INTRODUCTION
Hzailey-Hailey disease (HHD), or benign familial pemphigus, is an uncommon blistering disorder that presents as recurrent vesicles and erosions. These lesions often appear in the third to fourth decade of life,1 mostly in the axillae, neck, groin, or inframammary folds, where sweat, moisture, and friction likely play important roles in the development of the disease.2 The condition is typically worse in the summer.3 In affected individuals, skin trauma, bacterial infections, fungal infections, or sunburn may trigger lesions. HHD is an autosomal dominant disease due to mutation in a calcium ATPase (ATP2C1) on chromosome 3q21.3 This disease has variable penetrance, with 70% of patients showing a definite family history.2
Treatments for HHD include nonsurgical approaches such as topical4 and oral antimicrobial agents, topical antifungals, short courses of topical and oral corticosteroids,5 oral retinoids,6 methotrexate, dapsone, and cyclosporine.7 In addition, surgical methods have been used for the treatment of HHD. These treatments include surgical excision with healing by secondary intent,8 primary closure,9 grafting,10-12 dermabrasion,13-14 CO2 laser vaporization,15-18 and Er:YAG laser ablation.19
Onabotulinumtoxin A (Botox® Allergan) is a purified neurotoxin. Several published case reports have supported the use of onabotulinumtoxin A in treatment of HHD.1,20-23 This 6-month randomized, double-blind, placebo-controlled cross-over study was designed to test the hypothesis that treatment with onabotulinumtoxin A (BtxA) is safe and effective for subjects with HHD in individuals aged 12 years and older.
Treatments for HHD include nonsurgical approaches such as topical4 and oral antimicrobial agents, topical antifungals, short courses of topical and oral corticosteroids,5 oral retinoids,6 methotrexate, dapsone, and cyclosporine.7 In addition, surgical methods have been used for the treatment of HHD. These treatments include surgical excision with healing by secondary intent,8 primary closure,9 grafting,10-12 dermabrasion,13-14 CO2 laser vaporization,15-18 and Er:YAG laser ablation.19
Onabotulinumtoxin A (Botox® Allergan) is a purified neurotoxin. Several published case reports have supported the use of onabotulinumtoxin A in treatment of HHD.1,20-23 This 6-month randomized, double-blind, placebo-controlled cross-over study was designed to test the hypothesis that treatment with onabotulinumtoxin A (BtxA) is safe and effective for subjects with HHD in individuals aged 12 years and older.
MATERIALS AND METHODS
This was a single-center study, registered with clinicaltrials.gov (ID:100817). Enrolled patients had biopsy-proven HHD and/or a compelling family history of HHD. Patients were also at least 12 years of age and had a negative pregnancy test, if applicable, prior to injection. All treatments for HHD were discontinued for at least 14 days before their initial injection. After randomization, patients were treated with the placebo (saline) or Btx-A on day 1. Injections were prepared by a blinded third party. The investigator was blinded as to which study drug the subject received. Patients were seen for in-office visits at weeks 4, 8, 12, and at any point between week 12 and month 6 for relapse of the disease. If the subject did not achieve improvement of HHD by week 4, they were deemed to be non-responders and