Objective. The minimum clinically important difference (MCID) is a standard way of measuring clinical relevance. The objective of this work was to establish the MCID for the 6‐minute walking test (6minWT) and the Gross Motor Function Measure (GMFM‐88) in pediatric gait disorders. Methods. A cohort, pretest‐posttest study was conducted in a hospitalized care setting. A total of 182 patients with acquired brain injury (ABI) or cerebral palsy (CP) performed 20 robot‐assisted gait training sessions complemented with 20 sessions of physical therapy over 4 weeks. Separate MCIDs were calculated using 5 distribution‐based approaches, complemented with an anonymized survey completed by clinical professionals. Results. The MCID range for the 6minWT was 20‐38 m in the ABI cohort, with subgroup ranges of 20‐36 m for GMFCS I‐II, 23‐46 m for GMFCS III, and 24‐46 m for GMFCS IV. MCIDs for the CP population were 6‐23 m, with subgroup ranges of 4‐28 m for GMFCS I‐II, 9‐19 m for GMFCS III, and 10‐27 m for GMFCS IV. For GMFM‐88 total score, MCID values were 1.1%‐5.3% for the ABI cohort and 0.1%‐3.0% for the CP population. For dimension “D” of the GMFM, MCID ranges were 2.3%‐6.5% and 0.8%‐5.2% for ABI and CP populations, respectively. For dimension “E,” MCID ranges were 2.8%‐6.5% and 0.3%‐4.9% for ABI and CP cohorts, respectively. The survey showed a large interquartile range, but the results well mimicked the distribution‐based methods. Conclusions. This study identified for the first time MCID ranges for 6minWT and GMFM‐88 in pediatric patients with neurological impairments, offering useful insights for clinicians to evaluate the impact of treatments. Distribution‐based methods should be used with caution: methods based on pre‐post correlation may underestimate MCID when applied to patients with small improvements over the treatment period. Our results should be complemented with estimates obtained using consensus‐ and anchor‐based approaches.

Background The aim of this study was to determine the efficacy of the EAP regimen (etoposide, adriamycin and cisplatin) followed by the Machover schedule (fluorouracil and folinic acid) given as adjuvant treatment to patients with poor prognostic factors (N+ or T3/4). Patients and methods Before randomisation, the subjects were stratified on the basis of node involvement (N+ or N–) and the time from surgery to randomisation (≤21 days or >22 days). The surgical procedures for sub-total or total gastrectomy with D2 dissection were standardised among the participating centres. Results Between December 1992 and December 1997, 274 patients were enrolled: 137 in the treatment arm and 137 in the control arm. The majority of the patients (90%) were N+. After a median follow up of 66 months (range 2–83), the 5-year overall survival (OS) was 52% in the treatment arm and 48% in the control arm [hazard ratio (HR) 0.93; 95% confidence interval (CI) 0.65–1.34]; the 5-year disease-free survival (DFS) was 49% and 44%, respectively (HR: 0.83; 95% CI 0.59–1.17). Among the patients with N–/N+ (1–6), the 5-year OS was 61% in the treatment group and 60% in the control group; in those with N+ (1–6), it was 42% and 22%. The treatment was completed by 87% of patients. Drug-related grade 3/4 WHO toxicities included leukopenia (21%), nausea and vomiting (14%), mucositis (9%), neutropenia (3%) and thrombocytopenia (2%). There were two deaths due to sepsis. Conclusions Although our results are not statistically significant, there was a limited relative risk reduction in the patients receiving adjuvant therapy (17% in DFS and 7% in OS). The data suggest that D2 surgery may have a favourable impact on OS.

Objectives Neuroendocrine tumours (NETs) are heterogeneous neoplasms for which there is no standard treatment. We have previously proposed an effective polychemotherapy (5-fluorouracil, dacarbazine and epirubicin), which only produced objective responses of brief duration. The present study aimed to assess in a multidisciplinary manner the efficacy of the same regimen at intensified doses in patients with advanced NETs. Patients and methods Eighty-two consecutive patients entered the study, of whom 21 had inoperable, locally advanced disease and 61 had metastatic disease. Seventy-two patients were evaluated for objective, biochemical and subjective responses. Response rate, time to progression (TTP) and overall survival (OS) were evaluated based on histotype. Results An objective response was observed in 20 patients (intention-to-treat and standard analysis 24.4% and 27.8%, respectively). Complete biochemical and subjective responses were obtained in 25.1% and 38.9% of the cases. The median duration of treatment was 4 months and the objective responses had a median duration of 38 months. After a 60-month follow-up the median TTP and OS were 21 and 38 months, respectively. Conclusions Our polychemotherapy regimen is effective, with long duration, and is well tolerated both for gastroenteropancreatic and lung NETs, as well as for tumours with a more aggressive clinical behaviour. The new WHO endocrine tumour histotyping, examining also the tumour biology, may give additional information for selecting patients to chemotherapy.

The aims of this study are to quantify the movement limitation of upper limbs in hemiplegic children with traumatic brain injury (TBI) by using a clinical-functional scale and upper limb kinematics and to evaluate the effectiveness of constraint-induced movement therapy (CIMT) on upper limbs. Pre-post study. Clinical rehabilitation research laboratory. Ten children with TBI. The participants were evaluated by clinical examinations (Gross Motor Function Measure, Besta scale, Quality of Upper Extremities Skills Test, and Manual Ability Classification System) and 3D kinematic movement analysis of the upper limb before the CIMT program (pretest: 0.7 years after the injury) and at the end of the program (posttest: 10 weeks later). After the CIMT, most of the clinical measures improved significantly. Some significant improvements were present in terms of kinematics, in particular, in the movement duration and the velocity of movement execution of both tasks; the index of curvature and the average jerk improved, respectively, during reaching and hand-to-mouth task, while the adjusting sway parameter decreased during the 2 movements. Significant improvements were found in upper limb joint excursion after the rehabilitative programme too. Our results suggest that the CIMT program can improve movement efficiency and upper limb function in children after TBI. The integration of the clinical outcomes and upper limb kinematics revealed to be crucial in detecting the effects of the CIMT programme.

Summary: Background: Acquired brain injury (ABI) may result in lifelong impairment of physical, cognitive, and psychosocial functions. Several rehabilitative treatments are often needed to support walking recovery, thus participants’ engagement becomes a crucial aspect, especially when patients are children. In the last few years, traditional physiotherapy (PT) has been flanked by innovative technologies for rehabilitation in the fields of robotics and Virtual Reality (VR). Preliminary results have shown interesting perspectives in the use of a VR system, the GRAIL (Gait Real-time Analysis Interactive Lab), in improving walking abilities in a small group of children with ABI, although further insights are needed about its use as rehabilitative tool in the pediatric population.Objectives: To evaluate the efficacy of a rehabilitation treatment on a GRAIL system for the improvement of walking abilities, in a group of children suffering from ABI.Methods: 12 children with ABI (study group – SG; mean age = 12.1 ± 3.8 years old) underwent a 10-session treatment with the GRAIL, an instrumented multi-sensor platform based on immersive VR for gait training and rehabilitation in engaging VR environments. Before (T0) and at the end of the treatment (T1), the participants were assessed by means of functional scales (Gross Motor Function Measure (GMFM), Functional Assessment Questionnaire (FAQ), 6-Minute Walk Test (6minWT) and the 3D-Gait Analysis, over ground (OGA) and on GRAIL (GGA).Results: All the participants completed the rehabilitative treatment. The functional evaluations showed an improvement in Gross Motor abilities (GMFM-88, p = 0.008), especially in standing (GMFM-D, p = 0.007) and walking (GMFM-E, p = 0.005), an increase of the endurance (6minWT, p = 0.002), and enhanced autonomy in daily life activities (FAQ, p = 0.025). OGA identified a significant decrease of the Gillette Gait Index for the impaired side and a general increase of symmetry. GGA showed improvements in spatiotemporal parameters and joints range of motion that moved towards normality and symmetry recovery.Conclusions: A 10-session treatment with GRAIL on children with ABI led to improvements in their walking abilities and enhanced their engagement during the training. This is desirable when long life impairments are faced and children’s motor functions have to be regained and it supports the leading role that VR might have in the rehabilitation field.