Paediatric Behçet’s Disease: Data From A Single Center Experience In Turkey

ABSTRACT Objectives: Behçet’s disease (BD) is a multisystemic inflammatory disease with unknown etiology. It is characterized by recurrent oral and genital ulcerations, uveitis, and skin lesions, various musculoskeletal, gastrointestinal, central nervous system, and vascular manifestations. The aim of this study was to analyse the demographic characteristics and clinical features, treatment in Turkish paediatric BD from a single center experience.   Methods: The records 36 patients with BD who were diagnosed according to the International Study Group criteria between January 2017 and January 2019 in the department of paediatric rheumatology, were retrospectively reviewed. Data on demographic, clinical features and therapy were collected. Results: A total of 36 (19 male) patients were included in this study. Mean age at disease onset was 9.36 ± 4.45 years and mean age at diagnosis 13.99 ± 2.83 years. The frequencies of signs/symptoms were: recurrent oral aphtosis 100%, genital ulcers 80.6%, musculoskeletal 30.6%, ocular 16.7%, neurological 11.1% and vascular involvement 11.1%, gastrointestinal 2.8%. Colchicine and corticosteroids were the main treatments. Conclusions: In this single-center retrospective study, we analyzed the data of paediatric BD and their treatment from a single center in Turkey. The presented small series and the literature review suggest that paediatric BD is a heterogeneous disease with varied clinical manifestations

Lorlatinib in ALK- or ROS1-positive non-small cell lung cancer patients: Experience from an early access program in Turkey

Background: Lorlatinib, a third generation ALK and ROS1 inhibitor, is indicated for the treatment of patients with ALK+ metastatic NSCLC whose disease has progressed on crizotinib and at least one second-generation ALK inhibitor. The aim of this study is to evaluate the efficacy and safety of lorlatinib in an Expanded Access Program (EAP) in Turkey. Method: The EAP was open-label, multicenter, and single-arm. Patients were eligible to receive lorlatinib (100 mg po/day) if they had advanced stage ALK-or ROS1-positive NSCLC and had progressed on crizotinib and/or second generation ALK inhibitors such as ceritinib or alectinib. The primary endpoint was PFS with lorlatinib. Secondary endpoints were objective response rate, overall survival, and safety

Glass: Global lorlatinib for ALK(+) and ROS1(+) retrospective study: Real world data of 123 NSCLC patients

Lorlatinib is a third-generation tyrosine-kinases inhibitor (TKI) targeting ALK/ROS1 fusions. The FDA has approved lorlatinib for TKI-pretreated ALK(+) NSCLC, while its approval for ROS1( + ) is still pending. Here we present the largest real-world data of NSCLC patients harboring ALK/ROS1 rearrangements treated with lorlatinib.Methods: 123 patients were enrolled retrospectively (data cut-off 1/1/2019). Lorlatinib was administered through an early access program for patients with no other available therapy. Outcome and response were defined by each investigator upon RECIST 1.1 criteria.Results: 106 ALK(+) and 17 ROS1(+) patients recruited from 8 different countries. The ALK( + ) cohort included 50 % males, 73 % never-smokers and 68 % with brain metastases. Extracranial (EC) and intracranial (IC) response rates (RR) were 60 % and 62 %, with disease control rates (DCR) of 91 % and 88 % respectively. Mean duration of therapy (DoT) was 23.9 +/- 1.6 months and median overall survival (mOS) was 89.1 +/- 19.6 months. ROS1 cohort enrolled 53 % males, 65 % never-smokers and 65 % had brain metastases. EC and IC RR were 62 % and 67 % with DCR of 92 % and 78 % respectively. Median DoT was 18.1 +/- 2.5 months and mOS of 90.3 +/- 24.4 months. OS and DoT in both cohorts were not significantly correlated with line of therapy nor other parameters.The most common adverse events of any grade were peripheral edema (48 %), hyperlipidemia (47 %), weight gain (25 %) and fatigue (30 %). CNS adverse events such as cognitive effect of grade 1-2 were reported in 18 % of patients.Conclusion: Lorlatinib shows outstanding EC/IC efficacy in ALK/ROS1(+) NSCLC. The observed mOS of 89 +/- 19 months in ALK(+) NSCLC supports previous reports, while mOS from of 90 +/- 24 months is unprecedented for ROS1( + ) NSCLC.Pfize

Glass: Global lorlatinib for ALK(+) and ROS1(+) retrospective study: Real world data of 123 NSCLC patients

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