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A93: Childhood Granulomatosis With Polyangiitis: Standardized Disease Assessment in 6 Czech Children
Author(s) -
Böhm Marek,
Nemcová Dana,
Dallos Tomáš,
Lád Václav,
Suláková Terezie,
Jancová Eva,
Dolezalová Pavla
Publication year - 2014
Publication title -
arthritis and rheumatology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 4.106
H-Index - 314
eISSN - 2326-5205
pISSN - 2326-5191
DOI - 10.1002/art.38510
Subject(s) - granulomatosis with polyangiitis , czech , medicine , pediatrics , disease , vasculitis , pathology , linguistics , philosophy
Background/Purpose: Granulomatosis with polyangiitis is a rare primary systemic vasculitis affecting small blood vessels with predilection for the respiratory tract and kidneys. We describe clinical outcome of a small patient cohort as captured by the recently developed tools the Paediatric Vasculitis Activity Score (PVAS) and the Paediatric Vasculitis Damage Index (PVDI). Methods: A retrospective and prospective analysis of data gathered from 2006 to 2013 on patients fulfilling the EULAR/PRINTO/PRES criteria for GPA. Disease activity and damage items have been systematically recorded, data from the time of diagnosis and from the last follow‐up (F/U) are presented. Results: Characteristics of the 6 girls identified are summarized in the . The median F/U was 4 years. Disease manifestations were recorded under PVAS and PVDI sections with 6/9 and 8/10 systems affected with active disease or damage, respectively. General, ENT and pulmonary manifestations were observed in 4/6 patients. Nephritis developed in 2/6 patients. Cumulative damage was noted in all cases with ENT region most commonly affected. Persistent remission was achieved in one case with limited disease, in the other one subglottic stenosis caused an episode of airway obstruction and cerebral ischemia leading to apalic syndrome and later to death. Clinical inactivity on treatment was achieved in 2 girls. Patient characteristicsPatient 1 2 3 4 5 6 Age at onset (years) 14 14 13 12 15 14 F/U (years) 5 2 5 5 3 0.2 ANCA Atypical IF c‐ANCA p‐ANCA Atypical IF c‐ANCA c‐ANCA Treatment CS, CP, MMF CS, MTX CS, MTX CS, MTX, CP, AZA CS, CP, MMF, RTX CS, CP, RTX PVAS category (section score) Onset F/U Onset F/U Onset F/U Onset F/U Onset F/U Onset F/U General 0 0 3 0 1 0 0 0 3 0 2 N/A Cutaneous 0 0 0 0 0 0 0 0 3 0 5 N/A Mucous memb./Eyes 0 0 0 0 6 0 2 0 0 0 0 N/A ENT 0 0 6 0 0 0 6 0 4 0 6 N/A Chest 3 0 0 0 0 0 6 0 6 0 6 N/A Renal 12 4 0 0 0 0 0 0 12 0 0 N/A PVAS total 15 4 9 0 7 0 14 0 28 0 19 N/A PVDI category (section score) Onset F/U Onset F/U Onset F/U Onset F/U Onset F/U Onset F/U Musculoskeletal 0 0 0 0 0 0 0 0 0 2 0 N/A Skin/Mucous memb. 0 0 0 0 0 0 0 1 0 0 1 N/A Ocular 0 0 0 0 0 1 0 1 0 0 0 N/A ENT 0 0 0 3 0 1 0 2 0 1 1 N/A Chest 0 0 0 0 0 0 0 3 0 1 1 N/A Renal 0 1 0 0 0 0 0 0 0 1 0 N/A Nervous system 0 0 0 0 0 0 0 2 0 0 0 N/A Other 0 1 0 1 0 0 0 3 0 0 1 N/A PVDI total 0 2 0 4 0 2 0 12 0 5 4 N/AF/U–follow‐up, N/A‐non–available, CS‐Corticosteroids, CP‐Cyclophosphamide, MTX‐Methotrexate, MMFMycophenolate mofetil, AZA‐Azathioprine, RTX‐RituximabConclusion: This small series of patients illustrates a variety of GPA outcomes. A standardized scoring of disease activity and damage enables systematic patient F/U and drives appropriate treatment decision‐making.