A 2-year-old 10-month-old female patient diagnosed with NP-C 2 years later.
In April 2012 she started treatment with HPBCD.
All doses are administered intrathecally in bolus every 15 days starting at an initial dose of 175 mg followed by a subsequent dose of 250 mg, then increased to 300 mg once a new dose of 400 mg every other dose was then increased to 350 mg once
The medication management protocol has been adapted from the original Dr. Bailey protocol.
Caroline Hastings, USA3.
Intravenous administration was ruled out in our patient due to advanced symptoms and the fact that intravenous administration does not allow the medication to cross the neurological blood-brain barrier.
The name of the medicine is Trappsol® (LABORATORIO: Cyclodextrin Technologies Development).
It is available as a non-sterile powder.
The procedures for acquiring the medicine are carried out through the Spanish Agency for Medicines and Health Products, requesting the importation through the website of Drug Management in special situations.
The request is made by patient, providing medical report, authorization of the center where the treatment is to be administered and printed use of medications without indications.
The patient and/or guardian should sign the corresponding informed consent.
Confirmation of supply by the manufacturer's laboratory to the corresponding hospital must be attached to the Ministry for approval of the Spanish Medicines Agency.
The lab requires a payment by default to import the medication.
The preparation of all preparations was carried out in the Service Establishment following the rules for the correct elaboration and quality control of the formulas and office preparations, in adaptation to the sterile flow container bedroom R 175/2001.
All material used for weighing the medication was previously sterilized.
Weight and height were measured under aseptic conditions in a cleaning room ( ISO 5).
0.9% sequestration is performed with physiological serum for the dose of 175 mg; and in SF 0.675 % for the dose of 375 mg and 525 mg. Final sterility is achieved in a horizontal philate concentration 0.22 mg.
To date, he has received a total of 22 doses and has been able to stabilize the disease in terms of neurological sequelae.
The patient was in a similar clinical situation according to the evolution of the NPC clinical severity scale, but with improvement in auditory brainstem potentials.
The patient had two seizures with fever; it is unknown whether they are due to the disease itself or to medication.
