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Fabien Grimaud,1 Guillaume Penaranda,2 Chloé Stavris,3 Frédérique Retornaz,3 Véronique Brunel,4 Sylvie Cailleres,4 Hervé Pegliasco,5 Jacques Le Treut,5 Vincent Grisoni,6 Emilie Coquet,1 Laurent Chiche,3 Amélie Rognon1 1Department of Pharmacy, Hôpital Européen, Marseille, France; 2Department of Biostatistics, Hôpital Européen, Marseille, France; 3Department of Internal Medicine, Hôpital Européen, Marseille, France; 4Department of Haemato-Oncology, Hôpital Européen, Marseille, France; 5Department of Pulmonology, Hôpital Européen, Marseille, France; 6Department of Urology, Hôpital Européen, Marseille, FranceCorrespondence: Amélie RognonDepartment of Pharmacy, Hôpital Européen, 6 Rue Désirée Clary, Marseille, 13003, FranceTel +33 4 13 42 73 00Fax +33 4 13 42 76 80Email a.rognon@hopital-europeen.frAim: To assess the efficacy and tolerance of programmed death-1 (PD-1) and PD-ligand 1 (PD-L1) inhibitors and the impact of a standardised management-based protocol in a real-world setting.Patients and Methods: Data from patients who had received anti-PD-(L)1 were collected from our pharmacy database. Clinical response and toxicity were assessed using RECIST criteria and CTCAE version 5.0, respectively. Overall survival (OS) and progression-free survival (PFS) were estimated with the Kaplan–Meier method. Potential prognostic factors were identified using Cox’s model.Results: A total of 196 patients and 201 lines of treatment were included (median age: 66 (range: 38– 89) years). Types of cancer included non-small cell lung cancer (73%), transitional cell carcinoma (10%), renal cell carcinoma (6%), small cell lung cancer (5%), head and neck squamous cell carcinoma (4%) and classical Hodgkin’s lymphoma (1%). Twenty-five (12%) patients had pre-existing autoimmune conditions. Our standardised management-based protocol included 129 (64%) patients. Objective response rate was 29%, median OS was 10 months (IQR: 7– 15) and median PFS was 5 months (IQR: 1– 22). Patients with an abnormal baseline complete blood count had a worse OS (HR=2.48 [95% CI: 1.24– 4.96]; p=0.0103). Thirty-three (16%) patients experienced severe (grade 3 or 4) immune-related adverse event (irAE). There were three (1%) irAE-related deaths. AEs resolved faster when patients were assessed by an internist before anti-PD-(L)1 initiation (p=0.0205).Conclusion: PD-1 and PD-L1 inhibitors are effective and safe in a real-world setting. Implementation of a standardised management-based protocol with internal medicine specialists is an effective way to optimise irAE management.Keywords: immunotherapy, elderly, PD-1 inhibitor, PD-L1 inhibitor, PDL-1 inhibitor, safety, immune-related adverse events, solid tumours, prognostic biomarkers

Pauline Sahuc,1 Laurent Chiche,2 Bertrand Dussol,3 Jean Pouget,1 Jérôme Franques1,2,4 1Department of Neurology, La Timone Hospital, APHM, Aix-Marseille University, 2Department of Internal Medicine, European Hospital, 3Department of Nephrology, La Conception Hospital, APHM, Aix-Marseille University, 4Department of Neurology, La Casamance Hospital, Marseille, France Abstract: Hypohidrosis is a frequent and early symptom in patients with Fabry disease. Studies have reported improved sweating in patients treated with enzyme-replacement therapy. A new method, Sudoscan, has been developed that is noninvasive, is quantitative, and can quickly evaluate sweat gland function. It is based on the electrochemical reaction between sweat chlorides and stainless-steel electrodes in contact with the palms and soles. The aim of our study was to evaluate the Sudoscan as a tool to assess sudomotor dysfunction in patients with Fabry disease. Consecutive patients were prospectively recruited who had a diagnosis of Fabry disease, which had been confirmed genetically and/or by measurement of α-galactosidase activity in leukocytes. Healthy controls, matched (1:1) for age and sex, were also enrolled. Test results were expressed immediately as electrochemical skin conductance (ESC, µS) for hands and feet. Sudomotor dysfunction was considered absent, moderate, or severe if the ESC measured on the feet was >60 µS, between 60 and 40 µS, or

Laurent Chiche,1 Alessandra Brescianini,1 Julien Mancini,2 Hervé Servy,3 Jean-Marc Durand11Service de Médecine Interne, Centre de Compétence pour la prise en charge des Cytopénies Auto-immunes, Hôpital de la Conception, Marseille, 2Service de Santé Publique, Hôpital de la Timone, Marseille, 3Association AIMSU, Maison des Associations, La Ciotat, FranceBackground: Patients with rare diseases often lack information about the disease itself and appropriate health care, leading to poor quality of life. Personal health records provide health information which can then be shared between multiple health care providers. Personal health records may also offer a tool for capturing patients' reported outcomes, thus enhancing their empowerment and improving communication with health care professionals. We conducted a pilot study to evaluate the usability of Sanoia, a freely accessible personal health record, which was customized for patients with the rare disease, idiopathic thrombocytopenic purpura (ITP).Methods: The Sanoia interface was expanded with ITP-specific tools. A prospective study was conducted at the referent center to evaluate the usability of this new interface (referred to here as the “tool”) by patients. Forty-three patients were randomized into groups to use or to not use the tool. Its use was evaluated by a specific questionnaire and by surveying individual patient adherence profiles. Evaluation of health-related quality of life using the ITP patient assessment questionnaire, was performed at baseline and after 1, 3, and 6 months via postal mail.Results: The groups were similar at inclusion in terms of characteristics, including global quality of life. During the study period, the tool was used to update the personal records of 19/28 patients (68%), with a median of two connections to the tool (range 1–12) plus access by various health care professionals (n = 22). In addition, 15/19 (78%) patients used the "personal notes" section at least once. We observed no significant changes in quality of life between patients with or without the tool during the study period.Conclusion: This pilot study demonstrates the good usability of the new customized Sanoia interface for patients with ITP. Additional studies will increase its usability further, and its interface could be adapted for use with other rare chronic diseases.Keywords: electronic personal health records, rare diseases, idiopathic thrombocytopenic purpura

Laurent Chiche1,2, Noémie Jourde3, Guillemette Thomas1, Nathalie Bardin2, Charleric Bornet4, Albert Darque4, Julien Mancini51Department of Internal Medicine, Centre de Compétence Maladies Auto-immunes Systémiques PACA Ouest, 2Laboratory of Immunology, 3Department of Nephrology, 4Department of Pharmacy, Hôpital de la Conception, Marseille; 5Department of Public Health, Hôpital de la Timone, Marseille, FranceAbstract: Belimumab is the first biologic approved for patients with systemic lupus erythematosus (SLE). Belimumab is the first of a new class of drug targeting B cell-stimulating factors or their receptors to reach the market. Its target, BLyS, also known as BAFF (B cell-activating factor from the tumor necrosis factor family), is a type II transmembrane protein that exists in both membrane-bound and soluble forms. Additionally to a robust rational from murine experiments conducted in lupus prone mice, BLyS circulating levels are increased in SLE patients. After the negative results of a Phase II trial, two Phase III trials met their primary endpoints. Some SLE patients are still refractory to the standard options of care or necessitate prolonged high-dose corticotherapy and/or long-term immunosuppressive regimens. However, some experts still feel that the effect of this biologic might not be clinically relevant and blame the use of the new systemic lupus response index as well as the discrepancies between both trials and the noninclusion of the severe form of the disease as nephritis. In this review, we aim to discuss the characteristics of belimumab, critically evaluate the different steps of its development, and consider its future place in the arsenal against SLE, taking into account the patients’ perspectives.Keywords: systemic lupus erythematosus, belimumab, treatment, monoclonal antibodies, adverse effects, BLyS