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Lindsay Anne Magura Rein

Associate Professor of Medicine
Medicine, Hematologic Malignancies and Cellular Therapy
Dept of Medicine, BOX_31079 DUMC, Durham, NC 27710

Selected Publications


Avapritinib achieves long-term disease control with favorable safety in patients with indolent systemic mastocytosis over 3 years.

Journal Article J Allergy Clin Immunol · April 14, 2026 BACKGROUND: Indolent systemic mastocytosis (ISM), a clonal mast cell disease primarily driven by the KIT D816V mutation, can cause long-term debilitating symptoms and poor quality of life. Most patients rely on symptom-directed best supportive care (BSC) m ... Full text Link to item Cite

Expert consensus definition of treatment intolerance in chronic myeloid leukemia in chronic phase.

Journal Article Hematology · December 2025 INTRODUCTION: Chronic myeloid leukemia in the chronic phase (CML-CP) has undergone therapeutic transformation with the advent of BCR::ABL1 tyrosine kinase inhibitors (TKIs), improving 10-year survival rates from 20% to ∼90%. These outcomes place increasing ... Full text Link to item Cite

Beta-Arrestin 1 Deficiency Enhances Host Anti-Myeloma Immunity Through T Cell Activation and Checkpoint Modulation.

Journal Article Int J Mol Sci · November 27, 2025 Beta-arrestin 1 (ARRB1) is a multifunctional adaptor protein that regulates diverse signaling pathways beyond its canonical role in G-protein-coupled receptor desensitization. While ARRB1 has been implicated in cancer progression, its role in modulating ho ... Full text Open Access Link to item Cite

IMMU-81. Glioblastoma and other intracranial pathologies elicit systemic sympathetic hyperactivity that limits immunotherapeutic responses

Conference Neuro-Oncology · November 11, 2025 AbstractImmunotherapies remain largely ineffective against intracranial malignancies such as glioblastoma (GBM), in part due to profound systemic and local immune dysfunction. Hallmarks of systemic immune ... Full text Cite

The effect of bezuclastinib on the pathobiology of mastocytosis: Changes in BM mast cells, tryptase, and KIT p.D816V variant allele frequency from the pivotal summit trial

Conference Blood · November 3, 2025 AbstractBackground: Systemic mastocytosis (SM) includes a spectrum of hematologic malignancies characterized by a variable degree of neopla ... Full text Cite

Nuvisertib, an oral investigational selective PIM1 kinase inhibitor, showed clinical responses strongly correlating with cytokine modulation in patients with relapsed/refractory myelofibrosis in the ongoing global phase I/II study

Conference Blood · November 3, 2025 AbstractBackground: PIM1 is overexpressed in hematologic malignancies, including myelofibrosis (MF), contributing to disease progression by ... Full text Cite

Incidence, prevalence, and clinical outcomes of myelofibrosis with and without cytopenia in the United States

Conference Blood · November 3, 2025 AbstractIntroduction: Cytopenia in patients with myelofibrosis (MF) is characterized by presence of anemia and/or thrombocytopenia, and is ... Full text Cite

Mastocytosis: Part I: Pathogenesis, Clinical Presentation and Classification.

Journal Article J Am Acad Dermatol · June 19, 2025 Mast cells are tissue-resident immune cells which secrete numerous and varied immune mediators in response to a myriad of activation pathways and play an important physiological function in immunity, homeostasis, tissue repair, and nervous system regulatio ... Full text Link to item Cite

Mastocytosis: Part II: Diagnosis and Management of Mastocytosis through a Multidisciplinary Lens.

Journal Article J Am Acad Dermatol · June 19, 2025 Mastocytosis is a heterogeneous disorder marked by mast cell accumulation in various organs, presenting differently in children and adults. Red or brown maculopapular lesions on the skin should raise suspicion for mastocytosis. In children, presentations i ... Full text Link to item Cite

Prevalence of cytopenic myelofibrosis in the United States.

Conference Journal of Clinical Oncology · June 2025 e18575Background: Among patient (pts) with myelofibrosis (MF), cytopenias (anemia or thrombocytopenia) are a marker of advanc ... Full text Cite

β-Arrestin 2 as a Prognostic Indicator and Immunomodulatory Factor in Multiple Myeloma.

Journal Article Cells · March 26, 2025 β-arrestin 2 (ARRB2) is involved in the desensitization and trafficking of G protein-coupled receptors (GPCRs) and plays a critical role in cell proliferation, apoptosis, chemotaxis, and immune response modulation. The role of ARRB2 in the pathogenesis of ... Full text Link to item Cite

Top Ten Tips Palliative Care Clinicians Should Know About Caring for Patients with Myeloproliferative Neoplasms.

Journal Article J Palliat Med · February 2025 Myeloproliferative neoplasms (MPNs) are a group of rare chronic progressive blood cancers that vary widely in clinical presentation, yet all patients have a risk of disease progression and thrombotic complications. Diseases include primary myelofibrosis, p ... Full text Link to item Cite

Updated Efficacy and Safety Results of Patients Receiving Selected 100mg Bezuclastinib Dose and Participating in the Open-Label Extension of Summit: A Randomized, Double-Blind, Placebo Controlled Phase 2 Clinical Trial of Bezuclastinib in Adult Patients with Nonadvanced Systemic Mastocytosis

Journal Article Blood · November 5, 2024 Background: Systemic mastocytosis (SM) is a rare disease characterized by neoplastic mast cell (MC) infiltration of extracutaneous tissues. Nonadvanced SM (NonAdvSM) is associated with de ... Full text Cite

Cytokine Modulation Correlates Strongly with Symptom Improvement in Patients with Myelofibrosis Treated with Nuvisertib (TP-3654), an Investigational Selective PIM1 Kinase Inhibitor

Journal Article Blood · November 5, 2024 Background: PIM1 expression is upregulated in myelofibrosis (MF) and has been shown to modulate cytokine induced PI3K/AKT and JAK/STAT pathways. Clinical features of MF are partly attribu ... Full text Cite

Nuvisertib (TP-3654), an Investigational Selective PIM1 Kinase Inhibitor, Showed Durable Clinical Response and Sustained Hematological Improvement in Relapsed/Refractory Myelofibrosis Patients

Journal Article Blood · November 5, 2024 Background: PIM1 expression is upregulated in hematologic malignancies including myelofibrosis (MF). In preclinical models, PIM1 knockout (KO) was shown to prevent MF progression without ... Full text Cite

A Phase 1/2 Study of Nuvisertib (TP-3654), an Investigational Selective PIM1 Kinase Inhibitor, in Combination with JAK Inhibitors Ruxolitinib or Momelotinib in Patients with Myelofibrosis

Journal Article Blood · November 5, 2024 Background: JAK inhibitors are the current standard of care treatment for patients (pts) with myelofibrosis (MF). Ruxolitinib (RUX), the first approved JAK inhibitor in 2011, showed splee ... Full text Cite

Management and Outcomes of Patients Diagnosed with Chronic Myeloid Leukemia in Blast Phase: A Multicenter Analysis By the H Jean Khoury Cure CML Consortium

Conference Blood · November 5, 2024 Introduction:Despite advent of multiple tyrosine kinase inhibitors (TKIs) and newer treatment methods for both AML and ALL, the outcomes of patients (p ... Full text Cite

U.S. Expert Consensus on Defining Intolerance to Tyrosine Kinase Inhibitor Treatment in Chronic Phase Chronic Myeloid Leukemia (CML)

Conference Blood · November 5, 2024 Introduction: The incidence of CML in the United States (U.S) is 1-2 cases per 100,000 persons per year and the prevalence has risen, in part due to the effectiveness of BCR-ABL1 tyrosine ... Full text Cite

NCCN Guidelines® Insights: Systemic Mastocytosis, Version 3.2024.

Journal Article J Natl Compr Canc Netw · June 2024 Mastocytosis is a heterogeneous group of disorders comprising cutaneous mastocytosis, systemic mastocytosis, and mast cell sarcoma. It is associated with a variety of symptoms related to the release of mast cell mediators and mast cell tissue infiltration. ... Full text Link to item Cite

Systemic Mastocytosis, Version 3.2024: Featured Updates to the NCCN Guidelines

Journal Article JOURNAL OF THE NATIONAL COMPREHENSIVE CANCER NETWORK · June 2024 Link to item Cite

HJKC3-0006: Outcome of Patients with CML Treated on Second Line or Later Therapy in Routine Clinical Practice

Conference Blood · November 2, 2023 Background: First-line therapies for chronic-phase (CP) CML include the tyrosine kinase inhibitors(TKIs) dasatinib, nilotinib, bosutinib or imatinib, while ponatinib and asciminib are indicated only in later lines. Ther ... Full text Cite

HJKC3-007: Outcomes of Patients with Chronic Myeloid Leukemia in Chronic Phase Treated with Asciminib and Other Tyrosine Kinase Inhibitors in Routine Clinical Practice

Conference Blood · November 2, 2023 Background and significance: There are typically high response rates to first line TKI therapies in chronic phase chronic myeloid leukemia (CP-CML). However, responses in later lines (second line and beyond) are diminis ... Full text Cite

Initial Results from Summit: An Ongoing, 3-Part, Multi-Center, Randomized, Double-Blind, Placebo-Controlled Phase 2 Clinical Study of Bezuclastinib in Adult Patients with Nonadvanced Systemic Mastocytosis (NonAdvSM)

Journal Article Blood · November 2, 2023 Background: Systemic mastocytosis (SM) is a rare disease characterized by neoplastic mast cell infiltration of extracutaneous tissues and encompasses a spectrum of subtypes that can range from non-advanced to advanced d ... Full text Cite

Leveraging Natural Killer Cell Innate Immunity against Hematologic Malignancies: From Stem Cell Transplant to Adoptive Transfer and Beyond.

Journal Article Int J Mol Sci · December 22, 2022 Numerous recent advancements in T-cell based immunotherapies have revolutionized the treatment of hematologic malignancies. In the race towards the first approved allogeneic cellular therapy product, there is growing interest in utilizing natural killer (N ... Full text Link to item Cite

Randomized Phase 3 Trial of Ruxolitinib for COVID-19-Associated Acute Respiratory Distress Syndrome.

Journal Article Crit Care Med · December 1, 2022 OBJECTIVES: Evaluate the safety and efficacy of the Janus kinase (JAK)1/JAK2 inhibitor ruxolitinib in COVID-19-associated acute respiratory distress syndrome requiring mechanical ventilation. DESIGN: Phase 3 randomized, double-blind, placebo-controlled tri ... Full text Link to item Cite

Myeloproliferative Neoplasms, Version 3.2022, NCCN Clinical Practice Guidelines in Oncology.

Journal Article J Natl Compr Canc Netw · September 2022 The classic Philadelphia chromosome-negative myeloproliferative neoplasms (MPN) consist of myelofibrosis, polycythemia vera, and essential thrombocythemia and are a heterogeneous group of clonal blood disorders characterized by an overproduction of blood c ... Full text Link to item Cite

The Use of Allogeneic Hematopoietic Stem Cell Transplantation in Primary Myelofibrosis.

Journal Article J Pers Med · April 2, 2022 Primary myelofibrosis (PMF) is a BCR-ABL1 negative myeloproliferative neoplasm characterized by clonal proliferation of myeloid cells. This leads to reactive bone marrow fibrosis, ultimately resulting in progressive marrow failure, hepatosplenomegaly, and ... Full text Link to item Cite

The Evolving Landscape of Frontline Therapy in Chronic Phase Chronic Myeloid Leukemia (CML).

Journal Article Curr Hematol Malig Rep · October 2021 PURPOSE OF REVIEW: Chronic myeloid leukemia (CML) is a myeloproliferative neoplasm characterized by uncontrolled proliferation of mature and maturing granulocytes. The disease is characterized by the presence of translocation t(9;22) leading to the abnorma ... Full text Link to item Cite

A Phase I Trial of Incorporating Natural Killer (K-NK) Cells for Patients with Chronic Myeloid Leukemia (CML) and Molecular Residual Disease after Tyrosine Kinase Inhibitor (TKI) Therapy

Journal Article Blood · November 5, 2020 Tyrosine kinase inhibitors (TKIs) have revolutionized the treatment of patients with chronic myeloid leukemia (CML) ushering in an era where, in select patient populations, treatment planning goals have shifted towards the achievement of treatment ... Full text Cite

Myeloid/Lymphoid Neoplasms with Eosinophilia and TK Fusion Genes, Version 3.2021, NCCN Clinical Practice Guidelines in Oncology.

Journal Article J Natl Compr Canc Netw · September 2020 Eosinophilic disorders and related syndromes represent a heterogeneous group of neoplastic and nonneoplastic conditions, characterized by more eosinophils in the peripheral blood, and may involve eosinophil-induced organ damage. In the WHO classification o ... Full text Link to item Cite

The impact of bone marrow fibrosis and JAK2 expression on clinical outcomes in patients with newly diagnosed multiple myeloma treated with immunomodulatory agents and/or proteasome inhibitors.

Journal Article Cancer Med · August 2020 We determined the impact of bone marrow fibrosis (BMF) on the clinical outcomes of newly diagnosed multiple myeloma (NDMM) patients in the current era of myeloma therapy. A total of 393 MM patients were included in the final analysis. The median followup w ... Full text Link to item Cite

Systemic Mastocytosis, Version 2.2019, NCCN Clinical Practice Guidelines in Oncology.

Journal Article J Natl Compr Canc Netw · December 2018 Mastocytosis is a group of heterogeneous disorders resulting from the clonal proliferation of abnormal mast cells and their accumulation in the skin and/or in various extracutaneous organs. Systemic mastocytosis is the most common form of mastocytosis diag ... Full text Link to item Cite

Applications of Gene Editing Technologies to Cellular Therapies.

Journal Article Biol Blood Marrow Transplant · August 2018 Hematologic malignancies are characterized by genetic heterogeneity, making classic gene therapy with a goal of correcting 1 genetic defect ineffective in many of these diseases. Despite initial tribulations, gene therapy, as a field, has grown by leaps an ... Full text Link to item Cite

β-Arrestin2 mediates progression of murine primary myelofibrosis.

Journal Article JCI Insight · December 21, 2017 Primary myelofibrosis is a myeloproliferative neoplasm associated with significant morbidity and mortality, for which effective therapies are lacking. β-Arrestins are multifunctional adaptor proteins involved in developmental signaling pathways. One isofor ... Full text Link to item Cite

NCCN Guidelines Insights: Myeloproliferative Neoplasms, Version 2.2018.

Journal Article J Natl Compr Canc Netw · October 2017 Myeloproliferative neoplasms (MPNs) are a group of heterogeneous disorders of the hematopoietic system that include myelofibrosis (MF), polycythemia vera (PV), and essential thrombocythemia (ET). PV and ET are characterized by significant thrombohemorrhagi ... Full text Link to item Cite

Myeloproliferative Neoplasms, Version 2.2017, NCCN Clinical Practice Guidelines in Oncology.

Journal Article J Natl Compr Canc Netw · December 2016 Myelofibrosis (MF), polycythemia vera (PV), and essential thrombocythemia (ET) are a group of heterogeneous disorders of the hematopoietic system collectively known as Philadelphia chromosome-negative myeloproliferative neoplasms (MPNs). The diagnosis and ... Full text Link to item Cite

Clinical potential of elacytarabine in patients with acute myeloid leukemia.

Journal Article Ther Adv Hematol · December 2014 Acute myeloid leukemia (AML) has been treated for over four decades with standard induction chemotherapy including seven days of cytosine arabinoside (cytarabine, ara-C) infusion. Cytarabine, while effective in killing leukemic cells, is subject to develop ... Full text Link to item Cite

WT1 vaccination in acute myeloid leukemia: new methods of implementing adoptive immunotherapy.

Journal Article Expert Opin Investig Drugs · March 2014 INTRODUCTION: The Wilms tumor 1 (WT1) gene was originally identified as a tumor suppressor gene that, when mutated, would lead to the development of pediatric renal tumors. More recently, it has been determined that WT1 is overexpressed in 90% of patients ... Full text Link to item Cite

Targeting β-arrestin2 Enhances Survival in a Murine Model of Chronic Myeloid Leukemia

Conference Blood · November 15, 2013 AbstractBackgroundChronic myeloid leukemia (CML) is a myeloproliferative neoplasm of hematopoietic stem cells characterized by presenc ... Full text Cite

New approaches to manipulate minimal residual disease after allogeneic stem cell transplantation.

Journal Article Int J Hematol Oncol · February 2013 Minimal residual disease (MRD) is a complex topic that has been studied extensively in hematologic malignancies given its clinical implications related to prognosis. However, methods to monitor and treat MRD, especially after stem cell transplantation, are ... Full text Link to item Cite

Hypercholesterolemia and prostate cancer: a hospital-based case-control study.

Journal Article Cancer Causes Control · December 2008 OBJECTIVE: High levels of serum cholesterol have been proposed to increase the risk of prostate cancer but the epidemiologic evidence is limited. METHODS: We conducted a hospital-based case-control study in Fargo, ND, USA, to examine the association betwee ... Full text Link to item Cite