Tolerance and efficacy of targeted therapies prescribed for off-label indications in refractory systemic autoimmune diseases: data of the first 100 patients enrolled in the TATA registry (TArgeted Therapy in Autoimmune Diseases).

OBJECTIVES: To assess the tolerance and efficacy of targeted therapies prescribed off-label in refractory low-prevalence autoimmune and inflammatory systemic diseases. METHODS: The TATA registry (TArgeted Therapy in Autoimmune Diseases) is a prospective, observational, national and independent cohort follow-up. The inclusion criteria in the registry are as follows: age >18 years; low-prevalence autoimmune and inflammatory systemic disease treated with off-label drugs started after 1 January 2019. RESULTS: Hundred (100) patients (79 women) were enrolled. The median age was 52.5 years (95% CI 49 to 56) and the median disease duration before enrolment was 5 years (3 to 7). The targeted therapies at enrolment were as follows: Janus kinase/signal transducers and activators of transcription inhibitors (44%), anti-interleukin (IL)-6R (22%), anti-IL-12/23, anti-IL-23 and anti-IL-17 (9%), anti-B cell activating factor of the tumour necrosis factor family (5%), abatacept (5%), other targeted treatments (9%) and combination of targeted treatments (6%). 73% of patients were receiving corticosteroid therapy at enrolment (median dose 10 mg/day). The current median follow-up time is 9 months (8 to 10).Safety: 11 serious infections (incidence rate of 14.8/100 patient-years) and 1 cancer (1.3 cancers/100 patient-years) were observed. Two patients died from severe COVID-19 (2.7 deaths/100 patient-years).Efficacy: the targeted treatment was considered effective by the clinician in 56% of patients and allowed, in responders, a median reduction of oral corticosteroids of 15 (9 to 21) mg/day, below 7.5 mg/day in 76% of patients, while 28% discontinued. CONCLUSION: These initial results of the TATA registry confirm the diversity of targeted treatments prescribed off-label in refractory autoimmune diseases and their corticosteroid-sparing effect when effective. Tolerance was acceptable in these refractory patients with a long history of treatment with immunosuppressive drugs.

Current role for radioisotope synovectomy.

Radioisotope synovectomy has been extensively used to treat patients with chronic inflammatory joint disease but has moved to a less prominent position since the introduction of new and highly effective drugs. Remaining indications are refractory synovitis, pigmented villonodular synovitis as an adjunct to surgery, and hemophilic arthropathy. The three main radioisotopes used are yttrium-90, rhenium-186, and erbium-189. Radioisotope synovectomy should be performed only by highly experienced professionals, to minimize the risk of injection-related complications. The available safety data, in particular regarding the risk of malignancy, are reassuring. The efficacy of yttrium-90 in chronic inflammatory joint disease remains controversial.

Decreased patient exposure to ionizing radiation during interventional rheumatology procedures after optimization of protection.

OBJECTIVES: To decrease radiation exposure of patients undergoing interventional rheumatology procedures, without adversely affecting quality of care. METHODS: The radiation dose received, assessed by the dose-area product (DAP), was measured during 283 intraarticular injections performed under fluoroscopic guidance between May and July 2013. Then, three steps were taken to decrease patients' radiation exposure: a copper filter was added, the anti-scatter grid was removed, and exposure cell sensitivity was set at the highest value. DAP was measured during 158 intraarticular injections performed in 2014 with these measures in place. RESULTS: Mean DAP before optimization was 175µGray·m2 during facet joint injections (n=4) and 43µGray·m2 during hip injections but was less than 20µGray·m2 for injections into the shoulders (15.7µGray·m2), ankles (7.7µGray·m2), wrists (3.7µGray·m2), and fingers (3.3µGray·m2). After optimization, DAP decreased markedly for all injection sites, by 52% (shoulders) to 87% (facet joints, 22.7µGray·m2). Decreases occurred at all three steps of the procedure, i.e., patient installation, injection, and last image hold. Exposure during facet joint injections varied from 84 (54.5-108.5) µGray·m2 when body mass index (BMI) was <25kg/m2 to 228.9 (161.3-340.4)µGray·m2 when BMI was>30kg/m2. CONCLUSION: Simple technical changes translate into large decreases in patient radiation exposure during fluoroscopically-guided injections, particularly at the facet joints and in obese patients.

Sjögren's syndrome complicated by interstitial cystitis: A case series and literature review.

OBJECTIVES: To characterize the interstitial cystitis (IC) associated with Sjögren's syndrome (SS). METHODS: Report of three new cases. Only cases fulfilling the American-European consensus criteria for SS and the European Society for the Study of Interstitial Cystitis criteria with positive histological findings for IC were included. RESULTS: Thirteen cases of SS and IC have been reported in women, including the three reported here, with a mean age of 54 years. SS appeared first in 77% (n=10) of cases, a mean of 6.6 years before IC. The symptoms of IC included pollakiuria (n=11), lower abdominal pain (n=8), urinary urgency (n=5), painful micturition (n=6), hematuria (n=3) and dysuria (n=3). Urinary dilatation occurred in three cases, leading to acute renal failure in two patients. The diagnosis of IC was confirmed by anatomical evidence of cystitis inflammation on bladder biopsy in all (n=13) patients. Treatment was reported for nine patients, seven of whom (78%) received corticosteroid treatment, which was partially or completely effective in six cases. Immunosuppressive treatment was added in three cases (cyclosporine, n=2; azathioprine, n=1; cyclophosphamide, n=1). Local bladder treatments were performed, with hydraulic distension in five cases and DMSO instillation in one patient. A urinary catheter was inserted in the two cases of acute obstructive renal failure. CONCLUSIONS: Urinary symptoms without infection should lead the physician to consider a diagnosis of IC in SS patients. Urinary dilatation may occur, leading to acute obstructive renal failure. Corticosteroid treatment may be effective and local treatments have been tried.