Busulfan is effective second-line therapy for older patients with Philadelphia-negative myeloproliferative neoplasms intolerant of or unresponsive to hydroxyurea.

Genevieve Douglas, Claire Harrison, Cecily Forsyth, Michael Bennett, William Stevenson, John Hounsell, Sumita Ratnasingam, David Ritchie, David Ross, Andrew Grigg

Research output: Contribution to journalArticle

4 Citations (Scopus)

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.

Original languageEnglish
Pages (from-to)89-95
Number of pages7
JournalLeukemia and Lymphoma
Volume58
Issue number1
DOIs
Publication statusPublished - 2 Jan 2017

Keywords

  • busulfan
  • hydroxyurea
  • Philadelphia-negative myeloproliferative neoplasms

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