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Pulse cyclophosphamide therapy for refractory autoimmune thrombocytopenic
purpura [see comments]
A Reiner, T Gernsheimer and SJ Slichter
Medical & Research Division, Puget Sound Blood Center, Seattle, WA
98104-1256.
Autoimmune thrombocytopenic purpura (AITP) is generally a chronic disorder
in affected adults. Twenty-five percent of these patients will become
refractory to routine therapy (corticosteroids and splenectomy), as well as
most other available agents. Intravenous pulse cyclophosphamide therapy was
used to treat 20 patients with severe refractory AITP who had previously
failed to achieve a sustained remission with a mean of 4.8 agents (range 2
to 8). Patients received 1 to 4 doses (mean 2.0) of 1.0 to 1.5 g/m2
intravenous cyclophosphamide per course. Of the 20 patients treated with
pulse cyclophosphamide therapy, 13 patients (65%) achieved a complete
response (CR), four (20%) a partial response (PR), and three patients (15%)
failed to respond. Of the 13 complete responders, eight have remained in
remission with stable platelet counts during followup intervals of 7 months
to 7 years (median 2.5 years). Five patients developed recurrent AITP 4
months to 3 years following a CR. Of these, two patients responded to
subsequent courses of pulse cyclophosphamide therapy with current
remissions of 1 and 4 years. Of the four patients who obtained a PR, two
remain in partial remission after 10 months and 4 years; one relapsed after
18 months and, after retreatment, is still in remission at 6 months. Of the
patient characteristics examined, duration of disease was most strongly
associated with response to pulse cyclophosphamide. Side-effects of
treatment included neutropenia (three patients, one of whom developed
staphylococcal sepsis), acute deep venous thrombosis (two patients), and
psoas abscess (one patient). Intravenous pulse cyclophosphamide should be
strongly considered in the treatment of patients with refractory AITP.
There is a relatively low incidence of side-effects, and it can be
administered easily on an out- patient basis.
Volume 85,
Issue 2,
pp. 351-358,
01/15/1995
Copyright © 1995 by The American Society of Hematology
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