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- Title
A comparison of immediate release and delayed release cysteamine in 17 patients with nephropathic cystinosis.
- Authors
van Stein, Christina; Klank, Sabrina; Grüneberg, Marianne; Ottolenghi, Chris; Grebe, Jürgen; Reunert, Janine; Harms, Erik; Marquardt, Thorsten
- Abstract
<bold>Background: </bold>Nephropathic cystinosis is a rare and severe metabolic disease leading to an accumulation of cystine in lysosomes which especially harms kidney function. A lifelong therapy with the aminothiol cysteamine can delay the development of end-stage renal disease and the necessity of kidney transplantation. The purpose of our study was to compare the effectiveness of immediate-release and delayed-release cysteamine on cystine and cysteamine levels as well as assessing the onset of adverse effects.<bold>Methods: </bold>We retrospectively analysed cystine and cysteamine levels of 17 patients after a single dose of immediate-release cysteamine (Cystagon®, Mylan Pharmaceuticals, Canonsburg, PA and Recordati Pharma GmbH) as well as a single dose of delayed-release cysteamine (Procysbi®; Horizon Pharma USA and Chiesi Farmaceutici S.p.A., Parma, Italy) respectively. Data were collected during a period of three years in the context of optimizing the individual treatment regimens. The dose of DR-cysteamine was reduced to 70% of the equivalent dose of IR-cysteamine. The efficacy of both formulas in depleting white blood cells' cystine levels and their side effects were compared.<bold>Results: </bold>Immediate (IR)- and delayed-release (DR) cysteamine effectively decreased intracellular cystine levels under the target value of 0.5 nmol cystine/mg protein, while fewer side effects occurred under DR-cysteamine. Mean maximum levels of cysteamine were reached after 60 min with IR-cysteamine and after 180 min with DR-cysteamine.<bold>Conclusion: </bold>A therapy with DR-cysteamine is as effective as IR-cysteamine while less side effects were reported. Our data show that DR-cysteamine should be dosed higher than 70% of the equivalent dose of IR-cysteamine in order to decrease cystine levels over an extended period of time. Moreover, our data suggest increasing the dosing scheme of Procysbi® to three times daily, to prevent a rapid decrease and achieve a steadier decline in cystine levels. Due to the more convenient dosing scheme, DR-cysteamine might ameliorate therapy adherence and improve patients' quality of life.
- Subjects
PARMA (Italy); CYSTEAMINE; LEUCOCYTES; CHRONIC kidney failure; PATIENT compliance; BASILIXIMAB; QUALITY of life; LYSOSOMES; RAPAMYCIN
- Publication
Orphanet Journal of Rare Diseases, 2021, Vol 16, Issue 1, p1
- ISSN
1750-1172
- Publication type
journal article
- DOI
10.1186/s13023-021-01991-2