Publications by authors named "Nathalie Parquet"

Systemic sclerosis (SSc) is a rare autoimmune disease (AD), characterised by early diffuse vasculopathy, activation of the immune response and progressive skin and internal organ fibrosis. In severe progressive diffuse SSc (dSSc), autologous hematopoietic stem cell transplantation (aHSCT) improves survival, despite its own risk of complications and transplant related mortality (TRM). We present herein the case of a dSSc patient undergoing aHSCT with low dose cyclophosphamide conditioning and sudden acute myopericarditis and cardiogenic shock, four weeks after a second mRNA SARS-CoV-2 vaccine (Pfizer) injection.

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  • The study compares the clinical characteristics of primary myelofibrosis (PMF) and secondary myelofibrosis (SMF), highlighting key differences in patient presentation and symptoms.
  • It explores the molecular landscape of both conditions, analyzing genetic mutations and other molecular factors that may influence the disease.
  • The research also focuses on prognosis scoring systems, assessing how well they predict outcomes and survival rates for patients with PMF and SMF.
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  • Current risk scores for thrombotic events in myeloproliferative neoplasms (MPN) fail to differentiate between arterial and venous thrombosis, even though they have different causes and implications.
  • A new score called ARTS, which considers factors like prior arterial thrombosis, age over 60, cardiovascular issues, and specific gene mutations, effectively stratifies patients into low- and high-risk groups for arterial thrombosis.
  • Conversely, the VEnous Thrombosis Score (VETS), which only looks at prior venous thrombosis and JAK2 mutations, does not perform well, highlighting the need for better venous risk assessments that address its complexity.
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Erythropoietic protoporphyria (EPP) is a rare metabolic disease of the heme biosynthetic pathway where an enzymatic dysfunction results in protoporphyrin IX (PPIX) accumulation in erythroid cells. The porphyrins are photo-reactive and are responsible for severe photosensitivity in patients, thus drastically decreasing their quality of life. The liver eliminates PPIX and as such, the main and rare complication of EPP is progressive cholestatic liver disease, which can lead to liver failure.

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  • Chimeric Antigen Receptor T cells (CAR-T) are a promising treatment for relapsed/refractory diffuse large B-cell lymphoma (DLBCL), but come with significant toxicities like cytokine release syndrome (CRS) and neurotoxicity.
  • A study compared patients who received early G-CSF (granulocyte-colony stimulating factor) after CAR-T infusion to those treated before this protocol; early administration significantly reduced febrile neutropenia rates without affecting the overall toxicity levels.
  • The effectiveness of CAR-T in terms of T-cell expansion, response rates, and survival remained similar between both patient groups, indicating that early G-CSF is a safe strategy for managing side effects while preserving treatment efficacy.
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  • GCT patients with brain metastases treated with high-dose chemotherapy were studied to evaluate their outcomes, revealing a low 2-year survival rate of 36.9% and a median overall survival of just 12 months.
  • Among the 35 patients, 11 responded positively to treatment, with most of these favorable responses seen in those with metachronous brain metastases, typically isolated and without prior local therapy.
  • The findings suggest that even GCT patients with brain metastases can achieve long-term survival, and certain biological responses may indicate better outcomes with high-dose chemotherapy.
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Tisagenlecleucel therapy has shown promising efficacy for relapsed/refractory (R/R) B-cell precursor acute lymphoblastic leukemia (BCP-ALL). However, relapses occur in 30-50% of patients. Determinants for CD19 versus CD19 relapses are poorly characterized.

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Chimeric antigen receptor (CAR) T-cells are a new class of cancer treatments manufactured through autologous or allogeneic T cells genetic engineering to induce CAR expression directed against a membrane antigen present at the surface of malignant cells. In Europe, tisagenlecleucel (Kymriah™) has a marketing authorization for the treatment of relapsed/refractory B-cell acute lymphoblastic leukemia in children and young adults and for the relapsed/refractory diffuse large B-cell lymphoma (DLBCL). The marketing authorization for axicabtagene ciloleucel (Yescarta™) is the treatment of relapsed/refractory DLBCL and mediastinal B-cell lymphoma.

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Background Aims: According to European Directive 2001/83/EC, chimeric antigen receptor T (CAR T) cells belong to a new class of medicines referred to as advanced therapy medicinal products (ATMPs). The specific features and complexity of these products require a total reorganization of the hospital circuit, from cell collection from the patient to administration of the final medicinal product. In France, at the cell stage, products are under the responsibility of a cell therapy unit (CTU) that controls, manipulates (if necessary) and ships cells to the manufacturing site.

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The extraordinary and unexpected success of cellular immunotherapy using genetically engineered T-cells to express a chimeric antigen receptor (CAR) targeting CD19, in the treatment of refractory or relapsing B-hematological malignancies, has provided a real therapeutic hope. Indeed, remission rates reach more than 80 % in patients at a stage, without any other possibilities of treatment, notably in the child's acute lymphoblastic leukemia. These results, initially resulting from academic research, led to Food and Drug accreditation for market access of two innovative autologous therapy drugs, Kimryah® and Yescarta®.

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Although the risk of developing lymphoma has decreased in the highly active antiretroviral therapy era, this cancer remains the major cause of mortality in HIV-infected patients. Autologous hematopoietic stem cell transplantation (ASCT) outcome does not differ for HIV-infected versus HIV-uninfected patients. We propose to develop a new treatment for HIV-associated high-risk lymphoma based on autologous transplantation of two genetically modified products: CD4 T lymphocytes and CD34 hematopoietic stem cells (HSPCs).

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The Francophone society of bone marrow transplantation and cellular therapy (SFGM-TC) organized the 7th allogeneic hematopoietic stem cell transplantation clinical practices harmonization workshop series in September 2016 in Lille, France. The objective of our workshop is to discuss chronic graft versus host disease and to provide recommendations for the indications and treatment of this condition.

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Background: Data on plasma exchange therapy in the intensive care unit (ICU) setting are scarce. We aimed to describe the technical aspects and the adverse events associated with the procedure in critically ill patients.

Methods: All adult patients treated by plasma exchange in the medical ICU of the Saint-Louis university hospital between January 1, 2013 and March 31, 2015 were prospectively included.

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Optic neuritis could lead to severe visual impairment despite corticosteroids. Our aim was to evaluate the rate of visual improvement in patients treated with plasma exchange (PLEX) for severe steroid unresponsive optic neuritis and to identify predictive factors of outcome. Thirty-four patients (41 optic nerves damaged) with remaining visual acuity of 0.

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Interleukin 10 (IL-10)-producing B cells (regulatory B cells [Bregs]) regulate autoimmunity in mice and humans, and a regulatory role of IL-10-producing plasma cells has been described in mice. Dysfunction of B cells that maintain homeostasis may play a role in the pathogenesis of chronic graft-versus-host disease (cGVHD) after allogeneic stem cell transplantation. Here, we found a relation between decreased Breg frequencies and cGVHD severity.

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