Eric J. Niesor, Anne Perez, Serge Rezzi, Andrew Hodgson, Stephane Canarelli, Gregoire Millet, Tadej Debevec, Claire Bordat, Elie Nader, Philippe Connes
{"title":"Plasma monomeric ApoA1 and high-density lipoprotein bound ApoA1 are markedly decreased and associated with low levels of lipophilic antioxidants in sickle cell disease: A potential new pathway for therapy","authors":"Eric J. Niesor, Anne Perez, Serge Rezzi, Andrew Hodgson, Stephane Canarelli, Gregoire Millet, Tadej Debevec, Claire Bordat, Elie Nader, Philippe Connes","doi":"10.1111/ejh.14288","DOIUrl":"10.1111/ejh.14288","url":null,"abstract":"<p>Patients with sickle cell disease (SCD) exhibit high levels of reactive oxygen species and low plasma levels of lipophilic antioxidants, which may contribute to end-organ damage and disease sequelae. Apolipoprotein A1, the major apolipoprotein of high-density lipoprotein (HDL), is mainly secreted by the intestine and liver in the form of monomeric ApoA1 (mApoA1) present in plasma. Cholesterol and α-tocopherol are delivered to ApoA1 via the ATP-binding cassette transporter, subfamily A, member 1 (ABCA1). We measured cholesterol, mApoA1, ApoA1, and lipophilic antioxidants in the plasma of 17 patients with SCD and 40 healthy volunteers. Mean HDL cholesterol (-C) levels in SCD patients and healthy subjects were 59.3 and 48.1 mg/dL, respectively, and plasma lutein, zeaxanthin, and α-tocopherol were 64.0%, 68.7%, and 9.1% lower, respectively. To compare SCD to healthy subjects with similar HDL-C, we also performed subgroup analyses of healthy subjects with HDL-C above or below the mean. In SCD, the mApoA1 level was 30.4 μg/mL; 80% lower than 141 μg/mL measured in healthy volunteers with similar HDL-C (56.7 mg/dL). The mApoA1 level was also 38.4% greater in the higher versus lower HDL-C subgroups (<i>p</i> = .002). In the higher HDL-C subgroup, lutein and zeaxanthin transported by HDL were 48.9% (<i>p</i> = .01) and 41.9% (<i>p</i> = .02) higher, respectively, whereas α-tocopherol was 31.7% higher (<i>p</i> = .003), compared to the lower HDL-C subgroup. Plasma mApoA1 may be a marker of the capacity of HDL to capture and deliver liposoluble antioxidants, and treatments which raise HDL may benefit patients with high oxidative stress as exemplified by SCD.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"113 6","pages":"788-797"},"PeriodicalIF":2.3,"publicationDate":"2024-08-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14288","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142008486","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Sara Redondo, Irene García-Cadenas, Albert Esquirol, J. M. Portos, Eva Iranzo, Miguel Arguello-Tomas, Silvana Saavedra, Guadalupe Oñate, Ana-Carolina Caballero, Ana Garrido, Jordi López, Ana Muntañola, Annalisa Paviglianiti, Sara Miqueleiz, Jorge Sierra, Javier Briones, Rodrigo Martino
{"title":"Severity and organ distribution of graft-versus-host disease with post-transplant cyclophosphamide versus calcineurin inhibitor plus methotrexate/mycophenolate mofetil or sirolimus in allogenic HLA-matched or single-allele mismatched stem cell transplantation","authors":"Sara Redondo, Irene García-Cadenas, Albert Esquirol, J. M. Portos, Eva Iranzo, Miguel Arguello-Tomas, Silvana Saavedra, Guadalupe Oñate, Ana-Carolina Caballero, Ana Garrido, Jordi López, Ana Muntañola, Annalisa Paviglianiti, Sara Miqueleiz, Jorge Sierra, Javier Briones, Rodrigo Martino","doi":"10.1111/ejh.14294","DOIUrl":"10.1111/ejh.14294","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Objective</h3>\u0000 \u0000 <p>This retrospective single center study aims to describe changes in the severity and organ-specific distribution of GvHD, by comparing the outcomes of 3 distinct GvHD prophylaxis approaches.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Between January 2012 and June 2022, 226 patients underwent allogeneic hematopoietic stem cell transplantation from HLA-matched or 1-allele mismatched related or unrelated donors. Fifty-eight (26%) received prophylaxis with calcineurin inhibitor in combination with mycophenolate mofetil or a short course of methotrexate (Cohort-1), 87 (38%) tacrolimus plus sirolimus (Cohort-2), and 81 (36%) post-transplant cyclophosphamide (PTCy) plus tacrolimus (Cohort-3).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The incidence of grade II-IV aGvHD was 69% vs. 41.4% vs. 27.2%; <i>p <</i> .01. The most significant reduction with PTCy was observed in both stage 3–4 skin and lower gastrointestinal (GI) involvement (<i>p <</i> .01). The incidence of moderate-to-severe cGvHD at 12 months was 34.5% vs. 34.5% vs. 6.2%; <i>p</i> < .01. Moderate-to-severe skin and GI cGvHD was less common after PTCy (<i>p</i> < .01). The 1-year GvHD-free/relapse-free survival was higher with PTCy (<i>p</i> < .01).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Our study indicates that PTCy-based GvHD prophylaxis reduces the frequency and severity of both acute and chronic GvHD, with a notable decrease in severe GI and cutaneous manifestations. The higher GRFS may result in lower GvHD-related mortality, leading to an improved quality of life among survivors.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"113 6","pages":"776-787"},"PeriodicalIF":2.3,"publicationDate":"2024-08-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141999682","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Victoria Murillo, Paola Charry, María Suárez-Lledó, Laia Guardia, Cristina Moreno, Joan Cid, Miquel Lozano, Alexandra Pedraza, Raquel Salinas, Vanessa Vilas, Montserrat Duch, Marina Díaz-Beya, Laura Rosiñol, Jordi Esteve, Enric Carreras, Francesc Fernández-Avilés, Carmen Martínez, Montserrat Rovira, María Queralt Salas
{"title":"Outcomes of older adults undergoing allogeneic hematopoietic cell transplantation with post-transplant cyclophosphamide based prophylaxis","authors":"Victoria Murillo, Paola Charry, María Suárez-Lledó, Laia Guardia, Cristina Moreno, Joan Cid, Miquel Lozano, Alexandra Pedraza, Raquel Salinas, Vanessa Vilas, Montserrat Duch, Marina Díaz-Beya, Laura Rosiñol, Jordi Esteve, Enric Carreras, Francesc Fernández-Avilés, Carmen Martínez, Montserrat Rovira, María Queralt Salas","doi":"10.1111/ejh.14291","DOIUrl":"10.1111/ejh.14291","url":null,"abstract":"<p>This study evaluates the feasibility of using post-transplant cyclophosphamide (PTCY) prophylaxis in allo-hematopoietic cell transplantation (HCT) for adults aged 65 and older. PTCY is increasingly used to prevent graft-versus-host disease (GVHD) across all donor types, but concerns remain about potential risks, especially in older patients. Fifty-seven adults aged 65 or older with hematological malignancies, undergoing their first allo-HCT with PTCY prophylaxis between January 2011 and January 2023 were included. Overall, 94.8% of patients achieved primary engraftment. The median durations for neutrophil and platelet engraftments were 19 and 21 days. The day +30 cumulative incidence of bacterial bloodstream infection was 43.9%. No CMV reactivations occurred within the first 100 days after letermovir implementation. The day +180 cumulative incidences of grade II–IV and III–IV acute GVHD, and the 2-year cumulative incidence of moderate/severe chronic GVHD were 26.3%, 10.5%, and 4.8%. Eighteen patients (31.6%) relapsed, and 30 (52.6%) died, with relapse (16.4%) and infection (11.5%) being the main causes of death. The estimated 2-year overall survival, non-relapse mortality, cumulative incidence of relapse, and GVHD-free relapse-free survival rates were 45.5%, 27.1%, 33.9%, and 37.0%. Adults aged 70 or older had similar outcomes to those aged 65–69. This study confirms the safety and feasibility of PTCY-based allo-HCT in older adults.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"113 6","pages":"765-775"},"PeriodicalIF":2.3,"publicationDate":"2024-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141981994","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Graft-versus-host disease in patients with bone marrow transplants: A retrospective study analyzing outcomes and healthcare burden in US hospitals","authors":"Rushin Patel, Afoma Onyechi, Jessica Ohemeng-Dapaah, Mrunal Patel, Darshil Patel, Zalak Patel, Yu-Han Chen, Chieh Yang","doi":"10.1111/ejh.14281","DOIUrl":"10.1111/ejh.14281","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Graft-versus-host disease (GVHD) is a recognized complication among individuals undergoing bone marrow transplantation (BMT). There is a requirement for supplementary data regarding the in-patient outcomes of GVHD in individuals who have undergone BMT. Our analysis seeks to assess the healthcare burden and outcomes associated with GVHD in hospitalized patients who have undergone BMT.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Method</h3>\u0000 \u0000 <p>In this retrospective study, we used data from the National Inpatient Sample (NIS) database spanning from 2016 to 2019. Utilizing ICD-10 codes, we distinguished hospitalizations related to BMT and grouped them into two categories: those with GVHD and those without GVHD. Our areas of focus included in-hospital mortality, length of stay, charges, and associations related to GVHD. Unadjusted odds ratios/coefficients were computed through univariable analysis, followed by adjusted odds ratios (aORs)/coefficients from multivariable analysis that considered potential confounding factors.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>From 2016 to 2019, data were collected from 13,999 hospitalizations with bone marrow transplants. Among them, 836 had GVHD cases. Patient characteristics showed slight differences in mean age and demographics between the two groups, with GVHD patients having a mean age of 51.61 years and higher percentages of males and whites. Analyzing outcomes, patients with GVHD experienced significantly longer hospital stays (41.4 days vs. 21.3 days) and higher total hospital charges ($824,058 vs. $335,765). Adjusting for confounding factors, GVHD posed a substantial risk. The aOR for mortality in GVHD hospitalizations was 7.20 (95% CI: 5.54–9.36, <i>p</i> < .001). The coefficient for the length of stay was 19.36 days (95% CI: 17.29–21.42, <i>p</i> < .001), and the coefficient for total hospital charges was $453,733 (95% CI: $396,577 to $510,889, <i>p</i> < .001) in GVHD cases. Furthermore, GVHD in patients was associated with elevated risks of various medical conditions. The aORs for sepsis, pneumonia, acute respiratory failure, intubation and mechanical ventilation, <i>Clostridium difficile</i> infection, and acute kidney injury (AKI) in GVHD patients were 2.79 (95% CI: 2.28–3.41, <i>p</i> < .001), 3.30 (95% CI: 2.57–4.24, <i>p</i> < .001), 5.10 (95% CI: 4.01–6.49, <i>p</i> < .001), 4.88 (95% CI: 3.75–6.34, <i>p</i> < .001), 1.45 (95% CI: 1.13–1.86, <i>p</i> = .003), and 3.57 (95% CI: 2.97–4.29, <i>p</i> < .001).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 ","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"113 6","pages":"758-764"},"PeriodicalIF":2.3,"publicationDate":"2024-08-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14281","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141970909","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Venetoclax-based non-intensive induction followed by allogenic stem-cell transplantation in elderly acute myeloid leukemia patients with adverse cytogenetics","authors":"Amel Soua, Julia Gilhodes, Alexandre Iat, Yosr Hicheri, Colombe Saillard, Camille Rouzaud, Evelyne D'Incan, Jérôme Rey, Bilal Mohty, Aude Charbonnier, Antoine Ittel, Anne-Sophie Alary, Véronique Gelsi-Boyer, Anne Murati, Anne-Catherine Lhoumeau, Raynier Devillier, Jean-Marie Boher, Marie-Joelle Mozziconacci, Norbert Vey, Marie-Anne Hospital, Sylvain Garciaz","doi":"10.1111/ejh.14290","DOIUrl":"10.1111/ejh.14290","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Elderly acute myeloid leukemia (AML) patients with poor-risk cytogenetics have a poor outcome with intensive chemotherapy (IC). While Venetoclax (VEN) has changed the outcomes of elderly unfit patients treatment, it is unknown whether it could be effective in poor-risk cytogenetics 60–75 years old patients.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Materials and Methods</h3>\u0000 \u0000 <p>We included 60–75-year-old AML patients eligible to allogenic stem cell transplantation (allo-SCT) treated with VEN (combined with azacitidine or with Cladribin and Aracytine) at Institut Paoli Calmettes, between 2020 and 2023 and compared this cohort with patients treated by IC between 2010 and 2019.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Twenty six patients were treated with VEN (17 in combination with azacitidine and 9 with Cladribin and Aracytine) and 90 were treated with IC. Thirteen patients (50%) had a <i>TP53</i> mutation. The median time for leucocyte and platelet counts recovery was 26 days (range 0–103) and 26 days (range, 0–63). The median duration of the first hospitalization was 32 days (ranges, 7–79). The composite response rate was 69% (CR = 50%, CRi = 4%, MLFS = 15%). Allo-SCT could be performed in 42% of cases. Median overall survival (OS) was 7.9 months (20.9 months in the group of patients who transitioned to allo-SCT). We found no difference with the historical cohort of patients treated with IC except a trend toward less lower and upper tract gastro-intestinal (GI) tract infections in the VEN group (respectively 8% vs 26%, <i>p</i> = .06; and 0% vs. 13% <i>p</i> = .06).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>VEN-based treatment was found to be effective in high risk AML can be considered as an alternative to IC in patients aged 60–75 with adverse cytogenetics.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"113 6","pages":"751-757"},"PeriodicalIF":2.3,"publicationDate":"2024-08-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14290","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141916456","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Elliot Smith, Eshetu G. Atenafu, Aniket Bankar, Steven Chan, Marta Davidson, Vikas Gupta, Mark D. Minden, Guillaume Richard-Carpentier, Aaron Schimmer, Andre C. Schuh, Hassan Sibai, Karen Yee, Dawn Maze
{"title":"Evolution from an antecedent chronic myeloid malignancy does not impact survival outcomes in NPM1-mutated AML","authors":"Elliot Smith, Eshetu G. Atenafu, Aniket Bankar, Steven Chan, Marta Davidson, Vikas Gupta, Mark D. Minden, Guillaume Richard-Carpentier, Aaron Schimmer, Andre C. Schuh, Hassan Sibai, Karen Yee, Dawn Maze","doi":"10.1111/ejh.14283","DOIUrl":"10.1111/ejh.14283","url":null,"abstract":"<p>Nucleophosmin-1 (NPM1)-mutated AML is a molecularly defined subtype typically associated with favorable treatment response and prognosis; however, its prognostic significance in AML evolving from an antecedent chronic myeloid malignancy is unknown. This study's primary objective was to determine the impact of mutated NPM1 on the prognosis of AML evolving from an antecedent chronic myeloid malignancy. We conducted a retrospective chart review including patients with NPM1-mutated de novo and sAML. sAML was defined as those with a preceding chronic-phase myeloid malignancy before diagnosis of AML. Of 575 NPM1-mutated patients eligible for inclusion in our study, 51 (8.9%) patients were considered to have sAML. The median time from diagnosis of NPM1-mutated chronic myeloid malignancy to sAML evolution was 3.6 months (0.5–79.3 months). No significant differences in leukemia-free (2-year LKFS 52.0% vs. 51.2%, <i>p</i> = .9922) or overall survival (2-year OS 56.3% vs. 49.4%, <i>p</i> = .4246) were observed between patients with NPM1-mutated de novo versus sAML. Our study suggests that evolution from a preceding myeloid malignancy is not a significant predictor of poor prognosis in the setting of an NPM1 mutation. Our study demonstrated a short time to progression to sAML in most patients, which further supports the consideration of NPM1 as an AML-defining mutation.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"113 5","pages":"716-726"},"PeriodicalIF":2.3,"publicationDate":"2024-08-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14283","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141901386","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Camilla Eckert-Lind, Amani Meaidi, Brian Claggett, Niklas Dyrby Johansen, Mats Christian Højbjerg Lassen, Kristoffer Grundtvig Skaarup, Michael Fralick, Manan Pareek, Jens Ulrik Stæhr Jensen, Christian Torp-Pedersen, Gunnar Gislason, Tor Biering-Sørensen, Daniel Modin
{"title":"Recurrent venous thromboembolism and vaginal estradiol in women with prior venous thromboembolism: A nested case–control study","authors":"Camilla Eckert-Lind, Amani Meaidi, Brian Claggett, Niklas Dyrby Johansen, Mats Christian Højbjerg Lassen, Kristoffer Grundtvig Skaarup, Michael Fralick, Manan Pareek, Jens Ulrik Stæhr Jensen, Christian Torp-Pedersen, Gunnar Gislason, Tor Biering-Sørensen, Daniel Modin","doi":"10.1111/ejh.14287","DOIUrl":"10.1111/ejh.14287","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Objectives</h3>\u0000 \u0000 <p>Whether vaginal estradiol use is associated with an increased risk of recurrent venous thromboembolism (VTE) in women with prior VTE is unknown. We sought to evaluate the association between vaginal estradiol use and recurrent VTE in women with prior VTE.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We performed a nationwide nested case–control study among 44 024 women aged ≥45 years who developed a first VTE without a history of vaginal estrogen use prior to VTE diagnosis. Cases with recurrent VTE were matched 1:2 on birth year with controls using incidence density sampling. Exposure to vaginal estradiol tablets was categorized into current use (0–2 months before index), prior use (2–24 months before index) and past use (more than 24 months prior to index).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>We identified 5066 cases and 10 127 age-matched controls. In fully adjusted analysis vaginal estrogen was not associated with recurrent VTE with a hazard ratio of 0.75, <i>p</i> = .07 for current use, 0.83, <i>p</i> = .13 for prior use, and 1.24, <i>p</i> = .06 for past use.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Use of vaginal estradiol tablets in women with prior VTE was not associated with an increased rate of recurrent VTE. Our study indicates that vaginal estradiol therapy is unlikely to increase risk of recurrent VTE in women with prior VTE.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"113 6","pages":"745-750"},"PeriodicalIF":2.3,"publicationDate":"2024-08-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141901387","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Alexander Slowley, Sofie Weinmann, Tim d'Estrube, Kelesitse Phiri, Florian M. Karl, Erika Gleißner, Sabrina Mueller, Sophia Junker, Joachim R. Göthert
{"title":"Myelofibrosis and anemia: A German claims data analysis to describe epidemiology and current treatment","authors":"Alexander Slowley, Sofie Weinmann, Tim d'Estrube, Kelesitse Phiri, Florian M. Karl, Erika Gleißner, Sabrina Mueller, Sophia Junker, Joachim R. Göthert","doi":"10.1111/ejh.14284","DOIUrl":"10.1111/ejh.14284","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Objectives</h3>\u0000 \u0000 <p>There is limited data on the incidence, prevalence, and treatments for myelofibrosis (MF) in Germany. This retrospective study examined claims data from 3.3 million insured individuals, spanning from 2010 to 2021.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Four sensitivity scenarios were explored to identify cases of MF. Point prevalence and cumulative incidence of MF were determined as of December 31, 2021, and within 2021, respectively. A cross-sectional analysis used the main scenario definition of MF to identify cases and evaluate the period prevalence of patients receiving treatment for symptoms and/or splenomegaly, including first-line (1L) Janus kinase inhibitor (JAKi), second-line, or further (2L+) MF-related treatment therapies during 2021. The prevalence of anemia treatment was also reported.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The estimated standardized point prevalence of MF on December 31, 2021, was 9.9–12.4 cases per 100 000 persons, and cumulative incidence in 2021 was 1.2–1.8 cases per 100 000 persons. Standardized period prevalence in 2021 for MF patients receiving 1L JAKi and/or 2L+ MF-related treatment was 4.0 cases per 100 000. Among these patients, 47.1%–53.7% required treatment for anemia, resulting in a period prevalence of 1.9–2.2 cases per 100 000 individuals.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>The data reveal gaps in MF treatments and the need to improve patient quality of life.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"113 5","pages":"704-715"},"PeriodicalIF":2.3,"publicationDate":"2024-08-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14284","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141888849","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Health-related quality of life in older hematological cancer survivors (70+) compared to older general population—A German cancer-register-based cross-sectional comparative study","authors":"Florian Schatz, Anja Mehnert-Theuerkauf, Uwe Platzbecker, Franziska Springer, Heide Götze","doi":"10.1111/ejh.14285","DOIUrl":"10.1111/ejh.14285","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Objectives</h3>\u0000 \u0000 <p>The extent of health-related quality of life (HRQOL) impairments in older hematological cancer survivors (HCS) has not been sufficiently studied. We therefore examined HRQOL in older HCS compared to a community sample (CS) and investigated sociodemographic, disease- and treatment-specific, geriatric, and psychosocial factors associated with reduced HRQOL.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Materials and Methods</h3>\u0000 \u0000 <p>In this cancer-register-based cross-sectional comparative study 200 HCS, aged ≥70 years, and 252 persons of an age- and gender-matched CS completed validated questionnaires including the EORTC QLQ-C30 and EORTC QLQ-ELD14.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Older HCS reported a reduced HRQOL in the dimensions of global QOL, physical, role, and social functioning (small clinical significance) and higher symptom burden of fatigue, nausea and vomiting, appetite loss, and poorer mobility compared to the CS (fatigue and mobility with medium, the others with small clinical significance). Perceived disease burden of comorbidities, functional disabilities, psychological distress, and depression showed statistical significance for reduced HRQOL in older HCS in multiple linear regression analysis (<i>R</i><sup>2</sup> = .602, <i>p</i> < .001).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Discussion</h3>\u0000 \u0000 <p>The screening and treatment of functional limitations and individual symptoms and the integration of a geriatric assessment into oncological practice can help to identify supportive care needs, to implement individualized, patient-centered cancer survivorship care programs and to improve older HCS's HRQOL.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"113 5","pages":"693-703"},"PeriodicalIF":2.3,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14285","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141874516","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}