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8. Safety and efficacy of CPX-351 in younger patients (

Author

Przespolewski, Amanda, Goldberg, Aaron D., Talati, Chetasi, Fazal, Salman, Vachhani, Pankit, Sanikommu, Srinivasa R., Thota, Swapna, Waksal, Julian, Ball, Brian, Famulare, Christopher, Stahl, Maximilian, Baron, Jeffrey, Griffiths, Elizabeth A., Thompson, James E., Sweet, Kendra, and Wang, Eunice S.

Published
2023
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9. Safety and efficacy of CPX-351 in younger patients (< 60 years) with secondary acute myeloid leukemia

Author

Przespolewski, Amanda, primary, Goldberg, Aaron D, additional, Talati, Chetasi, additional, Fazal, Salman, additional, Vachhani, Pankit, additional, Sanikommu, Srinivasa Reddy, additional, Thota, Swapna, additional, Waksal, Julian A., additional, Ball, Brian John, additional, Famulare, Christopher A, additional, Stahl, Maximilian, additional, Baron, Jeffrey, additional, Griffiths, Elizabeth A, additional, Thompson, James E., additional, Sweet, Kendra L, additional, and Wang, Eunice S., additional

Published
2022
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11. Clinical Utility of Fedratinib in Myelofibrosis

Author

Waksal,Julian A, Tremblay,Douglas, and Mascarenhas,John

Subjects
OncoTargets and Therapy
Abstract

Julian A Waksal, Douglas Tremblay, John Mascarenhas Department of Hematology and Oncology, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USACorrespondence: John Mascarenhas Email John.Mascarenhas@mssm.eduAbstract: Myelofibrosis (MF) is a clonal hematologic malignancy characterized by bone marrow fibrosis, extramedullary hematopoiesis, splenomegaly, and constitutional symptoms with a propensity towards leukemic transformation. Constitutive activation of the JAK/STAT pathway is a well-described pathogenic feature of MF. Allogeneic stem cell transplant is the only curative therapy, but due to high morbidity and mortality this option is not available for most patients. There are two approved targeted therapy options for MF, ruxolitinib and fedratinib. In this review, we discuss the clinical utility of fedratinib in the myelofibrosis treatment paradigm. Fedratinib has shown impressive pre-clinical and clinical efficacy in patients with untreated MF as well as in those with ruxolitinib intolerance and those with relapsed/refractory MF. Here, we review the pre-clinical and clinical trials that led to the approval of fedratinib, and the ongoing late-phase trials. We highlight several areas regarding the clinical utility of fedratinib that remain unanswered. We discuss the limitations of fedratinib and address areas that are understudied and require further clinical evaluation and research. The approval of fedratinib has provided a significant expansion to the very limited treatment armamentarium available to patients with MF.Keywords: myelofibrosis, fedratinib, targeted therapy, JAK inhibitor

Published
2021

13. Comparing Outcomes between Liposomal Daunorubicin/Cytarabine (CPX-351) and HMA+Venetoclax As Frontline Therapy in Acute Myeloid Leukemia

Author

Grenet, Justin, primary, Jain, Akriti G, additional, Burkart, Madelyn, additional, Waksal, Julian, additional, Famulare, Christopher, additional, Numan, Yazan, additional, Stahl, Maximilian, additional, Mckinnell, Zoe, additional, Ball, Brian, additional, Ma, Xiaoyue, additional, Christos, Paul J, additional, Ritchie, Ellen, additional, Samuel, Michael B., additional, Kaner, Justin D., additional, Lee, Sangmin, additional, Goldberg, Aaron D, additional, Dinner, Shira, additional, Sweet, Kendra, additional, Roboz, Gail J., additional, and Desai, Pinkal, additional

Published
2021
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14. Safety and Efficacy of CPX-351 in Younger Patients < 60 Years Old with Secondary Acute Myeloid Leukemia: An Updated Analysis

Author

Przespolewski, Amanda, primary, Goldberg, Aaron D, additional, Talati, Chetasi, additional, Fazal, Salman, additional, Vachhani, Pankit, additional, Sanikommu, Srinivasa Reddy R., additional, Thota, Swapna, additional, Waksal, Julian, additional, Ball, Brian, additional, Famulare, Christopher, additional, Stahl, Maximilian, additional, Mckinnell, Zoe, additional, Baron, Jeffrey, additional, Griffiths, Elizabeth A., additional, Thompson, James E., additional, Sweet, Kendra, additional, and Wang, Eunice S., additional

Published
2021
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18. Novel therapeutics and targets in myelofibrosis.

Author

Waksal, Julian A., Harrison, Claire N., and Mascarenhas, John O.

Subjects
*MYELOFIBROSIS, *HEMATOPOIETIC stem cell transplantation, *EXTRAMEDULLARY hematopoiesis, *MYELOPROLIFERATIVE neoplasms, *THERAPEUTICS
Abstract

Myelofibrosis is a myeloproliferative neoplasm characterized by clonal proliferation of myeloid cells, bone marrow fibrosis and cytopenias, extramedullary hematopoiesis and hepatosplenomegaly, increased pro-inflammatory cytokine production, and systemic symptoms. Patients with MF also have a propensity toward leukemic transformation. Allogeneic hematopoietic stem cell transplantation (aHCT) is the only curative therapy for patients with MF; however, transplant-related morbidity and mortality precludes this option for the majority of patients. In the last decade, two targeted therapies have been approved for the treatment of MF, both JAK2 inhibitors, ruxolitinib and fedratinib. Despite the clinical efficacy of these two compounds in terms of splenomegaly and symptom burden reduction, there remain many areas of unmet need in the treatment of myelofibrosis. In this review, we discuss the limitations of currently approved treatment options and novel therapeutic targets with drug candidates in late-stage (phase II or III) clinical development for the treatment of MF. We delve into the mechanism of action and scientific rational of each candidate agent as well as the available clinical data, and ongoing trials that could lead to the approval of some of these novel therapies. [ABSTRACT FROM AUTHOR]

Published
2022
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19. Safety and Efficacy of CPX-351 in Younger Patients < 60 Years Old with Secondary Acute Myeloid Leukemia: An Updated Analysis

Author

Przespolewski, Amanda, Goldberg, Aaron D, Talati, Chetasi, Fazal, Salman, Vachhani, Pankit, Sanikommu, Srinivasa Reddy R., Thota, Swapna, Waksal, Julian, Ball, Brian, Famulare, Christopher, Stahl, Maximilian, Mckinnell, Zoe, Baron, Jeffrey, Griffiths, Elizabeth A., Thompson, James E., Sweet, Kendra, and Wang, Eunice S.

Published
2021
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20. Surveillance After Resection of Vestibular Schwannoma

Author

Tang, Shan, primary, Griffin, Andrew S., additional, Waksal, Julian A., additional, Phillips, C. Douglas, additional, Johnson, Carl E., additional, Comunale, Joseph P., additional, Karimi, Sasan, additional, Powell, Tiffany L., additional, Stieg, Philip E., additional, Gutin, Philip H., additional, Brown, Kevin D., additional, Sheehan, Matthew, additional, and Selesnick, Samuel H., additional

Published
2014
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