Dynamics of abdominal symptoms during the start of a new therapy with elexacaftor/tezacaftor/ivacaftor using the novel CFAbd-day2day questionnaire
Background: Elexacaftor–tezacaftor–ivacaftor (ETI) is a novel, highly effective CFTR modulator combination proven to enhance lung function and body weight in people with cystic fibrosis (pwCF) carrying a F508del mutation. Recently, we revealed significant reductions in abdominal symptoms (AS) in Ger...
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2023-10-01
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| Series: | Frontiers in Pharmacology |
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fphar.2023.1167407/full |
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| author | Jochen G. Mainz Jochen G. Mainz Anton Barucha Anton Barucha Pu Huang Pu Huang Lilith Bechinger Lilith Bechinger Franziska Duckstein Franziska Duckstein Louise Polte Louise Polte Pauline Sadrieh Pauline Sadrieh Lutz Nährlich Olaf Eickmeier Suzanne Van Dullemen Patience Eschenhagen Carsten Schwarz Stefan Lüth Carlos Zagoya Carlos Zagoya Ute Graepler-Mainka |
| author_facet | Jochen G. Mainz Jochen G. Mainz Anton Barucha Anton Barucha Pu Huang Pu Huang Lilith Bechinger Lilith Bechinger Franziska Duckstein Franziska Duckstein Louise Polte Louise Polte Pauline Sadrieh Pauline Sadrieh Lutz Nährlich Olaf Eickmeier Suzanne Van Dullemen Patience Eschenhagen Carsten Schwarz Stefan Lüth Carlos Zagoya Carlos Zagoya Ute Graepler-Mainka |
| author_sort | Jochen G. Mainz |
| collection | DOAJ |
| description | Background: Elexacaftor–tezacaftor–ivacaftor (ETI) is a novel, highly effective CFTR modulator combination proven to enhance lung function and body weight in people with cystic fibrosis (pwCF) carrying a F508del mutation. Recently, we revealed significant reductions in abdominal symptoms (AS) in German, British, and Irish pwCF after 24–26 weeks of ETI using the CFAbd-Score, the first patient-reported outcome measure (PROM) specifically developed and validated for pwCF following FDA guidelines. Notably, many pwCF reported marked changes in their AS during the first days of the new treatment. To capture these immediate effects, we developed the CFAbd-day2day, a CF-specific GI-diary, following FDA and COSMIN guidelines.Aim: To prospectively capture the immediate dynamics of AS using the CFAbd-day2day 14 days before and 14–28 days after ETI initiation. In addition, we aim to provide validation steps of the novel PROM concerning sensitivity to changes.Methods: To develop the CFAbd-day2day, focus groups (community voice = pwCF and their proxies and CF specialists from different fields) were repeatedly consulted. Before and during the new ETI therapy, pwCF prospectively scored AS on a daily basis with the CFAbd-day2day.Results: Altogether, 45 pwCF attended in five CF centers prospectively completed the CFAbd-day2day before (mean ± sd:14 ± 7 days) and after (mean ± sd: 28 ± 23 days) ETI initiation. On the one hand, cumulative scores significantly decreased during the 3–4-week time frame after ETI initiation, compared to 2 weeks prior to therapy. On the other hand, many patients who revealed a relatively stable level of AS before ETI reported changes during the first days of treatment with the highly effective CFTR modulators. Factors like pain and flatulence increased in up to 21% of patients during the first 14 days of therapy, but they improved during days 15–27.Conclusion: The CFAbd-day2day, specifically developed and in the process of validation to prospectively capture GI symptoms in pwCF, provides new substantial insights into the dynamics of AS in pwCF receiving a new treatment with ETI. This novel tool is also helpful in prospectively monitoring patients with specific GI problems. International implementation and further validation steps of the diary are ongoing. |
| first_indexed | 2024-03-11T12:19:41Z |
| format | Article |
| id | doaj.art-658abcaaf09940f9aee5d184e373a9e4 |
| institution | Directory Open Access Journal |
| issn | 1663-9812 |
| language | English |
| last_indexed | 2024-03-11T12:19:41Z |
| publishDate | 2023-10-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Pharmacology |
| spelling | doaj.art-658abcaaf09940f9aee5d184e373a9e42023-11-07T03:13:14ZengFrontiers Media S.A.Frontiers in Pharmacology1663-98122023-10-011410.3389/fphar.2023.11674071167407Dynamics of abdominal symptoms during the start of a new therapy with elexacaftor/tezacaftor/ivacaftor using the novel CFAbd-day2day questionnaireJochen G. Mainz0Jochen G. Mainz1Anton Barucha2Anton Barucha3Pu Huang4Pu Huang5Lilith Bechinger6Lilith Bechinger7Franziska Duckstein8Franziska Duckstein9Louise Polte10Louise Polte11Pauline Sadrieh12Pauline Sadrieh13Lutz Nährlich14Olaf Eickmeier15Suzanne Van Dullemen16Patience Eschenhagen17Carsten Schwarz18Stefan Lüth19Carlos Zagoya20Carlos Zagoya21Ute Graepler-Mainka22CF-Center, Klinikum Westbrandenburg, Brandenburg an der Havel, GermanyMedizinische Hochschule Brandenburg (MHB), Universitätsklinikum, Brandenburg an der Havel, GermanyCF-Center, Klinikum Westbrandenburg, Brandenburg an der Havel, GermanyMedizinische Hochschule Brandenburg (MHB), Universitätsklinikum, Brandenburg an der Havel, GermanyCF-Center, Klinikum Westbrandenburg, Brandenburg an der Havel, GermanyMedizinische Hochschule Brandenburg (MHB), Universitätsklinikum, Brandenburg an der Havel, GermanyCF-Center, Klinikum Westbrandenburg, Brandenburg an der Havel, GermanyMedizinische Hochschule Brandenburg (MHB), Universitätsklinikum, Brandenburg an der Havel, GermanyCF-Center, Klinikum Westbrandenburg, Brandenburg an der Havel, GermanyMedizinische Hochschule Brandenburg (MHB), Universitätsklinikum, Brandenburg an der Havel, GermanyCF-Center, Klinikum Westbrandenburg, Brandenburg an der Havel, GermanyMedizinische Hochschule Brandenburg (MHB), Universitätsklinikum, Brandenburg an der Havel, GermanyCF-Center, Klinikum Westbrandenburg, Brandenburg an der Havel, GermanyMedizinische Hochschule Brandenburg (MHB), Universitätsklinikum, Brandenburg an der Havel, GermanyCF Center for Children Justus-Liebig-Universität Gießen, Universitätsklinikum Gießen-Marburg GmbH, Gießen, GermanyDivision of Allergy, Pulmonology and Cystic Fibrosis, Department for Children and Adolescents, University Hospital, Frankfurt, GermanyDivision of Allergy, Pulmonology and Cystic Fibrosis, Department for Children and Adolescents, University Hospital, Frankfurt, GermanyCF-Center, Klinikum Westbrandenburg, Potsdam, GermanyCF-Center, Klinikum Westbrandenburg, Potsdam, GermanyDepartment of Gastroenterology, Brandenburg Medical School (Theodor Fontane), Brandenburg an der Havel, GermanyCF-Center, Klinikum Westbrandenburg, Brandenburg an der Havel, GermanyMedizinische Hochschule Brandenburg (MHB), Universitätsklinikum, Brandenburg an der Havel, GermanyChildren’s Hospital, Eberhard-Karls-University, Tübingen, GermanyBackground: Elexacaftor–tezacaftor–ivacaftor (ETI) is a novel, highly effective CFTR modulator combination proven to enhance lung function and body weight in people with cystic fibrosis (pwCF) carrying a F508del mutation. Recently, we revealed significant reductions in abdominal symptoms (AS) in German, British, and Irish pwCF after 24–26 weeks of ETI using the CFAbd-Score, the first patient-reported outcome measure (PROM) specifically developed and validated for pwCF following FDA guidelines. Notably, many pwCF reported marked changes in their AS during the first days of the new treatment. To capture these immediate effects, we developed the CFAbd-day2day, a CF-specific GI-diary, following FDA and COSMIN guidelines.Aim: To prospectively capture the immediate dynamics of AS using the CFAbd-day2day 14 days before and 14–28 days after ETI initiation. In addition, we aim to provide validation steps of the novel PROM concerning sensitivity to changes.Methods: To develop the CFAbd-day2day, focus groups (community voice = pwCF and their proxies and CF specialists from different fields) were repeatedly consulted. Before and during the new ETI therapy, pwCF prospectively scored AS on a daily basis with the CFAbd-day2day.Results: Altogether, 45 pwCF attended in five CF centers prospectively completed the CFAbd-day2day before (mean ± sd:14 ± 7 days) and after (mean ± sd: 28 ± 23 days) ETI initiation. On the one hand, cumulative scores significantly decreased during the 3–4-week time frame after ETI initiation, compared to 2 weeks prior to therapy. On the other hand, many patients who revealed a relatively stable level of AS before ETI reported changes during the first days of treatment with the highly effective CFTR modulators. Factors like pain and flatulence increased in up to 21% of patients during the first 14 days of therapy, but they improved during days 15–27.Conclusion: The CFAbd-day2day, specifically developed and in the process of validation to prospectively capture GI symptoms in pwCF, provides new substantial insights into the dynamics of AS in pwCF receiving a new treatment with ETI. This novel tool is also helpful in prospectively monitoring patients with specific GI problems. International implementation and further validation steps of the diary are ongoing.https://www.frontiersin.org/articles/10.3389/fphar.2023.1167407/fullgastrointestinalpatient-reported outcome measureprospectivesymptom scorediaryCFAbd-Score |
| spellingShingle | Jochen G. Mainz Jochen G. Mainz Anton Barucha Anton Barucha Pu Huang Pu Huang Lilith Bechinger Lilith Bechinger Franziska Duckstein Franziska Duckstein Louise Polte Louise Polte Pauline Sadrieh Pauline Sadrieh Lutz Nährlich Olaf Eickmeier Suzanne Van Dullemen Patience Eschenhagen Carsten Schwarz Stefan Lüth Carlos Zagoya Carlos Zagoya Ute Graepler-Mainka Dynamics of abdominal symptoms during the start of a new therapy with elexacaftor/tezacaftor/ivacaftor using the novel CFAbd-day2day questionnaire Frontiers in Pharmacology gastrointestinal patient-reported outcome measure prospective symptom score diary CFAbd-Score |
| title | Dynamics of abdominal symptoms during the start of a new therapy with elexacaftor/tezacaftor/ivacaftor using the novel CFAbd-day2day questionnaire |
| title_full | Dynamics of abdominal symptoms during the start of a new therapy with elexacaftor/tezacaftor/ivacaftor using the novel CFAbd-day2day questionnaire |
| title_fullStr | Dynamics of abdominal symptoms during the start of a new therapy with elexacaftor/tezacaftor/ivacaftor using the novel CFAbd-day2day questionnaire |
| title_full_unstemmed | Dynamics of abdominal symptoms during the start of a new therapy with elexacaftor/tezacaftor/ivacaftor using the novel CFAbd-day2day questionnaire |
| title_short | Dynamics of abdominal symptoms during the start of a new therapy with elexacaftor/tezacaftor/ivacaftor using the novel CFAbd-day2day questionnaire |
| title_sort | dynamics of abdominal symptoms during the start of a new therapy with elexacaftor tezacaftor ivacaftor using the novel cfabd day2day questionnaire |
| topic | gastrointestinal patient-reported outcome measure prospective symptom score diary CFAbd-Score |
| url | https://www.frontiersin.org/articles/10.3389/fphar.2023.1167407/full |
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