autologous blood and marrow transplantation - Blog Science ...

Rodenhuis et al. All
interest. One patient did respond to FE 120C, but had estrogen receptor-positive
lobular carcinoma. The authors believed that high-dose therapy was not the best
treatment option, and she was taken off protocol as a protocol violation. The
treatment of one further patient was too early for evaluation and will be reported in
a later publication.
A total of 80 tCTC courses were administered. Thirty-two patients received a
first tCTC course, 28 patients a second, and 20 patients completed the full planned
sequence of three tCTC courses. Two patients are still undergoing treatment after
tCTC-1 and will be reported elsewhere. The other two patients were taken off
protocol after tCTC-1 either because of lack of response (one patient) or because
of prolonged abnormalities of liver function tests (one patient). Six of eight patients
who did not proceed to a third course of tCTC after tCTC-2 stopped treatment
because of excessive toxicity (one unexplained death, three hemorrhagic cystitis,
one veno-occlusive disease, one refractory thrombocytopenia). A seventh patient
showed no further disease regression after the second tCTC course and was advised
to discontinue protocol treatment. The eighth patient was still on protocol at the
time of this writing.
The protocol required that second and third courses of tCTC be started on day
22-29 after the previous PBPC transplantation; the earliest time point of 22 days
was preferred. Following this policy, the median day after PBPC reinfusion on
which tCTC-2 was begun was 28 (range 21-53). This was day 35 (range 20-42)
for the third tCTC course. In addition, dose adaptations were required in three of
the third courses.
Major toxicity
The tCTC regimen was well tolerated by most patients, and the large majority
were able to leave the hospital on the first day after PBPC transplantation. Details
of the outpatient management of these transplantation patients will be published
elsewhere (A.M.W., et al., manuscript submitted); this section will focus on major
toxicities, such as grade IV gastrointestinal (GI) toxicity, organ toxicity (liver,
kidney, lung, heart, or lung), hemorrhagic cystitis, and other complications that are
important for the feasibility and acceptability of the triple tCTC regimen.
One patient died on the day 18 after reinfusion while apparently recovering
from her second tCTC course. Her death was sudden and in her sleep, at a time
when the neutrophil count had returned to normal but platelet transfusion
dependence had not yet resolved. Unfortunately, no autopsy permission could be
obtained and the cause of death remains unclear.
The major (organ) toxicity of tCTC is presented in Table 2. Although none of
this toxicity was life-threatening, it is clear that major organ toxicity such as
hemorrhagic cystitis and veno-occlusive disease of the liver do occur with some