Changes in Treatments and Outcomes After Implementation of a National Universal Access Program for Juvenile Idiopathic Arthritis

2021 ◽  
pp. jrheum.210011
Author(s):  
Sara Concha ◽  
Pamela S. Morales ◽  
Eduardo Talesnik ◽  
Arturo Borzutzky
Keyword(s):  
Juvenile Idiopathic Arthritis ◽  
Universal Access ◽  
Diagnostic Delay ◽  
Diagnosis And Treatment ◽  
Pediatric Rheumatologist ◽  
Biologic Drugs ◽  
Biologic Treatment ◽  
Clinical Records ◽  
Access Program

Objective To evaluate the clinical and demographic characteristics of patients with juvenile idiopathic arthritis (JIA) in Chile and compare treatments and outcomes before and after the introduction in 2010 of the Explicit Health Guarantees (GES) for JIA, a national universal access program for diagnosis and treatment of this condition. Methods The clinical records of 280 patients with JIA followed at a private tertiary academic health network between 2007 and 2018 were reviewed. Results Sixty-nine percent of JIA patients were female, mean age at diagnosis was 8.5±4.8 years and mean follow-up was 4±3.7 years. After GES implementation (post-GES), time to evaluation by pediatric rheumatologist and diagnostic delay were significantly reduced (15±4.5 vs. 9±4.2 months, p=0.004). In addition, use of magnetic resonance imaging significantly increased post-GES (p<0.001). In terms of JIA treatments, before GES implementation, no patients received biologics. Of the 67 patients diagnosed before 2010 with continued follow-up at our center, 34% began biologic treatment after GES implementation. Of 196 patients diagnosed post-GES, 46% were treated with biologics. JIA remission rates were significantly higher in patients diagnosed post-GES compared to pre- GES (43% vs. 29% p=0.02). Post-GES, we observed a significant decrease in uveitis complications among JIA patients (45% vs. 13%, p=0.037). Conclusion The implementation of a national legally-mandated universal access program for guaranteed JIA diagnosis and treatment led to earlier access to a pediatric rheumatologist and JIA diagnosis, increased rates of treatment with biologic drugs, higher rates of clinical remission, and lower rates of uveitis complications in Chilean children with JIA.

scholarly journals Addressing Healthcare Quality in Juvenile Idiopathic Arthritis With a Universal Access Program

2021 ◽  
pp. jrheum.210658
Author(s):  
Roberta Berard ◽  
Michelle Batthish
Keyword(s):  
Primary Care ◽  
Juvenile Idiopathic Arthritis ◽  
Universal Access ◽  
Healthcare Quality ◽  
Diagnosis And Treatment ◽  
Access Program

In this issue of The Journal of Rheumatology, Concha, et al1 describe the effects of the implementation, in 2010, of a national, legally mandated universal access program (Explicit Health Guarantees [GES]) for guaranteed juvenile idiopathic arthritis ( JIA) diagnosis and treatment in Chile. The GES program guarantees that evaluation by a specialist takes place less than 30 days after referral from primary care, and that treatment must start no later than 7 days after confirmation of diagnosis.

scholarly journals P357 Long-term outcomes of biologic therapy in Crohn’s disease complicated with internal fistulizing disease: BIOSCOPE study from GETECCU

2021 ◽  
Vol 15 (Supplement_1) ◽  
pp. S377-S378
Author(s):  
I Rodríguez-Lago ◽  
A Fernández-Clotet ◽  
F Mesonero ◽  
F J García-Alonso ◽  
M J Casanova ◽  
...  
Keyword(s):  
Crohn’S Disease ◽  
Crohn's Disease ◽  
Biologic Agent ◽  
Fistula Closure ◽  
Biologic Drugs ◽  
Biologic Treatment ◽  
Cross Sectional ◽  
Urinary Fistulas ◽  
Cross Sectional Imaging

Abstract Background The prevalence of penetrating disease in Crohn’s disease (CD) increases progressively over time, and evidence on the medical treatment of this complication is limited. The aim of this study was to evaluate the efficacy of biologic agents in CD complicated with internal fistulising disease. Methods A retrospective analysis of all adult patients from the ENEIDA registry (&gt;68,000 patients) with CD who received at least one biologic agent -anti-TNF, ustekinumab or vedolizumab- for penetrating disease was performed. Exclusion criteria comprised treatment for perianal disease, enterocutaneous, anastomotic or periostomal fistula tracts. The main outcomes were fistula-related surgery and fistula closure on cross-sectional imaging. Preestablished secondary outcomes included the rate of abdominal abscess, the need for percutaneous drainage, the changes in the number of fistula tracts, fistula closure rates, and the safety profile.The baseline characteristics were analyzed by means of descriptive statistics and were compared by non-parametric tests. Predictive factors associated with surgery and fistula closure were evaluated by a multivariable logistic regression and survival analyses. Results A total of 710 patients (median age 38 years [IQR, 28-48], 59% male, 55% L3) receiving 791 biologic treatments were included at 53 sites (701 anti-TNF, 71 ustekinumab, and 19 vedolizumab). Patients had a median of 1 (range 1-5) fistula tracts, 49% of them entero-enteric followed by entero-colic (28%). After a median follow-up of 59 months (IQR, 27-105), 244 patients (31%) required surgery due to internal fistulising complications after 8.5 months (IQR, 3-24). Patients with ileocolonic disease (OR 1.99 [1.22-3.23]), entero-urinary fistulas (OR 2.35 [1.17-4.73]), or with a stricture distal to the fistula (OR 2.1 [1.31-3.36]) showed a higher risk of surgery, with no differences between biologic drugs (Figure 1). Combination therapy reduced the risk of surgery (HR 0.58 [0.37-0.90]). Fistula closure was observed in 24% of patients after a median of 15 months (IQR, 7.5-27). Patients with a lower number of fistula tracts showed a higher probability of closure (OR 1.72 [1.09-2.7]). Conclusion A high proportion of patients with internal fistulizing CD benefit from biologic treatment after a median of 5 years. During this follow-up, around two thirds of patients are free of surgery and one in every four patients achieves fistula closure.

scholarly journals POS1317 PREDICTIVE FACTORS FOR RESPONSE TO TREATMENT IN A LONG-TERM COHORT OF PATIENTS WITH JUVENILE IDIOPATHIC ARTHRITIS-ASSOCIATED UVEITIS

2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 941.1-941
Author(s):  
L. Marelli ◽  
F. Minoia ◽  
G. Beretta ◽  
C. Mapelli ◽  
G. Leone ◽  
...  
Keyword(s):  
Juvenile Idiopathic Arthritis ◽  
Clinical Features ◽  
Predictive Factors ◽  
Disease Duration ◽  
Systemic Treatment ◽  
Response To Treatment ◽  
Conventional Dmards ◽  
Clinical Records

Background:Uveitis is the main extraarticular complication of juvenile idiopathic arthritis (JIA) with still a significant impact on JIA morbidity, despite continuous improvement in systemic treatment. Although antinuclear antibody positivity and early onset of JIA have been associated with a high risk of uveitis onset, so far no clinical features have been widely recognized as predictive factors for JIA-associated uveitis (JIA-U) response to treatment.Objectives:To investigate clinical features associated with response to systemic treatment in a long-term cohort of patients with JIA-UMethods:Clinical records of patients with JIA-U were retrospectively reviewed with regard to clinical features, therapeutic choices and outcome. Clinical and laboratory variables were compared by means of Mann-Whitney U test or chi-square/Fisher exact test, as appropriate.Results:Data from 164 JIA-U patients were analysed (81.7% female), with a median follow up of 12.1 years (7.1-17.3). Median age at JIA and uveitis onset was 2.6 (1.6-4.8) and 4.8 (2.9 – 7.0) years, respectively. Monotherapy with a conventional disease-modifying antirheumatic drug (DMARD) was used in 25.0% of patients, while 111 patients (67.7%) received at least one biologic DMARD (bDMARDs). Compared to patients responsive to DMARDs, children requiring a bDMARDs for uveitis had a lower median age at both JIA (2.4 vs 4.3 years, p 0.0234) and uveitis onset (4.1 vs 6.2 years, p 0.0023). Despite no differences in ocular damage at onset and median disease duration, patients not responsive to conventional DMARDs showed a higher frequency of ocular damage at the last visit (66.2% vs 33.3%, p 0.011). Children requiring more than one bDMARD for uveitis presented a more frequent polyarticular course (87.0% vs 20.2%, p 0.0022), a longer disease duration (median follow-up: 14.2 vs 10.4 years, p 0.0397) and a higher frequency of visual loss (best corrected visual acuity < 4/10: 23.3% vs 6.3%, p 0.0069).Conclusion:JIA-U patients with a lack of response to conventional DMARDs were significantly younger both at JIA and uveitis onset. Severe JIA-U requiring more than one bDMARDs was associated with polyarticular JIA course and longer disease duration. Children resistant to conventional treatment need prompt recognition and additional strategies to improve long-term outcome.References:[1]Heiligenhaus et al. Predictive factors and biomarkers for the 2-year outcome of uveitis in juvenile idiopathic arthritis. Rheumatology 2019.Disclosure of Interests:None declared

scholarly journals P731 Biologics use in elderly patients with IBD: Experience in a UK secondary care setting

2020 ◽  
Vol 14 (Supplement_1) ◽  
pp. S588-S588
Author(s):  
C Alexakis ◽  
J Robinson ◽  
R Yeates ◽  
N Ho-Yen ◽  
S Mathew ◽  
...  
Keyword(s):  
Biologic Therapy ◽  
Treatment Time ◽  
District General Hospital ◽  
Ageing Population ◽  
Biologic Treatment ◽  
Start Date ◽  
Morbidity Index ◽  
Clinical Records ◽  
Infective Complications

Abstract Background The combination of an ageing population coupled with a rising incidence in inflammatory bowel disease (IBD) will undoubtedly result in increased use of biologic agents in older patients with IBD, yet long-term data on the use of these drugs in this population remains scarce. We describe our experience of biologic treatment in IBD patients aged 60 years or greater at a UK district general hospital. Methods This was a retrospective descriptive study performed at Royal Surrey NHS Foundation Trust which serves a population of 300,000 people. IBD patients on biologic therapy are all registered on a prospectively collated database. Patients were included in this study if their biologic start date was on or after their 60th birthday. Each patient had their clinical records assessed and the following baseline data were recorded: sex, IBD subtype, concurrent medication use, relevant medical history and co-morbid status (using the Charleston Morbidity Index (CMI)), age at biologic start date and biologic subtype. Patients were followed up from biologic start date to biologic end date, death or end of study period (01.07.2019). Kaplan Meier analysis was used to generate biologic survival or ‘persistence’. During follow-up, we recorded the incidence of both infective and non-infective complications, and the incidents of both IBD surgery and malignancy. Results Of 220 patients on biologic therapy, 19 (8.7%) were aged ≥60 years at initiation, of whom 18 had data included in the analysis including 431 patient-months of follow-up. 78% were male. Half of patients had Crohn’s disease. Median CMI was 4. Only 1 patient had a history of malignancy prior to biologic initiation. Mean age at biologic initiation was 69 years. 72% of patients were on concurrent immune suppression medication. Most patients (72%) were commenced on Infliximab (Adalimumab 17%, Vedolizumab 11%). Median biologic treatment time was 17 months. The 6, 12 and 24 month biologic persistence rates were 78%, 67% and 28% respectively. 22% of patients were subsequently treated with a second biologic. 17% required surgery despite biologic use. Over half (56%) of patients suffered a complication post first biologic initiation, the most frequent being infection which affected 44% of patients during follow-up. Where recorded or available, 41% of patients developed antibodies to biologic, with a mean time of 11.4 months treatment. There were no recorded malignancies in any patient following biologic initiation. Conclusion Just under one in 10 of our biologic cohort is above the age of 60 on commencement of therapy. Despite a heavy burden of infective complications, there is reasonable biologic persistence in this group, and importantly a negligible malignancy risk following treatment initiation.

scholarly journals POS1315 USEFULNESS OF SYNOVIAL BIOPSY IN THE DIFFERENTIAL DIAGNOSIS AND AS POSSIBLE PREDICTOR OF RESPONSE TO TREATMENT IN JUVENILE IDIOPATHIC ARTHRITIS

2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 940.1-940
Author(s):  
S. Costi ◽  
F. Pregnolato ◽  
A. Parafioriti ◽  
E. Armiraglio ◽  
T. Giani ◽  
...  
Keyword(s):  
Differential Diagnosis ◽  
Juvenile Idiopathic Arthritis ◽  
Inflammatory Infiltrate ◽  
Clinical Status ◽  
Disease Onset ◽  
Synovial Biopsy ◽  
Response To Treatment ◽  
Biologic Drugs ◽  
Number Of Layers

Background:While synovial biopsy is an invasive procedure and is not required for the diagnosis of juvenile idiopathic arthritis (JIA), it may be useful in doubtful cases.Objectives:Aims of the study were i.to verify the role of synovial biopsy in the differential diagnosis of JIA and ii. to review the pathology slides in order to evaluate possible associations of a histologic pattern with response to treatment.Methods:We reviewed data from medical records of patients under the age of 18 years who underwent a synovial biopsy requested by our Pediatric Rheumatology Unit over the last 10 years. We collected information on demographic, clinical, laboratory, radiological, histopathological characteristics, as well as treatment response (in particular, remission at the last visit and number of examination, number of biologic drugs used). Among variables in the histologic score, number of layers in the synovial lining and inflammatory infiltrate (0-5) were compared to clinical status at last visit. Potential differences in variables between responders and non responders were assessed by unpaired t-test or non-parametric Mann-Whitney test, as appropriate.Results:We identified 64 patients (40F, 24M) with a median age at disease onset of 9 years (range 1-15) and a median follow-up time of 161 months (range 8-1160). We recognized two groups of interest: patients with a known JIA diagnosis (28/64) and patients with unknown diagnosis (36/64) at the moment of synovial biopsy. In the group with known JIA, most underwent the procedure during orthopedic surgery, and in all cases the histology was consistent with JIA. Among the unknown diagnosis group, in 19 cases results were consistent with a chronic synovitis, while among the other 17 histology could lead to a diagnosis of other conditions in 6 cases (foreign body and villonodular synovitis n=2 each, sarcoidosis and osteochondromatosis n=1 each). In the remaining 11 the final diagnoses were varied (mostly genetic forms eg skeletal dysplasia, CACP, Thiemann disease).Between the two groups we identified 46 patients with a definite JIA diagnosis. At the last follow-up visit 29 of them were in clinical remission, albeit on medication. The remaining 17 had a severe course of disease, with persistent activity and use of at least two biologic drugs. In 26 cases we could evaluate the correlation between status at last visit and number of layers/inflammatory infiltrate, but no statistical significant correlation was found.Conclusion:Despite its limited use nowadays, synovial biopsy may still be a useful tool in patients whose diagnosis is unclear. In our study, while it confirmed the suspicion in most cases, in other instances it allowed the diagnosis of rare conditions that would have been otherwise missed. No association between disease course and histological features in a small JIA cohort was found. We are currently expanding the study with a larger series.Disclosure of Interests:None declared

scholarly journals Importance of pediatric rheumatologists and transitional care for juvenile idiopathic arthritis-associated uveitis: A retrospective series of 9 cases.

2020 ◽  
Author(s):  
Susumu Yamazaki ◽  
Asami Shimbo ◽  
Yuko Akutsu ◽  
Horoshi Takase ◽  
Tomohiro Morio ◽  
...  
Keyword(s):  
Juvenile Idiopathic Arthritis ◽  
Transitional Care ◽  
Transition Period ◽  
Good Prognosis ◽  
Ophthalmic Surgery ◽  
Biologic Drugs ◽  
Speckled Pattern ◽  
Appropriate Treatment

Abstract Background: Juvenile idiopathic arthritis-associated uveitis (JIA-U) is a serious condition associated with the risk of blindness. However, pediatric rheumatologists rarely encounter cases of blindness, because most patients reach adulthood during the course of follow-up before blindness occurs. Here, we report the progress of 9 patients with JIA-U, including 2 patients who became blind after the transition period. We aimed to highlight the importance of the role of pediatric rheumatologists and transitional care in preventing blindness associated with JIA-U. Case presentation: We conducted a retrospective analysis of the case records of 9 JIA-U patients (1 male, 8 female; median age 16.8 years, range 5.5–19.8 years). All patients presented with oligo-juvenile idiopathic arthritis (oligo-JIA) (one presented with extended oligo-JIA); the median age of uveitis onset was 5.0 years (range 3.0–13.0 years), and the onset of uveitis preceded the onset of arthritis in 2 patients. The median disease duration was 12.5 years (range 3.5–24.7 years); 4 patients had anti-nuclear antibody (ANA) positivity (≧1:160) (all with a homogeneous and speckled-pattern subtype). All patients were negative for rheumatoid factor. Eight patients received methotrexate, 7 patients received one or more biologic drugs (etanercept, infliximab, adalimumab, and golimumab), and 6 patients required ophthalmic surgery at an early age (≦ 18 years). Two patients developed blindness after the transition period. Medical examination by pediatric rheumatologists and use of biologics had been delayed in both patients. One patient developed depression after transition and interrupted her own treatment. Conclusions: The reason for blindness in the 2 patients was thought to be the delay in the commencement of treatment and failure to provide transitional care. Inflammation is difficult to control in JIA-U even with appropriate treatment. Pediatric rheumatologists must be informed about the risk of JIA-U blindness, especially after transition. To ensure a good prognosis, the specialized treatment with the involvement of pediatric rheumatologists is necessary early on, and consideration for transitional medicine is essential. Therefore, this report reaffirms the importance of planned transitional care that has been advocated for globally.

scholarly journals Camptodactyly-Arthropathy-Coxa Vara-Pericarditis Syndrome Resembling Juvenile Idiopathic Arthritis: A Single-Center Experience from Southern Turkey

2021 ◽  
pp. 1-6
Author(s):  
Rabia Miray Kisla Ekinci ◽  
Sibel Balci ◽  
Haldun Dogan ◽  
Serdar Ceylaner ◽  
Celal Varan ◽  
...  
Keyword(s):  
Juvenile Idiopathic Arthritis ◽  
Pericardial Effusion ◽  
Early Onset ◽  
Joint Space ◽  
Coxa Vara ◽  
Single Center ◽  
The Third ◽  
Single Center Experience ◽  
Southern Turkey

Camptodactyly-arthropathy-coxa vara-pericarditis (CACP) syndrome, caused by biallelic pathogenic mutations in the <i>PRG4</i> gene, is characterized by early-onset camptodactyly, noninflammatory arthropathy, coxa vara deformity, and rarely, pericardial effusion. Herein, we report 3 patients with CACP syndrome from 2 unrelated families. All patients are female, born to consanguineous parents, and had camptodactyly since the first years of their lives. Two patients had a prior diagnosis of juvenile idiopathic arthritis. Hip changes were present in 2 patients, and 2 of 3 patients had undergone surgery for camptodactyly. Routine echocardiographic evaluations were normal during the 2-year follow-up. This paper represents the third study including CACP patients from Turkey. Clinically, all 3 patients resembled juvenile idiopathic arthritis cases and received unnecessary medication. There is also an ongoing need for improving awareness of CACP and an effective treatment focusing on the lubrication of the joint space in CACP patients.

scholarly journals Excision of Greater Superficial Petrosal Nerve Schwannoma Via a Pure Endoscopic Endonasal Approach

2021 ◽  
pp. 014556132110263
Author(s):  
Zhenlin Wang ◽  
Siyuan Zhang ◽  
Yan Qi ◽  
Lianjie Cao ◽  
Pu Li ◽  
...  
Keyword(s):  
Hearing Loss ◽  
Clinical Features ◽  
Middle Cranial Fossa ◽  
Nerve Sheath Tumor ◽  
Endoscopic Endonasal Approach ◽  
Endonasal Approach ◽  
Endoscopic Endonasal ◽  
Greater Superficial Petrosal Nerve ◽  
Clinical Records

Greater superficial petrosal nerve (GSPN) schwannomas are an exceedingly rare nerve sheath tumor. The current literature search was conducted using Medline and Embase database by key search terms. Only 31 cases have been reported in the literature so far. Facial palsy, hearing loss, and xerophthalmia accounted for 48.4% (15), 41.9% (13), and 29% (9) of all cases, respectively. The middle cranial fossa approach was used in all previous reports. A retrospective review of 2 GSPN schwannomas patients treated by endoscopic endonasal approach (EEA) in our center was collected. Clinical records, including clinical features, pre- and postoperative images, surgery, and follow-up information, were reviewed. In all cases, clinical features including facial numbness and headache were found, with tinnitus in case 1, hearing loss, xerophthalmia in case 2. Imaging studies showed a solid mass that originated in the anterior of the petrous bone. Two patients were treated by EEA. Furthermore, no recurrence was found during the follow-up period (15-29 months) in both of the 2 cases after the operation. Complete resection of GSPN schwannomas can be achieved via the pure EEA. Endoscopic endonasal approach for radical removal of tumors is safe and feasible.

scholarly journals Aromatic L-amino Acid Decarboxylase (AADC) deficiency: results from an Italian modified Delphi consensus

2021 ◽  
Vol 47 (1) ◽  
Author(s):  
Carlo Fusco ◽  
◽  
Vincenzo Leuzzi ◽  
Pasquale Striano ◽  
Roberta Battini ◽  
...  
Keyword(s):  
Amino Acid ◽  
Diagnostic Delay ◽  
Steering Committee ◽  
Outcome Data ◽  
Diagnosis And Treatment ◽  
Acid Decarboxylase ◽  
Delphi Consensus ◽  
Amino Acid Decarboxylase ◽  
Modified Delphi ◽  
Number Of Patients

Abstract Background Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare and underdiagnosed neurometabolic disorder resulting in a complex neurological and non-neurological phenotype, posing diagnostic challenges resulting in diagnostic delay. Due to the low number of patients, gathering high-quality scientific evidence on diagnosis and treatment is difficult. Additionally, based on the estimated prevalence, the number of undiagnosed patients is likely to be high. Methods Italian experts in AADC deficiency formed a steering committee to engage clinicians in a modified Delphi consensus to promote discussion, and support research, dissemination and awareness on this disorder. Five experts in the field elaborated six main topics, each subdivided into 4 statements and invited 13 clinicians to give their anonymous feedback. Results 100% of the statements were answered and a consensus was reached at the first round. This enabled the steering committee to acknowledge high rates of agreement between experts on clinical presentation, phenotypes, diagnostic work-up and treatment strategies. A research gap was identified in the lack of standardized cognitive and motor outcome data. The need for setting up an Italian working group and a patients’ association, together with the dissemination of knowledge inside and outside scientific societies in multiple medical disciplines were recognized as critical lines of intervention. Conclusions The panel expressed consensus with high rates of agreement on a series of statements paving the way to disseminate clear messages concerning disease presentation, diagnosis and treatment and strategic interventions to disseminate knowledge at different levels. Future lines of research were also identified.

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