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4. Lung function decline preceding chronic respiratory failure in spinal muscular atrophy: a national prospective cohort study

Author

Veldhoen, Esther S., Wijngaarde, Camiel A., van Eijk, Ruben P. A., Asselman, Fay-Lynn, Seddiqi, Negina, Otto, Louise A. M., Stam, Marloes, Cuppen, Inge, Wadman, Renske I., van Asperen, Roelie M. Wösten, Hulzebos, Erik H. J., van den Oudenrijn, Laura P. Verweij, Bartels, Bart, Boezer, Jasmijn, Gaytant, M., van der Ent, Cornelis K., and van der Pol, W. Ludo

Published
2023
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6. Natural history of respiratory muscle strength in spinal muscular atrophy: a prospective national cohort study

Author

Veldhoen, Esther S., Wijngaarde, Camiel A., Hulzebos, Erik H. J., Wösten-van Asperen, Roelie M., Wadman, Renske I., van Eijk, Ruben P. A., Asselman, Fay Lynn, Stam, Marloes, Otto, Louise A. M., Cuppen, Inge, Scheijmans, Feline E. V., den Oudenrijn, Laura P. Verweij-van, Bartels, Bart, Gaytant, Michael A., van der Ent, Cornelis K., and van der Pol, W. Ludo

Published
2022
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10. Early-Life Risk Factors for Carotid Intima-Media Thickness and Carotid Stiffness in Adolescence

Author

Speerpunt, Sociale Pediatrie patientenzorg, Child Health, Longziekten patientenzorg, Speerpunt Child Health, Infection & Immunity, HAG Onderzoek, Longziekten onderzoek 1, Sociale pediatrie onderzoek 2, Circulatory Health, van der Linden, Isabelle A, Roodenburg, Rozan, Nijhof, Sanne L, van der Ent, Cornelis K, Venekamp, Roderick P, van der Laan, Sabine E I, Schipper, Henk S, Speerpunt, Sociale Pediatrie patientenzorg, Child Health, Longziekten patientenzorg, Speerpunt Child Health, Infection & Immunity, HAG Onderzoek, Longziekten onderzoek 1, Sociale pediatrie onderzoek 2, Circulatory Health, van der Linden, Isabelle A, Roodenburg, Rozan, Nijhof, Sanne L, van der Ent, Cornelis K, Venekamp, Roderick P, van der Laan, Sabine E I, and Schipper, Henk S

Published
2024

11. Repeatability and reproducibility of the Forskolin-induced swelling (FIS) assay on intestinal organoids from people with Cystic Fibrosis

Author

Longziekten onderzoek 1, Regenerative Medicine and Stem Cells, Beleid & Communicatie, CTM & Statistical consultation, Circulatory Health, JC onderzoeksprogramma Methodologie, Arts Assistenten Longziekten, Longziekten patientenzorg, Speerpunt Child Health, Child Health, Infection & Immunity, Longziekten onderzoek 2, Onderzoek, Bierlaagh, Marlou C., Ramalho, Anabela S., Silva, Iris A.L., Vonk, Annelotte M., van den Bor, Rutger M., van Mourik, Peter, Pott, Johanna, Suen, Sylvia W.F., Boj, Sylvia F., Vries, Robert G.J., Lammertyn, Elise, Vermeulen, François, Amaral, Margarida D., de Boeck, Kris, van der Ent, Cornelis K., Beekman, Jeffrey M., Longziekten onderzoek 1, Regenerative Medicine and Stem Cells, Beleid & Communicatie, CTM & Statistical consultation, Circulatory Health, JC onderzoeksprogramma Methodologie, Arts Assistenten Longziekten, Longziekten patientenzorg, Speerpunt Child Health, Child Health, Infection & Immunity, Longziekten onderzoek 2, Onderzoek, Bierlaagh, Marlou C., Ramalho, Anabela S., Silva, Iris A.L., Vonk, Annelotte M., van den Bor, Rutger M., van Mourik, Peter, Pott, Johanna, Suen, Sylvia W.F., Boj, Sylvia F., Vries, Robert G.J., Lammertyn, Elise, Vermeulen, François, Amaral, Margarida D., de Boeck, Kris, van der Ent, Cornelis K., and Beekman, Jeffrey M.

Published
2024

12. Enhanced aortic stiffness in adolescents with chronic disease is associated with decreased left ventricular global longitudinal strain

Author

Team Onderzoek, CTI Boes, Cardiologie patientenzorg, Child Health, Circulatory Health, Immuno/reuma patientenzorg, Infection & Immunity, Longziekten patientenzorg, Speerpunt Child Health, MS Radiologie, Sociale pediatrie onderzoek 2, Verpalen, Victor A, Ververs, Francesca A, Slieker, Martijn, Nuboer, Roos, Swart, Joost F, van der Ent, Cornelis K, Fejzic, Zina, Westenberg, Jos J M, Leiner, Tim, Grotenhuis, Heynric B, Schipper, Henk S, Team Onderzoek, CTI Boes, Cardiologie patientenzorg, Child Health, Circulatory Health, Immuno/reuma patientenzorg, Infection & Immunity, Longziekten patientenzorg, Speerpunt Child Health, MS Radiologie, Sociale pediatrie onderzoek 2, Verpalen, Victor A, Ververs, Francesca A, Slieker, Martijn, Nuboer, Roos, Swart, Joost F, van der Ent, Cornelis K, Fejzic, Zina, Westenberg, Jos J M, Leiner, Tim, Grotenhuis, Heynric B, and Schipper, Henk S

Published
2024

13. A dyadic perspective on parent-child dyadic coping in children with a chronic condition

Author

Sociale Pediatrie patientenzorg, Child Health, Regenerative Medicine and Stem Cells, Longziekten patientenzorg, Speerpunt Child Health, Infection & Immunity, Sociale pediatrie onderzoek 1, van der Wal, Reine C., Nijhof, Sanne L., Leisten, Luca M., van de Putte, Elise M., van der Ent, Cornelis K., Hindriks-Keegstra, Alinde W., Bodenmann, Guy, Finkenauer, Catrin, Nap-van der Vlist, Merel M., Sociale Pediatrie patientenzorg, Child Health, Regenerative Medicine and Stem Cells, Longziekten patientenzorg, Speerpunt Child Health, Infection & Immunity, Sociale pediatrie onderzoek 1, van der Wal, Reine C., Nijhof, Sanne L., Leisten, Luca M., van de Putte, Elise M., van der Ent, Cornelis K., Hindriks-Keegstra, Alinde W., Bodenmann, Guy, Finkenauer, Catrin, and Nap-van der Vlist, Merel M.

Published
2024

14. OrgaSegment: deep-learning based organoid segmentation to quantify CFTR dependent fluid secretion

Author

Longziekten onderzoek 2, Cancer, Regenerative Medicine and Stem Cells, Longziekten patientenzorg, Speerpunt Child Health, Child Health, Infection & Immunity, Onderzoek, Lefferts, Juliet W, Kroes, Suzanne, Smith, Matthew B, Niemöller, Paul J, Nieuwenhuijze, Natascha D A, Sonneveld van Kooten, Heleen N, van der Ent, Cornelis K, Beekman, Jeffrey M, van Beuningen, Sam F B, Longziekten onderzoek 2, Cancer, Regenerative Medicine and Stem Cells, Longziekten patientenzorg, Speerpunt Child Health, Child Health, Infection & Immunity, Onderzoek, Lefferts, Juliet W, Kroes, Suzanne, Smith, Matthew B, Niemöller, Paul J, Nieuwenhuijze, Natascha D A, Sonneveld van Kooten, Heleen N, van der Ent, Cornelis K, Beekman, Jeffrey M, and van Beuningen, Sam F B

Published
2024

15. Clinical effects of the three CFTR potentiator treatments curcumin, genistein and ivacaftor in patients with the CFTR-S1251N gating mutation

Author

Berkers, Gitte, van der Meer, Renske, van Mourik, Peter, Vonk, Annelotte M, Kruisselbrink, Evelien, Suen, Sylvia WF, Heijerman, Harry GM, Majoor, Christof J, Koppelman, Gerard H, Roukema, Jolt, Janssens, Hettie M, de Rijke, Yolanda B, Kemper, E. Marleen, Beekman, Jeffrey M, van der Ent, Cornelis K, and de Jonge, Hugo R

Published
2020
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17. Forskolin-induced swelling of intestinal organoids correlates with disease severity in adults with cystic fibrosis and homozygous F508del mutations

Author

de Winter – de Groot, Karin M., Berkers, Gitte, Marck – van der Wilt, Rozemarijn E.P., van der Meer, Renske, Vonk, Annelotte, Dekkers, Johanna F., Geerdink, Margot, Michel, Sabine, Kruisselbrink, Evelien, Vries, Rob, Clevers, Hans, Vleggaar, Frank P., Elias, Sjoerd G., Heijerman, Harry G.M., van der Ent, Cornelis K., and Beekman, Jeffrey M.

Published
2020
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19. CFTR Function Restoration upon Elexacaftor/Tezacaftor/Ivacaftor Treatment in Patient-Derived Intestinal Organoids with Rare CFTR Genotypes

Author

Lefferts, Juliet W., primary, Bierlaagh, Marlou C., additional, Kroes, Suzanne, additional, Nieuwenhuijze, Natascha D. A., additional, Sonneveld van Kooten, Heleen N., additional, Niemöller, Paul J., additional, Verburg, Tibo F., additional, Janssens, Hettie M., additional, Muilwijk, Danya, additional, van Beuningen, Sam F. B., additional, van der Ent, Cornelis K., additional, and Beekman, Jeffrey M., additional

Published
2023
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25. Airway Epithelial Cultures of Children with Esophageal Atresia as a Model to Study Respiratory Tract Disorders

Author

Dreyer, Henriette H. M., primary, van Tuyll van Serooskerken, Eleonora Sofie, additional, Rodenburg, Lisa W., additional, Bittermann, Arnold J. N., additional, Arets, Hubertus G. M., additional, Reuling, Ellen M. B. P., additional, Verweij, Johannes W., additional, Haarman, Eric G., additional, van der Zee, David C., additional, Tytgat, Stefaan H. A. J., additional, van der Ent, Cornelis K., additional, Beekman, Jeffrey M., additional, Amatngalim, Gimano D., additional, and Lindeboom, Maud Y. A., additional

Published
2023
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27. Natural history of lung function in spinal muscular atrophy

Author

Wijngaarde, Camiel A., Veldhoen, Esther S., van Eijk, Ruben P. A., Stam, Marloes, Otto, Louise A. M., Asselman, Fay-Lynn, Wösten-van Asperen, Roelie M., Hulzebos, Erik H. J., Verweij-van den Oudenrijn, Laura P., Bartels, Bart, Cuppen, Inge, Wadman, Renske I., van den Berg, Leonard H., van der Ent, Cornelis K., and van der Pol, W. Ludo

Published
2020
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28. Airway Epithelial Cultures of Children with Esophageal Atresia as a Model to Study Respiratory Tract Disorders

Author

Dreyer, Henriette H. M., van Tuyll van Serooskerken, Eleonora Sofie, Rodenburg, Lisa W., Bittermann, Arnold J. N., Arets, Hubertus G. M., Reuling, Ellen M. B. P., Verweij, Johannes W., Haarman, Eric G., van der Zee, David C., Tytgat, Stefaan H. A. J., van der Ent, Cornelis K., Beekman, Jeffrey M., Amatngalim, Gimano D., and Lindeboom, Maud Y. A.

Abstract

Esophageal atresia (EA) is a rare birth defect in which respiratory tract disorders are a major cause of morbidity. It remains unclear whether respiratory tract disorders are in part caused by alterations in airway epithelial cell functions such as the activity of motile cilia. This can be studied using airway epithelial cell culture models of patients with EA. Therefore, the aim of this study was to evaluate the feasibility to culture and functionally characterize motile cilia function in the differentiated air–liquid interface cultured airway epithelial cells and 3D organoids derived from nasal brushings and bronchoalveolar lavage (BAL) fluid from children with EA. We demonstrate the feasibility of culturing differentiated airway epithelia and organoids of nasal brushings and BAL fluid of children with EA, which display normal motile cilia function. EA patient-derived airway epithelial cultures can be further used to examine whether alterations in epithelial functions contribute to respiratory disorders in EA.

Published
2023

29. Defining and Measuring Resilience in Children with a Chronic Disease: a Scoping Review

Author

van der Laan, Sabine E I, Berkelbach van der Sprenkel, Emma E, Lenters, Virissa C, Finkenauer, Catrin, van der Ent, Cornelis K, Nijhof, Sanne L, LS IRAS EEPI GRA (Gezh.risico-analyse), Leerstoel Finkenauer, Youth in Changing Cultural Contexts, LS IRAS EEPI GRA (Gezh.risico-analyse), Leerstoel Finkenauer, and Youth in Changing Cultural Contexts

Subjects
Children/adolescent, Measurement, Resilience, General Medicine, Defnition, Chronic disease
Abstract

UNLABELLED: More than 25% of all children grow up with a chronic disease. They are at higher risk for developmental and psychosocial problems. However, children who function resiliently manage to adapt positively to these challenges. We aim to systematically review how resilience is defined and measured in children with a chronic disease. A search of PubMed, Cochrane, Embase, and PsycINFO was performed on December 9, 2022, using resilience, disease, and child/adolescent as search terms. Two reviewers independently screened articles for inclusion according to predefined criteria. Extraction domains included study characteristics, definition, and instruments assessing resilience outcomes, and resilience factors. Fifty-five out of 8766 articles were identified as relevant. In general, resilience was characterized as positive adaptation to adversity. The included studies assessed resilience by the outcomes of positive adaptation, or by resilience factors, or both. We categorized the assessed resilience outcomes into three groups: personal traits, psychosocial functioning, and disease-related outcomes. Moreover, myriad of resilience factors were measured, which were grouped into internal resilience factors (cognitive, social, and emotional competence factors), disease-related factors, and external factors (caregiver factors, social factors, and contextual factors). Our scoping review provides insight into the definitions and instruments used to measure resilience in children with a chronic disease. More knowledge is needed on which resilience factors are related to positive adaptation in specific illness-related challenges, which underlying mechanisms are responsible for this positive adaptation, and how these underlying mechanisms interact with one another. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1007/s42844-023-00092-2.

Published
2023

30. 16S rRNA-Based Microbiota Profiling Assists Conventional Culture Analysis of Airway Samples from Pediatric Cystic Fibrosis Patients

Author

Kristensen, Maartje, primary, de Koff, Emma M., additional, Chu, Mei Ling, additional, Groendijk, Simone, additional, Tramper-Stranders, Gerdien A., additional, de Winter-de Groot, Karin M., additional, Janssens, Hettie M., additional, Tiddens, Harm A., additional, van Westreenen, Mireille, additional, Sanders, Elisabeth A. M., additional, Arets, Bert H. G. M., additional, van der Ent, Cornelis K., additional, Prevaes, Sabine M. P. J., additional, and Bogaert, Debby, additional

Published
2023
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32. A cross-sectional study on gaming intensity and social vulnerability in adolescents that have a chronic condition

Author

Alexandridis, Dionysis, primary, Nijhof, Sanne L., additional, van der Rijst, Vincent G., additional, van der Neut, Damian Y., additional, Spijkerman, Renske, additional, Stevens, Gonneke W. J. M., additional, Bakkes, Sander C. J., additional, Lesscher, Heidi M. B., additional, van den Eijnden, Regina J. J. M., additional, van der Ent, Cornelis K., additional, van den Berg, Gerrit, additional, and Peeters, Margot, additional

Published
2023
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34. Self-reported quantity and quality of sleep in children and adolescents with a chronic condition compared to healthy controls

Author

Biemans, Camille F.M., Nijhof, Sanne L., Gorter, Jan Willem, Stevens, Gonneke J.W.M., van de Putte, Elise, Hoefnagels, Johanna W., van den Berg, Anemone, van der Ent, Cornelis K., Dudink, Jeroen, Verschuren, Olaf W., Biemans, Camille F.M., Nijhof, Sanne L., Gorter, Jan Willem, Stevens, Gonneke J.W.M., van de Putte, Elise, Hoefnagels, Johanna W., van den Berg, Anemone, van der Ent, Cornelis K., Dudink, Jeroen, and Verschuren, Olaf W.

Abstract

To assess self-reported quantity and quality of sleep in Dutch children with a chronic condition compared to healthy controls and to the recommended hours of sleep for youth. Sleep quantity and quality were analyzed in children with a chronic condition (cystic fibrosis, chronic kidney disease, congenital heart disease, (auto-)immune disease, and medically unexplained symptoms (MUS); n = 291; 15 ± 3.1 years, 63% female. A subset of 171 children with a chronic condition were matched to healthy controls using Propensity Score matching, based on age and sex, ratio 1:4. Self-reported sleep quantity and quality were assessed with established questionnaires. Children with MUS were analyzed separately to distinguish between chronic conditions with and without an identified pathophysiological cause. Generally, children with a chronic condition met the recommended amount of sleep, however 22% reported poor sleep quality. No significant differences in sleep quantity and quality were found between the diagnosis groups. Children with a chronic condition and with MUS slept significantly more than healthy controls at ages 13, 15, and 16. Both at primary and secondary school, poor sleep quality was least frequent reported in children with a chronic condition and most often reported in children with MUS. Conclusion: Overall, children with chronic conditions, including MUS, met the recommended hours of sleep for youth, and slept more than healthy controls. However, it is important to obtain a better understanding of why a substantial subset of children with chronic conditions, mostly children with MUS, still perceived their sleep quality as poor.What is Known:• According to the Consensus statement of the American Academy of Sleep medicine, typically developing children (6 to 12 years) should sleep 9 to 12 h per night, and adolescents (13 to 18 years) should sleep 8 to 10 h per night.• Literature on the optimal quantity and quality of sleep in children with a chronic condition is v

Published
2023

35. Traits, trends and hits of orphan drug designations in cystic fibrosis

Author

Costa, Enrico, Girotti, Silvia, van den Ham, Hendrika A., Cipolli, Marco, van der Ent, Cornelis K., Taylor-Cousar, Jennifer L., Leufkens, Hubert G.M., Costa, Enrico, Girotti, Silvia, van den Ham, Hendrika A., Cipolli, Marco, van der Ent, Cornelis K., Taylor-Cousar, Jennifer L., and Leufkens, Hubert G.M.

Abstract

Background: In the United States (US) and in Europe, cystic fibrosis (CF) qualifies as a rare disease, thus positioning the field to benefit from regulatory incentives provided by orphan drug designation (ODD) to boost pharmaceutical research and development. In this study, we analyzed the pool of products for the treatment of CF that received such incentives from the US Food and Drug Administration (FDA) and/or the European Medicines Agency (EMA) over the past two decades. We describe the characteristics and trends in ODDs over time and explore factors that might be determinants of successful drug development. Methods: We collected the products that received the ODD from the registries of the FDA and the EMA from 2000 to 2021, characterizing their nature, development stage, and type of sponsor. We categorized the study drugs according to the therapeutic target addressed and described trends of drug development over the study period. A logistic regression analysis was done to assess how ODD characteristics were associated with the approval for market authorization. Results: From 2000–2021, 107 ODDs were collectively granted by the FDA and the EMA for products developed for the treatment of CF. Although the trends of the number of ODDs granted remained stable over time, those targeting the CF basic protein defect increased from 6 out of 54 (11.1%) in the first half of the study period up to 20 out of 54 (37.7%) in the second half, while those treating symptoms decreased from 48/54 (88.9%) to 33/53 (62.3%). Overall, 10 products obtained marketing approval: 7 in both the US and Europe, 3 only in Europe. All the approved ODDs were chemical products for chronic use. No statistically significant difference was found across the examinated variables, but we observed possible drivers of successful drug development for ODDs targeting CFTR, as well as for those with active substances previously marketed, and for those developed by large companies and companies with experien

Published
2023

36. Organized Sports Activities Are Safe for Children With Sickle Cell Disease: A Pilot Intervention Study

Author

Noordstar, Johannes J., Hulzebos, Erik H.J., Van Der Ent, Cornelis K., Suijker, Monique H., Bartels, Marije, Noordstar, Johannes J., Hulzebos, Erik H.J., Van Der Ent, Cornelis K., Suijker, Monique H., and Bartels, Marije

Abstract

Historically, children with sickle cell disease (SCD) are advised to refrain from sports activities, based on the assumption that physical exercise can trigger vaso-occlusive episodes. This pilot intervention study examined the safety (ie, no vaso-occlusive episodes) of a 10-week organized sports program for children with SCD. Eight children with SCD (5 boys/3 girls), aged 7 to 12 years old, received 10 training sessions (each 90 min) once a week. Training sessions were performed by a professional soccer club under the supervision of a medical team from the Wilhelmina Children's Hospital. During the study period, one child experienced a vaso-occlusive crisis, which could not be directly related to the organized sports program. None of the other children experienced vaso-occlusive episodes. The results of this study indicate that children with SCD can participate safely in moderate-intensity organized sports activities when personalized medical background and practical training information is shared with the trainer beforehand. All children continued their sports participation after the study period.

Published
2023

37. A cross-sectional study on gaming intensity and social vulnerability in adolescents that have a chronic condition

Author

Alexandridis, Dionysis, Nijhof, Sanne L, van der Rijst, Vincent G, van der Neut, Damian Y, Spijkerman, Renske, Stevens, Gonneke W J M, Bakkes, Sander C J, Lesscher, Heidi M B, van den Eijnden, Regina J J M, van der Ent, Cornelis K, van den Berg, Gerrit, Peeters, Margot, Alexandridis, Dionysis, Nijhof, Sanne L, van der Rijst, Vincent G, van der Neut, Damian Y, Spijkerman, Renske, Stevens, Gonneke W J M, Bakkes, Sander C J, Lesscher, Heidi M B, van den Eijnden, Regina J J M, van der Ent, Cornelis K, van den Berg, Gerrit, and Peeters, Margot

Abstract

BACKGROUND: Adolescents growing up with a chronic condition might experience more social vulnerabilities compared to their healthy peers as an indirect result of their conditions. This can lead to a relatedness need frustration for these adolescents. Consequently, they might spend more time playing video games compared to their peers. Research shows that both social vulnerability and gaming intensity are predictors for problematic gaming. Therefore, we investigated if social vulnerability and gaming intensity are more pronounced in adolescents that have a chronic condition compared to the general population; and if these levels reflect the levels of a clinical group being treated for Internet Gaming Disorder (IGD).METHODS: Data on peer problems and gaming intensity were compared from three separate samples: a national representative sample of adolescents, a clinical sample of adolescents that are undergoing treatment for IGD, and a sample of adolescents diagnosed with a chronic condition.RESULTS: No differences were found on either peer problems or gaming intensity between the group of adolescents that have chronic conditions and the national representative group. The group with chronic conditions scored significantly lower on gaming intensity than the clinical group. No significant differences were found between these groups on peer problems. We repeated the analyses for boys only. Similar results were found for the group with chronic conditions compared to the national representative group. The group with chronic conditions now scored significantly lower on both peer problems and gaming intensity than the clinical group.CONCLUSION: Adolescents growing up with a chronic condition appear similar in their gaming intensity and peer problems compared to their healthy peers.

Published
2023

38. A cross-sectional study on gaming intensity and social vulnerability in adolescents that have a chronic condition

Author

Sub Multimedia, Youth in Changing Cultural Contexts, Leerstoel Finkenauer, Leerstoel Stevens, AISS Behaviour Neuroscience, Multimedia, Alexandridis, Dionysis, Nijhof, Sanne L, van der Rijst, Vincent G, van der Neut, Damian Y, Spijkerman, Renske, Stevens, Gonneke W J M, Bakkes, Sander C J, Lesscher, Heidi M B, van den Eijnden, Regina J J M, van der Ent, Cornelis K, van den Berg, Gerrit, Peeters, Margot, Sub Multimedia, Youth in Changing Cultural Contexts, Leerstoel Finkenauer, Leerstoel Stevens, AISS Behaviour Neuroscience, Multimedia, Alexandridis, Dionysis, Nijhof, Sanne L, van der Rijst, Vincent G, van der Neut, Damian Y, Spijkerman, Renske, Stevens, Gonneke W J M, Bakkes, Sander C J, Lesscher, Heidi M B, van den Eijnden, Regina J J M, van der Ent, Cornelis K, van den Berg, Gerrit, and Peeters, Margot

Published
2023

39. Defining and Measuring Resilience in Children with a Chronic Disease: a Scoping Review

Author

LS IRAS EEPI GRA (Gezh.risico-analyse), Leerstoel Finkenauer, Youth in Changing Cultural Contexts, van der Laan, Sabine E I, Berkelbach van der Sprenkel, Emma E, Lenters, Virissa C, Finkenauer, Catrin, van der Ent, Cornelis K, Nijhof, Sanne L, LS IRAS EEPI GRA (Gezh.risico-analyse), Leerstoel Finkenauer, Youth in Changing Cultural Contexts, van der Laan, Sabine E I, Berkelbach van der Sprenkel, Emma E, Lenters, Virissa C, Finkenauer, Catrin, van der Ent, Cornelis K, and Nijhof, Sanne L

Published
2023

40. Traits, trends and hits of orphan drug designations in cystic fibrosis

Author

Afd Pharmacoepi & Clinical Pharmacology, Pharmacoepidemiology and Clinical Pharmacology, PECP - Centre for Pharmaceutical Policy and Regulation, Costa, Enrico, Girotti, Silvia, van den Ham, Hendrika A., Cipolli, Marco, van der Ent, Cornelis K., Taylor-Cousar, Jennifer L., Leufkens, Hubert G.M., Afd Pharmacoepi & Clinical Pharmacology, Pharmacoepidemiology and Clinical Pharmacology, PECP - Centre for Pharmaceutical Policy and Regulation, Costa, Enrico, Girotti, Silvia, van den Ham, Hendrika A., Cipolli, Marco, van der Ent, Cornelis K., Taylor-Cousar, Jennifer L., and Leufkens, Hubert G.M.

Published
2023

41. Self-reported quantity and quality of sleep in children and adolescents with a chronic condition compared to healthy controls

Author

Leerstoel Stevens, Youth in Changing Cultural Contexts, Leerstoel Thomaes, Social and personality development: A transactional approach, LS English Linguistics:Language Developm, Biemans, Camille F.M., Nijhof, Sanne L., Gorter, Jan Willem, Stevens, Gonneke J.W.M., van de Putte, Elise, Hoefnagels, Johanna W., van den Berg, Anemone, van der Ent, Cornelis K., Dudink, Jeroen, Verschuren, Olaf W., Leerstoel Stevens, Youth in Changing Cultural Contexts, Leerstoel Thomaes, Social and personality development: A transactional approach, LS English Linguistics:Language Developm, Biemans, Camille F.M., Nijhof, Sanne L., Gorter, Jan Willem, Stevens, Gonneke J.W.M., van de Putte, Elise, Hoefnagels, Johanna W., van den Berg, Anemone, van der Ent, Cornelis K., Dudink, Jeroen, and Verschuren, Olaf W.

Published
2023

42. CFTR Function Restoration upon Elexacaftor/Tezacaftor/Ivacaftor Treatment in Patient-Derived Intestinal Organoids with Rare CFTR Genotypes

Author

Lefferts, Juliet W., Bierlaagh, Marlou C., Kroes, Suzanne, Nieuwenhuijze, Natascha D.A., Sonneveld van Kooten, Heleen N., Niemöller, Paul J., Verburg, Tibo F., Janssens, Hettie M., Muilwijk, Danya, van Beuningen, Sam F.B., van der Ent, Cornelis K., Beekman, Jeffrey M., Lefferts, Juliet W., Bierlaagh, Marlou C., Kroes, Suzanne, Nieuwenhuijze, Natascha D.A., Sonneveld van Kooten, Heleen N., Niemöller, Paul J., Verburg, Tibo F., Janssens, Hettie M., Muilwijk, Danya, van Beuningen, Sam F.B., van der Ent, Cornelis K., and Beekman, Jeffrey M.

Abstract

Cystic fibrosis (CF) is caused by mutations in the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene. The combination of the CFTR modulators elexacaftor, tezacaftor, and ivacaftor (ETI) enables the effective rescue of CFTR function in people with the most prevalent F508del mutation. However, the functional restoration of rare CFTR variants remains unclear. Here, we use patient-derived intestinal organoids (PDIOs) to identify rare CFTR variants and potentially individuals with CF that might benefit from ETI. First, steady-state lumen area (SLA) measurements were taken to assess CFTR function and compare it to the level observed in healthy controls. Secondly, the forskolin-induced swelling (FIS) assay was performed to measure CFTR rescue within a lower function range, and to further compare it to ETI-mediated CFTR rescue in CFTR genotypes that have received market approval. ETI responses in 30 PDIOs harboring the F508del mutation served as reference for ETI responses of 22 PDIOs with genotypes that are not currently eligible for CFTR modulator treatment, following European Medicine Agency (EMA) and/or U.S. Food and Drug Administration (FDA) regulations. Our data expand previous datasets showing a correlation between in vitro CFTR rescue in organoids and corresponding in vivo ppFEV1 improvement upon a CFTR modulator treatment in published clinical trials, and suggests that the majority of individuals with rare CFTR variants could benefit from ETI. CFTR restoration was further confirmed on protein levels using Western blot. Our data support that CFTR function measurements in PDIOs with rare CFTR genotypes can help to select potential responders to ETI, and suggest that regulatory authorities need to consider providing access to treatment based on the principle of equality for people with CF who do not have access to treatment.

Published
2023

44. Is Measuring Physical Literacy in School-Aged Children With Cystic Fibrosis or Congenital Heart Disease Needed?

Author

Leerstoel Baar, Development and Treatment of Psychosocial Problems, Noordstar, Johannes J., Sprong, Maaike C.A., Slieker, Martijn G., Takken, Tim, van Brussel, Marco, van der Ent, Cornelis K., Hulzebos, Erik H.J., Leerstoel Baar, Development and Treatment of Psychosocial Problems, Noordstar, Johannes J., Sprong, Maaike C.A., Slieker, Martijn G., Takken, Tim, van Brussel, Marco, van der Ent, Cornelis K., and Hulzebos, Erik H.J.

Published
2023

45. 16S rRNA-Based Microbiota Profiling Assists Conventional Culture Analysis of Airway Samples from Pediatric Cystic Fibrosis Patients

Author

Kristensen, Maartje, de Koff, Emma M., Chu, Mei Ling, Groendijk, Simone, Tramper-Stranders, Gerdien A., de Winter-De Groot, Karin M., Janssens, Hettie M., Tiddens, Harm A., van Westreenen, Mireille, Sanders, Elisabeth A.M., Arets, Bert H.G.M., van der Ent, Cornelis K., Prevaes, Sabine M.P.J., Bogaert, Debby, Kristensen, Maartje, de Koff, Emma M., Chu, Mei Ling, Groendijk, Simone, Tramper-Stranders, Gerdien A., de Winter-De Groot, Karin M., Janssens, Hettie M., Tiddens, Harm A., van Westreenen, Mireille, Sanders, Elisabeth A.M., Arets, Bert H.G.M., van der Ent, Cornelis K., Prevaes, Sabine M.P.J., and Bogaert, Debby

Abstract

16S-based sequencing provides broader information on the respiratory microbial community than conventional culturing. However, it (often) lacks species- and strain-level information. To overcome this issue, we used 16S rRNA-based sequencing results from 246 nasopharyngeal samples obtained from 20 infants with cystic fibrosis (CF) and 43 healthy infants, which were all 0 to 6 months old, and compared them to both standard (blind) diagnostic culturing and a 16S-sequencing-informed “targeted” reculturing approach. Using routine culturing, we almost uniquely detected Moraxella catarrhalis, Staphylococcus aureus, and Haemophilus influenzae (42%, 38%, and 33% of samples, respectively). Using the targeted reculturing approach, we were able to reculture 47% of the top-5 operational taxonomical units (OTUs) in the sequencing profiles. In total, we identified 60 species from 30 genera with a median of 3 species per sample (range, 1 to 8). We also identified up to 10 species per identified genus. The success of reculturing the top-5 genera present from the sequencing profile depended on the genus. In the case of Corynebacterium being in the top 5, we recultured them in 79% of samples, whereas for Staphylococcus, this value was only 25%. The success of reculturing was also correlated with the relative abundance of those genera in the corresponding sequencing profile. In conclusion, revisiting samples using 16S-based sequencing profiles to guide a targeted culturing approach led to the detection of more potential pathogens per sample than conventional culturing and may therefore be useful in the identification and, consequently, treatment of bacteria considered relevant for the deterioration or exacerbation of disease in patients like those with CF. IMPORTANCE Early and effective treatment of pulmonary infections in cystic fibrosis is vital to prevent chronic lung damage. Although microbial diagnostics and treatment decisions are still based on conventional culture methods, r

Published
2023

46. CFTR Function Restoration upon Elexacaftor/Tezacaftor/Ivacaftor Treatment in Patient-Derived Intestinal Organoids with Rare CFTR Genotypes.

Author

Longziekten onderzoek 2, Cancer, Longziekten onderzoek 1, Arts Assistenten Longziekten, Longziekten patientenzorg, Speerpunt Child Health, Child Health, Infection & Immunity, Onderzoek, Regenerative Medicine and Stem Cells, Lefferts, Juliet W, Bierlaagh, Marlou C, Kroes, Suzanne, Nieuwenhuijze, Natascha D A, Sonneveld van Kooten, Heleen N, Niemöller, Paul J, Verburg, Tibo F, Janssens, Hettie M, Muilwijk, Danya, van Beuningen, Sam F B, van der Ent, Cornelis K, Beekman, Jeffrey M, Longziekten onderzoek 2, Cancer, Longziekten onderzoek 1, Arts Assistenten Longziekten, Longziekten patientenzorg, Speerpunt Child Health, Child Health, Infection & Immunity, Onderzoek, Regenerative Medicine and Stem Cells, Lefferts, Juliet W, Bierlaagh, Marlou C, Kroes, Suzanne, Nieuwenhuijze, Natascha D A, Sonneveld van Kooten, Heleen N, Niemöller, Paul J, Verburg, Tibo F, Janssens, Hettie M, Muilwijk, Danya, van Beuningen, Sam F B, van der Ent, Cornelis K, and Beekman, Jeffrey M

Published
2023

47. Childhood infection burden, recent antibiotic exposure and vascular phenotypes in preschool children

Author

Public Health Epidemiologie, Epi Infectieziekten Team 1, Child Health, Infection & Immunity, JC onderzoeksprogramma Infectieziekten, Longziekten patientenzorg, Speerpunt Child Health, Global Health, Circulatory Health, JC onderzoeksprogramma Cardiovasculaire Epidemiologie, Cardiovasculaire Epi Team 1, Yu, Angela, Jansen, Maria A.C., Dalmeijer, Geertje W., Bruijning-Verhagen, Patricia, van der Ent, Cornelis K., Grobbee, Diederick E., Burgner, David P., Uiterwaal, Cuno S.P.M., Public Health Epidemiologie, Epi Infectieziekten Team 1, Child Health, Infection & Immunity, JC onderzoeksprogramma Infectieziekten, Longziekten patientenzorg, Speerpunt Child Health, Global Health, Circulatory Health, JC onderzoeksprogramma Cardiovasculaire Epidemiologie, Cardiovasculaire Epi Team 1, Yu, Angela, Jansen, Maria A.C., Dalmeijer, Geertje W., Bruijning-Verhagen, Patricia, van der Ent, Cornelis K., Grobbee, Diederick E., Burgner, David P., and Uiterwaal, Cuno S.P.M.

Published
2023

48. Exploring intrinsic variability between cultured nasal and bronchial epithelia in cystic fibrosis

Author

Longziekten onderzoek 2, Regenerative Medicine and Stem Cells, CTI, Longziekten patientenzorg, Child Health, Speerpunt Child Health, Infection & Immunity, Onderzoek, Rodenburg, Lisa W, Metzemaekers, Mieke, van der Windt, Isabelle S, Smits, Shannon M A, den Hertog-Oosterhoff, Loes A, Kruisselbrink, Evelien, Brunsveld, Jesse E, Michel, Sabine, de Winter-de Groot, Karin M, van der Ent, Cornelis K, Stadhouders, Ralph, Beekman, Jeffrey M, Amatngalim, Gimano D, Longziekten onderzoek 2, Regenerative Medicine and Stem Cells, CTI, Longziekten patientenzorg, Child Health, Speerpunt Child Health, Infection & Immunity, Onderzoek, Rodenburg, Lisa W, Metzemaekers, Mieke, van der Windt, Isabelle S, Smits, Shannon M A, den Hertog-Oosterhoff, Loes A, Kruisselbrink, Evelien, Brunsveld, Jesse E, Michel, Sabine, de Winter-de Groot, Karin M, van der Ent, Cornelis K, Stadhouders, Ralph, Beekman, Jeffrey M, and Amatngalim, Gimano D

Published
2023

49. Development and validation of a novel personalized electronic patient-reported outcome measure to assess quality of life (Q-LIFE): a prospective observational study in people with Cystic Fibrosis

Author

Arts Assistenten Longziekten, Longziekten, Child Health, Infection & Immunity, Longziekten patientenzorg, Speerpunt Child Health, Muilwijk, Danya, van Paridon, Tessa J., van der Heijden, Doris C., Faber-Bisschop, Brenda M., Zomer-van Ommen, Domenique D., Heijerman, Harry G.M., van der Ent, Cornelis K., Arts Assistenten Longziekten, Longziekten, Child Health, Infection & Immunity, Longziekten patientenzorg, Speerpunt Child Health, Muilwijk, Danya, van Paridon, Tessa J., van der Heijden, Doris C., Faber-Bisschop, Brenda M., Zomer-van Ommen, Domenique D., Heijerman, Harry G.M., and van der Ent, Cornelis K.

Published
2023

50. High-throughput functional assay in cystic fibrosis patient-derived organoids allows drug repurposing

Author

Longziekten onderzoek 2, Longziekten onderzoek 1, Arts Assistenten Longziekten, Longziekten patientenzorg, Beleid & Communicatie, CTI, Speerpunt Child Health, Child Health, Infection & Immunity, Onderzoek, Regenerative Medicine and Stem Cells, Spelier, Sacha, de Poel, Eyleen, Ithakisiou, Georgia N., Suen, Sylvia W.F., Hagemeijer, Marne C., Muilwijk, Danya, Vonk, Annelotte M., Brunsveld, Jesse E., Kruisselbrink, Evelien, van der Ent, Cornelis K., Beekman, Jeffrey M., Longziekten onderzoek 2, Longziekten onderzoek 1, Arts Assistenten Longziekten, Longziekten patientenzorg, Beleid & Communicatie, CTI, Speerpunt Child Health, Child Health, Infection & Immunity, Onderzoek, Regenerative Medicine and Stem Cells, Spelier, Sacha, de Poel, Eyleen, Ithakisiou, Georgia N., Suen, Sylvia W.F., Hagemeijer, Marne C., Muilwijk, Danya, Vonk, Annelotte M., Brunsveld, Jesse E., Kruisselbrink, Evelien, van der Ent, Cornelis K., and Beekman, Jeffrey M.

Published
2023

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