Comparability of patients with ANCA-associated vasculitis enrolled in clinical trials or in observational cohorts

C Pagnoux; S Carette; N A Khalidi; M Walsh; T F Hiemstra; D Cuthbertson; C Langford; G Hoffman; C L Koening; P A Monach; L Moreland; L Mouthon; P Seo; U Specks; S Ytterberg; K Westman; P Hoglund; L Harper; O Flossman; R Luqmani; Caroline Savage; N Rasmussen; K de Groot; V Tesar; D Jayne; P A Merkel; L Guillevin; French Vasculitis Study Group (FVSG); European Vasculitis Society (EUVAS); Vasculitis Clinical Research Consortium (VCRC)

Comparability of patients with ANCA-associated vasculitis enrolled in clinical trials or in observational cohorts

C Pagnoux, S Carette, N A Khalidi, M Walsh, T F Hiemstra, D Cuthbertson, C Langford, G Hoffman, C L Koening, P A Monach, L Moreland, L Mouthon, P Seo, U Specks, S Ytterberg, K Westman, P Hoglund, L Harper, O Flossman, R LuqmaniCaroline Savage, N Rasmussen, K de Groot, V Tesar, D Jayne, P A Merkel, L Guillevin, French Vasculitis Study Group (FVSG), European Vasculitis Society (EUVAS), Vasculitis Clinical Research Consortium (VCRC)

Research output: Contribution to journal › Article › peer-review

33 Citations (Scopus)

Abstract

OBJECTIVE: To analyse the differences between patients with granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA) entered into randomised clinical trials (RCTs) and those followed in large observational cohorts.

METHODS: The main characteristics and outcomes of patients with generalised and/or severe GPA or MPA with a five-factor score ≥ 1 enrolled in the French Vasculitis Study Group (FVSG) or the US-Canadian-based Vasculitis Clinical Research Consortium cohorts were compared to those enrolled in one of 2 FVSG clinical RCTs (WEG91, WEGENT) or 3 European Vasculitis Society clinical trials (CYCLOPS, CYCAZAREM, IMPROVE).

RESULTS: 657 patients (65.3% with GPA) in RCTs were compared to 437 in cohorts (90.6% with GPA). RCT patients were older at diagnosis than the cohort patients (56.6 ± 13.9 vs. 46.8 ± 17.3 years), had higher Birmingham vasculitis activity score (19.5 ± 9.1 vs. 16.9 ± 7.4), and more frequent kidney disease (84.0% vs. 54.9%) but fewer ear, nose, and throat symptoms (56.8% vs. 72.2%). At 56 months post-diagnosis, mortality and relapse rates, adjusted for age and renal function, were higher for patients with GPA in RCTs vs. cohorts (10.7% vs. 2.5% [p=0.001] and 22.5% vs. 15.6% [p=0.03], respectively) but similar for patients with MPA (6.2% vs. 6.6% [p=0.92] and 16.6% vs. 10.1% [p=0.39], respectively).

CONCLUSION: Patients with GPA or MPA in RCTs and those in observational cohorts show important differences that should be remembered when interpreting results based on these study populations.

Original language	English
Pages (from-to)	S77-S83
Number of pages	7
Journal	Clinical and Experimental Rheumatology
Volume	33
Issue number	2, Suppl. 89
Publication status	Published - 29 May 2015

Keywords

Adult
Age Distribution
Aged
Antibodies, Antineutrophil Cytoplasmic
Cohort Studies
Female
Granulomatosis with Polyangiitis (Wegener’s granulomatosis)
Humans
Kidney Diseases
Male
Microscopic Polyangiitis
Middle Aged
Myeloblastin
Observational Study as Topic
Otorhinolaryngologic Diseases
Patient Selection
Peroxidase
Randomized Controlled Trials as Topic
Severity of Illness Index
ANCA
vasculitis

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Cite this

Pagnoux, C., Carette, S., Khalidi, N. A., Walsh, M., Hiemstra, T. F., Cuthbertson, D., Langford, C., Hoffman, G., Koening, C. L., Monach, P. A., Moreland, L., Mouthon, L., Seo, P., Specks, U., Ytterberg, S., Westman, K., Hoglund, P., Harper, L., Flossman, O., ... Vasculitis Clinical Research Consortium (VCRC) (2015). Comparability of patients with ANCA-associated vasculitis enrolled in clinical trials or in observational cohorts. Clinical and Experimental Rheumatology, 33(2, Suppl. 89), S77-S83. https://www.clinexprheumatol.org/abstract.asp?a=9032

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abstract = "OBJECTIVE: To analyse the differences between patients with granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA) entered into randomised clinical trials (RCTs) and those followed in large observational cohorts.METHODS: The main characteristics and outcomes of patients with generalised and/or severe GPA or MPA with a five-factor score ≥ 1 enrolled in the French Vasculitis Study Group (FVSG) or the US-Canadian-based Vasculitis Clinical Research Consortium cohorts were compared to those enrolled in one of 2 FVSG clinical RCTs (WEG91, WEGENT) or 3 European Vasculitis Society clinical trials (CYCLOPS, CYCAZAREM, IMPROVE).RESULTS: 657 patients (65.3% with GPA) in RCTs were compared to 437 in cohorts (90.6% with GPA). RCT patients were older at diagnosis than the cohort patients (56.6 ± 13.9 vs. 46.8 ± 17.3 years), had higher Birmingham vasculitis activity score (19.5 ± 9.1 vs. 16.9 ± 7.4), and more frequent kidney disease (84.0% vs. 54.9%) but fewer ear, nose, and throat symptoms (56.8% vs. 72.2%). At 56 months post-diagnosis, mortality and relapse rates, adjusted for age and renal function, were higher for patients with GPA in RCTs vs. cohorts (10.7% vs. 2.5% [p=0.001] and 22.5% vs. 15.6% [p=0.03], respectively) but similar for patients with MPA (6.2% vs. 6.6% [p=0.92] and 16.6% vs. 10.1% [p=0.39], respectively).CONCLUSION: Patients with GPA or MPA in RCTs and those in observational cohorts show important differences that should be remembered when interpreting results based on these study populations.",

keywords = "Adult, Age Distribution, Aged, Antibodies, Antineutrophil Cytoplasmic, Cohort Studies, Female, Granulomatosis with Polyangiitis (Wegener{\textquoteright}s granulomatosis), Humans, Kidney Diseases, Male, Microscopic Polyangiitis, Middle Aged, Myeloblastin, Observational Study as Topic, Otorhinolaryngologic Diseases, Patient Selection, Peroxidase, Randomized Controlled Trials as Topic, Severity of Illness Index, ANCA, vasculitis",

author = "C Pagnoux and S Carette and Khalidi, {N A} and M Walsh and Hiemstra, {T F} and D Cuthbertson and C Langford and G Hoffman and Koening, {C L} and Monach, {P A} and L Moreland and L Mouthon and P Seo and U Specks and S Ytterberg and K Westman and P Hoglund and L Harper and O Flossman and R Luqmani and Caroline Savage and N Rasmussen and {de Groot}, K and V Tesar and D Jayne and Merkel, {P A} and L Guillevin and {French Vasculitis Study Group (FVSG)} and {European Vasculitis Society (EUVAS)} and {Vasculitis Clinical Research Consortium (VCRC)}",

year = "2015",

month = may,

day = "29",

language = "English",

volume = "33",

pages = "S77--S83",

journal = "Clinical and Experimental Rheumatology",

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Pagnoux, C, Carette, S, Khalidi, NA, Walsh, M, Hiemstra, TF, Cuthbertson, D, Langford, C, Hoffman, G, Koening, CL, Monach, PA, Moreland, L, Mouthon, L, Seo, P, Specks, U, Ytterberg, S, Westman, K, Hoglund, P, Harper, L, Flossman, O, Luqmani, R, Savage, C, Rasmussen, N, de Groot, K, Tesar, V, Jayne, D, Merkel, PA, Guillevin, L, French Vasculitis Study Group (FVSG), European Vasculitis Society (EUVAS) & Vasculitis Clinical Research Consortium (VCRC) 2015, 'Comparability of patients with ANCA-associated vasculitis enrolled in clinical trials or in observational cohorts', Clinical and Experimental Rheumatology, vol. 33, no. 2, Suppl. 89, pp. S77-S83. <https://www.clinexprheumatol.org/abstract.asp?a=9032>

TY - JOUR

T1 - Comparability of patients with ANCA-associated vasculitis enrolled in clinical trials or in observational cohorts

AU - Pagnoux, C

AU - Carette, S

AU - Khalidi, N A

AU - Walsh, M

AU - Hiemstra, T F

AU - Cuthbertson, D

AU - Langford, C

AU - Hoffman, G

AU - Koening, C L

AU - Monach, P A

AU - Moreland, L

AU - Mouthon, L

AU - Seo, P

AU - Specks, U

AU - Ytterberg, S

AU - Westman, K

AU - Hoglund, P

AU - Harper, L

AU - Flossman, O

AU - Luqmani, R

AU - Savage, Caroline

AU - Rasmussen, N

AU - de Groot, K

AU - Tesar, V

AU - Jayne, D

AU - Merkel, P A

AU - Guillevin, L

AU - French Vasculitis Study Group (FVSG)

AU - European Vasculitis Society (EUVAS)

AU - Vasculitis Clinical Research Consortium (VCRC)

PY - 2015/5/29

Y1 - 2015/5/29

N2 - OBJECTIVE: To analyse the differences between patients with granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA) entered into randomised clinical trials (RCTs) and those followed in large observational cohorts.METHODS: The main characteristics and outcomes of patients with generalised and/or severe GPA or MPA with a five-factor score ≥ 1 enrolled in the French Vasculitis Study Group (FVSG) or the US-Canadian-based Vasculitis Clinical Research Consortium cohorts were compared to those enrolled in one of 2 FVSG clinical RCTs (WEG91, WEGENT) or 3 European Vasculitis Society clinical trials (CYCLOPS, CYCAZAREM, IMPROVE).RESULTS: 657 patients (65.3% with GPA) in RCTs were compared to 437 in cohorts (90.6% with GPA). RCT patients were older at diagnosis than the cohort patients (56.6 ± 13.9 vs. 46.8 ± 17.3 years), had higher Birmingham vasculitis activity score (19.5 ± 9.1 vs. 16.9 ± 7.4), and more frequent kidney disease (84.0% vs. 54.9%) but fewer ear, nose, and throat symptoms (56.8% vs. 72.2%). At 56 months post-diagnosis, mortality and relapse rates, adjusted for age and renal function, were higher for patients with GPA in RCTs vs. cohorts (10.7% vs. 2.5% [p=0.001] and 22.5% vs. 15.6% [p=0.03], respectively) but similar for patients with MPA (6.2% vs. 6.6% [p=0.92] and 16.6% vs. 10.1% [p=0.39], respectively).CONCLUSION: Patients with GPA or MPA in RCTs and those in observational cohorts show important differences that should be remembered when interpreting results based on these study populations.

AB - OBJECTIVE: To analyse the differences between patients with granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA) entered into randomised clinical trials (RCTs) and those followed in large observational cohorts.METHODS: The main characteristics and outcomes of patients with generalised and/or severe GPA or MPA with a five-factor score ≥ 1 enrolled in the French Vasculitis Study Group (FVSG) or the US-Canadian-based Vasculitis Clinical Research Consortium cohorts were compared to those enrolled in one of 2 FVSG clinical RCTs (WEG91, WEGENT) or 3 European Vasculitis Society clinical trials (CYCLOPS, CYCAZAREM, IMPROVE).RESULTS: 657 patients (65.3% with GPA) in RCTs were compared to 437 in cohorts (90.6% with GPA). RCT patients were older at diagnosis than the cohort patients (56.6 ± 13.9 vs. 46.8 ± 17.3 years), had higher Birmingham vasculitis activity score (19.5 ± 9.1 vs. 16.9 ± 7.4), and more frequent kidney disease (84.0% vs. 54.9%) but fewer ear, nose, and throat symptoms (56.8% vs. 72.2%). At 56 months post-diagnosis, mortality and relapse rates, adjusted for age and renal function, were higher for patients with GPA in RCTs vs. cohorts (10.7% vs. 2.5% [p=0.001] and 22.5% vs. 15.6% [p=0.03], respectively) but similar for patients with MPA (6.2% vs. 6.6% [p=0.92] and 16.6% vs. 10.1% [p=0.39], respectively).CONCLUSION: Patients with GPA or MPA in RCTs and those in observational cohorts show important differences that should be remembered when interpreting results based on these study populations.

KW - Adult

KW - Age Distribution

KW - Aged

KW - Antibodies, Antineutrophil Cytoplasmic

KW - Cohort Studies

KW - Female

KW - Granulomatosis with Polyangiitis (Wegener’s granulomatosis)

KW - Humans

KW - Kidney Diseases

KW - Male

KW - Microscopic Polyangiitis

KW - Middle Aged

KW - Myeloblastin

KW - Observational Study as Topic

KW - Otorhinolaryngologic Diseases

KW - Patient Selection

KW - Peroxidase

KW - Randomized Controlled Trials as Topic

KW - Severity of Illness Index

KW - ANCA

KW - vasculitis

M3 - Article

C2 - 26016754

SN - 0392-856X

VL - 33

SP - S77-S83

JO - Clinical and Experimental Rheumatology

JF - Clinical and Experimental Rheumatology

IS - 2, Suppl. 89

ER -

Comparability of patients with ANCA-associated vasculitis enrolled in clinical trials or in observational cohorts

Abstract

Keywords

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