Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy

Valeria Calbi; Francesca Fumagalli; Giulia Consiglieri; Rachele Penati; Serena Acquati; Daniela Redaelli; Vanessa Attanasio; Facchini Marcella; Maria Pia Cicalese; Maddalena Migliavacca; Federica Barzaghi; Francesca Ferrua; Andrea Assanelli; Paolo Silvani; Matteo Zoccolillo; Giovanna Chidini; Robert Chiesa; Ruchi Arora; Francesca Ciotti; Marina Sarzana; Gigliola Antonioli; Cristina Baldoli; Francesco Morena; Sabata Martino; Gian Luigi Ardissino; Maria Grazia Natali Sora; Luigi Naldini; Fabio Ciceri; Alessandro Aiuti; Maria Ester Bernardo

doi:10.1038/s41409-017-0085-1

Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy

Bone Marrow Transplant. 2018 Jul;53(7):913-917. doi: 10.1038/s41409-017-0085-1. Epub 2018 Jan 29.

Authors

Valeria Calbi^{1

2}, Francesca Fumagalli^{1

2

3}, Giulia Consiglieri^{2

4}, Rachele Penati², Serena Acquati¹, Daniela Redaelli¹, Vanessa Attanasio¹, Facchini Marcella¹, Maria Pia Cicalese^{1

2}, Maddalena Migliavacca^{1

2}, Federica Barzaghi^{1

2}, Francesca Ferrua^{1

2

4}, Andrea Assanelli^{2

5}, Paolo Silvani⁶, Matteo Zoccolillo¹, Giovanna Chidini⁷, Robert Chiesa⁸, Ruchi Arora⁹, Francesca Ciotti², Marina Sarzana², Gigliola Antonioli^{1

2}, Cristina Baldoli¹⁰, Francesco Morena¹¹, Sabata Martino¹¹, Gian Luigi Ardissino¹², Maria Grazia Natali Sora³, Luigi Naldini^{1

4}, Fabio Ciceri^{4

5}, Alessandro Aiuti^{13

14

15}, Maria Ester Bernardo^{1

2}

Affiliations

¹ San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
² Pediatric Immunohematology Unit and Stem Cell Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.
³ Neurology Department, IRCCS San Raffaele Scientific Institute, Milan, Italy.
⁴ Vita-Salute San Raffaele University, Milan, Italy.
⁵ Unit of Hematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, Milan, Italy.
⁶ Department of Anesthesia and Critical Care, IRCCS San Raffaele Scientific Institute, Milan, Italy.
⁷ Pediatric Intensive Care Unit, Department of Anesthesia and Critical Care, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy.
⁸ Bone Marrow Transplantation Unit, Great Ormond Street Hospital, London, UK.
⁹ Department of Paediatrics, Norfolk and Norwich University Hospital, Norwich, UK.
¹⁰ Neuroradiology Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
¹¹ Department of Chemistry, Biology and Biotechnologies, University of Perugia, Perugia, Italy.
¹² HUS Center, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
¹³ San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy. aiuti.alessandro@hsr.it.
¹⁴ Pediatric Immunohematology Unit and Stem Cell Program, IRCCS San Raffaele Scientific Institute, Milan, Italy. aiuti.alessandro@hsr.it.
¹⁵ Vita-Salute San Raffaele University, Milan, Italy. aiuti.alessandro@hsr.it.

No abstract available

Publication types

Case Reports

MeSH terms

Genetic Therapy / methods*
Hematopoietic Stem Cell Transplantation / methods*
Humans
Leukodystrophy, Metachromatic / drug therapy*
Leukodystrophy, Metachromatic / pathology
Leukodystrophy, Metachromatic / therapy*
Male
Platelet Aggregation Inhibitors / pharmacology
Platelet Aggregation Inhibitors / therapeutic use*
Polydeoxyribonucleotides / pharmacology
Polydeoxyribonucleotides / therapeutic use*
Transplantation Conditioning / methods*
Twins

Substances

Platelet Aggregation Inhibitors
Polydeoxyribonucleotides
defibrotide