Frontiers in Pediatrics (May 2019)
Propranolol 0.2% Eye Micro-Drops for Retinopathy of Prematurity: A Prospective Phase IIB Study
- Luca Filippi,
- Giacomo Cavallaro,
- Giacomo Cavallaro,
- Elettra Berti,
- Letizia Padrini,
- Gabriella Araimo,
- Giulia Regiroli,
- Giulia Regiroli,
- Genny Raffaeli,
- Genny Raffaeli,
- Valentina Bozzetti,
- Paolo Tagliabue,
- Barbara Tomasini,
- Annalisa Mori,
- Giuseppe Buonocore,
- Massimo Agosti,
- Angela Bossi,
- Gaetano Chirico,
- Salvatore Aversa,
- Pina Fortunato,
- Silvia Osnaghi,
- Barbara Cavallotti,
- Martina Suzani,
- Maurizio Vanni,
- Giulia Borsari,
- Simone Donati,
- Giuseppe Nascimbeni,
- Daniel Nardo,
- Stefano Piermarocchi,
- Giancarlo la Marca,
- Giulia Forni,
- Silvano Milani,
- Ivan Cortinovis,
- Maura Calvani,
- Paola Bagnoli,
- Massimo Dal Monte,
- Anna Maria Calvani,
- Alessandra Pugi,
- Eduardo Villamor,
- Gianpaolo Donzelli,
- Fabio Mosca,
- Fabio Mosca
Affiliations
- Luca Filippi
- Neonatal Intensive Care Unit, Medical Surgical Fetal-Neonatal Department, “A. Meyer” University Children's Hospital, Florence, Italy
- Giacomo Cavallaro
- Neonatal Intensive Care Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy
- Giacomo Cavallaro
- Department of Clinical Sciences and Community Health, University of Milan, Milan, Italy
- Elettra Berti
- Neonatal Intensive Care Unit, Medical Surgical Fetal-Neonatal Department, “A. Meyer” University Children's Hospital, Florence, Italy
- Letizia Padrini
- Neonatal Intensive Care Unit, Medical Surgical Fetal-Neonatal Department, “A. Meyer” University Children's Hospital, Florence, Italy
- Gabriella Araimo
- Neonatal Intensive Care Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy
- Giulia Regiroli
- Neonatal Intensive Care Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy
- Giulia Regiroli
- Department of Clinical Sciences and Community Health, University of Milan, Milan, Italy
- Genny Raffaeli
- Neonatal Intensive Care Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy
- Genny Raffaeli
- Department of Clinical Sciences and Community Health, University of Milan, Milan, Italy
- Valentina Bozzetti
- Neonatal Intensive Care Unit, MBBM Foundation, San Gerardo Hospital, Monza, Italy
- Paolo Tagliabue
- Neonatal Intensive Care Unit, MBBM Foundation, San Gerardo Hospital, Monza, Italy
- Barbara Tomasini
- Neonatal Intensive Care Unit, University Hospital of Siena, Policlinico Santa Maria alle Scotte, Siena, Italy
- Annalisa Mori
- Department of Molecular and Developmental Medicine, University of Siena, Siena, Italy
- Giuseppe Buonocore
- Department of Molecular and Developmental Medicine, University of Siena, Siena, Italy
- Massimo Agosti
- Neonatal Intensive Care Unit, Del Ponte Hospital, Varese, Italy
- Angela Bossi
- Neonatal Intensive Care Unit, Del Ponte Hospital, Varese, Italy
- Gaetano Chirico
- Neonatal Intensive Care Unit, Children's Hospital, University Hospital “Spedali Civili” of Brescia, Brescia, Italy
- Salvatore Aversa
- Neonatal Intensive Care Unit, Children's Hospital, University Hospital “Spedali Civili” of Brescia, Brescia, Italy
- Pina Fortunato
- Pediatric Ophthalmology, “A. Meyer” University Children's Hospital, Florence, Italy
- Silvia Osnaghi
- 0Department of Ophthalmology, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Università degli Studi di Milan, Milan, Italy
- Barbara Cavallotti
- 1Department of Ophthalmology, ASST Monza, San Gerardo Hospital, Monza, Italy
- Martina Suzani
- 1Department of Ophthalmology, ASST Monza, San Gerardo Hospital, Monza, Italy
- Maurizio Vanni
- 2Pediatric Ophthalmology, University Hospital of Siena, Policlinico Santa Maria alle Scotte, Siena, Italy
- Giulia Borsari
- 2Pediatric Ophthalmology, University Hospital of Siena, Policlinico Santa Maria alle Scotte, Siena, Italy
- Simone Donati
- 3Department of Surgical and Morphological Sciences, Section of Ophthalmology, University of Insubria, Varese, Italy
- Giuseppe Nascimbeni
- 4Department of Ophthalmology, University Hospital “Spedali Civili” of Brescia, Brescia, Italy
- Daniel Nardo
- 5Department of Women's and Children's Health, Azienda Ospedaliera di Padova, University of Padua, Padua, Italy
- Stefano Piermarocchi
- 6Department of Ophthalmology, University of Padua, Padua, Italy
- Giancarlo la Marca
- 7Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital, Florence, Italy
- Giulia Forni
- 7Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital, Florence, Italy
- Silvano Milani
- 8Laboratory “G.A. Maccacro,” Department of Clinical Sciences and Community Health, University of Milan, Milan, Italy
- Ivan Cortinovis
- 8Laboratory “G.A. Maccacro,” Department of Clinical Sciences and Community Health, University of Milan, Milan, Italy
- Maura Calvani
- 9Oncohematology Unit, Department of Pediatric Oncology, “A. Meyer” University Children's Hospital, Florence, Italy
- Paola Bagnoli
- 0Department of Biology, Unit of General Physiology, University of Pisa, Pisa, Italy
- Massimo Dal Monte
- 0Department of Biology, Unit of General Physiology, University of Pisa, Pisa, Italy
- Anna Maria Calvani
- 1Department of Pharmacy, “A. Meyer” University Children's Hospital, Florence, Italy
- Alessandra Pugi
- 2Clinical Trial Office, “A. Meyer” University Children's Hospital, Florence, Italy
- Eduardo Villamor
- 3Department of Pediatrics, Maastricht University Medical Center, School for Oncology and Developmental Biology, Maastricht, Netherlands
- Gianpaolo Donzelli
- Neonatal Intensive Care Unit, Medical Surgical Fetal-Neonatal Department, “A. Meyer” University Children's Hospital, Florence, Italy
- Fabio Mosca
- Neonatal Intensive Care Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy
- Fabio Mosca
- Department of Clinical Sciences and Community Health, University of Milan, Milan, Italy
- DOI
- https://doi.org/10.3389/fped.2019.00180
- Journal volume & issue
-
Vol. 7
Abstract
Background: Oral propranolol reduces retinopathy of prematurity (ROP) progression, although not safely. Propranolol 0.1% eye micro-drops administered to newborns with stage 2 ROP are well-tolerated, but not sufficiently effective.Methods: A multi-center open-label trial was conducted to assess the safety and efficacy of propranolol 0.2% eye micro-drops in newborns with stage 1 ROP. The progression of the disease was evaluated with serial ophthalmologic examinations. Hemodynamic, respiratory, biochemical parameters, and propranolol plasma levels were monitored. Demographic and perinatal characteristics, co-morbidities and co-intervention incidences, together with ROP progression, were compared with a historical control group in the same centers participating in the trial.Results: Ninety-eight newborns were enrolled and compared with the historical control group. Populations were not perfectly homogeneous (as demonstrated by the differences in the Apgar score and the different incidence rate in surfactant administration and oxygen exposure). The progression to ROP stage 2 or 3 plus was significantly lower than the incidence expected on the basis of historical data (Risk Ratio 0.521, 95% CI 0.297– 0.916). No adverse effects related to propranolol were observed and the mean propranolol plasma level was significantly lower than the safety cut-off of 20 ng/mL. Unexpectedly, three newborns treated with oral propranolol before the appearance of ROP, showed a ROP that was unresponsive to propranolol eye micro-drops and required laser photocoagulation treatment.Conclusion: Propranolol 0.2% eye micro-drops were well-tolerated and appeared to reduce the ROP progression expected on the basis of a comparison with a historical control group. Propranolol administered too early appears to favor a more aggressive ROP, suggesting that a β-adrenoreceptor blockade is only useful during the proliferative phase. Further randomized placebo-controlled trials are required to confirm the current results.Clinical Trial Registration The trial was registered at ClinicalTrials.gov with Identifier NCT02504944 and with EudraCT Number 2014-005472-29.
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