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Management of Patients With Cytogenetically Normal Acute Myeloid Leukemia Who Have Neither Favorable nor Unfavorable Markers

Alison R. Walker and Guido Marcucci

The presence or absence of cytogenetic and molecular abnormalities present at the time of diagnosis of acute myeloid leukemia (AML) not only provides important prognostic information, but also directs decisions regarding postremission therapy. In no other group has molecular analysis been more important than for the 40% to 50% of newly diagnosed patients in whom clonal chromosomal aberrations are not detected. Patients with cytogenetically normal (CN) AML were once considered a homogenous group, but are now classified into molecularly defined subgroups with distinct clinical outcomes. Evaluating FLT3, NPM1, and CEBPA mutational status is a routine component of the diagnostic evaluation for all patients with CN-AML and is used to determine outcome risk. However, in patients with FLT3 wild-type/NPM1 wild-type/CEBPA wild-type CN-AML, the optimal postremission therapy has not been well defined. This article reviews treatment outcomes for this group of patients after chemotherapy and autologous and allogeneic stem cell transplantation. New recurrent somatic mutations and their prognostic significance in patients with FLT3 wild-type/NPM1 wild-type CN-AML are also addressed.

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NCCN Task Force Report: Evaluating the Clinical Utility of Tumor Markers in Oncology

Phillip G. Febbo, Marc Ladanyi, Kenneth D. Aldape, Angelo M. De Marzo, M. Elizabeth Hammond, Daniel F. Hayes, A. John Iafrate, R. Kate Kelley, Guido Marcucci, Shuji Ogino, William Pao, Dennis C. Sgroi, and Marian L. Birkeland

The molecular analysis of biomarkers in oncology is rapidly advancing, but the incorporation of new molecular tests into clinical practice will require a greater understanding of the genetic changes that drive malignancy, the assays used to measure the resulting phenotypes and genotypes, and the regulatory processes that new molecular biomarkers must face to be accepted for clinical use. To address these issues and provide an overview of current molecular testing in 6 major malignancies, including glioma, breast cancer, colon cancer, lung cancer, prostate cancer, and acute myelogenous leukemia, an NCCN Task Force was convened on the topic of evaluating the clinical utility of tumor markers in oncology. The output of this meeting, contained within this report, describes the ways biomarkers have been developed and used; defines common terminology, including prognostic, predictive, and companion diagnostic markers, and analytic validity, clinical validity, and clinical utility; and proposes the use of a combination level of evidence score to aid in the evaluation of novel biomarker tests as they arise. The current state of regulatory oversight and anticipated changes in the regulation of molecular testing are also addressed.

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Acute Myeloid Leukemia, Version 2.2013

Margaret R. O’Donnell, Martin S. Tallman, Camille N. Abboud, Jessica K. Altman, Frederick R. Appelbaum, Daniel A. Arber, Eyal Attar, Uma Borate, Steven E. Coutre, Lloyd E. Damon, Jeffrey Lancet, Lori J. Maness, Guido Marcucci, Michael G. Martin, Michael M. Millenson, Joseph O. Moore, Farhad Ravandi, Paul J. Shami, B. Douglas Smith, Richard M. Stone, Stephen A. Strickland, Eunice S. Wang, Kristina M. Gregory, and Maoko Naganuma

These NCCN Guidelines Insights summarize several key updates to the NCCN Guidelines for Acute Myeloid Leukemia and discuss the clinical evidence that support the recommendations. The updates described in this article focus on the acute promyelocytic leukemia (APL) section, featuring recommendations for additional induction/consolidation regimens in patients with low- or intermediate-risk APL, and providing guidance on maintenance strategies for APL.

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Acute Myeloid Leukemia

Margaret R. O'Donnell, Camille N. Abboud, Jessica Altman, Frederick R. Appelbaum, Daniel A. Arber, Eyal Attar, Uma Borate, Steven E. Coutre, Lloyd E. Damon, Salil Goorha, Jeffrey Lancet, Lori J. Maness, Guido Marcucci, Michael M. Millenson, Joseph O. Moore, Farhad Ravandi, Paul J. Shami, B. Douglas Smith, Richard M. Stone, Stephen A. Strickland, Martin S. Tallman, Eunice S. Wang, Maoko Naganuma, and Kristina M. Gregory

Acute myeloid leukemia (AML) remains the most common form of acute leukemia among adults and accounts for the largest number of annual deaths due to leukemias in the United States. The NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for AML provide recommendations on the diagnostic evaluation and workup for AML, risk assessment based on cytogenetic and molecular features, treatment options for induction and consolidation therapies for younger and older (age ≥ 65 years) adult patients, and key supportive care considerations.

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Acute Myeloid Leukemia

Margaret R. O'Donnell, Camille N. Abboud, Jessica Altman, Frederick R. Appelbaum, Steven E. Coutre, Lloyd E. Damon, James M. Foran, Salil Goorha, Lori J. Maness, Guido Marcucci, Peter Maslak, Michael M. Millenson, Joseph O. Moore, Farhad Ravandi, Paul J. Shami, B. Douglas Smith, Richard M. Stone, Stephen A. Strickland, Martin S. Tallman, and Eunice S. Wang

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Acute Myeloid Leukemia, Version 3.2017, NCCN Clinical Practice Guidelines in Oncology

Margaret R. O'Donnell, Martin S. Tallman, Camille N. Abboud, Jessica K. Altman, Frederick R. Appelbaum, Daniel A. Arber, Vijaya Bhatt, Dale Bixby, William Blum, Steven E. Coutre, Marcos De Lima, Amir T. Fathi, Melanie Fiorella, James M. Foran, Steven D. Gore, Aric C. Hall, Patricia Kropf, Jeffrey Lancet, Lori J. Maness, Guido Marcucci, Michael G. Martin, Joseph O. Moore, Rebecca Olin, Deniz Peker, Daniel A. Pollyea, Keith Pratz, Farhad Ravandi, Paul J. Shami, Richard M. Stone, Stephen A. Strickland, Eunice S. Wang, Matthew Wieduwilt, Kristina Gregory, and Ndiya Ogba

Acute myeloid leukemia (AML) is the most common form of acute leukemia among adults and accounts for the largest number of annual deaths due to leukemias in the United States. This portion of the NCCN Guidelines for AML focuses on management and provides recommendations on the workup, diagnostic evaluation, and treatment options for younger (age <60 years) and older (age ≥60 years) adult patients.

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Acute Myeloid Leukemia, Version 3.2019, NCCN Clinical Practice Guidelines in Oncology

Martin S. Tallman, Eunice S. Wang, Jessica K. Altman, Frederick R. Appelbaum, Vijaya Raj Bhatt, Dale Bixby, Steven E. Coutre, Marcos De Lima, Amir T. Fathi, Melanie Fiorella, James M. Foran, Aric C. Hall, Meagan Jacoby, Jeffrey Lancet, Thomas W. LeBlanc, Gabriel Mannis, Guido Marcucci, Michael G. Martin, Alice Mims, Margaret R. O’Donnell, Rebecca Olin, Deniz Peker, Alexander Perl, Daniel A. Pollyea, Keith Pratz, Thomas Prebet, Farhad Ravandi, Paul J. Shami, Richard M. Stone, Stephen A. Strickland, Matthew Wieduwilt, Kristina M. Gregory, OCN, Lydia Hammond, and Ndiya Ogba

Acute myeloid leukemia (AML) is the most common form of acute leukemia among adults and accounts for the largest number of annual deaths due to leukemias in the United States. Recent advances have resulted in an expansion of treatment options for AML, especially concerning targeted therapies and low-intensity regimens. This portion of the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for AML focuses on the management of AML and provides recommendations on the workup, diagnostic evaluation and treatment options for younger (age <60 years) and older (age ≥60 years) adult patients.

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Acute Myeloid Leukemia, Version 3.2023, NCCN Clinical Practice Guidelines in Oncology

Daniel A. Pollyea, Jessica K. Altman, Rita Assi, Dale Bixby, Amir T. Fathi, James M. Foran, Ivana Gojo, Aric C. Hall, Brian A. Jonas, Ashwin Kishtagari, Jeffrey Lancet, Lori Maness, James Mangan, Gabriel Mannis, Guido Marcucci, Alice Mims, Kelsey Moriarty, Moaath Mustafa Ali, Jadee Neff, Reza Nejati, Rebecca Olin, Mary-Elizabeth Percival, Alexander Perl, Amanda Przespolewski, Dinesh Rao, Farhad Ravandi, Rory Shallis, Paul J. Shami, Eytan Stein, Richard M. Stone, Kendra Sweet, Swapna Thota, Geoffrey Uy, Pankit Vachhani, Carly J. Cassara, Deborah A. Freedman-Cass, and Katie Stehman

Acute myeloid leukemia (AML) is a heterogeneous hematologic malignancy characterized by the clonal expansion of myeloid blasts in the peripheral blood, bone marrow, and/or other tissues. It is the most common form of acute leukemia among adults and accounts for the largest number of annual deaths from leukemias in the United States. Like AML, blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a myeloid malignancy. It is a rare malignancy characterized by the aggressive proliferation of precursors of plasmacytoid dendritic cells that frequently involves the bone marrow, skin, central nervous system, and other organs and tissues. This discussion section focuses on the diagnosis and management of BPDCN as outlined in the NCCN Guidelines for AML.

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NCCN Guidelines Insights: Acute Myeloid Leukemia, Version 2.2021

Featured Updates to the NCCN Guidelines

Daniel A. Pollyea, Dale Bixby, Alexander Perl, Vijaya Raj Bhatt, Jessica K. Altman, Frederick R. Appelbaum, Marcos de Lima, Amir T. Fathi, James M. Foran, Ivana Gojo, Aric C. Hall, Meagan Jacoby, Jeffrey Lancet, Gabriel Mannis, Guido Marcucci, Michael G. Martin, Alice Mims, Jadee Neff, Reza Nejati, Rebecca Olin, Mary-Elizabeth Percival, Thomas Prebet, Amanda Przespolewski, Dinesh Rao, Farhad Ravandi-Kashani, Paul J. Shami, Richard M. Stone, Stephen A. Strickland, Kendra Sweet, Pankit Vachhani, Matthew Wieduwilt, Kristina M. Gregory, Ndiya Ogba, and Martin S. Tallman

The NCCN Guidelines for Acute Myeloid Leukemia (AML) provide recommendations for the diagnosis and treatment of adults with AML based on clinical trials that have led to significant improvements in treatment, or have yielded new information regarding factors with prognostic importance, and are intended to aid physicians with clinical decision-making. These NCCN Guidelines Insights focus on recent select updates to the NCCN Guidelines, including familial genetic alterations in AML, postinduction or postremission treatment strategies in low-risk acute promyelocytic leukemia or favorable-risk AML, principles surrounding the use of venetoclax-based therapies, and considerations for patients who prefer not to receive blood transfusions during treatment.