Pub Date : 2024-07-16DOI: 10.33590/emjhematol/pgqp6795
Elizabeth Macintyre
{"title":"EHA Congress Interview: Elizabeth Macintyre","authors":"Elizabeth Macintyre","doi":"10.33590/emjhematol/pgqp6795","DOIUrl":"https://doi.org/10.33590/emjhematol/pgqp6795","url":null,"abstract":"","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-07-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141642426","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 : 2024-07-15DOI: 10.33590/emjhematol/umdh9964
IMROZ represents a landmark study in multiple myeloma and is the first global Phase III study of an anti-CD38 monoclonal antibody (mAb) in combination with standard-of-care bortezomib, lenalidomide, and dexamethasone (VRd) to show a significant improvement in progression-free survival (PFS), together with deep and sustained responses, in newly-diagnosed patients not intended for transplant. During a joint interview conducted by the European Medical Journal (EMJ), two leading experts in the field of myeloma and co-investigators on the IMROZ study, Meral Beksac from the Division of Haematology, Ankara Liv Hospital Istinye University, Türkiye; and Mohamad Mohty from Sorbonne University, Saint-Antoine Hospital, Paris, France, discussed the findings and clinical implications of this important study of the isatuximab (Isa) plus VRd regimen. Beksac and Mohty reviewed the methodology and key efficacy and safety data from the IMROZ trial, and offered their perspectives on evaluating patient eligibility for haematopoietic stem cell transplantation (HSCT). The experts also considered the clinical impact of IMROZ on the management of newly diagnosed myeloma, and the potential positioning of quadruplet regimens such as Isa-VRd as the new first-choice frontline treatment for transplant-ineligible patients.
{"title":"What Does the Phase III ‘IMROZ’ Study Mean for Patients with Multiple Myeloma? An Interview with the Co-authors","authors":"","doi":"10.33590/emjhematol/umdh9964","DOIUrl":"https://doi.org/10.33590/emjhematol/umdh9964","url":null,"abstract":"IMROZ represents a landmark study in multiple myeloma and is the first global Phase III study of an anti-CD38 monoclonal antibody (mAb) in combination with standard-of-care bortezomib, lenalidomide, and dexamethasone (VRd) to show a significant improvement in progression-free survival (PFS), together with deep and sustained responses, in newly-diagnosed patients not intended for transplant. During a joint interview conducted by the European Medical Journal (EMJ), two leading experts in the field of myeloma and co-investigators on the IMROZ study, Meral Beksac from the Division of Haematology, Ankara Liv Hospital Istinye University, Türkiye; and Mohamad Mohty from Sorbonne University, Saint-Antoine Hospital, Paris, France, discussed the findings and clinical implications of this important study of the isatuximab (Isa) plus VRd regimen. Beksac and Mohty reviewed the methodology and key efficacy and safety data from the IMROZ trial, and offered their perspectives on evaluating patient eligibility for haematopoietic stem cell transplantation (HSCT). The experts also considered the clinical impact of IMROZ on the management of newly diagnosed myeloma, and the potential positioning of quadruplet regimens such as Isa-VRd as the new first-choice frontline treatment for transplant-ineligible patients.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141648227","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 : 2023-12-22DOI: 10.33590/emjhematol/10300884
N. Neupane, H. Kharel, M. Ammad-Ud-Din, S. Jamshed
Amyloidoma is a rare complication of plasmacytoma, that can involve the spine and present with compressive neurological symptoms. It is usually a diagnosis of exclusion, and is difficult to differentiate from other plasma cell disorders on imaging. Definite diagnosis requires a tissue biopsy. The treatment requires a multidisciplinary approach, with input from haematology, neurology, neurosurgery, and radiology for the optimum course of action, depending on the patient’s comorbidities and performance status. The authors hereby present a case of solitary sacral amyloidoma in a 52-year-old African American female. Only three other cases of solitary sacral amyloidoma have been reported in the literature.
{"title":"Spinal Plasmacytoma Transformed Into Solitary Sacral Amyloidoma: A Case Report","authors":"N. Neupane, H. Kharel, M. Ammad-Ud-Din, S. Jamshed","doi":"10.33590/emjhematol/10300884","DOIUrl":"https://doi.org/10.33590/emjhematol/10300884","url":null,"abstract":"Amyloidoma is a rare complication of plasmacytoma, that can involve the spine and present with compressive neurological symptoms. It is usually a diagnosis of exclusion, and is difficult to differentiate from other plasma cell disorders on imaging. Definite diagnosis requires a tissue biopsy. The treatment requires a multidisciplinary approach, with input from haematology, neurology, neurosurgery, and radiology for the optimum course of action, depending on the patient’s comorbidities and performance status. The authors hereby present a case of solitary sacral amyloidoma in a 52-year-old African American female. Only three other cases of solitary sacral amyloidoma have been reported in the literature.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-12-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138947657","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 : 2023-12-14DOI: 10.33590/emjhematol/10307569
Nicola Humphry
Pyruvate kinase (PK) deficiency is a hereditary haemolytic anaemia caused by mutations in the PKLR gene encoding PK, which is critical for maintaining red blood cell (RBC) energy levels. Defects in PK cause chronic haemolysis. There are currently no disease-modifying therapies approved for use in children with PK deficiency, and treatment can involve regular blood transfusions, iron chelation, splenectomy, and management of disease complications. In this interview, Rachael F. Grace, a paediatric haematologist at the Dana–Farber/Boston Children's Cancer and Blood Disorders Center, Massachusetts, USA; and Julián Sevilla, a haematologist at the Hospital Infantil Universitario Niño Jesús in Madrid, Spain, shared their experience of diagnosing and treating paediatric patients with PK deficiency. They discussed the substantial variability in symptoms between patients irrespective of their haemoglobin level, the risk of iron overload even in children not receiving regular transfusions, and the effects of jaundice on children’s self-esteem. Grace and Sevilla also examined the challenges in diagnosis and management of PK deficiency in paediatrics, and the importance of regular monitoring in all patients, both to identify potential complications, and to ensure optimal medical management of their disease. Finally, they discussed new therapies that are currently being developed, which have the potential to have a major impact on future treatments for paediatric patients with PK deficiency.
{"title":"Real-World Impact of Pyruvate Kinase Deficiency in Children","authors":"Nicola Humphry","doi":"10.33590/emjhematol/10307569","DOIUrl":"https://doi.org/10.33590/emjhematol/10307569","url":null,"abstract":"Pyruvate kinase (PK) deficiency is a hereditary haemolytic anaemia caused by mutations in the PKLR gene encoding PK, which is critical for maintaining red blood cell (RBC) energy levels. Defects in PK cause chronic haemolysis. There are currently no disease-modifying therapies approved for use in children with PK deficiency, and treatment can involve regular blood transfusions, iron chelation, splenectomy, and management of disease complications.\u0000In this interview, Rachael F. Grace, a paediatric haematologist at the Dana–Farber/Boston Children's Cancer and Blood Disorders Center, Massachusetts, USA; and Julián Sevilla, a haematologist at the Hospital Infantil Universitario Niño Jesús in Madrid, Spain, shared their experience of diagnosing and treating paediatric patients with PK deficiency. They discussed the substantial variability in symptoms between patients irrespective of their haemoglobin level, the risk of iron overload even in children not receiving regular transfusions, and the effects of jaundice on children’s self-esteem.\u0000Grace and Sevilla also examined the challenges in diagnosis and management of PK deficiency in paediatrics, and the importance of regular monitoring in all patients, both to identify potential complications, and to ensure optimal medical management of their disease. Finally, they discussed new therapies that are currently being developed, which have the potential to have a major impact on future treatments for paediatric patients with PK deficiency.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139003033","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 : 2023-08-01DOI: 10.33590/emjhematol/10301096
Nicola Humphry
This symposium was held on the first day of the 2023 International Conference on Malignant Lymphoma (ICML) Congress in Lugano, Switzerland. Björn Chapuy, a Haematologist in the department of Oncology and Tumour Immunology at Benjamin Franklin Campus, Charité – Universitätsmedizin Berlin, Germany, described the rapid pace of development of new treatment options for patients with diffuse large B cell lymphoma (DLBCL) who relapse after their first-line (1L) of therapy, and introduced an expert panel of speakers including both haematologists and a patient representative from the Lymphoma Coalition, Europe. Philipp Staber, Programme Director for Lymphoma and Chronic Lymphocytic Leukaemia at the Medical University of Vienna, Austria, discussed the importance of tumour boards, and how they are structured, while Natacha Bolaños, Head of membership and alliances for the Lymphoma Coalition Europe, shared insights from a global survey of patients living with DLBCL or relapsed/refractory (R/R) DLBCL. Eva González-Barca, Co-ordinator of the Lymphoma Unit at the Catalan Institute of Oncology, Barcelona, Spain, and Gabriel Brisou, a Haematologist at the Institut Paoli-Calmettes, Marseille, France, presented case studies of patients with R/R DLBCL treated with different therapies at second-line (2L). The panellists also described the supporting data for some of the options for 2L therapy. The overarching message from the symposium was that involvement of the patient, and potentially their caregiver, in treatment decisions is vital, and that recommendations for treatment should come from a multidisciplinary tumour board composed of pathologists, radiologists, and haemato-oncologists, rather than an individual clinician. Though there is currently no simple answer to which treatment approach should be chosen for each patient, the panel hopes that the next few years will bring a greater understanding of the best choices for individualised therapy.
{"title":"What to Do When Chimeric Antigen Receptor T Cells Are Not the Most Appropriate Option in Second-Line Diffuse Large B Cell Lymphoma: Current Treatment Options for Transplant-Ineligible Patients","authors":"Nicola Humphry","doi":"10.33590/emjhematol/10301096","DOIUrl":"https://doi.org/10.33590/emjhematol/10301096","url":null,"abstract":"This symposium was held on the first day of the 2023 International Conference on Malignant Lymphoma (ICML) Congress in Lugano, Switzerland. Björn Chapuy, a Haematologist in the department of Oncology and Tumour Immunology at Benjamin Franklin Campus, Charité – Universitätsmedizin Berlin, Germany, described the rapid pace of development of new treatment options for patients with diffuse large B cell lymphoma (DLBCL) who relapse after their first-line (1L) of therapy, and introduced an expert panel of speakers including both haematologists and a patient representative from the Lymphoma Coalition, Europe.\u0000\u0000Philipp Staber, Programme Director for Lymphoma and Chronic Lymphocytic Leukaemia at the Medical University of Vienna, Austria, discussed the importance of tumour boards, and how they are structured, while Natacha Bolaños, Head of membership and alliances for the Lymphoma Coalition Europe, shared insights from a global survey of patients living with DLBCL or relapsed/refractory (R/R) DLBCL. Eva González-Barca, Co-ordinator of the Lymphoma Unit at the Catalan Institute of Oncology, Barcelona, Spain, and Gabriel Brisou, a Haematologist at the Institut Paoli-Calmettes, Marseille, France, presented case studies of patients with R/R DLBCL treated with different therapies at second-line (2L). The panellists also described the supporting data for some of the options for 2L therapy.\u0000\u0000The overarching message from the symposium was that involvement of the patient, and potentially their caregiver, in treatment decisions is vital, and that recommendations for treatment should come from a multidisciplinary tumour board composed of pathologists, radiologists, and haemato-oncologists, rather than an individual clinician. Though there is currently no simple answer to which treatment approach should be chosen for each patient, the panel hopes that the next few years will bring a greater understanding of the best choices for individualised therapy.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"114530756","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 : 2023-08-01DOI: 10.33590/emjhematol/10308707
H. Moir
The thalassaemias are a heterogeneous group of inherited chronic blood disorders associated with impaired haemoglobin (Hb) synthesis, resulting in ineffective erythropoiesis, haemolysis, and the development of lifelong anaemia. The pathophysiology of thalassaemia is due, in part, to increased energy demand to clear globin aggregates and reactive O2 species, and maintain overall red blood cell (RBC) health, coupled with insufficient adenosine triphosphate (ATP) production. For this article, interviews were conducted by EMJ in December 2022 with two key opinion leaders. Kevin Kuo is a Clinician-Investigator and Staff Haematologist at the University Health Network, Toronto, Canada, and Associate Professor in the Division of Hematology, Department of Medicine, University of Toronto, Canada. Maria Cappellini is Professor of Medicine in the Department of Clinical Sciences and Community, University of Milan, Italy. Both haematologists have over 50 years of expertise and clinical experience between them in treating patients with thalassaemia. The two experts provided insights into two ongoing Phase III clinical trials for both α- and β-thalassaemia. These trials are investigating the effect of mitapivat, a first-in-class, small molecule pyruvate kinase activator, in patients across the full range of thalassaemia subtypes. The ENERGIZE trial is investigating mitapivat in patients with non–transfusion-dependent thalassaemia (NTDT), and the ENERGIZE-T trial is investigating mitapivat in patients with transfusion-dependent thalassaemia (TDT).
{"title":"Expert Insights: Phase III Clinical Trials in Non-Transfusion-Dependent (ENERGIZE) and Transfusion-Dependent (ENERGIZE-T) α- and β-Thalassaemia","authors":"H. Moir","doi":"10.33590/emjhematol/10308707","DOIUrl":"https://doi.org/10.33590/emjhematol/10308707","url":null,"abstract":"The thalassaemias are a heterogeneous group of inherited chronic blood disorders associated with impaired haemoglobin (Hb) synthesis, resulting in ineffective erythropoiesis, haemolysis, and the development of lifelong anaemia. The pathophysiology of thalassaemia is due, in part, to increased energy demand to clear globin aggregates and reactive O2 species, and maintain overall red blood cell (RBC) health, coupled with insufficient adenosine triphosphate (ATP) production.\u0000\u0000For this article, interviews were conducted by EMJ in December 2022 with two key opinion leaders. Kevin Kuo is a Clinician-Investigator and Staff Haematologist at the University Health Network, Toronto, Canada, and Associate Professor in the Division of Hematology, Department of Medicine, University of Toronto, Canada. Maria Cappellini is Professor of Medicine in the Department of Clinical Sciences and Community, University of Milan, Italy. Both haematologists have over 50 years of expertise and clinical experience between them in treating patients with thalassaemia. The two experts provided insights into two ongoing Phase III clinical trials for both α- and β-thalassaemia. These trials are investigating the effect of mitapivat, a first-in-class, small molecule pyruvate kinase activator, in patients across the full range of thalassaemia subtypes. The ENERGIZE trial is investigating mitapivat in patients with non–transfusion-dependent thalassaemia (NTDT), and the ENERGIZE-T trial is investigating mitapivat in patients with transfusion-dependent thalassaemia (TDT).","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"128856823","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 : 2023-07-20DOI: 10.33590/emjhematol/10301337.
{"title":"Review of the 28th Annual Congress of the European Hematology Association (EHA)","authors":"","doi":"10.33590/emjhematol/10301337.","DOIUrl":"https://doi.org/10.33590/emjhematol/10301337.","url":null,"abstract":"","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-07-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"125034273","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 : 2023-07-20DOI: 10.33590/emjhematol/10300820
{"title":"Infographic: Targeted Therapeutics in CLL and MCL","authors":"","doi":"10.33590/emjhematol/10300820","DOIUrl":"https://doi.org/10.33590/emjhematol/10300820","url":null,"abstract":"","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-07-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"127212920","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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