Alpesh Goyal (Assistant Professor), Nikhil Tandon (Professor and Head)
{"title":"Burosumab: Current status and future prospects","authors":"Alpesh Goyal (Assistant Professor), Nikhil Tandon (Professor and Head)","doi":"10.1016/j.beem.2023.101826","DOIUrl":"10.1016/j.beem.2023.101826","url":null,"abstract":"<div><p><span><span>Hypophosphatemic rickets/osteomalacia caused by FGF23 excess is conventionally treated with </span>multiple doses<span> of inorganic phosphate salts and active vitamin D analogs. However, conventional therapy targets the consequences of elevated FGF23 and not the elevated FGF23 itself and is associated with poor adherence and long-term complications such as </span></span>nephrocalcinosis<span><span><span> and secondary/tertiary hyperparathyroidism. Burosumab is a fully human </span>IgG1<span><span><span> monoclonal antibody that binds to and neutralises FGF-23, thereby leading to improvement in </span>phosphate homeostasis and healing of </span>rickets<span><span><span> and osteomalacia. Data from phase 2 and 3 trials report overall safety and efficacy and Burosumab is now FDA approved for </span>treatment of </span>XLH and TIO in children and adults. Cost and absence of long-term data are major issues with Burosumab which should be addressed in near future. At present, experts recommend Burosumab use </span></span></span>in patients with severe disease or those with mild-moderate disease and a failed response to a trial of conventional therapy.</span></p></div>","PeriodicalId":8810,"journal":{"name":"Best practice & research. Clinical endocrinology & metabolism","volume":"38 2","pages":"Article 101826"},"PeriodicalIF":7.4,"publicationDate":"2023-09-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"49686234","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Marina Tsoli (Consultant Endocrinologist) , Anna Koumarianou (Consultant Medical Oncologist) , Anna Angelousi (Associate Professor of Internal Medicine) , Gregory Kaltsas (Professor of Endocrinology)
{"title":"Established and novel circulating neuroendocrine tumor biomarkers for diagnostic, predictive and prognostic use","authors":"Marina Tsoli (Consultant Endocrinologist) , Anna Koumarianou (Consultant Medical Oncologist) , Anna Angelousi (Associate Professor of Internal Medicine) , Gregory Kaltsas (Professor of Endocrinology)","doi":"10.1016/j.beem.2023.101785","DOIUrl":"10.1016/j.beem.2023.101785","url":null,"abstract":"<div><p><span>The management of neuroendocrine tumors<span> (NETs) represents a clinical challenge due to heterogeneity of their clinical behaviour, molecular biology<span> and response to treatment. Over the years, several circulating biomarkers have been developed for the early diagnosis and follow-up of NETs. The specific secretory products of tumors associated with a secretory syndrome (functioning tumors) may be used as diagnostic and/or prognostic biomarkers while the most common non-specific circulating biomarkers, that may be increased in both functioning and non-functioning tumors, are </span></span></span>chromogranin A<span> and the neuron specific enolase. However, the diagnostic accuracy as well as the prognostic and predictive value of these biomarkers are limited and novel techniques of multianalyte analysis of regulators of tumor biology have been developed. The NETest has been most extensively studied and proved to be useful in NET diagnosis, early detection of post-operative recurrence and prediction of response to treatment but further investigation establishing higher level of evidence is required for implementation in clinical practice.</span></p></div>","PeriodicalId":8810,"journal":{"name":"Best practice & research. Clinical endocrinology & metabolism","volume":"37 5","pages":"Article 101785"},"PeriodicalIF":7.4,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10037552","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Kjetil Søreide , Julie Hallet , Nigel B. Jamieson , Stefan Stättner
{"title":"Optimal surgical approach for digestive neuroendocrine neoplasia primaries: Oncological benefits versus short and long-term complications","authors":"Kjetil Søreide , Julie Hallet , Nigel B. Jamieson , Stefan Stättner","doi":"10.1016/j.beem.2023.101786","DOIUrl":"10.1016/j.beem.2023.101786","url":null,"abstract":"<div><p>The rising incidence and the accumulating prevalence of neuroendocrine neoplasia (NEN) in the population makes this a common, prevalent and a clinically relevant disease group. Surgical resection represents the only potentially curative treatment for digestive NENs. Thus, resection should in principle be considered for all patients with NEN, although taking the patients age, relevant comorbidity, and performance status into account for operability. Patients with insulinomas, NEN of the appendix and rectal NENs are usually cured by surgery alone. However, less than a third of patients are amendable to curative surgery alone at time of diagnosis. Furthermore, recurrence is common and may occur years after primary surgery, hence the long follow-up time recommended in most NENs (>10 years). As many patients with NENs present with locoregional or metastatic disease, there is considerable debate regarding the role of debulking surgery in these settings. However, good long-term survival can be achieved in a considerable proportion of patients, with 50–70% alive up to 10 years after surgery. Location and grade are the main determinants of long-term survival. Here we present considerations to surgery for primary neuroendocrine tumors in the digestive tract.</p></div>","PeriodicalId":8810,"journal":{"name":"Best practice & research. Clinical endocrinology & metabolism","volume":"37 5","pages":"Article 101786"},"PeriodicalIF":7.4,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9641301","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Ashley Kieran Clift (Clinical Research Fellow) , Robert Thomas (Consultant Diagnostic and Interventional Radiologist) , Andrea Frilling (Chair in Endocrine Surgery)
{"title":"Developments in interventional management of hepatic metastases from neuroendocrine tumours","authors":"Ashley Kieran Clift (Clinical Research Fellow) , Robert Thomas (Consultant Diagnostic and Interventional Radiologist) , Andrea Frilling (Chair in Endocrine Surgery)","doi":"10.1016/j.beem.2023.101798","DOIUrl":"10.1016/j.beem.2023.101798","url":null,"abstract":"<div><p>Neuroendocrine tumours commonly metastasise to the liver, particularly those arising from the intestinal tract and pancreas. Whilst surgery offers the only approach with intent to cure, the vast majority of patients with neuroendocrine liver metastases are ineligible. Liver-directed interventional therapies seek to exploit the patho-anatomy of the blood supply of hepatic metastases to deliver therapy to liver deposits. This may involve percutaneous ablation, bland embolization, or the selective infusion of chemotherapeutics, targeted agents or radiolabelled embolic material. Retrospective case series evidence has characterised objective response rates, disease control rates, and longer-term outcomes associated with each approach. Recent advances in this field include ongoing comparative trials of different techniques, but more importantly, combinations of interventional liver-directed therapies and other systemic therapy in multimodal treatment concepts.</p></div>","PeriodicalId":8810,"journal":{"name":"Best practice & research. Clinical endocrinology & metabolism","volume":"37 5","pages":"Article 101798"},"PeriodicalIF":7.4,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9834725","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Maria Passhak (Consultant in Clinical Oncology) , Mairéad G. McNamara (Senior Lecturer and Consultant in Medical Oncology) , Richard A. Hubner (Consultant in Medical Oncology) , Irit Ben-Aharon (Professor in Oncology) , Juan W. Valle (Professor in Medical Oncology)
{"title":"Choosing the best systemic treatment sequence for control of tumour growth in gastro-enteropancreatic neuroendocrine tumours (GEP-NETs): What is the recent evidence?","authors":"Maria Passhak (Consultant in Clinical Oncology) , Mairéad G. McNamara (Senior Lecturer and Consultant in Medical Oncology) , Richard A. Hubner (Consultant in Medical Oncology) , Irit Ben-Aharon (Professor in Oncology) , Juan W. Valle (Professor in Medical Oncology)","doi":"10.1016/j.beem.2023.101836","DOIUrl":"10.1016/j.beem.2023.101836","url":null,"abstract":"<div><p>Gastro-enteropancreatic neuroendocrine tumours (GEP-NETs) represent a rare and highly heterogeneous entity with increasing incidence. Based on the results obtained from several trials performed in the last decade, various therapeutic options have been established for the treatment of patients with GEP-NETs. The options include somatostatin analogues, targeted therapies (sunitinib and everolimus), chemotherapy (with temozolomide or streptozocin-based regimens), and peptide receptor radionuclide therapy. The treatment choice is influenced by various clinico-pathological factors including tumour grade and morphology, the primary mass location, hormone secretion, the volume of the disease and the rate of tumour growth, as well as patient comorbidities and performance status. In this review, the efficacy and safety of treatment options for patients with GEP-NETs is discussed and the evidence to inform the best sequence of available therapies to control tumour growth, prolong patient survival, and to lower potential toxicity, while maintaining patient quality of life is explored.</p></div>","PeriodicalId":8810,"journal":{"name":"Best practice & research. Clinical endocrinology & metabolism","volume":"37 5","pages":"Article 101836"},"PeriodicalIF":7.4,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71430139","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Nicola Fazio (Oncologist, Director of Program in Digestive System and Neuroendocrine Tumors) , Anna La Salvia (Oncologist, Researcher)
{"title":"Precision medicine in gastroenteropancreatic neuroendocrine neoplasms: Where are we in 2023?","authors":"Nicola Fazio (Oncologist, Director of Program in Digestive System and Neuroendocrine Tumors) , Anna La Salvia (Oncologist, Researcher)","doi":"10.1016/j.beem.2023.101794","DOIUrl":"10.1016/j.beem.2023.101794","url":null,"abstract":"<div><p><em>Precision medicine</em><span><span> describes a target-related approach to tailoring diagnosis and treatment of the individual patient. While this personalized approach is revoluzionizing many areas of </span>oncology, it is quite late in the field of gastroenteropancreatic neuroendocrine neoplasms (GEP-NENs), in which there are few molecular alterations to be therapeutically targeted. We critically reviewed the current evidence about </span><em>precision medicine</em><span><span> in GEP NENs, focusing on potential clinically relevant actionable targets for GEP NENs, such as the mTOR pathway, </span>MGMT<span><span>, hypoxia<span><span> markers, RET, DLL-3, and some general agnostic targets. We analysed the main investigational approaches with solid and liquid biopsies. Furthermore, we reviewed a model of precision medicine more specific for NENs that is the theragnostic use of </span>radionuclides. Overall, currently no true </span></span>predictive factors for therapy have been validated so far in GEP NENs, and the personalized approach is based more on clinical thinking within a NEN-dedicated multidisciplinary team. However, there is a robust background to suppose that precision medicine, with the theragnostic model will yield new insights in this context soon.</span></span></p></div>","PeriodicalId":8810,"journal":{"name":"Best practice & research. Clinical endocrinology & metabolism","volume":"37 5","pages":"Article 101794"},"PeriodicalIF":7.4,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9749487","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Johannes Hofland (Consultant Endocrinologist), Wouter W. de Herder (Consultant Endocrinologist)
{"title":"Effective strategies for adequate control of hormonal secretion in functioning neuroendocrine neoplasms","authors":"Johannes Hofland (Consultant Endocrinologist), Wouter W. de Herder (Consultant Endocrinologist)","doi":"10.1016/j.beem.2023.101787","DOIUrl":"10.1016/j.beem.2023.101787","url":null,"abstract":"<div><p>Neuroendocrine neoplasms are a unique form of malignancies as they can be accompanied by specific functioning hormonal syndromes that can impair survival and quality of life in patients. Functioning syndromes are defined by the combination of specific clinical signs and symptoms in combination with inappropriately elevated circulating levels of hormones. Clinicians should remain vigilant for the presence of functioning syndromes in neuroendocrine neoplasm patients at presentation as well as during follow-up. The correct diagnostic work-up should be initiated in cases of clinical suspicion of a neuroendocrine neoplasm-associated functioning syndrome. Management of a functional syndrome includes options from supportive, surgical, hormonal and antiproliferative treatment modalities. Here, we review the patient and tumour characteristics for each functioning syndrome that should be taken into account when deciding the optimum treatment strategy in neuroendocrine neoplasm patients.</p></div>","PeriodicalId":8810,"journal":{"name":"Best practice & research. Clinical endocrinology & metabolism","volume":"37 5","pages":"Article 101787"},"PeriodicalIF":7.4,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9626513","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Updates in histopathological classification and tissue biomarkers of digestive neuroendocrine neoplasms: What the clinician should know","authors":"Anne Couvelard (Pathologist) , Aurélie Cazes Pathologist , Jérôme Cros (Pathologist)","doi":"10.1016/j.beem.2023.101795","DOIUrl":"10.1016/j.beem.2023.101795","url":null,"abstract":"<div><p><span>Histopathological classifications of neuroendocrine neoplasms (NEN) change regularly and the latest WHO classification published in 2022, which concerns all NEN in the body, attempts to standardize classifications in the different locations. Differentiation and proliferation mainly assessed by Ki-67 index are still the cornerstone of those classifications. However, many markers are now used for diagnostic (to check neuroendocrine differentiation<span>, to identify the site of origin of a metastasis, to help separating high-grade neuroendocrine tumors/NET and neuroendocrine carcinoma/NEC), prognostic or theranostic purposes. NENs are often heterogeneous and this can lead to difficulties in classifications, biomarker and </span></span>prognostic assessment. These different points are discussed successively in this review, insisting especially on the frequent digestive, gastro-entero-pancreatic (GEP) localizations.</p></div>","PeriodicalId":8810,"journal":{"name":"Best practice & research. Clinical endocrinology & metabolism","volume":"37 5","pages":"Article 101795"},"PeriodicalIF":7.4,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9823299","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Adeel Haq (Fellow Nuclear Medicine), Sampanna Rayamajhi (Fellow Nuclear Medicine), Maria Rosana Ponisio (Assoc Prof of Radiology), Vikas Prasad (Assoc Prof of Radiology)
{"title":"New horizon of radiopharmaceuticals in management of neuroendocrine tumors","authors":"Adeel Haq (Fellow Nuclear Medicine), Sampanna Rayamajhi (Fellow Nuclear Medicine), Maria Rosana Ponisio (Assoc Prof of Radiology), Vikas Prasad (Assoc Prof of Radiology)","doi":"10.1016/j.beem.2023.101797","DOIUrl":"10.1016/j.beem.2023.101797","url":null,"abstract":"<div><p><span>Neuroendocrine neoplasms are rare and heterogenous group of tumors with varying degrees of clinical presentations and involvement of multiple organ systems in the body. In the modern clinical practice somatostatin receptor </span>molecular imaging<span><span><span> and targeted radioligand therapy plays a vital role in the diagnosis and management of the disease. Several new and promising </span>radiotracers<span> for NET imaging and theranostics, belonging to various groups and classes are being studied and investigated. This exponential growth of radiotracers poses concerns about the indication, clinical benefit, and safety profile of the agents. We discuss the basis behind these radiotracers clinical use, receptor targeting and intra and inter tumor heterogeneity. Furthermore, role of dual tracer imaging, combination therapy and potential applications of </span></span>dosimetry<span> in predicting treatment outcome and safety profile is reviewed. Individualized precision medicine with better tumor characterization, maximum therapeutic benefit and minimum toxicity is the way forward for future medicine.</span></span></p></div>","PeriodicalId":8810,"journal":{"name":"Best practice & research. Clinical endocrinology & metabolism","volume":"37 5","pages":"Article 101797"},"PeriodicalIF":7.4,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9834720","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
C. Lacalle-González (Medical Oncologist) , A. Estrella Santos (Endocrinologist) , L.C. Landaeta Kancev (Nuclear Medicine Physician) , V.M. Castellano (Pathologist) , E. Macia Palafox (Cardiologist) , A. Paniagua Ruíz (Endocrinologist) , J. Luna Tirado (Radiation Oncologist) , B. Martínez-Amores (Medical Oncologist) , L. Martínez Dhier (Nuclear Medicine Physician) , A. Lamarca (Medical Oncologist)
{"title":"Management of non-hepatic distant metastases in neuroendocrine neoplasms","authors":"C. Lacalle-González (Medical Oncologist) , A. Estrella Santos (Endocrinologist) , L.C. Landaeta Kancev (Nuclear Medicine Physician) , V.M. Castellano (Pathologist) , E. Macia Palafox (Cardiologist) , A. Paniagua Ruíz (Endocrinologist) , J. Luna Tirado (Radiation Oncologist) , B. Martínez-Amores (Medical Oncologist) , L. Martínez Dhier (Nuclear Medicine Physician) , A. Lamarca (Medical Oncologist)","doi":"10.1016/j.beem.2023.101784","DOIUrl":"10.1016/j.beem.2023.101784","url":null,"abstract":"<div><p><span>Neuroendocrine neoplasms represent an uncommon disease with an increasing incidence. Thanks to improvements in diagnostic and therapeutic methods, metastases previously considered uncommon, such as </span>bone metastases<span>, or even very rare, such as brain, orbital and cardiac metastases, are more frequently found in daily practice. Due to the great heterogeneity of these neoplasms, there is a lack of high-quality evidence on the management of patients with these types of metastases. The aim of this review is to provide the current state of the art, reviewing neuroendocrine neoplasm specific studies and useful information from other tumor types and to propose a treatment recommendation with algorithms to consider in daily clinical practice.</span></p></div>","PeriodicalId":8810,"journal":{"name":"Best practice & research. Clinical endocrinology & metabolism","volume":"37 5","pages":"Article 101784"},"PeriodicalIF":7.4,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9569430","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}