{"title":"Haematological emergencies","authors":"Shahira Butt, Merrina Gregory, Priya Sriskandarajah, Natalia Curto-Garcia","doi":"10.1016/j.mpmed.2025.03.006","DOIUrl":"10.1016/j.mpmed.2025.03.006","url":null,"abstract":"<div><div>This article provides an overview of the management of common haematological emergencies. Individuals with haematological disorders often present with co-morbidities and/or undergo chemotherapy, which can pose challenges to clinicians who are unfamiliar with these conditions. Accurate interpretation of laboratory data, in-depth knowledge of disease-related complications and strong clinical skills are critical for diagnosing and managing haematological emergencies. This review focuses on the key principles involved in diagnosing and managing the most frequently encountered emergencies in these patients.</div></div>","PeriodicalId":74157,"journal":{"name":"Medicine (Abingdon, England : UK ed.)","volume":"53 5","pages":"Pages 343-350"},"PeriodicalIF":0.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143924661","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}
{"title":"Myelodysplastic neoplasms","authors":"Jiexin Zheng","doi":"10.1016/j.mpmed.2025.02.007","DOIUrl":"10.1016/j.mpmed.2025.02.007","url":null,"abstract":"<div><div>Myelodysplastic neoplasms (MDSs) are rare clonal haematological neoplasms that result from disordered haematopoiesis. MDSs typically present in elderly individuals with cytopenia in one or more blood cell lineages. MDSs are diagnosed based on clinical, morphological, cytogenetic and genetic changes, with the latest international MDS classifications in 2022 incorporating these disease features into different MDS subtypes. Prognostic scoring systems can be used in individuals with MDS at presentation, to confer an estimated overall survival as well as guide appropriate treatment; this should be tailored based on the prognostic score as well as individual clinical factors.</div></div>","PeriodicalId":74157,"journal":{"name":"Medicine (Abingdon, England : UK ed.)","volume":"53 5","pages":"Pages 278-281"},"PeriodicalIF":0.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143924749","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}
{"title":"Myeloproliferative neoplasms","authors":"Laura Li Gagnon, Claire N Harrison","doi":"10.1016/j.mpmed.2025.02.006","DOIUrl":"10.1016/j.mpmed.2025.02.006","url":null,"abstract":"<div><div>Myeloproliferative neoplasms (MPNs), polycythaemia vera, essential thrombocythaemia and myelofibrosis – are uncommon clonal haematological malignancies generally diagnosed from late middle age onwards, although they can occur in children and young adults. They should be suspected in patients with elevated blood counts, atypical thrombosis or splenomegaly. Their clinical courses share similarities, including thrombosis, haemorrhage and a tendency to progress to myelofibrosis or acute myeloid leukaemia. Myelofibrosis has a poorer prognosis and significant disease burden affecting quality of life. Advances in diagnostics and genomics have recently been used to stratify risk more accurately. Furthermore, the development of treatment modalities aimed at targeting specific molecular pathways, such as Janus kinase inhibitors, has resulted in a therapeutic paradigm shift. Several ground-breaking studies have proved the efficacy of the first such agents and they are now licensed as first-line therapy in myelofibrosis and as second line in polycythaemia vera. Additional targets, such as <em>CALR</em>, are under evaluation in clinical trials. However, the mainstay of treatment for polycythaemia vera and essential thrombocythaemia remains aggressive management of thrombotic risk factors, antiplatelet therapy for most patients, and cytoreductive agents such as hydroxycarbamide and interferon for patients at high risk of thrombosis. This is, nevertheless, a rapidly evolving landscape.</div></div>","PeriodicalId":74157,"journal":{"name":"Medicine (Abingdon, England : UK ed.)","volume":"53 5","pages":"Pages 282-287"},"PeriodicalIF":0.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143924650","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}
{"title":"AL amyloidosis","authors":"Sajitha Sachchithanantham, Majid Kazmi","doi":"10.1016/j.mpmed.2025.02.005","DOIUrl":"10.1016/j.mpmed.2025.02.005","url":null,"abstract":"<div><div>Amyloidosis is a rare group of disorders with protean manifestations characterized by tissue deposition of misfolded protein. This causes progressive accumulation of amyloid deposits and disruption of organ function. Among patients with systemic amyloidosis, AL-type protein is the most common. This article focuses on AL amyloidosis, formerly known as primary amyloidosis, which usually affects the kidneys, heart, liver and peripheral nervous system. Diagnosis is rarely made until symptoms are referable to a particular organ. Untreated, it is progressive and fatal. Prognosis is determined by the extent of cardiac involvement. The three-step approach to diagnosis and investigation involves confirmation of amyloid and type, assessing the extent of organ involvement and confirmation of an underlying plasma cell dyscrasia/lymphoma. Treatment aims to reduce production of amyloidogenic light chains and stop progressive organ function deterioration. New drug classes such as proteasome inhibitors, immunomodulatory agents and anti-CD38 monoclonal antibodies have shown promise in reducing the light-chain burden, halting amyloid production and subsequently improving organ response and survival. Autologous stem cell transplantation has improved survival rates but is available only to a limited patient cohort. Bispecific antibodies, chimeric antigen receptor T cell therapies and agents directly removing amyloid fibrils in development show encouraging results in selected group of patients.</div></div>","PeriodicalId":74157,"journal":{"name":"Medicine (Abingdon, England : UK ed.)","volume":"53 5","pages":"Pages 331-333"},"PeriodicalIF":0.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143924658","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}
Ethan Wong, Aaryan Godhamgaonkar, Donal P McLornan
{"title":"Principles of haemopoietic stem cell transplantation","authors":"Ethan Wong, Aaryan Godhamgaonkar, Donal P McLornan","doi":"10.1016/j.mpmed.2025.02.012","DOIUrl":"10.1016/j.mpmed.2025.02.012","url":null,"abstract":"<div><div>Rapid developments within the field of stem cell transplantation and cellular therapy have occurred over the last few decades. Improvements in conditioning regimens coupled with advances in supportive and anti-infective care have in general correlated with improved survival compared with historical cohorts. Rapid adoption of haploidentical donors has allowed more patients without a matched sibling or well-matched unrelated donor to proceed with allogeneic transplantation as a therapeutic modality. Here, we highlight the main indications for autologous and allogeneic stem cell transplantations, with a focus on stem cell harvesting and graft-versus-host disease. Key areas of current research focus are highlighted, as is the exploration of novel immunotherapeutic approaches including chimeric antigen receptor T cell therapies.</div></div>","PeriodicalId":74157,"journal":{"name":"Medicine (Abingdon, England : UK ed.)","volume":"53 5","pages":"Pages 334-339"},"PeriodicalIF":0.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143924659","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}
{"title":"Self-assessment/CPD answers","authors":"","doi":"10.1016/j.mpmed.2025.03.005","DOIUrl":"10.1016/j.mpmed.2025.03.005","url":null,"abstract":"","PeriodicalId":74157,"journal":{"name":"Medicine (Abingdon, England : UK ed.)","volume":"53 5","pages":"Pages 351-354"},"PeriodicalIF":0.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143924662","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}
{"title":"Chronic myeloid leukaemia","authors":"Samantha Drummond, Mhairi Copland","doi":"10.1016/j.mpmed.2025.03.001","DOIUrl":"10.1016/j.mpmed.2025.03.001","url":null,"abstract":"<div><div>Chronic myeloid leukaemia (CML) is a clonal myeloproliferative disorder resulting from a reciprocal translocation between the long arms of chromosomes 9 and 22. This is termed the Philadelphia chromosome, and leads to production of the fusion oncoprotein BCR::ABL1, a 210 kDa constitutively active tyrosine kinase. CML has two distinct phases: chronic and blast. Most patients (95%) present in the chronic phase, which is associated with leucocytosis and splenomegaly. Blast phase is associated with bone marrow failure and carries a poor prognosis. The introduction of tyrosine kinase inhibitors (TKIs; imatinib, dasatinib, nilotinib, bosutinib, ponatinib) and a STAMP (specifically targeting the ABL myristoyl pocket) inhibitor (asciminib) have altered the clinical course of CML for most patients, turning it from a fatal leukaemia to a chronic disorder managed with oral medication. Patients with chronic phase CML have excellent responses to TKIs, and individuals with an optimal response can expect a normal lifespan; some successfully discontinue TKI therapy. However, resistance to TKIs is seen, particularly in blast phase CML. In these patients, allogeneic stem cell transplantation is an important treatment option. With increasing experience in TKI use, different adverse effect profiles are emerging and require consideration when choosing the most suitable TKI for an individual patient.</div></div>","PeriodicalId":74157,"journal":{"name":"Medicine (Abingdon, England : UK ed.)","volume":"53 5","pages":"Pages 304-307"},"PeriodicalIF":0.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143924653","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}
Austin G Kulasekararaj, Shreyans Gandhi, Roochi Trikha
{"title":"Bone marrow failure: causes and complications","authors":"Austin G Kulasekararaj, Shreyans Gandhi, Roochi Trikha","doi":"10.1016/j.mpmed.2025.02.009","DOIUrl":"10.1016/j.mpmed.2025.02.009","url":null,"abstract":"<div><div>Aplastic anaemia (AA), a rare but serious form of bone marrow failure (BMF), is characterized by pancytopenia with hypocellular bone marrow. The pathophysiology of acquired AA is predominantly immunologically mediated, with damage to haemopoietic stem cells by autoreactive T lymphocytes. The aberrant immune response can be triggered by environmental triggers, such as drugs, toxins, chemicals and viral infections, especially after seronegative hepatitis. A careful medical and family history, physical examination and investigations are required to rule out rarer inherited causes of BMFs, as well as other acquired diseases, such as hypoplastic myelodysplastic syndrome, with a similar phenotype. The clinical spectrum of acquired AA varies: some patients with mild cytopenia do not require therapy; others have life-threatening complications resulting from severe pancytopenia. The autoimmune basis provides the rationale for treatment, with either combined immunosuppressive therapy using horse antithymocyte globulin and ciclosporin with eltrombopag, or allogeneic haemopoietic stem cell transplantation (HSCT). Long-term survival of 70–80% can be expected from these standard therapeutic modalities. Recurrence of pancytopenia, secondary to relapse, clonal evolution to myelodysplastic syndrome and paroxysmal nocturnal haemoglobinuria are late sequelae after immunosuppressive therapy. By contrast, HSCT provides the chance of long-term cure.</div></div>","PeriodicalId":74157,"journal":{"name":"Medicine (Abingdon, England : UK ed.)","volume":"53 5","pages":"Pages 273-277"},"PeriodicalIF":0.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143924748","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}
{"title":"Observational and real-world studies","authors":"Anthony Lockett","doi":"10.1016/j.mpmed.2025.03.010","DOIUrl":"10.1016/j.mpmed.2025.03.010","url":null,"abstract":"<div><div>While randomized studies provide the best evidence, they are not practical in some circumstances, for example rare diseases. Observational and real-world studies cannot replace randomized trials but they provide evidence of causality to be interpreted with other data. Observational studies are of two types – descriptive and analytical. Observational and real-world studies suffer from several biases, mainly selection bias. This is hard to overcome, but can be mitigated by the use of propensity analysis and sensitivity analysis.</div></div>","PeriodicalId":74157,"journal":{"name":"Medicine (Abingdon, England : UK ed.)","volume":"53 6","pages":"Pages 358-360"},"PeriodicalIF":0.0,"publicationDate":"2025-04-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144147590","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}
{"title":"Summarizing and presenting data from clinical trials","authors":"Gregory L Ginn, Clare Campbell-Cooper","doi":"10.1016/j.mpmed.2025.04.008","DOIUrl":"10.1016/j.mpmed.2025.04.008","url":null,"abstract":"<div><div>Clinical trials rely on rigorous data preparation and analysis to ensure robust, reliable outcomes. Key components include defining analysis populations, handling missing data and evaluating primary and secondary endpoints. Analysis populations, such as intent-to-treat and per-protocol, play a pivotal role in interpreting treatment efficacy under both real-world and ideal conditions. Handling of missing data, a critical challenge, employs techniques such as multiple imputation and maximum likelihood estimation to minimize bias and preserve validity. Efficacy data analysis revolves around predefined endpoints, with primary endpoints driving trial success and regulatory approval, and secondary endpoints providing broader insights into treatment effects. Subgroup and longitudinal analyses offer nuanced understandings of differential treatment effects and time-based outcomes, leveraging statistical tools such as mixed-effects models and Kaplan–Meier curves. Safety analyses, including adverse event reporting and time-to-event models, are essential for assessing treatment risks. Comparative safety analysis evaluates adverse events, serious adverse events and risk–benefit balances between treatments using methods such as logistic regression and Cox proportional hazards models. By integrating these methodologies, clinical trials provide comprehensive evaluations of treatments, guiding regulatory decisions and advancing medical knowledge. This systematic approach ensures that findings are both scientifically rigorous and clinically relevant.</div></div>","PeriodicalId":74157,"journal":{"name":"Medicine (Abingdon, England : UK ed.)","volume":"53 6","pages":"Pages 368-375"},"PeriodicalIF":0.0,"publicationDate":"2025-04-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144147491","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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