Evidence of Treatment Benefits in Patients with Mucopolysaccharidosis Type I-Hurler in Long-term Follow-up Using a New Magnetic Resonance Imaging Scoring System.

Adolescent Brain / diagnostic imaging Child Child, Preschool Female Follow-Up Studies Hematopoietic Stem Cell Transplantation Humans Magnetic Resonance Imaging Male Mucopolysaccharidosis I / diagnostic imaging Neuroimaging Retrospective Studies Spinal Cord / diagnostic imaging
ERT and MPS HSCT MPSI MPSI-H haematopoietic stem cell transplantation magnetic resonance neuroradiology and MPS

Journal

The Journal of pediatrics
ISSN: 1097-6833
Titre abrégé: J Pediatr
Pays: United States
ID NLM: 0375410

Informations de publication

Date de publication:
01 2022
Historique:
received: 06 01 2021
revised: 03 09 2021
accepted: 14 09 2021
pubmed: 22 9 2021
medline: 19 2 2022
entrez: 21 9 2021
Statut: ppublish

Résumé

We developed a brain and spine magnetic resonance scoring system based on a magnetic resonance assessment of 9 patients with mucopolysaccharidosis type I-Hurler who underwent hematopoietic stem-cell transplantation. The score is reliable and correlates with long-term clinical and cognitive outcome in patients with mucopolysaccharidosis type I-Hurler.

Identifiants

DOI: 10.1016/j.jpeds.2021.09.020 PMID: 34547335
pubmed: 34547335
pii: S0022-3476(21)00894-5
doi: 10.1016/j.jpeds.2021.09.020
pii:
doi:

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

297-301.e5

Informations de copyright

Copyright © 2021 Elsevier Inc. All rights reserved.

Auteurs

Silvia Pontesilli (S)

Department of Neuroradiology, IRCCS San Raffaele Scientific Institute, Milano, Italy.

Cristina Baldoli (C)

Department of Neuroradiology, IRCCS San Raffaele Scientific Institute, Milano, Italy.

Pasquale Anthony Della Rosa (PAD)

Department of Neuroradiology, IRCCS San Raffaele Scientific Institute, Milano, Italy.

Alessandro Cattoni (A)

Pediatric Department, Fondazione MBBM, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy.

Maria Ester Bernardo (ME)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milano, Italy; Vita-Salute San Raffaele University of Milano, Milano, Italy.

Pamela Meregalli (P)

Pediatric Department, Fondazione MBBM, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy.

Serena Gasperini (S)

Pediatric Department, Fondazione MBBM, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy.

Serena Motta (S)

Pediatric Department, Fondazione MBBM, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy.

Francesca Fumagalli (F)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milano, Italy.

Francesca Tucci (F)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milano, Italy.

Federica Baciga (F)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milano, Italy.

Giulia Consiglieri (G)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milano, Italy.

Francesco Canonico (F)

Department of Radiology, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy.

Paola De Lorenzo (P)

Centro Operativo di Ricerca Statistica, Fondazione Tettamanti, University of Milano-Bicocca, Monza, Italy.

Luisa Chiapparini (L)

Neuroradiology Unit, IRCCS Istituto Neurologico Carlo Besta, Milano, Italy.

Bernhard Gentner (B)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milano, Italy; Vita-Salute San Raffaele University of Milano, Milano, Italy.

Alessandro Aiuti (A)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milano, Italy; Vita-Salute San Raffaele University of Milano, Milano, Italy.

Andrea Biondi (A)

Pediatric Department, Fondazione MBBM, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy.

Attilio Rovelli (A)

Pediatric Department, Fondazione MBBM, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy.

Rossella Parini (R)

Pediatric Department, Fondazione MBBM, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milano, Italy. Electronic address: rossella.parini@unimib.it.

Articles similaires

[Redispensing of expensive oral anticancer medicines: a practical application].

Lisanne N van Merendonk, Kübra Akgöl, Bastiaan Nuijen
1.00
Humans Antineoplastic Agents Administration, Oral Drug Costs Counterfeit Drugs

Smoking Cessation and Incident Cardiovascular Disease.

Jun Hwan Cho, Seung Yong Shin, Hoseob Kim et al.
1.00
Humans Male Smoking Cessation Cardiovascular Diseases Female

Evaluation of Low-Value Services Across Major Medicare Advantage Insurers and Traditional Medicare.

Ciara Duggan, Adam L Beckman, Ishani Ganguli et al.
1.00
Humans United States Aged Cross-Sectional Studies Medicare Part C

Effectiveness of Virtual Yoga for Chronic Low Back Pain: A Randomized Clinical Trial.

Hallie Tankha, Devyn Gaskins, Amanda Shallcross et al.
1.00
Humans Yoga Low Back Pain Female Male

Classifications MeSH

questionsmedicales.fr › Personnes › Tranches d'âge › Adolescent › Evidence of Treatment Benefits in Patients...
questionsmedicales.fr › Système nerveux › Système nerveux central › Encéphale › Evidence of Treatment Benefits in Patients...
questionsmedicales.fr › Personnes › Tranches d'âge › Enfant › Evidence of Treatment Benefits in Patients...
questionsmedicales.fr › Personnes › Tranches d'âge › Enfant › Enfant d'âge préscolaire › Evidence of Treatment Benefits in Patients...
questionsmedicales.fr › Environnement et santé publique › Santé publique › Méthodes épidémiologiques › Caractéristiques des études épidémiologiques › Études épidémiologiques › Études de cohortes › Études de suivi › Evidence of Treatment Benefits in Patients...
questionsmedicales.fr › Procédures de chirurgie opératoire › Transplantation › Transplantation cellulaire › Transplantation de cellules souches › Transplantation de cellules souches hématopoïétiques › Evidence of Treatment Benefits in Patients...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Evidence of Treatment Benefits in Patients...
questionsmedicales.fr › Diagnostic › Techniques et procédures diagnostiques › Imagerie diagnostique › Tomographie › Imagerie par résonance magnétique › Evidence of Treatment Benefits in Patients...
questionsmedicales.fr › Maladies métaboliques et nutritionnelles › Maladies métaboliques › Erreurs innées du métabolisme › Maladies lysosomiales › Mucopolysaccharidoses › Mucopolysaccharidose de type I › Evidence of Treatment Benefits in Patients...
questionsmedicales.fr › Techniques d'investigation › Neuroimagerie › Evidence of Treatment Benefits in Patients...
questionsmedicales.fr › Environnement et santé publique › Santé publique › Méthodes épidémiologiques › Caractéristiques des études épidémiologiques › Études épidémiologiques › Études de cohortes › Études rétrospectives › Evidence of Treatment Benefits in Patients...
questionsmedicales.fr › Système nerveux › Système nerveux central › Moelle spinale › Evidence of Treatment Benefits in Patients...