Descriptive, Comparative Study of Filipino Myelofibrosis Patients: An Agematched Comparison of Patients on Ruxolitinib vs. Best Available Therapy
Autor: | Honorata Giongco Baylon, Flordeluna Zapata-Mesin, Susie L. Ponce, Narcisa Sonia Cornejo Comia, Priscilla B. Caguioa, Catherine C. Rosales, Ma Rosario Irene D. Castillo |
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Rok vydání: | 2014 |
Předmět: |
medicine.medical_specialty
Ruxolitinib Anemia business.industry Spleen International working group medicine.disease Bioinformatics Extramedullary hematopoiesis medicine.anatomical_structure Internal medicine medicine Hepatic dysfunction business Myelofibrosis Myeloproliferative neoplasm medicine.drug |
Zdroj: | Journal of Hematology & Thromboembolic Diseases. |
ISSN: | 2329-8790 |
DOI: | 10.4172/2329-8790.1000178 |
Popis: | Introduction: Myelofibrosis is characterized by bone marrow fibrosis, cytopenias and extramedullary hematopoiesis. Ruxolitinib is a Janus Kinase-2 inhibitor recently approved for myelofibrosis. In the Philippines, it was made available through the compassionate use program. Methods: This is a multicentre, retrospective case series of eleven patients in the compassionate use program and eleven age-matched historical controls on best available therapy. Results: The median age was 54.5+7.33, 82% females. Using the International Working Group on Myelofibrosis Research and Treatment (IWG-MRT) consensus criteria of 2013, 1 out of the 11 patients had both spleen response and anemia response, achieving transfusion independence for > 12 weeks. Nine patients experienced clinical improvement specifically spleen response. Decrease in spleen size was seen in the first 3 months of ruxolitinib use with a mean of 53.5% from baseline. Hematologic toxicities were anemia and thrombocytopenia while nonhematologic were gastrointestinal disorders 5(45%) and hepatic dysfunction 4(36%). These patients were matched by age with historical controls on best available therapy. There was a remarkable decrease in spleen size with ruxolitinib (53.5%) compared to BAT group (18% from baseline). Thrombocytopenia and anemia occurred more frequently in the ruxolitinib group. Conclusion: Ruxolitinib is a viable treatment option for Intermediate-2 to high risk myelofibrosis with massive splenomegaly. Anemia and thrombocytopenia were common hematologic toxicities during therapy. |
Databáze: | OpenAIRE |
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