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Congenital and Acquired Disorders of Macrophages and Histiocytes, An Issue of Hematology/Oncology Clinics of North America, E-Book

Congenital and Acquired Disorders of Macrophages and Histiocytes, An Issue of Hematology/Oncology Clinics of North America, E-Book

Nancy Berliner

(2016)

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Book Details

Abstract

This issue of Hematology/Oncology Clinics of North America is devoted to Congenital and Acquired Disorders of Macrophages and Histiocytes.  Guest Editors Nancy Berliner, MD and Barrett Rollins, MD have assembled a group of expert authors to review the following topics: Nosology of Langerhans Cell Histiocytosis (LCH); Cell of Origin of LCH; Genomic Changes in LCH; Clinical Treatment of LCH; Neurodegeneration in LCH; Pathogenesis of Hemophagocytic Lymphohistiocytosis (HLH); Familial HLH; HLH in Adults; Macrophage Activation Syndrome; and Stem Cell Transplant for HLH.

Table of Contents

Section Title Page Action Price
Front Cover Cover
Congenital and Acquired Disorders of Macrophages and \rHistiocytes i
Copyright\r ii
Contributors iii
CONSULTING EDITORS iii
EDITORS iii
AUTHORS iii
Contents vii
Dedication\r vii
Preface: Congenital and Acquired Disorders of Macrophages and Histiocytes\r vii
Nosology and Pathology of Langerhans Cell Histiocytosis\r vii
Cell(s) of Origin of Langerhans Cell Histiocytosis\r vii
Genomic Alterations in Langerhans Cell Histiocytosis\r vii
Clinical Characteristics and Treatment of Langerhans Cell Histiocytosis\r viii
Strategies for the Prevention of Central Nervous System Complications in\rPatients with Langerhans Cell Histiocytosis: The Problem of Neurodegenerative\rSyndrome\r viii
Pathogenesis of Hemophagocytic Lymphohistiocytosis\r viii
Familial Hemophagocytic Lymphohistiocytosis\r viii
Hemophagocytic Lymphohistiocytosis in Adults\r ix
Macrophage Activation Syndrome\r ix
The Role of Hematopoietic Stem Cell Transplantation in Treatment of Hemophagocytic Lymphohistiocytosis\r ix
HEMATOLOGY/ONCOLOGY\rCLINICS OF NORTH AMERICA\r x
FORTHCOMING ISSUES x
December 2015 x
February 2016 x
April 2016 x
RECENT ISSUES x
August 2015 x
June 2015 x
April 2015 x
Dedication xi
Preface\r xiii
Nosology and Pathology of Langerhans Cell Histiocytosis 799
Key points 799
PATHOLOGY OF THE HISTIOCYTOSES 799
GENERAL HISTOPATHOLOGY 800
HISTOPATHOLOGY IN SPECIFIC ORGANS 802
Bone 802
Skin 803
Lymph Nodes 805
High-Risk Organ Involvement 808
Bone marrow 808
Spleen 810
Liver 810
Thymus 812
Thyroid 813
Lung 813
Gastrointestinal Tract 815
Central Nervous System 815
SUMMARY 818
REFERENCES 818
Cell(s) of Origin of Langerhans Cell Histiocytosis 825
Key points 825
INTRODUCTION TO LANGERHANS CELL HISTIOCYTOSIS 825
The Histiocytoses 825
Clinical Overview 827
Rationale for Current Approaches to Langerhans Cell Histiocytosis 827
MOLECULAR INSIGHTS INTO PATHOGENESIS OF LANGERHANS CELL HISTIOCYTOSIS 827
Langerhans Cell Histiocytosis: The Debate 827
Somatic Mitogen-Activated Protein Kinase Mutations in Langerhans Cell Histiocytosis 827
BRANCHES OF DENDRITIC CELL DIFFERENTIATION 829
The Phenotype of Langerhans Cell Histiocytosis Dendritic Cells 829
Origin and Homeostasis of Langerhans Cells in the Steady State 830
Replenishment of Langerhans Cells During Inflammation 830
Dual Inflammatory Langerhans Cell Precursors 830
Langerhans Cells Are Not the Only Fruit: Other Human Dendritic Cells 831
Langerin-Positive Dendritic Cells 831
REVISITING THE CELL OF ORIGIN IN LANGERHANS CELL HISTIOCYTOSIS 832
Lineage Tracing Langerhans Cell Histiocytosis 832
An Updated Model of Langerhans Cell Histiocytosis 832
Juvenile Xanthogranuloma and Erdheim-Chester Disease: Cousins or Siblings of Langerhans Cell Histiocytosis? 833
Langerhans Cell Histiocytosis as a Myeloproliferative Disorder 833
SUMMARY/FUTURE DIRECTIONS 834
REFERENCES 834
Genomic Alterations in Langerhans Cell Histiocytosis 839
Key points 839
INTRODUCTION 839
MITOGEN-ACTIVATED PROTEIN KINASE PATHWAY ACTIVATION 840
BRAF 840
ARAF 842
MAP2K1 843
MAP3K1 845
NRAS 845
PIK3CA 846
Tumor Protein 53 846
Others 846
Translocations and Copy Number Changes 846
CLINICAL IMPLICATIONS 847
SUMMARY 847
REFERENCES 848
Clinical Characteristics and Treatment of Langerhans Cell Histiocytosis 853
Key points 853
INTRODUCTION 853
BIOLOGY 854
EPIDEMIOLOGY 854
PATHOLOGY 854
CLINICAL PRESENTATION 854
SITES OF INVOLVEMENT 855
Bone 855
Skin 856
Neuroendocrine and Central Nervous Systems 857
Pulmonary 857
Hematopoietic System 857
Hepatobiliary System 857
TREATMENT OF LANGERHANS CELL HISTIOCYTOSIS 857
Histiocyte Society’s Langerhans Cell Histiocytosis Studies 858
Langerhans Cell Histiocytosis-I (LCH-I) 858
Langerhans Cell Histiocytosis-II (LCH-II) 858
Langerhans Cell Histiocytosis-III (LCH-III) 858
Langerhans Cell Histiocytosis-IV 861
Stratum I 861
Stratum II 861
Stratum III 861
Stratum IV 861
Stratum V 861
Stratum VI 861
Stratum VII 861
DAL-HX Studies 862
Japan Langerhans Cell Histiocytosis Study Group and the Role Cytarabine 862
TREATMENT OF RECURRENT LANGERHANS CELL HISTIOCYTOSIS 863
2-Chlorodeoxyadenosine (Cladribine) 864
Clofarabine 864
Cytarabine 864
Bisphosphonates 864
Indomethacin 865
BRAF Inhibitors 865
Imatinib Mesylate 865
Hematopoietic Stem Cell Transplantation 865
SPECIAL CONSIDERATIONS 865
Adult Langerhans Cell Histiocytosis 865
Pulmonary Langerhans Cell Histiocytosis 866
Central Nervous System Langerhans Cell Histiocytosis 866
SUMMARY AND FUTURE DIRECTIONS 868
REFERENCES 868
Strategies for the Prevention of Central Nervous System Complications in Patients with Langerhans Cell Histiocytosis 875
Key points 875
INTRODUCTION 876
LABORATORY STUDIES OF RELEVANCE TO CENTRAL NERVOUS SYSTEM-LANGERHANS CELL HISTIOCYTOSIS DISEASE 877
Cytokines/Chemokines and Other Cerebrospinal Fluid Markers in Neuroinflammatory Diseases 877
Matrix Metalloproteinases and Other Cerebrospinal Fluid Molecules in Neuroinflammatory Diseases 880
Autoantibodies and Neuroinflammation 880
Complement Activation Cerebrospinal Fluid Markers in Neuroinflammatory and Neurodegenerative Diseases 881
Cerebrospinal Fluid Biomarkers in Alzheimer Disease and Parkinson Disease 882
LABORATORY AND THERAPEUTIC STUDIES TO PREVENT AND TREAT NEURODEGENERATION-CENTRAL NERVOUS SYSTEM-LANGERHANS CELL HISTIOCYTO ... 882
Patients at High Risk versus Low Risk for Central Nervous System-Langerhans Cell Histiocytosis 882
Subclinical Versus Clinical Central Nervous System-Langerhans Cell Histiocytosis Lesions 882
Cerebrospinal Fluid Biomarkers and the Pathogenesis of Central Nervous System-Langerhans Cell Histiocytosis 883
Determination of Cerebrospinal Fluid Cytokines/Chemokines Concentrations 884
Treatment of Neurodegenerative-Central Nervous System-Langerhans Cell Histiocytosis 884
Opportunities for Prevention of Neurodegenerative-Central Nervous System-Langerhans Cell Histiocytosis 885
SUMMARY AND FUTURE DIRECTIONS 886
REFERENCES 886
Pathogenesis of Hemophagocytic Lymphohistiocytosis 895
Key points 895
INTRODUCTION 895
DISEASE DESCRIPTION 896
GENETICS OF HEMOPHAGOCYTIC LMPHOHISTIOCYTOSIS 896
SECONDARY HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS 899
PATHOPHYSIOLOGY 900
TREATMENT 900
REFERENCES 901
Familial Hemophagocytic Lymphohistiocytosis 903
Key points 903
INTRODUCTION 903
INCIDENCE 904
FAMILIAL HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS SUBTYPES 904
DIAGNOSTIC WORK-UP 907
Flow Cytometry 908
Natural Killer Function 908
CD107a 908
TREATMENT 909
INITIAL THERAPY FOR FAMILIAL HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS 909
HEMATOPOIETIC CELL TRANSPLANTATION 910
SUMMARY/FUTURE DIRECTIONS 910
REFERENCES 910
Hemophagocytic Lymphohistiocytosis in Adults 915
Key points 915
INTRODUCTION 915
ACQUIRED HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS 916
Infection 917
Malignancy 917
Autoimmune Conditions, Macrophage Activation Syndrome 917
Diagnosis 918
Clinical Manifestations 918
Neurologic Manifestations 919
Dermatologic Manifestations 919
Laboratory Findings 919
Elevated ferritin 920
Hemophagocytosis 920
Liver function abnormality and coagulopathy 920
Soluble CD163 920
Depressed natural killer function 921
Elevated serum-soluble interleukin-2 receptor 921
Prognosis 921
Treatment 921
Refractory/Recurrent Disease 922
Treatment of Specific Populations 923
Treatment of the clinically stable patient 923
Infectious trigger 923
Rheumatologic trigger 923
Oncologic trigger 924
FUTURE DIRECTIONS 924
REFERENCES 924
Macrophage Activation Syndrome 927
Key points 927
INTRODUCTION 927
HISTORY, NOMENCLATURE, AND CLASSIFICATION 928
EPIDEMIOLOGY 928
PATHOGENESIS 928
CLINICAL, LABORATORY, AND HISTOPATHOLOGIC FEATURES 929
TRIGGERING FACTORS 931
DIAGNOSTIC GUIDELINES 932
THE NEW CLASSIFICATION CRITERIA 934
MANAGEMENT 935
REFERENCES 937
The Role of Hematopoietic Stem Cell Transplantation in Treatment of Hemophagocytic Lymphohistiocytosis 943
Key points 943
INTRODUCTION 943
ACHIEVING REMISSION IN PEDIATRIC HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS 944
RATIONALE FOR ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION IN PEDIATRIC HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS 945
ADVANCES IN CONDITIONING INTENSITY 947
USE OF ALTERNATIVE DONOR SOURCES 947
SPECIFIC ROLE OF ALEMTUZUMAB AND IMPORT OF POST–HEMATOPOIETIC STEM CELL TRANSPLANTATION CHIMERISM 948
ALGORITHM FOR HEMATOPOIETIC STEM CELL TRANSPLANTATION FAMILIAL VERSUS INFECTION-ASSOCIATED HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS 949
UNIQUE ASPECTS OF ADULT-ONSET HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS 951
RATIONALE FOR ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION IN ADULT HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS 952
LACK OF A CLEAR ALGORITHM IN ADULT-ONSET HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS 953
SUMMARY 955
REFERENCES 955
Index 961