Efficacy of Guselkumab in the Treatment of Hailey Hailey Disease: An Open-label, Proof of Concept Study
Hailey-Hailey disease (HHD) is a debilitating genetic skin disorder, affecting mainly body folds with erythema and painful erosions and blisters. Histopathological findings include epidermal hyperplasia, suprabasilar clefting, dyskeratosis and acantholysis of keratinocytes. A final diagnosis of HHD is usually confirmed based on clinical and histopathological findings in line with genetic testing. Several treatment options have been proposed for this chronic and disabling disorder, however, there is no reproducibly effective therapeutic for it. The primary objective is to evaluate the treatment response of guselkumab. Single-center, non-randomized, single-arm, open-label, phase II trial to evaluate the efficacy and safety of guselkumab for the treatment of patients with HHD.
• A documented diagnosis of Hailey-Hailey disease confirmed with clinical, and histopathologic findings
• Disease affecting more than one body site with at least moderate severity
• If patients are taking other systemic therapies for their HHD (antibiotics, prednisone), they must be taking a stable dose of the other medication(s) for at least 3 months with no plans to change the regimen in the next 6 months. With the exception of antibiotics, methotrexate or low dose prednisone (less than 5 mg daily), use of concomitant immunosuppressants, e.g. azathioprine, etc. and biologics other than TNF-α inhibitors will not be permitted.
• Patients must be willing to have skin biopsies, blood collection, and total body photography and to comply with clinic visits