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Treatment of Patients wIth Niemann-Pick Type Is Using Repeated Amniotic Epithelial Cells Implantation: Correction of Aggregation and Coagulation Abnormalities

Laboratorio d'Emostasi, Istituto per l'lnfanzia Burlo Garofolo, via dell'Istria 65/1, 34137 Trieste, Italy; Department and Laboratory of Pediatrics, Children Hospital "Burlo Garofolo," Trieste, Italy

Marino Andolina, MD

Roberto Simeone, SB

Rita Boscolo, SB

Giovanni Ciana, MD

Bruno Bembi, MD

Department and Laboratory of Pediatrics, Children Hospital "Burlo Garofolo," Trieste, Italy

Variations of platelet aggregation and plasma levels of clotting factors V, IX, XI, and XII were studied in 5 patients with Niemann-Pick disease type Is in the course of a 3-year study of treatment with periodic subcutaneous infusions of amniotic epithelial cells. Before commencement of treatment, the concentrations of these factors were found to be abnormal in four of five patients. It was possible to complete the study protocol in only two patients. Platelet aggregation and plasma levels of V, IX, XI, and XII clotting factors had been determined before each epithelial amniotic cells implantation and after 24, 48, and 72 hours. In both patients the aggregation test and the plasma levels of coagulation factors V, IX, XI, and XII were below the normal values of reference. Results showed that the epithelial amniotic cells treatment normalized platelet aggregation after each implantation in the two studied patients, both in terms of intensity of response (increase in light transmission after addition of adenosine diphosphate up to 350%) and in terms of obtaining an irreversible aggregation with 3 and 8 pM of adenosine diphosphate. The data related to clotting factors showed an increase of these concentrations up to 60% and some of these concentrations normalized completely.

Clinical Pediatrics, Vol. 36, No. 3, 141-146 (1997)
DOI: 10.1177/000992289703600304


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