An open-label pilot study of low-dose thalidomide in chronically active, steroid-dependent Crohn's disease.
OBJECTIVE
Thalidomide decreases production of tumor necrosis factor alpha, a proinflammatory cytokine associated with Crohn's disease (CD). In this study the safety, tolerance, and efficacy of low-dose thalidomide were evaluated for treatment of moderate-to-severe, steroid-dependent CD.


METHODS
Twelve adult male patients with Crohn's Disease Activity Index (CDAI) scores of > or = 250 and < or = 500 despite > or = 20 mg prednisone/day were enrolled. The first 6 patients received 50 mg thalidomide every night, the next 6 received 100 mg every night. Steroid doses were stable during the first 4 weeks of treatment, then tapered during weeks 5-12. CDAI was used to assess response.


RESULTS
(1) Disease activity improved consistently in all patients during weeks 1-4: 58% response, 17% remission. (2) Clinical improvement was generally maintained despite steroid taper during weeks 5-12. All patients were able to reduce steroids by >/=50%. Forty-four percent discontinued steroids entirely. In weeks 5-12, 70% of patients responded and 20% achieved remission. (3) Side effects were mild and mostly transient, with the most common being drowsiness, peripheral neuropathy, edema, and dermatitis.


CONCLUSIONS
Low-dose thalidomide appears to be well tolerated and effective over a 12-week period. Results of this pilot study support the need for controlled multicenter trials of thalidomide for treatment of CD.
Vasiliauskas
E A
EA
Inflammatory Bowel Disease Center, Division of Gastroenterology, Department of Medicine, Cedars-Sinai Medical Center, and UCLA School of Medicine, Los Angeles, CA 90048, USA.
Kam
L Y
LY
Abreu-Martin
M T
MT
Hassard
P V
PV
Papadakis
K A
KA
Yang
H
H
Zeldis
J B
JB
Targan
S R
SR
D000328:Adult; D000894:Anti-Inflammatory Agents, Non-Steroidal / Q000009:adverse effects / Q000627:therapeutic use*; D002908:Chronic Disease; D003424:Crohn Disease / Q000188:drug therapy*; D004576:Electromyography; D005402:Fistula; D006801:Humans; D008297:Male; D008875:Middle Aged; D010865:Pilot Projects; D011788:Quality of Life; D012074:Remission Induction; D013792:Thalidomide / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome; D014409:Tumor Necrosis Factor-alpha / Q000037:antagonists & inhibitors
D000894:Anti-Inflammatory Agents, Non-Steroidal; D014409:Tumor Necrosis Factor-alpha; D013792:Thalidomide