Thalidomide for severe refractory ankylosing spondylitis: a 6-month open-label trial.
OBJECTIVE
To examine the efficacy of thalidomide in the treatment of active ankylosing spondylitis (AS) refractory to conventional therapies.


METHODS
In a 6-month open-label trial, we studied 13 men with different subtypes of active AS: 3 juvenile AS, 9 adult AS, and one AS with psoriasis. All patients were resistant to conventional nonbiologic therapies including nonsteroidal antiinflammatory drugs, sulfasalazine, and methotrexate. After 3 months' observation on a preexisting regimen, oral thalidomide was added, starting at 100 mg/day for 1 week, then 200 mg/day for another 23 weeks. Outcome measures included the Bath AS Disease Activity Index (BASDAI), Functional Index (BASFI), Global Index (BAS-G), IgA, C-reactive protein (CRP), and eosinophil sedimentation rate (ESR). Response to treatment was defined following the Ankylosing Spondylitis Assessment criteria.


RESULTS
Three patients withdrew due to rash. Two patients were lost to followup due to lack of efficacy. Eight patients completed the trial. Four patients attained > 50% improvement (2 juvenile AS, 1 peripheral AS, and 1 psoriatic arthritis). Four patients attained > 20% improvement (2 axial and 2 peripheral AS). Total response rate accordingly was 80% (8/10). Mean BASDAI improved significantly from baseline to Week 24 (4.97 vs 3.1; p = 0.0156). Mean BASFI improved from baseline to Week 24 (5.24 vs 3.06; p = 0.0078), and BAS-G from 6.02 to 3.21 (p = 0.0078). Significant laboratory improvements were found in ESR (from 69.5 to 34.2 mm/h; p = 0.0156), but not CRP (from 6.08 to 3.01 mg/dl; p = 0.078) or IgA (from 496 to 505 mg/dl; p = 0.375). Dry mouth, constipation, and dizziness were common, but no severe adverse events were found.


CONCLUSIONS
Thalidomide is a promising treatment for patients with active AS who are resistant to conventional therapies other than biologics.
Wei
James Cheng-Chung
JC
Division of Allergy, Immunology and Rheumatology, Chung Shan Medical University, Sec. 1, 110 Chien Kuo N. Road, Taichung 402, Taiwan, China. wei3228@ms3.hinet.net
Chan
Tsai Wen
TW
Lin
Huey-Shyan
HS
Huang
Feng
F
Chou
Chan-Te
CT
D000328:Adult; D018501:Antirheumatic Agents / Q000627:therapeutic use; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D019233:Retreatment; D012720:Severity of Illness Index; D013167:Spondylitis, Ankylosing / Q000188:drug therapy* / Q000503:physiopathology; D013792:Thalidomide / Q000627:therapeutic use*; D017211:Treatment Failure; D016896:Treatment Outcome
D018501:Antirheumatic Agents; D007166:Immunosuppressive Agents; D013792:Thalidomide