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Pediatric Rheumatology
Published in: Pediatric Rheumatology 1/2017

Open Access 01-12-2017 | Research article

Clinical practice variation and need for pediatric-specific treatment guidelines among rheumatologists caring for children with ANCA-associated vasculitis: an international clinician survey

Authors: Clara Westwell-Roper, Joanna M. Lubieniecka, Kelly L. Brown, Kimberly A. Morishita, Cherry Mammen, Linda Wagner-Weiner, Eric Yen, Suzanne C. Li, Kathleen M. O’Neil, Sivia K. Lapidus, Paul Brogan, Rolando Cimaz, David A. Cabral, for ARChiVe Investigators Network within the PedVas initiative

Published in: Pediatric Rheumatology | Issue 1/2017

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Abstract

Background

Because pediatric antineutrophil cytoplasmic antibody-associated vasculitis is rare, management generally relies on adult data. We assessed treatment practices, uptake of existing clinical assessment tools, and interest in pediatric treatment protocols among rheumatologists caring for children with granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA).

Methods

A needs-assessment survey developed by an international working group of pediatric rheumatologists and two nephrologists was circulated internationally. Data were summarized with descriptive statistics. Pearson’s chi-square tests were used in inferential univariate analyses.

Results

The 209 respondents from 36 countries had collectively seen ~1600 children with GPA/MPA; 144 had seen more than two in the preceding 5 years. Standardized and validated clinical assessment tools to score disease severity, activity, and damage were used by 59, 63, and 36%, respectively; barriers to use included lack of knowledge and limited perceived utility. Therapy varied significantly: use of rituximab rather than cyclophosphamide was more common among respondents from the USA (OR = 2.7 [1.3-5.5], p = 0.0190, n = 139), those with >5 years of independent practice experience (OR = 3.8 [1.3-12.5], p = 0.0279, n = 137), and those who had seen >10 children with GPA/MPA in their careers (OR = 4.39 [2.1-9.1], p = 0.0011, n = 133). Respondents who had treated >10 patients were also more likely to continue maintenance therapy for at least 24 months (OR = 3.0 [1.4-6.4], p = 0.0161, n = 127). Ninety six percent of respondents believed in a need for pediatric-specific treatment guidelines; 46% supported adaptation of adult guidelines while 69% favoured guidelines providing a limited range of treatment options to allow comparison of effectiveness through a registry.

Conclusions

These data provide a rationale for developing pediatric-specific consensus treatment guidelines for GPA/MPA. While pediatric rheumatologist uptake of existing clinical tools has been limited, guideline uptake may be enhanced if outcomes of consensus-derived treatment options are evaluated within the framework of an international registry.
Appendix
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Metadata
Title
Clinical practice variation and need for pediatric-specific treatment guidelines among rheumatologists caring for children with ANCA-associated vasculitis: an international clinician survey
Authors
Clara Westwell-Roper
Joanna M. Lubieniecka
Kelly L. Brown
Kimberly A. Morishita
Cherry Mammen
Linda Wagner-Weiner
Eric Yen
Suzanne C. Li
Kathleen M. O’Neil
Sivia K. Lapidus
Paul Brogan
Rolando Cimaz
David A. Cabral
for ARChiVe Investigators Network within the PedVas initiative
Publication date
01-12-2017
Publisher
BioMed Central
Published in
Pediatric Rheumatology / Issue 1/2017
Electronic ISSN: 1546-0096
DOI
https://doi.org/10.1186/s12969-017-0191-z

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