Argumentaire pour une utilisation plus large de la photochimiothérapie extracorporelle chez l’enfant

International audienceThe management of immune diseases in children remains challenging , although significant advances have been made. In addition to pharmacological approaches, extracorporeal photochemotherapy (ECP) is distinctive in its ability to provide immunomodulation without immune suppression or toxicity. However, in practice, this therapy is not widely used because of logistical issues and the lack of robust clinical pediatric studies. Here, we discuss the potential clinical applications of ECP in children and emphasize the need for a rigorous and specifically pediatric clinical evaluation of ECP. ß 2010 Elsevier Masson SAS. All rights reserved. Résumé Malgré l'apport des biothérapies, le traitement des maladies dysim-munitaires sévères et des conflits allogéniques de l'enfant reste difficile et entaché de nombreuses complications. Dans ce contexte, la photo-chimiothérapie extracorporelle (PCE) (thérapie cellulaire qui repose sur l'effet immunomodulateur des cellules mononucléées du patient, prélevées par aphérèse et exposées ex vivo aux rayons ultraviolets A [UVA] en présence de psoralène) a l'avantage notable d'induire une tolérance immunitaire sans générer d'immunosuppression systémique ni de toxicité a ` court, moyen ou long terme. Cette immunomodulation fait intervenir notamment la génération de lymphocytes T régulateurs (T reg). Malgré cela, la PCE est peu utilisée en raison de ses contraintes logistiques et du manque de données cliniques. Nous proposons une revue des indications reconnues et potentielles de la PCE en pédiatrie. Nous insistons sur la nécessité d'une e ´valuation clinique spécifique a ` l'enfant qui ne peut se concevoir sans la participation active des cliniciens pédiatres en particulier dans le domaine de la transplanta-tion et des maladies auto-immunes et inflammatoires

A chemogenomic approach to identify personalized therapy for patients with relapse or refractory acute myeloid leukemia: results of a prospective feasibility study

International audienceTargeted next-generation sequencing (tNGS) and ex vivo drug sensitivity/resistance profiling (DSRP) have laid foundations defining the functional genomic landscape of acute myeloid leukemia (AML) and premises of personalized medicine to guide treatment options for patients with aggressive and/or chemorefractory hematological malignancies. Here, we have assessed the feasibility of a tailored treatment strategy (TTS) guided by systematic parallel ex vivo DSRP and tNGS for patients with relapsed/refractory AML (number NCT02619071). A TTS issued by an institutional personalized committee could be achieved for 47/55 included patients (85%), 5 based on tNGS only, 6 on DSRP only, while 36 could be proposed on the basis of both, yielding more options and a better rationale. The TSS was available in <21 days for 28 patients (58.3%). On average, 3 to 4 potentially active drugs were selected per patient with only five patient samples being resistant to the entire drug panel. Seventeen patients received a TTS-guided treatment, resulting in four complete remissions, one partial remission, and five decreased peripheral blast counts. Our results show that chemogenomic combining tNGS with DSRP to determine a TTS is a promising approach to propose patient-specific treatment options within 21 days

Actualisation des recommandations françaises de traitement du pemphigus

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Epidemiological changes in cutaneous lymphomas: an analysis of 8593 patients from the French Cutaneous Lymphoma Registry

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