Multicentre, phase II trial on the safety and efficacy of topical tacrolimus ointment for the treatment of lichen sclerosus.
BACKGROUND
Lichen sclerosus is a chronic inflammatory autoimmune disease causing significant sclerosis, atrophy and pruritus. Treatment remains unsatisfactory, with potent corticosteroids being the most effective therapy.


OBJECTIVE
To conduct a multicentre, phase II trial to assess the safety and efficacy of tacrolimus ointment 0.1% for the treatment of lichen sclerosus with a follow-up period of 18 months at 10 university and teaching hospitals in Germany and Austria.


METHODS
Eighty-four patients (49 women, 32 men and three girls) aged between 5 and 85 years with long-standing, active lichen sclerosus (79 with anogenital and five with extragenital localization) were treated with topical tacrolimus ointment 0.1% twice daily for 16 weeks. Computerized analysis of the lesional area was performed. The primary endpoint was clearance of active lichen sclerosus. Secondary endpoints were time to optimal response, reduction of sclerosis and duration of remission.


RESULTS
The primary endpoint (clearance of active lichen sclerosus) was reached by 43% of patients at 24 weeks of treatment. Partial resolution was reached in 34% of patients. Maximal effects occurred between week 10 and 24 of therapy. Treatment led to a significant reduction of the total lesional area (P < 0.01) and to a significant decline in the total symptom score (P < 0.005). Symptoms (e.g. itching) and findings (erythema, erosions and induration) showed significant improvement. No serious adverse events were observed. There were three (9%) recurrences during the follow-up period.


CONCLUSIONS
Topical tacrolimus ointment 0.1% was safe and effective for the treatment of long-standing active lichen sclerosus.
Hengge
U R
UR
Department of Andrology and Venereology, Philipps-University Marburg, Marburg, Germany. ulrich.hengge@uni-duesseldorf.de
Krause
W
W
Hofmann
H
H
Stadler
R
R
Gross
G
G
Meurer
M
M
Brinkmeier
T
T
Frosch
P
P
Moll
I
I
Fritsch
P
P
Müller
K
K
Meykadeh
N
N
Marini
A
A
Ruzicka
T
T
Gollnick
H
H
D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D001327:Autoimmune Diseases / Q000188:drug therapy* / Q000473:pathology; D002648:Child; D002675:Child, Preschool; D005260:Female; D005831:Genital Diseases, Female / Q000188:drug therapy / Q000473:pathology; D005832:Genital Diseases, Male / Q000188:drug therapy / Q000473:pathology; D006801:Humans; D007166:Immunosuppressive Agents / Q000009:adverse effects / Q000627:therapeutic use*; D018459:Lichen Sclerosus et Atrophicus / Q000188:drug therapy* / Q000473:pathology; D008297:Male; D008875:Middle Aged; D009824:Ointments; D012720:Severity of Illness Index; D016559:Tacrolimus / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome
D007166:Immunosuppressive Agents; D009824:Ointments; D016559:Tacrolimus