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Objectives: To evaluate disease activity status using the Acromegaly Disease Activity Tool (ACRODAT ® ) in a cohort of Spanish acromegaly patients, to assess the relationship between the level of disease activity according to both ACRODAT ® and the physicians' clinical evaluation, and to study the potential discrepancies in the perception of symptoms between physicians and patients., Design: Multicenter, observational, descriptive and cross-sectional study., Methods: Disease activity was assessed in adult patients with acromegaly under pharmacological treatment during at least 6 months using ACRODAT ® ., Results: According to ACRODAT ® , 48.2%, 31.8% and 20.0% of a total of 111 patients were classified as having a stable disease (S), mild disease activity (M-DA) and significant disease activity (S-DA) respectively. ACRODAT ® classification of disease activity significantly correlated with physicians' opinion, with a moderate inter-rater agreement and a specificity of 92.45% (PPV = 86.21%). No correlation was found between IGF-I levels and severity of symptoms or quality of life (QoL). A decision to take clinical action was significantly more frequent in S-DA and M-DA patients than S patients but no action was taken on 5 (22.7%) and 27 (77.1%) S-DA and M-DA patients, respectively CONCLUSIONS: ACRODAT ® detected disease activity in 51.8% of patients. Interestingly, although M-DA and S-DA patients were likely to be in the process of being controlled, action was not always taken on these patients. ACRODAT ® is a validated and highly specific tool that may be useful to routinely monitor acromegaly and to identify patients with non-obvious disease activity by incorporating "patient-centred" parameters like symptoms and QoL to the clinical evaluation of acromegaly., (© 2021. The Author(s).)

Objectives To study the efficacy and influence on metabolism of recombinant human growth hormone (rhGH) treatment in short children born small for gestational age (SGA). Methods Retrospective, observational, multicenter study in 305 short children born SGA, treated with rhGH during a mean ± SD of 5.03 ± 1.73 years at a mean ± SD dose of 37 ± 8 μg/kg/day. Auxological and metabolic assessment including glucose and lipids profile were collected. Results Mean ± SD age at the start of treatment was 7.11 ± 2.78 years. Height and weight improved significantly until the end of treatment from mean -2.72 (CI95%: -2.81 to -2.63) standard deviation score (SDS) to -1.16 (CI95%: -1.44 to -0.88) SDS and from -1.62 (CI95%: -1.69 to -1.55) SDS to -0.94 (CI95%: -1.14 to -0.74) SDS respectively. Mean height gain was 1.27 (CI95%: 0.99-1.54) SDS. Prepubertal patients showed higher height gain than pubertal children (mean [CI95%] = 1.44 [CI95%: 1.14-1.74] vs. 0.73 [CI95%: 0.22-1.24], p=0.02). Height gain SDS during treatment negatively correlated with chronological age (CA) and bone age (BA) delay and positively correlated with duration of treatment, height gain during first year of treatment, years on prepubertal treatment and height SDS from target height (TH). Glucose, insulin, and triglycerides increased significantly but remained within the normal range. Total and LDL-cholesterol decreased significantly, and HDL-cholesterol remained unchanged. Conclusions rhGH treatment in short SGA children effectively normalized height in most of the patients and showed a safe metabolic profile. Children who benefit the most are those with greater height SDS distance from TH, BA delay, longer duration of treatment and prepubertal treatment initiation.

Purpose: The burden of chronic daily subcutaneous administration of pegvisomant on adherence has not been previously studied. This study was aimed to determine the adherence to pegvisomant treatment in acromegaly patients in the real-world clinical practice setting in Spain., Methods: Multicenter, observational, descriptive, cross-sectional study in patients with acromegaly treated with pegvisomant for at least 12 months. Patient adherence was indirectly determined by Batalla and Haynes-Sackett questionnaires and directly by prescription record review. Additionally, treatment satisfaction was assessed by the Treatment Satisfaction with Medicines Questionnaire (SATMED-Q) and treatment convenience by an ad-hoc Pegvisomant questionnaire. Errors in reconstitution and administration process were determined by direct observation., Results: 108 patients were included in the analysis. Rates of adherence varied from 60.7 to 92.1% and did not correlate with disease control. Older patient age and alternative schedules other than daily pegvisomant dosing were associated with lower adherence. Treatment satisfaction and convenience was high, with a mean (SD) total SATMED-Q score of 74.6 ± 15.4 over 100 and a total ad-hoc Pegvisomant questionnaire score of 71.2 ± 15.2 over 100. 34.3% of patients made mistakes during the reconstitution /administration process., Conclusions: Patient adherence to pegvisomant was high (60.7-92.1%), but more than a third of the patients in the study made mistakes during the administration process, with a potential impact on disease control. Besides dosing compliance, correct administration of medication should be carefully assessed in these patients.

Background: Several potential predictive markers of efficacy of targeted agents in patients with metastatic renal cell carcinoma (mRCC) have been identified. Interindividual heterogeneity warrants further investigation., Patients and Methods: Multicenter, observational, retrospective study in patients with clear-cell mRCC treated with sunitinib. Patients were classified in two groups: long-term responders (LR) (progression-free survival (PFS)≥22 months and at least stable disease), and primary refractory (PR) (progressive disease within 3-months of sunitinib onset). Objectives were to compare baseline clinical factors in both populations and to correlate tumor expression of selected signaling pathways components with sunitinib PFS., Results: 123 patients were analyzed (97 LR, 26 PR). In the LR cohort, overall response rate was 79% and median duration of best response was 30 months. Median PFS and overall survival were 43.2 (95% confidence intervals[CI]:37.2-49.3) and 63.5 months (95%CI:55.1-71.9), respectively. At baseline PR patients had a significantly lower proportion of nephrectomies, higher lactate dehydrogenase and platelets levels, lower hemoglobin, shorter time to and higher presence of metastases, and increased Fuhrman grade. Higher levels of HEYL, HEY and HES1 were observed in LR, although only HEYL discriminated populations significantly (AUC[ROC]=0.704; cut-off=34.85). Increased levels of hsa-miR-27b, hsa-miR-23b and hsa-miR-628-5p were also associated with prolonged survival. No statistical significant associations between hsa-miR-23b or hsa-miR-27b and the expression of c-Met were found., Conclusions: Certain mRCC patients treated with sunitinib achieve extremely long-term responses. Favorable baseline hematology values and longer time to metastasis may predict longer PFS. HEYL, hsa-miR-27b, hsa-miR-23b and hsa-miR-628-5p could be potentially used as biomarkers of sunitinib response.

Background: Diagnosing and initiating treatment of psoriatric arthritis (PsA) as early as possible is essential to prevent irreversible joint destruction and poor clinical outcomes. Dermatologists are uniquely placed to identify early symptoms of PsA in psoriasis patients but levels of under- and late-diagnosis remain high., Objective: To evaluate the prevalence and clinical features of PsA in Spanish psoriatic patients attended by dermatologists and then referred to rheumatologic units for PsA diagnosis confirmation., Methods: a multicenter, non-interventional, cross-sectional trial conducted at 40 hospitals in Spain. Patients were initially screened for PsA by a dermatologist based on clinical evaluation and results from the Psoriatic Arthritis Screening and Evaluation (PASE) Questionnaire. All patients were then evaluated by a blinded rheumatologist for the presence of PsA using Moll and Wright criteria and Classification Criteria for Psoriatic Arthritis (CASPAR)., Results: Of 375 psoriatic patients enrolled at dermatology units, 28.6% patients scored ≥44 in PASE, whereas 32.3% patients screened positive for suspicion of PsA (clinical evaluation and/or PASE). Correlation of suspicion of PsA by dermatologists and PASE score was 0.368 (Pearson correlation coefficient). Following rheumatologic assessment, prevalence of PsA was 22.9% (86/375 patients) according to Moll and Wright and CASPAR criteria. The correlation of diagnosis of PsA between dermatologists and rheumatologists was 0.410 (Kappa Index)., Conclusions: Prevalence of PsA in our study was within the range reported in other studies. Our analyses found only a moderate correlation in the diagnosis of PsA between dermatologists and rheumatologists. The screening questionnaire, PASE, showed a moderate predictive value for the diagnosis of PsA.

Introduction and Objectives: Patients with peripheral artery disease have a high risk of cardiovascular events and death. The rate of prescription of evidence-based cardiovascular therapies and the attainment of therapeutic goals in this population is suboptimal. There are no previous studies evaluating the rate of prescription of these therapies in our country., Methods: PERIFERICA is a cross-sectional study conducted from May to December 2009 in 440 outpatient clinics of general practitioners, internal medicine, cardiology, vascular surgery, endocrinology, and nephrology specialists throughout Spain. Subjects were included if they were aged ≥45 years and had peripheral artery disease and a blood sample obtained during the previous 6 months. Patients were excluded if they had coronary or cerebrovascular diseases. Clinical and anthropometric variables and blood analysis were obtained in all participants., Results: In total, 4087 patients were included in the study (mean age, 68 years; 74% men). There was a high prevalence of diabetes (50%) and hypertension (90%); 79% of participants received lipid-lowering drugs (76% statins), 85.5% antihypertensive drugs (66% renin-angiotensin blockers) and 83% antithrombotics (75% antiplatelet drugs and 11% anticoagulants). In addition, 30% of subjects had a low-density lipoprotein cholesterol concentration n<100 mg/dL, 29.5% had optimal control of blood pressure, and 74.5% did not smoke. Only 8% had a good control of all of their cardiovascular risk factors., Conclusions: Although a high percentage of subjects with peripheral artery disease receives adequate treatment with evidence-based preventive therapies, the percentage of subjects with good control of all their risk factors is low., (Copyright © 2011 Sociedad Española de Cardiología. Published by Elsevier Espana. All rights reserved.)

Background. Renal transplantation is the best therapy for patients with hepatitis C virus (HCV) infection with end-stage renal disease. Patient and graft survival are lower in the long term compared with HCV-negative patients. The current study evaluated the results of renal transplantation in Spain in a long period (1990-2002), focusing on graft failure.Methods. Data on the Spanish Chronic Allograft Nephropathy Study Group including 4304 renal transplant recipients, 587 of them with HCV antibody, were used to estimate graft and patient survival at 4 years with multivariate Cox models.Results. Among recipients alive with graft function 1 year post-transplant, the 4-year graft survival was 92.8% in the whole group; this was significantly better in HCV-negative vs HCV-positive patients (94.4% vs 89.5%, P < 0.005). Notably, HCV patients showed more acute rejection, a higher degree of proteinuria accompanied by a diminution of renal function, more graft biopsies and lesions of de novo glomerulonephritis and transplant glomerulopathy. Serum creatinine and proteinuria at 1 year, acute rejection, HCV positivity and systolic blood pressure were independent risk factors for graft loss. Patient survival was 96.3% in the whole group, showing a significant difference between HCV-negative vs HCV-positive patients (96.6% vs 94.5%, P < 0.05). Serum creatinine and diastolic blood pressure at 1 year, HCV positivity and recipient age were independent risk factors for patient death.Conclusions. Renal transplantation is an effective therapy for HCV-positive patients with good survival but inferior than results obtained in HCV-negative patients in the short term. Notably, HCV-associated renal damage appears early with proteinuria, elevated serum creatinine showing chronic allograft nephropathy, transplant glomerulopathy and, less frequently, HCV-associated de novo glomerulonephritis. We suggest that HCV infection should be recognized as a true risk factor for graft failure, and preventive measures could include pre-transplant therapy with interferon.