Quantifying side effects and caregiver burdens of pediatric pulmonary hypertension therapies
Abstract Background and objectives Pulmonary hypertension (PH) is a rare, but serious disease among children. However, PH has been primarily evaluated among adults. Consequently, treatment therapies have not been fully evaluated among pediatric populations and are used in an ‘off label’ manner. The...
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| Format: | Article |
| Language: | English |
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BMC
2023-01-01
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| Series: | BMC Pediatrics |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s12887-023-03860-2 |
| _version_ | 1828051560204075008 |
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| author | Erik J. Nelson Ella Cook Samara Nelson Rebecca Brown Megan Pierce Ashley Bangerter Seelos Heather Stickle Michael Johansen |
| author_facet | Erik J. Nelson Ella Cook Samara Nelson Rebecca Brown Megan Pierce Ashley Bangerter Seelos Heather Stickle Michael Johansen |
| author_sort | Erik J. Nelson |
| collection | DOAJ |
| description | Abstract Background and objectives Pulmonary hypertension (PH) is a rare, but serious disease among children. However, PH has been primarily evaluated among adults. Consequently, treatment therapies have not been fully evaluated among pediatric populations and are used in an ‘off label’ manner. The purpose of this study was to estimate the side effect profiles of the most commonly prescribed pediatric PH therapies and to understand the burdens placed upon families caring for children living with PH. Methods Participants were recruited online through the “Families of children with pulmonary hypertension” Facebook group and asked to complete a survey about PH treatments. Results A total of 139 parents of a child living with PH completed the survey. Almost all children used ≥ 1 medication to treat PH, with 52% using ≥ 3 medications. The highest average number of side effects was reported by users of Treprostinil, Selexipag and type-5 phosphodiesterase (PDE5) inhibitors. The most common side effects were skin flushing, headache, nasal congestion, joint/muscle pain, and nausea. In terms of accessing care, 81% travel ≥ 20 miles and 68% travel for ≥ 60 min to receive care. Conclusions We found an array of treatment combinations employed to mitigate symptoms of PH in children, with a wide range of side effects. We also found a large, unseen economic, emotional, and time burden of caring for a child living with PH. Further research is warranted to understand the clinical implications of these side effects to move towards labeled usage of these therapies rather than post-hoc off-label usage. |
| first_indexed | 2024-04-10T19:40:29Z |
| format | Article |
| id | doaj.art-022c44189f484a1b85081a242fc65a24 |
| institution | Directory Open Access Journal |
| issn | 1471-2431 |
| language | English |
| last_indexed | 2024-04-10T19:40:29Z |
| publishDate | 2023-01-01 |
| publisher | BMC |
| record_format | Article |
| series | BMC Pediatrics |
| spelling | doaj.art-022c44189f484a1b85081a242fc65a242023-01-29T12:22:02ZengBMCBMC Pediatrics1471-24312023-01-012311910.1186/s12887-023-03860-2Quantifying side effects and caregiver burdens of pediatric pulmonary hypertension therapiesErik J. Nelson0Ella Cook1Samara Nelson2Rebecca Brown3Megan Pierce4Ashley Bangerter Seelos5Heather Stickle6Michael Johansen7Department of Public Health, Brigham Young UniversityDepartment of Public Health, Brigham Young UniversityEmma Eccles Jones College of Education & Human Services, Utah State UniversityDepartment of Public Health, Brigham Young UniversityDepartment of Public Health, Brigham Young UniversityDepartment of Public Health, Brigham Young UniversityDepartment of Public Health, Brigham Young UniversityIndiana University School of MedicineAbstract Background and objectives Pulmonary hypertension (PH) is a rare, but serious disease among children. However, PH has been primarily evaluated among adults. Consequently, treatment therapies have not been fully evaluated among pediatric populations and are used in an ‘off label’ manner. The purpose of this study was to estimate the side effect profiles of the most commonly prescribed pediatric PH therapies and to understand the burdens placed upon families caring for children living with PH. Methods Participants were recruited online through the “Families of children with pulmonary hypertension” Facebook group and asked to complete a survey about PH treatments. Results A total of 139 parents of a child living with PH completed the survey. Almost all children used ≥ 1 medication to treat PH, with 52% using ≥ 3 medications. The highest average number of side effects was reported by users of Treprostinil, Selexipag and type-5 phosphodiesterase (PDE5) inhibitors. The most common side effects were skin flushing, headache, nasal congestion, joint/muscle pain, and nausea. In terms of accessing care, 81% travel ≥ 20 miles and 68% travel for ≥ 60 min to receive care. Conclusions We found an array of treatment combinations employed to mitigate symptoms of PH in children, with a wide range of side effects. We also found a large, unseen economic, emotional, and time burden of caring for a child living with PH. Further research is warranted to understand the clinical implications of these side effects to move towards labeled usage of these therapies rather than post-hoc off-label usage.https://doi.org/10.1186/s12887-023-03860-2Pulmonary hypertensionTreatment side effectsFacebookAccess to careCaregiver burden |
| spellingShingle | Erik J. Nelson Ella Cook Samara Nelson Rebecca Brown Megan Pierce Ashley Bangerter Seelos Heather Stickle Michael Johansen Quantifying side effects and caregiver burdens of pediatric pulmonary hypertension therapies BMC Pediatrics Pulmonary hypertension Treatment side effects Access to care Caregiver burden |
| title | Quantifying side effects and caregiver burdens of pediatric pulmonary hypertension therapies |
| title_full | Quantifying side effects and caregiver burdens of pediatric pulmonary hypertension therapies |
| title_fullStr | Quantifying side effects and caregiver burdens of pediatric pulmonary hypertension therapies |
| title_full_unstemmed | Quantifying side effects and caregiver burdens of pediatric pulmonary hypertension therapies |
| title_short | Quantifying side effects and caregiver burdens of pediatric pulmonary hypertension therapies |
| title_sort | quantifying side effects and caregiver burdens of pediatric pulmonary hypertension therapies |
| topic | Pulmonary hypertension Treatment side effects Access to care Caregiver burden |
| url | https://doi.org/10.1186/s12887-023-03860-2 |
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