Blood ReviewsPub Date : 2025-09-27DOI: 10.1016/j.blre.2025.101339
Ali Mushtaq, Asfand Yar Cheema, Hossam M Ali, Yara Shatnawi, Omer Ashruf, Eman Nayaz Ahmad, Olga Lytvynova, Mishaal Munir, Muhammad Anns Asif, Maheen Ahmad, Hamza Hassan, Abdullah M Khan, Tara Roy, Aneela Majeed, Shahzad Raza, Sandra Mazzoni, Louis Williams, Jack Khouri, Jason Valent, Christy Samaras, Joslyn Rudoni, Beth M Faiman, Mikhaila Rice, Jonathan Kissam, Diana Basali, Faiz Anwer
{"title":"Management of relapsed refractory multiple myeloma: Evidence-based guide to community oncologists.","authors":"Ali Mushtaq, Asfand Yar Cheema, Hossam M Ali, Yara Shatnawi, Omer Ashruf, Eman Nayaz Ahmad, Olga Lytvynova, Mishaal Munir, Muhammad Anns Asif, Maheen Ahmad, Hamza Hassan, Abdullah M Khan, Tara Roy, Aneela Majeed, Shahzad Raza, Sandra Mazzoni, Louis Williams, Jack Khouri, Jason Valent, Christy Samaras, Joslyn Rudoni, Beth M Faiman, Mikhaila Rice, Jonathan Kissam, Diana Basali, Faiz Anwer","doi":"10.1016/j.blre.2025.101339","DOIUrl":"https://doi.org/10.1016/j.blre.2025.101339","url":null,"abstract":"<p><p>Multiple myeloma (MM), a clonal plasma cell malignancy, presents a therapeutic challenge, especially in selecting therapy for patients with relapsed/refractory MM (RRMM). Up to 1-3 prior lines of therapy in this population are considered early relapse. This review provides clinicians with a guide for personalized, evidence-based strategies for treatment of early RRMM. Factors influencing treatment selection, including patient-related factors (e.g., frailty and comorbidities), disease characteristics (e.g., high-risk cytogenetics), prior therapy response, and toxicity profiles, are highlighted. We outline current and emerging transformative novel therapeutics, including anti-CD38 and anti-SLAMF7 monoclonal antibodies, BCMA-directed immunotherapies, such as CAR T-cells, and bispecific antibodies. The review highlights key clinical trials on efficacy (response rates, progression-free survival, overall survival) and safety profiles (e.g., cytokine release syndrome, neurotoxicity, and infections). Crucially, it provides a practical framework for clinical decision-making, including guidance on selecting between different combination regimens, immunotherapy platforms, and considering meaningful therapeutic endpoints and survival. Relapse management following BCMA-directed therapy and potential salvage strategies are outlined. Future directions include next-generation cellular therapies, novel antibody constructs, CELMoDs, and strategies to enhance immunotherapy outcomes.</p>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":" ","pages":"101339"},"PeriodicalIF":5.7,"publicationDate":"2025-09-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145201470","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 : 2025-09-06DOI: 10.1016/j.blre.2025.101338
Sigrid van der Veen, Judith J M Jans, Eduard J van Beers, Bart J Biemond, Pablo Bartolucci, Maria Paola Boaro, Anna Collado Gimbert, Raffaella Colombatti, Mirco D'Agnolo, Karin J Fijnvandraat, Amira Idrizovic, Petros Kountouris, Mar Mañú Pereira, Elisabetta Mezzalira, Minke A E Rab, Anita W Rijneveld, Tiziana Sanavia, Nanda M Verhoeven-Duif, Marjon H Cnossen
{"title":"Metabolomics in sickle cell disease: Current knowledge and gaps - A scoping review.","authors":"Sigrid van der Veen, Judith J M Jans, Eduard J van Beers, Bart J Biemond, Pablo Bartolucci, Maria Paola Boaro, Anna Collado Gimbert, Raffaella Colombatti, Mirco D'Agnolo, Karin J Fijnvandraat, Amira Idrizovic, Petros Kountouris, Mar Mañú Pereira, Elisabetta Mezzalira, Minke A E Rab, Anita W Rijneveld, Tiziana Sanavia, Nanda M Verhoeven-Duif, Marjon H Cnossen","doi":"10.1016/j.blre.2025.101338","DOIUrl":"https://doi.org/10.1016/j.blre.2025.101338","url":null,"abstract":"<p><p>Sickle cell disease (SCD) has large phenotypic variability. Systematic metabolomic profiling may provide insights into phenotypes and treatment responses. We conducted a scoping review on associations between blood metabolites, SCD-related complications, and therapies in studies analyzing ≥10 metabolites in red blood cells, whole blood, and plasma. Lipidomics-focused studies were excluded. Fifteen studies were included, focusing on metabolic profiling, clinical outcomes, or therapies (hydroxyurea, transfusion, and mitapivat). Metabolic profiling differentiated SCD from healthy controls and patients with HbSS and HbSC genotypes. Associations with hemolysis, vaso-occlusive events, nephropathy, TRV, and mortality were identified. Overall, metabolites were involved in arginine, tryptophan, glutamate metabolism, glycolysis, pentose phosphate pathway, and the Lands cycle. Some metabolites showed opposite correlations across complications or sample types. Despite growing interest, gaps remain in study designs, metabolite selection, genotype representation, and underexplored complications and therapies. Standardized, large-scale metabolomics studies are needed to advance personalized treatment in SCD.</p>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":" ","pages":"101338"},"PeriodicalIF":5.7,"publicationDate":"2025-09-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145058841","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 : 2025-08-25DOI: 10.1016/j.blre.2025.101337
Magdalena Kriel, Jessica Opie, Jody Rusch, David Richardson, Vernon Louw
{"title":"Old tests and new paradigms: How to interpret iron studies and related biomarkers for the diagnosis of iron deficiency in adults.","authors":"Magdalena Kriel, Jessica Opie, Jody Rusch, David Richardson, Vernon Louw","doi":"10.1016/j.blre.2025.101337","DOIUrl":"https://doi.org/10.1016/j.blre.2025.101337","url":null,"abstract":"<p><p>Clinicians need a good understanding of available tools to diagnose iron deficiency (ID). Interpretation of commonly used laboratory tests can be challenging due to the dynamic nature of iron homeostasis and concurrent inflammation, which influence results. The misinterpretation of iron studies, inconsistencies in ID diagnostic guidelines, and low awareness of non-anaemic ID may lead to missed diagnoses and opportunities for treatment. Serum ferritin (SF) is the most specific routine test for diagnosing ID, but should be interpreted in the correct context. Low SF levels provide a clearcut diagnosis, whilst normal and raised SF levels in the setting of inflammation require a nuanced diagnostic approach. Limited evidence to support diagnostic SF cut-offs and uncertainty surrounding SF reference intervals represent ongoing challenges. This paper aims to provide clarity on the utility and limitations of various laboratory tests for ID and to demonstrate their interpretation with practical examples.</p>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":" ","pages":"101337"},"PeriodicalIF":5.7,"publicationDate":"2025-08-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145008633","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 : 2025-08-15DOI: 10.1016/j.blre.2025.101329
Rohit Singh, Happy Agarwal, Ryan Bynum, Eli J Isenberg, Erdmann Mary, Glover Joshua, Molly McNally, Jorge Gomez-Gutierrez, Lacey McNally, Holter-Chakrabarty Jennifer
{"title":"DecodingAcute GVHD: The role of serum biomarkers and imaging in diagnosis and management.","authors":"Rohit Singh, Happy Agarwal, Ryan Bynum, Eli J Isenberg, Erdmann Mary, Glover Joshua, Molly McNally, Jorge Gomez-Gutierrez, Lacey McNally, Holter-Chakrabarty Jennifer","doi":"10.1016/j.blre.2025.101329","DOIUrl":"10.1016/j.blre.2025.101329","url":null,"abstract":"<p><p>Graft-versus-host disease (GVHD) is one of the major barriers to the clinical success of hematopoietic stem cell transplantation (HCT). Despite improvements in GVHD incidence with the use of post-transplant cyclophosphamide, cord blood transplantation, and improvement in donor identification and matching, it remains a major cause of death in HCT. Early and accurate identification of GVHD is required to improve HCT outcomes. Diagnosis ambiguity continues to be problematic, despite improvements in PCR based stool studies, immunohistochemical markers in tissue biopsy, and improvement in histopathological examination. Unfortunately, therefore, early diagnosis of GVHD suffers from the lack of established diagnostic biomarkers which could improve prompt identification and treatment of GVHD at a time when therapeutic outcome is most impactful. This review discusses available diagnostic serum biomarkers and imaging advancements available to predict onset, severity, and prognosis in GVHD. Additionally, this review highlights the differential diagnosis of GVHD from other related pathological conditions.</p>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":" ","pages":"101329"},"PeriodicalIF":5.7,"publicationDate":"2025-08-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12483143/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144980125","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}
Blood ReviewsPub Date : 2025-08-05DOI: 10.1016/j.blre.2025.101328
Bruno Fattizzo, Leona E Ling, Wilma Barcellini
{"title":"Targeting the neonatal Fc receptor (FcRn) in hematologic conditions with a focus on warm autoimmune hemolytic anemia.","authors":"Bruno Fattizzo, Leona E Ling, Wilma Barcellini","doi":"10.1016/j.blre.2025.101328","DOIUrl":"https://doi.org/10.1016/j.blre.2025.101328","url":null,"abstract":"<p><p>Warm autoimmune hemolytic anemia (wAIHA) is the most prevalent form of autoimmune hemolytic anemia, where in most patients extravascular hemolysis is driven by immunoglobulin G (IgG) autoantibodies, with or without complement activation. While standard-of-care treatment with corticosteroids provides a high initial response rate, relapses are frequent, and most patients require additional immunosuppressive therapies. A high unmet medical need remains for patients with refractory or chronic relapsing wAIHA. Neonatal Fc receptor (FcRn) blockers are novel biologic therapies designed to provide a rapid, sustained decrease in circulating concentrations of IgG antibodies, including autoantibodies, and have been investigated in hematologic conditions like immune thrombocytopenia and hemolytic anemia of the fetus and newborn and other autoimmune conditions, such as generalized myasthenia gravis. FcRn blockade is currently under evaluation in patients with wAIHA to determine its potential as a safe, effective treatment option.</p>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":" ","pages":"101328"},"PeriodicalIF":5.7,"publicationDate":"2025-08-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144857134","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 : 2025-08-01DOI: 10.1016/j.blre.2025.101289
Akihiro Ohmoto , Yoshiyuki Yamada , Shigeo Fuji
{"title":"Transplant-acquired allergy in HCT-recipients: Reference for clinical management","authors":"Akihiro Ohmoto , Yoshiyuki Yamada , Shigeo Fuji","doi":"10.1016/j.blre.2025.101289","DOIUrl":"10.1016/j.blre.2025.101289","url":null,"abstract":"<div><div><span>Transplant-acquired allergy (TAA) is well known in pediatric patients undergoing liver transplantation. Regarding allogeneic hematopoietic </span>cell transplantation<span><span> (allo-HCT), the clinical characteristics of TAA have not been fully elucidated. Clinical manifestations of TAA include eczematous dermatitis, </span>allergic rhinitis<span><span>, and asthma. It is known that allergic diseases are transferable from allergic donors to non-allergic recipients via allo-HCT. The potential mechanism is the transfer of allergen-specific memory B cells resulting in Th2-skewed allergy-specific immune responses. Retrospective studies have suggested that </span>cord blood transplantation<span><span> (CBT) and the immunosuppressant<span> tacrolimus have a significant impact on the development of TAA. The reported prevalence varies according to study design, diagnostic methods, and study population, and the suspected allergens vary widely among individuals. </span></span>Prospective cohort studies and further mechanistic investigations are warranted to provide robust evidence on the prevalence and risk factors associated with TAA.</span></span></span></div></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"73 ","pages":"Article 101289"},"PeriodicalIF":5.7,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144051176","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 : 2025-08-01DOI: 10.1016/j.blre.2025.101300
Tomás José González-López , Drew Provan
{"title":"The new era of primary immune thrombocytopenia management in adults: A narrative review of current and emerging treatments","authors":"Tomás José González-López , Drew Provan","doi":"10.1016/j.blre.2025.101300","DOIUrl":"10.1016/j.blre.2025.101300","url":null,"abstract":"<div><div><span>The purpose of this review is to highlight the treatments currently available and those under- going evaluation in clinical trials for the treatment of ITP in order to achieve optimal use of the various existing ITP treatments. Specifically, we point out the indications for use of the various therapies available: corticosteroids, intravenous immunoglobulins (IVIG), thrombo- poietic agents (TPO-RAs), </span>Syk inhibitors<span><span>: Fostamatinib<span>, antiCD20 monoclonal antibodies i.e. </span></span>rituximab<span><span> and the use of splenectomy in ITP. A review of the use of new drugs in ITP is also included in our manuscript: Neonatal Fc receptor (FcRn) antagonists; </span>Bruton tyrosine kinase (BTK) inhibition; B-cell activating factor (BAFF) pathway inhibition; plasma cell depletion (an- tiCD38 monoclonal antibodies); new Syk inhibitors and complement inhibition. We believe that a reader with little knowledge of ITP can gain a clear understanding of the current treatment of ITP and its more or less immediate treatment prospects.</span></span></div></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"73 ","pages":"Article 101300"},"PeriodicalIF":5.7,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144082283","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 : 2025-08-01DOI: 10.1016/j.blre.2025.101288
Raffaele Palmieri , Anna Candoni , Francesco Di Raimondo , Giuseppe Rossi , Massimo Breccia , Fabrizio Pane , Paola Volpicelli , Benedetta Neri , Paola Finsinger , Morena Caira , Felicetto Ferrara
{"title":"Navigating acute myeloid leukemia towards better outcomes: Treatment pathways and challenges for patients ineligible for intensive chemotherapy","authors":"Raffaele Palmieri , Anna Candoni , Francesco Di Raimondo , Giuseppe Rossi , Massimo Breccia , Fabrizio Pane , Paola Volpicelli , Benedetta Neri , Paola Finsinger , Morena Caira , Felicetto Ferrara","doi":"10.1016/j.blre.2025.101288","DOIUrl":"10.1016/j.blre.2025.101288","url":null,"abstract":"<div><div>Acute myeloid leukemia (AML) is an aggressive hematologic malignancy that affects primarily older individuals. Patients ineligible to receive intensive standard chemotherapy followed by consolidation with/without hematopoietic stem cell transplant have a suboptimal prognosis. In recent years, significant advances have been made in the AML field leading to the development of new anti-leukemic approaches, including lower-intensity therapies specifically developed for patients who are ineligible for intensive chemotherapy. As the available options for this hard-to-manage and historically undertreated patient category are increasing, selecting the best treatment for each patient is crucial and ever more challenging. Accordingly, accurate patient evaluation is required to guide this decision-making process. There is currently no consensus on how to evaluate patients' fitness status, and the available tools that were originally developed for this purpose might not be adequate in the setting of the new treatment options. In this review we describe current management of AML patients unfit for intensive chemotherapy, aiming to highlight current challenges and suggest possible strategies for an accurate therapeutic selection. For this purpose, we will first provide an overview of epidemiology and classification of AML, and then move to current anti-leukemic treatments for unfit patients and the tools used for evaluating patient eligibility for a specific treatment. Finally, we will suggest possible measures to improve the management of AML patients in the era of novel lower-intensity regimens.</div></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"73 ","pages":"Article 101288"},"PeriodicalIF":5.7,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144065222","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 : 2025-08-01DOI: 10.1016/j.blre.2025.101298
Mohamed Yassin , Caterina Minniti , Nirmish Shah , Salam Alkindi , Fateen Ata , Mohammed Qari , Abdullah Al Zayed , Jaffer Altooq , Mona Al Rasheed , Maria Domenica Capellini
{"title":"Evidence and gaps in clinical outcomes of novel pharmacologic therapies for sickle cell disease: A systematic literature review highlighting insights from clinical trials and real-world studies","authors":"Mohamed Yassin , Caterina Minniti , Nirmish Shah , Salam Alkindi , Fateen Ata , Mohammed Qari , Abdullah Al Zayed , Jaffer Altooq , Mona Al Rasheed , Maria Domenica Capellini","doi":"10.1016/j.blre.2025.101298","DOIUrl":"10.1016/j.blre.2025.101298","url":null,"abstract":"<div><div>This systematic review aims to summarise the clinical outcomes of <span>l</span>-glutamine, crizanlizumab, and voxelotor in the treatment of sickle cell disease (SCD) based on clinical trials and real-world data and to identify any gaps in the observations. The review identified 97 studies reporting data until 31 May 2024.</div><div>A pivotal phase III study of <span>l</span>-glutamine showed that patients treated with <span>l</span>-glutamine had a 25 % reduction in pain crises and 33 % fewer hospital days compared to placebo. <span>l</span>-glutamine was generally well tolerated with minimal side effects. Real-world studies of <span>l</span>-glutamine emphasize patient adherence and obstacles to medication accessibility and approval as key concerns. In the SUSTAIN study, a 5-mg/kg dose of crizanlizumab reduced the occurrence of vaso-occlusive crises (VOCs) and hospitalizations by 45 % and 41 %, respectively. Real-world studies of crizanlizumab showed a reduction in complicated VOC events. The high discontinuation rate and results of the STAND trial led to a significant decrease in the use of crizanlizumab. The HOPE trial demonstrated a 51 % improvement in hemoglobin response and a reduction in hemolytic markers in patients treated with voxelotor. While some real-world studies have reported a decrease in VOCs and hospitalizations, the results are inconsistent and not conclusive. Further studies are needed to assess the impact of these novel therapies on end-organ-specific complications of SCD.</div></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"73 ","pages":"Article 101298"},"PeriodicalIF":5.7,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143998774","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 : 2025-08-01DOI: 10.1016/j.blre.2025.101299
Anam Ashfaque , Yara Shatnawi , Shahzad Raza , Diana Basali , Jack Khouri , Louis Williams , Sandra Mazzoni , Christy Samaras , Hamza Hassan , Utkarsh Acharya , Chakra Chaulagain , Faiz Anwer
{"title":"Suppression to removal, an emerging therapeutic approach for AL amyloidosis: A comprehensive review with early human data and pharmacokinetics of CAEL-101 antibody","authors":"Anam Ashfaque , Yara Shatnawi , Shahzad Raza , Diana Basali , Jack Khouri , Louis Williams , Sandra Mazzoni , Christy Samaras , Hamza Hassan , Utkarsh Acharya , Chakra Chaulagain , Faiz Anwer","doi":"10.1016/j.blre.2025.101299","DOIUrl":"10.1016/j.blre.2025.101299","url":null,"abstract":"<div><div>Light chain (AL amyloidosis) is a rare disorder characterized by the deposition of misfolded light chains in various organs, causing progressive organ damage. Current therapeutic agents do not remove amyloid aggregates already present in the organs, which are the major determinants of morbidity and mortality. Therefore, drugs targeting amyloid fibrils are currently being investigated. This article provides a comprehensive review of a novel, fibril-directed antibody, CAEL-101 (Anselamimab), which removes fibrillary aggregates from the organs, restoring organ function. Overall, CAEL-101 has demonstrated a favorable toxicity profile and improved organ responses, with faster treatment response time than current therapies in phase I/II trials, and is expected to have promising outcomes in the ongoing phase III studies. Combining anti-plasma cell dyscrasia (suppression) and fibril-directed agents (removal) is a novel therapeutic approach, providing optimism for organ function recovery and enhanced quality of life and survival, especially in patients with severe diseases.</div></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"73 ","pages":"Article 101299"},"PeriodicalIF":5.7,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144096009","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}