Rituximab in Idiopathic Pulmonary Hemosiderosis in Children: A Novel and Less Toxic Treatment Option
Idiopathic pulmonary hemosiderosis (IPH) is a rare, potentially life-threatening chronic disease. Steroids are the cornerstone of treatment, even though toxicity and side-effects are very common. Recently, rituximab (RTX) has been suggested as a treatment option, although evidence for its efficacy a...
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| Format: | Article |
| Language: | English |
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MDPI AG
2022-12-01
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| Series: | Pharmaceuticals |
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| Online Access: | https://www.mdpi.com/1424-8247/15/12/1549 |
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| author | Suzanne W. J. Terheggen-Lagro Eric G. Haarman Niels W. Rutjes J. Merlijn van den Berg Dieneke Schonenberg-Meinema |
| author_facet | Suzanne W. J. Terheggen-Lagro Eric G. Haarman Niels W. Rutjes J. Merlijn van den Berg Dieneke Schonenberg-Meinema |
| author_sort | Suzanne W. J. Terheggen-Lagro |
| collection | DOAJ |
| description | Idiopathic pulmonary hemosiderosis (IPH) is a rare, potentially life-threatening chronic disease. Steroids are the cornerstone of treatment, even though toxicity and side-effects are very common. Recently, rituximab (RTX) has been suggested as a treatment option, although evidence for its efficacy and long-term safety is lacking. We describe the disease course of two pediatric patients with IPH that were treated with RTX for over 4 years. Demographics, treatments, and clinical variables such as growth, infections, imaging follow-up by CT, and data from pulmonary function tests were retrospectively described. These are the first two cases described with a long-term follow-up of pediatric IPH patients treated with RTX. RTX was well-tolerated and prevented outbreaks of bleeding. In addition, RTX had a robust steroid-sparing effect resulting in the improvement of growth, pulmonary function, and CT abnormalities. |
| first_indexed | 2024-03-09T15:59:00Z |
| format | Article |
| id | doaj.art-0f434a4dfb9844c5b7ae2a4b76a1fbc0 |
| institution | Directory Open Access Journal |
| issn | 1424-8247 |
| language | English |
| last_indexed | 2024-03-09T15:59:00Z |
| publishDate | 2022-12-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Pharmaceuticals |
| spelling | doaj.art-0f434a4dfb9844c5b7ae2a4b76a1fbc02023-11-24T17:17:07ZengMDPI AGPharmaceuticals1424-82472022-12-011512154910.3390/ph15121549Rituximab in Idiopathic Pulmonary Hemosiderosis in Children: A Novel and Less Toxic Treatment OptionSuzanne W. J. Terheggen-Lagro0Eric G. Haarman1Niels W. Rutjes2J. Merlijn van den Berg3Dieneke Schonenberg-Meinema4Department of Pediatric Pulmonology and Allergy, Amsterdam University Medical Centres, University of Amsterdam, 1105 AZ Amsterdam, The NetherlandsDepartment of Pediatric Pulmonology and Allergy, Amsterdam University Medical Centres, University of Amsterdam, 1105 AZ Amsterdam, The NetherlandsDepartment of Pediatric Pulmonology and Allergy, Amsterdam University Medical Centres, University of Amsterdam, 1105 AZ Amsterdam, The NetherlandsDepartment of Pediatric Immunology, Rheumatology and Infectious Diseases, Amsterdam University Medical Centres, University of Amsterdam, 1105 AZ Amsterdam, The NetherlandsDepartment of Pediatric Immunology, Rheumatology and Infectious Diseases, Amsterdam University Medical Centres, University of Amsterdam, 1105 AZ Amsterdam, The NetherlandsIdiopathic pulmonary hemosiderosis (IPH) is a rare, potentially life-threatening chronic disease. Steroids are the cornerstone of treatment, even though toxicity and side-effects are very common. Recently, rituximab (RTX) has been suggested as a treatment option, although evidence for its efficacy and long-term safety is lacking. We describe the disease course of two pediatric patients with IPH that were treated with RTX for over 4 years. Demographics, treatments, and clinical variables such as growth, infections, imaging follow-up by CT, and data from pulmonary function tests were retrospectively described. These are the first two cases described with a long-term follow-up of pediatric IPH patients treated with RTX. RTX was well-tolerated and prevented outbreaks of bleeding. In addition, RTX had a robust steroid-sparing effect resulting in the improvement of growth, pulmonary function, and CT abnormalities.https://www.mdpi.com/1424-8247/15/12/1549pulmonary hemosiderosisrituximabpediatricpulmonary bleeding |
| spellingShingle | Suzanne W. J. Terheggen-Lagro Eric G. Haarman Niels W. Rutjes J. Merlijn van den Berg Dieneke Schonenberg-Meinema Rituximab in Idiopathic Pulmonary Hemosiderosis in Children: A Novel and Less Toxic Treatment Option Pharmaceuticals pulmonary hemosiderosis rituximab pediatric pulmonary bleeding |
| title | Rituximab in Idiopathic Pulmonary Hemosiderosis in Children: A Novel and Less Toxic Treatment Option |
| title_full | Rituximab in Idiopathic Pulmonary Hemosiderosis in Children: A Novel and Less Toxic Treatment Option |
| title_fullStr | Rituximab in Idiopathic Pulmonary Hemosiderosis in Children: A Novel and Less Toxic Treatment Option |
| title_full_unstemmed | Rituximab in Idiopathic Pulmonary Hemosiderosis in Children: A Novel and Less Toxic Treatment Option |
| title_short | Rituximab in Idiopathic Pulmonary Hemosiderosis in Children: A Novel and Less Toxic Treatment Option |
| title_sort | rituximab in idiopathic pulmonary hemosiderosis in children a novel and less toxic treatment option |
| topic | pulmonary hemosiderosis rituximab pediatric pulmonary bleeding |
| url | https://www.mdpi.com/1424-8247/15/12/1549 |
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