Hematology. American Society of Hematology. Education Program最新文献_第3页

Patient-reported outcomes after CAR T-cell therapy in patients with hematological malignancies.

IF 2.9 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2024-12-06 DOI: 10.1182/hematology.2024000536

Xin Shelley Wang, Samer A Srour

{"title":"Patient-reported outcomes after CAR T-cell therapy in patients with hematological malignancies.","authors":"Xin Shelley Wang, Samer A Srour","doi":"10.1182/hematology.2024000536","DOIUrl":"10.1182/hematology.2024000536","url":null,"abstract":"The remarkable improvement in survival among individuals with hematological malignancies receiving chimeric antigen receptor (CAR) T-cell therapy has highlighted the growing unmet need to incorporate patient-centered assessments in management guidelines for these patients. That CAR T-cell therapy is associated with unique toxicities and relatively high symptom burden in the first few weeks after cell infusion is well known. Magnifying the patient's voice by using patient-reported outcomes (PROs) might support personalized intervention in the acute-care setting, optimize the use of medical resources, improve satisfaction with therapy, and enhance survival benefit. However, various factors impede PRO use in routine patient care: (1) the feasibility of PRO assessment during the acute phase of treatment, especially in patients experiencing neurological toxicities, is not well established; (2) although PROs are widely used in drug- development trials, the assessment tools used in clinical trials primarily inform quality-of-life or safety comparisons among study arms and are rarely the proper tools for assessing and capturing clinically meaningful adverse events that should be monitored in routine patient care; (3) PRO data that could guide how best to monitor and capture the delayed effects of CAR T-cell therapy in long-term survivors are limited. There is a pressing need to overcome these barriers to integrating evidence-based PROs into standard-of-care guidelines for patients receiving CAR T-cell therapy. In this review, we present the current state of PRO utilization in CAR T-cell therapy. We also discuss practical approaches and future directions for successful implementation of PROs in the care of patients receiving CAR T-cell therapy.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2024 1","pages":"102-108"},"PeriodicalIF":2.9,"publicationDate":"2024-12-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142791366","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"教育学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Ph+ ALL: new approaches for upfront therapy.

IF 2.9 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2024-12-06 DOI: 10.1182/hematology.2024000532

Marlise R Luskin

{"title":"Ph+ ALL: new approaches for upfront therapy.","authors":"Marlise R Luskin","doi":"10.1182/hematology.2024000532","DOIUrl":"10.1182/hematology.2024000532","url":null,"abstract":"Philadelphia chromosome-positive (Ph+) ALL is the most common genetic subtype of ALL and primarily affects adults. Ph+ ALL is characterized by the constitutively active ABL1 kinase and is resistant to conventional chemotherapy. Thus, Ph+ ALL was historically associated with a dismal prognosis, particularly among patients who did not undergo allogeneic hematopoietic stem cell transplantation (alloHCT) in first complete remission (CR). Imatinib, the first tyrosine kinase inhibitor (TKI) effective against ABL1, transformed the treatment and prognosis of Ph+ ALL, allowing more patients to achieve CR and become eligible for alloHCT, thereby improving outcomes. In recent years, there has been an improved understanding of the biology of Ph+ ALL, including recognition of distinct subtypes (multilineage and lymphoblast-only Ph+ ALL). There has also been a dramatic expansion of effective therapeutic and diagnostic tools for management of Ph+ ALL, including more potent TKIs, which have activity against ABL kinase-resistance mutations; refinement of the chemotherapy and alloHCT regimens that accompany TKI therapy; introduction of immunotherapy (blinatumomab); and better assays for measurable residual disease monitoring. This article reviews recent advancements and future directions for the initial treatment of Ph+ ALL in adults.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2024 1","pages":"78-85"},"PeriodicalIF":2.9,"publicationDate":"2024-12-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142791512","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"教育学","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 in India: the journey and hope for the future.

IF 2.9 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2024-12-06 DOI: 10.1182/hematology.2024000678

Kalpna Gupta, Lakshmanan Krishnamurti, Dipty Jain

{"title":"Sickle cell disease in India: the journey and hope for the future.","authors":"Kalpna Gupta, Lakshmanan Krishnamurti, Dipty Jain","doi":"10.1182/hematology.2024000678","DOIUrl":"10.1182/hematology.2024000678","url":null,"abstract":"India, the most populous nation in the world, also has a high frequency of the sickle hemoglobin (HbS) allele globally. The Arab Indian HbS haplotype in India is characterized by a relatively high percentage of fetal Hb, with widely varying frequencies of α-thalassemia. Hence, sickle cell disease (SCD) in India was perceived to be mild. Advances in the past decade in screening and SCD management have revealed that the severity of SCD in India is comparable to many other parts of the world. Clinical features in India include vaso-occlusive crisis, acute chest syndrome, avascular necrosis, renal involvement, stroke, etc, at a relatively young age. Once a fatal disease of childhood, the majority of patients born with SCD are expected to survive into adulthood, largely because of improvements in comprehensive care programs including newborn screening, penicillin prophylaxis, transcranial Doppler, and hydroxyurea therapy. Several centers are performing hematopoietic stem cell transplants successfully for SCD. To address the urgent need to control and manage SCD in India's population, the Government of India launched the National Sickle Cell Anaemia Elimination Mission, with significant funding for large-scale measures to screen, treat, counsel, educate, and develop technologies and novel therapies and gene therapies.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2024 1","pages":"1-9"},"PeriodicalIF":2.9,"publicationDate":"2024-12-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142791545","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"教育学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Targeted medical therapies for vascular anomalies.

IF 2.9 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2024-12-06 DOI: 10.1182/hematology.2024000599

Alexandra Borst

引用次数: 0

The evolving frontline management of CLL: are triplets better than doublets? How will we find out?

IF 2.9 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2024-12-06 DOI: 10.1182/hematology.2024000571

Kerry A Rogers, Jennifer A Woyach

{"title":"The evolving frontline management of CLL: are triplets better than doublets? How will we find out?","authors":"Kerry A Rogers, Jennifer A Woyach","doi":"10.1182/hematology.2024000571","DOIUrl":"10.1182/hematology.2024000571","url":null,"abstract":"Frontline therapy for chronic lymphocytic leukemia (CLL) has substantially advanced in the previous decade. While monotherapy with a Bruton's tyrosine kinase (BTK) inhibitor is an excellent option for many patients, combination therapies are of high clinical interest as they can induce deep responses and durable remissions, and in many cases allow discontinuation of therapy. There are several doublet therapies that are currently in clinical use. These include combinations of BTK inhibitors (ibrutinib, acalabrutinib, zanubrutinib) or BCL2 inhibitors (venetoclax) with anti-CD20 monoclonal antibodies, and combinations of BTK and BCL2 inhibitors. While BTK inhibitors with anti-CD20 monoclonal antibodies still typically require indefinite therapy, combinations involving the BCL2 inhibitor venetoclax have allowed for successful therapy discontinuation. Triplets, which combine all 3 of these paradigms, are of interest especially for patients with higher-risk disease. While triplets have been mainly studied in single-arm trials with excellent outcomes, comparative data to doublets are limited. In this article, we outline the doublet and triplet regimens that have been evaluated in CLL as well as the data from trials comparing doublets and triplets.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2024 1","pages":"467-473"},"PeriodicalIF":2.9,"publicationDate":"2024-12-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142791555","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"教育学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Through thick and thin: confronting the aggressive cutaneous T-cell lymphomas.

IF 2.9 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2024-12-06 DOI: 10.1182/hematology.2024000529

Robert Stuver, Steven M Horwitz

{"title":"Through thick and thin: confronting the aggressive cutaneous T-cell lymphomas.","authors":"Robert Stuver, Steven M Horwitz","doi":"10.1182/hematology.2024000529","DOIUrl":"10.1182/hematology.2024000529","url":null,"abstract":"The cutaneous T-cell lymphomas (CTCLs) comprise a diverse set of diseases with equally diverse presentations ranging from asymptomatic solitary lesions to highly aggressive diseases with propensity for visceral spread. The more aggressive CTCLs, which herein we consider as certain cases of advanced-stage mycosis fungoides/Sézary syndrome (MF/SS), primary cutaneous CD8+ aggressive epidermotropic cytotoxic T-cell lymphoma (PCAETCL), and primary cutaneous gamma delta T-cell lymphoma (PCGDTCL), require systemic therapy. Over the last 5 years, treatment options for MF/SS have expanded with biological insights leading to new therapeutic options and increasingly unique management strategies. An enhanced appreciation of the compartmental efficacy of these agents (skin, blood, lymph nodes, visceral organs) is incorporated in current management strategies in MF/SS. In addition, approaches that combine modalities in attempts to increase depth and durability of responses across multiple compartments are being trialed. In contrast to MF/SS, PCAETCL and PCGDTCL remain diseases with few prospective studies to guide treatment. However, recent genomic insights on these diseases, such as the presence of JAK2 fusions in PCAETCL and cell of origin findings in PCGDTCL, have created options for new biomarker-driven strategies.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2024 1","pages":"62-68"},"PeriodicalIF":2.9,"publicationDate":"2024-12-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142791616","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"教育学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Trousseau syndrome: management of refractory VTE.

IF 2.9 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2024-12-06 DOI: 10.1182/hematology.2024000552

Jeffrey I Zwicker

引用次数: 0

What is the ideal approach-doublet, triplet, or quadruplet(s)?

IF 2.9 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2024-12-06 DOI: 10.1182/hematology.2024000581

Shaji K Kumar

{"title":"What is the ideal approach-doublet, triplet, or quadruplet(s)?","authors":"Shaji K Kumar","doi":"10.1182/hematology.2024000581","DOIUrl":"10.1182/hematology.2024000581","url":null,"abstract":"Significant progress has been made in the treatment of multiple myeloma (MM), with the introduction of several new drugs with different mechanisms of action. The treatment of newly diagnosed MM has evolved dramatically with the development of highly effective combinations that include 1 or more of the new drugs. Despite the continuing improvement in the overall survival of patients with MM, nearly a quarter of the patients have significantly inferior survival, often driven by a combination of factors, including tumor genetics and host frailty. The focus of initial therapy remains rapid control of the disease with reversal of the symptoms and complications related to the disease with minimal toxicity and a reduction in early mortality. The selection of the specific regimen, to some extent, depends on the ability of the patient to tolerate the treatment and the underlying disease risk. It is typically guided by results of randomized clinical trials demonstrating improvements in progression-free and/or overall survival. While increasing risk calls for escalating the intensity of therapy by using quadruplet combinations that can provide the deepest possible response and the use of autologous stem cell transplant, increasing frailty calls for a reduction in the intensity and selective use of triplet or doublet regimens. The choice of subsequent consolidation treatments and maintenance approaches, including duration of treatment, also depends on these factors, particularly the underlying disease risk. The treatment approaches for newly diagnosed myeloma continue to evolve, with ongoing trials exploring bispecific antibodies as part of initial therapy and CAR T cells for consolidation.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2024 1","pages":"551-560"},"PeriodicalIF":2.9,"publicationDate":"2024-12-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142791656","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"教育学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Molecular landscape and classification of vascular anomalies.

IF 2.9 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2024-12-06 DOI: 10.1182/hematology.2024000598

Emmanuel Seront, Angela Queisser, Laurence M Boon, Miikka Vikkula

引用次数: 0

Givosiran: a targeted treatment for acute intermittent porphyria.

IF 2.9 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2024-12-06 DOI: 10.1182/hematology.2024000663

Amy K Dickey, Rebecca K Leaf

{"title":"Givosiran: a targeted treatment for acute intermittent porphyria.","authors":"Amy K Dickey, Rebecca K Leaf","doi":"10.1182/hematology.2024000663","DOIUrl":"10.1182/hematology.2024000663","url":null,"abstract":"The acute hepatic porphyrias (AHPs) are a family of rare genetic diseases associated with attacks of abdominal pain, vomiting, weakness, neuropathy, and other neurovisceral symptoms. Pathogenic variants in 1 of 4 enzymes of heme synthesis are necessary for the development of AHP, and the onset of acute attacks also requires the induction of δ-aminolevulinic acid synthase 1 (ALAS1), the first and rate-limiting step of heme synthesis in the liver. Givosiran is an RNA interference medication that inhibits hepatic ALAS1 and was designed to treat AHP. In 2019 the US Food and Drug Administration approved givosiran for AHP based on positive results from a phase 3 clinical trial of 94 patients with AHP who demonstrated a marked improvement in AHP attacks and a substantial decrease in δ-aminolevulinic acid and porphobilinogen, the primary disease markers of AHP. A long-term follow-up study demonstrated continued improvement in AHP attack rates, biochemical measures of disease, and quality of life. Real-world studies have also confirmed these results. Common side effects include injection site reactions, hyperhomocysteinemia, and abnormalities of liver and renal biochemistries. This article reviews the studies that led to givosiran approval, discusses real-world clinical data, and highlights remaining questions in the treatment of AHP.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2024 1","pages":"426-433"},"PeriodicalIF":2.9,"publicationDate":"2024-12-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142791500","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"教育学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0