Safety and efficacy of fumagillin for the treatment of intestinal microsporidiosis. A French prospective cohort study. (30th October 2020)

• Record Type: Journal Article
• Authors: Maillard, Alexis; Scemla, Anne; Laffy, Benjamin; Mahloul, Nadir; Molina, Jean-Michel
• Is Part Of: Journal of antimicrobial chemotherapy. Volume 76:Number 2(2021)
• Page Start: 487
• Page End: 494
• Publication Date: 2020-10-30
• Subjects: Anti-infective agents -- Periodicals; Chemotherapy -- Periodicals; 615.58
• DOI: 10.1093/jac/dkaa438
• Languages: English
• ISSNs: 0305-7453

Abstract

Abstract: Background: Intestinal microsporidiosis due to Enterocytozoon bieneusi is a cause of chronic diarrhoea in immunocompromised patients. Fumagillin has been approved in France for its treatment. Objectives: To investigate the efficacy and safety of fumagillin in a real-life setting. Methods: As required by the French Medicine Agency, all patients receiving fumagillin were enrolled in a prospective study to evaluate its efficacy and safety. Stool examination with identification of E. bieneusi by PCR was performed at baseline, end of treatment and monthly thereafter for 6 months. Safety was monitored up to 6 months and full blood counts were monitored up to 42 days after treatment initiation. The primary endpoint was safety. Parasite clearance and relapses were secondary endpoints. Results: From 2007 to 2018, 166 patients received fumagillin, including 6 children. Patients were transplant recipients (84%), HIV-infected patients (13%) or had another cause of immunosuppression (5%). Serious adverse events were reported in 41 patients (25%), mainly thrombocytopenia (15%) and neutropenia (5%), with two haemorrhagic events leading to one death. Severe thrombocytopenia (<50 G/L) developed in 50 patients (29.6%), neutropenia (<1 G/L) in 20 patients (11.8%) and severe anaemia (<8 g/dL) in 21 patients (12.4%). At the end of treatment, 94% of patients with available stool examination ( n = 132) had no spores detected. Among 99 patients with available follow-up after the end of treatment, three parasite relapses were documented. Conclusions: E. bieneusi microsporidiosis was mainly diagnosed in transplant recipients. Fumagillin was associated with haematological toxicity but showed high efficacy with a low relapse rate.