Table 1.
Main characteristics of the studies on childhood-onset granulomatosis with polyangiitis and features of the patients at presentation
| First author | Stegmayr [20] | Cabral [6] | Rottem [24] | Akikusa [5] | Tahghighi [22] | Gajic-Veljic [23] | Iudici [11] | Sacri [12] | Arulkumaran [7] | Wong [19] | Orlowski [18] | Bohm [9] | Kosalka [10] |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Speciality | Paediatric Nephrology | Paediatric Rheumatology | Paediatrics | Paediatric Rheumatology | Paediatric Rheumatology | Paediatric Dermatology | Adult Internal Medicine | Paediatric Nephrology and Rheumatology | Adult Rheumatology | Paediatrics | Paediatrics | Paediatrics | Paediatrics |
| Country | Sweden, Germany | USA, Canada |
USA | Canada | Iran | Serbia | France | France | UK | UK | USA | International | Poland |
| Year of publication | 2000 | 2009 | 1993 | 2007 | 2013 | 2013 | 2015 | 2015 | 2011 | 1998 | 1978 | 2014 | 2014 |
| Years of enrolment | NA | since 2004 | NA | 1984–2005 | 2002–2011 | 1992–2011 | 1965–2014 | 1986–2011 | 1996–2010 | 1986–1991 | 1952–1976 | 2000–2010 | 1995–2013 |
| Study design | Retrospective | Cross-sectional 2004–2007: retrospectively collected March 2007-November 2008: prospectively collected |
Prospective | Retrospective | Cross-sectional Retrospective |
Retrospective | Retrospective | Retrospective | Retrospective | Retrospective | Retrospective | Retrospective | Retrospective |
| Number of patients | 7 | 65 | 23 | 25 | 11 | 3 | 25 | 28 | 7 | 12 | 6 | 56 | 9 |
| Ethnicity (n) | Caucasian | 41 Caucasian, 4 mixed, 3 East Indian, 3 African American, 2 Hispanic, 1 Asian, 1 Middle Eastern, 6 not available | NA | White 21/25 | NA | NA | 19 Caucasian | 21 Caucasian | 5 Caucasian, 2 Afro-Carribean | NA | 6 Caucasians | 46 Caucasian | NA |
| F (n) | 3 | 43 | 16 | 20 | 5 | 3 | 18 | 21 | 5 | 8 | 3 | 38 | 6 |
| Median age at onset (range), years | NA | NA | 15.4 (9.3–19.4) | NA | NA | 11 (6–16) | NA | NA | 11.5 (9–15) | 9 (0.5–14) | 17 (13–20) | 11.7 (1st and 3rd quartile 8.5–14.5) | 12 (88–16) |
| Median age at diagnosis (range), years | 14 (11–18) | 14.2 (4–17) | 14.5 (8.7–17.1) | 11 (6–15) | 14 (2–17) | 12.8 (10.1–14.6) | |||||||
| Classification/diagnostic tool used | Presence of kidney biopsy, upper and lower respiratory tract disease, ANCA+ | MD diagnosis | Clinical history compatible with GPA and histopathological evidence of vasculitis or granulomas, or both | ACR Criteria | EULAR/PRINTO/PRES | Clinical history compatible with GPA and histo-pathological evidence of vasculitis or granulomas, or both | EULAR/PRINTO/PRES and Revised Chapel-Hill | EULAR/PRINTO/PRES | EULAR/PRINTO/PRES | ACR Criteria | Histological examination in all patients | EULAR/PRINTO/PRES | EULAR/PRINTO/PRES or ACR criteria |
| cANCA positivity (ELISA), n (%) | 6/7 (85.7) | 43 (66.2) | NA | 13/15 (86.7) | 11 (100) | 2 (66.6) | 10/18 (55.5) | 18/28 (64.2) | NA | 4 (33.3) | NA | 34/51 (66.6) | NA |
| pANCA positivity (ELISA), n (%) | 1/7 (14.2) | 8 (12.3) | NA | NA | 2 (18) | 1 (33.3) | 4/18 (22.2) | 6/28 (21.4) | NA | 6 (50.0) | NA | 13/50 (26.0) | NA |
| cANCA positivity (IFI), n (%) | NA | 43 (66.2) | NA | 13/15 (86.7) | 11 (100) | 2 (66.6) | 12/20 (60.0) | NA | 4/7 (57.1) | 6 (50.0) | NA | NA | NA |
| pANCA positivity (IFI), n (%) | NA | 14 (21.5) | NA | 2/15 (13.3) | NA | 1 (33.3) | 4/20 (20.0) | NA | 1/7 (14.2) | 1 (14.2) | NA | NA | NA |
| Clinical manifestations | |||||||||||||
| Systemic, n (%) | 7 (100) | 58 (89.2) | 5 (71.4) | 24 (96.0) | 9 (81.8) | 3 (100) | 17 (68.0) | 23 (82.1) | 3 (42.8) | 0 | 5 (83.3) | 50 (89.2) | 8 (88.8) |
| Mucocutaneous, n (%) | 2 (28.5) | 23 (35.4) | 2 (8.6) | 8 (32.0) | 3 (27.2) | 3 (100) | 6 (24.0) | 15 (53.5) | 4 (57.1) | 10 (83.3) | 3 (50.0) | 36 (64.2) | 4 (44.4) |
| Musculoskeletal, n (%) | 4 (57.1) | 37 (56.9) | 7 (30.4) | 24 (96.0) | 3 (27.2) | 2 (66.6) | 9 (36.0) | 16 (57.1) | 5 (71.4) | 9 (75.0) | 2 (33.3) | 33 (58.9) | 3 (33.3) |
| Ocular, n (%) | 0 | 24 (36.9) | 3 (13.0) | 13 (52.0) | 2 (18.8) | 2 (66.6) | 7 (28.0) | 6 (21.4) | 3 (42.8) | 0 | 1 (16.6) | 19 (33.9) | 1 (11.1) |
| Ear, nose, and throat, n (%) | 7 (100) | 52 (80.0) | 20 (87) | 21 (84.0) | 8 (72.7) | 2 (33.3) | 21 (84.0) | 21 (75.0) | 4 (57.1) | 11 (91.6) | 6 (100) | 51 (91.0) | 5 (55.5) |
| Respiratory, n (%) | 6 (85.7) | 52 (80.0) | 5 (21.7) | 20 (80.0) | 2 (18.1) | 0 | 17 (68.0) | 19 (67.8) | 3 (42.8) | 7 (58.3) | 5 (83.3) | 44 (78.5) | 7 (77.7) |
| Cardiovascular, n (%) | 0 | 0 | 2 (8.7) | 5 (20.0) | 1 (9.0) | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Gastrointestinal, n (%) | 0 | 27 (41.5) | 0 | 3 (12.0) | 3 (27.2) | 0 | 5 (20.0) | 5 (17.8) | 1 (14.2) | 6 (50.0) | 0 | 9 (16.0) | 5 (55.5) |
| Neurological, n (%) | 0 | 16 (24.6) | 1 (4.3) | 2 (8.0) | 1 (9.0) | 0 | 1 (4.0) | 1 (3.5) | 1 (14.2) | 2 (16.6) | 1 (16.6) | 8 (14.2) | 1 (11.1) |
| Renal, n (%) | 7 (100) | 49 (75.4) | 2 (8.6) | 22 (88.0) | 4 (36.3) | 3 (100) | 9 (36.0) | 22 (78.5) | 4 (57.1) | 4 (33.3) | 5 (83.3) | 38 (67.8) | 8 (88.8) |
| Treatment | |||||||||||||
| Oral GCs ± IS, n (%) | 7 (100) | 60 (92.3) | 23 (100) | 25 (100) | NA | 3 (100) | 21 (84.0) | 28 (100) | NA | NA | 5 (83.3) | NA | 9 (100) |
| GCs ± CYC, n (%) | 6 (85.7) | 54 (83.0) | 18 (78.2) | 15 (60.0) | NA | 3 (100) | 18 (72.0) | NA | NA | NA | 3 (50.0) | NA | 9 (100) |
| GCs ± MTX, n (%) | 0 | 7 (10.7) | 1 (4.3) | 5 (20.0) | NA | 0 | 2 (8.0) | NA | NA | NA | 0 | NA | 0 |
| GCs ± AZA, n (%) | 0 | 0 | 2 (8.6) | 0 | NA | 0 | 2 (8.0) | NA | NA | NA | 0 | NA | 0 |
| Plasmapheresis, n (%) | 4 (57.1) | 9 (13.8) | 0 | 0 | NA | 1 (33.3) | 1 (4.0) | NA | NA | NA | 0 | NA | 0 |
NA not available, ELISA enzyme-linked immunosorbent assay, IFI indirect immunofluorence, GCs glucocorticoids, IS immunosoppressors, CYC cyclophosphamide, MTX methotrexate, AZA azathioprine, GPA granulomatosis with polyangiitis