Patients In Full Analysis Set Research Articles

Efficacy and safety of geptanolimab (GB226) for relapsed or refractory peripheral T cell lymphoma: an open-label phase 2 study (Gxplore-002)

BackgroundPeripheral T cell lymphoma (PTCL) is a rare disease and recent approved drugs for relapsed/refractory (r/r) PTCL provided limited clinical benefit. We conducted this study to evaluate the efficacy and safety of geptanolimab (GB226), an anti-PD-1 antibody, in r/r PTCL patients.MethodsWe did this single-arm, multicenter phase 2 study across 41 sites in China. Eligible patients with r/r PTCL received geptanolimab 3 mg/kg intravenously every 2 weeks until disease progression or intolerable toxicity. All patients who received at least one dose of geptanolimab and histological confirmed PTCL entered full analysis set (FAS). The primary endpoint was objective response rate (ORR) in FAS assessed by the independent radiological review committee (IRRC) per Lugano 2014 criteria.ResultsBetween July 12, 2018, and August 15, 2019, 102 patients were enrolled and received at least one dose of geptanolimab. At the data cutoff date (August 15, 2020), the median follow-up was 4.06 (range 0.30–22.9) months. For 89 patients in FAS, 36 achieved objective response (40.4%, 95% CI 30.2–51.4), of which 13 (14.6%) were complete response and 23 (25.8%) had partial response assessed by IRRC. The median duration of response (DOR) was 11.4 (95% CI 4.8 to not reached) months per IRRC. Patients with PD-L1 expression ≥ 50% derived more benefit from geptanolimab treatment compared to < 50% ones (ORR, 53.3% vs. 25.0%, p = 0.013; median PFS 6.2 vs. 1.5 months, p = 0.002). Grade ≥ 3 treatment-related adverse events occurred in 26 (25.5%) patients, and the most commonly observed were lymphocyte count decreased (n = 4) and platelet count decreased (n = 3). Serious adverse events were observed in 45 (44.1%) patients and 19 (18.6%) were treatment related.ConclusionsIn this study, geptanolimab showed promising activity and manageable safety profile in patients with r/r PTCL. Anti-PD-1 antibody could be a new treatment approach for this patient population.Trial registration: This clinical trial was registered at the ClinicalTrials.gov (NCT03502629) on April 18, 2018.

Efficacy and Safety of Geptanolimab (GB226) for Relapsed or Refractory Peripheral T-Cell Lymphoma: An Open-Label Phase 2 Study (Gxplore-002)

Background: Peripheral T cell lymphoma (PTCL) is a rare disease and recent approved drugs for relapsed/refractory (r/r) PTCL provided limited clinical benefit. We conducted this study to evaluate the efficacy and safety of geptanolimab (GB226), an anti-PD-1 antibody, in r/r PTCL patients. Methods: We did this single-arm, multicenter phase 2 study across 41 sites in China. Eligible patients with r/r PTCL received geptanolimab 3 mg/kg intravenously every 2 weeks until disease progression or intolerable toxicity. All patients who received at least one dose of geptanolimab and histological confirmed entered full analysis set (FAS). The primary endpoint was objective response rate (ORR) in FAS assessed by the independent radiological review committee (IRRC) per Lugano 2014 criteria. Findings: Between July 8, 2018 and August 15, 2019, 102 patients were enrolled and received at least one dose of geptanolimab. At data cutoff (Feb 15, 2020), the median follow-up was 4.06 months (range: 0.30-16.7). For 84 patients in FAS, 33 achieved objective response (39.3%, 95% CI 28.80-50.55), of which 10 (11.9%) were complete response and 23 (27.4%) had partial response assessed by IRRC. The 6-month duration of response (DOR) rate was 61.8% (95% CI 41.4, 76.9). Patients with PD-L1 expression ≥50% derived more benefit from geptanolimab treatment compared to <50% ones (ORR, 51.2 vs. 24.2%, p=0.020; median PFS 6.2 vs. 1.4 months, p=0.015). Grade ≥3 treatment-related adverse events occurred in 24 (23.5%) patients; and the most commonly observed were lymphocyte count decreased (n=4) and platelet count decreased (n=4). Serious adverse events were observed in 39 (38.2%) patients and 16 (15.7%) were treatment-related. Interpretation: In this study, geptanolimab showed potent activity with a manageable safety profile. Immune checkpoint inhibitor can be a new treatment approach for patient with r/r PTCL. Trial Registration: This trial was registered at the ClinicalTrials.gov (NCT03502629). Funding: Genor Biopharma Co., Ltd., China National Major Project, and Chinese Academy of Medical Sciences Declaration of Interests: FX and QW are employees of Genor Biopharma Co., Ltd., China. The other authors declared no conflicts of interest. Ethics Approval Statement: The protocol, protocol amendments, and patient informed consent were reviewed and approved by the relevant independent ethics committee at each participating centre prior to implementation. Written informed consent was obtained from all patients prior to enrolment.

PSS22 - Real World, Prospective, Observational, Patient Reported Outcome Study of Hydroxyzine Hydrochloride in Indian Patients with Chronic Pruritis

To assess effectiveness & tolerability of hydroxyzine hydrochloride in management of Indian patients with chronic pruritus in real world setting A prospective, observational, patient reported outcomes study of 400 patients across 7 centres (179 male, 221 female), >18 years of age, having chronic pruritus due to dermatological cause and treated with hydroxyzine hydrochloride as per clinician's discretion upto period of 12 weeks. Primary outcome was improvement from baseline in quality of life, assessed using 10-point Dermatology Quality of Life Index (DLQI) after 12 weeks of study period. Secondary outcomes were improvement in the pruritus scores (5D-Itch scale) after 12 weeks, improvements in DLQI and 5D-Itch scores at 2, 4, and 8 weeks, and clinical safety during the study period were recorded. Wilcoxon test is used to compare baseline and follow-up data for DLQI and pruritus scores. The per-protocol set (PPS) included 177 patients and full-analysis set (FAS) had 391 patients. There was significant improvement (p<0.0001) in DLQI scores at 2 weeks (13.10%), 4 weeks (37.40%), 8 weeks (57.01%) and 12 weeks (76.33%) period in PPS patients. Similar significant improvement in DLQI was noted in FAS at 2, 4, 8 & 12 weeks (p<0.0001). The pruritus scores also decreased significantly (p<0.0001) at 2 weeks (10.56%), 4 weeks (26.32%), 8 weeks (37.71%) and 12 weeks (57.76%) period in PPS patients. Similar significant reduction in pruritus scores was observed in FAS patients at 2, 4, 8 & 12 weeks (p<0001). Total 10 (2.5%) patients reported mild to moderate adverse events (total 11 events) which were dizziness (4), constipation (2), drowsiness (2), dry mouth (2) and sedation (1). All events resolved without any intervention. This real world, observational, patient reported outcome study demonstrates hydroxyzine hydrochloride to significantly decrease symptoms of pruritus and improve quality of life in patients with chronic pruritus over 12 weeks treatment period

The clinical value of China Savin pollen extract used for skin prick test

Objective:The aim of this study is to evaluate the effectiveness and safety of China Savin pollen extract which was used for skin prick test (SPT) in the diagnosis of China Savin pollen allergy. Method:Patients with diagnosis of allergic diseases were collected from Allergy Department of Peking Union Medical College Hospital. All patients were given SPT with China Savin pollen extract, and the mean wheal diameter (MWD) was measured after 15 minutes. Receiver operating characteristic curve (ROC) analysis was performed based on the results of serum specific immunoglobulin E (sIgE). The effectiveness of SPT in the diagnosis of China Savin pollen allergy was evaluated under different diagnostic cutoff values. Adverse events were also recorded to evaluate the safety. Result:A total of 1 029 patients were enrolled in this study without drop out case. There were 1 007 patients in full analysis set (FAS) and 765 patients in per protocol analysis set (PPS). The elimination rate was 25.66%. The area under the ROC curve of FAS is 0.814 (95%CI: 0.788-0.839); which of PPS is 0.829 (95%CI: 0.801-0.857). Based on the ROC curve of PPS, the optimal and the 95% specificity diagnostic cutoff values of MWD were 3.25 mm and 4.75 mm respectively. Based on different diagnostic cutoff value (3.00, 3.25 and 4.75 mm), the sensitivities of SPT with China Savin pollen extract were 0.740 0 (95%CI: 0.701 6-0.778 4), 0.700 (95%CI: 0.659 8-0.740 2) and 0.532 (95%CI: 0.488 3-0.575 7) respectively, whereas the specificity was gradually increased in sequence, which was 0.769 8 (95%CI: 0.719 1-0.820 5), 0.826 4 (95%CI: 0.780 8-0.872 0) and 0.950 9 (95%CI: 0.924 9-0.976 9) respectively. There were 7 adverse events observed among 6 patients (rate: 0.583%, 6/1 029). The manifestation was mild. There was no severe adverse event. Conclusion:SPT with China Savin pollen extract is an effective and safe tool for the diagnosis of China Savin pollen allergy. The effectiveness of diagnosis could be improved based on integration of medical history and different diagnostic threshold values of SPT.

Mirabegron improves quality-of-life, treatment satisfaction, and persistence in patients with overactive bladder: a multi-center, non-interventional, real-world, 12-month study

Objective: Observational studies can provide evidence about patient outcomes in routine clinical practice. This prospective, non-interventional study (BELIEVE) is the largest real-world European study to date to assess quality-of-life, treatment satisfaction, resource utilization, and persistence in patients with overactive bladder (OAB) who were prescribed mirabegron as part of routine clinical practice.Methods: The primary objective was to evaluate change from baseline in quality-of-life based on overactive bladder questionnaire (OAB-q) sub-scales. Secondary objectives included evaluation of treatment persistence, patient satisfaction, healthcare resource utilization and adverse events (AEs). Follow-up was for 12 months with visit windows at 2–4 and 10–12 months. Median change from baseline in total OAB-q and its sub-scales (Health-related quality-of-life [HRQoL] and symptom bother scale) were assessed.Results: Overall, 862 patients were enrolled from eight European countries. In the Full Analysis Set (FAS), 73.7% were female, mean age was 61.2 years; 47.7% ≥65 years. At baseline, 41.3% had switched from other OAB treatments, 42.2% were treatment naïve, 10.1% were lapsed, and 6.4% were on combination treatment. Symptom bother and HRQoL total scores improved from baseline to 2–4 and 10–12 months. There was a notable improvement in dry rate, increasing from 34.9% at baseline to 43.7% at 10–12 months in the FAS, and a reduction in pad use. Persistence was high, with 53.8% of FAS patients remaining on mirabegron at 10–12 months. Overall, no unexpected safety issues were observed and AEs were consistent with the known safety profile of mirabegron.Conclusion: Patients receiving mirabegron in a real-world setting reported meaningful improvements in QoL and health status, with a persistence rate of 53.8% at 12 months for the FAS. No unexpected safety issues were observed, and AEs were consistent with the known safety profile of mirabegron.

Efficacy of vortioxetine monotherapy compared with combined therapy vortioxetine and olanzapine in the treatment of major depression – first results

Abstract Vortioxetine is a novel antidepressant with two mechanisms of action – direct effect on several serotonin receptors and serotonin reuptake inhibition. Atypical antipsychotics, such as olanzapine, used in the augmentation of antidepressants causes not only a better response to treatment, but also increased number of remissions. The aim of our work was to evaluate the efficacy of vortioxetine monotherapy compared to the combined treatment vortioxetine and olanzapine in adult patients with depression during the acute phase of treatment lasting 6 weeks. Depressive symptomatology was assessed by the MADRS scale, anxiety symptoms were assessed by the HAM-A scale and global clinical impression were evaluated by the CGI-S scale. The number of patients in full-analysis set was 28. The results showed statistically significant improvement in CGI-S for both groups. Patients with vortioxetine monotherapy showed significant improvement in MADRS total score from the third week of treatment (p = 0.009) compared to patients with combined therapy that showed significant improvement since the end of first week of treatment (p = 0.036). Both groups showed significant improvement in HAM-A total score from the second week of treatment. Our results show the possibility of olanzapine in the augmentation strategy in treatment of major depressive disorder in adult patients.