Additional Information
Book Details
Abstract
This issue will assist the practicing pediatrician with providing evidence-based care to children with common, rare, inherited and acquired hematological disorders whom they regularly see in general pediatric practice. The information in this edition will support a general pediatrician’s understanding of recently developed diagnostic and therapeutic tools--for instance, techniques for the assessment of transfusional iron overload in chronically transfused patients – resulting in better surveillance of medication side effects and improved support for patients who are prescribed with complex chelation regimens. New, improved therapeutic approaches to treatment of children with venous thromboembolic disease have recently been introduced; affected patients often require close monitoring in their communities through the general pediatrician whose practice will be enhanced by information that has been prepared by pediatric specialists with pediatric patients in mind.
Table of Contents
| Section Title | Page | Action | Price |
|---|---|---|---|
| Front Cover | Cover | ||
| Pediatric Hematology | i | ||
| Copyright\r | ii | ||
| Contributors | v | ||
| Contents | ix | ||
| Pediatric Clinics Of North America\r | xiv | ||
| Preface\r | xv | ||
| Development of the Hematopoietic System and Disorders of Hematopoiesis that Present During Infancy and Early Childhood | 1273 | ||
| Key points | 1273 | ||
| Introduction | 1273 | ||
| Development of the hematopoietic system | 1274 | ||
| Fetal erythropoiesis | 1277 | ||
| Embryonic and fetal Hb | 1279 | ||
| Fetal thrombopoiesis | 1279 | ||
| Fetal myelopoiesis | 1283 | ||
| Fetal lymphopoiesis | 1283 | ||
| Disorders of hematopoiesis that manifest during infancy and early childhood | 1284 | ||
| Summary | 1286 | ||
| References | 1286 | ||
| The Inherited Bone Marrow Failure Syndromes | 1291 | ||
| Key points | 1291 | ||
| The ribosomopathies | 1292 | ||
| DBA | 1292 | ||
| DC | 1294 | ||
| Shwachman-Diamond Syndrome | 1295 | ||
| CHH | 1296 | ||
| FA/BRCA pathway | 1296 | ||
| Mitochondrial diseases | 1299 | ||
| Pearson Syndrome | 1299 | ||
| Reticular dysgenesis | 1300 | ||
| Congenital thrombocytopenias | 1300 | ||
| Amegakaryocytic Thrombocytopenia | 1300 | ||
| TAR Syndrome | 1300 | ||
| Congenital dyserythropoietic anemia | 1301 | ||
| Workup of a patient with bone marrow failure | 1302 | ||
| Cytogenetics | 1302 | ||
| DEB Chromosome Fragility Assay | 1302 | ||
| Flow Cytometry | 1302 | ||
| Genotype Analysis | 1302 | ||
| Infectious Causes | 1303 | ||
| Telomere Length Analysis | 1303 | ||
| Research | 1303 | ||
| References | 1303 | ||
| Acquired Aplastic Anemia in Children | 1311 | ||
| Key points | 1312 | ||
| Introduction | 1312 | ||
| Definitions | 1312 | ||
| Epidemiology | 1313 | ||
| Pathogenesis | 1314 | ||
| Clinical presentation | 1314 | ||
| Establishing the diagnosis | 1315 | ||
| Supportive care | 1318 | ||
| Definitive treatment | 1322 | ||
| Bone marrow transplant | 1322 | ||
| Matched Related Donor Bone Marrow Transplantation for Aplastic Anemia | 1322 | ||
| Conditioning | 1322 | ||
| Graft source | 1323 | ||
| Graft failure, GVHD, and donor chimerism | 1324 | ||
| Alternative Donor Bone Marrow Transplantation for Aplastic Anemia | 1324 | ||
| Donor selection | 1324 | ||
| Conditioning regimens | 1325 | ||
| Follow-Up and Late Effects After Stem Cell Transplantation for Acquired AA | 1325 | ||
| Immunosuppressive therapy | 1327 | ||
| G-CSF | 1328 | ||
| Alternative First-Line Therapies | 1328 | ||
| Moderate or nonsevere AA | 1328 | ||
| Response, relapse, and late effects after IST for acquired AA | 1329 | ||
| Response | 1329 | ||
| Relapse | 1329 | ||
| Second-Line Therapy | 1329 | ||
| Late Complications | 1329 | ||
| Summary | 1330 | ||
| Acknowledgments | 1331 | ||
| References | 1331 | ||
| Hematologic Manifestations of Systemic Disease (Including Iron Deficiency, Anemia of Inflammation and DIC) | 1337 | ||
| Key points | 1337 | ||
| Introduction | 1337 | ||
| Structure and function of hemoglobin | 1337 | ||
| Iron deficiency anemia | 1338 | ||
| Epidemiology | 1338 | ||
| Pathophysiology | 1338 | ||
| Clinical Manifestations | 1340 | ||
| Diagnostic Evaluation | 1340 | ||
| Treatment | 1341 | ||
| Anemia of inflammation | 1342 | ||
| Epidemiology | 1342 | ||
| Pathophysiology | 1342 | ||
| Clinical Manifestations | 1343 | ||
| Diagnostic Evaluation | 1343 | ||
| Treatment | 1343 | ||
| Disseminated intravascular coagulation | 1344 | ||
| Epidemiology | 1344 | ||
| Pathophysiology | 1344 | ||
| Diagnostic Evaluation | 1345 | ||
| Treatment | 1345 | ||
| References | 1346 | ||
| Abnormalities of the Erythrocyte Membrane | 1349 | ||
| Key points | 1349 | ||
| Introduction | 1349 | ||
| Hereditary spherocytosis | 1349 | ||
| Clinical manifestations and classification | 1350 | ||
| Initial assessment/physical examination | 1351 | ||
| Laboratory findings | 1352 | ||
| Erythrocyte Indices | 1352 | ||
| Peripheral Blood Smear | 1352 | ||
| EMA Binding and Osmotic Fragility | 1353 | ||
| Specialized Testing and Molecular Studies | 1354 | ||
| Other Laboratory Findings | 1354 | ||
| Diagnosis of HS in the neonate | 1354 | ||
| Imaging studies | 1355 | ||
| Differential diagnosis | 1355 | ||
| Complications | 1355 | ||
| Gallbladder Disease | 1355 | ||
| Crises | 1355 | ||
| Treatment | 1356 | ||
| Splenectomy | 1356 | ||
| Hereditary elliptocytosis, hereditary pyropoikilocytosis, and related disorders | 1357 | ||
| Introduction | 1357 | ||
| Clinical Manifestations | 1358 | ||
| Laboratory Findings | 1358 | ||
| Imaging Studies | 1358 | ||
| Differential Diagnosis | 1358 | ||
| Complications | 1359 | ||
| Treatment | 1359 | ||
| Summary | 1359 | ||
| References | 1359 | ||
| Sickle Cell Disease in Childhood | 1363 | ||
| Key points | 1363 | ||
| Introduction | 1363 | ||
| Hemoglobin, genetics, and basic pathophysiology | 1364 | ||
| Forms of SCD and diagnosis | 1364 | ||
| Clinical scenarios and complications of SCD | 1367 | ||
| The Newborn and Infant | 1367 | ||
| Hemolytic Anemia | 1367 | ||
| Hyposplenism, Fever, and Sepsis | 1368 | ||
| Splenic Sequestration | 1369 | ||
| Transient Aplastic Crisis | 1369 | ||
| Painful Events and Bony Complications | 1370 | ||
| Pulmonary Complications | 1372 | ||
| Neurologic Complications | 1373 | ||
| Renal and Genitourinary Complications | 1375 | ||
| Hepatobiliary Complications | 1375 | ||
| Surgery and Anesthesia | 1375 | ||
| Disease-modifying therapies | 1376 | ||
| Hydroxyurea | 1376 | ||
| Chronic Transfusions | 1377 | ||
| Hematopoietic Stem Cell Transplantation | 1378 | ||
| Disease severity and prognostication | 1378 | ||
| Survival and transition to adult medical care | 1378 | ||
| Summary | 1379 | ||
| References | 1379 | ||
| Thalassemias | 1383 | ||
| Key points | 1383 | ||
| Introduction/history | 1383 | ||
| Epidemiology | 1383 | ||
| α-Thalassemia | 1384 | ||
| β-Thalassemia | 1384 | ||
| Pathophysiology | 1385 | ||
| Diagnosis of thalassemia | 1385 | ||
| Complications of thalassemia | 1386 | ||
| Growth Impairment | 1386 | ||
| Bone Abnormalities | 1386 | ||
| Endocrinopathies | 1386 | ||
| Splenomegaly and Hypersplenism | 1387 | ||
| Hypercoagulability | 1387 | ||
| Pulmonary Hypertension | 1387 | ||
| Treatment for thalassemia | 1387 | ||
| Transfusion Therapy | 1387 | ||
| HbF Induction | 1388 | ||
| Hydroxyurea | 1388 | ||
| DNA methylation inhibitors | 1388 | ||
| Short-chain fatty acids | 1388 | ||
| Stem Cell Transplantation | 1388 | ||
| Splenectomy | 1389 | ||
| Management/observation of patients with thalassemia | 1390 | ||
| Summary | 1390 | ||
| References | 1390 | ||
| Evaluation and Treatment of Transfusional Iron Overload in Children | 1393 | ||
| Key points | 1393 | ||
| Introduction | 1393 | ||
| How transfusions lead to iron overload | 1394 | ||
| Toxicity of iron | 1394 | ||
| Evaluation of iron overload | 1394 | ||
| Goals of iron chelation | 1395 | ||
| Pharmacologic strategies | 1396 | ||
| Deferoxamine | 1396 | ||
| Deferiprone | 1397 | ||
| Deferasirox | 1397 | ||
| Evaluation/adjustment | 1401 | ||
| Combination chelation therapy | 1401 | ||
| Other oral chelators in development | 1402 | ||
| Summary | 1402 | ||
| References | 1402 | ||
| Developmental Hemostasis | 1407 | ||
| Key points | 1407 | ||
| Introduction | 1407 | ||
| Hemostasis physiology | 1408 | ||
| Hemostasis in the infant and child | 1408 | ||
| Coagulation testing in the infant and child | 1410 | ||
| Protective mechanisms | 1412 | ||
| Impact of developmental hemostasis in the clinic | 1412 | ||
| Vitamin K Deficiency | 1412 | ||
| Hemophilia | 1413 | ||
| Thrombosis | 1414 | ||
| Impact of developmental hemostasis on anticoagulation management | 1414 | ||
| Summary | 1415 | ||
| References | 1416 | ||
| Inherited Abnormalities of Coagulation | 1419 | ||
| Key points | 1419 | ||
| Introduction | 1419 | ||
| Overview of hemostasis | 1420 | ||
| Hemophilia | 1421 | ||
| Introduction | 1421 | ||
| Molecular Basis and Genetics | 1422 | ||
| Clinical Presentation and Diagnosis | 1423 | ||
| Clinical Management | 1426 | ||
| General overview | 1426 | ||
| Management of acute bleeds | 1427 | ||
| Factor replacement | 1427 | ||
| Local measures | 1427 | ||
| Desmopressin | 1427 | ||
| Antifibrinolytic agents | 1428 | ||
| Long-term management: prophylaxis versus episodic therapy | 1428 | ||
| The Future of Hemophilia Care | 1428 | ||
| Long-acting factor concentrates | 1428 | ||
| Gene therapy | 1429 | ||
| Inhibitors in Hemophilia | 1429 | ||
| von Willebrand disease | 1430 | ||
| Introduction | 1430 | ||
| Molecular Basis and Genetics | 1430 | ||
| Classification and Laboratory Diagnosis | 1431 | ||
| Type 1 VWD | 1431 | ||
| Type 2 VWD | 1432 | ||
| Type 2A VWD | 1432 | ||
| Type 2B VWD | 1432 | ||
| Type 2M VWD | 1432 | ||
| Type 2N (Normandy) VWD | 1433 | ||
| Type 3 VWD | 1433 | ||
| Clinical Management | 1433 | ||
| Estrogens | 1433 | ||
| Desmopressin | 1433 | ||
| VWF/FVIII concentrate | 1433 | ||
| Rare inherited coagulation disorders | 1433 | ||
| References | 1435 | ||
| Pediatric Thrombophilia | 1443 | ||
| Key points | 1443 | ||
| Introduction | 1443 | ||
| Thrombophilia in the neonatal period | 1444 | ||
| Inherited Thrombophilia Disorders: Hypercoagulable States | 1444 | ||
| Protein C and protein S deficiencies | 1444 | ||
| Mechanism | 1444 | ||
| Classification | 1444 | ||
| Epidemiology | 1444 | ||
| Diagnosis | 1445 | ||
| Acute treatment | 1445 | ||
| Long-term management | 1445 | ||
| Antithrombin deficiency | 1445 | ||
| Mechanism | 1445 | ||
| Classification | 1446 | ||
| Epidemiology | 1446 | ||
| Diagnosis | 1446 | ||
| Treatment | 1446 | ||
| Homozygous/heterozygous factor V Leiden mutation | 1447 | ||
| Antithrombotic Therapies | 1463 | ||
| Key points | 1463 | ||
| Objectives | 1463 | ||
| Epidemiology of VTE | 1464 | ||
| Conventional anticoagulants | 1464 | ||
| Thrombolysis | 1465 | ||
| Settings | 1465 | ||
| Agents | 1467 | ||
| Modalities | 1467 | ||
| Systemic thrombolysis | 1467 | ||
| Catheter-directed thrombolysis/thrombolytic infusion | 1468 | ||
| Percutaneous mechanical/pharmaco-mechanical thrombectomy | 1468 | ||
| Contraindications/Safety Issues | 1469 | ||
| New oral anticoagulants: direct factor Xa and IIa (thrombin) inhibitors | 1469 | ||
| Summary | 1470 | ||
| References | 1471 | ||
| Inherited Disorders of Platelet Function | 1475 | ||
| Key points | 1475 | ||
| Introduction: nature of the problem | 1475 | ||
| Patient history | 1477 | ||
| Physical examination | 1480 | ||
| Diagnostic testing | 1482 | ||
| Mucocutaneous bleeding of unknown cause | 1484 | ||
| Pharmacologic treatment options | 1484 | ||
| Nonpharmacologic treatment options | 1485 | ||
| Supportive care and other issues | 1486 | ||
| Summary and future directions | 1486 | ||
| References | 1486 | ||
| Diagnosis and Management of Autoimmune Cytopenias in Childhood | 1489 | ||
| Key points | 1489 | ||
| Introduction | 1489 | ||
| Primary autoimmune cytopenia syndromes | 1490 | ||
| ITP | 1490 | ||
| AIHA | 1492 | ||
| AIN | 1493 | ||
| ES | 1494 | ||
| Secondary autoimmune cytopenia syndromes | 1497 | ||
| ALPS | 1497 | ||
| Treatment of single-lineage and multi-lineage autoimmune cytopenias | 1502 | ||
| Summary | 1506 | ||
| References | 1506 | ||
| HUS and TTP in Children | 1513 | ||
| Key points | 1513 | ||
| Introduction | 1513 | ||
| Classification | 1514 | ||
| Pathophysiology | 1515 | ||
| STEC-HUS | 1515 | ||
| aHUS | 1515 | ||
| TTP | 1516 | ||
| Epidemiology | 1516 | ||
| Clinical causes | 1517 | ||
| STEC-HUS | 1517 | ||
| Nonfamilial aHUS | 1517 | ||
| Familial aHUS | 1518 | ||
| TTP | 1518 | ||
| Diagnosis | 1518 | ||
| Clinical features | 1520 | ||
| Treatment | 1520 | ||
| STEC-HUS | 1520 | ||
| Atypical HUS | 1521 | ||
| TTP | 1521 | ||
| Prognosis | 1522 | ||
| STEC-HUS | 1522 | ||
| aHUS | 1522 | ||
| TTP | 1523 | ||
| Summary | 1523 | ||
| References | 1523 | ||
| Transfusion and Hemovigilance in Pediatrics | 1527 | ||
| Key points | 1527 | ||
| Hemovigilance: definition | 1528 | ||
| Hemovigilance: aims | 1529 | ||
| Local incident review | 1530 | ||
| Case study: a good root cause analysis | 1530 | ||
| Hemovigilance contributes to a reduction in pathologic incidents | 1530 | ||
| Reduction in Bacterial Infections | 1530 | ||
| Reduction in Cases of Transfusion-Related Acute Lung Injury | 1531 | ||
| Transfusion-associated Graft Versus Host Disease | 1531 | ||
| Hemovigilance leads to recognition of particular at-risk groups | 1531 | ||
| Patients Undergoing Hemopoietic Stem Cell Transplant | 1531 | ||
| Mistakes with Anti-D Administration to Pregnant Women | 1531 | ||
| Patients with Hemoglobinopathies | 1531 | ||
| Case study: severe delayed hemolysis with hyperhemolysis and death in SCD | 1532 | ||
| Case study: failure to inform the laboratory about the diagnosis of SCD | 1532 | ||
| Case study: routine transfusion audit detects inadequate identity and monitoring issues in chronically transfused patient w ... | 1532 | ||
| Hemovigilance reporting has resulted in several national initiatives to improve practice | 1532 | ||
| Pediatric patients have particular risks | 1533 | ||
| What do we know about transfusion complications in pediatrics? | 1534 | ||
| Case study: ABO incompatible FFP transfusion | 1535 | ||
| Case study: a massively overtransfused infant | 1535 | ||
| Uncategorized Complications of Transfusion | 1535 | ||
| Case study: NEC associated with transfusion with a fatal outcome | 1536 | ||
| ATR (allergic, hypotensive, and severe febrile) | 1536 | ||
| “Near miss” reporting | 1536 | ||
| Case study: near miss detected after several errors | 1537 | ||
| Summary | 1537 | ||
| References | 1537 | ||
| Blood Banking/Immunohematology | 1541 | ||
| Key points | 1541 | ||
| Donor selection and collection | 1541 | ||
| Component preparation | 1542 | ||
| Whole Blood–Derived Products | 1542 | ||
| Apheresis-Derived Products | 1543 | ||
| RBC/Whole Blood Products | 1544 | ||
| Plasma Products | 1545 | ||
| Platelet Products | 1545 | ||
| Cryoprecipitate | 1546 | ||
| Granulocytes | 1547 | ||
| Storage and the Storage Lesion | 1547 | ||
| Blood component modifications | 1552 | ||
| Leukoreduction | 1552 | ||
| Irradiation | 1552 | ||
| Washed Cellular Components | 1553 | ||
| Volume Reduction | 1554 | ||
| Special pediatric population needs | 1554 | ||
| Special Considerations for Neonates | 1554 | ||
| Special Considerations for Patients on Extracorporeal Life Support and Other Critically Ill Patients | 1555 | ||
| Older versus fresher RBCs | 1555 | ||
| Special Considerations for Hematopoietic Stem Cell Transplant Recipients | 1555 | ||
| Special Considerations for Patients with Sickle Cell Disease | 1555 | ||
| Special products | 1557 | ||
| Recombinant Products/Plasma Derivatives | 1557 | ||
| IgA-Deficient Products | 1558 | ||
| HLA-Matched/Crossmatch Compatible Platelets | 1558 | ||
| Antigen-Negative Platelet Products for Neonatal Alloimmune Thrombocytopenia | 1559 | ||
| Pathogen-Inactivated Blood Products | 1559 | ||
| Frozen Deglycerolized RBCs | 1561 | ||
| Special blood bank situations | 1561 | ||
| Massive Transfusion | 1561 | ||
| Emergency Release Blood | 1562 | ||
| Immunohematology techniques | 1562 | ||
| Neonatal Immunohematological Issues | 1562 | ||
| Summary | 1563 | ||
| References | 1563 | ||
| Pediatric Therapeutic Apheresis | 1569 | ||
| Key points | 1569 | ||
| Introduction/Background | 1569 | ||
| Vascular Access | 1570 | ||
| Procedural Risks | 1570 | ||
| Indications | 1571 | ||
| Hematologic | 1571 | ||
| Hemoglobinopathies | 1571 | ||
| Immune-mediated cytopenias | 1572 | ||
| Oncologic | 1572 | ||
| Hyperleukocytosis with or without leukostasis | 1572 | ||
| Peripheral hematopoietic progenitor cell collection | 1572 | ||
| Graft-versus-host disease | 1573 | ||
| Neurologic | 1573 | ||
| Acute inflammatory demyelinating polyneuropathy/Guillain-Barré syndrome | 1573 | ||
| Chronic Inflammatory Demyelinating Polyneuropathy | 1573 | ||
| Myasthenia Gravis | 1573 | ||
| Multiple Sclerosis and Neuromyelitis Optica | 1573 | ||
| Acute Disseminated Encephalomyelitis | 1574 | ||
| Chronic Focal Encephalitis (Rasmussens Encephalitis) | 1574 | ||
| Phytanic Acid Storage Disease (Refsum Disease) | 1574 | ||
| Miscellaneous | 1574 | ||
| Pediatric Autoimmune Neuropsychiatric Disorder Associated with Group A Streptococci and Syndenham Chorea | 1574 | ||
| Renal | 1575 | ||
| Focal segmental glomerulosclerosis | 1575 | ||
| Vasculitides | 1575 | ||
| Systemic lupus erythematosus | 1575 | ||
| Antiphospholipid antibody syndrome | 1576 | ||
| Miscellaneous | 1576 | ||
| Solid Organ Transplant | 1576 | ||
| Renal | 1576 | ||
| Cardiac | 1576 | ||
| Pulmonary | 1577 | ||
| Liver | 1577 | ||
| Familial Hypercholesterolemia | 1577 | ||
| Infection | 1577 | ||
| Malaria and babesiosis | 1577 | ||
| Bordetella pertussis | 1577 | ||
| Sepsis | 1577 | ||
| References | 1578 | ||
| Index | 1581 |