Expert Review of Hematology最新文献_第5页

Gene therapies on the horizon for sickle cell disease: a clinician's perspective. 地平线上的镰状细胞病基因疗法：临床医生的视角。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2024-09-01 Epub Date: 2024-07-31 DOI: 10.1080/17474086.2024.2386366

Henna Butt, John F Tisdale

{"title":"Gene therapies on the horizon for sickle cell disease: a clinician's perspective.","authors":"Henna Butt, John F Tisdale","doi":"10.1080/17474086.2024.2386366","DOIUrl":"10.1080/17474086.2024.2386366","url":null,"abstract":"Introduction: Sickle cell disease (SCD) is a monogenic disorder that exerts several detrimental health effects on those affected, ultimately resulting in significant morbidity and early mortality. There are millions of individuals globally impacted by this disease. Research in gene therapy has been growing significantly over the past decade, now with two FDA approved products, aiming to find another cure for this complex disease.Areas covered: This perspective article aims to provide a clinician's insight into the current landscape of gene therapies, exploring the novel approaches, clinical advances, and potential impact on the management and prognosis of SCD. A comprehensive literature search encompassing databases such as PubMed, Web of Science and Google Scholar was employed. The search covered literature published from 1980 to 2024, focusing on SCD and curative therapy.Expert opinion: After careful evaluation of the risks and benefits associated with the use of gene therapy for affected patients, the need for a cure outweighs the risks associated with treatment in most cases of SCD. With advances in current technologies, gene therapies can increase access to cures for patients with SCD.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"555-566"},"PeriodicalIF":2.3,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141792361","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Improving care for systemic light-chain amyloidosis patients: is a multidisciplinary approach best? 改善对全身性轻链淀粉样变性患者的护理：多学科方法是否最佳？

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2024-09-01 Epub Date: 2024-08-20 DOI: 10.1080/17474086.2024.2388184

Nirija Ranjit Anderson, Dariusz Korczyk, Peter Mollee

{"title":"Improving care for systemic light-chain amyloidosis patients: is a multidisciplinary approach best?","authors":"Nirija Ranjit Anderson, Dariusz Korczyk, Peter Mollee","doi":"10.1080/17474086.2024.2388184","DOIUrl":"10.1080/17474086.2024.2388184","url":null,"abstract":"Introduction: Light chain (AL) amyloidosis is a rare and complex disease which can affect various systems of the body. In common with many rare and multisystemic diseases, the breadth of diagnostic, clinical, and supportive expertise required to care for such patients is best met by a multidisciplinary team.Areas covered: We outline different phases of the patients' journey, including diagnosis, staging, treatment, and response assessment, to highlight common clinical issues best resolved by a multidisciplinary approach.Expert opinion: To extend the benefit of multidisciplinary care to the majority of patients with AL amyloidosis, innovative healthcare models such as telehealth and multisite multidisciplinary team meetings need to be implemented. The need for a multidisciplinary approach where such a wide array of healthcare skills is required also highlights the shortcomings of our current diagnostic and monitoring assays. Better access to diagnostic and subtyping assays is necessary. The ability to characterize and measure the causative amyloidogenic light chain as well as imaging techniques to accurately diagnose and monitor response to therapy is also needed and is currently an area of research focus.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"567-579"},"PeriodicalIF":2.3,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141999710","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Optimizing maintenance therapy in acute myeloid leukemia: where do we stand in the year 2024? 优化急性髓性白血病的维持治疗：2024 年我们将何去何从？

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2024-08-01 Epub Date: 2024-07-21 DOI: 10.1080/17474086.2024.2382300

Caterina Alati, Martina Pitea, Maria Caterina Mico, Violetta Marafioti, Bruna Greve, Giulia Pratico, Barbara Loteta, Francesca Cogliandro, Gaetana Porto, Giorgia Policastro, Giovanna Utano, Annalisa Sgarlata, Lucrezia Imbalzano, Ilaria Maria Delfino, Elisa Montechiarello, Jessyca Germano, Gianfranco Filippelli, Massimo Martino

{"title":"Optimizing maintenance therapy in acute myeloid leukemia: where do we stand in the year 2024?","authors":"Caterina Alati, Martina Pitea, Maria Caterina Mico, Violetta Marafioti, Bruna Greve, Giulia Pratico, Barbara Loteta, Francesca Cogliandro, Gaetana Porto, Giorgia Policastro, Giovanna Utano, Annalisa Sgarlata, Lucrezia Imbalzano, Ilaria Maria Delfino, Elisa Montechiarello, Jessyca Germano, Gianfranco Filippelli, Massimo Martino","doi":"10.1080/17474086.2024.2382300","DOIUrl":"10.1080/17474086.2024.2382300","url":null,"abstract":"Introduction: Despite the prognosis of patients affected by acute myeloid leukemia (AML) improved in the last decade, most patients relapse. Maintenance therapy after a chemotherapy approach with or without allogeneic stem cell transplantation could be a way to control the undetectable residual burden of leukemic cells. Several studies are being carried out as maintenance therapy in AML. Some critical points need to be defined, how the physician can choose among the various drugs available.Areas covered: This review discusses the advances and controversies surrounding maintenance therapy for AML patients.Expert opinion: Patients withFLT3-positive AML should receive midostaurin or quizartinib in the first-linesetting. For a patient initially receiving midostaurin, consider switching to sorafenib in the post-transplant setting. Because of the improved safety profile and potency, many experts will lean toward using a second-generation FLT3 inhibitor such as quizartinib or gilteritinib. Finally, no data indicate whether maintenance therapy should be prolonged until progression or for a defined period.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"515-525"},"PeriodicalIF":2.3,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141626431","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

The impact of individual clinical features in cold agglutinin disease: hemolytic versus non-hemolytic symptoms. 冷凝集素病个体临床特征的影响：溶血症状与非溶血症状。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2024-08-01 Epub Date: 2024-06-28 DOI: 10.1080/17474086.2024.2372333

Sigbjørn Berentsen, Josephine M I Vos, Agnieszka Malecka, Geir E Tjønnfjord, Shirley D'Sa

{"title":"The impact of individual clinical features in cold agglutinin disease: hemolytic versus non-hemolytic symptoms.","authors":"Sigbjørn Berentsen, Josephine M I Vos, Agnieszka Malecka, Geir E Tjønnfjord, Shirley D'Sa","doi":"10.1080/17474086.2024.2372333","DOIUrl":"10.1080/17474086.2024.2372333","url":null,"abstract":"Introduction: During the last decades, the pathogenesis of cold agglutinin disease (CAD) has been well elucidated and shown to be complex. Several documented or investigational therapies have been made available. This development has resulted in major therapeutic advances, but also in challenges in choice of therapy.Areas covered: In this review, we address each step in pathogenesis: bone marrow clonal lymphoproliferation, composition and effects of monoclonal cold agglutinin, non-complement mediated erythrocyte agglutination, complement-dependent hemolysis, and other effects of complement activation. We also discuss the heterogeneous clinical features and their relation to specific steps in pathogenesis, in particular with respect to the impact of complement involvement. CAD can be classified into three clinical phenotypes with consequences for established treatments as well as development of new therapies. Some promising future treatment approaches - beyond chemoimmunotherapy and complement inhibition - are reviewed.Expert opinion: The patient's individual clinical profile regarding complement involvement and hemolytic versus non-hemolytic features is important for the choice of treatment. Further development of treatment approaches is encouraged, and some candidate drugs are promising irrespective of clinical phenotype. Patients with CAD requiring therapy should be considered for inclusion in clinical trials.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"479-492"},"PeriodicalIF":2.3,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141467350","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Joint health and pain in the changing hemophilia treatment landscape. 不断变化的血友病治疗环境中的关节健康和疼痛。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2024-08-01 Epub Date: 2024-07-15 DOI: 10.1080/17474086.2024.2378936

Maria Elisa Mancuso, Paul McLaughlin, Angela L Forsyth, Leonard A Valentino

{"title":"Joint health and pain in the changing hemophilia treatment landscape.","authors":"Maria Elisa Mancuso, Paul McLaughlin, Angela L Forsyth, Leonard A Valentino","doi":"10.1080/17474086.2024.2378936","DOIUrl":"10.1080/17474086.2024.2378936","url":null,"abstract":"Introduction: Hemophilia is an inherited bleeding disorder. Bleeding, and in particular joint hemorrhage results in chronic arthropathy and disability. Acute and chronic pain are frequent and limit activity and participation and result in decreased health-related quality of life. Remarkable progress has been made in the diagnosis and treatment of hemophilia but bleeding continues to prove recalcitrant to currently available treatments and joint disease remains problematic. Physiotherapy and pain management are mainstays of current multidisciplinary integrated care of people with hemophilia (PWH). The focus of this review is on preservation of joint health in the era of new and innovative therapies.Areas covered: A search of the PubMed Central was conducted on 1 February 2024 using the MeSH Major Topic terms identified as keywords for the manuscript. This review will highlight what is known and unknown about joint bleeding and arthropathy, including insights on pain as a related complication.Expert opinion: Recent advances in therapeutic interventions aimed at promoting healthy joints in PWH will be discussed, including both the pharmacological treatment landscape and related strategies to promote joint health.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"431-444"},"PeriodicalIF":2.3,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141563157","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Sickle cell disease and infertility risks: implications for counseling and care of affected girls and women. 镰状细胞病与不孕风险：对受影响女童和妇女的咨询和护理的影响。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2024-08-01 Epub Date: 2024-06-26 DOI: 10.1080/17474086.2024.2372320

Lydia H Pecker, Katie Cameron

{"title":"Sickle cell disease and infertility risks: implications for counseling and care of affected girls and women.","authors":"Lydia H Pecker, Katie Cameron","doi":"10.1080/17474086.2024.2372320","DOIUrl":"10.1080/17474086.2024.2372320","url":null,"abstract":"Introduction: Sickle cell disease (SCD), its treatments and cures present infertility risks. Fertility counseling is broadly indicated for affected girls and women and fertility preservation may appeal to some. Several streams of evidence suggest that the reproductive lifespan of women with SCD is reduced. Pregnancy is associated with high miscarriage rates. There are enduring questions about the effects of highly effective hydroxyurea treatment on female fertility. Current conditioning regimens for gene therapy or hematopoietic stem cell transplant are gonadotoxic. Fertility preservation methods exist as non-experimental standards of care for girls and women. Clinicians are challenged to overcome multifactorial barriers to incorporate fertility counseling and fertility preservation care into routine SCD care.Areas covered: Here we provide a narrative review of existing evidence regarding fertility and infertility risks in girls and women with SCD and consider counseling implications of existing evidence.Expert opinion: Addressing fertility for girls and women with SCD requires engaging concerns that emerge across the lifespan, acknowledging uncertainty and identifying barriers to care, some of which may be insurmountable without public policy changes. The contemporary SCD care paradigm can offer transformative SCD treatments alongside comprehensive counselling that addresses fertility risks and fertility preservation opportunities.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"493-504"},"PeriodicalIF":2.3,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11293988/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141445988","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Ultrasound-guided joint procedures in hemophilia: technique, indications and tips. 超声引导下的血友病关节手术：技术、适应症和提示。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2024-08-01 Epub Date: 2024-07-17 DOI: 10.1080/17474086.2024.2380477

Hortensia De la Corte-Rodriguez, E Carlos Rodriguez-Merchan, M Teresa Alvarez-Roman, Primitivo Gomez-Cardero, Victor Jimenez-Yuste

{"title":"Ultrasound-guided joint procedures in hemophilia: technique, indications and tips.","authors":"Hortensia De la Corte-Rodriguez, E Carlos Rodriguez-Merchan, M Teresa Alvarez-Roman, Primitivo Gomez-Cardero, Victor Jimenez-Yuste","doi":"10.1080/17474086.2024.2380477","DOIUrl":"10.1080/17474086.2024.2380477","url":null,"abstract":"Introduction: The therapeutic approach to pain in hemophilia should be multimodal. Intra-articular injections are a good option when joint lesions do not respond to hematological treatment or rehabilitation and orthopedic surgery is not yet indicated. Performing these procedures under ultrasound guidance has been shown to improve their accuracy and efficacy.Areas covered: This article provides a practical overview of the most frequently employed ultrasound-guided intra-articular procedures on the joints of people with hemophilia. The article describes the key elements for performing the technique on the elbow, knee and ankle as the most affected joints. The particularities of the most frequent indications, arthrocentesis, synoviorthesis and analgesic injections with various products are detailed.Expert opinion: Current hematological treatments have made it possible to incorporate new therapeutic tools for pain relief for people with hemophilia, including ultrasound-guided joint procedures, which offer excellent results.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"419-430"},"PeriodicalIF":2.3,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141616202","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Quality of life considerations in myelodysplastic syndrome: not only fatigue. 骨髓增生异常综合征的生活质量考量：不仅仅是疲劳。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2024-08-01 Epub Date: 2024-07-12 DOI: 10.1080/17474086.2024.2378035

Pasquale Niscola, Valentina Gianfelici, Marco Giovannini, Daniela Piccioni, Carla Mazzone, Paolo de Fabritiis

引用次数: 0

Targeting Ikaros and Aiolos: reviewing novel protein degraders for the treatment of multiple myeloma, with a focus on iberdomide and mezigdomide. 以 ikaros 和 Aiolos 为靶点：回顾用于治疗多发性骨髓瘤的新型蛋白降解剂，重点关注 iberdomide 和 mezigdomide。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2024-08-01 Epub Date: 2024-07-27 DOI: 10.1080/17474086.2024.2382897

Yuxin Liu, Clifton C Mo, Monique A Hartley-Brown, Adam S Sperling, Shonali Midha, Andrew J Yee, Giada Bianchi, Catherine Piper, Alice Tattersall, Omar Nadeem, Jacob P Laubach, Paul G Richardson

{"title":"Targeting Ikaros and Aiolos: reviewing novel protein degraders for the treatment of multiple myeloma, with a focus on iberdomide and mezigdomide.","authors":"Yuxin Liu, Clifton C Mo, Monique A Hartley-Brown, Adam S Sperling, Shonali Midha, Andrew J Yee, Giada Bianchi, Catherine Piper, Alice Tattersall, Omar Nadeem, Jacob P Laubach, Paul G Richardson","doi":"10.1080/17474086.2024.2382897","DOIUrl":"10.1080/17474086.2024.2382897","url":null,"abstract":"Introduction: The treatment of multiple myeloma (MM) is evolving rapidly. Quadruplet regimens incorporating proteasome inhibitors, immunomodulatory drugs (IMiDs), and CD38 monoclonal antibodies have emerged as standard-of-care options for newly diagnosed MM, and numerous novel therapies have been approved for relapsed/refractory MM. However, there remains a need for novel options in multiple settings, including refractoriness to frontline standards of care.Areas covered: Targeting degradation of IKZF1 and IKZF3 - Ikaros and Aiolos - through modulation of cereblon, an E3 ligase substrate recruiter/receptor, is a key mechanism of action of the IMiDs and the CELMoD agents. Two CELMoD agents, iberdomide and mezigdomide, have demonstrated substantial preclinical and clinical activity in MM and have entered phase 3 investigation. Using a literature search methodology comprising searches of PubMed (unlimited time-frame) and international hematology/oncology conference abstracts (2019-2023), this paper reviews the importance of Ikaros and Aiolos in MM, the mechanism of action of the IMiDs and CELMoD agents and their relative potency for targeting Ikaros and Aiolos, and preclinical and clinical data on iberdomide and mezigdomide.Expert opinion: Emerging data suggest that iberdomide and mezigdomide have promising activity, including in IMiD-resistant settings and, pending phase 3 findings, may provide additional treatment options for patients with MM.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"445-465"},"PeriodicalIF":2.3,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141758126","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Optimizing salvage therapy for Hodgkin lymphoma: progress and future challenges. 优化霍奇金淋巴瘤的挽救疗法：进展与未来的挑战。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2024-08-01 Epub Date: 2024-06-27 DOI: 10.1080/17474086.2024.2372325

Chathuri Abeyakoon, John Kuruvilla

{"title":"Optimizing salvage therapy for Hodgkin lymphoma: progress and future challenges.","authors":"Chathuri Abeyakoon, John Kuruvilla","doi":"10.1080/17474086.2024.2372325","DOIUrl":"10.1080/17474086.2024.2372325","url":null,"abstract":"Introduction: Despite clear advancements in the management of classical Hodgkin lymphoma (cHL) over the past decade including better risk stratification, the usage of 18F-flurodeoxyglucose positron emission tomography (FDG-PET)-guided approaches and incorporation of novel agents, approximately one-third of the patients will relapse. Important themes have been recently explored in the first salvage setting including the recognition of the positive prognostic value of a negative pre-autologous stem cell transplantation (ASCT) FDG-PET response and the incorporation of novel agents such as brentuximab vedotin (BV) and immune checkpoint inhibitors (CPIs) as salvage regimens to improve patient outcomes.Areas covered: The evolving treatment paradigm in optimizing salvage therapy in relapsed refractory cHL (RR-cHL) is discussed, including a vision to the future. The methodology included a literature search on PubMed using keywords. Selected articles were screened and evaluated by the authors of this review.Expert opinion: Achieving a complete remission by FDG-PET pre-ASCT is the most important prognostic factor in obtaining disease control and subsequent cure, and therefore should be a key goal of any salvage regimen. Although data from randomized controlled trials are currently lacking, retrospective evidence demonstrate superior event free survival with CPI-based regimens compared to conventional chemotherapy or BV-based therapy.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"467-478"},"PeriodicalIF":2.3,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141445950","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0