Docteur Patricia MORAN ALVAREZ

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Top publications · les plus citées

• 1

  Autologous CD19-Targeting CAR T Cells in a Patient With Refractory Juvenile Dermatomyositis

  Arthritis & rheumatology (Hoboken, N.J.) · 2024

  📚 51 citations🎯 RCR 8.63Top 3% NIH

  Lire l'abstract Crossref ↓

  ObjectiveThe aim of this study is to report the safety and efficacy of CD19‐targeting chimeric antigen receptor (CAR) T cells in a child with refractory juvenile dermatomyositis (JDM).MethodsWe describe a 12‐year‐old White male with severe, chronically active JDM refractory to multiple immunosuppressive treatment lines, including B cell depletion with rituximab. The patient received a single infusion of fresh, autologous, second‐generation anti‐CD19 CAR T cell product (lentiviral vector) manufactured on the Prodigy device (1 × 106 CAR T cells/kg), after lymphodepletion with cyclophosphamide (1,000 mg/m2 over two days) and fludarabine (90 mg/m2 over three days). Immunosuppressive and glucocorticoid treatment were withdrawn before leukapheresis and CAR T cell infusion.ResultsBefore anti‐CD19 CAR T cell therapy, the patient had persistent severe skin and muscular disease activity. CAR T cells expanded significantly (peak at day 7, 32.69 cells/μL). Complete B cell depletion was documented on day 5 in the blood and at week 2 in the bone marrow. The patient presented with fever as part of mild cytokine release syndrome (grade 1), transient anemia (grade 2), and neutropenia (grade 4). Neither infection nor neurotoxicity were observed. Laboratory tests, magnetic resonance imaging, Physician Global Assessment of disease activity, Childhood Myositis Assessment Scale, and Cutaneous Assessment Tool for myositis were performed at baseline and follow‐up to assess treatment response, showing remarkable progressive improvement that persists over time, even after B cell recovery.ConclusionThis patient with JDM with severe chronic disease, refractory to multiple treatments, achieved sustained B cell depletion and ongoing immunosuppressive drug‐free clinical and radiologic improvement eight months after a single infusion of anti‐CD19 CAR T cells.image

• 2

  Interface Gain-of-Function Mutations in TLR7 Cause Systemic and Neuro-inflammatory Disease

  Journal of clinical immunology · 2024

  📚 32 citations🎯 RCR 5.24Top 7% NIH🔓 Open Access📄 PDF gratuit ↗

  Lire l'abstract Crossref ↓

  AbstractTLR7 recognizes pathogen-derived single-stranded RNA (ssRNA), a function integral to the innate immune response to viral infection. Notably, TLR7 can also recognize self-derived ssRNA, with gain-of-function mutations in human TLR7 recently identified to cause both early-onset systemic lupus erythematosus (SLE) and neuromyelitis optica. Here, we describe two novel mutations in TLR7, F507S and L528I. While the L528I substitution arose de novo, the F507S mutation was present in three individuals from the same family, including a severely affected male, notably given that the TLR7 gene is situated on the X chromosome and that all other cases so far described have been female. The observation of mutations at residues 507 and 528 of TLR7 indicates the importance of the TLR7 dimerization interface in maintaining immune homeostasis, where we predict that altered homo-dimerization enhances TLR7 signaling. Finally, while mutations in TLR7 can result in SLE-like disease, our data suggest a broader phenotypic spectrum associated with TLR7 gain-of-function, including significant neurological involvement.

• 3

  Serological short-chain fatty acid and trimethylamine N-oxide microbial metabolite imbalances in young adults with acute myocardial infarction

  Heliyon · 2023

  📚 9 citations🎯 RCR 1.47🔓 Open Access📄 PDF gratuit ↗

Publications scientifiques (14) — classées par pathologie

Source PubMed · Recherche par auteur (homonymes possibles, vérifier l'affiliation).

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