M. Figueroa et al., COMBINATION CHEMOTHERAPY IN REFRACTORY IMMUNE THROMBOCYTOPENIC PURPURA, The New England journal of medicine, 328(17), 1993, pp. 1226-1229
Background. Chronic idiopathic thrombocytopenic purpura is a destructi
ve thrombocytopenia caused by an autoantibody. About 80 percent of pat
ients with chronic idiopathic thrombocytopenic purpura have remissions
after either corticosteroid therapy or splenectomy. Some patients wit
h resistant disease respond to other agents, but a substantial group a
re refractory to therapy. Methods. We used combination chemotherapy to
treat 10 patients with refractory immune thrombocytopenia. An average
of 6.8 (range, 3 to 10) previous therapies, including corticosteroids
and splenectomy, had been unsuccessful in these patients. The patient
s received from three to eight cycles of therapy consisting of cycloph
osphamide and prednisone combined with either vincristine (one patient
), vincristine and procarbazine (four patients), or etoposide (six pat
ients, including one patient who received four cycles each containing
procarbazine and etoposide) Results. Among the 10 patients, 6 had comp
lete responses (platelet count, >180,000 per cubic millimeter); of the
se, 4 patients had responses that persisted for more than 11, 30, 54,
or 126 months, 1 had a relapse 9 months after therapy but had a remiss
ion with further therapy and remained in remission for 48 months befor
e dying of an unrelated illness, and another relapsed just before her
fifth course of therapy. Two patients had partial responses (platelet
count, >50,000 per cubic millimeter); the platelet counts in one remai
ned stable for more than nine months after the end of therapy, and the
other patient relapsed. The remaining two patients had no response. C
omplete responses were associated with a disappearance or marked decre
ase in the level of platelet-associated auto-antibody. Conclusions. Co
mbination chemotherapy is beneficial in some patients in whom immune t
hrombocytopenia is refractory to corticosteroids and splenectomy.