Long-term analysis of the palliative benefit of 2-chlorodeoxyadenosine for myelofibrosis with myeloid metaplasia

Leonardo N. Faoro, Ayalew Tefferi, Ruben A. Mesa

Research output: Contribution to journalArticlepeer-review

48 Scopus citations


Objective: Therapeutic splenectomy in myelofibrosis with myeloid metaplasia (MMM) may result in extreme thrombocytosis and leukocytosis and accelerated hepatomegaly. We previously described initial palliative benefit from 2-chlorodeoxyadenosine (2-CdA) in such instances. The purpose of this study is to provide long-term follow-up on the durability of response in the initial cohort and in additional subsequent cases. Methods: We retrospectively identified patients with histologically confirmed MMM who had palliative therapy with 2-CdA. Clinical characteristics and information on subsequent clinical course were abstracted at the time of diagnosis of MMM and at initiation of 2-CdA therapy until death. Results: To date, we have used 2-CdA as palliative therapy in 14 patients with MMM. After a median of four cycles of therapy, responses for hepatomegaly occurred in 56% of patients, thrombocytosis 50%, leukocytosis 55%, and anemia 40%. Cytopenias were frequent but usually transient and without clinical consequence. Responses occurred usually by the second cycle; median duration of response was 6 months (range, 2-19) after completion of 2-CdA therapy. Conclusion: This study confirmed relevant and frequently durable palliation of symptoms in about half the patients. 2-CdA is a reasonable palliative option in postsplenectomy patients with MMM who have problematic myeloproliferation.

Original languageEnglish (US)
Pages (from-to)117-120
Number of pages4
JournalEuropean Journal of Haematology
Issue number2
StatePublished - Feb 2005


  • 2-Chlorodeoxyadenosine
  • Myelofibrosis
  • Myeloid metaplasia
  • Myeloproliferative diseases
  • Splenectomy
  • Treatment outcome

ASJC Scopus subject areas

  • Hematology


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