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Published on: June 16, 2020
Clinical Improvement in Early Onset Interstitial Lung Disease Using Rituximab in Children With Antimelanoma
Malki Peskin1, Marilyn Mostowy2, Jennifer Velez3
1M. Peskin, MD, Pediatric Rheumatology, The Children's Hospital at Montefiore Pediatrics, Bronx, New York, USA.
Insights
Rituximab (RTX) treatment showed significant improvements in children with juvenile dermatomyositis (JDM) and anti-MDA5 antibodies, effectively managing interstitial lung disease (ILD) and other symptoms. This study highlights RTX as a promising therapy for severe JDM complications.
Area of Science:
- Pediatric Rheumatology
- Immunology
- Pulmonology
Background:
- Juvenile dermatomyositis (JDM) patients with anti-MDA5 antibodies face a high risk of severe complications, notably interstitial lung disease (ILD).
- Limited data exists on effective treatments for ILD and other severe manifestations in this specific JDM subgroup.
- Rituximab (RTX) is an immunosuppressive agent with potential therapeutic value in autoimmune conditions.
Purpose of the Study:
- To evaluate the efficacy of rituximab (RTX) in treating children diagnosed with juvenile dermatomyositis (JDM) and positive anti-MDA5 antibodies.
- To assess the impact of RTX on disease course, particularly interstitial lung disease (ILD), cutaneous, and musculoskeletal manifestations in this patient cohort.
Main Methods:
- Retrospective review of clinical and laboratory data from pediatric patients (aged 2-21 years) diagnosed with JDM, anti-MDA5 antibodies, and ILD.
- Patients included in the study received treatment with rituximab (RTX) between July 2012 and August 2021.
- Disease course, treatment response, and complications were analyzed before and after RTX initiation.
Main Results:
- Of 8 patients with anti-MDA5 antibodies, 5 (62.5%) had evidence of ILD; data was available for 4 patients.
- All treated patients received at least 5 courses of RTX, leading to steroid discontinuation within 12 months and reduced concurrent medications.
- Significant improvements or complete resolution of ILD indicators (CT scans, PFTs), active cutaneous, and musculoskeletal disease were observed in all patients.
Conclusions:
- This study suggests rituximab (RTX) is effective in managing severe complications of JDM associated with anti-MDA5 antibodies, including ILD.
- RTX demonstrated notable improvements in cutaneous and musculoskeletal symptoms alongside pulmonary manifestations.
- Further research is warranted to establish the definitive efficacy of RTX for JDM-related complications.
Objective:
Children with juvenile dermatomyositis (JDM) and antibodies to antimelanoma differentiation-associated gene 5 (anti-MDA5) are at increased risk of severe disease complications, including interstitial lung disease (ILD). Data regarding treatment of disease complications in this patient population are limited. In this study, we examined the disease course of children with JDM and anti-MDA5 antibodies before and after treatment with rituximab (RTX).
Methods:
Patients aged 2-21 years and seen at the Children's Hospital at Montefiore between July 2012 and August 2021, with a diagnosis of JDM, positive anti-MDA5 antibodies, and evidence of ILD, and who were treated with RTX were eligible for inclusion. Retrospective clinical and laboratory data were reviewed.
Results:
Five of 8 patients with positive anti-MDA5 antibodies had evidence of ILD (62.5%). Four patients had data available for review. All patients received at least 5 courses of RTX infusions, with discontinuation of steroids by an average of 12 months after starting RTX and a decrease to fewer than 2 concurrent medications by the fifth course of RTX. Indicators of ILD on high-resolution computed tomography and pulmonary function tests either improved or fully resolved over the course of RTX treatment for all patients. Patients also demonstrated resolution of active cutaneous manifestations and musculoskeletal disease activity.
Conclusion:
To our knowledge, this is the first study to examine the use of RTX in children with JDM and anti-MDA5 antibodies, with notable improvements in ILD, cutaneous, and musculoskeletal manifestations. Further studies are needed to better understand the efficacy of RTX for JDM disease-related complications.
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