Michele Iudici1, Christian Pagnoux1, Pierre Quartier2, Matthias Büchler3, Ramiro Cevallos4, Pascal Cohen5, Claire de Moreuil6, Philippe Guilpain7, Alain Le Quellec7, Jacques Serratrice8, Benjamin Terrier5, Loïc Guillevin5, Luc Mouthon5, Xavier Puéchal9. 1. National Referral Center for Rare Systemic Autoimmune Diseases, Department of Internal Medicine, Hôpital Cochin, Assistance Publique - Hôpitaux de Paris (APHP), Paris, France. 2. Paris-Descartes University, Imagine Institute, Pediatric Immunology-Hematology and Rheumatology Unit, Necker-Enfants Malades, APHP, Paris, France. 3. CHU Bretonneau, Tours, France. 4. Centre Hospitalier Saint-Vincent, Strasbourg, France. 5. National Referral Center for Rare Systemic Autoimmune Diseases, Department of Internal Medicine, Hôpital Cochin, Assistance Publique - Hôpitaux de Paris (APHP), Paris, France; Université Paris Descartes, Paris, France. 6. CHU La Cavale Blanche, Brest, France. 7. CHU Saint-Eloi, Montpellier, France. 8. CHU La Timone, Marseille, France. 9. National Referral Center for Rare Systemic Autoimmune Diseases, Department of Internal Medicine, Hôpital Cochin, Assistance Publique - Hôpitaux de Paris (APHP), Paris, France; Université Paris Descartes, Paris, France. Electronic address: xavier.puechal@aphp.fr.
Abstract
OBJECTIVE: To investigate differences between childhood-onset ANCA-associated vasculitides (cAAVs) and matched adult-onset controls (aAAVs). METHODS: cAAV clinical pictures at onset and outcomes were compared to a randomly selected sample of aAAV patients from the French Vasculitis Study Group Registry. Cases and controls were matched for AAV (granulomatosis with polyangiitis [GPA], microscopic polyangiitis [MPA] or eosinophilic granulomatosis with polyangiitis [EGPA]), sex and year of enrollment. Medications, disease activity and damage were prospectively recorded. Kaplan-Meier curves and the log-rank test were used to analyze case-vs.-control differences for predefined outcomes. RESULTS: Comparing 35 cAAVs (25 GPA, 4 MPA, 6 EGPA) to 151 aAAVs (106 GPA, 17 MPA, 28 EGPA), their respective median follow-up durations were 71 and 64months (P=0.49), and, at baseline, children had less frequent myalgias (P=0.005) and peripheral neuropathy (P<0.001) but were more frequently febrile (P<0.05). Rates of renal involvement were comparable (13 [37%] cAAVs vs. 73 [48%] aAAVs; P=0.31). Initial GPA-associated ischemic abdominal pain and nasal cartilage damage were more common in cAAVs than aAAVs (P<0.05). During follow-up, the cAAV relapse rate was higher (24.5 vs. 18.7 flares per 100 patient-years; P<0.05) and, at last visit, cases had accumulated more damage, mostly ear, nose & throat sequelae (P=0.001), associated with longer maintenance therapy (P=0.03), than aAAV controls. Four (11.4%) cAAV and 13 (8.6%) aAAV patients died (P=0.53). CONCLUSION: cAAVs are severe diseases, characterized by a higher relapse rate, more accrued damage and longer maintenance therapy than for aAAVs.
OBJECTIVE: To investigate differences between childhood-onset ANCA-associated vasculitides (cAAVs) and matched adult-onset controls (aAAVs). METHODS: cAAV clinical pictures at onset and outcomes were compared to a randomly selected sample of aAAV patients from the French Vasculitis Study Group Registry. Cases and controls were matched for AAV (granulomatosis with polyangiitis [GPA], microscopic polyangiitis [MPA] or eosinophilic granulomatosis with polyangiitis [EGPA]), sex and year of enrollment. Medications, disease activity and damage were prospectively recorded. Kaplan-Meier curves and the log-rank test were used to analyze case-vs.-control differences for predefined outcomes. RESULTS: Comparing 35 cAAVs (25 GPA, 4 MPA, 6 EGPA) to 151 aAAVs (106 GPA, 17 MPA, 28 EGPA), their respective median follow-up durations were 71 and 64months (P=0.49), and, at baseline, children had less frequent myalgias (P=0.005) and peripheral neuropathy (P<0.001) but were more frequently febrile (P<0.05). Rates of renal involvement were comparable (13 [37%] cAAVs vs. 73 [48%] aAAVs; P=0.31). Initial GPA-associated ischemic abdominal pain and nasal cartilage damage were more common in cAAVs than aAAVs (P<0.05). During follow-up, the cAAV relapse rate was higher (24.5 vs. 18.7 flares per 100 patient-years; P<0.05) and, at last visit, cases had accumulated more damage, mostly ear, nose & throat sequelae (P=0.001), associated with longer maintenance therapy (P=0.03), than aAAV controls. Four (11.4%) cAAV and 13 (8.6%) aAAV patients died (P=0.53). CONCLUSION: cAAVs are severe diseases, characterized by a higher relapse rate, more accrued damage and longer maintenance therapy than for aAAVs.