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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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Autores principales: Terheggen-Lagro, Suzanne W. J., Haarman, Eric G., Rutjes, Niels W., van den Berg, J. Merlijn, Schonenberg-Meinema, Dieneke
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9784529/
https://www.ncbi.nlm.nih.gov/pubmed/36559000
http://dx.doi.org/10.3390/ph15121549
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author Terheggen-Lagro, Suzanne W. J.
Haarman, Eric G.
Rutjes, Niels W.
van den Berg, J. Merlijn
Schonenberg-Meinema, Dieneke
author_facet Terheggen-Lagro, Suzanne W. J.
Haarman, Eric G.
Rutjes, Niels W.
van den Berg, J. Merlijn
Schonenberg-Meinema, Dieneke
author_sort Terheggen-Lagro, Suzanne W. J.
collection PubMed
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.
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spelling pubmed-97845292022-12-24 Rituximab in Idiopathic Pulmonary Hemosiderosis in Children: A Novel and Less Toxic Treatment Option Terheggen-Lagro, Suzanne W. J. Haarman, Eric G. Rutjes, Niels W. van den Berg, J. Merlijn Schonenberg-Meinema, Dieneke Pharmaceuticals (Basel) Case Report 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. MDPI 2022-12-13 /pmc/articles/PMC9784529/ /pubmed/36559000 http://dx.doi.org/10.3390/ph15121549 Text en © 2022 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Case Report
Terheggen-Lagro, Suzanne W. J.
Haarman, Eric G.
Rutjes, Niels W.
van den Berg, J. Merlijn
Schonenberg-Meinema, Dieneke
Rituximab in Idiopathic Pulmonary Hemosiderosis in Children: A Novel and Less Toxic Treatment Option
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 Case Report
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9784529/
https://www.ncbi.nlm.nih.gov/pubmed/36559000
http://dx.doi.org/10.3390/ph15121549
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