TY - JOUR
T1 - Rituximab in Idiopathic Pulmonary Hemosiderosis in Children
T2 - A Novel and Less Toxic Treatment Option
AU - Terheggen-Lagro, Suzanne W. J.
AU - Haarman, Eric G.
AU - Rutjes, Niels W.
AU - van den Berg, J. Merlijn
AU - Schonenberg-Meinema, Dieneke
N1 - Publisher Copyright: © 2022 by the authors.
PY - 2022/12/1
Y1 - 2022/12/1
N2 - 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.
AB - 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.
KW - pediatric
KW - pulmonary bleeding
KW - pulmonary hemosiderosis
KW - rituximab
UR - http://www.scopus.com/inward/record.url?scp=85144730967&partnerID=8YFLogxK
U2 - https://doi.org/10.3390/ph15121549
DO - https://doi.org/10.3390/ph15121549
M3 - Article
C2 - 36559000
SN - 1424-8247
VL - 15
JO - Pharmaceuticals
JF - Pharmaceuticals
IS - 12
M1 - 1549
ER -