RESUMO
Introduction: Difelikefalin is to date the first and only specific treatment to be approved for the treatment of moderate-to-severe chronic kidney disease-associated pruritus (CKD-aP) in adult patients on hemodialysis. Patients and methods: This was a retrospective, single-center, real-life study in hemodialysis patients with CKD-aP treated with difelikefalin. The primary objective was to evaluate the evolution of the intensity of pruritus during treatment with difelikefalin using the Worst Itch Intensity-Numerical Rating Scale (WI-NRS). Adult patients were included if they had been on hemodialysis for at least 3 months and were suffering from moderate to severe CKD-aP (objectified by the WI-NRS score) for which difelikefalin had been prescribed. Results: 11 patients (7 men and 4 women; mean age, 63.8 years) with a mean (SD) weekly dialysis time of 13 h (2.4) were included. The mean hemodialysis duration was 5 (3.6) years and the mean pruritus duration was 4.3 (3.2) years. At inclusion, on-going treatments of CKD-aP were emollients in all patients and antihistamines in 9 patients. The mean WI-NRS score was 7.4 (1.1) at initiation of difelikefalin. At last assessment after a median follow-up of 9.0 months, the mean change of WI-NRS score was -5.1 (2.9) and 82% of patients had a decrease ≥ 3 points. Mild to moderate adverse reactions to difelikefalin were reported in 4 patients, all of whom recovered without sequelae. Conclusion: These results show that difelikefalin, prescribed according to its therapeutic indication, is effective in the treatment of CKD-aP under real-life conditions, outside the controlled conditions of a clinical trial.
Introduction: La difélikéfaline est à ce jour le premier et le seul traitement spécifique approuvé pour le traitement du prurit d'intensité modérée à sévère associé à la maladie rénale chronique (Pa-MRC) chez les patients adultes hémodialysés. Patients et méthodes: Il s'agit d'une étude rétrospective, monocentrique, en vie réelle, chez des patients hémodialysés souffrant de Pa-MRC et traités par difélikéfaline. L'objectif principal était d'évaluer l'évolution de l'intensité du prurit au cours du suivi à l'aide de l'échelle WI-NRS (Worst Itch Intensity-Numerical Rating Scale). Les patients adultes ont été inclus s'ils étaient hémodialysés depuis au moins trois mois et souffraient d'un Pa-MRC modéré à sévère (objectivé par le score WI-NRS) pour lequel la difélikéfaline avait été prescrite. Résultats: Onze patients (7 hommes et 4 femmes ; âge moyen : 63,8 ans) avec un temps de dialyse hebdomadaire moyen (SD) de 13 h (2,4) ont été inclus. La durée moyenne d'hémodialyse était de 5 ans (3,6) et la durée moyenne de prurit de 4,3 ans (3,2). À l'inclusion, les traitements du prurit en cours étaient des émollients pour tous les patients et des antihistaminiques pour 9 d'entre eux. Le score WI-NRS moyen était de 7,4 (1,1) au début du traitement par la difélikéfaline. À la dernière évaluation, après un suivi médian de 9 mois, la variation moyenne du score WI-NRS était de -5,1 (2,9) et 82 % des patients avaient une diminution ≥ 3 points. Des effets indésirables d'intensité légère à modérée liés à la difélikéfaline ont été rapportés chez 4 patients, tous rétablis sans séquelles. Conclusion: Ces résultats montrent que la difélikéfaline, prescrite conformément à son indication thérapeutique, est efficace en vie réelle dans le traitement du Pa-MRC, en dehors des conditions contrôlées d'un essai clinique.
RESUMO
Thrombotic microangiopathies (TMA) are usually associated with hematological features (RH-TMA). The epidemiology of TMA limited to kidneys (RL-TMA) is unclear Therefore, patients with TMA and native kidney biopsies were identified during 2009-2022 in 20 French hospitals and results evaluated. RL-TMA was present in 341/757 (45%) patients and associated with lower creatinine levels (median 184 vs 346 µmol/L) than RH-TMA. RL-TMA resulted from virtually all identified causes, more frequently from anti-VEGF treatment and hematological malignancies but less frequently from shigatoxin-associated hemolytic uremic syndrome (HUS), systemic sclerosis, gemcitabine and bacterial infection, and even less frequently when three or more causes/triggers were combined (RL-TMA: 5%; RH-TMA: 12%). RL-TMA was associated with significantly lower major cardiovascular events (10% vs 20%), kidney replacement therapy (23% vs 43%) and death (12% vs 20%) than RH-TMA during follow-up (median 28 months). Atypical HUS (aHUS) was found in 326 patients (RL-TMA: 43%, RH-TMA: 44%). Among the 69 patients with proven complement-mediated aHUS, eculizumab (anti-C5 therapy) was used in 43 (62%) (RL-TMA: 35%; RH-TMA: 71%). Among the 257 other patients with aHUS, including 51% with RL-TMA, eculizumab was used in 29 but with unclear effects of this treatment. Thus, RL-TMA represents a very high proportion of patients with TMA and results from virtually all known causes of TMA and includes 25% of patients with complement-mediated aHUS. Adverse outcomes of RL-TMA are lower compared to RH-TMA but remain significant. Anti-C5 therapy was rarely used in RL-TMA, even in proven complement-mediated aHUS, and its effects remain to be assessed.
Assuntos
Síndrome Hemolítico-Urêmica Atípica , Microangiopatias Trombóticas , Adulto , Humanos , Rim/patologia , Microangiopatias Trombóticas/epidemiologia , Microangiopatias Trombóticas/terapia , Microangiopatias Trombóticas/patologia , Síndrome Hemolítico-Urêmica Atípica/tratamento farmacológico , Síndrome Hemolítico-Urêmica Atípica/epidemiologia , Proteínas do Sistema Complemento , Testes de Função RenalRESUMO
Kidney Biopsy (KB) is a crucial diagnostic tool in the field of renal diseases and is routinely performed in nephrology departments. A previous survey conducted by the Société Francophone de Néphrologie Dialyse Transplantation (SFNDT) revealed significant disparities in clinical practices, sometimes conflicting with the existing literature and recently published recommendations. In response, the SFNDT wished to promote the development of best practice guidelines, under the auspices of the French National Authority for Health (HAS), to establish a standardized framework for performing kidney biopsies in France.
La biopsie rénale (BR) est un outil diagnostique crucial dans le domaine des maladies rénales et est pratiquée en routine dans les services de néphrologie. Une précédente enquête menée par la Société francophone de néphrologie, dialyse et transplantation (SFNDT) a révélé d'importantes disparités dans les pratiques cliniques, parfois en contradiction avec la littérature existante et les recommandations récemment publiées. En réponse, la SFNDT a souhaité promouvoir l'élaboration de recommandations de bonnes pratiques, sous l'égide de la Haute Autorité de santé (HAS), afin d'établir un cadre standardisé pour la réalisation des biopsies rénales en France.