Dr. Michelle Min
Michelle S Min , University of California, USA
Title: Novel Treatment Approaches in Challenging Connective Tissue Disease Cases
Biography
Biography: Dr. Michelle Min
Abstract
Introduction: Connective tissue diseases can be challenging to treat, and extensive cases often require systemic and/or procedural intervention. Recalcitrant disease significantly affects quality of life and can cause disability. Herein, we share our experiences and novel approaches to such challenging cases.
Janus kinase (JAK)-inhibitors for Dermatomyositis (DM): We will review our success in treating recalcitrant DM with tofacitinib, a JAK 1/3 inhibitor, in the largest case series available.1 Eleven patients with refractory DM were given tofacitinib, doses ranging from 5 mg po BID to 10 mg po BID. All patients had improvements in their skin appearance (Figure 1), pruritus, and muscle disease if present. The average Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) activity score improved by 17.8, with an average improvement of 19 points in amyopathic DM, 16.75 in classic DM, and 17 in juvenile DM. Therapy was well-tolerated.
Mycophenolate Mofetil for Salt-and-Pepper Dyspigmentation (S&P): Vitiligo-like depigmentation with perifollicular pigment retention, or salt-and-pepper dyspigmentation (S&P), is classically associated with severe systemic sclerosis (SSc). S&P can occur in other autoimmune sclerosing conditions including mixed connective tissue disease (MCTD) with scleroderma features. Unfortunately, such pigmentary changes are disproportionately seen in skin of color, and SSc clinical studies often neglect studying S&P. We report 3 African American patients with either SSc or MCTD with scleroderma features who suffered from S&P that responded to mycophenolate mofetil (MMF).2 All 3 patients experienced >75% improvement of S&P with MMF,
Hyaluronidase Injections for Oral Microstomia: Perioral changes are leading causes of concern to individuals with SSc. Few therapeutic options are available. We completed the first case series investigating local hyaluronidase intradermal injections in four individuals with debilitating oral microstomia (2 patients with limited SSc, 1 with diffuse SSc, and 1 MCTD with sclerodermoid features). Patients received 200 units of intradermal hyaluronidase monthly. All had improvements in mouth opening capacity (MOC) and Mouth Handicap in Systemic Sclerosis (MHISS). Patients’ MOC gained an average of 0.9 cm (range 0.5 to 1.6 cm) or 19.1%, and MHISS decreased 19.3 (17-24) or 61.9%. No serious adverse events were noted.
Conclusion: Patients with recalcitrant connective tissue disease require physicians to consider novel and/or unconventional therapeutic approaches. We share these cases to highlight potential treatments that physicians may want to consider for their sickest patients.