Pillon M.,University of Padua |
Mussolin L.,University of Padua |
Mussolin L.,Institute of Paediatric Research Fondazione Citta della Speranza |
Carraro E.,University of Padua |
And 16 more authors.
British Journal of Haematology | Year: 2016
Burkitt lymphoma (BL) and Diffuse Large B-Cell Lymphoma (DLBCL) account for most cases of non-Hodgkin lymphoma (NHL) in childhood. We report the clinical characteristics, outcome and prognostic factors in children with BL or DLBCL treated according to the Associazione Italiana Ematologia Oncologia Pediatrica (AIEOP) LNH-97 protocol. Patients aged up to 18 years that were newly diagnosed with BL/DLBCL were included in the study. Therapy consisted of pre-phase followed by 2–6 high-dose chemotherapy courses tailored according to lactate dehydrogenase (LDH) value and disease stage. A total of 442 patients (379 BL, 63 DLBCL) were enrolled between 1997 and 2014, of whom 18 failed to achieve remission, 6 experienced treatment-related death, 2 developed second malignancy and 20 relapsed. At a median follow-up time of 5 years, overall survival was 93% (±1%) and event-free survival was 90% (±1%). LDH value above the median value had an independently negative prognostic value (P < 0·0001). However, in the subgroup of 128 patients in which minimal disseminated disease (MDD) was analysed, MDD-positivity became the only unfavourable prognostic factor for progression-free survival. Tailored chemotherapy could be extremely effective with limited toxicity. Identification of MDD as a hallmark of a higher risk of treatment failure may provide a target population for treatment intensification by anti-CD20. © 2016 John Wiley & Sons Ltd
Tochon M.,Center Hospitalier University Timone Enfants |
Bosdure E.,Center Hospitalier University Timone Enfants |
Salles M.,Center Hospitalier University Timone Enfants |
Salles M.,Center Hospitalier |
And 9 more authors.
International Journal of Tuberculosis and Lung Disease | Year: 2011
SETTING: Drug-resistant tuberculosis (DR-TB) is increasing worldwide and may be a source of diagnostic and therapeutic problems in young exposed children. In France exposed children are systematically treated with 3-month isoniazid-rifampicin prophylaxis. OBJECTIVE: To describe the characteristics and management of children aged <2 years in contact with an adult case of DR-TB in France over a 5-year period (2004-2008). METHODS: Children were retrospectively identified by sending questionnaires to all the members of the Paediatric Infectious Diseases Group and the Paediatric Pulmonology Group of the French Paediatric Society. RESULTS: Ten children, all infants, in contact with an adult case of DR-TB were identified: six cases of DR-TB (mean age 4.6 months), one case of TB infection and three cases of exposure (mean age 3.1 months). The children were mainly in contact with poly- or multidrug-resistant TB. Time to initiation of appropriate treatment was 39 days for TB disease and 58 days for TB infection or exposure. One child with TB infection developed TB disease due to failure to adapt prophylaxis. Treatment was variable and centre-dependent. Short-term follow-up showed complete recovery of all children. CONCLUSION: Management of young children in contact with adult DR-TB requires rapid identification of the drug resistance profile. Molecular techniques should be used to reduce delays in initiating appropriate treatment. © 2011 The Union.
Benfer K.A.,University of Queensland |
Benfer K.A.,Paediatric Unit |
Weir K.A.,University of Queensland |
Weir K.A.,Griffith University |
And 5 more authors.
Disability and Rehabilitation | Year: 2016
Purpose: There is paucity of research investigating oropharyngeal dysphagia (OPD) in young children with cerebral palsy (CP), and most studies explore OPD in high-resource countries. This study aimed at determining the proportion and severity of OPD in preschool children with CP in Bangladesh, compared to Australia. Method: Cross-sectional, comparison of two cohorts. Two hundred and eleven children with CP aged 18–36 months, 81 in Bangladesh (mean = 27.6 months, 61.7% males), and 130 in Australia (mean = 27.4 months, 62.3% males). The Dysphagia Disorders Survey (DDS) – Part 2 was the primary OPD outcome for proportion and severity of OPD. Gross motor skills were classified using the Gross Motor Function Classification System (GMFCS), motor type/distribution. Results: (i) Bangladesh sample: proportion OPD = 68.1%; severity = 10.4 SD = 7.9. Australia sample: proportion OPD = 55.7%; severity = 7.0 SD = 7.5. (ii) There were no differences in the proportion or severity of OPD between samples when stratified for GMFCS (OR = 2.4, p = 0.051 and β = 1.2, p = 0.08, respectively). Conclusions: Despite overall differences in patterns of OPD between Bangladesh and Australia, proportion and severity of OPD (when adjusted for the functional gross motor severity of the samples) were equivalent. This provides support for the robust association between functional motor severity and OPD proportion/severity in children with CP, regardless of the resource context.Implications for RehabilitationThe proportion and severity of OPD according to gross motor function level were equivalent between high- and low-resource countries (LCs).Literature from high-resource countries may be usefully interpreted by rehabilitation professionals for low-resource contexts using the GMFCS as a framework.The GMFCS is a useful classification in LCs to improve earlier detection of children at risk of OPD and streamline management pathways for optimal nutritional outcomes.Rehabilitation professionals working in LCs are likely to have a caseload weighted towards GMFCS III–V, with less compensatory OPD management options available (such as non-oral nutrition through tubes). © 2016 Informa UK Limited, trading as Taylor & Francis Group
Stevens D.,University of Exeter |
Oades P.J.,Paediatric Unit |
Armstrong N.,University of Exeter |
Williams C.A.,University of Exeter
Journal of Cystic Fibrosis | Year: 2010
Background: Exercise testing is a valuable prognostic tool and exercise training has many health benefits in cystic fibrosis (CF). The objective of this study was to survey the provision of exercise testing and training in UK CF clinics. Methods: A three-page questionnaire was used to determine the extent of, scope and importance assigned to exercise testing and training. Results: Data from returned questionnaires showed that 38.9% of paediatric and 27.8% of adult patients had performed an exercise test in the preceding 12. months, most as part of an annual review process. Pulmonary rehabilitation programmes were accessible in only 31.3% of clinics, and only 26.0% provide exercise training programmes. When assigning importance for exercise testing on a scale from 1 'not important' to 5 'very important', the mean and median respondent scores were 3.5 and 4.0, respectively, and for the importance of training were 4.0 and 4.0, respectively. Conclusions: Despite the level of importance given to exercise testing and training by healthcare providers, exercise is underused as either an assessment tool or therapeutic intervention in the healthcare of patients with CF in the UK. © 2010 European Cystic Fibrosis Society.
Hussain N.,Paediatric Unit |
Siapkara A.,Paediatric Unit |
Branch S.,Paediatric Unit
International Journal of Pharmaceutics | Year: 2015
Abstract Since the advent of the EU Paediatric Regulation in 2007, 78 of the 1688 Paediatric Investigation Plans (PIPs) have been for monoclonal antibodies (Mabs). Of these, 22 have been assessed by the MHRA. The purpose of this mini-review is to aid those researching and developing this class of drugs to better understand regulatory concerns leading to improved medicinal products for children. Three principal quality issues were identified for PIPs under Article 7 and 8: i) the level of anti-aggregation stabilisers, ii) acceptability and tolerability of administration (i.e. multiple injections, infusion time and volume), and iii) the need to develop new presentational forms (e.g. pre-filled syringe). Overall, two types of concerns were ascertained - those which are potentially avoidable (e.g. through development of new presentational forms) and others which require the evolution of new technologies in the sector (e.g. production of concentrated, stabilised preparations). Crown Copyright © 2015 Published by Elsevier B.V.