David A Cabral1, Debra L Canter2, Eyal Muscal2, Kabita Nanda3, Dawn M Wahezi4, Steven J Spalding5, Marinka Twilt6, Susanne M Benseler6, Sarah Campillo7, Sirirat Charuvanij8, Paul Dancey9, Barbara A Eberhard10, Melissa E Elder11, Aimee Hersh12, Gloria C Higgins13, Adam M Huber14, Raju Khubchandani15, Susan Kim16, Marisa Klein-Gitelman17, Mikhail M Kostik18, Erica F Lawson19, Tzielan Lee20, Joanna M Lubieniecka21, Deborah McCurdy22, Lakshmi N Moorthy23, Kimberly A Morishita24, Susan M Nielsen25, Kathleen M O'Neil26, Andreas Reiff27, Goran Ristic28, Angela B Robinson29, Angelyne Sarmiento24, Susan Shenoi3, Mary B Toth30, Heather A Van Mater31, Linda Wagner-Weiner32, Jennifer E Weiss33, Andrew J White34, Rae S M Yeung35. 1. British Columbia Children's Hospital, Vancouver, British Columbia, Canada. dcabral@cw.bc.ca. 2. Texas Children's Hospital, Houston. 3. Seattle Children's Hospital, Seattle, Washington. 4. Children's Hospital at Montefiore, Bronx, New York. 5. Cleveland Clinic, Cleveland, Ohio. 6. Alberta Children's Hospital and University of Calgary, Calgary, Alberta, Canada. 7. Montreal Children's Hospital and McGill University Health Centre, Montreal, Quebec, Canada. 8. Siriraj Hospital and Mahidol University, Bangkok, Thailand. 9. Janeway Children's Health and Rehabilitation Centre, St. John's, Newfoundland, Canada. 10. Cohen Children's Medical Center of New York, New Hyde Park. 11. University of Florida, Gainesville. 12. University of Utah, Salt Lake City. 13. Nationwide Children's Hospital, Columbus, Ohio. 14. IWK Health Centre and Dalhousie University, Halifax, Nova Scotia. 15. Breach Candy Hospital, Mumbai, India. 16. Children's Hospital of Boston, Boston, Massachusetts. 17. Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois. 18. St. Petersburg State Pediatric Medical University, St. Petersburg, Russia. 19. University of California, San Francisco. 20. Stanford Children's Health and Stanford University School of Medicine, Stanford, California. 21. Simon Fraser University, Burnaby, British Columbia, Canada. 22. University of California, Los Angeles. 23. Rutgers Robert Wood Johnson Medical School, New Brunswick, New Jersey. 24. British Columbia Children's Hospital, Vancouver, British Columbia, Canada. 25. Rigshospitalet, Copenhagen, Denmark. 26. Riley Children's Hospital, Indianapolis, Indiana. 27. Children's Hospital Los Angeles, Los Angeles, California. 28. Mother and Child Health Care Institute of Serbia, Belgrade, Serbia. 29. University Hospitals Case Medical Center and Rainbow Babies and Children's Hospital, Cleveland, Ohio. 30. Akron Children's Hospital, Akron, Ohio. 31. Duke Children's Hospital and Health Center and Duke University Medical Center, Durham, North Carolina. 32. University of Chicago Medicine Comer Children's Hospital, Chicago, Illinois. 33. Joseph M. Sanzari Children's Hospital and Hackensack University Medical Center, Hackensack, New Jersey. 34. St. Louis Children's Hospital and Washington University School of Medicine, St. Louis, Missouri. 35. Hospital for Sick Children, Toronto, Ontario, Canada.
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.
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.
Authors: Sirada Panupattanapong; Dustin L Stwalley; Andrew J White; Margaret A Olsen; Anthony R French; Mary E Hartman Journal: Arthritis Rheumatol Date: 2018-12 Impact factor: 10.995
Authors: Paul E Moore; Jason T Poston; Debra Boyer; Emily Barsky; Jonathan Gaffin; Kathleen B Boyne; Kristie R Ross; Laura Beth Mann Dosier; Timothy J Vece; Alicia M Casey; Sebastian K Welsh; J Wells Logan; Edward G Shepherd; Pelton A Phinzy; Howard B Panitch; Christina M Papantonakis; Eric D Austin; Amir B Orandi; Maleewan Kitcharoensakkul; Mark K Abe; Amjad Horani; Jordan S Rettig; Jessica Pittman Journal: Ann Am Thorac Soc Date: 2017-08