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2. Relapsing and refractory peritoneal dialysis peritonitis caused by Corynebacterium amycolatum

Author

Habeeb, Shameer M., Yamin, Haneen, Simkova, Eva, Awad, Hazem S., Alhammadi, Entesar A., Eid, Loai A., and Lone, Rubina

Subjects
Continuous ambulatory peritoneal dialysis -- Complications and side effects -- Patient outcomes, Children -- Diseases, Peritoneal dialysis -- Complications and side effects -- Patient outcomes, Actinomycetales infections -- Care and treatment -- Patient outcomes, Health
Abstract

Background Peritonitis is an important complication and cause of morbidity in patients undergoing peritoneal dialysis (PD). Corynebacterium species, often considered skin and mucosal contaminants, are a rare cause of PD-associated peritonitis and have been acknowledged in published guidelines for the diagnosis and treatment of PD peritonitis only over the last decade. Case-Diagnosis/Treatment We present two children with difficult-to-treat episodes of PD peritonitis due to Corynebacterium amycolatum. Episodes were associated with fever, abdominal pain and cloudy dialysate, high dialysate polymorphonuclear leukocyte counts, and elevated serum C-reactive protein and procalcitonin concentrations. Symptoms persisted beyond 5 days in 4 of 5 peritonitis episodes, and peritonitis relapsed despite in vitro sensitivity of the bacterial isolates to guideline-recommended antibiotics. C. amycolatum was cultured from the PD catheter tip despite 4 weeks of intraperitoneal glycopeptide therapy and clinical peritonitis resolution suggestive of efficient biofilm formation. Our systematic literature search identified three previous (adult) case descriptions of C. amycolatum peritonitis, all with repeat episodes by the same organism. The incidence of C. amycolatum as a cause of PD peritonitis has not yet been established but is likely underreported due to challenges in species differentiation. Conclusions C. amycolatum is a rarely identified cause of refractory and/or relapsing PD peritonitis. Species differentiation of non-diphtheriae Corynebacterium isolates is critical, and prolonged antibiotic treatment, preferably with a glycopeptide antibiotic, is recommended, with a low threshold for PD catheter change or removal in case of repeat peritonitis., Author(s): Shameer M. Habeeb [sup.1] , Haneen Yamin [sup.1] , Eva Simkova [sup.1] , Hazem S. Awad [sup.1] , Entesar A. Alhammadi [sup.1] [sup.2] , Loai A. Eid [sup.1] [sup.2] [...]

Published
2023
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3. Randomized Clinical Trial on the Long-Term Efficacy and Safety of Lumasiran in Patients With Primary Hyperoxaluria Type 1

Author

Hulton, Sally A., Groothoff, Jaap W., Frishberg, Yaacov, Koren, Michael J., Overcash, J. Scott, Sellier-Leclerc, Anne-Laure, Shasha-Lavsky, Hadas, Saland, Jeffrey M., Hayes, Wesley, Magen, Daniella, Moochhala, Shabbir H., Coenen, Martin, Simkova, Eva, Garrelfs, Sander F., Sas, David J., Meliambro, Kristin A., Ngo, Taylor, Sweetser, Marianne T., Habtemariam, Bahru A., Gansner, John M., McGregor, Tracy L., and Lieske, John C.

Published
2022
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5. Cognitive Process as a Tool of Tourists’ Typology for Rural Destinations

Author

Šimková, Eva, Muzikantová, Alena, Hutchison, David, Series editor, Kanade, Takeo, Series editor, Kittler, Josef, Series editor, Kleinberg, Jon M., Series editor, Mattern, Friedemann, Series editor, Mitchell, John C., Series editor, Naor, Moni, Series editor, Pandu Rangan, C., Series editor, Steffen, Bernhard, Series editor, Terzopoulos, Demetri, Series editor, Tygar, Doug, Series editor, Weikum, Gerhard, Series editor, Christiansen, Henning, editor, Stojanovic, Isidora, editor, and Papadopoulos, George A., editor

Published
2015
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6. Lumasiran for Advanced Primary Hyperoxaluria Type 1: Phase 3 ILLUMINATE-C Trial

Author

Michael, Mini, primary, Groothoff, Jaap W., additional, Shasha-Lavsky, Hadas, additional, Lieske, John C., additional, Frishberg, Yaacov, additional, Simkova, Eva, additional, Sellier-Leclerc, Anne-Laure, additional, Devresse, Arnaud, additional, Guebre-Egziabher, Fitsum, additional, Bakkaloglu, Sevcan A., additional, Mourani, Chebl, additional, Saqan, Rola, additional, Singer, Richard, additional, Willey, Richard, additional, Habtemariam, Bahru, additional, Gansner, John M., additional, Bhan, Ishir, additional, McGregor, Tracy, additional, and Magen, Daniella, additional

Published
2023
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11. LUMASIRAN FOR PATIENTS WITH PRIMARY HYPEROXALURIA TYPE 1 WITH IMPAIRED KIDNEY FUNCTION: DATA FROM THE 6-MONTH ANALYSIS OF THE PHASE 3 ILLUMINATE-C TRIAL

Author

Saqan, Rola, Sellier-Leclerc, Anne-Laure, BAKKALOĞLU EZGÜ, SEVCAN AZİME, Willey, Richard, Habtemariam, Bahru, Simkova, Eva, Frishberg, Yaacov, Lieske, John, Shasha-Lavsky, Hadas, Michael, Mini, Groothoff, Jaap, Magen, Daniella, Bhan, Ishir, Gansner, John, Guebre-Egziabher, Fitsum, Arnaud, Devresse, Mourani, Chebl, and Singer, Richard

Published
2022

12. PD05-03 EFFICACY AND SAFETY OF LUMASIRAN IN PATIENTS WITH PRIMARY HYPEROXALURIA TYPE 1: 24-MONTH ANALYSIS OF THE ILLUMINATE-A TRIAL

Author

Lieske, John, primary, Groothoff, Jaap, additional, Frishberg, Yaacov, additional, Sellier-Leclerc, Anne-Laure, additional, Shasha-Lavsky, Hadas, additional, Saland, Jeffrey, additional, Hayes, Wesley, additional, Magen, Daniella, additional, Moochhala, Shabbir, additional, Coenen, Martin, additional, Simkova, Eva, additional, Ngo, Taylor, additional, Gansner, John, additional, and Hulton, Sally-Anne, additional

Published
2022
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13. FC070: Lumasiran for Patients with Primary Hyperoxaluria Type 1 with Impaired Kidney Function: Data from the 6-Month Analysis of the Phase 3 Illuminate-C Trial

Author

Groothoff, Jaap, primary, Michael, Mini, additional, Shasha-Lavsky, Hadas, additional, Lieske, John, additional, Frishberg, Yaacov, additional, Simkova, Eva, additional, Sellier-Leclerc, Anne-Laure, additional, Arnaud, Devresse, additional, Guebre-Egziabher, Fitsum, additional, Azime Bakkaloglu, Sevcan, additional, Mourani, Chebl, additional, Saqan, Rola, additional, Singer, Richard, additional, Willey, Richard, additional, Habtemariam, Bahru, additional, Bhan, Ishir, additional, Gansner, John, additional, and Magen, Daniella, additional

Published
2022
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14. Long-Term Outcome of Steroid-Resistant Nephrotic Syndrome in Children

Author

Trautmann, Agnes, Schnaidt, Sven, Lipska-Ziętkiewicz, Beata S., Bodria, Monica, Ozaltin, Fatih, Emma, Francesco, Anarat, Ali, Melk, Anette, Azocar, Marta, Oh, Jun, Saeed, Bassam, Gheisari, Alaleh, Caliskan, Salim, Gellermann, Jutta, Higuita, Lina Maria Serna, Jankauskiene, Augustina, Drozdz, Dorota, Mir, Sevgi, Balat, Ayse, Szczepanska, Maria, Paripovic, Dusan, Zurowska, Alexandra, Bogdanovic, Radovan, Yilmaz, Alev, Ranchin, Bruno, Baskin, Esra, Erdogan, Ozlem, Remuzzi, Giuseppe, Firszt-Adamczyk, Agnieszka, Kuzma-Mroczkowska, Elzbieta, Litwin, Mieczyslaw, Murer, Luisa, Tkaczyk, Marcin, Jardim, Helena, Wasilewska, Anna, Printza, Nikoleta, Fidan, Kibriya, Simkova, Eva, Borzecka, Halina, Staude, Hagen, Hees, Katharina, Schaefer, Franz, Azocar, M, Quiroz, Lily, Higuita, LM Serna, Dušek, Jiří, Ranchin, B, Fischbach, Michel, Davitaia, Tinatin, Gellerman, J, Oh, J, Melk, A, Schaefer, F, Wigger, Marianne, Printza, N, Sallay, Peter, Gheissari, Alaleh, Noris, Marina, Pasini, Andrea, Ghiggeri, Gian Marco, Ardissino, Gianluigi, Benetti, Elisa, Emma, F, Aoun, Bilal, Abou-Jaoudé, Pauline, Jankauskiene, A, Wasilewska, A, Gacka, Ewa, Zurowska, A, Drozdz, D, Tkaczyk, M, Lodz, Małgorzata Stańczyk, Borzecka, H, Silska, Magdalena, Jarmolinski, Tomasz, Firszt-Adamczyk, A, Ksiazek, Joanna, Kuzma-Mroczkowska, E, Medynska, Anna, Szczepanska, M, Afonso, Alberto Caldas, Jardim, H, Belgrade, Amira Peco-Antic, Bogdanovic, R, Krmar, Rafael T, Simonetti, Giacomo D, Saeed, B, Anarat, A, Balat, A, Baskin, E, Cakar, Nilgun, Erdogan, O, Özcakar, Birsin, Ozaltin, F, Sakallioglu, Onur, Soylemezoglu, Oguz, Akman, Sema, Gok, Faysal, Caliskan, S, Candan, Cengiz, Yilmaz, A, Mir, S, Akil, Ipek, Ertan, Pelin, Özkaya, Ozan, Kalyoncu, Mukaddes, Simkova, E, Alhammadi, Entesar, and Sobko, Roman

Published
2017
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15. The Phenotypic Spectrum of Nephropathies Associated with Mutations in Diacylglycerol Kinase ε

Author

Azukaitis, Karolis, Simkova, Eva, Majid, Mohammad Abdul, Galiano, Matthias, Benz, Kerstin, Amann, Kerstin, Bockmeyer, Clemens, Gajjar, Radha, Meyers, Kevin E., Cheong, Hae Il, Lange-Sperandio, Bärbel, Jungraithmayr, Therese, Frémeaux-Bacchi, Véronique, Bergmann, Carsten, Bereczki, Csaba, Miklaszewska, Monika, Csuka, Dorottya, Prohászka, Zoltán, Gipson, Patrick, Sampson, Matthew G., Lemaire, Mathieu, and Schaefer, Franz

Published
2017
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16. Analyse à 12 mois d’ILLUMINATE-A, une étude de phase 3 du lumasiran : réduction durable de l’oxalate et des taux d’évènements de calculs rénaux dans l’hyperoxalurie primitive de type 1

Author

Saland, Jeffrey, primary, Groothoff, Jaap, additional, Frishberg, Yaacov, additional, Hulton, Sally, additional, Koren, Michael, additional, Overcash, J. Scott, additional, Sellier-Leclerc, Anne-Laure, additional, Deschenes, Georges, additional, Shasha-Lavsky, Hadas, additional, Hayes, Wesley, additional, Fuster, Daniel, additional, Magen, Daniella, additional, Moochhala, Shabbir H., additional, Coenen, Martin, additional, Simkova, Eva, additional, Garrelfs, Sander, additional, Sas, David, additional, Meliambro, Kristin, additional, Ngo, Taylor, additional, Fujita, Kenji, additional, Gansner, John, additional, McGregor, Tracy, additional, and Lieske, John, additional

Published
2021
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17. MP07-09 12-MONTH ANALYSIS OF ILLUMINATE-A, A PHASE 3 STUDY OF LUMASIRAN: SUSTAINED OXALATE LOWERING AND KIDNEY STONE EVENT RATES IN PRIMARY HYPEROXALURIA TYPE 1

Author

Assimos, Dean, primary, Saland, Jeffrey M, additional, Groothoff, Jaap, additional, Frishberg, Yaacov, additional, Hulton, Sally-Anne, additional, Koren, Michael, additional, Overcash, J. Scott, additional, Sellier-Leclerc, Anne-Laure, additional, Deschenes, Georges, additional, Shasha-Lavsky, Hadas, additional, Hayes, Wesley, additional, Fuster, Daniel, additional, Magen, Daniella, additional, Moochhala, Shabbir H., additional, Coenen, Martin, additional, Simkova, Eva, additional, Garrelfs, Sander F., additional, Sas, David J., additional, Meliambro, Kristin, additional, Ngo, Taylor, additional, Fujita, Kenji, additional, Gansner, John M., additional, McGregor, Tracy L., additional, and Lieske, John C., additional

Published
2021
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18. 12-Month Analysis of ILLUMINATE-A, a Phase 3 Study of Lumasiran: Sustained Oxalate Lowering and Kidney Stone Event Rates in Primary Hyperoxaluria Type 1

Author

UCL - SSS/IREC/NEFR - Pôle de Néphrologie, UCL - (SLuc) Service de néphrologie, Devresse, Arnaud, Saland, Jeffrey M, Groothoff, Jaap, Frishberg, Yaacov, Hulton, Sally-Anne, Koren, Michael, Overcash, J. Scott, Sellier-Leclerc, Anne-Laure, Deschenes, Georges, Shasha-Lavsky,Hadas, Hayes, Wesley, Fuster, Daniel, Magen, Daniella, Moochhala Shabbir H, Coenen, Martin, Simkova, Eva, Garrelfs, Sander F, Sas, David J, Meliambro, Kristin, Ngo, Taylor, Fujita, Kenji, Gansner, John M, McGreogr Tracy L, Lieske, John C, SBN, UCL - SSS/IREC/NEFR - Pôle de Néphrologie, UCL - (SLuc) Service de néphrologie, Devresse, Arnaud, Saland, Jeffrey M, Groothoff, Jaap, Frishberg, Yaacov, Hulton, Sally-Anne, Koren, Michael, Overcash, J. Scott, Sellier-Leclerc, Anne-Laure, Deschenes, Georges, Shasha-Lavsky,Hadas, Hayes, Wesley, Fuster, Daniel, Magen, Daniella, Moochhala Shabbir H, Coenen, Martin, Simkova, Eva, Garrelfs, Sander F, Sas, David J, Meliambro, Kristin, Ngo, Taylor, Fujita, Kenji, Gansner, John M, McGreogr Tracy L, Lieske, John C, and SBN

Published
2021

19. Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1

Author

Garrelfs, Sander F., primary, Frishberg, Yaacov, additional, Hulton, Sally A., additional, Koren, Michael J., additional, O’Riordan, William D., additional, Cochat, Pierre, additional, Deschênes, Georges, additional, Shasha-Lavsky, Hadas, additional, Saland, Jeffrey M., additional, van’t Hoff, William G., additional, Fuster, Daniel G., additional, Magen, Daniella, additional, Moochhala, Shabbir H., additional, Schalk, Gesa, additional, Simkova, Eva, additional, Groothoff, Jaap W., additional, Sas, David J., additional, Meliambro, Kristin A., additional, Lu, Jiandong, additional, Sweetser, Marianne T., additional, Garg, Pushkal P., additional, Vaishnaw, Akshay K., additional, Gansner, John M., additional, McGregor, Tracy L., additional, and Lieske, John C., additional

Published
2021
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20. Global Variation of Nutritional Status in Children Undergoing Chronic Peritoneal Dialysis: A Longitudinal Study of the International Pediatric Peritoneal Dialysis Network

Author

Schaefer, Franz, Benner, Laura, Borzych-Duzalka, Dagmara, Zaritsky, Joshua, Xu, Hong, Rees, Lesley, Antonio, Zenaida L., Serdaroglu, Erkin, Hooman, Nakysa, Patel, Hiren, Sever, Lale, Vondrak, Karel, Flynn, Joseph, Rebori, Anabella, Wong, William, Holtta, Tuula, Yildirim, Zeynep Yuruk, Ranchin, Bruno, Grenda, Ryszard, Testa, Sara, Drozdz, Dorota, Szabo, Attila J., Eid, Loai, Basu, Biswanath, Vitkevic, Renate, Wong, Cynthia, Pottoore, Stephen J., Mueller, Dominik, Dusunsel, Ruhan, Celedon, Claudia Gonzalez, Fila, Marc, Sartz, Lisa, Sander, Anja, Warady, Bradley A., Adragna, M., Coccia, P. A., Suarez, A., Valles, P. G., Salim, R., Alconcher, L., Arbeiter, K., van Hoeck, K., Koch, V, Feber, J., Harvey, E., White, C., Valenzuela, M., Villagra, J., Cano, F., Contreras, M. A., Vogel, A., Zambrano, P., Hevia, P., Chiu, M. C., Ding, Jie, Vanegas, J. J., Higuita, L. M., Roussey, G., Ulinski, T., Krid, S., Fischbach, M., Harambat, J., Samaille, Ch, Buescher, R., Oh, J., Pape, L., John, U., Klaus, G., Billing, H., Stafanidis, C., Papachristou, F., Bagga, A., Kanitkar, M., Sinha, R., Sethi, S., Verrinam, E., Vidal, E., Leozappa, G., Landau, D., Paik, K. H., Bilal, A., Sahpazova, E., Lim, Y. N., Barbosa, L. Sanchez, Groothoff, J. W., Konijenberg, Y., Silva, Y., Al Ryami, M., Munarriz, R. Loza, Leszepanska, B., Szczepanska, M., Brumariu, O., Kari, J., Kruscic, D., Yap, H. K., Ariceta, G., Aguirre, M., Santos, F., Niwhiska-Faryna, B., Bayazit, A., Bakkaloglu, C. A. S., Bakkaloglu, S., Bilge, I, Yavascan, O., Mir, S., Simkova, Eva, Christian, M., Greenbaum, L., Neu, A., Askenazi, D., Al-Akash, A., Swartz, S., Brophy, P., Rheault, M., Pradhan, M., Pediatric surgery, Amsterdam Reproduction & Development (AR&D), Çukurova Üniversitesi, Büscher (R.), Rainer (Beitragende*r), Paediatric Nephrology, AGEM - Inborn errors of metabolism, APH - Methodology, APH - Quality of Care, ARD - Amsterdam Reproduction and Development, Ege Üniversitesi, Int Pediat Peritoneal Dial Network, Children's Hospital, Clinicum, and HUS Children and Adolescents

Subjects
Male, 0301 basic medicine, Pediatric Obesity, Longitudinal study, Pediatrics, medicine.medical_treatment, Medizin, lcsh:Medicine, Overweight, DISEASE, 0302 clinical medicine, Risk Factors, Prevalence, Longitudinal Studies, Registries, Child, lcsh:Science, RISK, 2. Zero hunger, OUTCOMES, Multidisciplinary, 3. Good health, Europe, OBESITY, Child, Preschool, GROWTH, Female, medicine.symptom, Underweight, Peritoneal Dialysis, Engineering sciences. Technology, medicine.medical_specialty, Asia, Adolescent, Nutritional Status, Article, Peritoneal dialysis, 03 medical and health sciences, Enteral Nutrition, Thinness, medicine, Humans, Dialysis, business.industry, MORTALITY, lcsh:R, Infant, nutritional and metabolic diseases, medicine.disease, Obesity, BODY-MASS INDEX, 030104 developmental biology, 3121 General medicine, internal medicine and other clinical medicine, Kidney Failure, Chronic, lcsh:Q, Americas, business, Body mass index, 030217 neurology & neurosurgery, Kidney disease
Abstract

WOS: 000461762600003, PubMed ID: 30894599, While children approaching end-stage kidney disease (ESKD) are considered at risk of uremic anorexia and underweight they are also exposed to the global obesity epidemic. We sought to investigate the variation of nutritional status in children undergoing chronic peritoneal dialysis (CPD) around the globe. The distribution and course of body mass index (BMI) standard deviation score over time was examined prospectively in 1001 children and adolescents from 35 countries starting CPD who were followed in the International Pediatric PD Network (IPPN) Registry. The overall prevalence of underweight, and overweight/obesity at start of CPD was 8.9% and 19.7%, respectively. Underweight was most prevalent in South and Southeast Asia (20%), Central Europe (16.7%) and Turkey (15.2%), whereas overweight and obesity were most common in the Middle East (40%) and the US (33%). BMI SDS at PD initiation was associated positively with current eGFR and gastrostomy feeding prior to PD start. Over the course of PD BMI SDS tended to increase on CPD in underweight and normal weight children, whereas it decreased in initially overweight patients. In infancy, mortality risk was amplified by obesity, whereas in older children mortality was markedly increased in association with underweight. Both underweight and overweight are prevalent in pediatric ESKD, with the prevalence varying across the globe. Late dialysis start is associated with underweight, while enteral feeding can lead to obesity. Nutritional abnormalities tend to attenuate with time on dialysis. Mortality risk appears increased with obesity in infants and with underweight in older children., International Society for Peritoneal Dialysis; Baxter Health Care; Fresenius Medical Care, The authors gratefully acknowledge the support by the International Society for Peritoneal Dialysis, Baxter Health Care, and Fresenius Medical Care. We also appreciate the continued dedicated support of the IPPN by the medical and nursing staff in all collaborating centers.

Published
2019

22. LB002ILLUMINATE-A, A PHASE 3 STUDY OF LUMASIRAN, AN INVESTIGATIONAL RNAI THERAPEUTIC, IN CHILDREN AND ADULTS WITH PRIMARY HYPEROXALURIA TYPE 1 (PH1)

Author

Garrelfs, Sander, primary, Frishberg, Yaacov, primary, Hulton, Sally, primary, Koren, Michael, primary, O'Riordan, William, primary, Cochat, Pierre, primary, Deschenes, Georges, primary, Shasha-Lavsky, Hadas, primary, Saland, Jeffrey, primary, Van't Hoff, William, primary, Fuster, Daniel Guido, primary, Magen, Daniella, primary, Moochhala, Shabbir, primary, Schalk, Gesa, primary, Simkova, Eva, primary, Groothoff, Jaap, primary, Sas, David, primary, Meliambro, Kristin, primary, Lu, Jiandong, primary, Garg, Pushkal, primary, Gansner, John, primary, McGregor, Tracy, primary, and Lieske, John, primary

Published
2020
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29. Lumasiran for Primary Hyperoxaluria Type 1 and Impaired Kidney Function: 24-Month Analysis of the Phase 3 ILLUMINATE-C Trial

Author

Lieske, John C., Magen, Daniella, Sellier-Leclerc, Anne-Laure A., Shasha-Lavsky, Hadas, Simkova, Eva, Devresse, Arnaud, Michael, Mini, Frishberg, Yaacov, Bakkaloglu, Sevcan A., Mourani, Chebl, Saqan, Rola, Singer, Richard F., Guzzo, Isabella, Makarova, Nune, Willey, Richard G., Kaspar, Cristin, Gansner, John M., and Groothoff, Jaap

Published
2023
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30. The Survey of the Main Trends of the ERP Applications in the Czech Republic and their Business Effects

Author

Basl Josef, Simkova Eva, and Pour Jan

Subjects
Czech, Process management, Scope (project management), media_common.quotation_subject, language.human_language, Information and Communications Technology, Informatics, language, Information system, ComputingMilieux_COMPUTERSANDSOCIETY, Quality (business), Business, Economic system, Enterprise information system, SWOT analysis, media_common
Abstract

The article deals with the analysis of the ERP market in the Czech Republic provided annually from 1993 (approx. 50 – 60 products each year). The main attention has been put on the functionality of the ERP but the scope is extended to the expected trends, SWOT analysis of the ERP products and market potential in the Czech Republic. The level of penetration of different ICT applications in the Czech companies and (mainly ERP, SCM,) and trends of future areas of application development are also included. Second part of the paper is oriented towards quality of enterprise informatics and its effect on the growth of competitiveness, increased performance related to enterprise management including analysis and planning of comprehensive objectives. All information are results of own research.

Published
2007

31. Genotype–phenotype associations in WT1 glomerulopathy

Author

Lipska, Beata S., primary, Ranchin, Bruno, additional, Iatropoulos, Paraskevas, additional, Gellermann, Jutta, additional, Melk, Anette, additional, Ozaltin, Fatih, additional, Caridi, Gianluca, additional, Seeman, Tomas, additional, Tory, Kalman, additional, Jankauskiene, Augustina, additional, Zurowska, Aleksandra, additional, Szczepanska, Maria, additional, Wasilewska, Anna, additional, Harambat, Jerome, additional, Trautmann, Agnes, additional, Peco-Antic, Amira, additional, Borzecka, Halina, additional, Moczulska, Anna, additional, Saeed, Bassam, additional, Bogdanovic, Radovan, additional, Kalyoncu, Mukaddes, additional, Simkova, Eva, additional, Erdogan, Ozlem, additional, Vrljicak, Kristina, additional, Teixeira, Ana, additional, Azocar, Marta, additional, and Schaefer, Franz, additional

Published
2014
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32. The choice of dialysis solutions in pediatric chronic peritoneal dialysis : guidelines by an AD HOC European committee

Author

Schroder, Cornelis, Edefonti, Alberto, Fischbach, Michel, Klaus, Gunther, Ronnholm, Kai, Schaefer, Franz, Simkova, Eva, Stefanides, Dinos, Strazdins, Vladimir, Vande Walle, Johan, Watson, Alan, and Zurowska, Alexandra

Subjects
icodextrin, amino acids, calcium, peritoneal dialysis, children, peritoneal dialysis solutions, Medicine and Health Sciences, dialysis, bicarbonate, glucose
Abstract

Objective: To provide guidelines on choosing dialysis solutions for children on chronic peritoneal dialysis (PD). Setting: European Paediatric Peritoneal Dialysis Working Group. Data Source: Literature on the application of PD solutions in children (Evidence), and discussions within the group (Opinion). Conclusions: Glucose is the standard osmotic agent for PD in children (Evidence). The lowest glucose concentration needed should be used (Opinion). Low calcium solution (1.25 mmol/L) should be applied, wherever possible, with careful monitoring of parathyroid hormone levels (Opinion). The use of amino acid-containing dialysis fluids can be considered in malnourished children, although aggressive enteral nutrition is preferred (Opinion). There is insufficient evidence documenting the efficacy of intraperitoneally administered amino acids (Evidence). When ultrafiltration and/or solute removal are insufficient, poly-glucose solutions are a welcome addition to the treatment of children on nocturnal intermittent PD (Evidence). However, in the absence of any reported long-term experience with children, their use must be closely monitored (Opinion). Bicarbonate would appear to be the preferred buffer for PD in children, but more in vivo studies are required before it replaces the present lactate-containing solutions (Evidence/Opinion).

Published
2001

34. Genetic screening in adolescents with steroid-resistant nephrotic syndrome

Author

Lipska, Beata S., primary, Iatropoulos, Paraskevas, additional, Maranta, Ramona, additional, Caridi, Gianluca, additional, Ozaltin, Fatih, additional, Anarat, Ali, additional, Balat, Ayse, additional, Gellermann, Jutta, additional, Trautmann, Agnes, additional, Erdogan, Ozlem, additional, Saeed, Bassam, additional, Emre, Sevinc, additional, Bogdanovic, Radovan, additional, Azocar, Marta, additional, Balasz-Chmielewska, Irena, additional, Benetti, Elisa, additional, Caliskan, Salim, additional, Mir, Sevgi, additional, Melk, Anette, additional, Ertan, Pelin, additional, Baskin, Esra, additional, Jardim, Helena, additional, Davitaia, Tinatin, additional, Wasilewska, Anna, additional, Drozdz, Dorota, additional, Szczepanska, Maria, additional, Jankauskiene, Augustina, additional, Higuita, Lina Maria Serna, additional, Ardissino, Gianluigi, additional, Ozkaya, Ozan, additional, Kuzma-Mroczkowska, Elzbieta, additional, Soylemezoglu, Oguz, additional, Ranchin, Bruno, additional, Medynska, Anna, additional, Tkaczyk, Marcin, additional, Peco-Antic, Amira, additional, Akil, Ipek, additional, Jarmolinski, Tomasz, additional, Firszt-Adamczyk, Agnieszka, additional, Dusek, Jiri, additional, Simonetti, Giacomo D., additional, Gok, Faysal, additional, Gheissari, Alaleh, additional, Emma, Francesco, additional, Krmar, Rafael T., additional, Fischbach, Michel, additional, Printza, Nikoleta, additional, Simkova, Eva, additional, Mele, Caterina, additional, Ghiggeri, Gian Marco, additional, and Schaefer, Franz, additional

Published
2013
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35. Management of Anemia in Children Receiving Chronic Peritoneal Dialysis

Author

Borzych-Duzalka, Dagmara, primary, Bilginer, Yelda, additional, Ha, Il Soo, additional, Bak, Mustafa, additional, Rees, Lesley, additional, Cano, Francisco, additional, Munarriz, Reyner Loza, additional, Chua, Annabelle, additional, Pesle, Silvia, additional, Emre, Sevinc, additional, Urzykowska, Agnieszka, additional, Quiroz, Lily, additional, Ruscasso, Javier Darío, additional, White, Colin, additional, Pape, Lars, additional, Ramela, Virginia, additional, Printza, Nikoleta, additional, Vogel, Andrea, additional, Kuzmanovska, Dafina, additional, Simkova, Eva, additional, Müller-Wiefel, Dirk E., additional, Sander, Anja, additional, Warady, Bradley A., additional, and Schaefer, Franz, additional

Published
2013
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36. Recessive mutations in DGKE cause atypical hemolytic-uremic syndrome

Author

Lemaire, Mathieu, primary, Frémeaux-Bacchi, Véronique, additional, Schaefer, Franz, additional, Choi, Murim, additional, Tang, Wai Ho, additional, Le Quintrec, Moglie, additional, Fakhouri, Fadi, additional, Taque, Sophie, additional, Nobili, François, additional, Martinez, Frank, additional, Ji, Weizhen, additional, Overton, John D, additional, Mane, Shrikant M, additional, Nürnberg, Gudrun, additional, Altmüller, Janine, additional, Thiele, Holger, additional, Morin, Denis, additional, Deschenes, Georges, additional, Baudouin, Véronique, additional, Llanas, Brigitte, additional, Collard, Laure, additional, Majid, Mohammed A, additional, Simkova, Eva, additional, Nürnberg, Peter, additional, Rioux-Leclerc, Nathalie, additional, Moeckel, Gilbert W, additional, Gubler, Marie Claire, additional, Hwa, John, additional, Loirat, Chantal, additional, and Lifton, Richard P, additional

Published
2013
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39. Relapsing Peritonitis in Children Who Undergo Chronic Peritoneal Dialysis

Author

Lane, Jerome C., primary, Warady, Bradley A., additional, Feneberg, Reinhard, additional, Majkowski, Nancy L., additional, Watson, Alan R., additional, Fischbach, Michel, additional, Kang, Hee Gyung, additional, Bonzel, Klaus E., additional, Simkova, Eva, additional, Stefanidis, Constantinos J., additional, Klaus, Günter, additional, Alexander, Steven R., additional, Ekim, Mesiha, additional, Bilge, Ilmay, additional, and Schaefer, Franz, additional

Published
2010
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40. ILLUMINATE-A, a phase 3 study of lumasiran, an investigational RNAi therapeutic, in children and adults with primary hyperoxaluria type 1 (PH1)

Author

Garrelfs, Sander, Frishberg, Yaacov, Hulton, Sally, Koren, Michael, O Riordan, William, Cochat, Pierre, Deschenes, Georges, Shasha-Laysky, Hadas, Saland, Jeffrey, T Hoff, William, Fuster, Daniel Guido, Daniel Fuster, Moochhala, Shabbir, Schalk, Gesa, Simkova, Eva, Groothoff, Jaap, Sas, David, Meliambro, Kristin, Lu, Jiandong, Garg, Pushkal, Gansner, John, Mcgregor, Tracy, and Lieske, John

41. ILLUMINATE-C, a Single-Arm, Phase 3 Study of Lumasiran in Patients with Primary Hyperoxaluria Type 1 and CKD3b-5, Including Those on Hemodialysis.

Author

Arnold, Philip, Michael, Mini, Groothoff, Jaap, Shasha-Lavsky, Hadas, Lieske, John, Frishberg, Yaacov, Simkova, Eva, Sellier-Leclerc, Devresse, Arnaud, Guebre-Egziabher, Fitsum, Bakkaloglu, Sevcan, Mourani, Chebl, Saqan, Rola, Singer, Richard, and Magen, Daniella

Subjects
*CHRONIC kidney failure, *NURSING, *RNA, *CONFERENCES & conventions, *NEPHROLOGY, *INBORN errors of carbohydrate metabolism, *HEMODIALYSIS
Abstract

We present the 6-month primary analysis data from ILLUMINATE-C, a single-arm, phase 3 study to evaluate lumasiran, an RNAi therapeutic which inhibits oxalate production, in patients with PH1 and impaired kidney function. Key inclusion criteria: genetically confirmed PH1 diagnosis, eGFR ≤45 mL/min/1.73 m², plasma oxalate (POx) ≥20 µmol/L (upper limit of normal=12 µmol/L). Cohort A: patients who did not require dialysis or kidney transplantation at study start. Cohort B: patients on hemodialysis. Primary endpoints: percent change in POx from baseline to Month (M) 6 (cohort A); percent change in pre-dialysis POx from baseline to M6 (cohort B). Baseline mean (SD) POx was 64.7 (41.3) µmol/L in cohort A (N=6) and 108.4 (29.5) µmol/L in cohort B (N=15). In cohorts A and B, respectively, lumasiran led to 33.33% (95%CI: -15.16, 81.82) and 42.43% (95%CI: 34.15, 50.71) least-square mean reductions in POx from baseline to M6 (averaged across M3-6). The most common lumasiran-related AEs were injection-site reactions (all mild). Lumasiran resulted in substantial reductions in POx in PH1 patients with CKD 3b-5, with an acceptable safety profile through M6. [ABSTRACT FROM AUTHOR]

Published
2022

42. Randomized Clinical Trial on the Long-Term Efficacy and Safety of Lumasiran in Patients With Primary Hyperoxaluria Type 1.

Author

Hulton SA, Groothoff JW, Frishberg Y, Koren MJ, Overcash JS, Sellier-Leclerc AL, Shasha-Lavsky H, Saland JM, Hayes W, Magen D, Moochhala SH, Coenen M, Simkova E, Garrelfs SF, Sas DJ, Meliambro KA, Ngo T, Sweetser MT, Habtemariam BA, Gansner JM, McGregor TL, and Lieske JC

Abstract

Introduction: Primary hyperoxaluria type 1 (PH1) is a rare genetic disease caused by hepatic overproduction of oxalate, leading to kidney stones, nephrocalcinosis, kidney failure, and systemic oxalosis. In the 6-month double-blind period (DBP) of ILLUMINATE-A, a phase 3, randomized, placebo-controlled trial in patients with PH1 ≥6 years old, treatment with lumasiran, an RNA interference therapeutic, led to substantial reductions in urinary oxalate (UOx) levels., Methods: We report data to month 12 in the extension period (EP) of ILLUMINATE-A, including patients who continued lumasiran (lumasiran/lumasiran) or crossed over from placebo to lumasiran (placebo/lumasiran)., Results: In the lumasiran/lumasiran group ( n  = 24), the reduction in 24-hour UOx level was sustained to month 12 (mean reduction from baseline, 66.9% at month 6; 64.1% at month 12). The placebo/lumasiran group ( n  = 13) had a similar time course and magnitude of 24-hour UOx reduction (mean reduction, 57.3%) after 6 months of lumasiran. Kidney stone event rates seemed to be lower after 6 months of lumasiran in both groups compared with the 12 months before consent, and this reduction was maintained at month 12 in the lumasiran/lumasiran group. At study start, 71% of patients in the lumasiran/lumasiran group and 92% in the placebo/lumasiran group had nephrocalcinosis. Nephrocalcinosis grade improved after 6 months of lumasiran in the lumasiran/lumasiran and placebo/lumasiran groups (13% and 8% of patients, respectively). After an additional 6 months of lumasiran, 46% of patients had improvement in nephrocalcinosis grade within the lumasiran/lumasiran group. Estimated glomerular filtration rate (eGFR) remained stable during the course of lumasiran treatment. The most common adverse events (AEs) related to lumasiran were mild, transient injection-site reactions (ISRs)., Conclusion: Long-term lumasiran treatment enabled sustained lowering of UOx levels with acceptable safety and encouraging results on clinical outcomes., (© 2022 International Society of Nephrology. Published by Elsevier Inc.)

Published
2021
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