预约演示

更新于：2025-05-07

Neutropenia, Nonimmune Chronic Idiopathic, Adult

成人非免疫性慢性特发性中性粒细胞减少症

更新于：2025-05-07

基本信息

别名

NEUTROPENIA, NONIMMUNE CHRONIC IDIOPATHIC, OF ADULTS、NI-CINA、NONIMMUNE CHRONIC IDIOPATHIC NEUTROPENIA OF ADULTS

+ [3]

简介-

关联

项与成人非免疫性慢性特发性中性粒细胞减少症相关的药物

Mavorixafor

靶点

CXCR4

作用机制

CXCR4拮抗剂

在研机构

X4 Pharmaceuticals, Inc. [+2]

原研机构

Genzyme Corp.

在研适应症

WHIM综合征 [+6]

非在研适应症

晚期肾细胞癌 [+4]

最高研发阶段批准上市

首次获批国家/地区

美国

首次获批日期2024-04-26

项与成人非免疫性慢性特发性中性粒细胞减少症相关的临床试验

NCT04154488

/ Completed临床1/2期

A Phase 1b/2, Open-Label, Multicenter Study of Mavorixafor in Patients With Congenital Neutropenia and Chronic Neutropenia Disorders

This is a 2-part study of mavorixafor in participants diagnosed with chronic neutropenia. The main goal of Part 1 (Phase 1b) is to help researchers learn more about how the investigational medicine, mavorixafor, impacts people living with chronic neutropenia (including congenital, idiopathic, and cyclic). In Part 2 (Phase 2), the safety and tolerability of chronic dosing of mavorixafor will be evaluated in a larger participant population and the impact of 6-month chronic dosing of mavorixafor on participant neutropenia.

开始日期2021-10-16

申办/合作机构

X4 Pharmaceuticals, Inc.

100 项与成人非免疫性慢性特发性中性粒细胞减少症相关的临床结果

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100 项与成人非免疫性慢性特发性中性粒细胞减少症相关的转化医学

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0 项与成人非免疫性慢性特发性中性粒细胞减少症相关的专利（医药）

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项与成人非免疫性慢性特发性中性粒细胞减少症相关的文献（医药）

2025-04-01·Fish Physiology and Biochemistry

The ability of dietary essential oils to mitigate nickel-induced growth retardation, immune-antioxidant suppression, and endoplasmic reticulum stress activation in Nile tilapia

Article

作者: Aly, Mohamed Y M ; Ghannam, Hala Elshahat ; Mahsoub, Fatma ; Abdo, Samar A ; Khamis, Tarek ; Ahmed, Shaimaa A A ; Ibrahim, Rowida E ; El-Rahman, Ghada I Abd ; Mohammed, Haiam A

AbstractFish immunity is significantly impacted by waterborne metal intoxication. Nickel is a ubiquitous metal in aquatic bodies which badly impacts fish immune responses and survival. In the current research, we looked into the possible protective effects of essential oils of tea tree (TTO) and basil (BEO) against nickel exposure in Nile tilapia, or Oreochromis niloticus. To achieve this, 240 Nile tilapia (27.92 ± 0.22 g) were categorized into six groups for 45 days; three groups were fed on basal control, TTO, and BEO diets without Ni exposure. The other three groups were subjected to Ni at a level of 3.6 mg/L and fed on basal, TTO, and BEO diets. The outcomes showed a substantial decrease in the growth measures and survival % in the Ni-exposed fish. Oxidative stress (higher splenic malondialdehyde and lower splenic catalase, reduced glutathione, and superoxide dismutase), immune suppression (lower serum lysozyme, myeloperoxidase, and nitric oxide), and elevated serum cortisol levels were observed in the Ni-intoxicated group. The Ni-intoxicated group exhibited various pathological alterations detected in the intestinal tissue mainly inflammatory and necrotic changes, moreover, moderate depletion of lymphoid elements represented by necrotic lymphocytes in the splenic tissue. Ni-exposure induced endoplasmic reticulum stress through up-regulation of the splenic expression of protein kinase R-like endoplasmic reticulum kinase, activating transcription factor 6, CCAAT/enhancer-binding protein homologous protein, X-box binding protein 1, α-subunit of eukaryotic initiation factor 2, inositol-requiring kinase 1a, mitogen-activated protein kinase, c-JunN-terminal kinase, and binding protein for immunoglobulins. Feeding on the TTO and BEO diets improved the growth measures, survival, and antioxidant capacity. Immunomodulation, enhancement of the intestinal and splenic architecture, and relief of the endoplasmic reticulum stress condition were noticed when the Ni-intoxicant groups were fed on TTO and BEO diets. Overall, the effect of the TTO diet was more pronounced than the BEO diet in mitigating the negative consequences of Ni-intoxication in Nile tilapia. Dietary fortification of 0.1% TTO and/or 0.1% BEO is recommended during Ni exposure in aquaculture practices.

2024-08-01·Annals of the Rheumatic Diseases

Altered serum metabolome as an indicator of paraneoplasia or concomitant cancer in patients with rheumatic disease

Article

作者: Carvalho, Rui A ; Christopoulos, Petros ; Luft, Thomas ; Hassel, Jessica C ; Graf, Jürgen ; Feisst, Manuel ; Marx, Dorothea ; Souto-Carneiro, M Margarida ; Klika, Karel D ; Gente, Karolina ; Will, Julia ; Lorenz, Hanns-Martin ; Müller-Tidow, Carsten

ObjectivesA timely diagnosis is imperative for curing cancer. However, in patients with rheumatic musculoskeletal diseases (RMDs) or paraneoplastic syndromes, misleading symptoms frequently delay cancer diagnosis. As metabolic remodelling characterises both cancer and RMD, we analysed if a metabolic signature can indicate paraneoplasia (PN) or reveal concomitant cancer in patients with RMD.MethodsMetabolic alterations in the sera of rheumatoid arthritis (RA) patients with (n=56) or without (n=52) a history of invasive cancer were quantified by nuclear magnetic resonance analysis. Metabolites indicative of cancer were determined by multivariable regression analyses. Two independent RA and spondyloarthritis (SpA) cohorts with or without a history of invasive cancer were used for blinded validation. Samples from patients with active cancer or cancer treatment, pulmonary and lymphoid type cancers, paraneoplastic syndromes, non-invasive (NI) precancerous lesions and non-melanoma skin cancer and systemic lupus erythematosus and samples prior to the development of malignancy were used to test the model performance.ResultsBased on the concentrations of acetate, creatine, glycine, formate and the lipid ratio L1/L6, a diagnostic model yielded a high sensitivity and specificity for cancer diagnosis with AUC=0.995 in the model cohort, AUC=0.940 in the blinded RA validation cohort and AUC=0.928 in the mixed RA/SpA cohort. It was equally capable of identifying cancer in patients with PN. The model was insensitive to common demographic or clinical confounders or the presence of NI malignancy like non-melanoma skin cancer.ConclusionsThis new set of metabolic markers reliably predicts the presence of cancer in arthritis or PN patients with high sensitivity and specificity and has the potential to facilitate a rapid and correct diagnosis of malignancy.

2023-01-01·Frontiers in nutrition

Neurological impairment and malnutrition in children: The role of home enteral nutrition in real life.

Article

作者: Mosca, Antonella ; Papa, Raffaele Edo ; De Angelis, Paola ; Mazzoli, Bianca ; Bolasco, Giulia ; Cerchiari, Antonella ; Maggiore, Giuseppe ; Caldaro, Tamara ; Trovato, Chiara Maria ; Staccioli, Susanna ; Capriati, Teresa ; De Peppo, Francesco ; Laureti, Francesca ; Diamanti, Antonella

ObjectiveRecent decades have brought an increased survival of children with Neurologic Impairment (NI) but malnutrition and digestive comorbidity remain important challenges to face. We designed the present study to assess the course of nutritional status following standardized Home Enteral Nutrition (HEN) program and to evaluate impact of changing mode of feeding, as a part of overall multidisciplinary management, on digestive co-morbidity as Gastro-Esophageal Reflux Disease (GERD), Oropharyngeal Dysphagia (OPD), constipation and airway aspiration.MethodsWe performed a retrospective analysis on NI children entered into Institutional HEN program due to NI disorders between January 2011 and 2019. Demographic, anthropometric characteristics (BMI z-score and weight for age z-score) and symptoms (GERD, OPD constipation and airway aspiration) were collected at the enrolment and during the follow up.ResultsWe enrolled 402 patients (median age: 39 months); overall survival was 97%. Nutritional status was significantly improved by HEN; in particular growth profile significantly changed within the first 2 years following HEN beginning; GERD and airways aspirations decreased after HEN beginning. Constipation and OPD remained unchanged over time.ConclusionsMalnutrition and digestive complaints are distinctive features of NI children. Nutritional status improve after 2 years from the beginning of standardized nutritional interventions. Overall multidisciplinary care, including standardized HEN protocols, seems to also impact on GERD and airway aspirations, which can decrease over time. It is possible that constipation and OPD, unchanged over time, are more dependent on underlying diseases than on overall treatments.

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