Blood ReviewsPub Date : 2024-09-04DOI: 10.1016/j.blre.2024.101240
Jean Escal, Geraldine Poenou, Xavier Delavenne, Souad Bezzeghoud, Valentine Mismetti, Marc Humbert, David Montani, Laurent Bertoletti
{"title":"Tailoring oral anticoagulant treatment in the era of multi-drug therapies for PAH and CTEPH.","authors":"Jean Escal, Geraldine Poenou, Xavier Delavenne, Souad Bezzeghoud, Valentine Mismetti, Marc Humbert, David Montani, Laurent Bertoletti","doi":"10.1016/j.blre.2024.101240","DOIUrl":"https://doi.org/10.1016/j.blre.2024.101240","url":null,"abstract":"<p><p>The use of oral anticoagulants in the management of pulmonary arterial hypertension (PAH) and chronic thromboembolic pulmonary hypertension (CTEPH) presents distinct therapeutic challenges and benefits. In PAH, the benefits of oral anticoagulation are uncertain, with studies yielding mixed results on their efficacy and safety. Conversely, oral anticoagulants are a cornerstone in the treatment of CTEPH, where their use is consistently recommended to prevent recurrent thromboembolic events. The choice between vitamin K antagonists (VKAs) and direct oral anticoagulants (DOACs) remains a significant clinical question, as each type presents advantages and potential drawbacks. Furthermore, drug-drug interactions (DDIs) with concomitant PAH and CTEPH treatments complicate anticoagulant management, necessitating careful consideration of individual patient regimens. This review examines the current evidence on oral anticoagulant use in PAH and CTEPH and discusses the implications of DDIs within a context of multi-drug treatments, including targeted drugs in PAH.</p>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":6.9,"publicationDate":"2024-09-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142156777","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Blood ReviewsPub Date : 2024-08-29DOI: 10.1016/j.blre.2024.101238
Dahniel Sastow, Hannah Levavi, Nicole Wagner, Keith Pratz, Douglas Tremblay
{"title":"Ven the dose matters: Venetoclax dosing in the frontline treatment of AML.","authors":"Dahniel Sastow, Hannah Levavi, Nicole Wagner, Keith Pratz, Douglas Tremblay","doi":"10.1016/j.blre.2024.101238","DOIUrl":"https://doi.org/10.1016/j.blre.2024.101238","url":null,"abstract":"<p><p>Older/unfit adults with AML have worse outcomes and fewer treatment options than their younger/fit counterparts. In vitro studies have found a synergistic effect of hypomethylating agents (HMA) with venetoclax (VEN) on AML cells and since the phase 3 VIALE-A trial demonstrated a survival benefit, HMA + VEN has become the standard of care in the frontline setting for older/unfit adults with AML. Unfortunately, the standard 28-day cycle of VEN is associated with a high degree of myelosuppression leading to treatment delays and dose modifications. Many small retrospective studies have successfully shown comparable outcomes to VIALE-A with reduced dose/duration of VEN. Furthermore, low dose metronomic dosing of HMA + VEN has shown clinical benefit while minimizing myelotoxicity. Future trials are vital to understand the appropriate dose of VEN in combination with HMA, to evaluate HMA + VEN compared to intensive therapy for younger/fit patients, and to explore its utility in the relapsed/refractory setting.</p>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":6.9,"publicationDate":"2024-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142115238","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Blood ReviewsPub Date : 2024-08-20DOI: 10.1016/j.blre.2024.101229
Mobil Akhmedov, J Luis Espinoza
{"title":"Addressing the surge of infections by multidrug-resistant Enterobacterales in hematopoietic cell transplantation.","authors":"Mobil Akhmedov, J Luis Espinoza","doi":"10.1016/j.blre.2024.101229","DOIUrl":"https://doi.org/10.1016/j.blre.2024.101229","url":null,"abstract":"<p><p>Patients undergoing hematopoietic cell transplantation (HCT) have an increased risk of developing severe infections. In recent years, bloodstream infections caused by Gram-negative bacteria have been increasingly reported among HCT recipients, and many of these infections are caused by bacterial strains of the Enterobacterales order. Among these pathogens, particularly concerning are the multidrug-resistant Enterobacterales (MDRE), such as Extended Spectrum β-lactamase-producing Enterobacterales and Carbapenem-resistant Enterobacterales, since infections caused by these pathogens are difficult to treat due to the limited antimicrobial options and are associated with worse transplant outcomes. We summarized the evidence from studies published in PubMed and Scopus on the burden of MDRE infections in HCT recipients, and strategies for the management and prevention of these infections, including strict adherence to recommended infection control practices and multidisciplinary antimicrobial stewardship, the use of probiotics, and fecal microbiota transplantation, are also discussed.</p>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":6.9,"publicationDate":"2024-08-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142115237","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Blood ReviewsPub Date : 2024-08-10DOI: 10.1016/j.blre.2024.101227
Marianela Iriarte-Gahete, Laura Tarancon-Diez, Vanesa Garrido-Rodríguez, Manuel Leal, Yolanda María Pacheco
{"title":"Absolute and functional iron deficiency: Biomarkers, impact on immune system, and therapy.","authors":"Marianela Iriarte-Gahete, Laura Tarancon-Diez, Vanesa Garrido-Rodríguez, Manuel Leal, Yolanda María Pacheco","doi":"10.1016/j.blre.2024.101227","DOIUrl":"https://doi.org/10.1016/j.blre.2024.101227","url":null,"abstract":"<p><p>Iron is essential for numerous physiological processes and its deficiency often leads to anemia. Iron deficiency (ID) is a global problem, primarily affecting reproductive-age women and children, especially in developing countries. Diagnosis uses classical biomarkers like ferritin or transferrin saturation. Recent advancements include using soluble transferrin receptor (sTfR) or hepcidin for improved detection and classification of absolute and functional iron deficiencies, though mostly used in research. ID without anemia may present symptoms like asthenia and fatigue, even without relevant clinical consequences. ID impacts not only red-blood cells but also immune system cells, highlighting its importance in global health and immune-related comorbidities. Managing ID, requires addressing its cause and selecting appropriate iron supplementation. Various improved oral and intravenous products are available, but further research is needed to refine treatment strategies. This review updates on absolute and functional iron deficiencies, their relationships with the immune system and advancements in diagnosis and therapies.</p>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":6.9,"publicationDate":"2024-08-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141984022","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Blood ReviewsPub Date : 2024-08-10DOI: 10.1016/j.blre.2024.101228
Patrick Hagen, Anita D'Souza
{"title":"Autologous stem cell transplantation in AL amyloidosis: Muddy waters.","authors":"Patrick Hagen, Anita D'Souza","doi":"10.1016/j.blre.2024.101228","DOIUrl":"https://doi.org/10.1016/j.blre.2024.101228","url":null,"abstract":"<p><p>Immunoglobulin light chain (AL) amyloidosis is a malignant plasma cell dyscrasia causing multi-organ morbidity. High dose melphalan and autologous stem cell transplantation (ASCT) is a preferred consolidation approach and is safe with improved patient selection criteria. With the advent of bortezomib and daratumumab based induction therapy, nearly all patients can achieve deep hematological responses but follow up for daratumumab based induction is short. Consequently, the traditional approach of induction followed by ASCT is called into question. Given the multi-organ involvement of AL, endpoints beyond depth of response and hematological progression free survival (PFS) are important. Major organ dysfunction PFS (MOD-PFS) adds to PFS and is a composite endpoint of PFS, renal and cardiac organ progression, and overall survival. It is currently unknown which consolidative approach (ASCT or non-ASCT) will generate improved outcomes across the MOD-PFS spectrum a question the recently opened S2213 trial will attempt to answer.</p>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":6.9,"publicationDate":"2024-08-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142047605","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Measurable residual disease (MRD)-testing in haematological cancers: A giant leap forward or sideways?","authors":"Qiujin Shen, Xiaowen Gong, Yahui Feng, Yu Hu, Tiantian Wang, Wen Yan, Wei Zhang, Saibing Qi, Robert Peter Gale, Junren Chen","doi":"10.1016/j.blre.2024.101226","DOIUrl":"10.1016/j.blre.2024.101226","url":null,"abstract":"<p><p>Measurable residual disease (MRD)-testing is used in many haematological cancers to estimate relapse risk and to direct therapy. Sometimes MRD-test results are used for regulatory approval. However, some people including regulators wrongfully believe results of MRD-testing are highly accurate and of proven efficacy in directing therapy. We review MRD-testing technologies and evaluate the accuracy of MRD-testing for predicting relapse and the strength of evidence supporting efficacy of MRD-guided therapy. We show that at the individual level MRD-test results are often an inaccurate relapse predictor. Also, no convincing data indicate that increasing therapy-intensity based on a positive MRD-test reduces relapse risk or improves survival. We caution against adjusting therapy-intensity based solely on results of MRD-testing.</p>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":6.9,"publicationDate":"2024-08-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142010021","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Blood ReviewsPub Date : 2024-08-02DOI: 10.1016/j.blre.2024.101225
Alex Hoover, Lucie M Turcotte, Rachel Phelan, Crystal Barbus, Arpana Rayannavar, Bradley S Miller, Erin E Reardon, Nicole Theis-Mahon, Margaret L MacMillan
{"title":"Longitudinal clinical manifestations of Fanconi anemia: A systematized review.","authors":"Alex Hoover, Lucie M Turcotte, Rachel Phelan, Crystal Barbus, Arpana Rayannavar, Bradley S Miller, Erin E Reardon, Nicole Theis-Mahon, Margaret L MacMillan","doi":"10.1016/j.blre.2024.101225","DOIUrl":"https://doi.org/10.1016/j.blre.2024.101225","url":null,"abstract":"<p><p>Fanconi anemia (FA) is a rare and complex inherited genetic disorder characterized by impaired DNA repair mechanisms leading to genomic instability. Individuals with FA have increased susceptibility to congenital anomalies, progressive bone marrow failure, leukemia and malignant tumors, endocrinopathies and other medical issues. In recent decades, steadily improved approaches to hematopoietic cell transplantation (HCT), the only proven curative therapy for the hematologic manifestations of FA, have significantly increased the life expectancy of affected individuals, illuminating the need to understand the long-term consequences and multi-organ ramifications. Utilizing a systematized review approach with narrative synthesis of each primary issue and organ system, we shed light on the challenges and opportunities for optimizing the care and quality of life for individuals with FA and identify knowledge gaps informing future research directions.</p>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":6.9,"publicationDate":"2024-08-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141899019","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Blood ReviewsPub Date : 2024-07-10DOI: 10.1016/j.blre.2024.101223
{"title":"Current challenges in conditioning regimens for MDS transplantation","authors":"","doi":"10.1016/j.blre.2024.101223","DOIUrl":"10.1016/j.blre.2024.101223","url":null,"abstract":"<div><p>Myelodysplastic syndrome (MDS) is a very heterogeneous clonal disorder. Patients with “higher-risk” MDS, defined by specific recurrent genetic abnormalities, have a poor prognosis because of a high risk of progression to secondary acute myeloid leukemia with low chemosensitivity. Allogeneic hematopoietic stem cell transplantation remains the only treatment that offers durable disease control because the donor immune system allows graft-versus-MDS effects. In terms of preparation steps before transplantation, targeting the malignant clone by increasing the conditioning regimen intensity is still a matter of intense debate. MDS is mainly diagnosed in older patients, and high toxicity related to common myeloablative conditioning regimens has been reported. Efforts to include new drugs in the conditioning regimen to achieve the best malignant clone control without increasing toxicity have been made over the past 20 years. We summarized these retrospective and prospective studies and evaluated the limitations of the available evidence to delineate the ideal conditioning regimen.</p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":6.9,"publicationDate":"2024-07-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0268960X24000560/pdfft?md5=b28710cbdba2c6aa48108ac802eca6b2&pid=1-s2.0-S0268960X24000560-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141700122","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}