📇 Rhumatologue · DRAVEIL · (91) · Libéral

MME Yen Thi NGUYEN-ARNAUD

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Auteur de référence en rhumatologie
38 articles scientifiques publiés — un praticien à la pointe de la recherche
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Rhumatologue

📍 DRAVEIL (91)LibéralRPPS 10001561876

✨ Génération du profil synthétique IA en cours…

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Top publications · les plus citées

Low flow cytometry events in detecting CNS involvement in pediatric B-cell precursor ALL: NGS validation and prognostic significance

Blood advances · 2026

📚 1 citations🔓 Open Access📄 PDF gratuit ↗

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Abstract Central nervous system (CNS) status in pediatric acute lymphoblastic leukemia (ALL) is a key prognostic factor that makes precise detection critical. Flow cytometry (FCM) offers a higher detection rate than conventional cytomorphology (CC); however, results are considered negative with &lt;10 FCM events, limiting its sensitivity. This study analyzed the cerebrospinal fluid from 173 pediatric patients with ALL using both CC and FCM, characterizing immunoglobulin heavy chain rearrangement clonotypes using next-generation sequencing (NGS). The sensitivities of CC and FCM were similar (P = .824) when the standard FCM positivity threshold was used. Receiver operating characteristic analysis based on NGS showed ≥3 events as the optimal FCM positivity cutoff, significantly improving sensitivity over CC (P = .005). Among patients treated according to the Taiwan Pediatric Oncology Group 2013 ALL protocol, FCM positivity correlated with lower event-free survival (EFS) and higher cumulative incidence of CNS relapse (CIRCNS). Patients with hyperdiploidy who were CC-negative/FCM-positive had worse EFS and CIRCNS when receiving less intensive CNS-directed treatments. The 3-event cutoff for FCM positivity enhances the detection of CNS involvement in pediatric ALL and, when combined with CC, identifies patients at a higher risk of CNS relapse, highlighting the potential need for treatment intensification.

Diazoxide Choline Extended-release Tablets in Prader-Willi Syndrome: A Randomized, Double-blind, Withdrawal Period Study

The Journal of clinical endocrinology and metabolism · 2026

📚 1 citations🔓 Open Access

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Abstract Context The hallmark condition of Prader-Willi syndrome, a rare, genetic neurobehavioral/metabolic disorder, is life-threatening hyperphagia. Objective We assessed the efficacy and safety of diazoxide choline extended-release (DCCR) tablets for the treatment of hyperphagia in adults and children four years of age and older with Prader-Willi syndrome. Methods We conducted a 16-week, randomized withdrawal study in children and adults with Prader-Willi syndrome and hyperphagia. Participants who previously completed randomized (13-week DCCR or placebo) and open-label (2.5-4.5 years DCCR) studies were randomized 1:1 to receive once-daily DCCR or placebo. The primary endpoint was Hyperphagia Questionnaire for Clinical Trials (HQ-CT) total score change from baseline to 16 weeks. Secondary endpoints included Clinical Global Impression of Severity (CGI-S) and Improvement (CGI-I); exploratory endpoints included weight and body mass index (BMI) z-score. Results Seventy-seven participants were randomized (DCCR:38; placebo:39). Statistically significant increases in HQ-CT from baseline to week 16 were observed with placebo vs DCCR [least square (LS) mean (standard error) change 7.6 (1.09)] with placebo and 2.6 (1.12) with DCCR; P = .0022. CGI scores favored DCCR but were not significantly changed. Consistent with the hyperphagia response, the placebo cohort gained more weight and increased their BMI z-score more than the DCCR cohort [LS mean weight difference (95% confidence interval) −1.6 kg (−3.1, −0.1)]; LS mean z-score difference was −0.09 (−0.17, −0.01). Adverse events were similar with both treatments, with no serious adverse events in the DCCR arm. Conclusion Continued DCCR treatment was superior to placebo for hyperphagia. DCCR appears to offer meaningful therapeutic benefits for people with Prader-Willi syndrome.

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MME Yen Thi NGUYEN-ARNAUD

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Top publications · les plus citées

Publications scientifiques (38) — classées par pathologie

Transversal22

Revue générale4

Qualité de vie / PROMs3

Épidémiologie & registres2

Case report / série1

Économie santé1

Essai clinique1

Ostéoporose1

Pédiatrie1

Revue / méta-analyse1

Santé mentale / fatigue1

Vraie vie / RWE1

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