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NEUROLOGY 2009;73:633-637
© 2009 American Academy of Neurology

Dopamine agonists in 6-pyruvoyl tetrahydropterin synthase deficiency

F. Porta, MD, A. Mussa, MD, D. Concolino, MD, M. Spada, MD, PhD and A. Ponzone, MD

From the Department of Pediatrics (F.P., A.M., M.S., A.P.), University of Torino, Italy; and Department of Pediatrics (D.C.), University "Magna Graecia" of Catanzaro, Italy.

Address correspondence and reprint requests to Prof. Alberto Ponzone, Department of Pediatrics, University of Torino, Piazza Polonia 94, 10126 Torino, Italy alberto.ponzone{at}unito.it

Objective: To report the efficacy, tolerability, and safety of the dopamine agonist pramipexole in a series of 5 patients affected by inherited 6-pyruvoyl tetrahydropterin synthase (PTPS) deficiency and needing l-3,4 dihydroxyphenylalanine (l-dopa) therapy.

Methods: Patients included 4 males and 1 female with ages ranging from 2 to 26 years. Their medication included tetrahydrobiopterin (BH4), 5-hydroxytryptophan, l-dopa, carbidopa, selegiline, and entacapone. All experienced residual symptoms of dopamine deficiency, movement and behavioral disability, and complications of l-dopa therapy, associated with fluctuating hyperprolactinemia. Patients had full assessment of clinical and biochemical condition, including evaluation by an adapted Unified Parkinson's Disease Rating Scale (UPDRS) and measurement of plasma prolactin (PRL) and catecholamines, before and after a 6-week trial with pramipexole. Pramipexole was administered twice daily as an adjunct to l-dopa therapy in dosages upwardly titrated, with a concurrent reduction of l-dopa dosage. Clinical follow-up went on for 1 year.

Results: Pramipexole was well tolerated by all patients, with marked improvement and stabilization of their clinical picture. The mean improvement on the total UPDRS score was 43% (range 33.3%–55.6%) from baseline. Diurnal profiles of plasma PRL normalized and plasma catecholamine levels lasted unchanged. The daily administrations of l-dopa were curtailed from 3 or 4 to 2, and the l-dopa dosage was reduced up to 40%.

Conclusions: The addition of pramipexole to the treatment of 6-pyruvoyl tetrahydropterin synthase deficiency improves the results of l-3,4 dihydroxyphenylalanine therapy. Similar benefits may be expected in other forms of inherited tetrahydrobiopterin deficiency.

Abbreviations: BH4 = tetrahydrobiopterin; 5-OH Trp = 5-hydroxytryptophan; Hb = hemoglobin; HVA = homovanillic acid; l-dopa = l-3,4 dihydroxyphenylalanine; ND = not done; PD = Parkinson disease; PRL = prolactin; PTPS = 6-pyruvoyl tetrahydropterin synthase; RBC = red blood cell; UPDRS = Unified Parkinson's Disease Rating Scale.

Disclosure: The authors report no disclosures.

Received December 18, 2008. Accepted in final form May 14, 2009.






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