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The Netherlands Journal of Medicine Apr 2020Anaemia is a common diagnosis for clinicians. This mini-review summarises criteria for diagnosing the cause of anaemia. Within the microcytic anaemias, iron-deficient... (Review)
Review
Anaemia is a common diagnosis for clinicians. This mini-review summarises criteria for diagnosing the cause of anaemia. Within the microcytic anaemias, iron-deficient anaemia is most common. In addition, we would like to raise awareness of thalassaemia as a differential diagnosis. A normocytic anaemia, such as anaemia of chronic disease, is a diagnosis of exclusion. A macrocytic anaemia scheme is provided and differentiates based on reticulocyte count. We aim to provide the readers a clear overview of anaemia and when to refer to haematologists.
Topics: Adolescent; Adult; Anemia; Child; Diagnosis, Differential; Female; Hemoglobins; Humans; Male; Pregnancy; Reference Values; Symptom Assessment; Young Adult
PubMed: 32332184
DOI: No ID Found -
Journal of Hematology & Oncology Sep 2021Immunotherapies such as immune checkpoint blockade (ICB) and adoptive cell therapy (ACT) have revolutionized cancer treatment, especially in patients whose disease was... (Review)
Review
Immunotherapies such as immune checkpoint blockade (ICB) and adoptive cell therapy (ACT) have revolutionized cancer treatment, especially in patients whose disease was otherwise considered incurable. However, primary and secondary resistance to single agent immunotherapy often results in treatment failure, and only a minority of patients experience long-term benefits. This review article will discuss the relationship between cancer immune response and mechanisms of resistance to immunotherapy. It will also provide a comprehensive review on the latest clinical status of combination therapies (e.g., immunotherapy with chemotherapy, radiation therapy and targeted therapy), and discuss combination therapies approved by the US Food and Drug Administration. It will provide an overview of therapies targeting cytokines and other soluble immunoregulatory factors, ACT, virotherapy, innate immune modifiers and cancer vaccines, as well as combination therapies that exploit alternative immune targets and other therapeutic modalities. Finally, this review will include the stimulating insights from the 2020 China Immuno-Oncology Workshop co-organized by the Chinese American Hematologist and Oncologist Network (CAHON), the China National Medical Product Administration (NMPA) and Tsinghua University School of Medicine.
Topics: Animals; Cancer Vaccines; Combined Modality Therapy; Humans; Immune Checkpoint Inhibitors; Immunotherapy; Neoplasms; Oncolytic Virotherapy
PubMed: 34579759
DOI: 10.1186/s13045-021-01164-5 -
Blood May 2022The treatment of patients with systemic light chain (AL) amyloidosis is a challenge to hematologists. Despite its generally small size, the underlying clone causes a...
The treatment of patients with systemic light chain (AL) amyloidosis is a challenge to hematologists. Despite its generally small size, the underlying clone causes a rapidly progressing, often devastating multiorgan dysfunction through the toxic light chains that form amyloid deposits. Clinical manifestations are deceitful and too often recognized at an irreversible stage. However, hematologists are in the unique position to diagnose AL amyloidosis at a presymptomatic stage, checking biomarkers of amyloid organ involvement in patients with monoclonal gammopathies at higher risk to develop the disease. Adequate technology and expertise are needed for a prompt and correct diagnosis, particularly for ruling out non-AL amyloidoses that are now also treatable. Therapy should be carefully tailored based on severity of organ involvement and clonal characteristics, and early and continual monitoring of response is critical. Three recent randomized clinical trials moved AL amyloidosis to evidence-based era. Above all, the daratumumab-bortezomib combination is a new standard-of-care for newly diagnosed patients, inducing rapid and deep responses that translate into high rates of organ response. The availability of new effective drugs allows to better personalize the therapy, reduce toxicity, and improve outcomes. Patients should be treated within clinical trials whenever possible.
Topics: Amyloidosis; Bortezomib; Humans; Immunoglobulin Light-chain Amyloidosis; Immunotherapy; Paraproteinemias
PubMed: 34517412
DOI: 10.1182/blood.2020008737 -
Turkish Journal of Haematology :... Jun 2022Gaucher disease (GD) is a rare hereditary lysosomal storage disease that arises due to deficiency of glucocerebrosidase. Early diagnosis is very important for starting... (Review)
Review
Gaucher disease (GD) is a rare hereditary lysosomal storage disease that arises due to deficiency of glucocerebrosidase. Early diagnosis is very important for starting proper treatment and preventing complications. Splenomegaly, anemia, and thrombocytopenia are the most common findings in GD and so most patients are initially referred to hematologists. The Turkish Society of Hematology established its Rare Hematological Diseases Subcommittee in 2015. One of the main topics of this subcommittee was to increase and improve awareness and education of rare diseases among hematologists in Turkey. This review presents GD with an overview of its clinical features, pathophysiology, and treatment options for hematologists.
Topics: Anemia; Gaucher Disease; Glucosylceramidase; Humans; Splenomegaly; Thrombocytopenia; Turkey
PubMed: 35439918
DOI: 10.4274/tjh.galenos.2021.2021.0683 -
Hematology. American Society of... Dec 2022Hematologists are often consulted for thrombocytopenia in pregnancy, especially when there is a concern for a non-pregnancy-specific etiology or an insufficient platelet... (Review)
Review
Hematologists are often consulted for thrombocytopenia in pregnancy, especially when there is a concern for a non-pregnancy-specific etiology or an insufficient platelet count for the hemostatic challenges of delivery. The severity of thrombocytopenia and trimester of onset can help guide the differential diagnosis. Hematologists need to be aware of the typical signs of preeclampsia with severe features and other hypertensive disorders of pregnancy to help distinguish these conditions, which typically resolve with delivery, from other thrombotic microangiopathies (TMAs) (eg, thrombotic thrombocytopenic purpura or complement-mediated TMA). Patients with chronic thrombocytopenic conditions, such as immune thrombocytopenia, should receive counseling on the safety and efficacy of various medications during pregnancy. The management of pregnant patients with chronic immune thrombocytopenia who are refractory to first-line treatments is an area that warrants further research. This review uses a case-based approach to discuss recent updates in diagnosing and managing thrombocytopenia in pregnancy.
Topics: Female; Pregnancy; Humans; Purpura, Thrombocytopenic, Idiopathic; Pregnancy Complications, Hematologic; Purpura, Thrombotic Thrombocytopenic; Thrombotic Microangiopathies; Platelet Count
PubMed: 36485110
DOI: 10.1182/hematology.2022000375 -
Hereditary hemorrhagic telangiectasia: diagnosis and management from the hematologist's perspective.Haematologica Sep 2018Hereditary hemorrhagic telangiectasia (HHT), also known as Osler-Weber-Rendu syndrome, is an autosomal dominant disorder that causes abnormal blood vessel formation. The... (Review)
Review
Hereditary hemorrhagic telangiectasia (HHT), also known as Osler-Weber-Rendu syndrome, is an autosomal dominant disorder that causes abnormal blood vessel formation. The diagnosis of hereditary hemorrhagic telangiectasia is clinical, based on the Curaçao criteria. Genetic mutations that have been identified include , , and , among others. Patients with HHT may have telangiectasias and arteriovenous malformations in various organs and suffer from many complications including bleeding, anemia, iron deficiency, and high-output heart failure. Families with the same mutation exhibit considerable phenotypic variation. Optimal treatment is best delivered a multidisciplinary approach with appropriate diagnosis, screening and local and/or systemic management of lesions. Anti-angiogenic agents such as bevacizumab have emerged as a promising systemic therapy in reducing bleeding complications but are not curative. Other pharmacological agents include iron supplementation, antifibrinolytics and hormonal treatment. This review discusses the biology of HHT, management issues that face the practising hematologist, and considerations of future directions in HHT treatment.
Topics: Animals; Biomarkers; Clinical Trials as Topic; Combined Modality Therapy; Disease Management; Disease Susceptibility; Genetic Association Studies; Humans; Mutation; Phenotype; Telangiectasia, Hereditary Hemorrhagic; Treatment Outcome
PubMed: 29794143
DOI: 10.3324/haematol.2018.193003 -
Chest Feb 2012Neonates and children differ from adults in physiology, pharmacologic responses to drugs, epidemiology, and long-term consequences of thrombosis. This guideline...
Antithrombotic therapy in neonates and children: Antithrombotic Therapy and Prevention of Thrombosis, 9th ed: American College of Chest Physicians Evidence-Based Clinical Practice Guidelines.
BACKGROUND
Neonates and children differ from adults in physiology, pharmacologic responses to drugs, epidemiology, and long-term consequences of thrombosis. This guideline addresses optimal strategies for the management of thrombosis in neonates and children.
METHODS
The methods of this guideline follow those described in the Methodology for the Development of Antithrombotic Therapy and Prevention of Thrombosis Guidelines: Antithrombotic Therapy and Prevention of Thrombosis, 9th ed: American College of Chest Physicians Evidence-Based Clinical Practice Guidelines.
RESULTS
We suggest that where possible, pediatric hematologists with experience in thromboembolism manage pediatric patients with thromboembolism (Grade 2C). When this is not possible, we suggest a combination of a neonatologist/pediatrician and adult hematologist supported by consultation with an experienced pediatric hematologist (Grade 2C). We suggest that therapeutic unfractionated heparin in children is titrated to achieve a target anti-Xa range of 0.35 to 0.7 units/mL or an activated partial thromboplastin time range that correlates to this anti-Xa range or to a protamine titration range of 0.2 to 0.4 units/mL (Grade 2C). For neonates and children receiving either daily or bid therapeutic low-molecular-weight heparin, we suggest that the drug be monitored to a target range of 0.5 to 1.0 units/mL in a sample taken 4 to 6 h after subcutaneous injection or, alternatively, 0.5 to 0.8 units/mL in a sample taken 2 to 6 h after subcutaneous injection (Grade 2C).
CONCLUSIONS
The evidence supporting most recommendations for antithrombotic therapy in neonates and children remains weak. Studies addressing appropriate drug target ranges and monitoring requirements are urgently required in addition to site- and clinical situation-specific thrombosis management strategies.
Topics: Anticoagulants; Blood Coagulation Tests; Cardiac Catheterization; Child; Child, Preschool; Cooperative Behavior; Dose-Response Relationship, Drug; Drug Administration Schedule; Evidence-Based Medicine; Factor Xa Inhibitors; Fibrinolytic Agents; Hemorrhage; Heparin; Heparin, Low-Molecular-Weight; Humans; Infant, Newborn; Interdisciplinary Communication; Platelet Aggregation Inhibitors; Renal Veins; Risk Factors; Secondary Prevention; Societies, Medical; Thrombosis; Upper Extremity Deep Vein Thrombosis; Vitamin K
PubMed: 22315277
DOI: 10.1378/chest.11-2308 -
Haematologica Aug 2020The acquired von Willebrand syndrome (AvWS) is a rare bleeding disorder with laboratory findings similar to those of inherited von Willebrand disease. However, unlike... (Review)
Review
The acquired von Willebrand syndrome (AvWS) is a rare bleeding disorder with laboratory findings similar to those of inherited von Willebrand disease. However, unlike the inherited disease, AvWS occurs in persons with no personal and family history of bleeding and is often associated with a variety of underlying diseases, most frequently lymphoproliferative, myeloproliferative and cardiovascular disorders. After the presentation of a typical case, in this narrative review we discuss the more recent data on the pathophysiology, clinical, laboratory and therapeutic aspects of this acquired bleeding syndrome. We chose to focus particularly on those aspects of greater interest for the hematologist.
Topics: Cardiovascular Diseases; Hemorrhage; Hemorrhagic Disorders; Humans; Syndrome; von Willebrand Diseases; von Willebrand Factor
PubMed: 32554559
DOI: 10.3324/haematol.2020.255117 -
Blood May 2017B deficiency is the leading cause of megaloblastic anemia, and although more common in the elderly, can occur at any age. Clinical disease caused by B deficiency usually... (Review)
Review
B deficiency is the leading cause of megaloblastic anemia, and although more common in the elderly, can occur at any age. Clinical disease caused by B deficiency usually connotes severe deficiency, resulting from a failure of the gastric or ileal phase of physiological B absorption, best exemplified by the autoimmune disease pernicious anemia. There are many other causes of B deficiency, which range from severe to mild. Mild deficiency usually results from failure to render food B bioavailable or from dietary inadequacy. Although rarely resulting in megaloblastic anemia, mild deficiency may be associated with neurocognitive and other consequences. B deficiency is best diagnosed using a combination of tests because none alone is completely reliable. The features of B deficiency are variable and may be atypical. Timely diagnosis is important, and treatment is gratifying. Failure to diagnose B deficiency can have dire consequences, usually neurological. This review is written from the perspective of a practicing hematologist.
Topics: Anemia, Megaloblastic; Anemia, Pernicious; Animals; Folic Acid; Humans; Intestinal Absorption; Vitamin B 12; Vitamin B 12 Deficiency
PubMed: 28360040
DOI: 10.1182/blood-2016-10-569186 -
Haematologica Mar 2019IgG4-related disease is a fibro-inflammatory condition that can affect nearly any organ system. Common presentations include major salivary and lacrimal gland... (Review)
Review
IgG4-related disease is a fibro-inflammatory condition that can affect nearly any organ system. Common presentations include major salivary and lacrimal gland enlargement, orbital disease, autoimmune pancreatitis, retroperitoneal fibrosis and tubulointerstitial nephritis. This review focuses on the hematologic manifestations of IgG4-related disease, including lymphadenopathy, eosinophilia, and polyclonal hypergammaglobulinemia. The disease can easily be missed by unsuspecting hematologists, as patients may present with clinical problems that mimic disorders such as multicentric Castleman disease, lymphoma, plasma cell neoplasms and hypereosinophilic syndromes. When IgG4-related disease is suspected, serum protein electrophoresis and IgG subclasses are helpful as initial tests but a firm histological diagnosis is essential both to confirm the diagnosis and to rule out mimickers. The central histopathological features are a dense, polyclonal, lymphoplasmacytic infiltrate enriched with IgG4-positive plasma cells (with an IgG4/IgG ratio >40%), storiform fibrosis, and obliterative phlebitis. Importantly for hematologists, the latter two features are seen in all tissues except bone marrow and lymph nodes, making these two sites suboptimal for histological confirmation. Many patients follow an indolent course and respond well to treatment, but a significant proportion may have highly morbid or fatal complications such as periaortitis, severe retroperitoneal fibrosis or pachymeningitis. Corticosteroids are effective but cause new or worsening diabetes in about 40% of patients. Initial response rates to rituximab are high but durable remissions are rare. More intensive lymphoma chemotherapy regimens may be required in rare cases of severe, refractory disease, and targeted therapy against plasmablasts, IgE and other disease biomarkers warrant further exploration.
Topics: Aged, 80 and over; Biomarkers; Biopsy; Combined Modality Therapy; Diagnosis, Differential; Diagnostic Imaging; Disease Management; Disease Susceptibility; Humans; Immunoglobulin G4-Related Disease; Male; Phenotype; Symptom Assessment; Treatment Outcome
PubMed: 30705099
DOI: 10.3324/haematol.2018.205526