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  • 1
    In: Blood, American Society of Hematology, Vol. 124, No. 21 ( 2014-12-06), p. 1313-1313
    Abstract: The aim of this study is to evaluate the management outcomes and hospital costs of acute leukemia (AL) patients over a 4-year period in France using the national Program for a Medicalised Information System (PMSI) database, which records all hospitalisations in the country. We identified patients with AL or another underlying hematologic malignancy prone to transform into AL (e.g. myelodysplastic syndromes) at diagnosis and hospitalized between 2006 and 2012 and retained only incident patients. We used a retrospective cohort approach, with a 5-year follow-up time from index date (i.e. 2008 to 2012), with an additional focus on the autologous and allogeneic stem cell transplanted (SCT) patient populations during the year 2012. Hospital costs calculations were based on the national scale of costs. A total of 21,757 patients were included with 5,655 acute lymphoblastic leukemias (ALL) (26%) and 16,102 acute myelogenous leukemias (AML) at an incidence rate of 4,300 newly diagnosed AL/year. 54.7% were males (57.6% for ALL and 53.7 for AML) with a median age of 18 years (range 0-100, peak between 0-10 years) for ALL and 71 years (range 0-104, peak between 71-80 years) for AML. In 96.7% of the cases patients were hospitalized in a public healthcare hospital. The median duration of the first hospitalization was 7 days (quartiles: 1-29), with an overall death rate during this first hospitalization of 10.5% (4.4% for ALL and 12.6% for AML). Fifty-nine percent of the patients received chemotherapy, (77% for ALL, and 52% for AML); 3,079 (14%) of patients received an autologous or allogeneic stem cell transplantation (SCT) [883 in ALL patients, 2,196 in AML patients]. Among SCT patients, 2,732 (89%) had an allogeneic SCT [774 in ALL patients and 1,958 in AML patients] . Over the 2,732 allogeneic SCT group, 1,243 (45%) suffered from an acute GVHD (55% of ALL patients and 42% of AML patients), and 21% from chronic GVHD (25% of ALL patients and 20% of AML patients). Post SCT transplant survival rates for AML and ALL were 83% and 86% at one year and 55% and 58% at 4 years. Without SCT transplant, survival rates were 40% and 77% at one year and 24% and 70% at 4 years for AML and ALL respectively. The overall survival rates were significantly improved with transplant for AML patients but not for ALL patients at 4 years (see figure 1). Figure 1: Overall survival rates for AL patients since diagnosis according to treatment procedures [ALL patients with transplant (n=883), without transplant (n=4,772), AML patients with transplant (n=3,079), without transplant (n=13,023)]. Figure 1:. Overall survival rates for AL patients since diagnosis according to treatment procedures [ALL patients with transplant (n=883), without transplant (n=4,772), AML patients with transplant (n=3,079), without transplant (n=13,023)] . Cost analyses showed an overall median hospitalization cost the first year after diagnosis of 44,453 for AL (72,360 for ALL, and 40,477 for AML), the second year after diagnosis the median overall hospitalization cost for AL was reduced to 2,185 (6,212 for ALL and 904 for AML). The median hospitalization costs of the stay for SCT were 42 068 for ALL and also 42 068 for ALM patients, while the cost of allogenic vs. autologous patients was nearly three times higher. In conclusion, this study in an extremely large national cohort illustrates the current characteristics of the care of acute leukemia patients in our country and provides significant information on the economic impact of the hospital care as well as the additional cost of stem cell transplant. Disclosures Nicolini: Novartis: Consultancy. Laurendeau:CEMKA: Employment. Bureau:CEMKA: Employment. Ricci:Wellmera: Employment. Gerbeau:Novartis: Employment. Bouee:CEMKA: Employment.
    Type of Medium: Online Resource
    ISSN: 0006-4971 , 1528-0020
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    Language: English
    Publisher: American Society of Hematology
    Publication Date: 2014
    detail.hit.zdb_id: 1468538-3
    detail.hit.zdb_id: 80069-7
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  • 2
    In: Blood Advances, American Society of Hematology, Vol. 4, No. 15 ( 2020-08-11), p. 3708-3715
    Abstract: Myeloproliferative neoplasms (MPNs) are the most frequent underlying causes of splanchnic vein thromboses (SVTs). MPN patients with SVTs (MPN-SVT) often have a unique presentation including younger age, female predominance, and low Janus kinase 2 (JAK2) mutation allele burden. This study aimed at identifying risk factors for adverse hematologic outcomes in MPN-SVT patients. We performed a retrospective study of a fully characterized cohort of MPN-SVT patients. The primary outcome was the incidence of evolution to myelofibrosis, acute leukemia, or death. Eighty patients were included in the testing cohort. Median follow-up was 11 years. Most of the patients were women with a mean age of 42 years and a diagnosis of polycythemia vera. The primary outcome was met in 13% of the patients and was associated with a JAK2V617F allele burden ≥50% (odds ratio [OR], 14.7) and presence of additional mutations in genes affecting chromatin/spliceosome (OR, 9). We identified high-risk patients (29% of the cohort) as those harboring at least 1 molecular risk factor: JAK2-mutant allele burden ≥50%, presence of chromatin/spliceosome/TP53 mutation. High-risk patients had worse event-free survival (81% vs 100%; P = .001) and overall survival at 10 years (89% vs 100%; P = .01) than low-risk patients. These results were confirmed in an independent validation cohort of 30 MPN-SVT patients. In conclusion, molecular profiling identified MPN-SVT patients with dismal outcome. In this high-risk population, a disease-modifying therapy should be taken into consideration to minimize the probability of transformation.
    Type of Medium: Online Resource
    ISSN: 2473-9529 , 2473-9537
    Language: English
    Publisher: American Society of Hematology
    Publication Date: 2020
    detail.hit.zdb_id: 2876449-3
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