Background. In myeloma patients resistance to both melphalan- and doxo
rubicin-containing regimens has been related to very short survival (a
pproximately 6 months). The development of effective regimens combined
with a low toxicity rate is mandatory in this patient subgroup. Metho
ds. Fourteen resistant myeloma patients were treated with cyclophospha
mide (a total of 3.6 g/m(2) was delivered in 2 doses on days 1 and 3)
and prednisone (2 mg/kg, days 1-4) every month for 4 cycles. G-CSF sup
port was administered to reduce myelosuppression. Results. This combin
ation was well tolerated. Granulocyte levels fell below 0.1x10(9)/L in
all patients after a median of 9 days (range 8-11), followed by recov
ery to 0.5 x 10(9)/L after a median of 12 days from the start of treat
ment (range 10-13 days). Platelets never fell below 50x10(9)/L. Ah pat
ients were treated on an outpatient basis and only 2 required hospital
ization for major complications (pneumonia and heart failure). Respons
e to cyclophosphamide was observed in 6/14 patients: 2 achieved comple
te remission, 4 showed a 50% or greater reduction of the M-component.
Five patients are still in remission after 2, 6, 7, 9 and 10 months; 1
relapsed after 10 months. Ah patients except one are alive 4-16 month
s from the start of treatment. Conclusions. This schedule may represen
t a new approach for resistant myeloma, and its very low toxicity allo
ws it to be delivered on an outpatient basis.