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

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13 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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