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

Eur J Haematol. 2005 Feb;74(2):117-20. doi: 10.1111/j.1600-0609.2004.00370.x.


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.

MeSH terms

  • Adult
  • Aged
  • Antineoplastic Agents / administration & dosage*
  • Antineoplastic Agents / adverse effects
  • Cladribine / administration & dosage*
  • Cladribine / adverse effects
  • Female
  • Follow-Up Studies
  • Hematologic Diseases / chemically induced
  • Hepatomegaly / chemically induced
  • Humans
  • Male
  • Middle Aged
  • Palliative Care*
  • Primary Myelofibrosis / complications
  • Primary Myelofibrosis / drug therapy*
  • Primary Myelofibrosis / pathology
  • Primary Myelofibrosis / surgery*
  • Retrospective Studies
  • Splenectomy*


  • Antineoplastic Agents
  • Cladribine