Bengisu Ece Duman , Melis Selin Kadıoğlu , Halil İbrahim Yüksel , Birol Güvenç
{"title":"真性红细胞增多症在长期羟基脲治疗后进展为急性髓性白血病:一个病例研究","authors":"Bengisu Ece Duman , Melis Selin Kadıoğlu , Halil İbrahim Yüksel , Birol Güvenç","doi":"10.1016/j.htct.2025.103929","DOIUrl":null,"url":null,"abstract":"<div><div>Polycythemia Vera (PV) is a chronic Myeloproliferative Neoplasm (MPN) with a well-documented risk of progression to Aute Myeloid Leukemia (AML), particularly in patients undergoing prolonged cytoreductive therapy. This report details the case of a 66-year-old male diagnosed with PV five years prior, initially managed with hydroxyurea. Over time, he developed progressive pancytopenia, ultimately leading to a diagnosis of AML. Following leukemic transformation, the patient was treated with azacitidine, a hypomethylating agent commonly utilized in myeloid malignancies. However, hematologic response was minimal, and disease progression ensued. Molecular analysis identified AML-associated mutations, which are implicated in disease evolution, therapeutic resistance, and poor prognosis. The transition from PV to AML represents a critical clinical challenge, significantly worsening patient outcomes. While hydroxyurea remains a widely used first-line therapy for PV, its potential role in leukemic transformation continues to be debated. Azacitidine, although a viable therapeutic option for post-MPN AML, frequently yields limited and non-durable responses, particularly in patients with high-risk genetic alterations. This case underscores the necessity of vigilant monitoring in PV patients receiving long-term cytoreductive therapy to enable early detection of leukemic progression. Alternative treatment approaches, including JAK inhibitors, interferon therapy, and early hematopoietic stem cell transplantation in eligible patients, may play a role in reducing leukemic transformation risk. Further research is essential to enhance the understanding of post-MPN AML pathogenesis and optimize treatment strategies to improve patient survival.</div></div>","PeriodicalId":12958,"journal":{"name":"Hematology, Transfusion and Cell Therapy","volume":"47 ","pages":"Article 103929"},"PeriodicalIF":1.8000,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"PROGRESSION OF POLYCYTHEMIA VERA TO ACUTE MYELOID LEUKEMIA FOLLOWING LONG-TERM HYDROXYUREA THERAPY: A CASE STUDY\",\"authors\":\"Bengisu Ece Duman , Melis Selin Kadıoğlu , Halil İbrahim Yüksel , Birol Güvenç\",\"doi\":\"10.1016/j.htct.2025.103929\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><div>Polycythemia Vera (PV) is a chronic Myeloproliferative Neoplasm (MPN) with a well-documented risk of progression to Aute Myeloid Leukemia (AML), particularly in patients undergoing prolonged cytoreductive therapy. This report details the case of a 66-year-old male diagnosed with PV five years prior, initially managed with hydroxyurea. Over time, he developed progressive pancytopenia, ultimately leading to a diagnosis of AML. Following leukemic transformation, the patient was treated with azacitidine, a hypomethylating agent commonly utilized in myeloid malignancies. However, hematologic response was minimal, and disease progression ensued. Molecular analysis identified AML-associated mutations, which are implicated in disease evolution, therapeutic resistance, and poor prognosis. The transition from PV to AML represents a critical clinical challenge, significantly worsening patient outcomes. While hydroxyurea remains a widely used first-line therapy for PV, its potential role in leukemic transformation continues to be debated. Azacitidine, although a viable therapeutic option for post-MPN AML, frequently yields limited and non-durable responses, particularly in patients with high-risk genetic alterations. This case underscores the necessity of vigilant monitoring in PV patients receiving long-term cytoreductive therapy to enable early detection of leukemic progression. Alternative treatment approaches, including JAK inhibitors, interferon therapy, and early hematopoietic stem cell transplantation in eligible patients, may play a role in reducing leukemic transformation risk. Further research is essential to enhance the understanding of post-MPN AML pathogenesis and optimize treatment strategies to improve patient survival.</div></div>\",\"PeriodicalId\":12958,\"journal\":{\"name\":\"Hematology, Transfusion and Cell Therapy\",\"volume\":\"47 \",\"pages\":\"Article 103929\"},\"PeriodicalIF\":1.8000,\"publicationDate\":\"2025-07-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Hematology, Transfusion and Cell Therapy\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S253113792500197X\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q3\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Hematology, Transfusion and Cell Therapy","FirstCategoryId":"1085","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S253113792500197X","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"HEMATOLOGY","Score":null,"Total":0}
PROGRESSION OF POLYCYTHEMIA VERA TO ACUTE MYELOID LEUKEMIA FOLLOWING LONG-TERM HYDROXYUREA THERAPY: A CASE STUDY
Polycythemia Vera (PV) is a chronic Myeloproliferative Neoplasm (MPN) with a well-documented risk of progression to Aute Myeloid Leukemia (AML), particularly in patients undergoing prolonged cytoreductive therapy. This report details the case of a 66-year-old male diagnosed with PV five years prior, initially managed with hydroxyurea. Over time, he developed progressive pancytopenia, ultimately leading to a diagnosis of AML. Following leukemic transformation, the patient was treated with azacitidine, a hypomethylating agent commonly utilized in myeloid malignancies. However, hematologic response was minimal, and disease progression ensued. Molecular analysis identified AML-associated mutations, which are implicated in disease evolution, therapeutic resistance, and poor prognosis. The transition from PV to AML represents a critical clinical challenge, significantly worsening patient outcomes. While hydroxyurea remains a widely used first-line therapy for PV, its potential role in leukemic transformation continues to be debated. Azacitidine, although a viable therapeutic option for post-MPN AML, frequently yields limited and non-durable responses, particularly in patients with high-risk genetic alterations. This case underscores the necessity of vigilant monitoring in PV patients receiving long-term cytoreductive therapy to enable early detection of leukemic progression. Alternative treatment approaches, including JAK inhibitors, interferon therapy, and early hematopoietic stem cell transplantation in eligible patients, may play a role in reducing leukemic transformation risk. Further research is essential to enhance the understanding of post-MPN AML pathogenesis and optimize treatment strategies to improve patient survival.