The Lancet Haematology最新文献

{"title":"Assessing authorship of clinical practice guidelines","authors":"Jeremy W Jacobs, Brian D Adkins, Deva Sharma, Allison P Wheeler, Laura D Stephens, Jennifer S Woo, Shazia S Khan, Garrett S Booth","doi":"10.1016/s2352-3026(23)00337-x","DOIUrl":"https://doi.org/10.1016/s2352-3026(23)00337-x","url":null,"abstract":"Abstract not available","PeriodicalId":501011,"journal":{"name":"The Lancet Haematology","volume":"46 9","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-11-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138455941","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":"Darbepoetin alfa to reduce transfusion episodes in infants with haemolytic disease of the fetus and newborn who are treated with intrauterine transfusions in the Netherlands: an open-label, single-centre, phase 2, randomised, controlled trial","authors":"Isabelle M C Ree, Masja de Haas, Nan van Geloven, Sandra E Juul, Derek de Winter, E J T Verweij, Dick Oepkes, Johanna G van der Bom, Enrico Lopriore","doi":"10.1016/s2352-3026(23)00285-5","DOIUrl":"https://doi.org/10.1016/s2352-3026(23)00285-5","url":null,"abstract":"<h3>Background</h3><p><span>Up to 88% of infants with haemolytic disease<span><span><span> of the fetus and newborn who are treated with </span>intrauterine transfusions<span> require erythrocyte transfusions after </span></span>birth. We aimed to investigate the effect of </span></span>darbepoetin alfa on the prevention of postnatal anaemia in infants with haemolytic disease of the fetus and newborn.</p><h3>Methods</h3><p><span>We conducted an open-label, single-centre, phase 2 randomised controlled trial<span> to evaluate the effect of darbepoetin alfa on the number of erythrocyte transfusions in infants with haemolytic disease of the fetus and newborn. All infants who were treated with intrauterine transfusion and born at 35 weeks of gestation or later at the Leiden University Medical Center, Leiden, Netherlands, were eligible for inclusion. Included infants were randomised by computer at birth to treatment with 10 μg/kg darbepoetin alfa subcutaneously once a week for 8 weeks or standard care (1:1 allocation, in varying blocks of four and six, with no stratification). Treating physicians and parents were not masked to treatment allocation, but the research team, data manager, and statistician were masked to treatment allocation during the process of data collection. The primary outcome was the number of erythrocyte transfusion episodes per infant from birth up to 3 months of life in the modified intention-to-treat population. This trial is registered with </span></span><span>ClinicalTrials.gov</span><svg aria-label=\"Opens in new window\" focusable=\"false\" height=\"8px\" viewbox=\"0 0 8 8\" width=\"8px\"><path d=\"M1.12949 2.1072V1H7V6.85795H5.89111V2.90281L0.784057 8L0 7.21635L5.11902 2.1072H1.12949Z\"></path></svg> (<span>NCT03104426</span><svg aria-label=\"Opens in new window\" focusable=\"false\" height=\"8px\" viewbox=\"0 0 8 8\" width=\"8px\"><path d=\"M1.12949 2.1072V1H7V6.85795H5.89111V2.90281L0.784057 8L0 7.21635L5.11902 2.1072H1.12949Z\"></path></svg>) and has been completed.</p><h3>Findings</h3><p>Between Oct 31, 2017, and April 31, 2022, we recruited 76 infants, of whom 44 (58%) were randomly assigned to a treatment group (20 [45%] were allocated to receive darbepoetin alfa and 24 [55%] were allocated to receive standard care). Follow-up lasted 3 months and one infant dropped out of the trial before commencement of treatment. A significant reduction in erythrocyte transfusion episodes was identified with darbepoetin alfa treatment compared with standard care (median 1·0 [IQR 1·0–2·0] transfusion episodes <em>vs</em> 2·0 [1·3–3·0] transfusion episodes; p=0·0082). No adverse events were reported and no infants died during the study.</p><h3>Interpretation</h3><p>Darbepoetin alfa reduced the transfusion episodes after intrauterine transfusion treatment for haemolytic disease of the fetus and newborn. Treatment with darbepoetin alfa or other types of erythropoietin should be considered as part of the postnatal treatment of severe haemolytic disease of the fe","PeriodicalId":501011,"journal":{"name":"The Lancet Haematology","volume":"49 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-11-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138455942","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":"CD19-directed CAR T cells as first salvage therapy for large B-cell lymphoma: towards a rational approach","authors":"Peter Dreger, Paolo Corradini, John G Gribben, Bertram Glass, Mats Jerkeman, Marie Jose Kersten, Franck Morschhauser, Alberto Mussetti, Andreas Viardot, Pier Luigi Zinzani, Anna Sureda","doi":"10.1016/s2352-3026(23)00307-1","DOIUrl":"https://doi.org/10.1016/s2352-3026(23)00307-1","url":null,"abstract":"<p>The approval of CD19-directed chimeric antigen receptor (CAR) T-cell therapies for the second-line treatment<span> of high-risk large B-cell lymphoma (LBCL) has greatly affected salvage algorithms for this condition, and such therapies could have the potential to improve the course of relapsed or refractory LBCL. In this Review, we provide guidance for a rational management approach to the use of commercial CD19-directed CAR T cells in the second-line treatment of LBCL, addressing crucial questions regarding eligible histologies; age, comorbidity, and tumour biology restrictions; the handling of very aggressive tumour behaviour; and holding and bridging therapies. The guidance was developed in a structured manner and, for each question, consists of a description of the clinical issue, a summary of the evidence, the rationale for a practical management approach, and recommendations. These recommendations could help to decide on the optimal management of patients with relapsed or refractory LBCL who are considered for second-line CAR T-cell treatment.</span></p>","PeriodicalId":501011,"journal":{"name":"The Lancet Haematology","volume":"48 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-11-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138455939","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":"Addition of danicopan to ravulizumab or eculizumab in patients with paroxysmal nocturnal haemoglobinuria and clinically significant extravascular haemolysis (ALPHA): a double-blind, randomised, phase 3 trial","authors":"Jong Wook Lee, Morag Griffin, Jin Seok Kim, Lily Wong Lee Lee, Caroline Piatek, Jun-ichi Nishimura, Cynthia Carrillo Infante, Deepak Jain, Peng Liu, Gleb Filippov, Flore Sicre de Fontbrune, Antonio Risitano, Austin G Kulasekararaj","doi":"10.1016/s2352-3026(23)00315-0","DOIUrl":"https://doi.org/10.1016/s2352-3026(23)00315-0","url":null,"abstract":"<h3>Background</h3><p><span><span>Symptoms of anaemia due to clinically significant extravascular haemolysis can affect patients with paroxysmal nocturnal haemoglobinuria (PNH) treated with C5 inhibitors (ravulizumab or eculizumab). The aim of this study was to assess the efficacy and safety of danicopan (ALXN2040), an investigational, first-in-class, oral </span>complement factor D inhibitor, as add-on therapy to </span>ravulizumab<span><span> or eculizumab </span>in patients with PNH and clinically significant extravascular haemolysis.</span></p><h3>Methods</h3><p><span>ALPHA is an ongoing, international, phase 3, randomised, double-blind, placebo-controlled trial evaluating danicopan as add-on therapy to ravulizumab or eculizumab. Eligible patients were adults (age ≥18 years) with PNH and clinically significant extravascular haemolysis (haemoglobin ≤9·5 g/dL; absolute reticulocyte count ≥120 × 10</span>⁹<span><span>/L) on ravulizumab or eculizumab for at least 6 months. Patients were randomly assigned (2:1) to danicopan or placebo added to ravulizumab or eculizumab for 12 weeks using an interactive response technology system. Randomisation was stratified based on transfusion history, haemoglobin, and patients enrolled from Japan. The initial oral danicopan dose was 150 mg three times a day; escalation to 200 mg three times a day was permitted based on clinical response. The infusion dose level of eculizumab (every 2 weeks) ranged from 900 mg to 1500 mg, and for ravulizumab (monthly or every 8 weeks) ranged from 3000 mg to 3600 mg. The primary endpoint was change in haemoglobin concentration from baseline to week 12. Here we present the protocol-prespecified interim analysis, planned when approximately 75% of participants were randomly assigned to </span>treatment and completed or discontinued at 12 weeks. This trial is registered with </span><span>ClinicalTrials.gov</span><svg aria-label=\"Opens in new window\" focusable=\"false\" height=\"8px\" viewbox=\"0 0 8 8\" width=\"8px\"><path d=\"M1.12949 2.1072V1H7V6.85795H5.89111V2.90281L0.784057 8L0 7.21635L5.11902 2.1072H1.12949Z\"></path></svg> (<span>NCT04469465</span><svg aria-label=\"Opens in new window\" focusable=\"false\" height=\"8px\" viewbox=\"0 0 8 8\" width=\"8px\"><path d=\"M1.12949 2.1072V1H7V6.85795H5.89111V2.90281L0.784057 8L0 7.21635L5.11902 2.1072H1.12949Z\"></path></svg>).</p><h3>Findings</h3><p><span><span>Individuals were randomly assigned between Dec 16, 2020, and Aug 29, 2022. At data cutoff (June 28, 2022), 73 individuals were randomly assigned, received treatment, and were analysed for safety (danicopan, n=49; placebo, n=24). The protocol-prespecified interim efficacy analysis set included the first 63 participants (danicopan, n=42; placebo, n=21). At week 12, danicopan plus ravulizumab or eculizumab increased haemoglobin versus placebo plus ravulizumab or eculizumab (least squares mean [LSM] change from baseline: danicopan, 2·94 g/dL [95% CI 2·52 to 3·36]; placebo, 0·50 g/dL [–0·13 to","PeriodicalId":501011,"journal":{"name":"The Lancet Haematology","volume":"48 6","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-11-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138455936","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":"Life after sickle cell disease, is it really uhuru?","authors":"Lydia H Pecker, Adeseye M Akinsete, C Patrick Carroll, Sophie Lanzkron, Kevin H M Kuo, Monica Hulbert, Elizabeth Stenger, Deepika S Darbari","doi":"10.1016/s2352-3026(23)00306-x","DOIUrl":"https://doi.org/10.1016/s2352-3026(23)00306-x","url":null,"abstract":"Abstract not available","PeriodicalId":501011,"journal":{"name":"The Lancet Haematology","volume":"48 5","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-11-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138455937","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":"Improving haematological care for adolescents","authors":"","doi":"10.1016/s2352-3026(23)00341-1","DOIUrl":"https://doi.org/10.1016/s2352-3026(23)00341-1","url":null,"abstract":"Abstract not available","PeriodicalId":501011,"journal":{"name":"The Lancet Haematology","volume":"48 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-11-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138455940","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":"Use of darbepoetin alfa in haemolytic disease of the fetus and newborn","authors":"Ravi M Patel, Robin Ohls","doi":"10.1016/s2352-3026(23)00316-2","DOIUrl":"https://doi.org/10.1016/s2352-3026(23)00316-2","url":null,"abstract":"Abstract not available","PeriodicalId":501011,"journal":{"name":"The Lancet Haematology","volume":"48 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-11-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138455938","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}