Hematology, transfusion and cell therapy最新文献

{"title":"Benefits of BV-AVD (Brentuximab vedotin, doxorubicin, vinblastine, and dacarbazine) versus ABVD (doxorubicin hydrochloride, bleomycin sulfate, vinblastine sulfate, dacarbazine) in patients with advanced-stage Hodgkin's lymphoma: an analysis of the ECHELON 1 trial by the GATLA group using the Delphi Method.","authors":"Astrid Pavlovsky, Juan Ignacio Garcia Altuve, Amalia Cerutti, Lorena Fiad, Nicolás Kurgansky, Fernando Warley, Florencia Negri Aranguren","doi":"10.1016/j.htct.2024.07.007","DOIUrl":"10.1016/j.htct.2024.07.007","url":null,"abstract":"<p><strong>Introduction: </strong>The BV-AVD (Brentuximab vedotin, doxorubicin, vinblastine, and dacarbazine) combination for first-line treatment of advanced stage Hodgkin's lymphoma has been approved by regulatory authorities and included in international guidelines. However, several factors influence its incorporation as standard of care.</p><p><strong>Materials and methods: </strong>A group of experts from different institutions was identified and, using the Delphi method, an analysis of the results of the ECHELON 1 trial for the indication of BV-AVD over ABVD (doxorubicin hydrochloride, bleomycin sulfate, vinblastine sulfate, dacarbazine) in patients with Hodgkin's lymphoma Stages III and IV in Argentina was done. The clinical and academic experience of the authors and the context of the Argentine healthcare system were considered.</p><p><strong>Results and discussion: </strong>Seven statements on general aspects of the management of Hodgkin's lymphoma and nine on specific aspects related to the use of BV-AVD over ABVD reached a consensus of agreement. There was a strong expert consensus in favor of indicating BV-AVD in the presence of extranodal disease or pulmonary disease. Moderate to severe neuropathy, pregnancy and drug allergy were considered absolute contraindications to prescribe BV.</p><p><strong>Conclusions: </strong>The authors agreed that BV-AVD could be considered a new treatment option in high-risk patients. However health system-dependent factors (such as high cost, lack of availability, reimbursement difficulties, irregular delivery, and issues with granulocyte-colony stimulating factor availability) could pose limitations for this prescription. While awaiting new data from clinical trials and real-world studies, these recommendations can represent a useful tool for hematologists in different parts of the world.</p>","PeriodicalId":94026,"journal":{"name":"Hematology, transfusion and cell therapy","volume":" ","pages":"S250-S256"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142376479","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":"Evaluation of blood cell count using an automatic hematology analyzer to optimize collection of peripheral blood progenitor cells by leukapheresis.","authors":"Paula Renata Machado Passos Pederzoli, Karen de Lima Prata, Nathália Gomide Cruz, Pedro Victorio de Almeida Marzano, Maurício Colombini Martins, Luciana de Almeida Costa, Roberta Kelly de Andrade, Marcia Regina Issa Salomão Libânio, Brian Custer, André Rolim Belisário","doi":"10.1016/j.htct.2024.04.117","DOIUrl":"10.1016/j.htct.2024.04.117","url":null,"abstract":"<p><strong>Background: </strong>Autologous stem cell transplantation is a treatment modality for several diseases. Prediction of successful mobilization may be useful to optimize hematopoietic stem cell collection.</p><p><strong>Study design and methods: </strong>This was a retrospective study with data from transplantation candidates between September 2015 and December 2021 being analyzed. The medical record of each patient was reviewed to mine mobilization information. The laboratory data analyzed were CD34⁺ cell enumeration and pre-collection peripheral blood cell count. The primary outcome, good mobilization, was defined as a CD34⁺ cell count ≥20/μL.</p><p><strong>Results: </strong>This study included 807 patients. Increased patient weight, low mean corpuscular volume, high nucleated red blood cells, peripheral blood mononuclear cell and immature granulocyte counts were significantly associated with good mobilization. In addition, patients diagnosed with multiple myeloma were two times more likely to be good mobilizers than patients with lymphoma. The model was applied to a validation set to identify patients who underwent apheresis (CD34⁺ cell count ≥10 µL), resulting in a sensitivity of 69 %, a specificity of 95 %, positive predictive value of 98 %, and a negative predictive value of 50 %.</p><p><strong>Conclusion: </strong>Success in mobilization was greater in patients who underwent the first mobilization cycle and who had a diagnosis of multiple myeloma. Furthermore, higher body weight, and nucleated red blood cells, immature granulocytes and mononuclear cell counts, as well as low mean corpuscular volumes, were associated with successful mobilization.</p>","PeriodicalId":94026,"journal":{"name":"Hematology, transfusion and cell therapy","volume":" ","pages":"S150-S156"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140893087","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":"Application of mass cytometry to characterize hematopoietic stem cells in apheresis products of patients with hematological malignancies.","authors":"Carlos Agustin Villegas-Valverde, Antonio Alfonso Bencomo-Hernandez, Yandy M Castillo-Aleman, Yendry Ventura-Carmenate, Imilla Casado-Hernandez, Rene Antonio Rivero-Jimenez","doi":"10.1016/j.htct.2023.10.008","DOIUrl":"10.1016/j.htct.2023.10.008","url":null,"abstract":"<p><strong>Introduction: </strong>Hematopoietic stem cell transplantation (HSCT) is a widely used therapy, but its success largely depends on the number and quality of stem cells collected. Current evidence shows the complexity of the hematopoietic system, which implies that, in the quality assurance of the apheresis product, the hematopoietic stem cells are adequately characterized and quantified, in which mass cytometry (MC) can provide its advantages in high-dimensional analysis.</p><p><strong>Objective: </strong>This research aimed to characterize and enumerate CD45^dim/CD34⁺ stem cells using the MC in apheresis product yields from patients with chronic lymphoid malignant diseases undergoing autologous transplantation at the Abu Dhabi Stem Cells Center.</p><p><strong>Methods: </strong>An analytical and cross-sectional study was performed on 31 apheresis products from 15 patients diagnosed with multiple myeloma (n = 9) and non-Hodgkin lymphomas (n = 6) eligible for HSCT. The MC was employed using the MaxPar Kit for stem cell immunophenotyping. The analysis was performed manually in the Kaluza and unsupervised by machine learning in Cytobank Premium.</p><p><strong>Results: </strong>An excellent agreement was found between mass and flow cytometry for the relative and absolute counts of CD45^dim/CD34⁺ cells (Bland-Altman bias: -0.029 and -64, respectively), seven subpopulations were phenotyped and no lineage bias was detected for any of the methods used in the pool of collected cells. A CD34+/CD38+/CD138+ population was seen in the analyses performed on four patients with multiple myeloma.</p><p><strong>Conclusions: </strong>The MC helps to characterize subpopulations of stem cells in apheresis products. It also allows cell quantification by double platform. Unsupervised analysis allows results completion and validation of the manual strategy. The proposed methodology can be extended to apheresis products for purposes other than HSCT.</p>","PeriodicalId":94026,"journal":{"name":"Hematology, transfusion and cell therapy","volume":" ","pages":"S59-S70"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139099423","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":"Familial acute myeloid leukemia due to a novel germline CEBPA pathogenic variant - a case report.","authors":"Flavia Gava, Luiz Fernando Bazzo Catto, Elvis Valera, Maristella Bergamo Francisco Dos Reis, Maria Carolina Tostes Pintão, Maria de Lourdes Chauffaille, Flavia Sacilotto Donaires Ramos, Carlos Alberto Scrideli, Lorena Lobo Figueiredo Pontes","doi":"10.1016/j.htct.2024.07.004","DOIUrl":"10.1016/j.htct.2024.07.004","url":null,"abstract":"","PeriodicalId":94026,"journal":{"name":"Hematology, transfusion and cell therapy","volume":" ","pages":"S417-S420"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142304628","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":"Autologous stem cell transplantation in patients older than 65 years with multiple myeloma: a real-world study.","authors":"Cristian Maximiliano Seehaus, Natalia Schutz, Erika Brulc, Gonzalo Ferini, Jorge Arbelbide, Dorotea Fantl, Ana Lisa Basquiera","doi":"10.1016/j.htct.2023.07.012","DOIUrl":"10.1016/j.htct.2023.07.012","url":null,"abstract":"<p><strong>Introduction: </strong>The treatment of elderly multiple myeloma (MM) patients with autologous stem cell transplantation (ASCT) is a controversial procedure. Most clinical trials evaluating the safety and efficacy of ASCT have primarily included patients younger than 65 years.</p><p><strong>Design and methods: </strong>This was a retrospective analysis of patients with MM who underwent ASCT between 2008 and 2018. Patients at or over 65 years were compared with patients under 65 years. We analyzed treatment-related mortality (TRM), response rate, progression-free survival (PFS) and overall survival (OS).</p><p><strong>Results: </strong>Two hundred and twenty-one patients were included: 50 patients at or over 65 years, (median age 68 years), including 7 patients over 70 years and 151 patients under 65 years, (median age 57 years). No differences were found in the neutrophil and platelet engraftment, median days of hospitalization and life support requirement during the hospitalization period for the ASCT. No statistically significant differences were found in the incidence of TRM between both groups at 100 days post-transplant (2% vs. 2.9%, p = 0.322). The ASCT improved complete response and stringent complete response rates (44% vs. 37%, p < 0.001). Survival was not modified by age: after a median follow-up of 53 months, the estimated PFS rates at three years were 63% and 60% (p = 0.88) and the OS rates at five years were 75% and 74% (p = 0.72), respectively.</p><p><strong>Conclusions: </strong>Our data suggest that the ASCT is feasible in selected elderly patients with MM over 65 years of age, achieving response and survival rates similar to those of younger patients.</p>","PeriodicalId":94026,"journal":{"name":"Hematology, transfusion and cell therapy","volume":" ","pages":"S13-S20"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41173007","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":"Quantitative detection of T315I mutations of BCR::ABL1 using digital droplet polymerase chain reaction.","authors":"Huijun Mu, Jian Zou, Haiping Zhang","doi":"10.1016/j.htct.2023.12.007","DOIUrl":"10.1016/j.htct.2023.12.007","url":null,"abstract":"<p><strong>Background: </strong>T315I mutations of the BCR::ABL1 gene lead to resistance to tyrosine kinase inhibitors (TKIs). This study evaluated the performance of digital droplet polymerase chain reaction (ddPCR) in quantifying T315I mutations and their frequency in Philadelphia chromosome (Ph) positive hematological patients.</p><p><strong>Methods: </strong>The course of disease and BCR::ABL1 fusion transcripts (e13a2, e14a2 and e1a2) were retrospectively reviewed in 21 patients with acute lymphoblastic leukemia (ALL) and 85 patients with chronic myeloid leukemia (CML). T315I mutation analysis was carried out using ddPCR and the limit of detection was assessed using mutant T315I DNA at varying variant allele fractions.</p><p><strong>Results: </strong>T315I mutations were found in two ALL patients and one CML patient without remission in molecular biology and with mutation burdens of 29.20%, 40.85%, and 3.00%, respectively. The mutation burden of ALL patients was higher than that of CML patients, but there was no significant difference between the two (p-value = 0.0536). The test's limit of detection was 0.02% with a correlation coefficient greater than 0.99 between the expected and actual detection abundances.</p><p><strong>Conclusion: </strong>T315I mutations have a high incidence in Ph-positive ALL patients even if the course of disease is short. In molecular biology, T315I mutation detection is indicated for CML patients not in remission.</p>","PeriodicalId":94026,"journal":{"name":"Hematology, transfusion and cell therapy","volume":" ","pages":"S79-S85"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139934768","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":"Patient-reported outcomes of treatment and adverse effects following acute lymphoblastic leukemia: a low- and middle-income country cross-sectional study.","authors":"Eduardo Cerello Chapchap, Nina Melo, Denise Martins, Maria Lucia Lee, Nelson Hamerschlak","doi":"10.1016/j.htct.2024.05.006","DOIUrl":"10.1016/j.htct.2024.05.006","url":null,"abstract":"<p><strong>Introduction: </strong>The scenario of adult patients with acute lymphoblastic leukemia treated in Brazil has not been well described yet.</p><p><strong>Methods: </strong>Four hundred patients diagnosed with acute lymphoblastic leukemia from 1981 to 2019, registered in the Brazilian lymphoma and leukemia association (ABRALE) or their caregivers were interviewed by telephone to evaluate patient-reported perceptions of diagnosis, treatment and adverse effects.</p><p><strong>Results: </strong>Overall, 203 were male with a mean age of 15.7 years and median follow-up of 6.2 years. Main presenting symptoms were fever (39 %), bleeding/ecchymosis (38 %), intense fatigue (30 %), and musculoskeletal pain (28 %). The proportion of patients diagnosed within one week of symptoms onset differed between public (17.9 %) and private healthcare (31.1 %; p-value = 0.019). Additionally, diagnostic difficulties were higher in public care: 35 % versus 22.6 % (p-value = 0.034). Only 36 patients were able to report their treatment protocols; from a list of eight reported protocols, the most common were the Brazilian Childhood Cooperative Group for Treatment of Acute Lymphoblastic Leukemia in Children (GBTLI - 10/27.8 %) and Berlin-Frankfurt-Münster (BFM - 8/22.2 %). Seventy patients (17.5 %) required treatment modification, 37.1 % due to severe adverse effects; 21.7 % received short treatment duration (≤6 months) and 16 % proceeded to allogeneic hematopoietic stem cell transplantation with 17/64 (27 %) reporting difficulties in this step, characterized as >3 months delay. Indication for transplantation was related to minimal residual disease and cranial radiotherapy; 41.7 % reported treatment-related adverse effects (range: 1-6), in particular: mood disorders (26.3 %), neurologic deficit (13.8 %), cognitive/memory impairment (12 %), and lung disease (15 %). Risk factors for adverse effects were age, indication of transplantation and living in a large city. Treatment disparities such as diagnostic and transplantation delays remain challenges in these patients.</p><p><strong>Conclusions: </strong>Urgent interventions are needed to optimize healthcare and reduce adverse effects, especially in adolescent and young adult patients.</p>","PeriodicalId":94026,"journal":{"name":"Hematology, transfusion and cell therapy","volume":" ","pages":"S182-S192"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141918359","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":"Fluvastatin suppresses hemin-induced cell death, reactive oxygen species generation, and elevated labile iron pool.","authors":"Shion Imoto, Katsuyasu Saigo, Mari Kono, Ayako Ohbuchi, Tohru Sawamura, Yuji Mizokoshi, Takashi Suzuki","doi":"10.1016/j.htct.2024.09.2480","DOIUrl":"10.1016/j.htct.2024.09.2480","url":null,"abstract":"<p><strong>Background: </strong>In transfusion-related iron overload, macrophage/reticuloendothelial cells are the first site of haem-derived iron accumulation. The prevention of haem-induced cytotoxicity in macrophages may represent a target for iron overload treatment. Deferasirox, an oral iron chelator, has been used to treat transfusion-related iron overload however, low adherence to the therapy is an issue. Statins, which are widely used for the prevention of atherosclerotic cardiovascular diseases, also have anti-oxidative and anti-inflammatory effects independent of their lipid lowering ones. Whether statins can suppress hemin-induced cytotoxicity and enhance the cytoprotective effects of deferasirox are important considerations to improve transfusion-related iron overload treatment. This study also evaluated the effects of eltrombopag, a thrombopoietin receptor agonist.</p><p><strong>Materials and methods: </strong>Human monocytic THP-1 cells were pretreated with statins, deferasirox, and/or eltrombopag, followed by treatment with hemin. Cell viability, reactive oxygen species generation, and the intracellular labile iron pool were measured using flow cytometry.</p><p><strong>Results: </strong>Fluvastatin and another four statins suppressed hemin-induced cell death, reactive oxygen species generation, and increases in the labile iron pool. Moreover, fluvastatin enhanced the suppressive effect of deferasirox on hemin-induced cell death. The effects of eltrombopag were similar to those of the statins.</p><p><strong>Conclusion: </strong>The safety of statins is well established. When used in combination with fluvastatin or other statins, the suppressive effects of deferasirox on hemin-induced cytotoxicity in THP-1 cells were amplified. Further research is necessary to see whether statins will act in the same way in vivo or in human primary monocytes/macrophages.</p>","PeriodicalId":94026,"journal":{"name":"Hematology, transfusion and cell therapy","volume":" ","pages":"S284-S290"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142635097","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":"Epidemiologic profile of hemoglobinopathies in Benin.","authors":"Selma Gomez, Adjile Edjide Roukiyath Amoussa, Edwige Dedjinou, Manasse Kakpo, Pélagie Gbédji, Nouhoum Amossou Soulé, Bernice Quenum","doi":"10.1016/j.htct.2024.07.008","DOIUrl":"10.1016/j.htct.2024.07.008","url":null,"abstract":"<p><strong>Background: </strong>Sickle cell disease is the most common inherited blood disorder in the world with the birth of approximately 300,000 newborns screened each year. In 2009, the World Health Organization ranked the fight against sickle cell disease among the priorities for the Africa regions. The best way to prevent this incurable disease remains, on one hand systematic screening at birth, and on the other the proscription of risky union between heterozygous subjects.</p><p><strong>Aim: </strong>The aim of this study was to analyze the epidemiological profile of sickle cell disease and other hemoglobinopathies in Benin and determine more up-to-date prevalence rates of the disease within the population.</p><p><strong>Methods: </strong>The hemoglobin profiles of 2910 study participants were determined by quantitative electrophoresis. Samples with abnormal hemoglobin results were subjected to a complete blood count.</p><p><strong>Results: </strong>Our study population was balanced between males (1528) and females (1382) with a sex ratio of 1.1. The mean age ranged from eight years in the pediatric group to 26 years in adults. The hemoglobin electrophoresis profiles found were as follows: 59.7 % Hb AA (normal), 21.7 % Hb AS, 10.2 % Hb AC, 3.1 % Hb SS, 3.7 % Hb SC, and 1.6 % of the rare phenotypes (Hb AD, Hb AE, Hb AF, Hb A/β-thal, Hb SD, Hb SF, Hb CC and Hb C/β-thal). Participants with abnormal hemoglobin presented a normochromic normocytic anemia. A total of 356 (12 %) people knew their profile compared to 2554 (88 %) who did not.</p><p><strong>Conclusion: </strong>The high prevalence of hemoglobinopathies found in this study highlights in importance of screening in the Benin population.</p>","PeriodicalId":94026,"journal":{"name":"Hematology, transfusion and cell therapy","volume":" ","pages":"S257-S262"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142484128","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":"Comprehensive health status and health-related quality of life of children at diagnosis of high-risk neuroblastoma: a multicentric pilot study.","authors":"Karina Viani, William Furlong, Vicente Odone Filho, Mariana Dos Santos Murra, Juliana Moura Nabarrete, Elena Ladas, Ronald Duncan Barr","doi":"10.1016/j.htct.2024.05.014","DOIUrl":"10.1016/j.htct.2024.05.014","url":null,"abstract":"<p><strong>Background: </strong>Neuroblastomas account for 8-10 % of all cancer diagnoses among children. Most patients present with advanced, high-risk disease and 90 % are less than five years old. The burden of morbidity and mortality is high and is quantifiable by measures of health-related quality of life (HRQL). Measuring quality of life in under five-year-old children is a particular challenge that has been met with the development of the Health Utilities Pre-School (HuPS) instrument. Quality of life studies in children with cancer are scarce in low- and middle-income countries and are usually conducted at a single center, thus limiting any conclusions drawn. This pilot study aimed to assess the health-related quality of life of children at the time of diagnosis of high-risk neuroblastomas.</p><p><strong>Method: </strong>This prospective cross-sectional multicentric study assessed the quality of life of children with high-risk neuroblastoma. The Health Utilities Pre-School instrument was applied to under five-year-olds, and the related Health Utilities Index Mark 3 instrument to over five-year olds.</p><p><strong>Main results: </strong>Eleven patients participated in this study. There was a high burden of morbidity at diagnosis, often equating to severe disability, indicative of states of health with scores worse than being dead in two under five-year-old children.</p><p><strong>Conclusion: </strong>The results of the current study will help to set research priorities for subsequent investigations and provide a basis to improve supportive care for children with high-risk neuroblastoma.</p>","PeriodicalId":94026,"journal":{"name":"Hematology, transfusion and cell therapy","volume":" ","pages":"S228-S233"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142304627","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}