Trial Information

Immunotherapy of the Paraneoplastic Syndromes

patients may stay either in-hospital while being treated with Tacrolimus, receive treatment
as an outpatient, or a combination of the two. Additionally, patients who are too sick to
be treated at Rockefeller University (eg. patients actively seizing), but are in need of
urgent treatment, may be treated at either Memorial Sloan-Kettering Cancer Center or New
York-Presbyterian Hospital in conjunction with MDs there. Dr. Robert Darnell, being
credentialed at both institutions, will be the primary person responsible for monitoring all
portions of the study performed at MSKCC and New York-Presbyterian Hospital. During
treatment, patients will undergo blood draws, at set intervals (see section g below),
clinical evaluation, possibly repeat leukapheresis or large volume blood draw, and lumbar
puncture (see below). Since many patients live far away from NY, some of these procedures
may be performed by RU staff or in conjunction with their local MDs.

Patients who are terminated from Tacrolimus treatment after 7-21 days will be followed up as
outpatients for evaluation of their neurologic and medical status. Wherever possible, these
patients will be seen on days 3 and 10 post treatment termination, and then on a biweekly
basis for two months. Since many patients live far away from NY, they may instead be
monitored in conjunction with their local MDs. Patients who show a definite clinical
response to Tacrolimus may be maintained on a therapeutic dose for up to one year, and will
be followed as outpatients. For patients receiving retreatment, they may be treated as
inpatients or on an outpatient basis, at the discretion of the PI, on the same schedule as
patients being treated initially (see 10.g.). Long term improvement or decline in neurologic
function will be objectively assessed by neurologic exam, which will be quantified by use of
the Karnofsky scale (a measure of functional neurologic status). Since the vast majority of
Hu patients decline over a 6-12 month period following diagnosis, a stable or improved
Karnofsky score over such a time period will be taken as a measure of successful treatment.
Repeat lumbar puncture (up to eight per year) and leukapheresis or large volume blood draw
(approx. 100 cc) may be performed (up to four of each per year), especially in the setting
of neurologic change, to assess the immune responses to the medications.