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Articles by Jason Gotlib
Predicting the clinical course of advanced systemic mastocytosis on treatment with avapritinib or midostaurin
Abstract Advanced systemic mastocytosis (AdvSM) is driven by KIT D816V in approximately 95% of cases. Accurate assessment and prediction of clinical course and disease outcome before and during treatment with the KIT inhibitors midostaurin and avapritinib may facilitate optimization of treatment strategies. We retrospectively evaluated clinical, morphologic, laboratory, and genetic predictors of outcome in 79 midostaurin- and 176 avapritinib-treated patients.
Recurrent Genomic Alterations in BCRi-Experienced CLL Patients Treated With Venetoclax: Extended Phase 2 Follow-Up
To the Editor, First-generation covalent Bruton tyrosine kinase inhibitors (cBTKi; ie, ibrutinib) and second-generation cBTKis, acalabrutinib and zanubrutinib, disrupt aberrant B-cell receptor (BCR)-signaling via inhibition of BTK. BTKis, along with idelalisib, a phosphatidylinositol 3-kinase delta inhibitor that also inhibits BCR signaling, have transformed outcomes for patients with chronic lymphocytic leukemia (CLL) [1].
Risk stratification of patients with TP53-mutated myeloproliferative neoplasms - Leukemia
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Abstract While TP53 mutations in myeloproliferative neoplasms (MPN) are associated with an increased risk of leukemic transformation, not all patients carrying a TP53 mutation progress. To better risk-stratify MPN patients with TP53 mutations, we analyzed data from 1540 patients treated at four specialized cancer centers. Among them, 1429 had wildtype TP53 and 111 had mutations in the TP53 gene.
By Benjamin Rolles, Nathan Fergusson, Jan Philipp Bewersdorf, Julia M Keating, Cecelia Perkins, Matthew Schwede, James England, Marlise R. Luskin, Daniel DeAngelo, Shai Shimony, Mifra Faiz, Addison C. Hillerbrand, Chulwoo Kim, Lachelle D. Weeks, Lauren Waldman, Mohammed Wazir, Joan How, Anna E. Marneth, Yiwen Liu, Martin J. Aryee, Raajit Rampal, Harrison K. Tsai, Jason Gotlib, Vikas Gupta, Maximilian Stahl, Ann Mullally
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Nature
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Prognostic impact of expression of CD2, CD25, and/or CD30 in/on mast cells in systemic mastocytosis: a registry study of the European Competence Network on Mastocytosis | Leukemia
Abstract Expression of CD2, CD25 and/or CD30 in extracutaneous mast cells (MC) is a minor diagnostic criterion for systemic mastocytosis (SM) in the classification of the World Health Organization and International Consensus Classification. So far, it remains unknown whether expression of these antigens on MC is of prognostic significance in SM.
World Health Organization and International Consensus Classification of eosinophilic disorders: 2024 update on diagnosis, risk stratification, and management
1 INTRODUCTION 1.1.1 Epidemiology The incidence and prevalence of HES is not well characterized.
Allogeneic haematopoietic cell transplantation for advanced systemic mastocytosis: Best practice recommendations on behalf of the EBMT Practice Harmonisation and Guidelines Committee | Leukemia
Abstract Systemic Mastocytosis (SM) is a multifaceted clinically heterogeneous disease. Advanced SM (AdvSM) comprises three entities: aggressive SM (ASM), mast cell leukaemia (MCL) and SM with an associated hematologic neoplasm (SM-AHN), the latter accounting for 60–70% of all AdvSM cases. Detection of a disease-triggering mutation in the KIT gene (esp.
Rusfertide, a Hepcidin Mimetic, for Control of Erythrocytosis in Polycythemia Vera
February 22, 2024 N Engl J Med 2024; 390:723-735 DOI: 10.1056/NEJMoa2308809 Abstract Polycythemia vera is a chronic myeloproliferative neoplasm characterized by erythrocytosis. Rusfertide, an injectable peptide mimetic of the master iron regulatory hormone hepcidin, restricts the availability of iron for erythropoiesis. The safety and efficacy of rusfertide in patients with phlebotomy-dependent polycythemia vera are unknown.
The international consensus classification of eosinophilic disorders and systemic mastocytosis
Abstract Based on new data and increased understanding of disease molecular genetics, the international consensus classification (ICC) has made several changes in the diagnosis and classification of eosinophilic disorders and systemic mastocytosis. Myeloid/lymphoid neoplasms with eosinophilia (M/LN-eo) and gene rearrangements have been renamed as M/LN-eo with tyrosine kinase gene fusions (M/LN-eo-TK).
Avapritinib Improved Symptoms and Quality of Life in Patients With Indolent Systemic Mastocytosis in the PIONEER Study
Indolent systemic mastocytosis (ISM), is a disorder that can be characterized by debilitating cutaneous, gastrointestinal, neurological, and musculoskeletal manifestations, high symptom burden, anaphylaxis, and poor quality of life (QoL). Avapritinib, an investigational selective KIT D816V inhibitor targets the primary driver of ISM.
Efficacy and Safety of Avapritinib in Indolent Systemic Mastocytosis (ISM): Results from the Double Blinded Placebo-Controlled PIONEER Study
ISM, primarily driven by the KIT D816V mutation, is the most frequent systemic mastocytosis subtype which may cause debilitating symptoms. PIONEER (NCT03731260) assessed safety and efficacy of avapritinib, an investigational selective KIT D816V inhibitor, vs placebo, both with best supportive care (BSC).
Systemic Mastocytosis Life Expectancy
What is the Life Expectancy for Systemic Mastocytosis? Being diagnosed with systemic mastocytosis (SM) may be scary, and you may be wondering how the disease might affect how long you’ll live. The answer isn’t simple. Life expectancy among patients with SM varies greatly - anywhere from months after diagnosis to a normal life expectancy.
Systemic Mastocytosis Treatment Options
How is Systemic Mastocytosis Treated? Treatment for people with systemic mastocytosis (SM) varies. The options are based on the subtype of the disease, the mast cell burden, symptoms, as well as the individual’s response to the first line of treatment. These treatments can be grouped into two categories: symptom management and cytoreductive therapy.
What Are the Types of Systemic Mastocytosis?
SM Can Be Either Non-Advanced or Advanced Systemic mastocytosis (SM) is a rare chronic blood disease that occurs when the body makes too many or abnormal mast cells, a type of white blood cell that plays an important role in the body’s immune response to pathogens and allergens. In contrast to cutaneous mastocytosis, which only affects the skin and mainly occurs in children, SM affects one or more other organs — sometimes, but not always, in addition to the skin — and mainly occurs in adults.
This paper is only available as a PDF. To read, Please Download here. Abstract Mast cell (MC) neoplasms are an emerging challenge in the fields of internal medicine, allergy, immunology, dermatology, laboratory medicine and pathology. In this review, we discuss the current standards for the diagnosis and prognostication of MC neoplasms with special reference to clinically relevant germline and somatic gene variants.
World Health Organization‐defined eosinophilic disorders: 2021 update on diagnosis, risk stratification, and management
ANNUAL CLINICAL UPDATES IN HEMATOLOGICAL MALIGNANCIES William Shomali, orcid.org/0000-0001-6500-350X Division of Hematology, Stanford Cancer Institute, Stanford University School of Medicine, Stanford, California, USA Search for more papers by this author Jason Gotlib, Corresponding Author jason.gotlib@stanford.edu Division of Hematology, Stanford Cancer Institute, Stanford University School of Medicine, Stanford, California, USA Correspondence Jason Gotlib, Stanford Cancer Institute, 875...
Diagnosis and Management of Neutrophilic Myeloid Neoplasms
Abstract: Chronic neutrophilia is commonly seen with persistent infections, inflammatory disorders, smoking, solid tumors, and specific medications. However, after reactive causes have been excluded, a workup for primary (clonal) neutrophilic disorders, such as myeloproliferative neoplasms (MPNs) and myelodysplastic/myeloproliferative overlap syndromes, should be pursued.
Proceedings from the Inaugural American Initiative in Mast Cell Diseases (AIM) Investigator Conference
The American Initiative in Mast Cell Diseases (AIM) held its inaugural investigator conference at Stanford University School of Medicine in May 2019. The overarching goal of this meeting was to establish a Pan-American organization of physicians and scientists with multidisciplinary expertise in mast cell disease.
Midostaurin improves quality of life and mediator-related symptoms in advanced systemic mastocytosis
Background Advanced systemic mastocytosis (advSM) is characterized by presence of the KIT D816V mutation and pathologic accumulation of neoplastic mast cells (MCs) in various tissues, leading to severe symptoms and organ damage (eg, cytopenias, liver dysfunction, portal hypertension, malabsorption, and weight loss).
Efficacy of Ruxolitinib in Patients With Chronic Neutrophilic Leukemia and Atypical Chronic Myeloid Leukemia
Chronic neutrophilic leukemia (CNL) and atypical chronic myeloid leukemia (aCML) are rare BCR-ABL1–negative myeloid neoplasms.
MDM2 Study: How Can Polycythemia Vera Patients Get Involved?
Published on November 15, 2019 Key Takeaways Learn about a current trial for polycythemia vera patients. Qualifying factors for current PV trial. Number of trail sites and phases for PV clinical trial. Patient Power Co-Founder, CLL patient and advocate, Andrew Schorr, talks with Dr. Jason Gotlib, from Stanford University Cancer Institute, about the current MDM2 clinical trial for polycythemia vera patients.
Emerging Role of MDM2 in Myelofibrosis Care
Published on September 18, 2019 What is MDM2 and what does it mean for myeloproliferative neoplasm (MPN) patients? Leading expert Dr. Srdan Verstovsek, from The University of Texas MD Anderson Cancer Center, joined Patient Power to discuss this novel treatment option currently under clinical investigation for myelofibrosis (MF). Here, Dr. Verstovsek explains the mechanism of action of the MDM2 inhibitor and which patient population it may benefit.
MPN Researcher Shares Optimism for Future Treatment
Patient Power founder Andrew Schorr is joined by expert Dr. Jason Gotlib, from the Stanford University School of Medicine, to discuss progress being made to improve treatment durability and efficacy for myeloproliferative neoplasm (MPN) patients. Dr. Gotlib gives an overview of the ever-evolving MPN treatment landscape, potential new drug approvals and a hopeful look at combination therapies in clinical trials. Watch now to learn his expert knowledge.
Experts Explain MDM2 Inhibition and Clinical Trial for MPN Patients
Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you. Andrew Schorr: Hello and welcome to Patient Power. I'm Andrew Schorr. Joining me once again is a leading expert in the MPNs. Today we're talking about a new trial for people with myelofibrosis.
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