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Epidemiology, clinical picture and long-term outcomes of FIP1L1-PDGFRA-positive myeloid neoplasm with eosinophilia: Data from 151 patients.
Rohmer, J, Couteau-Chardon, A, Trichereau, J, Panel, K, Gesquiere, C, Ben Abdelali, R, Bidet, A, Bladé, JS, Cayuela, JM, Cony-Makhoul, P, et al
American journal of hematology. 2020;(11):1314-1323
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Abstract
FIP1L1-PDGFRA-positive myeloid neoplasm with eosinophilia (F/P+ MN-eo) is a rare disease: robust epidemiological data are lacking and reported issues are scarce, of low sample-size and limited follow-up. Imatinib mesylate (IM) is highly efficient but no predictive factor of relapse after discontinuation has yet been identified. One hundred and fifty-one patients with F/P+ MN-eo (143 males; mean age at diagnosis 49 years; mean annual incidence: 0.18 case per million population) were included in this retrospective nationwide study involving all French laboratories who perform the search of F/P fusion gene (study period: 2003-2019). The main organs involved included the spleen (44%), skin (32%), lungs (30%), heart (19%) and central nervous system (9%). Serum vitamin B12 and tryptase levels were elevated in 74/79 (94%) and 45/57 (79%) patients, respectively, and none of the 31 patients initially treated with corticosteroids achieved complete hematologic remission. All 148 (98%) IM-treated patients achieved complete hematologic and molecular (when tested, n = 84) responses. Forty-six patients eventually discontinued IM, among whom 20 (57%) relapsed. In multivariate analysis, time to IM initiation (continuous HR: 1,01 [0.99-1,03]; P = .05) and duration of IM treatment (continuous HR: 0,97 [0,95-0,99]; P = .004) were independent factors of relapse after discontinuation of IM. After a mean follow-up of 80 (56) months, the 1, 5- and 10-year overall survival rates in IM-treated patients were 99%, 95% and 84% respectively. In F/P+ MN-eo, prompt initiation of IM and longer treatment durations may prevent relapses after discontinuation of IM.
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Randomized, Open-Label, Phase IV, Korean Study of Kidney Transplant Patients Converting From Cyclosporine to Prolonged-Release Tacrolimus Plus Standard- or Reduced-Dose Corticosteroids.
Baek, CH, Kim, CD, Lee, DR, Kim, YH, Yang, J, Kim, BS, Lee, JS, Han, SY, Kim, SW, Lee, S, et al
Transplantation proceedings. 2019;(3):749-760
Abstract
BACKGROUND This 24-week, multicenter, randomized, exploratory, comparative, open-label, phase-IV study assessed the safety and efficacy of prolonged-release tacrolimus (PR-T) with reduced-dose versus standard-dose corticosteroids in stable kidney transplant recipients in Korea after converting from cyclosporine-based therapy. METHODS At baseline, patients were converted from cyclosporine-based to PR-T-based immunosuppression and randomized (1:1) to receive either corticosteroids maintained at prestudy dose (standard-dose group) or tapered from week 4 to 50% of the prestudy dose by week 12 (reduced-dose group). Patients were seen at baseline and weeks 1, 4, 12, and 24. The primary endpoint was change in estimated glomerular filtration rate (Modification-of-Diet-in-Renal-Disease-4) between baseline and week 24. Secondary endpoints included either acute rejection or patient-reported satisfaction with PR-T. Adverse events (AEs) were recorded. RESULTS Overall, 150 patients were randomized into a reduced-dose group (n = 73) and a standard-dose group (n = 77). At week 24, mean ± standard deviation for corticosteroid dose was 2.5 ± 0.9 mg and 5.0 ± 1.3 mg, respectively. Mean change in estimated glomerular filtration rate from baseline to week 24 was +1.5 ± 9.1 mL/min/1.73 m2 (P = .1567) and +3.4 ± 10.6 mL/min/1.73 m2 (P = .0065), respectively, and not significantly different between groups. There were no acute rejection episodes. Most respondents (>70%) considered PR-T more convenient than cyclosporine. AE incidence was similar between groups. The most common AEs experienced by ≥3% of patients in either treatment group were gastrointestinal events (20.8% and 28.6% of patients receiving reduced- and standard-dose corticosteroids, respectively). Most AEs in both treatment groups were mild or moderate in severity. CONCLUSION Renal function was maintained following conversion from cyclosporine to PR-T, irrespective of corticosteroid regimen; PR-T enables reduced corticosteroid dosage.
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FCER2 T2206C variant associated with FENO levels in asthmatic children using inhaled corticosteroids: The PACMAN study.
Karimi, L, Vijverberg, SJH, Farzan, N, Ghanbari, M, Verhamme, KMC, Maitland-van der Zee, AH
Clinical and experimental allergy : journal of the British Society for Allergy and Clinical Immunology. 2019;(11):1429-1436
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Abstract
BACKGROUND The FCER2 gene, via encoding of the CD23 receptor, plays an important role in the regulation of IgE responses. A genetic variant of the FCER2 gene (T2206C) was previously shown to be associated with IgE levels in asthmatic children. IgE sensitization has also been linked to increased levels of fractional exhaled nitric oxide (FENO). OBJECTIVE To investigate whether the FCER2 T2206C variant influences FENO levels in asthmatic children with a reported use of inhaled corticosteroids (ICS). METHODS This cross-sectional study involved 593 asthmatic children with a reported use of ICS, availability of FENO measurements and genotyping data on the FCER2 T2206C variant (rs28364072). An additive genetic model was assumed, and the association between the FCER2 T2206C variant and the log-transformed (ln) FENO levels was evaluated using linear regression analysis, adjusted for age, sex, adapted British Thoracic Society (BTS) treatment steps and atopy. RESULTS The mean age of the population was 9.1 ± 2.2 years, and the median of FENO levels was 13.0 ppb with an interquartile range (IQR) of (8.0-27.5 ppb). The minor allele (G) frequency of rs28364072 was 29.6%, and each extra copy of the G allele was significantly associated with a lower level of the geometric mean of FENO (log scale, β = -0.12, 95% CI: -0.23, -0.02). CONCLUSION AND CLINICAL RELEVANCE Our results showed that the FCER2 T2206C variant was significantly associated with lower FENO levels in carriers of the G allele. Nevertheless, this SNP contributed little to the variability in FENO levels in this patient population. Our findings contribute to the present knowledge on FENO in asthmatic children; however, future replication studies are required to establish the role of this gene in relation to FENO.
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An Open-labeled, Multicenter Phase II Study of Tamibarotene in Patients with Steroid-refractory Chronic Graft-versus-Host Disease.
Maeda, Y, Nishimori, H, Inamoto, Y, Nakamae, H, Sawa, M, Mori, Y, Ohashi, K, Fujiwara, SI, Tanimoto, M
Acta medica Okayama. 2016;(5):409-412
Abstract
Chronic graft-versus-host disease (GVHD) is a major cause of late death and morbidity following allogeneic hematopoietic cell transplantation (HSCT). Retinoic acid (tamibarotene) exerts multiple effects on cell differentiation and is clinically used for the treatment of acute promyelocytic leukemia. Tamibarotene down-regulates both Th1 and Th17 differentiation in donor T cells after allogeneic HSCT, resulting in attenuation of experimental chronic GVHD. Based on preclinical data, we have launched a phase II study of tamibarotene in patients with steroid-refractory chronic GVHD. This study will clarify whether tamibarotene can exert beneficial effects in patients with steroid-refractory chronic GVHD.
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CINRG pilot trial of coenzyme Q10 in steroid-treated Duchenne muscular dystrophy.
Spurney, CF, Rocha, CT, Henricson, E, Florence, J, Mayhew, J, Gorni, K, Pasquali, L, Pestronk, A, Martin, GR, Hu, F, et al
Muscle & nerve. 2011;(2):174-8
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INTRODUCTION Corticosteroid treatment slows disease progression and is the standard of care for Duchenne muscular dystrophy (DMD). Coenzyme Q10 (CoQ10) is a potent antioxidant that may improve function in dystrophin-deficient muscle. METHODS We performed an open-label, "add-on" pilot study of CoQ10 in thirteen 5-10-year-old DMD patients on steroids. The primary outcome measure was the total quantitative muscle testing (QMT) score. RESULTS Twelve of 16 children (mean age 8.03 ± 1.64 years) completed the trial. Target serum levels of CoQ10 (≥2.5 μg/ml) were shown to be subject- and administration-dependent. Nine of 12 subjects showed an increase in total QMT score. Overall, CoQ10 treatment resulted in an 8.5% increase in muscle strength (P = 0.03). CONCLUSIONS Addition of CoQ10 to prednisone therapy in DMD patients resulted in an increase in muscle strength. These results warrant a larger, controlled trial of CoQ10 in DMD.
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Dural sac cross-sectional area does not correlate with efficacy of percutaneous adhesiolysis in single level lumbar spinal stenosis.
Park, CH, Lee, SH, Jung, JY
Pain physician. 2011;(4):377-82
Abstract
BACKGROUND Spinal stenosis is a narrowing of the spinal canal, which causes mechanical compression of spinal nerve roots. The compression of these nerve roots can cause low back pain and/or leg pain, as well as neurogenic claudication. Lumbar epidural steroid injections have commonly been used in patients with lumbar spinal stenosis (LSS). In cases that are refractory to epidural steroid injections, percutaneous epidural adhesiolysis has been used. OBJECTIVE The aim of our study is to determine the relationship between the severity of spinal stenosis and the participants' response to adhesiolysis, and to evaluate the mid-term effectiveness of adhesiolysis. STUDY DESIGN A prospective observational study. METHODS Sixty-six patients with degenerative LSS were enrolled in this prospective study. All participants underwent lumbar spine magnetic resonance imaging (MRI). The cross-sectional area of the dural sac was measured on the transverse angled sections through the central part of the disc on conventional MR images. All percutaneous adhesiolyses were performed in the operating room. One hour following the procedure, 6 mL of 8% sodium chloride solution was infused during 30 minutes in the recovery room while the patient underwent monitoring. Outcome measures were obtained using the 5-point patient satisfaction scale at 2 weeks and 6 months post-treatment. To evaluate outcome predictors, we divided the participants into 2 groups according to their response to treatment. LIMITATIONS Secondary outcomes were not measured and the study did not include a long-term follow-up period. RESULTS Improvement (including reports of slightly improved, much improved, and no pain) was observed in 49 participants (74.2%) at 2 weeks and 45 participants (66.7%) at 6 months after the procedure. The dural sac cross-sectional area (DSCSA) did not differ between participants who reported improvement and those who did not. There was no statistically significant correlation between pain relief and DSCSA, age, or participant sex. CONCLUSION Percutaneous adhesiolysis was shown to be effective for the treatment of LSS, with mid-term result, without affecting DSCSA.
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Interleukin-2 receptor antibody (basiliximab) for immunosuppressive induction therapy after liver transplantation: a protocol with early elimination of steroids and reduction of tacrolimus dosage.
Liu, CL, Fan, ST, Lo, CM, Chan, SC, Ng, IO, Lai, CL, Wong, J
Liver transplantation : official publication of the American Association for the Study of Liver Diseases and the International Liver Transplantation Society. 2004;(6):728-33
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Abstract
A prospective evaluation was performed to study the potential benefits of the use of interleukin-2 receptor antibody (IL-2Rab) in the induction therapy with early elimination of steroid and reduction of tacrolimus dosage in liver transplant recipients among whom 94% had chronic hepatitis B infection. Thirty-one liver transplant recipients who underwent right-lobe live donor (n = 19) or cadaveric (n = 12) liver transplantation received IL-2Rab, basiliximab 20 mg intravenously within 6 hours of graft reperfusion and on postoperative day 4 (IL-2ab group). Two doses of steroid injection were given intraoperatively and on postoperative day 1. Postoperative immunosuppression was maintained with oral tacrolimus and mycophenolate mofetil without the use of steroids. The operative outcomes were compared with those of 49 patients who received standard immunosuppressive regimen consisting of tacrolimus and corticosteroid (steroid group). The overall postoperative morbidity and hospital stay were comparable between the 2 groups. There were significantly lower incidences of postoperative new-onset diabetes (0% vs 28%, P =.011), acute cellular rejection (6% vs 27%, P =.038), and cytomegalovirus (CMV) antigenemia (0% vs 18%, P =.011) in the IL-2Rab group compared with the steroid group. The blood cholesterol level at 6 months after transplantation was significantly lower in the IL-2Rab group (median, 4.0 vs 4.4 mmol/L, P =.007). On follow-up, none of the patients in the IL-2Rab group had hepatitis B viral breakthrough or hepatocellular carcinoma (HCC) recurrence, whereas 1 and 3 patients in the steroid group developed these complications, respectively. In conclusion, treatment of liver transplant recipients with IL-2Rab with early withdrawal of steroids and reduction of tacrolimus dosage is associated with lower incidences of postoperative new-onset diabetes, acute cellular rejection, and CMV antigenemia, as well as a lower serum cholesterol level. Further studies and long-term follow-up are required to document their potential benefits on hepatitis B and HCC recurrences.
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The effect of perioperative corticosteroids on the outcome of microscopic lumbar disc surgery.
Lundin, A, Magnuson, A, Axelsson, K, Kogler, H, Samuelsson, L
European spine journal : official publication of the European Spine Society, the European Spinal Deformity Society, and the European Section of the Cervical Spine Research Society. 2003;(6):625-30
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Eighty adult patients with lumbar disc herniation verified by magnetic resonance imaging (MRI) and clinical findings corresponding to the radiological level underwent microscopic disc removal to evaluate the outcome of perioperatively given corticosteroids in a prospective randomized double-blind study. In the treatment group the patient received 250 mg Solu-Medrol intravenously and 160 mg Depo-Medrol intramuscularly. Before closure of the wound, a free fat transplant soaked in 80 mg Depo-Medrol was placed on the dural sac. In the control group the same procedure was performed, but sodium chloride was given instead of Depo-Medrol. All patients underwent a clinical examination before surgery and at 2, 6, 12, 26, 52 and 104 weeks postoperatively, rating their pain with the visual analog scale (VAS) and function with the Disability Rating Index (DRI). The postoperative hospital stay was significantly shorter (P=0.01) in the treatment group (1.7 days) compared to the control group (2.3 days). Time taken to return to full-time work was also significantly shorter in the treatment group (P=0.003). VAS-W (Worst Pain during last week) was significantly lower in the treatment group (P=0.02). Postoperative spondylitis occurred in one patient in the control group and no adverse corticosteroids effect was seen. Our study shows that perioperatively given corticosteroids improve the outcome of microscopic disc surgery in terms of length of hospital stay and time taken to return to full-time work. The results also indicate that corticosteroid treatment reduces pain and improves functional outcome.
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Montelukast or salmeterol combined with an inhaled steroid in adult asthma: design and rationale of a randomized, double-blind comparative study (the IMPACT Investigation of Montelukast as a Partner Agent for Complementary Therapy-trial).
Bjermer, L, Bisgaard, H, Bousquet, J, Fabbri, LM, Greening, A, Haahtela, T, Holgate, ST, Picado, C, Leff, JA
Respiratory medicine. 2000;(6):612-21
Abstract
Asthma patients who continue to experience symptoms despite taking regular inhaled corticosteroids represent a management challenge. Leukotrienes play a key role in asthma pathophysiology, and since pro-inflammatory leukotrienes are poorly suppressed by corticosteroids it seems rational to add a leukotriene receptor antagonist (LTRA) when a low to moderate dose of inhaled corticosteroids does not provide sufficient disease control. Long acting beta2-agonist (LABA) treatment represents an alternative to LTRAs and both treatment modalities have been shown to provide additional disease control when added to corticosteroid treatment. To compare the relative clinical benefits of adding either a LTRA or a LABA to asthma patients inadequately controlled by inhaled corticosteroids, a randomized, double-blind, multi-centre, 48-week study will be initiated at approximately 120 centres throughout Europe, Latin America, Middle East, Africa and the Asia-Pacific region in early 2000. The study will compare the oral LTRA montelukast with the inhaled LABA salmeterol, each administered on a background of inhaled fluticasone, on asthma attacks, quality of life, lung function, eosinophil levels, healthcare utilization, and safety, in approximately 1200 adult asthmatic patients. The requirements for study enrollment include a history of asthma, FEV1 or PEFR values between 50% and 90% of the predicted value together with > or = 12% improvement in FEV1 after beta-agonist administration, a minimum pre-determined level of asthma symptoms and daily beta-agonist medication. The study will include a 4-week run-in period, during which patients previously taking inhaled corticosteroids are switched to open-label fluticasone (200 microg daily), followed by a 48-week double-blind, treatment period in which patients continuing to experience abnormal pulmonary function and daytime symptoms are randomized to receive montelukast (10 mg once daily) and salmeterol placebo, or inhaled salmeterol (100 microg daily) and montelukast placebo. All patients will continue with inhaled fluticasone (200 microg daily). During the study, asthma attacks, overnight asthma symptoms, and morning peak expiratory flow rate will be assessed using patient diary cards; quality of life will also be assessed using an asthma-specific quality-of life questionnaire. The results of this study are expected to provide physicians with important clinical evidence to help them make a rational and logical treatment choice for asthmatic patients experiencing breakthrough symptoms on inhaled corticosteroids.
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Efficacy of intermittent etidronate therapy for corticosteroid-induced osteoporosis in patients with diffuse connective tissue disease.
Jinnouchi, Y
The Kurume medical journal. 2000;(3):219-24
Abstract
We conducted a one-year comparative study of 25 patients with corticosteroid-induced osteoporosis associated with diffuse connective tissue disease. The patients were randomly divided into 2 groups: group A (9 patients), monotherapy with active vitamin D3 (V.D3); and group B (16 patients), combination therapy with V.D3 and etidronate. Four markers were employed: as an bonegenic marker, serum alkaline phosphatase (ALP); as a bone resorption marker, urinary deoxypyridinoline (DPD); as a bone salt minerals assay level, young adult mean (YAM); and bonefracture ratio. Results showed that: ALP decreased in both groups with no significant difference between groups; DPD increased significantly from baseline (p < 0.05) in group A, but it decreased significantly from baseline (p < 0.05) in group B, but again without a significant difference between groups; YAM resulted in no significant improvement in group A, but a significant improvement from baseline (p < 0.01) was shown in group B, with a significant difference between groups (p < 0.05); and a new spinal compression fracture ratio was extremely lower in group A than in group B. The findings indicated cyclical/intermittent etidronate therapy is effective in preventing corticosteroid-induced osteoporosis.