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


CONCLUSIONS
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.
Faoro
Leonardo N
LN
Division of Hematology, Mayo Clinic, Rochester, MN, USA.
Tefferi
Ayalew
A
Mesa
Ruben A
RA
D000328:Adult; D000368:Aged; D000970:Antineoplastic Agents / Q000008:administration & dosage* / Q000009:adverse effects; D017338:Cladribine / Q000008:administration & dosage* / Q000009:adverse effects; D005260:Female; D005500:Follow-Up Studies; D006402:Hematologic Diseases / Q000139:chemically induced; D006529:Hepatomegaly / Q000139:chemically induced; D006801:Humans; D008297:Male; D008875:Middle Aged; D010166:Palliative Care; D055728:Primary Myelofibrosis / Q000150:complications / Q000188:drug therapy* / Q000473:pathology / Q000601:surgery*; D012189:Retrospective Studies; D013156:Splenectomy
D000970:Antineoplastic Agents; D017338:Cladribine