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1. Intravenously administered interleukin-7 to reverse lymphopenia in patients with septic shock: a double-blind, randomized, placebo-controlled trial [2023]

  • Daix, Thomas, Mathonnet, Armelle, Brakenridge, Scott, Dequin, Pierre-François, Mira, Jean-Paul, Berbille, Frederique, Morre, Michel, Jeannet, Robin, Blood, Teresa, Unsinger, Jacqueline, Blood, Jane, Walton, Andrew, Moldawer, Lyle L., Hotchkiss, Richard, and Francois, Bruno
  • Annals of Intensive Care. December, 2023, Vol. 13 Issue 1

2. The 24-hour food frequency assessment screening tool (FAST24): Development and evaluation of a novel dietary screener to identify foods associated with weight change. [2023]

  • Masheb RM, Vernarelli JA, Snow JL, Marsh AG, Ciszewski S, Dudley B, White CA, Purcell SA, and Lutes L
  • Clinical nutrition ESPEN [Clin Nutr ESPEN] 2023 Oct; Vol. 57, pp. 735-738. Date of Electronic Publication: 2023 Aug 24.
Subjects
United States, Female, Humans, Adolescent, Young Adult, Adult, Male, Databases, Factual, Diet, and Food
Abstract
Background & Aims: Brief screening questionnaires can identify 'at risk' behaviors in clinical settings. However, there is currently no screener for dietary intake specifically developed using foods associated with body weight change and increased risk for multiple chronic conditions and diseases.
Methods: We developed a novel brief dietary screener, the 24-Hour Food Frequency Assessment Screening Tool Questionnaire (FAST24), to identify intake of foods associated with weight change. University students completed the FAST24 and the Automated Self-Administered 24-Hour Dietary Assessment Tool (ASA24) at two time points to assess acceptability and determine preliminary criterion validity against food categories from the United States Department of Agriculture (USDA) Food Patterns Equivalents Database (FPED).
Results: 202 individuals (age 20.4 ± 3.6 years; 65.7% females) completed the FAST24 in an average time of 2 min compared to 24 min for the ASA24. Over half of the food items from the FAST24 were matched to, and correlated with, standard USDA food pattern components (r's ranging from .15 to .58, p's < .05). Food items from the dietary data from the FAST24 were also highly correlated with the more intensive ASA24 application (r's ranging from .23 to .82, p's < .01), and were less time-consuming and burdensome to complete (p's < .0001).
Conclusions: Findings support the continued refinement of the FAST24 as a rapid, valid primary care assessment tool for measuring USDA dietary intake patterns. Use of a short, simple screener such as the FAST24 has the potential for integration into large healthcare delivery settings to help establish a baseline for promoting relative behavior changes critical for long-term health and well-being.
Competing Interests: Declaration of competing interest The authors declare no conflicts of interest.
(Published by Elsevier Ltd.)

3. CMR-derived left ventricular intraventricular pressure gradients identify different patterns associated with prognosis in dilated cardiomyopathy. [2023]

  • Vos JL, Raafs AG, Henkens MTHM, Pedrizzetti G, van Deursen CJ, Rodwell L, Heymans SRB, and Nijveldt R
  • European heart journal. Cardiovascular Imaging [Eur Heart J Cardiovasc Imaging] 2023 Aug 23; Vol. 24 (9), pp. 1231-1240.
Subjects
Humans, Contrast Media, Ventricular Pressure, Magnetic Resonance Imaging, Cine, Gadolinium, Ventricular Function, Left, Stroke Volume, Magnetic Resonance Spectroscopy, Prognosis, Predictive Value of Tests, and Cardiomyopathy, Dilated
Abstract
Aims: Left ventricular (LV) blood flow is determined by intraventricular pressure gradients (IVPG). Changes in blood flow initiate remodelling and precede functional decline. Novel cardiac magnetic resonance (CMR) post-processing LV-IVPG analysis might provide a sensitive marker of LV function in dilated cardiomyopathy (DCM). Therefore, the aim of our study was to evaluate LV-IVPG patterns and their prognostic value in DCM.
Methods and Results: LV-IVPGs between apex and base were measured on standard CMR cine images in DCM patients (n = 447) from the Maastricht Cardiomyopathy registry. Major adverse cardiovascular events, including heart failure hospitalisations, life-threatening arrhythmias, and sudden/cardiac death, occurred in 66 DCM patients (15%). A temporary LV-IVPG reversal during systolic-diastolic transition, leading to a prolonged transition period or slower filling, was present in 168 patients (38%). In 14%, this led to a reversal of blood flow, which predicted outcome corrected for univariable predictors [hazard ratio (HR) = 2.57, 95% confidence interval (1.01-6.51), P = 0.047]. In patients without pressure reversal (n = 279), impaired overall LV-IVPG [HR = 0.91 (0.83-0.99), P = 0.033], systolic ejection force [HR = 0.91 (0.86-0.96), P < 0.001], and E-wave decelerative force [HR = 0.83 (0.73-0.94), P = 0.003] predicted outcome, independent of known predictors (age, sex, New York Heart Association class ≥ 3, LV ejection fraction, late gadolinium enhancement, LV-longitudinal strain, left atrium (LA) volume-index, and LA-conduit strain).
Conclusion: Pressure reversal during systolic-diastolic transition was observed in one-third of DCM patients, and reversal of blood flow direction predicted worse outcome. In the absence of pressure reversal, lower systolic ejection force, E-wave decelerative force (end of passive LV filling), and overall LV-IVPG are powerful predictors of outcome, independent of clinical and imaging parameters.
Competing Interests: Conflict of interest: None declared.
(© The Author(s) 2023. Published by Oxford University Press on behalf of the European Society of Cardiology.)

4. Brain Imaging Abnormalities in Mixed Alzheimer's and Subcortical Vascular Dementia. [2023]

  • Lee H, Wiggermann V, Rauscher A, Kames C, Beg MF, Popuri K, Tam R, Lam K, Jacova C, Shahinfard E, Sossi V, Pettersen JA, and Hsiung GR
  • The Canadian journal of neurological sciences. Le journal canadien des sciences neurologiques [Can J Neurol Sci] 2023 Jul; Vol. 50 (4), pp. 515-528. Date of Electronic Publication: 2022 May 26.
Subjects
Humans, Diffusion Tensor Imaging, Cross-Sectional Studies, Brain diagnostic imaging, Brain pathology, Magnetic Resonance Imaging methods, Dementia, Vascular diagnostic imaging, Alzheimer Disease diagnostic imaging, Alzheimer Disease pathology, and Mixed Dementias
Abstract
Background: A large proportion of Alzheimer's disease (AD) patients have coexisting subcortical vascular dementia (SVaD), a condition referred to as mixed dementia (MixD). Brain imaging features of MixD presumably include those of cerebrovascular disease and AD pathology, but are difficult to characterize due to their heterogeneity.
Objective: To perform an exploratory analysis of conventional and non-conventional structural magnetic resonance imaging (MRI) abnormalities in MixD and to compare them to those observed in AD and SVaD.
Methods: We conducted a cross-sectional, region-of-interest-based analysis of 1) hyperintense white-matter signal abnormalities (WMSA) on T2-FLAIR and hypointense WMSA on T1-weighted MRI; 2) diffusion tensor imaging; 3) quantitative susceptibility mapping; and 4) effective transverse relaxation rate (R2*) in N = 17 participants (AD:5, SVaD:5, MixD:7). General linear model was used to explore group differences in these brain imaging measures.
Results: Model findings suggested imaging characteristics specific to our MixD group, including 1) higher burden of WMSAs on T1-weighted MRI (versus both AD and SVaD); 2) frontal lobar preponderance of WMSAs on both T2-FLAIR and T1-weighted MRI; 3) higher fractional anisotropy values within normal-appear white-matter tissues (versus SVaD, but not AD); and 4) lower R2* values within the T2-FLAIR WMSA areas (versus both AD and SVaD).
Conclusion: These findings suggest a preliminary picture of the location and type of brain imaging characteristics associated with MixD. Future imaging studies may employ region-specific hypotheses to distinguish MixD more rigorously from AD or SVaD.

5. Left Atrial Strain Is an Independent Predictor of New-Onset Atrial Fibrillation in Dilated Cardiomyopathy. [2023]

  • Raafs AG, Vos JL, Henkens MTHM, Verdonschot JAJ, Sikking M, Stroeks S, Gerretsen S, Hazebroek MR, Knackstedt C, Nijveldt R, and Heymans SRB
  • JACC. Cardiovascular imaging [JACC Cardiovasc Imaging] 2023 Jul; Vol. 16 (7), pp. 991-992. Date of Electronic Publication: 2023 Mar 08.
Subjects
Humans, Predictive Value of Tests, Heart Atria diagnostic imaging, Echocardiography, Atrial Fibrillation diagnostic imaging, Atrial Fibrillation etiology, and Cardiomyopathy, Dilated diagnostic imaging

6. Innovative Technologies in CNS Trials: Promises and Pitfalls for Recruitment, Retention, and Representativeness. [2023]

  • LUTZ, JACQUELINE, PRATAP, ABHISHEK, LENZE, ERIC J., BESTHA, DURGA, LIPSCHITZ, JESSICA M., KARANTZOULIS, STELLA, VAIDYANATHAN, UMA, ROBIN, JESSICA, HORAN, WILLIAM, BRANNAN, STEPHEN, MITTOUX, AURELIA, DAVIS, MICHAEL C., LAKHAN, SHAHEEN E., and KEEFE, RICHARD
  • Innovations in Clinical Neuroscience; Jul-Sep2023, Vol. 20 Issue 7-9, p40-46, 7p
Abstract
Objective: Recruitment of a sufficiently large and representative patient sample and its retention during central nervous system (CNS) trials presents major challenges for study sponsors. Technological advances are reshaping clinical trial operations to meet these challenges, and the COVID-19 pandemic further accelerated this development. Method of Research: The International Society for CNS Clinical Trials and Methodology (ISCTM; www.isctm.org) Innovative Technologies for CNS Trials Working Group surveyed the state of technological innovations for improved recruitment and retention and assessed their promises and pitfalls. Results: Online advertisement and electronic patient registries can enhance recruitment, but challenges with sample representativeness, conversion rates from eligible prescreening to enrolled patients, data privacy and security, and patient identification remain hurdles for optimal use of these technologies. Electronic medical records (EMR) mining with artificial intelligence (AI)/machine learning (ML) methods is promising but awaits translation into trials. During the study treatment phase, technological innovations increasingly support participant retention, including adherence with the investigational treatment. Digital tools for adherence and retention support take many forms, including patient-centric communication channels between researchers and participants, real-time study reminders, and digital behavioral interventions to increase study compliance. However, such tools add technical complexities to trials, and their impact on the generalizability of results are largely unknown. Conclusion: Overall, the group found a scarcity of systematic data directly assessing the impact of technological innovations on study recruitment and retention in CNS trials, even for strategies with already high adoption, such as online recruitment. Given the added complexity and costs associated with most technological innovations, such data is needed to fully harness technologies for CNS trials and drive further adoption. [ABSTRACT FROM AUTHOR]

7. Author Correction: The power of genetic diversity in genome-wide association studies of lipids. [2023]

  • Graham SE, Clarke SL, Wu KH, Kanoni S, Zajac GJM, Ramdas S, Surakka I, Ntalla I, Vedantam S, Winkler TW, Locke AE, Marouli E, Hwang MY, Han S, Narita A, Choudhury A, Bentley AR, Ekoru K, Verma A, Trivedi B, Martin HC, Hunt KA, Hui Q, Klarin D, Zhu X, Thorleifsson G, Helgadottir A, Gudbjartsson DF, Holm H, Olafsson I, Akiyama M, Sakaue S, Terao C, Kanai M, Zhou W, Brumpton BM, Rasheed H, Ruotsalainen SE, Havulinna AS, Veturi Y, Feng Q, Rosenthal EA, Lingren T, Pacheco JA, Pendergrass SA, Haessler J, Giulianini F, Bradford Y, Miller JE, Campbell A, Lin K, Millwood IY, Hindy G, Rasheed A, Faul JD, Zhao W, Weir DR, Turman C, Huang H, Graff M, Mahajan A, Brown MR, Zhang W, Yu K, Schmidt EM, Pandit A, Gustafsson S, Yin X, Luan J, Zhao JH, Matsuda F, Jang HM, Yoon K, Medina-Gomez C, Pitsillides A, Hottenga JJ, Willemsen G, Wood AR, Ji Y, Gao Z, Haworth S, Mitchell RE, Chai JF, Aadahl M, Yao J, Manichaikul A, Warren HR, Ramirez J, Bork-Jensen J, Kårhus LL, Goel A, Sabater-Lleal M, Noordam R, Sidore C, Fiorillo E, McDaid AF, Marques-Vidal P, Wielscher M, Trompet S, Sattar N, Møllehave LT, Thuesen BH, Munz M, Zeng L, Huang J, Yang B, Poveda A, Kurbasic A, Lamina C, Forer L, Scholz M, Galesloot TE, Bradfield JP, Daw EW, Zmuda JM, Mitchell JS, Fuchsberger C, Christensen H, Brody JA, Feitosa MF, Wojczynski MK, Preuss M, Mangino M, Christofidou P, Verweij N, Benjamins JW, Engmann J, Kember RL, Slieker RC, Lo KS, Zilhao NR, Le P, Kleber ME, Delgado GE, Huo S, Ikeda DD, Iha H, Yang J, Liu J, Leonard HL, Marten J, Schmidt B, Arendt M, Smyth LJ, Cañadas-Garre M, Wang C, Nakatochi M, Wong A, Hutri-Kähönen N, Sim X, Xia R, Huerta-Chagoya A, Fernandez-Lopez JC, Lyssenko V, Ahmed M, Jackson AU, Yousri NA, Irvin MR, Oldmeadow C, Kim HN, Ryu S, Timmers PRHJ, Arbeeva L, Dorajoo R, Lange LA, Chai X, Prasad G, Lorés-Motta L, Pauper M, Long J, Li X, Theusch E, Takeuchi F, Spracklen CN, Loukola A, Bollepalli S, Warner SC, Wang YX, Wei WB, Nutile T, Ruggiero D, Sung YJ, Hung YJ, Chen S, Liu F, Yang J, Kentistou KA, Gorski M, Brumat M, Meidtner K, Bielak LF, Smith JA, Hebbar P, Farmaki AE, Hofer E, Lin M, Xue C, Zhang J, Concas MP, Vaccargiu S, van der Most PJ, Pitkänen N, Cade BE, Lee J, van der Laan SW, Chitrala KN, Weiss S, Zimmermann ME, Lee JY, Choi HS, Nethander M, Freitag-Wolf S, Southam L, Rayner NW, Wang CA, Lin SY, Wang JS, Couture C, Lyytikäinen LP, Nikus K, Cuellar-Partida G, Vestergaard H, Hildalgo B, Giannakopoulou O, Cai Q, Obura MO, van Setten J, Li X, Schwander K, Terzikhan N, Shin JH, Jackson RD, Reiner AP, Martin LW, Chen Z, Li L, Highland HM, Young KL, Kawaguchi T, Thiery J, Bis JC, Nadkarni GN, Launer LJ, Li H, Nalls MA, Raitakari OT, Ichihara S, Wild SH, Nelson CP, Campbell H, Jäger S, Nabika T, Al-Mulla F, Niinikoski H, Braund PS, Kolcic I, Kovacs P, Giardoglou T, Katsuya T, Bhatti KF, de Kleijn D, de Borst GJ, Kim EK, Adams HHH, Ikram MA, Zhu X, Asselbergs FW, Kraaijeveld AO, Beulens JWJ, Shu XO, Rallidis LS, Pedersen O, Hansen T, Mitchell P, Hewitt AW, Kähönen M, Pérusse L, Bouchard C, Tönjes A, Chen YI, Pennell CE, Mori TA, Lieb W, Franke A, Ohlsson C, Mellström D, Cho YS, Lee H, Yuan JM, Koh WP, Rhee SY, Woo JT, Heid IM, Stark KJ, Völzke H, Homuth G, Evans MK, Zonderman AB, Polasek O, Pasterkamp G, Hoefer IE, Redline S, Pahkala K, Oldehinkel AJ, Snieder H, Biino G, Schmidt R, Schmidt H, Chen YE, Bandinelli S, Dedoussis G, Thanaraj TA, Kardia SLR, Kato N, Schulze MB, Girotto G, Jung B, Böger CA, Joshi PK, Bennett DA, De Jager PL, Lu X, Mamakou V, Brown M, Caulfield MJ, Munroe PB, Guo X, Ciullo M, Jonas JB, Samani NJ, Kaprio J, Pajukanta P, Adair LS, Bechayda SA, de Silva HJ, Wickremasinghe AR, Krauss RM, Wu JY, Zheng W, den Hollander AI, Bharadwaj D, Correa A, Wilson JG, Lind L, Heng CK, Nelson AE, Golightly YM, Wilson JF, Penninx B, Kim HL, Attia J, Scott RJ, Rao DC, Arnett DK, Hunt SC, Walker M, Koistinen HA, Chandak GR, Yajnik CS, Mercader JM, Tusié-Luna T, Aguilar-Salinas CA, Villalpando CG, Orozco L, Fornage M, Tai ES, van Dam RM, Lehtimäki T, Chaturvedi N, Yokota M, Liu J, Reilly DF, McKnight AJ, Kee F, Jöckel KH, McCarthy MI, Palmer CNA, Vitart V, Hayward C, Simonsick E, van Duijn CM, Lu F, Qu J, Hishigaki H, Lin X, März W, Parra EJ, Cruz M, Gudnason V, Tardif JC, Lettre G, 't Hart LM, Elders PJM, Damrauer SM, Kumari M, Kivimaki M, van der Harst P, Spector TD, Loos RJF, Province MA, Psaty BM, Brandslund I, Pramstaller PP, Christensen K, Ripatti S, Widén E, Hakonarson H, Grant SFA, Kiemeney LALM, de Graaf J, Loeffler M, Kronenberg F, Gu D, Erdmann J, Schunkert H, Franks PW, Linneberg A, Jukema JW, Khera AV, Männikkö M, Jarvelin MR, Kutalik Z, Cucca F, Mook-Kanamori DO, van Dijk KW, Watkins H, Strachan DP, Grarup N, Sever P, Poulter N, Rotter JI, Dantoft TM, Karpe F, Neville MJ, Timpson NJ, Cheng CY, Wong TY, Khor CC, Sabanayagam C, Peters A, Gieger C, Hattersley AT, Pedersen NL, Magnusson PKE, Boomsma DI, de Geus EJC, Cupples LA, van Meurs JBJ, Ghanbari M, Gordon-Larsen P, Huang W, Kim YJ, Tabara Y, Wareham NJ, Langenberg C, Zeggini E, Kuusisto J, Laakso M, Ingelsson E, Abecasis G, Chambers JC, Kooner JS, de Vries PS, Morrison AC, North KE, Daviglus M, Kraft P, Martin NG, Whitfield JB, Abbas S, Saleheen D, Walters RG, Holmes MV, Black C, Smith BH, Justice AE, Baras A, Buring JE, Ridker PM, Chasman DI, Kooperberg C, Wei WQ, Jarvik GP, Namjou B, Hayes MG, Ritchie MD, Jousilahti P, Salomaa V, Hveem K, Åsvold BO, Kubo M, Kamatani Y, Okada Y, Murakami Y, Thorsteinsdottir U, Stefansson K, Ho YL, Lynch JA, Rader DJ, Tsao PS, Chang KM, Cho K, O'Donnell CJ, Gaziano JM, Wilson P, Rotimi CN, Hazelhurst S, Ramsay M, Trembath RC, van Heel DA, Tamiya G, Yamamoto M, Kim BJ, Mohlke KL, Frayling TM, Hirschhorn JN, Kathiresan S, Boehnke M, Natarajan P, Peloso GM, Brown CD, Morris AP, Assimes TL, Deloukas P, Sun YV, and Willer CJ
  • Nature [Nature] 2023 Jun; Vol. 618 (7965), pp. E19-E20.

8. Left Atrial Function in Patients with Titin Cardiomyopathy. [2023]

  • Henkens MTHM, Raafs AG, Vanloon T, Vos JL, Vandenwijngaard A, Brunner HG, Krapels IPC, Knackstedt C, Gerretsen S, Hazebroek MR, Vernooy K, Nijveldt R, Lumens J, and Verdonschot JAJ
  • Journal of cardiac failure [J Card Fail] 2023 May 23. Date of Electronic Publication: 2023 May 23.
Abstract
Background: Truncating variants in titin (TTNtv) are the most prevalent genetic etiology of dilated cardiomyopathy (DCM). Although TTNtv has been associated with atrial fibrillation, it remains unknown whether and how left atrial (LA) function differs between patients with DCM with and without TTNtv. We aimed to determine and compare LA function in patients with DCM with and without TTNtv and to evaluate whether and how left ventricular (LV) function affects the LA using computational modeling.
Methods and Results: Patients with DCM from the Maastricht DCM registry that underwent genetic testing and cardiovascular magnetic resonance (CMR) were included in the current study. Subsequent computational modeling (CircAdapt model) was performed to identify potential LV and LA myocardial hemodynamic substrates. In total, 377 patients with DCM (n = 42 with TTNtv, n = 335 without a genetic variant) were included (median age 55 years, interquartile range [IQR] 46-62 years, 62% men). Patients with TTNtv had a larger LA volume and decreased LA strain compared with patients without a genetic variant (LA volume index 60 mLm -2 [IQR 49-83] vs 51 mLm -2 [IQR 42-64]; LA reservoir strain 24% [IQR 10-29] vs 28% [IQR 20-34]; LA booster strain 9% [IQR 4-14] vs 14% [IQR 10-17], respectively; all P < .01). Computational modeling suggests that while the observed LV dysfunction partially explains the observed LA dysfunction in the patients with TTNtv, both intrinsic LV and LA dysfunction are present in patients with and without a TTNtv.
Conclusions: Patients with DCM with TTNtv have more severe LA dysfunction compared with patients without a genetic variant. Insights from computational modeling suggest that both intrinsic LV and LA dysfunction are present in patients with DCM with and without TTNtv.
Competing Interests: Declaration of Competing Interest The authors have no conflict of interest.
(Copyright © 2023 The Authors. Published by Elsevier Inc. All rights reserved.)

9. Does believing something to be fiction allow a form of moral licencing or a 'fictive pass' in understanding others' actions? [2023]

  • Thompson J, Teasdale B, van Emde Boas E, Budelmann F, Duncan S, Maguire L, and Dunbar R
  • Frontiers in psychology [Front Psychol] 2023 May 15; Vol. 14, pp. 1159866. Date of Electronic Publication: 2023 May 15 (Print Publication: 2023).
Abstract
Introduction: The human capacity to engage with fictional worlds raises important psychological questions about the mechanisms that make this possible. Of particular interest is whether people respond differently to fictional stories compared to factual ones in terms of how immersed they become and how they view the characters involved and their actions. It has been suggested that fiction provides us with a 'fictive pass' that allows us to evaluate in a more balanced, detached way the morality of a character's behaviour.
Methods: We use a randomised controlled experimental design to test this.
Results and Discussion: We show that, although knowing whether a substantial film clip is fact or fiction does not affect how engaged with ('transported' by) a troubling story an observer becomes, it does grant them a 'fictive pass' to empathise with a moral transgressor. However, a fictive pass does not override the capacity to judge the causes of a character's moral transgression (at least as indexed by a causal attribution task).
Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.
(Copyright © 2023 Thompson, Teasdale, van Emde Boas, Budelmann, Duncan, Maguire and Dunbar.)

10. Researching COVID to enhance recovery (RECOVER) pediatric study protocol: Rationale, objectives and design. [2023]

  • Gross R, Thaweethai T, Rosenzweig EB, Chan J, Chibnik LB, Cicek MS, Elliott AJ, Flaherman VJ, Foulkes AS, Witvliet MG, Gallagher R, Gennaro ML, Jernigan TL, Karlson EW, Katz SD, Kinser PA, Kleinman LC, Lamendola-Essel MF, Milner JD, Mohandas S, Mudumbi PC, Newburger JW, Rhee KE, Salisbury AL, Snowden JN, Stein CR, Stockwell MS, Tantisira KG, Thomason ME, Truong DT, Warburton D, Wood JC, Ahmed S, Akerlundh A, Alshawabkeh AN, Anderson BR, Aschner JL, Atz AM, Aupperle RL, Baker FC, Balaraman V, Banerjee D, Barch DM, Baskin-Sommers A, Bhuiyan S, Bind MC, Bogie AL, Buchbinder NC, Bueler E, Bükülmez H, Casey BJ, Chang L, Clark DB, Clifton RG, Clouser KN, Cottrell L, Cowan K, D'Sa V, Dapretto M, Dasgupta S, Dehority W, Dummer KB, Elias MD, Esquenazi-Karonika S, Evans DN, Faustino EVS, Fiks AG, Forsha D, Foxe JJ, Friedman NP, Fry G, Gaur S, Gee DG, Gray KM, Harahsheh AS, Heath AC, Heitzeg MM, Hester CM, Hill S, Hobart-Porter L, Hong TKF, Horowitz CR, Hsia DS, Huentelman M, Hummel KD, Iacono WG, Irby K, Jacobus J, Jacoby VL, Jone PN, Kaelber DC, Kasmarcak TJ, Kluko MJ, Kosut JS, Laird AR, Landeo-Gutierrez J, Lang SM, Larson CL, Lim PPC, Lisdahl KM, McCrindle BW, McCulloh RJ, Mendelsohn AL, Metz TD, Morgan LM, Müller-Oehring EM, Nahin ER, Neale MC, Ness-Cochinwala M, Nolan SM, Oliveira CR, Oster ME, Payne RM, Raissy H, Randall IG, Rao S, Reeder HT, Rosas JM, Russell MW, Sabati AA, Sanil Y, Sato AI, Schechter MS, Selvarangan R, Shakti D, Sharma K, Squeglia LM, Stevenson MD, Szmuszkovicz J, Talavera-Barber MM, Teufel RJ 2nd, Thacker D, Udosen MM, Warner MR, Watson SE, Werzberger A, Weyer JC, Wood MJ, Yin HS, Zempsky WT, Zimmerman E, and Dreyer BP
  • MedRxiv : the preprint server for health sciences [medRxiv] 2023 May 12. Date of Electronic Publication: 2023 May 12.
Abstract
Importance: The prevalence, pathophysiology, and long-term outcomes of COVID-19 (post-acute sequelae of SARS-CoV-2 [PASC] or "Long COVID") in children and young adults remain unknown. Studies must address the urgent need to define PASC, its mechanisms, and potential treatment targets in children and young adults.
Observations: We describe the protocol for the Pediatric Observational Cohort Study of the NIH's RE searching COV ID to E nhance R ecovery (RECOVER) Initiative. RECOVER-Pediatrics is an observational meta-cohort study of caregiver-child pairs (birth through 17 years) and young adults (18 through 25 years), recruited from more than 100 sites across the US. This report focuses on two of five cohorts that comprise RECOVER-Pediatrics: 1) a de novo RECOVER prospective cohort of children and young adults with and without previous or current infection; and 2) an extant cohort derived from the Adolescent Brain Cognitive Development (ABCD) study ( n =10,000). The de novo cohort incorporates three tiers of data collection: 1) remote baseline assessments (Tier 1, n=6000); 2) longitudinal follow-up for up to 4 years (Tier 2, n=6000); and 3) a subset of participants, primarily the most severely affected by PASC, who will undergo deep phenotyping to explore PASC pathophysiology (Tier 3, n=600). Youth enrolled in the ABCD study participate in Tier 1. The pediatric protocol was developed as a collaborative partnership of investigators, patients, researchers, clinicians, community partners, and federal partners, intentionally promoting inclusivity and diversity. The protocol is adaptive to facilitate responses to emerging science.
Conclusions and Relevance: RECOVER-Pediatrics seeks to characterize the clinical course, underlying mechanisms, and long-term effects of PASC from birth through 25 years old. RECOVER-Pediatrics is designed to elucidate the epidemiology, four-year clinical course, and sociodemographic correlates of pediatric PASC. The data and biosamples will allow examination of mechanistic hypotheses and biomarkers, thus providing insights into potential therapeutic interventions.
Clinical Trialsgov Identifier: Clinical Trial Registration: http://www.clinicaltrials.gov . Unique identifier: NCT05172011.

11. Is there equity of patient health outcomes across models of general practice in Aotearoa New Zealand? A national cross-sectional study [2023]

  • Sheridan, Nicolette, Love, Tom, Kenealy, Timothy, Aguirre-Duarte, Nelson, Arroll, Bruce, Atmore, Carol, Carryer, Jenny, Crampton, Peter, Dowell, Anthony, Fishman, Tana, Gauld, Robin, Harwood, Matire, Hoare, Karen, Jackson, Gary, Jansen, Rawiri McKree, Kerse, Ngaire, Lampshire, Debra, McBain, Lynn, MacRae, Jayden, Mills, Jane, Øvretveit, John, Percival, Teuila, Perera, Roshan, Roland, Martin, Ryan, Debbie, Schmidt-Busby, Jacqueline, Stokes, Tim, Stubbe, Maria, Hewitt, Sarah, Watt, Daniel, and Peck, Chris
  • International Journal for Equity in Health. May 4, 2023, Vol. 22 Issue 1

12. MP-453090-9 FINDINGS FROM TWELVE YEARS OF FAMILY SCREENING AFTER SUDDEN CARDIAC DEATH. [2023]

  • Brown, Stewart, Bailey, Janice, Wang, Robin, Kemp, Jacqueline, Strawbridge, Martyn, Sheppard, Mary, Dayer, Mark J., and Furniss, Guy O.
  • Heart Rhythm; 2023 Supplement, Vol. 20 Issue 5, pS117-S118, 2p

13. Patient-reported outcome measures and patient engagement in heart failure clinical trials: multi-stakeholder perspectives. [2023]

  • Zannad F, Alikhaani J, Alikhaani S, Butler J, Gordon J, Jensen K, Khatib R, Mantovani L, Martinez R, Moore WF, Murakami M, Roessig L, Stockbridge N, Van Spall HGC, Yancy C, and Spertus JA
  • European journal of heart failure [Eur J Heart Fail] 2023 Apr; Vol. 25 (4), pp. 478-487. Date of Electronic Publication: 2023 Mar 22.
Subjects
Humans, Patient Participation, Patient Reported Outcome Measures, Caregivers, Quality of Life, and Heart Failure therapy
Abstract
There are many consequences of heart failure (HF), including symptoms, impaired health-related quality of life (HRQoL), and physical and social limitations (functional status). These have a substantial impact on patients' lives, yet are not routinely captured in clinical trials. Patient-reported outcomes (PROs) can quantify patients' experiences of their disease and its treatment. Steps can be taken to improve the use of PROs in HF trials, in regulatory and payer decisions, and in patient care. Importantly, PRO measures (PROMs) must be developed with involvement of patients, family members, and caregivers from diverse demographic groups and communities. PRO data collection should become more routine not only in clinical trials but also in clinical practice. This may be facilitated by the use of digital tools and interdisciplinary patient advocacy efforts. There is a need for standardization, not only of the PROM instruments, but also in procedures for analysis, interpretation and reporting PRO data. More work needs to be done to determine the degree of change that is important to patients and that is associated with increased risks of clinical events. This 'minimal clinically important difference' requires further research to determine thresholds for different PROMs, to assess consistency across trial populations, and to define standards for improvement that warrant regulatory and reimbursement approvals. PROs are a vital part of patient care and drug development, and more work should be done to ensure that these measures are both reflective of the patient experience and that they are more widely employed.
(© 2023 European Society of Cardiology.)

14. Implementation of a Web-Based Communication System for Primary Care Providers and Cancer Specialists. [2023]

  • Petrovic B, Bender JL, Liddy C, Afkham A, McGee SF, Morgan SC, Segal R, O'Brien MA, Julian JA, Sussman J, Urquhart R, Fitch M, Schneider ND, and Grunfeld E
  • Current oncology (Toronto, Ont.) [Curr Oncol] 2023 Mar 21; Vol. 30 (3), pp. 3537-3548. Date of Electronic Publication: 2023 Mar 21.
Subjects
Humans, Continuity of Patient Care, Surveys and Questionnaires, Communication, Internet, and Neoplasms therapy
Abstract
Healthcare providers have reported challenges with coordinating care for patients with cancer. Digital technology tools have brought new possibilities for improving care coordination. A web- and text-based asynchronous system (eOncoNote) was implemented in Ottawa, Canada for cancer specialists and primary care providers (PCPs). This study aimed to examine PCPs' experiences of implementing eOncoNote and how access to the system influenced communication between PCPs and cancer specialists. As part of a larger study, we collected and analyzed system usage data and administered an end-of-discussion survey to understand the perceived value of using eOncoNote. eOncoNote data were analyzed for 76 shared patients (33 patients receiving treatment and 43 patients in the survivorship phase). Thirty-nine percent of the PCPs responded to the cancer specialist's initial eOncoNote message and nearly all of those sent only one message. Forty-five percent of the PCPs completed the survey. Most PCPs reported no additional benefits of using eOncoNote and emphasized the need for electronic medical record (EMR) integration. Over half of the PCPs indicated that eOncoNote could be a helpful service if they had questions about a patient. Future research should examine opportunities for EMR integration and whether additional interventions could support communication between PCPs and cancer specialists.

15. Patterns of failure in pediatric medulloblastoma and implications for hippocampal sparing. [2023]

  • Baliga S, Adams JA, Bajaj BVM, Van Benthuysen L, Daartz J, Gallotto SL, Lewy JR, DeNunzio N, Weyman EA, Lawell MP, Palmer JD, Yeap BY, Ebb DH, Huang MS, Perry AF, MacDonald SM, Jones RM, Tarbell NJ, and Yock TI
  • Cancer [Cancer] 2023 Mar 01; Vol. 129 (5), pp. 764-770. Date of Electronic Publication: 2022 Dec 11.
Subjects
Humans, Child, Organ Sparing Treatments methods, Organs at Risk, Protons, Prospective Studies, Radiotherapy Planning, Computer-Assisted methods, Radiotherapy Dosage, Cranial Irradiation adverse effects, Cranial Irradiation methods, Neoplasm Recurrence, Local epidemiology, Hippocampus diagnostic imaging, Medulloblastoma radiotherapy, Brain Neoplasms radiotherapy, Brain Neoplasms secondary, Radiotherapy, Intensity-Modulated methods, and Cerebellar Neoplasms radiotherapy
Abstract
Background: Hippocampal avoidance (HA) has been shown to preserve cognitive function in adult patients with cancer treated with whole-brain radiation therapy for brain metastases. However, the feasibility of HA in pediatric patients with brain tumors has not been explored because of concerns of increased risk of relapse in the peri-hippocampal region. Our aim was to determine patterns of recurrence and incidence of peri-hippocampal relapse in pediatric patients with medulloblastoma (MB).
Methods and Materials: We identified pediatric patients with MB treated with protons between 2002 and 2016 and who had recurrent disease. To estimate the risk of peri-hippocampal recurrence, three hippocampal zones (HZs) were delineated corresponding to ≤5 mm (HZ-1), 6 to 10 mm (HZ-2), and >10 mm (HZ-3) distance of the recurrence from the contoured hippocampi. To determine the feasibility of HA, three standard-risk patients with MB were planned using either volumetric-modulated arc therapy (VMAT) or intensity-modulated proton therapy (IMPT) plans.
Results: Thirty-eight patients developed a recurrence at a median of 1.6 years. Of the 25 patients who had magnetic resonance imaging of the recurrence, no patients failed within the hippocampus and only two patients failed within HZ-1. The crude incidence of peri-hippocampal failure was 8%. Both HA-VMAT and HA-IMPT plans were associated with significantly reduced mean dose to the hippocampi (p < .05). HA-VMAT and HA-IMPT plans were associated with decreased percentage of the third and lateral ventricles receiving the prescription craniospinal dose of 23.4 Gy.
Conclusions: Peri-hippocampal failures are uncommon in pediatric patients with MB. Hippocampal avoidance should be evaluated in a prospective cohort of pediatric patients with MB.
Plain Language Summary: In this study, the patterns of disease recurrence in patients with a pediatric brain tumor known as medulloblastoma treated with proton radiotherapy were examined. The majority of failures occur outside of an important structure related to memory formation called the hippocampus. Hippocampal sparing radiation plans using proton radiotherapy were generated and showed that dose to the hippocampus was able to be significantly reduced. The study provides the rationale to explore hippocampal sparing in pediatric medulloblastoma in a prospective clinical trial.
(© 2022 The Authors. Cancer published by Wiley Periodicals LLC on behalf of American Cancer Society.)

16. Volume of subcortical brain regions in social anxiety disorder: mega-analytic results from 37 samples in the ENIGMA-Anxiety Working Group. [2023]

  • Groenewold NA, Bas-Hoogendam JM, Amod AR, Laansma MA, Van Velzen LS, Aghajani M, Hilbert K, Oh H, Salas R, Jackowski AP, Pan PM, Salum GA, Blair JR, Blair KS, Hirsch J, Pantazatos SP, Schneier FR, Talati A, Roelofs K, Volman I, Blanco-Hinojo L, Cardoner N, Pujol J, Beesdo-Baum K, Ching CRK, Thomopoulos SI, Jansen A, Kircher T, Krug A, Nenadić I, Stein F, Dannlowski U, Grotegerd D, Lemke H, Meinert S, Winter A, Erb M, Kreifelts B, Gong Q, Lui S, Zhu F, Mwangi B, Soares JC, Wu MJ, Bayram A, Canli M, Tükel R, Westenberg PM, Heeren A, Cremers HR, Hofmann D, Straube T, Doruyter AGG, Lochner C, Peterburs J, Van Tol MJ, Gur RE, Kaczkurkin AN, Larsen B, Satterthwaite TD, Filippi CA, Gold AL, Harrewijn A, Zugman A, Bülow R, Grabe HJ, Völzke H, Wittfeld K, Böhnlein J, Dohm K, Kugel H, Schrammen E, Zwanzger P, Leehr EJ, Sindermann L, Ball TM, Fonzo GA, Paulus MP, Simmons A, Stein MB, Klumpp H, Phan KL, Furmark T, Månsson KNT, Manzouri A, Avery SN, Blackford JU, Clauss JA, Feola B, Harper JC, Sylvester CM, Lueken U, Veltman DJ, Winkler AM, Jahanshad N, Pine DS, Thompson PM, Stein DJ, and Van der Wee NJA
  • Molecular psychiatry [Mol Psychiatry] 2023 Mar; Vol. 28 (3), pp. 1079-1089. Date of Electronic Publication: 2023 Jan 19.
Subjects
Adult, Adolescent, Humans, Magnetic Resonance Imaging methods, Brain, Anxiety, Neuroimaging methods, and Phobia, Social
Abstract
There is limited convergence in neuroimaging investigations into volumes of subcortical brain regions in social anxiety disorder (SAD). The inconsistent findings may arise from variations in methodological approaches across studies, including sample selection based on age and clinical characteristics. The ENIGMA-Anxiety Working Group initiated a global mega-analysis to determine whether differences in subcortical volumes can be detected in adults and adolescents with SAD relative to healthy controls. Volumetric data from 37 international samples with 1115 SAD patients and 2775 controls were obtained from ENIGMA-standardized protocols for image segmentation and quality assurance. Linear mixed-effects analyses were adjusted for comparisons across seven subcortical regions in each hemisphere using family-wise error (FWE)-correction. Mixed-effects d effect sizes were calculated. In the full sample, SAD patients showed smaller bilateral putamen volume than controls (left: d = -0.077, p FWE  = 0.037; right: d = -0.104, p FWE  = 0.001), and a significant interaction between SAD and age was found for the left putamen (r = -0.034, p FWE  = 0.045). Smaller bilateral putamen volumes (left: d = -0.141, p FWE  < 0.001; right: d = -0.158, p FWE  < 0.001) and larger bilateral pallidum volumes (left: d = 0.129, p FWE  = 0.006; right: d = 0.099, p FWE  = 0.046) were detected in adult SAD patients relative to controls, but no volumetric differences were apparent in adolescent SAD patients relative to controls. Comorbid anxiety disorders and age of SAD onset were additional determinants of SAD-related volumetric differences in subcortical regions. To conclude, subtle volumetric alterations in subcortical regions in SAD were detected. Heterogeneity in age and clinical characteristics may partly explain inconsistencies in previous findings. The association between alterations in subcortical volumes and SAD illness progression deserves further investigation, especially from adolescence into adulthood.
(© 2023. The Author(s), under exclusive licence to Springer Nature Limited.)

17. The Dutch Lung Cancer Audit-Radiotherapy (DLCA-R): Real-World Data on Stage III Non-Small Cell Lung Cancer Patients Treated With Curative Chemoradiation. [2023]

  • Dieleman E, van der Woude L, van Os R, van Bockel L, Coremans I, van Es C, De Jaeger K, Knol HP, Kolff W, Koppe F, Pomp J, Reymen B, Schinagl D, Spoelstra F, Tissing-Tan C, van der Voort van Zyp N, van der Wel A, Wijsman R, Dielwart M, Wiegman E, Damhuis R, and Belderbos J
  • Clinical lung cancer [Clin Lung Cancer] 2023 Mar; Vol. 24 (2), pp. 130-136. Date of Electronic Publication: 2022 Nov 25.
Subjects
Humans, Male, Aged, Infant, Neoplasm Staging, Chemoradiotherapy adverse effects, Carcinoma, Non-Small-Cell Lung drug therapy, Lung Neoplasms drug therapy, and Radiation Oncology
Abstract
Introduction: Chemoradiotherapy (CRT) is the standard of care in inoperable non-small-cell lung cancer (NSCLC) patients, favoring concurrent (cCRT) over sequential CRT (seqCRT), with adjuvant immunotherapy in responders. Elderly and frail NSCLC patients have generally been excluded from trials in the past. In elderly patients however, the higher treatment related morbidity of cCRT, may outweigh the possible lower tumor control of seqCRT. For elderly patients with locally advanced NSCLC real-world data is essential to be able to balance treatment toxicity and treatment outcome. The aim of this study is to analyze acute toxicity and 3-month mortality of curative chemoradiation (CRT) in patients with stage III NSCLC and to analyze whether cCRT for elderly stage III NSCLC patients is safe.
Methods: The Dutch Lung Cancer Audit-Radiotherapy (DLCA-R) is a national lung cancer audit that started in 2013 for patients treated with curative intent radiotherapy. All Dutch patients treated for stage III NSCLC between 2015 and 2018 with seqCRT or cCRT for (primary or recurrent) stage III lung cancer are included in this population-based study. Information was collected on patient, tumor- and treatment characteristics and the incidence and severity of acute non-hematological toxicity (CTCAE-4 version 4.03) and mortality within 3 months after the end of radiotherapy. To evaluate the association between prognostic factors and outcome (acute toxicity and mortality within 3 months), an univariable and multivariable analysis was performed. The definition of cCRT was:radiotherapy started within 30 days after the start of chemotherapy.
Results: Out of all 20 Dutch departments of radiation oncology, 19 centers participated in the registry. A total of 2942 NSCLC stage III patients were treated with CRT. Of these 67.2% (n = 1977) were treated with cCRT (median age 66 years) and 32.8% (n = 965) were treated with seqCRT (median age 69 years). Good performance status (WHO 0-1) was scored in 88.6% for patients treated with cCRT and in 71.0% in the patients treated with seqCRT. Acute nonhematological 3-month toxicity (CTCAE grade ≥3 or radiation pneumonitis grade ≥2) was scored in 21.9% of the patients treated with cCRT and in 17.7% of the patients treated with seqCRT. The univariable analysis for acute toxicity showed significantly increased toxicity for cCRT (P = .008), WHO ≥2 (P = .006), and TNM IIIC (P = .031). The multivariable analysis for acute toxicity was significant for cCRT (P = .015), WHO ≥2 (P = .001) and TNM IIIC (P = .016). The univariable analysis for 3-month mortality showed significance for seqCRT (P = .025), WHO ≥2 (P < .001), higher cumulative radiotherapy dose (P < .001), higher gross tumor volume total (P = .020) and male patients (p < .001). None of these variables reached significance in the multivariable analysis for 3-month mortality.
Conclusion: In this national lung cancer audit of inoperable NSCLC patients, 3-month toxicity was significantly higher in patients treated with cCRT (21.9% vs. 17.7% for seqCRT) higher TNM stage IIIC, and poor performance (WHO≥2) patients.The 3-months mortality was not significantly different for tested parameters. Age was not a risk factor for acute toxicity, nor 3 months mortality.
(Copyright © 2022 The Author(s). Published by Elsevier Inc. All rights reserved.)

18. Kinetics of the Antibody Response to Symptomatic SARS-CoV-2 Infection in Vaccinated and Unvaccinated Individuals in the Blinded Phase of the mRNA-1273 COVID-19 Vaccine Efficacy Trial. [2023]

  • Follmann, Dean, Janes, Holly E, Chu, Eric, Jayashankar, Lakshmi, Petropoulos, Christos J, Serebryannyy, Leonid, Carroll, Robin, Jean-Baptiste, Naz, Narpala, Sandeep, Lin, Bob C, McDermott, Adrian, Novak, Richard M, Graciaa, Daniel S, Rolsma, Stephanie, Magaret, Craig A, Doria-Rose, Nicole, Corey, Lawrence, Neuzil, Kathleen M, Pajon, Rolando, and Miller, Jacqueline M
  • Open Forum Infectious Diseases. Mar2023, Vol. 10 Issue 3, p1-8. 8p.
Abstract
Background Hybrid immunity is associated with more durable protection against coronavirus disease 2019 (COVID-19). We describe the antibody responses following severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection in vaccinated and unvaccinated individuals. Methods The 55 vaccine arm COVID-19 cases diagnosed during the blinded phase of the Coronavirus Efficacy trial were matched with 55 placebo arm COVID-19 cases. Pseudovirus neutralizing antibody (nAb) activity to the ancestral strain and binding antibody (bAb) responses to nucleocapsid and spike antigens (ancestral and variants of concern [VOCs]) were assessed on disease day 1 (DD1) and 28 days later (DD29). Results The primary analysis set was 46 vaccine cases and 49 placebo cases with COVID-19 at least 57 days post–first dose. For vaccine group cases, there was a 1.88-fold rise in ancestral antispike bAbs 1 month post–disease onset, although 47% had no increase. The vaccine-to-placebo geometric mean ratios for DD29 antispike and antinucleocapsid bAbs were 6.9 and 0.04, respectively. DD29 mean bAb levels were higher for vaccine vs placebo cases for all VOCs. DD1 nasal viral load positively correlated with bAb levels in the vaccine group. Conclusions Following COVID-19, vaccinated participants had higher levels and greater breadth of antispike bAbs and higher nAb titers than unvaccinated participants. These were largely attributable to the primary immunization series. [ABSTRACT FROM AUTHOR]

19. Evaluation of left cardiac chamber function with cardiac magnetic resonance and association with outcome in patients with systemic sclerosis. [2023]

  • Butcher SC, Vos JL, Fortuni F, Galloo X, Liem SIE, Bax JJ, Delgado V, Vonk MC, van Leuven SI, Snoeren M, El Messaoudi S, de Vries-Bouwstra JK, Nijveldt R, and Ajmone Marsan N
  • Rheumatology (Oxford, England) [Rheumatology (Oxford)] 2023 Feb 06; Vol. 62 (SI), pp. SI20-SI31.
Subjects
Female, Humans, Male, Contrast Media, Gadolinium, Magnetic Resonance Imaging, Cine, Magnetic Resonance Spectroscopy, Predictive Value of Tests, Prognosis, Stroke Volume, Ventricular Function, Left, Middle Aged, Heart Failure, Scleroderma, Systemic complications, and Scleroderma, Systemic diagnostic imaging
Abstract
Objective: This study aimed to determine whether lower values of feature-tracking cardiovascular magnetic resonance (CMR)-derived left atrial reservoir strain (LARS) and impaired left ventricular (LV) global longitudinal strain (GLS) were associated with the presence of symptoms and long-term prognosis in patients with SSc.
Methods: A total of 100 patients {54 [interquartile range (IQR) 46-64] years, 42% male} with SSc who underwent CMR imaging at two tertiary referral centres were included. All patients underwent analysis of LARS and LV GLS using feature-tracking on CMR and were followed-up for the occurrence of all-cause mortality.
Results: The median LV GLS was -21.8% and the median LARS was 36%. On multivariable logistic regression, LARS [odds ratio (OR) 0.964 per %, 95% CI 0.929, 0.998, P = 0.049] was independently associated with New York Heart Association (NYHA) class II-IV heart failure symptoms. Over a median follow-up of 37 (21-62) months, a total of 24 (24%) patients died. Univariable Cox regression analysis demonstrated that LARS [hazard ratio (HR) 0.94 per 1%, 95% CI 0.91, 0.97, P < 0.0001) and LV GLS (HR 1.10 per %, 95% CI 1.03, 1.17, P = 0.005) were associated with all-cause mortality, while LV ejection fraction was not. Likelihood ratio tests demonstrated that LARS provided incremental value over prognostically important clinical and imaging parameters, including late gadolinium enhancement.
Conclusion: In patients with SSc, LARS was independently associated with the presence of NYHA class II-IV heart failure symptoms. Although both LARS and LV GLS were associated with all-cause mortality, only LARS provided incremental value over all evaluated variables known to be prognostically important in patients with SSc.
(© The Author(s) 2022. Published by Oxford University Press on behalf of the British Society for Rheumatology.)

20. A phase 1b study of venetoclax and azacitidine combination in patients with relapsed or refractory myelodysplastic syndromes. [2023]

  • Zeidan AM, Borate U, Pollyea DA, Brunner AM, Roncolato F, Garcia JS, Filshie R, Odenike O, Watson AM, Krishnadasan R, Bajel A, Naqvi K, Zha J, Cheng WH, Zhou Y, Hoffman D, Harb JG, Potluri J, and Garcia-Manero G
  • American journal of hematology [Am J Hematol] 2023 Feb; Vol. 98 (2), pp. 272-281. Date of Electronic Publication: 2022 Nov 10.
Subjects
Aged, Humans, Male, Azacitidine therapeutic use, Leukemia, Myeloid, Acute drug therapy, Neutropenia chemically induced, Sulfonamides, Treatment Outcome, Female, Antineoplastic Combined Chemotherapy Protocols adverse effects, and Myelodysplastic Syndromes drug therapy
Abstract
Patients with relapsed/refractory (R/R) higher-risk myelodysplastic syndromes (MDS) have a dismal median overall survival (OS) after failing hypomethylating agent (HMA) treatment. There is no standard of care for patients after HMA therapy failure; hence, there is a critical need for effective therapeutic strategies. Herein, we present the safety and efficacy of venetoclax + azacitidine in patients with R/R MDS. This phase 1b, open-label, multicenter study enrolled patients ≥18 years. Patients were treated with escalating doses of oral venetoclax: 100, 200, or 400 mg daily for 14 days every 28-day cycle. Azacitidine was administered on Days 1-7 every cycle at 75 mg/m 2 /day intravenously/subcutaneously. Responses were assessed per modified 2006 International Working Group (IWG) criteria. Forty-four patients (male 86%, median age 74 years) received venetoclax + azacitidine treatment. Median follow-up was 21.2 months. Hematological adverse events of Grade ≥ 3 included febrile neutropenia (34%), thrombocytopenia (32%), neutropenia (27%), and anemia (18%). Pneumonia (23%) was the most common Grade ≥ 3 infection. Marrow responses were seen including complete remission (CR, n = 3, 7%) and marrow CR (mCR, n = 14, 32%); 36% (16/44) achieved transfusion independence (TI) for RBCs and/or platelets, and 43% (6/14) with mCR achieved hematological improvement (HI). The median time to CR/mCR was 1.2 months, and the median duration of response for CR + mCR was 8.6 months. Median OS was 12.6 months. Venetoclax + azacitidine shows activity in patients with R/R MDS following prior HMA therapy failure and provides clinically meaningful benefits, including HI and TI, and encouraging OS.
(© 2022 The Authors. American Journal of Hematology published by Wiley Periodicals LLC.)

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