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CASE REPORT article

Front. Med.
Sec. Gene and Cell Therapy
Volume 11 - 2024 | doi: 10.3389/fmed.2024.1433217

Oral ATP treatment in alternating hemiplegia of childhood. (A case report and review)

Provisionally accepted
Marco Carrozzi Marco Carrozzi 1Maria Elisa Morelli Maria Elisa Morelli 1Mario Cirino Mario Cirino 2*Alessandra Maestro Alessandra Maestro 2Gilda Paternuosto Gilda Paternuosto 2Giulia Benericetti Giulia Benericetti 2Giada Bennati Giada Bennati 2Anna Flamigni Anna Flamigni 2Federico Pigato Federico Pigato 2Natalia Maximova Natalia Maximova 3Egidio Barbi Egidio Barbi 4,5Davide Zanon Davide Zanon 2
  • 1 Child Neuropsychiatry Unit, Department of Pediatrics, Institute for Maternal and Child Health Burlo Garofolo (IRCCS), Trieste, Italy
  • 2 Department of Pharmacy, Institute for Maternal and Child Health Burlo Garofolo (IRCCS), Trieste, Italy
  • 3 Oncohematology Unit, Department of Pediatrics, Institute for Maternal and Child Health Burlo Garofolo (IRCCS), Trieste, Friuli-Venezia Giulia, Italy
  • 4 Department of Pediatrics, Institute for Maternal and Child Health Burlo Garofolo (IRCCS), Trieste, Friuli-Venezia Giulia, Italy
  • 5 Department of Medicine, Surgery and Health Sciences, University of Trieste, Trieste, Italy

The final, formatted version of the article will be published soon.

    Alternating hemiplegia of childhood (AHC) is a rare neurological disorder that usually manifests before 18 months of age and is characterized by recurrent, alternating episodes of hemiparesis with variable frequency and can last from a few minutes to several days. We present a case of AHC in a little girl carrying a sporadic mutation in the ATP1A3 gene (p.Glu815Lys) refractory to flunarizine and non-compliant to topiramate due to adverse effects treated with oral compound of adenosine-5'triphosphate (ATP) capsules. Outcome was evaluated through the follow-up and side effects and safety were monitored regularly. Compounded drug showed effectiveness and safety. Indeed, during the fouryear follow-up, with the dose of adenosine-5'-triphosphate gradually increasing up to 21 mg/kg, the patient showed a substantial benefit in controlling the frequency and duration of hemiplegic episodes and an improvement in neurological deterioration.

    Keywords: Adenosine-5'-triphosphate, ATP, Alternating hemiplegia of childhood, rare disease, Compound, clinical galenic

    Received: 27 May 2024; Accepted: 11 Dec 2024.

    Copyright: © 2024 Carrozzi, Elisa Morelli, Cirino, Maestro, Paternuosto, Benericetti, Bennati, Flamigni, Pigato, Maximova, Barbi and Zanon. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

    * Correspondence: Mario Cirino, Department of Pharmacy, Institute for Maternal and Child Health Burlo Garofolo (IRCCS), Trieste, Italy

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