Efficacy of low dose nitisinone in the management of alkaptonuria.

AIM: To study the efficacy of low dosage of nitisinone in alkaptonuria. BACKGROUND: Alkaptonuria (AKU) is a rare genetic disease which induces deposition of homogentisic acid (HGA) in connective inducing premature arthritis, lithiasis, cardiac valve disease, fractures, muscle and tendon ruptures and osteopenia. Recent studies showed that nitisinone decreases HGA and is a beneficial therapy in AKU. This treatment induces an increase in tyrosine levels which can induces adverse effects as keratopathy. METHODS: We described the evolution HGA excretion and tyrosine evolution in 3 AKU patients treated by very low dosage of nitisinone with regards to their daily protein intakes. We also described the first pregnancy in an AKU patient treated by nitisinone. RESULTS: We found mild clinical signs of alkaptonuria on vertebra MRI in two young adults and homogentisate deposition in teeth of a 5 years old girl. Very low dose of nitisinone (10% of present recommended dose: 0.2 mg/day) allowed to decrease homogentisic acid by >90% without increasing tyrosine levels above 500 µmol/ in these three patients. INTERPRETATIONS: The analysis of the follow-up data shows that, in our three patients, a low-dosage of nitisinone is sufficient to decrease urinary HGA without increasing plasma tyrosine levels above the threshold of 500 µmol/L.

[Training of pharmacy personnel in the management of a centralized unit: the Oncolor network experience]. / Formation du personnel des pharmacies à la gestion d'une unité centralisée: expérience du réseau Oncolor.

The network of cancer care units in Lorraine area (Oncolor) developed management training for people working in chemotherapy units, and cytotoxic drug preparation. The programme was framed both for staff of executives (pharmacists), and technicians. Firstly, comparison between practices and theoretical recommendations lead to the elaboration of standardized operating procedures. Secondly, we elaborated a specific handbook for this education programme. A series of four-days independent sessions were organized for pharmacists and technicians. Each session combined theoretical and technical teaching for preparing antineoplastic drugs. Participants passing a successful final examination received a certificate from the Oncolor's network attesting their capacity to manage a chemotherapy unit. Four sessions were performed, with 35 participants. Only 31 passed at final examination. This preliminary experience will be enlarged to all members of the network and regularly brought up to date.