Prospective, systematically recorded mycophenolate safety data in Graves’ orbitopathy

Context The antiproliferative mechanism of mycophenolate acid (MPA) suggests a beneficial effect in patients with Graves’ orbitopathy (GO). Objective To systematically analyze for the first time adverse events (AEs) during MPA treatment in GO. Design Prospective longitudinal study. Setting Academic tertiary referral center with a joint thyroid-eye clinic. Patients Fifty-three consecutive, unselected patients with clinically active and moderate-to-severe GO. Methods MPA 0.720 g was given once daily for 24-weeks in GO patients. AEs were documented and coded according to the standardized medical dictionary for regulatory activities (MedDRA). AE were followed up and seriousness as defined by ICH-guideline E6 was documented. All AEs were analyzed regarding a possible underlying cause and if not, graded as side effect (SE). Results Fifty GO patients (93 %) had Graves’ disease, 37 (70 %) and 29 (54.7 %) were female and smoker, respectively. Thirty-six patients (68 %) reported at least one AE. A total of 88 AEs were documented, most frequent AEs were insomnia ( N = 6), fatigue ( N = 5) and optic neuropathy ( N = 5), while other AEs occurred in up to three patients (5.6 %), only. In 12 (23 %) patients, at least one SE occurred. All 17 reported SE, i.e. mild infections and gastrointestinal intolerance were within the known safety profile of MPA. No patient dropped MPA medication because of drug-induced SE. Most AEs showed a recovered (76 %) or recovering (16 %) outcome. Seven (13 %) patients were hospitalized, five (62 %) due to optic neuropathy, none of these events was graded as SE. Conclusions MedDRA-coded data documented the good tolerance of a moderate MPA dose in GO patients.