Hematology Reports最新文献

{"title":"Incidence, Risk Factors, and Outcomes of Thrombocytopenia in Older Medical Inpatients: A Prospective Cohort Study.","authors":"Ioanna Papakitsou, Andria Papazachariou, Theodosios D Filippatos, Petros Ioannou","doi":"10.3390/hematolrep16040076","DOIUrl":"10.3390/hematolrep16040076","url":null,"abstract":"<p><strong>Background: </strong>Thrombocytopenia, defined as a platelet count of less than 150 × 10⁹/L, is a frequent condition among hospitalized patients and presents unique challenges in diagnosis and management. Despite its commonality, data on incidence and related risk factors in medical inpatients remain limited, especially in older people.</p><p><strong>Methods: </strong>A 2-year prospective cohort study with a 3-year follow-up was conducted on inpatients aged ≥65 years admitted to a medical ward. Clinical data were collected, including demographics, comorbidities, laboratory results, and outcomes. Multivariate logistic regression analysis assessed risk factors associated with non-resolution of thrombocytopenia and mortality.</p><p><strong>Results: </strong>The study included 961 older inpatients with a mean age of 82 years. Thrombocytopenia occurred in 22.6% of the study population. The most common causes were infections (57.4%) and drug-induced thrombocytopenia (25.3%). The non-resolution of thrombocytopenia was noted in 59% of patients. In-hospital and 3-year mortality was significantly higher in this subgroup compared to the rest (24.5% vs. 12.7%, <i>p</i> = 0.015) and (72.4% vs. 59.8%, <i>p</i> = 0.04, respectively). In multivariate analysis, nadir platelet count and hematologic disease were independent factors associated with the non-resolution of thrombocytopenia. Furthermore, in individuals with thrombocytopenia, the administration of norepinephrine (<i>p</i> < 0.001) and a higher clinical frailty score (<i>p</i> < 0.001) were observed as independent mortality predictors.</p><p><strong>Conclusions: </strong>Thrombocytopenia in older medical inpatients is associated with poor prognosis, particularly in those with non-resolution thrombocytopenia. Early identification and targeted management may improve outcomes.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"804-814"},"PeriodicalIF":1.1,"publicationDate":"2024-12-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11675778/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142894029","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Incorporation of a Comorbidity Index in Treatment Decisions for Elderly AML Patients Can Lead to Better Disease Management-A Single-Center Experience.","authors":"Cristina Negotei, Iuliana Mitu, Silvana Angelescu, Florentina Gradinaru, Cristina Mambet, Oana Stanca, Mihai-Emilian Lapadat, Cristian Barta, Georgian Halcu, Carmen Saguna, Aurora Arghir, Mihaela Sorina Papuc, Andrei Turbatu, Nicoleta Mariana Berbec, Andrei Colita","doi":"10.3390/hematolrep16040074","DOIUrl":"10.3390/hematolrep16040074","url":null,"abstract":"<p><p><b>Introduction:</b> Acute myeloid leukemia (AML) is a form of cancer originating from precursor cells within the bone marrow. Elderly patients with acute leukemia require a personalized approach, considering age, performance status, and comorbidities, to determine suitability for intensive treatment. <b>Methods:</b> We studied the results of intense chemotherapy in 46 elderly, fit individuals with AML at a cancer center in Romania from January 2017 to December 2023. <b>Results:</b> The study involved a cohort of 46 patients, including 22 men and 24 women. The research indicated that 89.1% of the patients were diagnosed with de novo acute leukemia. Most patients had an ECOG score of 0-1, with one patient scoring ≥2. HCT-CI > 4 was found in 21 patients (45.7%), while CCI > 4 was present in 38 patients (82.6%). After the induction phase, 25 patients (54.3%) achieved complete remission (CR); the relapse rate was 56.8%. Upon completion of the study, nine individuals (19.6%) were still alive. The overall survival duration ranged from 0 to 33 months, with a median survival time of 8 months (CI 5.0-11.0). <b>Conclusions:</b> When considering treatment options for elderly patients, the Eastern Cooperative Oncology Group (ECOG) Performance Status, as well as comorbidity indices such as the Hematopoietic Cell Transplantation-Specific Comorbidity Index (HCT-CI) and the Charlson Comorbidity Index (CCI), have shown promising results in the literature, indicating their relevance in the decision-making process.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"781-794"},"PeriodicalIF":1.1,"publicationDate":"2024-12-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11727994/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142894031","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical Manifestations, Prognostic Factors, and Outcomes of Extranodal Natural Killer T-Cell Lymphoma: A Single-Center Experience in Thailand.","authors":"Wasinee Kaewboot, Lalita Norasetthada, Adisak Tantiworawit, Chatree Chai-Adisaksopha, Sasinee Hantrakool, Thanawat Rattanathammethee, Pokpong Piriyakhuntorn, Nonthakorn Hantrakun, Teerachat Punnachet, Ekarat Rattarittamrong","doi":"10.3390/hematolrep16040073","DOIUrl":"10.3390/hematolrep16040073","url":null,"abstract":"<p><p><b>Background/Objectives</b>: The primary objective of this study was to investigate clinical manifestations, time to diagnosis, and number of biopsies in patients with extranodal natural killer T-cell lymphoma (ENKTL). The secondary objectives were to determine response rates, survival outcomes, prognostic factor for overall survival (OS), and validation of the Prognostic Index of Natural Killer Lymphoma (PINK), Ann Arbor staging system (AASS), and the CA system. <b>Methods</b>: This retrospective study included data pertaining to patients with newly diagnosed ENKTL in Chiang-Mai University Hospital from 2004 to 2020. Comparisons between the areas under the receiver operating characteristic curve (AUC) of prognostic models (PINK, AASS, and CA system) were made. <b>Results</b>: Sixty patients were enrolled (n = 60) with a mean age of 49.1 ± 13.4 years. The most frequent symptom of ENKTL was nasal obstruction (66%). The median time to diagnosis was 22 days (ranging from 3 to 84 days), with 36.7% requiring more than one biopsy for diagnosis. Most patients presented with limited stage disease (75%). The median OS was 49 months. Factors associated with increased mortality were advanced stage, bone marrow involvement, gastrointestinal tract involvement, and receiving chemotherapy. Following prognostic model validation, the CA system model scored the highest level of accuracy (AUC 0.61), followed by AASS (AUC 0.58) and PINK (AUC 0.54). <b>Conclusions</b>: Patients with ENKTL commonly presented with nasal obstruction, with 36.7% requiring more than one biopsy for diagnosis. An advanced stage, bone marrow involvement, or gastrointestinal tract involvement were associated with poor OS. The CA system model has the highest level of accuracy for prognostic determination.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"769-780"},"PeriodicalIF":1.1,"publicationDate":"2024-11-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11727912/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142894024","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Role of 11C-Methionine PET Imaging for the Evaluation of Lymphomas: A Systematic Review.","authors":"Francesco Dondi, Maria Gazzilli, Gian Luca Viganò, Antonio Rosario Pisani, Cristina Ferrari, Giuseppe Rubini, Francesco Bertagna","doi":"10.3390/hematolrep16040072","DOIUrl":"10.3390/hematolrep16040072","url":null,"abstract":"<p><p><b>Background</b>: In the last years, different evidence has underlined a possible role for [11C]-methionine ([11C]MET) positron emission tomography (PET) imaging for the evaluation of lymphomas. The aim of this paper was, therefore, to review the available scientific literature focusing on this topic. <b>Methods</b>: A wide literature search of the PubMed/MEDLINE, Scopus and Cochrane Library databases was conducted in order to find relevant published articles investigating the role of [11C]MET in the assessment of lymphomas. <b>Results</b>: Eighteen studies were included in the systematic review and the main fields of application of this imaging modality were the evaluation of disease, therapy response assessment, prognostic evaluation and differential diagnosis with other pathological conditions. <b>Conclusion</b>: Even with heterogeneous evidence, a possible role for [11C]MET PET imaging in the assessment of lymphomas affecting both the whole body and the central nervous system was underlined. When compared to [18F]fluorodesoxyglucose ([18F]FDG) imaging, in general, similar results have been reported between the two modalities in these settings.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"752-768"},"PeriodicalIF":1.1,"publicationDate":"2024-11-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11675220/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142893940","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Serum Vitamin D in Children with Hemophilia A and Its Association with Joint Health and Quality of Life.","authors":"Aida M S Salem, Takwa Mohamed AbdEltwwab, Hanan Hosni Moawad, Marwa O Elgendy, Reham S Al-Fakharany, Ahmed Khames, Mohamed Hussein Meabed","doi":"10.3390/hematolrep16040071","DOIUrl":"10.3390/hematolrep16040071","url":null,"abstract":"<p><p><b>Background/Objectives</b>: Hemophilia A is an X-linked recessive illness produced by a deficiency of coagulation factor VIII. This study aimed to evaluate serum vitamin D in hemophilic pediatric patients and its correlation with joint health and quality of life. <b>Methods</b>: This case-control study was performed on ninety children under the age of 18 years old and separated into two groups: study group of 45 children with hemophilia A and control group of 45 healthy children at an outpatient pediatric hematology clinic at the Beni-Suef University hospitals. <b>Results</b>: Serum vitamin D levels were significantly lower in hemophilia A patients than in controls (<i>p</i> < 0.001). The level of serum vitamin D was deficient in 38 (84.4%), insufficient in 4 (8.8%) and sufficient in 3 (6.6%) in the study group while deficient in 8 (17.7%), insufficient in 16 (35.5%) and sufficient in 21 (46.6%) in the control group. Total hemophilia joint health score (HJHS) had a significant negative correlation with serum total calcium (R = -0.31, <i>p</i> = 0.038) and serum vitamin D level (R = -0.974, <i>p</i> < 0.001) while also positively correlated with alkaline phosphatase (R = 0.834, <i>p</i> < 0.001). A quality-of-life index that is specific to total hemophilia (Haemo-Qol/Haem-A-QoL) had a significant positive correlation with total hemophilia joint health score (HJHS) (R = 0.934, <i>p</i> < 0.001) and negatively correlated with serum vitamin D level (R = -0.924, <i>p</i>-value lower than 0.001), alkaline phosphatase (R = 0.842, <i>p</i> < 0.001), and severity of hemophilia (R = 0.67, <i>p</i> < 0.001). <b>Conclusions</b>: patients with hemophilia A had lower vitamin D levels than healthy controls. The severity of vitamin D deficiency is related positively to (HJHS) hemophilia and quality of life hemophilia cases according to Haemo-QoL.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"742-751"},"PeriodicalIF":1.1,"publicationDate":"2024-11-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11727777/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142894032","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Mesenchymal Stem Cells and Reticulated Platelets: New Horizons in Multiple Myeloma.","authors":"Cristian Alejandro Mera Azaín, Johan Leandro Vargas Pasquel, Sandra Milena Quijano Gómez, Viviana Marcela Rodríguez-Pardo","doi":"10.3390/hematolrep16040070","DOIUrl":"10.3390/hematolrep16040070","url":null,"abstract":"<p><p>Multiple myeloma (MM) is a malignant plasma cell disorder characterized by the accumulation of abnormal plasma cells in the bone marrow. Mesenchymal stem cells (MSCs) and reticulated platelets (RPs) have been implicated in the pathogenesis of MM. This narrative review aims to explore the role of MSCs and RPs in the pathophysiology of MM, particularly their clinical use as possible variables of prognostic value in this hematologic neoplasia. The interaction between MSCs and MM cells within the bone marrow microenvironment supports MM cell survival, proliferation, and drug resistance. MSCs contribute to the development and maintenance of MM through the secretion of various factors, including cytokines, chemokines, and growth factors. Moreover, RPs, young and highly reactive platelets, have been implicated in promoting angiogenesis, tumor growth, and metastasis in MM. Several studies show that cells such as MSCs and platelets participate actively in the biology of the disease. Still, in clinical practice, they are not considered part of evaluating affected patients. In this review, we explore the possibility of including the evaluation of MSCs and PRs in the clinical practice for patients with MM as part of the strategies to improve the outcomes of this disease.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"732-741"},"PeriodicalIF":1.1,"publicationDate":"2024-11-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11627159/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142709534","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Case of B-Cell Lymphoblastic Lymphoma Presenting with an Isolated Epidural Mass Treated Successfully with Radiotherapy Followed by United Kingdom Acute Lymphoblastic Leukemia (UKALL) Chemotherapy Protocol.","authors":"Musa Fares Alzahrani","doi":"10.3390/hematolrep16040069","DOIUrl":"10.3390/hematolrep16040069","url":null,"abstract":"<p><strong>Background: </strong>B-cell lymphoblastic lymphoma (B-LBL) is an aggressive type of non-Hodgkin lymphoma that usually involves lymph nodes, skin and soft tissue. Bone marrow and peripheral blood are normally spared from involvement in the disease. B-LBL typically forms solid masses that have similar pathologic and immunophenotypic features to their liquid counterpart, B-cell acute lymphoblastic leukemia (B-ALL). The presentation of B-LBL with a solitary epidural mass at the cervical spine is very rare and the optimal treatment of such cases is unknown. Most of the literature on the management of B-LBL comes from small case series, pediatric patients, or as part of retrospective data that combine B-LBL with B-ALL cases.</p><p><strong>Case presentation: </strong>The case presented herein is a unique presentation that was treated using three modalities, namely surgical resection, radiotherapy and consolidation with systemic chemotherapy, adopted from the United Kingdom acute lymphoblastic leukemia (UKALL14) protocol.</p><p><strong>Conclusions: </strong>The patient attained complete remission following the planned treatment and is still in remission for more than four and half years from the time of his initial diagnosis.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"724-731"},"PeriodicalIF":1.1,"publicationDate":"2024-11-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11586985/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142709201","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Efficacy of Anti-CD38 Monoclonal Antibodies for Relapsed or Refractory Multiple Myeloma in Stem Cell Transplant-Ineligible Patients Aged over 65 Years: A Propensity Score-Matched Study.","authors":"Satoshi Yamasaki, Michitoshi Hashiguchi, Nao Yoshida-Sakai, Hiroto Jojima, Koichi Osaki, Takashi Okamura, Yutaka Imamura","doi":"10.3390/hematolrep16040068","DOIUrl":"10.3390/hematolrep16040068","url":null,"abstract":"<p><strong>Background: </strong>The development of newer agents, including anti-CD38 monoclonal antibodies (mAbs), has significantly improved overall survival (OS) in patients with relapsed or refractory multiple myeloma (RRMM). However, the treatment of older patients with RRMM who are transplant-ineligible remains challenging.</p><p><strong>Methods: </strong>We retrospectively evaluated OS in 78 transplant-ineligible patients with RRMM who were aged ≥ 65 years and treated at our institution between February 2012 and November 2023.</p><p><strong>Results: </strong>Unadjusted OS was significantly longer in the anti-CD38 mAb-exposed group (i.e., those previously treated with daratumumab and receiving isatuximab plus pomalidomide and low-dose dexamethasone because of disease progression during treatment with daratumumab [<i>n</i> = 6], daratumumab plus pomalidomide and low-dose dexamethasone [<i>n</i> = 9], or isatuximab plus pomalidomide and low-dose dexamethasone without daratumumab-exposure [<i>n</i> = 14]) than in the anti-CD38 mAb-naïve group (no exposure to daratumumab or isatuximab [<i>n</i> = 49]) (<i>p</i> < 0.001). To address potential confounder factors associated with use or nonuse of anti-CD38 mAbs, we performed propensity score matching (PSM) using age, sex, performance status, and Geriatric 8 and Instrumental Activities of Daily Living scores. PSM identified 14 subjects from the anti-CD38 mAb-exposed group with baseline characteristics similar to those of 14 subjects from the anti-CD38 mAb-naïve group. After PSM, the adjusted OS was significantly longer in the anti-CD38 mAb-exposed group than in the anti-CD38 mAb-naïve group (<i>p</i> < 0.001).</p><p><strong>Conclusion: </strong>These findings provide insights into the optimal use of anti-CD38 mAbs in patients with RRMM who are transplant-ineligible and aged ≥65 years and on candidates who are appropriate for novel approaches, such as chimeric antigen receptor T-cell or bispecific T-cell engager therapy.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"714-723"},"PeriodicalIF":1.1,"publicationDate":"2024-11-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11587159/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142709529","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Treatment Strategies Used in Treating Myelofibrosis: State of the Art.","authors":"Massimo Martino, Martina Pitea, Annalisa Sgarlata, Ilaria Maria Delfino, Francesca Cogliandro, Anna Scopelliti, Violetta Marafioti, Simona Polimeni, Gaetana Porto, Giorgia Policastro, Giovanna Utano, Maria Pellicano, Giovanni Leanza, Caterina Alati","doi":"10.3390/hematolrep16040067","DOIUrl":"10.3390/hematolrep16040067","url":null,"abstract":"<p><strong>Background: </strong>Current drug therapy for myelofibrosis does not alter the natural course of the disease or prolong survival, and allogeneic stem cell transplantation is the only curative treatment modality. For over a decade, the Janus kinase (JAK) inhibitor ruxolitinib has been the standard of care. More recently, newer-generation JAK inhibitors have joined the ranks of accepted treatment options.</p><p><strong>Objectives: </strong>The primary goal of treatment is to reduce spleen size and minimize disease-related symptoms. Prognostic scoring systems are used to designate patients as being at lower or higher risk. For transplant-eligible patients, transplant is offered to those with a bridge of a JAK inhibitor; patients who are not eligible for transplant are usually offered long-term therapy with a JAK inhibitor. Limited disease-modifying activity, dose-limiting cytopenias, and other adverse effects have contributed to discontinuation of JAK inhibitor treatment.</p><p><strong>Conclusions: </strong>Novel JAK inhibitors and combination approaches are currently being explored to overcome these shortcomings. Further research will be essential to establish optimal therapeutic approaches in first-line and subsequent treatments.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"698-713"},"PeriodicalIF":1.1,"publicationDate":"2024-10-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11587016/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142709722","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-World Study of US Adults with Paroxysmal Nocturnal Hemoglobinuria Treated with Pegcetacoplan.","authors":"Brian Mulherin, Apeksha Shenoy, Lily Arnett, Weiqi Jiao, Joseph Guarinoni, Sujata Sarda, Jinny Min, David Dingli","doi":"10.3390/hematolrep16040065","DOIUrl":"10.3390/hematolrep16040065","url":null,"abstract":"<p><p><b>Background</b>: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, life-threatening disease characterized by complement-mediated hemolysis. OPERA is the first US longitudinal real-world study on C3 inhibitor therapy, known as pegcetacoplan. <b>Methods</b>: OPERA enrolled US patients with PNH, age ≥18, who were prescribed pegcetacoplan, and data were collected from routine care. Hemoglobin was reported by patients during regular follow-up (censored from transfusions). The Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue (0-52 score) and Patient-Reported Outcomes Measurement Information System scale for Cognitive Function Abilities (PROMIS-CF; 23.27-67.09 t-score) were completed electronically (low score = negative outcome). Patients self-reported incidence of healthcare resource utilization (HCRU). <b>Results</b>: By January 2024, 70 patients (mean age 44.6 years; 57.1% female) reported up to 9 months of pegcetacoplan treatment, with a median [IQR] follow-up of 6.6 [3.8] months. The latest reported hemoglobin levels improved by a mean (SD) of 2.6 (1.9) g/dL from baseline. At 3, 6 and 9 months, patients reported clinically meaningful improvements (≥5 points) in FACIT-F (53.3-69.0%) and (≥2 points) PROMIS-CF (46.7-55.2%). Patients reported a <10% incidence rate per person month of all HCRU events. <b>Conclusions</b>: This first longitudinal real-world US study indicates a positive trend in Hb, fatigue, and cognition with limited HCRU during pegcetacoplan treatment in adults with PNH.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"669-681"},"PeriodicalIF":1.1,"publicationDate":"2024-10-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11587045/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142709629","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}