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Factor X + Factor IX (Factor X P Behring)

✓ Approved

CSL Behring · F9 · 细胞治疗

什么是 Factor X + Factor IX？

Factor X + Factor IX 是一种细胞治疗，由CSL Behring研发。该药已获批，用于治疗相关适应症，给药途径：Injectable (Others)、Intravenous (IV)。

药物档案

商品名Factor X P Behring
公司CSL Behring
药物类别细胞治疗
分子靶点F9, F10
给药途径Injectable (Others), Intravenous (IV)
状态Approved
来源： ClinicalTrials.gov, CSL Behring

作用机制

分子靶点

Factor X + Factor IX 作用于 2 个分子靶点：

F9coagulation factor IX (P19, F9 p22)
F10coagulation factor X (FXA, FX)
需要更深入的分析？Noah AI 可解释复杂机制并与同类药物比较。

治疗适应症

Factor X + Factor IX 针对 2 个适应症，涉及 1 个治疗领域。

治疗领域疾病/病症分期
Congenital, familial and genetic disordersFactor IX deficiency✓ Approved
Congenital, familial and genetic disordersFactor X deficiency✓ Approved

相关研究文献

PubMedBlood coagulation & fibrinolysis : an international journal in haemostasis and thrombosis2026-07-27

Coagulation profiles and bleeding tendency in 12 patients with factor X deficiency: first report of a homozygous p.Gln249Pro mutation.

Wang Jianru J, Tang Ning N, Cao Weiliang W

To analyze the coagulation function and bleeding tendency in 12 patients with Factor X (FX) deficiency. Activated partial thromboplastin time (APTT), prothrombin time (PT), and FX activity (FX:C) were measured. F10 gene mutations were identified via Sanger sequencing. Clinical data and bleeding tendency were assessed using the ISTH-BAT score. Among the 12 patients, 10 had hereditary FX deficiency and 2 had acquired deficiency. Four patients with FX:C of 40-50% and heterozygous c.746A>C (p.Gln249Pro) mutations had no bleeding tendency. Five patients with F.X:C of 30-50% and heterozygous mutations (c.452G>A, c.871C>T, or c.1252G>A) showed mild bleeding. One patient with severe bleeding (FX:C <1.0%) had a homozygous c.746A>C (p.Gln249Pro) mutation - representing the first global report of this genotype. Two acquired cases had FX:C of 9% and 3%, with mild and moderate-severe bleeding, respectively. Severe bleeding in FX deficiency is associated with FX:C <5%, while milder deficiencies often present minimal symptoms. The novel homozygous p.Gln249Pro mutation causes a severe phenotype, highlighting the importance of genetic diagnosis.

PMID 42506896
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PubMedDiseases (Basel, Switzerland)2026-07-27

Hemophilia in Mexico: Updated Consensus Recommendations on Diagnosis, Treatment and Gene Therapy.

Alvarado Ibarra Martha M, Mera-González Alma B AB, Tapia-Enriquez Ana L AL, Ramirez-Hoyos Ana P AP et al.

Hemophilia is an X-linked inherited bleeding disorder, classified as type A or type B. Therapeutic advances offer new treatment options that improve disease control and reduce associated complications, including inhibitor development and hemophilic arthropathy. This document aims to update the Mexican hemophilia consensus, reviewing current evidence on diagnosis and management, and addressing gaps in the treatment and follow-up of patients in Mexico, aligning local needs with international recommendations. A PubMed literature search covering the last five years (up to September 2025) was conducted, prioritizing consensus statements, guidelines, and systematic reviews. Using the Delphi methodology, a structured questionnaire was submitted electronically to forty-four experts. Aspects without initial agreement were discussed at an in-person meeting. Consensus was defined as at least 80% of votes in favor. Recommendations were issued across six domains: laboratory diagnosis, genetic testing, management of hemophilia A and B without and with inhibitors, adjuvant treatments, and gene therapy. The recommendations address prophylaxis with coagulation factor concentrates, non-factor therapies, immune tolerance induction, perioperative management, pain management, and eligibility criteria and follow-up protocols for gene therapy with adeno-associated viral vectors. This consensus provides updated, evidence-based recommendations adapted to the Mexican healthcare context, identifying priority areas, including timely access to non-factor therapies and gene therapy, development of a national referral network for complex cases, and inclusion of novel therapeutic agents in the institutional essential medicines list, with the aim of improving the quality of life of people with hemophilia in Mexico.

PMID 42505587
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PubMedNursing reports (Pavia, Italy)2026-07-27

Adaptation and Validation of the Parental Stressor Scale: NICU for Spanish Populations.

Sánchez Regina Matey RM, Gómez Mónica Riaza MR, Reina Miguel A MA

Background/Objectives: Parental stress during neonatal intensive care unit (NICU) admission affects parental well-being, bonding, and neonatal outcomes. Reliable assessment requires instruments adapted to the linguistic and cultural context of each population. This study aimed to adapt the Parental Stressor Scale: NICU (PSS:NICU) for use in Spain and evaluate its psychometric properties. Methods: The adaptation comprised forward translation, back-translation, expert review, pilot testing with cognitive debriefing (n = 15), and psychometric evaluation. The adapted scale was administered to 160 parents (80 mothers, 80 fathers) of NICU-admitted neonates; 159 cases were retained for analysis. Internal consistency was assessed with Cronbach's alpha. Exploratory factor analysis (EFA; maximum likelihood, Promax rotation) with parallel analysis examined the factor structure. Confirmatory factor analysis (CFA) tested the original four-factor model, and a second-order model with five first-order factors was subsequently evaluated. Results: Internal consistency was excellent (total α = 0.968; subscales α = 0.866-0.961). Parallel analysis supported a five-factor EFA solution over a four-factor solution. CFA of the original four-factor model yielded poor fit (CFI = 0.755, TLI = 0.737, RMSEA = 0.125). A second-order model with five first-order factors and one general stress factor improved fit (CFI = 0.819, RMSEA = 0.107), though indices remained below conventional thresholds. Standardised factor loadings were moderate to high (0.57-0.96). Conclusions: The Spanish PSS:NICU demonstrates excellent reliability and provides preliminary evidence supporting its use for research and clinical assessment. The original four-factor structure was not replicated; the data suggest a five-factor organisation with evidence of a higher-order stress construct. Structural validity requires confirmation in larger, independent samples.

PMID 42506003
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PubMedJournal of trauma & dissociation : the official journal of the International Society for the Study of Dissociation (ISSD)2026-07-27

The Structure of Attachment Trauma: A Confirmatory Factor Analysis and Latent Profile Analysis on the Adult Attachment Trauma Questionnaire.

Zagaria Andrea A, Perinelli Enrico E, Lombardi Luigi L

The Adult Attachment Trauma Questionnaire (AATQ) is a newly developed measure to assess Attachment Trauma (AT) in adult romantic relationships. The current study builds upon the original validation study to further explore the psychometric properties of the AATQ in a general sample of 345 adults, using both variable-centered (Confirmatory Factor Analysis; CFA) and person-centered (Latent Profile Analysis; LPA) data analyses. The CFA revealed that the best model was a six-factor structure with a higher-order factor representing general AT. Invariance testing demonstrated that the AATQ is robust across sex, showing configural, metric, scalar, and strict invariance. The LPA identified three latent classes of AT (i.e. High, Medium, and Low). Discussions are provided on the correlates of the subscales. In summary, the AATQ has proven to possess robust psychometric properties.

PMID 42504866
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PubMedCells2026-07-27

Regenerative Potential of Autologously Processed White Adipose Tissue for Peripheral Nerve Regeneration: Evaluation of Growth Factor Profiles and Electrical Stimulation.

Egger Tobias T, Eigenberger Andreas A, Felthaus Oliver O, Ruewe Marc M et al.

Peripheral nerve injuries (PNI) present a major clinical and socioeconomic challenge due to limited regenerative capacity. Adipose-derived stem cells (ADSCs) within the stromal vascular fraction (SVF) of white adipose tissue offer a promising autologous source for regenerative support. This study evaluated the impact of mechanical processing CELT (Cell-Enriched Lipotransfer) and CELTPLUS and electrical stimulation on the regenerative secretome of human lipoaspirates. qPCR analysis revealed that CELTPLUS processing, which incorporates mechanical intersyringe shifting, significantly doubled the gene expression of nerve growth factor (NGF) (p = 0.015), vascular endothelial growth factor (VEGF) (p = 0.02), and brain-derived neurotrophic factor (BDNF) (p = 0.04) compared to CELT-processed lipoaspirate. Protein analysis via ELISA confirmed a time-dependent secretion of NGF and VEGF over 96 h. Furthermore, 24 h electrical stimulation (2 V) significantly enhanced NGF protein release (p < 0.001). These findings demonstrate that standardized mechanical processing effectively enriches regenerative cell populations and amplifies their neurogenic and angiogenic potential. The additional modulation of growth factor secretion via electrical stimulation highlights the potential of processed adipose tissue as a functional, autologous transplant for enhanced nerve reconstruction.

PMID 42505360
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PubMedHealth science reports2026-07-27

Prognostic Value of Cranial Nerve Invasion in T4-Stage Nasopharyngeal Carcinoma: A Retrospective Cohort Study With a Median Follow-Up of 136 Months.

Zhang Cuidai C, Liu Hui H, Liu Xuejia X, Ye Yihao Y et al.

The objective of this study was to evaluate the prognostic value of cranial nerve invasion (CNI) in T4-stage nasopharyngeal carcinoma (NPC) patients with nonmetastatic. We retrospectively analyzed 299 T4-stage NPC patients with nonmetastatic disease in the Cancer Hospital of Shantou University Medical College. The primary outcome was the overall survival (OS), and the secondary outcome was locoregional relapse-free survival (LRRFS). CNI was observed in 159 (53.2%) NPC patients with T4-stage disease. The mean follow-up period was 136 months (range, 3-168 months). Multivariate analyses confirmed that age, pericavernous extension, lower cervical lymph nodes metastasis, CN II and CN X invasion were significant for OS. Multivariate analyses confirmed that prevertebral muscle invasion (PMI) was significant for LRRFS, whereas CNI was not significant for LRRFS. In T4-stage NPC patients, significant differences were observed between the none and CNI groups in terms of the 5- and 10-year OS rates (70.7%, 61.9% vs. 61.0%, 51.4%; p = 0.03), respectively. No significant difference was observed between the none and CNI groups in terms of the 5- and 10-year LRRFS rates (87.4%, 78.1% vs. 88.1%, 79.8%; p = 0.71), respectively. In terms of OS, there were significant differences between the none and CN II, CN IX, CN X invasion, multiple CNI, unilateral CNI, and PMI groups, respectively (all p < 0.05). Significant differences were observed between the none and CN VII invasion and PMI groups in terms of LRRFS (both p < 0.05). CNI significantly affected T4-stage NPC patients' survival, although it had no discernible impact on local recurrence. This study elucidates the prognostic significance of CNI among patients with T4‑stage NPC, thereby enabling clinicians to precisely evaluate prognosis and implement optimal therapeutic interventions.

PMID 42504221
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