Jérémy Jost1, Claire Bahans1, Marie Courbebaisse1, Tu-Anh Tran1, Agnès Linglart1, Karelle Benistan1, Anne Lienhardt1, Hadile Mutar1, Elodie Pfender1, Voa Ratsimbazafy1, Vincent Guigonis1. 1. Pharmacie (J.J., V.R.), Inserm, Université de Limoges, Centre Hospitalo Universitaire de Limoges, Unité Mixte de Recherche Scientifique 1094, Institut d'Epidémiologie Neurologique et de Neurologie tropicale, Centre National de la Recherche Scinetifique Fédération de Recherche 3503 Génomique, Environnement, Immunité, Santé et Thérapie Pédiatrie (C.B., A.Lie., H.M., V.G.), Comité de l'HME pour la Recherche Clinique Centre Hospitalier Universitaire; and Centre d'Investigation Clinique (E.P.), Centre Hospitalier Universitaire, 87000 Limoges, France; Assistance Publique-Hôpitaux de Paris (M.C.), Hôpital Européen Georges Pompidou, Département de Physiologie; Université Paris Descartes, Faculté de Médecine; and Institut Necker Enfants-Malades, Inserm Unité 1151, Centre National de la Recherche Scientifique Unité Mixte de Recherche 8253, 75000 Paris, France; Pédiatrie (T.-A.T.), Centre Hospitalier Universitaire de Nîmes; Inserm Unité 1183, Faculté de Médecine Montpellier-Nîmes, 30000 Nîmes France; Assistance Publique-Hôpitaux de Paris (A.Lin.), Centre de Référence des Maladies Rare du Métabolisme Phosphocalcique et Plateforme d'Expertise Paris Sud Maladies Rares; and Inserm Unité 1169, Le Kremlin Bicêtre 94270, France; Génétique Médicale (K.B.), Hôpital Raymond Poincaré, 92380 Garches, France; and Centre National de la Recherche Scinetifique Unité Mixte de Recherche 7276 (V.G.), Université de Limoges, 87000 Limoges, France.
Abstract
CONTEXT: Hyperphosphatemic familial tumoral calcinosis (HFTC) and hyperphosphatemia hyperostosis syndrome (HHS) are rare diseases characterized by hyperphosphatemia and ectopic calcifications or recurrent episodes of diaphysitis. In the setting of metabolic or inflammatory diseases, recent data suggest that systemic administration of sodium thiosulfate (STS) could be effective in the treatment of ectopic calcifications but may also be poorly tolerated (digestive symptoms, metabolic acidosis). Our group developed a topical formulation of STS to treat ectopic calcifications locally, therefore limiting patient exposure to the drug and its adverse effects. OBJECTIVE: We aimed at describing efficacy and tolerance for a topical formulation of STS in treated patients. DESIGN: We performed a retrospective study wherein clinical, radiological, and biological data before and after the application of the topical STS treatment were collected and analyzed. PATIENTS OR OTHER PARTICIPANTS: Three patients admitted to 3 different hospitals with an ectopic calcification secondary to HFTC or HHS were treated with topical STS. INTERVENTION: The topical STS was applied daily by the patients. RESULTS: A significant clinical and radiological decrease of ectopic calcifications was observed after at least 5 months of treatment. The STS treatment was well tolerated and no clinical or biological side effects were observed. CONCLUSION: Topical STS appears to be a promising treatment for ectopic calcifications secondary to HFTC or HHS.
CONTEXT: Hyperphosphatemic familial tumoral calcinosis (HFTC) and hyperphosphatemia hyperostosis syndrome (HHS) are rare diseases characterized by hyperphosphatemia and ectopic calcifications or recurrent episodes of diaphysitis. In the setting of metabolic or inflammatory diseases, recent data suggest that systemic administration of sodium thiosulfate (STS) could be effective in the treatment of ectopic calcifications but may also be poorly tolerated (digestive symptoms, metabolic acidosis). Our group developed a topical formulation of STS to treat ectopic calcifications locally, therefore limiting patient exposure to the drug and its adverse effects. OBJECTIVE: We aimed at describing efficacy and tolerance for a topical formulation of STS in treated patients. DESIGN: We performed a retrospective study wherein clinical, radiological, and biological data before and after the application of the topical STS treatment were collected and analyzed. PATIENTS OR OTHER PARTICIPANTS: Three patients admitted to 3 different hospitals with an ectopic calcification secondary to HFTC or HHS were treated with topical STS. INTERVENTION: The topical STS was applied daily by the patients. RESULTS: A significant clinical and radiological decrease of ectopic calcifications was observed after at least 5 months of treatment. The STS treatment was well tolerated and no clinical or biological side effects were observed. CONCLUSION: Topical STS appears to be a promising treatment for ectopic calcifications secondary to HFTC or HHS.
Authors: Q-Y Zuo; X Cao; B-Y Liu; D Yan; Z Xin; X-H Niu; C Li; W Deng; Z-Y Dong; J-K Yang Journal: J Endocrinol Invest Date: 2019-09-18 Impact factor: 4.256
Authors: M Chakhtoura; M S Ramnitz; N Khoury; G Nemer; N Shabb; A Abchee; A Berberi; M Hourani; M Collins; S Ichikawa; G El Hajj Fuleihan Journal: Osteoporos Int Date: 2018-06-20 Impact factor: 4.507