Ruxolitinib in Aicardi-Goutières syndrome.

Aicardi-Goutières Syndrome (AGS) is a monogenic leukodystrophy with pediatric onset, clinically characterized by a variable degree of neurologic impairment. It belongs to a group of condition called type I interferonopathies that are characterized by abnormal overproduction of interferon alpha, an inflammatory cytokine which action is mediated by the activation of two of the four human Janus Kinases. Thanks to an ever-increasing knowledge of the molecular basis and pathogenetic mechanisms of the disease, Janus Kinase inhibitors (JAKIs) have been proposed as a treatment option for selected interferonopathies.

Comparison between an Ascenda and a silicone catheter in intrathecal baclofen therapy in pediatric patients: analysis of complications.

OBJECTIVE In this single-center study the authors investigated the complications occurring before and after the introduction of the new Ascenda intrathecal catheter (Medtronic Inc.) in pediatric patients treated with intrathecal baclofen therapy (ITB) for spasticity and/or dystonia. METHODS This was a retrospective review of 508 children who had received ITB, 416 with silicone catheters in the 13 years between September 1998 and September 2011 and 92 with Ascenda catheters in the 3 years between September 2011 and August 2014.

Analysis of complications in 430 consecutive pediatric patients treated with intrathecal baclofen therapy: 14-year experience.

Object: This single-center study investigated adverse events that occurred in children and adolescent patients treated with intrathecal baclofen (ITB) therapy for spasticity and/or dystonia.

Methods: In a 14-year period, 430 consecutive patients with a mean age of 13.3 ± 5.

Intrathecal baclofen and motor function in cerebral palsy.

Aim: The aim of this retrospective analysis was to determine the impact of intrathecal baclofen (ITB) therapy on motor function in patients with cerebral palsy (CP).

Method: We studied 37 patients (18 males, 19 females) with CP treated with ITB (mean age at implant 13 y 7 mo, SD 7 y). Eighteen patients were affected by spastic diplegia, 12 by spastic quadriplegia, six by dystonic quadriplegia, and one by hemidystonia.

Forced-use, without therapy, in children with hemiplegia: preliminary study of a new approach for the upper limb.

Background: Forced use (FU) is an emerging treatment for children with hemiplegic cerebral palsy (CP). It involves constraining the unaffected arm and no additional treatment of the affected arm. Our study examined a new approach to FU in children with hemiplegic CP: that is, restraint of the unaffected limb and no rehabilitation.

Upper limbs function after intrathecal baclofen therapy in children with secondary dystonia.

Background: Different studies show the efficacy of intrathecal baclofen therapy (ITB) in patients with secondary dystonia because of cerebral palsy. These studies only report improvements related to dystonia reduction; however, none of them have assessed whether such benefit is reflected in limb function. The purpose of our study is to determine whether the effects ITB therapy in patients with secondary dystonia to cerebral palsy, in addition to reducing dystonia, may also improve upper limb function.

Effect of intrathecal baclofen on dystonia in children with cerebral palsy and the use of functional scales.

Background: The literature available shows that spasticity in patients with cerebral palsy (CP) is reduced by intrathecal baclofen (ITB) treatment, and various studies suggest that this treatment can also be used in dystonic patients. The aim of the present study was to evaluate dystonia treated with ITB in children with CP.

Methods: Data of 19 patients affected by dystonia as an outcome of infant CP in patients belonging to level V of Gross Motor Function Classification System treated with ITB were collected.

Upper limb function after intrathecal baclofen treatment in children with cerebral palsy.

Background: The literature shows that intrathecal baclofen (ITB) treatment in patients with cerebral palsy (CP) is able to reduce spasticity. The purpose of this work is to evaluate the motor function of the upper limbs in patients with CP treated with ITB.

Methods: A consecutive series of 20 patients with spastic CP (mean age at implant, 11.