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Title: Busulfan is effective second-line therapy for older patients with Philadelphia-negative myeloproliferative neoplasms intolerant of or unresponsive to hydroxyurea
Austin Authors: Douglas, Genevieve ;Harrison, Claire;Forsyth, Cecily J;Bennett, Michael R;Stevenson, William;Hounsell, John;Ratnasingam, Sumita;Ritchie, David;Ross, David M;Grigg, Andrew P 
Affiliation: Austin Health, Heidelberg, Victoria, Australia
Department of Clinical Haematology, Austin Hospital, University of Melbourne, Heidelberg, Victoria, Australia
Department of Haematology, Guys and St Thomas' NHS Foundation Trust, London, UK
Wyong Hospital, Hamlyn Terrace, NSW, Australia
Royal North Shore Hospital, Sydney, NSW, Australia
Warrnambool Base Hospital, Warrnambool, Victoria, Australia
Royal Melbourne Hospital, Parkville, Victoria, Australia
Flinders University, Adelaide, SA, Australia
Flinders Medical Centre, Adelaide, SA, Australia
SA Pathology, Adelaide, SA, Australia
Issue Date: Jan-2017
Date: 2016-07-25
Publication information: Leukemia & Lymphoma 2017; 58(1): 89-95
Abstract: Hydroxyurea (Hu) is widely used as first-line cytoreductive therapy for patients with high-risk Philadelphia-negative myeloproliferative neoplasms (Ph-neg MPN), but a small proportion of patients have refractory disease or experience adverse effects. Studies have demonstrated busulfan (Bu) to be an active first-line agent, but data on its role as second-line or later therapy are minimal. To evaluate its efficacy and safety in this context, we undertook a multicenter audit of Ph-neg MPN patients who had received Bu as therapy for Hu intolerance or failure. Of 51 patients identified, 38 (75%) achieved either complete or partial hematological response following at least one Bu cycle. Bu was generally well tolerated, with only 21/135 (15%) cycles complicated by adverse effects, predominantly cytopenia; only 6% of cycles were ceased due to treatment complications. Bu is an effective and well-tolerated agent in patients with Ph-neg MPN in the setting of Hu intolerance or unresponsiveness.
DOI: 10.1080/10428194.2016.1187269
Journal: Leukemia & Lymphoma
PubMed URL:
Type: Journal Article
Subjects: Philadelphia-negative myeloproliferative neoplasms
Appears in Collections:Journal articles

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