Pub Date : 2020-07-30DOI: 10.33590/emjhematol/20-00037
Salma M. AlDallal
Haematological disorders are predominant in the tropical and subtropical countries where major problems of sickle-cell disease (SCD) and thalassaemias are often recorded. However, reports of these conditions have increased in the Western hemisphere more recently. Genetic counselling, early detection of the disease condition, and determining an appropriate treatment regimen remains the solution. Most molecular types of SCD have been determined and the pathological impact of individual types along with the degree of severity is known to clinical investigators and physicians. There is, however, a significant need for a proper counselling system for the clinical diagnosis in most countries. Lack of funding, trained personnel, relevant physicians, instruments, and laboratories are the challenges to overcome. Pregnancy-associated SCD and thromboembolism require special mention due to their mortality rate, complexity of treatment, and care necessities. This review considers some of the most important aspects of pregnancy-associated SCD and thromboembolism, shedding light on the present understanding of the disease condition, pathology, clinical issues, the association with venous thromboembolism, recent treatment measures, and clinical and social management of pregnant women and fetuses for patients with SCD. Integrated social and clinical care along with extensive timely medical and clinical counselling for patients can improve the present situation which is growing in different countries. To save future generations and pregnant mothers from the haematological disorders that could be either prevented or treated, essential genetic screening or counselling should be made a priority by governments. In addition, social education and campaigns related to the disease condition can help to improve the situation.
{"title":"Pregnancy-Related Thromboembolismin Sickle Cell Disease","authors":"Salma M. AlDallal","doi":"10.33590/emjhematol/20-00037","DOIUrl":"https://doi.org/10.33590/emjhematol/20-00037","url":null,"abstract":"Haematological disorders are predominant in the tropical and subtropical countries where major problems of sickle-cell disease (SCD) and thalassaemias are often recorded. However, reports of these conditions have increased in the Western hemisphere more recently. Genetic counselling, early detection of the disease condition, and determining an appropriate treatment regimen remains the solution. Most molecular types of SCD have been determined and the pathological impact of individual types along with the degree of severity is known to clinical investigators and physicians. There is, however, a significant need for a proper counselling system for the clinical diagnosis in most countries. Lack of funding, trained personnel, relevant physicians, instruments, and laboratories are the challenges to overcome. Pregnancy-associated SCD and thromboembolism require special mention due to their mortality rate, complexity of treatment, and care necessities. This review considers some of the most important aspects of pregnancy-associated SCD and thromboembolism, shedding light on the present understanding of the disease condition, pathology, clinical issues, the association with venous thromboembolism, recent treatment measures, and clinical and social management of pregnant women and fetuses for patients with SCD. Integrated social and clinical care along with extensive timely medical and clinical counselling for patients can improve the present situation which is growing in different countries. To save future generations and pregnant mothers from the haematological disorders that could be either prevented or treated, essential genetic screening or counselling should be made a priority by governments. In addition, social education and campaigns related to the disease condition can help to improve the situation.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2020-07-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"127345145","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2020-01-24DOI: 10.33590/emjhematolu/19-00187
W. Ammouri, H. Harmouche, H. Khibri, S. Benkirane, Masrar Azlarab, Z. Tazi, M. Maamar, M. Adnaoui
Pernicious anaemia (PA) is an autoimmune disease of multifactorial aetiology involving environmental and immunological factors. It is the most common cause of cobalamin deficiency anaemia worldwide. The disease is a macrocytic anaemia caused by a vitamin B12 deficiency, which, in turn, is the result of intrinsic factor deficiency, a protein that binds avidly to dietary vitamin B12 and promotes its transport to the terminal ileum for absorption. Despite the advances in understanding the pathogenesis and molecular biology, diagnosis of PA is still challenging for clinicians because of its complexity, diverse clinical presentations, and the limitations of the available diagnostic tools for the evaluation of cobalamin status and the presence of chronic autoimmune atrophic gastritis. Asymptomatic autoimmune gastritis, a chronic inflammatory disease of the gastric mucosa, precedes the onset of corpus atrophy by 10–20 years. Diagnostic dilemmas could occur when patients with PA present with spuriously normal or high cobalamin levels, normocytic or microcytic anaemia, nonanaemic macrocytosis, autoimmune haemolytic anaemia, pseudo-thrombotic microangiopathy, hyperhomocysteinemia-associated thromboembolism, pseudoleukemia, bone marrow failure, and neurologic manifestations without anaemia or macrocytosis. Other autoimmune disorders, especially thyroid disease, Type 1 diabetes mellitus, and vitiligo, are also commonly associated with PA. The present review focusses on novel aspects regarding the pathogenesis, clinical presentation, and the diagnostic approach of PA; the true usefulness of serum vitamin B12 levels; and the risk of adenocarcinoma and gastric carcinoids as well as their treatment and monitoring strategies.
{"title":"Pernicious Anaemia: Mechanisms, Diagnosis, and Management","authors":"W. Ammouri, H. Harmouche, H. Khibri, S. Benkirane, Masrar Azlarab, Z. Tazi, M. Maamar, M. Adnaoui","doi":"10.33590/emjhematolu/19-00187","DOIUrl":"https://doi.org/10.33590/emjhematolu/19-00187","url":null,"abstract":"Pernicious anaemia (PA) is an autoimmune disease of multifactorial aetiology involving environmental and immunological factors. It is the most common cause of cobalamin deficiency anaemia worldwide. The disease is a macrocytic anaemia caused by a vitamin B12 deficiency, which, in turn, is the result of intrinsic factor deficiency, a protein that binds avidly to dietary vitamin B12 and promotes its transport to the terminal ileum for absorption. Despite the advances in understanding the pathogenesis and molecular biology, diagnosis of PA is still challenging for clinicians because of its complexity, diverse clinical presentations, and the limitations of the available diagnostic tools for the evaluation of cobalamin status and the presence of chronic autoimmune atrophic gastritis. Asymptomatic autoimmune gastritis, a chronic inflammatory disease of the gastric mucosa, precedes the onset of corpus atrophy by 10–20 years. Diagnostic dilemmas could occur when patients with PA present with spuriously normal or high cobalamin levels, normocytic or microcytic anaemia, nonanaemic macrocytosis, autoimmune haemolytic anaemia, pseudo-thrombotic microangiopathy, hyperhomocysteinemia-associated thromboembolism, pseudoleukemia, bone marrow failure, and neurologic manifestations without anaemia or macrocytosis. Other autoimmune disorders, especially thyroid disease, Type 1 diabetes mellitus, and vitiligo, are also commonly associated with PA. The present review focusses on novel aspects regarding the pathogenesis, clinical presentation, and the diagnostic approach of PA; the true usefulness of serum vitamin B12 levels; and the risk of adenocarcinoma and gastric carcinoids as well as their treatment and monitoring strategies.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2020-01-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"124035072","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2020-01-24DOI: 10.33590/emjhematolus/19-00178
S. Uwaezuoke
Acute chest syndrome (ACS) is the most prominent cause of mortality in children with sickle cell disease. Its cause was initially not clearly understood, but there are now established concepts regarding its aetiopathogenesis. This narrative review discusses the current perspectives on sickle cell disease pathogenesis and treatment. The PubMed database was searched for articles that met the review objective. The major causative factors are pulmonary infections, pulmonary infarction, and pulmonary fat embolism from bone marrow necrosis. These factors initiate events that result in ACS, in which a vicious cycle of infarction, inflammation, and lung collapse occurs, leading to ventilation-perfusion mismatch and hypoxaemia. ACS is best managed in hospital settings because intensive care of the patient may be required. Despite its complex management, the primary treatment modalities are supportive care, transfusion therapy, and pharmacotherapy. Although the efficacy of several modalities in attenuating or preventing ACS are well established, the outcomes from instituting others are not convincing. More research is, therefore, needed to strengthen the evidence for their therapeutic efficacy.
{"title":"Acute Chest Syndrome in Children with Sickle Cell Disease: Current Perspectives on Pathogenesis and Treatment","authors":"S. Uwaezuoke","doi":"10.33590/emjhematolus/19-00178","DOIUrl":"https://doi.org/10.33590/emjhematolus/19-00178","url":null,"abstract":"Acute chest syndrome (ACS) is the most prominent cause of mortality in children with sickle cell disease. Its cause was initially not clearly understood, but there are now established concepts regarding its aetiopathogenesis. This narrative review discusses the current perspectives on sickle cell disease pathogenesis and treatment. The PubMed database was searched for articles that met the review objective. The major causative factors are pulmonary infections, pulmonary infarction, and pulmonary fat embolism from bone marrow necrosis. These factors initiate events that result in ACS, in which a vicious cycle of infarction, inflammation, and lung collapse occurs, leading to ventilation-perfusion mismatch and hypoxaemia. ACS is best managed in hospital settings because intensive care of the patient may be required. Despite its complex management, the primary treatment modalities are supportive care, transfusion therapy, and pharmacotherapy. Although the efficacy of several modalities in attenuating or preventing ACS are well established, the outcomes from instituting others are not convincing. More research is, therefore, needed to strengthen the evidence for their therapeutic efficacy.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2020-01-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"126212363","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2020-01-24DOI: 10.33590/emjhematolus/19-00186
S. Ahuja, V. Bharati, A. Gupta, P. Joshi, R. Kant, Ashutosh Kumar Singh
Peripheral T-cell lymphomas are aggressive lymphomas with a rapidly progressive clinical course and sinister prognosis even with the best available treatment modalities. Epstein–Barr virus-positive peripheral T-cell lymphoma is an unusual variant of the disease; it is extremely rare and associated with a fulminant course spanning weeks to months. Treatment protocols are different for this entity because of its rarity.
{"title":"Reed Sternberg-Like Cells in an Aggressive Lymphoma: Report of a Rare Case and Review of Literature","authors":"S. Ahuja, V. Bharati, A. Gupta, P. Joshi, R. Kant, Ashutosh Kumar Singh","doi":"10.33590/emjhematolus/19-00186","DOIUrl":"https://doi.org/10.33590/emjhematolus/19-00186","url":null,"abstract":"Peripheral T-cell lymphomas are aggressive lymphomas with a rapidly progressive clinical course and sinister prognosis even with the best available treatment modalities. Epstein–Barr virus-positive peripheral T-cell lymphoma is an unusual variant of the disease; it is extremely rare and associated with a fulminant course spanning weeks to months. Treatment protocols are different for this entity because of its rarity.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2020-01-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"126992233","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2020-01-24DOI: 10.33590/emjhematolus/19-00116
K. Manganas, S. Delicou, A. Xydaki, J. Koskinas
In this paper, the case of a 34-year-old male with sickle cell disease, recurrent episodes of ‘girdle syndrome’, and development of chronic ischaemic colitis is reported. At his last admission to the hospital, he presented with ileus attributed to severe intestinal ischaemia. During his hospitalisation, despite optimal supportive treatment, he developed acute liver failure, possibly as a result of acute intrahepatic cholestasis, a rare but fatal complication of sickle cell disease, and died from sepsis and multiorgan failure.
{"title":"‘Girdle Syndrome’ Progressing to Ischaemic Colitis and Acute Intrahepatic Cholestasis in a Patient with Sickle Cell Disease: A Case Report","authors":"K. Manganas, S. Delicou, A. Xydaki, J. Koskinas","doi":"10.33590/emjhematolus/19-00116","DOIUrl":"https://doi.org/10.33590/emjhematolus/19-00116","url":null,"abstract":"In this paper, the case of a 34-year-old male with sickle cell disease, recurrent episodes of ‘girdle syndrome’, and development of chronic ischaemic colitis is reported. At his last admission to the hospital, he presented with ileus attributed to severe intestinal ischaemia. During his hospitalisation, despite optimal supportive treatment, he developed acute liver failure, possibly as a result of acute intrahepatic cholestasis, a rare but fatal complication of sickle cell disease, and died from sepsis and multiorgan failure.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2020-01-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"124672777","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2019-08-01DOI: 10.33590/emjhematol/10314844
E. Paubelle, X. Thomas
Thalassaemia is a hereditary cause of hypochromic microcytic anaemia resulting from defects in haemoglobin production. β-thalassaemia, which is caused by a decrease in the production of β-globin chains, affects multiple organs and is associated with considerable morbidity and mortality. This review aims to highlight the significant progress being made in the areas of ineffective erythropoiesis control, metal chelation, and gene therapy, which is bringing new hope and should change patient management and prognosis in the near future.
{"title":"New Insights in β-Thalassaemia","authors":"E. Paubelle, X. Thomas","doi":"10.33590/emjhematol/10314844","DOIUrl":"https://doi.org/10.33590/emjhematol/10314844","url":null,"abstract":"Thalassaemia is a hereditary cause of hypochromic microcytic anaemia resulting from defects in haemoglobin production. β-thalassaemia, which is caused by a decrease in the production of β-globin chains, affects multiple organs and is associated with considerable morbidity and mortality. This review aims to highlight the significant progress being made in the areas of ineffective erythropoiesis control, metal chelation, and gene therapy, which is bringing new hope and should change patient management and prognosis in the near future.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2019-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"130580860","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2019-08-01DOI: 10.33590/emjhematol/10311051
Alan E. Corcoran
Multiple myeloma (MM), characterised by the clonal proliferation of malignant plasma cells, results in the overproduction of monoclonal immunoglobulins.1 Genetic heterogeneity of these clones confers treatment resistance and contributes to disease progression. Therefore, the use of combination therapies with different mechanisms of action can target the maximum number of clones simultaneously and may achieve long-term disease control.2 Current therapeutic strategies, such as chemotherapy, radiotherapy, proteasome inhibitors (PI), immunomodulatory drugs (IMiD), monoclonal antibodies, and autologous/allogeneic stem cell transplantation have resulted in improved outcomes for MM patients. However, these therapies rarely induce long-lasting complete remissions, and patients frequently develop resistance to treatments. As such, the search for novel treatment strategies, including personalised immunotherapies, is ongoing to overcome resistance and improve patient survival.
{"title":"Rebooting the Myeloma Treatment Programme","authors":"Alan E. Corcoran","doi":"10.33590/emjhematol/10311051","DOIUrl":"https://doi.org/10.33590/emjhematol/10311051","url":null,"abstract":"Multiple myeloma (MM), characterised by the clonal proliferation of malignant plasma cells, results in the overproduction of monoclonal immunoglobulins.1 Genetic heterogeneity of these clones confers treatment resistance and contributes to disease progression. Therefore, the use of combination therapies with different mechanisms of action can target the maximum number of clones simultaneously and may achieve long-term disease control.2 Current therapeutic strategies, such as chemotherapy, radiotherapy, proteasome inhibitors (PI), immunomodulatory drugs (IMiD), monoclonal antibodies, and autologous/allogeneic stem cell transplantation have resulted in improved outcomes for MM patients. However, these therapies rarely induce long-lasting complete remissions, and patients frequently develop resistance to treatments. As such, the search for novel treatment strategies, including personalised immunotherapies, is ongoing to overcome resistance and improve patient survival.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2019-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"128889243","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2019-08-01DOI: 10.33590/emjhematol/10310423
Z. Grudeva-Popova, E. Spasov, Vasko Graklanov, Katya N Sotirova, V. Popov, G. Balatzenko, I. Hristova
Since the late 1990s, when the first tyrosine kinase inhibitor (TKI) imatinib was introduced as a front-line treatment for chronic myeloid leukaemia, the disease’s course and prognosis has dramatically changed. The development of second-line and further-line more potent generations of TKI has further improved disease control and patients’ quality of life; however, during this time, many questions such as the duration of treatment, the depth of response, fertility, pregnancy, and family planning, have been raised. Recent prospective and retrospective discontinuation trials for TKI have shown encouraging results regarding the cessation of TKI treatment and maintaining complete molecular response. The authors report three cases of female patients diagnosed with chronic phase chronic myeloid leukaemia who achieved a long-term deep molecular response; had planned management during pregnancy, including regular molecular monitoring with or without INF-α; and all delivered healthy babies.
{"title":"Are Successful Pregnancies an Achievable Goal in Patients with Chronic Myeloid Leukaemia?","authors":"Z. Grudeva-Popova, E. Spasov, Vasko Graklanov, Katya N Sotirova, V. Popov, G. Balatzenko, I. Hristova","doi":"10.33590/emjhematol/10310423","DOIUrl":"https://doi.org/10.33590/emjhematol/10310423","url":null,"abstract":"Since the late 1990s, when the first tyrosine kinase inhibitor (TKI) imatinib was introduced as a front-line treatment for chronic myeloid leukaemia, the disease’s course and prognosis has dramatically changed. The development of second-line and further-line more potent generations of TKI has further improved disease control and patients’ quality of life; however, during this time, many questions such as the duration of treatment, the depth of response, fertility, pregnancy, and family planning, have been raised. Recent prospective and retrospective discontinuation trials for TKI have shown encouraging results regarding the cessation of TKI treatment and maintaining complete molecular response. The authors report three cases of female patients diagnosed with chronic phase chronic myeloid leukaemia who achieved a long-term deep molecular response; had planned management during pregnancy, including regular molecular monitoring with or without INF-α; and all delivered healthy babies.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2019-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"122193893","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2019-08-01DOI: 10.33590/emjhematol/10314307
A. Yacoub, J. Mahnken, L. Holcomb
This study consists of a retrospective chart review to evaluate the use of abdominal imaging to aid in the diagnosis of chronic cytopenia. Patients with unclear aetiology of chronic cytopenia often require complex work-up, and it would be beneficial to develop an algorithm to make this process more efficient. Abdominal imaging is a non-invasive procedure that may provide useful clinical diagnostic information as part of an algorithm for this subset of patients. The medical records of 36 patients were reviewed to determine the association between abdominal imaging results and other components of diagnostic work-up. Abdominal imaging was positive for 21 (58.33%) individuals. Following imaging, 8 individuals (22.22%) required bone marrow biopsy and 12 individuals (33.33%) required frequent monitoring, which were non-significant associations. However, positive imaging results were significantly associated with increased BMI as well as severity of thrombocytopaenia. This study suggests that abdominal imaging warrants further study as a potentially useful addition to diagnostic work-up for chronic cytopenia of unknown aetiology.
{"title":"Retrospective Review of the Role of Abdominal Imaging in Evaluation of Cytopenias","authors":"A. Yacoub, J. Mahnken, L. Holcomb","doi":"10.33590/emjhematol/10314307","DOIUrl":"https://doi.org/10.33590/emjhematol/10314307","url":null,"abstract":"This study consists of a retrospective chart review to evaluate the use of abdominal imaging to aid in the diagnosis of chronic cytopenia. Patients with unclear aetiology of chronic cytopenia often require complex work-up, and it would be beneficial to develop an algorithm to make this process more efficient. Abdominal imaging is a non-invasive procedure that may provide useful clinical diagnostic information as part of an algorithm for this subset of patients. The medical records of 36 patients were reviewed to determine the association between abdominal imaging results and other components of diagnostic work-up. Abdominal imaging was positive for 21 (58.33%) individuals. Following imaging, 8 individuals (22.22%) required bone marrow biopsy and 12 individuals (33.33%) required frequent monitoring, which were non-significant associations. However, positive imaging results were significantly associated with increased BMI as well as severity of thrombocytopaenia. This study suggests that abdominal imaging warrants further study as a potentially useful addition to diagnostic work-up for chronic cytopenia of unknown aetiology.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2019-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"125165326","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2019-08-01DOI: 10.33590/emjhematol/10313783
Iman Saleh Moustafa, M. Badawy, S. Moustafa, Fetouh
Immune thrombocytopenic purpura is a clinical syndrome of thrombocytopenia that manifests as a bleeding tendency, typical skin rashes, easy bruising, or extravasation of blood from the capillaries. Defects in the thrombopoietin-receptor (TPOR)/myeloproliferative leukaemia virus/JAK2 axis leads to haematological diseases such as thrombocytopenia or pancytopenia through the inhibition of the megakaryopoiesis process. Thrombopoietin-receptor agonists (TPORA), such as eltrombopag, increase platelet count by stimulating the TPOR. Bone marrow (BM) fibrosis has been reported in patients receiving TPORA. Myelofibrosis (MF) may be induced by mutations in JAK2, CALR, and MPL genes. This review gives an insight on MF as a serious side effect induced by eltrombopag. This review enriches the evidence of MF induced by eltrombopag after long-term administration ranging from 6 months to 7 years. MF is mostly spontaneous and decreases after discontinuation of medication; however, in a few cases it becomes persistent. This major issue should be treated with high concern. The authors recommend that any patient on eltrombopag treatment should be under vigilant observation and closely monitored for any sign of MF by clinical manifestation and any abnormal result from peripheral blood smear examination, and should additionally undergo BM biopsy for confirmation and detection of the severity of MF. The authors recommend discontinuing the medication if this side effect occurs. The authors also recommend to conduct larger studies for longer periods using serial BM before, and periodically after, eltrombopag treatment to evaluate the characteristics of this adverse effect.
{"title":"Eltrombopag-Induced Myelofibrosis in Patients with Adult Immune Thrombocytopenia: Scoping Review","authors":"Iman Saleh Moustafa, M. Badawy, S. Moustafa, Fetouh","doi":"10.33590/emjhematol/10313783","DOIUrl":"https://doi.org/10.33590/emjhematol/10313783","url":null,"abstract":"Immune thrombocytopenic purpura is a clinical syndrome of thrombocytopenia that manifests as a bleeding tendency, typical skin rashes, easy bruising, or extravasation of blood from the capillaries. Defects in the thrombopoietin-receptor (TPOR)/myeloproliferative leukaemia virus/JAK2 axis leads to haematological diseases such as thrombocytopenia or pancytopenia through the inhibition of the megakaryopoiesis process.\u0000\u0000Thrombopoietin-receptor agonists (TPORA), such as eltrombopag, increase platelet count by stimulating the TPOR. Bone marrow (BM) fibrosis has been reported in patients receiving TPORA. Myelofibrosis (MF) may be induced by mutations in JAK2, CALR, and MPL genes. This review gives an insight on MF as a serious side effect induced by eltrombopag.\u0000\u0000This review enriches the evidence of MF induced by eltrombopag after long-term administration ranging from 6 months to 7 years. MF is mostly spontaneous and decreases after discontinuation of medication; however, in a few cases it becomes persistent. This major issue should be treated with high concern. The authors recommend that any patient on eltrombopag treatment should be under vigilant observation and closely monitored for any sign of MF by clinical manifestation and any abnormal result from peripheral blood smear examination, and should additionally undergo BM biopsy for confirmation and detection of the severity of MF. The authors recommend discontinuing the medication if this side effect occurs. The authors also recommend to conduct larger studies for longer periods using serial BM before, and periodically after, eltrombopag treatment to evaluate the characteristics of this adverse effect.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2019-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"114689795","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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