Comparing Presenting Clinical Features in 48 Children With Microscopic Polyangiitis to 183 Children Who Have Granulomatosis With Polyangiitis (Wegener's)

An ARChiVe Cohort Study

the ARChiVe Investigators Network within the PedVas Initiative, Dawn M. Wahezi

Research output: Contribution to journalArticle

25 Citations (Scopus)

Abstract

Objective: To uniquely classify children with microscopic polyangiitis (MPA), to describe their demographic characteristics, presenting clinical features, and initial treatments in comparison to patients with granulomatosis with polyangiitis (Wegener's) (GPA). Methods: The European Medicines Agency (EMA) classification algorithm was applied by computation to categorical data from patients recruited to the ARChiVe (A Registry for Childhood Vasculitis: e-entry) cohort, with the data censored to November 2015. The EMA algorithm was used to uniquely distinguish children with MPA from children with GPA, whose diagnoses had been classified according to both adult- and pediatric-specific criteria. Descriptive statistics were used for comparisons. Results: In total, 231 of 440 patients (64% female) fulfilled the classification criteria for either MPA (n = 48) or GPA (n = 183). The median time to diagnosis was 1.6 months in the MPA group and 2.1 months in the GPA group (ranging to 39 and 73 months, respectively). Patients with MPA were significantly younger than those with GPA (median age 11 years versus 14 years). Constitutional features were equally common between the groups. In patients with MPA compared to those with GPA, pulmonary manifestations were less frequent (44% versus 74%) and less severe (primarily, hemorrhage, requirement for supplemental oxygen, and pulmonary failure). Renal pathologic features were frequently found in both groups (75% of patients with MPA versus 83% of patients with GPA) but tended toward greater severity in those with MPA (primarily, nephrotic-range proteinuria, requirement for dialysis, and end-stage renal disease). Airway/eye involvement was absent among patients with MPA, because these GPA-defining features preclude a diagnosis of MPA within the EMA algorithm. Similar proportions of patients with MPA and those with GPA received combination therapy with corticosteroids plus cyclophosphamide (69% and 78%, respectively) or both drugs in combination with plasmapheresis (19% and 22%, respectively). Other treatments administered, ranging in decreasing frequency from 13% to 3%, were rituximab, methotrexate, azathioprine, and mycophenolate mofetil. Conclusion: Younger age at disease onset and, perhaps, both gastrointestinal manifestations and more severe kidney disease seem to characterize the clinical profile in children with MPA compared to those with GPA. Delay in diagnosis suggests that recognition of these systemic vasculitides is suboptimal. Compared with adults, initial treatment regimens in children were comparable, but the complete reversal of female-to-male disease prevalence ratios is a provocative finding.

Original languageEnglish (US)
Pages (from-to)2514-2526
Number of pages13
JournalArthritis and Rheumatology
Volume68
Issue number10
DOIs
StatePublished - Oct 1 2016

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Microscopic Polyangiitis
Granulomatosis with Polyangiitis
Vasculitis
Registries
Cohort Studies
Mycophenolic Acid
Systemic Vasculitis
Lung
Plasmapheresis
Azathioprine
Kidney Diseases
Drug Combinations
Therapeutics
Proteinuria
Age of Onset
Methotrexate
Cyclophosphamide
Chronic Kidney Failure

ASJC Scopus subject areas

  • Immunology and Allergy
  • Rheumatology
  • Immunology

Cite this

Comparing Presenting Clinical Features in 48 Children With Microscopic Polyangiitis to 183 Children Who Have Granulomatosis With Polyangiitis (Wegener's) : An ARChiVe Cohort Study. / the ARChiVe Investigators Network within the PedVas Initiative; Wahezi, Dawn M.

In: Arthritis and Rheumatology, Vol. 68, No. 10, 01.10.2016, p. 2514-2526.

Research output: Contribution to journalArticle

@article{7ee2ce3ad4ca4ee1bf20541e3efaca34,
title = "Comparing Presenting Clinical Features in 48 Children With Microscopic Polyangiitis to 183 Children Who Have Granulomatosis With Polyangiitis (Wegener's): An ARChiVe Cohort Study",
abstract = "Objective: To uniquely classify children with microscopic polyangiitis (MPA), to describe their demographic characteristics, presenting clinical features, and initial treatments in comparison to patients with granulomatosis with polyangiitis (Wegener's) (GPA). Methods: The European Medicines Agency (EMA) classification algorithm was applied by computation to categorical data from patients recruited to the ARChiVe (A Registry for Childhood Vasculitis: e-entry) cohort, with the data censored to November 2015. The EMA algorithm was used to uniquely distinguish children with MPA from children with GPA, whose diagnoses had been classified according to both adult- and pediatric-specific criteria. Descriptive statistics were used for comparisons. Results: In total, 231 of 440 patients (64{\%} female) fulfilled the classification criteria for either MPA (n = 48) or GPA (n = 183). The median time to diagnosis was 1.6 months in the MPA group and 2.1 months in the GPA group (ranging to 39 and 73 months, respectively). Patients with MPA were significantly younger than those with GPA (median age 11 years versus 14 years). Constitutional features were equally common between the groups. In patients with MPA compared to those with GPA, pulmonary manifestations were less frequent (44{\%} versus 74{\%}) and less severe (primarily, hemorrhage, requirement for supplemental oxygen, and pulmonary failure). Renal pathologic features were frequently found in both groups (75{\%} of patients with MPA versus 83{\%} of patients with GPA) but tended toward greater severity in those with MPA (primarily, nephrotic-range proteinuria, requirement for dialysis, and end-stage renal disease). Airway/eye involvement was absent among patients with MPA, because these GPA-defining features preclude a diagnosis of MPA within the EMA algorithm. Similar proportions of patients with MPA and those with GPA received combination therapy with corticosteroids plus cyclophosphamide (69{\%} and 78{\%}, respectively) or both drugs in combination with plasmapheresis (19{\%} and 22{\%}, respectively). Other treatments administered, ranging in decreasing frequency from 13{\%} to 3{\%}, were rituximab, methotrexate, azathioprine, and mycophenolate mofetil. Conclusion: Younger age at disease onset and, perhaps, both gastrointestinal manifestations and more severe kidney disease seem to characterize the clinical profile in children with MPA compared to those with GPA. Delay in diagnosis suggests that recognition of these systemic vasculitides is suboptimal. Compared with adults, initial treatment regimens in children were comparable, but the complete reversal of female-to-male disease prevalence ratios is a provocative finding.",
author = "{the ARChiVe Investigators Network within the PedVas Initiative} and Cabral, {David A.} and Canter, {Debra L.} and Eyal Muscal and Wahezi, {Dawn M.} and Wahezi, {Dawn M.} and Spalding, {Steven J.} and Marinka Twilt and Benseler, {Susanne M.} and Sarah Campillo and Sirirat Charuvanij and Paul Dancey and Eberhard, {Barbara A.} and Elder, {Melissa E.} and Aimee Hersh and Higgins, {Gloria C.} and Huber, {Adam M.} and Raju Khubchandani and Susan Kim and Marisa Klein-Gitelman and Kostik, {Mikhail M.} and Lawson, {Erica F.} and Tzielan Lee and Lubieniecka, {Joanna M.} and Deborah McCurdy and Moorthy, {Lakshmi N.} and Morishita, {Kimberly A.} and Nielsen, {Susan M.} and O'Neil, {Kathleen M.} and Andreas Reiff and Goran Ristic and Robinson, {Angela B.} and Angelyne Sarmiento and Susan Shenoi and Toth, {Mary B.} and {Van Mater}, {Heather A.} and Linda Wagner-Weiner and Weiss, {Jennifer E.} and White, {Andrew J.} and Yeung, {Rae S M} and Cabral, {David A.} and Angelyne Sarmiento and Qun Yang and Victor Espinosa and Joanna Lubieniecki and Jaime Guzman and Kristin Houghton and Kimberly Morishita and Ross Petty and Lori Tucker and Ilowite, {Norman Todd}",
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month = "10",
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language = "English (US)",
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TY - JOUR

T1 - Comparing Presenting Clinical Features in 48 Children With Microscopic Polyangiitis to 183 Children Who Have Granulomatosis With Polyangiitis (Wegener's)

T2 - An ARChiVe Cohort Study

AU - the ARChiVe Investigators Network within the PedVas Initiative

AU - Cabral, David A.

AU - Canter, Debra L.

AU - Muscal, Eyal

AU - Wahezi, Dawn M.

AU - Wahezi, Dawn M.

AU - Spalding, Steven J.

AU - Twilt, Marinka

AU - Benseler, Susanne M.

AU - Campillo, Sarah

AU - Charuvanij, Sirirat

AU - Dancey, Paul

AU - Eberhard, Barbara A.

AU - Elder, Melissa E.

AU - Hersh, Aimee

AU - Higgins, Gloria C.

AU - Huber, Adam M.

AU - Khubchandani, Raju

AU - Kim, Susan

AU - Klein-Gitelman, Marisa

AU - Kostik, Mikhail M.

AU - Lawson, Erica F.

AU - Lee, Tzielan

AU - Lubieniecka, Joanna M.

AU - McCurdy, Deborah

AU - Moorthy, Lakshmi N.

AU - Morishita, Kimberly A.

AU - Nielsen, Susan M.

AU - O'Neil, Kathleen M.

AU - Reiff, Andreas

AU - Ristic, Goran

AU - Robinson, Angela B.

AU - Sarmiento, Angelyne

AU - Shenoi, Susan

AU - Toth, Mary B.

AU - Van Mater, Heather A.

AU - Wagner-Weiner, Linda

AU - Weiss, Jennifer E.

AU - White, Andrew J.

AU - Yeung, Rae S M

AU - Cabral, David A.

AU - Sarmiento, Angelyne

AU - Yang, Qun

AU - Espinosa, Victor

AU - Lubieniecki, Joanna

AU - Guzman, Jaime

AU - Houghton, Kristin

AU - Morishita, Kimberly

AU - Petty, Ross

AU - Tucker, Lori

AU - Ilowite, Norman Todd

PY - 2016/10/1

Y1 - 2016/10/1

N2 - Objective: To uniquely classify children with microscopic polyangiitis (MPA), to describe their demographic characteristics, presenting clinical features, and initial treatments in comparison to patients with granulomatosis with polyangiitis (Wegener's) (GPA). Methods: The European Medicines Agency (EMA) classification algorithm was applied by computation to categorical data from patients recruited to the ARChiVe (A Registry for Childhood Vasculitis: e-entry) cohort, with the data censored to November 2015. The EMA algorithm was used to uniquely distinguish children with MPA from children with GPA, whose diagnoses had been classified according to both adult- and pediatric-specific criteria. Descriptive statistics were used for comparisons. Results: In total, 231 of 440 patients (64% female) fulfilled the classification criteria for either MPA (n = 48) or GPA (n = 183). The median time to diagnosis was 1.6 months in the MPA group and 2.1 months in the GPA group (ranging to 39 and 73 months, respectively). Patients with MPA were significantly younger than those with GPA (median age 11 years versus 14 years). Constitutional features were equally common between the groups. In patients with MPA compared to those with GPA, pulmonary manifestations were less frequent (44% versus 74%) and less severe (primarily, hemorrhage, requirement for supplemental oxygen, and pulmonary failure). Renal pathologic features were frequently found in both groups (75% of patients with MPA versus 83% of patients with GPA) but tended toward greater severity in those with MPA (primarily, nephrotic-range proteinuria, requirement for dialysis, and end-stage renal disease). Airway/eye involvement was absent among patients with MPA, because these GPA-defining features preclude a diagnosis of MPA within the EMA algorithm. Similar proportions of patients with MPA and those with GPA received combination therapy with corticosteroids plus cyclophosphamide (69% and 78%, respectively) or both drugs in combination with plasmapheresis (19% and 22%, respectively). Other treatments administered, ranging in decreasing frequency from 13% to 3%, were rituximab, methotrexate, azathioprine, and mycophenolate mofetil. Conclusion: Younger age at disease onset and, perhaps, both gastrointestinal manifestations and more severe kidney disease seem to characterize the clinical profile in children with MPA compared to those with GPA. Delay in diagnosis suggests that recognition of these systemic vasculitides is suboptimal. Compared with adults, initial treatment regimens in children were comparable, but the complete reversal of female-to-male disease prevalence ratios is a provocative finding.

AB - Objective: To uniquely classify children with microscopic polyangiitis (MPA), to describe their demographic characteristics, presenting clinical features, and initial treatments in comparison to patients with granulomatosis with polyangiitis (Wegener's) (GPA). Methods: The European Medicines Agency (EMA) classification algorithm was applied by computation to categorical data from patients recruited to the ARChiVe (A Registry for Childhood Vasculitis: e-entry) cohort, with the data censored to November 2015. The EMA algorithm was used to uniquely distinguish children with MPA from children with GPA, whose diagnoses had been classified according to both adult- and pediatric-specific criteria. Descriptive statistics were used for comparisons. Results: In total, 231 of 440 patients (64% female) fulfilled the classification criteria for either MPA (n = 48) or GPA (n = 183). The median time to diagnosis was 1.6 months in the MPA group and 2.1 months in the GPA group (ranging to 39 and 73 months, respectively). Patients with MPA were significantly younger than those with GPA (median age 11 years versus 14 years). Constitutional features were equally common between the groups. In patients with MPA compared to those with GPA, pulmonary manifestations were less frequent (44% versus 74%) and less severe (primarily, hemorrhage, requirement for supplemental oxygen, and pulmonary failure). Renal pathologic features were frequently found in both groups (75% of patients with MPA versus 83% of patients with GPA) but tended toward greater severity in those with MPA (primarily, nephrotic-range proteinuria, requirement for dialysis, and end-stage renal disease). Airway/eye involvement was absent among patients with MPA, because these GPA-defining features preclude a diagnosis of MPA within the EMA algorithm. Similar proportions of patients with MPA and those with GPA received combination therapy with corticosteroids plus cyclophosphamide (69% and 78%, respectively) or both drugs in combination with plasmapheresis (19% and 22%, respectively). Other treatments administered, ranging in decreasing frequency from 13% to 3%, were rituximab, methotrexate, azathioprine, and mycophenolate mofetil. Conclusion: Younger age at disease onset and, perhaps, both gastrointestinal manifestations and more severe kidney disease seem to characterize the clinical profile in children with MPA compared to those with GPA. Delay in diagnosis suggests that recognition of these systemic vasculitides is suboptimal. Compared with adults, initial treatment regimens in children were comparable, but the complete reversal of female-to-male disease prevalence ratios is a provocative finding.

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