scholarly journals The Weighed Core Symptom Scale and Prediction of ADHD in Adults – Objective Measures of Remission and Response to Treatment with Methylphenidate

2013 ◽  
Vol 9 (1) ◽  
pp. 171-179
Author(s):  
Hanna Edebol ◽  
Lars Helldin ◽  
Torsten Norlander
Keyword(s):  
Adult Adhd ◽  
Response Rate ◽  
Optimal Dose ◽  
Response To Treatment ◽  
Therapeutic Effects ◽  
Modified Release ◽  
Symptom Scale ◽  
Core Symptom ◽  
Two Measures

Objective: Two measures of the response rate and the optimal treatment response for adult ADHD were evaluated using methylphenidate. The hypotheses were that Prediction of ADHD (PADHD) defines remission, the Weighed Core Symptom (WCS) scale registers direct effects of medication and that WCS may indicate the optimal dose level during titration. Design: PADHD and WCS were analyzed at baseline and after intake of low doses of either short-acting or modified-release formulations of methylphenidate, MPH (Study I), during titration with modified-release formulations of MPH (18/27, 36, 54, 72 mg) and at three months follow-up (Study II). Patients: Study I consisted of 63 participants (32 females) and Study II consisted of 10 participants (6 females) diagnosed with ADHD and who was to start with treatment. Outcome measures: Prediction of ADHD (PADHD) indicates the occurrence of ADHD (No, Yes) and the Weighed Core Symptom scale (WCS) quantifies ADHD from 0 to 100 (max-min). Results: The number of clinical cases of ADHD decreased after methylphenidate treatment according to PADHD. WCS increased (p < 0.001) from 9.75 (SD = 12.27) to 47.50 (SD = 29.75) with about 10 mg of methylphenidate (N = 63). During titration, symptoms improved after 18/27 mg and 36 mg of methylphenidate and baseline-follow up comparisons showed WCS increments (p = 0.005) from 31.00 (N = 10, SD = 26.85) to 69.00 (N = 10, SD = 22.34). Conclusions: PADHD defined remission and WCS measured therapeutic effects of methylphenidate in adult ADHD.

scholarly journals Eltrombopag Plus Pulsed Dexamethasone As First Line Therapy for Subjects with Immune Thrombocytopenic Purpura (ITP)

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 733-733 ◽  
Author(s):  
Lunqing Zhang ◽  
Mingjie Zhang ◽  
Xin Du ◽  
Yunfeng Cheng ◽  
Gregory Cheng
Keyword(s):  
Pilot Study ◽  
Response Rate ◽  
Response To Treatment ◽  
Platelet Response ◽  
First Line ◽  
Platelet Counts ◽  
First Line Therapy ◽  
Line Therapy ◽  
Prolonged Response

Abstract Background: Eltrombopag is an oral thrombopoietin receptor agonist that has been licensed for use as second line therapy in ITP patients. For most subjects, platelet counts usually return to baseline within 2 weeks of eltrombopag discontinuation, however, up to 15% of subjects may maintain a prolonged response after discontinuation (BJH Volume 165(6) 2014 865-869). Therefore, we conducted a pilot study to evaluate whether a 12-week course of eltrombopag plus pulsed dexamethasone as first line therapy can increase the proportion of patients with prolonged response. Methods: This multicenter, single arm, open-label pilot study was performed on subjects with newly diagnosed ITP. Eligible subjects had confirmed ITP and platelet counts <30×109/L or platelet counts < 50×109/L and significant bleeding symptoms (WHO bleeding scale 2 or above). Patients must have no prior ITP treatment except platelet transfusions. All patients provided written informed consent before enrolment. This study was conducted in accordance with the Declaration of Helsinki. Eligibility criteria included isolated thrombocytopenia with exclusions of secondary immune or drug-induced thrombocytopenia, cancer and pregnancy related thrombocytopenia and virus induced thrombocytopenia. Bone marrow examination is not mandatory but must be consistent with peripheral platelet consumptions with no features of dysplasia, extensive fibrosis, aplasia, marrow infiltration if performed. Treatment consisted of eltrombopag 25-75mg daily according to platelet response for 12 weeks plus pulsed dexamethasone, 40mg daily for 4 consecutive days every 4 weeks for 1-3 courses. The primary endpoint was prolonged response defined as platelet counts > 50×109/L for more than 6 months without any ITP therapy after completion of 12-week therapy. The reported prolonged response rate of dexamethasone alone is around 25%, a sample size of 60 subjects will be required to detect a doubling of the prolonged response rate at a significant level of 0.05 with power of 0.9. Differences between prolonged responder and relapsed subject groups were analyzed by the Student's t-test. P values <0.05 were considered significant. Results :46 subjects were enrolled from February 2015 to July 2018. The median age was 40 years, range 18 to 81; 29 were female and 17 were male. Median platelet counts at baseline were 18×109/L, range 1 to 44×109/L. One subject withdrew consent before starting treatment. One was withdrawn because of protocol violations. Three subjects did not have significant increase in platelet counts during the entire 12 weeks treatment despite a maximum of eltrombopag 75mg daily and 3 courses of pulsed dexamethasone. One of them subsequently turned out to be amegakaryocytic thrombocytopenia. These 5 subjects were still included in the final analysis. Twenty-nine subjects had good initial response to treatment and completed at least 6 months follow-up (Fig 1), 19 of them had achieved the primary end point of platelet counts count> 50×109/L for more than 6 months after discontinuation of eltrombopag. The median platelet counts at 6 months were 150×109/L (range 53 to 371×109/L), Average eltrombopag dose was 36.8mg daily and average pulsed dexamethasone was 2.2 courses. Eleven subjects maintained prolonged response for 12 months or more. Four subjects are still receiving treatment and 8 subjects are still on the 6 months off-therapy observation period (median follow up is 12 weeks, range 4 to 21 weeks). Ten subjects relapsed after discontinuation of eltrombopag(Table 1). The median time to relapse was 47.8 days, range 15 to 148 days. Average daily eltrombopag was 45mg and average pulsed dexamethasone was 2.5 courses. So far, the prolonged response rate of 56 % (19/34) among 34 evaluable subjects is very encouraging. According to statistical analysis, seven more prolonged responders among the remaining 26 subjects (14 yet enrolled and 12 pending final evaluation) would suggest that 12-week of eltrombopag plus pulsed dexamethasone as first line therapy may significantly improve prolonged response rate in ITP patients. All subjects tolerated the treatment well and no Grade 3 or above adverse effects were reported. Conclusions: Eltrombopag plus pulsed dexamethasone is an effective and safe treatment for ITP as a first line therapy that can provide sustained prolonged response in adults. This therapy could be a new treatment option for ITP subjects. Disclosures Cheng: Novartis: Research Funding; BMS: Honoraria, Speakers Bureau; Astrazeneca: Honoraria, Speakers Bureau.

scholarly journals High Response Rate to Antidepressants in the Management of Paroxysmal Hypertension (Pseudopheochromocytoma)

2021 ◽  
Author(s):  
Samuel J. Mann ◽  
Kaushal V. Solanki
Keyword(s):  
Response Rate ◽  
Case Series ◽  
Preventive Treatment ◽  
High Response Rate ◽  
High Response ◽  
Response To Treatment ◽  
Paroxysmal Hypertension ◽  
Treatment Data ◽  
Lost To Follow Up

There is no widely recognized preventive treatment for patients with paroxysmal hypertension (“pseudopheochromocytoma”) who suffer recurrent and often severe paroxysmal elevation of blood pressure, repeated emergency room visits and hospitalizations. In this case series we assessed the effectiveness of treatment with an antidepressant in preventing recurrent paroxysms. A secondary exploratory objective was to examine the psychological profile of patients and its relationship to response to treatment. The charts of 52 patients who reported having experienced at least 3 symptomatic episodes were selected; and and for whom treatment data were included. Treatment with an antidepressant was offered to, and follow-up data were available in 37..Two patients refused treatment, 6 were unable to tolerate an effective dose, 3 were lost to follow-up, and 1 was non-compliant with the medication. Of the remaining 25 evaluable patients, 92% (23/25) responded, with 80% (21/25) achieving complete and persisting cessation of paroxysms, and 8% (2/25), a reduction in frequency. Importantly, an antidepressant was effective in nearly all patients who reported that they were not suffering from anxiety or depression. The data were insufficient to determine superiority of any one antidepressant versus another. We conclude that treatment with an antidepressant is effective in preventing hypertensive paroxysms in a high proportion of patients with paroxysmal hypertension. Given the absence of any other pharmacologic intervention capable of preventing recurrent paroxysms, the similarly high response rate observed in previous reports, and the relatively safe profile of antidepressant agents, the findings support more widespread use of an antidepressant in patients with this disorder to prevent years of continued hypertensive paroxysms and their consequences.

Anti-MAG Neuropathy: a Single Center Retrospective Study In 61 Patients

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 3951-3951
Author(s):  
Marie-Anne HOSPITAL ◽  
Sonia Dragomir ◽  
Vincent Levy ◽  
Jean Neil ◽  
Lucile Musset ◽  
...  
Keyword(s):  
Retrospective Study ◽  
Board Of Directors ◽  
Response Rate ◽  
Immunoglobulin M ◽  
Response To Treatment ◽  
First Line ◽  
Advisory Committees ◽  
Significant Difference ◽  
Before And After

Abstract Abstract 3951 Background: Polyneuropathy is the most frequent neurological complication in Waldenstrom macroglobulinemia (WM). Most patients have an insidiously progressive distal symmetric sensorimotor and ataxic neuropathy due to a monoclonal immunoglobulin M anti-myelin-associated glycoprotein (MAG). There is still no treatment reference today. Recent studies underline the interest of Rituximab (RTX) but its efficacy is not yet proven. In order to identify the interest of immunotherapy, we performed a retrospective study in 61 patients with anti-MAG over a period of 12 years. Methods: All patients underwent neurological, biological (anti-MAG antibodies, IgM serum) and electrophysiological examination before and after each treatment. Clinically, patients were considered to be improved if they had a decrease of at least two points on the INCAT sensory sum score and/or a decrease of 20 mm on the visual analogue scale and/or motor strength improvement by at least two MRC points in the ankle dorsiflexor. Biologically, response anti-MAG antibodies and IgM serum were considered to be improved if their level was diminished by half. Electrophysiological studies were performed using standard procedures. Results: A total of 61 patients were analyzed. The median age at onset was 64 years (range 33–84), median serum IgM concentration 4.6 g/L (0-17), median anti-MAG antibodies 49900 BTU (23000>70000), RANKIN score 1 (35 patients), 2(20 patients), 3(6 patients). Twenty-five patients had WM with lymphoplasmacytic cells bone marrow infiltration. None of the patients had criteria for therapy initiation according to the 2th international workshop except symptomatic or evolving neuropathy. In first line, 45 patients were treated with Chlorambucil (CBL) (8 improved, 28 were stabilised, 9 worsened), 16 patients were treated with RTX alone (9) or in combination (7) (11 improved, 2 were stabilised, 3 worsened). RTX gave a significant higher response rate compared with CBL (p=2×10-4). With a median follow up of 96 months, 15 patients treated with CBL relapsed. Only one patient treated with RTX relapsed but the median follow up was not reached (60 months follow up). In the CBL group, 15 patients were treated at relapse with RTX and 11 improved, 3 were stabilised, 1 worsened. The average time follow up of the 2nd response was 48 months. Twelve patients in failure were treated with RTX: 8 improved, 1 was stabilised, 3 failed with an average follow up of 48 months. There was no significant difference between anti-MAG antibodies level before and after treatment (p=0.64) in patients in response but a low IgM level was associated with response to treatment (p<0.029). Conclusion: In first line, RTX alone or in combination is associated with a higher response rate than CBL. For patients who relapsed after CHL, patients favourably responded to RTX with an average time follow up of 48 months. IgM level is a prognosis factor (p<0.029) for clinical response to treatment. To better define the efficacy of RTX in this setting, results of a french randomized study comparing RTX to placebo are pending. Disclosures: Choquet: Roche: Consultancy. Leblond:ROCHE: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; MUNDIPHARMA: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; GENZYME: Membership on an entity's Board of Directors or advisory committees; CELGENE: Membership on an entity's Board of Directors or advisory committees; JANSSEN: Membership on an entity's Board of Directors or advisory committees.

NCCTG phase II trial N0531of weekly nab-paclitaxel (nab-p) in combination with gemcitabine (gem) in patients with metastatic breast cancer (MBC)

2007 ◽  
Vol 25 (18_suppl) ◽  
pp. 1048-1048 ◽  
Author(s):  
V. Roy ◽  
B. R. LaPlant ◽  
G. G. Gross ◽  
C. L. Bane ◽  
F. M. Palmieri ◽  
...  
Keyword(s):  
Phase Ii ◽  
Response Rate ◽  
Metastatic Breast ◽  
Progression Free Survival ◽  
Complete Response ◽  
Therapeutic Effects ◽  
Her2 Positive ◽  
Combination Methods ◽  
Dose Modifications

1048 Background: Previous trials with p plus gem show significant antitumor activity in MBC. Nab-p has lower toxicity compared to p with similar therapeutic effects. This study assessed proportion of confirmed tumor responses and toxicity profile of this combination. Methods: Single stage phase II study enrolled patients (pts) with measurable MBC eligible for 1st line chemotherapy (chemo). Pts treated (tx) with nab-p 125 mg/m2 followed by gem 1,000 mg/m2 days 1 and 8 of 21 day cycle. Dose modifications allowed for both drugs. Primary endpoint was proportion of confirmed responses; complete response (CR), partial response (PR) by RESIST criteria on two consecutive evaluations at least 6 weeks apart. 41 evaluable pts required to test the null hypothesis that the confirmed response rate was at most 40% against the alternative of at least 60%. Results: 50 pts enrolled 11/05 to 5/06. Median age 56 (29–86). 40 pts (80%) visceral disease, 34 (68%) ER positive, 28 (56%) PR positive, 1 (2%) HER2 positive, 1 (2%) prior trastuzumab treatment (tx). 25 (50%) had prior adjuvant chemo, including 15 (30%) prior taxane. Median follow-up 7 mos (3–12). Median of 6 cycles of tx completed (1–15). 41 pts discontinued tx, due to progression (41%), pt refusal (27%), adverse events (17%), alternate tx (7%). Confirmed response rate 48% (95% CI: 34–63%): 4 CR, 20 PR. Median duration of response 6.6 mos (95% CI: 5.7, not reached), median progression-free survival (PFS) 7.9 mos (95% CI: 5.3–9.3). Rate of overall survival (OS) at 6 mos 91% (95% CI: 84–100%), 6 month PFS 58% (95% CI: 45–74%). Median OS not reached. 30 pts required dose delays on total of 52 cycles, mostly due to hematologic AEs. 9 pts (19%) had grade (gr) 4 adverse event (AE), 32 (67%) had maximum gr 3 AE. Most common gr 3/4 AEs: neutropenia (52%), fatigue (26%), anemia (14%), dyspnea (14%), thrombocytopenia (12%). Four pts had gr 3 neuropathy. Conclusions: Nab-paclitaxel in combination with gem has clear activity in patients with MBC. Manageable hematologic, but no other significant toxicities. Study ongoing to correlate efficacy with caveolin-1 levels with results expected Spring 2007. Follow up study of this combination with an anti-angiogenesis agent is in development. No significant financial relationships to disclose.

Comparison of the Clinical and Treatment Characteristics of Patients Undergoing Electroconvulsive Therapy for Catatonia Indication in the Context of Gender

2021 ◽  
pp. 155005942110258
Author(s):  
Zozan Parsanoglu ◽  
Ozlem Devrim Balaban ◽  
Sakir Gica ◽  
Ozge Canbek Atay ◽  
Ozan Altin
Keyword(s):  
Clinical Data ◽  
Electroconvulsive Therapy ◽  
Response Rate ◽  
Signs And Symptoms ◽  
Response To Treatment ◽  
Global Improvement ◽  
Male And Female ◽  
Opposite Sex ◽  
The Mean

The aim of this study was to compare in the context of gender both clinical diagnosis and disease-related differences and electroconvulsive therapy (ECT)-related differences in data and efficacy in hospitalized patients with catatonic signs and symptoms. Data from 106 patients who received ECT with catatonia indication were retrospectively analyzed. Clinical data of male (n = 58) and female (n = 48) patients were compared. Hospitalization documents and outpatient files, sociodemographic and clinical data form, Clinical Global Improvement scores used by the ECT unit in the follow-up of patients who received ECT were used in the study. It was seen that the mean age of women at the onset of ECT was higher than in men and the presence of prolonged seizures was more common than men. In men, it was found out that the average number of sessions with the onset of clinical response to treatment was higher than the average of women. The distribution of diagnoses by gender showed that the presence of schizophrenia diagnosis in men and of bipolar disorder in women were significantly more frequent compared to the opposite sex. It was found out that there were no significant differences between genders in terms of response rate to ECT. Our study is important for being the first study in the literature investigating the gender differences in ECT used for catatonia. However, gender is not a distinctive factor in the effectiveness of treatment, there are some important differences between male and female patients showing signs and symptoms of catatonia and undergoing ECT.

scholarly journals Phase II study of carboplatin (CBDCA) in progressive low-grade gliomas

1998 ◽  
Vol 4 (4) ◽  
pp. E5 ◽  
Author(s):  
Albert Moghrabi ◽  
Henry S. Friedman ◽  
David M. Ashley ◽  
Krystal S. Bottom ◽  
Tracy Kerby ◽  
...  
Keyword(s):  
Partial Response ◽  
Stable Disease ◽  
Response Rate ◽  
Drug Regimen ◽  
Response To Treatment ◽  
Hematological Toxicity ◽  
Maximal Response ◽  
Low Grade ◽  
Juvenile Pilocytic Astrocytoma

In this study, the authors sought to investigate the response rate and toxicity of carboplatin in patients with progressive low-grade glioma (LGG). Thirty-two patients with progressive LGG were treated with carboplatin at a dosage of 560 mg/m2. Treatment was given at 4-week intervals and continued until the disease progressed, unacceptable toxicity supervened, or for 12 additional courses after achieving maximal response. Patients with stable disease were treated with a total of 12 cycles. All patients were treated as outpatients. Patients were evaluated for response to treatment and toxicity. All patients received a minimum of two cycles of carboplatin, and were examined for response. A partial response was achieved in nine patients (28%) and a minimal response in two (6%), for an overall response rate of 34% (11 of 32 patients). Eighteen patients (56%) had stable disease. A partial response was achieved in the nine patients after a median of six cycles (range 4-11 cycles), a minimal response was achieved in the two patients after five cycles. Glioma progression was noted in three patients after three, five, and five cycles, respectively. The 11 patients in whom some response was achieved had either an optic pathway tumor or a juvenile pilocytic astrocytoma. Twenty-six of the 32 patients had those characteristics, making the response rate in that group 42% (11 of 26 patients). Thirty-two patients received a total of 387 cycles of chemotherapy. Hematological toxicity was moderate. Twenty-one patients developed thrombocytopenia (platelet count < 50,000/μl); three patients required one platelet transfusion each. Nine patients developed neutropenia (absolute neutrophil count < 500/μl); one developed fever and required administration of antibiotic agents. One dose adjustment in each of the patients prevented further thrombocytopenia and neutropenia. Two patients with stable disease died of respiratory complications. One patient developed Grade III ototoxicity after receiving five cycles, one patient developed hypersensitivity to carboplatin, and none developed nephrotoxicity. Carboplatin given at a dosage of 560 mg/m2 every 4 weeks has activity in patients with progressive LGG. This drug regimen is relatively simple and well tolerated. Further investigation and longer follow-up study are warranted.

Response to Treatment of Alcoholism; a Follow-Up Study

1968 ◽  
Vol 29 (2) ◽  
pp. 364-381 ◽  
Author(s):  
Alex D. Pokorny ◽  
Byron A. Miller ◽  
Sidney E. Cleveland
Keyword(s):  
Response To Treatment ◽  
Follow Up Study

Knowledge, Perceptions and Practices on Modified Release Tablets among Prescribers: A Preliminary Study

2020 ◽  
Vol 01 (01) ◽  
pp. 05-14
Author(s):  
M.G.K.M. Fernando ◽  
K.I.J. Priyadarshi ◽  
L.G.T. Shanika ◽  
N.R. Samaranayake
Keyword(s):  
Online Survey ◽  
Product Information ◽  
Response Rate ◽  
Immediate Release ◽  
State Universities ◽  
Modified Release ◽  
Validated Questionnaire ◽  
Therapeutic Outcomes ◽  
Medical Faculties ◽  
Enteric Coated

Introduction: Modified release tablets (MRTs) are developed to achieve different therapeutic outcomes and are frequently prescribed. This study aims to evaluate the knowledge, perceptions and practices on using MRTs among a selected cohort of prescribers. Methods: A self administered online survey was conducted using a pre-validated questionnaire, prepared in-house to assess knowledge, perceptions and practices on using MRTs, among academics with an MBBS degree in medical faculties of State universities in Sri Lanka. Results: The response rate was 15.5% among 375 prescribers. Most were females (53.4%) and were 46-55 years (29.3%). Over 50% correctly expanded abbreviations related to MRTs. Most defined enteric coated (87.9%) and targeted release (77.6%) forms accurately. However, 87.0% mixed-up definitions of sustained release with controlled release. Most believed that inability to split tablets (70.7%) and high cost (70.7%), as disadvantages of MRTs. Nearly half did not identify the risk of dose dumping (53.5%) and inflexible dosing schedule (44.8%) as disadvantages. For frequency of administering MRTs, 86.2% referred the product information leaflet (PIL) while 29.0% depended on the frequency of the corresponding immediate release tablet. Most (79.3%) prescribed MRTs to increase patient compliance while 12.1% prescribed them to reduce cost. When problems regarding MRTs were encountered, most referred PILs (81.0%) and clarified with experts (75.9%). Conclusions: Although the response rate was low, a clear gap in knowledge, perceptions and practices on using MRTs were identified among prescribers who responded. Interventions are needed to improve the knowledge, perceptions, and practices on using MRTs among prescribers.

scholarly journals A206 EVOLUTION OF PEDIATRIC AUTOIMMUNE CHOLANGITIS AND PRIMARY SCLEROSING CHOLANGITIS INTO ADULTHOOD

2021 ◽  
Vol 4 (Supplement_1) ◽  
pp. 234-236
Author(s):  
P Willems ◽  
J Hercun ◽  
C Vincent ◽  
F Alvarez
Keyword(s):  
Primary Sclerosing Cholangitis ◽  
Sclerosing Cholangitis ◽  
Response To Treatment ◽  
Similar Proportion ◽  
Autoimmune Cholangitis ◽  
Significant Difference ◽  
One Year ◽  
Liver Tests

Abstract Background The natural history of primary sclerosing cholangitis (PSC) in children seems to differ from PSC in adults. However, studies on this matter have been limited by short follow-up periods and inconsistent classification of patients with autoimmune cholangitis (AIC) (or overlap syndrome). Consequently, it remains unclear if long-term outcomes are affected by the clinical phenotype. Aims The aims of this is study are to describe the long-term evolution of PSC and AIC in a pediatric cohort with extension of follow-up into adulthood and to evaluate the influence of phenotype on clinical outcomes. Methods This is a retrospective study of patients with AIC or PSC followed at CHU-Sainte-Justine, a pediatric referral center in Montreal. All charts between January 1998 and December 2019 were reviewed. Patients were classified as either AIC (duct disease on cholangiography with histological features of autoimmune hepatitis) or PSC (large or small duct disease on cholangiography and/or histology). Extension of follow-up after the age of 18 was done for patients followed at the Centre hospitalier de l’Université de Montréal. Clinical features at diagnosis, response to treatment at one year and liver-related outcomes were compared. Results 40 patients (27 PSC and 13 AIC) were followed for a median time of 71 months (range 2 to 347), with 52.5% followed into adulthood. 70% (28/40) had associated inflammatory bowel disease (IBD) (78% PSC vs 54% AIC; p=0.15). A similar proportion of patients had biopsy-proven significant fibrosis at diagnosis (45% PSC vs 67% AIC; p=0.23). Baseline liver tests were similar in both groups. At diagnosis, all patients were treated with ursodeoxycholic acid. Significantly more patients with AIC (77% AIC vs 30 % PSC; p=0.005) were initially treated with immunosuppressive drugs, without a significant difference in the use of Anti-TNF agents (0% AIC vs 15% PSC; p= 0.12). At one year, 55% (15/27) of patients in the PSC group had normal liver tests versus only 15% (2/13) in the AIC group (p=0.02). During follow-up, more liver-related events (cholangitis, liver transplant and cirrhosis) were reported in the AIC group (HR=3.7 (95% CI: 1.4–10), p=0.01). Abnormal liver tests at one year were a strong predictor of liver-related events during follow-up (HR=8.9(95% CI: 1.2–67.4), p=0.03), while having IBD was not (HR=0.48 (95% CI: 0.15–1.5), p=0.22). 5 patients required liver transplantation with no difference between both groups (8% CAI vs 15% CSP; p=0.53). Conclusions Pediatric patients with AIC and PSC show, at onset, similar stage of liver disease with comparable clinical and biochemical characteristics. However, patients with AIC receive more often immunosuppressive therapy and treatment response is less frequent. AIC is associated with more liver-related events and abnormal liver tests at one year are predictor of bad outcomes. Funding Agencies None

scholarly journals POS0677 THE ROLE OF MUSCULOSKELETAL ULTRASOUND IN PREDICTING THE RESPONSE TO JAK INHIBITORS: RESULTS FROM A LARGE MONOCENTRIC COHORT

2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 583-583
Author(s):  
C. Garufi ◽  
F. Ceccarelli ◽  
F. R. Spinelli ◽  
S. Mancuso ◽  
C. Pirone ◽  
...  
Keyword(s):  
Rheumatoid Arthritis ◽  
Disease Activity ◽  
Structural Damage ◽  
Power Doppler ◽  
Response To Treatment ◽  
Jak Inhibitors ◽  
Inflammatory Score ◽  
Inflammatory Status

Background:In the management of chronic arthritis, such as Rheumatoid Arthritis (RA), Ultrasound (US) assessment can provide relevant information about the joint inflammatory status in the diagnostic phase and even more in the monitoring of disease activity and structural damage1,2.Objectives:In this longitudinal study, we aimed to assesse the role of US in predicting the efficacy of JAK-inhibitors (JAKi) in RA patients.Methods:We enrolled RA patients starting baricitinib or tofacitinib. All patients were evaluated at baseline and after 4, 12, 24, 48 weeks. Disease activity was calculated by DAS28CRP. US examination in 22 joints (I–V MCPs and PIPs, wrists) aimed at evaluating inflammatory features (synovial effusion and hypertrophy, power Doppler-PD), through a semi-quantitative scale (0-3). The total US (0-198) and PD (0-66) scores were calculated. We scanned bilateral flexor (I–V fingers of hands) and extensor compartments (1-6) tendons: tenosynovitis was scored as absent/present (0/1), resulting in a total score (0-22).Results:We studied 102 patients (M/F 15/87; median age 59.2 years, IQR 17.75; median disease duration 144 months, IQR 126), 61 treated with baricitinib and 41 with tofacitinib. At baseline, the median total US score was 18 (IQR 19) and the median PD score 2 (4). We observed a significant reduction in both total and PD US scores at all time-points (p<0.0001) (Figure 1). At baseline, 75.4% of patients showed tenosynovitis involving at least one tendon, with a median score of 2 (IQR 3.5) significantly decreasing after 24 weeks (p=0.02). Multivariate analysis, adjusted for baseline DAS28CRP and other concomitant treatments (including glucocorticoids and methotrexate treatment), confirmed the independent association between baseline US (PD and tenosynovitis) scores and the reduction of disease activity at follow-up evaluations.Conclusion:The present study confirmed the early efficacy of JAKi in RA patients by using US evaluation. Furthermore, power doppler and tenosynovitis scores could play a predictive role in response to treatment.References:[1]MUELLER RB, HASLER C, POPP F, et al. Effectiveness, Tolerability, and Safety of Tofacitinib in Rheumatoid Arthritis: A Retrospective Analysis of Real-World Data from the St. Gallen and Aarau Cohorts. J Clin Med. 2019;8(10):1548.[2]COLEBATCH AN, EDWARDS CJ, ØSTERGAARD M, et al. EULAR recommendations for the use of imaging of the joints in the clinical management of rheumatoid arthritis. Ann Rheum Dis. 2013;72(6):804-14.Figure 1.Ultrasound inflammatory score (a) and Ultrasound Power Doppler (PD) score (b) at baseline and follow-up.Table 1.Baseline characteristics of 414 RA patients.WEEKS04122448US inflammatory score18 (19)11 (15.5)9.5 (11.7)7.5 (8)6 (11)US PD score2 (4)0 (2)0 (1)0 (1)0 (0.7)Disclosure of Interests:Cristina Garufi: None declared, Fulvia Ceccarelli: None declared, Francesca Romana Spinelli Speakers bureau: Abbvie, Eli Lilly, Consultant of: Gilead/Galapagos, Eli Lilly, Grant/research support from: Pfizer, Silvia Mancuso: None declared, Carmelo Pirone: None declared, Fabrizio Conti Speakers bureau: Abbvie, Eli Lilly, Sanofi, Pfizer, Consultant of: Gilead/Galapagos

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