Early Outcomes in Children With Antineutrophil Cytoplasmic Antibody–Associated Vasculitis

Abstract

This is the largest study to date to assess disease outcomes in pediatric AAV. Although the study showed that a significant proportion of patients did not achieve remission, the majority of patients responded to treatment. Unfortunately, more than one-half of this patient cohort experienced damage to various organ systems early in their disease course.

Epidemiology and Outcomes of Granulomatosis With Polyangiitis in Pediatric and Working‐Age Adult Populations In the United States

“…Our study demonstrated a slight female predominance in both pediatric and adult GPA cohorts, confirming the female predominance of pediatric GPA, which has been observed in nearly all previous studies (20)(21)(22)(27)(28)(29)37) and some prior studies focused on adult GPA patients (6,19,38). In contrast, higher male proportions (M:F ratio 1.2-1.6:1) or equal sex distributions have been reported in a number of other large adult population-based studies (7)(8)(9)(10)39,40).…”

“…Although we suspected that ESRD would take many years to develop and therefore would be found infrequently in children, we found similar rates of ESRD among pediatric and working-age adult GPA patients, and that the majority of patients progressed to ESRD in the first year of their disease. The ARChiVe registry very recently published 12 month outcomes for 105 pediatric patients with antineutrophil cytoplasmic antibody-associated vasculitis (AAV) of which 85 patients had GPA (although the data were aggregated and not separated from the other AAV patients)(37). This study found that 11.4% of the 105 pediatric AAV patients had ESRD at 1 year which is comparable to the 16.4% of pediatric GPA patients with ESRD in our cohort.…”

Coughing up clues: 16-year-old girl with acute haemoptysis

“…Other clinical features include constitutional symptoms (fever, lethargy, weight loss), ocular symptoms (conjunctivitis, keratitis), skin and musculoskeletal manifestations (arthralgia, muscle pain) symptoms 5 7. It is important to maintain a high index of suspicion for GPA in any patient who presents with haemoptysis and multisystem illness to ensure prompt recognition of the disease, as patients can experience acute and rapid deterioration even while appropriate workup is being conducted 6…”

“…The 5-year survival rate for paediatric GPA is >90% predominantly due to advancement in available therapies. Nonetheless, limited long-term outcome studies have shown that ongoing monitoring is paramount to ensure early detection of disease relapse, as this is common in many patients 7…”

Primary Vasculitis in Childhood: GPA and MPA in Childhood

“…Improvement of PVAS score of at least 50% from time of diagnosis to post-induction was seen in 92% of patients. Vasculitis associated disease damage with PVDI scores ≥1 was identified in more than 60% of the patient cohort early in their disease course ( 73 ).…”