971c741fd1075d9535c8697adfb259ad sfae049.pdf 5e7075d56f77b080dfd9160d2ea613e0ba121902 sfae049.pdf 7d1497b4d0fef8148de4463d140bbf9582c94dd9934b8446043eba9ada22b33b sfae049.pdf Title: Switching from immediate- to extended-release cysteamine in patients with nephropathic cystinosis: from clinical trials to clinical practice Subject: DOI: 10.1093/ckj/sfae049 Clinical Kidney Journal, 17, 4, 06-03-2024. Abstract: The purpose of this study is to evaluate the effectiveness and safety of switching from immediate-release (IR) to extended-release (ER) cysteamine in patients with nephropathic cystinosis (NC) in Spain.We conducted an observational, retrospective, multicentre study in NC patients who received IR cysteamine for at least 12 months, switched to ER cysteamine, and received it for at least 6 months before inclusion.Data were collected from nine patients (four children, five adults) 36 months before and after the switch. Despite the highly selected population, an improvement in growth, particularly in children and a significant reduction in hospitalization days was observed. A decrease in halitosis, body odour and gastrointestinal effects was reported in most of the patients who suffered before the switch, and the use of proton pump inhibitors (PPIs) decreased in some patients. The estimated glomerular filtration rate (eGFR) remained stable in patients with preserved kidney function. No significant changes in white blood cell (WBC) cystine levels were observed after the switch. There was no significant difference in the cysteamine dose received. However, some patients were receiving <50% of the recommended dose of cysteamine before and after the switch and showed elevated levels of WBC cystine.Switching from IR to ER cysteamine in clinical practice reduces hospital stays, improves nutritional status and growth in paediatric patients and could help to enhance treatment tolerability by reducing side effects. Furthermore, the dosing of ER cysteamine could promote therapeutic compliance and positively affect the quality of life of the NC population. Keywords: effectiveness, extended-release cysteamine, immediate-release cysteamine, nephropathic cystinosis, switching Author: Ariceta Gema, Santos Fernando, López Muñiz Andrés, Hermida Alvaro, Matoses Maria Luisa, Ventura Ana, Martin-Moreno Paloma Leticia, González Esther, Acuña Laura, Giner Elisa, Vara Julia Creator: OUP Producer: Acrobat Distiller 10.0.0 (Windows) CreationDate: Tue Apr 16 04:02:37 2024 CEST ModDate: Tue Apr 16 04:03:11 2024 CEST Custom Metadata: yes Metadata Stream: yes Tagged: yes UserProperties: no Suspects: no Form: none JavaScript: no Pages: 11 Encrypted: no Page size: 612.284 x 790.866 pts Page rot: 0 File size: 1461386 bytes Optimized: yes PDF version: 1.3 name type encoding emb sub uni object ID ------------------------------------ ----------------- ---------------- --- --- --- --------- FFCBGN+RMTMI Type 1C Custom yes yes yes 1215 0 FFCAKA+CaeciliaLTStd-Bold Type 1C WinAnsi yes yes no 1220 0 FFCBFM+CaeciliaLTStd-Heavy Type 1C WinAnsi yes yes no 1223 0 FFCAIN+CaeciliaLTStd-Italic Type 1C WinAnsi yes yes no 1226 0 FFCAIP+CaeciliaLTStd-Roman Type 1C Custom yes yes yes 1229 0 FFCAIO+dummy-space Type 1C WinAnsi yes yes no 1234 0 FFCKJC+Futura-Bold Type 1C WinAnsi yes yes no 18 0 FFCLDO+MTSY Type 1C Custom yes yes yes 183 0 FFCPLC+MyriadPro-Regular Type 1C WinAnsi yes yes no 190 0 FFDDGN+TimesNewRomanPS-ItalicMT Type 1C WinAnsi yes yes no 113 0 FFDJJG+TimesNewRomanPS-BoldItalicMT Type 1C WinAnsi yes yes no 116 0 FFDDGM+TimesNewRomanPS-BoldMT Type 1C WinAnsi yes yes no 119 0 FFDCPO+CaeciliaLTStd-BoldItalic Type 1C WinAnsi yes yes no 122 0 FFDDGO+TimesNewRomanPSMT Type 1C WinAnsi yes yes no 125 0 Jhove (Rel. 1.28.0, 2023-05-18) Date: 2026-10-03 04:50:08 CEST RepresentationInformation: sfae049.pdf ReportingModule: PDF-hul, Rel. 1.12.4 (2023-03-16) LastModified: 2026-10-02 18:55:14 CEST Size: 1461386 Format: PDF Version: 1.3 Status: Well-Formed and valid SignatureMatches: PDF-hul MIMEtype: application/pdf Profile: Linearized PDF, Tagged PDF PDFMetadata: Objects: 1293 FreeObjects: 1 IncrementalUpdates: 1 DocumentCatalog: ViewerPreferences: HideToolbar: false HideMenubar: false HideWindowUI: false FitWindow: false CenterWindow: false DisplayDocTitle: true NonFullScreenPageMode: UseNone Direction: L2R ViewArea: CropBox ViewClip: CropBox PrintArea: CropBox PageClip: CropBox PageLayout: OneColumn PageMode: UseOutlines Language: English Outlines: Item: Title: INTRODUCTION Destination: section*.2 Item: Title: MATERIALS AND METHODS Destination: section*.4 Item: Title: Study design Destination: section*.6 Item: Title: Study population Destination: section*.8 Item: Title: Study objectives Destination: section*.10 Item: Title: Statistical analysis Destination: section*.12 Item: Title: RESULTS Destination: section*.14 Item: Title: Study participants Destination: section*.16 Item: Title: Baseline characteristics Destination: section*.18 Item: Title: Clinical results Destination: section*.20 Item: Title: DISCUSSION Destination: section*.22 Item: Title: Study limitations Destination: section*.24 Item: Title: SUPPLEMENTARY DATA Destination: section*.26 Item: Title: ACKNOWLEDGEMENTS Destination: section*.28 Item: Title: FUNDING Destination: section*.30 Item: Title: AUTHORS’ CONTRIBUTIONS Destination: section*.32 Item: Title: DATA AVAILABILITY STATEMENT Destination: section*.34 Item: Title: CONFLICT OF INTEREST STATEMENT Destination: section*.36 Item: Title: REFERENCES Destination: section*.38 Info: Title: Switching from immediate- to extended-release cysteamine in patients with nephropathic cystinosis: from clinical trials to clinical practice Author: Ariceta Gema, Santos Fernando, López Muñiz Andrés, Hermida Alvaro, Matoses Maria Luisa, Ventura Ana, Martin-Moreno Paloma Leticia, González Esther, Acuña Laura, Giner Elisa, Vara Julia Subject: DOI: 10.1093/ckj/sfae049 Clinical Kidney Journal, 17, 4, 06-03-2024. Abstract: The purpose of this study is to evaluate the effectiveness and safety of switching from immediate-release (IR) to extended-release (ER) cysteamine in patients with nephropathic cystinosis (NC) in Spain.We conducted an observational, retrospective, multicentre study in NC patients who received IR cysteamine for at least 12 months, switched to ER cysteamine, and received it for at least 6 months before inclusion.Data were collected from nine patients (four children, five adults) 36 months before and after the switch. Despite the highly selected population, an improvement in growth, particularly in children and a significant reduction in hospitalization days was observed. A decrease in halitosis, body odour and gastrointestinal effects was reported in most of the patients who suffered before the switch, and the use of proton pump inhibitors (PPIs) decreased in some patients. The estimated glomerular filtration rate (eGFR) remained stable in patients with preserved kidney function. No significant changes in white blood cell (WBC) cystine levels were observed after the switch. There was no significant difference in the cysteamine dose received. However, some patients were receiving <50% of the recommended dose of cysteamine before and after the switch and showed elevated levels of WBC cystine.Switching from IR to ER cysteamine in clinical practice reduces hospital stays, improves nutritional status and growth in paediatric patients and could help to enhance treatment tolerability by reducing side effects. Furthermore, the dosing of ER cysteamine could promote therapeutic compliance and positively affect the quality of life of the NC population. 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Published by Oxford University Press on behalf of the ERA. This is an Open Access article distributed under the terms of the Creative Commons Attribution License (https://creativecommons.org/licenses/by/4.0/), which permits unrestricted reuse, distribution, and reproduction in any medium, provided the original work is properly cited. All Rights Reserved application/pdf doi:10.1093/ckj/sfae049 © The Author(s) 2024. Published by Oxford University Press on behalf of the ERA. This is an Open Access article distributed under the terms of the Creative Commons Attribution License (https://creativecommons.org/licenses/by/4.0/), which permits unrestricted reuse, distribution, and reproduction in any medium, provided the original work is properly cited. Ariceta Gema Santos Fernando López Muñiz Andrés Hermida Alvaro Matoses Maria Luisa Ventura Ana Martin-Moreno Paloma Leticia González Esther Acuña Laura Giner Elisa Vara Julia Oxford University Press DOI: 10.1093/ckj/sfae049 Clinical Kidney Journal, 17, 4, 06-03-2024. Abstract: The purpose of this study is to evaluate the effectiveness and safety of switching from immediate-release (IR) to extended-release (ER) cysteamine in patients with nephropathic cystinosis (NC) in Spain.We conducted an observational, retrospective, multicentre study in NC patients who received IR cysteamine for at least 12&#x00A0;months, switched to ER cysteamine, and received it for at least 6&#x00A0;months before inclusion.Data were collected from nine patients (four children, five adults) 36&#x00A0;months before and after the switch. Despite the highly selected population, an improvement in growth, particularly in children and a significant reduction in hospitalization days was observed. A decrease in halitosis, body odour and gastrointestinal effects was reported in most of the patients who suffered before the switch, and the use of proton pump inhibitors (PPIs) decreased in some patients. The estimated glomerular filtration rate (eGFR) remained stable in patients with preserved kidney function. No significant changes in white blood cell (WBC) cystine levels were observed after the switch. There was no significant difference in the cysteamine dose received. However, some patients were receiving &#x003C;50&#x0025; of the recommended dose of cysteamine before and after the switch and showed elevated levels of WBC cystine.Switching from IR to ER cysteamine in clinical practice reduces hospital stays, improves nutritional status and growth in paediatric patients and could help to enhance treatment tolerability by reducing side effects. Furthermore, the dosing of ER cysteamine could promote therapeutic compliance and positively affect the quality of life of the NC population. effectiveness extended-release cysteamine immediate-release cysteamine nephropathic cystinosis switching Switching from immediate- to extended-release cysteamine in patients with nephropathic cystinosis: from clinical trials to clinical practice Ariceta Gema, Santos Fernando, López Muñiz Andrés, Hermida Alvaro, Matoses Maria Luisa, Ventura Ana, Martin-Moreno Paloma Leticia, González Esther, Acuña Laura, Giner Elisa, Vara Julia uuid:efada41f-c676-3969-8284-11d823b81dbf uuid:67f9fa83-f401-40ca-b630-fda105aa07f4 True Pages: Page: Label: 1 Annotations: Annotation: Subtype: Link Rect: 62, 683, 116, 694 Annotation: Subtype: Link Rect: 314, 709, 426, 719 Annotation: Subtype: Link Rect: 151, 512, 165, 529 Annotation: Subtype: Link Rect: 163, 518, 170, 530 Destination: 1198 Annotation: Subtype: Link Rect: 282, 518, 289, 530 Destination: 1198 Annotation: 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