Long- term outcome of paediatric patients with ANCA vasculitis
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Author(s)
Type
Journal Article
Abstract
Background: Primary systemic vasculitis presenting in childhood is an uncommon but serious condition. As these
patients transfer to adult clinics for continuing care, defining long term outcomes with emphasis on disease and
treatment- related morbidity and mortality is important. The aim of this study is to describe the long- term clinical
course of paediatric patients with ANCA vasculitis.
Methods: The adult patients in our vasculitis clinics who had presented in childhood, with a follow up time of
greater than 10 years were included. We also reviewed the literature for articles describing the clinical outcome of
paediatric patients with ANCA vasculitis.
Results: We describe the clinical course of 8 adults who presented in childhood with ANCA vasculitis. 7 patients
had Wegener’s granulomatosis and 1 had microscopic polyangiitis. The median age at presentation was 11.5 years,
and follow up time ranged form 11 to 30 years. Induction therapy for all patients was steroids and/or
cyclophosphamide. Maintenance therapy was with azathioprine or mycophenolate mofetil. Biological agents were
used in 3 patients for relapsed disease in adulthood only.
Seven patients achieved complete remission. All patients experienced disease relapse, with a median of 4 episodes.
Kidney function was generally well preserved, with median eGFR 76 ml/min. Only one patient developed endstage
renal failure and one patient died after 25 years of disease. Treatment-related morbidity rates were high; 7
suffered from infections, 4 were infertile, 2 had skeletal complications, and 1 developed malignancy.
Conclusion: Close long- term follow up of paediatric patients with ANCA vasculitis is imperative, as this patient
cohort is likely to live long enough to develop significant treatment and disease- related morbidities. Prospective
cohort studies with novel therapies including paediatric patients are crucial to help us determine the best
approach to managing this complex group of patients. In addition, although not yet observed in our series, late
cardiovascular morbidity remains a major longer-term potential concern for adult survivors of paediatric vasculitis.
patients transfer to adult clinics for continuing care, defining long term outcomes with emphasis on disease and
treatment- related morbidity and mortality is important. The aim of this study is to describe the long- term clinical
course of paediatric patients with ANCA vasculitis.
Methods: The adult patients in our vasculitis clinics who had presented in childhood, with a follow up time of
greater than 10 years were included. We also reviewed the literature for articles describing the clinical outcome of
paediatric patients with ANCA vasculitis.
Results: We describe the clinical course of 8 adults who presented in childhood with ANCA vasculitis. 7 patients
had Wegener’s granulomatosis and 1 had microscopic polyangiitis. The median age at presentation was 11.5 years,
and follow up time ranged form 11 to 30 years. Induction therapy for all patients was steroids and/or
cyclophosphamide. Maintenance therapy was with azathioprine or mycophenolate mofetil. Biological agents were
used in 3 patients for relapsed disease in adulthood only.
Seven patients achieved complete remission. All patients experienced disease relapse, with a median of 4 episodes.
Kidney function was generally well preserved, with median eGFR 76 ml/min. Only one patient developed endstage
renal failure and one patient died after 25 years of disease. Treatment-related morbidity rates were high; 7
suffered from infections, 4 were infertile, 2 had skeletal complications, and 1 developed malignancy.
Conclusion: Close long- term follow up of paediatric patients with ANCA vasculitis is imperative, as this patient
cohort is likely to live long enough to develop significant treatment and disease- related morbidities. Prospective
cohort studies with novel therapies including paediatric patients are crucial to help us determine the best
approach to managing this complex group of patients. In addition, although not yet observed in our series, late
cardiovascular morbidity remains a major longer-term potential concern for adult survivors of paediatric vasculitis.
Date Issued
2011-06-19
Date Acceptance
2011-06-19
Citation
Pediatric Rheumatology, 2011, 9
ISSN
1546-0096
Publisher
BioMed Central
Journal / Book Title
Pediatric Rheumatology
Volume
9
Copyright Statement
© 2011 Arulkumaran et al; licensee BioMed Central Ltd. This is an Open Access article distributed under the terms of the Creative
Commons Attribution License (http://creativecommons.org/licenses/by/2.0), which permits unrestricted use, distribution, and
reproduction in any medium, provided the original work is properly cited.
Commons Attribution License (http://creativecommons.org/licenses/by/2.0), which permits unrestricted use, distribution, and
reproduction in any medium, provided the original work is properly cited.
License URL
Subjects
Science & Technology
Life Sciences & Biomedicine
Pediatrics
Rheumatology
PEDIATRICS
RHEUMATOLOGY
WEGENER GRANULOMATOSIS
KAWASAKI-DISEASE
CHILDREN
POLYARTERITIS
YOUNG
Publication Status
Published
Article Number
ARTN 12