Pasquale Niscola, Marco Giovannini, Valentina Gianfelici, Carla Mazzone, Paolo de Fabritiis, Esther Natalie Oliva
{"title":"阿扎胞苷清除伴有Del5q的高危骨髓增生异常综合征骨髓母细胞后对来那度胺的长期持续应答:1例报告","authors":"Pasquale Niscola, Marco Giovannini, Valentina Gianfelici, Carla Mazzone, Paolo de Fabritiis, Esther Natalie Oliva","doi":"10.2174/0118715257341062250130103015","DOIUrl":null,"url":null,"abstract":"<p><strong>Introduction: </strong>This report discusses a rare case involving a patient with high-risk (HR) Del(5q) myelodysplastic syndrome (MDS) who achieved a long-term response to lenalidomide after having received six cycles of azacytidine. The latter treatment led to the clearance of blast cells from the bone marrow (BM).</p><p><strong>Case representation: </strong>As per current clinical practice, patients with HR MDS receive azacytidine continuously until the disease progresses or the occurrence of unmanageable side effects. However, in this case, the patient decided to interrupt hypomethylation therapy. Due to the patient's preference for oral therapy at home, the absence of blast cells, the ongoing need for transfusions, and a cytogenetic abnormality-predictive response to lenalidomide, the choice of the latter agent allowed for a sustained response lasting up to 68 months.</p><p><strong>Conclusion: </strong>Our observations suggest that further studies could explore the sequential use of azacytidine followed by lenalidomide after achieving BM blast clearance in patients with HR MDS with del(5q).</p>","PeriodicalId":93924,"journal":{"name":"Cardiovascular & hematological agents in medicinal chemistry","volume":" ","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2025-02-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Long-term Sustained Response to Lenalidomide after Clearance of Bone Marrow Blasts by Azacytidine in High-risk Myelodysplastic Syndromes with Del5q: A Case Report.\",\"authors\":\"Pasquale Niscola, Marco Giovannini, Valentina Gianfelici, Carla Mazzone, Paolo de Fabritiis, Esther Natalie Oliva\",\"doi\":\"10.2174/0118715257341062250130103015\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Introduction: </strong>This report discusses a rare case involving a patient with high-risk (HR) Del(5q) myelodysplastic syndrome (MDS) who achieved a long-term response to lenalidomide after having received six cycles of azacytidine. The latter treatment led to the clearance of blast cells from the bone marrow (BM).</p><p><strong>Case representation: </strong>As per current clinical practice, patients with HR MDS receive azacytidine continuously until the disease progresses or the occurrence of unmanageable side effects. However, in this case, the patient decided to interrupt hypomethylation therapy. Due to the patient's preference for oral therapy at home, the absence of blast cells, the ongoing need for transfusions, and a cytogenetic abnormality-predictive response to lenalidomide, the choice of the latter agent allowed for a sustained response lasting up to 68 months.</p><p><strong>Conclusion: </strong>Our observations suggest that further studies could explore the sequential use of azacytidine followed by lenalidomide after achieving BM blast clearance in patients with HR MDS with del(5q).</p>\",\"PeriodicalId\":93924,\"journal\":{\"name\":\"Cardiovascular & hematological agents in medicinal chemistry\",\"volume\":\" \",\"pages\":\"\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2025-02-11\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Cardiovascular & hematological agents in medicinal chemistry\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.2174/0118715257341062250130103015\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Cardiovascular & hematological agents in medicinal chemistry","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.2174/0118715257341062250130103015","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
Long-term Sustained Response to Lenalidomide after Clearance of Bone Marrow Blasts by Azacytidine in High-risk Myelodysplastic Syndromes with Del5q: A Case Report.
Introduction: This report discusses a rare case involving a patient with high-risk (HR) Del(5q) myelodysplastic syndrome (MDS) who achieved a long-term response to lenalidomide after having received six cycles of azacytidine. The latter treatment led to the clearance of blast cells from the bone marrow (BM).
Case representation: As per current clinical practice, patients with HR MDS receive azacytidine continuously until the disease progresses or the occurrence of unmanageable side effects. However, in this case, the patient decided to interrupt hypomethylation therapy. Due to the patient's preference for oral therapy at home, the absence of blast cells, the ongoing need for transfusions, and a cytogenetic abnormality-predictive response to lenalidomide, the choice of the latter agent allowed for a sustained response lasting up to 68 months.
Conclusion: Our observations suggest that further studies could explore the sequential use of azacytidine followed by lenalidomide after achieving BM blast clearance in patients with HR MDS with del(5q).