{"title":"Current challenges and new approaches to implementing optimal management of sickle cell disease in sub-Saharan Africa","authors":"Mwashungi Ally, Emmanuel Balandya","doi":"10.1053/j.seminhematol.2023.08.002","DOIUrl":"10.1053/j.seminhematol.2023.08.002","url":null,"abstract":"Sickle cell disease (SCD) is the most common life-threatening monogenic disorder in the world. The disease is highly prevalent in malaria endemic areas with over 75% of patients residing in Sub-Saharan Africa (SSA). It is estimated that, without proper care, up to 90% of children with SCD will not celebrate their fifth birthday. Early identification and enrolment into comprehensive care has been shown to reduce the morbidity and mortality related with SCD complications. However, due to resource constraints, the SSA is yet to implement universal newborn screening programs for SCD. Furthermore, care for patients with SCD in the region is hampered by the shortage of qualified healthcare workers, lack of guidelines for the clinical management of SCD, limited infrastructure for inpatient and outpatient care, and limited access to blood and disease modifying drugs such as Hydroxyurea which contribute to poor clinical outcomes. Curative options such as bone marrow transplant and gene therapy are expensive and not available in many SSA countries. In addressing these challenges, various initiatives are ongoing in SSA which aim to enhance awareness on SCD, improve patient identification and retention to care, harmonize the standards of care for SCD, improve the skills of healthcare workers and conduct research on pertinent areas in SCD in the SSA context. Fortifying these measures is paramount to improving the outcomes of SCD in SSA.","PeriodicalId":21684,"journal":{"name":"Seminars in hematology","volume":"60 4","pages":"Pages 192-199"},"PeriodicalIF":3.6,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0037196323000598/pdfft?md5=88420ef74912b8813dee55a0e41485c5&pid=1-s2.0-S0037196323000598-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41151226","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}
Bach Quoc Khanh , Vo Thi Thanh Binh , Nguyen Ha Thanh , Dao Phan Thu Huong , Do Thi Thuy , Nguyen Khanh Ha , Richard W. Childs
{"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":"60 4","pages":"Pages 243-250"},"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}
{"title":"outside front cover, PMS 8883 metallic AND 4/C","authors":"","doi":"10.1053/S0037-1963(23)00084-7","DOIUrl":"10.1053/S0037-1963(23)00084-7","url":null,"abstract":"","PeriodicalId":21684,"journal":{"name":"Seminars in hematology","volume":"60 4","pages":"Page CO1"},"PeriodicalIF":3.6,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135782083","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}
{"title":"Access to essential therapy for sickle cell disease in Africa: Experience from a national program in Ghana","authors":"Christine Nyonator , Emefa Amoah , Etta Forson Addo , Maureen Mukanga , Augustine Kwabena Asubonteng , Kwaku Ohene-Frempong , Jonathan Michael Spector , Solomon Fiifi Ofori-Acquah","doi":"10.1053/j.seminhematol.2023.06.001","DOIUrl":"10.1053/j.seminhematol.2023.06.001","url":null,"abstract":"<div><p>Novartis, a global medicines company, and the Sickle Cell Foundation of Ghana (SCFG), an advocacy organization, have endeavored to support the implementation of global best practices in the care of people living with sickle cell disease (SCD) in Africa, and to address unmet needs relating to this condition on the continent. Beginning in 2019, a multifaceted SCD program was implemented in Ghana through a public-private partnership involving the government of Ghana, the SCFG, Novartis, and other partners. A key component of the program involved expanding the reach of hydroxyurea (HU), the only approved disease-modifying generic treatment for SCD, in ways that would promote sustainable access. The program helped to raise the profile of SCD in Ghana and, in 2022, the government adopted HU into its National Health Insurance Scheme. Features of the effort in Ghana are now being expanded to other countries in Africa through cocreated programs with in-country partners. This article reviews the program's history, progress, challenges, and lessons learned.</p></div>","PeriodicalId":21684,"journal":{"name":"Seminars in hematology","volume":"60 4","pages":"Pages 226-232"},"PeriodicalIF":3.6,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0037196323000483/pdfft?md5=5b792d8fcf97d42a6ca5fea7e938097b&pid=1-s2.0-S0037196323000483-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9853742","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}
{"title":"Special edition of the Seminars in Hematology series on Global Hematology Care","authors":"Anna Schuh","doi":"10.1053/j.seminhematol.2023.09.003","DOIUrl":"10.1053/j.seminhematol.2023.09.003","url":null,"abstract":"","PeriodicalId":21684,"journal":{"name":"Seminars in hematology","volume":"60 4","pages":"Pages 179-181"},"PeriodicalIF":3.6,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0037196323000732/pdfft?md5=81fc00d099d2c217fce807c5b115cae4&pid=1-s2.0-S0037196323000732-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41211669","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}
{"title":"Circulating tumor DNA in NK/T and peripheral T cell lymphoma","authors":"Yu-Jia Huo , Wei-Li Zhao","doi":"10.1053/j.seminhematol.2023.07.003","DOIUrl":"10.1053/j.seminhematol.2023.07.003","url":null,"abstract":"<div><p>Natural killer (NK)/T-cell lymphomas (NK/TCL) and peripheral T-cell lymphomas (PTCL) are aggressive hematological malignancies. With the development of next-generation sequencing, circulating tumor DNA (ctDNA) can be detected by several techniques with clinical implications. So far, the effect of ctDNA in pretreatment prognosis prediction, longitudinal monitoring of treatment response and surveillance of long-term remission or relapse in NK/TCL and PTCL has been reported in several researches.</p></div>","PeriodicalId":21684,"journal":{"name":"Seminars in hematology","volume":"60 3","pages":"Pages 173-177"},"PeriodicalIF":3.6,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10281525","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}
{"title":"Cell-free DNA in large B-cell lymphoma: MRD and beyond","authors":"Brian J. Sworder , David M. Kurtz","doi":"10.1053/j.seminhematol.2023.06.004","DOIUrl":"10.1053/j.seminhematol.2023.06.004","url":null,"abstract":"<div><p>Large B-cell lymphomas (LBCLs) are a strikingly diverse set of diseases<span>, including clinical, biological, and molecular heterogeneity. Despite a wealth of information resolving this heterogeneity in the research setting, applying molecular features routinely in the clinic remains challenging. The advent of circulating tumor DNA<span> (ctDNA) liquid biopsies promises to unlock additional molecular information in the clinic, including mutational genotyping, molecular classification, and minimal residual disease detection. Here, we examine the technologies, applications, and studies exploring the utility of ctDNA in LBCLs.</span></span></p></div>","PeriodicalId":21684,"journal":{"name":"Seminars in hematology","volume":"60 3","pages":"Pages 142-149"},"PeriodicalIF":3.6,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10528139/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10280055","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}
{"title":"outside front cover, PMS 8883 metallic AND 4/C","authors":"","doi":"10.1053/S0037-1963(23)00060-4","DOIUrl":"https://doi.org/10.1053/S0037-1963(23)00060-4","url":null,"abstract":"","PeriodicalId":21684,"journal":{"name":"Seminars in hematology","volume":"60 3","pages":"Page CO1"},"PeriodicalIF":3.6,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"50185625","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}
{"title":"Clinical applications of circulating tumor DNA in indolent B-cell lymphomas","authors":"Rahul Lakhotia, Mark Roschewski","doi":"10.1053/j.seminhematol.2023.06.003","DOIUrl":"10.1053/j.seminhematol.2023.06.003","url":null,"abstract":"<div><p><span><span><span>Indolent B-cell lymphomas are generally incurable with standard therapy and most patients have a prolonged disease course that includes multiple treatments and periods of time in which they do not require therapy. Currently available tools to monitor </span>disease burden and define response to treatment rely heavily on imaging scans that lack tumor specificity are unable to detect disease at the molecular level. </span>Circulating tumor DNA (ctDNA) is a versatile and promising biomarker being developed across multiple lymphoma subtypes. Advantages of ctDNA include high tumor specificity and limits of detection that are significantly lower than imaging scans. Potential clinical applications of ctDNA in indolent B-cell lymphomas include baseline prognostication, early signs of treatment resistance, measurements of minimal residual disease, and a noninvasive method to directly monitor disease burden and clonal evolution after therapy. Clinical applications of ctDNA have not yet proven clinical utility but are increasingly used as translational endpoints in </span>clinical trials testing novel approaches and the analytic techniques used for ctDNA continue to evolve. Advances in therapy for indolent B-cell lymphomas include novel targeted agents and combinations that achieve very high rates complete response which amplifies the need to improve our current methods to monitor disease.</p></div>","PeriodicalId":21684,"journal":{"name":"Seminars in hematology","volume":"60 3","pages":"Pages 164-172"},"PeriodicalIF":3.6,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10527907/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10332704","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}