median progression-free survival was 8.6 months. At 3 years, 37% of patients were alive and without evidence of disease. The patients with PCNSL had a significantly higher response rates (ORR 81 vs 47%; p = 0.015) and longer median OS (55.3 vs 4.8 months; p < 0.01) than those with secondary CNS DLBCL. Toxicities were mild and manageable.</p><p><strong>Conclusion: </strong>The rituximab, temozolomide and methotrexate regimen is an effective therapy for patients with PCNSL without the toxicities typically associated with consolidation therapy.</p>","PeriodicalId":14166,"journal":{"name":"International Journal of Hematologic Oncology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2017-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2217/ijh-2017-0020","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"36569750","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: 14
Untreated chronic lymphocytic leukemia in Lebanese patients: an observational study using standard karyotyping and FISH. 黎巴嫩患者中未经治疗的慢性淋巴细胞白血病:使用标准核型和 FISH 的观察性研究。
Pub Date : 2017-12-01 Epub Date: 2017-12-21 DOI: 10.2217/ijh-2017-0019
Elie El Rassy, Alain Chebly, Rima Korban, Warde Semaan, Ziad Bakouny, Tarek Assi, Hampig Raphael Kourie, Fadi El Karak, Eliane Chouery, Joseph Kattan

Aim: We aimed to understand the biology of chronic lymphocytic leukemia (CLL) patients in Lebanon.

Materials & methods: We applied conventional cytogenetic and FISH studies on Lebanese patients diagnosed with CLL and undergoing a watch and wait approach.

Results: Our study disclosed 53.6% of patients with aberrant karyotypes among which 26.7% were complex karyotypes. Genetic aberrations included del(13q14) 46.4%, 14q32 translocation in 25%, trisomy 12 in 14.3%, del(17p13) and del(11q22) in 7.1% each. The deletion of 6q21/6q23 was not found in any of our patients.

Conclusion: The higher prevalence of del(13q14) as a sole abnormality could be the primary event in inducing CLL. The del(17p13) and del(11q22) followed as potential drivers for progression in CLL patients with a watch and wait approach.

目的:我们旨在了解黎巴嫩慢性淋巴细胞白血病(CLL)患者的生物学特性:我们对确诊为慢性淋巴细胞白血病并接受观察和等待治疗的黎巴嫩患者进行了常规细胞遗传学和 FISH 研究:我们的研究发现,53.6%的患者核型异常,其中26.7%为复杂核型。基因畸变包括 del(13q14) 46.4%、14q32 易位 25%、12 三体 14.3%、del(17p13) 和 del(11q22) 各 7.1%。我们的患者中没有发现 6q21/6q23 缺失:结论:del(13q14)作为唯一异常的发生率较高,可能是诱发CLL的主要原因。Del(17p13)和Del(11q22)是导致CLL患者病情恶化的潜在因素,应采取观察和等待的方法。
{"title":"Untreated chronic lymphocytic leukemia in Lebanese patients: an observational study using standard karyotyping and FISH.","authors":"Elie El Rassy, Alain Chebly, Rima Korban, Warde Semaan, Ziad Bakouny, Tarek Assi, Hampig Raphael Kourie, Fadi El Karak, Eliane Chouery, Joseph Kattan","doi":"10.2217/ijh-2017-0019","DOIUrl":"10.2217/ijh-2017-0019","url":null,"abstract":"<p><strong>Aim: </strong>We aimed to understand the biology of chronic lymphocytic leukemia (CLL) patients in Lebanon.</p><p><strong>Materials & methods: </strong>We applied conventional cytogenetic and FISH studies on Lebanese patients diagnosed with CLL and undergoing a watch and wait approach.</p><p><strong>Results: </strong>Our study disclosed 53.6% of patients with aberrant karyotypes among which 26.7% were complex karyotypes. Genetic aberrations included del(13q14) 46.4%, 14q32 translocation in 25%, trisomy 12 in 14.3%, del(17p13) and del(11q22) in 7.1% each. The deletion of 6q21/6q23 was not found in any of our patients.</p><p><strong>Conclusion: </strong>The higher prevalence of del(13q14) as a sole abnormality could be the primary event in inducing CLL. The del(17p13) and del(11q22) followed as potential drivers for progression in CLL patients with a watch and wait approach.</p>","PeriodicalId":14166,"journal":{"name":"International Journal of Hematologic Oncology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2017-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6172003/pdf/ijh-06-105.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"36569749","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
Early mortality and survival in older adults with acute myeloid leukemia. 老年急性髓性白血病患者的早期死亡率和生存率。
Pub Date : 2017-11-01 Epub Date: 2017-11-20 DOI: 10.2217/ijh-2017-0013
Manisha Pant, Vijaya Raj Bhatt
Acute myeloid leukemia (AML) is a disease of older adults [1] and has a median age of 68 years at the time of diagnosis. The management of AML in older patients (>60 years) is far from optimal and often associated with poor outcomes. Over half of the older patients do not receive initial chemotherapy [1,2], remission rate is lower than in younger patients and lasts shorter duration, and long term survival is dismal. For example, 5-year survival is over 50% for patients aged 15–24 years that drops steadily to 13% for patients between 60 and 69 years of age and 3% for those aged 70–79 years [3]. Functional status varies widely even in older patients with similar biological age and affects outcome. For instance, data from the Southwest Oncology Group trials demonstrated that the early mortality at 1 month for patients older than 75 years is 14% with Eastern Cooperative Oncology Group performance score of 0 versus 82% for a score of 3 [4]. Higher incidence of comorbidities negatively affects outcomes [1]. This is reflected by a higher 8-week mortality (30 vs 19%) in patients with a higher modified Charlson Comorbidity Index of >1 versus ≤ 1, respectively [5]. Comorbidities may lead to poor tolerance of chemotherapy, enhanced toxicity, particularly following intensive chemotherapy and a lower rate of complete response. Malnutrition, decreased immunity with increased susceptibility to infection, cognitive decline and social isolation pose increased risk of toxicity from chemotherapy. Older adults have higher rates of multidrug resistance compared with the younger patients (57 vs 33%) [4]. The proportion with unfavorable cytogenetics increases with age, whereas translocation associated with favorable response drops. AML in younger patients may result from a limited number of mutational events restricting diversity of leukemic subclones and leaving many cell functions intact including mechanism of apoptosis. AML in the older patients, however, may arise due to string of mutational events producing multiple leukemic subclones giving rise to chemoresistance. Patients with pre-existing myelodysplastic or myeloproliferative disorders (i.e., secondary AML) have higher probability of harboring poor cytogenetics, which translates to poor survival. Also, the presence of secondary AML type mutations, commonly observed in older patients, is associated with poor chances of achieving remission and long-term disease control [6]. Overall care of older patients with AML may improve with refined risk-stratification and personalized therapy based on the principles of geriatric medicine. For example, careful assessment of comorbid conditions is vital in older patients. Comprehensive geriatric assessment is also valuable in determining tolerance to various intensities of chemotherapy. Geriatric assessment evaluates multiple health domains including comorbidities, polypharmacy, cognitive, nutritional, psychological, functional and social status, and can predict tolerance of
{"title":"Early mortality and survival in older adults with acute myeloid leukemia.","authors":"Manisha Pant,&nbsp;Vijaya Raj Bhatt","doi":"10.2217/ijh-2017-0013","DOIUrl":"https://doi.org/10.2217/ijh-2017-0013","url":null,"abstract":"Acute myeloid leukemia (AML) is a disease of older adults [1] and has a median age of 68 years at the time of diagnosis. The management of AML in older patients (>60 years) is far from optimal and often associated with poor outcomes. Over half of the older patients do not receive initial chemotherapy [1,2], remission rate is lower than in younger patients and lasts shorter duration, and long term survival is dismal. For example, 5-year survival is over 50% for patients aged 15–24 years that drops steadily to 13% for patients between 60 and 69 years of age and 3% for those aged 70–79 years [3]. Functional status varies widely even in older patients with similar biological age and affects outcome. For instance, data from the Southwest Oncology Group trials demonstrated that the early mortality at 1 month for patients older than 75 years is 14% with Eastern Cooperative Oncology Group performance score of 0 versus 82% for a score of 3 [4]. Higher incidence of comorbidities negatively affects outcomes [1]. This is reflected by a higher 8-week mortality (30 vs 19%) in patients with a higher modified Charlson Comorbidity Index of >1 versus ≤ 1, respectively [5]. Comorbidities may lead to poor tolerance of chemotherapy, enhanced toxicity, particularly following intensive chemotherapy and a lower rate of complete response. Malnutrition, decreased immunity with increased susceptibility to infection, cognitive decline and social isolation pose increased risk of toxicity from chemotherapy. Older adults have higher rates of multidrug resistance compared with the younger patients (57 vs 33%) [4]. The proportion with unfavorable cytogenetics increases with age, whereas translocation associated with favorable response drops. AML in younger patients may result from a limited number of mutational events restricting diversity of leukemic subclones and leaving many cell functions intact including mechanism of apoptosis. AML in the older patients, however, may arise due to string of mutational events producing multiple leukemic subclones giving rise to chemoresistance. Patients with pre-existing myelodysplastic or myeloproliferative disorders (i.e., secondary AML) have higher probability of harboring poor cytogenetics, which translates to poor survival. Also, the presence of secondary AML type mutations, commonly observed in older patients, is associated with poor chances of achieving remission and long-term disease control [6]. Overall care of older patients with AML may improve with refined risk-stratification and personalized therapy based on the principles of geriatric medicine. For example, careful assessment of comorbid conditions is vital in older patients. Comprehensive geriatric assessment is also valuable in determining tolerance to various intensities of chemotherapy. Geriatric assessment evaluates multiple health domains including comorbidities, polypharmacy, cognitive, nutritional, psychological, functional and social status, and can predict tolerance of","PeriodicalId":14166,"journal":{"name":"International Journal of Hematologic Oncology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2017-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2217/ijh-2017-0013","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"36568755","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: 6
Defibrotide for the treatment of hepatic veno-occlusive disease/sinusoidal obstruction syndrome with multiorgan failure. 除颤肽治疗肝静脉闭塞性疾病/窦梗阻综合征伴多器官衰竭。
Pub Date : 2017-11-01 Epub Date: 2017-08-11 DOI: 10.2217/ijh-2017-0015
Paul G Richardson, Stephan A Grupp, Antonio Pagliuca, Amrita Krishnan, Vincent T Ho, Selim Corbacioglu

Hepatic veno-occlusive disease, also called sinusoidal obstruction syndrome (VOD/SOS), is a potentially life-threatening and unpredictable complication of hematopoietic stem cell transplantation (HSCT). Characterized by a prothrombotic-hypofibrinolytic state, VOD/SOS typically presents with hyperbilirubinemia, ascites, weight gain and painful hepatomegaly; VOD/SOS with multiorgan failure may be associated with >80% mortality. Treatment has been mainly supportive. However, defibrotide is now approved in the USA for treatment of hepatic VOD/SOS with renal or pulmonary dysfunction following HSCT and in the European Union for treatment of severe hepatic VOD/SOS post-HSCT. In vitro evidence suggests defibrotide may restore thrombotic-fibrinolytic balance at the endothelial level and protect endothelial cells. Defibrotide has demonstrated significant reduction in VOD/SOS-related mortality and resolved VOD/SOS-related symptoms, with a manageable safety profile.

肝静脉闭塞性疾病,也称为正弦阻塞综合征(VOD/SOS),是造血干细胞移植(HSCT)的一种潜在的危及生命和不可预测的并发症。VOD/SOS以血栓前低纤溶状态为特征,通常表现为高胆红素血症、腹水、体重增加和疼痛性肝肿大;VOD/SOS合并多器官功能衰竭可能与>80%的死亡率相关。治疗主要是支持性的。然而,降纤肽目前在美国被批准用于治疗HSCT后伴有肾或肺功能障碍的肝脏VOD/SOS,在欧盟被批准用于HSCT后严重的肝脏VOD/SOS。体外证据表明,降纤肽可以在内皮水平上恢复血栓性纤溶平衡并保护内皮细胞。除颤肽已证明显著降低了VOD/SOS相关死亡率,并解决了VOD/SOS相关症状,具有可控的安全性。
{"title":"Defibrotide for the treatment of hepatic veno-occlusive disease/sinusoidal obstruction syndrome with multiorgan failure.","authors":"Paul G Richardson,&nbsp;Stephan A Grupp,&nbsp;Antonio Pagliuca,&nbsp;Amrita Krishnan,&nbsp;Vincent T Ho,&nbsp;Selim Corbacioglu","doi":"10.2217/ijh-2017-0015","DOIUrl":"10.2217/ijh-2017-0015","url":null,"abstract":"<p><p>Hepatic veno-occlusive disease, also called sinusoidal obstruction syndrome (VOD/SOS), is a potentially life-threatening and unpredictable complication of hematopoietic stem cell transplantation (HSCT). Characterized by a prothrombotic-hypofibrinolytic state, VOD/SOS typically presents with hyperbilirubinemia, ascites, weight gain and painful hepatomegaly; VOD/SOS with multiorgan failure may be associated with >80% mortality. Treatment has been mainly supportive. However, defibrotide is now approved in the USA for treatment of hepatic VOD/SOS with renal or pulmonary dysfunction following HSCT and in the European Union for treatment of severe hepatic VOD/SOS post-HSCT. <i>In vitro</i> evidence suggests defibrotide may restore thrombotic-fibrinolytic balance at the endothelial level and protect endothelial cells. Defibrotide has demonstrated significant reduction in VOD/SOS-related mortality and resolved VOD/SOS-related symptoms, with a manageable safety profile.</p>","PeriodicalId":14166,"journal":{"name":"International Journal of Hematologic Oncology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2017-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2217/ijh-2017-0015","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"36609876","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: 24
期刊
International Journal of Hematologic Oncology
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