{"title":"An instrument of screening criteria to assess individualized high-risk sexual behavior to be applied in the Brazilian population: translation and cross-cultural adaptation.","authors":"Miriane Lucindo Zucoloto, Guilherme Galdino, Edson Zangiacomi Martinez","doi":"10.1016/j.htct.2024.06.009","DOIUrl":"https://doi.org/10.1016/j.htct.2024.06.009","url":null,"abstract":"<p><strong>Background: </strong>Alternative approaches have been proposed to ensure a safe and equitable screening process for blood donation that treats all people equally, regardless of gender identity or sexual orientation. The terms 'neutral approach' and 'individualized risk assessment' have been used to describe this goal. To facilitate research and implementation of these concepts in blood donation contexts and health services in Brazil, we propose a Portuguese version of the 'for the assessment of individualized risk screening criteria' (FAIR) screening criteria.</p><p><strong>Methods: </strong>The FAIR screening criteria are 12 questions that assess sex, sexuality, ethnicity, and the extent to which participants engaged in each targeted sexual behavior. The aim of FAIR is to reduce error while increasing reliable and accurate reporting of sexual behaviors associated with both objective and subjective estimates of infection risk. The FAIR screening criteria were translated and cross-culturally adapted using a systematic approach with standardized procedures appropriate for adapting instruments that track behaviors.</p><p><strong>Results: </strong>A version that is appropriate for use with the Brazilian population was produced employing the following steps: expert translations, harmonization, consensus version, expert back-translation, revision, panel of experts, cognitive interviewing, and finalization.</p><p><strong>Conclusion: </strong>The Portuguese version of FAIR was proposed, and because of its straightforward, simple language and focus on specific and frequent behaviors in some populations, it has the potential to be used in a variety of contexts involving the screening of high-risk sexual behavior in Brazil.</p>","PeriodicalId":94026,"journal":{"name":"Hematology, transfusion and cell therapy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-09-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142304623","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":"Improvement of blood transfusion safety using the chemiluminescence technique for viral marker screening of blood donors in sub Saharan Africa.","authors":"Macoura Gadji, Aissata Ba, Youssou Bamar Gueye, Alioune Badara Senghor, Tandakha Ndiaye Dieye, Saliou Diop","doi":"10.1016/j.htct.2024.04.120","DOIUrl":"https://doi.org/10.1016/j.htct.2024.04.120","url":null,"abstract":"<p><strong>Introduction: </strong>Sub-Saharan Africa struggles continuously with insufficient resources and inadequate infrastructure that hinder the establishment of a safer blood supply despite improvements in transfusion safety over recent decades. This study aimed to evaluate the impact of the chemiluminescence technique in combination with immunoenzymatic and immunochromatographic tests for viral marker screening of hepatitis B (HBV), hepatitis C (HCV) and human immunodeficiency virus (HIV) in donated blood in a country of sub-Saharan Africa.</p><p><strong>Method: </strong>This study was conducted in a population of 113,406 blood donors at the National Centre of Blood Transfusion in Senegal. The data were obtained from the 'INLOG' software and donor registers. Statistical analyses used Excel 2010 and Epi Info v6. Screening for HBsAg viral markers, anti-HCV Ab, HIV p24 Ag, anti-HIV1 and anti-HIV2 antibodies were first carried out using the chemiluminescence technique. Blood donations screened positive for HBV or HCV were retested in a second chemiluminescence equipment. HIV-positive donations and their controls were subjected to solid phase immunochromatographic and indirect enzyme immunoassay techniques.</p><p><strong>Results: </strong>The prevalence among donors of HBV was 8.39 %, 0.56 % for HCV and 0.18 % for HIV. Of the donors tested positive for HIV in screenings and in doubled-controls, only 61.54 % were confirmed by the alternative tests; 34.02 % were negative and 4.44 % discordant between the three techniques.</p><p><strong>Conclusion: </strong>This study shows the importance of introducing the chemiluminescence technique in association with serological screening of transfusion-transmitted viruses to improve blood supply safety in low-income countries.</p>","PeriodicalId":94026,"journal":{"name":"Hematology, transfusion and cell therapy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-09-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142335171","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}
Fabíola Reis de Oliveira, Adriane Souza Lima, Carlos Roberto Faria, Thaise Oliveira Quaresma, Marcio M Mourani, Lauro Wichert-Ana, Paulo Louzada, Fernanda Gutierrez-Rodrigues, Neal S Young, Rodrigo T Calado
{"title":"Complex somatic mutation landscape in myeloid cells in a patient with VEXAS syndrome: First Brazilian case report.","authors":"Fabíola Reis de Oliveira, Adriane Souza Lima, Carlos Roberto Faria, Thaise Oliveira Quaresma, Marcio M Mourani, Lauro Wichert-Ana, Paulo Louzada, Fernanda Gutierrez-Rodrigues, Neal S Young, Rodrigo T Calado","doi":"10.1016/j.htct.2024.05.013","DOIUrl":"https://doi.org/10.1016/j.htct.2024.05.013","url":null,"abstract":"","PeriodicalId":94026,"journal":{"name":"Hematology, transfusion and cell therapy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-09-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142304626","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}
Jorge Antonio Elias Godoy Carlos, Keli Lima, Eduardo Magalhães Rego, Leticia Veras Costa-Lotufo, João Agostinho Machado-Neto
{"title":"The survivin/XIAP suppressant YM155 impairs clonal growth and induces apoptosis in JAK2<sup>V617F</sup> cells.","authors":"Jorge Antonio Elias Godoy Carlos, Keli Lima, Eduardo Magalhães Rego, Leticia Veras Costa-Lotufo, João Agostinho Machado-Neto","doi":"10.1016/j.htct.2024.05.012","DOIUrl":"https://doi.org/10.1016/j.htct.2024.05.012","url":null,"abstract":"<p><p>The central role of the control of apoptosis in the pathophysiology of Philadelphia chromosome-negative myeloproliferative neoplasms has recently been reinforced in genetic and pharmacological studies. The inhibitor of apoptosis protein family has eight members and plays an important role in apoptosis, with the most studied being survivin (BIRC5) and X-linked inhibitor of apoptosis (XIAP). YM155 is a small molecule with antineoplastic potential that has been described as a suppressant of survivin and XIAP. In the present study, BIRC5 expression was significantly increased in primary myelofibrosis patients compared to healthy donors. On the other hand, XIAP expression was reduced in myeloproliferative neoplasms patients. In JAK2<sup>V617F</sup> cells, YM155 reduces cell viability and autonomous clonal growth and induces apoptosis, cell cycle arrest, and autophagy. HEL cells that show greater malignancy are more sensitive to the drug than SET2 cells. In the molecular scenario, YM155 modulates apoptosis-, cell cycle-, DNA damage- and autophagy-related genes. Protein expression analysis corroborates the observed cellular phenotype and exploratory gene expression findings. In summary, our results indicate that survivin/BIRC5 and XIAP are differently expressed in myeloproliferative neoplasms and YM155 has multiple antineoplastic effects on JAK2<sup>V617F</sup> cells suggesting that inhibitor of apoptosis proteins may be a target for pharmacological interventions in the treatment of these diseases.</p>","PeriodicalId":94026,"journal":{"name":"Hematology, transfusion and cell therapy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-09-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142304648","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}
Frank Nunes, Breno Moreno de Gusmão, Franciely Bueno Wiginesk, Euler Manenti, Juliana Soares, Mizianne Garcia Freitas, Juliane Dantas Seabra-Garcez, Alexandre Manoel Varela, João Pedro Passos Dutra, Bruno Cesar Bacchiega, Tânia Félix Lorenzato da Fonseca Peixoto, Carolina Maria Pinto Domingues de Carvalho E Silva, Renato D Lopes, Ariane Vieira Scarlatelli Macedo
{"title":"From the mechanism of action to clinical management: A review of cardiovascular toxicity in adult treated with CAR-T therapy.","authors":"Frank Nunes, Breno Moreno de Gusmão, Franciely Bueno Wiginesk, Euler Manenti, Juliana Soares, Mizianne Garcia Freitas, Juliane Dantas Seabra-Garcez, Alexandre Manoel Varela, João Pedro Passos Dutra, Bruno Cesar Bacchiega, Tânia Félix Lorenzato da Fonseca Peixoto, Carolina Maria Pinto Domingues de Carvalho E Silva, Renato D Lopes, Ariane Vieira Scarlatelli Macedo","doi":"10.1016/j.htct.2024.06.008","DOIUrl":"https://doi.org/10.1016/j.htct.2024.06.008","url":null,"abstract":"<p><p>Chimeric antigen receptor T-cell therapy represents an innovative approach to immunotherapy and currently stands out, particularly for oncohematological patients refractory to traditional treatments. Ongoing trials are further expanding its clinical use for new oncological and non-oncological indications, potentially leading to newer treatment options soon. This new approach, however, also presents challenges, including cardiovascular toxicity. Little is reported in pivotal studies, and some recent retrospective observations suggest a non-negligible incidence of side effects with presentation ranging from mild adverse cardiovascular events to fatal complications in which, in most cases, there is a direct or indirect association with cytokine release syndrome. In this literature review, the hypotheses of an important interface between cytokine release syndrome and cardiotoxicity by chimeric antigen receptor T-cell therapy will be addressed, as will current knowledge about risk factors for cardiotoxicity and recommendations for pre-therapy evaluation, post-infusion monitoring and clinical management of these complications.</p>","PeriodicalId":94026,"journal":{"name":"Hematology, transfusion and cell therapy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-09-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142304629","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}
Sara Montolio Chiva, Paula Gomez Fernandez, Antonio Manuel Gutiérrez Garcia, Maria Del Carmen Ballester Ruiz, Antonia Sampol Mayol, Albert Perez Montaña
{"title":"An approach to autologous stem cell mobilization: trying to define good mobilizers.","authors":"Sara Montolio Chiva, Paula Gomez Fernandez, Antonio Manuel Gutiérrez Garcia, Maria Del Carmen Ballester Ruiz, Antonia Sampol Mayol, Albert Perez Montaña","doi":"10.1016/j.htct.2024.04.126","DOIUrl":"https://doi.org/10.1016/j.htct.2024.04.126","url":null,"abstract":"<p><strong>Background and objectives: </strong>Stem cell mobilization is a well-known procedure to harvest hematopoietic stem cells for autologous stem cell transplantation in certain hematologic diseases. Numerous studies have been conducted to identify risk factors for poor mobilization but there are no studies that identify good mobilizers. In our hospital, we decided to explore good mobilizers, defining them as those with ≥40 CD34<sup>+</sup> cells/μL on Day +4 in order to start early apheresis.</p><p><strong>Material and methods: </strong>A descriptive retrospective study was performed at Hospital Universitari Son Espases. A total of 198 patients mobilized with doses of around 10 µg/kg of granulocyte colony-stimulating factor (G-CSF) every 12 h were analyzed for autologous collection between January 2015 and September 2022. Fifty patients who had ≥40 CD34<sup>+</sup> cells/μL on Day +4 started early apheresis; the rest continued mobilization as planned. Success was defined as obtaining over 2.5 × 10<sup>6</sup> CD34<sup>+</sup> cells/kg in a single apheresis.</p><p><strong>Results: </strong>The necessary number of CD34<sup>+</sup> cells/kg to perform an autologous stem cell transplantation was reached in a single apheresis session in 62 % of patients with ≥40 CD34<sup>+</sup> cells/μL in peripheral blood. A cutoff of 102 CD34<sup>+</sup> cells/μL on Day +4 was shown to have the best success rate (94 %). In an analysis of success, age, previously failed mobilization and having one or more adverse factors for bad mobilization were statistically significant.</p><p><strong>Conclusion: </strong>Patients considered as good mobilizers were matched with our factors of poor mobilization, revealing that most patients (79 %) had none or only one risk factor for poor mobilization. Apheresis on Day +4 in good mobilizers was shown to be an effective alternative to reduce mobilization duration and decrease the amount of granulocyte-colony stimulating factor administered.</p>","PeriodicalId":94026,"journal":{"name":"Hematology, transfusion and cell therapy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-09-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142304622","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}
Larissa Hilario Dulley, Arthur Gomes Oliveira Braga, Guilherme Garcia Rodrigues, Sergio Costa Fortier, Carlos Sérgio Chiattone, Talita Maira Bueno da Silveira
{"title":"Lower doses of dacarbazine (modified BEACODD) as a safer strategy with equal effectiveness in an intensive treatment protocol of Hodgkin's lymphoma: a preliminary retrospective analysis of a single public center in Brazil.","authors":"Larissa Hilario Dulley, Arthur Gomes Oliveira Braga, Guilherme Garcia Rodrigues, Sergio Costa Fortier, Carlos Sérgio Chiattone, Talita Maira Bueno da Silveira","doi":"10.1016/j.htct.2024.06.003","DOIUrl":"https://doi.org/10.1016/j.htct.2024.06.003","url":null,"abstract":"<p><p>The German Hodgkin Study Group developed the escalated BEACOPP (bleomycin, etoposide, doxorubicin, cyclophosphamide, vincristine, procarbazine, and prednisone) protocol as a treatment strategy for advanced-stage Hodgkin's lymphoma. In Brazil, as well as in other countries, procarbazine has been replaced with dacarbazine due to the limited availability of procarbazine. The Hematology Center at Irmandade da Santa Casa de Misericórdia in São Paulo adopted and modified the escalated BEACOPP protocol, substituting prednisone with dexamethasone and incorporating two different doses of dacarbazine: 375 mg/m<sup>2</sup>/day on Day 8 or the original dose of 250 mg/m<sup>2</sup>/day on Days 2 and 3. This adjustment was made in response to the anticipated toxicity profile. This study aimed to compare the two different doses in the protocols (375 mg/m<sup>2</sup>/cycle versus 500 mg/m<sup>2</sup>/cycle) administered to patients with advanced Hodgkin's lymphoma in similar periods. This retrospective study analyzed the data of 31 patients at a single center in Brazil from 2019 to 2021. Seventeen of the 31 patients received 500 mg/m<sup>2</sup>/cycle (500 Group), while 14 received 375 mg/m<sup>2</sup>/cycle (375 Group). At the end of the protocol, 71% of the patients in the 375 Group and 76% in the 500 Group achieved complete remission. On analyzing the number of cycles that patients presented with febrile neutropenia, the 500 Group had three times more events (17.9%) than the 375 Group (6.09% - p-value = 0.04). In the 500 Group, 47.1% needed to change the protocol to ABVD (doxorubicin hydrochloride, bleomycin sulfate, vinblastine sulfate, and dacarbazine) due to toxicity. In this limited cohort from a single public center in Brazil, the use of 375 mg/m<sup>2</sup> of dacarbazine per cycle of the modified escalated BEACOPP protocol emerged as a safer strategy, maintaining treatment efficacy without compromising response in patients with advanced Hodgkin's lymphoma.</p>","PeriodicalId":94026,"journal":{"name":"Hematology, transfusion and cell therapy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-09-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142304631","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}
Thiago Henrique Costa, Carolina Bonet-Bub, José Mauro Kutner
{"title":"Platelet antibody detection assays: a single-laboratory comparison of MAIPA, PIFT, and microsphere-based multiplex assays Pak-Lx.","authors":"Thiago Henrique Costa, Carolina Bonet-Bub, José Mauro Kutner","doi":"10.1016/j.htct.2024.06.004","DOIUrl":"https://doi.org/10.1016/j.htct.2024.06.004","url":null,"abstract":"<p><strong>Background and objectives: </strong>The identification of platelet antibodies is essential for diagnosing and managing conditions such as fetal and neonatal alloimmune thrombocytopenic purpura, post-transfusion purpura, and immune platelet refractoriness. Monoclonal antibody immobilization of platelet antigens (MAIPA) is the standard method for detecting anti-human platelet antigen (HPA) antibodies, while the detection of anti-HLA antibodies once relied on the complement-dependent cytotoxicity method, however advanced technologies such as enzyme-linked immunosorbent assay and Luminex have significantly improved sensitivity and accuracy in identifying these antibodies. Flow cytometry-based techniques (platelet immunofluorescence test - PIFT) and Luminex platform-driven microsphere-based multiplex assays (Pak-Lx) are widely employed in platelet immunology laboratories owing to their remarkable flexibility and versatility. The present study compared the sensitivity, specificity, and concordance of these different serological techniques used in platelet antibody identification.</p><p><strong>Material and methods: </strong>One hundred serum samples from patients suspected of immune-mediated platelet disorders were examined. Initially, the samples underwent testing using the MAIPA method. Subsequently, the results were compared with three alternative methods: PIFT and microsphere-based multiplex assays for both HLA and HPA antibodies.</p><p><strong>Results: </strong>Pak-Lx demonstrated a 94 % agreement with MAIPA, while PIFT had 88 % agreement for HPA antibodies. For HLA antibody detection, Pak-Lx versus DLX had 75 % concordance, MAIPA versus DLX showed 77 %, and PIFT versus DLX displayed an 81 % concordance rate. Remarkably, there were no significant differences in concordance levels between Pak-Lx and PIFT compared to MAIPA and DLX for anti-HPA and HLA antibodies, respectively.</p><p><strong>Conclusion: </strong>This study found no significant differences in concordance among the tested assays for detecting anti-HPA and anti-HLA antibodies. These data suggest that no single method can detect all clinically important antibodies. Therefore, it is advisable that each laboratory develops customized protocols based on their expertise and employs complementary methods for comprehensive patient assessments.</p>","PeriodicalId":94026,"journal":{"name":"Hematology, transfusion and cell therapy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-09-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142304646","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}
Liana Ariel de Siqueira Lira, Jorge David Aivazoglou Carneiro, Maria do Carmo Menezes Bezerra Duarte
{"title":"Pediatric venous thromboembolism: incidence and patient profile in a single Brazilian institution.","authors":"Liana Ariel de Siqueira Lira, Jorge David Aivazoglou Carneiro, Maria do Carmo Menezes Bezerra Duarte","doi":"10.1016/j.htct.2024.06.006","DOIUrl":"https://doi.org/10.1016/j.htct.2024.06.006","url":null,"abstract":"<p><strong>Background: </strong>As the diagnosis of Pediatric venous thromboembolism has dramatically increased in recent decades, this study aims to evaluate these patients, determining the incidence and describing their biological and clinical characteristics.</p><p><strong>Methods: </strong>An observational, cross-sectional study was conducted at a Brazilian quaternary hospital between January 2022 and February 2023. Under 18-year-old hospitalized patients with a confirmed diagnosis of venous thromboembolism were included, while those with arterial or chronic thrombosis were excluded. Data on biological and clinical characteristics, diagnosis and treatment were evaluated. A descriptive data analysis was performed and the incidence of hospital-associated thrombosis was calculated.</p><p><strong>Results: </strong>Thirty-nine pediatric patients were evaluated. The incidence of hospital-associated thrombosis was 19.9 cases per 10,000 pediatric hospitalizations. Median age at diagnosis was four months (range: 12 days-17 years). Most of the patients (66.7%) were asymptomatic, with venous thromboembolism being diagnosed incidentally. In all cases, at least one risk factor was identified and in 74.6% of cases four or more factors were present. The principal risk factors were the presence of a central venous catheter (89.7%) and infection (89.7%). Thrombogenic comorbidities, particularly congenital heart disease, were present in 48.7% of patients.</p><p><strong>Conclusions: </strong>The incidence of venous thromboembolism found in the present study was lower than rates reported in developed countries. The principal characteristics of this sample were a greater frequency of central venous catheter and infection as risk factors, and the fact that the cases consisted mainly of newborns and individuals with heart disease.</p>","PeriodicalId":94026,"journal":{"name":"Hematology, transfusion and cell therapy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-09-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142304644","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}
Karina Viani, William Furlong, Vicente Odone Filho, Mariana Dos Santos Murra, Juliana Moura Nabarrete, Elena Ladas, Ronald Duncan Barr
{"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":"https://doi.org/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":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-09-07","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}