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A Review of Real-World Experience With Ruxolitinib for Myelofibrosis.
Clin Lymphoma Myeloma Leuk
December 2024
Incyte Corporation, Wilmington, DE.
Myelofibrosis (MF) is a rare myeloproliferative neoplasm characterized by progressive bone marrow fibrosis and splenomegaly. Ruxolitinib is the standard-of-care first-line treatment option for MF. This review summarizes real-world effectiveness and safety of ruxolitinib in more than 4500 patients with MF from real-world settings, including expanded-access and phase 4 trials, as well as registry, postmarketing, and retrospective studies in the 10 years since regulatory approval.
View Article and Find Full Text PDFPharmacokinetics of Zilurgisertib With and Without Food from Single and Multiple Ascending Dose Phase 1 Studies in Healthy Adults.
Eur J Drug Metab Pharmacokinet
December 2024
Department of Early Development, Incyte Corporation, Wilmington, DE, USA.
Background And Objectives: The oral, potent, and highly selective activin receptor-like kinase 2 (ALK2) inhibitor zilurgisertib (INCB000928) is in development as a treatment for fibrodysplasia ossificans progressiva (FOP), and for anemia due to myelofibrosis, myelodysplastic syndromes, and multiple myeloma. Saliva is an attractive alternative to blood for drug monitoring and pharmacokinetic analysis, as it is non-invasive to retrieve. This is beneficial for patients, such as those with FOP, for whom blood draws can be challenging due to soft tissue damage susceptibility that can cause progressive heterotopic ossification, and for whom tourniquet time and blood draws must be minimized.
View Article and Find Full Text PDFLongitudinal Assessment of Transfusion Intensity in Patients With JAK Inhibitor-Naive or -Experienced Myelofibrosis Treated With Momelotinib.
Clin Lymphoma Myeloma Leuk
October 2024
Washington University School of Medicine, St Louis, MO.
Purpose: Anemia is a cardinal feature of myelofibrosis often managed with red blood cell (RBC) transfusions, which may contribute to negative prognostic, quality-of-life, and healthcare-related economic impacts. The Janus kinase (JAK) 1/JAK2/activin A receptor type 1 inhibitor momelotinib was approved for the treatment of patients with myelofibrosis and anemia based on clinical trial evidence of anemia, spleen, and symptom benefits illustrated using binomial response/nonresponse endpoints. In the present post hoc, descriptive analyses, the impact of momelotinib on RBC transfusion burden over time was further characterized across JAK inhibitor-naive and -experienced patients.
View Article and Find Full Text PDFSOHO State of the Art Updates and Next Questions | Updates on Myelofibrosis With Cytopenia.
Clin Lymphoma Myeloma Leuk
September 2024
Department of Medicine, Mayo Clinic Arizona, Phoenix, AZ, USA. Electronic address:
Myelofibrosis (MF) is a rare hematologic malignancy that is characterized by dysregulation of the JAK-STAT pathway resulting in fibrosis of the bone marrow, splenomegaly, and abnormalities in peripheral blood counts including anemia, leukocytosis, and thrombocytopenia. This disease has 2 phenotypic extremes - myeloproliferative and cytopenic. Cytopenic myelofibrosis presents with pronounced cytopenia and a different landscape of genetic mutations which results in worse clinical outcomes and a poor prognosis.
View Article and Find Full Text PDFClinical features and treatment of newly diagnosed multiple myeloma with secondary myelofibrosis: a retrospective study.
Ther Adv Hematol
October 2024
Department of Hematology, The Affiliated Hospital of Qingdao University, 1677 Wutaishan Road, Qingdao, Shandong 225009, China.
Background: Secondary myelofibrosis (SMF) is characterized by the excessive deposition of fibrous tissue on top of the primary disease, often causing clinical manifestations to be overshadowed by the primary disease. Unfortunately, current staging systems do not incorporate myelofibrosis, leading to potential treatment delays for SMF.
Objectives: To evaluate the prognosis of patients with multiple myeloma (MM) complicated with myelofibrosis.
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