Successful Management of Refractory Pediatric-Onset Complex Aphthosis With LenalidomideKalampokis, Ioannis MD, MPH*; Rabinovich, C. Egla MD, MPH†JCR: Journal of Clinical Rheumatology: June 2014 - Volume 20 - Issue 4 - p 221–223 doi: 10.1097/RHU.0000000000000100 Case Reports Abstract Author Information Clinicians are frequently confronted with patients presenting with oral aphthous ulcers or orogenital aphthae. Patients with complex aphthosis are characterized by the nearly constant presence of more than 3 oral aphthous ulcers or recurrent orogenital aphthae but do not satisfy the criteria for Behçet disease. We report a severe case of pediatric-onset complex aphthosis with poor response and/or significant toxicity to first-line medications. Lenalidomide, a second-generation immunomodulatory drug, induced a complete disease remission within few days of therapy. The patient has been treated for nearly 3 years with significant subjective and clinical improvement and no adverse effects. Thereby, lenalidomide may represent a well-tolerated and effective medication for patients with complex aphthosis who are intolerant or not responsive to first-line agents. Nevertheless, in view of the rare but serious potential adverse effects of lenalidomide such as teratogenicity and cancer, a clinical trial is necessary to assess the true risk-to-benefit ratio for the use of lenalidomide in patients with complex aphthosis. From the *Department of Immunology and †Division of Pediatric Rheumatology, Department of Pediatrics, Duke University Medical Center, Durham, NC. The authors declare no conflict of interest. Correspondence: C. Egla Rabinovich, MD, MPH, Division of Pediatric Rheumatology, Department of Pediatrics, Duke University Medical Center, Box 3212, Durham, NC 27710. E-mail: email@example.com. © 2014 by Lippincott Williams & Wilkins, Inc.