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    1 OF 135

    A Practical Guide for Treatment of Pain in Patients with Systemic Mast Cell Activation Disease.
    Pain Physician 2017 Sep;20(6):E849-E861
    Institute of Human Genetics, University Hospital of Bonn, Germany.
    Systemic mast cell activation disease (MCAD, a subclass of mastocytosis), which has a prevalence of around 17% (at least in the German population), is characterized by accumulation of genetically altered dysfunctional mast cells with abnormal release of these cells' mediators. Since mast cells affect functions in potentially every organ system, often without causing abnormalities in routine laboratory or radiologic testing, this disease has to be considered routinely in the differential diagnosis of patients with chronic multisystem polymorbidity of a generally inflammatory and allergic theme. Pain in its different manifestations is a common symptom in MCAD found in more than three-quarters of the MCAD patients. Read More

    Prevalence and risk factors for fragility fracture in systemic mastocytosis.
    Bone 2017 Sep 14;105:219-225. Epub 2017 Sep 14.
    Department of Dermatology, Mastocytosis Expert Center of Midi-Pyrénées, Paul Sabatier University, Toulouse University Hospital, Toulouse, France. Electronic address:
    Objectives: Systemic mastocytosis (SM) is characterized by the accumulation of mast cells in tissues other than the skin. Bone involvement although frequent has not been thoroughly evaluated. Primary objective was to determine risk factors associated with fragility fractures (FF) in SM. Read More

    Siglec-8 as mast cell selective target: developing paradigms amidst inconvenient truths.
    Naunyn Schmiedebergs Arch Pharmacol 2017 Sep 14. Epub 2017 Sep 14.
    Institute of Human Genetics, University Hospital of Bonn, Sigmund-Freud-Strasse 25, 53127, Bonn, Germany.
    Due to the limited efficacy of current drugs in treating systemic mast cell activation disease, there is an urgent need for more effective drugs selectively acting at mast cells. In the past, a large number of compounds have been claimed to be effective and mast cell selective on the basis of cell culture experiments and studies on blood leukocytes which could not be verified in organ and animal studies. Nevertheless, over time in review papers about potential mast cell targets mast cell selectivity of these targets has been no longer challenged. Read More

    Fatal Anaphylaxis: Mortality Rate and Risk Factors.
    J Allergy Clin Immunol Pract 2017 Sep - Oct;5(5):1169-1178
    Department of Paediatric Allergy, Imperial College London, London, United Kingdom. Electronic address:
    Up to 5% of the US population has suffered anaphylaxis. Fatal outcome is rare, such that even for people with known venom or food allergy, fatal anaphylaxis constitutes less than 1% of total mortality risk. The incidence of fatal anaphylaxis has not increased in line with hospital admissions for anaphylaxis. Read More

    Characterization of midostaurin as a dual inhibitor of FLT3 and SYK and potentiation of FLT3 inhibition against FLT3-ITD-driven leukemia harboring activated SYK kinase.
    Oncotarget 2017 Aug 6;8(32):52026-52044. Epub 2017 Jul 6.
    Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts, USA.
    Oncogenic FLT3 kinase is a clinically validated target in acute myeloid leukemia (AML), and both multi-targeted and selective FLT3 inhibitors have been developed. Spleen tyrosine kinase (SYK) has been shown to be activated and increased in FLT3-ITD-positive AML patients, and has further been shown to be critical for transformation and maintenance of the leukemic clone in these patients. Further, over-expression of constitutively activated SYK causes resistance to highly selective FLT3 tyrosine kinase inhibitors (TKI). Read More

    Mast Cell Disorders, Melanoma and Pancreatic Carcinoma: From a Clinical Observation to a Brief Review of the Literature.
    Acta Dermatovenerol Croat 2017 Jul;25(2):112-119
    Dario Didona, MD Istituto Dermopatico dell'Immacolata-IRCCS Via dei Monti di Creta 104, 00167 Rome Italy
    Mastocytosis can be associated with other clonal or non-clonal hematologic diseases as well as a variety of non-hematologic malignancies. A 75-year-old Caucasian male patient was referred to us with a 5-month history of neutrophilic leukocytosis and mild splenomegaly. He had developed a cutaneous melanoma sixteen years ago. Read More

    Management around invasive procedures in mastocytosis: An update.
    Ann Allergy Asthma Immunol 2017 Aug 30. Epub 2017 Aug 30.
    Department of Internal Medicine, Section of Allergy, Erasmus University Medical Center, Rotterdam, The Netherlands; Department of Internal Medicine, Section of Clinical Immunology, Erasmus University Medical Center, Rotterdam, The Netherlands.
    Objective: Mastocytosis is a chronic hematologic disorder that is characterized by the accumulation of aberrant mast cells and typically involves the skin and/or bone marrow. Patients with mastocytosis are at increased risk of anaphylaxis. Based on theoretical assumptions, medical procedures requiring general anesthesia or radiocontrast media are deemed hazardous for patients with mastocytosis. Read More

    Case Report: Unusual Manifestation of KIT Negative Systemic Mastocytosis.
    Am J Hematol Oncol 2016 Dec;12(12):24-27
    Department of Hematology-Oncology of the VA Caribbean Healthcare System, 10 Calle Casia, San Juan, Puerto Rico 00921.
    Aggressive systemic mastocytosis is a rare hematologic neoplastic disease that presents with a poor prognosis and low survival rate. It typically manifests with symptoms associated to mast cell release of bioactive substances, causing anaphylaxis, flushing, autonomic and hemodynamic instability, gastric distress and headache. Moreover, more than 95% of cases are related to a mutation in codon 816 of the KIT gene, located on human chromosome 4q12 which codes for a type III receptor tyrosine kinase. Read More

    Oesophageal mastocytosis: eosinophilic oesophagitis without eosinophils?
    BMJ Case Rep 2017 Aug 28;2017. Epub 2017 Aug 28.
    Division of Gastroenterology, Hepatology and Nutrition, College of Medicine, University of Florida, Gainesville, Florida, USA.
    A 59-year-old male with a history of lifelong asthma, allergic rhinitis and hypercholesterolaemia presented to the emergency department for management of severe substernal chest pain with radiating pain to his left arm, nausea and diaphoresis. Physical examination was unrevealing and a cardiac workup including cardiac enzymes, ECG, chest radiographs were negative for an underlying ischaemic event. A subsequent gastrointestinal workup including oesophageal manometry and oesophagogastroduodenoscopy revealed elevated lower oesophageal pressures and histopathology suggestive of mast cell proliferation, respectively. Read More

    Mast cell inhibition as a therapeutic approach in fibrodysplasia ossificans progressiva (FOP).
    Bone 2017 Aug 26. Epub 2017 Aug 26.
    Department of Medicine, Mayo Clinic School of Medicine, Mayo Clinic, Rochester, MN, United States. Electronic address:
    Background: Episodic flare-ups of fibrodysplasia ossificans progressiva (FOP) are characterized clinically by severe, often posttraumatic, connective tissue swelling and intramuscular edema, followed histologically by an intense and highly angiogenic fibroproliferative reaction. This early inflammatory and angiogenic fibroproliferative response is accompanied by the presence of abundant mast cells far in excess of other reported myopathies.

    Results: Using an injury-induced, constitutively-active transgenic mouse model of FOP we show that mast cell inhibition by cromolyn, but not aprepitant, results in a dramatic reduction of heterotopic ossification. Read More

    Beyond IgE-When Do IgE-Crosslinking and Effector Cell Activation Lead to Clinical Anaphylaxis?
    Front Immunol 2017 10;8:871. Epub 2017 Aug 10.
    Allergy Clinic, Copenhagen University Hospital at Gentofte, Hellerup, Denmark.
    Anaphylaxis in humans is inherently difficult to study due to the acuteness of symptoms and the lack of biomarkers serving as risk predictors. Most cases are related to IgE sensitizations to foods, insect venoms, and drugs with mastocytosis patients forming a smaller risk group. However, identifying the relatively small fraction of persons at risk has been exceedingly difficult. Read More

    Etiologies and management of cutaneous flushing: Malignant causes.
    J Am Acad Dermatol 2017 Sep;77(3):405-414
    Department of Dermatology, Tulane University, New Orleans, Louisiana.
    The second article in this 2-part continuing medical education series reviews the following malignant causes of flushing: mastocytosis, medullary thyroid carcinoma, pheochromocytoma, carcinoid tumors, gastroenteropancreatic neuroendocrine tumors, bronchogenic carcinoma, vasointestinal polypeptide secreting tumors, and renal cell carcinoma. The information provided will allow physicians to better distinguish patients who have worrisome presentations that require a more thorough investigation. Appropriate diagnostic workup and treatment options for these malignancies are reviewed. Read More

    Hymenoptera-induced anaphylaxis: is it a mast cell driven hematological disorder?
    Curr Opin Allergy Clin Immunol 2017 Oct;17(5):356-362
    aDepartment of Dermatology and Allergology, Technical University of Munich, Munich, Germany bDivision of Allergy and Clinical Immunology, Department of Medicine, University of Michigan, Ann Arbor, Michigan, USA.
    Purpose Of Review: Hymenoptera-induced allergy (HVA) is a common cause of anaphylaxis and may be fatal. It is associated with systemic mastocytosis in about 7% of adult patients. Systemic mastocytosis is a proliferative disorder of hematopoietic mast cell progenitors. Read More

    Bidirectional Mast Cell-Eosinophil Interactions in Inflammatory Disorders and Cancer.
    Front Med (Lausanne) 2017 24;4:103. Epub 2017 Jul 24.
    Department of Translational Medical Sciences (DiSMeT), Center for Basic and Clinical Immunology Research (CISI), University of Naples Federico II, Naples, Italy.
    Human mast cells (MCs) and eosinophils were first described and named by Paul Ehrlich. These cells have distinct myeloid progenitors and differ morphologically, ultrastructurally, immunologically, biochemically, and pharmacologically. However, MCs and eosinophils play a pivotal role in several allergic disorders. Read More

    Neuroimaging evidence of brain abnormalities in mastocytosis.
    Transl Psychiatry 2017 Aug 8;7(8):e1197. Epub 2017 Aug 8.
    Centre de Référence des Mastocytoses, Université Paris Descartes, Sorbonne, Paris Cité, Hôpital Necker-Enfants Malades, Fondation Imagine, Paris, France.
    Mastocytosis is a rare disease in which chronic symptoms are related to mast cell accumulation and activation. Patients can display depression-anxiety-like symptoms and cognitive impairment. The pathophysiology of these symptoms may be associated with tissular mast cell infiltration, mast cell mediator release or both. Read More

    Mast cell activation syndromes.
    J Allergy Clin Immunol 2017 Aug;140(2):349-355
    Division of Allergy and Clinical Immunology, University of Michigan, Ann Arbor, Mich. Electronic address:
    Mast cell activation is common and possibly necessary for maintenance of survival. Disordered mast cell activation occurs when mast cells are pathologically overproduced or if their activation is out of proportion to the perceived threat to homeostasis. Mast cell activation syndrome refers to a group of disorders with diverse causes presenting with episodic multisystem symptoms as the result of mast cell mediator release. Read More

    Neurobiological Basis of Hypersomnia.
    Sleep Med Clin 2017 Sep 7;12(3):265-277. Epub 2017 Jun 7.
    International Institute of Integrative Sleep Medicine, Tsukuba University, 1-1-1 Tennodai, Tsukuba, Ibaraki 305-8575, Japan. Electronic address:
    Narcolepsy is the most well-characterized hypersomnia in both clinical and basic research fields. Narcolepsy is caused by degeneration of hypocretin-producing neurons in the hypothalamus. Although hypocretin receptor antagonists have been developed as sleep-inducing drugs, a high dose of suvorexant, a hypocretin receptor antagonist, inhibits gene expression of prepro-hypocretin to induce narcoleptic attack in wild-type mice. Read More

    Targeted Treatment Options in Mastocytosis.
    Front Med (Lausanne) 2017 20;4:110. Epub 2017 Jul 20.
    French Reference Center for Mastocytosis (CEREMAST), Department of Hematology, Necker Children's Hospital, APHP, Paris, France.
    Mastocytosis refers to a heterogeneous group of disorders resulting from the clonal proliferation of abnormal mast cells and their accumulation in the skin (cutaneous mastocytosis when only in the skin, CM) or in various organs (systemic mastocytosis, SM). This leads to a wide variety of clinical manifestations resulting from excessive mediator release in CM and benign forms of SM (indolent SM, ISM) and from tissue mast cell infiltration causing multiorgan dysfunction and failure in more aggressive subtypes (aggressive SM, ASM, or mast cell leukemia). In addition, SM may be associated with hematological neoplasms (AHN). Read More

    Natural history and treatment of cutaneous and systemic mastocytosis.
    Postgrad Med 2017 Aug 21:1-6. Epub 2017 Aug 21.
    a Division of Allergy, Immunology and Dermatology, Department of Pediatrics , McGill University Health Centre, Montreal Children's Hospital , Montreal , Canada.
    Introduction: Mastocytosis, a heterogeneous group of disorders, is characterized by an abnormal increase in the number of mast cells that is limited to the skin (cutaneous mastocytosis), involving extracutaneous tissues (systemic mastocytosis), or presenting as solid tumours (mastocytoma and mast cell sarcoma). Recent studies estimate that 1 in 10,000 people are diagnosed with mastocytosis. Although prompt diagnosis and appropriate management are crucial, little is known about the natural history and currently there are no established management guidelines. Read More

    Dystonia as an unusual presentation of systemic mastocytosis: possible link between histamine release and movement disorders.
    J Allergy Clin Immunol Pract 2017 Jul 27. Epub 2017 Jul 27.
    Division of Allergy and Clinical Immunology, Department of Pediatrics, National Jewish Health, Denver, Colo; Department of Pediatrics, University of Colorado School of Medicine, Aurora, Colo. Electronic address:

    Efficacy and safety of midostaurin in patients with advanced systemic mastocytosis: 10-year median follow-up of a phase II trial.
    Leukemia 2017 Jul 24. Epub 2017 Jul 24.
    Division of Hematology, Department of Medicine, Stanford University School of Medicine/Stanford Cancer Institute, Stanford, CA, USA.
    Patients with advanced systemic mastocytosis (SM) (e.g. aggressive SM (ASM), SM with an associated hematologic neoplasm (SM-AHN) and mast cell leukemia (MCL)) have limited treatment options and exhibit reduced survival. Read More

    Insights in Anaphylaxis and Clonal Mast Cell Disorders.
    Front Immunol 2017 10;8:792. Epub 2017 Jul 10.
    Instituto de Estudios de Mastocitosis de Castilla La Mancha (CLMast), Hospital Virgen del Valle, Toledo, Spain.
    The prevalence of anaphylaxis among patients with clonal mast cell disorders (MCD) is clearly higher comparing to the general population. Due to a lower frequency of symptoms outside of acute episodes, clonal MCD in the absence of skin lesions might sometimes be difficult to identify which may lead to underdiagnosis, and anaphylaxis is commonly the presenting symptom in these patients. Although the release of mast cell (MC) mediators upon MC activation might present with a wide variety of symptoms, particular clinical features typically characterize MC mediator release episodes in patients with clonal MCD without skin involvement. Read More

    Management of Mastocytosis in Pregnancy: A Review.
    J Allergy Clin Immunol Pract 2017 Sep - Oct;5(5):1217-1223. Epub 2017 Jul 21.
    Department of Internal Medicine, Division of Allergy and Clinical Immunology, University of Michigan, Ann Arbor, Mich. Electronic address:
    In this article, the authors present a case of pregnancy complicated by the need for management of indolent systemic mastocytosis. The diagnosis of mastocytosis is reviewed, as well as subtypes of mastocytosis and management options particularly in pregnancy. A table of pregnancy/lactaction categories for common medications used in mastocytosis is presented. Read More

    Leveraging Siglec-8 endocytic mechanisms to kill human eosinophils and malignant mast cells.
    J Allergy Clin Immunol 2017 Jul 20. Epub 2017 Jul 20.
    Division of Allergy and Immunology, Department of Medicine, Feinberg School of Medicine, Northwestern University, Chicago, Ill; Division of Allergy and Clinical Immunology, Department of Medicine, The Johns Hopkins University School of Medicine, Baltimore, Md. Electronic address:
    Background: Sialic acid-binding immunoglobulin-like lectin (Siglec)-8 is a cell-surface protein expressed selectively on human eosinophils, mast cells, and basophils, making it an ideal target for the treatment of diseases involving these cell types. However, the effective delivery of therapeutic agents to these cells requires an understanding of the dynamics of Siglec-8 surface expression.

    Objectives: We sought to determine whether Siglec-8 is endocytosed in human eosinophils and malignant mast cells, identify mechanisms underlying its endocytosis, and demonstrate whether a toxin can be targeted to Siglec-8-bearing cells to kill these cells. Read More

    Mastocytosis: from a Molecular Point of View.
    Clin Rev Allergy Immunol 2017 Jul 19. Epub 2017 Jul 19.
    Floridsdorf Allergy Center (FAZ), Vienna, Austria.
    Mast cells (MCs) are physiologically activated by binding of stem cell factor (SCF) to the extracellular domains of the Kit receptor. This binding increases the proliferation and prolongs the survival of normal mature MCs, as well as intensifies the release of mediators. In mastocytosis, somatic mutations of the coding Kit gene cause autocrine dysregulation and lead to constitutive KIT activation even in the absence of its ligand SCF. Read More

    House Dust Mite-induced Allergic Airway Disease is Independent of IgE and FcɛRIα.
    Am J Respir Cell Mol Biol 2017 Jul 12. Epub 2017 Jul 12.
    University of Cincinnati, 2514, Medicine, Cincinnati, Ohio, United States.
    IgE contributes to disease exacerbations but not to baseline airway hyperresponsiveness (AHR) in human asthma. In rodent allergic airway disease (AAD), mast cell and IgE dependence for the induction of AHR has only been observed when mice are immunized with a relatively weak allergen without adjuvant. To evaluate the role of IgE in murine AAD that is induced by a potent allergen, we inoculated BALB/c and FVB/N background wild-type, and IgE- or FcεRIα-deficient mice intratracheally with large or limiting doses of house dust mite extract (HDM) and evaluated AHR, pulmonary eosinophilia, goblet cell metaplasia, serum IgE, and lung mastocytosis. Read More

    Ultraviolet radiation and skin mast cells: Effects, mechanisms and relevance for skin diseases.
    Exp Dermatol 2017 Jul 4. Epub 2017 Jul 4.
    Department of Dermatology and Allergy, Allergie-Centrum-Charité, Charité - Universitätsmedizin, Berlin, Germany.
    Mast cells (MCs) are well known as versatile effector cells in allergic reactions and several other immune responses. Skin MCs and cutaneous MC responses are subject to the effects of environmental factors including ultraviolet radiation (UVR). Numerous studies have assessed the effects of UVR on MCs, in vitro and in vivo. Read More

    Tyrosine Kinase Inhibitors in the Treatment of Eosinophilic Neoplasms and Systemic Mastocytosis.
    Hematol Oncol Clin North Am 2017 Aug;31(4):643-661
    Division of Hematology, Stanford Cancer Institute/Stanford University School of Medicine, 875 Blake Wilbur Drive, Room 2324, Stanford, CA 94305-5821, USA. Electronic address:
    The World Health Organization's semimolecular classification of eosinophilias emphasizes neoplasms driven by fusion tyrosine kinases. More than 80% of patients with systemic mastocytosis carry the KIT D816V mutation, the primary driver of disease pathogenesis. Genetic annotation of these diseases is critical and affords opportunities for targeted therapy. Read More

    SETD2 and histone H3 lysine 36 methylation deficiency in advanced systemic mastocytosis.
    Leukemia 2017 Jun 16. Epub 2017 Jun 16.
    Department of Experimental, Diagnostic and Specialty Medicine, Hematology/Oncology 'L. e A. Seràgnoli', University of Bologna, Bologna, Italy.
    The molecular basis of advanced systemic mastocytosis (SM) is not fully understood and despite novel therapies the prognosis remains dismal. Exome sequencing of an index-patient with mast cell leukemia (MCL) uncovered biallelic loss-of-function mutations in the SETD2 histone methyltransferase gene. Copy-neutral loss-of-heterozygosity at 3p21. Read More

    Omalizumab prevents anaphylaxis and improves symptoms in systemic mastocytosis: Efficacy and safety observations.
    Allergy 2017 Jun 29. Epub 2017 Jun 29.
    Department of Dermatology and Allergy Centre, Odense Research Centre for Anaphylaxis (ORCA), Odense University Hospital, Odense, Denmark.
    Background: Patients with systemic mastocytosis (SM) may suffer from mast cell (MC) mediator-related symptoms insufficiently controlled by conventional therapy. Omalizumab is an established treatment in other MC-driven diseases, but experiences in SM are limited.

    Objective: To assess the efficacy and safety of omalizumab in SM. Read More

    Acquired bilateral telangiectasia macularis eruptiva perstans: A unique clinical feature of photodamaging rather than a subtype of cutaneous mastocytosis.
    J Dermatol 2017 Jun 23. Epub 2017 Jun 23.
    Department of Dermatology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea.
    Telangiectasia macularis eruptiva perstans (TMEP) is a rare subtype of cutaneous mastocytosis, characterized by telangiectatic tan to brown macules on the trunk and extremities. Although TMEP has been descried as an uncommon disease in the literature, we often encounter patients with TMEP lesions in the outpatient clinic. We aimed to assess the clinical and histopathological characteristics of acquired bilateral TMEP, and the pathophysiological mechanism of acquired bilateral TMEP among these patients. Read More

    Angioedema: Perioperative management.
    SAGE Open Med Case Rep 2017 8;5:2050313X17713912. Epub 2017 Jun 8.
    Department of Anesthesiology and Critical Care Medicine, Vanderbilt University Medical Center, Nashville, TN, USA.
    Objective: To describe the perioperative management of a patient with acquired angioedema (AAE).

    Methods: A 66-year-old Caucasian male presented from an outside hospital with a history of acquired angioedema and gastrointestinal stromal tumor-related intractable urticaria and mastocytosis. He was admitted for urgent laparoscopic partial gastrectomy, secondary to gastric outlet obstruction symptomatology. Read More

    Management of poorly controlled indolent systemic mastocytosis using narrowband UVB phototherapy.
    Cutis 2017 May;99(5):E30-E33
    Georgetown University Hospital/Washington Hospital Center, Washington, DC, USA.
    The mastocytoses comprise a group of proliferative stem cell disorders defined by the abnormal accumulation of mast cells (MCs) in the skin or other body tissues including the bone marrow, gastrointestinal tract, and liver. Systemic mastocytosis is defined by the presence of one major and one minor criterion or 3 minor criteria delineated by the World Health Organization (WHO). We present the case of a 57-year-old woman with a 10-year history of red-brown pruritic maculopapular lesions on the upper and lower extremities and trunk who was originally diagnosed with cutaneous mastocytosis. Read More

    A distinct biomolecular profile identifies monoclonal mast cell disorders in patients with idiopathic anaphylaxis.
    J Allergy Clin Immunol 2017 Jun 16. Epub 2017 Jun 16.
    Laboratory of Allergic Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md.
    Background: Clonal mast cell disorders are known to occur in a subset of patients with systemic reactions to Hymenoptera stings. This observation has prompted the question of whether clonal mast cell disorders also occur in patients with idiopathic anaphylaxis (IA).

    Objective: We sought to determine the prevalence of clonal mast cell disorders among patients with IA, criteria to identify those patients who require a bone marrow biopsy, and whether the pathogenesis of IA involves a hyperresponsive mast cell compartment. Read More

    Mastocytosis: A case series of 107 consecutive patients.
    Br J Dermatol 2017 Jun 16. Epub 2017 Jun 16.
    The University of Melbourne - Faculty of Medicine,Dentistry and Health Sciences, Melbourne, Victoria, Australia.
    Mastocytosis is classified by the World-Health-Organisation (WHO)(1) as cutaneous-mastocytosis (CM) and systemic-mastocytosis (SM). CM is subdivided into maculopapular (MPCM), diffuse-CM and mastocytomas. SM is subdivided into indolent (ISM), with-an-associated-haematologic-neoplasm (SM-AHN)(2) , aggressive (ASM) and mast-cell-leukemia (MCL). Read More

    Midostaurin: First Global Approval.
    Drugs 2017 Jul;77(11):1251-1259
    Springer, Private Bag 65901, Mairangi Bay, 0754, Auckland, New Zealand.
    Midostaurin (Rydapt(®)) is a multikinase inhibitor being developed by Novartis Pharmaceuticals. In April 2017, midostaurin was approved in the USA for the treatment of adult patients with newly diagnosed, FMS-like tyrosine kinase 3 (FLT3) mutation-positive acute myeloid leukaemia (AML) [in combination with standard cytarabine and daunorubicin induction, and cytarabine consolidation], or aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated haematological neoplasm (SM-AHN) or mast cell leukaemia (MCL) [collectively known as advanced SM]. The article summarizes the milestones in the development of midostaurin leading to this first global approval. Read More

    Midostaurin for the treatment of acute myeloid leukemia.
    Future Oncol 2017 Sep 14;13(21):1853-1871. Epub 2017 Jun 14.
    Department of Hematology, Mayo Clinic, Rochester, MN 55905, USA.
    Midostaurin is a multikinase tyrosine kinase inhibitor acting against targets known to be expressed in hematologic malignancies, especially acute myeloid leukemia. Midostaurin combined with chemotherapy followed by single-agent maintenance therapy elicited statistically significant and clinically meaningful improvement in overall survival versus placebo in patients with newly diagnosed FLT3-mutant acute myeloid leukemia. Although gastrointestinal events were more common with midostaurin, overall the drug was relatively well tolerated. Read More

    Mast cell leukemia (MCL): Clinico-pathologic and molecular features and survival outcome.
    Leuk Res 2017 Aug 1;59:105-109. Epub 2017 Jun 1.
    Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX, United States. Electronic address:
    Mast cell leukemia (MCL) is a very rare subtype of systemic mastocytosis (SM). We have identified 13 such patients (5.9%) among 218 patients with SM seen at our institution between 1994 and 2016. Read More

    KIT(D816V) Induces SRC-Mediated Tyrosine Phosphorylation of MITF and Altered Transcription Program in Melanoma.
    Mol Cancer Res 2017 Sep 5;15(9):1265-1274. Epub 2017 Jun 5.
    Division of Translational Cancer Research, Lund Stem Cell Center, Lund University, Medicon Village and Department of Oncology, Skåne University Hospital, Lund, Sweden.
    The oncogenic D816V mutation of the KIT receptor is well characterized in systemic mastocytosis and acute myeloid leukemia. Although KIT(D816V) has been found in melanoma, its function and involvement in this malignancy is not understood. Here we show that KIT(D816V) induces tyrosine phosphorylation of MITF through a triple protein complex formation between KIT, MITF, and SRC family kinases. Read More

    Acute Myeloid Leukemia With Inv(16)(p13q22) Associated With Hidden Systemic Mastocytosis: Case Report and Review of Literature.
    Clin Med Insights Blood Disord 2017 30;10:1179545X17700858. Epub 2017 Mar 30.
    Department of Hematology/Oncology, National Center for Cancer Care and Research, Hamad Medical Corporation, Doha, Qatar.
    Systemic mastocytosis (SM) is a condition associated with clonal neoplastic proliferation of mast cells. In up to 40% of systemic mastocytosis cases, an associated clonal hematological disease of non-mast cell lineage, such as acute myeloid leukemia (AML), is diagnosed before, simultaneously with, or after the diagnosis of SM. Herein, we report a case of a 30-year-old man diagnosed with AML with inv(16) (p13;q22) CBFB:MYH11. Read More

    Improved efficacy of allergen-specific immunotherapy by JAK inhibition in a murine model of allergic asthma.
    PLoS One 2017 1;12(6):e0178563. Epub 2017 Jun 1.
    Center of Allergy and Environment (ZAUM), Technical University of Munich and Helmholtz Center Munich, Member of the German Center for Lung research (DZL), Munich, Germany.
    Background: Allergen-specific immunotherapy (AIT) is the only curative treatment for type-1 allergies, but sometimes shows limited therapeutic response as well as local and systemic side effects. Limited control of local inflammation and patient symptoms hampers its widespread use in severe allergic asthma.

    Objective: Our aim was to evaluate whether AIT is more effective in suppression of local inflammation if performed under the umbrella of short-term non-specific immunomodulation using a small molecule inhibitor of JAK pathways. Read More

    Pharmacotherapy of mast cell disorders.
    Curr Opin Allergy Clin Immunol 2017 Aug;17(4):295-303
    aDepartment of Respiratory Medicine and Allergy, Karolinska University Hospital, Huddinge bDepartment of Medicine Solna, Immunology and Allergy Unit, Karolinska Institutet and Karolinska University Hospital cMastocytosis Centre Karolinska, Karolinska University Hospital and Karolinska Institutet, Stockholm, Sweden dDepartment of Internal Medicine, Division of Allergy and Clinical Immunology, University of Michigan, Ann Arbor, Michigan, USA.
    Purpose Of Review: Mast cell disorders (MCDs) comprise mastocytosis and disorders referred to as mast cell activation syndrome and are caused by abnormal accumulation and/or activation of mast cells in tissues. Clinical signs and symptoms are protean; therefore, finding suitable treatment options for individual patients entails a challenge for clinicians. The purpose of this manuscript is to review the literature on the available therapeutic interventions in patients with MCD. Read More

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