OBJECTIVE: To assess at 24 months corrected age (CA) the neurological, respiratory, and general health status of children born prematurely from 27+0 to 33+6 weeks' gestation who were treated in a first-in-human study with a new fully synthetic surfactant (CHF5633) enriched with SP-B and SP-C proteins. OUTCOME MEASURES: Children were assessed using Bayley Scales of Infant Development (BSID), with a score below normal defined as BSID-II Mental Development Index score <70, or BSID-III cognitive composite score <85. In addition, a health status questionnaire was used to check for functional disability including respiratory problems and related treatments, sensory and neurodevelopment assessments, communication skills as well as the number of hospitalizations. RESULTS: 35 of 39 survivors had a neurodevelopmental assessment, 24 infants being evaluated by Bayley's Scales and 11 by health status questionnaires only. 23 children had scores within normal limits and one had BSID-III <85. The remaining 11 were judged clinically to have normal development. Health status questionnaires detected only issues that would normally be expected in preterm-born children. CONCLUSIONS: This assessment offers reassurance that treatment with CHF5633 surfactant was not associated with adverse neurodevelopmental, respiratory, or health outcomes by two years corrected age.
- Klíčová slova
- 24-month developmental assessment, Synthetic surfactant, first-in-human study, respiratory distress syndrome,
- MeSH
- fosfatidylcholiny terapeutické užití MeSH
- kojenec MeSH
- lidé MeSH
- nemoci nedonošenců * farmakoterapie MeSH
- novorozenec MeSH
- peptidové fragmenty MeSH
- předškolní dítě MeSH
- protein B asociovaný s plicním surfaktantem MeSH
- protein C asociovaný s plicním surfaktantem MeSH
- syndrom respirační tísně novorozenců * farmakoterapie MeSH
- vývoj dítěte MeSH
- Check Tag
- kojenec MeSH
- lidé MeSH
- novorozenec MeSH
- předškolní dítě MeSH
- Publikační typ
- časopisecké články MeSH
- Názvy látek
- CHF5633 MeSH Prohlížeč
- fosfatidylcholiny MeSH
- peptidové fragmenty MeSH
- protein B asociovaný s plicním surfaktantem MeSH
- protein C asociovaný s plicním surfaktantem MeSH
OBJECTIVES: To provide an overview of the paediatric rheumatology (PR) services in Europe, describe current delivery of care and training, set standards for care, identify unmet needs and inform future specialist service provision. METHODS: An online survey was developed and presented to national coordinating centres of the Paediatric Rheumatology International Trials Organisation (PRINTO) (country survey) and to individual PR centres (centre and disease surveys) as a part of the European Union (EU) Single Hub and Access point for paediatric Rheumatology in Europe project. The survey contained components covering the organization of PR care, composition of teams, education, health care and research facilities and assessment of needs. RESULTS: Response rates were 29/35 (83%) for country surveys and 164/288 (57%) for centre surveys. Across the EU, approximately one paediatric rheumatologist is available per million population. In all EU member states there is good access to specialist care and medications, although biologic drug availability is worse in Eastern European countries. PR education is widely available for physicians but is insufficient for allied health professionals. The ability to participate in clinical trials is generally high. Important gaps were identified, including lack of standardized clinical guidelines/recommendations and insufficient adolescent transition management planning. CONCLUSION: This study provides a comprehensive description of current specialist PR service provision across Europe and did not reveal any major differences between EU member states. Rarity, chronicity and complexity of diseases are major challenges to PR care. Future work should facilitate the development, dissemination and implementation of standards of care, treatment and service recommendations to further improve patient-centred health care across Europe.
- Klíčová slova
- paediatric rheumatology, service provision, standards of care,
- MeSH
- biologické přípravky terapeutické užití MeSH
- biomedicínský výzkum statistika a číselné údaje MeSH
- dítě MeSH
- dostupnost zdravotnických služeb organizace a řízení normy MeSH
- lidé MeSH
- mezisektorová spolupráce MeSH
- monitorování léčiv metody MeSH
- poskytování zdravotní péče organizace a řízení normy MeSH
- přechod k lékaři pro dospělé organizace a řízení normy MeSH
- průzkumy zdravotní péče MeSH
- revmatické nemoci terapie MeSH
- revmatologie výchova organizace a řízení normy MeSH
- služby zdravotní péče o dítě organizace a řízení normy MeSH
- spotřeba léčiv statistika a číselné údaje MeSH
- standardní péče MeSH
- studium lékařství organizace a řízení normy MeSH
- výzkum zdravotnických služeb metody MeSH
- zdravotnické služby - potřeby a požadavky statistika a číselné údaje MeSH
- Check Tag
- dítě MeSH
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
- multicentrická studie MeSH
- práce podpořená grantem MeSH
- Geografické názvy
- Evropa MeSH
- Názvy látek
- biologické přípravky MeSH
The Paediatric Rheumatology European Society (PReS) has over many years, developed a portfolio of educational activities to address increasing educational needs of workforce and support young clinicians to acquire skills to develop new knowledge and deliver clinical care in the future. These educational activities aim to facilitate growth of paediatric rheumatology and ultimately improve the clinical care for children and families. This article describes the current portfolio of PReS educational activities and their relevance to the international paediatric rheumatology community.
- Klíčová slova
- Education, PReS, Paediatric rheumatology, Resources, Training, Workforce,
- MeSH
- dítě MeSH
- lidé MeSH
- pediatrie výchova MeSH
- revmatologie výchova MeSH
- studium lékařství metody MeSH
- Check Tag
- dítě MeSH
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
- přehledy MeSH
- Geografické názvy
- Evropa MeSH
Lupus nephritis (LN) occurs in 50%-60% of patients with childhood-onset systemic lupus erythematosus (cSLE), leading to significant morbidity. Timely recognition of renal involvement and appropriate treatment are essential to prevent renal damage. The Single Hub and Access point for paediatric Rheumatology in Europe (SHARE) initiative aimed to generate diagnostic and management regimens for children and adolescents with rheumatic diseases including cSLE. Here, we provide evidence-based recommendations for diagnosis and treatment of childhood LN. Recommendations were developed using the European League Against Rheumatism standard operating procedures. A European-wide expert committee including paediatric nephrology representation formulated recommendations using a nominal group technique. Six recommendations regarding diagnosis and 20 recommendations covering treatment choices and goals were accepted, including each class of LN, described in the International Society of Nephrology/Renal Pathology Society 2003 classification system. Treatment goal should be complete renal response. Treatment of class I LN should mainly be guided by other symptoms. Class II LN should be treated initially with low-dose prednisone, only adding a disease-modifying antirheumatic drug after 3 months of persistent proteinuria or prednisone dependency. Induction treatment of class III/IV LN should be mycophenolate mofetil (MMF) or intravenous cyclophosphamide combined with corticosteroids; maintenance treatment should be MMF or azathioprine for at least 3 years. In pure class V LN, MMF with low-dose prednisone can be used as induction and MMF as maintenance treatment. The SHARE recommendations for diagnosis and treatment of LN have been generated to support uniform and high-quality care for all children with SLE.
- Klíčová slova
- corticosteroids, disease activity, lupus nephritis, systemic lupus erythematosus, treatment,
- MeSH
- antirevmatika terapeutické užití MeSH
- azathioprin terapeutické užití MeSH
- cyklofosfamid terapeutické užití MeSH
- dítě MeSH
- hormony kůry nadledvin terapeutické užití MeSH
- imunosupresiva terapeutické užití MeSH
- indukce remise metody MeSH
- kyselina mykofenolová terapeutické užití MeSH
- lidé MeSH
- management nemoci MeSH
- medicína založená na důkazech normy MeSH
- nefritida při lupus erythematodes diagnóza farmakoterapie MeSH
- směrnice pro lékařskou praxi jako téma * MeSH
- věk při počátku nemoci MeSH
- výsledek terapie MeSH
- Check Tag
- dítě MeSH
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
- Geografické názvy
- Evropa MeSH
- Názvy látek
- antirevmatika MeSH
- azathioprin MeSH
- cyklofosfamid MeSH
- hormony kůry nadledvin MeSH
- imunosupresiva MeSH
- kyselina mykofenolová MeSH
