Jakafi (ruxolitinib)

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

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.

P3, N=105, Recruiting, Chengdu Zenitar Biomedical Technology Co., Ltd | Not yet recruiting --> Recruiting

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.

P1/2, N=33, Recruiting, M.D. Anderson Cancer Center | Active, not recruiting --> Recruiting

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.

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.

This review traces the 20-year journey of the statin-in-MPN concept, from its mechanistic rationale to the present, where a confluence of evidence from comorbid disease prevention and onco-immunology is supportive of their upfront use. I posit that we are at a watershed moment, where statins should be integrated into the upfront treatment of MPNs and in combination therapy strategies, particularly with IFN-α, and potentially with ruxolitinib and metformin, to extinguish the chronic inflammatory drive and fundamentally alter the natural history of these neoplasms.

P1, N=1, Terminated, Children's Hospital Medical Center, Cincinnati | N=30 --> 1 | Trial completion date: Jun 2028 --> Sep 2025 | Recruiting --> Terminated | Trial primary completion date: Jun 2028 --> Aug 2025; Study closed due to slow enrollment.

P1, N=140, Active, not recruiting, Incyte Corporation | Trial completion date: Dec 2026 --> Apr 2027 | Trial primary completion date: Dec 2025 --> Apr 2027

P2, N=7, Active, not recruiting, Children's Hospital Medical Center, Cincinnati | Recruiting --> Active, not recruiting | N=40 --> 7