Clinical characteristics and outcomes of childhood-onset ANCA-associated vasculitis: a French nationwide study

Sacri, Anne‐Sylvia; Chambaraud, T.; Ranchin, Bruno; Florkin, Benoît; See, Hélène; Decramer, Stéphane; Flodrops, Hugues; Ulinski, Tim; Allain-Launay, Emma; Boyer, Olivia; Dunand, Olivier; Fischbach, Michel; Hachulla, É.; Piètrement, Christine; Pogamp, P. Le; Stéphan, Jean-Louis; Belot, Alexandre; Nivet, Hubert; Nobili, François; Guillevin, Loı̈c; Quartier, Pierre; Deschênes, Georges; Salomon, Rémi; Essig, Marie; Harambat, Jérôme

doi:10.1093/ndt/gfv011

Cited by 62 publications

(118 citation statements)

References 55 publications

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“…Until now, the 2 largest pediatric studies in which outcomes have been described have included respective total sample sizes of 66 patients and 35 patients with AAV (20,35). This is the largest cohort study to date to report pediatric outcomes in this group of diseases.…”

Section: Discussionmentioning

confidence: 99%

“…Despite these advances, disease relapses occur in up to 30-50% of adult patients (17)(18)(19). A recent retrospective study of 66 children with AAV from France assessed outcomes in children who had been followed up for a median of 5.2 years (20); 90% of the patients achieved remission after a median time of 6.7 months (interquartile range [IQR] 4.9-10.1), but 41% experienced at least 1 relapse after a median time of 29 months (IQR . A recent retrospective study of 66 children with AAV from France assessed outcomes in children who had been followed up for a median of 5.2 years (20); 90% of the patients achieved remission after a median time of 6.7 months (interquartile range [IQR] 4.9-10.1), but 41% experienced at least 1 relapse after a median time of 29 months (IQR .…”

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confidence: 99%

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Early Outcomes in Children With Antineutrophil Cytoplasmic Antibody–Associated Vasculitis

Morishita

Moorthy

Lubieniecka

et al. 2017

Arthritis & Rheumatology

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Section: Discussionmentioning

confidence: 99%

mentioning

confidence: 99%

Early Outcomes in Children With Antineutrophil Cytoplasmic Antibody–Associated Vasculitis

Morishita

Moorthy

Lubieniecka

et al. 2017

Arthritis & Rheumatology

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“…In these diseases, autoantibodies directed against neutrophil granule components myeloperoxidase and proteinase-3 cause inflammation and destruction of pulmonary capillaries and small vessels, leading to pulmonary hemorrhage [3]. Children with ANCA-associated vasculitis often present with fever, malaise, and renal disease in addition to pulmonary hemorrhage [4-6]. Systemic lupus erythematosis (SLE) has also been recognized as cause of immune mediated pulmonary hemorrhage in children and has been associated with a variety of immunological aberrations and associations [7].…”

Section: Introductionmentioning

confidence: 99%

Copa Syndrome: a Novel Autosomal Dominant Immune Dysregulatory Disease

et al. 2016

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Inherently defective immunity typically results in either ineffective host defense, immune regulation, or both. As a category of primary immunodeficiency diseases, those that impair immune regulation can lead to autoimmunity and/or autoinflammation. In this review we focus on one of the most recently discovered primary immunodeficiencies that leads to immune dysregulation: “Copa syndrome”. Copa syndrome is named for the gene mutated in the disease, which encodes the alpha subunit of the coatomer complex-I that, in aggregate, is devoted to transiting molecular cargo from the Golgi complex to the endoplasmic reticulum (ER). Copa syndrome is autosomal dominant with variable expressivity and results from mutations affecting a narrow amino acid stretch in the COPA gene-encoding COPα protein. Patients with these mutations typically develop arthritis and interstitial lung disease with pulmonary hemorrhage representing a striking feature. Immunologically Copa syndrome is associated with autoantibody development, increased Th17 cells and pro-inflammatory cytokine expression including IL-1β and IL-6. Insights have also been gained into the underlying mechanism of Copa syndrome, which include excessive ER stress owing to the impaired return of proteins from the Golgi, and presumably resulting aberrant cellular autophagy. As such it represents a novel cellular disorder of intracellular trafficking associated with a specific clinical presentation and phenotype.

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“…In contrast, because bronchoscopy and imaging (Fig 2) cannot distinguish capillaritis from idiopathic pulmonary hemosiderosis, a lung biopsy is required to diagnose idiopathic pulmonary capillaritis. 68 While most reports on pulmonary vasculitis in children have been small case series, a larger study of 66 children with ANCA-associated vasculitis from Sacri et al 69 reported a female predominance and an average age of diagnosis of 11.5 years. Importantly, this cohort illustrated the chronic nature of the disease, with a relapse rate of > 50% within 5 years of diagnosis.…”

Section: Pulmonary Vasculitismentioning

confidence: 99%