P2, N=30, Active, not recruiting, Memorial Sloan Kettering Cancer Center | Trial completion date: Feb 2026 --> Feb 2027 | Trial primary completion date: Feb 2026 --> Feb 2027
2 days ago
Trial completion date • Trial primary completion date
Treatment with ruxolitinib resulted in a marked improvement in splenomegaly and symptoms related to portal hypertension...This case represents a rare instance of PMF with FVL mutation encountered in Japan. In cases of atypical-site thrombosis, particularly among Caucasian patients, comprehensive evaluation including MPN driver mutations and other thrombophilic factors is essential to avoid underdiagnosis.
The patient was treated with subcutaneous immunoglobulin and ruxolitinib, and clinical exam remained stable at 2-year follow up. STAT3-GOF syndrome may present with bilateral optic disc edema, retinitis, and retinal vascular leakage in the setting of recurrent infections and polyendocrinopathy. This case illustrates the role of monogenic mutations in non-neoplastic autoimmune retinopathy and highlights the importance of multidisciplinary management including genetic consultation for accurate diagnosis and targeted immunomodulatory treatment.
3 days ago
Journal
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STAT3 (Signal Transducer And Activator Of Transcription 3)
STAT1, EGR1, FOXO1, and SMAD7 are associated with glycolytic-inflammatory crosstalk underlying PMF progression and ruxolitinib resistance, with experimental validation supporting STAT1 and EGR1 as potential diagnostic and therapeutic targets.
8 days ago
Journal
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STAT1 (Signal Transducer And Activator Of Transcription 1) • SMAD7 (SMAD Family Member 7) • EGR1 (Early Growth Response 1)
Treatment with intermittent phlebotomy and ruxolitinib led to hematologic improvement and complete normalization of serum sodium levels without the need for continued SIADH-specific therapy...Clinicians should consider MPNs in the differential diagnosis of unexplained SIADH. Further research is warranted to elucidate the underlying pathophysiological link.
Fedratinib is the second drug approved by the FDA for adult patients with myelofibrosis (MF) following ruxolitinib; however, the mechanism of its dose-limiting toxicity, particularly hepatotoxicity, remains poorly unclear. The results of risk assessment indicated that clinically relevant doses of fedratinib could significantly increase the area under curve (AUC) of drugs mainly metabolized by UGT1A1 and UGT1A3, suggesting a potential risk of clinically significant drug-drug interactions (DDIs). The current research provides useful information for the possible hepatotoxicity mechanism and clinical safe medication of fedratinib.
8 days ago
Journal
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UGT2B15 (UDP Glucuronosyltransferase Family 2 Member B15) • UGT1A3 (UDP Glucuronosyltransferase Family 1 Member A3)