Non-severe eosinophilic granulomatosis with polyangiitis: long-term outcomes after remission-induction trial

Rheumatology ◽  
2019 ◽  
Vol 58 (12) ◽  
pp. 2107-2116 ◽  
Author(s):  
Xavier Puéchal ◽  
Christian Pagnoux ◽  
Gabriel Baron ◽  
François Lifermann ◽  
Loïk Geffray ◽  
...  
Keyword(s):  
Granulomatosis With Polyangiitis ◽  
Task Force ◽  
Controlled Trial ◽  
Remission Induction ◽  
Eosinophilic Granulomatosis With Polyangiitis ◽  
Long Term Outcomes ◽  
First Relapse ◽  
Eosinophilic Granulomatosis

Abstract Objective In a previous controlled trial, 1-year adjunction of AZA to glucocorticoids (GC) for patients with non-severe, newly diagnosed eosinophilic granulomatosis with polyangiitis (EGPA) failed to lower remission failure, vasculitis relapse and isolated asthma/rhinosinus exacerbation rates, or cumulative GC use at month (M) 24. The aim of this study was to analyse longer-term outcomes to determine whether subsequent vasculitis relapse or isolated asthma/rhinosinus exacerbation (IARE) rates differed. Methods After M24, patients were followed prospectively, being treated based on physicians’ best judgment. Flares and reasons for increased GC dose or immunosuppressant use were recorded, and reviewed according to randomization group to distinguish vasculitis relapses from IAREs according to EGPA Task Force recommendations. Results Fifty EGPA trial participants were followed for a median (interquartile range) of 6.3 (5.4–7.6) years; two (4%) died 11 months post-inclusion. By M24, vasculitis had relapsed in 21/49 (43%) patients and 14/50 (28%) had IAREs. Another patient died 4.8 years post-inclusion (infection). Among nine patients with subsequent vasculitis relapses, three had a major relapse and three had their first relapse after M24; among 25 patients with later IAREs, 17 occurred after M24. At 5 years, respective vasculitis relapse and IARE rates were 48% (95% CI 34.0, 62.6) and 56% (95% CI 41.7, 70.8), with no between-arm differences (P = 0.32 and 0.13). No entry clinical or biological parameter was associated with these outcomes during follow-up. Conclusion These results confirmed that 1-year AZA and GC induction obtained good overall survival but no long-term benefit for non-severe EGPA patients. Vasculitis relapses, occurring mostly during the first 2 years, and IAREs, occurring throughout follow-up, require other preventive treatments. Trial registration ClinicalTrials.gov, https://clinicaltrials.gov, NCT00647166.

scholarly journals A real-world assessment of mycophenolate mofetil for remission induction in eosinophilic granulomatosis with polyangiitis

2021 ◽  
Author(s):  
Mariana Philobos ◽  
Amy Perkins ◽  
Maira Karabayas ◽  
Paula Dospinescu ◽  
Nick Fluck ◽  
...  
Keyword(s):  
Mycophenolate Mofetil ◽  
Real World ◽  
Granulomatosis With Polyangiitis ◽  
Controlled Trial ◽  
Remission Induction ◽  
Eosinophilic Granulomatosis With Polyangiitis ◽  
Newly Diagnosed ◽  
Real World Data ◽  
Disease Remission ◽  
Eosinophilic Granulomatosis

AbstractEosinophilic granulomatosis with polyangiitis (EGPA) is a form of ANCA-associated vasculitis (AAV). Clinical trials demonstrating the efficacy of mycophenolate mofetil (MMF) for remission induction in AAV excluded patients with EGPA. Despite this, MMF is commonly used in these patients. The objective of this study was to evaluate, for the first time, the effectiveness and tolerance of MMF in EGPA remission induction. A retrospective, two-center, real-world study was conducted in patients with EGPA who received MMF in addition to prednisolone for newly diagnosed or relapsing disease between 2009 and 2019. Baseline, 3-, 6- and 12-month outcome data were extracted from electronic health records. The primary outcome was disease remission, defined as a Birmingham Vasculitis Activity Score of 0 at 6 months. Secondary outcomes included disease relapse, median prednisolone dose at 12 months and drug tolerance. In total, 15 patients (73% male, median age 57) with EGPA (11 newly diagnosed/4 relapsing) were identified. At 6 months, 67% had achieved disease remission. At 12 months, this was maintained (66.7%) and 4 patients had relapsed. All but one patient remained on MMF at study completion and all patients tolerated MMF. Our real-world data suggest that MMF is an effective and well-tolerated agent for achieving disease remission in EGPA. A future randomized controlled trial of MMF in this neglected orphan disease is now warranted.

scholarly journals POS0250 SIGNIFICANT DAMAGE OCCURS EARLY IN THE COURSE OF EOSINOPHILIC GRANULOMATOSIS WITH POLYANGIITIS AND IS MAINLY DUE TO DISEASE-RELATED SEQUELAE

2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 347.1-347
Author(s):  
P. Delvino ◽  
A. Milanesi ◽  
F. Brandolino ◽  
S. Monti ◽  
C. Montecucco
Keyword(s):  
Side Effects ◽  
Granulomatosis With Polyangiitis ◽  
Age At Diagnosis ◽  
Systemic Hypertension ◽  
Eosinophilic Granulomatosis With Polyangiitis ◽  
Short Term ◽  
Damage Accrual ◽  
Eosinophilic Granulomatosis

Background:Following the introduction of effective immunosuppressive treatments, ANCA-associated vasculitides (AAV) have become chronic diseases with a remitting-relapsing course. Therefore, preventing chronic damage accrual during follow-up is critical, as relapses, treatment-related side effects, and comorbidities may significantly affect the long-term outcomes of AAV patients. At present, no study specifically evaluated the burden of damage in patients with eosinophilic granulomatosis with polyangiitis (EGPA).Objectives:To describe short-term (6 months) and long-term (5 years) damage accrual in patients with newly diagnosed EGPA.Methods:Patients diagnosed with EGPA, according to ACR criteria and/or Chapel Hill definitions and regularly followed-up in our vasculitis center for ≥5 years were included. Damage accrual was assessed with the Vasculitis Damage Index (VDI). Short-term and long-term damage accrual was defined by VDI at 6 months and at 5 years, respectively, and categorized as related to vasculitis or its treatment.Results:VDI data at 6 months were available for 45 EGPA patients: 24 (53.3%) female, mean age at diagnosis 51.6±13.0 years. ANCA were positive in 17 patients (37.8%), with MPO being the only detected enzyme immunoassay (EIA)-specificity. At 6 months mean VDI was 2.8±1.3; 25/45 (55.6%) and 6/45 patients (13.3%) presented ≥3 and ≥5 items, respectively, whilst only 1 patient (2.2%) showed no items of damage. VDI data at 5 years were available for 32/45 EGPA patients (71.1%): 16 (50%) female, mean age at diagnosis 51.5±13.1 years. MPO-ANCA were positive in 13 patients (40.6%). At 5 years mean VDI was 3.5±1.3, with 26/32 (81.3%) and 7/32 patients (21.9%) presenting ≥3 and ≥5 items, respectively; notably, no patients presented a VDI=0 at 5 years.The most frequent disease-related VDI items at 6 months and at 5 years were asthma, chronic sinusitis, peripheral neuropathy, cardiomyopathy, pulmonary function tests abnormalities and nasal blockage (Figure 1). Osteoporotic fractures, diabetes and systemic hypertension were the most commonly reported treatment-related items at 6 months and at 5 years (Figure 1). Damage accrual progressively rose during the 5-year follow-up (P=0.023), mainly due to disease-related items rather than treatment-related items both at 6 months (disease related VDI 2.6±1.2, treatment-related VDI 0.3±0.6) and at 5 years (disease related VDI 2.9±1.2, treatment-related VDI 0.6±0,7). No significant difference in terms of damage accrual was observed between ANCA-positive and ANCA-negative patients (P >0.5).Conclusion:In our cohort of EGPA patients damage accrual occurs early, with more than half of the patients displaying ≥3 VDI items already at 6 months. Poor control of previous disease activity, particularly ENT and respiratory manifestations, contributes to progressive damage accrual more than treatment side effects.Figure 1.Disease-related and treatment-related VDI items at 6 months and at 5 years in patients with EGPA.Disclosure of Interests:None declared

scholarly journals Randomized Controlled Trial of the Clinical Recovery and Biodegradation of Polylactide-co-glycolide Implants Used in the Intramedullary Nailing of Children’s Forearm Shaft Fractures with at Least Four Years of Follow-Up

2021 ◽  
Vol 10 (5) ◽  
pp. 995
Author(s):  
Marja Perhomaa ◽  
Tytti Pokka ◽  
Linda Korhonen ◽  
Antti Kyrö ◽  
Jaakko Niinimäki ◽  
...  
Keyword(s):  
Intramedullary Nailing ◽  
Controlled Trial ◽  
Long Term Outcomes ◽  
Intramedullary Nails ◽  
Metal Implants ◽  
Shaft Fractures ◽  
Fractures In Children ◽  
Significant Difference

The preferred surgical fixation of forearm shaft fractures in children is Elastic Stable Intramedullary Nailing (ESIN). Due to known disadvantageous effects of metal implants, a new surgical method using biodegradable polylactide-co-glycolide (PLGA) intramedullary nails has been developed but its long-term outcomes are unclear. The aim of this study was to compare the long-term outcomes of Biodegradable Intramedullary Nailing (BIN) to ESIN and assess the biodegradation of the study implants via magnetic resonance imaging (MRI). The study population of the prospective, randomized trial consisted of paediatric patients whose forearm shaft fractures were treated with BIN (n = 19) or ESIN (n = 16). Forearm rotation at minimally four years’ follow-up was the main outcome. There was no clinically significant difference in the recovery of the patients treated with the BIN as compared to those treated with the ESIN. More than half of the implants (57.7%, n = 15/26) were completely degraded, and the rest were degraded almost completely. The PLGA intramedullary nails used in the treatment of forearm shaft fractures in this study resulted in good function and anatomy. No unexpected disadvantages were found in the degradation of the implants. However, two implant failures had occurred in three months postoperatively.

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