Pub Date : 2023-11-01Epub Date: 2023-11-23DOI: 10.1053/j.seminhematol.2023.11.001
Allison Barraclough, Eliza A Hawkes
Diffuse large B-cell lymphoma (DLBCL) is the most common lymphoma and a heterogeneous B-cell disease. The majority of patients with newly diagnosed disease are cured with first-line combination immunochemotherapy treatment however, those who experience treatment failure have dismal outcomes. Antibody therapies and immunotherapy have provided the single most major advance in the treatment of DLBCL in the last 4 decades. Rituximab, the first immunotherapy, and a monoclonal antibody targeting CD20, improved DLBCL overall survival when added to chemotherapy 2 decades ago. Since then, the advent of further "naked" monoclonal antibodies that target malignant B-cells or stimulate the immune system to kill cancer, as well as antibody-drug conjugates and bispecific antibodies have all entered the DLBCL armamentarium; with 5 antibody therapy approvals in the last 6 years alone. Here we review the literature on antibodies and immunotherapies for DLBCL and the future directions involving this successful group of drugs.
弥漫大 B 细胞淋巴瘤(DLBCL)是最常见的淋巴瘤,也是一种异质性 B 细胞疾病。大多数新确诊的患者都能通过一线免疫化疗联合疗法治愈,但治疗失败的患者的预后却令人沮丧。抗体疗法和免疫疗法是过去 40 年中治疗 DLBCL 的最大进步。利妥昔单抗是第一种免疫疗法,也是一种靶向 CD20 的单克隆抗体,20 年前在化疗中加入利妥昔单抗后,DLBCL 的总生存率得到了改善。从那时起,更多靶向恶性B细胞或刺激免疫系统杀灭癌症的 "裸 "单克隆抗体以及抗体-药物共轭物和双特异性抗体都进入了DLBCL的治疗领域;仅在过去6年中,就有5种抗体疗法获得批准。在此,我们回顾了有关 DLBCL 抗体和免疫疗法的文献,以及这组成功药物的未来发展方向。
{"title":"Antibody and immunotherapy in diffuse large B-cell lymphoma.","authors":"Allison Barraclough, Eliza A Hawkes","doi":"10.1053/j.seminhematol.2023.11.001","DOIUrl":"10.1053/j.seminhematol.2023.11.001","url":null,"abstract":"<p><p>Diffuse large B-cell lymphoma (DLBCL) is the most common lymphoma and a heterogeneous B-cell disease. The majority of patients with newly diagnosed disease are cured with first-line combination immunochemotherapy treatment however, those who experience treatment failure have dismal outcomes. Antibody therapies and immunotherapy have provided the single most major advance in the treatment of DLBCL in the last 4 decades. Rituximab, the first immunotherapy, and a monoclonal antibody targeting CD20, improved DLBCL overall survival when added to chemotherapy 2 decades ago. Since then, the advent of further \"naked\" monoclonal antibodies that target malignant B-cells or stimulate the immune system to kill cancer, as well as antibody-drug conjugates and bispecific antibodies have all entered the DLBCL armamentarium; with 5 antibody therapy approvals in the last 6 years alone. Here we review the literature on antibodies and immunotherapies for DLBCL and the future directions involving this successful group of drugs.</p>","PeriodicalId":21684,"journal":{"name":"Seminars in hematology","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138804479","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-09-01DOI: 10.1053/j.seminhematol.2023.07.004
Clara Chamba, William Mawalla
The world is moving towards precision medicine for cancer. This movement goes hand in hand with the development of newer advanced technologies for early, precise diagnosis of cancer and personalized treatment plans with fewer adverse effects for the patient. Liquid biopsy is one such advancement. At the same time, it has the advantage of minimal invasion and avoids serial invasive biopsies. In countries with limited access to pathology services, such as sub-Saharan Africa, liquid biopsy may provide an opportunity for early detection and prognostication of lymphoma. We discuss the current diagnostic modalities for lymphoma, highlighting the existing challenges with tissue biopsy, and how feasible it is for countries with limited pathology resources to leverage advancements made in the clinical application of liquid biopsy to improve lymphoma care.
{"title":"The future of lymphoma diagnosis, prognosis, and treatment monitoring in countries with limited access to pathology services","authors":"Clara Chamba, William Mawalla","doi":"10.1053/j.seminhematol.2023.07.004","DOIUrl":"10.1053/j.seminhematol.2023.07.004","url":null,"abstract":"<div><p>The world is moving towards precision medicine for cancer. This movement goes hand in hand with the development of newer advanced technologies for early, precise diagnosis of cancer and personalized treatment plans with fewer adverse effects for the patient. Liquid biopsy is one such advancement. At the same time, it has the advantage of minimal invasion and avoids serial invasive biopsies. In countries with limited access to pathology services, such as sub-Saharan Africa, liquid biopsy may provide an opportunity for early detection and prognostication of lymphoma. We discuss the current diagnostic modalities for lymphoma, highlighting the existing challenges with tissue biopsy, and how feasible it is for countries with limited pathology resources to leverage advancements made in the clinical application of liquid biopsy to improve lymphoma care.</p></div>","PeriodicalId":21684,"journal":{"name":"Seminars in hematology","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0037196323000562/pdfft?md5=5d043a805c643eede0343a10554cf43e&pid=1-s2.0-S0037196323000562-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10028555","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Patients with hematologic malignancies often experience fatigue, lack of vitality, and energy, and high psychological distress. High levels of unmet care needs of patients with hematologic malignancies in Asia were identified. This review provides an overview of current evidence on the experiences and palliative care needs of patients with hematologic malignancies and their families and the barriers and challenges of integrating palliative care into hematology care in Asia. Patients with hematologic malignancies who received palliative care could benefit from less aggressive end-of-life treatments. However, the uncertain and variable nature of the prognosis and illness trajectories of hematologic malignancies increase the difficulties of integrating palliative care into hematologic care. Patients and their families are often referred to palliative care services late, which leaves a short window for palliative care teams to provide holistic needs assessment and person-centered care for those who need it. In addition, cultural differences in medical decision-making patterns and complex social norms and interactions among patients, families, and healthcare staff make it even more challenging to initiate palliative care conversations in Asia. Future research should focus on the development and evaluation of culturally appropriate palliative care for patients with hematologic malignancies and their family caregivers in Asia, given that the low rate of service intake and poor public awareness of the important role of palliative care in disease trajectories were reported. The socio-cultural context surrounding individuals should be taken into consideration to ensure the provision of person-centered care for this group of patients. Digital health could be one of the possible solutions forward to address local needs and challenges.
{"title":"Palliative care needs of patients with hematologic malignancies and family caregivers and challenges of palliative care provision in Asia: A review of evidence","authors":"Yupawadee Kantabanlang , Cheng-Pei Lin , Kittikorn Nilmanat , Ping Guo","doi":"10.1053/j.seminhematol.2023.07.002","DOIUrl":"10.1053/j.seminhematol.2023.07.002","url":null,"abstract":"Patients with hematologic malignancies often experience fatigue, lack of vitality, and energy, and high psychological distress. High levels of unmet care needs of patients with hematologic malignancies in Asia were identified. This review provides an overview of current evidence on the experiences and palliative care needs of patients with hematologic malignancies and their families and the barriers and challenges of integrating palliative care into hematology care in Asia. Patients with hematologic malignancies who received palliative care could benefit from less aggressive end-of-life treatments. However, the uncertain and variable nature of the prognosis and illness trajectories of hematologic malignancies increase the difficulties of integrating palliative care into hematologic care. Patients and their families are often referred to palliative care services late, which leaves a short window for palliative care teams to provide holistic needs assessment and person-centered care for those who need it. In addition, cultural differences in medical decision-making patterns and complex social norms and interactions among patients, families, and healthcare staff make it even more challenging to initiate palliative care conversations in Asia. Future research should focus on the development and evaluation of culturally appropriate palliative care for patients with hematologic malignancies and their family caregivers in Asia, given that the low rate of service intake and poor public awareness of the important role of palliative care in disease trajectories were reported. The socio-cultural context surrounding individuals should be taken into consideration to ensure the provision of person-centered care for this group of patients. Digital health could be one of the possible solutions forward to address local needs and challenges.","PeriodicalId":21684,"journal":{"name":"Seminars in hematology","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0037196323000550/pdfft?md5=d02193cb245ff3dd12fb337015a070ad&pid=1-s2.0-S0037196323000550-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10273670","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-09-01DOI: 10.1053/j.seminhematol.2023.09.001
David J. Roberts , Aggrey Dhabangi
Setting priorities in healthcare is always contentious given the array of possible services at primary, secondary, and tertiary levels of care, not to mention potential public health interventions. The central goals in global policy have been reducing inequity within and between countries, protecting vulnerable groups (particularly women and children) and reducing the major communicable diseases which have historically been a major burden in lower- and middle-income countries. Here limited relative and absolute spending on healthcare have spurred a series of initiatives in Global Health over the last 50 years which have led to significant gains in measures of morbidity and mortality.
Against this background there remains the continuing question of how to adapt current medical practice in higher income countries for training and planning of services in lower- and middle-income countries. Here, the historical development of Global Health is outlined, and lessons drawn from the surveys of the global burden of disease and health economic analysis to understand how we can apply these principles to define Global Hematology.
It remains likely that in lower-income countries effort should be concentrated on developing laboratory services and blood transfusion, to allow safe and effective support for the assessment of treatment of anemia, sickle cell disease, maternal and child health and urgent surgery and obstetric services. However, the principles of Global Health, could also be used for hematological malignancies to develop a framework for Global Hematology for all settings.
{"title":"Debate: Should the loss of disability adjusted life years (DALY) define the focus of Global Hematology?","authors":"David J. Roberts , Aggrey Dhabangi","doi":"10.1053/j.seminhematol.2023.09.001","DOIUrl":"10.1053/j.seminhematol.2023.09.001","url":null,"abstract":"<div><p>Setting priorities in healthcare is always contentious given the array of possible services at primary, secondary, and tertiary levels of care, not to mention potential public health interventions. The central goals in global policy have been reducing inequity within and between countries, protecting vulnerable groups (particularly women and children) and reducing the major communicable diseases which have historically been a major burden in lower- and middle-income countries. Here limited relative and absolute spending on healthcare have spurred a series of initiatives in Global Health over the last 50 years which have led to significant gains in measures of morbidity and mortality.</p><p>Against this background there remains the continuing question of how to adapt current medical practice in higher income countries for training and planning of services in lower- and middle-income countries. Here, the historical development of Global Health is outlined, and lessons drawn from the surveys of the global burden of disease and health economic analysis to understand how we can apply these principles to define Global Hematology.</p><p>It remains likely that in lower-income countries effort should be concentrated on developing laboratory services and blood transfusion, to allow safe and effective support for the assessment of treatment of anemia, sickle cell disease, maternal and child health and urgent surgery and obstetric services. However, the principles of Global Health, could also be used for hematological malignancies to develop a framework for Global Hematology for all settings.</p></div>","PeriodicalId":21684,"journal":{"name":"Seminars in hematology","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0037196323000719/pdfft?md5=01a879d8520949e2a9404160a1c0ca58&pid=1-s2.0-S0037196323000719-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"49681996","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-09-01DOI: 10.1053/j.seminhematol.2023.09.002
Rodrigo T. Calado
Aplastic anemia (AA) is a rare serious hematologic disorder caused by hematopoietic stem cell failure in maintaining hematopoiesis. AA is virtually fatal if not treated, and diagnosis and therapy require extensive hematologic infrastructure. Academic medical centers in Brazil have continuously and significantly contributed to diagnostic tools and therapy development, from novel transplant strategies to drug combinations and implementation science in the national public health system. In the present review, we discuss how the collaborative effort among academic centers in hematology has contributed to improving health care for patients with aplastic anemia. We also discuss what needs are still unmet and how to overcome these challenges.
{"title":"The future of aplastic anemia treatment in Brazil: Lessons learned for global hematology","authors":"Rodrigo T. Calado","doi":"10.1053/j.seminhematol.2023.09.002","DOIUrl":"10.1053/j.seminhematol.2023.09.002","url":null,"abstract":"<div><p>Aplastic anemia (AA) is a rare serious hematologic disorder caused by hematopoietic stem cell failure in maintaining hematopoiesis. AA is virtually fatal if not treated, and diagnosis and therapy require extensive hematologic infrastructure. Academic medical centers in Brazil have continuously and significantly contributed to diagnostic tools and therapy development, from novel transplant strategies to drug combinations and implementation science in the national public health system. In the present review, we discuss how the collaborative effort among academic centers in hematology has contributed to improving health care for patients with aplastic anemia. We also discuss what needs are still unmet and how to overcome these challenges.</p></div>","PeriodicalId":21684,"journal":{"name":"Seminars in hematology","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0037196323000720/pdfft?md5=d33844a46c2bac667fdb1d6a743cbccd&pid=1-s2.0-S0037196323000720-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41127002","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-09-01DOI: 10.1053/j.seminhematol.2023.06.002
Ahlam Nasser , Hamisa Iddy
Over the last 2 decades, the introduction of targeted therapies and the advances in the detection of BCR::ABL1 oncogene have dramatically improved comprehensive care for patients with Chronic myeloid leukemia (CML). The once deadly malignancy has now transformed into a chronic disease with an overall patient survival approaching that of the age-matched general population. While excellent prognoses have been reported among CML patients in high-income countries, it is unfortunately not the same for those living in low and middle-income (LMIC) countries such as Tanzania. This disparity is largely contributed by barriers associated with the provision of comprehensive care including early diagnosis, access to treatment, and regular monitoring of the disease. In this review, we will share our experiences and lessons learned in setting up a network of comprehensive care for patients with CML in Tanzania.
{"title":"Setting up a network of comprehensive care for patients with chronic myeloid leukemia: Lessons learned from Tanzania","authors":"Ahlam Nasser , Hamisa Iddy","doi":"10.1053/j.seminhematol.2023.06.002","DOIUrl":"10.1053/j.seminhematol.2023.06.002","url":null,"abstract":"<div><p>Over the last 2 decades, the introduction of targeted therapies and the advances in the detection of <em>BCR::ABL1</em> oncogene have dramatically improved comprehensive care for patients with Chronic myeloid leukemia (CML). The once deadly malignancy has now transformed into a chronic disease with an overall patient survival approaching that of the age-matched general population. While excellent prognoses have been reported among CML patients in high-income countries, it is unfortunately not the same for those living in low and middle-income (LMIC) countries such as Tanzania. This disparity is largely contributed by barriers associated with the provision of comprehensive care including early diagnosis, access to treatment, and regular monitoring of the disease. In this review, we will share our experiences and lessons learned in setting up a network of comprehensive care for patients with CML in Tanzania.</p></div>","PeriodicalId":21684,"journal":{"name":"Seminars in hematology","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0037196323000471/pdfft?md5=679d3c885e6933e0cadbb597b2037baf&pid=1-s2.0-S0037196323000471-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9767702","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-09-01DOI: 10.1053/j.seminhematol.2023.08.005
Satish Gopal
A clear case for can be made for prioritizing malignant hematology services in low- and middle-income countries based on large public health burden, convincing demonstrations of cure and control, innovation opportunities with likely worldwide implications, and sizable returns on investment for health systems and societies. We must now ensure that need and opportunity are matched by commensurate levels of investment and attention.
{"title":"The case for prioritizing malignant hematology services in low- and middle-income countries","authors":"Satish Gopal","doi":"10.1053/j.seminhematol.2023.08.005","DOIUrl":"10.1053/j.seminhematol.2023.08.005","url":null,"abstract":"<div><p>A clear case for can be made for prioritizing malignant hematology services in low- and middle-income countries based on large public health burden, convincing demonstrations of cure and control, innovation opportunities with likely worldwide implications, and sizable returns on investment for health systems and societies. We must now ensure that need and opportunity are matched by commensurate levels of investment and attention.</p></div>","PeriodicalId":21684,"journal":{"name":"Seminars in hematology","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0037196323000707/pdfft?md5=1ade57a2a0a4092714809b20f34ce37f&pid=1-s2.0-S0037196323000707-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10301512","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-09-01DOI: 10.1053/j.seminhematol.2023.07.001
María José Mela Osorio, Carolina Pavlovsky, Miguel Arturo Pavlovsky
Health equity is today an important objective to evenly reach the population among different health care systems. This article will focus on diagnosis and treatment access inequalities in Argentina.
Although different aspects must be optimized to overcome access barriers worldwide, access inequalities in some regions of Argentina may depend basically on the type of health coverage or insurance. Health care in Argentina is divided into Public, Social security and Private care systems. Access to diagnosis and disease monitoring will vary according whether the patient is under each of these systems. Reducing inequalities may help target some important aspects not covered today and that may directly impact patients' outcome. Disparities in health cancer care were analyzed according to Public, Social security and Private sectors. A disadvantage in resource access, inadequate funding and limited medical infrastructures are common characteristics of the public health care systems. In our country the disparity between the public and private sectors in terms of timely diagnosis, stage of disease at diagnosis, accuracy of diagnosis, access to novel agents and transplantation is notorious, with the public sector lagging behind in access to diagnostic and treatment resources. While the Private sector has treatment outcomes comparable to those of high-income countries, challenges remain in the Public sector for patients who rely on early and accurate diagnosis and timely access to treatment. There is an urgent need to provide equitable care for multiple myeloma and CLL patients and reduce the emotional and financial consequences of the disease for the patient.
A survey about diagnosis and therapeutic resources was conducted between April and May 2023 among large centers in the Public, Social security and Private systems. A total of 49 hematologists from 31 institutions from five provinces of Argentina participated in the survey. We observed differences between the different systems with more access for the Private system on genetic diagnosis (FISH-IGVH access). More CLL patients in the public and social security systems were treated with CIT reflecting the inaccessibility in these sectors of more expensive targeted therapies rather than a gap in information since the Public centers surveyed were large hospitals with knowledgeable physicians. Access to different treatments both in first-line and relapsed settings was more equitable in the treatment of multiple myeloma for the different systems with the exception of access to daratumumab in first-line that was extremely infrequent in the public coverage. With increasing cost and treatment complexity as the introduction of CARTs and BITEs for CLL and MM, the gap will probably deepen more if the problem is not treated comprehensively by all the actors of the health sector: government, physicians, patients' organizations and pharmaceutical companies.
{"title":"Inequalities in access to treatment in Argentina: Differences in management of CLL and multiple myeloma?","authors":"María José Mela Osorio, Carolina Pavlovsky, Miguel Arturo Pavlovsky","doi":"10.1053/j.seminhematol.2023.07.001","DOIUrl":"10.1053/j.seminhematol.2023.07.001","url":null,"abstract":"<div><p>Health equity is today an important objective to evenly reach the population among different health care systems. This article will focus on diagnosis and treatment access inequalities in Argentina.</p><p>Although different aspects must be optimized to overcome access barriers worldwide, access inequalities in some regions of Argentina may depend basically on the type of health coverage or insurance. Health care in Argentina is divided into Public, Social security and Private care systems. Access to diagnosis and disease monitoring will vary according whether the patient is under each of these systems. Reducing inequalities may help target some important aspects not covered today and that may directly impact patients' outcome. Disparities in health cancer care were analyzed according to Public, Social security and Private sectors. A disadvantage in resource access, inadequate funding and limited medical infrastructures are common characteristics of the public health care systems. In our country the disparity between the public and private sectors in terms of timely diagnosis, stage of disease at diagnosis, accuracy of diagnosis, access to novel agents and transplantation is notorious, with the public sector lagging behind in access to diagnostic and treatment resources. While the Private sector has treatment outcomes comparable to those of high-income countries, challenges remain in the Public sector for patients who rely on early and accurate diagnosis and timely access to treatment. There is an urgent need to provide equitable care for multiple myeloma and CLL patients and reduce the emotional and financial consequences of the disease for the patient.</p><p>A survey about diagnosis and therapeutic resources was conducted between April and May 2023 among large centers in the Public, Social security and Private systems. A total of 49 hematologists from 31 institutions from five provinces of Argentina participated in the survey. We observed differences between the different systems with more access for the Private system on genetic diagnosis (FISH-IGVH access). More CLL patients in the public and social security systems were treated with CIT reflecting the inaccessibility in these sectors of more expensive targeted therapies rather than a gap in information since the Public centers surveyed were large hospitals with knowledgeable physicians. Access to different treatments both in first-line and relapsed settings was more equitable in the treatment of multiple myeloma for the different systems with the exception of access to daratumumab in first-line that was extremely infrequent in the public coverage. With increasing cost and treatment complexity as the introduction of CARTs and BITEs for CLL and MM, the gap will probably deepen more if the problem is not treated comprehensively by all the actors of the health sector: government, physicians, patients' organizations and pharmaceutical companies.</p></div>","PeriodicalId":21684,"journal":{"name":"Seminars in hematology","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0037196323000549/pdfft?md5=74ca2ab37ff8f79f3c026b7a4d7af25b&pid=1-s2.0-S0037196323000549-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10255087","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-09-01DOI: 10.1053/j.seminhematol.2023.08.004
Eduardo Flávio Oliveira Ribeiro , Ana Amélia Morais de Lacerda Mangueira Belmiro , Lenisa Cezar Vilas Boas , Carsten Utoft Niemann
In health care, innovation is a core part of the process that pushes advances forward. Drug and device development follow a step-by-step process from the discovery of a molecule to the final product. While patent filing and preclinical studies are usually performed by academic centers or start-ups, the clinical development is usually performed by pharmaceutical companies. To assess safety, efficacy and fulfil regulatory demands, clinical trials must be performed in sequential Phase I, II, and III stages prior to market access. In this context, clinical research centers have been established around the globe, also outside traditional academic centers, aiming to increase the access for patients to participate in clinical trials and the capacity for clinical development. The increasing number of clinical trial sites across the world, gives pharmaceutical companies, investigators and developers an improved access to properly test the exponentially increasing number of potential medicinal products and treatment approaches in trials in different parts of the world. Historically, Low- and Middle-Income Countries (LMIC) did not significantly take part in clinical trial development. As participation in all steps of clinical research provides earlier access to novel treatment options in LMIC along with creating data on efficacy and toxicity within more diverse populations, it is warranted to improve clinical trial access in LMIC. With the goal to provide input on how to tackle the challenges during the built of a clinical research center, we here describe the experience from setting up a clinical trial unit within a private hospital network in Brasília, Brazil, a Middle-Income country, to provide inspiration, “how to” knowledge and a recipe for those with a similar road ahead in LMIC.
{"title":"How to set up a clinical research center in Brazil, as an example of a middle-income country","authors":"Eduardo Flávio Oliveira Ribeiro , Ana Amélia Morais de Lacerda Mangueira Belmiro , Lenisa Cezar Vilas Boas , Carsten Utoft Niemann","doi":"10.1053/j.seminhematol.2023.08.004","DOIUrl":"10.1053/j.seminhematol.2023.08.004","url":null,"abstract":"<div><p>In health care, innovation is a core part of the process that pushes advances forward. Drug and device development follow a step-by-step process from the discovery of a molecule to the final product. While patent filing and preclinical studies are usually performed by academic centers or start-ups, the clinical development is usually performed by pharmaceutical companies. To assess safety, efficacy and fulfil regulatory demands, clinical trials must be performed in sequential Phase I, II, and III stages prior to market access. In this context, clinical research centers have been established around the globe, also outside traditional academic centers, aiming to increase the access for patients to participate in clinical trials and the capacity for clinical development. The increasing number of clinical trial sites across the world, gives pharmaceutical companies, investigators and developers an improved access to properly test the exponentially increasing number of potential medicinal products and treatment approaches in trials in different parts of the world. Historically, Low- and Middle-Income Countries (LMIC) did not significantly take part in clinical trial development. As participation in all steps of clinical research provides earlier access to novel treatment options in LMIC along with creating data on efficacy and toxicity within more diverse populations, it is warranted to improve clinical trial access in LMIC. With the goal to provide input on how to tackle the challenges during the built of a clinical research center, we here describe the experience from setting up a clinical trial unit within a private hospital network in Brasília, Brazil, a Middle-Income country, to provide inspiration, “how to” knowledge and a recipe for those with a similar road ahead in LMIC.</p></div>","PeriodicalId":21684,"journal":{"name":"Seminars in hematology","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0037196323000690/pdfft?md5=eb24959eb8709e2753f2a20c6bac79a4&pid=1-s2.0-S0037196323000690-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41177035","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-09-01DOI: 10.1053/j.seminhematol.2023.08.003
Bach Quoc Khanh , Vo Thi Thanh Binh , Nguyen Ha Thanh , Dao Phan Thu Huong , Do Thi Thuy , Nguyen Khanh Ha , Richard W. Childs
Through collaboration with international experts, our institution established a highly active and successful hematopoietic stem cell transplant program, providing access to this potentially curative treatment modality for patients with a variety of benign and malignant hematological diseases. The initial development of an autologous stem cell transplant program provided our institution with the infrastructure, equipment, and expertise needed for the subsequent development of an allogeneic stem cell transplant program. Key transplant staff received training from international transplant experts at the NHLBI/NIH, the Mayo Clinic, the Johns Hopkins Hospital, and Nagoya Japan, providing them with the expertise to conduct a variety of different transplant approaches, including PBSC transplants from HLA-matched relatives, unrelated cord blood transplants, haploidentical transplants, and CD34 selected stem cell transplants. Patient characteristics were varied among all groups. The number of allogeneic and autologous transplants performed at the NIHBT has increased steadily every year since the initiation of our transplant program. By 2022, 547 transplant procedures had been performed, including 268 autologous and 279 allogeneic transplants. Allogeneic transplants were performed for both malignant and nonmalignant hematological diseases, with acute leukemia (AL) being the most common indication for allogeneic HCT. The majority of recipients undergoing allogeneic transplantation received G-CSF mobilized PBSC allografts from either HLA identical or haplo-identical relatives, with a smaller percentage of patients receiving a UCB transplant or a PBSC allograft that had been CD34+ selected. Amongst the 279 recipients of an allogeneic transplant, mortality rates within day 100 and beyond day 100 were 12.6% and 26.2% respectively. Overall survival (OS) and event-free survival at 5 years in benign and malignant subgroups were 81% and 73% vs 52% and 48% respectively. Through collaboration with international transplant experts, the National Institute of Hematology and Blood Transfusion in Hanoi has stood up the most active transplant center in the northern region of Vietnam. Patients coming from low-income financial backgrounds are now able to receive a variety of different state-of-the-art transplant approaches that are affordable and have been associated with excellent long-term outcomes.
{"title":"Forging international collaboration and alliances to establish the largest transplant center in the north of Vietnam","authors":"Bach Quoc Khanh , Vo Thi Thanh Binh , Nguyen Ha Thanh , Dao Phan Thu Huong , Do Thi Thuy , Nguyen Khanh Ha , Richard W. Childs","doi":"10.1053/j.seminhematol.2023.08.003","DOIUrl":"10.1053/j.seminhematol.2023.08.003","url":null,"abstract":"<div><p>Through collaboration with international experts, our institution established a highly active and successful hematopoietic stem cell transplant program, providing access to this potentially curative treatment modality for patients with a variety of benign and malignant hematological diseases. The initial development of an autologous stem cell transplant program provided our institution with the infrastructure, equipment, and expertise needed for the subsequent development of an allogeneic stem cell transplant program. Key transplant staff received training from international transplant experts at the NHLBI/NIH, the Mayo Clinic, the Johns Hopkins Hospital, and Nagoya Japan, providing them with the expertise to conduct a variety of different transplant approaches, including PBSC transplants from HLA-matched relatives, unrelated cord blood transplants, haploidentical transplants, and CD34 selected stem cell transplants. Patient characteristics were varied among all groups. The number of allogeneic and autologous transplants performed at the NIHBT has increased steadily every year since the initiation of our transplant program. By 2022, 547 transplant procedures had been performed, including 268 autologous and 279 allogeneic transplants. Allogeneic transplants were performed for both malignant and nonmalignant hematological diseases, with acute leukemia (AL) being the most common indication for allogeneic HCT. The majority of recipients undergoing allogeneic transplantation received G-CSF mobilized PBSC allografts from either HLA identical or haplo-identical relatives, with a smaller percentage of patients receiving a UCB transplant or a PBSC allograft that had been CD34+ selected. Amongst the 279 recipients of an allogeneic transplant, mortality rates within day 100 and beyond day 100 were 12.6% and 26.2% respectively. Overall survival (OS) and event-free survival at 5 years in benign and malignant subgroups were 81% and 73% vs 52% and 48% respectively. Through collaboration with international transplant experts, the National Institute of Hematology and Blood Transfusion in Hanoi has stood up the most active transplant center in the northern region of Vietnam. Patients coming from low-income financial backgrounds are now able to receive a variety of different state-of-the-art transplant approaches that are affordable and have been associated with excellent long-term outcomes.</p></div>","PeriodicalId":21684,"journal":{"name":"Seminars in hematology","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0037196323000689/pdfft?md5=66822187ba53e03ea1d954df08ef043c&pid=1-s2.0-S0037196323000689-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10309883","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}