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 Abstract: 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: firstname.lastname@example.org. © 2014 by Lippincott Williams & Wilkins, Inc.